NCT07222371

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

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 1 (actual)
• Sponsor: University of California, San Diego · Academic / Other
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

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

Detailed description

The study is an investigational interventional drug created for the study subject's specific pathogenic genetic mutation.

Conditions

• Genetic Disease
• TUBB4A-Related Leukodystrophy

Interventions

Type	Name	Description
DRUG	nL-TUBB4-001	Personalized antisense oligonucleotide

Timeline

• Start date: 2025-09-19
• Primary completion: 2027-09-01
• Completion: 2027-09-01
• First posted: 2025-10-29
• Last updated: 2025-10-29

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study