A Phase 3 interventional study of AOC-1020 and Placebo in Facioscapulohumeral Muscular Dystrophy, FSHD and FSHD - Facioscapulohumeral Muscular Dystrophy, sponsored by Avidity Biosciences, Inc.. Recruiting at 46 sites in 10 countries. Open to participants aged 16 Years to 70 Years. Per ClinicalTrials.gov, last updated 2026-07-27.
Sponsored by Avidity Biosciences, Inc. · Phase 3, Interventional, and Treatment
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)
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.
Exclusion Criteria:
Del-brax (AOC 1020) will be administered 13 times
Drug: AOC-1020
Saline will be administered 13 times
Drug: Placebo
Del-brax will be administered by intravenous (IV) infusion.
Also known as: Del-brax
Placebo will be administered by intravenous (IV) infusion.
Also known as: Saline
Change from Baseline to Week 78 in Quantitative Muscle Testing (QMT) total composite score (PPN)
Assessment of muscle strength using standardized quantitative measurements
Time frame: Baseline through Week 78
Change from Baseline to Week 78 in 10MWRT
Assessment of the time required to walk or run 10 meters as quickly and safely as possible
Time frame: Baseline through Week 78
Change from Baseline to Week 78 in Timed Up-and-Go (TUG)
Assessment of functional mobility by measuring the time required to stand up from a chair, walk 3 meters, turn, return, and sit down
Time frame: Baseline through Week 78
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
Assessment of patient-reported physical function using the PROMIS Physical Function Short Form 20a questionnaire
Time frame: Baseline through Week 78
Change from Baseline to Week 78 in PROMIS Fatigue
Assessment of patient-reported fatigue using the PROMIS Fatigue questionnaire
Time frame: Baseline through Week 78
Change from Baseline to Week 78 in Worst Pain Numeric Rating Scale (NRS)
Assessment of the severity and impact of fatigue over the previous 7 days using the self-reported PROMIS Fatigue Short Form 6a questionnaire
Time frame: Baseline through Week 78
Change from Baseline to Week 78 in Patient Global Impression of Severity/Change (PGI-S/PGI-C)
Assessment of participant-reported disease severity and change in disease status using the Patient Global Impression of Severity/Change (PGI-S/PGI-C) scales
Time frame: Baseline through Week 78
Change from Baseline to Week 78 in Quality of Life in Neurological Disorders (NeuroQoL) Upper Extremity Function
Assessment of health-related quality of life using the Quality of Life in Neurological Disorders (Neuro-QoL) questionnaire
Time frame: Baseline through Week 78
Change from Baseline to Week 78 in NeuroQoL Sleep Disturbance
Assessment of participant-reported sleep disturbance using the Neuro-QoL Sleep Disturbance questionnaire
Time frame: Baseline through Week 78
Change from Baseline to Week 78 in DUX4-regulated plasma KHDC1L
Assess of changes from baseline in circulating PD biomarkers relevant to FSHD
Time frame: Baseline through Week 78
Change from Baseline to Week 78 in Serum CK
Assess of changes from baseline in circulating PD biomarkers relevant to FSHD
Time frame: Baseline through Week 78
Plan to share: Undecided
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
Contact study teamGet an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Muscular Dystrophy, Facioscapulohumeral→
Avidity Biosciences, Inc.