Clinical Trials on Adrenomyeloneuropathy Form (AMN) of X-linked Adrenoleukodystrophy (X-ALD)
Total 1565 results
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Eunice Kennedy Shriver National Institute of Child...CompletedSmith-Lemli-Opitz SyndromeUnited States
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Eunice Kennedy Shriver National Institute of Child...CompletedEstimation of the Carrier Frequency and Incidence of Smith-Lemli-Opitz Syndrome in African AmericansSmith-Lemli-Opitz SyndromeUnited States
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Mayo ClinicRecruitingCystinuria | Hyperoxaluria | Dent Disease | Lowe Syndrome | Adenine Phosphoribosyltransferase DeficiencyUnited States, Canada, Iceland, Israel
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Oregon Health and Science UniversityTerminatedSmith-Lemli-Opitz SyndromeUnited States
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Eunice Kennedy Shriver National Institute of Child...WithdrawnSmith-Lemi-Opitz Syndrome
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Eunice Kennedy Shriver National Institute of Child...CompletedPregnancy | Smith-Lemli-Opitz SyndromeUnited States
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National Center for Research Resources (NCRR)Oregon Health and Science UniversityUnknownSmith-Lemli-Opitz SyndromeUnited States
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M.A.G.I.C. Clinic LTDHanalytics Solutions Inc.RecruitingMitochondrial Diseases | Fabry Disease | Metabolic Disease | Gaucher Disease | Pompe DiseaseCanada
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University of Texas Southwestern Medical CenterRecruitingDuchenne Muscular Dystrophy | Becker Muscular Dystrophy | Dilated Cardiomyopathy | Cardiomyopathy, Hypertrophic | Heart Failure With Reduced Ejection Fraction | Cardiac Sarcoidosis | Heart Failure With Preserved Ejection FractionUnited States
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University Hospital, GhentCompletedBelgian Screening Project for the Detection of Anderson-Fabry Disease in Hypertrophic CardiomyopathyLeft Ventricular HypertrophyBelgium
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CENTOGENE GmbH RostockCompletedScoliosis | Muscular Atrophy | Duchenne Muscular Dystrophy | Lordosis | Muscular Weakness | Increased Lordosis/Scoliosis | Hyporeflexia | Red-Green Color BlindnessEgypt, India, Albania, Pakistan, Georgia, Lebanon, Romania, Sri Lanka
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University of AarhusLundbeck Foundation; AP Moeller Foundation; The Korning Foundation DenmarkCompletedAortic Aneurysm | Turner Syndrome | Klinefelter Syndrome | Triple X Syndrome | 47 XYY SyndromeDenmark
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University Hospital, MontpellierRecruiting
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Johann Wolfgang Goethe University HospitalWithdrawnSDF-1 is an Important Cytokine for Neovascularization. Cleavage of SDF-1 is Reduced by DPPIV InhibitorsGermany
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Qilu Hospital of Shandong UniversityRecruitingFabry Disease | Hypertrophic Cardiomyopathy | Left Ventricular HypertrophyChina
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National Taiwan University HospitalRecruitingStroke | Magnetic Resonance Imaging | Fabry Disease | Cerebral Small Vessel Diseases | Cadasil | HTRA1-Related Autosomal Dominant Cerebral Angiopathy | COL4A1-Related Brain Small Vessel Disease With Haemorrhage | Next-generation SequencingTaiwan
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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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University of Colorado, DenverRecruitingKlinefelter Syndrome | Trisomy X | XYY Syndrome | XXXY and XXXXY Syndrome | Xxyy Syndrome | Xyyy Syndrome | Xxxx Syndrome | Xxxxx Syndrome | Xxxyy Syndrome | Xxyyy Syndrome | Xyyyy Syndrome | Male With Sex Chromosome MosaicismUnited States
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Boston Children's HospitalCompletedSmith-Lemli-Opitz SyndromeUnited States
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Forbes Porter, M.D.Eunice Kennedy Shriver National Institute of Child Health and Human Development...Completed
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National Center for Research Resources (NCRR)James Whitcomb Riley Hospital for ChildrenCompletedThrombocytopenia | Dyskeratosis Congenita | Fanconi's Anemia | Shwachman Syndrome
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Massachusetts General HospitalGenzyme, a Sanofi Company; Scott and White Hospital & ClinicTerminatedKidney Diseases | Genetic Diseases, X-Linked | End Stage Renal Disease | ESRD | Fabry Disease | Rare DiseasesUnited States
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American Thrombosis and Hemostasis NetworkNovo Nordisk A/S; Sanofi; Pfizer; Genentech, Inc.; CSL Behring; Hemophilia of Georgia... and other collaboratorsRecruitingHemophilia | Thrombosis | Thrombophilia | Sickle Cell Disease | Thalassemia | Bleeding Disorder | Von Willebrand Diseases | Factor IX Deficiency | Connective Tissue Disorder | Factor VIII Deficiency | Hematologic Disorder | Rare Bleeding Disorder | Platelet DisorderUnited States
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Wuerzburg University HospitalAcademisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA); St George... and other collaboratorsRecruitingFabry Disease | Rare Diseases | Hypertrophic Cardiomyopathy | Fabry Disease, Cardiac VariantGermany
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Eunice Kennedy Shriver National Institute of Child...Completed
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University of NebraskaUniversity of Colorado, Denver; University of Pittsburgh; Children's Hospital...Completed
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University Hospital FreiburgCompletedMyelodysplastic Syndromes | Fanconi Anemia | Dyskeratosis Congenita | Pearson Marrow-pancreas Syndrome | Shwachman-diamond SyndromeSpain, Germany, Switzerland, Austria, Netherlands, Italy, Czech Republic, Belgium, Denmark, Ireland
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National Institutes of Health Clinical Center (CC)CompletedChronic Granulomatous Disease | Wegener's Granulomatosis | Job's Syndrome | Bronchogenic Carcinoma | Mycobacterium InfectionUnited States
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Boston Children's HospitalCompletedAML | ALL | Biphenotypic Leukemia | Refractory Anemia | Refractory Cytopenia With Multilineage Dysplasia | Refractory Anemia With Ringed Sideroblasts | Undifferentiated Leukemia | Ref. Cytopenia w Multilineage Dysplasia & Ringed Sideroblasts | Refractory Anemia With Excess Blasts-1 (5-10% Blasts) | Refractory... and other conditionsUnited States
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CENTOGENE GmbH RostockWithdrawnFructose Metabolism, Inborn Errors | Glycogen Storage Disease Type II | Glycogen Storage Disease | Glycogen Storage Disease Type V | Glycogen Storage Disease Type I | Glycogen Storage Disease Type III | Glycogen Storage Disease Type VII | Glycogen Storage Disease Type IV | Glycogen Storage Disease Type... and other conditionsGermany, India, Sri Lanka
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Caroline Michaela KistorpSanofiNot yet recruitingChronic Kidney Diseases | Fabry DiseaseDenmark
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Wuerzburg University HospitalTakedaActive, not recruitingLysosomal Storage Diseases | Fabry Disease | Fabry Disease, Cardiac Variant | HCM - Hypertrophic Cardiomyopathy | Anderson Fabry DiseaseGermany
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CENTOGENE GmbH RostockWithdrawnChronic Kidney Disease | Hearing Loss | Angiokeratomas | Ocular AbnormalitiesSri Lanka, India, Germany
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Eunice Kennedy Shriver National Institute of Child...RecruitingCHILD Syndrome | Smith Lemli Opitz Syndrome | Lathosterolosis | DesmosterolosisUnited States
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Massachusetts General HospitalCompletedFabry Disease | Mitochondrial Disease | Small Fiber Neuropathy | Ehlers Danlos SyndromeUnited States
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O & O Alpan LLCUnknownFabry Disease | Niemann-Pick Disease | Gaucher Disease | Pompe Disease | Lysosomal Storage DisordersUnited States
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Institute of Hematology & Blood Diseases HospitalRecruitingLymphoma | Acute Myeloid Leukemia | Leukemia | Hemophilia A | Hemophilia B | Hemophilia | Multiple Myeloma | Myelodysplastic Syndrome | Bone Marrow Transplantation | MDS | Aplastic Anemia | Bleeding Disorder | Myeloma, Multiple | Myelomas, Multiple | Leukemias, Acute Myeloid | Myeloid Leukemias, Acute | Hemophilia As | Hemophilia... and other conditionsChina
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National Taiwan University HospitalUnknownHypertension | Diabetes | Dementia | Alzheimer Disease | Fabry Disease | Dry Eye Syndrome | Metabolic Disease | Healthy Individuals | Ocular Rosacea | Limbal Insufficiency | Phlyctenulosis | Graft Versus Host Disease in EyeTaiwan
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Makerere UniversityUniversity of MinnesotaRecruitingThe Study Will Focus on Assessing the Survival Benefit on an Enhanced Package of Care for Patients With Advanced HIV DiseaseUganda
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Baylor College of MedicineThe Methodist Hospital Research Institute; Center for Cell and Gene Therapy...Active, not recruitingAcute Myeloid Leukemia | Acute Lymphoblastic Leukemia | Non Hodgkin Lymphoma | Myelodysplastic Syndrome | Chronic Myelogenous Leukemia | Hemophagocytic Lymphohistiocytosis | Epstein Barr Virus Infection | X-linked Lymphoproliferative Disease | Hemophagocytic Syndrome | Familial Hemophagocytic LymphohistiocytosisUnited States
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National Heart, Lung, and Blood Institute (NHLBI)CompletedCystic Fibrosis | Chronic Granulomatous Disease | Primary Ciliary Dyskinesia | Pseudohypoaldosteronism | Nontuberculous Mycobacterial InfectionUnited States
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University of Colorado, DenverRecruitingHearing Loss | Smith-Lemli-Opitz Syndrome | Cone-Rod DystrophyUnited States
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ResMedClinical Trial Center North Hamburg Germany; CRI-The Clinical Research Institute...TerminatedPulmonary Disease, Chronic Obstructive | Amyotrophic Lateral Sclerosis | Muscular Dystrophies | Duchenne Muscular Dystrophy | Myotonic Dystrophy | Spinal Cord Injury | Obesity Hypoventilation Syndrome | Kyphoscoliosis | Congenital Central Hypoventilation Syndrome | MyopathiesGermany
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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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Paul SzabolcsEnrolling by invitationChronic Granulomatous Disease (CGD) | Wiskott-Aldrich Syndrome | Severe Chronic Neutropenia | Severe Combined Immunodeficiency (SCID) | Immunodeficiency With Predominant T-cell Defect, Unspecified | Hyper IgE Syndromes | Hyper IgM Deficiencies | Mendelian Susceptibility to Mycobacterial Disease | Common...United 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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National Human Genome Research Institute (NHGRI)RecruitingMetabolic Disease | Purine-Pyrimidine Metabolism | AICDA, OMIM *605257, Immunodeficiency With Hyper-IgM, Type 2; HIGM2 | UNG, OMIM *191525, Hyper-IgM Syndrome 5 | NT5C3A<TAB>, OMIM *606224, Anemia, Hemolytic, Due to UMPH1 Deficiency | UMPS, OMIM *613891, Orotic Aciduria | DHODH, OMIM *126064,... and other conditionsUnited States
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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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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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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