Status:

ENROLLING_BY_INVITATION

Phase 2 Open-label Extension Study of AOC 1020 in Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)

Lead Sponsor:

Avidity Biosciences, Inc.

Conditions:

FSHD

FSHD1

Eligibility:

All Genders

16-70 years

Phase:

PHASE2

Brief 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 (FS...

Detailed Description

This study will continue to evaluate the safety, tolerability, and efficacy of AOC 1020 in participants who were treated in the randomized, placebo-controlled, Phase 1/2 AOC 1020-CS1 clinical study. ...

Eligibility Criteria

Inclusion

  • 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.
  • Completion of AOC 1020-CS1 with the following as judged by the Investigator and Sponsor:
  • No significant tolerability issues
  • Satisfactory compliance with the AOC 1020-CS1 protocol requirements

Exclusion

  • Pregnancy, intent to become pregnant during the clinical study, or active breastfeeding.
  • Unwilling or unable to continue to comply with contraceptive requirements for the length of AOC 1020-CS2.
  • 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.)

Key Trial Info

Start Date :

July 25 2024

Trial Type :

INTERVENTIONAL

Allocation :

ESTIMATED

End Date :

April 1 2028

Estimated Enrollment :

84 Patients enrolled

Trial Details

Trial ID

NCT06547216

Start Date

July 25 2024

End Date

April 1 2028

Last Update

December 20 2024

Active Locations (17)

Enter a location and click search to find clinical trials sorted by distance.

Page 1 of 5 (17 locations)

1

University of California Los Angeles

Los Angeles, California, United States, 90095

2

Stanford University

Palo Alto, California, United States, 94304

3

University of California San Diego

San Diego, California, United States, 92093

4

University of Colorado

Denver, Colorado, United States, 80045