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
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.
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