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Condition: Leukodystrophy Synced from ClinicalTrials.gov · July 29, 2026

Recruiting studies: Leukodystrophy

Studies currently listed as recruiting on the public registry for this search term. Each page explains the study in plain language, with locations and the study team's contact details. Clinicians: generate a printable list for your patient →

Krabbe Disease Global Patient Registry
Phase N/A · 1 location(s) · State University of New York at Buffalo · NCT02993796UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
Phase 1 · 1 location(s) · Joanne Kurtzberg, MD · NCT02254863Direct Lentiviral Injection Gene Therapy for MLD
NA · 1 location(s) · Shenzhen Geno-Immune Medical Institute · NCT03725670Home Exercise for Individuals with Neurodegenerative Disease
NA · 1 location(s) · Hugo W. Moser Research Institute at Kennedy Krieger, Inc. · NCT05443906Lentiviral Hematopoietic Stem Cell Gene Therapy for MLD
NA · 1 location(s) · Shenzhen Geno-Immune Medical Institute · NCT07046338Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Phase N/A · 2 location(s) · Sanford Health · NCT01793168Modeling Macrophages Activation Pattern in X-linked Adrenoleukodystrophy, Metachromatic Leukodystrophy and Adult Onset L
Phase N/A · 2 location(s) · Assistance Publique - Hôpitaux de Paris · NCT04925349The Myelin Disorders Biorepository Project
Phase N/A · 23 location(s) · Children's Hospital of Philadelphia · NCT03047369Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC
Phase N/A · 1 location(s) · Paul Szabolcs · NCT04528355Natural History, Diagnosis, and Outcomes for Leukodystrophies
Phase N/A · 1 location(s) · University of Utah · NCT03639285A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)
Phase 1/Phase 2 · 4 location(s) · Aspa Therapeutics · NCT04998396Longitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases.
Phase N/A · 1 location(s) · Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta · NCT04880356Longitudinal Study of Neurodegenerative Disorders
Phase N/A · 1 location(s) · University of Pittsburgh · NCT03333200Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT
Phase 2 · 1 location(s) · Paul Szabolcs · NCT01962415
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This page is independently generated by Eichor from the public ClinicalTrials.gov record and re-synced daily. It is not the sponsor's official website unless claimed. Nothing here is medical advice; eligibility is always determined by the study team — talk to your own doctor first.