Search clinical trials for: Inherited mitochondrial disease
Total 378374 results
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University College, LondonNot yet recruiting
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UK Kidney AssociationRecruitingVasculitis | AL Amyloidosis | Tuberous Sclerosis | Fabry Disease | Cystinuria | Focal Segmental Glomerulosclerosis | IgA Nephropathy | Bartter Syndrome | Pure Red Cell Aplasia | Membranous Nephropathy | Atypical Hemolytic Uremic Syndrome | Autosomal Dominant Polycystic Kidney Disease | Cystinosis | Nephronophthisis | BK Nephropathy and other conditionsUnited Kingdom
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Omeicos Therapeutics GmbHRecruitingPrimary Mitochondrial DiseaseGermany, Italy
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Centre Hospitalier Régional de la CitadelleSanofi; Takeda; University of Liege; Orchard Therapeutics; Centre Hospitalier Universitaire... and other collaboratorsRecruitingCongenital Adrenal Hyperplasia | Hemophilia A | Hemophilia B | Mucopolysaccharidosis I | Mucopolysaccharidosis II | Cystic Fibrosis | Alpha 1-Antitrypsin Deficiency | Sickle Cell Disease | Fanconi Anemia | Chronic Granulomatous Disease | Wilson Disease | Severe Congenital Neutropenia | Ornithine Transcarbamylase... and other conditionsBelgium
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Foundation Fighting BlindnessRecruitingRetinitis Pigmentosa | Choroideremia | Usher Syndrome | Batten Disease | Leber Congenital Amaurosis | Goldmann-Favre Syndrome | Kearns-Sayre Syndrome | Retinal Disease | Bardet-Biedl Syndrome | Stargardt Disease | Cone Dystrophy | Retinoschisis | Achromatopsia | Gyrate Atrophy | Eye Diseases Hereditary | Bassen-Kornzweig... and other conditionsUnited States
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Masonic Cancer Center, University of MinnesotaRecruitingHurler Syndrome | Sphingolipidoses | Peroxisomal Disorders | Metachromatic Leukodystrophy | Alpha-Mannosidosis | Hunter Syndrome | Mucopolysaccharidosis Disorders | Maroteaux Lamy Syndrome | Sly Syndrome | Fucosidosis | Aspartylglucosaminuria | Glycoprotein Metabolic Disorders | Recessive Leukodystrophies | Globoid... and other conditionsUnited States
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Sanford HealthNational Ataxia Foundation; Beyond Batten Disease Foundation; Pitt Hopkins Research... and other collaboratorsRecruitingMitochondrial Diseases | Retinitis Pigmentosa | Myasthenia Gravis | Eosinophilic Gastroenteritis | Multiple System Atrophy | Leiomyosarcoma | Leukodystrophy | Anal Fistula | Spinocerebellar Ataxia Type 3 | Friedreich Ataxia | Kennedy Disease | Lyme Disease | Hemophagocytic Lymphohistiocytosis | Spinocerebellar Ataxia... and other conditionsUnited States, Australia
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Columbia UniversityNational Institute of Neurological Disorders and Stroke (NINDS)RecruitingMitochondrial Diseases | Mitochondrial Disorders | Mitochondrial Genetic Disorders | Disorder of Mitochondrial Respiratory Chain Complexes | Deletion and Duplication of Mitochondrial DNAUnited States, Canada
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RTI InternationalEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsEnrolling by invitationPrimary Hyperoxaluria Type 3 | Diabetes Mellitus | Hemophilia A | Hemophilia B | Hereditary Fructose Intolerance | Cystic Fibrosis | Factor VII Deficiency | Phenylketonurias | Sickle Cell Disease | Dravet Syndrome | Duchenne Muscular Dystrophy | Prader-Willi Syndrome | Fragile X Syndrome | Chronic Granulomatous Disease and other conditionsUnited States
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PTC TherapeuticsEnrolling by invitationInherited Mitochondrial DiseaseUnited States, France, Spain, United Kingdom, Italy, Poland, Japan
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Khondrion BVJulius Clinical; ProPharma Group; CertaraCompletedMitochondrial Diseases | Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation | Maternally Inherited Diabetes and Deafness (MIDD) | Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke Like Episodes (MELAS) | Chronic Progressive External Ophthalmoplegia (CPEO)Netherlands, Denmark, United Kingdom, Germany
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PTC TherapeuticsNo longer available
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GenSight BiologicsNo longer availableLeber Hereditary Optic Neuropathy (Optic, Atrophy, Hereditary, Leber)
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GenSight BiologicsActive, not recruitingLeber Hereditary Optic NeuropathyBelgium, Spain, United States, France, United Kingdom, Taiwan, Italy
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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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GenSight BiologicsCompletedLeber Hereditary Optic NeuropathyUnited States, France, Italy, Spain, United Kingdom
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National Heart, Lung, and Blood Institute (NHLBI)CompletedCancer | Muscle Weakness | Skin FibroblastsUnited States
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Horizon Pharma USA, Inc.Terminated
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Horizon Pharma USA, Inc.CompletedInherited Mitochondrial Disease, Including Leigh SyndromeUnited States
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Enzyre B.V.Indiana Hemophilia & Thrombosis CenterNot yet recruiting
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University Hospital, BonnSwedish Orphan BiovitrumRecruitingHaemophilia A | Synovitis | Hemophilia Arthropathy | SonographyGermany
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Equilibra Bioscience LLCNot yet recruitingHemophilia A | Hemophilia B | Healthy Participants
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Jirair Krikor BedoyanUltragenyx Pharmaceutical IncRecruitingPyruvate Dehydrogenase Complex DeficiencyUnited States
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Washington University School of MedicineNational Cancer Institute (NCI); The Foundation for Barnes-Jewish HospitalRecruitingMultiple Myeloma | Rectal Cancer | Cholangiocarcinoma | Colon CancerUnited States
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IRCCS Fondazione Stella MarisFondazione I.R.C.C.S. Istituto Neurologico Carlo Besta; IRCCS Ospedale San... and other collaboratorsRecruiting
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Texas Tech UniversityNot yet recruiting
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The University of QueenslandNot yet recruitingAtaxia TelangiectasiaAustralia
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Dr. Sascha DrewloNot yet recruitingFetal Growth Retardation | Preeclampsia | Cervix; Pregnancy | Inherited Genetic Conditions (Diagnosis)
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Kathelijn FischerRadboud University Medical Center; University Medical Center Groningen; Maastricht... and other collaboratorsRecruitingAdolescent | Child | Hemophilia A With Inhibitor | Adult | Hemophilia A Without Inhibitor | Hemophilia A, SevereNetherlands
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Niguarda HospitalIstituto di Fisiologia Clinica CNRRecruitingCoronary Artery DiseaseItaly
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Suzhou Alphamab Co., Ltd.RecruitingHemophilia A With Inhibitor | Hemophilia B With InhibitorChina
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Chia Tai Tianqing Pharmaceutical Group Nanjing...Not yet recruiting
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Spark Therapeutics, Inc.Recruiting
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University College, LondonMoorfields Eye Hospital NHS Foundation TrustRecruiting
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Staidson (Beijing) Biopharmaceuticals Co., LtdRecruiting
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Investigación en Hemofilia y FisioterapiaNot yet recruiting
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Bispebjerg HospitalNot yet recruitingInflammation | Muscle Strain, Multiple Sites
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Radboud University Medical CenterEnzyre B.V.Not yet recruiting
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Investigación en Hemofilia y FisioterapiaNot yet recruiting
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Investigación en Hemofilia y FisioterapiaRecruiting
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Institute of Hematology & Blood Diseases Hospital...Not yet recruiting
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All India Institute of Medical Sciences, BhubaneswarRecruiting
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BioMarin PharmaceuticalRecruiting
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BayerRecruitingHemophilia A | Prophylaxis of Bleeding | Treatment of BleedingKorea, Republic of
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University Hospital, BordeauxRecruiting
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Hospices Civils de LyonRecruiting
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Richard FryeAutism Discovery and Treatment FoundationRecruitingEpilepsy | Neurodevelopmental Disorders | Autism Spectrum Disorder | Down Syndrome | Mitochondrial Encephalomyopathies | Pediatric Acute-Onset Neuropsychiatric Syndrome | Cerebral Folate Deficiency | Pediatric Autoimmune Neuropsychiatric Disorder Associated With Streptococcal InfectionUnited States
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Innostellar Biotherapeutics Co.,LtdRecruitingInherited Retinal DystrophyChina
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Hemab ApSRecruitingGlanzmann ThrombastheniaUnited States, Belgium, France, Italy, Netherlands, United Kingdom
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University School of Physical Education, Krakow...Ministry of Education and Science,PolandRecruiting