for people ages 18-65 (full criteria)
study started
completion around



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 Adult Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)

Official Title

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 Adult Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)


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 adult participants with FSHD Type 1 (FSHD1) and FSHD Type 2 (FSHD2).

Part A is a dose titration design which includes a single and multiple dose schedule with 1 cohort. Part B is a single-ascending and multiple-ascending dose design with 2 cohorts. Part C is a parallel, multi-dose cohort design with 1 cohort. For each of Part A, B, and C the patient duration is 12 months as the active treatment period is approximately 9 months followed by a 3-month follow-up period. Once participants have completed active treatment and the follow-up period, 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 through a 6-month safety follow-up period.


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, FORTITUDE, Avidity, Avidity Biosciences, AOC 1020, Facioscapulohumeral Muscular Dystrophy, Atrophy


You can join if…

Open to people ages 18-65

  • FSHD1 or FSHD2 diagnosis confirmed by documented genetic testing (testing provided by Sponsor)
  • Ambulatory and able to walk 10 meters (with or without assistive devices such as one cane, 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

You CAN'T join if...

  • Pregnancy, intent to become pregnant within 9 months after last planned dose of Study Drug, 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 to undergo any nonstudy muscle biopsies over the duration of the study
  • History of bleeding disorders, significant keloid, or other skin or muscle conditions (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 the investigator or Sponsor, would make the participant unsuitable for the study or could interfere with participation or completion of the study
  • Treatment with any investigative medication within 1 month (or 5 half-lives of the drug, whichever is longer) of Screening


  • University of California Los Angeles accepting new patients
    Los Angeles California 90095 United States
  • University of California San Diego accepting new patients
    San Diego California 92093 United States
  • Stanford University accepting new patients
    Palo Alto California 94304 United States


accepting new patients
Start Date
Completion Date
Avidity Biosciences, Inc.
Avidity Biosciences Website FORTITUDE Study Website
Phase 1/2 research study
Study Type
Expecting 72 study participants
Last Updated