AAV9 U7snRNA Gene Therapy to Treat Boys With DMD Exon 2 Duplications.
Public ClinicalTrials.gov record NCT04240314. 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
Phase I/IIa Systemic Gene Delivery Clinical Trial of scAAV9.U7.ACCA for Exon 2 Duplication-Associated Duchenne Muscular Dystrophy
Study identification
- NCT ID
- NCT04240314
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 1, Phase 2
- Lead sponsor
- Megan Waldrop
- Other
- Enrollment
- 3 participants
Conditions and interventions
Conditions
Interventions
- scAAV9.U7.ACCA Biological
Biological
Eligibility (public fields only)
- Age range
- 6 Months to 13 Years
- Sex
- Male
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Jan 14, 2020
- Primary completion
- Nov 12, 2023
- Completion
- Jun 30, 2025
- Last update posted
- Sep 10, 2025
2020 – 2025
United States locations
- U.S. sites
- 1
- U.S. states
- 1
- U.S. cities
- 1
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | — |
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 NCT04240314, 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 Sep 10, 2025 · Synced Jul 22, 2026
Related: full search, browse by condition, browse by drug or therapy, browse by sponsor, browse by U.S. city.
Open the official record
The complete protocol, eligibility criteria, and contact information for NCT04240314 live on ClinicalTrials.gov.