Safety, Tolerability, and Exploratory Efficacy Study of Intrathecally Administered Gene Therapy AMT-162 in Adult Participants With SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS) (NCT06100276)
A Phase 1/2, Multicenter, Single Ascending Dose Study to Evaluate the Safety, Tolerability, and Exploratory Efficacy of Intrathecally Administered Gene Therapy AMT-162 in Adult Participants With SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS).
🩺 Plain-English Summary (8th-Grade Level)
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.
Who can join: Review the eligibility criteria below.
Location: Barrow Neurological Institute — Phoenix, Arizona
Age range: 18 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (12 Sites)
- Barrow Neurological Institute — Phoenix, Arizona
- University of California Irvine — Irvine, California
- California Pacific Medical Center — San Francisco, California
- Mayo Clinic Florida — Jacksonville, Florida
- Winship Cancer Institute of Emory University — Atlanta, Georgia
- Northwestern University Feinberg School of Medicine — Chicago, Illinois
- University of Kansas Medical Center — Fairway, Kansas
- Massachusetts General Hospital, Sean M. Healey and AMG Center for ALS Research — Boston, Massachusetts
- Mayo Clinic Rochester — Rochester, Minnesota
- Columbia University Irving Medical Center — New York, New York
- University of Pennsylvania School of Medicine — Philadelphia, Pennsylvania
- Norrlands Universitetssjukhus — Umeå, Vasterbottens Ian
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
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- Rho Kinase Inhibitor in Amyotrophic Lateral Sclerosis (REAL) — Phoenix, Arizona
- TRIAL READY (Clinical Trial Readiness) — Miami, Florida
- Phase III Trial of AMX0035 for Amyotrophic Lateral Sclerosis Treatment — Phoenix, Arizona
- Personalized Antisense Oligonucleotide for a Single Participant With CHCHD10 ALS — New York, New York
Explore by condition: All Amyotrophic Lateral Sclerosis clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.