NCT06244082

Ph2 Open-label Study of AOC 1044 in Duchenne Muscular Dystrophy Participants With Mutations Amenable to Exon44 Skipping

A Phase 2 Open-label Study to Evaluate the Pharmacodynamics and Long-Term Safety and Tolerability of AOC 1044 Administered Intravenously to DMD Participants With Mutations Amenable to Exon 44 Skipping

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 39 (actual)
• Sponsor: Avidity Biosciences, Inc. · Industry
• Sex: Male
• Age: 7 Years – 27 Years
• Healthy volunteers: Not accepted

Summary

AOC 1044-CS2 (EXPLORE44-OLE) is an Open-label Study to Evaluate the Long-Term Safety and Tolerability of AOC 1044 Administered Intravenously to DMD Participants with Mutations Amenable to Exon 44 Skipping.

Detailed description

AOC 1044-CS2 (EXPLORE44-OLE) is an open label, extension study to Part B of AOC 1044-CS1 (EXPLORE44). AOC 1044-CS2 is designed to evaluate the long-term safety, tolerability, pharmacokinetics, and exploratory efficacy of AOC 1044. All participants who enroll in AOC 1044-CS2 will receive active treatment. The treatment period is 2 years with IV dosing every 6 weeks. Once participants have completed active treatment, they will be followed through a 3-month safety follow-up period. The sponsor may extend active treatment beyond 2 years at a future timepoint.

Conditions

• DMD
• Duchenne Muscular Dystrophy
• Duchenne
• Exon 44

Interventions

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

Timeline

• Start date: 2024-01-22
• Primary completion: 2027-04-30
• Completion: 2027-07-31
• First posted: 2024-02-06
• Last updated: 2025-05-14

Locations

10 sites across 1 country: United States

Regulatory

• FDA-regulated drug study