drugset

drugset / Trial / NCT07222371

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

NCT07222371 ↗

Phase 1/2 Active not recruiting 1 enrolled University of California, San Diego

NaSingle-groupOpen-labelTreatment

Summary

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

Timeline

Start: 2025-09-19
Primary completion: 2027-09
Completion: 2027-09

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	nL-TUBB4-001	Antisense oligonucleotide	—	—

Indications