Department of General Medicine, Okayama University Graduate School of Medicine, Dentistry and Pharmaceutical Sciences, Okayama, Japan.
Division of Diabetes and Endocrinology, Department of Internal Medicine, Kobe University Graduate School of Medicine, Kobe, Japan.
Clin Endocrinol (Oxf). 2020 Nov;93(5):620-628. doi: 10.1111/cen.14273. Epub 2020 Aug 14.
Somapacitan is a long-acting, reversible albumin-binding growth hormone (GH) derivative in development. This study aimed to evaluate the safety and efficacy of once-weekly somapacitan versus daily GH over 52 weeks in Japanese patients with adult growth hormone deficiency (AGHD).
Phase 3, multicentre, randomized, parallel-group, open-label, active-controlled trial (NCT03075644).
Previously GH-treated Japanese patients with AGHD were randomized 3:1 to somapacitan (n = 46) or daily GH (n = 16) for 20 weeks' dose titration and 32 weeks' fixed-dose treatment.
Primary endpoint was the incidence of adverse events (AEs). Secondary endpoints included change from baseline to week 52 in visceral, subcutaneous and total adipose tissue (VAT, SAT and TAT).
Mean (SD) prescribed doses after titration were 1.780 (1.058) mg/week for somapacitan and 0.197 (0.083) mg/day for daily GH. Rate of AEs per 100 patient-years was similar between arms (somapacitan, 312.7; daily GH, 309.8). Four AEs in the somapacitan arm were serious; none were considered treatment-related. Mean insulin-like growth factor-I standard deviation score (IGF-I SDS) was maintained from baseline in both arms. No significant differences were observed between arms for change from baseline to week 52 in VAT, SAT or TAT (estimated difference, somapacitan - daily GH [95% CI]: -1.74 [-18.13; 14.66], -11.53 [-35.54; 12.48] and - 12.85 [-47.31; 21.62] cm , respectively).
Treatment in both groups was well tolerated, with no unexpected safety findings. Impact on adipose tissue was similar to somapacitan and daily GH in patients with AGHD. A short visual summary of our work is available at https://bit.ly/3946YNF.
Somapacitan 是一种长效、可逆的白蛋白结合型生长激素（GH）衍生物，目前正在研发中。本研究旨在评估每周一次的 Somapacitan 与每日 GH 相比，在 52 周内治疗日本成年生长激素缺乏症（AGHD）患者的安全性和疗效。
这是一项为期 3 期、多中心、随机、平行组、开放标签、活性对照试验（NCT03075644）。
先前接受过 GH 治疗的日本 AGHD 患者按 3:1 的比例随机分为 Somapacitan（n=46）或每日 GH（n=16）组，分别进行 20 周的剂量滴定和 32 周的固定剂量治疗。
主要终点是不良事件（AE）的发生率。次要终点包括从基线到第 52 周时内脏、皮下和总脂肪组织（VAT、SAT 和 TAT）的变化。
滴定后的平均（SD）处方剂量Somapacitan 为 1.780（1.058）mg/周，每日 GH 为 0.197（0.083）mg/天。每个 100 患者年的 AE 发生率在两组间相似（Somapacitan，312.7；每日 GH，309.8）。Somapacitan 组有 4 例 AE 为严重事件，但均与治疗无关。两组 IGF-I 标准差评分（IGF-I SDS）均从基线维持。从基线到第 52 周，两组间 VAT、SAT 或 TAT 的变化无显著差异（估计差值，Somapacitan-每日 GH[95%CI]：-1.74[-18.13；14.66]、-11.53[-35.54；12.48]和-12.85[-47.31；21.62]cm）。
两组治疗均耐受良好，无意外安全性发现。对脂肪组织的影响与 Somapacitan 和每日 GH 相似，在 AGHD 患者中。我们工作的简短视觉摘要可在 https://bit.ly/3946YNF 查看。
