Clinical Trial

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

Active, Not Recruiting Phase 1/2
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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.
Protocol Amendment History 12 amendments
This ClinicalTrials.gov record has been amended 12 times since 2023-10-20; most recent amendment 2025-10-20.
Status change: Recruiting → Active, Not Recruiting 2025-10-20
Status change: Not Yet Recruiting → Recruiting 2024-08-01
Trial Details
NCT Number NCT06100276
Lead Sponsor UniQure Biopharma B.V.
Conditions Amyotrophic Lateral Sclerosis
Enrollment 20 participants
Start Date 2024-08-01
Primary Completion 2026-09-30 (estimated)
Study Completion 2031-06-30 (estimated)
Updated on ClinicalTrials.gov 2025-10-22