Not applicableStudy completedNCT02952482What this trial is testingNewborn Screening for AdrenoleukodystrophyWho this might be right forAdrenoleukodystrophy National Taiwan University Hospital 45,796
Not applicableLooking for participantsNCT05443906What this trial is testingHome Exercise for Individuals with Neurodegenerative DiseaseWho this might be right forNeurodegenerative DiseasesLeukodystrophyAtaxia+5 more Hugo W. Moser Research Institute at Kennedy Krieger, Inc. 30
Not applicableStudy completedNCT00004348What this trial is testingStudy of Protein Translocation in Patients With Beta-Oxidation DisordersWho this might be right forBeta-Oxidation DisorderPeroxisomal Disorders National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) 20
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
Large-scale testing (Phase 3)Study completedNCT01896102What this trial is testingThe Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD)Who this might be right forCerebral Adrenoleukodystrophy (CALD) Genetix Biotherapeutics Inc. 32
Not applicableEnrolling By InvitationNCT03655223What this trial is testingEarly Check: Expanded Screening in NewbornsWho this might be right forSpinal Muscular AtrophyFragile X SyndromeFragile X - Premutation+182 more RTI International 30,000
Not applicableLooking for participantsNCT05939232What this trial is testingRegistry of X-linked AdrenoleukodystrophyWho this might be right forX-linked Adrenoleukodystrophy Beijing Tiantan Hospital 200
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 applicableStudy completedNCT00004450What this trial is testingRandomized Study of Beta Interferon and Thalidomide in Patients With AdrenoleukodystrophyWho this might be right forAdrenoleukodystrophy FDA Office of Orphan Products Development 60
Large-scale testing (Phase 3)Ended earlyNCT00654433What this trial is testingALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic DiseasesWho this might be right forInherited Metabolic DiseasesLysosomal Storage DisordersPeroxisomal Storage Diseases+2 more Aldagen 40
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
Not applicableNot Yet RecruitingNCT05911919What this trial is testingValidation of a Prognostic Biomarker Using Brain Diffusion MRI in X-linked AdrenoleukodystrophyWho this might be right forX-linked Adrenoleukodystrophy Assistance Publique - Hôpitaux de Paris 200
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
Testing effectiveness (Phase 2)UnknownNCT02559830What this trial is testingAutologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and AdrenoleukodystrophyWho this might be right forMetachromatic LeukodystrophyAdrenoleukodystrophy Shenzhen Second People's Hospital 50
Testing effectiveness (Phase 2)Study completedNCT01043640What this trial is testingAllogeneic Bone Marrow Transplant for Inherited Metabolic DisordersWho this might be right forMucopolysaccharidosisHurler SyndromeHunter Syndrome+10 more Masonic Cancer Center, University of Minnesota 46
Not applicableStudy completedNCT02699190What this trial is testingLeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for LeukodystrophiesWho this might be right forLeukodystrophyWhite Matter Disease4H Syndrome+61 more Children's Hospital of Philadelphia 236
Not applicableLooking for participantsNCT01668186What this trial is testingLongitudinal Natural History Study of Patients With Peroxisome Biogenesis Disorders (PBD)Who this might be right forPeroxisome Biogenesis DisorderZellweger Spectrum DisorderRCDP - Rhizomelic Chondrodysplasia Punctata+8 more McGill University Health Centre/Research Institute of the McGill University Health Centre 244
Early research (Phase 1)Study completedNCT03500627What this trial is testingEvaluate the Safety, Tolerability, and Pharmacokinetics of OP-101 After Intravenous Administration in Healthy VolunteersWho this might be right forHealthy Orpheris, Inc. 8
Not applicableEnded earlyNCT02204904What this trial is testingObservational Study to Evaluate Allogeneic HSCT Outcomes for Cerebral Adrenoleukodystrophy (CALD)Who this might be right forX-Linked Adrenoleukodystrophy (X-ALD)Cerebral Adrenoleukodystrophy (CALD)Adrenoleukodystrophy (ALD) Genetix Biotherapeutics Inc. 59
Early research (Phase 1)Study completedNCT01586455What this trial is testingHuman Placental-Derived Stem Cell TransplantationWho this might be right forMucopolysaccharidosis IMucopolysaccharidosis VIAdrenoleukodystrophy+13 more New York Medical College 43