The Efficacy and Safety of Inpegsomatropin Injection in Children With Idiopathic Short Stature

A Multicenter, Randomized, Open-Label, Active-Controlled Phase III Clinical Study to Evaluate the Efficacy and Safety of Inpegsomatropin Injection, s.c Once a Week, Compared With Recombinant Human Growth Hormone (rhGH) Injection in Children With Idiopathic Short Stature (ISS).

This is a multicenter, randomized, open-label, active-controlled Phase III clinical trial to evaluate the efficacy and safety of Inpegsomatropin injection，once a week，compared with recombinant human growth hormone (rhGH) in children with idiopathic short stature (ISS). It plans to enroll 300 children with ISS, who will be randomized , stratified by gender and age, and assigned to either the experimental group or the positive controlled group. Each participant will undergo a screening period (up to 12 weeks), a treatment period (52 weeks), and a post-treatment follow-up period (5 weeks). And the safety and efficacy will be evaluated.

Study Overview

• Status: Recruiting
• Conditions: Idiopathic Short Stature
• Intervention / Treatment: Drug: Inpegsomatropin Injection; Drug: Recombinant Human Growth Hormone Injection

• Study Type: Interventional
• Enrollment (Estimated): 300
• Phase: Phase 3

Contacts and Locations

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

Study Locations

• China
  • Hainan, China
    • Recruiting
    • Sanya Central Hospital
    • Contact: Hua Zhang
  • Hangzhou, China
    • Recruiting
    • Zhejiang Provincial People's Hospital
    • Contact: Xiaoming Luo
  • Hangzhou, China
    • Recruiting
    • Hangzhou First People's Hospital
    • Contact: Chunming Jiang
  • Henan, China
    • Recruiting
    • The First Affiliated Hospital of Henan University of Science and Technology
    • Contact: Dan Lou
  • Jiaxing, China
    • Recruiting
    • Jiaxing First Hospital
    • Contact: Shunfeng Mao
  • Jining, China
    • Recruiting
    • Affiliated Hospital of Jining Medical University
    • Contact: Meiyun Xin
  • Kunming, China
    • Recruiting
    • Kunming Children's Hospital
    • Contact: Na Tao
  • Nanjing, China
    • Recruiting
    • Nanjing Children's Hospital
    • Contact: Wei Gu
  • Qingdao, China
    • Recruiting
    • Qingdao Women and Children's Hospital
    • Contact: Tang Li
  • Shanghai, China
    • Recruiting
    • Shanghai Children's Medical Center
    • Contact: Xiumin Wang
    • Contact: Yu Ding
  • Shanghai, China
    • Recruiting
    • Xinhua Hospital Affiliated to Shanghai Jiao Tong University School of Medicine
    • Contact: Huiwen Zhang
  • Shanghai, China
    • Recruiting
    • Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine
    • Contact: Wenli Lu
  • Shijiazhuang, China
    • Recruiting
    • The First Hospital of Hebei Medical University
    • Contact: Huijuan Ma
  • Wuhan, China
    • Recruiting
    • Renmin Hospital of Wuhan University
    • Contact: Jingping Ye
  • Xi'an, China
    • Recruiting
    • The Second Affiliated Hospital of Xi'an Jiaotong University
    • Contact: Erdi Xu
  • Xiangyang, China
    • Recruiting
    • Xiangyang Central Hospital
    • Contact: Rui Pan
  • Xinjiang, China
    • Recruiting
    • The First Affiliated Hospital of Xinjiang Medical University
    • Contact: Miregul Mamat
  • Yuncheng, China
    • Recruiting
    • Yuncheng Central Hospital
    • Contact: Sufang Chang
  • Zhuzhou, China
    • Recruiting
    • ZhuZhou Central Hospital
    • Contact: Xianglan Wen
  • Anhui
    • Hefei, Anhui, China
      • Recruiting
      • Anhui Provincial Children's Hospital
      • Contact: Yuqing Chen
    • Hefei, Anhui, China
      • Recruiting
      • The Second Affiliated Hospital of Anhui Medical University
      • Contact: Deyun Liu
  • Beijing Municipality
    • Beijing, Beijing Municipality, China
      • Recruiting
      • Capital Institute of Pediatrics, Beijing (SHOUER)
      • Contact: Fuying Song
  • Chongqing Municipality
    • Chongqing, Chongqing Municipality, China
      • Recruiting
      • Children's Hospital of Chongqing Medical University
      • Contact: Min Zhu
  • Fujian
    • Xiamen, Fujian, China
      • Recruiting
      • The First Affiliated Hospital of Xiamen University
      • Contact: Qun Lian
  • Gansu
    • Lanzhou, Gansu, China
      • Recruiting
      • Gansu Provincial Maternity and Child Care Hospital
      • Contact: Bo Sun
  • Guangdong
    • Guangzhou, Guangdong, China
      • Recruiting
      • Sun Yat-Sen Memorial Hospital, Sun Yat-Sen University
      • Contact: Liyang Liang
    • Guangzhou, Guangdong, China
      • Recruiting
      • Guangzhou Women and Children's Medical Center
      • Contact: Wen Zhang
    • Guangzhou, Guangdong, China
      • Recruiting
      • The Third Affiliated Hospital, Sun Yat-sen University
      • Contact: Shunye Zhu
  • Henan
    • Zhengzhou, Henan, China
      • Recruiting
      • Henan Children's Hospital Zhengzhou Children's Hospital
      • Contact: Haiyan Wei
  • Hubei
    • Wuhan, Hubei, China
      • Recruiting
      • Wuhan Children's Hospital
      • Contact: Hui Yao
  • Hunan
    • Changsha, Hunan, China
      • Recruiting
      • Hunan Children's Hospital
      • Contact: Yan Zhong
  • Jiangsu
    • Suzhou, Jiangsu, China
      • Recruiting
      • Children's Hospital Affiliated to Soochow University
      • Contact: Haiying Wu
  • Jiangxi
    • Nanchang, Jiangxi, China
      • Recruiting
      • Jiangxi Provincial Children's Hospital
      • Contact: Yu Yang
  • Shanghai Municipality
    • Shanghai, Shanghai Municipality, China
      • Recruiting
      • Children's Hospital of Shanghai
      • Contact: Pin Li
  • Sichuan
    • Chengdu, Sichuan, China
      • Recruiting
      • Chengdu Women's and Children's Central Hospital
      • Contact: Xinran Cheng
    • Chengdu, Sichuan, China
      • Recruiting
      • West China Second University Hospital, Sichuan University
      • Contact: Fan Yang
  • Zhejiang
    • Ningbo, Zhejiang, China
      • Recruiting
      • The Affiliated Women's and Children's Hospital of Ningbo University
      • Contact: Shuxia Ding
    • Wenzhou, Zhejiang, China
      • Recruiting
      • The Second Affiliated Hospital and Yuying Children's Hospital of Wenzhou Medical University
      • Contact: Xiaoou Shan

Participation Criteria

Eligibility Criteria

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

Description

Inclusion Criteria:

• Prepubertal children: boys aged ≥3 years and <11 years at screening with testicular volume <4 mL; girls aged ≥3 years and <10 years at screening with breast development at Tanner Stage I, i.e., no palpable breast glandular tissue.
• Compared to chronological age, bone age is advanced by no more than 1 year or delayed by no more than 2 years (i.e., -2 years ≤ bone age - chronological age ≤ 1 year).
• Height at screening is below -2 standard deviations (SD) from the mean for age and sex, with height reference to Appendix 1.
• Body mass index (BMI) is within the 5th to 95th percentile for age and sex of healthy children, with reference to Appendix 2.
• Peak GH level ≥10.0 ng/ml in at least one GH stimulation test.
• No prior systemic pharmacological treatment for growth promotion (continuous use ≥1 month), including but not limited to growth hormone, insulin-like growth factor 1 (IGF-1), etc.
• The legal guardian understands and signs the informed consent form. If the participants is ≥8 years old, they must also sign the informed consent form. For participants under 8 years old who are able to express their consent, their assent should be clearly documented.

Exclusion Criteria:

• Individuals with closed epiphyses;
• Other types of growth and developmental abnormalities, including confirmed or highly suspected growth hormone deficiency (GHD), Noonan syndrome, Prader-Willi syndrome, Russell-Silver syndrome, Turner syndrome, small for gestational age, short stature due to SHOX gene abnormalities, growth delay due to malnutrition, growth delay due to hypothyroidism, and other short statures with a clear etiology; genetic testing results must be obtained before randomization to exclude short stature caused by definite genetic abnormalities;
• Individuals who have participated in any other clinical trial within 3 months prior to screening and have received pharmacological or non-pharmacological interventions;
• Individuals who received inhaled corticosteroids for more than 2 consecutive weeks, or oral or intravenous corticosteroids for more than 1 consecutive week, within the 3 months prior to screening;
• Individuals who are currently receiving or require long-term treatment with other therapies that may affect growth, including but not limited to methylphenidate, sex hormones, gonadotropin-releasing hormone analogs, aromatase inhibitors, anabolic agents, insulin, etc.;
• Individuals with abnormal liver or kidney function at screening (ALT > 1.5 times the upper limit of normal, Cr > upper limit of normal);
• Individuals diagnosed with diabetes mellitus, or with fasting blood glucose ≥6.1 mmol/L on two consecutive occasions;
• Individuals with chronic infectious diseases, such as chronic hepatitis B;
• Individuals with systemic chronic diseases, such as moderate to severe anemia, malnutrition, hypothyroidism, chronic kidney disease, cardiovascular diseases (e.g., dilated cardiomyopathy, etc.), psychiatric and psychological disorders, or those with congenital anomalies requiring clinical intervention as determined by the investigator;
• Individuals with congenital skeletal abnormalities, or those with scoliosis exceeding 15°, limping, or a history of slipped capital femoral epiphysis;
• Individuals with a history of increased intracranial pressure;
• Individuals with a history of malignancy or currently having active malignancy, including intracranial tumors;
• Individuals with severe allergic constitutions, or known allergies to growth hormone or its excipients;
• Individuals with any other disease that the investigator deems may endanger the subject's safety or compromise compliance with the study protocol.

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: Inpegsomatropin group	Drug: Inpegsomatropin Injection; Inpegsomatropin injection, 280 μg/kg/week, s.c., once weekly, for 52 consecutive weeks.
Active Comparator: rhGH group	Drug: Recombinant Human Growth Hormone Injection; Recombinant Human Growth Hormone Injection, 350 μg/kg/week, s.c., divided into 7 doses, i.e., 50 μg/kg/day (0.15 IU/kg/day), once daily for 52 consecutive 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 growth velocity from baseline (△HV)	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
The ratio of the change in bone age to the change in chronological age (△BA/△CA)	From baseline to all follow-up time points at Week 52

Collaborators and Investigators

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

Study record dates

Study Major Dates

• Study Start (Actual): April 28, 2025
• Primary Completion (Estimated): March 1, 2026
• Study Completion (Estimated): June 1, 2028

Study Registration Dates

• First Submitted: April 6, 2025
• First Submitted That Met QC Criteria: April 13, 2025
• First Posted (Actual): April 15, 2025

Study Record Updates

• Last Update Posted (Actual): March 11, 2026
• Last Update Submitted That Met QC Criteria: March 10, 2026
• Last Verified: March 1, 2026

More Information

Terms related to this study

• Keywords: Inpegsomatropin Injection; Recombinant Human Growth Hormone Injection
• Additional Relevant MeSH Terms: Endocrine System Diseases; Bone Diseases; Musculoskeletal Diseases; Genetic Diseases, Inborn; Bone Diseases, Developmental; Dwarfism; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Peptide Hormones; Peptides; Amino Acids, Peptides, and Proteins; Pituitary Hormones; Pituitary Hormones, Anterior; Growth Hormone
• Other Study ID Numbers: PEGPESEN-4-2-001

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