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

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: 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 Amaurosi

Brief 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 Summary:

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.


Sponsor: Applied Genetic Technologies Corp

Current Primary Outcome: Number of Participants Experiencing Ocular or Non-ocular Adverse Events [ Time Frame: 2 years ]

Original Primary Outcome: Number and proportion of participants experiencing ocular or non-ocular adverse events

Current Secondary Outcome:

  • Participants With Changes in Visual Fields [ Time Frame: 2 years ]
    Improvement in the central 30 degree visual field, measured by static perimetry, at one or more time points after treatment, that was greater than the limit of agreement for baseline values .
  • Participants With Changes in Best Corrected Visual Acuity [ Time Frame: 2 years ]
    Increase in BCVA of 7 or more letters at Year 2 visit compared to average baseline value


Original Secondary Outcome:

  • Changes in visual fields
  • Changes in best corrected visual acuity


Information By: Applied Genetic Technologies Corp

Dates:
Date Received: September 8, 2008
Date Started: December 2008
Date Completion: December 2027
Last Updated: April 7, 2016
Last Verified: April 2016