A Phase 1/2, Randomized, Placebo-controlled, Double-blind, Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamic Effects of Single and Multiple Ascending Doses of AOC 1044 Administered Intravenously to Healthy Adult Volunteers and Participants With DMD Mutations Amenable to Exon 44 Skipping
Latest Information Update: 29 Apr 2024
At a glance
- Drugs AOC 1044 (Primary)
- Indications Duchenne muscular dystrophy
- Focus Adverse reactions
- Acronyms EXPLORE44
- Sponsors Avidity Biosciences
- 18 Apr 2024 Trial design, presented at the 76th Annual Meeting of the American Academy of Neurology 2024
- 28 Feb 2024 According to an Avidity Biosciences media release, company is planning to share 5 mg/kg cohort data from this Phase 1/2 EXPLORE44 trial of people living with DMD44 in the second half of 2024.
- 20 Feb 2024 According to an Avidity Biosciences media release, the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation to AOC 1044, for the treatment of Duchenne muscular dystrophy, based on data from this trial.