drugset / Trial / NCT05791864
A First-in-Human, Open-Label, Dose-Escalation Study to Evaluate the Safety and Tolerability of Gene Therapy With TTX-381 for the Ocular Manifestations Associated With Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) Disease
Non-randomizedSequentialSingle-blindTreatment
Summary
This is a first-in-human, open-label, single ascending dose study of TTX-381 for the treatment of ocular manifestations of CLN2 (Batten disease).
Timeline
- Start
- 2023-05-17
- Primary completion
- 2026-07-30
- Completion
- 2031-07-30
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | TTX-381 | Unknown | 2e+10 vg | Other |
| Subject | TTX-381 | Unknown | 6e+10 vg | Other |