Phase
Condition
Muscular Dystrophy
Treatment
Placebo
AOC 1020
Clinical Study ID
Ages 16-70 All Genders
Study Summary
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
Study Description
Connect with a study center
University of Ottawa
Ottawa, Ontario K1Y 4E9
CanadaSite Not Available
Centre for Human Drug Research
Leiden,
NetherlandsSite Not Available
University College London
London, WIT 7HA
United KingdomSite Not Available
University of Sheffield
Sheffield, S10 2TN
United KingdomSite Not Available
University of California, Irvine
Irvine, California 92697
United StatesSite Not Available
University of California Los Angeles
Los Angeles, California 90095
United StatesSite Not Available
Stanford University
Palo Alto, California 94304
United StatesSite Not Available
University of California San Diego
San Diego, California 92093
United StatesSite Not Available
University of Colorado
Denver, Colorado 80045
United StatesSite Not Available
University of Florida
Gainesville, Florida 32608
United StatesSite Not Available
Rare Disease Research
Atlanta, Georgia 30329
United StatesSite Not Available
Kansas University Medical Center
Kansas City, Kansas 66205
United StatesSite Not Available
University of Minnesota
Minneapolis, Minnesota 55455
United StatesSite Not Available
University of Rochester Medical Center
Rochester, New York 14642
United StatesSite Not Available
Duke University
Durham, North Carolina 27708
United StatesSite Not Available
Ohio State University
Columbus, Ohio 43221
United StatesSite Not Available
University of Pennsylvania
Philadelphia, Pennsylvania 19104
United StatesSite Not Available
University of Texas Southwestern
Dallas, Texas 75390
United StatesSite Not Available
University of Utah
Salt Lake City, Utah 84112
United StatesSite Not Available
Virginia Commonwealth University
Richmond, Virginia 23298
United StatesSite Not Available
University of Washington
Seattle, Washington 98104
United StatesSite Not Available
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