Clinical trial NCT06927310: The Efficacy and Safety of Inpegsomatropin Injection in Children With Idiopathic Short Stature
Official title: 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).
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE3
Conditions: Idiopathic Short Stature
Keywords: Inpegsomatropin Injection
Recombinant Human Growth Hormone Injection
Lead sponsor: Xiamen Amoytop Biotech Co., Ltd.
Sponsor class: INDUSTRY
Collaborators: name: Tongji Hospital
First posted: 2025-04-15
Enrollment: 300
Sex: ALL
Minimum age (years): 3
Maximum age (years): 11
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: 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.
* Bone age ≤ chronological age,with a delay of no more than 2 years compared to chronological age.
* 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 have received inhaled corticosteroids for more than 2 consecutive weeks, or oral or intravenous corticosteroids for more than 1 week within 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 anemia, malnutrition, hypothyroidism, chronic kidney disease, cardiovascular diseases (e.g., congenital heart disease, dilated cardiomyopathy, etc.), psychiatric disorders, or those with congenital anomalies;
* 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.

Primary outcomes:
measure: Growth velocity (HV, cm/year).
timeFrame: Week 52
Secondary outcomes:
measure: Change in height standard deviation score from baseline (△HT SDS)
timeFrame: From baseline to all follow-up time points at Week 52
measure: Change in growth velocity from baseline (△HV)
timeFrame: From baseline to all follow-up time points at Week 52
measure: Change in insulin-like growth factor 1 standard deviation score from baseline (△IGF-1 SDS)
timeFrame: From baseline to all follow-up time points at Week 52
measure: The ratio of the change in bone age to the change in chronological age (△BA/△CA)
timeFrame: From baseline to all follow-up time points at Week 52
Other outcomes:
Locations:
city: Hefei
country: China
facility: The Second Affiliated Hospital of Anhui Medical University
state: Anhui
city: Beijing
country: China
facility: Capital Institute of Pediatrics, Beijing (SHOUER)
state: Beijing
city: Chongqing
country: China
facility: Children's Hospital of Chongqing Medical University
state: Chongqing
city: Xiamen
country: China
facility: The First Affiliated Hospital of Xiamen University
state: Fujian
city: Guangzhou
country: China
facility: Guangzhou Women and Children's Medical Center
state: Guangdong
city: Guangzhou
country: China
facility: Sun Yat-sen Memorial Hospital, Sun Yat-sen University
state: Guangdong
city: Guangzhou
country: China
facility: The Third Affiliated Hospital, Sun Yat-sen University
state: Guangdong
city: Zhengzhou
country: China
facility: Henan Children's Hospital Zhengzhou Children's Hospital
state: Henan
city: Wuhan
country: China
facility: Wuhan Children's Hospital
state: Hubei
city: Changsha
country: China
facility: Hunan Children's Hospital
state: Hunan
city: Suzhou
country: China
facility: Children's Hospital Affiliated to Soochow University
state: Jiangsu
city: Nanchang
country: China
facility: Jiangxi Provincial Children's Hospital
state: Jiangxi
city: Shanghai
country: China
facility: Children's Hospital of Shanghai
state: Shanghai
city: Chengdu
country: China
facility: Chengdu Women's and Children's Central Hospital
state: Sichuan
city: Chengdu
country: China
facility: West China Second University Hospital, Sichuan University
state: Sichuan
city: Ningbo
country: China
facility: The Affiliated Women's and Children's Hospital of Ningbo University
state: Zhejiang
city: Wenzhou
country: China
facility: The Second Affiliated Hospital and Yuying Children's Hospital of Wenzhou Medical University
state: Zhejiang
Study officials:
affiliation: Tongji Hospital
name: Xiaoping Luo, Ph.D
role: PRINCIPAL_INVESTIGATOR
References: