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ACTIVE NOT RECRUITING
NCT06244082
PHASE2

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

Sponsor: Avidity Biosciences, Inc.

View on ClinicalTrials.gov

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.

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

Key Details

Gender

MALE

Age Range

7 Years - 27 Years

Study Type

INTERVENTIONAL

Enrollment

39

Start Date

2024-01-22

Completion Date

2027-07-31

Last Updated

2025-05-14

Healthy Volunteers

No

Interventions

DRUG

AOC 1044

AOC 1044 will be administered via intravenous (IV) infusion

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

Rare Disease Research - Atlanta

Atlanta, Georgia, United States

University of Massachusetts Medical School

Worcester, Massachusetts, United States

Gillette Children's Specialty Healthcare

Saint 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