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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

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Trial Profile

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

Status: Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 29 Apr 2024

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At a glance

  • Drugs AOC 1044 (Primary)
  • Indications Duchenne muscular dystrophy
  • Focus Adverse reactions
  • Acronyms EXPLORE44
  • Sponsors Avidity Biosciences
  • Most Recent Events

    • 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.
    • 15 Feb 2024 According to an Avidity Biosciences media release, data from this study will be presented at the 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference being held March 3-6, 2024, in Orlando, Florida.
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