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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 55 Years
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Anemia, Sickle Cell, Complex and Transfusion-dependent Hemoglobinopathies, Thalassemia, Diamond-Blackfan Anemia, Bone Marrow Failure Syndromes, Alpha-Thalassemia, Beta-Thalassemia
Interventions: Enriched Hematopoetic Stem Cell Infusion; Biological
Lead sponsor: Talaris Therapeutics Inc.; Industry
Eligibility: Up to 45 Years
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2016
U.S. locations: 3
States / cities: Chicago, Illinois • Louisville, Kentucky • Durham, North Carolina

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: 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: 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: 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: Blood and Lymphatic Diseases
Interventions: BIVV003, ST-400; Drug
Lead sponsor: Sangamo Therapeutics; Industry
Eligibility: 18 Years to 45 Years
Enrollment: 8 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2038
U.S. locations: 7
States / cities: Oakland, California • Sacramento, California • Atlanta, Georgia + 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

Conditions: Sickle Cell Anemia
Interventions: Ambrisentan, Placebo; Drug
Lead sponsor: Augusta University; Other
Eligibility: 18 Years to 65 Years
Enrollment: 26 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2019
U.S. locations: 1
States / cities: Augusta, Georgia

Conditions: Transfusional Iron Overload, Beta-Thalassemia
Interventions: SPD602; Drug
Lead sponsor: Shire; Industry
Eligibility: 6 Years to 17 Years
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2014
U.S. locations: 2
States / cities: Boston, Massachusetts • Philadelphia, Pennsylvania

Conditions: Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis, Creutzfeld-Jakob Disease, Cystic Fibrosis, Duchenne Muscular Dystrophy, Early-Onset Alzheimer Disease, Ehlers-Danlos Syndrome, Huntington Disease, Gaucher Disease, GM1 Gangliosidosis, Myasthenia Gravis, Pompe Disease, Sickle Cell Disease, Transthyretin Amyloid Cardiomyopathy, Rare Diseases
Interventions: Not listed
Lead sponsor: xCures; Industry
Eligibility: Not listed
Enrollment: 1,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2026
U.S. locations: 1
States / cities: Los Altos, California

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: 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: Myeloid Chimerism
Interventions: CliniMACS CD34 Reagent; Device
Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI); NIH
Eligibility: 2 Years to 80 Years
Enrollment: 32 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2019 – 2037
U.S. locations: 1
States / cities: Bethesda, Maryland

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: 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: 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
Interventions: Pociredir; Drug
Lead sponsor: Fulcrum Therapeutics; Industry
Eligibility: 18 Years and older
Enrollment: 50 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2030
U.S. locations: 8
States / cities: Little Rock, Arkansas • Los Angeles, California • Baton Rouge, Louisiana + 5 more

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 – 2026
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Neutropenia, Sickle Cell Anemia, Thalassemia Major, Red-Cell Aplasia, Pure
Interventions: anti-thymocyte globulin, busulfan, cyclophosphamide, Bone marrow transplantation; Drug · Procedure
Lead sponsor: Fairview University Medical Center; Other
Eligibility: 0 Years and older
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2000
U.S. locations: 1
States / cities: Minneapolis, Minnesota

Conditions: Sickle Cell Disease, Hb-SS Disease, Hemoglobin S, Disease Sickle Cell Anemia, Sickle Cell Disorders, Hemoglobin Beta Thalassemia Disease
Interventions: Fostamatinib; Drug
Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI); NIH
Eligibility: 18 Years to 65 Years
Enrollment: 25 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2024 – 2027
U.S. locations: 1
States / cities: Bethesda, Maryland

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 – 2025
U.S. locations: 1
States / cities: Research Triangle Park, North Carolina

Conditions: Beta Thalassemia Intermedia, Sickle Cell Disease
Interventions: Benserazide Only Product; Drug
Lead sponsor: Phoenicia BioScience; Other
Eligibility: 18 Years and older
Enrollment: 36 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2026
U.S. locations: 4
States / cities: Oakland, California • Boston, Massachusetts • Weston, Massachusetts + 1 more

Conditions: Sickle Cell Disease, Thalassemia, Stem Cell Transplantation, Graft vs Host Disease
Interventions: Alemtuzumab, Sirolimus, Cyclophosphamide, Pentostatin, Radiotherapy, Filgrastim; Drug · Procedure
Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI); NIH
Eligibility: 4 Years to 80 Years
Enrollment: 56 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2014 – 2027
U.S. locations: 1
States / cities: Bethesda, Maryland

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Expanded Access Protocol for Gene Therapy Utilizing shmiR Lentivirus Vector to Induce Fetal Hemoglobin in Sickle Cell Disease

Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies

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

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

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

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

Long - Term Follow Up of Sickle Cell Disease and Beta-thalassemia Subjects Previously Exposed to BIVV003 or ST-400.

Decision-Making and Quality of Life Surrounding Hematologic Disease and Gene Therapy

The Role of Endothelin-1 in Sickle Cell Disease

Safety, Efficacy and Pharmacokinetics of an Oral Iron Chelator Given for a Year to Pediatric Patients With Iron Overload

FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases

CD34+Selection for Partially Matched Family or Matched Unrelated Adult Donor Transplant

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

Repeat Peripheral Blood Stem Cell Transplantation for Patients With Sickle Cell Disease and Falling Donor Myeloid Chimerism Levels

A Study of IMR-687 in Healthy Adult Volunteers

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)

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

Open-Label Extension Study to Pioneer Study 6058-SCD-101

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

Study of Allogeneic Bone Marrow Transplantation Using Matched, Related Donors in Patients With Nonmalignant Hematologic Disorders

Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease

Early Check: Expanded Screening in Newborns

The BENeFiTS Trial in Beta Thalassemia Intermedia

Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant Failure