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

Phase 3

Recruiting

• 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
• Interventions: Drug: AOC-1020; Drug: Placebo

• Registration Number: NCT07038200
• Lead Sponsor: Avidity Biosciences, Inc.

Brief 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.

Study Timeline

• Study Start: 2025-06-10
• Study First Submitted: 2025-06-13
• Study First Submitted QC: 2025-06-23
• Study First Posted: 2025-06-26
• Status Verified: 2025-07
• Last Update Submitted: 2025-07-25
• Last Update Posted: 2025-07-29
• Primary Completion: 2028-05
• Study Completion: 2028-07-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 200

Inclusion Criteria

• Clinical and genetic diagnosis of FSHD1 or FSHD2
• Ability to walk independently at pre-specified walking speed (orthoses and ankle braces allowed) for at least 10 meters at screening
• Adequate muscle strength based on QMT composite score

Exclusion Criteria

• Breastfeeding, pregnancy, or intent to become pregnant during the study
• Unwilling or unable to comply with contraceptive requirements
• Abnormal lab values, conditions or diseases that would make the participant unsuitable for the study
• Blood Pressure > 140/90 mmHg at Screening
• Treatment with another investigational drug or biological agent within 1 month of Screening or 5 half-lives of the drug, whichever is longer
• Treatment with an oligonucleotide within 9 months of Screening

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
del-brax	AOC-1020	Del-brax (AOC 1020) will be administered 13 times
placebo	Placebo	Saline will be administered 13 times

Primary Outcome Measures

Name	Time	Method
Quantitative Muscle Testing (QMT) composite score	Baseline through Week 78

Secondary Outcome Measures

Name	Time	Method
10-Meter Walk/Run Test (10MWRT)	Baseline through Week 78
Timed Up-and-Go (TUG)	Baseline through Week 78
Patient-Reported Outcomes Measurement Information System (PROMIS) Physical Function Item Bank v2.0-Physical Function-Short Form 20a Questionnaire	Baseline through Week 78
Worst Pain Numeric Rating Scale (NRS)	Baseline through Week 78
Patient Global Impression of Severity/Change (PGI-S/PGI-C)	Baseline through Week 78
DUX-4 Regulated Circulating Biomarker of FSHD Disease Biology	Baseline through Week 78
Circulating Creatine Kinase (CK)	Baseline through Week 78

Trial Locations

Locations (13)

University of California Irvine; 🇺🇸 Orange, California, United States

Stanford University; 🇺🇸 Palo Alto, California, United States

University of Colorado; 🇺🇸 Denver, Colorado, United States

University of Florida; 🇺🇸 Gainesville, Florida, United States

University of Iowa; 🇺🇸 Iowa City, Iowa, United States

Kansas University Medical Center; 🇺🇸 Kansas City, Kansas, United States

Kennedy Krieger Institute; 🇺🇸 Baltimore, Maryland, United States

University of Massachusetts; 🇺🇸 Worcester, Massachusetts, United States

University of Rochester Medical Center; 🇺🇸 Rochester, New York, United States

Duke University; 🇺🇸 Durham, North Carolina, United States

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