- ICH GCP
- US Clinical Trials Registry
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Clinical Trials on Mental Retardation, X-linked 102
Total 2011 results
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Minoryx Therapeutics, S.L.Active, not recruitingAdrenoleukodystrophySpain, United States, Hungary, France, Germany, Italy, Netherlands, United Kingdom
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National Taiwan University HospitalCompleted
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Masonic Cancer Center, University of MinnesotaWithdrawn
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MedDay Pharmaceuticals SACompletedAdrenoleukodystrophy | Adrenomyeloneuropathy | AMNFrance, Germany, Spain
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Eunice Kennedy Shriver National Institute of Child...Food and Drug Administration (FDA)TerminatedAdrenoleukodystrophy | AdrenomyeloneuropathyUnited States
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Jirair Krikor BedoyanUltragenyx Pharmaceutical IncRecruitingPyruvate Dehydrogenase Complex DeficiencyUnited States
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University of FloridaEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsActive, not recruitingPyruvate Dehydrogenase Complex DeficiencyUnited States
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Fondazione TelethonCompletedPyruvate Dehydrogenase Complex DeficiencyItaly
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Masonic Cancer Center, University of MinnesotaRecruiting
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Minoryx Therapeutics, S.L.RecruitingCerebral Adrenoleukodystrophy (cALD)United States, Brazil, Argentina
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Poxel SANot yet recruiting
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bluebird bioCompletedCerebral Adrenoleukodystrophy (CALD)United States, France, Germany, Italy, Netherlands, United Kingdom
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bluebird bioCompletedCerebral Adrenoleukodystrophy (CALD)United States, Germany, Argentina, Australia, France, United Kingdom
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Masonic Cancer Center, University of MinnesotaWithdrawnCerebral Adrenoleukodystrophy
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FDA Office of Orphan Products DevelopmentHugo W. Moser Research Institute at Kennedy Krieger, Inc.Completed
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Far Eastern Memorial HospitalNational Taiwan University Hospital; Fu Jen Catholic UniversityUnknownAdrenoleukodystrophyTaiwan
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University of California, San DiegoBioMarin PharmaceuticalWithdrawnBehavioral Manifestations of Lesch-Nyhan DiseaseUnited States
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Retina Foundation of the SouthwestDSM Nutritional Products, Inc.; Foundation Fighting BlindnessCompletedRetinitis Pigmentosa | X-linked Genetic DiseasesUnited States
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Duke UniversityRecruiting
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ShireCompletedHunter SyndromeUnited States, United Kingdom
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bluebird bioCenter for International Blood and Marrow Transplant ResearchRecruitingCerebral Adrenoleukodystrophy (CALD)United States
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Poxel SANot yet recruiting
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Masonic Cancer Center, University of MinnesotaRecruitingCerebral Adrenoleukodystrophy | Adrenoleukodystrophy | ALD (Adrenoleukodystrophy)United States
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Onofre, Aurora Pujol, M.D.Completed
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Hugo W. Moser Research Institute at Kennedy Krieger...TerminatedAdrenoleukodystrophyUnited States
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Onofre, Aurora Pujol, M.D.Ministerio de Sanidad, Servicios Sociales e Igualdad; Fundacion HesperiaCompleted
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Emalex Biosciences Inc.Psyadon PharmaTerminatedLesch-Nyhan Disease | Self-injurious BehaviorSpain, United States
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Oslo University HospitalActive, not recruiting
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REGENXBIO Inc.Active, not recruitingMucopolysaccharidosis Type II (MPS II)United States, Brazil
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ShireCompleted
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ShireCompletedHunter Syndrome | Mucopolysaccharidosis (MPS)United States, Argentina, Mexico, Spain, United Kingdom
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TakedaTakeda Development Center Americas, Inc.RecruitingHunter Syndrome | Mucopolysaccharidosis (MPS)United States
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ShireCompletedHunter SyndromeUnited Kingdom, United States, Brazil
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Astellas Gene TherapiesCompletedX-linked Myotubular MyopathyUnited States, France, United Kingdom, Canada, Germany
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University of FloridaAudentes TherapeuticsCompletedX-linked Myotubular MyopathyUnited States
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University of PittsburghNational Institute of Neurological Disorders and Stroke (NINDS); Rare Diseases...RecruitingPyruvate Dehydrogenase Complex Deficiency DiseaseUnited States
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Great Ormond Street Hospital for Children NHS Foundation...UnknownX-linked Severe Combined ImmunodeficiencyUnited Kingdom
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TakedaRecruitingHunter SyndromeCanada, France
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REGENXBIO Inc.Active, not recruitingMucopolysaccharidosis Type II (MPS II)United States, Canada
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Lundquist Institute for Biomedical Innovation at...TerminatedMucopolysaccharidosis I | Mucopolysaccharidosis II | Mucopolysaccharidosis VIUnited States
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Massachusetts General HospitalEuropean Leukodystrophy AssociationRecruitingRestless Legs Syndrome | AdrenoleukodystrophyUnited States, Netherlands
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James DowlingCanadian Institutes of Health Research (CIHR); Cures Within Reach; The Joshua... and other collaboratorsRecruitingX Linked Myotubular MyopathyUnited Kingdom, Canada, United States
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National Institute of Allergy and Infectious Diseases...RecruitingX-linked Severe Combined Immunodeficiency (XSCID)United States
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St. Jude Children's Research HospitalNational Heart, Lung, and Blood Institute (NHLBI); Assisi Foundation; California...SuspendedSevere Combined Immunodeficiency Disease, X-linkedUnited States
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Assistance Publique - Hôpitaux de ParisCompletedX-linked Severe Combined ImmunodeficiencyFrance
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Eunice Kennedy Shriver National Institute of Child...TerminatedFragile X Syndrome | Premature Ovarian Failure | Premature Menopause | FMR1 Premutation | Primary Ovairan InsufficiencyUnited States
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ShireCompletedHunter SyndromeSpain, United States, Canada, United Kingdom, France, Australia, Mexico
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University Hospital HeidelbergCompletedMucopolysaccharidosis Type I | Mucopolysaccharidosis Type II | Coping Behavior | Mucopolysaccharidosis Type III | Behavior DisordersGermany
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Shenzhen Geno-Immune Medical InstituteUnknownSCID, X LinkedChina