- 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
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 697–720
of 2,014
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Dyskeratosis Congenita, Aplastic Anemia
- Interventions
- Alemtuzumab, Fludarabine, Cyclophosphamide, Total Body Irradiation, Stem Cell Transplant, Anti-thymocyte globulin
- Drug · Radiation · Biological
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Eligibility
- 0 Years to 70 Years
- Enrollment
- 61 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2025
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Congenital Bleeding Disorder, Haemophilia A
- Interventions
- turoctocog alfa pegol
- Drug
- Lead sponsor
- Novo Nordisk A/S
- Industry
- Eligibility
- 18 Years to 65 Years · Male only
- Enrollment
- 27 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2011
- U.S. locations
- 5
- States / cities
- Iowa City, Iowa • Lexington, Kentucky • Baltimore, Maryland + 2 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
- Severe Hemophilia A, Moderate Hemophilia A
- Interventions
- Emicizumab
- Drug
- Lead sponsor
- Hoffmann-La Roche
- Industry
- Eligibility
- 13 Years to 69 Years
- Enrollment
- 136 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2027
- U.S. locations
- 3
- States / cities
- Los Angeles, California • Miami, Florida • Oklahoma City, Oklahoma
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Myelodysplastic/Myeloproliferative Diseases
- Interventions
- clofarabine, cyclophosphamide
- Drug
- Lead sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
- Other
- Eligibility
- 2 Years and older
- Enrollment
- 70 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 2005
- U.S. locations
- 1
- States / cities
- Baltimore, Maryland
- Conditions
- Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions
- Interventions
- data collection
- Drug
- Lead sponsor
- Paul Szabolcs
- Other
- Eligibility
- 2 Months to 60 Years
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2028
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania
- Conditions
- Von Willebrand Disease
- Interventions
- Vonicog alfa, Antihemophilic Factor (Recombinant)
- Biological
- Lead sponsor
- Baxalta now part of Shire
- Industry
- Eligibility
- Up to 17 Years
- Enrollment
- 47 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2026
- U.S. locations
- 16
- States / cities
- Aurora, Colorado • Washington D.C., District of Columbia • Jacksonville, Florida + 12 more
- Conditions
- Sickle Cell Disease
- Interventions
- Isoquercetin, Placebo
- Drug
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
- NIH
- Eligibility
- 18 Years to 70 Years
- Enrollment
- 46 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2022
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Fanconi Anemia
- Interventions
- Cyclophosphamide, Fludarabine, Total Body Irradiation, Bone Marrow Transplantation, Mycophenolate Mofetil, Sirolimus
- Drug · Procedure
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Other
- Eligibility
- Not listed
- Enrollment
- 83 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2020
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Protein C Deficiency
- Interventions
- Protein C Concentrate (Human) Vapor Heated
- Drug
- Lead sponsor
- Baxalta now part of Shire
- Industry
- Eligibility
- Not listed
- Enrollment
- 11 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005
- U.S. locations
- 10
- States / cities
- Los Angeles, California • Denver, Colorado • Fort Carson, Colorado + 7 more
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Interventions
- fenretinide
- Drug
- Lead sponsor
- California Cancer Consortium
- Network
- Eligibility
- 18 Years to 120 Years
- Enrollment
- 29 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2017
- U.S. locations
- 4
- States / cities
- Los Angeles, California • Bethesda, Maryland • Houston, Texas + 1 more
- Conditions
- Hemophilia A, Hemophilia B
- Interventions
- Counseling, Control
- Behavioral
- Lead sponsor
- New York Presbyterian Hospital
- Other
- Eligibility
- 12 Years to 20 Years · Male only
- Enrollment
- 14 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2008
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Myeloid Malignancy, Inherited Bone Marrow Failure Syndrome, Clonal Expansion, Cytopenia, Bone Marrow Failure Syndrome, Clonal Cytopenia of Undetermined Significance, Clonal Hematopoiesis of Indeterminate Potential, Hematologic Neoplasms, Hematopoietic and Lymphatic System Neoplasm, Hereditary Neoplastic Syndrome, Idiopathic Cytopenia of Undetermined Significance, Idiopathic Dysplasia of Uncertain Significance, Low Risk Myelodysplastic Syndrome
- Interventions
- Biospecimen Collection, Bone Marrow Biopsy, Punch Biopsy, Buccal Swab, Clinical Evaluation, Genetic Counseling, Quality-of-Life Assessment, Electronic Health Record Review
- Procedure · Other
- Lead sponsor
- Mayo Clinic
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 2,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2035
- U.S. locations
- 3
- States / cities
- Scottsdale, Arizona • Jacksonville, Florida • Rochester, Minnesota
- Conditions
- Hemophilia
- Interventions
- Fitusiran
- Drug
- Lead sponsor
- Genzyme, a Sanofi Company
- Industry
- Eligibility
- 12 Years and older · Male only
- Enrollment
- 281 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2026
- U.S. locations
- 10
- States / cities
- Phoenix, Arizona • Los Angeles, California • Orange, California + 7 more
- Conditions
- Von Willebrand Disease
- Interventions
- Neumega (Oprelvekin, Interleukin 11, IL-11)
- Drug
- Lead sponsor
- Margaret Ragni
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2012
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania
- Conditions
- Sickle Cell Disease
- Interventions
- Anumigilimab, Placebo
- Biological · Drug
- Lead sponsor
- CSL Behring
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 63 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2028
- U.S. locations
- 9
- States / cities
- Little Rock, Arkansas • Orange, California • Hollywood, Florida + 6 more
- Conditions
- Hemarthrosis, Hemophilia
- Interventions
- Intra articular PRP Injection
- Biological
- Lead sponsor
- University of Utah
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 14 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2025
- U.S. locations
- 1
- States / cities
- Salt Lake City, Utah
- Conditions
- Fetal Conditions, Maternal; Procedure, Pregnancy Related, Twin to Twin Transfusion Syndrome, In Utero Procedure Affecting Fetus or Newborn, Chorion; Abnormal, Twin Reversal Arterial Perfusion Syndrome, Twin Monochorionic Monoamniotic Placenta, Chorioangioma, Vasa Previa
- Interventions
- Karl Storz Curved Scope, Karl Storz Straight Scope
- Device
- Lead sponsor
- Boston Children's Hospital
- Other
- Eligibility
- 18 Years to 45 Years · Female only
- Enrollment
- 50 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2027
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Sickle Cell Disease, Stroke
- Interventions
- Hydroxyurea, Placebo
- Drug
- Lead sponsor
- Johns Hopkins University
- Other
- Eligibility
- 12 Months to 54 Months
- Enrollment
- 28 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2022
- U.S. locations
- 5
- States / cities
- Birmingham, Alabama • Baltimore, Maryland • Kansas City, Missouri + 2 more
- 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
- Von Willebrand Disease (VWD), Heavy Menstrual Bleeding (HMB)
- Interventions
- ALN-6400
- Drug
- Lead sponsor
- Alnylam Pharmaceuticals
- Industry
- Eligibility
- 16 Years to 45 Years · Female only
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2028
- U.S. locations
- 6
- States / cities
- Orange, California • San Diego, California • San Francisco, California + 3 more
- Conditions
- Hemophilia A, Hemophilia B
- Interventions
- Virtual Reality Distraction, Standard of Care Distraction
- Other
- Lead sponsor
- Nationwide Children's Hospital
- Other
- Eligibility
- 6 Years to 19 Years
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016
- U.S. locations
- 1
- States / cities
- Columbus, Ohio
- Conditions
- Iron Overload, Sickle Cell Disease, Other Anemias
- Interventions
- Deferiprone
- Drug
- Lead sponsor
- ApoPharma
- Industry
- Eligibility
- 3 Years and older
- Enrollment
- 134 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2019
- U.S. locations
- 5
- States / cities
- Oakland, California • Ann Arbor, Michigan • Detroit, Michigan + 2 more