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Conditions: Hemophilia A
Interventions: ReFacto; Drug
Lead sponsor: Wyeth is now a wholly owned subsidiary of Pfizer; Industry
Eligibility: 6 Years and older · Male only
Enrollment: 15 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2003 – 2007
U.S. locations: 4
States / cities: Los Angeles, California • New Brunswick, New Jersey • Cincinnati, Ohio + 1 more

Conditions: Hemophilia A, Hemophilia B
Interventions: Non-treatment, seroprevalence; Other
Lead sponsor: Baxalta now part of Shire; Industry
Eligibility: 18 Years to 75 Years · Male only
Enrollment: 242 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2021
U.S. locations: 7
States / cities: Los Angeles, California • Aurora, Colorado • East Lansing, Michigan + 4 more

Conditions: Hemophilia A or B
Interventions: PF-06741086; Biological
Lead sponsor: Pfizer; Industry
Eligibility: 18 Years to 64 Years · Male only
Enrollment: 27 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2018
U.S. locations: 4
States / cities: Aurora, Colorado • Chicago, Illinois

Conditions: Hemophilia A With Inhibitor
Interventions: Eloctate ITI, Emicizumab; Drug
Lead sponsor: Margaret Ragni; Other
Eligibility: Male only
Enrollment: 1 participant
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2022
U.S. locations: 2
States / cities: Pittsburgh, Pennsylvania

Conditions: Severe Hemophilia A
Interventions: rF.VIII; Drug
Lead sponsor: University of Pittsburgh; Other
Eligibility: 18 Years and older · Male only
Enrollment: 4 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2013
U.S. locations: 6
States / cities: Washington D.C., District of Columbia • Philadelphia, Pennsylvania • Pittsburgh, Pennsylvania + 2 more

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

Conditions: Hemophilia B
Interventions: REGV131, LNP1265; Drug
Lead sponsor: Regeneron Pharmaceuticals; Industry
Eligibility: 2 Years and older · Male only
Enrollment: 130 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2047
U.S. locations: 16
States / cities: Phoenix, Arizona • Los Angeles, California • Sacramento, California + 11 more

Conditions: Hemophilia A
Interventions: Recombinant Factor VIII (Kogenate FS, BAY14-2222); Biological
Lead sponsor: Bayer; Industry
Eligibility: 12 Years to 50 Years · Male only
Enrollment: 84 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2013
U.S. locations: 28
States / cities: Tucson, Arizona • Little Rock, Arkansas • Orange, California + 23 more

Conditions: Hemophilia B, Hemophilia A
Interventions: Standard of Care FIX Replacement therapy, Standard of Care FVIII Replacement therapy; Drug
Lead sponsor: Pfizer; Industry
Eligibility: 18 Years to 64 Years · Male only
Enrollment: 212 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2024
U.S. locations: 13
States / cities: Palo Alto, California • San Francisco, California • Stanford, California + 7 more

Conditions: Congenital Bleeding Disorder, Haemophilia A
Interventions: Turoctocog alfa pegol; Drug
Lead sponsor: Novo Nordisk A/S; Industry
Eligibility: Male only
Enrollment: 160 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2020
U.S. locations: 18
States / cities: Phoenix, Arizona • Long Beach, California • Iowa City, Iowa + 15 more

Conditions: Hemophilia A
Interventions: Antihemophilic factor, recombinant, manufactured protein-free; Biological
Lead sponsor: Baxalta now part of Shire; Industry
Eligibility: 12 Years to 65 Years
Enrollment: 38 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006 – 2007
U.S. locations: 8
States / cities: Little Rock, Arkansas • Los Angeles, California • Peoria, Illinois + 5 more

Conditions: Hemophilia A, Hemophilia B
Interventions: ALN-AT3SC, Sterile Normal Saline (0.9% NaCl); Drug
Lead sponsor: Sanofi; Industry
Eligibility: 18 Years to 65 Years · Male only
Enrollment: 51 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2014 – 2017
U.S. locations: 1
States / cities: Pittsburgh, Pennsylvania

Conditions: Hemophilia A or B
Interventions: PF-06741086; Biological
Lead sponsor: Pfizer; Industry
Eligibility: 12 Years to 74 Years · Male only
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2020
U.S. locations: 1
States / cities: Aurora, Colorado

Conditions: Hemophilia A
Interventions: assessment of joint health and bone density; Other
Lead sponsor: Washington Institute for Coagulation; Other
Eligibility: 16 Years and older · Male only
Enrollment: 40 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2026
U.S. locations: 4
States / cities: Los Angeles, California • San Diego, California • San Antonio, Texas + 1 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 A, Hemophilia B
Interventions: Not listed
Lead sponsor: Weill Medical College of Cornell University; Other
Eligibility: 1 Month and older · Female only
Enrollment: 22 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2010
U.S. locations: 18
States / cities: Duarte, California • Aurora, Colorado • Washington D.C., District of Columbia + 15 more

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: Severe Haemophilia A
Interventions: Human cl rhFVIII; Biological
Lead sponsor: Octapharma; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 58 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2018
U.S. locations: 9
States / cities: Sacramento, California • Aurora, Colorado • Washington D.C., District of Columbia + 6 more

Conditions: Hemophilia
Interventions: Fitusiran, Clotting factor concentrates (CFC) or bypassing agents (BPA), Antithrombin concentrate (ATIIIC); Drug
Lead sponsor: Sanofi; Industry
Eligibility: 12 Years and older · Male only
Enrollment: 91 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2029
U.S. locations: 8
States / cities: Orange, California • Minneapolis, Minnesota • Hackensack, New Jersey + 5 more

Conditions: Hemophilia A
Interventions: ASC618; Biological
Lead sponsor: ASC Therapeutics; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 12 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2026
U.S. locations: 1
States / cities: Little Rock, Arkansas

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: Haemophilia A
Interventions: Recombinant Factor VIII (Kovaltry, BAY81-8973); Biological
Lead sponsor: Bayer; Industry
Eligibility: 0 Years to 12 Years · Male only
Enrollment: 94 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2020
U.S. locations: 4
States / cities: New Orleans, Louisiana • Cincinnati, Ohio • Cleveland, Ohio + 1 more

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
Interventions: Not listed
Lead sponsor: Boston Children's Hospital; Other
Eligibility: 2 Years to 18 Years · Male only
Enrollment: 36 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2020
U.S. locations: 1
States / cities: Boston, Massachusetts

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Study Evaluating ReFacto® in Hemophilia A Undergoing Major Surgery

Global Epidemiologic Study of Preexisting Immunity to AAV in Adults With Severe Hemophilia

PF-06741086 Multiple Dose Study in Severe Hemophilia

The Hemophilia Inhibitor Eradication Trial

Hemophilia Adult Prophylaxis Study: Factor VIII in Severe Hemophilia A

A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors

A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B

Trial to Evaluate the Effect of Secondary Prophylaxis With rFVIII Therapy in Severe Hemophilia A Adult and/or Adolescent Subjects Compared to That of Episodic Treatment

A Study to Evaluate Prospective Efficacy and Safety Data of Current FIX Prophylaxis Replacement Therapy in Adult Hemophilia B Subjects (FIX:C≤2%) or Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Subjects (FVIII:C≤1%)

A Research Study Looking at How a Factor VIII Medicine Called Turoctocog Alfa Pegol (N8-GP) Works in People With Haemophilia A

Dose-Response Study of Recombinant Factor VIII Manufactured Protein-Free (rAHF-PFM) in Patients With Hemophilia A

A Phase 1 Study of an Investigational Drug, ALN-AT3SC, in Healthy Volunteers and Hemophilia A or B Patients

PF-06741086 Long-term Treatment in Severe Hemophilia

Effects of Emicizumab vs. Factor VIII Prophylaxis on Joint and Bone Health in Severe Hemophilia A

Early Check: Expanded Screening in Newborns

Females With Severe or Moderate Hemophilia A or B: A Multi-Center Study

BAX 855 PK-guided Dosing

Assess the Efficacy and Safety of Personalized Prophylaxis Human-cl rhFVIII in Patients With Severe Haemophilia A

A Study to Test a Medicine (Fitusiran) Injected Under the Skin for Preventing Bleeding Episodes in Male Adolescent or Adult Participants With Severe Hemophilia

ASC618 Gene Therapy in Hemophilia A Patients

Health-Related Quality of Life Outcomes and Bleeding Rates Among Patients With Severe Hemophilia A on Emicizumab

BAY81-8973 Pediatric Safety and Efficacy Trial

A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients With Inhibitors

Platelet Function in Patients With Hemophilia A