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Study of AOC 1044 in Healthy Adult Volunteers and Participants With Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping

NCT05670730 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 70

Last updated 2025-07-14

No results posted yet for this study

Summary

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.

Conditions

Interventions

DRUG

AOC 1044

AOC 1044 will be administered via intravenous (IV) infusion

DRUG

Placebo

Placebo will be administered via intravenous (IV) infusion.

Sponsors & Collaborators

  • Avidity Biosciences, Inc.

    lead INDUSTRY

Principal Investigators

  • Carmen Castrillo, MD · Avidity Biosciences, Inc.

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
SEQUENTIAL

Eligibility

Min Age
7 Years
Max Age
55 Years
Sex
MALE
Healthy Volunteers
Yes

Timeline & Regulatory

Start
2022-11-09
Primary Completion
2024-11-25
Completion
2024-11-25
FDA Drug
Yes

Countries

  • United States

Study Locations

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Entities

Drugs
Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05670730 on ClinicalTrials.gov