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

NCT ID: NCT07038200

Last Updated: 2026-01-20

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

200 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-06-10

Study Completion Date

2028-07-31

Brief Summary

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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

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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

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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

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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del-brax

Del-brax (AOC 1020) will be administered 13 times

Group Type EXPERIMENTAL

AOC-1020

Intervention Type DRUG

Placebo

placebo

Saline will be administered 13 times

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Placebo will be administered by intravenous (IV) infusion.

Interventions

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AOC-1020

Placebo

Intervention Type DRUG

Placebo

Placebo will be administered by intravenous (IV) infusion.

Intervention Type DRUG

Other Intervention Names

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del-brax saline

Eligibility Criteria

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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
Minimum Eligible Age

16 Years

Maximum Eligible Age

70 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Avidity Biosciences, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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University of California Irvine

Orange, California, United States

Site Status RECRUITING

Stanford University

Palo Alto, California, United States

Site Status RECRUITING

University of Colorado

Denver, Colorado, United States

Site Status RECRUITING

University of Florida

Gainesville, Florida, United States

Site Status RECRUITING

University of Iowa

Iowa City, Iowa, United States

Site Status RECRUITING

Kansas University Medical Center

Kansas City, Kansas, United States

Site Status RECRUITING

Kennedy Krieger Institute

Baltimore, Maryland, United States

Site Status RECRUITING

University of Massachusetts

Worcester, Massachusetts, United States

Site Status RECRUITING

University of Rochester Medical Center

Rochester, New York, United States

Site Status RECRUITING

Duke University

Durham, North Carolina, United States

Site Status RECRUITING

Ohio State University

Columbus, Ohio, United States

Site Status RECRUITING

University of Pennsylvania

Philadelphia, Pennsylvania, United States

Site Status RECRUITING

University of Texas Health Science Center at San Antonio

San Antonio, Texas, United States

Site Status RECRUITING

Virginia Commonwealth University

Richmond, Virginia, United States

Site Status RECRUITING

University of Calgary - Cumming School of Medicine

Calgary, Alberta, Canada

Site Status RECRUITING

University of Alberta Hospital

Edmonton, Alberta, Canada

Site Status NOT_YET_RECRUITING

The Ottawa Hospital

Ottawa, Ontario, Canada

Site Status RECRUITING

Genge Partners Inc

Montreal, Quebec, Canada

Site Status NOT_YET_RECRUITING

National Hospital Organization Osaka Toneyama Medical Center

Osaka, , Japan

Site Status RECRUITING

The University of Osaka Hospital

Osaka, , Japan

Site Status RECRUITING

National Hospital Organization Sendai Nishitaga Hospital

Sendai, , Japan

Site Status RECRUITING

National Center of Neurology and Psychiatry

Tokyo, , Japan

Site Status RECRUITING

Countries

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United States Canada Japan

Central Contacts

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Avidity Biosciences, Inc.

Role: CONTACT

858-771-7038

Related Links

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Other Identifiers

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AOC 1020-CS3

Identifier Type: -

Identifier Source: org_study_id

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