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A Randomized, Double-Blind, Placebo-Controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple-Doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) Patients

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Trial Profile

A Randomized, Double-Blind, Placebo-Controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple-Doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) Patients

Status: Completed
Phase of Trial: Phase I/II

Latest Information Update: 05 Mar 2024

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At a glance

  • Drugs AOC 1001 (Primary)
  • Indications Myotonic dystrophy
  • Focus Adverse reactions; First in man; Proof of concept
  • Acronyms MARINA
  • Sponsors Avidity Biosciences

    • Most Recent Events

    • 07 Oct 2023 New positive AOC 1001 data from this trial presented in an Avidity Biosciences media release.
    • 26 Sep 2023 According to an Avidity Biosciences media release, data from this study will be presented at the 28th Annual Congress of the World Muscle Society (WMS).
    • 31 Aug 2023 According to an Avidity Biosciences media release, company will be presenting topline data analysis from this trial at a poster presentation at the 2023 Myotonic Dystrophy Foundation Annual Conference, being held September 7-9, 2023, in Washington, D.C.

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