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Clinical Trials in the USA / NCT06244082
Active, not recruiting Phase 2

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

NCT06244082 · tracked via the Priya Life Science USA tracker
Sponsor
Avidity Biosciences, Inc.
Phase
Phase 2
Started
2024-01-22
Last updated
2026-07-30

Condition(s) studied

DMDDuchenne Muscular DystrophyDuchenneExon 44

Investigational drug(s) / intervention(s)

AOC 1044

AOC 1044: AOC 1044 will be administered via intravenous (IV) infusion

Study 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.

Eligibility

Sex
MALE
Min age
7 Years
Max age
27 Years
Healthy volunteers
No
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

Primary outcome measure(s)

Trial sites (10)

FacilityCityRegionStatus
Arkansas Children's Hospital Little Rock Arkansas
University of California, San Diego, Rady's Children's Hospital La Jolla California
UC Davis Medical Center Sacramento California
Lucille Packard Children's Hospital at Stanford San Carlos California
Rare Disease Research - Atlanta Atlanta Georgia
University of Massachusetts Medical School Worcester Massachusetts
Gillette Children's Specialty Healthcare Saint Paul Minnesota
Rare Disease Research Hillsborough North Carolina
Abigail Wexner Research Institute at Nationwide Children's Hospital Columbus Ohio
Neurology Rare Disease Center Denton Texas

More Avidity Biosciences, Inc. trials in the USA

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06244082 on ClinicalTrials.gov ↗ ← All trials in the USA