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Conditions: Diamond Blackfan Anemia
Interventions: Sotatercept, Sotatercept with prednisone boost; Drug
Lead sponsor: Northwell Health; Other
Eligibility: 18 Years and older
Enrollment: 19 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2021
U.S. locations: 1
States / cities: Manhasset, New York

Conditions: Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases, Precancerous/Nonmalignant Condition
Interventions: tetradecanoylphorbol acetate; Drug
Lead sponsor: University of Medicine and Dentistry of New Jersey; Other
Eligibility: 18 Years and older
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1998 – 2004
U.S. locations: 1
States / cities: New Brunswick, New Jersey

Conditions: Pyruvate Kinase Deficiency, Pyruvate Kinase Deficiency Anemia, Hereditary Hemolytic Anemia, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasm, Clonal Myeloid Neoplasm, Myeloproliferative Neoplasm, Acute Myeloid Leukemia, Clonal Cytopenia of Undetermined Significance, Other Clonal Myeloid Neoplasm, Unexplained Coombs-negative Non-immune Hemolytic Anemia
Interventions: Blood Draw; Procedure
Lead sponsor: Massachusetts General Hospital; Other
Eligibility: 18 Years and older
Enrollment: 18 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2026
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Sickle Cell Disease
Interventions: Crizanlizumab; Drug
Lead sponsor: Novartis Pharmaceuticals; Industry
Eligibility: 6 Months to 100 Years
Enrollment: 130 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2031
U.S. locations: 7
States / cities: Birmingham, Alabama • Washington D.C., District of Columbia • Augusta, Georgia + 3 more

Conditions: Twin Twin Transfusion Syndrome
Interventions: Selective Laser Photocoagulation of Communicating Vessels; Device
Lead sponsor: University of South Florida; Other
Eligibility: 18 Years to 55 Years · Female only
Enrollment: 77 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006 – 2009
U.S. locations: 1
States / cities: Tampa, Florida

Conditions: Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(15;17)(q22;q12), Adult Acute Myeloid Leukemia With t(16;16)(p13;q22), Adult Acute Myeloid Leukemia With t(8;21)(q22;q22), Blastic Phase Chronic Myelogenous Leukemia, Chronic Myelomonocytic Leukemia, de Novo Myelodysplastic Syndromes, Previously Treated Myelodysplastic Syndromes, Recurrent Adult Acute Lymphoblastic Leukemia, Recurrent Adult Acute Myeloid Leukemia, Refractory Anemia With Excess Blasts, Refractory Anemia With Excess Blasts in Transformation, Relapsing Chronic Myelogenous Leukemia, Secondary Acute Myeloid Leukemia, Secondary Myelodysplastic Syndromes
Interventions: becatecarin, laboratory biomarker analysis; Drug · Other
Lead sponsor: National Cancer Institute (NCI); NIH
Eligibility: 18 Years and older
Enrollment: 40 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2004
U.S. locations: 1
States / cities: Houston, Texas

Conditions: Sickle Cell Disease
Interventions: Plerixafor, Busulfan, Gene-modified CD34+ cells, Motixafortide; Drug · Biological
Lead sponsor: St. Jude Children's Research Hospital; Other
Eligibility: 18 Years to 24 Years
Enrollment: 25 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2032
U.S. locations: 1
States / cities: Memphis, Tennessee

Conditions: Acne Vulgaris
Interventions: ACZONE Gel, 5%, Vehicle; Drug
Lead sponsor: Allergan; Industry
Eligibility: 12 Years and older
Enrollment: 64 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2006
U.S. locations: 29
States / cities: Birmingham, Alabama • San Diego, California • San Francisco, California + 24 more

Conditions: Skin Ulcers, Sickle Cell Anemia
Interventions: Arginine Butyrate, Standard local care dressing; Drug · Other
Lead sponsor: Susan P. Perrine; Other
Eligibility: 16 Years to 60 Years
Enrollment: 23 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1997 – 2005
U.S. locations: 4
States / cities: Chicago, Illinois • Boston, Massachusetts • New York, New York + 1 more

Conditions: Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(15;17)(q22;q12), Adult Acute Myeloid Leukemia With t(16;16)(p13;q22), Adult Acute Myeloid Leukemia With t(8;21)(q22;q22), Blastic Phase Chronic Myelogenous Leukemia, Childhood Acute Myeloid Leukemia in Remission, Childhood Chronic Myelogenous Leukemia, Childhood Myelodysplastic Syndromes, Previously Treated Myelodysplastic Syndromes, Recurrent Adult Acute Myeloid Leukemia, Recurrent Childhood Acute Myeloid Leukemia, Refractory Anemia With Excess Blasts
Interventions: busulfan, etoposide, intensity-modulated radiation therapy, allogeneic hematopoietic stem cell transplantation, allogeneic bone marrow transplantation, peripheral blood stem cell transplantation, tomotherapy; Drug · Radiation · Procedure
Lead sponsor: City of Hope Medical Center; Other
Eligibility: 6 Years to 55 Years
Enrollment: 25 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2011
U.S. locations: 1
States / cities: Duarte, California

Conditions: Bone Marrow Failure Syndrome, Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia, Acquired Neutropenia in Newborn, Acquired Anemia Hemolytic, Acquired Thrombocytopenia, Hemophagocytic Lymphohistiocytoses, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, Common Variable Immunodeficiency, X-linked Lymphoproliferative Disease, Severe Combined Immunodeficiency, Hurler Syndrome, Mannosidosis, Adrenoleukodystrophy
Interventions: Thiotepa--single daily dose, Thiotepa--escalated dose; Drug
Lead sponsor: University of Florida; Other
Eligibility: 3 Months to 39 Years
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2023
U.S. locations: 1
States / cities: Gainesville, Florida

Conditions: Sickle Cell Disease
Interventions: Rilzabrutinib, Placebo; Drug
Lead sponsor: Sanofi; Industry
Eligibility: 10 Years to 65 Years
Enrollment: 192 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2028
U.S. locations: 21
States / cities: Birmingham, Alabama • Phoenix, Arizona • Clovis, California + 17 more

Conditions: Sickle Cell Disease
Interventions: Interview; Other
Lead sponsor: St. Jude Children's Research Hospital; Other
Eligibility: 8 Years and older
Enrollment: 145 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2023 – 2027
U.S. locations: 1
States / cities: Memphis, Tennessee

Conditions: Sickle Cell Anemia, Sickle Cell Thalassemia, Sickle Cell Pain, Hbss, Hbsc, Sickle Beta Thalassemia, Sickle Beta Zero Thalassemia, Sickle Cell Syndrome Variant
Interventions: Not listed
Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI); NIH
Eligibility: 16 Years to 80 Years
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2027
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Sickle Cell Disease, Stroke, Neurologic Manifestations, Neurological Morbidity, Sickle Cell Anemia in Children, Sickle Cell Anemia
Interventions: This study does not entail offering any intervention; all data collected will be based on standard of care.; Other
Lead sponsor: Vanderbilt University Medical Center; Other
Eligibility: 5 Years to 26 Years
Enrollment: 350 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2023
U.S. locations: 1
States / cities: Nashville, Tennessee

Conditions: Anemia, Sickle Cell
Interventions: Nitric Oxide, Placebo; Drug
Lead sponsor: Mallinckrodt; Industry
Eligibility: 10 Years and older
Enrollment: 150 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2004 – 2008
U.S. locations: 11
States / cities: Birmingham, Alabama • Oakland, California • Aurora, Colorado + 7 more

Conditions: Fanconi Anemia
Interventions: Hematopoietic Stem Cell Transplant, Thymic Shielding During Radiation, Total Body Irradiation, Cyclophosphamide, Fludarabine; Procedure · Drug
Lead sponsor: Masonic Cancer Center, University of Minnesota; Other
Eligibility: 1 Day to 18 Years
Enrollment: 16 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2004 – 2008
U.S. locations: 1
States / cities: Minneapolis, Minnesota

Conditions: Sickle Cell Disease, Vaso-occlusive Pain Episode
Interventions: L-citrulline, Placebo; Drug · Other
Lead sponsor: Suvankar Majumdar; Other
Eligibility: 4 Years to 21 Years
Enrollment: 99 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2029
U.S. locations: 1
States / cities: Washington D.C., District of Columbia

Conditions: Sickle Cell Disease, Chronic Pain
Interventions: Not listed
Lead sponsor: Duke University; Other
Eligibility: 12 Years to 100 Years
Enrollment: 21 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2014
U.S. locations: 1
States / cities: Durham, North Carolina

Conditions: Thalassemia
Interventions: Not listed
Lead sponsor: Carelon Research; Other
Eligibility: 12 Years to 90 Years
Enrollment: 252 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2010
U.S. locations: 7
States / cities: Oakland, California • Atlanta, Georgia • Chicago, Illinois + 4 more

Conditions: Sickle Cell Disease
Interventions: Prasugrel, Placebo; Drug
Lead sponsor: Eli Lilly and Company; Industry
Eligibility: 2 Years to 17 Years
Enrollment: 341 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2015
U.S. locations: 20
States / cities: Oakland, California • Palo Alto, California • Hartford, Connecticut + 16 more

Conditions: Acute Respiratory Distress Syndrome
Interventions: Sigh breaths; Other
Lead sponsor: University of Minnesota; Other
Eligibility: 18 Years to 89 Years
Enrollment: 524 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2022
U.S. locations: 15
States / cities: Fresno, California • Los Angeles, California • Sacramento, California + 12 more

Conditions: Sickle Cell Disease, Pharmacokinetics
Interventions: L-glutamine; Drug
Lead sponsor: Emmaus Medical, Inc.; Industry
Eligibility: 5 Years and older
Enrollment: 13 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2021
U.S. locations: 1
States / cities: Cincinnati, Ohio

Conditions: Sickle Cell Disease
Interventions: Low Dose Rifaximin ER, Low Dose Rifaximin DER, High Dose Rifaximin ER, High Dose Rifaximin DER, Placebo; Drug
Lead sponsor: Bausch Health Americas, Inc.; Industry
Eligibility: 18 Years to 70 Years
Enrollment: 44 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2023
U.S. locations: 5
States / cities: Orange, California • Denver, Colorado • Atlanta, Georgia + 2 more

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Safety and Efficacy Study of Sotatercept in Adults With Transfusion Dependent Diamond Blackfan Anemia

12-O-Tetradecanoylphorbol-13-acetate in Treating Patients With Hematologic Cancer or Bone Marrow Disorder

Acquired Pyruvate Kinase Deficiency In Clonal Myeloid Neoplasms

Rollover Study for Patients With Sickle Cell Disease Who Have Completed a Prior Novartis-Sponsored Crizanlizumab Study

Selective Laser Photocoagulation of Communicating Vessels in Twin-Twin Transfusion Syndrome

Rebeccamycin Analog in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia, Myelodysplastic Syndrome, Acute Lymphoblastic Leukemia, or Chronic Myelogenous Leukemia

Gene Editing For Sickle Cell Disease

Phase IV Study to Gather More Information About the Safety of ACZONE Gel, 5% in Treating Subjects With Acne Who Have G6PD Deficiency

Phase II Randomized Trial:Arginine Butyrate Plus Standard Local Therapy in Patients With Refractory Sickle Cell Ulcers

Busulfan, Etoposide, and Intensity-Modulated Radiation Therapy Followed By Donor Stem Cell Transplant in Treating Patients With Advanced Myeloid Cancer

Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation

The Efficacy and Safety of Rilzabrutinib in Participants Aged 10 to 65 Years With Sickle-cell Disease

Gene Therapy Communication: Use of a Needs Assessment to Drive Decision-AIDS for Gene Therapy for Rare Diseases (GENETX)

Investigating the Mechanistic Effects of Mitapivat in Subjects With Sickle Cell Disease

The Afolabi Stroke Registry for Children and Young Adults With SCD in Northern Nigeria

Nitric Oxide Inhalation to Treat Sickle Cell Pain Crises

Stem Cell Transplantation for Fanconi Anemia

Intravenous L-Citrulline for Vaso-occlusive Pain Episode in Sickle Cell Disease

Use of a Mobile-based App for SCD Patients

Assessment of Pain in People With Thalassemia

A Study of Prasugrel in Pediatric Participants With Sickle Cell Disease (SCD)

Sigh Ventilation to Increase Ventilator-Free Days in Victims of Trauma at Risk for Acute Respiratory Distress Syndrome

Pharmacokinetics and Safety of Endari (L-glutamine) in Sickle Cell Disease Patients

Pharmacokinetics and Pharmacodynamics of Rifaximin Novel Formulations in Patients With Sickle Cell Disease