A Phase 3 interventional study of AOC 1044 and Placebo in Muscular Dystrophies, Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy) and Muscular Disorders, Atrophic, sponsored by Avidity Biosciences, Inc.. Recruiting at 20 sites in 10 countries. Open to male participants aged 7 Years to 16 Years. Per ClinicalTrials.gov, last updated 2026-11-10.
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 1044 for the treatment of Duchenne Muscular Dystrophy (DMD) with Gene Mutations Amenable to Exon 44 Skipping
The study consists of a Screening Period of up to 44 Days, a Randomized 54 Week Treatment Period, a 54 Week Open Label Extension (OLE), and a Follow-Up Period of 6 weeks. Participants will be randomized to receive an intravenous infusion of either delpacibart-zotadirsen or placebo at the clinical study site every 6 weeks for a total of 9 doses. After completion of the randomized treatment period, all participants may enter the OLE portion of the study consisting of 9 doses of AOC 1044 regardless of group assignment in the randomized period. The final dose will occur at Week 102, followed by a final assessment at Week 108 and a safety follow-up visit at Week 114. 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.
126 studies on the registry are indexed under Myotonic Dystrophy; 50 are open to participants now.
This study's planned enrollment of 70 is above the median of 40 across 67 interventional studies indexed under Myotonic Dystrophy.
Browse Myotonic Dystrophy 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 after Avidity Biosciences, Inc. became part of Novartis.
Compared within Myotonic Dystrophy, this study's primary condition as ClinicalTrials.gg classifies it; the registry's first condition term is Muscular Dystrophies. The counts are of registry records indexed under each term, refreshed daily. How we classify →
Key Inclusion Criteria:
Key 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.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participants will receive AOC 1044 intravenously (IV) every 6 weeks (Q6W) during the 54-week double-blind treatment period, followed by a 54-week open-label treatment period in which the same participants will continue to receive AOC 1044 IV Q6W
Drug: AOC 1044
Participants will receive placebo (saline) IV Q6W during the 54-week double-blind treatment period, followed by a 54-week open-label treatment period in which all participants will then receive AOC 1044 IV Q6W
Drug: Placebo
AOC 1044 will be administered by intravenous (IV) infusion
Placebo will be administered by intravenous (IV) infusion
Change from Baseline in Time to Rise (TTR) Velocity at Week 54
Time frame: Baseline, Week 54
Change from Baseline in Creatine Kinase (CK) at Week 54
Time frame: Baseline, Week 54
Change from Baseline in 4-Stair Climb (4SC) Velocity at Week 54
Time frame: Baseline, Week 54
Change from Baseline in 10-Meter Walk/Run Test (10MWRT) Velocity at Week 54
Time frame: Baseline, Week 54
Change from Baseline in Stride Velocity 95th Centile (SV95C) at Week 54
Time frame: Baseline, Week 54
Change from Baseline in North Star Ambulatory Assessment (NSAA) Total Score at Week 54
Time frame: Baseline, Week 54
Change from Baseline in DMD Quality of Life (DMD-QoL) Score at Week 54
Time frame: Baseline, Week 54
Change from Baseline in Patient Global Impression of Severity (PGI-S) at Week 54
Time frame: Baseline, Week 54
Change from Baseline in Caregiver Global Impression of Severity (CaGI-S) at Week 54
Time frame: Baseline, Week 54
Patient Global Impression of Change (PGI-C) at Week 54
Time frame: Week 54
Caregiver Global Impression of Change (CaGI-C) at Week 54
Caregiver Global Impression of Change (CaGI-C) at Week 54
Time frame: Week 54
Change from Baseline in Quantitative Muscle Testing (QMT) at Week 54
Time frame: Baseline, Week 54
Plan to share: Undecided
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
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Studies of the drug tested here: Delpacibart zotadirsen