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AOC 1044 · 1 trial · 14 indications
| Arm | Type | Description |
|---|---|---|
| AOC 1044 | EXPERIMENTAL | 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 |
| Placebo | PLACEBO_COMPARATOR | 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 |
| Name | Type | Description |
|---|---|---|
| AOC 1044 | DRUG | AOC 1044 will be administered by intravenous (IV) infusion |
| Placebo | DRUG | Placebo will be administered by intravenous (IV) infusion |
Key Inclusion Criteria: * Ambulatory males with clinical and genetic diagnosis of DMD * Acceptable genetic test confirming dystrophin gene mutation amenable to exon 44 skipping * 7 to 16 years of age at time of consent * TTR and NSAA assessment completed within the protocol specified parameters at ...
AOC 1044, also known as delpacibart zotadirsen, is an investigational therapy for Duchenne muscular dystrophy (DMD) in patients with gene mutations amenable to exon 44 skipping. It is being studied in a Phase 3 clinical trial for this rare genetic muscle disease.
AOC 1044 targets dystrophin, a protein that is deficient or defective in Duchenne muscular dystrophy. The therapy is designed to address the underlying genetic cause of the disease by promoting exon 44 skipping, which aims to restore dystrophin production.
AOC 1044 is being developed by Avidity Biosciences, Inc., a biopharmaceutical company traded on the stock exchange under the ticker RNAM. The company is conducting clinical research on this investigational drug for Duchenne muscular dystrophy.
AOC 1044 is currently in Phase 3 clinical development. It is an investigational drug, meaning it has not been approved by regulatory authorities and is still being evaluated for safety and efficacy in clinical trials.
AOC 1044 is being studied in a Phase 3 clinical trial registered as NCT07587242. This randomized, double-blind, placebo-controlled study is recruiting approximately 70 male participants aged 7 years and older with Duchenne muscular dystrophy amenable to exon 44 skipping. The trial is enrolling in Belgium, France, Germany, Italy, Spain, and the United Kingdom.
Yes, AOC 1044 is also referred to as delpacibart zotadirsen. Both names refer to the same investigational drug being developed by Avidity Biosciences for Duchenne muscular dystrophy with mutations amenable to exon 44 skipping.