Original articleRandomized Trial of Ciliary Neurotrophic Factor Delivered by Encapsulated Cell Intraocular Implants for Retinitis Pigmentosa
Section snippets
Study Design
A total of 65 and 68 patients were enrolled at 13 sites in the United States for the CNTF3 and CNTF4 studies, respectively (Table 1). Approvals were received from the National Institutes of Health Recombinant DNA Advisory Committee, from the Food and Drug Administration (FDA), and from the Institutional Review Board and Institutional Biosafety Committee at each site prior to enrollment. The Institutional Review Boards responsible for these studies are listed in the acknowledgment at the end of
Study Patients
Between January 8, 2007 and October 31, 2007, 65 patients and 68 patients were enrolled into the CNTF3 and CNTF4 studies, respectively, and were randomly assigned to study treatment. Groups were balanced for demographic and baseline ocular characteristics (Table 1). All patients completed the 12-month primary endpoint follow-up and no patients dropped out of the study.
Safety Profile
Cumulative adverse events for the 12-month study period (CNTF3 and CNTF4) are summarized in Table 2. The most frequent adverse
Discussion
CNTF3 and CNTF4 studies were prospective, masked, randomized studies to evaluate the safety profile of the encapsulated cell–ciliary neurotrophic factor implant, to determine the effect of ciliary neurotrophic factor on retinal structure and visual function, and to explore the dose and primary endpoint for future studies in patients with RP. Neither study demonstrated a significant improvement in their respective endpoints: BCVA at 12 months for CNTF3 and visual field sensitivity at 12 months
Dr David G. Birch, PhD, takes a multidisciplinary approach to retinal degenerative diseases, utilizing electrophysiology, psychophysics and retinal imaging. He has served as principal investigator on several single site and multi-center clinical trials in retinal degenerative diseases, received an Achievement Award from the American Academy of Ophthalmology, is a Fellow of the Association for Research in Vision and Ophthalmology and is the author of over 250 scientific papers.
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2022, Advanced Drug Delivery ReviewsCitation Excerpt :The cell-based system was implanted in the vitreous following scleral incision and was anchored in place with a single stitch [80,81]. The encapsulated cells were transfected with ciliary neurotrophic factor (CNTF) gene, and the implant was able to sustain the production and release of CNTF for at least two years [82]. Nevertheless, development was terminated as it failed to demonstrate long-term benefits (clinical trials: NCT00447980, NCT00447993) [82].
Dr David G. Birch, PhD, takes a multidisciplinary approach to retinal degenerative diseases, utilizing electrophysiology, psychophysics and retinal imaging. He has served as principal investigator on several single site and multi-center clinical trials in retinal degenerative diseases, received an Achievement Award from the American Academy of Ophthalmology, is a Fellow of the Association for Research in Vision and Ophthalmology and is the author of over 250 scientific papers.