Open Actively Recruiting

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

About

Brief 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 Adult Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)

Primary Purpose
Treatment
Study Type
Interventional
Phase
Phase 1/Phase 2

Eligibility

Gender
All
Healthy Volunteers
No
Minimum Age
18 Years
Maximum Age
65 Years

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 Investigator

Exclusion Criteria:

  • 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

Join this Trial

Contact our clinical trial navigators for opportunities that may be suitable for you
Share:
Study Stats
Protocol No.
23-000872
Category
Genetic and Rare Diseases
Contact
Brenden Roberts
Location
  • UCLA Westwood
For Providers
NCT No.
NCT05747924
For detailed technical eligibility, visit ClinicalTrials.gov.