AMX0114 in Adult Participants With Amyotrophic Lateral Sclerosis (NCT06665165)
Phase 1, Randomized, Double-blind, Placebo-controlled, Multiple Ascending Dose Study to Evaluate Safety, Tolerability, PK and PD of Antisense Oligonucleotide AMX0114 Administered to Adult Participants With Amyotrophic Lateral Sclerosis
🩺 Plain-English Summary (8th-Grade Level)
This study is a placebo-controlled Phase I study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of the antisense oligonucleotide (ASO) AMX0114 in adult participants with amyotrophic lateral sclerosis (ALS).
Who can join: Review the eligibility criteria below.
Location: University of California, San Diego — La Jolla, California
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 (14 Sites)
- University of California, San Diego — La Jolla, California
- Georgetown University Hospital Pasquerilla Healthcare Center — Washington D.C., District of Columbia
- University of Florida — Gainesville, Florida
- Mayo Clinic in Florida — Jacksonville, Florida
- Orlando Regional Medical Center, Orlando Health Neuroscience Institute — Orlando, Florida
- Massachusetts General Hospital, Healey & AMG Center for ALS — Boston, Massachusetts
- Mayo Clinic in Rochester — Rochester, Minnesota
- Temple University of the Commonwealth System of Higher Education — Philadelphia, Pennsylvania
- Alliance for Multispecialty Research, LLC — Knoxville, Tennessee
- Houston Methodist Neurological Institute — Houston, Texas
- University of Calgary — Calgary, Alberta
- McMaster University — Hamilton, Ontario
- London Health Sciences Centre — London, Ontario
- McGill University Health Centre - Centre for Innovative Medicine — Montreal, Quebec
📞 Contact the Study Coordinator
Phone: (857) 320-6200
Email: clinicaltrials@amylyx.com
Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.
💰 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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Explore by condition: All ALS clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Contact the study coordinator using the phone/email above.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.