Home › Trials › NCT06411288

Treatment of Myotonic Dystrophy Type 1 with Del-desiran

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

Phase 3 Interventional Avidity Biosciences, Inc. · NCT06411288

This study is testing if an intravenous treatment called del-desiran can help people with Myotonic Dystrophy Type 1 feel better compared to a placebo over a year.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment150 (estimated)
Ages16 Years to 65 Years
SexAll
SponsorAvidity Biosciences, Inc. Industry-sponsored
Locations34 sites (Stanford, California and 33 other locations)
Trial IDNCT06411288 on ClinicalTrials.gov

What this trial studies

This Phase 3 clinical trial evaluates the efficacy and safety of del-desiran, an intravenous treatment, for patients with Myotonic Dystrophy Type 1 (DM1). Participants will be randomly assigned to receive either del-desiran or a placebo every 8 weeks for a total of 7 doses over a 54-week treatment period. The study includes a screening phase of up to 6 weeks, followed by assessments at various intervals to monitor safety and effectiveness. An Independent Data Monitoring Committee will oversee the study to ensure participant safety and data integrity.

Who should consider this trial

Good fit: Ideal candidates are individuals with a clinical and genetic diagnosis of DM1, specifically those with a CTG repeat of 100 or more and the ability to walk independently.

Not a fit: Patients who are pregnant, breastfeeding, or have uncontrolled diabetes may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve the quality of life for patients with Myotonic Dystrophy Type 1.

How similar studies have performed: Previous studies on similar treatments for Myotonic Dystrophy have shown promise, but this specific approach with del-desiran is novel.

Eligibility criteria

Show full inclusion / exclusion criteria 
Key Inclusion Criteria:

* Clinical and genetic diagnosis (CTG repeat ≥ 100) of DM1
* Ability to walk independently (orthoses and ankle braces allowed) for at least 10 meters at screening

Key Exclusion Criteria:

* Breastfeeding, pregnancy, or intent to become pregnant during the study
* Unwilling or unable to comply with contraceptive requirements
* Abnormal lab values, conditions or diseases that would make the participant unsuitable for the study
* Diabetes that is not adequately controlled
* History of decompensated heart failure within 3 months of screening. Participants with preexisting pacemaker/ICD are not excluded.
* Body Mass Index \> 35 kg/m2 at Screening
* Recently treated with an investigational drug or biological agent
* Treatment with anti-myotonic medication within 5 half-lives or 14 days of baseline, whichever is longer, prior to baseline.

Note: Additional protocol defined Inclusion and Exclusion criteria apply

Where this trial is running

Stanford, California and 33 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions DM1Myotonic DystrophyMyotonic Dystrophy 1MyotoniaMyotonic Dystrophy Type 1Dystrophy MyotonicMyotonic DisordersSteinert Disease
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.