Global Study of Del-desiran for the Treatment of DM1

Launched by AVIDITY BIOSCIENCES, INC. · May 8, 2024

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called del-desiran for people with Myotonic Dystrophy Type 1 (DM1), a genetic condition that affects muscles and can cause weakness and stiffness. The trial is in its final phase and is designed to see if this treatment is safe and effective. It involves giving participants either the new treatment or a placebo (a treatment that doesn’t contain the active drug) through an intravenous (IV) line, which means it will be administered directly into the bloodstream.

To be eligible for the study, participants should have a confirmed diagnosis of DM1 with a specific genetic marker and be able to walk independently for a short distance. The trial is open to adults of all genders, aged between 18 and 80 years. Participants will need to meet certain health criteria and may not qualify if they have specific medical conditions or are pregnant. Those who join can expect regular check-ups and monitoring throughout the study to help ensure their safety. This trial is important as it could lead to new treatment options for DM1 patients, helping improve their quality of life.

Gender

ALL

Eligibility 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