Clinical Trials on Maternally-inherited Leigh Syndrome
Total 124 results
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Horizon Pharma USA, Inc.CompletedInherited Mitochondrial Disease, Including Leigh SyndromeUnited States
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PTC TherapeuticsAxio Research. LLCCompleted
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PTC TherapeuticsCompletedLeigh SyndromeUnited States
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Aadi Bioscience, Inc.Withdrawn
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Taysha Gene Therapies, Inc.Withdrawn
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Sohag UniversityAssiut University; Kyoto UniversityRecruitingInherited BMF SyndromeEgypt
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Franziska WachterHarvard Clinical and Translational Science Center (Harvard Catalyst)RecruitingAcute Myeloid Leukemia | Myelodysplastic Syndromes | MDS | Aml | Myeloid Neoplasm | Myeloid Malignancies | Inherited Bone Marrow Failure SyndromeUnited States
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The University of Texas Health Science Center,...RecruitingLeigh Disease | Leigh Syndrome | Leigh's Necrotizing Encephalopathy | Subacute Necrotizing Encephalomyopathy | Subacute Necrotizing EncephalomyelopathyUnited States
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Khondrion BVDrug Research Unit Ghent, BelgiumCompletedMitochondrial Disease | MELAS | Leigh Syndrome | LHON | Mitochondrial DNA tRNALeu(UUR) m.3243A<G MutationBelgium
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Peter MacCallum Cancer Centre, AustraliaNational Health and Medical Research Council, Australia; University of MelbourneRecruitingHematologic Diseases | Inherited Platelet Disorder | Inherited BMF SyndromeAustralia
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PTC TherapeuticsCompletedMitochondrial Diseases | Drug Resistant Epilepsy | Leigh Disease | Leigh Syndrome | Mitochondrial Encephalopathy (MELAS) | Pontocerebellar Hypoplasia Type 6 (PCH6) | Alpers Disease | Alpers SyndromeUnited States, Spain, United Kingdom, Canada, France, Italy, Poland, Sweden, Japan
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University of WyomingCompletedMaternal Obesity SyndromeUnited States
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Mayo ClinicRecruitingHematologic Neoplasms | Myeloid Malignancy | Cytopenia | Bone Marrow Failure Syndrome | Clonal Cytopenia of Undetermined Significance | Clonal Hematopoiesis of Indeterminate Potential | Low Risk Myelodysplastic Syndrome | Inherited Bone Marrow Failure Syndrome | Clonal Expansion | Hereditary Neoplastic... and other conditionsUnited States
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LMU KlinikumUniversity of Pisa; German Federal Ministry of Education and Research; European...RecruitingMitochondrial Diseases | MDS | Mitochondrial Myopathies | MELAS Syndrome | MIDD | Kearns-Sayre Syndrome | MERRF Syndrome | Barth Syndrome | Leigh Syndrome | MNGIE | LHON | Pearson Syndrome | NARP Syndrome | Coenzyme Q10 Deficiency | SANDO | SCAE | MIRAS | CPEOAustria, Germany, Italy
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National Institute of Allergy and Infectious Diseases...CompletedInherited Immune DeficienciesUnited States
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National Cancer Institute (NCI)RecruitingInherited Bone Marrow Failure Syndrome | Familial Platelet Disorder With Predisposition to Myeloid MalignanciesUnited States
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Massachusetts General HospitalNational Cancer Institute (NCI); Boston Medical CenterCompletedGenetic TestingUnited States
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Vanderbilt-Ingram Cancer CenterNational Cancer Institute (NCI); University of South FloridaRecruitingBreast Cancer | Colorectal Cancer | Prostate Cancer | Endometrial Cancer | Inherited Cancer SyndromeUnited States
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Fondazione TelethonOspedale San RaffaeleCompleted
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Mongi Slim HospitalUnknownCesarean | Maternal Hypotension Syndrome
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Cardiff UniversityCardiff and Vale University Health BoardRecruitingSepsis | SIRS | Pregnancy; Infection | Maternal Sepsis During Labor | Maternal SepsisUnited Kingdom
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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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National Human Genome Research Institute (NHGRI)Active, not recruitingWiskott- Aldrich Syndrome | ADA Deficient SCIDUnited States
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Fondazione TelethonOspedale San RaffaeleActive, not recruitingWiskott-Aldrich SyndromeItaly, United States
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David WilliamsActive, not recruitingWiskott-Aldrich SyndromeUnited States
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GenethonInstitute of Child Health; Great Ormond Street Hospital for Children NHS Foundation...Completed
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GlaxoSmithKlineCompletedWiskott-Aldrich SyndromeUnited States
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GenethonHôpital Necker-Enfants MaladesCompleted
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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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Efferon JSCRecruitingMaternal sepsIsRussian Federation
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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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National Eye Institute (NEI)TerminatedInherited Retinal Degeneration | Inherited Ophthalmic DiseasesUnited States
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National Cancer Institute (NCI)RecruitingFanconi Anemia | Inherited Bone Marrow Failure SyndromeUnited States
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National Cancer Institute (NCI)RecruitingFanconi Anemia | Dyskeratosis Congenita | Diamond Blackfan Anemia | Shwachman Diamond Syndrome | Inherited Bone Marrow Failure Syndrome, Aplastic AnemiaUnited States
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Soma JyonouchiBaylor College of MedicineCompletedX-linked Thrombocytopenia | Wiskott-Aldrich Syndrome (WAS)United States
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Bellicum PharmaceuticalsTerminatedHemoglobinopathies | Hemophagocytic Lymphohistiocytosis | Metabolic Disorders | Primary Immune Deficiency Disorders | Inherited Bone Marrow Failure SyndromeUnited States
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National Heart, Lung, and Blood Institute (NHLBI)RecruitingSevere Aplastic Anemia | Telomere Biology Disorders | Inherited Bone Marrow Failure SyndromesUnited States
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Federal Research Institute of Pediatric Hematology...UnknownHematopoietic Stem Cell Transplantation | Wiskott-Aldrich Syndrome | Graft FailureRussian Federation
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Federal Research Institute of Pediatric Hematology...CompletedWiskott-Aldrich SyndromeRussian Federation
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Federal Research Institute of Pediatric Hematology...RecruitingWiskott-Aldrich SyndromeRussian Federation
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Weill Medical College of Cornell UniversityNovartis PharmaceuticalsTerminatedBleeding | Thrombocytopenia | Wiskott-Aldrich SyndromeUnited States
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Eunice Kennedy Shriver National Institute of Child...University of WashingtonUnknownWiskott-Aldrich Syndrome | Leukocyte Adhesion Deficiency Syndrome | X-Linked Agammaglobulinemia | X-Linked Hyper IgM SyndromeUnited States
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NICHD Global Network for Women's and Children's...Boston University; University of Colorado, Denver; University of Alabama at Birmingham and other collaboratorsActive, not recruitingMaternal Death | Maternal Infections Affecting Fetus or Newborn | Neonatal SEPSIS | Maternal Sepsis During Labor | Neonatal Death | Postpartum SepsisZambia, Bangladesh, Congo, The Democratic Republic of the, Guatemala, India, Kenya, Pakistan
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Bio Products LaboratoryCompletedCommon Variable Immunodeficiency | Wiskott-Aldrich Syndrome | X-linked Agammaglobulinemia | Primary Immune Deficiency Disorders | Hyper-IgM SyndromeUnited States, Chile, Israel
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NovartisCompletedMyelodysplastic Syndromes | Hemoglobinopathies | Diamond-Blackfan Anemia | Transfusional Iron Overload | Other Inherited or Acquired Anaemia | MPD Syndrome | Other Rare AnaemiasAustralia
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University Hospital, MontpellierInstitut National de la Santé Et de la Recherche Médicale, FranceActive, not recruitingPediatric ALL | Long QT Syndrome | Inherited Cardiac Conduction Disorder | Congenital Long Qt SyndromeFrance
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Columbia UniversityEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsEnrolling by invitationMitochondrial Disease | Mitochondrial Disorders | Melas | Kearns Sayer | NARP | MNGIE | LHON | Mitochondrial Depletion Syndrome | Leigh's DiseaseUnited States