🏛️ Verified NIH Record • Updated 2026-01-20

Global Study of Del-desiran for the Treatment of DM1 (NCT06411288)

A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous AOC 1001 for the Treatment of Myotonic Dystrophy Type 1

🔵 Active, Not Recruiting Phase3 💊 Investigational Drug / Biologic Sponsor: Avidity Biosciences, Inc.

🩺 Plain-English Summary (8th-Grade Level)

A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous Delpacibart Etedesiran (abbreviated del-desiran, formerly AOC 1001) for the Treatment of Myotonic Dystrophy Type 1

Who can join: Review the eligibility criteria below.

Location: Stanford University — Stanford, California

Age range: 16 Years to 65 Years

🧪 Interventions in This Study

📋 "Do I Qualify?" — 1-Minute Self Screener

Instant Checklist

Full eligibility criteria are available on the official NIH protocol. View the criteria ↗

📍 All Participating Trial Locations (34 Sites)

Showing the first 25 of 34 sites.

  • Stanford University — Stanford, California
  • University of Colorado — Denver, Colorado
  • University of Florida — Gainesville, Florida
  • University Research Center of South Florida — Tampa, Florida
  • Indiana University (IU) — Indianapolis, Indiana
  • Kansas University Medical Center — Kansas City, Kansas
  • Kennedy Krieger Institute — Baltimore, Maryland
  • Beth Israel Deaconess Medical Center — Boston, Massachusetts
  • University of Minnesota — Minneapolis, Minnesota
  • University of Rochester Medical Center — Rochester, New York
  • Duke University Medical Center — Durham, North Carolina
  • Wake Forest — Winston-Salem, North Carolina
  • University of Cincinnati Gardner Neuroscience Institute — Cincinnati, Ohio
  • Ohio State University — Columbus, Ohio
  • University of Pennsylvania — Philadelphia, Pennsylvania
  • Houston Methodist Neurological Institute — Houston, Texas
  • Virginia Commonwealth University — Richmond, Virginia
  • University of Washington — Seattle, Washington
  • The Ottawa Hospital — Ottawa, Ontario
  • Montreal Neurological Institute — Montreal, Quebec
  • Aarhus University Hospital — Aarhus N,
  • Rigshospitalet — Copenhagen,
  • AP-HP Hopital Pitie-Salpetriere — Paris,
  • Klinikum der Ludwig-Maximilians-Universitaet Muenchen — Munich,
  • Fondazione Serena Onlus - Centro Clinico NeMO Milano — Milan,

💰 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

  1. Check the checklist above to see if you may qualify.
  2. Review the official NIH record below to find the coordinating site.
  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.
🏛️ View Official NIH Record ↗