AOC 1044 for Duchenne Muscular Dystrophy

(EXPLORE44 Trial)

Recruiting at10 trial locations

Age: < 65

Sex: Male

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Waitlist Available

Sponsor: Avidity Biosciences, Inc.

Trial Summary

What is the purpose of this trial?

AOC 1044-CS1 (EXPLORE44) is a Phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of single and multiple ascending doses of AOC 1044 in healthy adult volunteers and participants with DMD mutations amenable to exon 44 skipping. Part A is a single dose design with multiple cohorts (dose levels) in healthy adult volunteers. Part B is a multiple-ascending dose design with 3 cohorts (dose levels) in participants with Duchenne.

Research Team

Carmen Castrillo, MD

Principal Investigator

Avidity Biosciences, Inc.

Eligibility Criteria

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.

Inclusion Criteria

I weigh at least 23 kg.

I was diagnosed with DMD or showed symptoms by age 6.

Part A: Body mass index (BMI) of 18.5 to 32.0 kg/m2

Exclusion Criteria

I have previously undergone cell or gene therapy.

Part B: Serum hemoglobin < lower limit of normal

My biceps muscles cannot be biopsied.

Treatment Details

Interventions

• AOC 1044 (Exon Skipping Agent)

Trial Overview 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.

Participant Groups

Group I: AOC 1044-CS1 Part B - Multiple Ascending Dose Levels 1-3 Experimental Treatment 1 Intervention

AOC 1044 will be administered three times.

Group II: AOC 1044-CS1 Part A - Single Dose Levels 1-5 Experimental Treatment 1 Intervention

AOC 1044 will be administered once.

Group III: AOC 1044-CS1 Part A - Single Dose: Placebo Placebo Group 1 Intervention

Placebo will be administered once.

Group IV: AOC 1044-CS1 Part B - Multiple Ascending Dose: Placebo Placebo Group 1 Intervention

Placebo will be administered three times.

Who Is Running the Clinical Trial?

Avidity Biosciences, Inc.

Lead Sponsor

Trials

8

Recruited

960+