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NCT00430768 · ClinicalTrials.gov registry record · Phase 1
Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin (AAT) Deficiency
A Phase 1 study, sponsored by University of Massachusetts, Worcester.
- Completed
- Registry status
- Phase 1
- Development phase
- 9
- Enrollment target
NCT00430768: Completed Phase 1 study, sponsored by University of Massachusetts, Worcester.
NCT00430768 is a Phase 1 study that has completed, run by University of Massachusetts, Worcester. The registered enrollment target is 9 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (85% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00430768, a Phase 1 study, has completed, sponsored by University of Massachusetts, Worcester.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 9 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 with the expectation that the AAT protein may be produced at normal levels. This study will evaluate the safety of the experimental gene transfer procedure in individuals with AAT deficiency. The study will also determine what dose may be required to achieve normal levels of AAT.
Primary Outcome
Adverse events considered possibly, probably or definitely related to study drug/study drug procedure Criteria to evaluate severity according to Attachment 2 of the Protocol 1. Mild toxicity, usually transient, requiring no special treatment and generally not interfering with usual daily activities 2. Moderate toxicity which may be ameliorated by simple therapeutic maneuvers, and impairs usual activities 3. Severe toxicity which requires therapeutic intervention and interrupts usual activities.
Interventions
- BIOLOGICAL rAAV1-CB-hAAT
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 9 participants |
| Start Date | 2006-02 |
| Est. Completion | 2015-01 |
| Phase | Phase 1 |
What the finished NCT00430768 record still lists
NCT00430768 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 9 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (85% lower).
The record links to 0 conditions, and to 1 intervention - of which rAAV1-CB-hAAT is the first listed.
NCT00430768 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT00430768 about?
NCT00430768 is a clinical study titled "Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin (AAT) 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 with the expectation that the AAT protein may be produced at normal levels. This study will e...
What is the current status of trial NCT00430768?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 9 participants. The study started on 2006-02. Estimated completion is 2015-01.
What interventions are being tested in trial NCT00430768?
The interventions under investigation include: rAAV1-CB-hAAT (BIOLOGICAL).
Who is sponsoring clinical trial NCT00430768?
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 NCT00430768's enrollment target sits among peer trials
9 1955th of 2000 higher than 42 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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