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341 Study Matches

A Phase 2, multicenter, open-label, non-randomized, proof-of-concept study evaluating the efficacy, safety, and tolerability of BIVV020 in adults with chronic inflammatory demyelinating polyneuropathy (CIDP)

The purpose of the study is to see if the drug (BIVV020) works to improve symptoms in three populations of adults who have chronic inflammatory demyelinating polyneuropathy (CIDP): • Participants currently on the standard of care (SOC) treatment. • Participants treated previously with the SOC but with no meaningful improvement. • Participants that have not been treated with the SOC. Additional purposes of the study are to find out how safe and tolerable the drug is.

Jeff Allen
This study is NOT accepting healthy volunteers
STUDY00014727
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Inclusion Criteria:

• adults at least 18 years of age
• documented definite or probable diagnosis of CIDP (typical CIDP, pure motor CIDP, or Lewis-Sumner Syndrome)
• treated by Standard of care, or refractory to treatment or have not yet been treated (study staff will review)
• female and male participants must be willing to use two methods of highly effective contraception for a year after the last dose of study medication
Exclusion Criteria:

• polyneuropathy due to other causes
• other neurological or systemic disease that can cause symptoms and signs interfering with treatment or outcome assessment
• Poorly controlled diabetes (HbA1c >7%).
• history of suicidality in the six months prior to screening or currently at risk of committing suicide
• conditions (medical history or laboratory assessments) that may predispose the participant to excessive bleeding or increased risk of infection
• additional diagnosis or treatments (study staff will review)
Rare Diseases
CIDP, Clinics and Surgery Center (CSC), chronic inflammatory demyelinating polyneuropathy
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A Randomized, Parallel-Arm, Active Control, Multicenter Study Assessing the Safety and Efficacy of DEXTENZA for the Treatment of Ocular Pain and Inflammation Following Surgery for Pediatric Cataract

This randomized trial will compare the insertion of a DEXTENZA plug versus the standard prednisolone acetate suspension in the form of an eye drop to treat ocular pain and inflammation following cataract surgery. Its primary objective is to assess the safety of DEXTENZA compared to the control (prednisolone acetate) in children under the age of 6 years who are undergoing cataract surgery.

Raymond Areaux
STUDY00010045
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Inclusion Criteria:
Pediatric cataract diagnosis Subject is >0 - 3 years of age
Exclusion Criteria:
Any intraocular inflammation in the study eye Ocular hypertension or glaucoma Evidence of acute external ocular infections
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A Phase 3 Randomized, Placebo-controlled, Double-blind Study to Evaluate the Efficacy and Safety of BBP-418 (ribitol) in Patients with Limb Girdle Muscular Dystrophy 21 (LGMD21) (Fortify)

This study will use BBP-418 study drug in patients with LGMD to assess the clinical biomarkers, efficacy and safety of BBP-418 during the 36 months treatment phase.

Peter Kang
STUDY00018570
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Inclusion Criteria:

• 12 to 60 years of age
• genetically confirmed diagnosis of limb girdle muscular dystrophy
• have clinical symptoms of weakness
• weight at least 30 kg (66 lbs.)
• willing to use a highly effective method of birth control until 12 weeks after last dose of study medication
Exclusion Criteria:

• any significant medical or mental health diagnosis including abnormal lab values (study staff will review)
• surgery for scoliosis or other indication planned during the time of the study
• use of ribose or other sugar alcohol-containing supplement within 90 days of staring the study
• use of a systemic corticosteroid for the treatment of muscular dystrophy within 90 days of starting the study
Rare Diseases
Clinics and Surgery Center (CSC), Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I), Muscular Dystrophy
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PEPN2121 : A Phase 1/2 Study of Tiragolumab (NSC# 827799, IND# 161266) and Atezolizumab (NSC# 783608, IND# 161266) in Patients with Relapsed or Refractory SMARCB1 or SMARCA4 Deficient Tumors

This phase I/II trial studies how well tiragolumab and atezolizumab works when given to children and adults with SMARCB1 or SMARCA4 deficient tumors that that has either come back (relapsed) or does not respond to therapy (refractory). SMARCB1 or SMARCA4 deficiency means that tumor cells are missing the SMARCB1 and SMARCA4 genes, which is related to having more aggressive cancers that are harder to treat. Immunotherapy with monoclonal antibodies, such as tiragolumab and atezolizumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread.

Emily Greengard
STUDY00017037
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Inclusion Criteria:
Patients must be >= 12 months of age at the time of study enrollment. For part A, patients must be <18 years old at enrollment. For part B, there is no upper age limit The Part B (phase 2) cohorts will initially open concurrently with the part A but will only enroll patients at least 18 years of age. Patients <18 years of age will be included in the part B cohorts only after the tiragolumab monotherapy dose has been assessed to be safe in the part A portion Patients must have SMARCB1 (INI1) or SMARCA4 deficient tumors verified through institutional immunohistochemistry (IHC) or molecular confirmation of a pathologic tumor bi-allelic SMARCB1 (INI1) or SMARCA4 loss or mutation from a Clinical Laboratory Improvement Act (CLIA) certified lab with the following disease histologies: Renal medullary carcinoma Malignant rhabdoid tumor (extra-CNS) Atypical teratoid rhabdoid tumor (CNS) Poorly differentiated chordoma Epithelioid sarcoma Other SMARCB1 or SMARCA4 deficient tumors Note: Documentation of the institutional IHC or molecular testing must be uploaded via the RAVE system Part A: Patients must have either measurable or evaluable disease Part B: Patients must have either measurable disease per RECIST v1.1 for non-CNS tumors or CNS response criteria for CNS tumors Patients must have relapsed, refractory disease or newly diagnosed disease 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 (Karnofsky/Lansky score of > 50). Use Karnofsky for patients > 16 years of age and Lansky for patients =< 16 years of age. Note: Neurologic deficits in patients with CNS tumors 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: See Developmental Therapeutics (DVL) homepage on the Children's Oncology Group (COG) Members site for commercial and investigational agent classifications. For agents not listed, the duration of this interval must be discussed with the study chair and the study-assigned Research Coordinator prior to enrollment >= 21 days after the last dose of myelosuppressive chemotherapy (42 days if prior nitrosourea). Please refer to the table of myelosuppressive/Anticancer Agents on the COG website: https://www.cogmembers.org/uploadedFiles/Site/Disc/DVL/Documents/TableOfMyelosuppressiveAnti-CancerAgents.pdf 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. See the DVL homepage on the COG Members site for commercial and investigational agent classifications. For agents not listed, the duration of this interval must be discussed with the study chair and the study-assigned Research Coordinator prior to enrollment 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 Hematopoietic growth factors: >= 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 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]): Autologous stem cell infusion including boost infusion: >= 30 days Cellular therapy: >= 30 days after the completion of any type of cellular therapy (e.g., modified T cells, natural killer [NK] cells, dendritic cells, etc.) External 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 (e.g., radiolabeled antibody, iodine I 131 metaiodobenzylguanidine [131I MIBG]): >= 42 days after systemically administered radiopharmaceutical therapy Patients must not have had prior TIGIT targeting therapy Patients must not have received prior therapy with an anti- PD-1, anti-PD-L1, anti-PD-L2, or anti-CTLA4 agent or with an agent directed to another stimulatory or co-inhibitory T cell receptor (i.e. OX-40, CD137) Patients must not have received live/attenuated vaccine within 30 days of first dose of treatment Patients must not be receiving concomitant systemic steroid medications and > 14 days must have elapsed since last dose of systemic corticosteroid with the following exceptions: The use of physiologic doses of corticosteroids (5 mg/m^2/day up to 10 mg/day of prednisone equivalent) is acceptable The use of topical, inhaled, or ophthalmic corticosteroids are acceptable The use of acute, low-dose systemic immunosuppressant medication or a one-time pulse dose of systemic immunosuppressant medication (e.g., 48 hours of corticosteroids for a contrast allergy) are acceptable Treatment with systemic immunosuppressive medication (including, but not limited to, cyclophosphamide, azathioprine, methotrexate, thalidomide, and anti-tumor necrosis factor-alpha [TNF-alpha] agents) must have concluded >= 14 days prior to study enrollment For patients with solid tumors without known bone marrow involvement Peripheral absolute neutrophil count (ANC) >= 1000/uL (must be performed within 7 days prior to enrollment) 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) (must be performed within 7 days prior to enrollment) Patients with known bone marrow metastatic disease will be eligible for study provided they meet the blood counts above (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 A creatinine based on age/gender as follows (must be performed within 7 days prior to enrollment): Age; Maximum Serum Creatinine (mg/dL) 1 to < 2 years; Male: 0.6; Female: 0.6 2 to < 6 years; Male: 0.8; Female: 0.8 6 to < 10 years; Male: 1; Female: 1 10 to < 13 years; Male: 1.2; Female: 1.2 13 to < 16 years; Male: 1.5; Female: 1.4 >= 16 years; Male: 1.7; Female: 1.4 OR- a 24 hour urine creatinine clearance >= 70 mL/min/1.73 m^2 (must be performed within 7 days prior to enrollment) OR- a glomerular filtration rate (GFR) >= 70 mL/min/1.73 m^2. GFR must be performed using direct measurement with a nuclear blood sampling method OR direct small molecule clearance method (iothalamate or other molecule per institutional standard) (must be performed within 7 days prior to enrollment) Note: Estimated GFR (eGFR) from serum creatinine, cystatin C or other estimates are not acceptable for determining eligibility Bilirubin (sum of conjugated + unconjugated or total) =< 1.5 x upper limit of normal (ULN) for age (must be performed within 7 days prior to enrollment) Patients with known Gilbert disease: Total bilirubin < 3 x ULN Serum glutamic pyruvic transaminase (SGPT) (alanine aminotransferase [ALT]) =< 135 U/L (must be performed within 7 days prior to enrollment). For the purpose of this study, the ULN for SGPT is 45 U/L Albumin >= 2 g/dL (must be performed within 7 days prior to enrollment) 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 Nervous system disorders (Common Terminology Criteria for Adverse Events [CTCAE] v5) resulting from prior therapy must be =< grade 2, with the exception of decreased tendon reflex (DTR). Any grade of DTR is eligible International normalized ratio (INR) =< 1.5 (must be performed within 7 days prior to enrollment) Serum amylase =< 1.5 x ULN (must be performed within 7 days prior to enrollment) Serum lipase =< 1.5 x ULN (must be performed within 7 days prior to enrollment) Grade 1 or lower calcium level Note: can have history of hypercalcemia as long as controlled and asymptomatic
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 therapy and at least 90 days after final dose of tiragolumab and 150 days after final dose of atezolizumab, whichever is later. Abstinence is an acceptable method of birth control. It is not known if atezolizumab or tiragolumab are present in breast milk; however, IgG immunoglobulins are found in milk. Due to the potential for serious adverse reactions in the breastfed infant, breastfeeding is not recommended during therapy and for at least 150 days after the last dose of atezolizumab and 90 days after the last dose of tiragolumab, whichever is later Concomitant medications: Corticosteroids: Patients must not be receiving concomitant systemic steroid medications and >= 14 days must have elapsed since last dose of systemic corticosteroid with the following exceptions: The use of physiologic doses of corticosteroids (5 mg/m^2/day up to 10 mg/day of prednisone equivalent) is acceptable The use of topical, inhaled, or ophthalmic corticosteroids are acceptable The use of acute, low-dose systemic immunosuppressant medication or a one-time pulse dose of systemic immunosuppressant medication (e.g. 48 hours of corticosteroids for a contrast allergy) are acceptable 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 Systemic immunosuppressive medications (including, but not limited to, cyclophosphamide, azathioprine, methotrexate, and thalidomide) during study treatment because these agents could potentially alter the efficacy and safety of study treatments would not be eligible Patients must not have a known hypersensitivity to any component of tiragolumab or atezolizumab injection History of severe allergic anaphylactic reactions to chimeric or humanized antibodies or fusion proteins Known hypersensitivity to Chinese hamster ovary cell products or to any component of the atezolizumab or tiragolumab formulation Patients who have undergone allogeneic bone marrow or stem cell transplant are not eligible Patients with known, untreated CNS metastases will be excluded with the following exceptions: Patients with a history of CNS metastases that have been previously treated may enroll if sequential imaging shows no evidence for active disease in the CNS Patients must not have active autoimmune disease that has required systemic treatment in the past 12 months, or a documented history of clinically severe autoimmune disease, or a syndrome that requires systemic steroids or immunosuppressive agents. Subjects with vitiligo or resolved childhood asthma/atopy are not excluded. Replacement therapy (e.g. thyroxine, insulin, physiologic corticosteroid replacement therapy for adrenal or pituitary insufficiency, etc.) is not considered a form of systemic treatment and these patients are eligible Patients who have active immune deficiency are not eligible Patients who have known active tuberculosis are not eligible Hepatitis B or C infection: Patients < 18 years old at enrollment, who have known hepatitis B or C Patients >= 18 years old at enrollment with: Positive hepatitis B surface antigen (HBsAg), OR Positive total hepatitis B core antibody (HBcAb) who have a quantitative hepatitis B virus (HBV) deoxyribonucleic acid (DNA) >= 500 IU/mL, OR Positive hepatitis C virus (HCV) antibody with a positive HCV ribonucleic acid (RNA) test Note: For adults (>= 18 years old at enrollment), hepatitis B serology testing is required to determine eligibility. The HBV DNA test is required only for patients who have a negative HBsAg test, a negative HBsAb test, and a positive total HBcAb test. For adults (>= 18 years old at enrollment), hepatitis C serology testing is required to determine eligibility. The HCV RNA test is required only for patients who have a positive HCV antibody test Patients who have a known, recent Epstein-Barr virus (EBV) infection or known history of chronic, active infection are not eligible Patients who have history of or active human immunodeficiency virus (HIV) are not eligible except patients who are stable on anti-retroviral therapy, have a CD4 count >= 200/uL, and have an undetectable viral load Patients who have significant cardiovascular disease (such as New York Heart Association class III or IV congestive heart failure, myocardial infarction, or cerebrovascular accident) within 3 months prior to study enrollment, unstable arrhythmia, or unstable angina are not eligible Patients who have a major surgical procedure, other than for diagnosis, within 4 weeks prior to study enrollment, or the anticipation of the need for a major surgical procedure during the study are not eligible Patients who have a history of idiopathic pulmonary fibrosis, organizing pneumonia, drug-induced pneumonitis, idiopathic pneumonitis, or known active pneumonitis are not eligible. History of radiation pneumonitis in the radiation field is permitted Patients who have uncontrolled pleural effusion, pericardial effusion, or ascites requiring recurrent drainage procedures (once monthly or more frequently) are not eligible. Patients with indwelling catheters (e.g., PleurX) are allowed 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
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MT2021-25: Phase I/II Multicenter study evaluating the Safety and Efficacy of Allogeneic GDA-201 Natural Killer cells in patients with relapsed/refractory B-Cell Non-Hodgkin Lymphoma

This study is designed to assess the safety of GDA-201 + rituximab, as well as the maximum tolerated dose in patients with B cell lymphomas in phase I; in phase II, it will assess safety and efficacy of GDA-201 in cohorts of patients with follicular lymphoma, high grade B cell lymphoma (including diffuse large B-cell), high grade B cell lymphoma not otherwise specified, and primary mediastinal B cell lymphoma.

Veronika Bachanova, MD
STUDY00015044
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Inclusion Criteria:
Patients must have relapsed/refractory FL or HGBCL/DLBCL that has failed conventional therapy defined as follows: Received at least 2 prior lines of therapy Transplant ineligible patients allowed assuming they meet criterion a. Patients who received prior chimeric antigen receptor modified T-cells (CAR-T) cell therapy or are considered ineligible for CAR-T therapy per the investigator's discretion FL transformed to HGBCL: Must have received at least 1 line of therapy after transformation to DLBCL/HGBCL Patients must be at least 18 years of age Patients must have adequate hematologic, hepatic, renal, cardiac and pulmonary function prior to any study treatment.
Exclusion Criteria:
CNS lymphoma Time between previous treatment and first dose of study treatment (rituximab): Allogeneic HSCT < 6 months prior to study treatment Autologous HSCT < 3 months prior to study treatment CAR-T < 2 months prior to study treatment
Clinics and Surgery Center (CSC)
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A Randomized, Double-Blind, Placebo-Controlled Multiple-Center, Efficacy and Safety Study of ZYN002 Administered as a Transdermal Gel to Children and Adolescents with Fragile X Syndrome - RECONNECT (RECONNECT)

This is a randomized, double-blind, placebo-controlled, multiple-center study, to assess the efficacy and safety of CBD administered as ZYN002, a transdermal gel, for the treatment of children and adolescent patients with FXS. Qualified male and female patients with FXS will enter a two-week single-blind placebo lead-in period. Following the placebo lead-in, patients meeting randomization criteria will receive double-blind treatment for 16 weeks. The study will be comprised of a Screening visit and a combination of seven onsite (face-to-face) and virtual study visits. Approximately 204 male and female patients, ages 3 to < 18 years, will be randomized 1:1 to either trial drug or placebo. Randomization will be stratified by gender (male, female), methylation status (complete, partial), and by weight (≤50 kg, >50 kg).

Amy Esler
STUDY00014264
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Inclusion Criteria:
Male or female children and adolescents aged 3 to < 23 years, at the time of Screening. Patient resides with caregiver who will continue to provide consistent care throughout the study. Judged by the Investigator to be in generally good health at Screening based upon the results of medical history, physical exam, 12-lead ECG and clinical laboratory test results. -Laboratory results outside the reference range must be documented as not clinically significant by both the Investigator and Sponsor. Participants must have a diagnosis of FXS through molecular documentation of full mutation of the FMR1 gene documented through genetic testing at Screening. Patients with a history of seizure disorders must currently be receiving treatment with a stable regimen of no more than two anti-seizure medications (ASMs) for the four weeks preceding study Screening; or must be seizure-free for one year if not currently receiving ASMs. Patients taking psychoactive medication(s) should be on a stable regimen of not more than three such medications for at least fours weeks preceding Screening and must maintain that regimen throughout the study. Psychoactive medications include (but are not limited to) antipsychotics, antidepressants, anxiolytics, attention-deficit / hyperactivity disorder (ADHD) medications, and medications for sleep. If patients are receiving non-pharmacological, behavioral and/or dietary interventions, they must be stable and have been doing so for three months prior to screening. Patients have a body mass index between 12-30 kg/m2 (inclusive). Females of childbearing potential must have a negative serum pregnancy test at the Screening Visit and a negative serum or urine pregnancy test at all designated visits. Patients and parents/caregivers must be adequately informed of the nature and risks of the study and given written informed consent prior to Screening. Patients and parents/caregivers agree to abide by all study restrictions and comply with all study procedures, and in the Investigator's opinion, are reliable and willing and able to comply with all protocol requirements and procedures.
Exclusion Criteria:
Females who are pregnant, nursing or planning a pregnancy; females of childbearing potential and male patients with a partner of childbearing potential who are unwilling or unable to use an acceptable method of contraception as outlined below for the duration of therapy and for three months after the last dose of study medication. Standard acceptable methods of contraception include abstinence (defined as refraining from heterosexual intercourse from screening to three months after the last dose of study medication) or the use of a highly effective method of contraception, including hormonal contraception, diaphragm, cervical cap, vaginal sponge, condom, spermicide, vasectomy, or intrauterine device. The reliability of sexual abstinence needs to be evaluated in relation to the duration of the clinical trial and the preferred and usual lifestyle of the subject. Periodic abstinence (calendar, symptothermal, post-ovulation methods) is not an acceptable method of contraception. Patient has transitioned to independent living or living in a residential facility such as a university setting or congregate care. History of significant allergic condition, significant drug-related hypersensitivity, or allergic reaction to any compound or chemical class related to ZYN002 or its excipients. Exposure to any investigational drug or device less than or equal to 30 days prior to Screening or at any time during the study. Alanine aminotransferase (ALT), aspartate aminotransferase (AST) or total bilirubin levels greater than or equal to 2 times the upper limit of normal or alkaline phosphatase levels greater than or equal to 3 times the upper limit of normal. Use of cannabis or any THC or CBD-containing product within 3 months of Screening Visit or during the study (aside from ZYN002). Patient has a positive drug screen, including ethanol, cocaine, THC, barbiturates, amphetamines (unless prescribed), benzodiazepines (except midazolam or comparable administered for blood draws and ECG collection), and opiates. Patient is using the following AEDs (medications for the treatment of seizures and/ or epilepsy): clobazam, phenobarbital, ethosuximide, felbamate, carbamazepine, phenytoin, or vigabatrin. Patient is using a strong inhibitor/inducer of CYP3A4 or sensitive substrate of CYP3A4 including but not limited to the following medications: midazolam (except single doses administered for the purposes of obtaining blood samples and ECG's), oral ketoconazole, fluconazole, nefazadone, rifampin, alfentanil, alfuzosin, amiodarone, cyclosporine, dasatinib, docetaxol, eplerenone, ergotamine, everolimus, fentanyl, halofantrine, irinotecan, lapatinib, levomethadyl, lumefantrine, nilotinib, pimozide, quinidine, ranolazine, sirolimus, tacrolimus, temsirolimus, toremifene, tretinioin, vincristine, vinorelbine, St. John's Wort, and grapefruit Juice/products. Patients may not be taking any benzodiazepines (except single doses administered for the purposes of obtaining blood samples and ECGs) at screening or throughout the study. Patient is expected to initiate or change pharmacologic or non-pharmacologic interventions during the course of the study. Patient has an advanced, severe, or unstable disease that may interfere with the study outcome evaluations. Patient has acute or progressive neurological disease, psychosis, schizophrenia or any other psychiatric disorder or severe mental abnormalities (other than FXS) that are likely to require changes in drug therapy or interfere with the study objectives or ability to adhere to protocol requirements. Patient has a positive result for the presence of HBsAg, HCV, or HIV antibodies. Patient has known history of cardiovascular disease, advanced arteriosclerosis, structural cardiac abnormality, cardiomyopathy, serious heart rhythm abnormalities, coronary artery disease, cardiac conduction problems, exercise-related cardiac events including syncope and pre-syncope, risk factors for Torsades de pointes (TdP) (e.g., heart failure, hypokalemia, family history of Long QT Syndrome), or other serious cardiac problems. Any clinically significant condition or abnormal findings at the Screening Visit that would, in the opinion of the Investigator, preclude study participation or interfere with the evaluation of the study medication. Any skin disease or condition including eczema, psoriasis, melanoma, acne, contact dermatitis, scarring, imperfections, lesions, tattoos, or discoloration that may affect treatment application, application site assessments or absorption of the trial drug. History of treatment for, or evidence of, drug abuse within the past year. Previous participation in a ZYN002 study (with the exception of patients who were screen failures in Study ZYN2-CL-016 and did not enter Study ZYN2-CL-017). Patient responds "yes" to Question 4 or 5 on the C-SSRS (Children) during Screening or at any time on study.
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A Parallel-group, Two-staged, Phase 2/3, Randomized, Multicenter Study to Evaluate the Efficacy and Safety of REC-2282 in Participants with Progressive NF2 Mutated Meningiomas (POPLAR-NF2)

This study will investigate the efficacy and safety of REC-2282 in patients with progressive NF2 mutated meningiomas who have either NF2 disease-related meningioma or recurrent sporadic meningiomas that have NF2 mutations. This study is a parallel-group, two-staged, Phase 2/3, randomized, multi-center study with two cohorts: Cohort A followed by Cohort B. The purpose of Cohort A is to provide early data on efficacy and safety of REC-2282 in participants with progressive NF2 mutated meningiomas, and provide guidance for the correct dose, population, sample size, and endpoint for the confirmatory part of the study (Cohort B). Additional goals for Cohort A are to assess effects of food on drug absorption. The purpose of Cohort B of the study is to assess the efficacy and safety of REC-2282 compared with placebo in participants with progressive NF2 mutated meningiomas.

Christopher Moertel, MD
STUDY00015488
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Inclusion Criteria:
≥12 years of age and weighing at least 40 kg Progressive meningioma that is amenable to volumetric analysis Has either 1) sporadic meningioma with confirmed NF2 mutation; or, 2) confirmed diagnosis of NF2 disease (revised Manchester criteria); or, 3) at least one NF2-related tumor (with pathogenic germline or proven mosaic NF2 variant) Adequate bone marrow function Has provided written informed consent/assent to participate in the study
Exclusion Criteria:
Progressive disease associated with significant or disabling clinical symptoms likely to require surgery or radiation therapy within the next 3 months. Received prior surgery, radiosurgery, or laser interstitial thermal therapy in the target tumor, or immediately adjacent to the target tumor within 6 months prior to screening. Received an anti- tumor agent for meningioma within 3 months, or 5 half-lives (whichever is longer), prior to screening. History of an active malignancy within the previous 3 years except for localized cancers that are considered cured, and, in the opinion of the investigator, present a low risk of recurrence. Received another investigational drug within 30 days prior to screening Pregnant, lactating, or is planning to attempt to become pregnant or impregnate someone during this study or within 90 days after the last dose of IMP.
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PEPN2011 - A Phase 1/2 Study of Tegavivint (IND#156033, NSC#826393) in Children, Adolescents, and Young Adults with Recurrent or Refractory Solid Tumors, Including Lymphomas and Desmoid Tumors

This phase I/II trial evaluates the highest safe dose, side effects, and possible benefits of tegavivint in treating children, adolescents, and young adults with recurrent or refractory solid tumors, including lymphomas and desmoid tumors.

Emily Greengard
STUDY00014319
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Inclusion Criteria:
PART A: Patients must be >= 12 months and =< 21 years of age at the time of study enrollment PART B: Patients must be >= 12 months and =< 30 years of age at the time of study enrollment Patients with recurrent or refractory solid tumors including non-Hodgkin lymphoma and desmoid tumors are eligible. Patients must have had histologic verification of malignancy at original diagnosis or relapse PART A: Patients with relapsed or refractory solid tumors, including patients with non-Hodgkin lymphoma and desmoid tumors PART B: Patients with recurrent or refractory Ewing sarcoma, desmoid tumors, osteosarcoma, liver tumors (HCC and hepatoblastoma), Wilms tumor, and tumors with Wnt pathway aberrations. For the Wnt pathway aberrations cohort we will include the most common CTNNB1 mutations (S37F, S45F, T41A, S45P, S33C, S37C, D32Y, S33F, T41I, G34R, G34V, D32N, S33P, G34E, D32G) as well as any loss of function mutations in the APC, Axin2FBXW7, TCF7L2, and RNF43 genes or any gain-of-function mutations in the GSK3B, LRP6, and LGR5 genes. For patients without prior sequencing, immunohistochemistry (IHC), is required. IHC showing strong nuclear beta-catenin staining will be accepted for the following tumor types: colorectal carcinoma, melanoma, endometrial cancer, ovarian cancer, neuroblastoma, non-Hodgkin lymphoma, pancreatic ductal adenocarcinoma, and solid pseudopapillary tumor of the pancreas PART A: Patients must have either measurable or evaluable disease. For desmoid tumors, the patient must have disease that the investigator deems unresectable or sufficiently morbid or potentially life-threatening that there is favorable risk/benefit to the patient to participate in the trial PART B: Patients must have measurable disease. For desmoid tumors, the patient must have measurable disease that the investigator deems unresectable or sufficiently morbid or potentially life-threatening that there is favorable risk/benefit to the patient to participate in the trial 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 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 Solid tumor patients: >= 21 days after the last dose of myelosuppressive chemotherapy (42 days if prior nitrosourea) Non-Hodgkin lymphoma patients A waiting period prior to enrollment is not required for patients receiving standard maintenance chemotherapy (i.e., corticosteroid, vincristine, thioguanine [6MP], and/or methotrexate) >= 14 days must have elapsed after the completion of other cytotoxic therapy, with the exception of hydroxyurea, for patients not receiving standard maintenance therapy NOTE: Cytoreduction with hydroxyurea must be discontinued >= 24 hours prior to the start of protocol therapy Anti-cancer agents not known to be myelosuppressive (e.g., not associated with reduced platelet or absolute neutrophil counts [ANC]): >= 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 (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 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 infusion (DLI) or boost infusion: >= 84 days after infusion and no evidence of graft versus host disease (GVHD) Autologous stem cell infusion including boost infusion: >= 42 days. Cellular therapy: >= 42 days after the completion of any type of cellular therapy (e.g., modified T cells, natural killer [NK] cells, dendritic cells, etc.). External beam 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 (e.g., radiolabeled antibody, iobenguane I-131 [131I MIBG]): >= 42 days after systemically administered radiopharmaceutical therapy Patients must not have received prior exposure to tegavivint PATIENTS WITH SOLID TUMORS WITHOUT KNOWN BONE MARROW INVOLVEMENT: Peripheral absolute neutrophil count (ANC) >= 1000/uL (within 7 days prior to enrollment) 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) (within 7 days prior to enrollment) PATIENTS WITH SOLID TUMORS WITHOUT KNOWN BONE MARROW INVOLVEMENT: Hemoglobin >= 8.0 g/dL at baseline (may receive red blood cell [RBC] transfusions) (within 7 days prior to enrollment) Patients with known bone marrow metastatic disease will be eligible for study provided they meet blood counts (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 on Part A must be evaluable for hematologic toxicity Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70 mL/min/1.73 m^2 or a creatinine based on age/gender as follows (within 7 days prior to enrollment): Age; maximum serum creatinine Age 1 to < 2 years; 0.6 mg/dL (male); 0.6 mg/dL (female) Age 2 to < 6 years; 0.8 mg/dL (male); 0.8 mg/dL (female) Age 6 to < 10 years; 1 mg/dL (male); 1 mg/dL (female) Age 10 to < 13 years; 1.2 mg/dL (male); 1.2 mg/dL (female) Age 13 to < 16 years; 1.5 mg/dL (male); 1.4 mg/dL (female) Age >= 16 years; 1.7 mg/dL (male); 1.4 mg/dL (female) PATIENTS WITH SOLID TUMORS: Bilirubin (sum of conjugated + unconjugated or total) =< 1.5 x upper limit of normal (ULN) for age (within 7 days prior to enrollment) PATIENTS WITH SOLID TUMORS: Serum glutamic pyruvic transaminase (SGPT) (alanine aminotransferase [ALT]) =< 135 U/L. For the purpose of this study, the ULN for SGPT is 45 U/L (within 7 days prior to enrollment) PATIENTS WITH SOLID TUMORS: Albumin >= 2 g/dL (within 7 days prior to enrollment)
Exclusion Criteria:
Pregnant or breast-feeding women will not be entered on this study 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. Abstinence is an acceptable method of birth control 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 the 1st dose of tegavivint to the end of the study Patients who have received bisphosphonates within 4 weeks prior to study enrollment will be excluded Patients who have received denosumab within 180 days prior to study enrollment will be excluded Patients with primary brain tumors are ineligible Patients with known central nervous system (CNS) metastasis, except for craniopharyngeal tumors, will be excluded Patients with a known metabolic bone disease (ex: hyperparathyroidism, Paget's disease, osteomalacia) are not eligible Patients with a disorder associated with abnormal bone metabolism will be excluded Patients with grade >= 2 hypocalcemia that is not corrected with oral calcium supplementation will be excluded Patients with vitamin D < 20 ng/mL will require supplementation, or will otherwise be excluded. Patients must agree to take vitamin D +/- calcium supplements (if necessary) according to institutional or published guidelines. Additional calcium supplementation is not required if adequate dietary intake can be ascertained Patients with pre-existing grade 3 osteoporosis are excluded 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
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COG ACNS1831 - A Phase 3 Randomized Study of Selumetinib (IND # 77782) versus Carboplatin/Vincristine in Newly Diagnosed or Previously Untreated Neurofibromatosis Type 1 (NF1) Associated Low-Grade Glioma (LGG)

Christopher Moertel, MD
STUDY00008583
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Inclusion Criteria:
Patients must be >= 2 years and =< 21 years at the time of enrollment Patients must have a body surface area (BSA) of >= 0.5 m^2 at enrollment Patients must have neurofibromatosis type 1 (NF1) based on clinical criteria and/or germline genetic testing Patients must be newly diagnosed or have previously diagnosed NF-1 associated LGG that has not been treated with any modality other than surgery For patients with optic pathway gliomas (OPGs): Newly-diagnosed patients with OPG are eligible if there are neurologic symptoms (including visual dysfunction, as defined below) or other exam findings associated with the tumor Previously-diagnosed patients with OPG are eligible if they have new or worsening neurologic symptoms (including visual dysfunction, as defined below) or have tumor growth For both newly-diagnosed and previously-diagnosed OPG, the patient may be eligible, irrespective of whether there has been tumor growth or other neurological symptoms or worsening, if they meet at least one of the following visual criteria: Visual worsening, defined as worsening of visual acuity (VA) or visual fields (VF) documented within the past year (by examination or history); OR Significant visual dysfunction (defined as VA worse than normal for age by 0.6 logMAR [20/80, 6/24, or 2.5/10] or more in one or both eyes) For patients with LGG in other locations (i.e., not OPGs): Newly-diagnosed patients with LGG are eligible if there are neurologic symptoms or other exam findings associated with the tumor NOTE: Newly-diagnosed patients with LGG without associated neurologic symptoms or exam findings are not eligible Previously-diagnosed patients with LGG are eligible if they have new or worsening neurologic symptoms or have tumor growth Although not required, if a biopsy/tumor resection is performed, eligible histologies will include all tumors considered LGG or low-grade astrocytoma (World Health Organization [WHO] grade I and II) by 5th edition WHO classification of central nervous system (CNS) tumors with the exception of subependymal giant cell astrocytoma Patients must have two-dimensional measurable tumor >= 1 cm^2 Patients with metastatic disease or multiple independent primary LGGs are allowed on study Creatinine clearance or radioisotope glomerular filtration Rate (GFR) >= 70 mL/min/1.73 m^2 OR a serum creatinine based on age/gender (within 7 days prior to enrollment) as follows: Age; maximum serum creatinine (mg/dL) 2 to < 6 years; 0.8 (male) and 0.8 (female) 6 to < 10 years; 1 (male) and 1 (female) 10 to < 13 years; 1.2 (male) and 1.2 (female) 13 to < 16 years; 1.5 (male) and 1.4 (female) >= 16 years; 1.7 (male) and 1.4 (female) Total bilirubin =< 1.5 x upper limit of normal (ULN) for age (within 7 days prior to enrollment) (children with a diagnosis of Gilbert's syndrome will be allowed on study regardless of their total and indirect [unconjugated] bilirubin levels as long as their direct [conjugated] bilirubin is < 3.1 mg/dL) Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) =< 3 x upper limit of normal (ULN) = 135 U/L (within 7 days prior to enrollment). For the purpose of this study, the ULN for SGPT is 45 U/L Albumin >= 2 g/dL (within 7 days prior to enrollment) Left ventricular ejection fraction (LVEF) >= 53% (or institutional normal; if the LVEF result is given as a range of values, then the upper value of the range will be used) by echocardiogram (within 4 weeks prior to enrollment) Corrected QT (QTc) interval =< 450 msec by electrocardiography (EKG) (within 4 weeks prior to enrollment) Absolute neutrophil count >= 1,000/uL (unsupported) (within 7 days prior to enrollment) Platelets >= 100,000/uL (unsupported) (within 7 days prior to enrollment) Hemoglobin >= 8 g/dL (may be supported) (within 7 days prior to enrollment) Patients with a known seizure disorder should be stable and should have not experienced a significant increase in seizure frequency within 2 weeks prior to enrollment Patients 2-17 years of age must have a blood pressure that is =< 95th percentile for age, height, and gender at the time of enrollment. Patients >= 18 years of age must have a blood pressure =< 130/80 mmHg at the time of enrollment (with or without the use of antihypertensive medications). Note: Adequate blood pressure can be achieved using medication for the treatment of hypertension All patients must have ophthalmology toxicity assessments performed within 4 weeks prior to enrollment For all patients, an MRI of the brain (with orbital cuts for optic pathway tumors) and/or spine (depending on the site(s) of primary disease) with and without contrast must be performed within 4 weeks prior to enrollment For patients who undergo a surgery on the target tumor (not required), a pre- and post-operative* MRI of the brain (with orbital cuts for optic pathway tumors) or spine (depending on the site(s) of primary disease) with and without contrast must also be performed within 4 weeks prior to enrollment The post-operative MRIs should be performed ideally within 48 hours after surgery if possible 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 Patients must have the ability to swallow whole capsules Patients must have receptive and expressive language skills in English or Spanish to complete the quality of life (QOL) and neurocognitive assessments All patients and/or their parents or legal guardians must sign a written informed consent. All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met.
Exclusion Criteria:
Patients must not have received any prior tumor-directed therapy including chemotherapy, radiation therapy, immunotherapy, or bone marrow transplant. Prior surgical intervention is permitted Patients with a concurrent malignancy or history of treatment (other than surgery) for another tumor within the last year are ineligible Patients may not be receiving any other investigational agents Patients with any serious medical or psychiatric illness/ condition, including substance use disorders likely in the judgement of the investigator to interfere or limit compliance with study requirements/treatment are not eligible Patients who, in the opinion of the investigator, are not able to comply with the study procedures are not eligible Female patients who are pregnant are not eligible since fetal toxicities and teratogenic effects have been noted for several of the study drugs. A pregnancy test is required for female patients of childbearing potential Lactating females who plan to breastfeed their infants are not eligible Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation and for 12 weeks after stopping study therapy are not eligible Note: Women of child-bearing potential and males with sexual partners who are pregnant or who could become pregnant (i.e., women of child-bearing potential) should use effective methods of contraception for the duration of the study and for 12 weeks after stopping study therapy to avoid pregnancy and/or potential adverse effects on the developing embryo Cardiac conditions: Known genetic disorder that increases risk for coronary artery disease. Note: The presence of dyslipidemia in a family with a history of myocardial infarction is not in itself an exclusion unless there is a known genetic disorder documented Symptomatic heart failure New York Heart Association (NYHA) class II-IV prior or current cardiomyopathy Severe valvular heart disease History of atrial fibrillation Ophthalmologic conditions: Current or past history of central serous retinopathy Current or past history of retinal vein occlusion or retinal detachment Patients with uncontrolled glaucoma If checking pressure is clinically indicated, patients with intraocular pressure (IOP) > 22 mmHg or ULN adjusted by age are not eligible Ophthalmological findings secondary to long-standing optic pathway glioma (such as visual loss, optic nerve pallor, or strabismus) or longstanding orbito-temporal plexiform neurofibroma (PN), such as visual loss, strabismus) will NOT be considered a significant abnormality for the purposes of the study Treatments and/or medications patient is receiving that would make her/him ineligible, such as: Supplementation with vitamin E greater than 100% of the daily recommended dose. Any multivitamin containing vitamin E must be stopped prior to study enrollment even if less than 100% of the daily recommended dosing for vitamin E Surgery within 2 weeks prior to enrollment, with the exception of surgical placement for vascular access or cerebrospinal fluid (CSF) diverting procedures such as endoscopic third ventriculostomy (ETV) and ventriculo-peritoneal (VP) shunt. Note: Patients must have healed from any prior surgery prior to enrollment Patients who have an uncontrolled infection are not eligible
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A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of SPR001 (Tildacerfont) in Reducing Supraphysiologic Glucocorticoid Use in Adult Subjects with Classic Congenital Adrenal Hyperplasia

SPR001-204 will be a randomized, double-blind, placebo-controlled study that will evaluate the potential of tildacerfont to reduce GC burden in adult subjects with classic CAH who have lower limit of detection (LLD) ≤ A4 ≤ 1.5x upper limit of normal (ULN) and are on supraphysiologic doses of GC therapy. SPR001-204 will be the first study of tildacerfont to evaluate GC dose reduction. In addition, Study SPR001-204 will characterize clinical outcomes after up to 52 weeks of treatment with tildacerfont.

Kyriakie Sarafoglou
This study is NOT accepting healthy volunteers
STUDY00011764
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Inclusion Criteria:

• at least 18 years old
• CAH due to 21-hydroxylase deficiency based on genetic mutation in CYP21A2 and/or documented elevated 17-OHP - currently treatment with HC, HC acetate, prednisone, prednisolone, methylprednisolone (or a combination)
• on a stable dose of GC replacement for at least 1 month
• people with the salt-wasting form of CAH on been on a stable dose of mineralocorticoid replacement for at least 1 month
Exclusion Criteria:

• diagnosis of any other known form of classic CAH (not due to 21-hydroxylase deficiency)
• history of bilateral adrenalectomy or hypopituitarism
• allergy or hypersensitivity to tildacerfont, any of its forms or any other CRF1 receptor antagonist
• clinical signs or symptoms of adrenal insufficiency
Diabetes & Endocrine
congenital adrenal hyperplasia, CAH
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A double-blind, randomized, placebo-controlled study to assess the safety and efficacy of nebulized PC945 when added to systemic antifungal therapy for the treatment of refractory invasive pulmonary aspergillosis

Jo-Anne Young, MD
This study is NOT accepting healthy volunteers
STUDY00017568
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Inclusion Criteria:

• diagnosis of invasive pulmonary aspergillosis that hasn't responded to treatment
Exclusion Criteria:

• surgical or medical condition that makes participation difficult or potentially unsafe
• require care in an intensive care unit
Breathing, Lung & Sleep Health, Infectious Diseases
Clinics and Surgery Center (CSC), antifungal therapy, aspergillosis, fungal infection, lung infection, pulmonary aspergillosis
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A Multicenter, Randomized, Double-Blind, PlaceboControlled, Operationally Seamless, Adaptive Phase 2/3 Study to Evaluate the Efficacy and Safety of Baricitinib in Adult Patients with Severe or Very Severe Alopecia Areata

This study will test the hypothesis that the higher dose of baricitinib is superior to placebo in the treatment of patients with severe or very severe Alopecia Areata (AA) and safety and efficacy of both doses of baricitinib. Baricitinib is an oral inhibitor of JAKs, and preclinical information and clinical reports support the use of JAK inhibitors for AA.

Maria Hordinsky
This study is NOT accepting healthy volunteers
STUDY00005177
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Inclusion Criteria:

• at least 18 years and no more than 60 years old for men and no more than 70 years of age for women
• self-identify as Black or African American race
• severe or very severe AA
• agree not to use any AA treatments during the study
• not pregnant or breastfeeding and must agree to not become pregnant while on the study
Exclusion Criteria:

• primarily diffuse type of AA
• currently experiencing other forms of alopecia
• currently experiencing or have a history of other medical conditions (heart, lung, liver, etc.)
• recent use of other treatments for AA (study staff will review)
Dermatology (Skin, Hair & Nails)
AA, Alopecia Areata, Baldness, Hair Loss
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MT2021-08: Phase II, Open-Label, Prospective Study of T Cell Receptor Alpha/Beta Depletion (A/B TCD) Peripheral Blood Stem Cell (PBSC) Transplantation for Children and Adults with Hematological Malignancies

Margaret MacMillan, MD
This study is NOT accepting healthy volunteers
STUDY00016450
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Inclusion Criteria:

• hematological cancer needing stem cell transplant
• 60 years old or younger
Exclusion Criteria:

• pregnant or breast feeding
• active infection
• positive for HIV, Hepatitis B or C
• brain metastasis
Cancer
Blood Cancers, Hematologic Malignancy, Leukemia, Stem Cell Transplant, Clinics and Surgery Center (CSC)
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MT2019-06: A Phase 3 Study Evaluating Gene Therapy by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo with the LentiGlobin BB305 Lentiviral Vector in Subjects with Sickle Cell Disease.

Evaluate the efficacy of treatment with bb1111 (also known as LentiGlobin BB305 Drug Product for Sickle Cell Disease) in subjects with sickle cell disease (SCD).

Ashish Gupta
STUDY00006923
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Inclusion Criteria:
Have a diagnosis of SCD, with either βS/βS, βS/β0, or βS/β+ genotype. Be ≥2 and ≤50 years of age at time of consent. Weigh a minimum of 6 kg. Have a Karnofsky performance status of ≥60 (≥16 years of age) or a Lansky performance status of ≥60 (<16 years of age). Be treated and followed for at least the past 24 months prior to Informed Consent in medical center(s) that maintained detailed records on sickle cell disease history. In the setting of appropriate supportive care measures (e.g., pain management plan), have experienced at least 4 protocol-defined VOEs in the 24 months prior to informed consent. Have either experienced HU failure at any point in the past or must have intolerance to HU (intolerance is defined as the patient being unable to continue to take HU per PI judgment). Female and male subjects of childbearing potential agree to use 1 method of highly effective contraception from Screening to at least 6 months after drug product infusion. Provision of written informed consent for this study by subject, or as applicable, subject's parent(s)/legal guardian(s).
Exclusion Criteria:
Subjects for whom allogeneic hematopoietic stem cell transplantation (allo-HSCT) is medically appropriate per PI judgment and a willing, human leukocyte antigen (HLA)-matched related hematopoietic stem cell donor is available. Severe cerebral vasculopathy, defined by any history of overt ischemic or hemorrhagic stroke, a history of abnormal transcranial Doppler (TCD) or TCD imaging (TCDI) for subjects ≤ 16 years of age (e.g. TCD velocity >200 cm/sec) requiring ongoing chronic transfusions, a Screening TCD or TCDI velocity > 200 cm/sec (central read), a Screening MRA showing > 50% stenosis or occlusion in the circle of Willis (central read), or a Screening MRA showing the presence of Moyamoya (central read). Positive for presence of human immunodeficiency virus type 1 or 2 (HIV-1 or HIV-2), hepatitis B, hepatitis C, human T-lymphotropic virus-1 (HTLV-1), active syphilis. Clinically significant, active bacterial, viral, fungal, or parasitic infection Advanced liver disease, such as clear evidence of liver cirrhosis, active hepatitis or significant fibrosis (based on MRI or liver biopsy) liver iron concentration ≥15 mg/g unless liver biopsy shows no evidence of cirrhosis, active hepatitis or significant fibrosis Inadequate bone marrow function, as defined by an absolute neutrophil count of <1×10^9/L (<0.5×10^9/L for subjects on hydroxyurea treatment) or a platelet count <100×10^9/L. Any contraindications to the use of plerixafor during the mobilization of hematopoietic stem cells and any contraindications to the use of busulfan and any other medicinal products required during the myeloablative conditioning, including hypersensitivity to the active substances or to any of the excipients. Patients needing therapeutic anticoagulation treatment during the period of conditioning through platelet engraftment Unable to receive pRBC transfusion. Prior receipt of an allogeneic transplant. Prior receipt of gene therapy. Any prior or current malignancy or immunodeficiency disorder, except previously treated, non-life threatening, cured tumors such as squamous cell carcinoma of the skin. Immediate family member with a known or suspected Familial Cancer Syndrome. Female subject is breastfeeding, pregnant or will attempt to become pregnant from Screening to at least 6 months after drug product infusion. Any other condition that would render the subject ineligible for HSCT. Participation in another clinical study with an investigational drug within 30 days of screening. Presence of a chromosomal abnormality or genetic mutation that may put the subject at an increased risk of myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) per Investigator's judgment. Presence of genetic mutations that result in the inactivation of 2 or more α-globin genes
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Disparities in REsults of Immune Checkpoint Inhibitor Treatment (DiRECT): A Prospective Cohort Study of Cancer Survivors Treated With Anti-PD-1/Anti-PD-L1 Immunotherapy in a Community Oncology Setting (DiRECT)

This study compares treatment outcomes between patients of African American/Black (AA) ancestry and European American/White (EA) ancestry currently receiving immune checkpoint inhibitor treatment. Collecting samples of blood and saliva and health and treatment information from racially diverse patients receiving immune checkpoint inhibitor treatment over time may help doctors better understand health care disparities among all cancer patients.

Edward Greeno, M.D.
This study is NOT accepting healthy volunteers
MMCORC058
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Inclusion Criteria:

• self-identify as African/African American/black (AA), or European American/ Caucasian/white (EA), or Hispanic/Latino ethnicity in combination with an AA or EA racial identity
• current diagnosis of invasive cancer at stage I-IV
• scheduled to receive anti-PD-1/-L1 ICI-containing therapy as standard of care treatment alone or in combination with co-treatments
Exclusion Criteria:

• identify as Asian, Pacific Islander, or American Indian/Alaskan Native
• diagnosed with melanoma (because melanoma is very rare in AAs)
• received prior immunotherapy for cancer,
Cancer
Immune checkpoint inhibitor, Cancer, Disparities
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A PHASE 2, OPEN-LABEL, SINGLE-ARM, COHORT STUDY TO EVALUATE THE SAFETY, EFFICACY, AND PHARMACOKINETICS OF SPARSENTAN TREATMENT IN PEDIATRIC SUBJECTS WITH SELECTED PROTEINURIC GLOMERULAR DISEASES&#13;&#10;(EPPIK) (EPPIK)

Currently, there are no approved treatment options for pediatric subjects with proteinuric kidney conditions. The study will look at the safety, efficacy, and pharmacokinetic (PK) trial in children ≥1 to <18 years treated for up to 108 weeks with the drug sparsentan.

Michelle Rheault
This study is NOT accepting healthy volunteers
STUDY00013216
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Inclusion Criteria:

• Child 1 to 18 years old
• Diagnosed by biopsy with specific types of glomerular disease & protein in the urine
• Blood pressure is within normal range for age
• Maintained on a stable dose of immunosuppressive medications
Exclusion Criteria:

• Weight less than 7.3 kg at screening.
• Disease due to to viral infections, drug toxicities, or cancer.
• Kidney function is below the minimum required
Rare Diseases, Children's Health, Kidney, Prostate & Urinary
Glomerulosclerosis, Immunoglobulin A Nephropathy, IgA Vasculitis, Alport Syndrome
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A Prospective, Multi-Center, Single-Arm, Open-Label, Observational Study on the Safety and Effectiveness of the ProSomnus EVO Sleep and Snore Device in the Treatment of Severe Obstructive Sleep Apnea

The primary objectives of this study are to evaluate the therapeutic effectiveness and safety profile of the ProSomnus EVO Sleep and Snore Device in individuals with severe obstructive sleep apnea (OSA). The secondary objective is to evaluate the effect of the EVO Sleep and Snore Device on Epworth Sleepiness Scale (ESS) scores.

Bimaje Akpa
This study is NOT accepting healthy volunteers
STUDY00016436
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Inclusion Criteria:

• Age: 18-80 years
• Diagnosed with severe obstructive sleep apnea (OSA)
• Body mass index (BMI) < 40 kg/m2
Exclusion Criteria:

• Unable to breathe comfortably through the nose
• History of surgery to correct OSA
• History of temporomandibular joint (TMJ) disorder
• Loose teeth or gum disease
• Uncontrolled blood pressure
• Pregnancy
• Other significant medical history
Breathing, Lung & Sleep Health
Oral Appliance, Obstructive Sleep Apnea, Sleep Apnea, ProSomnus EVO Sleep and Snore Device
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KIN-1902-2001: A Randomized, Double-blind, Placebo-controlled Phase 2 Study with Open-label Extension to Assess the Efficacy and Safety of Namilumab in Subjects with Chronic Pulmonary Sarcoidosis (RESOLVE-Lung)

We are studying a new medication, namilumab, given to treat Chronic Pulmonary Sarcoidosis. We are looking at the effectiveness, how well the drug is tolerated, and the side effects that occur. For the first part of the study, some people will receive the namilumab; the others will receive an inactive (placebo) drug. In the second part of the study, everyone may receive the namilumab.

Maneesh Bhargava
This study is NOT accepting healthy volunteers
STUDY00014721
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Inclusion Criteria:

• Diagnosis of pulmonary sarcoidosis with some respiratory symptoms
• If receiving prednisone, must be on a stable dose of 25 mg or less for 4 weeks
• If receiving immunosuppressive therapy, must agree to stop if eligible to participate in the study
• Completion of primary series of COVID-19 vaccination
Exclusion Criteria:

• Pregnancy or breast-feeding
• Smoking or using any form of inhaled tobacco or cannabis within 6 months
Rare Diseases, Respiratory System
Clinics and Surgery Center (CSC), Sarcoidosis
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A Study Evaluating the Efficacy and Safety of Adjuvant Giredestrant Compared With Physician's Choice of Adjuvant Endocrine Monotherapy in Participants With Estrogen Receptor-Positive, HER2-Negative Early Breast Cancer (lidERA Breast Cancer)

We are studying long-term endocrine therapy for people who have Stage I-III estrogen receptor (ER)-positive, HER2- early breast cancer. We are comparing the effectiveness and side effects of a drug, giredestrant, to other endocrine therapy selected by the physician.

Kiran Lassi
This study is NOT accepting healthy volunteers
MMCORC041
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Inclusion Criteria:

• estrogen receptor (ER)-positive and HER2-negative breast tumor
• definitive surgery of primary breast tumor(s) and axillary lymph nodes is complete
• if receiving adjuvant chemotherapy after surgery, it must be complete
Exclusion Criteria:

• women who are pregnant or breastfeeding
• active cardiac disease or history
• history of any other malignancy within 3 years prior to screening (except cervical, nonmelanoma skin carcinoma, or Stage I uterine cancer)
Cancer
Breast Cancer, Breast Cancer, chemotherapy, Hormone positive breast cancer
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MT2018-19: COG ANBL1531 - A Phase 3 Study of 131I-Metaiodobenzylguanidine (131I-MIBG) or ALK Inhibitor Therapy Added to Intensive Therapy for Children with Newly Diagnosed High-Risk Neuroblastoma (NBL) (IND# 134379)

This partially randomized phase III trial studies iobenguane I-131 or ALK Inhibitor Therapy and standard therapy in treating younger patients (365 days to 30 years of age) with newly-diagnosed high-risk neuroblastoma or ganglioneuroblastoma.

Emily Greengard
STUDY00003568
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Inclusion Criteria:
Patients must be enrolled on ANBL00B1 (NCT00904241) or APEC14B1 (NCT02402244) prior to enrollment on ANBL1531 (NCT03126916) Patient must be >= 365 days and =< 30 years of age at diagnosis Patients must have a diagnosis of neuroblastoma or ganglioneuroblastoma (nodular) verified by tumor pathology analysis or demonstration of clumps of tumor cells in bone marrow with elevated urinary catecholamine metabolites; the following disease groups are eligible: Patients with International Neuroblastoma Risk Group (INRG) stage M disease are eligible if found to have either of the following features: MYCN amplification (> 4-fold increase in MYCN signals as compared to reference signals), regardless of additional biologic features; OR Age > 547 days regardless of biologic features Patients with INRG stage MS disease with MYCN amplification Patients with INRG stage L2 disease with MYCN amplification Patients > 547 days of age initially diagnosed with INRG stage L1, L2 or MS disease who progressed to stage M without prior chemotherapy may enroll within 4 weeks of progression to stage M Patients >= 365 days of age initially diagnosed with MYCN amplified INRG stage L1 disease who progress to stage M without systemic therapy may enroll within 4 weeks of progression to stage M Patients initially recognized to have high-risk disease must have had no prior systemic therapy (other than topotecan/cyclophosphamide initiated on an emergent basis and within allowed timing); patients observed or treated with a single cycle of chemotherapy per a low or intermediate risk neuroblastoma regimen (e.g., as per ANBL0531, ANBL1232 or similar) for what initially appeared to be non-high risk disease but subsequently found to meet the criteria will also be eligible; patients who receive localized emergency radiation to sites of life-threatening or function-threatening disease prior to or immediately after establishment of the definitive diagnosis will be eligible Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70 mL/min/1.73 m^2 or a serum creatinine based on age/sex as follows: 1 to < 2 years: male = 0.6; female = 0.6 2 to < 6 years: male = 0.8; female = 0.8 6 to < 10 years: male = 1; female = 1 10 to < 13 years: male = 1.2; female = 1.2 13 to < 16 years: male = 1.5; female = 1.4 >= 16 years: male = 1.7; female = 1.4 Total bilirubin =< 1.5 x upper limit of normal (ULN) for age, and Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) < 10 x ULN; for the purposes of this study, ULN for SGPT (ALT) is 45 Shortening fraction of >= 27% by echocardiogram, or ejection fraction of > 50% by echocardiogram or radionuclide angiogram No known contraindication to peripheral blood stem cell (PBSC) collection; examples of contraindications might be a weight or size less than the collecting institution finds feasible, or a physical condition that would limit the ability of the child to undergo apheresis catheter placement (if necessary) and/or the apheresis procedure
Exclusion Criteria:
Patients with INRG stage L2 tumors without amplification of MYCN regardless of tumor histology (may meet criteria for high risk classification but are not eligible for this trial) Patients with bone marrow failure syndromes Patients for whom targeted radiopharmaceutical therapy would be contraindicated due to underlying medical disorders Female patients who are pregnant since fetal toxicities and teratogenic effects have been noted for several of the study drugs; a pregnancy test is required for female patients of childbearing potential Lactating females who plan to breastfeed their infants Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation
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Composur, A Patient-centric, Phase IV, Open-label, Prospective, Real World US Study to Evaluate Vibegron on Patient Treatment Satisfaction, Quality of Life, and Healthcare Resource Utilization in Patients with Overactive Bladder

This study will evaluate treatment satisfaction, discontinuation, reasons for discontinuation, quality of life, healthcare resource utilization, and safety with vibegron for the treatment of OAB in the context of real-world clinical practice.

Nissrine Nakib
STUDY00016936
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Inclusion Criteria:
Diagnosis of overactive bladder (OAB) with or without urgency urinary incontinence Symptoms of OAB for at least 3 months prior to the Baseline Visit Willing and able to complete electronic patient-reported outcomes questionnaires monthly for a minimum of 1 year Previous exposure to anticholinergics or mirabegron monotherapy and/or mirabegron plus solifenacin combination therapy prior to initiation of vibegron
Exclusion Criteria:
Any contraindication to the use of vibegron per the United States label History of OAB treatment with botulinum toxin A; sacral neuromodulation; percutaneous tibial nerve stimulation; external beam radiation therapy; urinary stents within the last 6 months; pelvic or lower urinary tract surgery within the last 6 months; and urethral catheterizations within the last 3 months prior to the Baseline Visit History of mixed incontinence where stress incontinence is the predominant form (as determined by the investigator) Participants at risk of urinary retention (as determined by the investigator) Neurologic conditions associated with OAB symptoms, e.g., multiple sclerosis Pregnant or breastfeeding or plans to do so during the study Use of vibegron prior to the Baseline Visit either prescribed or in a previous vibegron clinical trial where the participant was on vibegron Anyone who, at the discretion of the investigator, is not suitable for treatment with a beta 3 agonist for OAB for any reason
Clinics and Surgery Center (CSC)
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Phase 1/2 Study to Evaluate Palbociclib (IBRANCE?) in Combination With Irinotecan and Temozolomide or in Combination with Topotecan and Cyclophosphamide in Pediatric Patients With Recurrent or Refractory Solid Tumors Protocol No.: ADVL1921/A5481092

This is a Phase 1/2 multicenter, open-label study to evaluate palbociclib in combination with either irinotecan (IRN) and temozolomide (TMZ) or topotecan (TOPO) and cyclophosphamide (CTX) chemotherapy in children, adolescents and young adults with recurrent or refractory solid tumors. The study consists of a non- randomized Phase 1 portion for recurrent or refractory solid tumors followed by potential non- randomized tumor specific cohort(s) and a randomized, Phase 2 portion for recurrent or refractory EWS.

Emily Greengard
STUDY00007068
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Inclusion: Histologically confirmed relapsed or refractory solid tumor as follows: For dose escalation and dose determination parts: Histologically confirmed relapsed or refractory solid tumor (including CNS tumors but not lymphomas). Patients with Diffuse Intrinsic Pontine Glioma do not require histological only radiographic confirmed relapse to enroll. For dose expansion and tumor specific cohorts: Histologically confirmed relapsed or refractory solid tumor including but not limited to EWS, rhabdoid tumor, rhabdomyosarcoma, neuroblastoma, and medulloblastoma. Patients with Diffuse Intrinsic Pontine Glioma do not require histological only radiographic confirmed relapse to enroll. EWS is not eligible for TOPO and CTX tumor-specific cohorts. For randomized Phase 2 part: Histologically confirmed Ewing sarcoma at diagnosis or at relapse, with presence of EWSR1-ETS or FUS-ETS rearrangement. Histopathology confirmation of both EWSR1-ETS or FUS-ETS rearrangement partners is required OR availability of formalin fixed paraffin embedded (FFPE) tumor tissue sample for central testing. Patient must have relapsed or have refractory disease and at least evaluable disease in at least one site other than bone marrow that can be followed by imaging. Age ≥2 and <21 years at the time of study entry. Lansky performance status ≥50% for patients ≤16 years of age, or Eastern Cooperative Oncology Group (ECOG) 0, 1 or 2 for patients >16 years of age. Adequate bone marrow function. Absolute neutrophil count ≥1000/mm3; Platelet count ≥100,000/mm3 (transfusion independent, no platelet transfusion in past 7 days prior study entry); Hemoglobin ≥8.5 g/dL (transfusion allowed). Adequate renal function: Serum creatinine level based on age/gender must within protocol specified limits. Adequate liver function, including: Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤2.5 × upper limit of normal (ULN) or ≤5 × ULN for age, if attributable to disease involvement of the liver; Total bilirubin ≤1.5 × ULN for age, unless the patient has documented Gilbert's syndrome. Patients enrolled to Phase 1 portion of the study and tumor specific cohorts must have measurable disease as defined by RECIST version 1.1 or modified RANO criteria for CNS disease or INRC for neuroblastoma. Patients with EWS enrolled to Phase 2 portion of the study are eligible with evaluable disease (eg, bone only disease with no soft tissue component). Recovered to CTCAE Grade ≤1, or to baseline, from any non-hematological acute toxicities of prior surgery, chemotherapy, immunotherapy, radiotherapy, differentiation therapy or biologic therapy, with the exception of alopecia. Serum/urine pregnancy test (for all girls ≥8 years of age) negative at screening and at the baseline visit. Exclusion: Phase 1 and tumor specific cohorts: For palbociclib with IRN and TMZ combination, prior treatment with a CDK4/6 inhibitor or progression while on treatment with an IRN-containing regimen that includes TMZ. Patients who have received the combination of IRN and TMZ and did not progress while on these medications are eligible. For patients enrolling in the palbociclib with TOPO and CTX combination, prior treatment with a CDK4/6 inhibitor or progression while on treatment with a TOPO-containing regimen that includes CTX. Patients who have received the combination of TOPO and CTX and did not progress while on these medications are eligible. Phase 2 portion: prior treatment with a CDK4/6 inhibitor or progression while on treatment with an IRN-containing or TMZ-containing regimen. Patients who have received IRN and/or TMZ and did not progress while on these medications are eligible. Prior intolerability to IRN and/or TMZ plus/minus palbociclib with IRN and TMZ combination and prior intolerability to TOPO and/or CTX for TOPO and CTX combination. Use of strong cytochrome P450 (CYP) 3A inhibitors or inducers. Patients who are receiving strong uridine diphosphate-glucuronosyl transferase 1A1 (UGT1A1) inhibitors within 12 days of Cycle 1 Day 1 (C1D1) are not eligible for the palbociclib with IRN and TMZ combination. Patients who are receiving strong UGT1A1 inhibitors within 12 days of C1D1 are eligible for the palbociclib with TOPO and CTX combination (See Section 5.7.1 for list of products.) Systemic anti cancer therapy within 2 weeks prior to study entry and 6 weeks for nitrosoureas. Prior irradiation to >50% of the bone marrow (see Appendix 9). Participation in other studies involving investigational drug(s) within 2 weeks or 5 half lives, whichever is longer, prior to study entry. Major surgery within 4 weeks prior to study entry. Surgical biopsies or central line placement are not considered major surgeries. For IRN and TMZ with/without palbociclib combinations: known or suspected hypersensitivity to palbociclib, IRN and/or TMZ. For combination of palbociclib with TOPO and CTX: known or suspected hypersensitivity to palbociclib, TOPO and/or CTX. Patients with known symptomatic brain tumors or brain metastases and require steroids, unless they have been on a stable or on a decreasing steroid dose for >14 days. Patients with previously diagnosed brain metastases are eligible if they have completed their prior treatment and have recovered from the acute effects of radiation therapy or surgery prior to study entry for these metastases for at least 14 days post radiation and 4 weeks post-surgery and are neurologically stable. Hereditary bone marrow failure disorder. QTc >470 msec. History of clinically significant or uncontrolled cardiac disease, including: History of or active congestive heart failure; if patient had congestive heart failure resolve and >1 year from resolution, patient will be considered eligible; Clinically significant ventricular arrhythmia (such as ventricular tachycardia, ventricular fibrillation or Torsades de Pointes); Diagnosed or suspected congenital or acquired prolonged QT syndrome; Need for medications known to prolong the QT interval; Uncorrected hypomagnesemia or hypokalemia because of potential effects on the QT interval; Left ventricular ejection fraction <50% or shortening fraction <28%. Recent or ongoing clinically significant gastrointestinal disorder that may interfere with absorption of orally administered drugs (eg, gastrectomy). Severe acute or chronic medical or laboratory test abnormality that may increase the risk associated with study participation or investigational product administration or may interfere with the interpretation of study results, and in the judgment of the Investigator, would make the patient inappropriate for entry into this study. Investigator site staff members directly involved in the conduct of the study and their family members, site staff members otherwise supervised by the investigator, or patients who are Pfizer employees, including their family members, directly involved in the conduct of the study.
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MT2021-36: A Randomized, Open Label Phase 3 Study Evaluating Safety and Efficacy of Venetoclax in combination with Azacitidine after allogeneic Stem Cell Transplantation in Subjects with Acute Myeloid Leukemia (AML) (VIALE-T) (VIALE-T)

Part 1 Primary: To determine the recommended Phase 3 dose of venetoclax in combination with azacitidine in AML subjects when given as maintenance therapy following allogeneic SCT. Part 2 primary: 1. To determine the efficacy of venetoclax in combination with azacitidine to improve RFS in AML subjects compared to BSC when given as maintenance therapy following allogeneic SCT

Mark Juckett
STUDY00014681
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Inclusion Criteria:
Participants must be at least 18 years old for Part 1 and, at least 12 years old for Part 2. Participant must be diagnosed with Acute Myeloid Leukemia (AML) by World Health Organization (WHO) criteria (2017) and either be planning for allogeneic stem cell transplantation or have received allogeneic stem cell transplantation within the past 60 days. Blast percentage in bone marrow before transplant must be < 10%. Blast count in peripheral blood must be "0" and Blast percentage in bone marrow must be < 5% after transplant. Participant meet adequate renal, hepatic and hematologic criteria as described in the protocol. Participants >= 17 years old must have a Karnofsky Performance Scale (KPS) score > 50 and participants between 12 to 16 years old must have a Lansky Play Performance Scale score > 40.
Exclusion Criteria:
History of disease progression during prior treatment with venetoclax. History of any other malignancy within 2 years prior to study entry, except for: Adequately treated in situ carcinoma of the cervix uteri or carcinoma in situ of breast; basal cell carcinoma of the skin or localized squamous cell carcinoma of the skin; previous malignancy confined and surgically resected (or treated with other modalities) with curative intent; Myelodysplastic Syndrome, Myeloproliferative neoplasm (only allowed if it transformed to AML and AML should be the indication for marrow transplantation). Participant has known infection with HIV or history of being positive for hepatitis B virus (HBV) or hepatitis C virus (HCV) infection. Presence of clinical or laboratory symptoms/signs of extramedullary myeloid malignancy.
Clinics and Surgery Center (CSC)
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MT2020-24: A Phase 1 Dose Escalation and Cohort Expansion Study of the Safety and Efficacy of Anti-CD19 Allogeneic CRISPR-Cas9 Engineered T Cells (CTX110) in Subjects With Relapsed or Refractory B Cell Malignancies

Primary objective, Part A (dose escalation): To assess the safety of escalating doses of CTX110 in subjects with relapsed or refractory B cell malignancies to determine the recommended Part B dose Primary objective, Part B (cohort expansion): To assess the efficacy of CTX110 in subjects with relapsed or refractory B cell malignancies, as measured by objective response rate (ORR)

Joseph Maakaron
STUDY00010648
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Key
Inclusion Criteria:
For NHL patients: Age ≥18 years. For B cell ALL patients: age ≥18 years to ≤70 years Refractory or relapsed non-Hodgkin lymphoma, as evidenced by 2 or more lines of prior therapy, or histologically confirmed B cell ALL, refractory or relapsed. Eastern Cooperative Oncology Group performance status 0 or 1. Adequate renal, liver, cardiac and pulmonary organ function Female subjects of childbearing potential and male subjects must agree to use acceptable method(s) of contraception from enrollment through at least 12 months after CTX110 infusion. Key
Exclusion Criteria:
For NHL patients: prior allogeneic HSCT. For B cell ALL patients: prior allogeneic HSCT within 6 months, and/or any evidence of GvHD. History of central nervous system (CNS) involvement by malignancy History of a seizure disorder, cerebrovascular ischemia/hemorrhage, dementia, cerebellar disease, or any autoimmune disease with CNS involvement. Presence of bacterial, viral, or fungal infection that is uncontrolled. Positive for HIV, or active hepatitis B virus or hepatitis C virus infection. Previous or concurrent malignancy, except basal cell or squamous cell skin carcinoma, adequately resected and in situ carcinoma of cervix, or a previous malignancy that was completely resected and has been in remission for ≥5 years. For NHL patients: Use of systemic anti-tumor therapy or investigational agent within 14 days or 5 half-lives, whichever is longer, of CTX110 infusion. For B cell ALL patients: Use of systemic antitumor therapy within 7 days of CTX110 infusion. Primary immunodeficiency disorder or active autoimmune disease requiring steroids and/or other immunosuppressive therapy. Women who are pregnant or breastfeeding.
Clinics and Surgery Center (CSC)
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A Phase I/II, Multicenter, Open-Label, Single-Dose, Dose-Ranging Study to Assess the Safety and Tolerability of ST-920, a AAV2/6 Human Alpha Galactosidase A Gene Therapy, in Subjects with Fabry Disease

Chester Whitley, MD, PhD
This study is NOT accepting healthy volunteers
STUDY00007094
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Inclusion Criteria:

• at least 18 years of age
• diagnosis of Fabry disease
• one or more of the following symptoms: i) cornea verticillata, ii) acroparesthesia, iii) anhidrosis, iv) angiokeratoma
• fully vaccinated for COVID- 19 per CDC guidance
• additional requirements apply for cardiac and renal groups (study staff will review)
Exclusion Criteria:

• history of liver disease
• current or history of use in the last six months of systemic steroids
• other significant medical & mental health diagnosis (study staff will review)
Rare Diseases
Fabry Disease
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A Phase 1/2 Study of the Oral RET Inhibitor LOXO-292 in Pediatric Patients with Advanced RET-Altered Solid or Primary Central Nervous System Tumors; Protocol Number: LOXO-RET-18036 (J2G-OX-JZJJ) (LIBRETTO-121)

This is an open-label, multi-center, Phase 1/2 study of oral LOXO-292 in pediatric patients with an activating RET alteration and an advanced solid or primary CNS tumor.

Emily Greengard
STUDY00008874
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Inclusion Criteria:
Advanced or metastatic solid or primary CNS tumor which has failed standard of care therapies Evidence of an activating RET gene alteration in the tumor and/or blood Measurable or non-measurable disease Karnofsky (participants 16 years and older) or Lansky (participants younger than 16) performance score of at least 50 Participant with primary CNS tumors or cerebral metastases must be neurologically stable for 7 days prior and must not have required increasing doses of steroids within the last 7 days Adequate hematologic, hepatic and renal function. Ability to receive study drug therapy orally or via gastric access Willingness of men and women of reproductive potential to observe conventional and effective birth control
Exclusion Criteria:
Major surgery within two weeks prior to planned start of LOXO-292 Clinically significant, uncontrolled cardiac, cardiovascular disease or history of myocardial infarction within 6 months prior to planned start of LOXO-292 Active uncontrolled systemic bacterial, viral, fungal or parasitic infection Clinically significant active malabsorption syndrome Pregnancy or lactation Uncontrolled symptomatic hyperthyroidism or hypothyroidism (i.e. the participant required a modification to current thyroid medication in the 7 days before start of LOXO-292) Uncontrolled symptomatic hypercalcemia or hypocalcemia Known hypersensitivity to any of the components of the investigational agent, LOXO-292 or Ora-Sweet® SF and OraPlus®, for participants who will receive LOXO-292 suspension Prior treatment with a selective RET inhibitor(s) (including investigational selective RET inhibitor[s])
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A Phase II, Open Label, Two Arm Study of Therapeutic Iobenguane (131I) as Single Agent or in Combination with Vorinostat for Recurrent or Progressive High-Risk Neuroblastoma Subjects (OPTIMUM Trial) Protocol Number: MIBG 2014-01 (OPTIMUM)

This will be a Phase II, two-arm, nonrandomized, non-comparative, open-label study in participants ≥ 1 year of age with iobenguane avid, recurrent or progressive high-risk neuroblastoma. Participants not eligible for vorinostat treatment may receive 131I-MIBG as monotherapy.

Emily Greengard
STUDY00005792
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Inclusion Criteria:
Subjects with a diagnosis of iobenguane avid, high-risk neuroblastoma based on Revised INRC criteria at the time of study enrollment with recurrent or progressive disease at any time prior to enrollment, regardless of overall response to frontline therapy, where frontline therapy includes a minimum of 4 cycles of induction therapy at any time prior to enrollment. May have had prior 131I-MIBG therapy, provided: It has been at least 6 months from the date of last 131I-MIBG ; Response was other than progressive disease on first restaging after 131I-MIBG ; Prior 131I-MIBG was given as monotherapy and not in combination with systemic anticancer agents; Cumulative lifetime dose of 131I-MIBG at enrollment does not exceed 18 mCi/kg. All soft tissue lesions identified on CT/MRI scans must be iobenguane avid lesions on an (123I)-iobenguane scan, or any progressive non-iobenguane avid lesion is proven by biopsy to be a non-neuroblastoma lesion. any other non-avid lesion is comprised of a fibrotic or scarred mass as shown by routine imaging and confirmed by the investigator. Adequate cryopreserved autologous peripheral blood stem cells or bone marrow (at least 2 aliquots of 2.0 × 10exp6 CD34/kg at the time of study enrollment). If a male, must agree to use an adequate contraception method as deemed appropriate by the Investigator (e.g., vasectomy, condoms) or partner using effective contraception and to not donate sperm during the study and for 90 days after receiving the last dose of study drug. If a female of childbearing potential, have a negative serum pregnancy test result prior to each dosing and, if sexually active, be practicing an effective method of birth control [e.g., intrauterine device, double-barrier method (i.e., diaphragm, or a cervical cap) with intravaginal spermicidal foam, cream or gel], or male partner sterilization throughout the study. Age at study entry ≥1 year. Previous platelet transfusions are permitted, as long as the subject has a platelet count ≥50,000/μL without transfusion support for at least 1 week. Subjects must have a minimum pulse oximetry measurement of at least 94% at baseline. An absolute neutrophil count ≥750/μL without growth factor for 5 days. Liver function parameter results: total bilirubin ≤2 × upper limit of normal for age, and Serum alanine aminotransferase (glutamic-pyruvic transaminase) and serum aspartate aminotransferase (glutamic-oxaloacetic transaminase) ≤ 10 times the upper limit of normal (for all sites, the upper limit of normal for alanine aminotransferase is defined as 45 U/L). Normal thyroid function as measured by T4 or TSH or have abnormal results that are not considered clinically important by the Investigator or may be receiving levothyroxine. Cardiac Function: shortening fraction of ≥ 27% by echocardiogram or ejection fraction ≥ 50% documented by echocardiogram or radionuclide angiogram within 1 month prior to Visit 1 (Baseline). Karnofsky Performance Status (for subjects >16 years of age) or the Lansky Performance Status Performance Status (for subjects 1 to 16 years of age) ≥50%. Full recovery from the toxic effects of any prior therapy. Coagulation Function: International Normalized Ratio (INR) < 1.5 Partial thromboplastin time (PTT) < 1.5 times upper limit of normal.
Exclusion Criteria:
Subjects within 5 half-lives after any antibody-based immunotherapy, or have not recovered from effects of any biologic therapy. Subjects <12 weeks after myeloablative therapy with autologous stem cell transplant. Subjects who have had an allogeneic stem cell treatment less than 4 months from Visit 1 are excluded. Those who have received allogeneic stem cell treatment more than 4 months from Visit 1 must have recovered and have no active graft versus host disease (GVHD) to be eligible. Subjects must not have received radiation for a minimum of 2 weeks prior to study enrollment. Subjects whose only site(s) of disease have been radiated are eligible as long as the subject has MIBG avidity 2 weeks after completion of radiation. A minimum of 12 weeks prior to study enrollment is required following prior large field radiation therapy (ie, craniospinal, whole abdominal, total lung, > 50% marrow space) History of total body irradiation. Subjects do not have adequate renal function defined as GFR ≥ 70 mL/min/1.73 m2 either by creatinine clearance or radioisotope direct measurement or by calculation with the Schwartz formula Subjects who are on hemodialysis. Pregnancy or breastfeeding. Significant active infections including active hepatitis B, or hepatitis C infection, or known infection with human immunodeficiency virus (HIV) (testing for HIV is not required prior to study entry). Clinically important cardiac, pulmonary, and hepatic impairment. Vorinostat treatment exclusion criteria (subjects, who meet any one of these criteria and otherwise meet eligibility criteria, are still eligible for 131I-MIBG monotherapy) Since valproic acid has HDAC inhibitory activity, patients must not have received valproic acid within 30 days of study entry. Since vorinostat may prolong the QT interval, patients must not be receiving other medications known to prolong the QT interval at the time of study entry . Pentamidine must not have been received within 1 week of study enrollment. Patients with a history of deep venous thrombosis that was not associated with the presence of a central venous catheter. Patients who are receiving Coumadin.
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EA5163/S1709 INSIGNA: A Randomized, Phase III Study of Firstline Immunotherapy alone or in Combination with Chemotherapy in Induction/Maintenance or Postprogression in Advanced Nonsquamous Non Small Cell Lung Cancer (NSCLC) with Immunobiomarker SIGNature driven Analysis

We are studying the use of pembrolizumab to treat people who have stage IV non-squamous, non-small cell lung cancer. Pembrolizumab may help the body’s own immune system attack cancer so tumor cells cannot grow and spread. We are looking at when it is most effective to give the pembrolizumab and when to combine it with other anticancer drugs, pemetrexed and carboplatin.

Edward Greeno, M.D.
This study is NOT accepting healthy volunteers
MMCORC012
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Inclusion Criteria:

• confirmed stage IV non-squamous non-small cell lung cancer (NSCLC)
• PD-L1 expression Tumor Proportion Score (TPS) >= 1% in tumor cells -
Exclusion Criteria:

• Prior systemic chemotherapy or immunotherapy for advanced metastatic NSCLC
• EGFR mutations (except exon 20 insertion), BRAF mutations (V600) or ALK or ROS1 translocations
• significant gastrointestinal disorders with diarrhea as a major symptom
• history of auto-immune condition (including Guillain-Barre Syndrome or Multiple Sclerosis) requiring ongoing or intermittent systemic treatment in the past 2 years
• pregnant or breast-feeding
Cancer
Lung Cancer, Lung Cancer, Immunotherapy, Nonsquamous Non-Small Cell Lung Cancer (NSCLC)
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Pragmatica-Lung: A Prospective Randomized Study of Ramucirumab (LY3009806; NSC 749128) Plus Pembrolizumab (MK-3475; NSC 776864) Versus Standard of Care for Participants Previously Treated With Immunotherapy for Stage IV or Recurrent Non-Small Cell Lung Cancer

We are comparing the effectiveness of the combination of ramucirumab and pembrolizumab compared to the usual chemotherapy for the treatment of non-small cell lung cancer that is stage IV or that has come back after a period of improvement (recurrent). The drugs work in different ways to stop the growth of tumor cells. We will compare how well the treatment regimens work and the side effects that occur.

Nicole Hartung
This study is NOT accepting healthy volunteers
MMCORC068
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Inclusion Criteria:

• at least 18 years old
• confirmed non-small cell lung cancer (NSCLC) which is stage IV (metastatic) or has recurred
• must have received at least one previous treatment of immune therapy
• must have received platinum-based chemotherapy
• able to care for self with occasional assistance
Exclusion Criteria:

• may not have received more than one treatment with immunotherapy for stage IV or recurrent disease
• may not receive receive another investigational drug during study participation
Cancer
Immunotherapy, Lung Cancer, Lung Cancer, Non Small cell lung cancer, NSCLC, Stage IV lung cancer
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COG ACNS1931 - A Phase 3 Study of Selumetinib (NSC# 748727, IND# 77782) or Selumetinib in Combination with Vinblastine for non-NF1, non-TSC Patients with Recurrent or Progressive Low-Grade Gliomas (LGGs) Lacking BRAFV600E or IDH1 Mutations

This phase III trial investigates the best dose of vinblastine in combination with selumetinib and the benefit of adding vinblastine to selumetinib compared to selumetinib alone in treating children and young adults with low-grade glioma (a common type of brain cancer) that has come back after prior treatment (recurrent) or does not respond to therapy (progressive). Selumetinib is a drug that works by blocking a protein that lets tumor cells grow without stopping. Vinblastine blocks cell growth by stopping cell division and may kill cancer cells. Giving selumetinib in combination with vinblastine may work better than selumetinib alone in treating recurrent or progressive low-grade glioma.

Christopher Moertel, MD
This study is NOT accepting healthy volunteers
STUDY00018221
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Inclusion Criteria:

• ages 2 to 21 at time of starting the study
• diagnosis of low-grade glioma or low-grade astrocytoma brain tumor
• tumor size increased or returned after treatment with at least one cancer therapy
• high blood pressure (hypertension) must be under control
• must be able to swallow whole capsules
• contact study staff for additional criteria
Exclusion Criteria:

• treatment for another tumor in the past year
• any serious medical or mental health diagnosis, including substance use disorders or ophthalmological conditions (study staff will review)
• women who are pregnant or breast feeding
Cancer
Astrocytoma, Brain Cancer, Glioma, Low Grade Astrocytoma, Low Grade Glioma
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