Study of AOC 1044 in Healthy Adult Volunteers and Participants With Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping
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.
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
- Enrollment
- 70 participants
Conditions and interventions
Conditions
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 8, 2022
- Primary completion
- Nov 24, 2024
- Completion
- Nov 24, 2024
- Last update posted
- Jul 13, 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 13, 2025 · Synced Sep 11, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT05670730 live on ClinicalTrials.gov.