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Showing 49–72 of 117 matching trials from the live ClinicalTrials.gov search.
Conditions
Hemophilia B with Inhibitor, Hemophilia B
Interventions
SerpinPC
Drug
Lead sponsor
ApcinteX Ltd
Industry
Eligibility
12 Years to 65 Years · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
3
States / cities
Aurora, Colorado • Tampa, Florida • Greenville, North Carolina
Conditions
HIV Infections, Hemophilia A
Interventions
Indinavir sulfate, Lamivudine, Stavudine, Zidovudine, Zalcitabine, Didanosine
Drug
Lead sponsor
Merck Sharp & Dohme LLC
Industry
Eligibility
16 Years and older · Male only
Enrollment
55 participants
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
7
States / cities
San Francisco, California • Washington D.C., District of Columbia • Atlanta, Georgia + 4 more
Conditions
Hemophilia A
Interventions
OBI-1
Biological
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
6 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
1
States / cities
Indianapolis, Indiana
Conditions
Hemophilia B, Allergic Reactions
Interventions
Not listed
Lead sponsor
Wyeth is now a wholly owned subsidiary of Pfizer
Industry
Eligibility
Not listed
Enrollment
166 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2007
U.S. locations
15
States / cities
Phoenix, Arizona • Orange, California • Atlanta, Georgia + 12 more
Conditions
Congenital Bleeding Disorder, Haemophilia A, Haemophilia A With Inhibitors, Haemophilia B, Haemophilia B With Inhibitors
Interventions
No treatment given
Other
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
Male only
Enrollment
381 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
1
States / cities
Plainsboro, New Jersey
Conditions
Hemophilia
Interventions
Coagulation Factor VIIa (Recombinant)
Biological
Lead sponsor
Laboratoire français de Fractionnement et de Biotechnologies
Industry
Eligibility
6 Months to 75 Years · Male only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
1
States / cities
Dallas, Texas
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
Terminated Phase 3 Interventional Results available

BAX 802 in CHA With Inhibitors

NCT02895945
Conditions
Hemophilia A
Interventions
Antihemophilic Factor (Recombinant), Porcine Sequence (BAX 802)
Biological
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
12 Years to 75 Years · Male only
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2021
U.S. locations
3
States / cities
Peoria, Illinois • Winston-Salem, North Carolina • Cleveland, Ohio
Conditions
Hemophilia A, Hemophilia B
Interventions
fitusiran, factor concentrates
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
12 Years and older · Male only
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
8
States / cities
Little Rock, Arkansas • Gainesville, Florida • Tampa, Florida + 5 more
Completed Phase 2 Interventional Results available

Dose Confirmation Trial of AAV5-hFIXco-Padua

NCT03489291
Conditions
Hemophilia B
Interventions
AAV5-hFIXco-Padua (AMT-061)
Genetic
Lead sponsor
CSL Behring
Industry
Eligibility
18 Years and older · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
4
States / cities
Phoenix, Arizona • Sacramento, California • San Diego, California + 1 more
Conditions
Hemophilia a, Hemophilia B
Interventions
SerpinPC
Drug
Lead sponsor
ApcinteX Ltd
Industry
Eligibility
12 Years to 65 Years · Male only
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
10
States / cities
Aurora, Colorado • Tampa, Florida • Indianapolis, Indiana + 7 more
Conditions
Hemophilia A
Interventions
Moroctocog alfa (AF-CC)
Biological
Lead sponsor
Pfizer
Industry
Eligibility
6 Months to 15 Years · Male only
Enrollment
66 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2018
U.S. locations
7
States / cities
Cincinnati, Ohio • Portland, Oregon • Dallas, Texas + 1 more
Conditions
Congenital Bleeding Disorder, Haemophilia B
Interventions
nonacog beta pegol
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
13 Years to 70 Years · Male only
Enrollment
74 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
12
States / cities
Los Angeles, California • Jacksonville, Florida • Augusta, Georgia + 9 more
Completed Not applicable Interventional

Virtual Reality for Hemophilia

NCT03507582
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
Hemophilia A, Hemophilia B
Interventions
fitusiran, Bypassing agents
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
12 Years and older · Male only
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
12
States / cities
Phoenix, Arizona • Los Angeles, California • Orange, California + 9 more
Conditions
Hemophilia A, Hemophilia B
Interventions
Not listed
Lead sponsor
Dana-Farber Cancer Institute
Other
Eligibility
21 Years and older
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
1
States / cities
Boston, Massachusetts
Terminated Phase 1Phase 2 Interventional Results available

Phase 1/2 Dose Confirmation Study of FLT180a in Hemophilia B

NCT05164471
Conditions
Hemophilia B
Interventions
verbrinacogene setparvovec
Genetic
Lead sponsor
Spur Therapeutics
Industry
Eligibility
18 Years to 65 Years · Male only
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
3
States / cities
Los Angeles, California • Tampa, Florida • Ann Arbor, Michigan
Conditions
Congenital Bleeding Disorder, Haemophilia A With Inhibitors, Haemophilia B With Inhibitors
Interventions
activated recombinant human factor VII, long acting
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
12 Years to 65 Years · Male only
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2011
U.S. locations
7
States / cities
Little Rock, Arkansas • Los Angeles, California • Orange, California + 4 more
Conditions
Hemophilia A, Hemophilia B
Interventions
Hemophilia B standard half-life, Hemophilia B extended half-life, Hemophilia A standard half-life, Hemophilia A extended half-life
Drug
Lead sponsor
Pfizer
Industry
Eligibility
Not listed
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2018
U.S. locations
3
States / cities
Gainesville, Florida • Las Vegas, Nevada • Fort Worth, Texas
Conditions
Significant Bleeding Risk
Interventions
Octaplex, Beriplex P/N (Kcentra)
Drug
Lead sponsor
Octapharma
Industry
Eligibility
18 Years and older
Enrollment
208 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
22
States / cities
Aurora, Colorado • New Haven, Connecticut • Miami, Florida + 14 more
Conditions
Bone Mineral Density, Hemophilia A, Hemophilia B
Interventions
DXA scan + MRI
Other
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
25 Years and older · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2012
U.S. locations
1
States / cities
Los Angeles, California
Conditions
Haemophilia A, Haemophilia A With Inhibitors, Haemophilia B, Haemophilia B With Inhibitors
Interventions
No treatment given
Other
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
12 Years and older · Male only
Enrollment
231 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
29
States / cities
Los Angeles, California • Sacramento, California • Aurora, Colorado + 24 more
Conditions
Hemophilia A With Inhibitors, Hemophilia B With Inhibitors
Interventions
Coagulation FVIIa (Recombinant)
Biological
Lead sponsor
Laboratoire français de Fractionnement et de Biotechnologies
Industry
Eligibility
Up to 11 Years · Male only
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
3
States / cities
Aurora, Colorado • Oklahoma City, Oklahoma • Dallas, Texas
Conditions
Hemophilia A, Hemophilia B
Interventions
Testing of hepatic AAV Vector integration
Diagnostic Test
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older · Male only
Enrollment
173 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2040
U.S. locations
15
States / cities
Rancho Cordova, California • Sacramento, California • San Francisco, California + 6 more