Study of AOC 1044 in Healthy Adult Volunteers and Participants With Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping
Completed · Phase 1/Phase 2 · Has a placebo group
Conditions studied: Duchenne Muscular Dystrophy, Exon 44
In brief
AOC 1044-CS1 (EXPLORE44) is a Phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of single and multiple ascending doses of AOC 1044 in healthy adult volunteers and participants with DMD mutations amenable to exon 44 skipping. Part A is a single dose design with multiple cohorts (dose levels) in healthy adult volunteers. Part B is a multiple-ascending dose design with 3 cohorts (dose levels) in participants with Duchenne.
Key facts
- Study ID
- NCT05670730
- Run by
- Avidity Biosciences, Inc.
- People needed
- 70
- Starts
- 2022-11-09
- Expected to finish
- 2024-11-25
- Last updated by the study team
- 2025-07-14
Who can join
Age: 7 and older, up to 55. Sex: male. Healthy volunteers: accepted.
Where it is running
- Arkansas Children's — Little Rock, Arkansas, United States
- UCSD — La Jolla, California, United States
- Stanford University — Palo Alto, California, United States
- Rare Disease Research - Atlanta — Atlanta, Georgia, United States
- University of Iowa — Iowa City, Iowa, United States
- University of Massachusetts Chan Medical School — Worcester, Massachusetts, United States
- Gillette Children's — Saint Paul, Minnesota, United States
- Rare Disease Research NC — Hillsborough, North Carolina, United States
- Abigail Research Institute at Nationwide Children's Hospital — Columbus, Ohio, United States
- Worldwide Clinical Trials (Part A only) — San Antonio, Texas, United States
Full record on ClinicalTrials.gov
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