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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 B
Interventions: DTX101; Genetic
Lead sponsor: Ultragenyx Pharmaceutical Inc; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2017
U.S. locations: 6
States / cities: Little Rock, Arkansas • Los Angeles, California • Gainesville, Florida + 3 more

Conditions: Hemophilia B
Interventions: rFIXFc; Drug
Lead sponsor: Bioverativ Therapeutics Inc.; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2009
U.S. locations: 6
States / cities: Chicago, Illinois • Indianapolis, Indiana • Boston, Massachusetts + 3 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 B
Interventions: BeneFIX; Drug
Lead sponsor: Wyeth is now a wholly owned subsidiary of Pfizer; Industry
Eligibility: Up to 5 Years
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2002 – 2007
U.S. locations: 6
States / cities: Aurora, Colorado • Detroit, Michigan • New Brunswick, New Jersey + 3 more

Conditions: Hemophilia B
Interventions: IB1001; Biological
Lead sponsor: Cangene Corporation; Industry
Eligibility: 12 Years and older
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2015
U.S. locations: 1
States / cities: Chicago, Illinois

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 B, Hemophilia B, Moderately Severe or Severe
Interventions: BE-101; Drug
Lead sponsor: Be Biopharma; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 5 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2026
U.S. locations: 5
States / cities: Davis, California • Washington D.C., District of Columbia • Ann Arbor, Michigan + 2 more

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 B
Interventions: SB-FIX; Biological
Lead sponsor: Sangamo Therapeutics; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 1 participant
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2021
U.S. locations: 1
States / cities: Washington D.C., District of Columbia

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: 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: BAY2599023 (DTX201); Drug
Lead sponsor: Bayer; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 11 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2026
U.S. locations: 3
States / cities: Little Rock, Arkansas • Ann Arbor, Michigan • Madison, Wisconsin

Conditions: Severe Hemophilia B
Interventions: rFIXFc; Biological
Lead sponsor: Bioverativ Therapeutics Inc.; Industry
Eligibility: Male only
Enrollment: 120 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2017
U.S. locations: 10
States / cities: Phoenix, Arizona • Sacramento, California • Aurora, Colorado + 7 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 – 2025
U.S. locations: 1
States / cities: Research Triangle Park, North Carolina

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: 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: 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 B
Interventions: AAV5-hFIXco-Padua, Factor IX (FIX); Genetic · Biological
Lead sponsor: CSL Behring; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 67 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2025
U.S. locations: 19
States / cities: Phoenix, Arizona • Little Rock, Arkansas • Los Angeles, California + 14 more

Conditions: Hemophilia B
Interventions: CSL222 (AAV5-hFIXco-Padua); Genetic
Lead sponsor: CSL Behring; Industry
Eligibility: 18 Years and older
Enrollment: 35 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2032
U.S. locations: 3
States / cities: San Diego, California • Ann Arbor, Michigan • Pittsburgh, Pennsylvania

Conditions: Hemophilia A
Interventions: ReFacto AF; Biological
Lead sponsor: Wyeth is now a wholly owned subsidiary of Pfizer; Industry
Eligibility: 12 Years and older · Male only
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006 – 2008
U.S. locations: 8
States / cities: La Jolla, California • Detroit, Michigan • Chapel Hill, North Carolina + 4 more

Conditions: Hemophilia B
Interventions: Not listed
Lead sponsor: Ultragenyx Pharmaceutical Inc; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2021
U.S. locations: 3
States / cities: Little Rock, Arkansas • Gainesville, Florida • Ann Arbor, Michigan

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: AAV8-hFIX19; Biological
Lead sponsor: Spark Therapeutics, Inc.; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 4 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2016
U.S. locations: 2
States / cities: Philadelphia, Pennsylvania • Pittsburgh, Pennsylvania

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

Safety and Dose Finding Study of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B

Phase I/IIa Study of FIXFc in Hemophilia B Patients

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

Study Evaluating rFIX; BeneFIX in Severe Hemophilia B

Study of Recombinant Factor IX Product, IB1001, in Previously Treated Subjects With Hemophilia B

PF-06741086 Multiple Dose Study in Severe Hemophilia

BeCoMe-9: A Clinical Study of BE-101 for the Treatment of Adults With Moderately Severe or Severe Hemophilia B

Study Evaluating ReFacto® in Hemophilia A Undergoing Major Surgery

Ascending Dose Study of Genome Editing by Zinc Finger Nuclease Therapeutic SB-FIX in Subjects With Severe Hemophilia B

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

PF-06741086 Long-term Treatment in Severe Hemophilia

Study to Test the Safety and How Well Patients With Severe Hemophilia A Respond to Treatment With BAY 2599023 (DTX 201), a Drug Therapy That Delivers a Healthy Version of the Defective Factor VIII Gene Into the Nucleus of Liver Cells Using an Altered, Non-infectious Virus (AAV) as a "Shuttle"

Long-Term Safety and Efficacy of rFIXFc in the Prevention and Treatment of Bleeding Episodes in Previously Treated Participants With Hemophilia B

Early Check: Expanded Screening in Newborns

Safety of a New Type of Treatment Called Gene Transfer for the Treatment of Severe Hemophilia B

Study of a Gene Therapy Treatment for Hemophilia A

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

HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients

Efficacy and Safety of CSL222 (Etranacogene Dezaparvovec) Gene Therapy in Adults With Hemophilia B With Pretreatment Adeno-associated Virus Serotype 5 (AAV5) Neutralizing Antibodies (Nabs)

Study Evaluating B-Domain Deleted Recombinant Factor VIII (BDDrFVIII, ReFacto AF) in Patients With Hemophilia A Undergoing Elective Major Surgery

Long-Term Safety, Tolerability, and Efficacy of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B

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

Hemophilia B Gene Therapy With AAV8 Vector