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

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)

Inclusion Criteria: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 InvestigatorExclusion Criteria:Diagnosed with congenital or infantile FSHDPregnancy, intent to become pregnant within 9 months after last planned dose of Study Drug, or active breastfeedingUnwilling or unable to continue to comply with contraceptive requirementsBody mass index (BMI) >35.0 kg/m2 at ScreeningHistory of muscle biopsy within 30 days of the screening biopsy or planning to undergo any nonstudy muscle biopsies over the duration of the studyHistory 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 biopsyAnticipated survival less than 2 yearsBlood or plasma donation within 16 weeks of Study Day 1Any contraindication to MRIAny 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 studyTreatment with any investigative medication within 1 month (or 5 half-lives of the drug, whichever is longer) of Screening