Date: 2016-04-22
Type of information: Publication of results in a medical journal
phase: 1-2
Announcement: publication of results in Ophthalmology
Company: AGTC (USA - FL)
Product: rAAV2-CB-hRPE65
Action
mechanism: gene therapy. rAAV2-CB-hRPE65 is a nonreplicating recombinant AAV vector that contains cDNA encoding the human retinal pigment epithelium (RPE)-specific protein hRPE65.
Disease: Leber congenital amaurosis (LCA), severe early-childhood-onset retinal degeneration (SECORD)
Therapeutic area: Rare diseases - Genetic diseases - Ophtalmological diseases
Country: USA
Trial
details: 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. (NCT00749957)
Latest
news: * On April 22, 2016, Applied Genetic Technologies Corporation, a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, announced data evaluating an experimental recombinant AAV vector gene delivery in patients with Leber congenital amaurosis (LCA) or severe early-childhood-onset retinal degeneration (SECORD), two related retinal diseases caused by mutations in the RPE65 gene that cause severe loss of vision in infancy. Results from the study, entitled "Results at 2 Years after Gene Therapy for RPE65-deficient Leber Congenital Amaurosis and Severe Early-Childhood Onset Retinal Dystrophy," were published in the peer-reviewed journal Ophthalmology. The study was conducted at Oregon Health and Science University (OHSU) Casey Eye Institute (CEI) and University of Massachusetts. The study enrolled eight adults and four children with LCA or SECORD who received a subretinal injection of a recombinant AAV expressing RPE65 (rAAV2-CB-hRPE65) at one of two dose levels in the poorer seeing eye. All subjects then underwent serial assessments of visual function and adverse events during two-year clinical follow-up to evaluate safety and efficacy parameters. All subjects tolerated the surgery and study agent administration with no treatment-related serious adverse events reported. In the treated eye, best-corrected visual acuity (BCVA) increased in five subjects, static perimetry hill of vision measurements for the central 30° of the visual field increased in six subjects, total visual field hill of vision measurements increased in five subjects, and kinetic visual field area improved in three subjects. One study participant had a decrease in BCVA and two subjects had a decrease in kinetic visual field area. Common adverse events associated with the injection included subconjunctival hemorrhage in eight subjects and ocular hyperemia (redness) in five subjects. The investigators concluded that the treatment with rAAV2-CB-hRPE65 was not associated with serious adverse events and improvement in one or more measures of visual function was observed in 9 of 12 (75 percent) subjects.
