Overview

This trial is active, not recruiting.

Condition leber congenital amaurosis
Treatment raav2-cb-hrpe65
Phase phase 1/phase 2
Sponsor Applied Genetic Technologies Corp
Collaborator Oregon Health and Science University
Start date December 2008
End date September 2014
Trial size 12 participants
Trial identifier NCT00749957, AGTC-RPE65-002, R01 FD003694

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

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation non-randomized
Endpoint classification safety/efficacy study
Intervention model parallel assignment
Masking open label
Primary purpose treatment
Arm
(Experimental)
Subjects at least 6 y/o treated with a lower dose of the vector by subretinal injection
raav2-cb-hrpe65
Recombinant adeno-associated virus vector expressing RPE65
(Experimental)
Subjects at least 6 y/o treated with a higher dose of the vector by subretinal injection
raav2-cb-hrpe65
Recombinant adeno-associated virus vector expressing RPE65

Primary Outcomes

Measure
Number and proportion of participants experiencing ocular or non-ocular adverse events
time frame: 2 years

Secondary Outcomes

Measure
Changes in visual fields
time frame: 2 years
Changes in best corrected visual acuity
time frame: 2 years

Eligibility Criteria

Male or female participants at least 6 years old.

Inclusion Criteria: - Retinal disease consistent with a diagnosis of Leber congenital amaurosis and documented mutations in the RPE65 gene (including null mutations and mutations that code for abnormal RPE65 protein); - At least 6 years of age; - Good general health without significant physical examination findings or clinically significant abnormal laboratory results; - Able to perform tests of visual and retinal function; - Visual acuity not better than 20/60 and not worse than hand motion in both the treated eye and the fellow eye; - Visible photoreceptor (outer nuclear) layer on a standard optical coherence tomography (OCT) scan; - Acceptable hematology, clinical chemistry and urine laboratory parameters; - For females of childbearing potential, a negative pregnancy test at screening and at baseline, and agreement to use effective contraception for 12 months after administration of rAAV2-CB-hRPE65, for sexual activity that could lead to pregnancy; - For males of reproductive potential, agreement to use effective contraception for 12 months after administration of rAAV2-CB-hRPE65, for sexual activity that could lead to pregnancy Exclusion Criteria: - Pre-existing eye conditions that would preclude the planned surgery or interfere with interpretation of study endpoints or complications of surgery (e.g. glaucoma, corneal or lenticular opacities, or history or retinal detachment); - Presence of epiretinal membrane on OCT; - History of immunodeficiency or other medical conditions that might increase the risk of rAAV2-CB-hRPE65 administration; - Use of anticoagulants or anti-platelet agents within 7 days prior to study agent administration; - History of allergy or sensitivity to medications planned for use in the peri-operative period; - For females of childbearing potential, a positive pregnancy test at screening or baseline (within 2 days before rAAV2-CB-hRPE65 administration); - Females who are breast feeding; - Use of any investigational agent, or systemic corticosteroids or other immunosuppressive drug(s), within 3 months prior to enrollment; - Prior receipt of any AAV gene therapy product; - Any condition which leads the investigator to believe that the participant cannot comply with the protocol requirements or that may place the participant at an unacceptable risk for participation.

Additional Information

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
Principal investigator J Timothy Stout, MD, PhD, MBA
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.
Trial information was received from ClinicalTrials.gov and was last updated in June 2015.
Information provided to ClinicalTrials.gov by Applied Genetic Technologies Corp.