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

Launched by AVIDITY BIOSCIENCES, INC. · May 29, 2025

Keywords

ClinConnect Summary

This clinical trial is studying a treatment called Del-desiran for people with Myotonic Dystrophy Type 1 (DM1), a genetic condition that affects muscle function and can lead to weakness and other health issues. The trial is in its third phase and aims to evaluate how safe and effective this treatment is over a longer period. It is an open-label study, meaning that both the participants and the researchers will know that Del-desiran is being given.

To be eligible for this study, participants should have completed earlier studies involving the same treatment and followed the study rules. They must also be between the ages of 58 and 43, and it’s important that they are not pregnant, breastfeeding, or planning to become pregnant during the trial. If someone has new health issues or their current health problems worsen, they may not be able to participate. Those who take part can expect to receive regular doses of Del-desiran and will be monitored closely for any side effects and how well the treatment works. This trial is not yet recruiting participants, so interested individuals will need to wait for further announcements.

Gender

ALL

Eligibility criteria

• 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.