Phase 1/2 Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)

Last updated: March 31, 2025
Sponsor: Avidity Biosciences, Inc.
Overall Status: Active - Not Recruiting

Phase

1/2

Condition

Muscular Dystrophy

Treatment

Placebo

AOC 1020

Clinical Study ID

NCT05747924
AOC 1020-CS1
2022-002704-20
2022-502096-32-00
  • Ages 16-70
  • All Genders

Study Summary

A Randomized, Double-blind, Placebo-controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of AOC 1020 Administered Intravenously to Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • FSHD1 or FSHD2 diagnosis confirmed by documented genetic testing (testing providedby Sponsor)

  • Ambulatory and able to walk 10 meters (with or without assistive devices such as onecane, walking stick or braces)

  • At least 1 muscle region suitable for biopsy (testing provided by Sponsor)

  • Muscle weakness in both upper and lower body, as determined by Investigator

Exclusion

Exclusion Criteria:

  • Pregnant or intends to become pregnant while on study, or active breastfeeding

  • Unwilling or unable to continue to comply with contraceptive requirements

  • Body mass index (BMI) >35.0 kg/m2 at Screening

  • History of muscle biopsy within 30 days of the screening biopsy or planning toundergo any nonstudy muscle biopsies over the duration of the study

  • History of bleeding disorders, significant keloid, or other skin or muscleconditions (e.g., severe muscle wasting) that, in the opinion of the Investigator,makes the participant unsuitable for serial muscle biopsy

  • Anticipated survival less than 2 years

  • Blood or plasma donation within 16 weeks of Study Day 1

  • Any contraindication to MRI

  • Any abnormal lab values, conditions or diseases that, in the opinion of theinvestigator or Sponsor, would make the participant unsuitable for the study orcould interfere with participation or completion of the study

  • Treatment with any investigative medication within 1 month (or 5 half-lives of thedrug, whichever is longer) of Screening

Study Design

Total Participants: 90
Treatment Group(s): 2
Primary Treatment: Placebo
Phase: 1/2
Study Start date:
April 04, 2023
Estimated Completion Date:
April 30, 2027

Study Description

AOC 1020-CS1 is a first-in-human, 3-part, multi-center, Phase 1/2, randomized, double-blind, placebo-controlled study designed to evaluate safety, tolerability, pharmacokinetics and to explore pharmacodynamics and efficacy of single and multiple-doses of AOC 1020 administered intravenously in participants with FSHD Type 1 (FSHD1) and FSHD Type 2 (FSHD2).

Cohort A comprises a placebo-controlled dose titration cohort (Cohort A1) which includes a nested single and multiple dose schedule. Cohort B comprises a placebo-controlled, nested single ascending dose (SAD)/multiple ascending dose (MAD) cohort (Cohort B1). Cohort C comprises a randomized, placebo-controlled, expansion cohort (Cohort C1). For each of Cohorts A, B, and C the study duration is 12 months as the active treatment period is approximately 9 months for Cohorts A & B and approximately 10 months for Cohort C followed by a 12-week follow-up period for Cohorts A & B and a 7-week follow-up period for Cohort C. Once participants have completed active treatment with follow-up through 12 months, they may have the option to participate in a planned open-label extension. If patients do not enroll in the open-label extension, they will be followed for 12-weeks after their last dose of study medication.

Connect with a study center

  • University of Ottawa

    Ottawa, Ontario K1Y 4E9
    Canada

    Site Not Available

  • Centre for Human Drug Research

    Leiden,
    Netherlands

    Site Not Available

  • University College London

    London, WIT 7HA
    United Kingdom

    Site Not Available

  • University of Sheffield

    Sheffield, S10 2TN
    United Kingdom

    Site Not Available

  • University of California, Irvine

    Irvine, California 92697
    United States

    Site Not Available

  • University of California Los Angeles

    Los Angeles, California 90095
    United States

    Site Not Available

  • Stanford University

    Palo Alto, California 94304
    United States

    Site Not Available

  • University of California San Diego

    San Diego, California 92093
    United States

    Site Not Available

  • University of Colorado

    Denver, Colorado 80045
    United States

    Site Not Available

  • University of Florida

    Gainesville, Florida 32608
    United States

    Site Not Available

  • Rare Disease Research

    Atlanta, Georgia 30329
    United States

    Site Not Available

  • Kansas University Medical Center

    Kansas City, Kansas 66205
    United States

    Site Not Available

  • University of Minnesota

    Minneapolis, Minnesota 55455
    United States

    Site Not Available

  • University of Rochester Medical Center

    Rochester, New York 14642
    United States

    Site Not Available

  • Duke University

    Durham, North Carolina 27708
    United States

    Site Not Available

  • Ohio State University

    Columbus, Ohio 43221
    United States

    Site Not Available

  • University of Pennsylvania

    Philadelphia, Pennsylvania 19104
    United States

    Site Not Available

  • University of Texas Southwestern

    Dallas, Texas 75390
    United States

    Site Not Available

  • University of Utah

    Salt Lake City, Utah 84112
    United States

    Site Not Available

  • Virginia Commonwealth University

    Richmond, Virginia 23298
    United States

    Site Not Available

  • University of Washington

    Seattle, Washington 98104
    United States

    Site Not Available

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