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

May 12, 2025 updated by: Avidity Biosciences, Inc.

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

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.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Actual)

39

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Arkansas
      • Little Rock, Arkansas, United States, 72202
        • Arkansas Children's Hospital
    • California
      • La Jolla, California, United States, 92037
        • University of California, San Diego, Rady's Children's Hospital
      • Sacramento, California, United States, 95817
        • UC Davis Medical Center
      • San Carlos, California, United States, 94070
        • Lucille Packard Children's Hospital at Stanford
    • Georgia
      • Atlanta, Georgia, United States, 30329
        • Rare Disease Research - Atlanta
    • Massachusetts
      • Worcester, Massachusetts, United States, 01655
        • University of Massachusetts Medical School
    • Minnesota
      • St. Paul, Minnesota, United States, 55101
        • Gillette Children's Specialty Healthcare
    • North Carolina
      • Hillsborough, North Carolina, United States, 27278
        • Rare Disease Research
    • Ohio
      • Columbus, Ohio, United States, 43215
        • Abigail Wexner Research Institute at Nationwide Children's Hospital
    • Texas
      • Denton, Texas, United States, 76208
        • Neurology Rare Disease Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult

Accepts Healthy Volunteers

No

Description

Key Inclusion Criteria

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

Key Exclusion Criteria

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: AOC 1044 Multiple Dose Levels
AOC 1044 will be IV infused every 6 weeks for approximately 2 years.
Drug: AOC 1044
AOC 1044 will be administered via intravenous (IV) infusion

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Incidence of Treatment Emergent Adverse Events (TEAEs)
Time Frame: Through study completion (approximately 2 years)
Through study completion (approximately 2 years)

Secondary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Avidity Biosciences, Inc.

Investigators

  • Study Director: Carmen Castrillo, MD, Avidity Biosciences, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 22, 2024

Primary Completion (Estimated)

April 30, 2027

Study Completion (Estimated)

July 31, 2027

Study Registration Dates

First Submitted

January 26, 2024

First Submitted That Met QC Criteria

February 2, 2024

First Posted (Actual)

February 6, 2024

Study Record Updates

Last Update Posted (Actual)

May 14, 2025

Last Update Submitted That Met QC Criteria

May 12, 2025

Last Verified

May 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • AOC 1044-CS2

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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