A Study to Evaluate Del-brax (Also Referred to as AOC 1020) in Participants With FSHD

a study on Facioscapulohumeral Muscular Dystrophy Facioscapulohumeral Dystrophy Facio-Scapulo-Humeral Dystrophy

Summary

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

Description

Summary

A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1020 for the Treatment of Facioscapulohumeral Muscular Dystrophy (FSHD)

Official Title

A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1020 for the Treatment of Facioscapulohumeral Muscular Dystrophy (FSHD)

Details

The study consists of a Screening Period of up to 6 weeks and 72-week Treatment Period. The anticipated duration is approximately 78 weeks.

Participants will be randomized to receive an intravenous infusion of either del-brax or placebo at the clinical study site every 6 weeks for a total of 13 doses. The final dose will occur at Week 72, followed by a final assessment at Week 78.

After completion of the Week 78 visit, eligible participants will have the option to enroll into an open label extension (OLE) study, pending regulatory approval. Participants who decline participation in the OLE will be followed for a period of 12 weeks for safety.

An Independent Data Monitoring Committee (IDMC) comprising members independent and external to the Sponsor will review safety, tolerability, and efficacy (as needed) data of this study at regular intervals.

Keywords

Facioscapulohumeral Muscular Dystrophy, FSHD, FSHD - Facioscapulohumeral Muscular Dystrophy, FSHD1, FSHD2, Fascioscapulohumeral Muscular Dystrophy, Fascioscapulohumeral Muscular Dystrophy Type 1, Fascioscapulohumeral Muscular Dystrophy Type 2, Facioscapulohumeral Muscular Dystrophy 1, Facioscapulohumeral Dystrophy, Facio-Scapulo-Humeral Dystrophy, Facioscapulohumeral Muscular Dystrophy 2, Facioscapulohumeral Muscular Dystrophy Type 1 (FSHD1), FSH Muscular Dystrophy, Landouzy Dejerine Dystrophy, Landouzy-Dejerine Muscular Dystrophy, Landouzy-Dejerine Syndrome, Avidity, Avidity Biosciences, del-brax, del brax, delbrax, AOC1020, AOC 1020, delpacibart braxlosiran, FORTITUDE-3, FORTITUDE Phase 3, FORTITUDE, FORTITUDE 3, Facioscapulohumeral Muscular Dystrophy 1B, Facioscapulohumeral muscular dystrophy 1a, Sodium Chloride, AOC-1020

Eligibility

You can join if…

Open to people ages 16-70

  • Clinical and genetic diagnosis of FSHD1 or FSHD2
  • Ability to walk independently at pre-specified walking speed (orthoses and ankle braces allowed) for at least 10 meters at screening
  • Adequate muscle strength based on QMT composite score

You CAN'T join if...

  • Breastfeeding, pregnancy, or intent to become pregnant during the study
  • Unwilling or unable to comply with contraceptive requirements
  • Abnormal lab values, conditions or diseases that would make the participant unsuitable for the study
  • Blood Pressure > 140/90 mmHg at Screening
  • Treatment with another investigational drug or biological agent within 1 month of Screening or 5 half-lives of the drug, whichever is longer
  • Treatment with an oligonucleotide within 9 months of Screening

Locations

  • University of California Irvine accepting new patients
    Orange 5379513 California 5332921 92868 United States
  • Stanford University accepting new patients
    Palo Alto 5380748 California 5332921 94304 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Avidity Biosciences, Inc.
Links
Related Info
ID
NCT07038200
Phase
Phase 3 research study
Study Type
Interventional
Participants
Expecting 200 study participants
Last Updated
Contact us about this trial