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Leukoencephalopathies clinical trials recruiting in the U.S.

7 studies for Leukoencephalopathies are recruiting participants at sites across the United States.

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Recently updated studies

An Open-Label Exploratory Study of Fosigotifator in Participants With Vanishing White Matter Disease

RecruitingPhase 1/2
6 Months · All sexes · Updated Sep 24, 2026 · NCT05757141

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

North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)

RecruitingObservational studyHealthy volunteers welcome
All ages · All sexes · Updated Feb 4, 2026 · NCT01694940

The Myelin Disorders Biorepository Project

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

Natural History Study of Patients with HPDL Mutations

RecruitingObservational study
All ages · All sexes · Updated Mar 30, 2025 · NCT05848271

Natural History Study of Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation (LBSL)

RecruitingObservational study
All ages · All sexes · Updated Jul 3, 2024 · NCT03624374

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