Clinical trials for Leukodystrophy
8 recruiting or active trials synced from ClinicalTrials.gov.
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Clinical trial records are synced from ClinicalTrials.gov through automated extraction.
Report missing data- ACTIVE NOT RECRUITINGPHASE1, PHASE2NCT07222371An Open-label, Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for TUBB4A-related LeukodystrophyUniversity of California, San Diego
- ENROLLING BY INVITATIONPHASE1, PHASE2NCT06369974Single Participant Study of an Experimental ASO Treatment for TUBB4A-related LeukodystrophyMassachusetts General Hospital · PI: Florian Eichler, MD
- RECRUITINGN/ANCT06224413A Study of Participants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene AutotemcelGenetix Biotherapeutics Inc. · PI: Kiran Bhirangi, MD
- RECRUITINGPhase 3NCT05819866A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adult Male Subjects With Cerebral AdrenoleukodystrophyMinoryx Therapeutics, S.L.
- RECRUITINGPHASE1, PHASE2NCT05757141An Open-Label Exploratory Study of Fosigotifator in Participants With Vanishing White Matter DiseaseCalico Life Sciences LLC · PI: ABBVIE INC.
- RECRUITINGN/ANCT03047369The Myelin Disorders Biorepository ProjectChildren's Hospital of Philadelphia · PI: Adeline Vanderver, MD
- RECRUITINGPhase 1NCT02254863UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like CellsJoanne Kurtzberg, MD · PI: Joanne Kurtzberg, MD
- RECRUITINGNCT03639285Natural History, Diagnosis, and Outcomes for LeukodystrophiesUniversity of Utah