Clinical Trials on Creatine Deficiency, X-linked
Total 1559 results
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Janssen Research & Development, LLCJanssen Research & Development, LLCActive, not recruitingX-Linked Retinitis PigmentosaBelgium, Canada, United States, Israel, United Kingdom, Spain, Denmark, France, Italy, Netherlands, Switzerland
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University Hospital, MontpellierAssociation Xtraordinaire sub-group DDX3X; GenidaUnknownIntellectual Developmental Disorder | X-LINKEDFrance
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SwanBio Therapeutics, Inc.RecruitingAMN | AMN Gene Mutation | X-ALDNetherlands, United States
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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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National Institute of Allergy and Infectious Diseases...RecruitingChronic Granulomatous Disease | InfectionUnited States
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National Institute of Allergy and Infectious Diseases...Enrolling by invitationChronic Granulomatous Disease | p47 | Autosomal RecessiveUnited States
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GenethonActive, not recruitingWiskott-Aldrich SyndromeUnited Kingdom, France
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Capital Research Institute of PediatricsUnknownWiskott-Aldrich Syndrome
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St. Jude Children's Research HospitalCompletedWiskott-Aldrich SyndromeUnited States
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Medical College of WisconsinNational Cancer Institute (NCI); National Heart, Lung, and Blood Institute... and other collaboratorsCompletedChronic Granulomatous Disease | Hemophagocytic Lymphohistiocytosis | Leukocyte Adhesion Deficiency | Chronic Active Epstein-Barr Virus Infection | HIGM-1 | IPEXUnited States, Canada
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Assistance Publique - Hôpitaux de ParisRecruitingX-linked HypophosphatemiaFrance
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Wuerzburg University HospitalKyowa Kirin, Inc.Completed
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Hospices Civils de LyonCompleted
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Kyowa Hakko Kirin Pharma, Inc.Kyowa Kirin Co., Ltd.CompletedX-linked HypophosphatemiaUnited States, Canada
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Peking Union Medical College HospitalUnknownX-linked HypophosphatemiaChina
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Kyowa Kirin, Inc.Kyowa Kirin Co., Ltd.CompletedX-linked HypophosphatemiaUnited States, Korea, Republic of, Japan, Ireland, United Kingdom, France, Italy
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Kyowa Kirin Pharmaceutical Development LtdCompletedX-linked HypophosphatemiaUnited Kingdom, France, Ireland, Italy
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Kyowa Hakko Kirin Pharma, Inc.Completed
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Grifols Therapeutics LLCCompletedCommon Variable Immunodeficiency | Severe Combined Immunodeficiency | Wiskott-Aldrich Syndrome | Agammaglobulinemia | Immunologic Deficiency SyndromeCanada, United States
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Janssen Research & Development, LLCActive, not recruitingX-Linked Retinitis PigmentosaUnited States, United Kingdom
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National Institute of Allergy and Infectious Diseases...Rare Diseases Clinical Research Network; Primary Immune Deficiency Treatment...CompletedWiskott-Aldrich SyndromeUnited States, Canada
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The Korean Society of Pediatric Hematology OncologyCompletedWiskott-Aldrich SyndromeKorea, Republic of
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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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National Institute of Neurological Disorders and...CompletedMotor Neuron DiseaseUnited States
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Psyadon PharmaCompletedLesch-Nyhan DiseaseUnited States
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Duke UniversityRecruiting
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National Institute of Arthritis and Musculoskeletal...CompletedIchthyosis | Ichthyosis, X-Linked | Ichthyosis, Lamellar | Darier Disease | Hailey-Hailey Disease | Hyperkeratosis, Epidermolytic | KeratodermaUnited States
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ShireCompletedHunter SyndromeUnited States, United Kingdom
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National Taiwan University HospitalUnknownRett Syndrome | Tourette Syndrome | Tic DisordersTaiwan
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Kyowa Kirin, Inc.Kyowa Kirin Co., Ltd.CompletedX-linked HypophosphatemiaUnited States, Korea, Republic of, Canada, Japan, France, Denmark
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Kyowa Hakko Kirin Pharma, Inc.Kyowa Kirin Co., Ltd.CompletedX-linked HypophosphatemiaUnited States, Canada
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University Hospital, MontpellierAgence de La BiomédecineActive, not recruitingFragile X Syndrome | Huntington Disease | Myotonic Dystrophy 1France
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University of OxfordUniversity College, London; University of Southampton; Moorfields Eye Hospital... and other collaboratorsCompleted
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University of British ColumbiaVancouver General HospitalWithdrawn
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TakedaTakeda Development Center Americas, Inc.RecruitingHunter Syndrome | Mucopolysaccharidosis (MPS)United States
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National Institute of Neurological Disorders and...RecruitingMotor Neuron Disease | Spinal and Bulbar Muscular Atrophy | Kennedys DiseaseUnited States
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ShireCompletedHunter SyndromeUnited Kingdom, United States, Brazil
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NightstaRx Ltd, a Biogen CompanyCompletedX-Linked Retinitis PigmentosaUnited States, France, United Kingdom, Finland, Netherlands, Germany, Canada
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Jerry Vockley, MD, PhDUltragenyx Pharmaceutical IncNo longer availableBarth Syndrome | Mitochondrial Trifunctional Protein Deficiency | Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency | Carnitine Palmitoyltransferase Deficiencies (CPT1, CPT2) | Long-chain Hydroxyacyl-CoA Dehydrogenase Deficiency | Glycogen Storage Disorders | Pyruvate Carboxylase Deficiency... and other conditionsUnited States
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National Institute for Mother and Child Health...Marie Curie Emergency Children's Hospital, Bucharest; Victor Gomoiu Children...RecruitingChronic Granulomatous DiseaseRomania
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Fondazione TelethonOspedale San RaffaeleCompleted
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Federal Research Institute of Pediatric Hematology...UnknownHematopoietic Stem Cell Transplantation | Wiskott-Aldrich Syndrome | Graft FailureRussian Federation
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Kiora Pharmaceuticals, Inc.RecruitingRetinitis Pigmentosa | ChoroideremiaAustralia
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Center for Eye Research AustraliaAustralian National University; Commonwealth Scientific and Industrial Research... and other collaboratorsEnrolling by invitation
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MeiraGTx UK II LtdSyne Qua Non Limited; Bionical EmasCompletedGene Therapy for X-linked Retinitis Pigmentosa (XLRP) - Retinitis Pigmentosa GTPase Regulator (RPGR)X-Linked Retinitis PigmentosaUnited Kingdom, United States
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Mobius Medical Pty Ltd.University of Melbourne; Australian National University; Centre for Eye Research... and other collaboratorsCompleted
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Seoul National University HospitalRecruitingDuchenne Muscular Dystrophy | Scoliosis Neuromuscular | Lordosis LumbarKorea, Republic of