NCT00749957

Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis

A Multiple-Site, Phase 1/2, Safety and Efficacy Trial of a Recombinant Adeno-associated Virus Vector Expressing RPE65 (rAAV2-CB-hRPE65) in Patients With Leber Congenital Amaurosis Type 2

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 12 (actual)
• Sponsor: Beacon Therapeutics · Industry
• Sex: All
• Age: 6 Years
• Healthy volunteers: Not accepted

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

Detailed description

This will be a non-randomized, open label study. A total of 12 participants will be enrolled into two groups of 6 each. Each participant will receive rAAV2 CB hRPE65 by subretinal injection in one eye on a single occasion. Participants in Group 1 will receive 450 µL at a dosage level of 4 x 10\^11 vg/mL containing a total of 1.8 x 10\^11 vg of rAAV2-CB-hRPE65. Participants in Group 2 will receive 450 µL at a dosage level of 1.33 x 10\^12 vg/mL containing a total of 6 x 10\^11 vg of rAAV2-CB-hRPE65. A retinal surgeon will administer the vector by subretinal injection. Enrollment will begin with Group 1 and will proceed to Group 2 after review of safety data by a Data and Safety Monitoring Committee. Safety will be monitored by evaluation of ocular and non ocular adverse events and hematology and clinical chemistry parameters. Efficacy will be measured by evaluation of visual fields, visual acuity and electroretinography.

Conditions

• Leber Congenital Amaurosis

Interventions

Type	Name	Description
BIOLOGICAL	rAAV2-CB-hRPE65	Recombinant adeno-associated virus vector expressing RPE65

Timeline

• Start date: 2009-06-17
• Primary completion: 2014-09-23
• Completion: 2017-09-22
• First posted: 2008-09-10
• Last updated: 2017-12-28
• Results posted: 2016-04-08

Locations

2 sites across 1 country: United States