- Conditions
- Thalassemia Major
- Interventions
- Vitamin D3, Placebo
- Drug
- Lead sponsor
- Weill Medical College of Cornell University
- Other
- Eligibility
- 6 Years to 60 Years
- Enrollment
- 40 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2014
- U.S. locations
- 1
- States / cities
- New York, New York
Search Results
Search by objective public record fields.
Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results come from ClinicalTrials.gov. When the live registry is unavailable, a clearly labeled stored copy may be shown.
These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Showing 73–96
of 115
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Acute Leukemia, Severe Aplastic Anemia, Non-hodgkin Lymphoma, Hodgkin Lymphoma, Kostmann, Diamond Blackfan Anemia, Amegakaryocytic Thrombocytopenia, Sickle Cell Disease, Beta-Thalassemia
- Interventions
- alpha beta depletion
- Drug
- Lead sponsor
- Mitchell Cairo
- Other
- Eligibility
- 1 Day to 30 Years
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2027
- U.S. locations
- 1
- States / cities
- Valhalla, New York
- Conditions
- Thalassemia
- Interventions
- Luspatercept, Placebo, Best Supportive Care (BSC)
- Drug · Other
- Lead sponsor
- Celgene
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 145 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2022
- U.S. locations
- 4
- States / cities
- Los Angeles, California • Oakland, California • Chicago, Illinois
- Conditions
- THALASSEMIA MAJOR
- Interventions
- Blood Draw/Semen Exam, Retrospective data/Chart Review/Relevant Clinical Results, Pituitary MRI
- Other
- Lead sponsor
- UCSF Benioff Children's Hospital Oakland
- Other
- Eligibility
- 12 Years and older
- Enrollment
- 59 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2013 – 2020
- U.S. locations
- 1
- States / cities
- Oakland, California
- Conditions
- Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases, Hemoglobinopathies
- Interventions
- CTX001
- Biological
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Industry
- Eligibility
- 2 Years to 11 Years
- Enrollment
- 16 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2027
- U.S. locations
- 1
- States / cities
- Nashville, Tennessee
- Conditions
- Transfusion-dependent Alpha-Thalassemia, Transfusion-dependent Beta-Thalassemia
- Interventions
- Mitapivat Matched Placebo, Mitapivat
- Drug
- Lead sponsor
- Agios Pharmaceuticals, Inc.
- Industry
- Eligibility
- 1 Year to 17 Years
- Enrollment
- 54 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2032
- U.S. locations
- 4
- States / cities
- Atlanta, Georgia • New York, New York • Philadelphia, Pennsylvania + 1 more
- Conditions
- Sickle Cell Disease, Sickle-Cell; Hb-SC, Sickle Beta 0 Thalassemia
- Interventions
- IMR-687, Placebo Oral Capsule
- Drug
- Lead sponsor
- Cardurion Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years to 55 Years
- Enrollment
- 66 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2016 – 2017
- U.S. locations
- 1
- States / cities
- Overland Park, Kansas
- Conditions
- Thrombocytopenia, Metachromatic Leukodystrophy, Fanconi's Anemia, Thalassemia Major, Pure Red-Cell Aplasia, Inborn Errors of Metabolism
- Interventions
- Stem Cell Transplantation
- Procedure
- Lead sponsor
- National Center for Research Resources (NCRR)
- NIH
- Eligibility
- 0 Years to 17 Years
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 1995
- U.S. locations
- 1
- States / cities
- Los Angeles, California
- Conditions
- Chronic Myeloid Leukemia (CML), Acute Myelogenous Leukemia (AML), Myelodysplastic Syndrome (MDS), Juvenile Myelomonocytic Leukemia (JMML), Acute Lymphoblastic Leukemia (ALL), Lymphoma (Hodgkin's and Non-Hodgkin's)
- Interventions
- CliniMACS CD34+ Reagent System, Thiotepa, Cyclophosphamide, Alemtuzumab, Tacrolimus, Melphalan, Busulfan, Fludarabine, Methylprednisolone
- Device · Drug
- Lead sponsor
- Diane George
- Other
- Eligibility
- Up to 22 Years
- Enrollment
- 14 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2026
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Transfusional Iron Overload, Thalassemia Major, Sickle Cell Disease, Myelodysplastic Syndromes, Aplastic Anemia
- Interventions
- Hepatic biomagnetic susceptibility measurement
- Device
- Lead sponsor
- Columbia University
- Other
- Eligibility
- 5 Years to 80 Years
- Enrollment
- 99 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2011 – 2016
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Leukemia, Lymphoma, Bone Marrow Failure, Immunodeficiencies, Histiocytosis, Sickle Cell Disease, Beta Thalassemia, Inborn Errors of Metabolism
- Interventions
- Full Intensity with TBI, Full Intensity, Reduced Intensity, Reduced Intensity (Fanconi)
- Drug
- Lead sponsor
- New York Medical College
- Other
- Eligibility
- Up to 70 Years
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2018
- U.S. locations
- 1
- States / cities
- Valhalla, New York
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0), Beta-Thalassemia, Transfusion Dependent Beta-Thalassaemia
- Interventions
- autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein, Sequencing Assay for Variant rs114518452
- Biological · Device
- Lead sponsor
- Daniel Bauer
- Other
- Eligibility
- 13 Years to 40 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2030
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Sickle Red Blood Cell, Fetal Hemoglobin, Oxidative Stress
- Interventions
- Broccosprouts® (Brassica Protection Products LLC) homogenate
- Dietary Supplement
- Lead sponsor
- Duke University
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 21 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2012 – 2015
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- Conditions
- Sickle Cell Disease, Sickle Beta Zero Thalassemia, Sickle B+ Thalassemia, Sickle Cell Hemoglobin C
- Interventions
- MED-Go App
- Behavioral
- Lead sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
- Other
- Eligibility
- 12 Years to 21 Years
- Enrollment
- 36 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2025
- U.S. locations
- 1
- States / cities
- Chicago, Illinois
- Conditions
- β-thalassemia, Ineffective Erythropoiesis
- Interventions
- PTG-300
- Drug
- Lead sponsor
- Protagonist Therapeutics, Inc.
- Industry
- Eligibility
- 12 Years to 65 Years
- Enrollment
- 34 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2020
- U.S. locations
- 2
- States / cities
- Oakland, California • Boston, Massachusetts
- Conditions
- β-thalassemia Major
- Interventions
- LentiGlobin BB305 Drug Product
- Genetic
- Lead sponsor
- Genetix Biotherapeutics Inc.
- Industry
- Eligibility
- 12 Years to 35 Years
- Enrollment
- 19 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2018
- U.S. locations
- 4
- States / cities
- Los Angeles, California • Oakland, California • Chicago, Illinois + 1 more
- Conditions
- Hemoglobinopathies, Anemia, Sickle Cell, Hemoglobin SC Disease, Thalassemia, Thalassemia Major
- Interventions
- Busulfan, Fludarabine, FK506, Prednisone
- Drug
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
- NIH
- Eligibility
- 18 Years and older
- Enrollment
- 2 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2001 – 2003
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions, Systemic Juvenile Idiopathic Arthritis (sJIA), Juvenile Rheumatoid Arthritis (JRA)
- Interventions
- Hydroxyurea, Alemtuzumab, Fludarabine, Melphalan, Thiotepa
- Drug
- Lead sponsor
- Paul Szabolcs
- Other
- Eligibility
- 2 Months to 55 Years
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2027
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy, Hyperinsulinemic Hypoglycemia, Familial 1, Diabetes Mellitus, Adrenoleukodystrophy, Neonatal, Medium-chain Acyl-CoA Dehydrogenase Deficiency, Very Long Chain Acyl Coa Dehydrogenase Deficiency, Beta-ketothiolase Deficiency, Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency, Primary Hyperoxaluria Type 1, Congenital Bile Acid Synthesis Defect Type 2, Pyridoxine-Dependent Epilepsy, Hereditary Fructose Intolerance, Hypophosphatasia, Hyperargininemia, Mucopolysaccharidosis Type 6, Argininosuccinic Aciduria, Citrullinemia, Type I, Wilson Disease, Maple Syrup Urine Disease, Type 1A, Maple Syrup Urine Disease, Type 1B, Biotinidase Deficiency, Neonatal Severe Primary Hyperparathyroidism, Intrinsic Factor Deficiency, Usher Syndrome Type 1D/F Digenic (Diagnosis), Cystic Fibrosis, Stickler Syndrome Type 2, Stickler Syndrome Type 1, Alport Syndrome, Autosomal Recessive, Alport Syndrome, X-Linked, Carbamoyl Phosphate Synthetase I Deficiency Disease, Carnitine Palmitoyl Transferase 1A Deficiency, Carnitine Palmitoyltransferase II Deficiency, Cystinosis, Chronic Granulomatous Disease, Cerebrotendinous Xanthomatoses, Maple Syrup Urine Disease, Type 2, Severe Combined Immunodeficiency Due to DCLRE1C Deficiency, Thyroid Dyshormonogenesis 6, Thyroid Dyshormonogenesis 5, Supravalvar Aortic Stenosis, Factor X Deficiency, Hemophilia A, Hemophilia B, Tyrosinemia, Type I, Fructose 1,6 Bisphosphatase Deficiency, Glycogen Storage Disease Type I, G6PD Deficiency, Glycogen Storage Disease II, Galactokinase Deficiency, Mucopolysaccharidosis Type IV A, Galactosemias, Guanidinoacetate Methyltransferase Deficiency, Agat Deficiency, Glutaryl-CoA Dehydrogenase Deficiency, Gtp Cyclohydrolase I Deficiency, Hyperinsulinism-Hyperammonemia Syndrome, Primary Hyperoxaluria Type 2, 3-Hydroxyacyl-CoA Dehydrogenase Deficiency, Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency, Mitochondrial Trifunctional Protein Deficiency, Sickle Cell Disease, Beta-Thalassemia, Holocarboxylase Synthetase Deficiency, 3-Hydroxy-3-Methylglutaric Aciduria, Primary Hyperoxaluria Type 3, Hermansky-Pudlak Syndrome 1, Hermansky-Pudlak Syndrome 4, Apparent Mineralocorticoid Excess, HSDB, CBAS1, Mucopolysaccharidosis Type 2, Mucopolysaccharidosis Type 1, Severe Combined Immunodeficiency, X Linked, Severe Combined Immunodeficiency Due to IL-7Ralpha Deficiency, Diabetes Mellitus, Permanent Neonatal, Isovaleric Acidemia, Severe Combined Immunodeficiency T-Cell Negative B-Cell Positive Due to Janus Kinase-3 Deficiency (Disorder), Jervell and Lange-Nielsen Syndrome 2, Hyperinsulinemic Hypoglycemia, Familial, 2, Diabetes Mellitus, Permanent Neonatal, With Neurologic Features, Jervell and Lange-Nielsen Syndrome 1, Lysosomal Acid Lipase Deficiency, CblF, 3-Methylcrotonyl CoA Carboxylase 1 Deficiency, 3-Methylcrotonyl CoA Carboxylase 2 Deficiency, Waardenburg Syndrome Type 2A, Methylmalonic Aciduria cblA Type, Methylmalonic Aciduria cblB Type, Methylmalonic Aciduria and Homocystinuria Type cblC, MAHCD, Methylmalonic Aciduria Due to Methylmalonyl-CoA Mutase Deficiency, Congenital Disorder of Glycosylation Type 1B, Mthfr Deficiency, Methylcobalamin Deficiency Type Cbl G (Disorder), Methylcobalamin Deficiency Type cblE, Usher Syndrome, Type 1B, N-acetylglutamate Synthase Deficiency, Ornithine Transcarbamylase Deficiency, Phenylketonurias, Waardenburg Syndrome Type 1, Congenital Hypothyroidism, Propionic Acidemia, Usher Syndrome, Type 1F, Pancreatic Agenesis 1, Hereditary Hypophosphatemic Rickets, Glycogen Storage Disease IXB, Glycogen Storage Disease IXC, MOWS, Epilepsy, Early-Onset, Vitamin B6-Dependent, Pyridoxal Phosphate-Responsive Seizures, Pituitary Hormone Deficiency, Combined, 1, Ptsd, Dihydropteridine Reductase Deficiency, Severe Combined Immunodeficiency Due to RAG1 Deficiency, Severe Combined Immunodeficiency Due to RAG2 Deficiency, Retinoblastoma, Multiple Endocrine Neoplasia Type 2B, Pseudohypoaldosteronism, Type I, Liddle Syndrome, Biotin-Responsive Basal Ganglia Disease, SCD, DIAR1, GSD1C, Acrodermatitis Enteropathica, Thyroid Dyshormonogenesis 1, Riboflavin Transporter Deficiency, Waardenburg Syndrome, Type 2E, SRD, Congenital Lipoid Adrenal Hyperplasia Due to STAR Deficiency, Barth Syndrome, Adrenocorticotropic Hormone Deficiency, Transcobalamin II Deficiency, Thyroid Dyshormonogenesis 3, Segawa Syndrome, Autosomal Recessive, Autosomal Recessive Nonsyndromic Hearing Loss, Thyroid Dyshormonogenesis 2A, Congenital Isolated Thyroid Stimulating Hormone Deficiency, Hypothyroidism Due to TSH Receptor Mutations, Usher Syndrome Type 1C, Usher Syndrome Type 1G (Diagnosis), Von Willebrand Disease, Type 3, Combined Immunodeficiency Due to ZAP70 Deficiency, Adenine Phosphoribosyltransferase Deficiency, Metachromatic Leukodystrophy, Canavan Disease, Menkes Disease, Carbonic Anhydrase VA Deficiency, Developmental and Epileptic Encephalopathy 2, 17 Alpha-Hydroxylase Deficiency, Smith-Lemli-Opitz Syndrome, Krabbe Disease, Glutathione Synthetase Deficiency, Mucopolysaccharidosis Type 7, Rett Syndrome, Molybdenum Cofactor Deficiency, Type A, Niemann-Pick Disease, Type C1, Niemann-Pick Disease Type C2, Ornithine Aminotransferase Deficiency, 3-Phosphoglycerate Dehydrogenase Deficiency, Leber Congenital Amaurosis 2, Dravet Syndrome, Mucopolysaccharidosis Type 3 A, Ornithine Translocase Deficiency, Carnitine-acylcarnitine Translocase Deficiency, Glucose Transporter Type 1 Deficiency Syndrome, Creatine Transporter Deficiency, Niemann-Pick Disease Type A, Pitt Hopkins Syndrome, Tuberous Sclerosis 1, Tuberous Sclerosis 2, Ataxia With Isolated Vitamin E Deficiency, Angelman Syndrome, Prader-Willi Syndrome, Homocystinuria, Permanent Neonatal Diabetes Mellitus, Transient Neonatal Diabetes Mellitus, Factor VII Deficiency, Glycogen Storage Disease Type IXA1, Glycogen Storage Disease, Type IXA2, Glycogen Storage Disease IC, Glycogen Storage Disease Type IB, Central Hypoventilation Syndrome With or Without Hirschsprung Disease
- Interventions
- Confirmatory Testing
- Diagnostic Test
- Lead sponsor
- RTI International
- Other
- Eligibility
- 1 Day to 31 Days
- Enrollment
- 30,000 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2018 – 2026
- U.S. locations
- 1
- States / cities
- Research Triangle Park, North Carolina
- Conditions
- Bone Marrow Failure Syndrome, Severe Aplastic Anemia, Severe Congenital Neutropenia, Amegakaryocytic Thrombocytopenia, Diamond-Blackfan Anemia, Schwachman Diamond Syndrome, Primary Immunodeficiency Syndromes, Acquired Immunodeficiency Syndromes, Histiocytic Syndrome, Familial Hemophagocytic Lymphocytosis, Lymphohistiocytosis, Macrophage Activation Syndrome, Langerhans Cell Histiocytosis (LCH), Hemoglobinopathies, Sickle Cell Disease, Sickle Cell-beta-thalassemia
- Interventions
- CD34 Stem Cell Selection Therapy
- Biological
- Lead sponsor
- Diane George
- Other
- Eligibility
- Up to 40 Years
- Enrollment
- 37 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2030
- U.S. locations
- 1
- States / cities
- New York, New York
- 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
- 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
- Transfusion-dependent Beta-Thalassemia
- Interventions
- Safety and efficacy assessments
- Other
- Lead sponsor
- Genetix Biotherapeutics Inc.
- Industry
- Eligibility
- 0 Years to 50 Years
- Enrollment
- 66 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2035
- U.S. locations
- 6
- States / cities
- Oakland, California • Chicago, Illinois • Bethesda, Maryland + 3 more
- Conditions
- Transfusion Dependent Beta Thalassemia, Sickle Cell Disease
- Interventions
- Not listed
- Lead sponsor
- Georgetown University
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2026
- U.S. locations
- 1
- States / cities
- Washington D.C., District of Columbia