Not applicableLooking for participantsNCT02714764What this trial is testingEvaluation of Outcome Metrics in Alexander DiseaseWho this might be right forAlexander Disease Children's Hospital of Philadelphia 200
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)Active Not RecruitingNCT04849741What this trial is testingEvaluate the Safety and Efficacy of Zilganersen (ION373) in Patients With Alexander Disease (AxD)Who this might be right forAlexander Disease Ionis Pharmaceuticals, Inc. 54
Not applicableAvailableNCT07487389What this trial is testingZilganersen Expanded Access Program for Individuals With Alexander DiseaseWho this might be right forAlexander Disease Ionis Pharmaceuticals, Inc.
Not applicableLooking for participantsNCT03047369What this trial is testingThe Myelin Disorders Biorepository ProjectWho this might be right forLeukodystrophyWhite Matter DiseaseLeukoencephalopathies+63 more Children's Hospital of Philadelphia 12,000
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