A Global Phase 3 OLE Study to Assess AOC 1001 for the Treatment of Myotonic Dystrophy Type 1
Research type
Research Study
Full title
A Global Phase 3 Open-Label Extension Study to Assess the Long-Term Safety, Tolerability, and Efficacy of Intravenous AOC 1001 for the Treatment of Myotonic Dystrophy Type 1
IRAS ID
1013077
Contact name
Jazmine Cruz-Maddox
Contact email
Sponsor organisation
Avidity Biosciences, Inc.
Clinicaltrials.gov Identifier
Research summary
A Global Phase 3 Open-Label Extension Study to Assess AOC 1001 in Patients with DM1
Study Objective
The purpose of this Phase 3 open-label extension study is to continue evaluating the safety, tolerability, and efficacy of AOC 1001 long-term in DM1 participants who have completed another study assessing AOC 1001.
Study Design
This Phase 3 study is planned to be conducted in multiple different clinics in several countries in approximately 217 participants with DM1. All participants will receive AOC 1001 for up to approximately 4 years. Participants may be eligible for the study if they have satisfactorily completed any prior AOC 1001 studies (e.g., MARINA-OLE® Phase 2 study, HARBOR™ Phase 3 study) and are willing and able to perform the tests in the study as described in the consent form. Certain medical conditions may prevent a participant from participating. Pregnant people, people planning to become pregnant, or people who are breastfeeding will not be included. Study staff will explain the details of the study to prospective participants.
AOC 1001 will be given by intravenous infusion (through a small tube inserted with a needle directly into a vein in the arm). All participants will receive a dose of AOC 1001 once approximately every 8 weeks.
Throughout the study, participants will be asked to perform tests such as those that measure hand function, grip strength, and the strength of arm and leg muscles. Participants will also answer questions about their health and day-to-day activities while enrolled in the study.
Study participants will provide blood samples to study staff according to the study schedule to assess safety based on laboratory analyses.REC name
Wales REC 5
REC reference
25/WA/0313
Date of REC Opinion
18 Nov 2025
REC opinion
Further Information Favourable Opinion