NCT06547216

Phase 2 Open-label Extension Study of AOC 1020 in Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)

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)

• Status: Enrolling By Invitation
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 84 (estimated)
• Sponsor: Avidity Biosciences, Inc. · Industry
• Sex: All
• Age: 16 Years – 70 Years
• Healthy volunteers: Not accepted

Summary

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)

Detailed description

This study will continue to evaluate the safety, tolerability, and efficacy of AOC 1020 in participants who were treated in the randomized, placebo-controlled, Phase 1/2 AOC 1020-CS1 clinical study. Participants from AOC 1020-CS1 are eligible to enroll in AOC 1020-CS2 if they have satisfactorily completed AOC 1020-CS1. All participants who enroll in AOC 1020-CS2 will receive AOC 1020 regardless of whether they received AOC 1020 or placebo in AOC 1020-CS1. Dosing will occur every 6 to 7 weeks (twice quarterly). The total duration of active treatment in AOC 1020-CS2 is approximately 22 months. Once participants have completed active treatment, they will be followed through a 12-week safety follow-up period to monitor for adverse events (AEs), concomitant medications, and pregnancy status (as applicable). Total duration on study is up to approximately 25 months (2 years 1 month), excluding the potential 8-week screening period.

Conditions

• FSHD
• FSHD1
• FSHD2
• FMD
• FMD2
• Fascioscapulohumeral Muscular Dystrophy
• Fascioscapulohumeral Muscular Dystrophy Type 1
• Fascioscapulohumeral Muscular Dystrophy Type 2
• Dystrophies, Facioscapulohumeral Muscular
• Dystrophy, Facioscapulohumeral Muscular
• Facioscapulohumeral Muscular Dystrophy 1
• Facioscapulohumeral Muscular Dystrophy 2
• Facio-Scapulo-Humeral Dystrophy
• Atrophy, Facioscapulohumeral
• Atrophies, Facioscapulohumeral
• Facioscapulohumeral Atrophy
• Muscular Dystrophies
• Muscular Dystrophy, Facioscapulohumeral
• FSH Muscular Dystrophy
• Landouzy Dejerine Dystrophy
• Landouzy-Dejerine Muscular Dystrophy
• Dystrophies, Landouzy-Dejerine
• Dystrophy, Landouzy-Dejerine
• Landouzy-Dejerine Syndrome
• Muscular Dystrophy, Landouzy Dejerine
• Progressive Muscular Dystrophy
• FSH

Interventions

Type	Name	Description
DRUG	AOC 1020	AOC 1020 will be administered via intravenous (IV) infusion

Timeline

• Start date: 2024-07-25
• Primary completion: 2028-04-01
• Completion: 2028-04-01
• First posted: 2024-08-09
• Last updated: 2024-12-20

Locations

17 sites across 3 countries: United States, Canada, United Kingdom

Regulatory

• FDA-regulated drug study