Ph2 Open-label Study of AOC 1044 in Duchenne Muscular Dystrophy Participants With Mutations Amenable to Exon44 Skipping — EXPLORE44OLE  

Duchenne Muscular Dystrophy Clinical Trial

Official title: 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 Enrolling by invitation

Clinical Trial 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.

Clinical Trial 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. ;

Related Conditions & MeSH terms

• DMD
• Duchenne Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

• NCT number: NCT06244082
• Study type: Interventional
• Source: Avidity Biosciences, Inc.
• Status: Enrolling by invitation
• Phase: Phase 2
• Start date: January 22, 2024
• Completion date: December 31, 2026