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

NCT06244082 | Active Not Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: AOC 1044-CS2
• Study Type: interventional
• Target: 39
• Locations: 1 country
• Sites: 10

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.

Conditions

DMD; Duchenne Muscular Dystrophy; Duchenne; Exon 44

Keywords

EXPLORE44; EXPLORE44-OLE; EXPLORE44 OLE; Avidity Biosciences; Avidity; AOC 1044; AOC 1044-CS1; AOC 1044-CS2; AOC

Outcome Measures

Primary Outcomes (1)

• Incidence of Treatment Emergent Adverse Events (TEAEs)

  Through study completion (approximately 2 years)

Secondary Outcomes (1)

• Change from baseline in serum creatine kinase concentration at Study Weeks 24, 48, and 102

  Through study completion (approximately 2 years)

Study Arms (1)

AOC 1044 Multiple Dose Levels

EXPERIMENTAL

AOC 1044 will be IV infused every 6 weeks for approximately 2 years.

Drug: AOC 1044

Interventions

AOC 1044 DRUG

AOC 1044 will be administered via intravenous (IV) infusion

AOC 1044 Multiple Dose Levels

Eligibility Criteria

• Age: 7 Years - 27 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Rollover Participants:
• Satisfactorily completed AOC 1044-CS1 (EXPLORE44) as determined by the Investigator and Sponsor
• No significant tolerability issues with AOC 1044
• De novo Participants:
• Aged 7 to 27 years, inclusive, at the time of informed consent
• Clinical diagnosis of DMD or clear onset of DMD symptoms at or before the age of 6 years
• Confirmation of DMD gene mutation amenable to exon 44 skipping
• Weight ≥ 23 kg
• Ambulatory or non-ambulatory
• Ambulatory participants: LVEF ≥50% and FVC≥50%
• Non-ambulatory participants: LVEF ≥45% and FVC≥40%
• PUL 2.0 entry item A ≥3
• If on corticosteroids, stable dose for 30 days before screening and throughout the study

You may not qualify if:

• Rollover Participants:
• Presence of any new condition or worsening of existing condition that could affect a participant\'s safety or ability to comply with study procedures
• De novo Participants:
• Serum hemoglobin \< lower limit of normal
• Uncontrolled hypertension or diabetes
• Prior treatment with any cell or gene therapy
• Prior treatment with another exon 44 skipping agent within 6 months prior to informed consent
• Recently treated with an investigational drug
• History of multiple drug allergies

Sponsors & Collaborators

• Avidity Biosciences, Inc.: lead

Study Sites (10)

Arkansas Children's Hospital

Little Rock, Arkansas, 72202, United States

Location

University of California, San Diego, Rady's Children's Hospital

La Jolla, California, 92037, United States

Location

UC Davis Medical Center

Sacramento, California, 95817, United States

Location

Lucille Packard Children's Hospital at Stanford

San Carlos, California, 94070, United States

Location

Rare Disease Research - Atlanta

Atlanta, Georgia, 30329, United States

Location

University of Massachusetts Medical School

Worcester, Massachusetts, 01655, United States

Location

Gillette Children's Specialty Healthcare

Saint Paul, Minnesota, 55101, United States

Location

Rare Disease Research

Hillsborough, North Carolina, 27278, United States

Location

Abigail Wexner Research Institute at Nationwide Children's Hospital

Columbus, Ohio, 43215, United States

Location

Neurology Rare Disease Center

Denton, Texas, 76208, United States

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Carmen Castrillo, MD

  Avidity Biosciences, Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Masking Details: Randomization from the parent study, AOC 1044-CS1 will remain blinded.
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 26, 2024
• First Posted: February 6, 2024
• Study Start: January 22, 2024
• Primary Completion (Estimated): April 30, 2027
• Study Completion (Estimated): July 31, 2027
• Last Updated: May 14, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (10)