A Phase 3, Open-Label, Single-Arm Trial of the Efficacy and Safety of Triptorelin 6-Month Formulation in Chinese Children with Central Precocious Puberty

IF 3.4 3区医学 Q2 MEDICINE, RESEARCH & EXPERIMENTAL

Advances in Therapy Pub Date : 2024-10-16 DOI:10.1007/s12325-024-02991-x

Xiao Yu, Xinran Cheng, Haiyan Wei, Xu Xu, Chunxiu Gong, Guimei Li, Hui Yao, Li Zhou, Yan Zhong, Yu Yang, Feihong Luo, Yining Zhang, Frank Huang, Xiaofeng Shi, Patrick Cabri, Xiaoping Luo

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引用次数: 0

Abstract

Introduction

This phase 3 study assessed the efficacy, safety, and pharmacokinetics of the 6-month prolonged release (PR) formulation in Chinese children with central precocious puberty (CPP).

Methods

In this open-label study (NCT05029622), Chinese children (girls < 9 years, boys < 10 years) received two doses of triptorelin pamoate 22.5 mg (day 1 and month 6). Primary endpoint was the proportion at month 6 with luteinizing hormone (LH) suppression (stimulated peak LH ≤ 5 IU/L after gonadotropin-releasing hormone stimulation). Secondary endpoints included safety assessments, hormone level changes, and clinical parameters from baseline.

Results

Overall, 66 children completed the study (93.9% girls; median age 8.0 [range 5–9] years). At month 6, all patients had LH suppression; this was maintained at month 12 in 98.5% of patients. Mean basal and peak LH and follicle-stimulating hormone levels were suppressed throughout follow-up. All patients at months 3 to 12 had sex hormone suppression to prepubertal levels. Stable or reduced breast development was seen for 98.4% and 93.5% of girls at month 6 and 12, respectively; all boys had regression or stable genital development until month 12. Compared with baseline (9.82 cm/year), mean growth velocity was 5.88 cm/year at month 6 and 5.17 cm/year at month 12. Mean bone age/chronological age ratio decreased from 1.27 at baseline to 1.23 and 1.21 at month 6 and 12, respectively. In girls, 64.5% showed decreased uterine length at month 6 and 12 versus baseline, while 75.0% of boys showed stable testicular volume versus baseline. Thirteen patients (19.7%) had 22 drug-related treatment emergent adverse events (TEAEs); no grade ≥ 3 TEAEs were reported.

Conclusion

The efficacy and safety profile of triptorelin 6-month PR in Chinese children with CPP was consistent with data previously reported in non-Chinese children with CPP, supporting this as a viable treatment option for Chinese children with CPP.

Trial Registration

Trial registration: ClinicalTrials.gov identifier, NCT05029622.

查看原文本刊更多论文

特普瑞林 6 个月制剂对中国中枢性性早熟儿童疗效和安全性的 3 期、开放标签、单臂试验。

简介：本3期研究评估了6个月长效缓释制剂（PR）对中国中枢性性早熟（CPP）儿童的疗效、安全性和药代动力学：本 3 期研究评估了中枢性性早熟（CPP）患儿服用 6 个月长效缓释制剂（PR）的疗效、安全性和药代动力学：在这项开放标签研究（NCT05029622）中，中国儿童（女孩结果：共有 66 名儿童完成了研究：共有 66 名儿童完成了研究（93.9% 为女孩；中位年龄为 8.0 [5-9]岁）。第 6 个月时，所有患者的 LH 均得到抑制；第 12 个月时，98.5% 的患者的 LH 均得到抑制。在整个随访期间，LH和卵泡刺激素的基础和峰值平均水平均受到抑制。在第 3 至 12 个月期间，所有患者的性激素水平都被抑制到了青春期前的水平。在第 6 个月和第 12 个月，分别有 98.4% 和 93.5% 的女孩乳房发育稳定或减弱；所有男孩的生殖器发育在第 12 个月前都有所减弱或稳定。与基线（9.82 厘米/年）相比，第 6 个月和第 12 个月的平均生长速度分别为 5.88 厘米/年和 5.17 厘米/年。平均骨龄/同步年龄比从基线时的1.27降至第6个月时的1.23和第12个月时的1.21。与基线相比，64.5%的女孩在第 6 个月和第 12 个月的子宫长度有所减少，而 75.0%的男孩的睾丸体积与基线相比保持稳定。13名患者（19.7%）发生了22起与药物相关的治疗突发不良事件（TEAEs）；没有≥3级TEAEs的报告：结论：曲普瑞林6个月PR在中国儿童CPP患者中的疗效和安全性与之前在非中国儿童CPP患者中报道的数据一致，支持将其作为中国儿童CPP患者的可行治疗方案：试验注册ClinicalTrials.gov identifier，NCT05029622。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Advances in Therapy 医学-药学

CiteScore

7.20

自引率

2.60%

发文量

353

审稿时长

6-12 weeks

期刊介绍： Advances in Therapy is an international, peer reviewed, rapid-publication (peer review in 2 weeks, published 3–4 weeks from acceptance) journal dedicated to the publication of high-quality clinical (all phases), observational, real-world, and health outcomes research around the discovery, development, and use of therapeutics and interventions (including devices) across all therapeutic areas. Studies relating to diagnostics and diagnosis, pharmacoeconomics, public health, epidemiology, quality of life, and patient care, management, and education are also encouraged. The journal is of interest to a broad audience of healthcare professionals and publishes original research, reviews, communications and letters. The journal is read by a global audience and receives submissions from all over the world. Advances in Therapy will consider all scientifically sound research be it positive, confirmatory or negative data. Submissions are welcomed whether they relate to an international and/or a country-specific audience, something that is crucially important when researchers are trying to target more specific patient populations. This inclusive approach allows the journal to assist in the dissemination of all scientifically and ethically sound research.