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

Phase 2

Active, not recruiting

• Conditions: DMD; Duchenne Muscular Dystrophy; Duchenne; Exon 44
• Interventions: Drug: AOC 1044

• Registration Number: NCT06244082
• Lead Sponsor: Avidity Biosciences, Inc.

Brief 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.

Study Timeline

• Study Start: 2024-01-22
• Study First Submitted: 2024-01-26
• Study First Submitted QC: 2024-02-02
• Study First Posted: 2024-02-06
• Status Verified: 2025-05
• Last Update Submitted: 2025-05-12
• Last Update Posted: 2025-05-14
• Primary Completion: 2027-04-30
• Study Completion: 2027-07-31

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: Male
• Target Recruitment: 39

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
AOC 1044 Multiple Dose Levels	AOC 1044	AOC 1044 will be IV infused every 6 weeks for approximately 2 years.

Primary Outcome Measures

Name	Time	Method
Incidence of Treatment Emergent Adverse Events (TEAEs)	Through study completion (approximately 2 years)

Secondary Outcome Measures

Name	Time	Method
Change from baseline in serum creatine kinase concentration at Study Weeks 24, 48, and 102	Through study completion (approximately 2 years)

Trial Locations

Locations (10)

Rare Disease Research - Atlanta; 🇺🇸 Atlanta, Georgia, United States

University of Massachusetts Medical School; 🇺🇸 Worcester, Massachusetts, United States

Gillette Children's Specialty Healthcare; 🇺🇸 St. Paul, Minnesota, United States

Arkansas Children's Hospital; 🇺🇸 Little Rock, Arkansas, United States

University of California, San Diego, Rady's Children's Hospital; 🇺🇸 La Jolla, California, United States

Rare Disease Research; 🇺🇸 Hillsborough, North Carolina, United States

UC Davis Medical Center; 🇺🇸 Sacramento, California, United States

Lucille Packard Children's Hospital at Stanford; 🇺🇸 San Carlos, California, United States

Abigail Wexner Research Institute at Nationwide Children's Hospital; 🇺🇸 Columbus, Ohio, United States

Neurology Rare Disease Center; 🇺🇸 Denton, Texas, United States