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Showing 1–24 of 60 matching trials from the live ClinicalTrials.gov search.
Conditions
Factor VIII Deficiency
Interventions
efanesoctocog alfa (BIVV001)
Biological
Lead sponsor
Bioverativ, a Sanofi company
Industry
Eligibility
12 Years and older
Enrollment
159 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
8
States / cities
Los Angeles, California • San Diego, California • Gainesville, Florida + 4 more
Conditions
Von Willebrand Disease
Interventions
VWF/FVIII products
Drug
Lead sponsor
Skane University Hospital
Other
Eligibility
Not listed
Enrollment
105 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2013
U.S. locations
2
States / cities
Chapel Hill, North Carolina • Milwaukee, Wisconsin
Conditions
Hemophilia A
Interventions
PLASMA DERIVED Factor VIII, Recombinant FVIII
Drug
Lead sponsor
Fondazione Angelo Bianchi Bonomi
Other
Eligibility
1 Minute to 6 Years · Male only
Enrollment
303 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2015
U.S. locations
10
States / cities
Duarte, California • Los Angeles, California • Aurora, Colorado + 7 more
Active, not recruiting No phase listed Observational Accepts healthy volunteers

Early Check: Expanded Screening in Newborns

NCT03655223
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
Von Willebrand Disease, Type 3
Interventions
Von Willebrand Factor Concentrates, Von Willebrand Factor Concentrates and Factor VIII Concentrates, Factor VIII Concentrates, Recombinant Activated Factor VII, Activated Prothrombin Complex Concentrate
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
2 Years and older
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
3
States / cities
Sacramento, California • Minneapolis, Minnesota • St Louis, Missouri
Conditions
Von Willebrand Disease (VWD), Von Willebrand Disease (VWD), Type 1, Von Willebrand Disease (VWD), Type 2, Von Willebrand Disease (VWD), Type 3, Von Willebrand Disease, Type 2A, Von Willebrand Disease, Type 2M, Von Willebrand Disease, Type 2N
Interventions
Clinical outcomes of patients with VWD, Type 1, Clinical outcomes of patients with VWD, Type 2A, Type 2M, Type 2N, or Type 3
Other
Lead sponsor
Hemab ApS
Industry
Eligibility
16 Years and older
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
13
States / cities
Phoenix, Arizona • Little Rock, Arkansas • Los Angeles, California + 10 more
Conditions
Von Willebrand Diseases
Interventions
Not listed
Lead sponsor
American Thrombosis and Hemostasis Network
Network
Eligibility
Not listed
Enrollment
108 participants
Timeline
2019 – 2026
U.S. locations
22
States / cities
Orange, California • Aurora, Colorado • Farmington, Connecticut + 19 more
Conditions
Von Willebrand Diseases
Interventions
recombinant von Willebrand factor, tranexamic acid
Drug
Lead sponsor
Margaret Ragni
Other
Eligibility
13 Years to 45 Years · Female only
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
19
States / cities
Gilbert, Arizona • Orange, California • San Francisco, California + 16 more
Conditions
Hemophilia A
Interventions
Advate (Low Dose), Advate (High Dose), BIVV001 (Low Dose), BIVV001 (High Dose)
Biological
Lead sponsor
Bioverativ, a Sanofi company
Industry
Eligibility
18 Years to 65 Years · Male only
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2018
U.S. locations
7
States / cities
Los Angeles, California • Indianapolis, Indiana • Iowa City, Iowa + 4 more
Conditions
Von Willebrand Disease
Interventions
recombinant interleukin-11
Drug
Lead sponsor
University of Pittsburgh
Other
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2007
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Not listed No phase listed Observational

Low VW Activity in Adolescent HMB

NCT02933411
Conditions
Von Willebrand Factor Deficiency
Interventions
Genetic Analysis, Medical Record Data Abstraction, Pictorial Blood Assessment Chart (PBAC) score, Complete Bleeding Symptom ISTH Bleeding Assessment Tool
Other
Lead sponsor
Baylor College of Medicine
Other
Eligibility
Up to 21 Years · Female only
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2025
U.S. locations
12
States / cities
Atlanta, Georgia • East Lansing, Michigan • Kansas City, Missouri + 9 more
Conditions
Alagille Syndrome, Pulmonary Artery Stenoses, Acquired Von Willebrand Disease
Interventions
Assessment for bleeding disorder with tailored post-operative care
Other
Lead sponsor
Stanford University
Other
Eligibility
Up to 17 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
Palo Alto, California
Conditions
Bleeding Disorder
Interventions
Recombinant von Willebrand Factor
Drug
Lead sponsor
University of Virginia
Other
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
1
States / cities
Charlottesville, Virginia
Conditions
Intracranial Embolism, Cerebral Thromboembolism, Carotid Stenosis
Interventions
ARC1779 Injection, Placebo (normal saline)
Drug
Lead sponsor
Archemix Corp.
Industry
Eligibility
18 Years to 80 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
2
States / cities
Hackensack, New Jersey • Houston, Texas
Conditions
Von Willebrand Disease
Interventions
Wilate
Drug
Lead sponsor
Octapharma
Industry
Eligibility
Up to 5 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
1
States / cities
New Orleans, Louisiana
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
Von Willebrand Disease
Interventions
Recombinant von Willebrand Factor (rVWF)
Biological
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
18 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
12
States / cities
Aurora, Colorado • Miami, Florida • Augusta, Georgia + 8 more
Conditions
Hemophilia A
Interventions
efanesoctocog alfa (BIVV001)
Drug
Lead sponsor
Bioverativ, a Sanofi company
Industry
Eligibility
Up to 11 Years · Male only
Enrollment
74 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
10
States / cities
Los Angeles, California • Gainesville, Florida • Atlanta, Georgia + 7 more
Conditions
Von Willebrand Disease (VWD)
Interventions
VGA039
Drug
Lead sponsor
Vega Therapeutics, Inc
Industry
Eligibility
12 Years to 75 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
14
States / cities
Little Rock, Arkansas • Los Angeles, California • Palo Alto, California + 10 more
Conditions
Von Willebrand Diseases, Postpartum Hemorrhage
Interventions
Recombinant Von Willebrand factor, Tranexamic Acid Injection [Cyklokapron]
Drug
Lead sponsor
Nicoletta C Machin
Other
Eligibility
18 Years and older · Female only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Completed No phase listed Observational

T-TAS® wS Method Comparison

NCT06710262
Conditions
Antiplatelet Therapy, Healthy Donors, Von Willebrand Disease (VWD)
Interventions
T-TAS PL Assay
Diagnostic Test
Lead sponsor
Hikari Dx, Inc.
Other
Eligibility
21 Years and older
Enrollment
115 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
2
States / cities
Jacksonville, Florida • Baltimore, Maryland
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
Prevent Bleeding in Major Surgery
Interventions
human VWF/FVIII concentrate
Biological
Lead sponsor
Octapharma
Industry
Eligibility
6 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
3
States / cities
Indianapolis, Indiana • Chapel Hill, North Carolina • Milwaukee, Wisconsin
Completed No phase listed Observational Accepts healthy volunteers

Biomarkers in Aortic Stenosis - B.A.S.S.

NCT01334801
Conditions
Aortic Stenosis, Disorder of Prosthetic Cardiac Valve, Aortic Valve Insufficiency, Mitral Valve Insufficiency, Hypertrophic Cardiomyopathy, Tricuspid Valve Insufficiency
Interventions
Blood Draw
Procedure
Lead sponsor
Mayo Clinic
Other
Eligibility
21 Years and older
Enrollment
378 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010 – 2020
U.S. locations
1
States / cities
Jacksonville, Florida