Not applicableNo Longer AvailableNCT02233257What this trial is testingExpanded Access for Lorenzo's Oil (GTO/GTE) in AdrenoleukodystrophyWho this might be right forX-linked Adrenoleukodystrophy University of Minnesota
Not applicableLooking for participantsNCT04880356What this trial is testingLongitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases.Who this might be right forInherited DiseaseRare DiseasesMetabolic Disease+3 more Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta 100
Large-scale testing (Phase 3)Study completedNCT00007020What this trial is testingCompassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic AcidWho this might be right forInfantile Refsum's DiseaseZellweger SyndromeAdrenoleukodystrophy+2 more Mirum Pharmaceuticals, Inc. 85
Not applicableLooking for participantsNCT05939232What this trial is testingRegistry of X-linked AdrenoleukodystrophyWho this might be right forX-linked Adrenoleukodystrophy Beijing Tiantan Hospital 200
Large-scale testing (Phase 3)Study completedNCT03852498What this trial is testingA Clinical Study to Assess the Efficacy and Safety of Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy (CALD)Who this might be right forCerebral Adrenoleukodystrophy (CALD) Genetix Biotherapeutics Inc. 35
Large-scale testing (Phase 3)Study completedNCT01838941What this trial is testingBetaine and Peroxisome Biogenesis DisordersWho this might be right forPeroxisome Biogenesis Disorders McGill University Health Centre/Research Institute of the McGill University Health Centre 12
Testing effectiveness (Phase 2)Ended earlyNCT00668564What this trial is testingHematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of MetabolismWho this might be right forHurler's SyndromeMaroteaux-Lamy SyndromeSly Syndrome+8 more Masonic Cancer Center, University of Minnesota 18
Large-scale testing (Phase 3)Study completedNCT02961803What this trial is testingMD1003-AMN MD1003 in AdrenomyeloneuropathyWho this might be right forAdrenomyeloneuropathyAdrenoleukodystrophyAMN MedDay Pharmaceuticals SA 67
Not applicableLooking for participantsNCT06796920What this trial is testingEpidural Spinal Cord Stimulation for Lower-limb Impairment in AdrenomyeloneuropathyWho this might be right forAdrenomyeloneuropathy Without Cerebral Involvement Third Military Medical University 10
Large-scale testing (Phase 3)Study completedNCT00176904What this trial is testingStem Cell Transplant for Inborn Errors of MetabolismWho this might be right forAdrenoleukodystrophyMetachromatic LeukodystrophyGloboid Cell Leukodystrophy+8 more Masonic Cancer Center, University of Minnesota 135
Testing effectiveness (Phase 2)WithdrawnNCT03196765What this trial is testingSafety, Pharmacokinetics and Pharmacodynamics of NV1205 in Pediatric Male Subjects With AdrenoleukodystrophyWho this might be right forX-Linked Adrenoleukodystrophy NeuroVia, Inc.
Not applicableLooking for participantsNCT06497829What this trial is testingGeranylgeraniol Supplementation in Patients With Mevalonate Kinase DeficiencyWho this might be right forMevalonate Kinase Deficiency University Hospital, Motol 6
Testing effectiveness (Phase 2)Study completedNCT03856866What this trial is testingHydroxychloroquine Administration for Reduction of PexophagyWho this might be right forZellweger SyndromePeroxisome Biogenesis Disorders The Hospital for Sick Children 3
Not applicableUnknownNCT00278044What this trial is testingClinical Study and Gene Mutation Analysis of Adrenoleukodystrophy in Taiwanese ChildrenWho this might be right forAdrenoleukodystrophy Far Eastern Memorial Hospital 20
Not applicableLooking for participantsNCT06224413What this trial is testingParticipants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene AutotemcelWho this might be right forCerebral Adrenoleukodystrophy (CALD) Genetix Biotherapeutics Inc. 120
Not applicableStudy completedNCT01594853What this trial is testingExercise Study of Function and Pathology for Women With X-linked AdrenoleukodystrophyWho this might be right forX-linked Adrenoleukodystrophy Hugo W. Moser Research Institute at Kennedy Krieger, Inc. 31
Testing effectiveness (Phase 2)Study completedNCT00383448What this trial is testingHSCT for High Risk Inherited Inborn ErrorsWho this might be right forAdrenoleukodystrophyMetachromatic LeukodystrophyGloboid Cell Leukodystrophy+6 more Masonic Cancer Center, University of Minnesota 38
Not applicableLooking for participantsNCT06190626What this trial is testingLongitudinal Prospective Natural History Study of Retinopathy in Zellweger Spectrum DisorderWho this might be right forZellweger Spectrum Disorder McGill University Health Centre/Research Institute of the McGill University Health Centre 30
Not applicableUnknownNCT04031287What this trial is testingRCDP Natural History StudyWho this might be right forRhizomelic Chondrodysplasia Punctata MED-LIFE DISCOVERIES LP 75
Not applicableLooking for participantsNCT04675749What this trial is testingQuality of Life in Women with X-linked AdrenoleukodystrophyWho this might be right forX-linked Adrenoleukodystrophy Leipzig University Medical Center 200