From the Foundation Fighting Blindness: new RPE65 trial recruiting!

Applied Genetic Technologies, the Oregon Health & Science University and the Foundation Fighting Blindness Announce Open Recruitment for a Phase 1/Phase 2 Research Clinical Trial

  

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

 

Applied Genetic Technologies Corporation is the gene therapy development company providing the study drug and is the sponsor of this research trial.  The Casey Eye Institute at the Oregon Health & Science University, Portland, OR (eIRB# 4892) is open for enrollment of patients in this clinical trial. 

 

The RPE 65 gene makes the RPE65 protein that is needed for the retina to work normally.  The study drug contains a normal copy of the RPE65 gene and also contains part of a virus called AAV.  The AAV virus does not cause illness in humans, and it is used in the study drug to help the normal RPE65 gene get into cells of the retina after an eye surgeon injects the study drug beneath the retina in the back of one eye. 

The purpose of this research study is to test whether the study drug, called rAAV2-CB-hRPE65, is safe and to see if it can improve vision in individuals who have a mutation in their RPE65 gene.  Right now, rAAV2-CB-hRPE65 is not approved by the United States Food and Drug Administration (FDA) to treat people with changes in their RPE65 genes because not enough is known about the drug.  The FDA will allow testing of the drug only in a research study such as this one. 

 

Potential participants will be asked to come to the clinical site in Portland, Oregon.  If their home is not close to the site, they will have to stay at a nearby hotel.  During the first visit, there will be examinations and tests done to see if the individual is eligible to join the study.  At the second visit, there will be more tests and if these results are acceptable, the participant will receive the study drug two days later.  An eye surgeon will inject the study drug beneath the retina in one eye only. 

 After receiving the study drug, participants will be examined and have tests performed 1, 7 and 14 days later and then again at 1, 2, 3, 6, 9, 12, 18, and 24 months.  Patients are expected to travel to the site where the injection was done for each of these visits, since special equipment is used to measure their visual function.  After the 24-month visit, they will be contacted once a year for 15 years and asked to answer questions.  They will also be asked to have a follow-up visit once a year from years 3 to 15 after treatment.  These follow-up visits can be performed by their local ophthalmologist.

 Participants are not paid for being in the study.  However, participants will be reimbursed for reasonable costs related to the travel to attend the initial evaluation visit and, if accepted into the study, subsequent study visits. 

 

Some of the basic criteria for inclusion or exclusion in this trial are as follows:

 

Inclusion Criteria

 

Retina disease consistent with a diagnosis of Leber Congenital Amaurosis caused by a documented mutation in the RPE65 gene;

At least 6 years of age;

Good general health;

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

 

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 of retinal detachment)

History of immunodeficiency or other medical conditions that might increase the risk of study drug (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;

Use of any investigational agent or systemic corticosteroids or other immunosuppressive drug(s) within 3 months prior to enrollment. 

 

Additional and detailed inclusion and exclusion criteria can be reviewed at www.clinicaltrials.gov.  Study identifier is NCT00749957.  Questions regarding criteria for possible participation can also be directed to the investigators at Casey Eye Institute. 

 

Casey Eye Institute, Oregon Health & Science University   

Portland, Oregon, United States, 97239

Contact: Principal Investigator - J Timothy Stout, MD, PhD, MBA - 503-494-2435 - stoutt@ohsu.edu

Contact: Peter Francis, MD, PhD - (503) 418-1627 - francisp@ohsu.edu

Contact: Maureen Toomey, BS - (503) 494-3795 - toomeym@ohsu.edu

 

Applied Genetic Technologies Corporation – www.agtc.com

 

Foundation Fighting Blindness – www.FightBlindness.org