Foundation News » Other Retinal Diseases
New LCA Gene Therapy Clinical Trial Recruiting Participants
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Applied Genetic Technologies Corporation (AGTC) has launched a Phase I/II clinical trial of gene therapy to treat Leber congenital amaurosis, extending the work being done for similar treatments in three other Phase I clinical trials taking place at The Children’s Hospital of Philadelphia, Universities of Pennsylvania and Florida, and Moorfield’s Eye Hospital in the U.K. In this trial, AGTC will be evaluating higher doses of the treatment in younger participants with the hope of restoring even more vision than has been restored in the other three studies. AGTC is recruiting a total of 12 people for the study, which is being conducted at two clinical trial centers: Casey Eye Institute, Oregon Health & Science University and University of Massachusetts Medical School. Participants should be in good general health and at least eight years of age, and have been diagnosed with LCA caused by variations (mutations) in the gene RPE65. If individuals have been diagnosed with LCA, but do not know if a variation in RPE65 is causing the condition, they may contact the investigators listed at the end of the article to discuss a possible genetic test. AGTC is also planning to launch clinical trials of gene therapy to treat achromatopsia (day blindess) and retinoschisis. The Foundation Fighting Blindness is currently funding preclinical studies of these treatments. AGTC’s LCA Phase I/II Clinical Trial Sites: Casey Eye Institute, Oregon Health & Science University, Portland, Oregon Contact Principal Investigator J. Timothy Stout, M.D., Ph.D., (503) 494-2435 or This e-mail address is being protected from spambots. You need JavaScript enabled to view it . University of Massachusetts Medical School, Worcester, Massachusetts Contact Principal Investigator Shalesh Kaushal, M.D., Ph.D., at This e-mail address is being protected from spambots. You need JavaScript enabled to view it or (508) 334-0687. Click here for more information, including inclusion and exclusion criteria, for the clinical study. Click here to learn more about the Foundation’s collaboration with AGTC. |
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