Turner Syndrome
Conditions
Keywords
Inpegsomatropin Injection, Givopegsomatropin Solution Injection
Brief summary
This is a multicenter, randomized, open-label, positive-controlled phase III confirmatory clinical study. A total of 84 children with short stature due to Turner Syndrome (TS) are planned to be enrolled. Stratified by age and karyotype, subjects will be randomized at a 1:1 ratio to either the test group or the positive control group with continuous treatment for 52 weeks. The study aims to compare the efficacy and safety of Inpegsomatropin-Injection versus Givopegsomatropin Solution Injection in children with TS-related short stature, so as to provide evidence for the new indication application of the investigational drug.
Interventions
DRUGInpegsomatropin-Injection
Inpegsomatropin injection, 280 μg/kg/week, s.c., once weekly, for 52 weeks.
DRUGGivopegsomatropin Solution Injection
Givopegsomatropin Solution Injection, 200 μg/kg/week, s.c., once weekly, for 52 weeks.
Sponsors
Xiamen Amoytop Biotech Co., Ltd.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
FEMALE
Age
2 Years to 12 Years
Healthy volunteers
No
Inclusion criteria
* 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.
Exclusion criteria
* Subjects with closed epiphyses. * 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. * 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. * 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. * 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 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 a history of malignant tumor or current active malignant tumor, including intracranial tumors. * Known hypersensitivity to growth hormone or its excipients. * Subjects with celiac disease who have not maintained a gluten-free diet within 12 months prior to screening. * Any other conditions deemed inappropriate for enrollment in this clinical trial by the investigator.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Growth velocity (HV, cm/year). | Week 52 |
Secondary
| 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 height 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 |
| Ratio of change in bone age from baseline to change in chronological age from baseline (△BA/△CA) | Week 52 |
Countries
China
Contacts
CONTACTXiaoping Luo
PRINCIPAL_INVESTIGATORXiaoping Luo
Tongji Hospital
Outcome results
None listed