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

Launched by XIAMEN AMOYTOP BIOTECH CO., LTD. · Apr 13, 2025

Keywords

ClinConnect Summary

This clinical trial is studying a new medication called Inpegsomatropin, which is an injection given once a week, to see how well it works and how safe it is for children with idiopathic short stature (ISS). ISS means that these children are shorter than most kids their age and there isn't a clear medical reason for their height. The trial will include 300 prepubertal boys and girls aged between 3 and 11 years who meet specific height and growth criteria. To participate, children must have a certain bone age and height that is significantly below average for their age and gender.

During the trial, participants will go through a screening period of up to 12 weeks, a treatment period lasting 52 weeks, and then a follow-up period of 5 weeks. Throughout this time, researchers will monitor the children's growth and any side effects from the treatment. It's important to know that children with certain health issues, like specific growth disorders or chronic diseases, will not be eligible to join the study. This trial is not currently recruiting participants, but if you think your child may qualify, it's a good idea to discuss it with your doctor for more information.

Gender

ALL

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.