- Conditions
- Hematologic Malignancies
- Interventions
- UCB and HPDSC
- Procedure
- Lead sponsor
- Celgene Corporation
- Industry
- Eligibility
- Up to 55 Years
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2013
- U.S. locations
- 1
- States / cities
- New Orleans, Louisiana
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 25–48
of 100
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Hydrops Fetalis, Birth Defect, Fetal Anomaly
- Interventions
- Exome sequencing
- Diagnostic Test
- Lead sponsor
- University of California, San Francisco
- Other
- Eligibility
- 18 Years to 55 Years
- Enrollment
- 500 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2027
- U.S. locations
- 1
- States / cities
- San Francisco, California
- 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
- Sickle Cell Anemia, Thalassemia
- Interventions
- L-Glutamine, Placebo
- Drug
- Lead sponsor
- Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2004 – 2009
- U.S. locations
- 1
- States / cities
- Torrance, California
- Conditions
- Sickle Cell Disease, Thalassemia, Anemia, Granuloma, Wiskott-Aldrich Syndrome, Chediak Higashi Syndrome, Osteopetrosis, Neutropenia, Thrombocytopenia, Hurler Disease, Niemann-Pick Disease, Fucosidosis
- Interventions
- Hematopoietic stem cell transplantation
- Procedure
- Lead sponsor
- Children's Hospital Los Angeles
- Other
- Eligibility
- Up to 21 Years
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2015
- U.S. locations
- 1
- States / cities
- Los Angeles, California
- 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
- 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
- Hydrops Fetalis, Fetal Growth Retardation
- Interventions
- Tandem MS test for inborn errors of metabolism
- Procedure
- Lead sponsor
- Obstetrix Medical Group
- Industry
- Eligibility
- 18 Years to 60 Years · Female only
- Enrollment
- 48 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2009
- U.S. locations
- 11
- States / cities
- Phoenix, Arizona • Laguna Hills, California • Long Beach, California + 6 more
- 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
- 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
- Healthy Volunteers, Sickle Cell Disease, Thalassemia
- Interventions
- Etavopivat, Moxifloxacin, Placebo
- Drug
- Lead sponsor
- Novo Nordisk A/S
- Industry
- Eligibility
- 18 Years to 55 Years
- Enrollment
- 33 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2025
- U.S. locations
- 1
- States / cities
- Baltimore, Maryland
- 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
- Beta-Thalassemia
- Interventions
- VIT-2763 60 mg QD, VIT-2763 60 mg BID, VIT-2763 120 mg BID, Placebo
- Drug
- Lead sponsor
- Vifor (International) Inc.
- Industry
- Eligibility
- 18 Years to 65 Years
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2023
- U.S. locations
- 1
- States / cities
- Whittier, California
- Conditions
- Thalassemia Major
- Interventions
- DST-0509, Jadenu, Exjade
- Drug
- Lead sponsor
- DisperSol Technologies, LLC
- Industry
- Eligibility
- 8 Years and older
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2021
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Sickle Cell Disease
- Interventions
- IMR-687, Placebo
- Drug
- Lead sponsor
- Cardurion Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years to 55 Years
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2020
- U.S. locations
- 7
- States / cities
- Oakland, California • Farmington, Connecticut • Hollywood, Florida + 3 more
- Conditions
- Sickle Cell Anemia, Sickle Cell Disease, Chronic Hemolytic Disorders
- Interventions
- Sodium Nitrite Cream
- Drug
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
- NIH
- Eligibility
- 18 Years to 99 Years
- Enrollment
- 28 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2018
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Age-Related Macular Degeneration, Allergies, Alpha-Gal Syndrome, Alzheimer Disease, Amyloidosis, Ankylosing Spondylitis, Arthritis, Alopecia Areata, Asthma, Atopic Dermatitis, Autism, Autoimmune Hepatitis, Behcet's Disease, Beta-Thalassemia, Cancer, Celiac Disease, Kidney Diseases, COPD, Crohn Disease, Cystic Fibrosis, Diabetes, Dravet Syndrome, DMD, Fibromyalgia, Graves Disease, Thyroid Diseases, Hepatitis, Hidradenitis Suppurativa, ITP, Leukemia, ALS, Lupus or SLE, Lymphoma, Multiple Sclerosis, Myasthenia Gravis, Heart Diseases, Parkinson Disease, Pemphigus Vulgaris, Cirrhosis, Psoriasis, Schizophrenia, Scleroderma, Sickle Cell Disease, Stroke, Ulcerative Colitis, Vasculitis, Vitiligo
- Interventions
- Specimen sample
- Diagnostic Test
- Lead sponsor
- Sanguine Biosciences
- Industry
- Eligibility
- 18 Years to 85 Years
- Enrollment
- 20,000 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2021 – 2025
- U.S. locations
- 1
- States / cities
- Waltham, Massachusetts
- 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
- 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 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
- Thalassemia
- Interventions
- AG-348
- Drug
- Lead sponsor
- Agios Pharmaceuticals, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2030
- U.S. locations
- 2
- States / cities
- Oakland, California • Boston, Massachusetts
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia
- Interventions
- vorinostat
- Drug
- Lead sponsor
- Dana-Farber Cancer Institute
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 5 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2014
- U.S. locations
- 3
- States / cities
- Boston, Massachusetts
- Conditions
- Sickle Cell Disease, Sickle Cell Disorder, Hemoglobinopathies, Thalassemia, Anemia, Sickle Cell
- Interventions
- Cyclophosphamide, Pentostatin, Rabbit anti-thymocyte globulin, Tacrolimus, Mycophenolate mofetil, CD4+ T-cell-depleted Haploidentical Hematopoietic Transplant
- Drug · Biological
- Lead sponsor
- City of Hope Medical Center
- Other
- Eligibility
- 18 Years to 45 Years
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2027
- U.S. locations
- 1
- States / cities
- Duarte, California
- Conditions
- Sickle Cell Disease, Thalassemia
- Interventions
- Etavopivat A, Etavopivat B, Etavopivat C
- Drug
- Lead sponsor
- Novo Nordisk A/S
- Industry
- Eligibility
- 2 Years and older
- Enrollment
- 480 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2030
- U.S. locations
- 36
- States / cities
- Birmingham, Alabama • Phoenix, Arizona • Los Angeles, California + 27 more