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CompletedPhase 3

Global Study of Del-desiran for the Treatment of DM1

Key facts

Who runs it
Avidity Biosciences, Inc.
Where
Baltimore, Boston, Cincinnati, Columbus, Denver, Durham, Gainesville, Houston, Indianapolis, Kansas City, Minneapolis, Philadelphia, Richmond, Rochester, Seattle, Stanford, Tampa, and Winston-Salem, United StatesSee the 18 sitesAlso in 9 other countries, 16 sites
Trial code
Status
Completed
Phase
Phase 3
People taking part
159 people enrolled
Started
30 May 2024
Ended
29 Jul 2026
Type of study
Interventional, 2 groupsOne group gets a placebo (a dummy treatment), for comparison.
Treatments tested
  • AOC 1001 (del-desiran)
  • Placebo
Record updated
1 September 2026

Who can take part

Age
16 years to 65 years
Sex
Women and men
Healthy volunteers
Cannot take part

The registry criteria, word for word

Show all 10 criteriaShow less

Who can take part

  • 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

Who cannot take part

  • 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
  • Diabetes that is not adequately controlled
  • History of decompensated heart failure within 3 months of screening. Participants with preexisting pacemaker/ICD are not excluded
  • Abnormal lab values, conditions or diseases that would make the participant unsuitable for the study
  • Breastfeeding, pregnancy, or intent to become pregnant during the study
  • Unwilling or unable to comply with contraceptive requirements
  • Body Mass Index > 35 kg/m2 at Screening

Where in United States

Also in 9 other countries, 16 sites

  • Kennedy Krieger InstituteBaltimore, United States
  • Beth Israel Deaconess Medical CenterBoston, United States
  • University of Cincinnati Gardner Neuroscience InstituteCincinnati, United States
  • Ohio State UniversityColumbus, United States
  • University of ColoradoDenver, United States
  • Duke University Medical CenterDurham, United States
  • University of FloridaGainesville, United States
  • Houston Methodist Neurological InstituteHouston, United States
  • Indiana University (IU)Indianapolis, United States
  • Kansas University Medical CenterKansas City, United States
  • University of MinnesotaMinneapolis, United States
  • University of PennsylvaniaPhiladelphia, United States
  • Virginia Commonwealth UniversityRichmond, United States
  • University of Rochester Medical CenterRochester, United States
  • University of WashingtonSeattle, United States
  • Stanford UniversityStanford, United States
  • University Research Center of South FloridaTampa, United States
  • Wake ForestWinston-Salem, United States

About the trial

What the trial wants to find out

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

The 2 groups in the trial

  • Del-desiranTreatment being tested

    Del-desiran (AOC 1001) will be administered seven times

    Treatment: AOC 1001 (del-desiran)

  • PlaceboPlacebo, for comparison

    Saline will be administered seven times

Data and sources

Registry record
Listed on ClinicalTrials.gov.
Official title
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
Record updated
1 September 2026