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NCT00749957 · ClinicalTrials.gov registry record · Phase 1
Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis
A Phase 1 study, sponsored by Beacon Therapeutics.
- Completed
- Registry status
- Phase 1
- Development phase
- 12
- Enrollment target
NCT00749957: Completed Phase 1 study, sponsored by Beacon Therapeutics.
NCT00749957 is a Phase 1 study that has completed, run by Beacon Therapeutics. The registered enrollment target is 12 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (80% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00749957, a Phase 1 study, has completed, sponsored by Beacon Therapeutics.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 12 participants
- Enrollment target
Study Summary
The purpose of the study is to evaluate the safety and efficacy of an adeno-associated virus vector expressing RPE65 in patients with Leber congenital amaurosis caused by mutations in the RPE65 gene. Funding Source - FDA OOPD
Interventions
- BIOLOGICAL rAAV2-CB-hRPE65
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 12 participants |
| Start Date | 2009-06-17 |
| Est. Completion | 2017-09-22 |
| Phase | Phase 1 |
What the finished NCT00749957 record still lists
NCT00749957 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 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (80% lower).
The record links to 0 conditions, and to 1 intervention - of which rAAV2-CB-hRPE65 is the first listed.
NCT00749957 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT00749957 about?
NCT00749957 is a clinical study titled "Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis". The purpose of the study is to evaluate the safety and efficacy of an adeno-associated virus vector expressing RPE65 in patients with Leber congenital amaurosis caused by mutations in the RPE65 gene. Funding Source - FDA OOPD
What is the current status of trial NCT00749957?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 12 participants. The study started on 2009-06-17. Estimated completion is 2017-09-22.
What interventions are being tested in trial NCT00749957?
The interventions under investigation include: rAAV2-CB-hRPE65 (BIOLOGICAL).
Who is sponsoring clinical trial NCT00749957?
This trial is sponsored by Beacon Therapeutics, which has 12 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT00749957's enrollment target sits among peer trials
12 1852nd of 2000 higher than 90 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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