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Abstract：
To evaluate the safety and efficacy of daily somatropin (Jintropin®), a recombinant human growth hormone, in prepubertal children with ISS in China.
This study was a multicenter, randomized, controlled, open-label, phase 3 study. All subjects were randomized 3:1 to daily somatropin 0.05 mg/kg/day or no treatment for 52 weeks. A total of 481 subjects with a mean baseline age of 5.8 years were enrolled in the study. The primary endpoint was change in (△) height standard deviation score (HT-SDS) for chronological age (CA). Secondary endpoints included △height from baseline; △bone age (BA)/CA; △height velocity (HV) and △insulin-like growth factor 1 (IGF-1 SDS).
△HT-SDS at week 52 was 1.04 ± 0.31 in the treatment group and 0.20 ± 0.33 in the control group (P < 0.001). At week 52, statistical significance was observed in the treatment group compared with control for △height (10.19 ± 1.47 cm vs. 5.85 ± 1.80 cm; P < 0.001), △BA/CA (0.04 ± 0.09 vs. 0.004 ± 0.01; P < 0.001), △HV (5.17 ± 3.70 cm/year vs. 0.75 ± 4.34 cm/year; P < 0.001), and △IGF-1 SDS (2.31 ± 1.20 vs. 0.22 ± 0.98; P < 0.001). The frequencies of treatment-emergent adverse events (TEAEs) were similar for the treatment and the control groups (89.8% vs. 82.4%); most TEAEs were mild to moderate in severity and 23 AEs were considered study-drug related.
Daily subcutaneous administration of somatropin at 0.05 mg/kg/day for 52 weeks demonstrated improvement in growth outcomes and was well tolerated with a favorable safety profile.
ClinicalTrials.gov (identifier: NCT03635580). URL: https://clinicaltrials.gov/ct2/show/NCT03635580.
This study was a multicenter, randomized, controlled, open-label, phase 3 study. All subjects were randomized 3:1 to daily somatropin 0.05 mg/kg/day or no treatment for 52 weeks. A total of 481 subjects with a mean baseline age of 5.8 years were enrolled in the study. The primary endpoint was change in (△) height standard deviation score (HT-SDS) for chronological age (CA). Secondary endpoints included △height from baseline; △bone age (BA)/CA; △height velocity (HV) and △insulin-like growth factor 1 (IGF-1 SDS).
△HT-SDS at week 52 was 1.04 ± 0.31 in the treatment group and 0.20 ± 0.33 in the control group (P < 0.001). At week 52, statistical significance was observed in the treatment group compared with control for △height (10.19 ± 1.47 cm vs. 5.85 ± 1.80 cm; P < 0.001), △BA/CA (0.04 ± 0.09 vs. 0.004 ± 0.01; P < 0.001), △HV (5.17 ± 3.70 cm/year vs. 0.75 ± 4.34 cm/year; P < 0.001), and △IGF-1 SDS (2.31 ± 1.20 vs. 0.22 ± 0.98; P < 0.001). The frequencies of treatment-emergent adverse events (TEAEs) were similar for the treatment and the control groups (89.8% vs. 82.4%); most TEAEs were mild to moderate in severity and 23 AEs were considered study-drug related.
Daily subcutaneous administration of somatropin at 0.05 mg/kg/day for 52 weeks demonstrated improvement in growth outcomes and was well tolerated with a favorable safety profile.
ClinicalTrials.gov (identifier: NCT03635580). URL: https://clinicaltrials.gov/ct2/show/NCT03635580.
摘要：
为了评估每日生长激素（Jintropin®）的安全性和有效性，重组人生长激素,在中国患有ISS的青春期前儿童中。
这项研究是多中心的,随机化，控制,开放标签，第三阶段研究。所有受试者均以3：1随机分配至每日生长激素0.05mg/kg/天或不治疗52周。总共481名平均基线年龄为5.8岁的受试者被纳入研究。主要终点是实际年龄（CA）的（△）身高标准差评分（HT-SDS）的变化。次要终点包括从基线开始的△身高；△骨龄（BA）/CA；△身高速度（HV）和△胰岛素样生长因子1（IGF-1SDS）。
治疗组52周时△HT-SDS为1.04±0.31,对照组为0.20±0.33(P<0.001)。在第52周，治疗组与对照组相比，△身高有统计学意义（10.19±1.47cmvs.5.85±1.80cm;P<0.001),△BA/CA(0.04±0.09vs.0.004±0.01；P<0.001），△HV(5.17±3.70cm/年vs.0.75±4.34厘米/年；P<0.001），和△IGF-1SDS(2.31±1.20vs.0.22±0.98；P<0.001）。治疗组和对照组的治疗引起的不良事件（TEAE）的频率相似（89.8％vs.82.4%）；大多数TEAE的严重程度为轻度至中度，23个AE被认为与研究药物相关。
每天皮下施用0.05mg/kg/天的生长激素持续52周证明了生长结果的改善，并且耐受性良好，具有良好的安全性。
ClinicalTrials.gov（标识符：NCT03635580）。URL：https://clinicaltrials.gov/ct2/show/NCT03635580。
这项研究是多中心的,随机化，控制,开放标签，第三阶段研究。所有受试者均以3：1随机分配至每日生长激素0.05mg/kg/天或不治疗52周。总共481名平均基线年龄为5.8岁的受试者被纳入研究。主要终点是实际年龄（CA）的（△）身高标准差评分（HT-SDS）的变化。次要终点包括从基线开始的△身高；△骨龄（BA）/CA；△身高速度（HV）和△胰岛素样生长因子1（IGF-1SDS）。
治疗组52周时△HT-SDS为1.04±0.31,对照组为0.20±0.33(P<0.001)。在第52周，治疗组与对照组相比，△身高有统计学意义（10.19±1.47cmvs.5.85±1.80cm;P<0.001),△BA/CA(0.04±0.09vs.0.004±0.01；P<0.001），△HV(5.17±3.70cm/年vs.0.75±4.34厘米/年；P<0.001），和△IGF-1SDS(2.31±1.20vs.0.22±0.98；P<0.001）。治疗组和对照组的治疗引起的不良事件（TEAE）的频率相似（89.8％vs.82.4%）；大多数TEAE的严重程度为轻度至中度，23个AE被认为与研究药物相关。
每天皮下施用0.05mg/kg/天的生长激素持续52周证明了生长结果的改善，并且耐受性良好，具有良好的安全性。
ClinicalTrials.gov（标识符：NCT03635580）。URL：https://clinicaltrials.gov/ct2/show/NCT03635580。
