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

> **NCT07222371** · PHASE1,PHASE2 · ACTIVE_NOT_RECRUITING · sponsor: **University of California, San Diego** · enrollment: 1 (actual)

## Conditions studied

- Genetic Disease
- TUBB4A-Related Leukodystrophy

## Interventions

- **DRUG:** nL-TUBB4-001

## Key facts

- **NCT ID:** NCT07222371
- **Lead sponsor:** University of California, San Diego
- **Sponsor class:** OTHER
- **Phase:** PHASE1,PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** ACTIVE_NOT_RECRUITING
- **Start date:** 2025-09-19
- **Primary completion:** 2027-09
- **Final completion:** 2027-09
- **Target enrollment:** 1 (ACTUAL)
- **Last updated:** 2025-10-29


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT07222371

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT07222371, "An Open-label, Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for TUBB4A-related Leukodystrophy". Retrieved via AI Analytics 2026-05-26 from https://api.ai-analytics.org/clinical/NCT07222371. Licensed CC0.

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*[Clinical trials dataset](/datasets/clinical-trials) · CC0 1.0*