# Single Participant Study of an Experimental ASO Treatment for TUBB4A-related Leukodystrophy

> **NCT06369974** · PHASE1,PHASE2 · ENROLLING_BY_INVITATION · sponsor: **Massachusetts General Hospital** · enrollment: 1 (estimated)

## Conditions studied

- Genetic Disease

## Interventions

- **DRUG:** Antisense oligonucleotide treatment (ASO)

## Key facts

- **NCT ID:** NCT06369974
- **Lead sponsor:** Massachusetts General Hospital
- **Sponsor class:** OTHER
- **Phase:** PHASE1,PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** ENROLLING_BY_INVITATION
- **Start date:** 2024-09-18
- **Primary completion:** 2026-06
- **Final completion:** 2026-06
- **Target enrollment:** 1 (ESTIMATED)
- **Last updated:** 2026-03-02

## Collaborators

- [object Object]

## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT06369974

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT06369974, "Single Participant Study of an Experimental ASO Treatment for TUBB4A-related Leukodystrophy". Retrieved via AI Analytics 2026-06-08 from https://api.ai-analytics.org/clinical/NCT06369974. Licensed CC0.

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