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387 Study Matches

A STUDY TO EVALUATE MAINTENANCE OF HAIR REGROWTH FOLLOWING DOSE REDUCTION OF CTP-543 IN ADULT PATIENTS WITH MODERATE TOSEVERE ALOPECIA AREATA

You are being asked to take part in a research study of an investigational study drug called CTP-543. Investigational means that the study drug is currently being tested. It is not approved by the U.S. Food and Drug Administration (FDA). This is a two part research study that will evaluate the regrowth of hair of research subjects with alopecia areata (sudden hair loss) when treated with CTP-543 at two different doses twice daily for 24 weeks. The first part of the study will also evaluate what happens to hair re-growth when the dose of CTP-543 is reduced or changed to placebo (looks identical to the study drug but contains an inactive substance) after the initial 24 weeks of treatment. In the second part of the study, subjects who experienced significant hair loss following dose reduction or being changed to placebo will be re-treated with the original CTP-543 dose that they received for an additional 24 weeks to further evaluate the effects of the dose reduction or drug discontinuation (being changed to placebo) on hair re-growth. Other purposes of the study are to determine how you and the Study Doctor feel about your alopecia areata during the study. CTP-543 is a modified version of another drug called Jakafi, which is approved by the FDA for other uses, but not for alopecia areata. This study will involve approximately 300 subjects at about 25 different study sites in the United States. The study will take place for up to a maximum of 80 weeks. You will have a maximum of 16 visits to the study site in Part A, and a maximum of 9 visits to the study site in Part B. You were selected as a possible subject in this study because you have at least 50% hair loss on your head due to alopecia areata, and are between the ages of 18 and 65. You will not be able to participate if you are currently undergoing treatment with another drug or with other treatments that might affect your hair regrowth or if you are taking medications that weaken the immune system; these medications may not allow your body to protect against infection and foreign substances like bacteria and viruses. The Study Doctor can explain this to you and will make the necessary assessments and tell you if you are eligible to participate.

Maria Hordinsky
All
18 Years to 65 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT04784533
STUDY00012359
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Inclusion Criteria:

• Definitive diagnosis of alopecia areata with a current episode of scalp hair loss lasting at least 6 months and not exceeding 10 years at the time of Screening. Total disease duration greater than 10 years is permitted.
• At least 50% scalp hair loss, as defined by a SALT score ≥50, at Screening and Baseline.
• Willing to comply with the study visits and requirements of the study protocol.
Exclusion Criteria:

• Treatment with other medications or agents within 1 month of Baseline or during the study that may affect hair regrowth or immune response.
• Active scalp inflammation, psoriasis, or seborrheic dermatitis requiring topical treatment to the scalp, significant trauma to the scalp, or other scalp condition that may interfere with the SALT assessment, or untreated actinic keratosis anywhere on the body at Screening and/or Baseline.
• Treatment with systemic immunosuppressive medications within 3 months of Screening or during the study, or biologics within 6 months of Screening or during the study.
• Females who are nursing, pregnant, or planning to become pregnant while in the study, and for 30 days after last dose of study drug.
• Clinically significant medical condition, psychiatric disease, or social condition, as determined by the Investigator, that may unfavorably alter the risk-benefit of study participation, adversely affect study compliance, or confound interpretation of study results.
Drug: CTP-543, Drug: Placebo
Alopecia Areata
CTP-543
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RANDOMIZED, DOUBLE-BLINDED PHASE III STUDY OF CABOZANTINIB VERSUS PLACEBO IN PATIENTS WITH ADVANCED NEUROENDOCRINE TUMORS AFTER PROGRESSION ON PRIOR THERAPY (CABINET)

Nicole Hartung
Phase III
This study is NOT accepting healthy volunteers
NCT03375320
Atypical Carcinoid Tumor, Carcinoid Tumor, Functioning Pancreatic Neuroendocrine Tumor, Intermediate Grade Lung Neuroendocrine Neoplasm, Locally Advanced Pancreatic Neuroendocrine Tumor, Locally Advanced Unresectable Digestive System Neuroendocrine Neoplasm, Low Grade Lung Neuroendocrine Neoplasm, Lung Atypical Carcinoid Tumor, Lung Carcinoid Tumor, Metastatic Digestive System Neuroendocrine Neoplasm, Metastatic Digestive System Neuroendocrine Tumor G1, Metastatic Pancreatic Neuroendocrine Tumor, Metastatic Thymic Neuroendocrine Neoplasm, Neuroendocrine Neoplasm, Non-Functioning Pancreatic Neuroendocrine Tumor, Pancreatic Serotonin-Producing Neuroendocrine Tumor, Stage IIIA Digestive System Neuroendocrine Tumor AJCC v7, Stage IIIB Digestive System Neuroendocrine Tumor AJCC v7, Stage IV Digestive System Neuroendocrine Tumor AJCC v7
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UP0085: A POSTMARKETING, MULTICENTER, LONGITUDINAL, PROSPECTIVE, PHARMACOKINETIC, PHASE 1B STUDY IN PREGNANT WOMEN WITH CHRONIC INFLAMMATORY DISEASES TREATED WITH CIMZIA? (CERTOLIZUMAB PEGOL)

A postmarketing, multicenter, longitudinal, prospective, pharmacokinetic, phase 1B study in pregnant women with chronic inflammatory diseases treated with cimzia

Eugenia Shmidt
Phase IV
This study is NOT accepting healthy volunteers
NCT04163016
STUDY00009260
Digestive & Liver Health, Axial Spondyloarthritis, Crohn's Disease, Plaque Psoriasis, Psoriatic Arthritis, Rheumatoid Arthritis
inflammatory bowel disease, Axial Spondyloarthritis (AxSpA), CZP, Certolizumab Pegol, Cimzia, Clinics and Surgery Center (CSC), Crohn's Disease (CD), Pharmacokinetics, Plaque Psoriasis (PSO), Pregnant Women, Psoriatic Arthritis (PsA), Rheumatoid Arthritis (RA)
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A Phase 1, Open-label Study of ASP9801, an Oncolytic Virus, Administered by Intratumoral Injection in Patients with Advanced/Metastatic Solid Tumors

The purpose of this study is to assess the safety and tolerability of ASP9801 in participants with cancer who have tumors that cannot be removed or have spread to a different part of the body, as well as to determine the maximum tolerated dose and/or recommended phase 2 dose of ASP9801.

Manish Patel
All
18 Years and over
Phase 1
This study is NOT accepting healthy volunteers
NCT03954067
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Inclusion Criteria:

• Subject must have histologically- or cytologically-confirmed diagnosis of advanced or metastatic solid tumor(s).
• Subject has measurable disease as determined by Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1. At least 1 lesion must be suitable for intratumoral (IT) injection. Lesions for injection must be ≥ 10 mm and ≤ 60 mm in longest diameter.
• Subject has had disease progression after, been intolerant to, or has refused all available therapies that are known to confer clinical benefit. Note: There is no limit to the number of prior treatment regimens.
• Subject has a predicted life expectancy ≥ 12 weeks.
• Subject has at least 2 sites of disease suitable for biopsy and is willing and able to undergo required tumor biopsies according to the treating institution's guidelines at screening and during study treatment.
• Subject has an Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1.
• A female subject is eligible to participate if she is not pregnant as documented by negative pregnancy test within 72 hours prior to treatment and at least 1 of the following conditions applies:
• Not a woman of childbearing potential (WOCBP) OR
• WOCBP who agrees to follow the contraceptive guidance throughout the treatment period and for at least 180 days after the final study investigational product (IP) administration.
• Female subject must agree not to breastfeed starting at screening, and throughout the study period and 180 days after the final study IP administration.
• Female subject must not donate ova starting at screening, and throughout the study period and for 180 days after the final study IP administration.
• Male subject must agree to remain abstinent or use a condom throughout the study period and for 180 days after the final study IP administration.
• Male subject with female partner(s) of childbearing potential must agree to use contraception during the treatment period and for at least 180 days after the final study IP administration.
• Male subject must not donate sperm during the treatment period and for at least 180 days after the final study IP administration.
• Subject must be willing and able to comply with the study requirements including prohibited concomitant medication restrictions.
• Subject agrees not to participate in another interventional study while receiving study IP.
• Subject has the ability to understand and the willingness to sign a written informed consent document.
Exclusion Criteria:

• Subject has ongoing toxicity ≥ National Cancer Institute's Common Terminology Criteria for Adverse Events (NCI CTCAE) grade 2 attributable to prior antineoplastic therapies considered clinically significant.
• Subject who has had major surgery ≤ 4 weeks of screening. Subjects must have recovered from prior procedures and/or any complications from surgery prior to starting study treatment.
• Subject is concurrently participating in another interventional study or has received an investigational product ≤ 30 days or 5 half-lives whichever is shorter, prior to first IP administration.
• Subject with symptomatic or untreated central nervous system (CNS) metastases or leptomeningeal disease. Subjects with treated symptomatic brain metastases should be neurologically stable (without evidence of progression by imaging for at least 4 weeks prior to screening and any neurologic symptoms have returned to baseline) and off steroids for at least 2 weeks prior to first IP administration. Subjects with carcinomatous meningitis are excluded regardless of clinical stability.
• Subject with active or prior autoimmune or inflammatory disorders requiring systemic therapy within past 2 years (including inflammatory skin conditions or severe eczema, inflammatory bowel disease (e.g., colitis or Crohn's disease), diverticulitis (with the exception of diverticulosis), celiac disease, systemic lupus erythematosus, Sarcoidosis syndrome, Wegener syndrome (granulomatosis with polyangiitis), Graves' disease, rheumatoid arthritis, hypophysitis, uveitis, etc. The following are exceptions to this criterion:
• Subject with vitiligo or alopecia
• Subject with hypothyroidism (e.g., following Hashimoto syndrome) stable on hormone replacement
• Any chronic skin condition that does not require systemic therapy
• Subject with another malignancy that currently requires treatment.
• Subject with only tumors encasing major vascular structures such as the carotid artery, tumors adjacent to vital neurovascular structures or tumors in locations that are at high risk for adverse events (AEs) or otherwise not considered appropriate for IT injection. Subjects with such tumors that have other injectable tumors would be eligible.
• Subject with inadequate organ and marrow functions meeting any of the below criteria:
• Leukocytes < 3000/μL
• Absolute neutrophil count < 1500/μL
• Platelets < 100,000/μL
• Hemoglobin (Hgb) < 9 g/dL (Criteria must be met without packed red blood cell transfusion within the prior 2 weeks. Subjects can be on stable dose of erythropoietin [≥ approximately 3 months])
• International normalized ratio (INR) > 1.5 × ULN and/or activated partial thromboplastin time (aPTT) > 1.5 × institutional normal limits, except for subjects in Group B (Visceral Lesions) escalation and expansion groups where INR and aPTT must be normal
• Total Bilirubin (TBL) > 1.5 × institutional normal limits (subjects with known Gilbert syndrome who are excluded if TBL > 3.0 × institutional normal limits or direct bilirubin > 1.5 × institutional normal limits)
• Aspartate aminotransferase (AST) and Alanine transaminase (ALT) > 2.5 × institutional normal limits. Subjects with tumors in the liver AST and ALT > 5 × institutional normal limits.
• Albumin < 3.0 g/dL
• Creatinine > 1.5 × institutional normal limits
• Subject with a condition requiring systemic treatment with either corticosteroids (> 10 mg daily prednisone equivalents) or other immunosuppressive medications within 14 days of first administration of study IP. Inhaled or topical steroids and adrenal replacement doses > 10 mg daily prednisone equivalents are permitted in the absence of active autoimmune disease.
• Subject has an uncontrolled intercurrent illness including, but not limited to, symptomatic congestive heart failure, any form of substance abuse or psychiatric illness/social situations that would limit compliance with study visits or requirements or a condition that could invalidate communication with the investigator.
• Subject is positive for human immunodeficiency virus, hepatitis B surface antigen, hepatitis B core immunoglobulin or immunoglobulin G (IgG) antibody or hepatitis C (IgG or ribonucleic acid (RNA) test) indicating acute or chronic infection.
• Subject has a history of moderate to severe ascites, clinically significant and/or rapidly accumulating ascites, bleeding esophageal varices, hepatic encephalopathy or pericardial and/or pleural effusions related to liver insufficiency within 6 months of screening. Mild ascites that does not preclude safe IT injection of ASP9801 is allowed.
• Subject has a clinically significant abnormal electrocardiogram (ECG) at screening.
• Subject has symptomatic cardiovascular disease within the preceding 12 months unless cardiology consultation and clearance has been obtained for study participation, including but not limited to the following: significant coronary artery disease (e.g., requiring angioplasty or stenting), acute myocardial infarction or unstable angina pectoris < 3 months prior to screening, uncontrolled hypertension, clinically significant arrhythmia or congestive heart failure (New York Heart Association grade ≥ 2).
• Subject has medical conditions that predispose the subject to untoward medical risk in the event of volume loading (e.g., intravenous fluid bolus infusion), tachycardia or hypotension during or following treatment with ASP9801.
• Subject has a known or suspected hypersensitivity to ASP9801 or any components of the formulation used, including prior adverse reaction to vaccinia (e.g., as smallpox vaccine).
• Subject has had previous exposure with ASP9801.
• Subject has an active infection requiring systemic therapy.
• Subject with known history of active Bacillus Tuberculosis.
• Subject has received prior therapy with an anti-PD-1, anti-programmed cell death ligand 1 (PD-L1), or anti-programmed cell death ligand 2 (PD-L2) agent or with an agent directed to another stimulatory or co-inhibitory T-cell receptor (e.g., cytotoxic T-lymphocyte-associated protein 4, OX 40, CD137) and was discontinued from that treatment due to an immune-related adverse event.
• Subject has received prior radiation therapy within 2 weeks of start of study treatment. Subject must have recovered from all radiation-related toxicities, not require corticosteroids, and not have had radiation pneumonitis. A 1-week washout is permitted for palliative radiation (≤ 2 weeks of radiotherapy) to non-CNS disease.
• Subject has received a live vaccine within 30 days prior to the first dose of study drug. Examples of live vaccines include, but are not limited to, the following: measles, mumps, rubella, varicella/zoster (chicken pox), yellow fever, rabies, bacillus Calmette-Guérin and typhoid vaccine. Seasonal influenza vaccines for injection are generally killed virus vaccines and are allowed; however, intranasal influenza vaccines (e.g., FluMist®) are live attenuated vaccines and are not allowed.
• Subject has severe hypersensitivity (≥ grade 3) to pembrolizumab and/or any of its excipients, or history of ≥ grade 2 infusion reactions that were not prevented by adequate premedication.
• Subject has a history of (non-infectious) pneumonitis that required steroids or has current pneumonitis.
• Subject has had an allogeneic tissue/solid organ transplant.
Biological: ASP9801, Combination Product: Pembrolizumab
Metastatic Cancer, Solid Tumors, Advanced Cancer
immunotherapy, vaccinia virus, visceral lesions, cutaneous lesions, intratumoral injection, ASP9801, subcutaneous lesions, oncolytic virus, Clinics and Surgery Center (CSC), Phase I Clinic
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COG AOST2121 - An Open-Label Phase 2 Study of Maintenance Therapy with OST31-164 After Resection of Recurrent Osteosarcoma (OST-164-01)

This phase II trial studies OST31-164 as a single agent every 3 weeks for 48 weeks, with 4 doses constituting 1 treatment cycle (12 weeks per cycle). Each patient will receive treatment at a dose of 1x109 CFU until Week 48 or until disease progression, unacceptable toxicity, or the patient meets any other treatment discontinuation criteria. Following treatment discontinuation, all patients will enter a 3-year survival follow-up period. The primary endpoints are disease control during the first 12 months after enrollment and safety assessments (adverse events [AEs], physical examinations, clinical laboratory tests, vital sign measurements, performance status, and tests to monitor for the persistence of Lm).

Emily Greengard
All
12 Years to 39 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT04974008
STUDY00013667
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Inclusion Criteria:

• Note: Patients enrolled on AOST2031 are eligible for enrollment in the present study. Patients are eligible to be included in the study only if all the following criteria apply: Age and Weight
• Between 12 years of age and 39 years of age at the time the Informed Consent/ Assent form is signed.
• Weight at least 40 kg. Diagnosis
• Has histologic confirmation of osteosarcoma at diagnosis.
• Has at least one episode of disease recurrence in the lungs without limitation on the number of episodes of recurrence as long as the following criteria are met:
• Surgical resection of all possible sites of suspected pulmonary metastases to achieve a complete remission within 8 weeks prior to study enrollment
• Pathological confirmation of osteosarcoma from at least one resected tumor.
• Patients with no clinical evidence of osteosarcoma at the time of resection will not require radiographic confirmation of complete remission for enrollment Note: The definition of complete resection is: gross resection of all disease as per the operating surgeon. (For further details see https://members.childrensoncologygroup.org/files/Disc/surgery/handbooks/OsteoBoneHandb ook.pdf) Performance Status
• Patient must have a performance status corresponding to ECOG scores of 0, 1, or 2. Use Karnofsky scale for patients > 16 years of age and Lansky scale for patients < years of age Prior Therapy
• Patient must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, radiotherapy, or surgery prior to entering this study. Organ Function Requirements
• Patient has adequate organ function as defined below: a. Hematological: i. Absolute neutrophil count (ANC) is at least 1,000/µL without transfusion or growth factor support. ii. Platelet count ≥ 50,000/µL without transfusion or growth factor support. b. Adequate renal function defined as: i. Creatine clearance or radioisotope glomerular filtration rate (GFR) > 70 mL/min/1.73 m2 or ii. A serum creatine based on age/gender as follows: Maximum Serum Creatinine (mg/dL) Age: 12 to < 13 years Male :1.2 Female:1.2 Age:13 to < 16 years Male :1.5 Female:1.4 Age: ≥ 16 years Male :1.7 Female:1.4 Note: the threshold for creatinine values in this table were derived from the Schwartz formula for estimating GFR. c. Adequate liver function defined as: i. Total bilirubin < 1.5 x upper limit of normal (ULN) for age ii. Serum glutamic-pyruvic transaminase (SGPT) / alanine aminotransferase (ALT) < 110 U/L (for the purpose of this study the ULN for SGPT is 45 U/L) iii. Serum albumin > 2 g/dL d. Adequate coagulation i. International normalized ratio (INR) or prothrombin time (PT) < 1.5 x ULN unless patient is receiving anticoagulant therapy as long as PT or INR is within therapeutic range of intended use of anticoagulants. ii. Activated partial thromboplastin time (aPTT) < 1.5 x ULN unless patient is receiving anticoagulant therapy as long as aPTT is within therapeutic range of intended use of anticoagulants. e. Adequate cardiac function defined as: i. Shortening fraction of > 27% by echocardiogram, or ii. Ejection fraction of > 50% by radionuclide angiogram or echocardiogram f. Adequate pulmonary function defined as: i. No evidence of dyspnea at rest, no history of exercise intolerance, and a pulse oximetry of > 94%. g. Central nervous system (CNS) function defined as: i. Patients with a known seizure disorder may be enrolled if on anticonvulsants and/or are well-controlled. ii. CNS toxicity including peripheral neuropathy < Grade 2.
• Patient and/or patient's parent or legal guardian must be capable of understanding the investigational nature, potential risks, and benefits of the study. The patient and/or the parent or legal guardian must sign a written informed consent. Age-appropriate assent will be obtained per institutional guidelines. Contraception: Female patients :
• A female patient is eligible to participate if she is not pregnant, not breastfeeding, and at least one of the following conditions applies:
• Not a woman of childbearing potential (WOCBP) as defined in the protocol OR
• A WOCBP who agrees to follow the contraceptive guidance in the protocol during the treatment period and for at least 120 days after the last dose of study treatment.
• A female patient of childbearing potential must have a negative urine or serum pregnancy test within 72 hours prior to receiving any dose of study treatment. If the urine test is positive or cannot be confirmed as negative, a serum pregnancy test will be required. Male patients:
• A male patient is eligible to participate if he agrees to follow the contraceptive guidance in the protocol during the study treatment period and for at least 120 days after the last dose of study treatment.
Exclusion Criteria:

• Has clinically evident metastatic or recurrent disease.
• Has concurrent pulmonary recurrence and local recurrence at the primary tumor site.
• Has primary refractory disease with progression of the primary tumor on initial-therapy.
• Has CNS or any extrapulmonary disease involvement at the time of the most recent episode of disease recurrence proceeding enrollment.
• Has active infection requiring systemic therapy or is dependent on or is currently receiving systemic antibiotics that cannot be discontinued before dosing. (Note: Patients who discontinue an antibiotic prior to dosing must wait at least 5 half-lives after the last dose of antibiotic before receiving any OST31-164 infusion). Inhaled prophylactic PJP (pneumocystis jiroveci pneumonia) treatment is acceptable per Investigator discretion.
• Is currently dependent on or has received corticosteroids within the past 4 weeks (topical corticosteroids and occasional inhaled corticosteroids are allowed).
• Is currently participating in or has participated in a study of an investigational agent or is using an investigational device within 4 weeks of the first dose of treatment.
• Has a history of other active malignancy for < 2 years prior to enrollment. Basal cell carcinoma of the skin, squamous cell carcinoma of the skin, or in situ cervical cancer that has undergone potentially curative therapy or is felt by the Investigator to be at low risk for recurrence is allowed.
• Has an active autoimmune disease requiring systemic treatment within the past 3 months or a documented history of clinically severe autoimmune disease, or a syndrome that requires systemic steroids or immunosuppressive agents. Replacement therapy (e.g., thyroxine, insulin, or physiologic corticosteroid replacement therapy for adrenal or pituitary insufficiency) is not considered a form of systemic treatment. Patients with vitiligo or resolved childhood asthma/atopy would be an exception to this rule. Patients who require intermittent use of bronchodilators or local steroid injections will not be excluded from the study. Patients with hypothyroidism stable on hormone replacement or Sjogren's syndrome will not be excluded from the study.
• Has known psychiatric or substance abuse disorders that would interfere with cooperation with the requirements of the trial.
• Is pregnant or breastfeeding or expecting to conceive or father children within the projected duration of the trial, starting with the pre-screening or screening visit through 120 days after the last dose of study treatment.
• Has a known allergy to any component of the study treatment(s) formulations.
• Has a contraindication (e.g., sensitivity/allergy) to both trimethoprim/ sulfamethoxazole and amoxicillin.
• Has contraindication to administration of NSAIDs.
• Is currently receiving or will be receiving any chemotherapy, including PI3K inhibitors, during the treatment phase.
• Has had a prior monoclonal antibody therapy within 2 weeks prior to study Day 1.
• Requires or anticipates requiring tumor necrosis factor (TNF) blocking agent (e.g., infliximab) therapy for diagnosis of rheumatologic disease or inflammatory bowel disease (e.g., ankylosing spondylitis, Crohn's disease, plaque psoriasis, psoriatic arthritis, rheumatoid arthritis, or ulcerative colitis).
• Has previous history of listeriosis.
• Has a known history of human immunodeficiency virus (HIV) (HIV 1/2 antibodies).
• Has known active hepatitis B (e.g., HBsAg reactive) or hepatitis C (e.g., HCV RNA [qualitative] is detected).
• Has received a live vaccine within 30 days prior to Study Day 1.
• Patient is or has an immediate family member (spouse, children, or parent) who is directly involved with this study or is employed by the investigational site or Sponsor, unless prospective Institutional Review Board (IRB) approval (by chair or designee) is given allowing exception to this criterion for a specific patient. Regulatory Requirements:
• All patients and/or their parents or legal guardians must sign a written informed consent.
• All institutional, FDA, and NCI requirements for human studies must be met.
Drug: OST31-164
Bone Cancer
Osteosarcoma, Bone Cancer
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A Randomized, Comparative Effectiveness Study of Staged Complete Revascularization with&#13;&#10;Percutaneous Coronary Intervention to Treat Coronary Artery Disease vs Medical Management Alone&#13;&#10;in Patients with Symptomatic Aortic Valve Stenosis undergoing Elective Transfemoral Transcatheter&#13;&#10;&#13;&#10;Aortic Valve Replacement: The COMPLETE TAVR Study (COMPLETE TAVR)

The study will be a randomized, multicenter, open-label trial with blinded adjudication of outcomes.

Greg Helmer
All
Not specified
N/A
This study is NOT accepting healthy volunteers
NCT04634240
STUDY00012707
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Inclusion Criteria:

• Men and women with severe symptomatic aortic valve stenosis defined as: [aortic valve area ≤ 1.0 cm2 or aortic valve area index ≤ 0.6 cm2/m2] AND [Jet velocity ≥ 4.0 m/s or mean gradient ≥ 40 mmHg] AND [NYHA Functional Class ≥ 2 OR abnormal exercise test with severe SOB, abnormal blood pressure response, or arrhythmia] AND
• Coronary artery disease defined as: (at least 1 coronary artery lesion of ≥70% visual angiographic diameter stenosis in a native segment that is at least 2.5 mm in diameter that is not a CTO and is amenable to treatment with percutaneous coronary intervention (PCI)) AND
• Consensus by the Multidisciplinary Heart Team that the patient is suitable for elective transfemoral transcatheter aortic valve replacement (TAVR) with a balloon expandable transcatheter heart valve AND would receive a bypass with an anastomosis distal to the coronary artery lesion(s) if they were undergoing surgical aortic valve replacement. AND
• Successful TAVR defined as the implantation of a single transcatheter aortic valve within the past 96 hours with freedom from more than minimal aortic insufficiency, stroke, or major vascular complications
Exclusion Criteria:

• PCI already performed within 90 days prior to TAVR or at the same time as the index transfemoral TAVR procedure
• Planned PCI of coronary artery lesion(s)
• Planned surgical revascularization of coronary artery lesion(s)
• Non-cardiovascular co-morbidity reducing life expectancy to < 5 years
• Any factor precluding 5-year follow-up
• Prior coronary artery bypass grafting surgery or surgical valve replacement
• Severe mitral regurgitation (> 3+)
• Severe left ventricular dysfunction (LVEF < 30%)
• Low coronary takeoff (high risk for coronary obstruction)
• Acute myocardial infarction within 90 days
• Stroke or transient ischemic attack within 90 days
• Renal insufficiency (eGFR < 30 ml/min) and/or renal replacement Rx
• Hemodynamic or respiratory instability
Procedure: Percutaneous Coronary Intervention (PCI)
Aortic Stenosis, Coronary Artery Disease, Coronary Stenosis
Transcatheter Aortic Valve Replacement, Percutaneous Coronary Intervention, Coronary Artery Disease, Aortic Stenosis, Clinics and Surgery Center (CSC)
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An Open-Label Rollover Study of Levosimendan in Patients with Pulmonary Hypertension with Heart Failure and Preserved Left Ventricular Ejection Fraction (PH-HFpEF)

This is an open-label rollover study to continue treatment Levosimendan of subjects who were participated in the Tenax sponsored study after they have completed the 04 parent study.

Thenappan Thenappan
All
18 Years and over
Phase 2
This study is NOT accepting healthy volunteers
NCT03624010
STUDY00006731
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Inclusion Criteria:

• Provide a personally signed and dated informed consent document prior to initiation of any study-related procedures that are not considered standard of care.
• Completed double-blind therapy in a PH-HFpEF clinical study sponsored by Tenax Therapeutics, Inc.
• May, in the opinion of the Investigator, benefit from continued levosimendan treatment.
• Female patients of childbearing potential must agree to use a highly effective method of contraception.
• Willingness and ability to comply with scheduled visits, treatment plan, laboratory tests and other study procedures.
Exclusion Criteria:

• Discontinued treatment in the parent study for any reason other than study completion or Sponsor termination of the study.
• Pregnant or breastfeeding women.
• Local access to commercially available levosimendan
• Inability to comply with planned study procedures
• Patients with scheduled lung or heart transplant or cardiac surgery
• Dialysis developed since enrollment in parent study (either hemodialysis, peritoneal dialysis, continuous venovenous hemofiltration, or ultrafiltration)
• Estimated glomerular filtration rate (eGFR) <30 mL/min/1.73m2
• Liver dysfunction with Child Pugh Class B or C (see Attachment 2)
• Evidence of systemic bacterial, systemic fungal, or viral infection refractory to treatment
• Weight >150kg
• Systolic blood pressure (SBP) cannot be managed to ensure SBP >100 mmHg at initiation of study drug
• Heart rate >100 bpm with study drug, persistent for at least 10 minutes at screening.
• Hemoglobin < 80 g/L
• Serum potassium < 3.0 mmol/L or > 5.5 mmol/L at baseline that is unresponsive to management
Drug: Levosimendan 2.5 mg/ml Injectable Solution
Hypertension Pulmonary Secondary Heart Failure, Right Sided Heart Failure With Normal Ejection Fraction, Heart Failure With Normal Ejection Fraction
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An Open-label, Single-arm Study of Letermovir (LTV) for Prevention of Recurrent CMV Infection in High-risk Hematopoietic Cell Transplant (HCT) Recipients

This is an open-label single arm trial of letermovir (LTV, Prevymis) for prevention of recurrent CMV infection in allogeneic hematopoietic cell transplantation (HCT) recipients with history of CMV infection. The study is being conducted at MSKCC and at the University of Minnesota. Thirty-six HCT recipients who are ≥12 years of age, had clinically significant CMV infection treated with CMV antivirals and are at high risk for recurrent CMV infection, defined as receiving a transplant from an HLA mismatched donor (including cord blood), acute or chronic graft versus host disease (GVHD) requiring either topical and/or systemic steroid treatment within 14 days prior to enrollment, or T cell-depleted (CD34+-selected) allograft, will be eligible to participate in the study.

Jo-Anne Young, MD
All
12 Years and over
Phase 2
This study is NOT accepting healthy volunteers
NCT04017962
STUDY00011646
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Inclusion Criteria:

• Age >/= 12 years (any weight)
• Have received allogenic HCT
• Have received preemptive therapy for clinically significant CMV infection post-HCT and have completed preemptive therapy no longer than 7 days prior to enrollment. Preemptive treatment includes ganciclovir, valganciclovir, foscarnet or cidofovir. Clinically significant CMV infection is defined as CMV viremia requiring preemptive therapy or CMV EOD. Patients who have received LTV prophylaxis prior to onset of clinically significant CMV infection prior to enrollment (see also exclusion criteria below).
• Have one or more risk factors for recurrent CMV infection:
• Human leukocyte antigen (HLA) mismatch
• HLA-related (sibling) donor with at least one mismatch at the HLA-A, -B or -DR gene loci
• Haploidentical donor
• Unrelated donor with at least one mismatch at the HLA-A, -B, -C or -DRC1gene loci, or
• Cord blood as stem cell source
• Acute or chronic GVHD requiring either topical steroids for gastrointestinal GVHD and/or systemic steroid treatment (>/= 1mg/kg/day of prednisone or equivalent dose of another corticosteroid) within 14 days prior to enrollment
• T-cell-depleted allograft ex-vivo or in-vivo T-cell depleting agents including but not limited to ATG, alemtuzimab and post HCT cyclophosphamide.
• For adult patients, able to provide written consent and complete the informed consent. For patients under 18 years, the patient's parent(s) or legal guardian(s) must provide informed consent and the patient must provide written assent to participation in the study.
• Willing and able to comply with trial instructions and requirements
• Male and female patients of childbearing potential must be willing to use a highly effective method of contraception for the course of the study. Abstinence is acceptable if this is the usual lifestyle and preferred contraception for the patient. Subject eligibility criteria for the observational cohort:
• Age 18 years or older
• First allogenic peripheral blood or marrow HCT
• LTV prophylaxis starting <30 days post HCT and given for at least 6 weeks
• T-cell count >/=100 at day +100
Exclusion Criteria:

• Clinically significant CMV infection present at enrollment
• Breakthrough CMV infection while on primary LTV prophylaxis (unless patient non-adherent or unable to adequately absorb letermovir or documented resistance to LTV.
• Glomerular filtration rate (GFR) • Severe hepatic impairment
• Routine use of high-dose acyclovir (doses of > 800 mg twice daily), valacyclovir (doses of > 500mg twice daily), or famciclovir (doses > 500mg/day) for varicella zoster virus (VZV)/herpes simplex virus prophylaxis; limited treatment courses at higher doses for VZV infections are permissible if treatment duration dose not exceed 14 days total. Short courses of IV cidofovir for adeno virus (ADV) are permissible
• Suspected or known hypersensitivity to active or inactive ingredients of LTV formulations
• Patients treated with a medication whose administration with LTV is ontraindicated and whose discontinuation is not possible. Contraindicated medications include pimozide, ergot alkaloids and pitavastatin or simvastatin when co-administered with cyclosporine.
• Imminent demise (expected survival <6 weeks)
• Documented positive result for human immunodeficiency virus antibody (HIV-Ab) or for hepatitis C virus antibody (HCV-Ab) with detectable HCV RNA, or hepatitis B surface antigen (HBsAg) at any time prior to HCT
• Need for mechanical ventilation and/or vasopressor support at the time of enrollment
• Pregnancy or breastfeeding
• Plans to conceive or father children within the projected duration of the trial
• History of current evidence of any condition, therapy, lab abnormality, or other circumstance that might confound the results of the study, interfere with the subject's participation for the full duration of the study, or would place the subject at undue risk as judged by the investigator, such that it is not in the best interest of the subject to participate in this study.
• The following antivirals are allowed up to the listed dose limits:
• Acyclovir up to 800 mg twice daily
• Valacyclovir up to 500 mg twice daily
• Famciclovir up to 500 mg/day for VZV/HSV prophylaxis; limited treatment courses at higher doses for VZV infections are permissible if treatment duration does not exceed 14 days total.
• Short courses of IV cidofovir for ADV (up to two doses) Exclusion criteria for observational cohort:
• Clinically significant CMV infection during the 100 days following HCT. Clinically significant CMV infection defined as either CMV viremia requiring preemptive therapy with CMV antivirals or CMV end organ disease (EOD)
• Grade 3-4 GVHD
• Cord blood as cell source for HCT
• Treatment with systemic steroids (>0.5mg/kg for 2 weeks or longer) within 3 weeks prior to enrollment
Drug: Letermovir Pill, Other: blood draw
CMV, CMV Infection, Hematopoietic Cell Transplant
HCT, CMV infection, Letermovir, LTV, Memorial Sloan Kettering Cancer Center, 19-174, Clinics and Surgery Center (CSC)
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A Study to Assess the Safety, Tolerability and Preliminary Efficacy of ASP0367 (MA-0211) in Pediatric Male Participants With Duchenne Muscular Dystrophy (DMD)

Double Blind Phase 1b Study to assess ASP0367 in pediatric male patients with Duchenne Muscular Dystrophy

Male
8 Years to 16 Years old
Phase 1
This study is NOT accepting healthy volunteers
NCT04184882
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Inclusion Criteria:

• Subject has a diagnosis of Duchenne muscular dystrophy (DMD) (confirmed by Central Genetic Counselor) defined as a clinical picture consistent with typical DMD and 1 of the following:
• Dystrophin immunofluorescence and/or Western blot showing severe dystrophin deficiency consistent with the diagnosis of DMD.
• Identifiable mutation within the DMD gene (deletion/duplication of 1 or more exons), where reading frame can be predicted as "out-of-frame"
• Complete dystrophin gene sequencing showing an alteration (point mutation, duplication or other) that is expected to preclude production of the functional dystrophin protein (i.e., nonsense mutation or deletion/duplication leading to a downstream stop codon).
• A male subject of reproductive potential (Tanner Stage 2 and above) must agree to do either of the following from screening throughout the study until 30 days after the last dose of the investigational product (IP):
• Abstain from sexual intercourse, OR
• If having heterosexual intercourse, must use a condom and their female partners who are of childbearing potential must use a highly effective contraception method.
• Subject has been on a stable regimen of corticosteroids for 6 months prior to the time of enrollment (at baseline).
• Subject has been on stable cardiac therapy for 3 months prior to the time of enrollment (at baseline), if used, which may include prophylactic angiotensin-converting enzyme inhibitors (ACE), angiotensin II receptor blocker (ARB), aldosterone receptor antagonists (e.g., spironolactone, eplerenone), and/or beta-blocker therapy or a combination therapy thereof.
• Subject is unable to complete the 10 meter run/walk in <6 seconds at screening.
• Subject has a PUL 2.0 entry item A score of 4, 5 or 6 at screening.
• Subject and subject's parent(s) or legal guardian agrees not to participate in another interventional study while participating in the present study.
• For those subjects receiving exon-skipping therapy, the subject has been on a stable dose regimen with a single commercially-available product for at least 6 months prior to randomization at baseline.
• For those subjects using metformin, the subject has been on a stable dose of metformin for 3 months prior to the time of enrollment (at baseline) and the investigator expects the subject to maintain the current metformin dose.
Exclusion Criteria:

• Subject has had an acute illness (i.e., upper respiratory or viral infection) within 4 weeks prior to study enrollment (at baseline), which precludes participation.
• Subject has a cardiac ejection fraction < 55% on echocardiogram at screening.
• Subject has a mean QT interval from triplicate electrocardiogram (ECG) using Fridericia's correction (QTcF) of > 450 msec at screening. If the mean QTcF exceeds the limits stated above, 1 additional triplicate ECG can be taken and utilized at screening.
• Subject has cardiac troponin I (cTnI) above the upper limit of normal (ULN) at screening and is assessed clinically significant.
• Subject has used coenzyme Q10 (CoQ10), idebenone, carnitine, or other mitochondrial focused supplements or drugs within 4 weeks prior to randomization at baseline. In addition, subject has used any peroxisome proliferator-activated receptors (PPAR) ligands such as fibrates and thiazolidinediones 4 weeks prior to randomization at baseline.
• Subject has a known or suspected hypersensitivity to ASP0367, or any components of the formulation used.
• Subject has inadequate renal function, as defined by serum Cystatin C > 2 x ULN at screening.
• Subject who has any of the following liver function tests elevated: gamma-glutamyl transferase [GGT] and/or total bilirubin [TBL]) > 1.5 x ULN at screening.
• Subject who has a positive test result for hepatitis A virus (HAV) antibodies (immunoglobulin M [IgM]), hepatitis B surface (HBs) antigen, hepatitis C virus (HCV) antibody or human immunodeficiency virus (HIV) antigen/antibody at screening.
• Subject has mental conditions such as schizophrenia, bipolar disorder or major depressive disorder.
• Subject has a history of suicide attempt, suicidal behavior or has any suicidal ideation within 1 year prior to screening that meets criteria at a level of 4 or 5 by using the Columbia Suicide Severity Rating Scale (C-SSRS) or who is at significant risk to commit suicide, as assessed at screening or at baseline.
• Subject has severe behavioral or cognitive problems that preclude participation in the study.
• Subject has any condition, which makes the subject unsuitable for study participation.
• Subject is taking any other investigational therapy currently or has taken any other investigational therapy within 3 months prior to the time of enrollment (at baseline).
• Subject and parent/guardian are unwilling and unable to comply with scheduled visits, IP administration plan and study procedures.
• Subject has had clinical signs and symptoms consistent with coronavirus (SARSCoV-2) infection or who has tested positive within 2 months prior to randomization at baseline.
• Subject whose parent(s) and/or caregiver(s) have increased risk of coronavirus (SARS-CoV-2) exposure from work history (e.g., nursing home, meat processing facility and correctional facility) or recent travel history unless the subject's parent(s) and/or caregiver(s) have been appropriately vaccinated with one of the COVID-19 vaccines.
Drug: Bocidelpar, Drug: Placebo
Duchenne Muscular Dystrophy (DMD)
tolerability, ASP0367, MA-0211, safety
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A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study of NPC-21 for Kidney Transplant Recipients at High-Risk of Cytomegalovirus Infection, LionHeart21

This is a Phase 2, Randomized, Double-Blind, Placebo-Controlled Study of NPC-21 for Kidney Transplant Recipients at High-Risk of Cytomegalovirus Infection. The objective is to assess the efficacy and safety of the study drug, NPC-21, prophylactically for CMV seronegative (CMV-) patients receiving a first kidney transplant from a CMV seropositive (CMV+) donor.

Jo-Anne Young, MD
All
18 Years to 76 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT04225923
STUDY00010524
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Inclusion Criteria:

• Male or female patients 18 to 75 years of age in the United States or 20 to 75 years of age in Japan at the time of obtaining informed consent.
• Patients must be CMV seronegative pre-transplant and scheduled to receive or have received (within 7 days prior to first study drug administration) a first kidney transplant from a CMV seropositive donor.
• Patients must be willing and able to give written informed consent for participation in the study.
• Patients must be eligible to undergo kidney transplantation from a living or deceased donor, as per institutional standards.
• Patients must agree with contraception by using appropriate contraceptive measures.
Exclusion Criteria:

• Patients who have received a previous solid organ transplantation or hematopoietic stem cell transplantation.
• Patients who receive a multi-organ transplant.
• Patients who have CMV disease or CMV viremia at Screening.
• Patients who have a positive donor-specific antibody within 90 days prior to Randomization confirmed via medical records.
• Patients whose body weight is more than 120 kg at Screening.
• Patients who have received the following anti-CMV therapy within 7 days prior to Randomization and/or plan to receive the following anti-CMV therapy during the study: ・ Anti-CMV agents (eg, foscarnet, ganciclovir, valganciclovir, letermovir, high dose acyclovir, high dose valacyclovir, high dose famciclovir, or cidofovir). Note: The use of anti-CMV agents per local standard of care during the Rescue Phase of the study is permitted. Note: The use of anti-herpes simplex virus and anti-varicella zoster virus prophylaxis for at-risk patients is recommended (as long as the doses are below the one specified above).
• Patients who have received the following therapy within 28 days prior to Randomization and/or plan to receive the following anti-CMV therapy during the study:
• CMV hyperimmune globulin (eg, CytoGam).
• Intravenous immunoglobulin.
• Plasmapheresis (receipt prior to first study drug administration is acceptable).
• Patients with a history of a serious drug allergy to proteins, immunoglobulins, transfusions, or vaccines or any excipient of the NPC-21 formulation.
• Patients with severe hepatic insufficiency at Screening (eg, Child-Pugh Class C).
• Patients with active and untreated hepatitis B virus or hepatitis C virus, as documented as part of the pre-transplant screening.
• Patients with known human immunodeficiency virus infection, based on medical records serology.
• Patients with any uncontrolled infection at Randomization or a history of serious and uncontrolled infection within 6 months prior to Randomization.
• Patients who are pregnant or lactating.
• Patients with a history of malignancy within 5 years prior to Randomization other than curatively treated in situ cervical carcinoma, cutaneous basal cell carcinoma, or cutaneous squamous cell carcinoma.
• Patients with a history of alcohol or drug abuse or dependence within 1 year prior to Randomization that, in the opinion of the Investigator, would preclude study participation.
• Patients who have previously participated in this study or any other study involving NPC-21.
• Patients who have previously participated or are currently participating in any study involving the administration of a CMV vaccine or another CMV investigational agent.
• Patients who have participated in another interventional clinical study and received another investigational product (ie, not approved by the Food and Drug Administration in the United States or the Ministry of Health, Labour and Welfare in Japan) within 90 days before Randomization.
• Patients who are unable or unwilling, in the opinion of the Investigator, to comply with the protocol.
Drug: NPC-21 Low dose, Drug: NPC-21 High dose, Drug: NPC-21 Placebo
Cytomegalovirus Disease
Clinics and Surgery Center (CSC)
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BEGIN-OB-19: A Prospective Study to Evaluate Biological and Clinical Effects of Significantly Corrected CFTR Function in Infants and Young Children (BEGIN)

This is a two-part, multi-center, prospective longitudinal, exploratory study of highly effective CFTR modulators and their impact in children with CF on endocrine growth factors, the gut microbiome, respiratory microbiome, liver and pancreatic function, lung function, sweat chloride, and inflammatory markers.

Elissa Downs
NA
This study is NOT accepting healthy volunteers
NCT04509050
STUDY00010861
Cystic Fibrosis
CF, CFTR Modulator, Cystic Fibrosis, elexacaftor, ivacaftor, tezacaftor, triple combination therapy
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PEPN2112 - A Phase 1/2 Study of BAY 1895344 (elimusertib, IND#152153, NSC#810486) in Pediatric Patients with Relapsed or Refractory Solid Tumors

This phase I/II trial tests the safety, best dose, and whether BAY 1895344 (elimusertib) monotherapy works in treating patients (≥ 12 months and ≤ 30 years) with solid tumors that have come back (relapsed) or does not respond to treatment (refractory). Elimusertib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

Emily Greengard
All
12 Months to 30 Years old
Phase 1/Phase 2
This study is NOT accepting healthy volunteers
NCT05071209
STUDY00014678
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Inclusion Criteria:

• Part A: Patients between >= 12 months and < 18 years of age
• Part B:
• Patients between >= 12 months and =< 30 years of age for the phase 2 expansion cohorts for both EWS and PAX3-FOXO1 ARMS.
• Patients between >= 12 months and =< 21 years of age for the phase 2 DDR expansion cohort
• The Phase 2 cohorts will initially open concurrently with the Phase 1 portion but will only enroll patients at least 18 years of age. Patients < 18 years of age will be included in the Phase 2 cohorts only after the RP2D/MTD has been estimated in the Phase 1 portion
• All patients for both Parts A and B must have a minimum body surface area (BSA) >=
• 74 m^2
• All patients for both Parts A and B must have the ability to swallow BAY 1895344 (elimusertib) tablets intact
• Patients with recurrent or refractory solid tumors. Patients must have had histologic verification of malignancy at original diagnosis or relapse
• Part A: Any (non-CNS primary) solid tumor diagnosis including lymphoma which meets one of the following criteria:
• Any Ewing Sarcoma (histological confirmation alone is adequate) or any EWS-fusion positive solid tumor (i.e. including related Ewing's family of tumors with EWS fusions such as EWS-WT1, EWS-ATF1, etc.)
• Alveolar rhabdomyosarcoma (ARMS) with the PAX3-FOXO1 fusion. This does not include PAX7-FOXO1 or other variant fusion ARMS
• Any (non-CNS primary) solid tumor including lymphoma with inactivation (monoallelic or biallelic) inactivation of any of the DNA Damage Repair (DDR) genes: ATM, ATRX, BRCA1, BRCA2, CDK12, CHEK1, CHEK2, FANCA, MSH2, MRE11, PALB2, PARP1, POLD1, RAD51, or XRCC2
• Part B: Any (non-CNS primary) solid tumor diagnosis including lymphoma which meets one of the following criteria:
• B1, EWS Cohort:
• Any Ewing Sarcoma (histological confirmation alone is adequate) or any EWS-fusion positive solid tumor (i.e. including related Ewing's family of tumors with EWS fusions such as EWS-WT1, EWS-ATF1, etc.)
• B2, PAX3-FOXO1 ARMS Cohort:
• Alveolar rhabdomyosarcoma (ARMS) with the PAX3-FOXO1 fusion. This does not include PAX7-FOXO1 or other variant fusion ARMS
• B3, DDR Non-statistical Cohort:
• Any (non-CNS primary) solid tumor including lymphoma with inactivation (monoallelic or biallelic) inactivation of any of the DNA Damage Repair (DDR) genes: ATM, ATRX, BRCA1, BRCA2, CDK12, CHEK1, CHEK2, FANCA, MSH2, MRE11, PALB2, PARP1, POLD1, RAD51, or XRCC2
• All the genes on the DDR panel are annotated with OncoKB, a precision oncology knowledge base which is publicly available here: https://www.oncokb.org/. Alterations which are considered either 'Oncogenic' or 'Likely Oncogenic' would be considered sufficient for eligibility on either the phase 1 or phase 2 portions of this study. Alterations which are not annotated in OncoKB will need to be reviewed with locally qualified experts in molecular pathology, such as via an established molecular tumor board, in order to determine the likely oncogenicity AND will require approval by the study chair, Dr. Michael Ortiz. If such experts are not available at any institution, the study chair will review
• In cases where multiple mutations are present or multiple samples are available, either at different locations or different points in time, the presence of a single qualifying genomic alteration in any of those samples will is considered sufficient for eligibility on the phase 2 portions of this study
• Qualifying aberrations must be detected in either DNA or ribonucleic acid (RNA) in any tumor tissue sample (i.e. detection of a variant on circulating tumor DNA/RNA is not sufficient to qualify) using a somatic (and/or germline) mutational testing approach with either a targeted panel or whole exome/genome sequencing in the context of a Clinical Laboratory Improvement Amendments (CLIA)-certified laboratory setting. Any CLIA certified laboratory is acceptable to use
• Part A: Patients must have either measurable or evaluable disease
• Part B (1, 2, 3): Patients must have measurable disease
• Patients with a prior history of CNS metastases may enroll on study provided there is no current evidence of active disease at the time of enrollment
• Patient's current disease state must be one for which there is no known curative therapy or therapy proven to prolong survival with an acceptable quality of life
• Patients must have a performance status corresponding to Eastern Cooperative Oncology Group (ECOG) scores of 0, 1 or 2. Use Karnofsky >= 50% for patients > 16 years of age and Lansky >= 50% for patients =< 16 years of age. Note that neurologic deficits in patients with tumors previously metastatic to the CNS (or other non-oncologic reasons) must have been stable for at least 7 days prior to study enrollment. Patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score
• Patients must have fully recovered from the acute toxic effects of all prior anti-cancer therapy and must meet the following minimum duration from prior anti-cancer directed therapy prior to enrollment. If after the required timeframe, the numerical eligibility criteria are met, e.g., blood count criteria, the patient is considered to have recovered adequately
• Cytotoxic chemotherapy or other anti-cancer agents known to be myelosuppressive: >= 21 days after the last dose of myelosuppressive chemotherapy (42 days if prior nitrosourea)
• Anti-cancer agents not known to be myelosuppressive (eg, not associated with reduced platelet or absolute neutrophil count [ANC] counts): >= 7 days after the last dose of agent
• Antibodies: >= 21 days must have elapsed from infusion of last dose of antibody, and toxicity related to prior antibody therapy must be recovered to grade =< 1
• Corticosteroids: If used to modify immune adverse events related to prior therapy, >= 14 days must have elapsed since last dose of corticosteroid
• Hematopoietic growth factors: >= 14 days after the last dose of a long-acting growth factor (eg, pegfilgrastim) or 7 days for short acting growth factor. For agents that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur
• Interleukins, interferons and cytokines (other than hematopoietic growth factors): >= 21 days after the completion of interleukins, interferon or cytokines (other than hematopoietic growth factors)
• Stem cell Infusions (with or without total-body irradiation [TBI]):
• Allogeneic (non-autologous) bone marrow or stem cell transplant, or any stem cell infusion including donor lymphocyte infusions (DLI) or boost infusion: >= 84 days after infusion and no evidence of graft versus host disease (GVHD)
• Autologous stem cell infusion including boost infusion: >= 30 days
• Cellular therapy: >= 42 days after the completion of any type of cellular therapy (eg, modified T cells, natural killer [NK] cells, dendritic cells, etc.)
• Radiation therapy (XRT)/external beam irradiation including protons: >= 14 days after local XRT; >= 150 days after TBI, craniospinal XRT or if radiation to >= 50% of the pelvis; >= 42 days if other substantial bone marrow (BM) radiation
• Radiopharmaceutical therapy (eg, radiolabeled antibody, 131I MIBG): >= 42 days after systemically administered radiopharmaceutical therapy
• Study specific prior therapy: Patients must not have received prior exposure to BAY 1895344 (elimusertib) or any other specific ATR inhibitors including berzosertib (M6620, VX-970), ceralasertib (AZD6738), M4344 (VX-803), M1774, and RP-3500. Treatment with other DNA damage repair inhibitors which do not specifically inhibit ATR (e.g. PARP inhibitors, WEE1 inhibitors, CHEK1 inhibitors, etc.) does not exclude them from eligibility on this study
• For patients with solid tumors without known bone marrow involvement
• Peripheral absolute neutrophil count (ANC) >= 1000/uL
• For patients with solid tumors without known bone marrow involvement
• Platelet count >= 100,000/uL (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment)
• For patients with solid tumors without known bone marrow involvement
• Hemoglobin >= 8.0 g/dL at baseline (may receive red blood cell [RBC] transfusions)
• Patients with known or possible bone marrow metastatic disease will be eligible for study provided they meet the blood counts in above inclusion criteria (may receive transfusions provided they are not known to be refractory to red cell or platelet transfusions). These patients will not be evaluable for hematologic toxicity. At least 5 of every cohort of 6 patients must be evaluable for hematologic toxicity for the dose-escalation part of the study. If dose-limiting hematologic toxicity is observed, all subsequent patients enrolled must be evaluable for hematologic toxicity
• Serum creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70 mL/min/1.73 m^2 or a creatinine based on age/gender as follows:
• Age: 1 to < 2 years; Maximum serum creatinine (mg/dL): 0.6 (male); 0.6 (female)
• Age: 2 to < 6 years; Maximum serum creatinine (mg/dL): 0.8 (male); 0.8 (female)
• Age: 6 to < 10 years; Maximum serum creatinine (mg/dL): 1 (male); 1 (female)
• Age: 10 to < 13 years; Maximum serum creatinine (mg/dL): 1.2 (male); 1.2 (female)
• Age: 13 to < 16 years; Maximum serum creatinine (mg/dL): 1.5 (male); 1.4 (female)
• Age: >= 16 years; Maximum serum creatinine (mg/dL): 1.7 (male); 1.4 (female)
• Bilirubin (sum of conjugated + unconjugated or total) =< 1.5 x upper limit of normal (ULN) for age
• Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) =< 135 U/L. For the purpose of this study, the ULN for SGPT is 45 U/L
• Patients with seizure disorder may be enrolled if on anticonvulsants and well controlled as evidenced by no increase in seizure frequency in the prior 7 days. For patients a history of seizure but not on anticonvulsants, no seizure in the past 3 months
• Nervous system disorders (Common Terminology Criteria for Adverse Events [CTCAE] version [v]5) resulting from prior therapy must be =< grade 2, with the exception of decreased tendon reflex (DTR). Any grade of DTR is eligible
Exclusion Criteria:

• Pregnant or breast-feeding women will not be entered on this study due to risks of fetal and teratogenic adverse events as seen in animal/human studies, OR because there is yet no available information regarding human fetal or teratogenic toxicities. Pregnancy tests must be obtained in girls who are post-menarchal. Males or females of reproductive potential may not participate unless they have agreed to use two effective methods of birth control, including a medically accepted barrier or contraceptive method (e.g., male or female condom) for the duration of the study and for 3 months + 2 days for males and 6 months + 2 days for females after receiving the last dose of BAY 1895344 (elimusertib) on the study. Abstinence is an acceptable method of birth control. Female patients must not breastfeed during treatment and until 4 months after last study drug administration
• Corticosteroids: Patients receiving corticosteroids who have not been on a stable or decreasing dose of corticosteroid for at least 7 days prior to enrollment are not eligible. If used to modify immune adverse events related to prior therapy, >= 14 days must have elapsed since last dose of corticosteroid
• Patients who are currently receiving another investigational drug are not eligible
• Patients who are currently receiving other anti-cancer agents are not eligible
• Patients who are receiving cyclosporine, tacrolimus or other agents to prevent graft-versus-host disease post bone marrow transplant are not eligible for this trial
• Patients who are currently receiving drugs that are strong inducers or inhibitors of CYP3A4 are not eligible. Strong inducers or inhibitors of CYP3A4 should be avoided from 14 days prior to enrollment to the end of the study. Drugs that are considered sensitive or narrow therapeutic range CYP3A4 substrates should be avoided for the duration of protocol therapy
• Dedicated CNS imaging is not required but patients with current active CNS metastasis whether symptomatic or discovered incidentally without clinical symptoms, will be excluded from study participation
• Patients who have an uncontrolled infection are not eligible
• Patients who have received a prior solid organ transplantation are not eligible
• Patients who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study are not eligible
Drug: Elimusertib
Recurrent Alveolar Rhabdomyosarcoma, Recurrent Ewing Sarcoma, Recurrent Lymphoma, Recurrent Malignant Solid Neoplasm, Refractory Alveolar Rhabdomyosarcoma, Refractory Ewing Sarcoma, Refractory Lymphoma, Refractory Malignant Solid Neoplasm
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COG ADVL1521 - A Phase 2 Study of the MEK inhibitor Trametinib (IND #119346, NSC# 763093) in Children with Relapsed or Refractory Juvenile Myelomonocytic Leukemia

This phase II trial studies how well trametinib works in treating patients (≥ 2 years and < 22 years of age) with juvenile myelomonocytic leukemia that has come back or does not respond to treatment. Trametinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

Emily Greengard
All
1 Month to 21 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT03190915
STUDY00003883
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Inclusion Criteria:

• Patients must have had histologic verification of juvenile myelomonocytic leukemia (JMML) at original diagnosis and currently have relapsed or refractory disease; the diagnosis is made based on the following criteria
• JMML category 1 (all of the following): the diagnostic criteria must include all features in category 1 and EITHER (i) one of the features in category 2 OR (ii) two features from category 3 to make the diagnosis
• Splenomegaly
• > 1000 (1 x 10^9/uL) circulating monocytes
• < 20% blasts in the bone marrow or peripheral blood
• Absence of the t(9;22) or BCR/ABL fusion gene
• JMML category 2 (at least one of the following if at least two category 3 criteria are not present):
• Somatic mutation in RAS or PTPN11
• Clinical diagnosis of NF1 or NF1 gene mutation
• Homozygous mutation in CBL
• Monosomy 7
• JMML category 3 (at least two of the following if no category 2 criteria are met):
• Circulating myeloid precursors
• White blood cell count, > 10 000 (10 x 10^9/ uL)
• Increased hemoglobin F for age
• Clonal cytogenetic abnormality
• GM-CSF hypersensitivity
• Patients with refractory or relapsed JMML must have had at least one cycle of intensive frontline therapy or at least 2 cycles of a deoxyribonucleic acid (DNA) demethylating agent with persistence of disease, defined by clinical symptoms or the presence of a clonal abnormality; frontline therapy is defined as one cycle of intravenous chemotherapy that includes of any of the following agents: fludarabine, cytarabine, or any anthracycline but specifically excludes oral 6-mercaptopurine; frontline therapy will also include any conditioning regimen as part of a stem cell transplant; patients who transform to AML at any point with more than 20% blasts are not eligible for this trial
• Patients must have a Lansky or Karnofsky performance status score of >= 50, corresponding to Eastern Cooperative Oncology Group (ECOG) categories 0, 1 or 2; use Karnofsky for patients > 16 years of age and Lansky for patients =< 16 years of age; patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score
• Patients must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to study enrollment
• Myelosuppressive chemotherapy: patients must have completely recovered from all acute toxic effects of chemotherapy, immunotherapy or radiotherapy prior to study enrollment; at least 14 days must have elapsed since the completion of cytotoxic therapy, with the exception of hydroxyurea
• Note: cytoreduction with hydroxyurea can be initiated and continued for up to 24 hours prior to the start of protocol therapy
• Hematopoietic growth factors: at least 14 days after the last dose of a long-acting growth factor (e.g., pegfilgrastim) or 7 days for short-acting growth factor; for agents that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur
• Biologic (anti-neoplastic agent): at least 7 days must have elapsed since completion of therapy with a biologic agent; for agents that have known adverse events occurring beyond 7 days after administration, this period prior to enrollment must be extended beyond the time during which adverse events are known to occur
• Monoclonal antibodies:
• At least 30 days after the completion of any type of immunotherapy, e.g. tumor vaccines
• At least 3 half-lives must have elapsed since prior therapy that included a monoclonal antibody
• Radiotherapy:
• >= 2 weeks must have elapsed since local palliative external radiation therapy (XRT) (small port)
• >= 6 months must have elapsed if prior craniospinal XRT was received, if >= 50% of the pelvis was irradiated, or if traumatic brain injury (TBI) was received
• >= 4 weeks must have elapsed if other substantial bone marrow irradiation was given
• Stem cell transplant or rescue without TBI: no evidence of active graft versus (vs.) host disease and >= 3 months must have elapsed since transplant; >= 4 weeks must have elapsed since any donor lymphocyte infusion
• Patients must not be known to be refractory to red blood cell or platelet transfusions
• Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70 mL/min/1.73 m^2 or a serum creatinine based on age/gender as follows:
• Age: Maximum serum creatinine (mg/dL)
• 1 month to < 6 months: 0.4 (male) 0.4 (female)
• 6 months to < 1 year: 0.5 (male) 0.5 (female)
• 1 to < 2 years: 0.6 (male) 0.6 (female)
• 2 to < 6 years: 0.8 (male) 0.8 (female)
• 6 to < 10 years: 1 (male) 1 (female)
• 10 to < 13 years: 1.2 (male) 1.2 (female)
• 13 to < 16 years: 1.5 (male) 1.4 (female)
• >= 16 years: 1.7 (male) 1.4 (female)
• Total bilirubin =< 1.5 x upper limit of normal (ULN) for age
• Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) =< 3 x ULN (=< 135 U/L) (for the purpose of this study, the ULN for SGPT is 45 U/L)
• Serum albumin >= 2 g/dL
• Shortening fraction of >= 27% by echocardiogram OR ejection fraction of >= 50% by multi-gated acquisition (MUGA)
• Corrected QT (by Bazett's formula [QTcB]) interval < 450 msecs
• Patients must be able to swallow tablets or liquid; use of a nasogastric or gastrostomy (G) tube is also allowed
Exclusion Criteria:

• Patients who are pregnant or breast-feeding are not eligible for this study as there is yet no available information regarding human fetal or teratogenic toxicities; negative pregnancy tests must be obtained in girls who are post-menarchal; patients of reproductive potential may not participate unless they have agreed to use an effective contraceptive method for the duration of study therapy; women of childbearing potential should be advised to use effective contraception for 4 months after the last dose of trametinib; trametinib may also potentially be secreted in milk and therefore breastfeeding women are excluded; female patients should not breastfeed during treatment with trametinib, and for 4 months following the last dose; male patients must use a condom during intercourse and agree not to father a child during therapy and for 4 months following discontinuation of trametinib to avoid unnecessary exposure of trametinib to the fetus
• Concomitant Medications
• Corticosteroids: patients requiring corticosteroids who have not been on a stable or decreasing dose of corticosteroid for the 7 days prior to enrollment are not eligible; if used to modify immune adverse events related to prior therapy, >= 14 days must have elapsed since last dose of corticosteroid
• Note: hydrocortisone used as a pre-medication to prevent transfusion related reactions is not considered a concomitant corticosteroid
• Investigational drugs: patients who are currently receiving another investigational drug are not eligible
• Anti-cancer agents: patients who are currently receiving other anti-cancer agents are not eligible (except patients receiving hydroxyurea, which may be continued until 24 hours prior to start of protocol therapy)
• Anti-graft versus host disease (GVHD) or agents to prevent organ rejection post-transplant: patients who are receiving cyclosporine, tacrolimus or other agents to prevent either graft-versus-host disease post bone marrow transplant or organ rejection post-transplant are not eligible for this trial
• Cardiac medications: any medications for treatment of left ventricular systolic dysfunction
• Patients who have an uncontrolled infection are not eligible
• Patients who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study are not eligible
• Patients with a history of hepatic sinusoid obstructive syndrome (veno-occlusive disease) within the prior 3 months are not eligible
• Patients with a history of or current evidence/risk of retinal vein occlusion (RVO) or central serous retinopathy (CSR) are not eligible
• Patients with a history of RVO or CSR, or predisposing factors to RVO or CSR (e.g., uncontrolled glaucoma or ocular hypertension
• Patients with uncontrolled systemic disease(s) such as hypertension or diabetes mellitus are not eligible; blood pressure must be =< the 95th percentile for age, height, and gender
• Patients with a history of allergic reaction attributed to compounds of similar chemical or biologic composition to the MEK inhibitor, trametinib are not eligible
• Patients with a clinical diagnosis of Noonan syndrome are not eligible; Note: patients with Casitas B-lineage lymphoma (CBL) syndrome, also known as Noonan-like syndrome, are eligible to enroll
Drug: Trametinib
Juvenile Myelomonocytic Leukemia, Neurofibromatosis Type 1
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An Open-label, Phase 1/2 Study to Evaluate the Safety and Efficacy of Single-dose PR001A in Infants with Type 2 Gaucher Disease

This is a study to assess the safety and efficacy of PR001A, an Aden-associated (AAV9) viral vector to treat neuronopathic Gaucher disease type 2 (GD2) in infants. PRA001A will be administered via suboccipital injection to the cisterna magna during a single neurosurgical session. GD2 is a fatal disease of early infancy that does not have any therapeutic options beyond palliative care. This study will enroll infants 0-24 months of age.

Chester Whitley, MD, PhD
All
up to 24 Months old
Phase 1/Phase 2
This study is NOT accepting healthy volunteers
NCT04411654
STUDY00008823
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Inclusion Criteria:

• Bi-allelic GBA1 mutations consistent with a diagnosis of GD2 confirmed by the central laboratory.
• Clinical diagnosis of GD2
• Parent/legal guardian has the ability to understand the purpose and risks of the study and provide written informed consent and authorization to use protected health information in accordance with national and local privacy regulations.
• Patient has a reliable informant (i.e., parent/legal guardian) willing and able to participate in the study as a source of information on the patient's health status and cognitive and functional abilities (including providing input into the rating scales).
Exclusion Criteria:

• Diagnosis of a significant CNS disease other than GD2 that may be a cause for the patient's GD symptoms or may confound study objectives.
• Achieved independent gait.
• Severe peripheral symptoms of GD which, in the opinion of the Investigator, would pose an unacceptable risk to the patient or interfere with the patient's ability to comply with study procedures or interfere with the conduct of the study.
• Concomitant disease, condition, or treatment which, in the opinion of the Investigator, would pose an unacceptable risk to the patient or interfere with the patient's ability to comply with study procedures or interfere with the conduct of the study.
• Use of any GD treatment-related substrate reduction therapy.
• Use of strong inhibitors or inducers of cytochrome P450 3A4 (CYP3A4) or P-glycoprotein (P-gp) medications, herbals, or over-the-counter agents.
• Any type of prior gene or cell therapy.
• Live vaccine Immunizations within 4 weeks, or non-live vaccines within 2 weeks prior to the start of required immunosuppressive regimen.
• Use of blood thinners. Antiplatelet therapies are acceptable if the patient is medically able to temporarily stop them from 7 days prior to dosing and through at least 48 hours after the intracisternal injection and lumbar puncture.
• Use of systemic immunosuppressant or corticosteroid therapy other than protocol-specified (topical or inhaled preparations for dermatological conditions or asthma are allowed).
• Participation in another investigational drug or device study within the past 3 months.
• Brain MRI (magnetic resonance imaging) and MRA (magnetic resonance angiography) showing clinically significant abnormality deemed a contraindication to intracisternal injection.
• Clinically significant laboratory test result abnormalities assessed at screening.
• Contraindications or intolerance to radiographic visualization methods (e.g. MRI, MRA, CT), and intolerance to contrast agents used for MRI or CT scans.
• Contraindications to general anesthesia or sedation. Other protocol-defined inclusion/exclusion criteria may apply.
Biological: LY3884961, Drug: Methylprednisolone, Drug: Sirolimus, Drug: Prednisone
Gaucher Disease, Type 2
Gaucher Disease, GD, Gaucher, Type 2 Gaucher, Neuronopathic Gaucher, nGD, AAV9, GBA, Gene Therapy, Glucocerebrosidase, GBA1 mutation, Infants
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A Prospective Multicenter Study Evaluating the Effect of Implant Material and/or Surface Structure on Progression of Fusion in XLIF? Surgery (AMS in XLIF)

This study is a prospective, non-concurrent, multicenter study to compare the clinical and radiographic outcomes of smooth Polyetheretherketone (PEEK), 3D-printed titanium, and Porous PEEK interbody implants when used with cancellous allograft chips with Bone Marrow Aspirate (BMA) or cellular allograft in subjects who undergo lumbar lateral interbody fusion at one or two levels. Patients will be followed up to 24 months post-surgery. Fusion rates and clinical outcomes of the 3 groups will be evaluated.

Jonathan Sembrano
All
18 Years to 80 Years old
NA
This study is NOT accepting healthy volunteers
NCT03649490
STUDY00004973
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Inclusion Criteria:

• Male and female patients who are 18-80 years of age;
• Planned interbody fusion surgery, including at least one level of extreme lateral interbody fusion (XLIF) prior to enrollment in the research at one or two consecutive lumbar levels for degenerative disc disease, including those with up to Grade 2 spondylolisthesis, with one of the following NuVasive, Inc. interbody implants:
• Coroent® XL PEEK interbody implant; or
• Modulus® 3D-printed titanium interbody implant; or
• Cohere® XLIF Porous PEEKTM interbody implant. If a transforaminal lumbar interbody fusion or anterior lumbar interbody fusion (TLIF or ALIF) is planned adjacent to the XLIF level, the same NuVasive, Inc. interbody implant material type must be used as determined by the implant enrollment schedule. For example:
• Smooth PEEK: Coroent XL PEEK (XLIF) + Coroent L (TLIF) or Coroent XLR (ALIF) or Brigade (ALIF)
• 3D-printed titanium: Modulus XLIF + Modulus TLIF or Modulus ALIF
• Porous PEEK: Cohere XLIF + Coalesce TLIF (currently no ALIF option)
• The planned procedure must include placement of bilateral posterior screw fixation with or without intrafacet fusion using autograft (with or without the assigned allograft used at the XLIF level(s)) at the treated level(s). Direct posterior decompression at the index interbody fusion level(s) is acceptable.
• Preoperative coronal Cobb angle of < 10°;
• Able to undergo surgery based on physical exam, medical history, and surgeon judgment;
• Understands the conditions of enrollment and willing to sign an informed consent to participate in the evaluation.
Exclusion Criteria:

• Use of BMP, synthetic bone graft substitutes, allografts, or any other graft material in the interbody or intrafacet spaces other than those under study;
• Posterior grafting other than the allowed intrafacet fusion at the treated level(s);
• Revision of prior fusion at treated level(s) (adjacent level interbody fusion is acceptable);
• XLIF procedure that requires or results in the release of the anterior longitudinal ligament or posterior osteotomy;
• Preoperative coronal Cobb angle of ≥ 10°;
• Procedures performed with XLIF interbody implants with integrated vertebral body screw(s);
• Active smoking six (6) weeks prior to surgery;
• Systemic or local infection (active or latent);
• Diseases that significantly inhibit bone healing (e.g., prior diagnosis of osteoporosis, metabolic bone disease, uncontrolled diabetes, dialysis dependent renal failure, symptomatic liver disease);
• Rheumatoid arthritis or other autoimmune disease that, in the option of the investigator, would interfere with bone healing and/or fusion;
• Treatment with pharmaceuticals interfering with calcium metabolism;
• Undergoing chemotherapy or radiation treatment or chronic use of steroids (defined as more than 6 weeks of steroid use within 12 months of surgery or anytime postoperatively, other than episodic use or inhaled corticosteroids);
• Use of bone stimulators postoperatively;
• Non-ambulatory, wheelchair-bound;
• Involvement in active litigation relating to the spine (worker's compensation claim is allowed if it is not contested);
• Significant general illness (e.g., HIV, active metastatic cancer of any type, uncontrolled diabetes, dialysis dependent renal failure, symptomatic liver disease);
• Spinal metastases or active spinal tumor malignancy;
• Immunocompromised or is being treated with immunosuppressive agents;
• Pregnant, or plans to become pregnant during the study;
• Mental or physical condition that would limit the ability to comply with study requirements;
• Prisoners;
• Participating in another clinical study that would confound study data.
Degenerative Disc Disease, Spondylolisthesis
Clinics and Surgery Center (CSC)
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A Study to Evaluate the Efficacy and Safety of Recombinant Human Pentraxin-2 (rhPTX-2; PRM-151) in Participants With Idiopathic Pulmonary Fibrosis (STARSCAPE)

This is a multi-center, randomized, double-blind, placebo-controlled Phase 3 study including patients with IPF. The combination of PRM-151 with standard of care pirfenidone or nintedanib for 52 weeks will be evaluated, with primary objective as the absolute change from baseline to Week 52 in forced vital capacity (FVC).

Hyun Kim
All
40 Years to 85 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT04552899
STUDY00011372
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Inclusion Criteria:

• Documented diagnosis of IPF per the 2018 American Thoracic Society (ATS)/European Respiratory Society (ERS)/Japanese Respiratory Society (JRS)/Latin American Thoracic Society (ALAT) Clinical Practice Guideline
• High-resolution computed tomography (HRCT) pattern consistent with the diagnosis of IPF, confirmed by central review of Chest HRCT and central review of any available lung biopsy (LB)
• Minimum 6 minute walk distance (6MWD) of 150 meters with maximum use of 6 L/min at sea-level and up-to 8 L/min at altitude of supplemental oxygen while maintaining oxygen saturation of greater than or equal to (>/= )83% during the 6 minute walk test (6MWT) during screening
• FVC >/= 45% predicted during screening as determined by the over-reader
• Forced expiratory volume in 1 second (FEV1)/FVC ratio greater than (>) 0.70 during screening determined by the over-reader
• Diffusing capacity for carbon monoxide (DLCO) >/= 30% and less than or equal to ( • If receiving pirfenidone or nintedanib treatment for IPF, the participant must have been on treatment for at least 3 months and a stable dose for at least 4 weeks prior to screening, and during screening
• If not currently receiving nintedanib or pirfenidone treatment (either treatment naïve or having previously taken and discontinued) must have discontinued such treatment >/= 4 weeks prior to screening and during screening
• Anticipated life expectancy of at least 12 months at baseline
• Participant and investigator considered all medicinal treatment options and/or possibly lung transplantation prior to considering participation in the study.
• For women of childbearing potential (excluding participant enrolling in Japan): agreement to remain abstinent or use contraception
• For men: agreement to remain abstinent or use a condom, and agreement to refrain from donating sperm
• Anticipated life expectancy of at least 12 months at baseline, according to the investigator's judgment
• For participant enrolled in the extended China enrollment phase: current resident of mainland China, Hong Kong, or Taiwan, and of Chinese ancestry
Exclusion Criteria:

• Evidence of other known causes of Interstitial Lung Disease (ILD)
• FVC% predicted value showing repeated increase in the 6 months period prior to screening and including screening value
• Emphysema present on greater than or equal to (>/=) 50% of the HRCT, or the extent of emphysema is greater than the extent of fibrosis, according to central review of the HRCT
• Receiving nintedanib in combination with pirfenidone
• Received cytotoxic, immunosuppressive, cytokine modulating, or receptor antagonist agents (including but not limited to methotrexate, azathioprine, mycophenolate mofetil, cyclophosphamide, cyclosporine or other steroid sparing agent) within 4 weeks prior to or during screening
• Receiving systemic corticosteroids equivalent to prednisone > 10 mg/day or equivalent within 2 weeks prior to or during screening
• Acute respiratory or systemic bacterial, viral, or fungal infection either during screening or prior to screening and not successfully resolved 4 weeks prior to screening visit
• Participants with active or latent tuberculosis (confirmed within the 6 months prior to or during screening, by a positive screening test [interferon gamma release assay])
• Resting oxygen saturation of < 89% using up to 4 L/min of supplemental oxygen at sea level and up to 6 L/min at altitude (>/= 5000 feet [1524 meters] above sea level) during screening
• Class IV New York Heart Association chronic heart failure
• Historical evidence of left ventricular ejection fraction < 35%
• Presence of pulmonary hypertension that, in the investigator's opinion, would substantially limit the ability to comply with study requirements or may influence any of the safety or efficacy assessments included in the study
• Cardiopulmonary rehabilitation program based on exercise training that has been completed within 8 weeks prior to screening or planned to start during the participant enrollment in this trial
• History of smoking, alcohol or substance abuse disorder, or a malignancy
• Previous treatment with PRM-151
• Clinically significant abnormality on ECG during screening that, in the opinion of the investigator, may pose an additional risk in administering study drug to the participant including prolonged corrected QT interval > 450 ms (for men) or > 470 ms (for women) on ECG during screening based on the Fridericia correction formula
• Clinically significant laboratory test abnormalities during screening (hematology, serum chemistry, and urinalysis) that, in the opinion of the investigator, may pose an additional risk in administering study drug to the participant
• Pregnant or breastfeeding, or become pregnant during the study or within 8 weeks after the final dose of PRM-151
• Women of childbearing potential (Only for participants enrolling in Japan)
Drug: PRM-151 (Zinpentraxin Alfa), Drug: Placebo
Idiopathic Pulmonary Fibrosis
Clinics and Surgery Center (CSC)
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HM2021-05:A PHASE 1, MULTICENTER, OPEN-LABEL, DOSE FINDING STUDY OF CC-96673 IN SUBJECTS WITH RELAPSED OR REFRACTORY NON-HODGKIN S LYMPHOMA

The purpose of this study is to determine if the investigational drug called CC-96673 is safe and effective to treat your lymphoma. CC-96673 has not been approved for the treatment of lymphoma or any other disease and its use in this study is investigational. “Investigational” means that this treatment is being studied to learn more about it. This will be the first study testing CC-96673 in people.

Veronika Bachanova, MD
All
18 Years and over
Phase 1
This study is NOT accepting healthy volunteers
NCT04860466
STUDY00013124
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Inclusion Criteria:
Participants must satisfy the following criteria to be enrolled in the study:
• Participant (male or female) is ≥ 18 years of age at the time of signing the informed consent form (ICF).
• Participant must understand and voluntarily sign an ICF prior to any study-related assessments/procedures being conducted.
• Participant is willing and able to adhere to the study visit schedule and other protocol requirements.
• Participant must have a history of NHL that has relapsed or progressed.
• Participant has tumor accessible for biopsies.
• Participant has an ECOG PS of 0 or 1.
• Participants must have acceptable laboratory values as specified in the protocol.
Exclusion Criteria:

• Participant has cancer with symptomatic central nervous system (CNS) involvement
• Participant is on chronic systemic immunosuppressive therapy or corticosteroids or subjects with clinically significant graft-versus-host disease (GVHD). Intranasal, inhaled, topical, or local corticosteroid injections, or steroids as premedication for hypersensitivity reactions are exceptions to this criterion.
• Inadequate cardiac function or significant cardiovascular disease
• Participant has received prior investigational therapy directed at CD47 or SIRPα.
• Participant had major surgery ≤ 2 weeks prior to starting CC-96673.
• Participant is a pregnant or lactating female or intends to become pregnant during participation of the study.
• Participant has known active human immunodeficiency virus (HIV) infection.
• Participant has active hepatitis B or C (HBV/HCV) infection.
• Ongoing treatment with chronic, therapeutic dosing of anti-coagulants.
• History of autoimmune hemolytic anemia or autoimmune thrombocytopenia.
• History of concurrent second cancers requiring active, ongoing systemic treatment.
• Participant has any significant medical condition, laboratory abnormality, or psychiatric illness that would prevent the subject from participating in the study.
• Participant has active, uncontrolled, or suspected infection. Other protocol defined inclusion/exclusion criteria could apply.
Drug: CC-96673
Lymphoma, Non-Hodgkin
Relapsed or Refractory, Non-Hodgkin's Lymphoma, CC-96673, Dose Finding, Clinics and Surgery Center (CSC)
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MT2018-35 A Randomized Phase 2 Study of Obinutuzumab for Prevention of Chronic Graft vs. Host Disease After Allogeneic Peripheral Blood Stem Cell Transplantation

This is a prospective, multicenter, randomized phase II trial of prophylactic obinutuzumab vs. placebo for prevention of chronic Graft vs. Host Disease (cGVHD) after Hematopoietic Cell Transplantation (HCT). A single dose of obinutuzumab or placebo will be administered prophylactically at 3, 6, 9 and 12 months after HCT. Clinical and immunological endpoints are measured sequentially. 200 subjects will be randomized and analyzed in a modified intention-to-treat fashion. The primary objective is to determine the effect of obinutuzumab prophylaxis on the incidence of corticosteroid-requiring cGVHD after allogeneic HCT. The primary endpoint of this phase II trial is the rate of corticosteroid-requiring cGVHD one year after HCT.

Najla El Jurdi
Phase II
This study is NOT accepting healthy volunteers
NCT02867384
STUDY00005258
Graft vs. Host Disease
Clinics and Surgery Center (CSC), Graft vs. Host Disease
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A STUDY EVALUATING THE PREGNANCY OUTCOMES AND SAFETY OF INTERRUPTING ENDOCRINE THERAPY FOR YOUNG WOMEN WITH ENDOCRINE RESPONSIVE BREAST CANCER WHO DESIRE PREGNANCY (POSITIVE)

NA
This study is NOT accepting healthy volunteers
NCT02308085
0123456789
Early Breast Cancer
Breast, Pregnancy, Premenopausal, endocrine responsive
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A Double-blind, Placebo-controlled Phase 2b Study to Evaluate the Efficacy and Safety of ARO-APOC3 in Adults with Severe Hypertriglyceridemia (SHASTA-2)

The primary objective of the study is to evaluate the safety and efficacy of ARO-APOC3 in adults with SHTG and to select a dosing regimen for later stage clinical studies in this patient population.

Daniel Duprez
All
18 Years and over
Phase 2
This study is NOT accepting healthy volunteers
NCT04720534
STUDY00013050
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Inclusion Criteria:

• Based on medical history, evidence of TG ≥ 500 mg/dL and ≤ 4000 mg/dL at Screening
• Fasting TG ≥ 500 mg/dL at Screening
• Willing to follow diet counseling per Investigator judgment based on local standard of care
• Women of childbearing potential must have a negative pregnancy test, cannot be breastfeeding, and must be willing to use contraception
• Willing to provide written informed consent and to comply with study requirements
Exclusion Criteria:

• Active pancreatitis within 12 weeks prior to first dose
• Any planned bariatric surgery or similar procedures to induce weight loss from consent to end of study
• Acute coronary syndrome event within 24 weeks of first dose
• Major surgery within 12 weeks of first dose
• Planned coronary intervention (e.g., stent placement or heart bypass) or any non-cardiac major surgical procedure throughout the study
• Uncontrolled hypertension
• Human immunodeficiency virus (HIV) infection, seropositive for Hepatitis B (HBV), seropositive for Hepatitis C (HCV)
• Uncontrolled hypothyroidism or hyperthyroidism
• Hemorrhagic stroke within 24 weeks of first dose
• Malignancy within the last 2 years prior to date of consent requiring systemic treatment (some exceptions apply) Note: additional inclusion/exclusion criteria may apply per protocol
Drug: ARO-APOC3, Drug: Placebo
Severe Hypertriglyceridemia
Clinics and Surgery Center (CSC)
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STRIKE-PE: A Prospective, Multicenter Study of the IndigoTM Aspiration System Seeking to Evaluate the Long-Term Safety and Outcomes of Treating Pulmonary Embolism

The purpose of this study is to collect information on how patients with PE recover after treatment with the Indigo Aspiration System. The Indigo Aspiration System is a medical device that has been cleared by the U.S. Food and Drug Administration (FDA) for removing blood clots from the blood vessels throughout the body, excluding the head. The device is commercially available globally. Participants will be in this research study for about one year. Participants will be asked to complete a screening and baseline visit, device procedure in-patient visit as part of routine treatment for their PE, one post-procedure visit in the hospital and two follow-up visits. The study team will collect information on tests and procedures done during these visits from their medical records. They will also be asked to complete a quality of life questionnaire.

Michael Rosenberg
Post Market Monitoring
This study is NOT accepting healthy volunteers
NCT04798261
STUDY00013412
Pulmonary Embolism
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RPC01-3203: A Phase 3, Multicenter, Randomized, Double Blind, Placebo-Controlled Study of Oral Ozanimod as Maintenance Therapy for Moderately to Severely Active Chron's Disease

This is a Phase 3, randomized, double-blind, placebo-controlled study to demonstrate the effect of oral ozanimod as maintenance therapy in subjects with moderately to severely active CD, defined as a CDAI score of ≥ 220 to ≤ 450. Subjects who complete the initial 12 weeks of treatment (Induction Studies RPC01-3201 or RPC01-3202) and are in clinical response (CDAI reduction from baseline of ≥ 100 points or CDAI score of < 150) and/or clinical remission (CDAI score < 150 and/or average stool frequency score ≤ 3 with a stool frequency no worse than baseline and an average abdominal pain score ≤ 1) will be eligible to participate in the Maintenance Study.

Eugenia Shmidt
All
18 Years to 75 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT03464097
STUDY00003228
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For more information regarding Bristol-Myers Squibb Clinical Trial participation, please visit: www.BMSStudyConnect.com
Inclusion Criteria:

• Fulfilled the inclusion criteria at time of entry into the induction study and completed the week 12 efficacy assessments of the induction study
• In clinical response and/or clinical remission and/or an average daily stool frequency score ≤ 3 and an average abdominal pain score ≤ 1 with abdominal pain and stool frequency no worse than baseline at Week 12 of the Induction Study
Exclusion Criteria:

• Partial or total colectomy, small bowel resection, or an ostomy since day 1 of the induction studies or has developed a symptomatic fistula
• Had a rectal steroid therapy, rectal 5-aminosalicylates, parenteral corticosteroids, immunomodulatory agents, investigational agents or apheresis Other protocol-defined inclusion/exclusion criteria apply
Drug: Ozanimod, Other: Placebo
Digestive & Liver Health, Crohn Disease
Crohn's Disease, Crohn Disease, Oral, Ozanimod, Moderately active, Severely active, RPC01, RPC01-3203, Clinics and Surgery Center (CSC), inflammatory bowel disease
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HEM-POWR: Observational Study Evaluating Effectiveness and Safety of Real-World Treatment with Damoctocog alfa pegol in Previously Treated Patients with Hemophilia A (HEM-POWR)

Multinational, open-label, prospective, non-interventional, multicenter, cohort study. The objectives of this study are to assess the effectiveness and long term safety of prophylaxis with damoctocog alfa pegol in the real-world setting through the collection of total bleeding events and analysis of the annualized bleeding rate (ABR) in the different prophylaxis regimens (following approved local label or any other regimen prescribed by the physician as part of normal clinical practice) in patients with hemophilia A. The analyses will be stratified, based on severity of hemophilia, severity of patient bleeding profile, disease characteristics, prophylaxis regimen, age, and time on treatment (i.e., damoctocog alfa pegol-naive or not).

Joan Beckman
All
Not specified
Post Market Monitoring
This study is NOT accepting healthy volunteers
NCT03932201
STUDY00006977
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Inclusion Criteria:

• Diagnosis of hemophilia A.
• Patients previously treated for Hemophilia A.
• Patients without previous history of inhibitors or patients with previous history of inhibitors on standard prophylaxis therapy for at least 1 year prior to study entry.
• No current evidence of FVIII inhibitor or clinical suspicion of FVIII inhibitor.
• Initiation of or currently on damoctocog alfa pegol with any kind of treatment modality (on-demand, prophylaxis, or intermittent prophylaxis).
• Signed informed consent/assent.
Exclusion Criteria:

• Concurrent participation in an investigational program with interventions outside of routine clinical practice.
• Diagnosis of any other bleeding/coagulation disorder other than hemophilia A.
• Contra-indications according to the local marketing authorization.
• Patient on immune tolerance induction (ITI) treatment at the time of enrollment.
Drug: Damoctocog alfa pegol (Jivi, Bay94-9027)
Hemophilia A
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COG ANBL1821 - A Phase 2 Randomized Study of Irinotecan/Temozolomide/Dinutuximab with or without Eflornithine (DFMO) (IND# 141913) in Children with Relapsed, Refractory or Progressive Neuroblastoma

The purpose of this prospective, randomized Phase 2 study is to find out if giving eflornithine (DFMO) along with dinutuximab, irinotecan, and temozolomide is tolerated in patients ≥ 1 year of age. It will also investigate how effective the drug combination is against relapsed or refractory neuroblastoma (NBL).

Emily Greengard
All
1 Year and over
Phase 2
This study is NOT accepting healthy volunteers
NCT03794349
STUDY00006850
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Inclusion Criteria:

• Patients must have had histologic verification of neuroblastoma or ganglioneuroblastoma or demonstration of neuroblastoma cells in the bone marrow with elevated urinary catecholamines (i.e. > 2 x upper limit of normal [ULN]), at the time of initial diagnosis.
• For the purposes of this study, aggressive multidrug chemotherapy is defined as chemotherapy including 2 or more agents that must include an alkylating agent and a platinum-containing compound as intended to treat high-risk disease. The doses of chemotherapy must be comparable to those used in frontline high-risk neuroblastoma therapies (examples include A3973, ANBL0532, ANBL09P1, ANBL12P1, and ANBL1531). Patients must have ONE of the following:
• First episode of recurrent high-risk disease following completion of aggressive multi-drug frontline high-risk therapy.
• First episode of progressive high-risk disease during aggressive multi-drug frontline therapy.
• Primary resistant/refractory disease (less than partial response by International Neuroblastoma Response Criteria [INRC]) detected at the conclusion of at least 4 cycles of aggressive multidrug induction chemotherapy on or according to a high-risk neuroblastoma protocol (examples include A3973, ANBL0532, ANBL09P1, ANBL12P1, ANBL1531, etc.).
• Patients must have at least ONE of the following at the time of enrollment:
• Measurable tumor on magnetic resonance imaging (MRI) or computed tomography (CT) scan. Measurable is defined as >= 10 mm in at least one dimension on spiral/helical CT that is metaiodobenzylguanidine (MIBG) avid or demonstrates increased fludeoxyglucose F-18 (FDG) uptake on positron emission tomography (PET) scan.
• MIBG-avid lesion detected on MIBG scan with positive uptake at a minimum of one site. This site must represent disease recurrence after completion of therapy, progressive disease on therapy, or refractory disease during induction.
• Patients with resistant/refractory soft tissue disease that is not MIBG avid or does not demonstrate increased FDG uptake on PET scan must undergo biopsy to document the presence of viable neuroblastoma. Biopsy is not required for patients who have a new site of soft tissue disease (radiographic evidence of disease progression) regardless of whether progression occurs while receiving therapy or after completion of therapy.
• Patients with bone marrow disease only will be eligible if they have more than 5% disease involvement (documented neuroblastoma cells) in at least one sample from bilateral bone marrow biopsies.
• Note: Patients with elevated catecholamines (i.e. > 2 x ULN) only are NOT eligible for this study.
• Patients must have a performance status corresponding to Eastern Cooperative Oncology Group (ECOG) scores of 0, 1 or 2. Use Karnofsky for patients > 16 years of age and Lansky for patients =< 16 years of age.
• Primary refractory/resistant patients must have received at least 4 cycles of frontline high-risk chemotherapy. Frontline therapy may also have included surgery, chemotherapy, autologous stem cell transplantation (SCT) +/- MIBG, immunotherapy, radiotherapy, and retinoids but must NOT have received second line therapy for resistant/refractory, relapsed, or progressive disease. Patients who received intensified therapy for poor induction response or refractory disease (e.g. MIBG) will be considered to have received second line therapy and will not be eligible.
• At least 14 days must have elapsed since completion of myelosuppressive therapy.
• Anti-cancer agents not known to be myelosuppressive (e.g. not associated with reduced platelet or absolute neutrophil count [ANC] counts): >= 7 days after the last dose of agent.
• Antibodies: >= 21 days must have elapsed from infusion of last dose of antibody, and toxicity related to prior antibody therapy must be recovered to grade =< 1.
• No interim time prior to study entry is required following prior radiation therapy (RT) for non-target lesions. However, patients must not have received radiation for a minimum of 4 weeks prior to study entry at the site of any lesion that will be identified as a target lesion to measure tumor response. Lesions that have been previously radiated cannot be used as target lesions unless there is radiographic evidence of progression at the site following radiation or a biopsy done following radiation shows viable neuroblastoma. Palliative radiation while on study is not permitted.
• Patients are eligible >= 6 weeks after autologous stem cell transplants or stem cell infusions (including stem cell infusions given as supportive care following 131 I-MIBG therapy) as long as hematologic and other eligibility criteria have been met.
• Patients are eligible >= 6 weeks after therapeutic 131 I-MIBG provided that all other eligibility criteria are met.
• Subjects who have previously received anti-GD2 monoclonal antibodies with or without retinoids for biologic therapy are eligible unless they have had progressive disease while receiving prior anti-GD2 therapy or progressed/relapsed within 3 months of receiving anti-GD2 therapy. However, eligible patients may NOT have received anti-GD2 monoclonal antibodies in combination with chemotherapy.
• Subjects who have received autologous marrow infusions or autologous stem cell infusions that were purged using monoclonal antibody linked to beads are eligible.
• Subjects who have previously received DFMO are eligible for this study provided they have not had progressive disease while receiving DFMO or progressed/relapsed within 3 months of completing DFMO.
• Patients must not have received long-acting myeloid growth factors (e.g. pegfilgrastim) within 14 days of entry on this study. Seven days must have elapsed since administration of a short-acting myeloid growth factor.
• For patients with solid tumors (without marrow involvement) including status post SCT: peripheral absolute neutrophil count (ANC) >= 750/uL (within 7 days prior to enrollment).
• For patients with solid tumors (without marrow involvement) including status post SCT: platelet count >= 75,000/uL (transfusion independent) (within 7 days prior to enrollment).
• Patients known to have bone marrow involvement with neuroblastoma are eligible provided that minimum ANC and transfusion independent platelet count criteria are met (as above). However, these patients are not evaluable for hematological toxicity.
• Creatinine clearance or radioisotope GFR >= 70 mL/min/1.73 m^2 or a serum creatinine based on age/gender as follows:
• 1 to < 2 years (male 0.6 mg/dL, female 0.6 mg/dL)
• 2 to < 6 years (male 0.8 mg/dL, female 0.8 mg/dL)
• 6 to < 10 years (male 1 mg/dL, female 1 mg/dL)
• 10 to < 13 years (male 1.2 mg/dL, female 1.2 mg/dL)
• 13 to < 16 years (male 1.5 mg/dL, female 1.4 mg/dL)
• >= 16 years (male 1.7 mg/dL, female 1.4 mg/dL) (within 7 days prior to enrollment).
• Total bilirubin =< 1.5 x ULN for age (within 7 days prior to enrollment).
• Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) =< 5.0 x ULN for age (=< 225 U/L). For the purpose of this study, the ULN for SGPT is 45 U/L (within 7 days prior to enrollment).
• Shortening fraction of >= 27% by echocardiography (ECHO) (within 7 days prior to enrollment).
• Ejection fraction of >= 50% by ECHO or gated radionuclide study (within 7 days prior to enrollment).
• No evidence of dyspnea at rest, no exercise intolerance, no chronic oxygen requirement, and room air pulse oximetry > 94% if there is a clinical indication for pulse oximetry. Normal pulmonary function tests in patients who are capable of cooperating with testing (including diffusion capacity of the lung for carbon monoxide [DLCO)] are required if there is a clinical indication for determination. For patients who do not have respiratory symptoms, full pulmonary function tests (PFTs) are NOT required.
• Patients with a history of central nervous system (CNS) disease must have no clinical or radiological evidence of active CNS disease at the time of study enrollment.
• Patients with seizure disorders may be enrolled if seizures are well controlled on anti-convulsants.
• CNS toxicity =< grade 2.
Exclusion Criteria:

• Men and women of childbearing potential and their partners must agree to use adequate contraception while enrolled on this study. Based on the established teratogenic potential of alkylating agents, pregnant women will be excluded from this study. Because of potential risks to breastfed infants due to drug metabolites that could be excreted in breast milk, female patients who are lactating must agree to stop breastfeeding or will otherwise be excluded from this study. Females of childbearing potential must have a negative pregnancy test to be eligible for this study.
• Patients with only elevated catecholamines (i.e. > 2 x ULN) are NOT eligible for this study.
• Patients must have been off pharmacologic doses of systemic steroids for at least 7 days prior to enrollment. Patients who require or are likely to require pharmacologic doses of systemic corticosteroids while receiving treatment on this study are ineligible. The only exception is for patients known to require 2 mg/kg or less of hydrocortisone (or an equivalent dose of an alternative corticosteroid) as premedication for blood product administration in order to avoid allergic transfusion reactions. The use of conventional doses of inhaled steroids for the treatment of asthma is permitted, as is the use of physiologic doses of steroids for patients with known adrenal insufficiency. Patients on any other immunosuppressive medications (e.g. cyclosporine, tacrolimus) are not eligible.
• Patients must not have received prior treatment with irinotecan and temozolomide.
• Patients must not have received enzyme-inducing anticonvulsants including phenytoin, phenobarbital, or carbamazepine for at least 7 days prior to study enrollment. Patients receiving non-enzyme inducing anticonvulsants such as gabapentin, valproic acid, or levetiracetam will be eligible.
• Patients who have received drugs that are strong inducers or inhibitors of CYP3A4 within 7 days prior to study enrollment are not eligible.
• Patients must not have been diagnosed with myelodysplastic syndrome or with any malignancy other than neuroblastoma.
• Patients with symptoms of congestive heart failure are not eligible.
• Patients must not have >= grade 2 diarrhea.
• Patients who are unable to tolerate oral/nasogastric/gastrostomy medications will not be eligible for this trial. Additionally, patients with significant malabsorption will not be eligible for this trial.
• Patients must not have uncontrolled infection.
• Patients with a history of grade 4 allergic reactions to anti-GD2 antibodies or reactions that required permanent discontinuation of the anti-GD2 therapy are not eligible.
• Patients with a significant intercurrent illness (any ongoing serious medical problem unrelated to cancer or its treatment) that is not covered by the detailed exclusion criteria and that is expected to interfere with the action of study agents or to significantly increase the severity of the toxicities experienced from study treatment are not eligible.
Biological: Dinutuximab, Drug: Eflornithine Hydrochloride, Drug: Irinotecan Hydrochloride, Biological: Sargramostim, Drug: Temozolomide
High Risk Neuroblastoma, Recurrent Neuroblastoma, Refractory Neuroblastoma
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A Phase I/II Study of Nivolumab, Ipilimumab and Plinabulin in Patients with Recurrent Small Cell Lung Cancer: Big Ten Cancer Research Consortium BTCRC-LUN17-127

This is an open-label Phase I/II study, with a dose escalation part (Phase I) and a 2-arm randomized part (Phase II), in patients with recurrent small cell lung cancer. In the Phase I part, patients will receive plinabulin at escalating doses in combination with nivolumab and ipilimumab. In the Phase II part, approximately 40 patients will be randomized in a 1:1 ratio to receive either nivolumab + ipilimumab (Arm NI) or the triple combination of plinabulin (at the recommended phase 2 dose) + nivolumab + ipilimumab (Arm PNI). Patients will continue treatment until disease progression, development of unacceptable toxicity or one of the protocol-defined reasons for treatment discontinuation occurs.

Naomi Fujioka
All
18 Years and over
Phase 1/Phase 2
This study is NOT accepting healthy volunteers
NCT03575793
STUDY00004593
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Inclusion Criteria:
The patients must satisfy all of the following inclusion/exclusion criteria in order to be eligible for the study:
• Must have signed and dated written informed consent form in accordance with regulatory and institutional guidelines.
• Males and females aged >18 years at time of consent.
• Histological or cytological confirmed extensive-stage SCLC
• Patients who progressed after at least 1 platinum-based chemotherapy regimen. Patients with platinum resistance (defined as recurrence or progression of disease within 90 days of completion of the platinum-based regimen) are eligible. For phase II, patients also must have been treated with at least one prior line of PD-1/PD-L1 therapy.
• Measurable disease according to RECIST v1.1 (Section 8) obtained by imaging within 28 days prior to study registration.
• Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1 within 14 days before registration and minimum life expectancy of at least 12 weeks.
• Treatment to be initiated at least 2 weeks since last dose of prior systemic anticancer therapy (chemotherapy, radiation, and/or surgery.
• Recovery to grade 1 of any clinically significant toxicity (excluding alopecia, grade 2 fatigue, vitiligo, endocrinopathies on stable replacement therapy) prior to initiation of study drugs.
• Female patients of childbearing potential have a negative pregnancy test at baseline. Females of childbearing potential are defined as sexually mature women without prior hysterectomy or who have had any evidence of menses in the past 12 months. However, women who have been amenorrheic for 12 or more months are still considered to be of childbearing potential if the amenorrhea is possibly due to prior chemotherapy, anti estrogens, or ovarian suppression.
• Women of childbearing potential (i.e., menstruating women) must have a negative urine pregnancy test (positive urine tests are to be confirmed by serum test) documented within 14 days of study registration and within the 24-hour period prior to the first dose of study drug.
• Sexually active women of childbearing potential enrolled in the study must agree to use 2 forms of accepted methods of contraception during the course of the study and for 23 weeks after their last dose of study drug. Effective birth control includes (a) intrauterine device plus 1 barrier method; (b) on stable doses of hormonal contraception for at least 3 months (e.g., oral, injectable, implant, transdermal) plus one barrier method; (c) 2 barrier methods. Effective barrier methods are male or female condoms, diaphragms, and spermicides (creams or gels that contain a chemical to kill sperm); or (d) a vasectomized partner.
• For male patients who are sexually active and who are partners of premenopausal women: agreement to use 2 forms of contraception as in criterion 9b above during the treatment period and for 31 weeks after the last dose of study drug.
• Adequate laboratory values.
• Absolute neutrophil count ≥1,000/µL
• Platelet count ≥100,000/µL
• Hemoglobin ≥9.0 g/dL
• Total bilirubin ≤1.5 x upper limit of normal (ULN) or ≤3 x ULN for subjects with Gilbert's disease
• Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤3.0 x ULN (≤5 x ULN if evidence of hepatic involvement by malignant disease)
• Creatinine ≤ 1.5 x ULN or estimated glomerular filtration rate (eGFR) ≥40 mL/min/1.73m2
• Lipase and Amylase ≤1.5 x ULN. Subjects with Lipase >1.5 x ULN may enroll if there are neither clinical nor radiographic signs of a pancreatitis. Exclusion Criteria Patients with any of the following will be excluded from participation in the study.
• Active interstitial lung disease (ILD) or pneumonitis or a history of ILD or pneumonitis requiring treatment with steroids. Prior history of radiation pneumonitis is allowed if pneumonitis was restricted to the field of radiation.
• History of ileus or other significant gastrointestinal disorder known to increase the risk of ileus or chronic bowel hypomotility
• Pregnant or breastfeeding, or expecting to conceive or father children within the projected duration of the trial, starting with the pre-screening or screening visit through 23 weeks (female) or 31 weeks (male) after the last dose of study drug.
• Must not have received CTLA-4 targeted therapy previously
• Treatment with any investigational agent within 28 days prior to registration for protocol therapy. Vaccination for SARS-CoV-2 is allowed as well as any therapy as required for the treatment of active COVID 19 infection.
• Known active symptomatic central nervous system (CNS) metastases and/or carcinomatous meningitis. Patients with neurological symptoms must undergo a head computed tomography (CT) scan or brain magnetic resonance imaging (MRI) to exclude brain metastasis. Patients whose brain metastases have been treated may participate provided there is no evidence of progression for at least 2 weeks after CNS-directed treatment, as ascertained by clinical examination or brain imaging.
• Known history of human immunodeficiency virus (HIV) or active hepatitis B (by surface antigen expression or polymerase chain reaction [PCR]) or active hepatitis C (by PCR) infection. NOTE: HIV testing is not required; Hepatitis B and C testing are required at screening.
• Diagnosis of immunodeficiency or is receiving systemic steroid therapy or any other form of immunosuppressive therapy within 7 days prior to study registration.
• Has active autoimmune disease that has required systemic treatment in the past 2 years (i.e., with use of disease modifying agents, corticosteroids, or immunosuppressive drugs) or a documented history of clinically severe autoimmune disease, or a syndrome that requires systemic steroids or immunosuppressive agents. Vitiligo, alopecia, hypothyroidism only requiring hormone replacement, psoriasis not requiring systemic treatment, celiac disease controlled by diet alone or conditions not expected to recur in the absence of an external trigger are permitted.
• A condition requiring systemic treatment with corticosteroids (>10 mg daily prednisone equivalent) or other immunosuppressive medications within 14 days prior to administration of study drugs.
• History of psychiatric illness or social situations that would limit compliance with study requirements. Has a history or current evidence of any condition, therapy, or laboratory abnormality that might confound the results of the trial, interfere with the patient's participation for the full duration of the trial, or is not in the best interest of the patient to participate, in the opinion of the treating investigator.
• Prior malignancies (except non-melanoma skin cancers, and the following in situ cancers: bladder, gastric, colon, endometrial, cervical/dysplasia, melanoma, or breast) unless a complete remission was achieved at least 2 years prior to study entry.
• Documented history of a cerebral vascular event (stroke or transient ischemic attack), unstable angina, myocardial infarction, or cardiac symptoms consistent with New York Heart Association (NYHA) Class III-IV within 6 months prior to their first dose of study drugs.
• Evidence of ongoing inadequately controlled hypertension (defined as baseline systolic blood pressure >160 mmHg or diastolic blood pressure >100 mmHg).
• Any active grade 3 or higher viral, bacterial, or fungal infection within 2 weeks of the first dose of the study drugs. Routine antimicrobial prophylaxis is permitted.
Drug: Nivolumab, Drug: Plinabulin, Drug: Ipilimumab
Lung Cancer, SCLC
Clinics and Surgery Center (CSC)
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An Open-label, Multicenter, Multicohort, Phase 2 Study to Evaluate Enfortumab Vedotin in Subjects with Previously Treated Locally Advanced or Metastatic Malignant Solid Tumors (EV-202)

The primary objective is to determine the antitumor activity of enfortumab vedotin as measured by confirmed objective response rate (ORR) per investigator assessment.

Naomi Fujioka
All
18 Years and over
Phase 2
This study is NOT accepting healthy volunteers
NCT04225117
STUDY00011453
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Inclusion Criteria:

• Subject is considered an adult according to local regulation at the time of signing the informed consent form (ICF).
• Subject has measurable disease by RECIST Version 1.1.
• Subject has accessible archival tumor tissue from either the primary tumor or a metastatic site, for which source and availability have been confirmed prior to study treatment. If no archival tumor tissue is available, the subject will have a biopsy to obtain tumor tissue prior to study treatment. If the subject is unable to undergo a biopsy due to safety concerns, enrollment into the study must be discussed with the medical monitor.
• Subject has ECOG performance status of 0 or 1.
• Subject has the following baseline laboratory data. If a subject has received a recent blood transfusion, the hematology tests must be obtained ≥ 28 days after any blood transfusion.
• absolute neutrophil count (ANC) ≥ 1.0 × 10^9/L
• platelet count ≥ 100 × 10^9/L
• hemoglobin ≥ 9 g/dL
• serum total bilirubin ≤ 1.5 × upper limit of normal (ULN) or ≤ 3 × ULN for subjects with Gilbert's disease
• creatinine clearance (CrCl) ≥ 30 mL/min as estimated per institutional standards or as measured by 24-hour urine collection (glomerular filtration rate [GFR] can also be used instead of CrCl).
• alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 3 × ULN
• Subject agrees not to participate in another interventional study while receiving study treatment in the present study.
• Additional contraceptive requirements exist for male and female subjects. Disease Specific
Inclusion Criteria:

• Evidence of progression on or after the last regimen received.
• Locally advanced or metastatic disease that is not amenable to curative intent treatment. Cohort 1: HR+/HER2- breast cancer
• Subject has evidence of radiographic progression on or after the last regimen received.
• Subject has histologically- or cytologically-confirmed HR+/HER2- (estrogen receptor [ER] positive and/or progesterone receptor [PR] positive, and HER2 negative) breast cancers and are not considered a candidate for further hormonal therapy. Subject will be considered HR+ if biopsies show ≥ 1% expression of ER or PR as per current American Society of Clinical Oncology/College of American Pathologists (ASCO/CAP) guidelines.
• Subject has locally advanced or metastatic disease that is not amenable to curative intent treatment.
• Subject must have received a taxane or anthracycline in the neoadjuvant, adjuvant or incurable, locally advanced or metastatic setting.
• Prior cytotoxic regimen received in the neoadjuvant or adjuvant setting will count as a prior cytotoxic regimen if disease recurrence occurred during or within 6 months of completing the regimen.
• Subject has progressed, relapsed, or discontinued for toxicity during or after at least 1 prior standard of care cytotoxic regimen in the incurable, unresectable locally advanced or metastatic setting, and has not received > 2 prior lines of cytotoxic therapy in the locally advanced or metastatic setting. No limit applies to endocrine therapies. Poly(ADP-ribose) polymerases (PARP) inhibitors do not count as a line of cytotoxic therapy.
• Subject has progressed, relapsed, or discontinued for toxicity during or after receiving endocrine therapy or with hormonally-directed therapy with cyclin-dependent kinase (CDK) inhibitors. Prior therapy with CDK inhibitors is not required. Cohort 2: triple negative breast cancer (TNBC)
• Subject has evidence of radiographic progression on or after the last regimen received.
• Subject has histologically- or cytologically-confirmed TNBC; defined as unequivocal TNBC histology (ER-negative/PR-negative/HER2-negative). This is defined by < 1% expression of ER and PR by immunohistochemistry (IHC), and that are, for HER2, either 0 to 1+ by IHC, or IHC 2+ and fluorescence in situ hybridization (FISH) negative (not amplified) as per current ASCO/CAP guidelines.
• Subject has locally advanced or metastatic disease that is not amenable to curative intent treatment.
• Subject must have received a taxane or anthracycline in the neoadjuvant, adjuvant or incurable, locally advanced or metastatic setting.
• Prior cytotoxic regimen received in the neoadjuvant or adjuvant setting will count as a prior cytotoxic regimen if disease recurrence occurred during or within 6 months of completing the regimen.
• Subject has progressed, relapsed, or discontinued for toxicity during or after at least 1 prior standard of care cytotoxic regimen in the incurable, unresectable locally advanced or metastatic setting, and has not received > 2 prior lines of cytotoxic therapy in the locally advanced or metastatic setting. Poly(ADP-ribose) polymerases (PARP) inhibitors do not count as a line of cytotoxic therapy.
• Subject has received prior therapy with an anti-programmed cell death protein-1 (PD-1) or an anti-programmed cell death-ligand 1 (PD-L1) based on subject's tumor PD-1 or PD-L1 expression and local treatment guidelines and has progressed or discontinued treatment due to toxicity, or therapy is contraindicated for subject. Cohort 3: squamous non-small cell lung cancer (NSCLC)
• Subject has evidence of radiographic progression on or after the last regimen received.
• Subject has histologically or cytologically-confirmed squamous NSCLC.
• Subjects with mixed histology NSCLC are eligible provided there is not any component of neuroendocrine histology.
• Subjects with known epidermal growth factor receptor (EGFR), anaplastic lymphoma kinase (ALK), reactive oxygen species (ROS), BRAF, or other actionable mutations are eligible if treated with mutation targeted therapy and have progressed, relapsed, or discontinued treatment due to toxicity.
• Subject has locally advanced or metastatic disease that is not amenable to curative intent treatment.
• Subject has progressed, relapsed, or discontinued treatment due to toxicity after 1 platinum-based standard of care regimen for locally advanced or metastatic disease, and has not received > 2 prior lines of cytotoxic anticancer therapy in the locally advanced or metastatic setting.
• Subjects with locally advanced disease who previously received curative intent treatment with platinum-based standard of care regimen in the adjuvant or neoadjuvant setting or as part of concomitant chemoradiation therapy are eligible if they have progressed or relapsed within 6 months of completion.
• Maintenance therapy does not constitute a new chemotherapy regimen provided there was no progression after the initial platinum-based regimen.
• Changing chemotherapy agents during platinum-based treatment for the management of toxicities does not constitute a new chemotherapy regimen provided no progression had occurred while on the initial therapy.
• Subject has received prior therapy with an anti-programmed cell death protein-1 (PD-1) or anti-programmed cell death-ligand 1 (PD-L1) based on subject's tumor PD-1 or PD-L1 expression and local treatment guidelines and has progressed, relapsed, or discontinued treatment due to toxicity, or therapy is contraindicated for subject. Cohort 4: non-squamous non-small cell lung cancer
• Subject has evidence of radiographic progression on or after the last regimen received.
• Subject has histologically- or cytologically-confirmed non-squamous NSCLC.
• Subjects with mixed histology NSCLC are eligible provided there is not any component of neuroendocrine histology.
• Subjects with known EGFR, ALK, ROS, BRAF, or other actionable mutations are eligible if treated with mutation targeted therapy and have progressed, relapsed, or discontinued treatment due to toxicity.
• Subject has locally advanced or metastatic disease that is not amenable to curative intent treatment.
• Subject has progressed, relapsed, or discontinued treatment due to toxicity after 1 platinum-based standard of care regimen for locally advanced or metastatic disease, and has not received > 2 prior lines of cytotoxic anticancer therapy in the locally advanced or metastatic setting.
• Subjects with locally advanced disease who previously received curative intent treatment with platinum-based standard of care regimen in the adjuvant or neoadjuvant setting or as part of concomitant chemoradiation therapy are eligible if they have progressed or relapsed within 6 months of completion.
• Maintenance therapy does not constitute a new chemotherapy regimen provided there was no progression after the initial platinum-based regimen.
• Changing chemotherapy agents during platinum-based treatment for the management of toxicities does not constitute a new chemotherapy regimen provided no progression has occurred while on the initial therapy.
• Subject has received prior therapy with an anti-PD-1 or anti-PD-L1 based on subject's tumor PD-1 or PD-L1 expression and local treatment guidelines and has progressed, relapsed, or discontinued treatment due to toxicity, or therapy is contraindicated for subject. Cohort 5: head and neck cancer
• Subject has evidence of radiographic progression on or after the last regimen received.
• Subject has histologically- or cytologically-confirmed head and neck cancer.
• Primary tumor site must arise from the oral cavity, oropharynx, hypopharynx, and larynx; tumors arising from the nasopharynx are excluded. Salivary gland tumors and/or parotid gland tumors are not eligible for Cohort 5.
• Subject has locally advanced or metastatic disease that is not amenable to curative intent treatment.
• Subject has progressed, relapsed, or discontinued treatment due to toxicity after 1 platinum-based standard of care regimen for locally advanced or metastatic disease, and has not received > 2 prior lines of cytotoxic anticancer therapy in the locally advanced or metastatic setting.
• Subjects with locally advanced disease who previously received curative intent treatment with platinum-based standard of care regimen in the adjuvant or neoadjuvant setting or as part of concomitant chemoradiation therapy are eligible if they have progressed or relapsed within 6 months after completion.
• Subject has received prior therapy with an anti-PD-1 or anti-PD-L1 based on subject's tumor PD-1 or PD-L1 expression and local treatment guidelines and has progressed, relapsed, or discontinued treatment due to toxicity, or therapy is contraindicated for subject. Cohorts 6, 7 and 8: gastric or gastroesophageal junction (GEJ) or esophageal adenocarcinoma
• Subject has evidence of radiographic progression on or after the last regimen received.
• Subject has histologically- or cytologically-confirmed gastric, GEJ, or esophageal cancer.
• Subject has locally advanced or metastatic disease that is not amenable to curative intent treatment.
• Subject has progressed, relapsed, or discontinued due to toxicity after 1 platinum-based standard of care regimen for locally advanced or metastatic disease, and has not received > 2 prior lines of cytotoxic anticancer therapy in the locally advanced or metastatic setting.
• Neoadjuvant or adjuvant cytotoxic regimens will count as a prior regimen if relapsed or progressed ≤ 6 months after completion.
• Subject must have received a HER2 directed therapy if known to have HER2 positive cancer.
• Subject has received prior therapy with an anti-PD-1 or anti-PD-L1 based on subject's tumor PD-1 or PD-L1 expression and local treatment guidelines and has progressed, relapsed, or discontinued treatment due to toxicity, or therapy is contraindicated for subject.
Exclusion Criteria:

• Subject has preexisting sensory or motor neuropathy Grade ≥ 2.
• Subject has active central nervous system (CNS) metastases. Subjects with treated CNS metastases are permitted on study if all the following are true:
• CNS metastases have been clinically stable for ≥ 6 weeks prior to screening
• If requiring steroid treatment for CNS metastases, the subject is on a stable dose ≤ 20 mg/day of prednisone or equivalent for ≥ 2 weeks
• Baseline imaging scans show no evidence of new or enlarged brain metastasis
• Subject does not have leptomeningeal disease
• Subject has ongoing clinically significant toxicity (Grade 2 or higher with the exception of alopecia) associated with prior treatment (including systemic therapy, radiotherapy or surgery).
• Subjects with ongoing ≥ Grade 3 immunotherapy-related hypothyroidism or panhypopituitarism are excluded. Subjects with ongoing immunotherapy-related colitis, uveitis, myocarditis or pneumonitis, or subjects with other immunotherapy-related AEs requiring high doses of steroids (> 20 mg/day of prednisone or equivalent), are excluded. Subject with ≤ Grade 2 immunotherapy-related hypothyroidism or panhypopituitarism may be enrolled when well maintained/controlled on a stable dose of hormone replacement therapy (if indicated).
• Subject has a history of uncontrolled diabetes mellitus within 3 months before the first dose of study treatment. Uncontrolled diabetes (within 3 months before first dose) is defined as hemoglobin A1c (HbA1c) ≥ 8% or HbA1c between 7 and < 8% with associated diabetes symptoms (polyuria or polydipsia) that are not otherwise explained. The lowest HbA1c during the screening period will be used to determine eligibility.
• Subject has prior treatment with enfortumab vedotin or other monomethyl auristatin E (MMAE) based antibody-drug conjugates (ADCs).
• Subject has a second malignancy diagnosed within 3 years before first dose of study drug, or any evidence of residual disease from a previously diagnosed malignancy. Subjects with non-melanoma skin cancer, localized prostate cancer treated with curative intent with no evidence of progression, low-risk or very low-risk (per standard guidelines) localized prostate cancer under active surveillance/watchful waiting without intent to treat, or carcinoma in situ of any type (if complete resection was performed) are allowed.
• Subject is currently receiving systemic antimicrobial treatment for viral, bacterial, or fungal infection at the time of first dose of study treatment. Routine antimicrobial prophylaxis is permitted.
• Subject has known active hepatitis B (e.g., hepatitis B surface antigen [HBsAg] reactive) or active hepatitis C (e.g., hepatitis C virus [HCV] RNA [qualitative] is detected).
• Subject has known history of human immunodeficiency virus (HIV) infection (HIV 1 or 2).
• Subject has documented history of a cerebral vascular event (stroke or transient ischemic attack), unstable angina, myocardial infarction or cardiac symptoms (including congestive heart failure) consistent with New York Heart Association Class III-IV within 6 months prior to the first dose of study drug.
• Subject has major surgery within 4 weeks prior to first dose of study drug.
• Subject had radiotherapy, chemotherapy, biologics, investigational agents, and/or antitumor treatment with immunotherapy that is not completed 2 weeks prior to first dose of study drug.
• Subject has known hypersensitivity to enfortumab vedotin or to any excipient contained in the drug formulation of enfortumab vedotin (including histidine, trehalose dihydrate and polysorbate 20) OR subject has known hypersensitivity to biopharmaceutical produced in Chinese hamster ovary cells.
• Subject has known active keratitis or corneal ulcerations. Subject with superficial punctate keratitis is allowed if the disorder is being adequately treated.
• Subject has any condition which makes the subject unsuitable for study participation.
Drug: enfortumab vedotin
Locally Advanced or Metastatic Malignant Solid Tumors
Squamous NSCLC, Triple negative breast cancer (TNBC), ASG-22CE, non-small cell lung cancer (NSCLC), Hormone receptor-positive/ human epidermal growth factor receptor 2-negative (HR+/HER2-) breast cancer, non-squamous NSCLC, locally advanced or metastatic malignant solid tumors, Head and neck cancer, EV-202, Gastric or gastroesophageal junction (GEJ) or esophageal cancer, Clinics and Surgery Center (CSC), Phase I Clinic
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A Randomized, Double-Blind, Placebo-Controlled, Dose-Ranging Study to Evaluate the Efficacy and Safety of SPR001 (Tildacerfont) in Adult Subjects with Classic Congenital Adrenal Hyperplasia

Study SPR001-203 will be a larger, randomized, double-blind, placebo-controlled, dose-ranging study that will investigate the efficacy and safety of up to 52 weeks of treatment with tildacerfont in subjects with classic CAH who have elevated biomarkers at baseline (A4 >1.5x upper limit of normal [ULN] and ACTH >2x ULN) on their current Glucocorticoid regimen.

Kyriakie Sarafoglou
Phase III
This study is NOT accepting healthy volunteers
NCT04457336
STUDY00009488
Congenital Adrenal Hyperplasia
Adrenal Disorder, CAH, Congenital Adrenal Hyperplasia
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MT2021-14 A PHASE I/II, DOSE-ESCALATION STUDY OF MGTA-117 IN PATIENTS WITH ADULT ACUTE MYELOID LEUKEMIA (AML) AND MYELODYSPLASIA-EXCESS BLASTS (MDS-EB)

The overall objective of this study is to investigate the safety, tolerability, PK, PD, and potential anti-leukemia activity of MGTA-117 given intravenously (IV) as a single dose in adults with R/R AML or MDS-EB. Once adequate data are available, the protocol will be amended to assess the safety/tolerability and preliminary efficacy of MGTA-117 in combination with reduced intensity conditioning (RIC) in patients with AML who are proceeding to HSCT.

Mark Juckett
All
18 Years to 75 Years old
Phase 1
This study is NOT accepting healthy volunteers
NCT05223699
STUDY00013345
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Inclusion Criteria:

• Participant must have a World Health Organization (WHO)-defined diagnosis of R/R AML and meet one of the following criteria:
• The participant has experienced primary AML induction failure or R/R AML OR The participant has a WHO-defined diagnosis of MDS-EB and has failed/is refractory to HMA
• CD117+ based on IHC or flow cytometry
• Participant must have an identified HSC donor (related donor or unrelated donor), haplo-identical transplant donor, or umbilical blood donor.
• Participant's Eastern Cooperative Oncology Group (ECOG) performance status must be ≤2.
• Participant must have adequate baseline hepatic function. Alanine aminotransferase (ALT), aspartate aminotransferase (AST), and alkaline phosphatase (ALP) ≤2 x upper limit of normal (ULN), and serum bilirubin ≤1.5 x ULN.
• Estimated creatinine clearance ≥60 mL/min
• Adequate cardiac function as demonstrated by cardiac left ventricular ejection fraction ≥40% or perform New York Heart Association (NYHA) classification I and II
Exclusion Criteria:

• Acute promyelocytic leukemia (APL).
• Known active central nervous system (CNS) leukemia or chloroma (granulocyte sarcoma).
• Received HSCT within 6 months prior to dosing
• Has active graft-versus-host disease (GVHD).
• Active hepatitis B (Hep-B) or hepatitis C (Hep-C) infection or history of human immunodeficiency virus (HIV).
• Participant with a QTc value >470 msec
• Participant has received another investigational drug or device within 14 days or 5 half-lives of dosing, whichever is longer.
• Participant has any clinically significant medical condition, which in the opinion of the Investigator may place the participant at an unacceptable risk.
• Active uncontrolled systemic bacterial, fungal, or viral infection
• Participant has a history of serious allergic reactions, which in the opinion of the Investigator may pose an increased risk of serious infusion reactions.
• Participant has had any systemic antileukemia treatment within 14 days except hydroxyurea, which is permitted until 24 hours prior to MGTA-117 dosing.
• Participant has received prior anti-CD117 antibody treatment.
• Participant has received gemtuzumab ozogamicin (Mylotarg) within the last 3 months prior to dosing.
• Participant has received recent monoclonal antibody as anti-leukemic therapy within the last 30 days or 5 half-lives, whichever is longer.
• Participant has received recent vaccination within the last 14 days prior to dosing.
• Participant has Grade 2 or higher electrolyte abnormality at screening
Biological: MGTA-117
Acute Myeloid Leukemia, Myelodysplasia
Acute Myeloid Leukemia, Myelodysplasia-Excess Blasts, MGTA-117, Hematopoietic stem cell transplant, Clinics and Surgery Center (CSC)
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APD334-206: A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety and Efficacy of Etrasimod in Adult Subjects with Eosinophilic Esophagitis (VOYAGE)

The purpose of this study is to determine whether oral etrasimod is a safe and effective treatment for active eosinophilic esophagitis (EoE) in adult participants.

Tyson Sievers
All
18 Years to 65 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT04682639
STUDY00012609
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Inclusion Criteria:

• Have an eosinophilic esophagitis (EoE) diagnosis prior to screening and histologically active disease with an esophageal peak eosinophil count (PEC) of ≥ 15 eosinophils per high powered field (eos/hpf)
• Have dysphagia, defined as solid food going down slowly or getting stuck in the throat with an average frequency of ≥ 2 episodes per week over 2 weeks during the Screening period Inclusion Criteria for the Extension Treatment Period
• Completion of the Week 24 study visit [including esophagogastroduodenoscopy (EGD)]
• Compliance with study procedures during the Double-Blind Treatment Period as assessed by the Investigator
• No notable safety concerns during the Double-Blind Treatment Period, as determined by the Investigator
• Willing to comply with all study visits and procedures for the Extension Treatment Period
Exclusion Criteria:

• History of any of the non-EoE conditions or procedures that may interfere with the evaluation of or affect the histologic (eg eosinophilic gastritis), endoscopic (eg, high-grade esophageal stenosis), or symptom endpoints (eg, esophageal resection) of the study
• Undergone dilation of an esophageal stricture within 12 weeks prior to Screening EGD
• Use of corticosteroids for the treatment of EoE within 8 weeks prior to Screening EGD
• Discontinue, initiate, or change dosing (dosage/frequency) of the following therapies for EoE within 8 weeks prior to Screening EGD. Participants on any of the following therapy need to stay on a stable regimen during study participation:
• Elemental diet
• EoE food trigger elimination diet
• Proton pump inhibitor (PPI) therapy
• Used any immunotherapy/desensitization including oral immunotherapy (OIT) or sublingual immunotherapy (SLIT) within 12 months prior to the Screening EGD. Note: Stable (ie, ≥ 6 months prior to the Screening EGD) subcutaneous immunotherapy (SCIT) is permitted. Participants on SCIT need to stay on a stable treatment during study participation
• Used any protocol-specified immunomodulatory therapies within the protocol-specified timeframe prior to Baseline (eg, dupilumab, benralizumab, omalizumab, or infliximab within 12 weeks; a sphingosine-1-phosphate receptor modulator at any time)
• Use of any investigational agent or device within 12 weeks prior to Baseline
• Females who are pregnant
Drug: Etrasimod, Drug: Placebo, Drug: Etrasimod
Eosinophilic Esophagitis
Eosinophilic esophagitis, Esophageal eosinophilia, Etrasimod, APD334, EoE, Eosinophilic oesophagitis, Clinics and Surgery Center (CSC)
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A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Rosnilimab (ANB030) in the Treatment of Subjects with Alopecia Areata

This research is studying a new treatment, rosnilimab, in a small number of people to learn about the safety, potential effect on alopecia areata, and how it interacts with the body. Researchers want to understand if rosnilimab may cause hair regrowth in people with alopecia areata and how it may work.

Maria Hordinsky
18 Years and over
Phase II
This study is NOT accepting healthy volunteers
NCT05205070
STUDY00014901
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Inclusion Criteria:
Men and women, adults 18- 65 years of age who are in good health Diagnosed with severe or very severe Alopecia Areata and have greater than or equal to 50% scalp hair loss Must be willing to stop using other types of medications to treat Alopecia Areata throughout the study Attend 10 visits over 28 weeks Other requirements, which the study team will review with you
Exclusion Criteria:
Subjects cannot participate in this study if they have another cause of hair loss in addition to alopecia areata. Subjects also cannot participate if they have had surgery in the last 4 weeks, if immune system is compromised, if are allergic to anything in rosnilimab, if recently used a prescription treatment for alopecia areata, if have tuberculosis, if person is pregnant or breastfeeding, or if person intends to become pregnant while part of this study. Subjects cannot participate in this study if they are currently part of another study that is using an experimental drug or device (medical tool).
Alopecia Areata, Dermatology (Skin, Hair & Nails), Immune Diseases
hair loss, Alopecia areata, PD-1 receptor, Rosnilimab
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