Humphrey Perimetry 10-2 (JapicCTI-090748) assessment of Retinitis Pigmentosa Patients in Phase 2 Study of UF-021

Abstract

Purpose: : To test the effect of UF-021 in improving the central vision in retinitis pigmentosa (RP) patients.

Methods: : The safety and efficacy of UF-021 were determined by a randomized, double-masked, parallel 3-arm comparative study of UF-021 in a 0.15% ophthalmic solution. One hundred and nine mid- to late-stage RP patients with visual acuity of ≥0.5 at 6 ophthalmological centers were given placebo, or one, or two drops of UF-021 twice daily for 24 weeks. The primary endpoint was a change in the microperimetry (MP-1) sensitivity from the baseline in the central 2 degrees. The secondary endpoints included change in retinal sensitivity from baseline within the central 2 degrees, and improvements of the MD by Humphrey perimetry 10-2 and VFQ-25. The background luminance of Humphrey perimetry was 31.5 asb (10.0 cd/m²) which provided cone-dominant retinal sensitivity thresholds.

Results: : Simple linear regression analysis showed that significant improvements of the Humphrey perimetry MDs from the baseline in the central 2 degrees in a dose-dependent way (P=0.02). The central vision improved by >3 dB in the high dose group which was significantly better (P=0.04) than in the placebo group. Patients with improvement of retinal sensitivity >3 dB had significant improvement of visual acuity (P<0.05) and contrast sensitivity (P<0.01). The VFQ-25 within groups was significantly (P=0.048) improved from the baseline in the high-dose patients only and in the dose-responsiveness (P=0.12) by simple linear regression analysis of the 3 arms. The change of VFQ-25 subscale value for "Social Life Functions Due to Vision" showed significant (P=0.001) dose-responsiveness by simple linear regression analysis across arms. The change of VFQ-25 score was significantly (P<0.001) better in the high-dose vs placebo patients.

Conclusions: : UF-021 can improve the central 2°vision of mid- to late-stage RP patients and was safe. These data support further clinical testing of UF-021.

Clinical Trial: : http://www.clinicaltrials.jp/user/ctiMain_e.jsp, JapicCTI-090748

Keywords: drug toxicity/drug effects • neuroprotection • retina