NCT07038200

A Study to Evaluate Del-brax (Also Referred to as AOC 1020) in Participants With FSHD

A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1020 for the Treatment of Facioscapulohumeral Muscular Dystrophy (FSHD)

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 200 (estimated)
• Sponsor: Avidity Biosciences, Inc. · Industry
• Sex: All
• Age: 16 Years – 70 Years
• Healthy volunteers: Not accepted

Summary

A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1020 for the Treatment of Facioscapulohumeral Muscular Dystrophy (FSHD)

Detailed description

The study consists of a Screening Period of up to 6 weeks and 72-week Treatment Period. The anticipated duration is approximately 78 weeks. Participants will be randomized to receive an intravenous infusion of either del-brax or placebo at the clinical study site every 6 weeks for a total of 13 doses. The final dose will occur at Week 72, followed by a final assessment at Week 78. After completion of the Week 78 visit, eligible participants will have the option to enroll into an open label extension (OLE) study, pending regulatory approval. Participants who decline participation in the OLE will be followed for a period of 12 weeks for safety. An Independent Data Monitoring Committee (IDMC) comprising members independent and external to the Sponsor will review safety, tolerability, and efficacy (as needed) data of this study at regular intervals.

Conditions

• Facioscapulohumeral Muscular Dystrophy
• FSHD
• FSHD - Facioscapulohumeral Muscular Dystrophy
• FSHD1
• FSHD2
• Fascioscapulohumeral Muscular Dystrophy
• Fascioscapulohumeral Muscular Dystrophy Type 1
• Fascioscapulohumeral Muscular Dystrophy Type 2
• Facioscapulohumeral Muscular Dystrophy 1
• Facioscapulohumeral Dystrophy
• Facio-Scapulo-Humeral Dystrophy
• Facioscapulohumeral Muscular Dystrophy 2
• Facioscapulohumeral Muscular Dystrophy Type 1 (FSHD1)
• FSH Muscular Dystrophy
• Landouzy Dejerine Dystrophy
• Landouzy-Dejerine Muscular Dystrophy
• Landouzy-Dejerine Syndrome

Interventions

Type	Name	Description
DRUG	AOC-1020	Placebo
DRUG	Placebo	Placebo will be administered by intravenous (IV) infusion.

Timeline

• Start date: 2025-06-10
• Primary completion: 2028-05-01
• Completion: 2028-07-01
• First posted: 2025-06-26
• Last updated: 2026-01-20

Locations

22 sites across 3 countries: United States, Canada, Japan

Regulatory

• FDA-regulated drug study