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

Trial ID: NCT06411288

Interventional - Phase 3 - Completed

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

Conditions

• DM1
• Myotonic Dystrophy
• Myotonic Dystrophy 1
• Myotonia
• Myotonic Dystrophy Type 1 (DM1)
• Dystrophy Myotonic
• Myotonic Disorders
• Steinert Disease
• Steinert
• Myotonic Muscular Dystrophy

Interventions

• AOC 1001 (del-desiran)
• Placebo

Eligibility

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

Sponsor

Avidity Biosciences, Inc.

Source: ClinicalTrials.gov

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