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)

NCT06100276 ↗

Phase 1/2 Active not recruiting 20 enrolled UniQure Biopharma B.V.
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

EvaluationDrugModalityDoseRoute
Subject AMT-162 Unknown — Intrathecal