This study is accepting new patients by invitation only

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

a study on Facioscapulohumeral Muscular Dystrophy FMD FMD2 Facio-Scapulo-Humeral Dystrophy Muscular Dystrophies Progressive Muscular Dystrophy FSH

Summary

Eligibility
for people ages 16-70 (full criteria)
Location
at UCLA UCSD
Dates
study started
study ends around

Description

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)

Official Title

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)

Details

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.

Participants from AOC 1020-CS1 are eligible to enroll in AOC 1020-CS2 if they have satisfactorily completed AOC 1020-CS1. All participants who enroll in AOC 1020-CS2 will receive AOC 1020 regardless of whether they received AOC 1020 or placebo in AOC 1020-CS1. Dosing will occur every 6 to 7 weeks (twice quarterly).

The total duration of active treatment in AOC 1020-CS2 is approximately 22 months. Once participants have completed active treatment, they will be followed through a 12-week safety follow-up period to monitor for adverse events (AEs), concomitant medications, and pregnancy status (as applicable). Total duration on study is up to approximately 25 months (2 years 1 month), excluding the potential 8-week screening period.

Keywords

FSHD, FSHD1, FSHD2, FMD, FMD2, Fascioscapulohumeral Muscular Dystrophy, Fascioscapulohumeral Muscular Dystrophy Type 1, Fascioscapulohumeral Muscular Dystrophy Type 2, Dystrophies, Facioscapulohumeral Muscular, Dystrophy, Facioscapulohumeral Muscular, Facioscapulohumeral Muscular Dystrophy 1, Facioscapulohumeral Muscular Dystrophy 2, Facio-Scapulo-Humeral Dystrophy, Atrophy, Facioscapulohumeral, Atrophies, Facioscapulohumeral, Facioscapulohumeral Atrophy, Muscular Dystrophies, Muscular Dystrophy, Facioscapulohumeral, FSH Muscular Dystrophy, Landouzy Dejerine Dystrophy, Landouzy-Dejerine Muscular Dystrophy, Dystrophies, Landouzy-Dejerine, Dystrophy, Landouzy-Dejerine, Landouzy-Dejerine Syndrome, Muscular Dystrophy, Landouzy Dejerine, Progressive Muscular Dystrophy, FSH, FORTITUDE, Avidity, Avidity Biosciences, AOC 1020, FORTITUDE-OLE, FORTITUDE OLE, Facioscapulohumeral Muscular Dystrophy, Facioscapulohumeral Muscular Dystrophy 1B, Facioscapulohumeral muscular dystrophy 1a

Eligibility

You can join if…

Open to people ages 16-70

  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

You CAN'T join if...

  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.)

Locations

  • University of California Los Angeles
    Los Angeles 5368361 California 5332921 90095 United States
  • University of California San Diego
    San Diego 5391811 California 5332921 92093 United States
  • Stanford University
    Palo Alto 5380748 California 5332921 94304 United States

Details

Status
accepting new patients by invitation only
Start Date
Completion Date
(estimated)
Sponsor
Avidity Biosciences, Inc.
Links
Avidity Biosciences Website
ID
NCT06547216
Phase
Phase 2 research study
Study Type
Interventional
Participants
Expecting 84 study participants
Last Updated