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NCT00377416 · ClinicalTrials.gov registry record · Early Phase 1
Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin Deficiency
A Early Phase 1 study, sponsored by University of Massachusetts, Worcester.
- Completed
- Registry status
- Early Phase 1
- Development phase
- 12
- Enrollment target
NCT00377416: Completed Early Phase 1 study, sponsored by University of Massachusetts, Worcester.
NCT00377416 is a Early Phase 1 study that has completed, run by University of Massachusetts, Worcester. The registered enrollment target is 12 participants, below the 53-participant average among 2,764 other Early Phase 1 trials with a reported enrollment target (77% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00377416, a Early Phase 1 study, has completed, sponsored by University of Massachusetts, Worcester.
- COMPLETED
- Registry status
- Early Phase 1
- Development phase
- 12 participants
- Enrollment target
Study Summary
Individuals with a deficiency of the Alpha 1-antitrypsin (AAT) protein are at risk for developing emphysema and liver damage. Researchers have developed a way to introduce normal AAT genes into muscle cells so that the AAT protein is produced at normal levels. This study will evaluate the safety of the experimental gene transfer procedure in individuals with AAT deficiency.
Interventions
- GENETIC rAAV2-CB-hAAT Gene Vector
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 12 participants |
| Start Date | 2004-03 |
| Est. Completion | 2020-01 |
| Phase | Early Phase 1 |
What the finished NCT00377416 record still lists
NCT00377416 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 12 participants, a relatively small participant target, below the 53-participant average among 2,764 other Early Phase 1 trials with a reported enrollment target (77% lower).
The record links to 0 conditions, and to 1 intervention - of which rAAV2-CB-hAAT Gene Vector is the first listed.
NCT00377416 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT00377416 about?
NCT00377416 is a clinical study titled "Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin Deficiency". Individuals with a deficiency of the Alpha 1-antitrypsin (AAT) protein are at risk for developing emphysema and liver damage. Researchers have developed a way to introduce normal AAT genes into muscle cells so that the AAT protein is produced at normal levels. This study will evaluate the safety of ...
What is the current status of trial NCT00377416?
This trial is currently completed. It is a Early Phase 1 study. The enrollment target is 12 participants. The study started on 2004-03. Estimated completion is 2020-01.
What interventions are being tested in trial NCT00377416?
The interventions under investigation include: rAAV2-CB-hAAT Gene Vector (GENETIC).
Who is sponsoring clinical trial NCT00377416?
This trial is sponsored by University of Massachusetts, Worcester, which has 240 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT00377416's enrollment target sits among peer trials
12 1530th of 2000 higher than 401 of 2,000 other Early Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Early 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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