@article {1347432, title = {Effect of Ciliary Neurotrophic Factor on Retinal Neurodegeneration in Patients with Macular Telangiectasia Type 2: A Randomized Clinical Trial}, journal = {Ophthalmology}, volume = {126}, number = {4}, year = {2019}, month = {2019 Apr}, pages = {540-549}, abstract = {PURPOSE: To test the effects of an encapsulated cell-based delivery of a neuroprotective agent, ciliary neurotrophic factor (CNTF), on progression of macular telangiectasia type 2, a neurodegenerative disease with no proven effective therapy. DESIGN: Randomized sham-controlled clinical trial. PARTICIPANTS: Ninety-nine study eyes of 67 eligible participants were enrolled. METHODS: Single-masked randomized clinical trial of 24 months{\textquoteright} duration conducted from May 2014 through April 2017 in 11 clinical centers of retinal specialists in the United States and Australia. Participants were randomized 1:1 to surgical implantation of intravitreal sustained delivery of human CNTF versus a sham procedure. MAIN OUTCOME MEASURES: The primary outcome was the difference in the area of neurodegeneration as measured in the area of the ellipsoid zone disruption (or photoreceptor loss) measured on spectral-domain (SD) OCT images at 24 months from baseline between the treated and untreated groups. Secondary outcomes included comparison of visual function changes between treatment groups. RESULTS: Among the 67 participants who were randomized (mean age, 62{\textpm}8.9 years; 41 women [61\%]; 58 white persons [86\%]), 65 (97\%) completed the study. Two participants (3 study eyes) died and 3 participants (4 eyes) were found ineligible. The eyes receiving sham treatment had 31\% greater progression of neurodegeneration than the CNTF-treated eyes. The difference in mean area of photoreceptor loss was 0.05{\textpm}0.03 mm (P\ = 0.04) at 24 months. Retinal sensitivity changes, measured using microperimetry, were correlated highly with the changes in the area of photoreceptor loss (r\ = 0.86; P \< 0.0001). The mean retinal sensitivity loss of the sham group was 45\% greater than that of the treated group (decrease, 15.81{\textpm}8.93 dB; P\ = 0.07). Reading speed deteriorated in the sham group (-13.9 words per minute) with no loss in the treated group (P\ = 0.02). Serious adverse ocular effects were found in 2 of 51 persons (4\%) in the sham group and 2 of 48 persons (4\%) in the treated group. CONCLUSIONS: In participants with macular telangiectasia type 2, a surgical implant that released CNTF into the vitreous cavity, compared with a sham procedure, slowed the progression of retinal degeneration. Further research is needed to assess longer-term clinical outcomes and safety.}, issn = {1549-4713}, doi = {10.1016/j.ophtha.2018.09.041}, author = {Chew, Emily Y and Clemons, Traci E and Jaffe, Glenn J and Johnson, Charles A and Farsiu, Sina and Lad, Eleonora M and Guymer, Robyn and Rosenfeld, Philip and Hubschman, Jean-Pierre and Constable, Ian and Wiley, Henry and Singerman, Lawrence J and Gillies, Mark and Comer, Grant and Blodi, Barbara and Eliott, Dean and Yan, Jiong and Bird, Alan and Friedlander, Martin and Macular Telangiectasia Type 2-Phase 2 CNTF Research Group} }