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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: Hemophilia A With Inhibitor, Hemophilia B With Inhibitor
Interventions: coagulation factor VIIa [recombinant]-jncw; Drug
Lead sponsor: American Thrombosis and Hemostasis Network; Network
Eligibility: 12 Years to 100 Years
Enrollment: 55 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2027
U.S. locations: 21
States / cities: Phoenix, Arizona • Little Rock, Arkansas • Los Angeles, California + 17 more

Conditions: Hemophilia A, Factor VIII Deficiency, Hemophilia, Hemophilia B, Factor IX Deficiency
Interventions: Peripheral Vein Blood draw; Procedure
Lead sponsor: Children's Mercy Hospital Kansas City; Other
Eligibility: 1 Year to 21 Years · Male only
Enrollment: 3 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2018
U.S. locations: 1
States / cities: Kansas City, Missouri

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: Not listed
Lead sponsor: Spark Therapeutics, Inc.; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 4 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2017
U.S. locations: 2
States / cities: Philadelphia, Pennsylvania • Pittsburgh, Pennsylvania

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: ReFacto, Advante; Drug
Lead sponsor: Wyeth is now a wholly owned subsidiary of Pfizer; Industry
Eligibility: 18 Years to 64 Years · Male only
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005
U.S. locations: 4
States / cities: Washington D.C., District of Columbia • Atlanta, Georgia • Iowa City, Iowa + 1 more

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: Hemophilia A, Hemophilia B, Hemarthroses
Interventions: ultrasound; Other
Lead sponsor: Northwell Health; Other
Eligibility: 6 Months to 18 Years · Male only
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2019
U.S. locations: 1
States / cities: New Hyde Park, New York

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: 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: 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
Interventions: rhFVIIa; Biological
Lead sponsor: rEVO Biologics; Industry
Eligibility: 18 Years to 75 Years · Male only
Enrollment: 15 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2013
U.S. locations: 2
States / cities: Sacramento, California • Chicago, Illinois

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: Congenital Bleeding Disorder, Haemophilia B
Interventions: nonacog beta pegol; Drug
Lead sponsor: Novo Nordisk A/S; Industry
Eligibility: 0 Years to 6 Years · Male only
Enrollment: 54 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2022
U.S. locations: 23
States / cities: Long Beach, California • Los Angeles, California • Gainesville, Florida + 20 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, 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

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: 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 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: 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: 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
Interventions: Fitusiran; Drug
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 1 Year to 11 Years · Male only
Enrollment: 32 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2026
U.S. locations: 4
States / cities: Los Angeles, California • Hackensack, New Jersey • Cleveland, Ohio + 1 more

Search by objective public record fields.

A Trial Comparing Nonacog Beta Pegol (N9-GP) and ALPROLIX® in Patients With Haemophilia B

SEVENFACT® for Bleeding Events in Hemophilia With Inhibitors

A Study Comparing Factor Level and Inhibitor Titer Testing Results Drawn From Central Venous Lines and Venipuncture

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

LTFU for Gene Transfer Subjects With Hemophilia B

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

Study Comparing Blood Levels of ReFacto and Advante in Hemophilia A

ATHN 8: Previously Untreated Patients (PUPs) Matter Study

Ultrasonography in Hemophilic Joint Disease and Serum Markers

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 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

Early Check: Expanded Screening in Newborns

A Phase I Safety, Pharmacokinetics and Pharmacodynamics Study of Recombinant Factor VIIa in Adult Patients With Hemophilia A or B

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

Safety and Efficacy of Nonacog Beta Pegol (N9-GP) in Previously Untreated Patients With Haemophilia B

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

Socialization of Adult Men With Congenital Hemophilia A or B

Study of Prophylaxis, ACtivity and Effectiveness (SPACE) in Hemophilia Patients Currently Treated With ADVATE or RIXUBIS

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)

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

Research Study to Look at How Well the Drug Concizumab Works in Your Body if You Have Haemophilia With Inhibitors

A Factor IX Gene Therapy Study (FIX-GT)

Fitusiran Prophylaxis in Male Pediatric Subjects Aged 1 to Less Than 12 Years With Hemophilia A or B