Phase 1/2 Study of AOC 1020 in Adults With Facioscapulohumeral Muscular Dystrophy (FSHD) (FORTITUDE)
FSHD, FSHD1, FSHD2
About this trial
This is an interventional treatment trial for FSHD focused on measuring FORTITUDE, Avidity, Avidity Biosciences, AOC 1020
Eligibility Criteria
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: Diagnosed with congenital or infantile FSHD 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
Sites / Locations
- University of California, Irvine
- University of California Los AngelesRecruiting
- Stanford UniversityRecruiting
- University of California San DiegoRecruiting
- University of ColoradoRecruiting
- University of FloridaRecruiting
- Rare Disease ResearchRecruiting
- Kansas University Medical CenterRecruiting
- University of Minnesota
- University of Rochester Medical CenterRecruiting
- Duke University
- Ohio State University
- University of PennsylvaniaRecruiting
- University of Texas SouthwesternRecruiting
- University of Utah
- Virginia Commonwealth UniversityRecruiting
- University of Ottawa
- Centre for Human Drug Research
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Experimental
Experimental
Experimental
Placebo Comparator
AOC 1020 Regimen 1
AOC 1020 Regimen 2
AOC 1020 Regimen 3
Placebo (Saline)
Part A: AOC 1020 Dose Regimen 1; Five doses administered intravenously over 9 months
Part B1 & C: AOC 1020 Dose Regimen 2; Five doses administered intravenously over 9 months
Part B2 & C: AOC 1020 Dose Regimen 3; Five doses administered intravenously over 9 months
Part A, B & C: Placebo; Five doses administered intravenously over 9 months