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387 Study Matches

HM2017-39 Phase III Randomized Study of Crenolanib versus Midostaurin Administered Following Induction Chemotherapy and Consolidation Therapy in Newly Diagnosed Subjects with FLT3 Mutated Acute Myeloid Leukemia

This study is meant to compare the efficacy of crenolanib with midostaurin administered following induction chemotherapy and consolidation therapy on event-free survival (EFS) in newly diagnosed acute myeloid leukemia subjects with FLT3 mutation.

Mark Juckett
All
18 Years to 60 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT03258931
STUDY00002581
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Inclusion Criteria:

• Confirmed diagnosis of de novo AML according to World Health Organization (WHO) 2016 classification
• Presence of FLT3-ITD and/or D835 mutation(s) in bone marrow or peripheral blood
• Age ≥ 18 years and ≤ 60 years
• Adequate hepatic function within 48 hours prior to induction chemotherapy
• Adequate renal functions within 48 hours prior to induction chemotherapy
• ECOG performance status within 48 hours prior to induction chemotherapy ≤ 3
• Eligible for intensive cytarabine/daunorubicin (7+3) chemotherapy specified
Exclusion Criteria:

• Acute promyelocytic leukemia (APL)
• Known clinically active central nervous system (CNS) leukemia
• Severe liver disease
• Active infections
• Known, active infection with hepatitis B virus (HBV) or hepatitis C virus (HCV)
• Known infection with human immunodeficiency virus (HIV)
• Prior systemic anti-cancer treatment (e.g. chemotherapy, tyrosine kinase inhibitors, immunotherapy, or investigational agents)(except for hydroxyurea and/or leukapheresis)
Drug: Crenolanib, Drug: Midostaurin, Drug: Cytarabine, Drug: Duanorubicin
Newly Diagnosed FLT3 Mutated AML
Clinics and Surgery Center (CSC)
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A DOUBLE-BLIND, RANDOMIZED, PLACEBO-CONTROLLED STUDY TO EVALUATE THE EFFICACY AND SAFETY OF CTP-543 IN ADULT PATIENTS&WITH MODERATE TO SEVERE ALOPECIA AREATA (THRIVE-AA1)

This is a double-blind, randomized, placebo-controlled multicenter study to evaluate the efficacy and safety of CTP-543 in adult patients with moderate to severe alopecia areata. Patients will be between 18 and 65 years of age and experiencing an episode of hair loss associated with alopecia areata lasting at least 6 months and not exceeding 10 years.

Maria Hordinsky
All
18 Years to 65 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT04518995
STUDY00010630
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Inclusion Criteria:

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

• Treatment with other medications or agents within 1 month of Baseline or during the study that may affect hair regrowth or immune response.
• Active scalp inflammation, psoriasis, or seborrheic dermatitis requiring topical treatment to the scalp, significant trauma to the scalp, or other scalp condition that may interfere with the SALT assessment, or untreated actinic keratosis anywhere on the body at Screening and/or Baseline.
• Treatment with systemic immunosuppressive medications within 3 months of Screening or during the study, or biologics within 6 months of Screening or during the study.
• Females who are nursing, pregnant, or planning to become pregnant while in the study, and for 30 days after last dose of study drug.
• Clinically significant medical condition, psychiatric disease, or social condition, as determined by the Investigator, that may unfavorably alter the risk-benefit of study participation, adversely affect study compliance, or confound interpretation of study results.
Drug: CTP-543, 8 mg BID, Drug: CTP-543, 12 mg BID, Drug: Placebo, BID
Alopecia Areata
Alopecia, Hair loss, Hair disease, CTP-543, Clinics and Surgery Center (CSC)
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A Phase 1b/2, Open-Label, Multicenter, Dose Escalation And Dose Expansion Study Of Nktr-255 In Combination With Cetuximab As A Salvage Regimen For Solid Tumors

This study is a Phase 1b (Dose Escalation) / 2 (Dose Expansion), open-label, multicenter dose escalation and dose expansion study in patients with relapsed or refractory (R/R) head and neck squamous cell carcinoma (HNSCC) or colorectal carcinoma (CRC). The intervention is FDA-approved cetuximab combined with an investigational drug, NKTR-255. Patients will receive a loading dose of cetuximab alone, followed 7 days later by the first combination treatment of cetuximab and NKTR-255 on Cycle 1 Day 1. Thereafter, NKTR-255 will be given in 21-day cycles in combination with weekly IV cetuximab. After determination of the recommended Phase 2 dose (RP2D) of NKTR-255 in combination with cetuximab, this dose of NKTR-255 will be further studied in patients with HNSCC (Cohort A) and CRC (Cohort B) in Phase 2 of the study. Patients will remain on treatment until meeting one of the criteria for discontinuation.

Manish Patel
All
18 Years and over
Phase 1/Phase 2
This study is NOT accepting healthy volunteers
NCT04616196
STUDY00011716
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Key
Inclusion Criteria:

• Histologically confirmed diagnosis of a locally advanced or metastatic HNSCC, CRC, cSCC, ASCC, or cervical cancer.
• Life expectancy > 12 weeks as determined by the Investigator.
• Eastern Cooperative Oncology Group (ECOG) performance status 0 or 1.
• Measurable disease per RECIST 1.1. HNSCC:
• Progression on any first or second line platinum-based chemotherapy and/or anti-PD-1 or programmed death-ligand 1 antibody. CRC:
• Patients must have received or were intolerant to at least 2 prior cancer therapy regimens administered for metastatic disease. cSCC
• Patients must have received prior therapy including anti-PD-1 and platinum-based chemotherapy, have documented platinum-refractory disease, or be ineligible/unfit for platinum-based therapy. aSCC
• Patients must have received prior therapy including anti-PD-1 and platinum-based chemotherapy, have documented platinum-refractory disease, or be ineligible/unfit for platinum-based therapy.
• If human immunodeficiency virus (HIV)-positive, patients must also have CD4+ count ≥ 300/μL, undetectable viral load, and be receiving highly active antiretroviral therapy at the time of screening. Cervical Cancer
• Patients must have experienced progression (or toxicity precluding additional treatment) on any first- or second-line platinum-based chemotherapy and anti-PD-(L)1, have documented platinum-refractory disease, or be ineligible/unfit for platinum-based therapy.
• Patients must have known status by pathology for HPV Key
Exclusion Criteria:

• Use of an investigational agent or an investigational device within 28 days before administration of first dose of study drug(s)
• Prior surgery or radiotherapy within 14 days of initiating study drug(s)
• Evidence of clinically significant interstitial lung disease or active, noninfectious pneumonitis; active infection requiring systemic therapy within 7 days prior to dosing
• Patients who have been previously treated with IL-2 or IL-15
• Known Grade 3 or 4 hypersensitivity reaction to cetuximab, history of allergy to red meat or tick bites, or history of positive test results for immunoglobulin E antibodies against cetuximab
• Patients who have an active, known, or suspected autoimmune disease NOTE: Other protocol defined inclusion/exclusion criteria may apply
Drug: NKTR-255, Drug: Cetuximab
Head and Neck Squamous Cell Carcinoma, Colorectal Cancer, Cutaneous Squamous Cell Carcinoma, Anal Squamous Cell Carcinoma, Cervical Cancer
HNSCC, Head and Neck Squamous Cell Carcinoma, CRC, Colorectal Cancer, cSCC, Cutaneous Squamous Cell Carcinoma, ASCC, Anal Squamous Cell Carcinoma, Cervical Cancer, NKTR-255, Cetuximab, Erbitux®, Clinics and Surgery Center (CSC), Phase I Clinic
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A Randomized Phase II Study Comparing Single-Agent Olaparib, Single Agent Cediranib, and the Combination of Cediranib/Olaparib in Women with Recurrent, Persistent or Metastatic Endometrial Cancer

To compare the efficacy of single-agent olaparib and the combination of olaparib and cediranib (and potentially other combination arms that may be added by subsequent amendment) versus single agent cediranib as measured by progression free survival (PFS), in patients with recurrent, persistent or metastatic endometrial cancer.

Phase II
This study is NOT accepting healthy volunteers
NCT03660826
0123456789
Endometrial Undifferentiated Carcinoma, Endometrioid Adenocarcinoma, Recurrent Endometrial Serous Adenocarcinoma, Recurrent Uterine Corpus Cancer, Stage IV Uterine Corpus Cancer AJCC v7, Stage IVA Uterine Corpus Cancer AJCC v7, Stage IVB Uterine Corpus Cancer AJCC v7
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A Randomized Phase III Study of the addition of Venetoclax to Ibrutinib and Obinutuzumab versus Ibrutinib and Obinutuzumab in Untreated Younger Patients with Chronic Lymphocytic Leukemia (CLL)

Birendra Kumar
Phase III
This study is NOT accepting healthy volunteers
NCT03701282
0123456789
Chronic Lymphocytic Leukemia, Small Lymphocytic Lymphoma
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A Randomized, Double-Blind, Multicenter, Placebo-Controlled Phase 3 Study with Open-Label Period to Evaluate the Efficacy and Safety of Inebilizumab in Adults with Myasthenia Gravis

This is a randomized, double-blind, placebo-controlled, parallel-group study with an optional open-label extension testing the safety and efficacy of inebilizumab in participants with Myasthenia Gravis.

Georgios Manousakis
Phase III
This study is NOT accepting healthy volunteers
NCT04524273
STUDY00010270
Myasthenia Gravis, Rare Diseases
Clinics and Surgery Center (CSC), Myasthenia Gravis
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MT2021-11: An Open-label, Single-arm, Multicohort, Phase 2 Study to Assess the Efficacy and Safety of Tabelecleucel in Subjects with Epstein-Barr Virus-associated Diseases

This study is intended to determine the clinical benefit of tabelecleucel (EBV-specific cytotoxic T-lymphocytes) in subjects with EBV-associated diseases.

Joseph Maakaron
All
Not specified
Phase 2
This study is NOT accepting healthy volunteers
NCT04554914
STUDY00013494
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Inclusion Criteria:

• Diagnosis of EBV+ disorder
• Eastern Cooperative Oncology Group performance status <= 3 for participants aged >= 16 years; Lansky score >= 20 for participants from 1 year to < 16 years
• Adequate organ function test results, unless organ dysfunction is considered to be due to the underlying EBV-associated disease by the investigator Cohort-specific
Inclusion Criteria:

• For participants with PID LPD:
• Newly diagnosed or relapsed/refractory LPD confirmed by biopsy-proven EBV+ LPD or positive cerebrospinal fluid (CSF) cytology with or without radiographically measurable intracranial disease with EBV detected in CSF
• Participant must have systemic measurable disease and/ or CNS measurable disease
• Definitive therapy (eg, allogeneic HCT, gene therapy) for the underlying PID is planned
• Participants with newly diagnosed disease should be ineligible for standard first-line therapy for EBV+ LPD, as determined by the investigator
• For participants with AID LPD:
• Newly diagnosed or relapsed/refractory LPD confirmed by biopsy-proven EBV+ LPD or positive CSF cytology, with or without radiographically measurable intracranial disease, with EBV detected in CSF
• Participant must have systemic measurable disease and/ or CNS measurable disease
• Participants who are human immunodeficiency virus positive (HIV+) must meet both of the following criteria: Have an HIV viral load assessed by reverse transcription-polymerase chain reaction (RT-PCR) below the lower limit of detection and CD4 >= 50 cells/μL within 6 months prior to the first dose of tabelecleucel
• Participants with newly diagnosed disease should be ineligible for standard first-line therapy for EBV+ LPD, as determined by the investigator
• For participants with CNS PTLD:
• Newly diagnosed or relapsed/refractory EBV+ CNS PTLD histologically confirmed by biopsy-proven EBV+ CNS PTLD or positive CSF cytology with or without radiographically measurable intracranial disease with EBV detected in CSF
• Participant may have systemic and CNS disease or CNS disease only
• Participants with newly diagnosed disease should be ineligible for standard first-line therapy for EBV+ LPD, as determined by the investigator
• For participants with EBV+ PTLD, where standard first line therapy (rituximab and/or chemotherapy) is not appropriate, including CD20-negative disease:
• Newly diagnosed, biopsy-proven EBV+ PTLD
• Ineligible for standard first-line therapy for EBV+ PTLD, as determined by the investigator
• Participants must have systemic disease measurable per Lugano Classification criteria, except when contraindicated or mandated by local practice, then MRI may be used.
• For participants with sarcoma, including LMS:
• Newly diagnosed or failed systemic first-line therapy for EBV+ sarcoma. Participants with newly diagnosed disease should be ineligible for standard first-line therapy for EBV+ sarcoma, as determined by the investigator.
• Biopsy-proven EBV+ sarcoma
• Measurable disease using diagnostic PET/CT and/or MRI following RECIST 1.1 criteria
• For participants with CAEBV:
• Newly diagnosed or previously treated CAEBV
• Detectable EBV viremia on at least 2 occasions at a minimum of 90 days apart
• At least 3 active clinical findings (per Kimura H, et al. Front Immunol. 2017;8:1867) as: Fever >= 38.5°C; splenomegaly, lymphadenopathy, and/or hepatomegaly; cytopenia affecting at least 2 or 3 lineages in the peripheral blood (hemoglobin < 9 g/dL, platelets < 100 × 10^3/mL, neutrophils < 1 × 10^3/mL); hypogammaglobulinemia; hemophagocytosis; hepatitis; neuropathy; rash; and hydroa vacciniforme
• For participants with EBV+ viremia with HLH:
• Newly diagnosed or previously treated EBV+ viremia with HLH
• A molecular diagnosis consistent with HLH-2004 trial (per Henter JI, et al. Pediatr Blood Cancer. 2007;48:124-31) OR 5 or more of the clinical symptoms (per Jordan MB, et al. Blood. 2011;118:4041-4052): Fever >= 38.5°C; splenomegaly; cytopenia affecting at least 2 or 3 lineages in the peripheral blood (hemoglobin < 9 g/dL, platelets < 100 × 10^3/mL, neutrophils < 1 × 10^3/mL); hypertriglyceridemia (fasting >= 265 mg/dL) and/or hypofibrinogenemia (<= 150 mg/dL); hemophagocytosis in bone marrow, spleen, lymph nodes, or liver; low or absent natural killer cell (NK-cell) activity; ferritin >= 500 ng/mL; and elevated soluble CD25
Exclusion Criteria:

• Burkitt, T-cell (except in the setting of HLH), natural killer/T-cell lymphoma/LPD, Hodgkin, or transformed lymphoma
• Serious known active infections, defined as ongoing uncontrolled adenovirus infection or infections requiring systemic therapy at the time of enrollment
• Suspected or confirmed Grade >= 2 acute graft-versus-host disease (GvHD) per the Center for International Blood and Marrow Transplant Research (CIBMTR) consensus grading system or extensive chronic GvHD per National Institutes of Health (NIH) consensus criteria at the time of the enrollment
• Need for vasopressor or ventilatory support
• Prior therapy (in order of increasing washout period) prior to enrollment as:
• Within 4 weeks or 5 half-lives (whichever is shorter) for any investigational product and/ or any chemotherapy (systemic or intrathecal), targeted small molecule therapy, or antibody/biologic therapy. Note: prior anti-CD20 antibody use is permitted within the washout period if a subsequent disease response assessment indicates disease progression
• Within <= 8 weeks for cellular therapies (EBV-CTLs, chimeric antigen receptor therapies directed at T cells or T-cell subsets, donor lymphocyte infusion, other CTLs); and/or therapies which could impact tabelecleucel function (anti-thymocyte globulin, alemtuzumab)
• Unwilling to use protocol specified contraceptive methods
• Women who are pregnant or breastfeeding
• Ongoing need for daily steroids of > 0.5 mg/kg prednisone or glucocorticoid equivalent, ongoing methotrexate, or extracorporeal photopheresis (protocol-specified dexamethasone is permitted and concludes by the time of enrollment)
• For participants with PID LPD or AID LPD: history of prior allogeneic HCT or solid organ transplant
Biological: Tabelecleucel
Epstein-Barr Virus (EBV)-Associated Diseases, EBV+ Lymphoproliferative Disease With Primary Immunodeficiency (PID LPD), EBV+ Lymphoproliferative Disease With Acquired (Non-congenital) Immunodeficiency (AID LPD), EBV+ Posttransplant Lymphoproliferative Disease in Central Nervous System (CNS PTLD), EBV+ Post-transplant Lymphoproliferative Disease (EBV+ PTLD), Solid Organ Transplant Complications, Lymphoproliferative Disorders, Allogeneic Hematopoietic Cell Transplant, Stem Cell Transplant Complications, EBV+ Sarcomas, Leiomyosarcoma, Chronic Active Epstein-Barr Virus (CAEBV), Chronic Active Epstein-Barr Virus With Hemophagocytic Lymphohistiocytosis (HLH), Lymphohistiocytosis, Hemophagocytic
Allogeneic, Off-The-Shelf T-cell Immunotherapy, Epstein-Barr Virus (EBV), Epstein-Barr Virus-specific Cytotoxic T lymphocyte (EBV-CTL), Solid Organ Transplant (SOT), Hematopoietic Cell Transplant (HCT), Clinics and Surgery Center (CSC)
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A Phase I Study of HCW9218, a Bifunctional TGF-B; Antagonist/IL-15 Protein Complex, in Select Advanced Solid Tumors After Failing at Least Two Prior Therapies

This is a single center, Phase I dose finding study of HCW9218 for the treatment of select advanced solid tumor cancers, including, but not limited to breast, ovarian, prostate and colorectal. HCW9218 potently activates natural killer (NK) cells and CD8+ T cells in vitro and in vivo to promote their proliferative and metabolic activities and enhance their cytotoxicity against tumor targets. The fusion complex also exhibits TGF-β neutralizing activity in vitro and sequesters plasma TGF-β in vivo. It is hypothesized that HCW9218 may serve as a novel therapeutic to simultaneously provide immunostimulation and lessen immunosuppression associated with solid tumors. The primary objective of this study is to determine the maximum tolerated dose (MTD) of HCW9218 as monotherapy in advanced solid tumor cancers except pancreatic cancer and primary brain tumors.

Melissa Geller, MD
All
18 Years and over
Phase 1
This study is NOT accepting healthy volunteers
NCT05322408
STUDY00015102
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Inclusion Criteria:

• Histologically or cytologically confirmed advanced/metastatic solid tumor cancer (except pancreatic and primary brain cancers), has failed at least 2 prior lines of therapy given either in the recurrent or metastatic setting and must be refractory to or intolerant of existing therapy(ies) known to provide clinical benefit for their condition.
• Measurable disease per RECIST v 1.1.
• Acute effects of any prior therapy must have resolved to baseline or Grade ≤1 NCI CTCAE v5 except for AEs not constituting a safety risk by enrolling Investigator judgment.
• Age 18 years or older at the time of consent.
• ECOG Performance Status 0 or 1.
• Evidence of adequate organ function within 14 days prior to enrollment as defined in Section 4.1.6.
• Adequate pulmonary function with PFTs >50% FEV1 if symptomatic or known impairment.
• Sexually active persons of child-bearing potential or with partners of childbearing potential must agree to use a highly effective form of contraception (refer to Section
• 1.10 for acceptable methods) for at least 28 days after the last dose of HCW9218.
• Provides voluntary written consent prior to the performance of any research related activity.
Exclusion Criteria:

• Pregnant or breastfeeding.
• History of clinically significant vascular disease, including any of the following within 6 months prior to start of study treatment: MI or unstable angina, percutaneous coronary intervention, bypass grafting, ventricular arrhythmia requiring medication, stroke or transient ischemic attack, symptomatic peripheral arterial disease.
• Marked baseline prolongation of QT/QTc interval (e.g., demonstration of a QTc interval greater or equal to 470 milliseconds by Fridericia's correction).
• Known or suspected untreated CNS metastases.
• Anti-cancer treatment including surgery, radiotherapy, chemotherapy, other immunotherapy, or investigational therapy within 14 days before treatment start.
• Other prior malignancy except for the following: adequately treated basal cell or squamous cell skin cancer, in situ cervical cancer, adequately treated Stage I or II cancer from which the subject is currently in complete remission, or any other cancer from which the subject has been disease-free for 3 years after surgical treatment.
• Known hypersensitivity or history of allergic reactions attributed to compounds of similar chemical or biologic composition to the agents used in the study.
• Prior therapy with TGF-β antagonist, IL-15 or analogs.
• Concurrent use of St. John's wort and and/or other herbal CYP modulators within 7 days of Day 1. Must agree to not use during study treatment through the end of treatment visit to be eligible.
• Known autoimmune disease requiring active treatment. Persons with a condition requiring systemic treatment with either corticosteroids (> 10 mg daily prednisone equivalent) or other immunosuppressive medications within 14 days of enrollment. Inhaled or topical steroids, and adrenal replacement steroid doses ≤ 10 mg daily prednisone equivalent, are permitted in the absence of active autoimmune disease.
• Active systemic infection requiring parenteral antibiotic therapy. All prior infections must have resolved following optimal therapy.
• Prior organ allograft or allogeneic transplantation.
• Known HIV-positive or AIDS.
• Psychiatric illness/social situations that would limit compliance with study requirements.
• Other illness or a medical issue that in the opinion of the Investigator would exclude the subject from participating in this study
Drug: HCW9218
Solid Tumor
Clinics and Surgery Center (CSC), Phase I Clinic
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Phase 1/2, dose-escalation study to evaluate the safety, tolerability and efficacy of a single intravenous infusion of SPK-3006 in adults with late-onset Pompe disease

This is a prospective, multinational, multicenter, open-label, non-randomized, first-in human Phase 1/2a, dose-escalation study to evaluate the safety, tolerability, and exploratory efficacy of a single intravenous infusion of SPK-3006 in adults with clinically moderate, late-onset Pompe disease. Participants will be treated in sequential, dose-level cohorts. The number of participants in each cohort will be determined by levels of circulating GAA, safety, and immunogenicity evaluations. Data from participants treated early in the study will enable potential adaptation of the dosing regimen for an optional additional cohort(s) or for an expanded cohort (or cohorts) at selected dose levels.

Chester Whitley, MD, PhD
All
18 Years and over
Phase 1/Phase 2
This study is NOT accepting healthy volunteers
NCT04093349
STUDY00008038
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Inclusion Criteria:

• Provide written informed consent;
• Males and Females ≥18 years of age with late-onset Pompe disease;
• Received ERT for at least the previous 24 months
• Have clinically moderate, late-onset Pompe disease characteristics;
• Agree to use reliable contraception.
Exclusion Criteria:

• Active hepatitis B and/or C;
• Significant underlying liver disease;
• Human immunodeficiency virus (HIV) infection;
• Prior hypersensitivity to rhGAA;
• Pre-existing anti-AAV neutralizing antibody titers;
• High titer antibody responses to rhGAA;
• Requires any invasive ventilation or requires noninvasive ventilation while awake and upright;
• Received any prior vector or gene transfer agent;
• Active malignancy (except non-melanoma skin cancer);
• History of liver cancer;
• Pregnant or nursing women;
• Any evidence of active infection at the time of SPK-3006 infusion.
Genetic: SPK-3006
Pompe Disease, Pompe Disease (Late-onset), Glycogen Storage Disease Type 2, Glycogen Storage Disease Type II, LOPD, Lysosomal Storage Diseases, Acid Maltase Deficiency
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A LONG-TERM, OPEN-LABEL STUDY TO EVALUATE THE SAFETY, PHARMACODYNAMICS, AND EFFICACY OF MIGALASTAT IN SUBJECTS > 12 YEARS OF AGE WITH FABRY DISEASE AND AMENABLE GLA VARIANTS

This an extension study assessing the use of migalastat (AT1001) in pediatric populations. AT1001, under the trade name Galafold, is approved for use in the US in adults, but not children. The parent study is approved by the IRB under STUDY00006216.

Chester Whitley, MD, PhD
All
12 Years to 17 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT04049760
STUDY00009760
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Inclusion Criteria:

• Male or female subjects diagnosed with Fabry disease > 12 years of age who completed Study AT1001-020
• Subject's parent or legally-authorized representative is willing and able to provide written informed consent and authorization for use and disclosure of personal health information or research-related health information, and subject provides assent, if applicable
• Subject's parent or legally-authorized representative is willing and able to provide written informed consent and authorization for use and disclosure of personal health information or research-related health information, and subject provides assent, if applicable
Exclusion Criteria:

• Has moderate or severe renal impairment (eGFR <60 ml/min/1.73 m2 at screening)
• Has advanced kidney disease requiring dialysis or kidney transplantation
• History of allergy or sensitivity to study medication (including excipients) or other iminosugars (eg, miglustat, miglitol)
• Has received any gene therapy at any time or anticipates starting gene therapy during the study period
• Requires treatment with Glyset (miglitol), Zavesca (miglustat) within 6 months before screening or throughout the study
• Requires treatment with Replagal (agalsidase alfa), or Fabrazyme (agalsidase beta) within 14 days before screening or throughout the study
• Subject is treated or has been treated with any investigational/experimental drug, biologic or device within 30 days before screening
• Any intercurrent illness or condition or concomitant medication use considered to be a contraindication at screening or baseline or that may preclude the subject from fulfilling the protocol requirements or suggests to the investigator that the potential subject may have an unacceptable risk by participating in this study
• Pregnant or breast-feeding
• Otherwise unsuitable for the study in the opinion of the investigator
Drug: migalastat HCl 150 mg
Fabry Disease
Lysosomal storage disease, migalastat
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OMS721-IGA-001: A Randomized, Double-blind, Placebo-controlled, Phase 3 Study of the Safety and Efficacy of OMS721 in Patients with Immunoglobulin A (IgA) Nephropathy (ARTEMIS - IGAN)

This is a Phase 3, double-blind, randomized, placebo-controlled, study in patients aged 18 years and above with a biopsy-confirmed diagnosis of IgAN and with 24-hour UPE that is > 1 g/day at baseline. During the study, all patients will continue optimized renin-angiotensin system (RAS) blockade. The study consists of five periods: Screening, Run-In, Initial Treatment (Weeks 1-12), Response Evaluation (Weeks 13-24), and Follow-Up (Weeks 25 to end-of-study). The study duration for each patient is expected to last up to 160 weeks.

Patrick Nachman
All
18 Years and over
Phase 3
This study is NOT accepting healthy volunteers
NCT03608033
STUDY00002971
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Inclusion Criteria:

• Age 18 years or older at the onset of Screening
• Biopsy confirmed diagnosis of IgAN within 8 years prior to Screening
• Proteinuria of > 1 g/day within 6 months prior to Screening or uPCR > 0.75 by spot urine at Screening
• Mean of two proteinuria measurements > 1 g/day at baseline
• Estimated glomerular filtration rate of ≥ 30 mL/min/1.73 m2 at Screening and baseline
Exclusion Criteria:

• Treatment with immunosuppressants (e.g., azathioprine or cyclophosphamide), or cytotoxic drugs, for IgA within 8 weeks prior to Screening. Treatment with immunosuppressants or cytotoxic drugs for IgAN is not allowed during the Run-In Period. Treatment with immunosuppressants are allowed if such treatment is for indications other than IgAN.
• Treatment with eculizumab within 8 weeks prior to Screening. Treatment with eculizumab is not allowed during the Run-In Period.
• Treatment with systemic corticosteroids within 8 weeks prior to Screening. Treatment with systemic corticosteroids is not allowed during the Run-In Period.
• Uncontrolled BP, a systolic BP of > 150 mmHg and a diastolic BP of > 100 mmHg at rest despite the combination of two or more anti-hypertensives including ACEIs, ARBs, or direct renin inhibitors at Screening and baseline
• Female patients who are pregnant, breast feeding, or planning to become pregnant up through 12 weeks after the last dose of study drug, including possible retreatments
• Clinical or biological evidence of Type 1 diabetes mellitus (DM), or poorly controlled DM with hemoglobin A1c > 7.5 or with evidence of diabetic nephropathy on biopsy, systemic lupus erythematosus, IgA vasculitis (Henoch-Schonlein purpura), secondary IgAN, or other renal disease during Screening and Run-In
• History of renal transplantation
• Have a known hypersensitivity to any constituent of the investigational product
• Rapidly progressive glomerulonephritis
• Significant abnormalities in clinical laboratory values
• History of human immunodeficiency virus (HIV), evidence of immune suppression, active HCV infection (patients with positive anti-HCV antibody but a non-detected HCV RNA PCR can enroll), HBV infection (patients with positive HBsAg are excluded. For patients with isolated positive anti-HBc antibody, HBV DNA test by PCR must be non-detectable to enroll).
• Diagnosis of a malignancy except for adequately treated and cured basal or squamous cell skin cancer, curatively treated in situ disease, or other cancer from which the patient has been disease-free for ≥ 5 years
• Have received any other investigational drug or device or experimental procedures within 30 days of the Screening Visit (SV)
Biological: OMS721, Other: Vehicle (D5W or saline)
IgA Nephropathy
Clinics and Surgery Center (CSC)
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HM2020-10 A Randomized, Double-blind, Multicenter Study Comparing Magrolimab in Combination with Azacitidine versus Azacitidine Plus Placebo in Treatment-naive Patients with Higher Risk Myelodysplastic Syndrome (ENHANCE)

This study seeks to compare the effects of magrolimab in combination with azacitidine, to those of azacitidine in combination with placebo, to find out which is better for treating patients with MDS. Other purposes of this study include determining the quantity of magrolimab in the blood, aspects of patients' disease management (e.g. if a patient can have less frequent blood transfusions), quality of life and the side effects that magrolimab has on the body.

Joseph Maakaron
All
18 Years and over
Phase 3
This study is NOT accepting healthy volunteers
NCT04313881
STUDY00010290
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Key
Inclusion Criteria:

• Participants with Myelodysplastic Syndrome (MDS) defined according to World Health Organization classification, with Revised International Prognostic Scoring System (IPSS-R) prognostic risk category of intermediate, high, or very high risk.
• Adequate performance status and hematological, liver, and kidney function Key
Exclusion Criteria:

• Immediate eligibility for allogenic stem cell transplant (SCT), as determined by the investigator, with an available donor
• Prior treatment with Cluster of Differentiation (CD) 47 or Signal-regulatory protein alpha (SIRPα)-targeting agents
• Any prior antileukemic therapy for treatment of intermediate, high, very high risk MDS per IPSS-R
• Second malignancy, except treated basal cell or localized squamous skin carcinomas, localized prostate cancer, or other malignancies for which participants are not on active anticancer therapies and have had no evidence of active malignancy for at least ≥ 1 year
• Contraindications to azacitidine
• Clinical suspicion of active central nervous system (CNS) involvement by MDS
• Known active or chronic hepatitis B or C infection or human immunodeficiency virus in medical history
• Active hepatitis B virus and/or active hepatitis C virus, and/or HIV following testing at screening
• Pregnancy or active breastfeeding Note: Other protocol defined Inclusion/Exclusion criteria may apply.
Drug: Magrolimab, Drug: Azacitidine, Drug: Placebo
Myelodysplastic Syndromes
Clinics and Surgery Center (CSC)
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A RANDOMIZED PHASE II/III OPEN-LABEL STUDY OF IPILIMUMAB AND NIVOLUMAB VERSUS TEMOZOLOMIDE IN PATIENTS WITH NEWLY DIAGNOSED MGMT (TUMOR O-6-METHYLGUANINE DNA METHYLTRANSFERASE) UNMETHYLATED GLIOBLASTOMA

Elizabeth Neil
Phase II/III
This study is NOT accepting healthy volunteers
NCT04396860
0123456789
Gliosarcoma, MGMT-Unmethylated Glioblastoma
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Recombinant tPA by Endovascular Administration for the treatment of Submassive pulmonary embolism using pharmaco-mechanical Catheter directed thrombolysis for the redUction of thrombus burdEn - The RESCUE Study (RESCUE)

RESCUE is a prospective, non-randomized, multicenter study of the Bashir™ Endovascular Catheter (BEC), a device intended for the localized infusion of therapeutic agents into the pulmonary artery and peripheral vasculature. The study objective is to demonstrate the efficacy and safety of the Bashir™ Endovascular Catheter for the administration of pharmaco-mechanical catheter directed therapy using low dose r-tPA for the treatment of acute submassive pulmonary embolism.

Michael Rosenberg
All
18 Years to 75 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT04248868
STUDY00010186
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Inclusion Criteria:

• Willing and able to provide informed consent;
• Age 18 to ≤ 75 years of age;
• PE symptom duration ≤ 14 days.
• Filling defect in at least one main or lobar pulmonary artery as determined by contrast enhanced chest CT (CTA);
• RV/LV diameter ratio ≥ 0.9 by CTA as determined by the investigative site;
• Willing and able to comply with all study procedures and follow-up.
Exclusion Criteria:

• CVA or TIA within one (1) year;
• Head trauma, active intracranial, or intraspinal disease ≤ one (1) year prior to inclusion in the study;
• Active bleeding from a major organ within one (1) month prior to inclusion in the study;
• Intracranial condition(s) that may increase the risk of bleeding (e.g., neoplasms, arteriovenous malformations, or aneurysms);
• Patients with bleeding diatheses;
• Hematocrit < 30%;
• Platelets < 100,000/μL;
• INR > 1.5 if currently on warfarin (Coumadin®);
• aPTT > 50 seconds in the absence of anticoagulants;
• Major surgery ≤ 14 days prior to inclusion in the study;
• Serum creatinine > 2.0mg/dL;
• Clinician deems high-risk for catastrophic bleeding;
• History of heparin-induced thrombocytopenia (HIT Syndrome);
• Pregnancy;
• SBP < 90 mmHg > 15 minutes within two (2) hours prior to BEC procedure and is not resolved with IV fluids;
• Any vasopressor support;
• Cardiac arrest (including pulseless electrical activity and asystole) requiring active cardiopulmonary resuscitation (CPR) during this hospitalization at treating institution and/or referring institution;
• Evidence of irreversible neurological compromise;
• Life expectancy < one (1) year;
• Use of thrombolytics or glycoprotein IIb/IIIa inhibitor within 3 days prior to inclusion in the study;
• Use of non-vitamin K oral anti-coagulants (NOACs), such as rivaroxaban (Xarelto®), apixaban (Eliquis®), dabigatran (Pradaxa®), edoxaban (Savaysa®) within 48 hours prior to inclusion in the study;
• Profound bradycardia requiring a temporary pacemaker and/or inotropic support;
• Previous enrollment in this study;
• Morbidly obese patient who by the judgement of the investigator is high risk for bleeding;
• BMI > 45kg/m2;
• Absolute contraindication to anticoagulation;
• Uncontrolled hypertension defined as SBP > 175mmHg and / or DBP > 110mmHg with pharmacotherapy within two (2) hours prior to inclusion in the study;
• Currently participating in another study;
• Any arterial line placement;
• Current positive COVID diagnosis, or ≤ 8 weeks negative of COVID, or > 8 weeks from positive COVID test and with current symptoms, or current active viral pneumonia on chest CT scan;
• In the opinion of the investigator, the subject is not a suitable candidate for the study.
Device: The Bashir™ Endovascular Catheter, Drug: r-tPA
Pulmonary Embolism
Pulmonary Embolism, Catheter Directed Therapy
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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 3 years who are undergoing cataract surgery.

Raymond Areaux
Phase III
This study is NOT accepting healthy volunteers
NCT04539548
STUDY00010045
Cataract
Dextenza, dexamethasone ophthalmic insert
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A Phase I, Multi-Center, Open Label, Single Ascending Dose Study to Evaluate the Safety, Tolerability, and Pharmakokinetics of CSL889 in Adult Patients with Stable Sickle Cell Disease

This study is being done to measure levels of CSL889 in the blood and see how well it is tolerated. The study will also look for changes in several blood tests related to sickle cell disease to see how the study drug might affect these measures.

Alexander Boucher
All
18 Years to 60 Years old
Phase 1
This study is NOT accepting healthy volunteers
NCT04285827
STUDY00012191
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Inclusion Criteria:

• Diagnosis of SCD characterized by HbSS or SCD characterized by the compound heterozygous state of the βS mutation with β0 thalassemia mutations (HbSβ0)
• Aged 18 to 60 years, inclusive
• Stable SCD for at least 30 days before CSL889 infusion (Part A) or subject hospitalized for uncomplicated VOC (Part B)
• Subject is either not taking hydroxyurea and / or L-glutamine, or subject has been taking hydroxyurea and / or L-glutamine for at least 30 days before Day 1 on a stable, well tolerated regimen that is planned to continue without change throughout the study
Exclusion Criteria:

• History of primary hemorrhagic stroke
• History or evidence of inherited bleeding diathesis or significant coagulopathy at risk for bleeding
• Weight >110 kg (242 lbs)
• Surgery within 30 days before Day 1 or any preplanned surgeries during the study (minor surgeries may be permitted under local anesthesia before screening, with permission of the medical monitor)
• Female subjects who are pregnant or breastfeeding
• Female subject of childbearing potential or fertile male subject either not using or not willing to use an acceptable method of contraception to avoid pregnancy during the study and for 30 days after receipt of CSL889.
• Treatment with any other drug / biologic that is newly approved for SCD during the conduct of this study within 90 days before Day 1.
• Treatment with another investigational product within 30 days or within 5 half-lives of the product (whichever is greater) before Day 1
• Vaccination within 30 days before Day 1, or planned vaccination during the study
• Body-mass index < 16 kg/m2 or weight < 50 kg (110 lbs)
• History of anaphylactic-type reactions, transfusion related reaction, asthma, or autoimmune disease
Biological: CSL889
Sickle Cell Disease
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Open-Label Study to Evaluate the Efficacy and Safety of SCY-078 in Patients with Fungal Diseases that are Refractory to or Intolerant of Standard Antifungal Treatment (FURI) (FURI)

The purpose of this study is to see how well the experimental drug, SCY-078, works at treating people with fungal diseases that are resistant to, or unable to be treated due to bad side effects of, the Standard Antifungal Treatment that is currently used by doctors. This study will compare the effects of SCY-078 to Standard Antifungal Treatment.

Jo-Anne Young, MD
All
18 Years and over
Phase 3
This study is NOT accepting healthy volunteers
NCT03059992
STUDY00000611
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Key
Inclusion Criteria:

• Must have a documented eligible invasive and/or severe fungal disease that is refractory or intolerant to Standard-of-Care treatment
• Be able to tolerate medication orally or through a nasogastric (NG) tube or percutaneous endoscopic gastrostomy (PEG) tube
• Be able to understand and sign a written informed consent form (ICF), which must be obtained prior to treatment and any study-related procedures.
• Be able to understand and sign a consent or authorization form which shall permit the use, disclosure and transfer of the subject's personal health information. (e.g., in the U.S. HIPAA Authorization form).
• Be able to understand and follow all study-related procedures including study drug administration.
• Agree to use a medically acceptable method of contraception while receiving protocol-assigned product. Key
Exclusion Criteria:

• An invasive fungal disease with CNS involvement.
• Subject has an inappropriately controlled fungal disease source (e.g., persistent catheters that cannot be removed and are likely the source of infection).
• Subject is hemodynamically unstable, requiring vasopressor medication for blood pressure support.
• A life expectancy < 30 days.
• Subject with abnormal liver test parameters: aspartate aminotransferase (AST), alanine aminotransferase (ALT) >10 x the upper limit of normal (ULN), and/or total bilirubin > 5 x ULN.
• Subject is pregnant or lactating.
• Subject has used an investigational drug within 30 days prior to the baseline visit.
Drug: Ibrexafungerp
Invasive Candidiasis, Mucocutaneous Candidiasis, Coccidioidomycosis, Histoplasmosis, Blastomycosis, Chronic Pulmonary Aspergillosis, Allergic Bronchopulmonary Aspergillosis, Invasive Pulmonary Aspergillosis, Recurrent Vulvovaginal Candidiasis, Other Emerging Fungi
Clinics and Surgery Center (CSC)
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A Randomized, Open Label, Multi-Center, Phase 3 Efficacy Study of Sub-Q Abatacept in&#13;&#10;Preventing Extension of Oligoarticular Juvenile Idiopathic Arthritis JIA (Limit-JIA) (Limit-JIA)

To evaluate the effectiveness of a 24-week course of treatment with a T-cell co-stimulation inhibitor (abatacept (Orencia)) plus usual care versus usual care to prevent polyarthritis (≥5 joints), uveitis, or treatment with other systemic medication (s) for JIA (e.g one or more of the following: glucocorticoids, DMARD or biologic) medication within 18 months of randomization in children with recent-onset limited JIA

Colleen Correll
All
2 Years to 16 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT03841357
STUDY00007938
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To be eligible for this trial, participants must meet all of the following criteria in order to be include in the study:
• Age ≥ 2 years old and ≤16.5 years old
• Clinical diagnosis of JIA by a pediatric rheumatologist within the past 6 months
• Arthritis affecting ≤4 joints between disease onset and randomization
• Enrollment in the CARRA Registry
• Participants of childbearing potential must agree to remain abstinent or agree to use an effective and medically acceptable form of birth control from the time of written or verbal assent to at least 66 days after taking the last dose of study drug.
• Weight ≥50 kg (Canadian Sites only) ¹ Enrollment is defined as having signed consent to participate in the Limit-JIA study. The presence of any of the following will exclude a study participant from inclusion in the study:
• 1. Systemic JIA as defined by 2004 ILAR criteria1
• Sacroiliitis (clinical or radiographic)
• Inflammatory bowel disease (IBD)
• History of psoriasis or currently active psoriasis
• History of uveitis or currently active uveitis
• Prior treatment with systemic medication(s) for JIA (e.g. one or more of the following: DMARD or biologic medication)
• Current or previous (within 30 days of enrollment) treatment with systemic glucocorticoids (A short course of oral prednisone [≤ 14 days] is allowed)
• History of active or chronic liver disease
• Chronic or acute renal disorder
• AST (SGOT), ALT (SGPT) or BUN >2 x ULN (upper limit of normal) or creatinine >1.5 mg/dL or any other laboratory abnormality considered by the examining physician to be clinically significant within 2 months of the randomization visit
• Presence of any medical or psychological condition or laboratory result which would make the participant, in the opinion of the investigator, unsuitable for the study
• Participation in another concurrent clinical interventional study within 30 days of randomization
• Known positive human immunodeficiency virus (HIV)
• Received a live virus vaccine within 1 month of the baseline visit
• Current or prior positive Purified Protein Derivative (PPD) test or Quantiferon Gold TB
• Pregnant, breast feeding, or planned breast feeding during the study duration
• Planned transfer to non-participating pediatric rheumatology center or adult rheumatologist in the next 12 months
• Active malignancy of any type or history of malignancy
• Chronic or active infection or any major episode of infection requiring hospitalization or treatment with intravenous (IV) antibiotics within 30 days or oral antibiotics within 14 days prior to screening
• Primary language other than English or Spanish
• Positive for Hepatitis B surface antigen or core antibody
• <10 Kg in weight
• If a potential subject has symptoms consistent with COVID-19 and/or known COVID-19 exposure at screening, it is recommended that the site follow CDC guidance regarding testing and quarantine requirements. The subject can be re-screened when there is no longer concern for active infection. A subject with a positive COVID -19 test may be re-screened.
Drug: Abatacept Injection, Other: Usual Care
Juvenile Idiopathic Arthritis
Polyarthritis, abatacept, uveitis, prevention
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A Phase IIa, Double-Blind, Randomized, Intracohort Placebo-Controlled, Multicenter Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of EHP-101 in Patients with Diffuse Cutaneous Systemic Sclerosis

The primary objective of the study is to evaluate the safety and tolerability of selected doses of EHP-101 in patients with diffuse cutaneous systemic sclerosis (dcSSc) administered for up to 84 days (12 weeks).The secondary objectives of the study are to evaluate the:Treatment effect of selected doses of EHP-101 as measured by the Composite Response Index in dcSSc (CRISS) as well as all individual components of the CRISS following treatment of 84 days (12 weeks).

Jerry Molitor
All
18 Years to 74 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT04166552
STUDY00010677
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Inclusion Criteria:

• Patients male and female ≥18 years and ≤74 years at the time of consent;
• American College of Rheumatology/ European League Against Rheumatism 2013 Criteria for SSc; dcSSc (skin thickening on upper arms, upper legs, or trunk);
• Documented SSc for up to 6 years from the first non-Raynaud's phenomenon with a total mRSS of ≥15;
• No new or increased doses of immunosuppressants medications within 3 months prior to Screening;
• Effective method of contraception for participants and their partners.
Exclusion Criteria:

• Severe or unstable Systemic Sclerosis (SSc) or SSc with end-stage organ failure;
• Patient with FVC <60%;
• History of clinically significant medical condition or concurrent medical therapies that would exclude the patient, preclude participation in the clinical trial, influence response to study product, or interfere with study assessments;
• History of gastrointestinal dysmotility requiring total parenteral nutrition or hospitalization within 6 months before Visit 1;
• Any one of the following values for laboratory tests at screening:
• Haemoglobin <9 g/dL;
• Neutrophils <1.0 x 10^9/L;
• Platelets <75 x 10^9/L;
• Estimated creatinine clearance <50 mL/min according to the Cockcroft-Gault equation;
• Serum transaminases >2.0 x upper normal limit;
• Total bilirubin ≥1.5 x upper limit of normal.
Drug: Patients will be randomized to receive EHP-101 or Placebo, Drug: Patients will be randomized to receive EHP-101 or Placebo
Diffuse Cutaneous Systemic Sclerosis
Scleroderma, Systemic sclerosis, EHP-101 Oral Solution, dcSSc, diffuse, pathological processes, connective tissue disease, skin disease
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A RANDOMIZED PHASE III STUDY OF IBRUTINIB PLUS OBINUTUZUMAB VERSUS IBRUTINIB PLUS VENETOCLAX AND OBINUTUZUMAB IN UNTREATED OLDER PATIENTS (&#8805; 70 YEARS OF AGE) WITH CHRONIC LYMPHOCYTIC LEUKEMIA (CLL)

Birendra Kumar
Phase III
This study is NOT accepting healthy volunteers
NCT03737981
0123456789
Chronic Lymphocytic Leukemia, Small Lymphocytic Lymphoma
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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
18 Years and over
Phase II
This study is NOT accepting healthy volunteers
NCT04544410
STUDY00011764
Congenital Adrenal Hyperplasia, Diabetes & Endocrine
Adrenal Disorder, CAH, Congenital Adrenal Hyperplasia, congenital adrenal hyperplasia
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MT2020-36: A Phase 2, Open-label, Multicenter Study Evaluating the Safety and Efficacy of Autologous Tumor-infiltrating Lymphocytes (TILs) in Subjects with Advanced Melanoma (DELTA-1) (DELTA-1)

Protocol ITIL-168-101 is a phase 2, open-label, multicenter study evaluating the safety and efficacy of ITIL-168 in adult subjects with advanced melanoma who relapsed after or were refractory to at least 1 prior line of systemic therapy. Subjects will be assessed for study eligibility during the screening period, which occurs within 21 days after the signing of the informed consent form. Once a subject is deemed eligible to participate, they will undergo surgical resection of tumor tissue necessary to harvest TILs for the manufacturing of ITIL-168. Tumor resection should take place within 30 days after providing consent.

Evidio Domingo Musibay
All
18 Years and over
Phase 2
This study is NOT accepting healthy volunteers
NCT05050006
STUDY00011954
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Inclusion Criteria:

• Histologically confirmed advanced (unresectable or metastatic) cutaneous melanoma.
• Cohort 1: Disease that is relapsed after or refractory to at least 1 prior line of systemic therapy that must include a PD-1 inhibitor and, if positive for proto- oncogene BRAF V600 activating mutation, targeted therapy.
• Cohort 2: Disease that is persistent after discontinuing PD-1 due to toxicity. Patients with a proto-oncogene BRAF V600 activating mutation must have progressed after targeted therapy.
• Cohort 3: Disease that is stable (SD) after at least 4 doses of a PD-1 inhibitor. Patients with a proto-oncogene BRAF V600 activating mutation must have progressed after targeted therapy.
• Medically suitable for surgical resection of tumor tissue
• Following tumor resection for TIL harvest, will have, at minimum, 1 remaining measurable lesion as identified by CT or MRI per Response Evaluation Criteria in Solid Tumors (RECIST) v1.1
• Eastern Cooperative Oncology Group (ECOG) performance status 0 or 1
• Adequate bone marrow and organ function Key
Exclusion Criteria:

• History of another primary malignancy within the previous 3 years
• Melanoma of uveal, acral, or mucosal origin
• Previously received an allogeneic stem cell transplant or organ allograft
• Previously received TIL or engineered cell therapy ( eg, CAR T-cell)
• Significant cardiac disease
• Stroke or transient ischemic attack within 12 months of enrollment
• History of significant central nervous system (CNS) disorder
• Symptomatic and/or untreated CNS metastases
• History of significant autoimmune disease within 2 years prior to enrollment
• Known history of severe, immediate hypersensitivity reaction attributed to cyclophosphamide, fludarabine, or IL-2.
Biological: ITIL-168
Advanced Cutaneous Melanoma
ITIL-168, Cell Therapy, melanoma, Autologous cell therapy, Cellular Immunotherapy, TIL, Autologous Adoptive Cell Transfer, Immuno-oncology, IL-2, Autologous Adoptive Cell Therapy, Tumor Infiltrating Lymphocytes, T-cell therapy, Clinics and Surgery Center (CSC)
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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
All
12 Months to 30 Years old
Phase 1/Phase 2
This study is NOT accepting healthy volunteers
NCT04851119
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:
• 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) =<
• 5 x upper limit of normal (ULN) for age
• 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
• PATIENTS WITH SOLID TUMORS: Albumin >= 2 g/dL
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 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
Drug: Tegavivint
Colorectal Carcinoma, Endometrial Carcinoma, Melanoma, Neuroblastoma, Ovarian Carcinoma, Pancreatic Ductal Adenocarcinoma, Recurrent Desmoid Fibromatosis, Recurrent Ewing Sarcoma, Recurrent Hepatoblastoma, Recurrent Hepatocellular Carcinoma, Recurrent Malignant Solid Neoplasm, Recurrent Non-Hodgkin Lymphoma, Recurrent Osteosarcoma, Refractory Desmoid Fibromatosis, Refractory Ewing Sarcoma, Refractory Hepatoblastoma, Refractory Hepatocellular Carcinoma, Refractory Malignant Solid Neoplasm, Refractory Non-Hodgkin Lymphoma, Refractory Osteosarcoma, Solid Pseudopapillary Neoplasm of the Pancreas, Wilms Tumor
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Study to Evaluate the Efficacy and Safety of Pemigatinib in Participants With Previously Treated Glioblastoma or Other Primary Central Nervous System Tumors Harboring Activating FGFR1-3 Alterations (FIGHT-209)

All
18 Years to 99 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT05267106
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Inclusion Criteria:

• Histological, cytological, or molecular confirmation of recurrent GBM or other adult-type, diffuse glioma or circumscribed astrocytic tumors.
• For Cohorts A and C: Prior histopathologically proven WHO grade 4, IDH-wild-type GBM OR molecular diagnosis of IDH-wild-type, diffuse astrocytic glioma with molecular features of WHO grade 4 GBM (astrocytic glioma requires presence of either amplification of EGFR, whole chromosome 7 gain and whole chromosome 10 loss, or TERT-promoter mutation; Louis et al 2021) that has recurred or progressed on or after treatment with at least 1 line of standard of care therapy (eg, temozolomide and radiotherapy or radiotherapy).
• For Cohorts B and C: Prior histopathologically proven, per WHO criteria, adult-type diffuse gliomas other than GBM, including IDH-mutant astrocytoma and IDH-mutant and 1p/19q codeleted oligodendroglioma, and circumscribed astrocytic tumors, including pilocytic astrocytomas, that are recurrent or progressed on or after at least 1 line of standard of care therapy (eg, radiotherapy and/or treatment with an alkylating chemotherapy such as TMZ, CCNU, or BCNU-containing chemotherapy). For Cohort C, all gliomas and glioneuronal and neuronal tumors with a known or likely FGFR 1-3 activating mutation are also eligible.
• Radiographically measurable disease. Tumor lesions located in a previously irradiated area, or in an area subjected to other loco-regional therapy, are considered measurable if progression has been clearly demonstrated in the lesion.
• Karnofsky performance status ≥ 60.
• Life expectancy ≥ 12 weeks.
• Documentation of an FGFR1-3 gene mutation or fusion/rearrangement from tissue (cfDNA from a qualified laboratory such as FMI or Guardant Health may be acceptable after review by medical monitor).
• Cohort A: Participants with prior, histopathologically proven, WHO grade 4,IDH-wild-type GBM OR molecular diagnosis of IDH-wild-type, diffuse astrocytic glioma with molecular features of WHO grade 4 GBM (astrocytic glioma requires presence of either amplification of EGFR, whole chromosome 7 gain and whole chromosome 10 loss, or TERT-promoter mutation; Louis et al 2021) that has recurred, harboring FGFR1-3 fusions or rearrangements (FGFR1-3 in-frame fusions, any FGFR2 rearrangement, or FGFR1/3 rearrangement with known partner). Only FGFR fusions or rearrangements with an intact kinase domain are eligible.
• Cohort B: Participants with other histopathologically proven, per WHO criteria dult-type, diffuse gliomas other than GBM, including IDH-mutant astrocytoma and IDH-mutant and 1p/19q codeleted oligodendroglioma, and circumscribed astrocytic tumors, including pilocytic astrocytomas that are recurrent, harboring FGFR1-3 fusions or rearrangements (FGFR1-3 in-frame fusions, any FGFR2 rearrangement, or FGFR1/3 rearrangement with known partner). Only FGFR fusions or rearrangements with an intact kinase domain are eligible.
• Cohort C: Participants with prior, histopathologically proven, WHO grade 4, IDH-wild-type GBM or molecular diagnosis of IDH-wild-type, diffuse astrocytic glioma with molecular features of WHO grade 4 GBM that has recurred or histopathologically proven, per WHO criteria, adult-type, diffuse gliomas other than GBM, including IDH-mutant astrocytoma and IDH-mutant and 1p/19q codeleted oligodendroglioma, and circumscribed astrocytic tumors, including pilocytic astrocytomas, that are recurrent with a known or likely activating mutation or FGFR1-3 mutation. All gliomas and glioneuronal and neuronal tumors with a known or likely FGFR 1-3 activating mutation are also eligible.
• MRI-documented objective progression after prior therapy and must have no therapy available that is likely to provide clinical benefit. An interval of at least 12 weeks after prior radiotherapy is required unless there is either histopathological confirmation of recurrent tumor or new enhancement on MRI outside the radiotherapy field. Participants who are intolerant of or decline the approved therapy are eligible only if they have no therapy available that is likely to provide clinical benefit.
• Baseline archival tumor specimen less than 24 months from date of screening. Must be a tumor block or a minimum of 15 unstained slides from biopsy or resection of primary tumor or metastasis.
• Willingness to avoid pregnancy or fathering children.
Exclusion Criteria:

• Prior receipt of a selective FGFR inhibitor.
• Receipt of anticancer medications or investigational drugs for any indication or reason within 28 days before first dose of study drug. Participants must have recovered (≤ Grade 1 as per CTCAE v5.0 or at pretreatment baseline) from AEs from previously administered therapies (excluding alopecia).
• Participants may have had treatment for an unlimited number of prior relapses but must not have had prior bevacizumab or other VEGF/VEGFR inhibitors (exception: prior bevacizumab is allowed if it was administered for the treatment of radiation necrosis rather than progressive tumor and was stopped at least 12 weeks prior to MRI showing tumor progression).
• Concurrent anticancer therapy (eg, chemotherapy, immunotherapy, biologic therapy, hormonal therapy, investigational therapy, or tumor embolization).
• Candidate for potentially curative surgery.
• Dexamethasone (or equivalent) > 4 mg daily at the time of study registration (higher doseof steroid for symptom control is allowed during the study).
• Current evidence of clinically significant corneal (including but not limited to bullous/band keratopathy, corneal abrasion, inflammation/ulceration, and keratoconjunctivitis) or retinal disorder (including but not limited to macular/retinal degeneration, diabetic retinopathy, and retinal detachment) as confirmed by ophthalmologic examination.
• Diffuse leptomeningeal disease.
• Radiation therapy administered within 12 weeks before enrollment/first dose of study drug. An interval of at least 12 weeks after prior radiotherapy is required unless there is either histopathological confirmation of recurrent tumor or new enhancement on MRI outside the radiotherapy field.
• Known additional malignancy that is progressing or requires active systemic treatment. Exceptions include basal cell carcinoma of the skin, squamous cell carcinoma of the skin,or in situ cervical cancer that has undergone potentially curative therapy.
• Participants with defined laboratory values at screening.
Drug: Pemigatinib
Glioblastoma, Adult-type Diffuse Gliomas
glioblastoma, GBM, adult-type diffuse gliomas, gliomas, oligodendroglioma, FGFR1-3 Alteration, FGFR1-3 fusions, FGFR1-3 rearrangements, Central nervous system tumor, isocitrate dehydrogenase, IDH-mutant astocytoma, IDH-wild-type GBM
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MT2020-23: A Phase I, Open-Label, Multicenter Study of FT538 as Monotherapy in Relapsed/Refractory Acute Myelogenous Leukemia and in Combination with Monoclonal Antibodies in Relapsed/Refractory Multiple Myeloma

This is a Phase I, open-label, multicenter study to evaluate the safety, pharmacokinetics, and anti-tumor activity of FT538 in subjects with relapsed or refractory (r/r) AML and r/r MM. Subjects will be enrolled in two stages: a dose-escalation stage and a dose-expansion stage.

Mark Juckett
All
18 Years and over
Phase 1
This study is NOT accepting healthy volunteers
NCT04614636
STUDY00010225
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Inclusion Criteria:

• Diagnosis of one of the following by treatment regimen: Regimen A (FT538 monotherapy in r/r AML)
• Primary refractory AML, or
• Relapsed AML, defined as not in CR after one or more re-induction attempts; if >60 years of age, prior re-induction therapy is not required Regimens B or C (FT538 + mAb in r/r MM)
• Regimen B only: MM that has relapsed or progressed after at least two lines of therapies, including a proteasome inhibitor and an immunomodulatory drug
• Regimen C only: MM that has relapsed or progressed after proteasome inhibitor therapy, and immunomodulatory therapy
• Regimen B and Regimen C: Measurable disease as defined in the protocol
• Capable of giving signed informed consent
• Age ≥18 years old
• Agreement to comply with study procedures as described in the Schedule of Activities
• Contraceptive use as described in the protocol
Exclusion Criteria:

• Females who are pregnant or breastfeeding
• ECOG Performance Status ≥ 2
• Evidence of insufficient hematologic function as defined in the protocol
• Evidence of insufficient organ function defined as defined by the protocol
• Clinically significant cardiovascular disease as defined by the protocol
• Known active central nervous system (CNS) involvement by malignancy
• Non-malignant CNS disease such as stroke, epilepsy, CNS vasculitis, or neurodegenerative disease or receipt of medications for these conditions in the 2-year period leading up to study enrollment
• Currently receiving or likely to require systemic immunosuppressive therapy for any reason during the treatment period
• Clinically significant infections including HIV, HBV and HCV
• Live vaccine <6 weeks prior to start of lympho-conditioning
• Receipt of an allograft organ transplant
• Prior allogeneic HSCT or allogeneic CAR-T within 6 months of Day 1, or ongoing requirement for systemic graft-versus-host therapy
• Known allergy to albumin (human) or DMSO
• Presence of any medical or social issues that are likely to interfere with study conduct or may cause increased risk to subject
• Any medical condition or clinical laboratory abnormality that per investigator or Medical Monitor judgement precludes safe participation in and completion of the study, or which could affect compliance with protocol conduct or interpretation of results Exclusion Criteria Specific to Regimen A (r/r AML)
• Diagnosis of promyelocytic leukemia with t(15;17) translocation
• Receipt of any biological therapy, chemotherapy, or radiation therapy, except for palliative purposes, within 2 weeks prior to Day 1 or five half-lives, whichever is shorter; or any investigational therapy within 28 days prior to Day 1 Exclusion Criteria Specific to Regimens B and C (r/r MM)
• Plasma cell leukemia defined as a plasma cell count >2000/mm3
• Leptomeningeal involvement of MM
• Receipt of any biological therapy, chemotherapy, or radiation therapy, except for palliative purposes, within 2 weeks prior to Day 1 or five half-lives, whichever is shorter; or any investigational therapy within 28 days prior to the first dose of mAb
• Allergy or hypersensitivity to antibodies or antibody-related proteins
Drug: FT538, Drug: Cyclophosphamid, Drug: Fludarabine, Drug: Daratumumab, Drug: Elotuzumab
Acute Myeloid Leukemia, AML, Adult, Multiple Myeloma, Myeloma
Acute Myeloid Leukemia, AML, Multiple Myeloma, daratumumab, elotuzumab, NK cell, cellular therapy, Clinics and Surgery Center (CSC)
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A Multicenter, 6-Month, Randomized, Open-Label, Active Control, Parallel Arm, Phase 2b Study of Daily Oral LUM-201 in Naive-to-Treatment, Prepubertal Children with Growth Hormone Deficiency (GHD)

This research study is studying LUM-201 as a possible treatment for Growth Hormone Deficiency (GHD) in pre-pubertal (before puberty) children. Lumos Pharma is sponsoring this research study. Your child is being asked to be in this study because she or he has been diagnosed to have growth hormone deficiency; and your child’s study doctor thinks that your child might be a good candidate for this study. Growth hormone deficiency can result in growth failure. One of the most visible signs of growth failure is a height that is much shorter than most other children of the same age. This is called short stature. However, some children can have growth failure even if they do not have short stature. The standard treatment for growth hormone deficiency is daily injections under the skin of recombinant human growth hormone (rhGH). This study seeks to see if oral LUM-201 at various doses may achieve similar catch-up growth compared to rhGH and provide a safe and effective oral treatment alternative to daily injections. LUM-201 is to be taken by mouth and it is thought that it can increase the body’s ability to release growth hormone.

Brad Miller, MD, PhD
Phase II
This study is NOT accepting healthy volunteers
NCT04614337
STUDY00011599
Growth Hormone Deficiency
Catch-up growth, GHD, Growth hormone secretagogue, Height, LUM-201, Oral, PEM, PGHD, Predictive Enrichment Marker
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A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Crinecerfont (NBI-74788) in Pediatric Subjects with Classic Congenital Adrenal Hyperplasia, Followed by Open-Label Treatment

A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Crinecerfont (NBI-74788) in Pediatric Subjects with Classic Congenital Adrenal Hyperplasia, Followed by Open-Label Treatment Participants will be randomized to placebo or Crinecerfont for 28 weeks followed by an open-label period where all participants will receive Crinecerfont for 24 weeks. The purpose of this research study in children (ages 2 to 17 years) with CAH are: • To study whether crinecerfont can lower high levels of adrenal androgens (male hormones) and high doses of glucocorticoid medication • To study whether improving high androgen levels and high glucocorticoid doses can lead to improvements in CAH • To learn about what happens to crinecerfont in the body by measuring levels in your child’s body after starting the study drug. • To study the safety and tolerability of crinecerfont The study will last for about 60 weeks with 14 study visits. The main activities in this study are: physical exams, height and weight measurements, vital signs, study drug dosing, glucocorticoid dose adjustments, blood draws, saliva and urine tests, electrocardiogram (ECG) testing, x-ray for bone age, ultrasound of testes (boys), and questionnaires.

Kyriakie Sarafoglou
Phase III
This study is NOT accepting healthy volunteers
NCT04806451
STUDY00012858
Congenital Adrenal Hyperplasia
21-hydroxylase deficiency, classic congenital adrenal hyperplasia (CAH)
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A Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study to Evaluate the Efficacy and Safety of Obinutuzumab in Patients with ISN/RPs 2003 Class III or IV Lupus Nephritis (REGENCY) (REGENCY)

Study to Evaluate the Efficacy and Safety of Obinutuzumab in Patients with ISN/RPS 2003 Class III or IV Lupus Nephritis

Surabhi Thakar
All
18 Years to 75 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT04221477
STUDY00010015
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Key
Inclusion Criteria:

• Diagnosis of active or active/chronic ISN/RPS 2003 Class III or IV proliferative LN as evidenced by renal biopsy performed within 6 months. Participants may co-exhibit Class V disease in addition to either Class III or Class IV disease
• Urine protein to creatinine ratio greater than or equal to (>/=) 1 on a 24-hour collection
• Other inclusion criteria may apply Key
Exclusion Criteria:

• Pregnancy or breastfeeding
• Severe renal impairment or the need for dialysis or renal transplantation
• Receipt of an excluded therapy, including any anti-CD20 therapy less than 9 months prior to screening or during screening; or cyclophosphamide, tacrolimus, ciclosporin, or voclosporin during the 2 months prior to screening or during screening
• Significant or uncontrolled medical disease which, in the investigator's opinion, would preclude patient participation
• Known active infection of any kind or recent major episode of infection
• Intolerance or contraindication to study therapies
• Other exclusion criteria may apply
Drug: Obinutuzumab, Drug: MMF, Drug: Prednisone, Drug: Placebo, Drug: Methylprednisolone, Drug: Acetaminophen, Drug: Diphenhydramine
Lupus Nephritis
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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
All
2 Years to 50 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT04293185
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 severe VOEs in the 24 months prior to informed consent as defined below. A protocol-defined severe VOE is: (a) an event of acute priapism: defined as a sustained, unwanted painful erection lasting more than 2 hours and requiring care at a medical facility (with or without hospitalization) or (b) an event that requires a ≥ 24-hour hospital or emergency room (ER) observation unit visit or at least 2 visits to a day unit or ER over 72 hours with both visits requiring intravenous treatment.
• 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 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) (e.g. TCD velocity >200 cm/sec) requiring 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-lymphotrophic virus-1 (HTLV-1) or -2 (HTLV-2), 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 in the bone marrow that may put the subject at an increased risk of myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) per Investigator's judgment.
Genetic: bb1111
Sickle Cell Disease
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MT2020-06: A PHASE 1/2 STUDY TO EVALUATE THE SAFETY, TOLERABILITY, AND EFFICACY OF JSP191 FOR HEMATOPOIETIC CELL TRANSPLANTATION CONDITIONING TO ACHIEVE ENGRAFTMENT AND IMMUNE RECONSTITUTION IN SUBJECTS WITH SCID

Phase 1: To evaluate the safety and tolerability of JSP191 and to determine Phase 2 doses of JSP191 as a conditioning agent prior to allogeneic hematopoietic cell transplantation (HCT) in two populations of subjects with severe combined immunodeficiency (SCID): • SCID subjects with history of prior allogeneic HCT but with poor graft function • SCID subjects who are HCT-naïve Phase 2: • To evaluate the efficacy of JSP191 conditioning to enable engraftment of allogeneic CD34+ hematopoietic cells, as determined by CD15+ donor myeloid chimerism • To evaluate the efficacy of JSP191 conditioning to enable immune reconstitution determined by the production of naïve T cells

Christen Ebens
All
3 Months and over
Phase 1/Phase 2
This study is NOT accepting healthy volunteers
NCT02963064
STUDY00010559
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Inclusion Criteria:
All patient groups must have:
• Typical SCID as defined by Primary Immune Deficiency Treatment Consortia including but not limited to the following subtypes:
• T-, B+, NK-: IL-2Rcγ deficient, JAK3-deficient
• T-, B-, NK+: RAG1/2 deficient, Artemis-deficient
• T-, B+, NK+: IL7Rα deficient, CD3 subunit deficient, CD45 deficient OR Variant SCID with absent or low T cell function, Omenn syndrome, Leaky SCID, Reticular dysgenesis, Adenosine deaminase deficiency, and Purine nucleoside phosphorylase deficiency may be included after consultation with the medical monitor.
• Patients with human leukocyte antigen (HLA) matched related or unrelated donors
• Adequate end organ function as defined in study protocol Key
Exclusion Criteria:

• Patients with any acute or uncontrolled infections
• Patients receiving any other investigational agents, or concurrent biological, chemotherapy, or radiation therapy
• Patients with active malignancies
• Active GVHD within 6 months prior to enrollment, or on immunosuppressive therapy for GVHD
Biological: Humanized anti-CD117 Monoclonal Antibody (JSP191)
SCID
Immunodeficiency, Pediatric, SCID, Bone Marrow Transplantation, GVHD, Stem Cells, Chimerism, Transplant, BMT
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