Phase 1/2 Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)
NCT ID: NCT05747924
Last Updated: 2025-04-01
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE1/PHASE2
90 participants
INTERVENTIONAL
2023-04-04
2027-04-30
Brief Summary
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Detailed Description
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Cohort A comprises a placebo-controlled dose titration cohort (Cohort A1) which includes a nested single and multiple dose schedule. Cohort B comprises a placebo-controlled, nested single ascending dose (SAD)/multiple ascending dose (MAD) cohort (Cohort B1). Cohort C comprises a randomized, placebo-controlled, expansion cohort (Cohort C1). For each of Cohorts A, B, and C the study duration is 12 months as the active treatment period is approximately 9 months for Cohorts A \& B and approximately 10 months for Cohort C followed by a 12-week follow-up period for Cohorts A \& B and a 7-week follow-up period for Cohort C. Once participants have completed active treatment with follow-up through 12 months, 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 for 12-weeks after their last dose of study medication.
Conditions
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Study Design
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RANDOMIZED
SEQUENTIAL
TREATMENT
QUADRUPLE
Study Groups
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AOC 1020 Regimen 1
Cohort A: AOC 1020 Dose Regimen 1; Five doses administered intravenously over 9 months
AOC 1020
AOC 1020 will be administered via intravenous (IV) infusion
AOC 1020 Regimen 2
Cohort B1: AOC 1020 Dose Regimen 2; Five doses administered intravenously over 9 months
AOC 1020
AOC 1020 will be administered via intravenous (IV) infusion
AOC 1020 Regimen 3
Cohort C: AOC 1020 Dose Regimen 3; Eight doses administered intravenously over approximately 10 months
AOC 1020
AOC 1020 will be administered via intravenous (IV) infusion
Placebo (Saline) Regimen 1
Cohort A \& B: Placebo; Five doses administered intravenously over 9 months
Placebo
Placebo will be administered via intravenous (IV) infusion
Placebo (Saline) Regimen 2
Cohort C: Placebo; Eight doses administered intravenously over approximately 10 months
Placebo
Placebo will be administered via intravenous (IV) infusion
Interventions
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AOC 1020
AOC 1020 will be administered via intravenous (IV) infusion
Placebo
Placebo will be administered via intravenous (IV) infusion
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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
* 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
16 Years
70 Years
ALL
No
Sponsors
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Avidity Biosciences, Inc.
INDUSTRY
Responsible Party
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Locations
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University of California Los Angeles
Los Angeles, California, United States
Stanford University
Palo Alto, California, United States
University of California San Diego
San Diego, California, United States
University of Colorado
Denver, Colorado, United States
University of Florida
Gainesville, Florida, United States
Rare Disease Research
Atlanta, Georgia, United States
Kansas University Medical Center
Kansas City, Kansas, United States
University of Rochester Medical Center
Rochester, New York, United States
Duke University
Durham, North Carolina, United States
Ohio State University
Columbus, Ohio, United States
University of Pennsylvania
Philadelphia, Pennsylvania, United States
University of Texas Southwestern
Dallas, Texas, United States
Virginia Commonwealth University
Richmond, Virginia, United States
University of Washington
Seattle, Washington, United States
University of Ottawa
Ottawa, Ontario, Canada
University College London
London, , United Kingdom
University of Sheffield
Sheffield, , United Kingdom
Countries
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Related Links
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Avidity Biosciences Website
FORTITUDE Study Website
Other Identifiers
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AOC 1020-CS1
Identifier Type: -
Identifier Source: org_study_id
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