Study of ATX-01 in Participants With DM1 (NCT06300307)
A Phase 1/2a Double-Blind, Placebo-controlled, Single- and Multiple Ascending Dose Study to Assess the Safety, Tolerability, PK, PD and Efficacy of IV Administration of ATX-01 In Male and Female Participants Aged 18 to 64 With Classic DM1
🩺 Plain-English Summary (8th-Grade Level)
The goal of this clinical trial is to test ATX-01 in participants with myotonic dystrophy type 1 (DM1). The main question it aims to answer is if ATX-01 is safe and well tolerated. The trial will compare the safety and tolerability of ATX-01 and a matching placebo. There will be a single-ascending dose part of the trial and a multiple-ascending dose part. In the single-ascending dose, participants will receive one dose of ATX-01 or placebo. In the multiple-ascending dose part, participants will receive three doses of ATX-01 or placebo. ATX-01 is a novel anti-miR (synthetic single stranded oligonucleotide) that inhibits a microRNA called miR-23b.
Who can join: Review the eligibility criteria below.
Location: UCLA — Los Angeles, California
Age range: 18 Years to 64 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)
- UCLA — Los Angeles, California
- University of Florida — Gainesville, Florida
- University of Iowa Health Care - Department of Neurology — Iowa City, Iowa
- University of Kansas Medical Center, Department of Neurology — Fairway, Kansas
- Virginia Commonwealth University — Richmond, Virginia
- Centre Intégré Universitaire de Santé et Services Sociaux du Saguenay-Lac-St-Jean — Chicoutimi, Quebec
- Institute of Myology — Paris,
- The NeMO Clinical Center in Milan, Neurorehabilitation Unit, University of Milan — Milan,
- Fondazione Policlinico A. Gemelli- IRCCS — Rome,
- Radboudumc — Nijmegen,
- Hospital Universitario Donostia — Donostia / San Sebastian,
- St. George's University Hospital — London,
📞 Contact the Study Coordinator
Phone: +34676229821
Email: clinical@arthexbiotech.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.
More studies in Myotonic Dystrophy 1
- Study of SRP-1003 in Participants With Type 1 Myotonic Dystrophy — Liverpool, New South Wales
- A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1 — Calgary, Alberta
- Estab Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1) — La Jolla, California
- An Open-Label Extension Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1 (FREEDOM-OLE) — Calgary, Alberta
Explore by condition: All Myotonic Dystrophy 1 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.