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Global Open-Label Extension Study of Del-desiran for the Treatment of DM1
Global Open-Label Extension Study of Del-desiran for the Treatment of DM1

NCT07008469

Enrolling By InvitationPhase 3

Sponsor: Avidity Biosciences, Inc.

Conditions: Myotonic Dystrophy Type 1, DM1, Myotonic Dystrophy, Myotonia, Myotonic Dystrophy 1

Interventions: Del-desiran (AOC 1001)

Countries: United States, Canada, Denmark, France, Germany, Italy, Japan, Netherlands

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

Eligibility overview

Sex: ALL

Age: 16 Years to —

Healthy volunteers: No

Study type: INTERVENTIONAL

Eligibility criteria
FROM A PARENT STUDY

Key Inclusion Criteria:

* 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.

Key Exclusion Criteria:

* Breastfeeding, pregnancy, or intent to become pregnant during the study.
* Unwilling to comply with contraceptive requirements.
* 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

Key Inclusion Criteria:

* Clinical and genetic diagnosis of DM1 and clinically significant hand myotonia.

Key Exclusion Criteria:

* Breastfeeding, pregnancy, or intent to become pregnant during the study
* Unwilling 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

Note: Additional protocol defined Inclusion and Exclusion criteria apply
Locations (35)
  • Los Angeles, California, United States
  • Stanford, California, United States
  • Denver, Colorado, United States
  • Gainesville, Florida, United States
  • Tampa, Florida, United States
  • Indianapolis, Indiana, United States
  • Kansas City, Kansas, United States
  • Baltimore, Maryland, United States
  • Boston, Massachusetts, United States
  • Minneapolis, Minnesota, United States
  • Rochester, New York, United States
  • Durham, North Carolina, United States
  • Winston-Salem, North Carolina, United States
  • Cincinnati, Ohio, United States
  • Columbus, Ohio, United States
  • Philadelphia, Pennsylvania, United States
  • Houston, Texas, United States
  • Richmond, Virginia, United States
  • Seattle, Washington, United States
  • Ottawa, Ontario, Canada
  • Montreal, Quebec, Canada
  • Aarhus, Denmark
  • Copenhagen, Denmark
  • Paris, France
  • München, Germany
  • Milan, Italy
  • Aomori, Aomori, Japan
  • Osaka, Japan
  • Osaka, Japan
  • Tokyo, Japan
  • + 5 more on CT.gov