Active, not recruitingPHASE1, PHASE2NCT07222371
An Open-label, Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for TUBB4A-related Leukodystrophy
Studying Leukodystrophy
Last synced from ClinicalTrials.gov
ℹ
Clinical trial records are synced from ClinicalTrials.gov through automated extraction.
Report missing dataKey facts
- Sponsor
- University of California, San Diego
- Intervention
- nL-TUBB4-001(drug)
- Enrollment
- 1 target
- Eligibility
- All sexes
- Timeline
- 2025 – 2027
Study locations (1)
- Rady Children's Hospital San Diego, San Diego, California, United States
Primary source
Recruitment status, site addresses, contacts, and full eligibility criteria can change between syncs. Always verify with the trial team before planning travel or treatment.
Open NCT07222371 on ClinicalTrials.govOther trials for Leukodystrophy
Additional recruiting or active studies for the same condition.
- ENROLLING BY INVITATIONPHASE1, PHASE2NCT06369974Single Participant Study of an Experimental ASO Treatment for TUBB4A-related LeukodystrophyMassachusetts General Hospital
- RECRUITINGNANCT06224413A Study of Participants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene AutotemcelGenetix Biotherapeutics Inc.
- RECRUITINGPHASE3NCT05819866A 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
- RECRUITINGNANCT03047369The Myelin Disorders Biorepository ProjectChildren's Hospital of Philadelphia
- RECRUITINGPHASE1NCT02254863UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like CellsJoanne Kurtzberg, MD
- RECRUITINGNCT03639285Natural History, Diagnosis, and Outcomes for LeukodystrophiesUniversity of Utah