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

Ki vehet részt

• 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

Ki nem vehet részt

• 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
• 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 celiac disease who have not maintained a gluten-free diet within 12 months prior to screening
• 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
• 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 a history of malignant tumor or current active malignant tumor, including intracranial tumors
• Known hypersensitivity to growth hormone or its excipients
• Any other conditions deemed inappropriate for enrollment in this clinical trial by the investigator
• 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
• Subjects with closed epiphyses