Clinical Trials on X-linked Myotubular Myopathy
Total 1543 results
-
Weill Medical College of Cornell UniversityNovartis PharmaceuticalsTerminatedBleeding | Thrombocytopenia | Wiskott-Aldrich SyndromeUnited States
-
Novo Nordisk A/SCompletedHaemophilia A, Haemophilia B, ObesityUnited States
-
Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia BUnited States, Netherlands, Germany, Macedonia, The Former Yugoslav Republic of, United Kingdom, Italy, Malaysia, Hungary, Thailand, France, Russian Federation, Turkey, Japan, South Africa, Canada
-
National Institute of Allergy and Infectious Diseases...CompletedGranulomatous Disease, Chronic | Infection | Job's SyndromeUnited States
-
Novo Nordisk A/SCompletedHealthy | Congenital Bleeding Disorder | Haemophilia A | Haemophilia BUnited Kingdom
-
Novo Nordisk A/SCompletedHealthy | Congenital Bleeding Disorder | Haemophilia A | Haemophilia BUnited Kingdom
-
Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia A | Haemophilia BSpain
-
Andrea GropmanNational Center for Research Resources (NCRR); Rare Diseases Clinical Research... and other collaboratorsCompletedBrain Diseases, Metabolic, Inborn | Ornithine Transcarbamylase Deficiency | Urea Cycle DisorderUnited States
-
Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia A | Haemophilia BBelgium, Germany, France, United Kingdom, Italy, Hungary, Austria, Sweden, Iran, Islamic Republic of, Argentina, Ireland, Poland, Portugal, Slovakia
-
University of California, San FranciscoRecruitingMucopolysaccharidosis I | Mucopolysaccharidosis II | Mucopolysaccharidosis VI | Wolman Disease | Pompe Disease Infantile-Onset | Mucopolysaccharidosis IV A | Mucopolysaccharidosis VII | Neuronopathic Gaucher DiseaseUnited States
-
University of Milano BicoccaComitato Maria Letizia Verga,ItalyRecruitingLymphoma, Non-Hodgkin | Leukemia, Myeloid, Acute | Leukemia, B-cell | Adrenoleukodystrophy | Lymphoma, Hodgkin | Thalassemia Major | Leukemia, T Cell | DrepanocytosisItaly
-
University of CincinnatiChildren's Hospital Medical Center, CincinnatiTerminatedAdrenoleukodystrophy | Zellweger Syndrome | Infantile Refsum's Disease | Bifunctional Enzyme Deficiency
-
Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With InhibitorsJapan
-
Novo Nordisk A/SCompletedHealthy | Congenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With InhibitorsFrance
-
Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With InhibitorsFrance, United Kingdom, Spain, Israel, Poland, Turkey, Hungary
-
Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With InhibitorsUnited States, Denmark, France, United Kingdom
-
University of ChicagoNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsCompletedKrabbe Disease | Mucopolysaccharidosis Type II (MPS II) | Mucopolysaccharidosis Type I (MPS I) | Mucopolysaccharidosis Type III (MPS III) | Mucopolysaccharidosis Type VI (MPS VI)United States
-
Children's Hospital of PhiladelphiaRecruitingLeukemia | Bone Marrow Failure Syndromes | Immunodeficiencies | Inborn Errors of Metabolism | Immunodysregulation Polyendocrinopathy Enteropathy X-linked SyndromeUnited States
-
National Human Genome Research Institute (NHGRI)Active, not recruitingWiskott- Aldrich Syndrome | ADA Deficient SCIDUnited States
-
Boston Children's HospitalMassachusetts General Hospital; Mayo Clinic; Karolinska University Hospital; Baylor... and other collaboratorsActive, not recruitingAplastic Anemia | Dyskeratosis Congenita | Hoyeraal Hreidarsson Syndrome | Revesz SyndromeUnited States, Norway, Sweden
-
Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With InhibitorsUnited States
-
National Institute of Allergy and Infectious Diseases...WithdrawnChronic Granulomatous Disease | Inflammatory Bowel DiseaseUnited States
-
Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With InhibitorsJapan
-
Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With InhibitorsSaudi Arabia, Algeria, Turkey, Germany, Poland, Netherlands, Italy, Belgium, South Africa, United Kingdom, Sweden, Austria, Portugal, France, Czech Republic, Venezuela
-
Shanghai Children's HospitalRecruitingAlport Syndrome, X-LinkedChina
-
Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With InhibitorsUnited States
-
Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With InhibitorsUnited States, Austria, Spain, Japan, Croatia, Italy, United Kingdom, Israel, Sweden, Ukraine, Malaysia, Greece, Denmark, Canada
-
Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With InhibitorsUnited States, Serbia, Malaysia, Thailand, Greece, Japan, Romania
-
Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With InhibitorsFrance, United States, Sweden, Serbia, Turkey, United Kingdom, Malaysia, Brazil, Former Serbia and Montenegro, Japan, South Africa
-
Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With InhibitorsJapan
-
Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With InhibitorsUnited States
-
Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With InhibitorsSlovakia, Spain, Argentina, Germany, Croatia, Italy, Canada, United Kingdom, Ireland, Sweden, France, United States, Czech Republic, Switzerland
-
University of Sao Paulo General HospitalCompletedHemophilia A | Hemophilia B | Osteoarthritis | HemarthrosisBrazil
-
National Institute of Allergy and Infectious Diseases...Not yet recruitingHyper-Immunoglobulin E. Syndrome (HEIS) | Chronic Granulomatous Disease (CGD)United States
-
Novo Nordisk A/SCompletedEpidemiological Study on Haemophilia Care and Orthopaedic Status in Developing Countries (HAEMOcare)Congenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With Inhibitors | Haemophilia A | Haemophilia BIndia, Morocco, South Africa, Oman
-
Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With InhibitorsUnited States
-
Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With Inhibitors | Haemophilia A | Haemophilia BSpain
-
Mirum Pharmaceuticals, Inc.Children's Hospital Medical Center, CincinnatiCompletedCholestasis | Peroxisomal Disorders | Adrenoleukodystrophy | Zellweger Syndrome | Infantile Refsum's DiseaseUnited States
-
Duke UniversityNational Institute of Allergy and Infectious Diseases (NIAID); University of... and other collaboratorsRecruitingCommon Variable Immunodeficiency | X-linked Agammaglobulinemia | Primary Immune Deficiency | Primary Antibody Deficiencies | CVID | XLA | Secondary HypogammaglobulinemiaUnited States
-
National Institute of Allergy and Infectious Diseases...TerminatedIFN-Gamma Therapy | CGD Gene Mutation | CGD Response to IFNg | CGD - Chronic Granulomatous Disease | Immunodeficiency DiseaseUnited States
-
University of PennsylvaniaTerminatedOrnithine Transcarbamylase Deficiency Disease
-
Novo Nordisk A/SWithdrawnHealthy | Congenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With Inhibitors | Haemophilia A | Haemophilia B
-
National Institute of Allergy and Infectious Diseases...RecruitingChronic Granulomatous Disease-associated ColitisUnited States
-
National Institute of Allergy and Infectious Diseases...Enrolling by invitationChronic Granulomatous Disease | Inflammatory Bowel DiseaseUnited States
-
National Institute of Allergy and Infectious Diseases...WithdrawnChronic Granulomatous Diseases (CGD) and Liver LesionsUnited States
-
National Institute of Allergy and Infectious Diseases...TerminatedEctodermal Dysplasia | Hyper-IgM SyndromeUnited States
-
Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With InhibitorsTaiwan, United Kingdom, Thailand, Serbia, Croatia, Italy, Poland, Romania, Hungary, Malaysia, United States, Austria, Brazil, Greece, Japan, Puerto Rico, Russian Federation, South Africa, Turkey
-
Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With InhibitorsFrance, United States, Spain, Brazil, Philippines, South Africa, Poland, Turkey, Romania, Bulgaria, Russian Federation, Argentina
-
Eunice Kennedy Shriver National Institute of Child...Children's National Research InstituteTerminatedPhase I Pilot Study of Liver-Directed Gene Therapy for Partial Ornithine Transcarbamylase DeficiencyOrnithine Transcarbamylase Deficiency Disease
-
Chiesi Farmaceutici S.p.A.ICON plcNot yet recruiting