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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: Not listed
Lead sponsor: Arkansas Children's Hospital Research Institute; Other
Eligibility: 30 Years to 50 Years · Male only
Enrollment: 50 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2032
U.S. locations: 1
States / cities: Little Rock, Arkansas

Conditions: Haemostasis, Haemophilia A
Interventions: Concizumab, Turoctocog alfa; Drug
Lead sponsor: Novo Nordisk A/S; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 36 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2020
U.S. locations: 5
States / cities: Los Angeles, California • Indianapolis, Indiana • Oklahoma City, Oklahoma + 2 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: 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
Interventions: Not listed
Lead sponsor: Victor J Marder, M.D.; Other
Eligibility: 2 Years and older · Male only
Enrollment: 55 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2014
U.S. locations: 1
States / cities: Detroit, Michigan

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: 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: Not listed
Lead sponsor: BioMarin Pharmaceutical; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 172 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2040
U.S. locations: 13
States / cities: Los Angeles, California • Sacramento, California • San Diego, California + 10 more

Conditions: Hemophilia A
Interventions: rFVIII-FS/pegylated liposomes (BAY79-4980), rFVIII-FS/WFI (BAY14-2222); Biological
Lead sponsor: Bayer; Industry
Eligibility: 12 Years to 70 Years · Male only
Enrollment: 143 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2010
U.S. locations: 20
States / cities: Orange, California • Sacramento, California • San Francisco, California + 17 more

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: Attention Deficit Disorder With Hyperactivity, Asthma, COPD, Breast Cancer, Coronary Artery Disease, Congestive Heart Failure, Diabetes, Glaucoma, Hemophilia, Hypertension, Hyperthyroidism, Hypothyroidism, Myasthenia Gravis, Osteoporosis, Osteopenia, Renal Failure, Renal Insufficiency, Sickle Cell Disease, Stroke
Interventions: MAPLE; Other
Lead sponsor: Brigham and Women's Hospital; Other
Eligibility: 18 Years and older
Enrollment: 140 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2017
U.S. locations: 1
States / cities: Boston, Massachusetts

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 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: 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: Congenital Bleeding Disorder, Haemophilia A
Interventions: turoctocog alfa pegol; Drug
Lead sponsor: Novo Nordisk A/S; Industry
Eligibility: 0 Years to 6 Years · Male only
Enrollment: 124 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2023
U.S. locations: 34
States / cities: Phoenix, Arizona • Long Beach, California • Los Angeles, California + 28 more

Conditions: Severe Hemophilia A
Interventions: Human-cl rhFVIII; Biological
Lead sponsor: Octapharma; Industry
Eligibility: Male only
Enrollment: 48 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2018
U.S. locations: 1
States / cities: Sacramento, California

Conditions: Hemophilia A
Interventions: Fitusiran (SAR439774), Clotting factor concentrates (CFC) or bypassing agents (BPA), Antithrombin concentrate (ATIIIC), Emicizumab; Drug · Biological
Lead sponsor: Sanofi; Industry
Eligibility: 12 Years and older · Male only
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2030
U.S. locations: 2
States / cities: Los Angeles, California • Flowood, Mississippi

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: Hematologic Disease, Hemophilia A
Interventions: Kogenate (BAY14-2222); Drug
Lead sponsor: Bayer; Industry
Eligibility: 30 Years to 45 Years · Male only
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006 – 2008
U.S. locations: 3
States / cities: Aurora, Colorado • Chapel Hill, North Carolina • Houston, Texas

Conditions: Hemophilia A
Interventions: BAX855; Biological
Lead sponsor: Baxalta now part of Shire; Industry
Eligibility: Up to 75 Years
Enrollment: 218 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2018
U.S. locations: 21
States / cities: Phoenix, Arizona • Aurora, Colorado • Gainesville, Florida + 18 more

Conditions: Hemophilia B
Interventions: PF-06838435/ fidanacogene elaparvovec; Biological
Lead sponsor: Pfizer; Industry
Eligibility: 18 Years to 65 Years · Male only
Enrollment: 51 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2031
U.S. locations: 17
States / cities: San Francisco, California • Aurora, Colorado • Indianapolis, Indiana + 3 more

Conditions: Hemophilia A
Interventions: rFVIII (BAY81-8973) on demand, rFVIII (BAY81-8973) prophylaxis low-dose, rFVIII (BAY81-8973) prophylaxis high-dose; Biological
Lead sponsor: Bayer; Industry
Eligibility: 12 Years to 65 Years · Male only
Enrollment: 80 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2012
U.S. locations: 2
States / cities: Dayton, Ohio • Houston, Texas

Conditions: Hemophilia A
Interventions: rFVIIIFc; Biological
Lead sponsor: Bioverativ, a Sanofi company; Industry
Eligibility: Up to 5 Years · Male only
Enrollment: 108 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2019
U.S. locations: 20
States / cities: Little Rock, Arkansas • Duarte, California • Sacramento, California + 16 more

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

A Prospective Assessment of Bone Health in Patients With Severe Hemophilia A on Factor VIII vs Factor Mimetic Prophylaxis (Efa Emi Bone Health Study)

A Trial Evaluating Efficacy and Safety of Prophylactic Administration of Concizumab in Patients With Severe Haemophilia A Without Inhibitors

Early Check: Expanded Screening in Newborns

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

Personalized Prediction of Tolerance and Immunogenicity in Hemophilia

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

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

A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401)

BAY79-4980 Compared to rFVIII-FS in Previously Treated Patients With Severe Hemophilia A

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

Study of an Intervention to Improve Problem List Accuracy and Use

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

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

ASC618 Gene Therapy in Hemophilia A Patients

Safety and Efficacy of Turoctocog Alfa Pegol (N8-GP) in Previously Untreated Patients With Haemophilia A

Study to Investigate Immunogenicity, Efficacy and Safety of Treatment With Human-cl rhFVIII

A Study to Test a Medicine (Fitusiran) for Preventing Bleeds in People With Severe Hemophilia Who Previously Received Preventive Treatment With Emicizumab

Study of a Gene Therapy Treatment for Hemophilia A

BAY14-2222 Prophylaxis and Joint Function Improvement (Adults)

BAX 855 Continuation

A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B

A Trial to Compare Prophylaxis Therapy to On-demand Therapy With a New Full Length Recombinant FVIII in Patients With Severe Hemophilia A

An Open Label Study to Determine the Safety and Efficacy of Replacement Factor VIII Protein (Known as rFVIIIFc) in Previously Untreated Males With Severe Hemophilia A