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Clinical Trials on X-linked Severe Combined Immunodeficiency
Total 1627 results
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Caroline Michaela KistorpRigshospitalet, Denmark; Amicus TherapeuticsActive, not recruiting
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Spark TherapeuticsCompletedHematologic Diseases | Blood Coagulation Disorders, Inherited | Coagulation Protein Disorders | Hemorrhagic Disorders | Genetic Diseases, Inborn | Genetic Diseases, X-Linked | Gene Therapy | Blood Coagulation Disorder | Gene Transfer | Adeno-Associated Virus (AAV) | Factor VIII (FVIII) | Factor VIII (FVIII)... and other conditionsUnited States
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University Hospital ErlangenCompletedHypohidrotic Ectodermal DysplasiaGermany
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Ospedale San DonatoAmicus Therapeutics; Institute of Biomedicine and Molecular Immunology - CNRCompletedHeart Diseases | Fabry DiseaseItaly
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Cooperative International Neuromuscular Research...Carolinas Medical Center lead study siteCompletedBecker Muscular Dystrophy | Limb-Girdle Muscular Dystrophy, Type 2A (Calpain-3 Deficiency) | Limb-Girdle Muscular Dystrophy, Type 2B (Miyoshi Myopathy, Dysferlin Deficiency) | Limb-Girdle Muscular Dystrophy, Type 2I (FKRP-deficiency)United States
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Masonic Cancer Center, University of MinnesotaCompletedMucopolysaccharidosis | Hurler Syndrome | Sphingolipidoses | Peroxisomal Disorders | Krabbe Disease | Adrenoleukodystrophy (ALD) | Hunter Syndrome | Sly Syndrome | Fucosidosis | Aspartylglucosaminuria | Alpha Mannosidosis | Metachromatic Leukodystrophy (MLD) | Maroteaux-Lamy SyndromeUnited States
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Mackay Memorial HospitalRecruiting
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Universidade do PortoHospital da Luz, Lisboa; Hospital Pedro Hispano; Centro Hospitalar de Leiria; Centro... and other collaboratorsRecruitingFabry DiseasePortugal
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Yonsei UniversityRecruitingFabry DiseaseKorea, Republic of
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Merve KurtRecruitingDuchenne Muscular Dystrophy | Virtual Reality | Spinal Muscular Atrophy | Neuromuscular Disease | BiofeedbackTurkey
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Yonsei UniversityRecruitingFabry DiseaseKorea, Republic of
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Yonsei UniversityRecruitingFabry's DiseaseKorea, Republic of
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Genzyme, a Sanofi CompanyTerminatedFabry Disease | Alpha Galactosidase A DeficiencyUnited States, Austria, United Kingdom
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Columbia UniversityMuscular Dystrophy Association; Stevens Institute of TechnologyCompletedDuchenne Muscular Dystrophy | Spinal Muscular Atrophy Type 3United States
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Resverlogix CorpWithdrawn
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Kyowa Kirin Co., Ltd.CompletedA Study of KRN23 in Adult and Pediatric Patients With X-linked Hypophosphatemic Rickets/OsteomalaciaXLHJapan, Korea, Republic of
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University Children's Hospital, ZurichCompletedPhenylketonurias | OTC Deficiency | Inborn Errors of Metabolism | Urea Cycle Disorder | Maple Syrup Urine Disease | Methylmalonicacidemia | Aminoacidopathy | Patient Reported Outcome MeasurementsSwitzerland
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University of Maryland, BaltimoreCompletedOsteoporosis PseudogliomaUnited States
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Ulla Feldt-RasmussenCompleted
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Wolfson Medical CenterUnknownFabry Disease in the Young StrokeIsrael
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Genzyme, a Sanofi CompanyWithdrawn
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National Institute of Neurological Disorders and...CompletedHealthy | Fabry Disease | Cerebrovascular AccidentUnited States
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Assistance Publique - Hôpitaux de ParisNot yet recruitingHemophilia A | Hemophilia B | Cystic Fibrosis | Sickle Cell Disease | Muscular Dystrophy, Duchenne | Fragile X Syndrome | Huntington Disease | Myotonic Dystrophy | Autosomal Recessive Polycystic Kidney Disease | Neurofibromatosis-Noonan Syndrome | Muscular Dystrophy, Becker | Invasive PreNatal Diagnosis in a... and other conditionsFrance
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Brigham and Women's HospitalNortheastern UniversityCompletedVenous Thromboembolism | Patient Fall | Adverse Drug Event | Severe Hypoglycemia | Hospital Acquired Pressure Ulcer | Central Line-Associated Bloodstream Infection (CLABSI) | Catheter-Associated Infection | Opioid-Related Severe Adverse Drug Event | Severe Hospital Acquired Delerium | Rapid Response Related...United States
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iECURE, Inc.RecruitingUrea Cycle Disorders, Inborn | Ornithine Transcarbamylase Deficiency | Ornithine Transcarbamylase Deficiency Disease | Ornithine Carbamoyltransferase Deficiency (Disorder)United Kingdom
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Yonsei UniversityRecruitingFabry DiseaseKorea, Republic of
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ProtalixChiesi Farmaceutici S.p.A.CompletedFabry DiseaseUnited States, Netherlands, Hungary, United Kingdom, Czechia, Norway, Slovenia, Spain, Finland, France, Italy, Switzerland
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Mads Peter Godtfeldt StemmerikEdgewise Therapeutics, Inc.CompletedBecker Muscular Dystrophy | McArdle Disease | Limb-Girdle Muscular Dystrophy Type 2Denmark
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NYU Langone HealthNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)CompletedCystinuria | Primary Hyperoxaluria | Dent Disease | Adenine Phosphoribosyl Transferase DeficiencyUnited States, Iceland
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Novo Nordisk A/SCompletedHealthy | Congenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With Inhibitors | Acquired Bleeding Disorder | Acquired Haemophilia | Congenital FVII Deficiency | Glanzmann's DiseaseFrance
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Lysosomal and Rare Disorders Research and Treatment...Recruiting
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Chiesi Farmaceutici S.p.A.Active, not recruitingFabry DiseaseUnited States, Netherlands, Australia, Hungary, United Kingdom, Canada, Czechia, Finland, France, Italy, Norway, Slovenia, Spain
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University of Maryland, BaltimoreChildren's Hospital of PhiladelphiaWithdrawnOsteoporosis Pseudoglioma SyndromeUnited States
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CENTOGENE GmbH RostockWithdrawnStroke, Acute | Cerebrovascular Accident | Cerebral StrokeGermany
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Children's Hospital Medical Center, CincinnatiCompletedDuchenne Muscular Dystrophy | Spinal Muscular Atrophy | Congenital Muscular Dystrophy
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Rennes University HospitalCompleted
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University College, LondonUniversity of Sydney; University Hospital BirminghamUnknownFabry DiseaseUnited Kingdom, Australia
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Assistance Publique - Hôpitaux de ParisTerminatedNeuromuscular Diseases | Amyotrophic Lateral Sclerosis | Duchenne Muscular DystrophyFrance
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Genzyme, a Sanofi CompanyCompletedFabry DiseaseUnited States, United Kingdom, Canada, Hungary, Poland, Czech Republic
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National Institute of Neurological Disorders and...CompletedFabry DiseaseUnited States
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University of CambridgeSanofiRecruiting
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Fundacion para la Investigacion Biomedica del Hospital...Fundación Mutua MadrileñaRecruiting
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Baylor College of MedicineChildren's Hospital of Philadelphia; Children's Hospital Colorado; Seattle Children... and other collaboratorsRecruitingOrnithine Transcarbamylase Deficiency | Urea Cycle Disorder | Argininosuccinic Aciduria | Hyperargininemia | Citrullinemia 1 | ARGI Deficiency | ASL Deficiency | ASS DeficiencyUnited States
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University Hospital, AngersRecruiting
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Chiesi Farmaceutici S.p.A.Active, not recruitingFabry DiseaseBelgium, United States, United Kingdom, Italy, Czechia, Denmark, Norway
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Assistance Publique - Hôpitaux de ParisShire International GmbHCompleted
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Children's Mercy Hospital Kansas CityCompletedHypophosphatemic Rickets, X-Linked DominantUnited States
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National Human Genome Research Institute (NHGRI)Completed