The Efficacy and Safety of Inpegsomatropin Injection in Children With Turner Syndrome(TS) and Short Stature

Multicenter, Randomized, Open-Label, Positive-Controlled Phase III Clinical Study to Evaluate the Efficacy and Safety of Inpegsomatropin-Injection Versus Givopegsomatropin Solution Injection in the Treatment of Short Stature in Children With Turner Syndrome (TS)

This is a multicenter, randomized, open-label, positive-controlled phase III confirmatory clinical study. A total of 84 children with short stature due to Turner Syndrome (TS) are planned to be enrolled. Stratified by age and karyotype, subjects will be randomized at a 1:1 ratio to either the test group or the positive control group with continuous treatment for 52 weeks. The study aims to compare the efficacy and safety of Inpegsomatropin-Injection versus Givopegsomatropin Solution Injection in children with TS-related short stature, so as to provide evidence for the new indication application of the investigational drug.

Study Overview

• Status: Not yet recruiting
• Conditions: Turner Syndrome
• Intervention / Treatment: Drug: Inpegsomatropin-Injection; Drug: Givopegsomatropin Solution Injection

• Study Type: Interventional
• Enrollment (Estimated): 84
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Xiaoping Luo; Phone Number: 15671671188; Email: xpluo@tjh.tjmu.edu.cn

Study Locations

• China
  • Anyang, China
    • Anyang Maternal and Child Health Hospital
    • Contact: Huiping An
  • Beijing, China
    • Peking University First Hospital
    • Contact: Yanling Yang
  • Beijing, China
    • Peking Union Medical College Hospital, Chinese Academy of Medical Sciences
    • Contact: Hui Pan
  • Beijing, China
    • Beijing Children's Hospital, Capital Medical University
    • Contact: Di Wu
  • Beijing, China
    • Capital Institute of Pediatrics, Capital Medical University
    • Contact: Bingyan Cao
  • Changsha, China
    • The Second Xiangya Hospital of Central South University
    • Contact: Xingxing Zhang
  • Chengdu, China
    • Chengdu Women's and Children's Central Hospital
    • Contact: Xinran Cheng
  • Chongqing, China
    • Children's Hospital of Chongqing Medical University
    • Contact: Min Zhu
  • Dalian, China
    • The First Affiliated Hospital of Dalian Medical University
    • Contact: Luyi Ma
  • Guangzhou, China
    • Sun Yat-sen Memorial Hospital, Sun Yat-sen University
    • Contact: Liyang Liang
  • Guangzhou, China
    • The First Affiliated Hospital of Sun Yat-sen University
  • Guangzhou, China
    • The third affiliated hospital of Sun Yat-Sen University
    • Contact: Shunye Zhu
  • Guangzhou, China
    • Guangdong Provincial Maternal and Child Health Hospital
    • Contact: Shu Liu
  • Hangzhou, China
    • The First Affiliated Hospital, Zhejiang University School of Medicine
    • Contact: Chunlin Wang
  • Hangzhou, China
    • Zhejiang Provincial Hospital of Traditional Chinese Medicine
    • Contact: Lan Li
  • Hebei, China
    • The First Affiliated Hospital of Hebei North University
    • Contact: Zhengrong Xu
  • Henan, China
    • The First Affiliated Hospital of Henan University of Science and Technology
    • Contact: Dan Lou
  • Huzhou, China
    • Huzhou Central Hospital
    • Contact: Wei Qiu
  • Jiangxi, China
    • Jiangxi Provincial Children's Hospital
    • Contact: Yu Yang
  • Jilin, China
    • The First Hospital of Jilin University
    • Contact: Yining Zhang
  • Jinan, China
    • Shandong Provincial Hospital
    • Contact: Yan Sun
  • Jining, China
    • Affiliated Hospital of Jining Medical University
    • Contact: Mei Zhang
  • Kunming, China
    • Kunming Children's Hospital
    • Contact: Na Tao
  • Lanzhou, China
    • Gansu Provincial People's Hospital
    • Contact: Juxiang Liu
  • Nanning, China
    • The Second Affiliated Hospital of Guangxi Medical University
    • Contact: Xin Fan
  • Pingxiang, China
    • Pingxiang People's Hospital
    • Contact: Guangping Qin
  • Qilu, China
    • Qilu Hospital of Shandong University
    • Contact: Aihua Cao
  • Shanghai, China
    • Ruijin Hospital, Shanghai Jiao Tong University School of Medicine
    • Contact: Wenli Lu
  • Shanghai, China
    • Shanghai Children's Hospital
    • Contact: Fei Wang
  • Shanghai, China
    • Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
    • Contact: Wenjuan Qiu
  • Shanghai, China
    • Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine
    • Contact: Yu Ding
  • Shanxi, China
    • The Second Affiliated Hospital of Shaanxi University of Chinese Medicine
    • Contact: Haibo Lin
  • Shaoxing, China
    • Shaoxing People's Hospital
    • Contact: Dihua Huang
  • Shenyang, China
    • Shengjing Hospital of China Medical University
    • Contact: Ying Xin
  • Shenzhen, China
    • Shenzhen Children's Hospital
    • Contact: Zhe Su
  • Sichuan, China
    • West China Second University Hospital, Sichuan University
    • Contact: Jin Wu
  • Suzhou, China
    • Children's Hospital of Soochow University
    • Contact: Fengyun Wang
  • Taizhou, China
    • Taizhou First People's Hospital
    • Contact: Xiaoming Ying
  • Taizhou, China
    • Taizhou People's Hospital
    • Contact: Shufang Yang
  • Tangshan, China
    • Tangshan Maternal and Child Health Hospital
    • Contact: Lanying Wang
  • Wenzhou, China
    • The First Affiliated Hospital of Wenzhou Medical University
    • Contact: Hong Zhu
  • Wuhan, China
    • Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology
    • Contact: Xiaoping Luo
  • Wuhan, China
    • Wuhan Children's Hospital
    • Contact: Hui Yao
  • Wuxi, China
    • Affiliated Hospital of Jiangnan University
    • Contact: Yaping Ma
  • Xi'an, China
    • The Second Affiliated Hospital of Xi'an Jiaotong University
    • Contact: Erdi Xu
  • Xiamen, China
    • Xiamen Maternal and Child Health Hospital
    • Contact: Xiaoqing Yang
  • Xiangyang, China
    • Xiangyang First People's Hospital
    • Contact: Xi Yang
  • Xinjiang, China
    • The First Affiliated Hospital of Xinjiang Medical University
    • Contact: Mirigul Maimaiti
  • Yibin, China
    • Yibin Second People's Hospital
    • Contact: Lan Yao
  • Zhengzhou, China
    • Henan Children's Hospital Zhengzhou Children's Hospital
    • Contact: Haiyan Wei
  • Zibo, China
    • Zibo Central Hospital
    • Contact: Xiaodong Zhao

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Prepubertal girls at Tanner stage I, with age ≥ 2 years and < 12 years at the time of informed consent signature.
• With clinical manifestations of Turner syndrome and a confirmed diagnosis of Turner syndrome based on peripheral blood karyotype analysis (karyotype analysis of at least 30 metaphase cells).
• At screening, bone age is delayed relative to chronological age or advanced by no more than 1 year (i.e., bone age - chronological age ≤ 1 year).
• At screening, height is below -2 standard deviations (-2SD) of the mean for age and gender; height reference is shown in Appendix 1.
• No prior systematic pharmacological growth-promoting treatment (continuous use for ≥ 1 month), including but not limited to growth hormone, insulin-like growth factor 1 (IGF-1), etc.
• Thyroid hormone replacement therapy (if applicable) received prior to randomization should be maintained on a stable regimen for at least 4 weeks.
• The legal guardian understands and signs the informed consent form; participants aged ≥ 8 years shall also sign the informed consent form. For participants aged under 8 years who are capable of expressing assent, their assent shall be clearly documented.

Exclusion Criteria:

• Subjects with closed epiphyses.
• Patients with Turner syndrome carrying Y chromosome or Y-chromosome-derived fragments and without gonadectomy.
• Other types of growth and development abnormalities, including but not limited to growth hormone deficiency (GHD), Noonan syndrome, Prader-Willi syndrome, and growth retardation caused by malnutrition.
• Participation in any other clinical trial within 3 months prior to screening with pharmacological or non-pharmacological intervention received.
• Inhaled glucocorticoids used continuously for more than 2 weeks, or oral/intravenous glucocorticoids used continuously for more than 1 week within 3 months prior to screening.
• Receiving other treatments that may affect growth, including but not limited to methylphenidate, sex hormones, gonadotropin-releasing hormone analogs, aromatase inhibitors, anabolic agents, etc.
• Abnormal liver and renal function at screening (ALT > 2 times the upper limit of normal; Cr > upper limit of normal).
• Subjects with abnormal glucose metabolism, including: a. Diagnosed diabetes mellitus; b. Fasting blood glucose ≥ 6.1 mmol/L on two consecutive measurements; c. Glycated hemoglobin (HbA1c) ≥ 6.5%; d. Impaired glucose tolerance judged by the investigator as unsuitable for participation in this study.
• Presence of chronic infectious diseases judged by the investigator to interfere with study participation, such as chronic hepatitis B.
• Subjects with systemic chronic diseases, such as chronic kidney disease, severe cardiovascular diseases (e.g., aortic dissection, uncontrolled hypertension), psychiatric and psychological disorders.
• Subjects with severe congenital skeletal dysplasia; or those with scoliosis > 20°, significant kyphosis, claudication, or a prior diagnosis of slipped capital femoral epiphysis.
• Subjects with a prior history of intracranial hypertension.
• Subjects with a history of malignant tumor or current active malignant tumor, including intracranial tumors.
• Known hypersensitivity to growth hormone or its excipients.
• Subjects with celiac disease who have not maintained a gluten-free diet within 12 months prior to screening.
• Any other conditions deemed inappropriate for enrollment in this clinical trial by the investigator.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Experimental Group	Drug: Inpegsomatropin-Injection; Inpegsomatropin injection, 280 μg/kg/week, s.c., once weekly, for 52 weeks.
Active Comparator: Control Group	Drug: Givopegsomatropin Solution Injection; Givopegsomatropin Solution Injection, 200 μg/kg/week, s.c., once weekly, for 52 weeks.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Growth velocity (HV, cm/year).	Week 52

Secondary Outcome Measures

Outcome Measure	Time Frame
Change in height standard deviation score from baseline (△HT SDS)	From baseline to all follow-up time points at Week 52
Change in insulin-like growth factor 1 standard deviation score from baseline (△IGF-1 SDS)	From baseline to all follow-up time points at Week 52
Change in height velocity from baseline (△HV)	From baseline to all follow-up time points at Week 52
Ratio of change in bone age from baseline to change in chronological age from baseline (△BA/△CA)	Week 52

Collaborators and Investigators

• Sponsor: Xiamen Amoytop Biotech Co., Ltd.
• Investigators: Principal Investigator: Xiaoping Luo, Tongji Hospital

Study record dates

Study Major Dates

• Study Start (Estimated): June 15, 2026
• Primary Completion (Estimated): March 30, 2028
• Study Completion (Estimated): February 28, 2030

Study Registration Dates

• First Submitted: May 21, 2026
• First Submitted That Met QC Criteria: May 21, 2026
• First Posted (Actual): May 29, 2026

Study Record Updates

• Last Update Posted (Actual): May 29, 2026
• Last Update Submitted That Met QC Criteria: May 21, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Keywords: Inpegsomatropin Injection; Givopegsomatropin Solution Injection
• Additional Relevant MeSH Terms: Urogenital Diseases; Endocrine System Diseases; Cardiovascular Diseases; Male Urogenital Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Heart Diseases; Genetic Diseases, Inborn; Gonadal Disorders; Congenital Abnormalities; Cardiovascular Abnormalities; Heart Defects, Congenital; Disorders of Sex Development; Urogenital Abnormalities; Sex Chromosome Disorders; Chromosome Disorders; Sex Chromosome Disorders of Sex Development; Gonadal Dysgenesis; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Turner Syndrome
• Other Study ID Numbers: PEGPESEN-4-2-004

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No