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

Phase 2

• 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 Pharmacodynamics and 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 pharmacodynamic effect of AOC 1044 on dystrophin protein production in skeletal muscle as well as the long-term safety, tolerability, pharmacokinetics, and exploratory efficacy of AOC 1044.

Participants from AOC 1044-CS1 Part B are eligible to enroll in AOC 1044-CS2 if they have satisfactorily completed AOC 1044-CS1.

All participants who enroll in AOC 1044-CS2 will receive active treatment regardless of whether they received active treatment or placebo in AOC 1044-CS1. The treatment period is 2 years with IV dosing every 6 or 8 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
• Status Verified: 2024-02
• Study First Submitted QC: 2024-02-02
• Last Update Submitted: 2024-02-02
• Study First Posted: 2024-02-06
• Last Update Posted: 2024-02-06
• Primary Completion: 2025-12-31
• Study Completion: 2026-12-31

Recruitment & Eligibility

• Status: ENROLLING_BY_INVITATION
• Sex: Male
• Target Recruitment: 24

Inclusion Criteria

Not provided

Read More

Exclusion Criteria

Not provided

Read More

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 or 8 weeks for approximately 2 years.

Primary Outcome Measures

Name	Time	Method
Change from baseline to biopsy visit in dystrophin protein level as measured in skeletal muscle by western blot	Baseline collection in AOC 1044-CS1 to 8 months after targeted dose starts in AOC 1044-CS2

Secondary Outcome Measures

Name	Time	Method
Incidence of treatment emergent adverse events (TEAEs)	Through study completion (approximately 2 years)
Change from baseline to biopsy visit in dystrophin protein levels as measured in skeletal muscle by mass spectrometry	Baseline collection in AOC 1044-CS1 to 8 months after targeted dose starts in AOC 1044-CS2.
Change from baseline to biopsy visit in exon skipping as measured in skeletal muscle	Baseline collection in AOC 1044-CS1 to 8 months after targeted dose starts in AOC 1044-CS2.

Trial Locations

Locations (10)

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

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

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

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

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

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

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

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

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

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