drugset / Trial / NCT06100276
Safety, Tolerability, and Exploratory Efficacy Study of Intrathecally Administered Gene Therapy AMT-162 in Adult Participants With SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS)
Non-randomizedSequentialOpen-labelTreatment
Summary
This is the study of AMT-162 in Participants with SOD1-ALS and is designed to evaluate the safety, tolerability, and exploratory efficacy of intrathecally administered gene therapy AMT-162. AMT-162-001 is a Phase 1/2, multi-center, single ascending dose study.
Timeline
- Start
- 2024-08-01
- Primary completion
- 2026-09-30
- Completion
- 2031-06-30
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | AMT-162 | Unknown | — | Intrathecal |