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Global Study of Del-desiran for the Treatment of DM1

Myotonic Dystrophy 1 | Steinert Disease | Myotonic Dystrophy | DM1 | Myotonic Muscular Dystrophy | Myotonic...

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

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Participation Requirements

  • Sex:

    ALL
  • Eligible Ages:

    16 to 65

Participation 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

Study Location

The Ottawa Hospital
The Ottawa Hospital
Ottawa, Ontario
Canada

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CSSS de Chicoutimi
CSSS de Chicoutimi
Chicoutimi, Quebec
Canada

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Montreal Neurological Institute
Montreal Neurological Institute
Montreal, Quebec
Canada

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Study Sponsored By
Avidity Biosciences, Inc.
Participants Required
More Information
Study ID: NCT06411288