NCT01208389

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

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

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 12 (estimated)
• Sponsor: Spark Therapeutics, Inc. · Industry
• Sex: All
• Age: 8 Years
• Healthy volunteers: Not accepted

Summary

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

Detailed 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.

Conditions

• Leber Congenital Amaurosis

Interventions

Type	Name	Description
BIOLOGICAL	voretigene neparvovec-rzyl	One time, subretinal administration of 1.5E11 vg AAV2-hRPE65v2 vector in 300 microliters to the contralateral, previously uninjected eye.

Timeline

• Start date: 2010-11-01
• Primary completion: 2027-10-01
• Completion: 2027-10-01
• First posted: 2010-09-24
• Last updated: 2025-04-29

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study