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Exon Skipping Agent

AOC 1044 for Duchenne Muscular Dystrophy (EXPLORE44OLE Trial)

Phase 2
Recruiting
Research Sponsored by Avidity Biosciences, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be younger than 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights
No Placebo-Only Group

Summary

This trial is testing the safety and effectiveness of a drug called AOC 1044 when given through a vein to participants with a specific type of mutation in Duchenne muscular dystrophy.

Who is the study for?
This trial is for individuals with Duchenne Muscular Dystrophy (DMD) who have specific genetic mutations that can be treated by skipping exon 44. Participants must have completed a prior study, EXPLORE44, without significant issues tolerating the treatment.
What is being tested?
The trial is testing AOC 1044, an intravenous medication designed to skip exon 44 in DMD patients. It aims to assess the drug's long-term safety and its effect on the body over time in an open-label setting.
What are the potential side effects?
While specific side effects of AOC 1044 are not listed here, common side effects for treatments like this may include reactions at the infusion site, potential kidney or liver function changes, and general symptoms like fatigue or fever.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: AOC 1044 Multiple Dose LevelsExperimental Treatment1 Intervention
AOC 1044 will be IV infused every 6 weeks or 8 weeks for approximately 2 years.

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Who is running the clinical trial?

Avidity Biosciences, Inc.Lead Sponsor
7 Previous Clinical Trials
911 Total Patients Enrolled
Marc Morris, JD, MD, MSStudy DirectorAvidity Biosciences, Inc.
Mark Stahl, MD, PhDStudy DirectorAvidity Biosciences, Inc.
1 Previous Clinical Trials
64 Total Patients Enrolled
~18 spots leftby Dec 2025
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