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Conditions: Congenital Hemolytic Anemia, Sickle Cell Disease
Interventions: Peripheral blood hematopoietic progenitor cell (PBPC) transplant, Alemtuzumab, Peripheral blood hematopoietic progenitor cell Apheresis, Sirolimus; Procedure · Drug
Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI); NIH
Eligibility: 2 Years to 80 Years
Enrollment: 130 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2004 – 2028
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Sickle Cell Disease
Interventions: Pociredir; Drug
Lead sponsor: Fulcrum Therapeutics; Industry
Eligibility: 18 Years to 65 Years
Enrollment: 13 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2026
U.S. locations: 6
States / cities: Miami, Florida • Orlando, Florida • Riverdale, Georgia + 3 more

Conditions: Sickle Cell Disease
Interventions: Autologous CD34+ HSC cells transduced with the lentiviral vector containing a shRNA targeting BCL11a; Biological
Lead sponsor: David Williams; Other
Eligibility: 13 Years to 40 Years
Enrollment: 25 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2027
U.S. locations: 9
States / cities: Los Angeles, California • Oakland, California • Sacramento, California + 4 more

Conditions: Alpha Thalassemia Major, Hemoglobinopathy; With Thalassemia, Hemoglobinopathies, Fetal Anemia, Fetal Hydrops, Alpha; Thalassemia, Thalassemia Major, Thalassemia Alpha, A-Thalassemia
Interventions: in utero hematopoietic stem cell transplantation; Biological
Lead sponsor: University of California, San Francisco; Other
Eligibility: 18 Weeks to 26 Weeks
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2024
U.S. locations: 1
States / cities: San Francisco, California

Conditions: Beta-Thalassemia, Thalassemia, Hematologic Diseases, Genetic Diseases, Inborn, Hemoglobinopathies, Sickle Cell Disease, Sickle Cell Anemia
Interventions: CTX001; Biological
Lead sponsor: Vertex Pharmaceuticals Incorporated; Industry
Eligibility: 12 Years to 35 Years
Enrollment: 26 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2027
U.S. locations: 3
States / cities: New York, New York • Charlotte, North Carolina • Nashville, Tennessee

Conditions: Beta Thalassemia
Interventions: 5-Azacytidine; Drug
Lead sponsor: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); NIH
Eligibility: Not listed
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2000 – 2003
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Transfusion Dependent Beta-thalassemia
Interventions: ST-400 Investigational product; Genetic
Lead sponsor: Sangamo Therapeutics; Industry
Eligibility: 18 Years to 40 Years
Enrollment: 5 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2022
U.S. locations: 6
States / cities: Los Angeles, California • Oakland, California • Atlanta, Georgia + 3 more

Conditions: Thalassemia
Interventions: Decitabine (USAN, INN); Drug
Lead sponsor: Carelon Research; Other
Eligibility: 18 Years and older
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2010
U.S. locations: 2
States / cities: Oakland, California • Philadelphia, Pennsylvania

Conditions: All Diagnosed Health Conditions, ADD/ADHD, Alopecia Areata, Ankylosing Spondylitis, Asthma, Atopic Dermatitis Eczema, Beta Thalassemia, Bipolar Disorder, Breast Cancer, Celiac Disease, Cervical Cancer, Chronic Inflammatory Demyelinating Polyneuropathy, Chronic Kidney Diseases, Chronic Obstructive Pulmonary Disease, Colon Cancer, Colorectal Cancer, Crohn's Disease, Cystic Fibrosis, Depression, Diabetes Mellitus, Duchenne Muscular Dystrophy, Endometriosis, Epilepsy, Facioscapulohumeral Muscular Dystrophy, G6PD Deficiency, General Anxiety Disorder, Hepatitis B, Hereditary Hemorrhagic Telangiectasia, HIV/AIDS, Human Papilloma Virus, Huntington's Disease, Idiopathic Thrombocytopenic Purpura, Insomnia, Kidney Cancer, Leukemia, Lung Cancer, Lupus Nephritis, Lymphoma, Melanoma, Multiple Myeloma, Multiple Sclerosis, Myositis, Myotonic Dystrophy, Ovarian Cancer, Pancreatic Cancer, Parkinson's Disease, Polycystic Kidney Diseases, Prostate Cancer, Psoriasis, Psoriatic Arthritis, Rosacea, Scleroderma, Sickle Cell Anemia, Sickle Cell Trait, Sjogren's Syndrome, Skin Cancer, Spinal Muscular Atrophy, Systemic Lupus Erythematosus, Thrombotic Thrombocytopenic Purpura, Trisomy 21, Ulcerative Colitis
Interventions: Not listed
Lead sponsor: Sanguine Biosciences; Industry
Eligibility: 18 Years to 100 Years
Enrollment: 17,667 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2013 – 2024
U.S. locations: 1
States / cities: Los Angeles, California

Conditions: Myelodysplastic Syndromes (MDS), Beta-thalassemia, Myeloproliferative Neoplasm(MPN)-Associated Myelofibrosis
Interventions: Luspatercept; Drug
Lead sponsor: Celgene; Industry
Eligibility: 18 Years and older
Enrollment: 665 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2028
U.S. locations: 12
States / cities: Los Angeles, California • Oakland, California • Stanford, California + 9 more

Conditions: Sickle Cell Disease, Thalassemia, Beta, Thalassemia
Interventions: Fludarabine, Cyclophosphamide, Tacrolimus, Mycophenolate Mofetil, Rabbit ATG, Dexamethasone, Bortezomib, Rituximab; Drug · Biological
Lead sponsor: Memorial Sloan Kettering Cancer Center; Other
Eligibility: 2 Years to 50 Years
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2027
U.S. locations: 6
States / cities: Basking Ridge, New Jersey • Middletown, New Jersey • Montvale, New Jersey + 3 more

Conditions: Beta-Thalassemia
Interventions: arginine butyrate, epoetin alfa; Drug
Lead sponsor: Boston University; Other
Eligibility: 3 Years and older
Enrollment: 15 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 1999
U.S. locations: 6
States / cities: Oakland, California • New Haven, Connecticut • Bethesda, Maryland + 2 more

Conditions: Beta-Thalassemia
Interventions: LJPC-401; Drug
Lead sponsor: La Jolla Pharmaceutical Company; Industry
Eligibility: 14 Years and older
Enrollment: 84 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2020
U.S. locations: 2
States / cities: Oakland, California • New York, New York

Conditions: Beta-Thalassemia
Interventions: Deferitrin (GT56-252), desferoxamine (DFO); Drug
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 18 Years and older
Enrollment: 25 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2003 – 2005
U.S. locations: 1
States / cities: New York, New York

Conditions: Beta-Thalassemia, Thalassemia, Sickle Cell Disease, Hematologic Diseases, Hemoglobinopathies, Genetic Diseases, Inborn, Sickle Cell Anemia
Interventions: CTX001; Biological
Lead sponsor: Vertex Pharmaceuticals Incorporated; Industry
Eligibility: 2 Years and older
Enrollment: 160 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2039
U.S. locations: 9
States / cities: Palo Alto, California • Chicago, Illinois • New York, New York + 5 more

Conditions: β-thalassemia, Ineffective Erythropoiesis, Chronic Anemia
Interventions: PTG-300; Drug
Lead sponsor: Protagonist Therapeutics, Inc.; Industry
Eligibility: 12 Years to 65 Years
Enrollment: 63 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2020
U.S. locations: 3
States / cities: Oakland, California • Boston, Massachusetts • Philadelphia, Pennsylvania

Conditions: Sickle Cell Anemia, Beta-thalassemia Major, Diamond-blackfan Anemia
Interventions: CD3/CD19 depleted leukocytes, CD45RA depleted leukocytes, Hydroxyurea, Rituximab, Alemtuzumab, Fludarabine, Thiotepa; Biological · Drug
Lead sponsor: Paul Szabolcs; Other
Eligibility: 5 Years to 40 Years
Enrollment: 5 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2028
U.S. locations: 1
States / cities: Pittsburgh, Pennsylvania

Conditions: Non-transfusion Dependent Thalassemia
Interventions: deferasirox, placebo; Drug
Lead sponsor: Novartis Pharmaceuticals; Industry
Eligibility: 10 Years and older
Enrollment: 166 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2012
U.S. locations: 3
States / cities: Oakland, California • Chicago, Illinois • New York, New York

Conditions: Alpha and Beta Thalassemia, Sickle Cell Disease, Malaria, Human Physiology
Interventions: Not listed
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 18 Years to 70 Years
Enrollment: 300 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2019 – 2029
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Sickle Cell Disease, Transfusion-dependent Beta-Thalassemia, Hemoglobinopathies
Interventions: Safety and efficacy assessments; Other
Lead sponsor: Editas Medicine, Inc.; Industry
Eligibility: 12 Years to 50 Years
Enrollment: 54 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2040
U.S. locations: 16
States / cities: Oakland, California • Aurora, Colorado • New Haven, Connecticut + 12 more

Conditions: Transfusional Iron Overload, Beta-thalassemia
Interventions: FBS0701; Drug
Lead sponsor: FerroKin BioSciences, Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2009
U.S. locations: 3
States / cities: Oakland, California • Boston, Massachusetts • Philadelphia, Pennsylvania

Conditions: Sickle Cell Disease, Sickle B+ Thalassemia, Sickle Beta Zero Thalassemia, Sickle Cell Hemoglobin C
Interventions: HU-Go app; Other
Lead sponsor: Ann & Robert H Lurie Children's Hospital of Chicago; Other
Eligibility: 12 Years to 21 Years
Enrollment: 68 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2025
U.S. locations: 1
States / cities: Chicago, Illinois

Conditions: Non-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-Thalassemia
Interventions: Placebo Matching Mitapivat, Mitapivat; Drug
Lead sponsor: Agios Pharmaceuticals, Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 194 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2028
U.S. locations: 6
States / cities: La Jolla, California • Palo Alto, California • Boston, Massachusetts + 3 more

Conditions: Sickle Cell Disease
Interventions: Patient Navigator, Recruitment into Specialty Care; Behavioral
Lead sponsor: Virginia Commonwealth University; Other
Eligibility: 15 Years and older
Enrollment: 353 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2018
U.S. locations: 1
States / cities: Richmond, Virginia

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Improving the Results of Bone Marrow Transplantation for Patients With Severe Congenital Anemias

Single-dose Pharmacokinetics of Pociredir in Participants With Sickle Cell Disease

A Gene Transfer Study Inducing Fetal Hemoglobin in Sickle Cell Disease (GRASP, BMT CTN 2001)

In Utero Hematopoietic Stem Cell Transplantation for Alpha-thalassemia Major (ATM)

Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease

5-Azacytidine and Phenylbutyrate to Treat Severe Thalassemia

A Study to Assess the Safety, Tolerability, and Efficacy of ST-400 for Treatment of Transfusion-Dependent Beta-thalassemia (TDT)

Evaluating the Safety and Effectiveness of Decitabine in People With Thalassemia Intermedia

At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic Conditions

A Study to Evaluate Long-term Safety in Participants Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials

A Study of Immune Suppression Treatment for People With Sickle Cell Disease or β-Thalassemia Who Are Going to Receive an Allogeneic Hematopoietic Cell Transplantation (HCT)

Phase II Study of Arginine Butyrate With or Without Epoetin Alfa in Patients With Thalassemia Intermedia

A Study With LJPC-401 for the Treatment of Myocardial Iron Overload in Patients With Transfusion-Dependent Beta Thalassemia

Iron Balance Study of DFO and GT56-252 in Patients With Transfusional Iron Overload Secondary to Beta-Thalassemia

A Long-term Follow-up Study in Participants Who Received CTX001

Study of PTG-300 in Non-Transfusion Dependent and Transfusion-Dependent Beta-Thalassemia Subjects With Chronic Anemia

T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias

Efficacy and Safety of Deferasirox in Non-transfusion Dependent Thalassemia Patients With Iron Overload and a One Year Open-label Extension Study

Collection of Human Biospecimens for Basic and Clinical Research Into Globin Variants

A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301

Safety and Pharmacokinetic Study of Escalating Multiple Doses of an Iron Chelator in Patients With Iron Overload

The Longitudinal Relationship of HU Adherence to HRQOL, Barriers to Adherence and Habit in SCD.

A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)

Enhancing Use of Hydroxyurea In Sickle Cell Disease Using Patient Navigators