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Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2

Leber Congenital Amaurosis Clinical Trial

Official title:
A Follow-On Study to Evaluate the Safety of Re-Administration of Adeno-Associated Viral Vector Containing the Gene for Human RPE65 [AAV2-hRPE65v2] to the Contralateral Eye in Subjects With Leber Congenital Amaurosis (LCA) Previously Enrolled in a Phase 1 Study

Clinical Trial Summary

The study is a follow-on to a Phase 1 dose-escalation and safety study.

Clinical Trial Description

The study is a follow-on to a Phase 1 dose-escalation and safety study (closed to enrollment as of June 2009). Up to twelve adults and children with a molecular diagnosis of biallelic RPE65 mutations, who have participated in the earlier Phase 1 study, and who meet all study eligibility criteria, will receive AAV2-hRPE65v2 vector in the previously uninjected, contralateral eye to evaluate the safety of bilateral, sequential subretinal administration of AAV2-hRPE65v2. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT01208389
Study type Interventional
Source Spark Therapeutics
Contact
Status Active, not recruiting
Phase Phase 1/Phase 2
Start date November 2010
Completion date June 2030
View Details
See also
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