- 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
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 457–480
of 857
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Congenital Hemophilia A
- Interventions
- rVIII-SingleChain
- Biological
- Lead sponsor
- CSL Behring
- Industry
- Eligibility
- Up to 11 Years · Male only
- Enrollment
- 84 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2015
- U.S. locations
- 2
- States / cities
- Aurora, Colorado • Chicago, Illinois
- Conditions
- Wiskott-Aldrich Syndrome (WAS), X-linked Thrombocytopenia
- Interventions
- Interleukin-2
- Drug
- Lead sponsor
- Soma Jyonouchi
- Other
- Eligibility
- 24 Months and older
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2016
- U.S. locations
- 3
- States / cities
- Philadelphia, Pennsylvania • Houston, Texas
- Conditions
- Hemophilia B
- Interventions
- DTX101
- Genetic
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Industry
- Eligibility
- 18 Years and older · Male only
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2017
- U.S. locations
- 6
- States / cities
- Little Rock, Arkansas • Los Angeles, California • Gainesville, Florida + 3 more
- Conditions
- Hemophilia A
- Interventions
- SPK-8011QQ
- Drug
- Lead sponsor
- Hoffmann-La Roche
- Industry
- Eligibility
- 18 Years and older · Male only
- Enrollment
- 5 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2032
- U.S. locations
- 6
- States / cities
- Orange, California • San Francisco, California • Vallejo, California + 3 more
- Conditions
- Hemophilia A or B
- Interventions
- PF-06741086
- Biological
- Lead sponsor
- Pfizer
- Industry
- Eligibility
- 18 Years to 64 Years · Male only
- Enrollment
- 27 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2018
- U.S. locations
- 4
- States / cities
- Aurora, Colorado • Chicago, Illinois
- Conditions
- Idiopathic Thrombocytopenic Purpura
- Interventions
- Eltrombopag, Placebo
- Drug
- Lead sponsor
- GlaxoSmithKline
- Industry
- Eligibility
- 1 Year to 17 Years
- Enrollment
- 92 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2014
- U.S. locations
- 4
- States / cities
- Los Angeles, California • St. Petersburg, Florida • Brooklyn, New York + 1 more
- Conditions
- Thrombotic Thrombocytopenic Purpura
- Interventions
- Octaplas, Standard Plasma
- Biological · Drug
- Lead sponsor
- Octapharma
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 1 participant
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017
- U.S. locations
- 2
- States / cities
- Manhasset, New York • Rochester, New York
- Conditions
- Myeloproliferative Neoplasms
- Interventions
- INCA033989, Ruxolitinib
- Drug
- Lead sponsor
- Incyte Corporation
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 290 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2028
- U.S. locations
- 13
- States / cities
- Duarte, California • Palo Alto, California • Miami, Florida + 9 more
- Conditions
- Hermansky-Pudlak Syndrome (HPS)
- Interventions
- Not listed
- Lead sponsor
- National Human Genome Research Institute (NHGRI)
- NIH
- Eligibility
- 1 Month to 115 Years
- Enrollment
- 600 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 1995
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Vasculitis, Behcet's Disease, CNS Vasculitis, Cryoglobulinemic Vasculitis, Eosinophilic Granulomatosis With Polyangiitis (EGPA), Churg-Strauss Syndrome (CSS), Granulomatosis With Polyangiitis (GPA), Wegener's Granulomatosis, IgA Vasculitis, Henoch-Schoenlein Purpura (HSP), Microscopic Polyangiitis (MPA), Polyarteritis Nodosa (PAN), Takayasu Arteritis (TAK), Urticarial Vasculitis, Systemic Vasculitis
- Interventions
- Online questionnaires
- Other
- Lead sponsor
- University of Pennsylvania
- Other
- Eligibility
- 18 Years to 50 Years · Female only
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2027
- U.S. locations
- 1
- States / cities
- Tampa, Florida
- Conditions
- Hemophilia B
- Interventions
- Non-Interventional
- Other
- Lead sponsor
- Regeneron Pharmaceuticals
- Industry
- Eligibility
- 16 Years and older · Male only
- Enrollment
- 11 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2026
- U.S. locations
- 9
- States / cities
- Aurora, Colorado • New Haven, Connecticut • Washington D.C., District of Columbia + 6 more
- Conditions
- HIV Infections, Hemophilia A
- Interventions
- Zidovudine
- Drug
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
- NIH
- Eligibility
- 12 Years and older
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Ends 1989
- U.S. locations
- 2
- States / cities
- Buffalo, New York • Rochester, New York
- Conditions
- Hemophilia A, Hemophilia B
- Interventions
- rFVIIIFc, rFIXFc, non-Fc FVIII replacement products, non-Fc FIX replacement products
- Biological · Drug
- Lead sponsor
- Bioverativ Therapeutics Inc.
- Industry
- Eligibility
- Male only
- Enrollment
- 3 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015
- U.S. locations
- 2
- States / cities
- Los Angeles, California • East Lansing, Michigan
- Conditions
- Thrombotic Thrombocytopenic Purpura
- Interventions
- Rituximab
- Drug
- Lead sponsor
- Weill Medical College of Cornell University
- Other
- Eligibility
- 17 Years and older
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2009
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Brain and Central Nervous System Tumors, Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Lymphoproliferative Disorder, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms, Pain, Precancerous Condition, Unspecified Adult Solid Tumor, Protocol Specific
- Interventions
- fentanyl citrate, pharmacological study
- Drug · Other
- Lead sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 2 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2009
- U.S. locations
- 1
- States / cities
- Baltimore, Maryland
- Conditions
- Polycythemia Vera
- Interventions
- P1101 (Ropeginterferon alfa-2b-njft), Ropeginterferon alfa-2b-njft (P1101)
- Drug
- Lead sponsor
- PharmaEssentia
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 111 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2027
- U.S. locations
- 17
- States / cities
- Jacksonville, Florida • Fort Wayne, Indiana • Westwood, Kansas + 14 more
- Conditions
- Hemophilia A
- Interventions
- Not listed
- Lead sponsor
- Octapharma
- Industry
- Eligibility
- Male only
- Enrollment
- 80 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2020
- U.S. locations
- 5
- States / cities
- Gainesville, Florida • Miami, Florida • New Orleans, Louisiana + 2 more
- Conditions
- Immune Thrombocytopenia
- Interventions
- Avatrombopag, Placebo
- Drug
- Lead sponsor
- Sobi, Inc.
- Industry
- Eligibility
- 1 Year to 17 Years
- Enrollment
- 75 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2025
- U.S. locations
- 21
- States / cities
- Phoenix, Arizona • Long Beach, California • Orange, California + 18 more
- Conditions
- Primary Myelofibrosis, Post-polycythemia Vera Myelofibrosis, Post-essential Thrombocythemia Myelofibrosis
- Interventions
- Pacritinib, Best Available Therapy
- Drug
- Lead sponsor
- CTI BioPharma
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 327 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2016
- U.S. locations
- 4
- States / cities
- Scottsdale, Arizona • Omaha, Nebraska • Morristown, New Jersey + 1 more
- Conditions
- Hemophilia, Hemorrhage, Facelift Surgery
- Interventions
- 1%Tranexamic acid with standard local, 3% TXA, 1% TXA with local plus 3% TXA-soaked pledgets
- Drug
- Lead sponsor
- University of Minnesota
- Other
- Eligibility
- 18 Years and older
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2026
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- Conditions
- Immune Thrombocytopenic Purpura
- Interventions
- Fostamatinib Disodium
- Drug
- Lead sponsor
- Rigel Pharmaceuticals
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 123 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2020
- U.S. locations
- 9
- States / cities
- Tucson, Arizona • Peoria, Illinois • Lafayette, Indiana + 5 more
- Conditions
- Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia, Asparagine Synthetase Deficiency, CHARGE Syndrome, Early Infantile Epileptic Encephalopathy, FOXG1 Syndrome, KBG Syndrome, Noonan Syndrome, Severe Hemophilia A, Short Bowel Syndrome, Beta-Propeller Protein-Associated Neurodegeneration, Brain Injury of Prematurity With Periventricular Leukomalacia, Chromosome 17p13.3 Microdeletion Syndrome, Chromosome 1q43-1q44 Deletion, Cockayne Syndrome, Congenital Diaphragmatic Hernia, End-Stage Renal Disease With Cloacal Anomaly, Mitochondrial Depletion Disorder, Severe Factor VII Deficiency
- Interventions
- Family Centered pediatric palliative care for family caregivers of children with rare diseases.
- Behavioral
- Lead sponsor
- Children's National Research Institute
- Other
- Eligibility
- 12 Months to 99 Years
- Enrollment
- 480 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2025 – 2029
- U.S. locations
- 1
- States / cities
- Washington D.C., District of Columbia
- Conditions
- Haemophilia A, Haemophilia B, Obesity
- Interventions
- No treatment given
- Other
- Lead sponsor
- Novo Nordisk A/S
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 317 participants
- Timeline
- 2017 – 2018
- U.S. locations
- 1
- States / cities
- Plainsboro, New Jersey