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Conditions: Hemophilia A
Interventions: Recombinant coagulation factor (rFVIIIFc); Biological
Lead sponsor: Swedish Orphan Biovitrum; Industry
Eligibility: Male only
Enrollment: 16 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2020
U.S. locations: 1
States / cities: Washington D.C., District of Columbia

Conditions: Hemophilia A
Interventions: FVIII concentrate; Drug
Lead sponsor: The University of Texas Health Science Center, Houston; Other
Eligibility: Not listed
Enrollment: 25 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2020
U.S. locations: 13
States / cities: Atlanta, Georgia • Indianapolis, Indiana • Lexington, Kentucky + 9 more

Conditions: Hemophilia A
Interventions: Recombinant Protein-Free Factor VIII (rAHF-PFM); Drug
Lead sponsor: Baxalta now part of Shire; Industry
Eligibility: 18 Years to 70 Years
Enrollment: 85 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006 – 2015
U.S. locations: 10
States / cities: Los Angeles, California • Washington D.C., District of Columbia • Chicago, Illinois + 7 more

Conditions: Hemophilia A
Interventions: Damoctocog alfa pegol (Jivi, BAY94-9027); Drug
Lead sponsor: Bayer; Industry
Eligibility: Not listed
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2024
U.S. locations: 2
States / cities: Orange, California • New Orleans, Louisiana

Conditions: Factor VIII Deficiency
Interventions: Efanesoctocog alfa; Drug
Lead sponsor: Sanofi; Industry
Eligibility: 12 Years and older
Enrollment: 35 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2027
U.S. locations: 4
States / cities: Los Angeles, California • Indianapolis, Indiana • Iowa City, Iowa + 1 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 A, Hemophilia B
Interventions: Virtual Reality Distraction, Standard of Care Distraction; Other
Lead sponsor: Nationwide Children's Hospital; Other
Eligibility: 6 Years to 19 Years
Enrollment: 25 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016
U.S. locations: 1
States / cities: Columbus, Ohio

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
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 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 A, Factor VIII (FVIII)
Interventions: Half-life factor VIII based replacement therapy, Non-FVIII based replacement therapy; Drug
Lead sponsor: University of Texas Southwestern Medical Center; Other
Eligibility: 6 Months and older
Enrollment: 60 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2033
U.S. locations: 1
States / cities: Dallas, Texas

Conditions: Hemophilia A
Interventions: Available Recombinant, and Human Factor VIII products incl. Kogenate FS (Recombinant Factor VIII, BAY14-2222); Drug
Lead sponsor: Bayer; Industry
Eligibility: 16 Years and older · Male only
Enrollment: 272 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2018
U.S. locations: 7
States / cities: Phoenix, Arizona • Aurora, Colorado • Washington D.C., District of Columbia + 4 more

Conditions: Haemophilia A With or Without Inhibitors
Interventions: Mim8; Drug
Lead sponsor: Novo Nordisk A/S; Industry
Eligibility: 1 Year to 11 Years
Enrollment: 70 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2024
U.S. locations: 9
States / cities: Los Angeles, California • Aurora, Colorado • Tampa, Florida + 6 more

Conditions: Hemophilia A
Interventions: Factor VIII; Biological
Lead sponsor: Baxalta now part of Shire; Industry
Eligibility: 5 Years to 65 Years
Enrollment: 44 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016
U.S. locations: 2
States / cities: Peoria, Illinois • Portland, Oregon

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: Hemophilia A
Interventions: Desmopressin; Drug
Lead sponsor: Emory University; Other
Eligibility: 18 Years to 60 Years · Female only
Enrollment: 2 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2018
U.S. locations: 2
States / cities: Atlanta, Georgia

Conditions: Hemophilia, Menstrual Flow Excessive
Interventions: Recombinant FVIII Fc fusion product Eloctate, Recombinant FIX Fc fusion product Alprolix, Patient-operated diagnostic device for anemia AnemoCheck.; Drug · Device
Lead sponsor: Emory University; Other
Eligibility: 14 Years to 55 Years · Female only
Enrollment: 3 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2019
U.S. locations: 1
States / cities: Atlanta, Georgia

Conditions: Von Willebrand Disease, Blood Coagulation Disorders, Blood Platelet Disorders, Hematologic Disease
Interventions: Blood coagulation Factor VIII and vWF, human; Drug
Lead sponsor: CSL Behring; Industry
Eligibility: Not listed
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2001 – 2006
U.S. locations: 1
States / cities: Milwaukee, Wisconsin

Conditions: Thrombosis
Interventions: Kcentra, Apixaban, Placebo; Drug
Lead sponsor: Thomas Jefferson University; Other
Eligibility: 18 Years to 55 Years
Enrollment: 12 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2014
U.S. locations: 1
States / cities: Philadelphia, Pennsylvania

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: Hemophilia A
Interventions: SPK-8011QQ; Drug
Lead sponsor: Hoffmann-La Roche; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 5 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2031
U.S. locations: 2
States / cities: Orange, California • Vallejo, California

Conditions: Hemophilia A
Interventions: ADYNOVI/ADYNOVATE; Biological
Lead sponsor: Baxalta now part of Shire; Industry
Eligibility: Not listed
Enrollment: 207 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2030
U.S. locations: 1
States / cities: Gainesville, Florida

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

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Study of rFVIIIFc for Immune Tolerance Induction (ITI) in Haemophilia A Patients With Inhibitors Who Have Failed Previous ITI Therapies

Hemophilia Inhibitor Previously Untreated Patient Study

Phase 3/4 Study of a Recombinant Protein-Free Factor VIII (rAHF-PFM): Comparison of Continuous Infusion Versus Intermittent Bolus Infusion in Hemophilia A Subjects Undergoing Major Orthopedic Surgery

Study to Learn More About the Physical Activity Level of Patients Suffering From Hemophilia A Treated With Damoctocog Alfa Pegol (LIFE ACTIVE Study)

Efanesoctocog Alfa Prophylaxis in Patients With Hemophilia A With Synovial Hypertrophy

Study Comparing Blood Levels of ReFacto and Advante in Hemophilia A

Virtual Reality for Hemophilia

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

Study of a Gene Therapy Treatment for Hemophilia A

Early Check: Expanded Screening in Newborns

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

TG-INSIGHT With Joint POCUS, Hemostatic Potential in Patients With Severe Hemophilia A on Novel Replacement and Substitution FVIII Therapies

Patient Functioning and Well-being, Economic, and Clinical Impact of Hemophilia A and Its Treatment

A Research Study Looking at Mim8 in Children With Haemophilia A With or Without Inhibitors

GAS-Hem Feasibility Study

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

Characterization of Laboratory Response to DDAVP in Adult Hemophilia A Carriers

Extended Half Life Factor (EHF) Products For Heavy Menstrual Bleeding in Hemophilia Carriers

Study of Safety and Efficacy of Antihemophilic Factor/Von Willebrand Factor Complex in Surgical Subjects With Von Willebrand Disease (vWD)

Reversibility of Apixaban Anticoagulation With the Four Factor Prothrombin Complex Concentrate Kcentra

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

A Gene Therapy Study of SPK-8011QQ in Adults With Severe or Moderately Severe Hemophilia A

A Long-term Study of ADYNOVI/ADYNOVATE in Participants With Haemophilia A

BAX 855 PK-guided Dosing