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

May 12, 2026 updated by: Avidity Biosciences, Inc.

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)

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)

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

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 46 months. Once participants have completed active treatment, they will be followed through a 18-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 50 months (4 years 2 month), excluding the potential 8-week screening period.

Study Type

Interventional

Enrollment (Estimated)

84

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Canada
    • Ontario
      • Ottawa, Ontario, Canada, K1Y 4E9
        • University of Ottawa
  • United Kingdom
      • London, United Kingdom, WIT 7HA
        • University College London
      • Sheffield, United Kingdom, S10 2TN
        • University of Sheffield
  • United States
    • California
      • Los Angeles, California, United States, 90095
        • University of California Los Angeles
      • Palo Alto, California, United States, 94304
        • Stanford University
      • San Diego, California, United States, 92093
        • University Of California San Diego
    • Colorado
      • Denver, Colorado, United States, 80045
        • University of Colorado
    • Florida
      • Gainesville, Florida, United States, 32608
        • University of Florida
    • Georgia
      • Atlanta, Georgia, United States, 30329
        • Rare Disease Research
    • Kansas
      • Kansas City, Kansas, United States, 66205
        • Kansas University Medical Center
    • New York
      • Rochester, New York, United States, 14642
        • University of Rochester Medical Center
    • North Carolina
      • Durham, North Carolina, United States, 27708
        • Duke University
    • Ohio
      • Columbus, Ohio, United States, 43221
        • Ohio State University
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • University of Pennsylvania
    • Texas
      • Dallas, Texas, United States, 75390
        • University of Texas Southwestern
    • Virginia
      • Richmond, Virginia, United States, 23298
        • Virginia Commonwealth University
    • Washington
      • Seattle, Washington, United States, 98104
        • University of Washington

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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.)

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: AOC 1020 Regimen
AOC 1020 Dose Regimen; Thirty-two doses administered intravenously over 46 months. All participants will receive AOC 1020 at a dose level of 2mg/kg.
Drug: AOC 1020
AOC 1020 will be administered via intravenous (IV) infusion
Other Names:
  • delpacibart braxlosiran
  • del-brax

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Incidence of treatment-emergent adverse events
Time Frame: Through study completion, up to Day 729
Through study completion, up to Day 729

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Avidity Biosciences, Inc.

Investigators

  • Study Director: Amy Halseth, Ph.D., Avidity Biosciences, Inc.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

July 25, 2024

Primary Completion (Estimated)

April 1, 2030

Study Completion (Estimated)

June 1, 2030

Study Registration Dates

First Submitted

August 7, 2024

First Submitted That Met QC Criteria

August 7, 2024

First Posted (Actual)

August 9, 2024

Study Record Updates

Last Update Posted (Actual)

May 14, 2026

Last Update Submitted That Met QC Criteria

May 12, 2026

Last Verified

May 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • AOC 1020-CS2

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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