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Exon Skipping Agent

AOC 1044 Multiple Dose Levels for Duchenne Muscular Dystrophy (EXPLORE44OLE Trial)

Phase 2

Waitlist Available

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 through study completion (approximately 2 years)

Awards & highlights

EXPLORE44OLE Trial 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.Check my eligibility

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.See study design

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.

EXPLORE44OLE Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ through study completion (approximately 2 years)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~through study completion (approximately 2 years)

This trial's timeline: 3 weeks for screening, Varies for treatment, and through study completion (approximately 2 years) for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Change from baseline to biopsy visit in dystrophin protein level as measured in skeletal muscle by western blot

Secondary outcome measures

Change from baseline to biopsy visit in dystrophin protein levels as measured in skeletal muscle by mass spectrometry

Change from baseline to biopsy visit in exon skipping as measured in skeletal muscle

Incidence of treatment emergent adverse events (TEAEs)

EXPLORE44OLE 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.

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

Avidity Biosciences, Inc.Lead Sponsor

5 Previous Clinical Trials

661 Total Patients Enrolled

Mark Stahl, MD, PhDStudy DirectorAvidity Biosciences, Inc.

1 Previous Clinical Trials

64 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Has the Food and Drug Administration granted approval for multiple dose levels of AOC 1044?

"Based on our evaluations at Power, the safety rating for AOC 1044 Multiple Dose Levels is a 2. This assessment reflects the Phase 2 trial status where there is existing safety data but limited evidence of effectiveness."

Answered by AI

Are individuals still eligible to apply for participation in this ongoing trial?

"According to information from clinicaltrials.gov, patient recruitment for this trial has concluded. The trial was first listed on 1/22/2024 and last updated on 2/2/2024. Despite the closure of this particular study, there are currently 97 other trials actively seeking participants."

Answered by AI

Which individuals are eligible to participate in this research investigation?

"Individuals aged between 7 and 27 years with a confirmed diagnosis of Duchenne muscular dystrophy are eligible for enrollment in this study, which has space for up to 24 participants."

Answered by AI

Is the clinical trial open to individuals younger than 55 years old?

"Patients aged between 7 and 27 are eligible for participation in this clinical study. There are a total of 66 trials focused on minors under 18 years old, while there are 55 studies targeting individuals above the age of 65."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

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

2024. "Ph2 Open-label Study of AOC 1044 in Duchenne Muscular Dystrophy Participants With Mutations Amenable to Exon44 Skipping". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT06244082.

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