Overview

Safety, Tolerability, and Exploratory Efficacy Study of Intrathecally Administered Gene Therapy AMT-162 in Adult Participants With SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS)

Status:
Not yet recruiting
Trial end date:
2031-03-30
Target enrollment:
Participant gender:
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.
Phase:
Phase 1/Phase 2
Details
Lead Sponsor:
UniQure Biopharma B.V.