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Clinical Trials on X-linked Severe Combined Immunodeficiency
Total 1627 results
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University Hospital, BordeauxCompletedPain | Fabry's DiseaseFrance
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Wuerzburg University HospitalCharite University, Berlin, Germany; University Hospital Muenster; Health Care...RecruitingQuality of Life | Adherence, Medication | Fabry Disease | Rare DiseasesGermany
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Mayo ClinicNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsRecruitingCystinuria | Primary Hyperoxaluria | Dent Disease | APRT DeficiencyUnited States, Iceland
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Ultragenyx Pharmaceutical IncKyowa Kirin Co., Ltd.AvailableX-linked Hypophosphatemia | Tumor-Induced Osteomalacia
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National Institute of Allergy and Infectious Diseases...Enrolling by invitationSafety and Efficacy of Tofacitinib for Chronic Granulomatous Disease With Inflammatory ComplicationsChronic Granulomatous Disease | Inflammatory Gastrointestinal Disease | Inflammatory Skin Disease | Inflammatory Lung DiseaseUnited States
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Kyowa Kirin, Inc.Kyowa Kirin Co., Ltd.CompletedX-linked HypophosphatemiaNetherlands, United States, United Kingdom, France
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Orthopedic Hospital Vienna SpeisingCompleted
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Lokman Hekim ÜniversitesiHacettepe UniversityCompletedQuality of Life | Lower Urinary Tract Symptoms | Muscle Weakness | Duchenne Muscular Dystrophy | Pelvic Floor Muscle Weakness | Posture Disorders in Children | Lumbar LordosisTurkey
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University of MinnesotaGenzyme, a Sanofi CompanyWithdrawnNeuropathic Pain | Fabry DiseaseUnited States
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University of WashingtonNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsUnknown
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University of RostockCompletedStroke, Acute | Cerebrovascular Accident | Cerebral StrokeGermany, Poland
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Assistance Publique - Hôpitaux de ParisCompletedOculocerebrorenal SyndromeFrance
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University of MichiganNational Institute of Dental and Craniofacial Research (NIDCR)Not yet recruitingHealing Wound | Alveolar Bone Loss | Image | Dent Disease | Alveolar; Wound
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SanofiRecruitingFabry DiseaseChina, Finland, Germany, Norway, United Kingdom, United States, Denmark, Austria, Greece, Romania, Argentina, Brazil, Canada, Japan, Mexico, Poland, Italy, France, Turkey, Australia, Switzerland
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Albina Nowak, MDSwiss National Science FoundationRecruiting
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Ultragenyx Pharmaceutical IncKyowa Kirin Co., Ltd.Active, not recruitingX-linked Hypophosphatemia | Hypophosphatemic RicketsUnited States, Brazil, Argentina, Canada, Chile, Colombia
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Assistance Publique - Hôpitaux de ParisCompletedDuchenne Muscular Dystrophy | Spinal Muscular AtrophyFrance
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Turkish Society of Pediatric Gastroenterology,...CompletedDuchenne Muscular Dystrophy | Pompe Disease (Late-onset)Turkey
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Universidad de Zaragozafundación española para el estudio y tratamiento de la Enfermedad de Gaucher... and other collaboratorsCompleted
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Bicetre HospitalUnknownRare Diseases | X-linked HypophosphatemiaFrance
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Boston Children's HospitalTerminatedFanconi Anemia | Dyskeratosis CongenitaUnited States
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Amicus TherapeuticsCompleted
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University of Alabama at BirminghamCompleted
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University of Alabama at BirminghamCompletedProteinuria | Fabry DiseaseUnited States, Slovenia
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Medical University of ViennaMedical University of GrazUnknownFabry Disease | Left Ventricular HypertrophyAustria
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Baylor College of MedicineChildren's National Research InstituteRecruitingOrnithine Transcarbamylase Deficiency | Urea Cycle Disorder | Carbamyl Phosphate Synthetase Deficiency | Argininosuccinic Aciduria | Hyperargininemia | Citrullinemia 1 | ARGI Deficiency | ASL Deficiency | ASS Deficiency | NAGS DeficiencyUnited States
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Mayo ClinicRecruitingCystinuria | Primary Hyperoxaluria | Dent Disease | APRT DeficiencyUnited States
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Amicus TherapeuticsEngage Health Inc.CompletedFabry DiseaseUnited States
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Yale UniversityCompleted
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University Hospital, BordeauxCompletedChronic Pain | Fabry's DiseaseFrance
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Yale UniversityNational Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)CompletedHyperparathyroidism | Hypophosphatemia, FamilialUnited States
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Baylor Research InstituteNational Institute of Neurological Disorders and Stroke (NINDS)Completed
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Universität Duisburg-EssenWeinmann Geräte für Medizin GmbH + Co. KGCompletedDuchenne Muscular Dystrophy | Spinal Muscular Atrophy | Chronic Respiratory InsufficiencyGermany
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Johannes Gutenberg University MainzUnknownMucopolysaccharidosis | Fabry DiseaseGermany
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University of RostockUnknownGrowth Disorders | Hypophosphatemic Rickets | Somatropin TherapyGermany
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St. Jude Children's Research HospitalBaylor College of Medicine; Children's Hospital of Philadelphia; Dana-Farber... and other collaboratorsRecruitingMyelodysplastic Syndromes | Sickle Cell Disease | Fanconi Anemia | Severe Congenital Neutropenia | Bone Marrow Failure Syndromes | Blood Coagulation Disorder | Erythrocyte Disorder | Leukocyte Disorder | Hemostasis | Dyskeratosis Congenita | Diamond-Blackfan Anemia | Congenital Thrombocytopenia | Myeloproliferative...United States
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Yang I. Pachankis, PhDS for ScienceActive, not recruitingPTSD | Anxiety Disorders | OCD | Borderline Personality Disorder | Social Anxiety Disorder | Depression, Anxiety | Depression, Unipolar | ADHD - Combined Type | Grief | Depression Anxiety Disorder | Depression and Suicide | Autism Spectrum Disorder High-Functioning | Avoidant Personality Disorder | Agoraphobia With... and other conditionsChina
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Masonic Cancer Center, University of MinnesotaCompletedNiemann-Pick Disease | Wolman Disease | Adrenoleukodystrophy | Batten Disease | Metachromatic Leukodystrophy | GM1 Gangliosidosis | Fucosidosis | Globoid Cell Leukodystrophy | Gaucher's Disease | Tay Sachs Disease | Sandhoff DiseaseUnited States
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Eunice Kennedy Shriver National Institute of Child...RecruitingAndrogen Insensitivity Syndrome | Metabolic Parameters in AIS, CAIS, PAIS and MAIS | Tumor Formation in AIS, CAIS, PAIS and MAIS | Sexual Function AIS, CAIS, PAIS and MAISUnited States
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University Hospital Birmingham NHS Foundation TrustNorthern Care Alliance NHS Foundation Trust; Cambridge University Hospitals... and other collaboratorsActive, not recruitingArrhythmia Burden, Risk of Sudden Cardiac Death and Stroke in Patients With Fabry Disease (RaILRoAD)Fabry DiseaseUnited Kingdom, Australia
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University of AarhusCompletedX-linked Hypophosphatemia | Hereditary HypophosphatemiaDenmark
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Ultragenyx Pharmaceutical IncTerminatedWilson Disease | Ornithine Transcarbamylase Deficiency | Glycogen Storage Disease Type IASpain, United Kingdom, Canada, United States, Brazil
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CENTOGENE GmbH RostockCompleted
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Hospices Civils de LyonUnknownEnd Stage Renal Disease | Fabry Disease | Renal DialysisFrance
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Baylor Research InstituteCompleted
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Centre Hospitalier Universitaire de NiceUnknown
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Baylor College of MedicineThe Methodist Hospital Research Institute; Center for Cell and Gene Therapy...RecruitingHaploidentical Hematopoietic Cell Transplantation Using TCR Alpha/Beta and CD19 Depletion (HAPLOTAB)Myelodysplastic Syndromes | Primary Immunodeficiency Diseases | Hemoglobinopathies | Chronic Myeloid Leukemia | Cytopenia | Severe Aplastic Anemia | Bone Marrow Failure Syndrome | Acute Myeloid Leukemia in Remission | Hemophagocytic Lymphohistiocytoses | Acute Lymphoblastic Leukemia in Remission | Severe...United States
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Fondazione Policlinico Universitario Agostino Gemelli...Recruiting
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AbiliTech Medical Inc.Richard M. Schulze Family FoundationRecruitingCerebral Palsy | Muscular Dystrophies | Duchenne Muscular Dystrophy | Becker Muscular Dystrophy | Spinal Muscular Atrophy | Limb Girdle Muscular Dystrophy | FSHDUnited States