๐Ÿ›๏ธ Verified NIH Record โ€ข Updated 2026-07-09

Personalized Antisense Oligonucleotide Therapy for A Single Participant With CHCHD10 ALS (NCT06392126)

An Open-label Study of an Experimental Antisense Oligonucleotide Treatment for a Single Participant With Amyotrophic Lateral Sclerosis (ALS) Due to a Pathogenic Variant in CHCHD10

๐Ÿ”ต Active, Not Recruiting Phase1 / Phase2 ๐Ÿ’Š Investigational Drug / Biologic Sponsor: n-Lorem Foundation

๐Ÿฉบ Plain-English Summary (8th-Grade Level)

This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with amyotrophic lateral sclerosis (ALS) due to a pathogenic variant in CHCHD10

Who can join: Review the eligibility criteria below.

Location: Mayo Clinic โ€” Jacksonville, Florida

Age range: 48 Years

๐Ÿงช Interventions in This Study

๐Ÿ“‹ "Do I Qualify?" โ€” 1-Minute Self Screener

Instant Checklist

Full eligibility criteria are available on the official NIH protocol. View the criteria โ†—

๐Ÿ“ All Participating Trial Locations (1 Sites)

  • Mayo Clinic โ€” Jacksonville, Florida

๐Ÿ“ž Contact the Study Coordinator

Phone: (800) 664-4580

Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.

๐Ÿ’ฐ 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 ยง 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.

๐Ÿ’ก How to Participate

  1. Check the checklist above to see if you may qualify.
  2. Contact the study coordinator using the phone/email above.
  3. Ask about the visit schedule, what is covered at no cost, and any travel support.
  4. Confirm with your own doctor before making a decision.
๐Ÿ›๏ธ View Official NIH Record โ†—