Del-brax (AOC 1020) intravenous treatment for people with FSHD
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)
This study will try regular IV infusions of del-brax (AOC 1020) in adults with FSHD to see if it slows muscle weakness and helps preserve walking ability.
Quick facts
| Phase | Phase 3 |
|---|---|
| Study type | Interventional |
| Enrollment | 200 (estimated) |
| Ages | 16 Years to 70 Years |
| Sex | All |
| Sponsor | Avidity Biosciences, Inc. Industry-sponsored |
| Locations | 22 sites (Orange, California and 21 other locations) |
| Trial ID | NCT07038200 on ClinicalTrials.gov |
What this trial studies
This is a randomized, double-blind, placebo-controlled Phase 3 trial testing intravenous del-brax (AOC 1020) given every 6 weeks for 72 weeks (13 doses) after up to 6 weeks of screening. Participants are randomized to del-brax or placebo and undergo clinical assessments through a final visit at Week 78, with eligible participants offered an open-label extension thereafter. Safety and tolerability will be reviewed periodically by an independent data monitoring committee. The study includes on-site IV infusions and functional outcome measures, and excludes people with recent oligonucleotide treatment or certain abnormal labs or blood pressure.
Who should consider this trial
Good fit: Adults with a clinical and genetic diagnosis of FSHD1 or FSHD2 who can walk independently for at least 10 meters and meet the study's muscle strength and safety criteria are ideal candidates.
Not a fit: People who cannot walk independently for 10 meters, who have disqualifying lab abnormalities, are pregnant or breastfeeding, or who recently received other oligonucleotide therapies may not benefit from this study.
Why it matters
Potential benefit: If successful, del-brax could slow disease progression and help maintain muscle strength and walking function in people with FSHD.
How similar studies have performed: Early-phase work with del-brax and other antibody-oligonucleotide conjugates has shown preliminary safety and target engagement, but large Phase 3 proof of clinical benefit in FSHD has not yet been established.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * 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 Exclusion Criteria: * 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
Where this trial is running
Orange, California and 21 other locations
- University of California Irvine — Orange, California, United States (Recruiting)
- Stanford University — Palo Alto, California, United States (Recruiting)
- University of Colorado — Denver, Colorado, United States (Recruiting)
- University of Florida — Gainesville, Florida, United States (Recruiting)
- University of Iowa — Iowa City, Iowa, United States (Recruiting)
- Kansas University Medical Center — Kansas City, Kansas, United States (Recruiting)
- Kennedy Krieger Institute — Baltimore, Maryland, United States (Recruiting)
- University of Massachusetts — Worcester, Massachusetts, United States (Recruiting)
- University of Rochester Medical Center — Rochester, New York, United States (Recruiting)
- Duke University — Durham, North Carolina, United States (Recruiting)
- Ohio State University — Columbus, Ohio, United States (Recruiting)
- University of Pennsylvania — Philadelphia, Pennsylvania, United States (Recruiting)
- University of Texas Health Science Center at San Antonio — San Antonio, Texas, United States (Recruiting)
- Virginia Commonwealth University — Richmond, Virginia, United States (Recruiting)
- University of Calgary - Cumming School of Medicine — Calgary, Alberta, Canada (Recruiting)
- University of Alberta Hospital — Edmonton, Alberta, Canada (Not_yet_recruiting)
- The Ottawa Hospital — Ottawa, Ontario, Canada (Recruiting)
- Genge Partners Inc — Montreal, Quebec, Canada (Not_yet_recruiting)
- National Hospital Organization Osaka Toneyama Medical Center — Osaka, Japan (Recruiting)
- The University of Osaka Hospital — Osaka, Japan (Recruiting)
- National Hospital Organization Sendai Nishitaga Hospital — Sendai, Japan (Recruiting)
- National Center of Neurology and Psychiatry — Tokyo, Japan (Recruiting)
Study contacts
- Study coordinator: Avidity Biosciences, Inc.
- Email: medinfo@aviditybio.com
- Phone: 858-771-7038
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.