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Showing 1–24 of 43 matching trials from the live ClinicalTrials.gov search.
Conditions
Congenital Bleeding Disorder, Haemophilia B
Interventions
nonacog beta pegol
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
0 Years to 12 Years · Male only
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2023
U.S. locations
26
States / cities
Los Angeles, California • Sacramento, California • Aurora, Colorado + 22 more
Conditions
Congenital Bleeding Disorder, Haemophilia A With Inhibitors, Haemophilia B With Inhibitors
Interventions
activated recombinant human factor VII, factor IX, factor VIII
Drug · Other
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
5 Years and older
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2004
U.S. locations
14
States / cities
Los Angeles, California • Chicago, Illinois • New Orleans, Louisiana + 9 more
Conditions
Congenital Bleeding Disorder, Haemophilia A With Inhibitors, Haemophilia B With Inhibitors
Interventions
activated recombinant human factor VII
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
2 Years and older · Male only
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2005
U.S. locations
13
States / cities
Berkeley, California • Los Angeles, California • Chicago, Illinois + 9 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
Terminated Phase 1Phase 2 Interventional Results available

A Factor IX Gene Therapy Study (FIX-GT)

NCT03369444
Conditions
Hemophilia B
Interventions
FLT180a
Biological
Lead sponsor
University College, London
Other
Eligibility
18 Years and older · Male only
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
1
States / cities
Memphis, Tennessee
Conditions
Hemophilia A, Hemophilia B
Interventions
ADVATE (Antihemophilic Factor [Recombinant]), RIXUBIS (Coagulation Factor IX [Recombinant])
Biological
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
13 Years to 65 Years
Enrollment
64 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
17
States / cities
Los Angeles, California • San Diego, California • Aurora, Colorado + 14 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
71 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2014
U.S. locations
13
States / cities
Los Angeles, California • San Francisco, California • Washington D.C., District of Columbia + 10 more
Conditions
Congenital Bleeding Disorder, Haemophilia A With Inhibitors, Haemophilia B With Inhibitors
Interventions
eptacog alfa (activated)
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
5 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011
U.S. locations
1
States / cities
Princeton, New Jersey
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
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
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
7
States / cities
Los Angeles, California • San Francisco, California • Washington D.C., District of Columbia + 4 more
Conditions
Haemophilia A Without Inhibitors, Haemophilia B Without Inhibitors
Interventions
Concizumab
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
12 Years and older · Male only
Enrollment
156 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2028
U.S. locations
12
States / cities
Los Angeles, California • Orange, California • Augusta, Georgia + 9 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
Congenital Bleeding Disorder, Haemophilia A With Inhibitors, Haemophilia B With Inhibitors
Interventions
eptacog alfa (activated), Feiba VH
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
Up to 20 Years · Male only
Enrollment
42 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2006
U.S. locations
20
States / cities
Little Rock, Arkansas • Berkeley, California • Los Angeles, California + 16 more
Completed Phase 2 Interventional Results available

A Gene Therapy Study for Hemophilia B

NCT02484092
Conditions
Hemophilia B
Interventions
SPK-9001
Biological
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
9
States / cities
Sacramento, California • Jackson, Mississippi • Madison, Mississippi + 2 more
Conditions
Hemophilia B
Interventions
Adeno-Associated Viral with Human Factor IX
Genetic
Lead sponsor
Avigen
Industry
Eligibility
18 Years and older · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2004
U.S. locations
3
States / cities
Palo Alto, California • Philadelphia, Pennsylvania • Pittsburgh, Pennsylvania
Conditions
Haemostasis, Congenital Bleeding Disorder, Haemophilia B
Interventions
No treatment given
Other
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
18 Years and older
Enrollment
449 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015
U.S. locations
1
States / cities
Plainsboro, New Jersey
Conditions
Congenital Bleeding Disorder, Haemophilia B
Interventions
N9-GP, ALPROLIX®
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
18 Years to 70 Years · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017
U.S. locations
7
States / cities
Phoenix, Arizona • Chicago, Illinois • Peoria, Illinois + 4 more
Conditions
Haemophilia A With Inhibitors, Haemophilia B With Inhibitors
Interventions
Concizumab
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
Male only
Enrollment
134 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2027
U.S. locations
7
States / cities
Orange, California • Atlanta, Georgia • Indianapolis, Indiana + 4 more
Conditions
Hematologic Disorder, Bleeding Disorder, Connective Tissue Disorder, Hemophilia, Thrombosis, Von Willebrand Diseases, Thrombophilia, Rare Bleeding Disorder, Platelet Disorder, Factor IX Deficiency, Factor VIII Deficiency, Thalassemia, Sickle Cell Disease
Interventions
Not listed
Lead sponsor
American Thrombosis and Hemostasis Network
Network
Eligibility
Not listed
Enrollment
3,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2035
U.S. locations
76
States / cities
Phoenix, Arizona • Little Rock, Arkansas • Los Angeles, California + 60 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
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
Liver Cirrhosis, Hemophilia, Hemophilia A, Hemophilia B, Liver Transplant Disorder, Liver Failure, Coagulation Factor Deficiency
Interventions
Not listed
Lead sponsor
Stanford University
Other
Eligibility
18 Years and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
Palo Alto, California
Conditions
Congenital Bleeding Disorder, Haemophilia A With Inhibitors, Haemophilia B With Inhibitors
Interventions
activated recombinant human factor VII, Feiba VH
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
Male only
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
1
States / cities
Plainsboro, New Jersey
Completed No phase listed Observational Results available

PROPACT: Retrospective Prophylaxis Patient Case Collection

NCT00882778
Conditions
Congenital Bleeding Disorder, Haemophilia A With Inhibitors, Haemophilia B With Inhibitors
Interventions
eptacog alfa (activated)
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
Male only
Enrollment
86 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
1
States / cities
Princeton, New Jersey