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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
Sponsor
Avidity Biosciences, Inc.
Phase
Phase 2
Started
2024-07-25
Last updated
2026-07-14

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: 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

Sex
ALL
Min age
16 Years
Max age
72 Years
Healthy volunteers
No
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)

Trial sites (17)

FacilityCityRegionStatus
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

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