the efficacy and safety of inpegsomatropin injection in children with idiopathic short stature

Conditions: Idiopathic Short Stature

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.

Keywords: inpegsomatropin injection recombinant human growth hormone injection