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Conditions: Hemophilia A
Interventions: SPK-8011; Genetic
Lead sponsor: Spark Therapeutics, Inc.; Industry
Eligibility: 18 Years and older · Male only
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2035
U.S. locations: 27
States / cities: Phoenix, Arizona • Loma Linda, California • Los Angeles, California + 23 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
Interventions: Fitusiran; Drug
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 12 Years and older · Male only
Enrollment: 281 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2026
U.S. locations: 10
States / cities: Phoenix, Arizona • Los Angeles, California • Orange, California + 7 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 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: 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 A, Hemophilia B
Interventions: Not listed
Lead sponsor: Weill Medical College of Cornell University; Other
Eligibility: Female only
Enrollment: 200 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2011
U.S. locations: 1
States / cities: New York, New York

Conditions: Hemophilia A With Inhibitors, Hemophilia B With Inhibitors
Interventions: Coagulation Factor VIIa (Recombinant); Biological
Lead sponsor: rEVO Biologics; Industry
Eligibility: 12 Years to 75 Years · Male only
Enrollment: 27 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2015
U.S. locations: 5
States / cities: Los Angeles, California • Sacramento, California • Aurora, Colorado + 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: 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, Hemophilia B
Interventions: BAY86-6150, eptacog alfa [activated]; Drug
Lead sponsor: Bayer; Industry
Eligibility: 12 Years to 62 Years · Male only
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2014
U.S. locations: 2
States / cities: Sacramento, California • Chattanooga, Tennessee

Conditions: Hemophilia A, Hemophilia B
Interventions: Recombinant Factor VIIa BI (rFVIIa BI); Biological
Lead sponsor: Baxalta now part of Shire; Industry
Eligibility: 12 Years to 65 Years · Male only
Enrollment: 40 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2014
U.S. locations: 1
States / cities: Tampa, Florida

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 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: 14
States / cities: Rancho Cordova, California • Sacramento, California • San Francisco, California + 5 more

Conditions: Hemophilia A, Hemophilia B
Interventions: Fitusiran (SAR439774); Drug
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 34 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2023
U.S. locations: 2
States / cities: Ann Arbor, Michigan • Pittsburgh, Pennsylvania

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
Interventions: Fitusiran, Clotting factor concentrates (CFC) or bypassing agents (BPA), Antithrombin concentrate (ATIIIC); Drug · Biological
Lead sponsor: Sanofi; Industry
Eligibility: 1 Year to 11 Years · Male only
Enrollment: 85 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2031
U.S. locations: 5
States / cities: Los Angeles, California • Orange, California • Las Vegas, Nevada + 2 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: Hemophilia A, Hemophilia B
Interventions: MOD-5014; Drug
Lead sponsor: OPKO Health, Inc.; Industry
Eligibility: 18 Years to 65 Years · Male only
Enrollment: 27 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2018
U.S. locations: 11
States / cities: Duarte, California • Los Angeles, California • Sacramento, California + 7 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: 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: 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

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Study of a Gene Therapy Treatment for Hemophilia A

Early Check: Expanded Screening in Newborns

Long-term Safety and Efficacy Study of Fitusiran in Patients With Hemophilia A or B, With or Without Inhibitory Antibodies to Factor VIII or IX

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)

PF-06741086 Long-term Treatment in Severe Hemophilia

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

Females With Severe or Moderate Hemophilia A or B: an International Multi-center Study

Phase III Study of Coagulation FVIIa (Recombinant) in Congenital Hemophilia A or B Patients With Inhibitors

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

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

A Phase 2/ 3 Trial to Evaluate the Efficacy and Safety of BAY86-6150

Recombinant Factor VIIa BI (rFVIIa BI) Treatment of Acute Bleeding Episodes Per an On-demand Regimen

The Efficacy and Safety of SerpinPC in Participants with Severe Hemophilia a or Moderately Severe to Severe Hemophilia B

A Phase III Study on the Safety, Pharmacokinetics and Efficacy of Coagulation Factor VIIa

Safety and Effectiveness of Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec in Patients With Hemophilia A or B Respectively

An Open-label Extension Study of an Investigational Drug, Fitusiran, in Patients With Moderate or Severe Hemophilia A or B

Socialization of Adult Men With Congenital Hemophilia A or B

A Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12 Years With Hemophilia A or B

Safety and Efficacy of Activated Recombinant Human Factor VII in Haemophilia Patients With Inhibitors During and After Major Surgery

Efficacy of NovoSeven® in Bleeding Prophylaxis in Hemophilia

A Study of a Long-Acting r-Factor 7a (Factor VIIa) in Adult Men With Hemophilia A or B

PF-06741086 Multiple Dose Study in Severe Hemophilia

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

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