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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 · NCT02254863

Krabbe Disease Global Patient Registry

RecruitingObservational study
All ages · All sexes · Updated Jul 17, 2026 · NCT02993796

A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)

RecruitingPhase 1/2
30 Months · All sexes · Updated Apr 17, 2026 · NCT04998396

Longitudinal Study of Neurodegenerative Disorders

RecruitingObservational study
All ages · All sexes · Updated Feb 9, 2026 · NCT03333200

Data 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 · NCT04528355

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

RecruitingPhase 2
2 Months – 55 Years · All sexes · Updated Dec 15, 2025 · NCT01962415

The Myelin Disorders Biorepository Project

RecruitingObservational study
All ages · All sexes · Updated Oct 23, 2025 · NCT03047369

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

RecruitingObservational study
All ages · All sexes · Updated May 29, 2025 · NCT01793168

From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.