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Showing 169–192 of 322 matching trials from the live ClinicalTrials.gov search.
Conditions
Hemophilia A
Interventions
recombinant porcine coagulation factor VIII (OBI-1)
Drug
Lead sponsor
Octagen Corporation
Industry
Eligibility
12 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2007
U.S. locations
1
States / cities
Blue Bell, Pennsylvania
Completed No phase listed Observational

GAS-Hem Feasibility Study

NCT02674997
Conditions
Hemophilia A
Interventions
Factor VIII
Biological
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
5 Years to 65 Years
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016
U.S. locations
2
States / cities
Peoria, Illinois • Portland, Oregon
Conditions
Severe Hemophilia A
Interventions
VWF/FVIII concentrates
Drug
Lead sponsor
City of Hope Medical Center
Other
Eligibility
Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2020
U.S. locations
1
States / cities
Duarte, California
Conditions
Hemophilia A
Interventions
Emicizumab, rFVIIa, aPCC
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
12 Years and older
Enrollment
113 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2020
U.S. locations
11
States / cities
Los Angeles, California • Santa Monica, California • Aurora, Colorado + 8 more
Conditions
Acquired Hemophilia A
Interventions
OBI-1
Biological
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
18 Years and older
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
8
States / cities
Indianapolis, Indiana • New Orleans, Louisiana • Bethesda, Maryland + 4 more
Conditions
Hemophilia, Hemophilia A
Interventions
Not listed
Lead sponsor
Boston VA Research Institute, Inc.
Other
Eligibility
Male only
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2021
U.S. locations
1
States / cities
Boston, Massachusetts
Conditions
Hemophilia A
Interventions
rFVIIIFc
Biological
Lead sponsor
Bioverativ, a Sanofi company
Industry
Eligibility
Up to 5 Years · Male only
Enrollment
108 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
20
States / cities
Little Rock, Arkansas • Duarte, California • Sacramento, California + 16 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
Hemophilia, AIDS
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
4 Years to 100 Years
Enrollment
333 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2020
U.S. locations
15
States / cities
La Jolla, California • Los Angeles, California • Indianapolis, Indiana + 11 more
Conditions
Congenital Bleeding Disorder, Haemophilia A
Interventions
turoctocog alfa pegol
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
12 Years and older · Male only
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2018
U.S. locations
8
States / cities
Orlando, Florida • Iowa City, Iowa • East Lansing, Michigan + 5 more
Completed Phase 4 Interventional Results available

Prophylaxis Regimen for Hemophilia A Patients

NCT05036278
Conditions
Hemophilia A, Prophylaxis of Bleeding
Interventions
Damoctocog alfa-pegol is a recombinant B-domain deleted human coagulation FVIII variant site specifically conjugated with a 60 kDa, branched (30 kDa each) polyethylene glycol (PEG).
Biological
Lead sponsor
Bayer
Industry
Eligibility
12 Years and older
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
12
States / cities
Loma Linda, California • Los Angeles, California • Aurora, Colorado + 8 more
Conditions
Congenital Bleeding Disorder, Haemophilia A, Haemophilia B
Interventions
eptacog alfa (activated), vatreptacog alfa (activated)
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
12 Years and older · Male only
Enrollment
51 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2010
U.S. locations
12
States / cities
Los Angeles, California • Augusta, Georgia • Chicago, Illinois + 9 more
Conditions
Hematologic Disease, Blood Coagulation Disorders
Interventions
Recombinant Factor VIII (Kogenate, BAY 14-2222)
Behavioral
Lead sponsor
Bayer
Industry
Eligibility
14 Years to 29 Years · Male only
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2010
U.S. locations
7
States / cities
Duarte, California • Denver, Colorado • Peoria, Illinois + 4 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
Completed Phase 3 Interventional Results available

BAX 855 PK-guided Dosing

NCT02585960
Conditions
Hemophilia A
Interventions
PEGylated Recombinant Factor VIII
Biological
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
12 Years to 65 Years
Enrollment
135 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
14
States / cities
Phoenix, Arizona • Tucson, Arizona • Los Angeles, California + 11 more
Conditions
Joint Bleed, Hemophilia A
Interventions
handheld ultrasound: Philips Lumify integrated tele-ultrasound powered by Reacts collaborative platform
Device
Lead sponsor
Washington Institute for Coagulation
Other
Eligibility
18 Years and older · Male only
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
1
States / cities
Seattle, Washington
Conditions
Hemophilia A
Interventions
GreenGene™ F
Biological
Lead sponsor
Green Cross Corporation
Industry
Eligibility
Not listed
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
2
States / cities
Little Rock, Arkansas • New Hyde Park, New York
Conditions
Haemophilia A and B With and Without Inhibitors
Interventions
Concizumab
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
Not listed
Enrollment
153 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2029
U.S. locations
19
States / cities
San Diego, California • Aurora, Colorado • Orlando, Florida + 14 more
Conditions
Hemophilia B
Interventions
Gene Transfer, scAAV2/8-LP1-hFIXco
Genetic · Drug
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
18 Years and older · Male only
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2026
U.S. locations
3
States / cities
Stanford, California • Memphis, Tennessee • Temple, Texas
Conditions
Hemophilia A
Interventions
BAY2599023 (DTX201)
Drug
Lead sponsor
Bayer
Industry
Eligibility
18 Years and older · Male only
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2026
U.S. locations
3
States / cities
Little Rock, Arkansas • Ann Arbor, Michigan • Madison, Wisconsin
Conditions
Hemophilia A
Interventions
NXT007, Human Coagulation Factor VIII
Combination Product · Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
12 Years and older
Enrollment
138 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
2
States / cities
Aurora, Colorado • Iowa City, Iowa
Conditions
Hemophilia a
Interventions
Case scenarios - questionnaire, Case scenarios - Focus groups, Use scenarios - Usability test, Use scenarios - interview
Other
Lead sponsor
Enzyre B.V.
Industry
Eligibility
12 Years and older
Enrollment
74 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024
U.S. locations
1
States / cities
Indianapolis, Indiana
Conditions
Hemophilia A
Interventions
Emicizumab
Drug
Lead sponsor
Newark Beth Israel Medical Center
Other
Eligibility
4 Years and older
Enrollment
50 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2025
U.S. locations
1
States / cities
Newark, New Jersey
Conditions
Hemophilia A
Interventions
Auto CD34+PBSC, transduced with a lentiviral vector encoding the B domain deleted from of human coagulation factor VIII
Biological
Lead sponsor
Medical College of Wisconsin
Other
Eligibility
18 Years and older · Male only
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2033
U.S. locations
1
States / cities
Milwaukee, Wisconsin