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

NCT06547216 | Enrolling By Invitation Phase 2 DMC FDA Drug

• 1 other identifier: AOC 1020-CS2
• Study Type: interventional
• Target: 84
• Locations: 3 countries
• Sites: 17

Brief Summary

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)

Conditions

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

FORTITUDE; Avidity; Avidity Biosciences; AOC 1020; FORTITUDE-OLE; FORTITUDE OLE

Outcome Measures

Primary Outcomes (1)

• Incidence of treatment-emergent adverse events

  Through study completion, up to Day 729

Study Arms (1)

AOC 1020 Regimen

EXPERIMENTAL

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

Drug: AOC 1020

Interventions

AOC 1020 DRUG

AOC 1020 will be administered via intravenous (IV) infusion

Also known as: delpacibart braxlosiran, del-brax

AOC 1020 Regimen

Eligibility Criteria

• Age: 16 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• Completion of AOC 1020-CS1 with the following as judged by the Investigator and Sponsor:
• No significant tolerability issues
• Satisfactory compliance with the AOC 1020-CS1 protocol requirements

You may not qualify if:

• Pregnancy, intent to become pregnant during the clinical study, or active breastfeeding.
• Unwilling or unable to continue to comply with contraceptive requirements for the length of AOC 1020-CS2.

Sponsors & Collaborators

• Avidity Biosciences, Inc.: lead

Study Sites (17)

University of California Los Angeles

Los Angeles, California, 90095, United States

Location

Stanford University

Palo Alto, California, 94304, United States

Location

University of California San Diego

San Diego, California, 92093, United States

Location

University of Colorado

Denver, Colorado, 80045, United States

Location

University of Florida

Gainesville, Florida, 32608, United States

Location

Rare Disease Research

Atlanta, Georgia, 30329, United States

Location

Kansas University Medical Center

Kansas City, Kansas, 66205, United States

Location

University of Rochester Medical Center

Rochester, New York, 14642, United States

Location

Duke University

Durham, North Carolina, 27708, United States

Location

Ohio State University

Columbus, Ohio, 43221, United States

Location

University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

Location

University of Texas Southwestern

Dallas, Texas, 75390, United States

Location

Virginia Commonwealth University

Richmond, Virginia, 23298, United States

Location

University of Washington

Seattle, Washington, 98104, United States

Location

University of Ottawa

Ottawa, Ontario, K1Y 4E9, Canada

Location

University College London

London, WIT 7HA, United Kingdom

Location

University of Sheffield

Sheffield, S10 2TN, United Kingdom

Location

Related Links

• Avidity Biosciences Website

MeSH Terms

Conditions

Muscular Dystrophy, Facioscapulohumeral; Facioscapulohumeral Muscular Dystrophy 1B; Muscular Dystrophies; Facioscapulohumeral muscular dystrophy 1a

Condition Hierarchy (Ancestors)

Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Amy Halseth, Ph.D.

  Avidity Biosciences, Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 7, 2024
• First Posted: August 9, 2024
• Study Start: July 25, 2024
• Primary Completion (Estimated): April 1, 2028
• Study Completion (Estimated): April 1, 2028
• Last Updated: December 20, 2024

Record last verified: 2024-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (14); GB (2); CA (1)