Clinical Trial

An Open-label, Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for TUBB4A-related Leukodystrophy

Name: An Open-label, Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for TUBB4A-related Leukodystrophy — clinical trial record (NCT07222371)
Creator: DataLookout
License: https://creativecommons.org/licenses/by/4.0/

Study acronym: ASOTUBB4A

Active, Not Recruiting Phase 1/2

View on ClinicalTrials.gov →

Summary

This current study is aimed for the treatment of an individual participant with a form of TUBB4A-related leukodystrophy with hypomyelination.

Trial Details

NCT Number	NCT07222371
Lead Sponsor	University of California, San Diego
Conditions	Genetic Disease, TUBB4A-Related Leukodystrophy
Enrollment	1 participants
Start Date	2025-09-19
Primary Completion	2027-09 (estimated)
Study Completion	2027-09 (estimated)
Updated on ClinicalTrials.gov	2025-10-29