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Enrolling by invitationPhase 3

Global Open-Label Extension 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, Los Angeles, Minneapolis, Philadelphia, Richmond, Rochester, Seattle, Stanford, Tampa, and Winston-Salem, United StatesSee the 19 sitesAlso in 9 other countries, 16 sites
Trial code
Status
Enrolling by invitation
Phase
Phase 3
People taking part
199 people, planned
Started
26 Jun 2025
Expected to end
Oct 2030
Type of study
Interventional
Treatments tested
  • Del-desiran (AOC 1001)
Record updated
19 August 2026

Who can take part

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

The registry criteria, word for word

Show all 13 criteriaShow less

Who can take part

  • Completion of any prior AOC 1001 studies with satisfactory completion of dosing and follow-up assessments and satisfactory compliance with the protocol requirements of the parent study, as determined by the Investigator
  • Clinical and genetic diagnosis of DM1 and clinically significant hand myotonia

Who cannot take part

  • Recently treated with an investigational drug or biological agent; Note: Additional protocol defined Inclusion and Exclusion criteria apply
  • Any new conditions or worsening of existing condition that in the opinion of the Investigator would make the participant unsuitable for the study; FIXED-DOSE PK COHORT
  • 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 to comply with contraceptive requirements
  • Breastfeeding, pregnancy, or intent to become pregnant during the study
  • Unwilling to comply with contraceptive requirements
  • Body Mass Index > 35 kg/m2 at Screening

Other criteria

  • FROM A PARENT STUDY

Where in United States

Also in 9 other countries, 16 sites

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

About the trial

What the trial wants to find out

A Global Phase 3 Open-Label Extension Study to Assess the Long-Term Safety, Tolerability, and Efficacy of Intravenous Delpacibart Etedesiran (abbreviated del-desiran, formerly AOC 1001) for the Treatment of Myotonic Dystrophy Type 1

The group in the trial

  • Del-desiranTreatment being tested

    Del-desiran (AOC 1001) will be administered seven times per year for up to 4 years.

    Treatment: Del-desiran (AOC 1001)

Questions to ask the study team

  1. How often do I need to come in, and for how long each time?
  2. Are travel costs paid?
  3. Can I keep my own doctor?
  4. What happens if I want to stop?

Contact the study team: see the registry record → Contacts and Locations

Print a page for my doctor

Data and sources

Registry record
Listed on ClinicalTrials.gov.
Official title
A Global Phase 3 Open-Label Extension Study to Assess the Long-Term Safety, Tolerability, and Efficacy of Intravenous AOC 1001 for the Treatment of Myotonic Dystrophy Type 1
Record updated
19 August 2026