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

NCT ID: NCT06547216

Last Updated: 2024-12-20

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

ENROLLING_BY_INVITATION

Clinical Phase

PHASE2

Total Enrollment

84 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-07-25

Study Completion Date

2028-04-30

Brief Summary

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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

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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

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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

Keywords

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FORTITUDE Avidity Avidity Biosciences AOC 1020 FORTITUDE-OLE FORTITUDE OLE

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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AOC 1020 Regimen

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

Group Type EXPERIMENTAL

AOC 1020

Intervention Type DRUG

AOC 1020 will be administered via intravenous (IV) infusion

Interventions

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AOC 1020

AOC 1020 will be administered via intravenous (IV) infusion

Intervention Type DRUG

Other Intervention Names

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delpacibart braxlosiran del-brax

Eligibility Criteria

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Inclusion Criteria

1. Ability to provide written informed consent (signed and dated) and any authorizations required by local law and be willing and able to comply with all study requirements. When enrolling participants who are minors, it is necessary to also obtain consent from a legally designated representative and the participant will receive information in a way adapted to their age and mental maturity.
2. Completion of AOC 1020-CS1 with the following as judged by the Investigator and Sponsor:

1. No significant tolerability issues
2. Satisfactory compliance with the AOC 1020-CS1 protocol requirements

Exclusion Criteria

1. Pregnancy, intent to become pregnant during the clinical study, or active breastfeeding.
2. Unwilling or unable to continue to comply with contraceptive requirements for the length of AOC 1020-CS2.
3. Any new conditions or worsening of existing condition(s) that in the opinion of the Investigator or Sponsor would make the participant unsuitable for the study or could interfere with participation or completion of the study. (Note: FSHD progression is not exclusionary, even if the participant no longer has the ability to walk 10 meters without a walker or two canes.)
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

Principal Investigators

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Amy Halseth, Ph.D.

Role: STUDY_DIRECTOR

Avidity Biosciences, Inc.

Locations

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University of California Los Angeles

Los Angeles, California, United States

Site Status

Stanford University

Palo Alto, California, United States

Site Status

University of California San Diego

San Diego, California, United States

Site Status

University of Colorado

Denver, Colorado, United States

Site Status

University of Florida

Gainesville, Florida, United States

Site Status

Rare Disease Research

Atlanta, Georgia, United States

Site Status

Kansas University Medical Center

Kansas City, Kansas, United States

Site Status

University of Rochester Medical Center

Rochester, New York, United States

Site Status

Duke University

Durham, North Carolina, United States

Site Status

Ohio State University

Columbus, Ohio, United States

Site Status

University of Pennsylvania

Philadelphia, Pennsylvania, United States

Site Status

University of Texas Southwestern

Dallas, Texas, United States

Site Status

Virginia Commonwealth University

Richmond, Virginia, United States

Site Status

University of Washington

Seattle, Washington, United States

Site Status

University of Ottawa

Ottawa, Ontario, Canada

Site Status

University College London

London, , United Kingdom

Site Status

University of Sheffield

Sheffield, , United Kingdom

Site Status

Countries

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United States Canada United Kingdom

Related Links

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https://www.aviditybiosciences.com/

Avidity Biosciences Website

Other Identifiers

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AOC 1020-CS2

Identifier Type: -

Identifier Source: org_study_id