Clinical Trials in the USA / NCT06547216
Active, not recruiting
Phase 2
Phase 2 Open-label Extension Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)
NCT06547216 · tracked via the Priya Life Science USA tracker
Condition(s) studied
FSHDFSHD1FSHD2FMDFMD2Fascioscapulohumeral Muscular DystrophyFascioscapulohumeral Muscular Dystrophy Type 1Fascioscapulohumeral Muscular Dystrophy Type 2Dystrophies, Facioscapulohumeral MuscularDystrophy, Facioscapulohumeral MuscularFacioscapulohumeral Muscular Dystrophy 1Facioscapulohumeral Muscular Dystrophy 2Facio-Scapulo-Humeral DystrophyAtrophy, FacioscapulohumeralAtrophies, FacioscapulohumeralFacioscapulohumeral AtrophyMuscular DystrophiesMuscular Dystrophy, FacioscapulohumeralFSH Muscular DystrophyLandouzy Dejerine DystrophyLandouzy-Dejerine Muscular DystrophyDystrophies, Landouzy-DejerineDystrophy, Landouzy-DejerineLandouzy-Dejerine SyndromeMuscular Dystrophy, Landouzy DejerineProgressive Muscular DystrophyFSH
Investigational drug(s) / intervention(s)
AOC 1020: AOC 1020 will be administered via intravenous (IV) infusion
Study summary
A Phase 2 Open-label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of AOC 1020 Administered Intravenously to Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)
Eligibility
Inclusion Criteria:
1. Ability to provide written informed consent (signed and dated) and any authorizations required by local law and be willing and able to comply with all study requirements. When enrolling participants who are minors, it is necessary to also obtain consent from a legally designated representative and the participant will receive information in a way adapted to their age and mental maturity.
2. Completion of AOC 1020-CS1 with the following as judged by the Investigator and Sponsor:
1. No significant tolerability issues
2. Satisfactory compliance with the AOC 1020-CS1 protocol requirements
Exclusion Criteria:
1. Pregnancy, intent to become pregnant during the clinical study, or active breastfeeding.
2. Unwilling or unable to continue to comply with contraceptive requirements for the length of AOC 1020-CS2.
3. Any new conditions or worsening of existing condition(s) 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. (Note: FSHD progression is not exclusionary, even if the participant no longer has the ability to walk 10 meters without a walker or two canes.)
Primary outcome measure(s)
- Incidence of treatment-emergent adverse events — Through study completion, up to Day 729
Trial sites (17)
| Facility | City | Region | Status |
|---|---|---|---|
| University of California Los Angeles | Los Angeles | California | |
| Stanford University | Palo Alto | California | |
| University of California San Diego | San Diego | California | |
| University of Colorado | Denver | Colorado | |
| University of Florida | Gainesville | Florida | |
| Rare Disease Research | Atlanta | Georgia | |
| Kansas University Medical Center | Kansas City | Kansas | |
| University of Rochester Medical Center | Rochester | New York | |
| Duke University | Durham | North Carolina | |
| Ohio State University | Columbus | Ohio | |
| University of Pennsylvania | Philadelphia | Pennsylvania | |
| University of Texas Southwestern | Dallas | Texas | |
| Virginia Commonwealth University | Richmond | Virginia | |
| University of Washington | Seattle | Washington | |
| University of Ottawa | Ottawa | Ontario | |
| University College London | London | United Kingdom | |
| University of Sheffield | Sheffield | United Kingdom |
More Avidity Biosciences, Inc. trials in the USA
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Other trials for the same condition
Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT06547216 on ClinicalTrials.gov ↗ ← All trials in the USA