Leber Congenital Amaurosis Clinical Trial
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 Amaurosis Type 2
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
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.
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