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NCT00428935 · ClinicalTrials.gov registry record · Phase 1
Safety Study of Mini-dystrophin Gene to Treat Duchenne Muscular Dystrophy
A Phase 1 study, sponsored by Nationwide Children's Hospital.
- Completed
- Registry status
- Phase 1
- Development phase
- 6
- Enrollment target
NCT00428935: Completed Phase 1 study, sponsored by Nationwide Children's Hospital.
NCT00428935 is a Phase 1 study that has completed, run by Nationwide Children's Hospital. The registered enrollment target is 6 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (90% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00428935, a Phase 1 study, has completed, sponsored by Nationwide Children's Hospital.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 6 participants
- Enrollment target
Study Summary
The purpose of this study is to determine the safety of a miniature dystrophin gene in the treatment of progressive muscle weakness due to Duchenne Muscular Dystrophy (DMD).
Primary Outcome
Physical Exams assessing major organ systems and safety labs (GGT, Bilirubin, Glucose, Amylase, CBC/Diff, AFP, Platelets, PT/PTT, Creatinine, Electrolytes, Total protein, Alkaline phosphatase, and Urinalysis)
Interventions
- BIOLOGICAL rAAV2.5-CMV-minidystrophin (d3990)
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 6 participants |
| Start Date | 2006-03 |
| Est. Completion | 2010-07 |
| Phase | Phase 1 |
What the finished NCT00428935 record still lists
NCT00428935 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 6 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (90% lower).
The record links to 0 conditions, and to 1 intervention - of which rAAV2.5-CMV-minidystrophin (d3990) is the first listed.
NCT00428935 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT00428935 about?
NCT00428935 is a clinical study titled "Safety Study of Mini-dystrophin Gene to Treat Duchenne Muscular Dystrophy". The purpose of this study is to determine the safety of a miniature dystrophin gene in the treatment of progressive muscle weakness due to Duchenne Muscular Dystrophy (DMD).
What is the current status of trial NCT00428935?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 6 participants. The study started on 2006-03. Estimated completion is 2010-07.
What interventions are being tested in trial NCT00428935?
The interventions under investigation include: rAAV2.5-CMV-minidystrophin (d3990) (BIOLOGICAL).
Who is sponsoring clinical trial NCT00428935?
This trial is sponsored by Nationwide Children's Hospital, which has 188 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT00428935's enrollment target sits among peer trials
6 1974th of 2000 higher than 12 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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