Ph2 Open-label Study of AOC 1044 in Duchenne Muscular Dystrophy Participants With Mutations Amenable to Exon44 Skipping
NCT ID: NCT06244082
Last Updated: 2025-05-14
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE2
39 participants
INTERVENTIONAL
2024-01-22
2027-07-31
Brief Summary
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Detailed Description
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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.
Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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AOC 1044 Multiple Dose Levels
AOC 1044 will be IV infused every 6 weeks for approximately 2 years.
AOC 1044
AOC 1044 will be administered via intravenous (IV) infusion
Interventions
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AOC 1044
AOC 1044 will be administered via intravenous (IV) infusion
Eligibility Criteria
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Inclusion Criteria
* 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
Exclusion Criteria
* 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
7 Years
27 Years
MALE
No
Sponsors
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Avidity Biosciences, Inc.
INDUSTRY
Responsible Party
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Principal Investigators
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Carmen Castrillo, MD
Role: STUDY_DIRECTOR
Avidity Biosciences, Inc.
Locations
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Arkansas Children&#39;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&#39;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
Countries
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Other Identifiers
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AOC 1044-CS2
Identifier Type: -
Identifier Source: org_study_id
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