1 active not recruiting. 1 Phase 1, 1 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 1 | Active not recruiting 1 |
| Phase 2 | 1 | Active not recruiting 1 |
Follow TUBB4A-Related Leukodystrophy: new trials by email Literature Review of TUBB4A-Related Leukodystrophy trials Research Report on TUBB4A-Related Leukodystrophy
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| An Open-label, Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for TUBB4A-related Leukodystrophy nL-TUBB4-001 - Genetic Disease, TUBB4A-Related Leukodystrophy | Phase 1 / Phase 2 | Active not recruiting | University of California, San Diego | 1 | 2025-09-19 | 2025-10-29 |
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (1)
University of California, San Diego (1)
United States (1)