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NCT Number: NCT07038200

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

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Key information

Conditions

Facioscapulohumeral Muscular Dystrophy Congenital, Hereditary, and Neonatal Diseases and Abnormalities FSH Muscular Dystrophy FSHD FSHD - Facioscapulohumeral Muscular Dystrophy FSHD1 FSHD2 Facio-Scapulo-Humeral Dystrophy Facioscapulohumeral Dystrophy Facioscapulohumeral Muscular Dystrophy 1 Facioscapulohumeral Muscular Dystrophy 1B Facioscapulohumeral Muscular Dystrophy 2 Facioscapulohumeral Muscular Dystrophy Type 1 (FSHD1) Facioscapulohumeral muscular dystrophy 1a Fascioscapulohumeral Muscular Dystrophy Fascioscapulohumeral Muscular Dystrophy Type 1 Fascioscapulohumeral Muscular Dystrophy Type 2 Genetic Diseases, Inborn Landouzy Dejerine Dystrophy Landouzy-Dejerine Muscular Dystrophy Landouzy-Dejerine Syndrome Muscular Diseases Muscular Disorders, Atrophic Muscular Dystrophies Muscular Dystrophy, Facioscapulohumeral Musculoskeletal Diseases Nervous System Diseases Neuromuscular Diseases

Age range

16 year–70 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

University of Calgary - Cumming School of Medicine, Calgary, Alberta, Canada

Loading trial locations.

About this study

The study duration is approximately 22 months, consisting of a screening period of up to 6 weeks, a 78-week active treatment period, and a 12-week follow-up period (18 weeks from the last dose).

Participants will be randomized to receive an intravenous infusion of either delbrax 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 in an open-label extension (OLE) study, pending regulatory approval. Participants who decline participation in the OLE will be followed for 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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

AOC-1020

Drug

Del-brax will be administered by intravenous (IV) infusion.

Other names: Del-brax

Placebo

Drug

Placebo will be administered by intravenous (IV) infusion.

Other names: Saline

Primary outcomes

  1. Change from Baseline to Week 78 in Quantitative Muscle Testing (QMT) total composite score (PPN)

    Time frame: Baseline through Week 78

    Assessment of muscle strength using standardized quantitative measurements

Secondary outcomes

  1. Change from Baseline to Week 78 in 10MWRT

    Time frame: Baseline through Week 78

    Assessment of the time required to walk or run 10 meters as quickly and safely as possible

  2. Change from Baseline to Week 78 in Timed Up-and-Go (TUG)

    Time frame: Baseline through Week 78

    Assessment of functional mobility by measuring the time required to stand up from a chair, walk 3 meters, turn, return, and sit down

  3. Change from Baseline to Week 78 in Patient-Reported Outcomes Measurement Information System (PROMIS) Physical Function Item Bank v2.0-Physical Function-Short Form 20a Questionnaire

    Time frame: Baseline through Week 78

    Assessment of patient-reported physical function using the PROMIS Physical Function Short Form 20a questionnaire

  4. Change from Baseline to Week 78 in PROMIS Fatigue

    Time frame: Baseline through Week 78

    Assessment of patient-reported fatigue using the PROMIS Fatigue questionnaire

  5. Change from Baseline to Week 78 in Worst Pain Numeric Rating Scale (NRS)

    Time frame: Baseline through Week 78

    Assessment of the severity and impact of fatigue over the previous 7 days using the self-reported PROMIS Fatigue Short Form 6a questionnaire

  6. Change from Baseline to Week 78 in Patient Global Impression of Severity/Change (PGI-S/PGI-C)

    Time frame: Baseline through Week 78

    Assessment of participant-reported disease severity and change in disease status using the Patient Global Impression of Severity/Change (PGI-S/PGI-C) scales

  7. Change from Baseline to Week 78 in Quality of Life in Neurological Disorders (NeuroQoL) Upper Extremity Function

    Time frame: Baseline through Week 78

    Assessment of health-related quality of life using the Quality of Life in Neurological Disorders (Neuro-QoL) questionnaire

  8. Change from Baseline to Week 78 in NeuroQoL Sleep Disturbance

    Time frame: Baseline through Week 78

    Assessment of participant-reported sleep disturbance using the Neuro-QoL Sleep Disturbance questionnaire

  9. Change from Baseline to Week 78 in DUX4-regulated plasma KHDC1L

    Time frame: Baseline through Week 78

    Assess of changes from baseline in circulating PD biomarkers relevant to FSHD

  10. Change from Baseline to Week 78 in Serum CK

    Time frame: Baseline through Week 78

    Assess of changes from baseline in circulating PD biomarkers relevant to FSHD

Study contacts

Contact information is provided by the study sponsor or research team.

Avidity Biosciences, Inc., A Novartis Company

CONTACT

[email protected]

1-888-669-6682

Avidity Biosciences, Inc., A Novartis Company

CONTACT

[email protected]

+41613241111

Sponsors and collaborators

Lead sponsor

Avidity Biosciences, Inc.

Industry

Registry information

Official study 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)

Acronym: FORTITUDE-3

Important dates

Study start
2025
Primary completion
2028
Study completion
2028
First posted
Jun 26, 2025
Registry last updated
Jul 27, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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