Del-brax (AOC 1020) intravenous treatment for people with FSHD

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• University of Calgary - Cumming School of Medicine
  Calgary, Alberta, Canada Recruiting
• University of Alberta Hospital
  Edmonton, Alberta, Canada Recruiting
• The Ottawa Hospital
  Ottawa, Ontario, Canada Recruiting
• Genge Partners Inc
  Montreal, Quebec, Canada Recruiting
• Rigshospitalet
  Copenhagen, Denmark Recruiting
• AP-HP Hopital Pitie-Salpetriere
  Paris, France Recruiting

Study contacts

• Study coordinator: Avidity Biosciences, Inc., A Novartis Company
• Email: novartis.email@novartis.com
• Phone: 1-888-669-6682

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• BetterLife FSHD: a patient-driven health platform

  This project tries to connect people with FSHD to personalized resources and collect their health experiences so they can be matched to relevant studies and help improve care.

  Recruiting FSHD Society · Randolph, Massachusetts · NCT07409142

• Apitegromab for FSHD in Adults (Phase 2)

  This study tests whether apitegromab works as a standalone treatment for adults with FSHD.

  Recruiting Phase 2 Scholar Rock, Inc. · Austin, Texas · NCT07435129

• Assessing respiratory function in FSHD patients using diaphragmatic ultrasound

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  Recruiting Fondazione Policlinico Universitario Agostino Gemelli IRCCS · Roma · NCT06078852

• Amino acids plus diet and exercise for people with FSHD

  This trial will test whether taking essential amino acid supplements together with a structured diet and exercise program helps adults with FSHD build muscle, reduce fat, and improve physical performance compared with placebo and matched healthy adults.

  Recruiting University of Pavia · Voghera, Pavia · NCT07543016

• Wearable monitoring of walking and arm movement in adults with neuromuscular or metabolic movement disorders

  This project uses a wrist/ankle wearable to continuously monitor walking and upper-limb movement in ambulant adults with neuromuscular diseases or obesity to see if digital measures like stride speed track real-life motor function.

  Recruiting Centre Hospitalier Universitaire de Liege · Liège · NCT07136844

• Umbilical cord lining stem cell treatment for FSHD

  This trial will try two intravenous doses of umbilical cord lining–derived stem cells (ULSC) versus placebo to see if they are safe and help people aged 15+ with genetically confirmed FSHD and moderate disease (Ricci score ≥3).

  Recruiting Phase 1 Restem, LLC. · Palo Alto, California · NCT07086521

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.