AOC 1020 for Facioscapulohumeral Muscular Dystrophy
(FORTITUDE-OLE Trial)

Recruiting in Palo Alto (17 mi)

+16 other locations

Age: Any Age

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

Approved in 1 Jurisdiction

Trial Summary

What is the purpose of this trial?A Phase 2 Open-label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of AOC 1020 Administered Intravenously to Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)

Eligibility Criteria

This trial is for adults with Facioscapulohumeral Muscular Dystrophy (FSHD), a type of muscular dystrophy that affects the muscles of the face, shoulder blades, and upper arms. Participants should have completed prior studies with AOC 1020 or meet specific health criteria.

Inclusion Criteria

Ability to provide written informed consent and comply with all study requirements

Completion of AOC 1020-CS1 with no significant tolerability issues and satisfactory compliance with protocol requirements

Exclusion Criteria

Pregnancy, intent to become pregnant during the clinical study, or active breastfeeding

I don't have new or worsening conditions that would stop me from completing the study.

I cannot or will not follow the birth control rules during the AOC 1020-CS2 study.

Participant Groups

The study tests long-term safety and effectiveness of AOC 1020 when given through an IV. It's an open-label extension, meaning everyone knows they're getting AOC 1020 and there's no placebo group.

1Treatment groups

Experimental Treatment

Group I: AOC 1020 RegimenExperimental Treatment1 Intervention

AOC 1020 Dose Regimen; Sixteen doses administered intravenously over 22 months. All participants will receive AOC 1020 at a dose level of 2mg/kg.

AOC 1020 is already approved in United States for the following indications:

🇺🇸 Approved in United States as AOC 1020 for: