- 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
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- Conditions
- Heart Failure, Respiratory Failure
- Interventions
- Not listed
- Lead sponsor
- University of California, San Francisco
- Other
- Eligibility
- 14 Days to 18 Years
- Enrollment
- 304 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2016
- U.S. locations
- 25
- States / cities
- Los Angeles, California • Oakland, California • Orange, California + 19 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
- Acquired Bleeding Disorder, Cardiac Surgery Requiring Cardiopulmonary Bypass
- Interventions
- catridecacog, placebo
- Drug
- Lead sponsor
- Novo Nordisk A/S
- Industry
- Eligibility
- 35 Years to 70 Years
- Enrollment
- 43 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2008
- U.S. locations
- 15
- States / cities
- Tucson, Arizona • San Francisco, California • Denver, Colorado + 10 more
- Conditions
- Lower Gastrointestinal Bleeding, Hemostatic Disorder
- Interventions
- Pure-Vu Cleansing Device
- Device
- Lead sponsor
- The Cleveland Clinic
- Other
- Eligibility
- 22 Years and older
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2022
- U.S. locations
- 1
- States / cities
- Cleveland, Ohio
- 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
- Embolism and Thrombosis, Embolism, Vascular Diseases, Warfarin, Venous Thromboembolism, Thrombosis, Thromboembolism
- Interventions
- CYP 2C9 and VKORC1 Testing for Warfarin
- Other
- Lead sponsor
- Medco Health Solutions, Inc.
- Industry
- Eligibility
- 40 Years to 75 Years
- Enrollment
- 1,635 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2010
- U.S. locations
- 1
- States / cities
- Franklin Lakes, New Jersey
- Conditions
- Albinism, Inborn Errors of Metabolism, Oculocutaneous Albinism, Platelet Storage Pool Deficiency, Pulmonary Fibrosis
- Interventions
- Pirfenidone, Placebo
- Drug
- Lead sponsor
- William Gahl, M.D.
- NIH
- Eligibility
- 18 Years and older
- Enrollment
- 35 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2016
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Hypofibrinogenemia, Bleeding
- Interventions
- Traditional Cryoprecipitate, Pathogen-Reduced Cryoprecipitate
- Biological
- Lead sponsor
- Weill Medical College of Cornell University
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 208 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2023 – 2025
- U.S. locations
- 1
- States / cities
- New York, New York
- 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
- Coagulation
- Interventions
- Whole Blood Clotting Time
- Device
- Lead sponsor
- Perosphere Technologies Inc.
- Industry
- Eligibility
- 18 Years to 80 Years
- Enrollment
- 150 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2027
- U.S. locations
- 4
- States / cities
- Danbury, Connecticut • Rockville, Maryland • Denville, New Jersey + 1 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
- Brain and Central Nervous System Tumors, Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Lymphoproliferative Disorder, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms, Unspecified Adult Solid Tumor, Protocol Specific
- Interventions
- 3'-deoxy-3'-[18F]fluorothymidine
- Device
- Lead sponsor
- Barbara Ann Karmanos Cancer Institute
- Other
- Eligibility
- 18 Years to 120 Years
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2028
- U.S. locations
- 1
- States / cities
- Detroit, Michigan
- Conditions
- Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(15;17)(q22;q12), Adult Acute Myeloid Leukemia With t(16;16)(p13;q22), Adult Acute Myeloid Leukemia With t(8;21)(q22;q22), B-cell Adult Acute Lymphoblastic Leukemia, B-cell Childhood Acute Lymphoblastic Leukemia, Childhood Chronic Myelogenous Leukemia, Childhood Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia, Essential Thrombocythemia, Polycythemia Vera, Previously Treated Myelodysplastic Syndromes, Recurrent Adult Acute Lymphoblastic Leukemia, Recurrent Adult Acute Myeloid Leukemia, Recurrent Childhood Acute Lymphoblastic Leukemia, Recurrent Childhood Acute Myeloid Leukemia, Refractory Anemia With Excess Blasts, Refractory Anemia With Excess Blasts in Transformation, Relapsing Chronic Myelogenous Leukemia, Secondary Acute Myeloid Leukemia, T-cell Adult Acute Lymphoblastic Leukemia, T-cell Childhood Acute Lymphoblastic Leukemia
- Interventions
- therapeutic allogeneic lymphocytes, aldesleukin, peripheral blood stem cell transplantation, allogeneic bone marrow transplantation, laboratory biomarker analysis, gene expression analysis, immunologic technique, flow cytometry, polymerase chain reaction, cytogenetic analysis, staining method
- Biological · Procedure · Other + 1 more
- Lead sponsor
- Fred Hutchinson Cancer Center
- Other
- Eligibility
- Not listed
- Enrollment
- 37 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2002 – 2013
- U.S. locations
- 1
- States / cities
- Seattle, Washington
- 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
- Giant Cell Arteritis, Takayasu's Arteritis, Polyarteritis Nodosa, Wegener's Granulomatosis, Microscopic Polyangiitis, Churg-Strauss Syndrome, Behcet's Disease, Kawasaki Disease, Henoch-schoenlein Purpura, Vasculitis, Central Nervous System, Drug-induced Necrotizing Vasculitis
- Interventions
- Not listed
- Lead sponsor
- University of South Florida
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 467 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2012
- U.S. locations
- 1
- States / cities
- Tampa, Florida
- Conditions
- Blood Coagulation Disorders, Trauma, Acute Coagulopathy
- Interventions
- Blood collection
- Other
- Lead sponsor
- University of Rochester
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 2 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2014
- U.S. locations
- 1
- States / cities
- Rochester, New York
- Conditions
- Hemostasis, General Surgery
- Interventions
- sFilm-FS, TACHOSIL®
- Combination Product
- Lead sponsor
- Sealantium Medical Ltd.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 33 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2023
- U.S. locations
- 1
- States / cities
- St Louis, Missouri
- 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
- Coagulopathy
- Interventions
- Correlation of the Quantra with the TEG
- Device
- Lead sponsor
- Stanford University
- Other
- Eligibility
- Up to 8 Years
- Enrollment
- 40 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2023
- U.S. locations
- 1
- States / cities
- Palo Alto, California
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Multiple Myeloma and Malignant Plasma Cell Neoplasms, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms
- Interventions
- busulfan, Cyclophosphamide, VP-16, Fractionated Total Body Irradiation (FTBI)
- Drug · Radiation
- Lead sponsor
- H. Lee Moffitt Cancer Center and Research Institute
- Other
- Eligibility
- 15 Years to 55 Years
- Enrollment
- 125 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1993 – 2007
- U.S. locations
- 1
- States / cities
- Tampa, Florida