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NCT00481546 · ClinicalTrials.gov registry record · Phase 1
Phase I Trial of Gene Vector to Patients With Retinal Disease Due to RPE65 Mutations
A Phase 1 study of Retinal Diseases and Amaurosis of Leber, sponsored by University of Pennsylvania.
- Active
- Registry status
- Phase 1
- Development phase
- 15
- Enrollment target
- 2
- Study locations
NCT00481546: Active Phase 1 study of Retinal Diseases and Amaurosis of Leber, sponsored by University of Pennsylvania.
NCT00481546 is a Phase 1 study of Retinal Diseases and Amaurosis of Leber that is active but no longer recruiting, run by University of Pennsylvania. The registered enrollment target is 15 participants, below the 395-participant average among 8 other Retinal Diseases trials with a reported enrollment target (96% lower). The trial reports 2 study locations across 2 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00481546, a Phase 1 study of Retinal Diseases and Amaurosis of Leber, is active but no longer recruiting, sponsored by University of Pennsylvania.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 15 participants
- Enrollment target
- 2
- Study locations
Study Summary
A recombinant adeno-associated virus serotype 2 (rAAV2) vector has been altered to carry the human RPE65 (hRPE65) gene. This vector has been shown to restore vision in animal models that resemble human RPE65-associated Leber congenital amaurosis (LCA), an incurable retinal degeneration that causes severe vision loss. The proposed study is an open label, Phase I clinical trial of subretinal rAAV2-CBSB-hRPE65 administration to individuals with RPE65-associated retinal disease. Five cohorts will be included in this trial. Cohorts 1, 2 and 4 will consist of individuals 18 years of age and older. Cohorts 3 and 5 will consist of individuals between the ages of 8 and 17, inclusive. Enrollment in Cohorts 3 and 5 will begin only after confirming the safety of rAAV2-CBSB-hRPE65 administration in the older groups of participants. This trial will lead to a greater understanding of the safety and thereby potential value of gene transfer in RPE65-associated retinal disease and will have implications for other forms of retinal degenerative disease amenable to this type of intervention. The goal of this clinical trial is to determine the safety of uniocular subretinal administration of rAAV2-CBSB-hRPE65 in individuals with RPE65-associated retinal disease. Ocular and systemic toxicity will be assessed prior to and following vector administration to determine if there are adverse changes that may be associated with vector administration.
Conditions Studied
Interventions
- GENETIC rAAV2-CBSB-hRPE65
Study Locations (2)
Florida
- Shands Children's Hospital, University of Florida - Gainesville
Pennsylvania
- Scheie Eye Institute, University of Pennsylvania - Philadelphia
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 15 participants |
| Start Date | 2007-07 |
| Est. Completion | 2026-06 |
| Phase | Phase 1 |
What the registry record for NCT00481546 still lists
NCT00481546 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 15 participants, a relatively small participant target, below the 395-participant average among 8 other Retinal Diseases trials with a reported enrollment target (96% lower).
The record links to 2 conditions, with Retinal Diseases appearing as the primary indexed condition, and to 1 intervention - of which rAAV2-CBSB-hRPE65 is the first listed.
NCT00481546 reports a single indexed study location in Florida, Pennsylvania.
Frequently Asked Questions
What is clinical trial NCT00481546 about?
NCT00481546 is a clinical study titled "Phase I Trial of Gene Vector to Patients With Retinal Disease Due to RPE65 Mutations". A recombinant adeno-associated virus serotype 2 (rAAV2) vector has been altered to carry the human RPE65 (hRPE65) gene. This vector has been shown to restore vision in animal models that resemble human RPE65-associated Leber congenital amaurosis (LCA), an incurable retinal degeneration that causes s...
What is the current status of trial NCT00481546?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 15 participants. The study started on 2007-07. Estimated completion is 2026-06.
What conditions does trial NCT00481546 study?
This clinical trial studies the following conditions: Retinal Diseases, Amaurosis of Leber.
What interventions are being tested in trial NCT00481546?
The interventions under investigation include: rAAV2-CBSB-hRPE65 (GENETIC).
Who is sponsoring clinical trial NCT00481546?
This trial is sponsored by University of Pennsylvania, which has 1,423 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT00481546 being conducted?
This trial has 2 study locations across Florida, Pennsylvania. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Where NCT00481546's enrollment target sits among peer trials
15 6th of 8 higher than 3 of 8 other Retinal Diseases trials
participants (enrollment target), bucketed by value
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