NCT05670730

Study of AOC 1044 in Healthy Adult Volunteers and Participants With Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping

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: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 70 (actual)
• Sponsor: Avidity Biosciences, Inc. · Industry
• Sex: Male
• Age: 7 Years – 55 Years
• Healthy volunteers: Accepted

Summary

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.

Conditions

• Duchenne Muscular Dystrophy
• Exon 44

Interventions

Type	Name	Description
DRUG	AOC 1044	AOC 1044 will be administered via intravenous (IV) infusion
DRUG	Placebo	Placebo will be administered via intravenous (IV) infusion.

Timeline

• Start date: 2022-11-09
• Primary completion: 2024-11-25
• Completion: 2024-11-25
• First posted: 2023-01-04
• Last updated: 2025-07-14

Locations

10 sites across 1 country: United States

Regulatory

• FDA-regulated drug study