Boyer David S, Gonzalez Victor H, Kunimoto Derek Y, Maturi Raj K, Roe Richard H, Singer Michael A, Xavier Samantha, Kornfield Julie A, Kuppermann Baruch D, Quiroz-Mercado Hugo, Aubel Janine, Karageozian Hampar L, Park John Y, Karageozian Vicken H, Karageozian Lisa, Sarayba Melvin A, Kaiser Peter K
Ophthalmic Surg Lasers Imaging Retina. 2021 Jun;52(6):327-335. doi: 10.3928/23258160-20210528-05. Epub 2021 Jun 1.
To evaluate the safety and efficacy of 1.0 mg risuteganib in subjects with nonexudative age-related macular degeneration (AMD).
This was a phase 2a, prospective, double-masked, sham-controlled study. Eyes with nonexudative (dry) AMD and Early Treatment Diabetic Retinopathy Study (ETDRS) best-corrected visual acuity (BCVA) between 20/40 and 20/200 were included. Subjects were randomized to intravitreal 1.0 mg risuteganib or sham injection. At Week 16, subjects in the risuteganib group received a second 1.0-mg dose and the sham group crossed over to receive a dose of 1.0 mg risuteganib and were evaluated at Week 28. The primary endpoint was proportion of subjects with 8 letters ETDRS or more BCVA gain from baseline to Week 28 in the risuteganib group versus baseline to Week 12 for the sham group. BCVA was tested and subjects were observed for adverse events (AEs) every 4 weeks until completion of the study at 32 weeks.
Forty-five subjects (risuteganib, n = 29; sham, n = 16) were enrolled in the study, of whom 39 (risuteganib, n = 25; sham, n = 14) completed the study and were included in the per protocol efficacy analysis. At baseline, mean age was 78.8 and 75.9 years and mean BCVA was 67.1 and 64.4 letters in the sham and risuteganib groups, respectively. The primary endpoint was met by 48% of the risuteganib group at Week 28 and 7% of the sham group at Week 12 ( = .013). Of the risuteganib subjects, 20% gained 15 letters or more at Week 28, whereas no patients in the sham group at Week 12 achieved this visual acuity gain. The only ocular treatment-related treatment-emergent AE was vitreous floaters, which spontaneously recovered without sequelae. No drug-related serious AE was reported.
Risuteganib demonstrated significant BCVA improvement in patients with non-exudative AMD. No drug-related AEs were seen during a 32-week observation period. .
评估1.0毫克瑞舒替尼对非渗出性年龄相关性黄斑变性（AMD）患者的安全性和有效性。
这是一项2a期前瞻性双盲、假手术对照研究。纳入患有非渗出性（干性）AMD且早期糖尿病性视网膜病变研究（ETDRS）最佳矫正视力（BCVA）在20/40至20/200之间的患者。受试者被随机分为玻璃体内注射1.0毫克瑞舒替尼组或假注射组。在第16周时，瑞舒替尼组的受试者接受第二次1.0毫克剂量注射，假手术组交叉接受1.0毫克瑞舒替尼注射，并在第28周进行评估。主要终点是瑞舒替尼组从基线到第28周BCVA提高8个或更多ETDRS字母的受试者比例，与假手术组从基线到第12周的情况进行对比。每4周测试一次BCVA，并观察受试者的不良事件（AE），直至在32周完成研究。
45名受试者（瑞舒替尼组n = 29；假手术组n = 16）纳入研究，其中39名（瑞舒替尼组n = 25；假手术组n = 14）完成研究并纳入符合方案的疗效分析。基线时，假手术组和瑞舒替尼组的平均年龄分别为78.8岁和75.9岁，平均BCVA分别为67.1和64.4个字母。瑞舒替尼组在第28周时48%的受试者达到主要终点，假手术组在第12周时为7%（P = 0.013）。在瑞舒替尼组受试者中，20%在第28周时视力提高了15个或更多字母，而假手术组在第12周时没有患者达到这一视力提高水平。唯一与眼部治疗相关的治疗中出现的AE是玻璃体漂浮物，可自发恢复且无后遗症。未报告与药物相关的严重AE。
瑞舒替尼在非渗出性AMD患者中显示出显著的BCVA改善。在32周的观察期内未观察到与药物相关的AE。
