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Completed Phase 1Phase 2 Interventional Accepts healthy volunteers

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

ClinicalTrials.gov ID: NCT05670730

Public ClinicalTrials.gov record NCT05670730. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

ClinicalTrials.gov public records Last synced Oct 27, 2026, 6:59 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Official title

A Phase 1/2, Randomized, Placebo-controlled, Double-blind, Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamic Effects of Single and Multiple Ascending Doses of AOC 1044 Administered Intravenously to Healthy Adult Volunteers and Participants With DMD Mutations Amenable to Exon 44 Skipping

Brief summary

Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.

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.

Study identification

NCT ID
NCT05670730
Recruitment status
Completed
Study type
Interventional
Phase
Phase 1, Phase 2
Lead sponsor
Avidity Biosciences, Inc.
Industry
Enrollment
70 participants

Conditions and interventions

Eligibility (public fields only)

Age range
7 Years to 55 Years
Sex
Male
Healthy volunteers
Accepts healthy volunteers

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date
Nov 9, 2022 (Actual)
Primary completion
Nov 25, 2024 (Actual)
Completion
Nov 25, 2024 (Actual)
Last update posted
Jul 14, 2025

2022 – 2024

United States locations

U.S. sites
10
U.S. states
9
U.S. cities
10
Facility City State ZIP Site status
Arkansas Children's Little Rock Arkansas 72202 —
UCSD La Jolla California 92037 —
Stanford University Palo Alto California 94304 —
Rare Disease Research - Atlanta Atlanta Georgia 30329 —
University of Iowa Iowa City Iowa 52242 —
University of Massachusetts Chan Medical School Worcester Massachusetts 01655 —
Gillette Children's Saint Paul Minnesota 55101 —
Rare Disease Research NC Hillsborough North Carolina 27278 —
Abigail Research Institute at Nationwide Children's Hospital Columbus Ohio 43215 —
Worldwide Clinical Trials (Part A only) San Antonio Texas 78217 —

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

About this trial record page

What this page shows
Public field values for ClinicalTrials.gov record NCT05670730, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do
No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from
Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh
Last update posted Jul 14, 2025 · Synced Oct 27, 2026

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Open the official record

The complete public registry record, listed eligibility criteria, and available contact information for NCT05670730 live on ClinicalTrials.gov.

View official ClinicalTrials.gov record →