A 12-month, Open-Label, Single-Arm, Phase 3 Trial of the Efficacy and Safety of Triptorelin 6-Month Formulation in Chinese Children with Central Precocious Puberty

Xiao Yu; Xinran Cheng; 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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ESPE Abstracts (2024) 98 P1-80

ESPE2024 Poster Category 1 Pituitary, Neuroendocrinology and Puberty 1 (9 abstracts)

A 12-month, Open-Label, Single-Arm, Phase 3 Trial of the Efficacy and Safety of Triptorelin 6-Month Formulation in Chinese Children with Central Precocious Puberty

Xiao Yu ¹ , Xinran Cheng ² , Xu Xu ³ , Chunxiu Gong ⁴ , Guimei Li ⁵ , Hui Yao ⁶ , Li Zhou ⁷ , Yan Zhong ⁸ , Yu Yang ⁹ , Feihong Luo ¹⁰ , Yining Zhang ¹¹ , Frank Huang ¹² , Xiaofeng Shi ¹² , Patrick Cabri ¹³ & Xiaoping Luo ¹

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¹Tongji Hospital, Wuhan, China. ²Chengdu Women's and Children's Central Hospital, Chengdu, China. ³Wuxi Children's Hospital, Wuxi, China. ⁴Beijing Children's Hospital, Beijing, China. ⁵Shandong Provincial Hospital, Jinan, China. ⁶Wuhan Children's Hospital, Wuhan, China. ⁷Pingxiang Maternity and Childcare, Pingxiang, China. ⁸Hunan Children's Hospital, Changsha, China. ⁹Children's Hospital of Nanchang University and Jiangxi Provincial Children's Hospital, Nanchang, China. ¹⁰Children's Hospital of Fudan University, Shanghai, China. ¹¹The First Hospital of Jilin University, Changchun, China. ¹²Ipsen (Shanghai) Innovation Pharmaceutical Co. Ltd, Shanghai, China. ¹³Ipsen Boulogne-Billancourt, Boulogne-Billancourt, France

Objectives: The gonadotropin-releasing hormone (GnRH) analogue triptorelin is used to treat central precocious puberty (CPP) in 1-, 3- and 6-month prolonged-release (PR) formulations. Currently, only the 1- and 3-month formulations are approved for CPP in China. However, the 6-month formulation is available for CPP treatment in numerous countries; this study assessed the efficacy and safety of this formulation in Chinese children with CPP.

Methods: In this open-label study (NCT05029622), participants received two doses (Day 1 and Month 6) of triptorelin pamoate 22.5 mg. The primary endpoint was the proportion of patients with luteinising hormone (LH) suppression (stimulated peak LH ≤5 IU/L after GnRH stimulation) at Month 6. Secondary endpoints included changes from baseline in hormone levels, pubertal stage, auxological parameters, bone age/chronological age ratio, uterine length or testicular volume, and treatment-emergent adverse events (TEAEs). Summary statistics (computed by SAS^®) are reported.

Results: Overall, 66 patients completed the study (93.9% female; mean age 7.5 [SD, 0.9] years). At Month 6, 100% of patients had LH suppression, maintained at Month 12 in 98.5% of patients. Mean basal and peak LH and follicle-stimulating hormone levels were substantially 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 females at Month 6 and 12, respectively; all males 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. Relative to baseline, there was no bone age/chronological age ratio rise in 95.5% of patients at Month 6 and 92.4% at Month 12, and it decreased from 1.27 at baseline to 1.23 and 1.21 at Month 6 and 12, respectively. In females, 64.5% showed decreased uterine length at Month 6 and 12 vs baseline, while 75.0% of males showed no testicular volume increase vs baseline. Thirteen patients (19.7%) had 22 drug-related TEAEs (most common: weight increase (9.1%) and obesity, vaginal haemorrhage, hepatic stenosis (all 3.0%); no grade ≥3 TEAEs nor deaths 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 populations. Clinically relevant improvements were observed using two injections/year of the triptorelin 6-month PR formulation, supporting it as a viable option for Chinese children with CPP.