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NCT01054339 · ClinicalTrials.gov registry record · Phase 2
Safety & Efficacy Study of rAAV1-CB-hAAT for Alpha-1 Antitrypsin Deficiency
A Phase 2 study of Alpha-1 Antitrypsin Deficiency, sponsored by Beacon Therapeutics.
- Completed
- Registry status
- Phase 2
- Development phase
- 9
- Enrollment target
- 4
- Study locations
NCT01054339: Completed Phase 2 study of Alpha-1 Antitrypsin Deficiency, sponsored by Beacon Therapeutics.
NCT01054339 is a Phase 2 study of Alpha-1 Antitrypsin Deficiency that has completed, run by Beacon Therapeutics. The registered enrollment target is 9 participants, below the 2,341-participant average among 24 other Alpha-1 Antitrypsin Deficiency trials with a reported enrollment target (100% lower). The trial reports 4 study locations across 4 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01054339, a Phase 2 study of Alpha-1 Antitrypsin Deficiency, has completed, sponsored by Beacon Therapeutics.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 9 participants
- Enrollment target
- 4
- Study locations
Study Summary
Assessment of the safety and efficacy of intramuscular (IM) administration of a recombinant adenoassociated virus (rAAV) alpha-1 antitrypsin (AAT) vector (rAAV1-CB-hAAT) in AAT-deficient adults at three dosage levels \[6.0 × 10e11, 1.9 × 10e12 and 6.0 × 10e12 vector genome particles (vg) per kg body weight\]. Funding Sources - The FDA Office of Orphan Products Development and NIH National Heart, Lung, and Blood Institute
Conditions Studied
Interventions
- DRUG rAAV1-CB-hAAT
Study Locations (4)
Colorado
- National Jewish Health - Denver
Massachusetts
- University of Massachusetts Medical Center - Worcester
Ohio
- Cincinnati Children's Hospital Medical Center - Cincinnati
Other
- Beaumont Hospital - Dublin
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 9 participants |
| Start Date | 2010-06 |
| Est. Completion | 2015-10 |
| Phase | Phase 2 |
What the finished NCT01054339 record still lists
NCT01054339 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 2,341-participant average among 24 other Alpha-1 Antitrypsin Deficiency trials with a reported enrollment target (100% lower).
The record links to 1 condition, with Alpha-1 Antitrypsin Deficiency appearing as the primary indexed condition, and to 1 intervention - of which rAAV1-CB-hAAT is the first listed.
NCT01054339 reports a single indexed study location in Colorado, Massachusetts, Ohio.
Frequently Asked Questions
What is clinical trial NCT01054339 about?
NCT01054339 is a clinical study titled "Safety & Efficacy Study of rAAV1-CB-hAAT for Alpha-1 Antitrypsin Deficiency". Assessment of the safety and efficacy of intramuscular (IM) administration of a recombinant adenoassociated virus (rAAV) alpha-1 antitrypsin (AAT) vector (rAAV1-CB-hAAT) in AAT-deficient adults at three dosage levels \[6.0 × 10e11, 1.9 × 10e12 and 6.0 × 10e12 vector genome particles (vg) per kg body...
What is the current status of trial NCT01054339?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 9 participants. The study started on 2010-06. Estimated completion is 2015-10.
What conditions does trial NCT01054339 study?
This clinical trial studies the following conditions: Alpha-1 Antitrypsin Deficiency.
What interventions are being tested in trial NCT01054339?
The interventions under investigation include: rAAV1-CB-hAAT (DRUG).
Who is sponsoring clinical trial NCT01054339?
This trial is sponsored by Beacon Therapeutics, which has 12 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT01054339 being conducted?
This trial has 4 study locations across Colorado, Massachusetts, Ohio. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Where NCT01054339's enrollment target sits among peer trials
9 24th of 24 higher than 1 of 24 other Alpha-1 Antitrypsin Deficiency trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Alpha-1 Antitrypsin Deficiency 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.
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