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Exon Skipping Agent

AOC 1044 for Duchenne Muscular Dystrophy (EXPLORE44 Trial)

Phase 1 & 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 day 1-2 (0-24 hours after first dose) (part a); day 1-2 (0-24 hours after first dose) (part b)

Summary

This trial studies a new drug to treat Duchenne muscular dystrophy. It tests safety, how well it works and how it affects the body in healthy adults and people with Duchenne.

Who is the study for?
This trial is for healthy adults and those with Duchenne Muscular Dystrophy (DMD) who can benefit from exon 44 skipping. Participants must be aged 18-55 years with a BMI of 18.5 to 32 kg/m2.
What is being tested?
The study tests AOC 1044, which could potentially treat DMD by skipping exon 44 in the gene mutation. It's given in single or multiple doses to see how safe it is and how the body responds.
What are the potential side effects?
Possible side effects are not detailed here but will likely include reactions at the injection site, potential liver enzyme changes, and other symptoms related to altering muscle protein production.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~day 1-2 (0-24 hours after first dose) (part a); day 1-2 (0-24 hours after first dose) (part b)
This trial's timeline: 3 weeks for screening, Varies for treatment, and day 1-2 (0-24 hours after first dose) (part a); day 1-2 (0-24 hours after first dose) (part b) for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Secondary study objectives
Plasma pharmacokinetic (PK) parameters
Urine pharmacokinetic parameters

Trial Design

4Treatment groups
Experimental Treatment
Placebo Group
Group I: AOC 1044-CS1 Part B - Multiple Ascending Dose Levels 1-3Experimental Treatment1 Intervention
AOC 1044 will be administered three times.
Group II: AOC 1044-CS1 Part A - Single Dose Levels 1-5Experimental Treatment1 Intervention
AOC 1044 will be administered once.
Group III: AOC 1044-CS1 Part A - Single Dose: PlaceboPlacebo Group1 Intervention
Placebo will be administered once.
Group IV: AOC 1044-CS1 Part B - Multiple Ascending Dose: PlaceboPlacebo Group1 Intervention
Placebo will be administered three times.

Find a Location

Logistics

Participation is compensated

You will be compensated for participating in this trial.

Who is running the clinical trial?

Avidity Biosciences, Inc.Lead Sponsor
7 Previous Clinical Trials
894 Total Patients Enrolled
Mark Stahl, MD, PhDStudy DirectorAvidity Biosciences, Inc.
1 Previous Clinical Trials
35 Total Patients Enrolled
Marc Morris, MD, JD, MSStudy DirectorAvidity Biosciences, Inc.

Media Library

AOC 1044 (Exon Skipping Agent) Clinical Trial Eligibility Overview. Trial Name: NCT05670730 — Phase 1 & 2
Duchenne Muscular Dystrophy Research Study Groups: AOC 1044-CS1 Part B - Multiple Ascending Dose Levels 1-3, AOC 1044-CS1 Part A - Single Dose: Placebo, AOC 1044-CS1 Part A - Single Dose Levels 1-5, AOC 1044-CS1 Part B - Multiple Ascending Dose: Placebo
Duchenne Muscular Dystrophy Clinical Trial 2023: AOC 1044 Highlights & Side Effects. Trial Name: NCT05670730 — Phase 1 & 2
AOC 1044 (Exon Skipping Agent) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05670730 — Phase 1 & 2
~21 spots leftby Jan 2026
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