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. (part of Novartis) · 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.
79 studies on the registry are indexed under Muscular Dystrophy, Facioscapulohumeral; 26 are open to participants now.
This study's planned enrollment of 200 is above the median of 48 across 49 interventional studies indexed under Muscular Dystrophy, Facioscapulohumeral.
Browse Muscular Dystrophy, Facioscapulohumeral studies →Avidity Biosciences, Inc. is the lead sponsor of 11 studies on the registry; 4 are open to participants now.
Of its 5 completed or terminated interventional studies of FDA-regulated products, 1 (20%) have results posted.
First submitted before Avidity Biosciences, Inc. became part of Novartis.
Counted across the registry records on this site, refreshed daily.
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Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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
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Plan to share: Undecided
Started Jun 2025 (actual). Primary completion is estimated for May 2028, 35 months after the start.
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Studies of the drug tested here: AOC 1020
Muscular Dystrophy, Facioscapulohumeral →