AOC 1001 for Myotonic Dystrophy
(MARINA-OLE Trial)

Recruiting in Palo Alto (17 mi)

+7 other locations

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: Avidity Biosciences, Inc.

No Placebo Group

Prior Safety Data

Trial Summary

What is the purpose of this trial?This trial is testing a new medicine called AOC 1001 to see if it is safe and effective for adults with a muscle disease called Myotonic Dystrophy Type 1. The medicine is given through an IV, and researchers want to know if it helps muscles work better.

Eligibility Criteria

This trial is for adults with Myotonic Dystrophy Type 1 who completed the MARINA study without major issues. They must be able to continue using contraception and not be pregnant or breastfeeding. Those with new or worsening conditions that could affect their participation are excluded.

Inclusion Criteria

Completion of AOC 1001-CS1 (MARINA) study with satisfactory compliance and no significant tolerability issues

Exclusion Criteria

Unwilling or unable to continue to comply with contraceptive requirements

Pregnancy, intent to become pregnant, or active breastfeeding

Any new conditions or worsening of existing conditions that in the opinion of the investigator or sponsor would make the participant unsuitable for the study or could interfere with participation or completion of the study

Participant Groups

The trial is an extension of a previous study, testing multiple doses of AOC 1001 administered intravenously against a placebo to assess its safety, tolerability, effectiveness, and how it's processed by the body in DM1 patients.

2Treatment groups

Experimental Treatment

Group I: AOC 1001 (with Placebo at Day 43)Experimental Treatment2 Interventions

AOC 1001 will initially be administered quarterly. On Day 43 patients will receive an additional dose. Treatment assignment will be based on treatment received in AOC 1001-CS1. If participant received AOC 1001 on Day 43 in AOC 1001-CS1, participant will receive blinded placebo treatment on Day 43 in AOC 1001-CS2. Beginning in September 2024, AOC 1001 will be administered every 8 weeks.

Group II: AOC 1001Experimental Treatment1 Intervention

AOC 1001 will initially be administered quarterly. On Day 43 patients will receive an additional dose. Treatment assignment will be based on treatment received in AOC 1001-CS1. If participant did not receive AOC 1001 on Day 43 in AOC 1001-CS1, participant will receive AOC 1001 treatment on Day 43 in AOC 1001-CS2. Beginning in September 2024, AOC 1001 will be administered every 8 weeks.