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Leukodystrophies clinical trials recruiting in the U.S.
8 studies for Leukodystrophies are recruiting participants at sites across the United States.
Leukodystrophies are rare diseases that affect the cells of the brain and are often genetic. Find more information on these progressive diseases.
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Recently updated studies
UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
RecruitingPhase 1
1 Week – 22 Years · All sexes · Updated Sep 3, 2026 · NCT02254863Krabbe Disease Global Patient Registry
RecruitingObservational study
All ages · All sexes · Updated Jul 17, 2026 · NCT02993796A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)
RecruitingPhase 1/2
30 Months · All sexes · Updated Apr 17, 2026 · NCT04998396Longitudinal Study of Neurodegenerative Disorders
RecruitingObservational study
All ages · All sexes · Updated Feb 9, 2026 · NCT03333200Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC
RecruitingObservational study
2 Months – 60 Years · All sexes · Updated Jan 13, 2026 · NCT04528355Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT
RecruitingPhase 2
2 Months – 55 Years · All sexes · Updated Dec 15, 2025 · NCT01962415The Myelin Disorders Biorepository Project
RecruitingObservational study
All ages · All sexes · Updated Oct 23, 2025 · NCT03047369Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
RecruitingObservational study
All ages · All sexes · Updated May 29, 2025 · NCT01793168From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.