🏛️ Verified NIH Record • Updated 2026-04-09
AMX0035 in Adult Patients With Wolfram Syndrome (NCT05676034)
A Phase II Study of Safety and Efficacy of AMX0035 in Adult Patients With Wolfram Syndrome
🔵 Active, Not Recruiting Phase2 💊 Investigational Drug / Biologic Sponsor: Amylyx Pharmaceuticals Inc.
🩺 Plain-English Summary (8th-Grade Level)
This study is an open label Phase II study to evaluate the safety and efficacy of AMX0035 in adults with Wolfram syndrome.
Who can join: Review the eligibility criteria below.
Location: Washington University — St Louis, Missouri
Age range: 17 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 (1 Sites)
- Washington University — St Louis, Missouri
💰 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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💡 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.