Ignite Creation Date:
2024-05-05 @ 5:34 PM
Last Modification Date:
2024-10-26 @ 9:33 AM
Study NCT ID:
NCT00481546
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2023-07-03
First Post:
2007-05-31
Brief Title:
Phase I Trial of Gene Vector to Patients With Retinal Disease Due to RPE65 Mutations
Sponsor:
University of Pennsylvania
Organization:
University of Pennsylvania
Study Overview
Official Title:
Phase I Trial of Ocular Subretinal Injection of a Recombinant Adeno-Associated Virus rAAV2-CBSB-hRPE65 Gene Vector to Patients With Retinal Disease Due to RPE65 Mutations Clinical Trials of Gene Therapy for Leber Congenital Amaurosis
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2024-07
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
LCA
Brief 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
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
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| Grant 1 U10 EY017280-01 | None | None | None |
| UP IRB 804582 | None | None | None |
| UP IBC 06-105 | None | None | None |
| UF GCRC 675 | None | None | None |
| UF IBC RD 2795 | None | None | None |
| WIRB 20061300 | None | None | None |