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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: 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

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: 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: Factor VII Deficiency, Glanzmann Thrombasthenia, Hemophilia A With Inhibitor
Interventions: Coagulation Factor VIIa variant; Biological
Lead sponsor: Catalyst Biosciences; Industry
Eligibility: 12 Years and older
Enrollment: 19 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021
U.S. locations: 7
States / cities: Sacramento, California • San Francisco, California • Aurora, Colorado + 4 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

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

Conditions: Hemophilia, Hemophilia A, Hemophilia B, Hemophilia A With Inhibitor, Hemophilia B With Inhibitor, Haemophilia B Without Inhibitor, Haemophilia A Without Inhibitor, Haemophilia
Interventions: Not listed
Lead sponsor: American Thrombosis and Hemostasis Network; Network
Eligibility: 0 Years to 13 Years
Enrollment: 237 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2022
U.S. locations: 27
States / cities: Phoenix, Arizona • Madera, California • San Diego, California + 24 more

Conditions: Congenital Bleeding Disorder, Haemophilia A
Interventions: turoctocog alfa; Drug
Lead sponsor: Novo Nordisk A/S; Industry
Eligibility: 0 Years to 6 Years · Male only
Enrollment: 60 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2018
U.S. locations: 25
States / cities: Phoenix, Arizona • Long Beach, California • Sacramento, California + 20 more

Conditions: Haemophilia A, Haemophilia B
Interventions: No treatment given; Other
Lead sponsor: Novo Nordisk A/S; Industry
Eligibility: 1 Year to 21 Years · Male only
Enrollment: 563 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2019
U.S. locations: 22
States / cities: Los Angeles, California • Orange, California • San Diego, California + 19 more

Conditions: Congenital Bleeding Disorder, Haemophilia A
Interventions: turoctocog alfa pegol; Drug
Lead sponsor: Novo Nordisk A/S; Industry
Eligibility: 18 Years to 65 Years · Male only
Enrollment: 27 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2011
U.S. locations: 5
States / cities: Iowa City, Iowa • Lexington, Kentucky • Baltimore, Maryland + 2 more

Conditions: Congenital Bleeding Disorder, Haemophilia A
Interventions: turoctocog alfa; Drug
Lead sponsor: Novo Nordisk A/S; Industry
Eligibility: 12 Years to 65 Years · Male only
Enrollment: 150 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2011
U.S. locations: 18
States / cities: Little Rock, Arkansas • Long Beach, California • Tampa, Florida + 15 more

Conditions: Severe Hemophilia A
Interventions: Nuwiq; Drug
Lead sponsor: Octapharma; Industry
Eligibility: 12 Years and older · Male only
Enrollment: 28 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2026
U.S. locations: 1
States / cities: Dallas, Texas

Conditions: Congenital Bleeding Disorder, Haemophilia A
Interventions: turoctocog alfa; Drug
Lead sponsor: Novo Nordisk A/S; Industry
Eligibility: Male only
Enrollment: 69 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2020
U.S. locations: 6
States / cities: Mobile, Alabama • Torrance, California • Peoria, Illinois + 3 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: 186 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2018
U.S. locations: 31
States / cities: Phoenix, Arizona • Long Beach, California • Sacramento, California + 26 more

Conditions: Inherited Bleeding Disorders
Interventions: Power Doppler Sonography, MRI; Procedure
Lead sponsor: New York Presbyterian Hospital; Other
Eligibility: 1 Year to 60 Years
Enrollment: 43 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2009
U.S. locations: 1
States / cities: New York, New York

Conditions: Haemostasis, Haemophilia A
Interventions: Concizumab, Turoctocog alfa; Drug
Lead sponsor: Novo Nordisk A/S; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 36 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2020
U.S. locations: 5
States / cities: Los Angeles, California • Indianapolis, Indiana • Oklahoma City, Oklahoma + 2 more

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: Healthy Participants, Hemophilia A, Hemophilia B, Factor VII Deficiency
Interventions: SR604, Placebo; Drug
Lead sponsor: Equilibra Bioscience LLC; Industry
Eligibility: 18 Years to 60 Years
Enrollment: 31 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2024 – 2026
U.S. locations: 9
States / cities: Glendale, California • Los Angeles, California • Chicago, Illinois + 6 more

Conditions: Hemophilia A, Hemophilia A, Severe, Factor VIII (FVIII), FVIII Deficiency, Carrier of Hemophilia A, Inhibitors, Pregnancy, Maternal Blood Loss, Pregnancy Complications, Bleeding Disorder, Hemorrhage, Postpartum, Alloimmunization, X-Linked
Interventions: Not listed
Lead sponsor: University of Washington; Other
Eligibility: Not listed
Enrollment: 500 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2024 – 2029
U.S. locations: 1
States / cities: Seattle, Washington

Conditions: Congenital Bleeding Disorder, Haemophilia A With Inhibitors, Haemophilia B With Inhibitors
Interventions: No treatment given; Other
Lead sponsor: Novo Nordisk A/S; Industry
Eligibility: 12 Years and older · Male only
Enrollment: 19 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2016
U.S. locations: 5
States / cities: Tucson, Arizona • Tampa, Florida • Atlanta, Georgia + 2 more

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Study of Modified Recombinant Factor VIII (OBI-1) in Subjects With Acquired Hemophilia A

Early Check: Expanded Screening in Newborns

Host Genetic Factors Influencing HIV1 and HCV Viral Loads and AIDS Clinical Progression in a Hemophilia Cohort (HGDS-3)

Safety, Tolerability, and Pharmacokinetics Study of Turoctocog Alfa Pegol Injected Under the Skin in Patients With Haemophilia A

Haemophilia Patients With Inhibitors Being Treated for Acute Joint Bleeds

A Research Study on How Well Concizumab Works for You if You Have Haemophilia A or B With or Without Inhibitors

Study of Coagulation Factor VIIa Marzeptacog Alfa (Activated) in Subjects With Inherited Bleeding Disorders

Alterations in Coagulation Factor Levels in Patients With End Stage Liver Disease

Observational Patient Diary Study of Treatment Doses for Patients With Haemophilia With Inhibitors to Factors VIII and IX

PROPACT: Retrospective Prophylaxis Patient Case Collection

ATHN 8: Previously Untreated Patients (PUPs) Matter Study

Safety and Efficacy of Turoctocog Alfa in Prevention and Treatment of Bleeds in Previously Untreated Children With Haemophilia A

A Study of the Impact of Hemophilia and Its Treatment on Brain Development, Thinking and Behaviour in Children With Hemophilia

Safety and Pharmacokinetics of NNC 0129-0000-1003 in Subjects With Haemophilia A

Safety and Efficacy of Turoctocog Alfa in Haemophilia A Subjects

Nuwiq for Perioperative Management Of Patients With Haemophilia A on Emicizumab Regular Prophylaxis Study

Safety and Efficacy of Turoctocog Alfa During Long-Term Treatment of Severe and Moderately Severe Haemophilia A

Evaluation of Safety and Efficacy, Including Pharmacokinetics, of NNC 0129-0000-1003 When Administered for Treatment and Prophylaxis of Bleeding in Subjects With Haemophilia A

Study to Evaluate the Use of Ultrasound in the Diagnosis of Hemophilic Joint Disease

A Trial Evaluating Efficacy and Safety of Prophylactic Administration of Concizumab in Patients With Severe Haemophilia A Without Inhibitors

Effect of Indinavir Plus Two Other Anti-HIV Drugs on Blood Clotting in HIV-Positive Males With Hemophilia

A Study of Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of SR604 in Two Participants Groups (Part A: Healthy Participants, and Part B: Participants With Hemophilia A or Hemophilia B or Factor VII Deficiency)

Hemophilia A Research Program

Pharmacogenetic Testing of Saliva Samples From Patients With Five or More Exposure Days to rFVIIa Analogue in the Adept™2 Trial