The Developing Regorafenib Eye drops for neovascular Age‐related Macular degeneration (DREAM) study: an open‐label phase II trial

摘要: Aims This programme investigated topical regorafenib, a multikinase inhibitor, in patients with neovascular age-related macular degeneration (nAMD). Methods Topical regorafenib was an open-label, phase IIa/b study which choroidal neovascularization (CNV) secondary to nAMD received (25 μl, 30 mg ml-1 ) three times day for 12 weeks. The primary endpoint of the II/a/b mean change best-corrected visual acuity (BCVA) from baseline weeks 4 and 12. Results In (N = 51), changes BCVA were +1.2 [90% confidence interval (CI) -0.61, 2.97] -2.4 (90% CI -4.18, -0.54) letters at 12, respectively. Ocular treatment-emergent adverse events (TEAEs) (study eye) reported 21 by week There one serious ocular TEAE (visual reduced) that not drug related. Twenty required rescue (intravitreal ranibizumab). Conclusions terminated after IIa ended because efficacy lower than current treatments. According elaborate post hoc analyses, most likely reason insufficient exposure target compartment (back eye).