A randomized, placebo-controlled, single-blind, parallel design, multi-center, phase II clinical trial to evaluate the efficacy and safety of GV1001 in patients with benign prostatic hyperplasia (BPH)

Introduction & Objectives: The active compound of GV1001 is a single peptide consisting of 16 amino acids and the peptide corresponds to a fragment from the catalytic site of the enzyme telomerase. It has been recently documented that the weights of prostate decreased in a study on the animal study. We studied the efficacy and safety of GV1001. Materials & Methods: We divided 4 groups (Study group 1: GV1001 0.4 mg, 2‐week interval, Study group 2: GV1001 0.56 mg, 2‐week interval, Study group 3: GV1001 0.56 mg, 4‐week interval, Control group) in patients with BPH. After a run‐in period with 4 weekly (2 times at a 2‐week interval) administrations of placebo intradermally, eligible patients were randomly assigned to receive any of the 3 treatments at different GV1001 dosages and regimen or the placebo treatment in a 1:1:1:1. The 161 randomized patients were enrolled. Efficacy was evaluated at weeks 4, 8, 12, 13, and 16; safety was evaluated throughout the whole 16‐week period. International prostate symptoms score (IPSS), maximum urinary flow rate (Qmax), residual urine volume (RU), prostate volume, International Index of Erectile Function (IIEF), testosterone, dihydrotestosterone (DHT), prostate specific antigen (PSA) were evaluated. Results: At the Evaluation visit (week 13), there was statistically significant differences in the mean change of IPSS from baseline compared with the control group both in study group 1 and study group 2 (‐3.5vs‐7.2vs‐6.8: control vs group 1 vs group 2, p<0.05). Statistically significant differences in changes from baseline were also observed at weeks 8,12,13 and 16 in study group 1 and 2 compared to the control group. At Week 16, there was a statistically significant difference in the change of prostate volume from baseline compared with the control group in all of the 3 study groups (0.8vs‐4.6vs‐2.5vs‐4.2: control vs group 1 vs group 2 vs group 3, p<0.05). No statistically significant differences in Qmax, RU volume were observed in the 3 study groups compared to the control group, but an obvious trend of improvement in mean change from baseline (CFB) at weeks 4, 8, 12, 13, and 16 was observed. There were no statistically significant difference in mean CFB PSA, the IIEF, testosterone and DHT among groups at weeks 4 8,12,13, and 16 were observed. 88.8% received all planned doses of study treatment. There were no significant differences among the 4 groups for AE rate (all p‐values≥0.05). One patient each in study group 2 and the control group experienced severe TEAEs of clavicle fracture and dry eye, respectively, none of which were considered to be treatment‐related. There were no TEAEs which were definitely related or probably related to the study drug. Conclusions: At all treatment regimens tested, GV1001 was safe and effective and well‐tolerated in BPH patients.