Idiopathic Short Stature (ISS)
Conditions
Keywords
Idiopathic Short Stature, ISS
Brief summary
This study is a multicenter, randomized, open-label, active-controlled, non-inferiority Phase III clinical trial. It aims to evaluate the efficacy and safety of Human Growth Hormone (hGH) Injection (Sinotropin AQ) in pediatric participants with Idiopathic Short Stature (ISS). Eligible participants will be randomized to receive either the study drug or an active comparator for 52 weeks. The primary efficacy endpoint is height velocity (HV, cm/year) over the treatment period. The study drug will be administered subcutaneously once daily at a dose of 0.15 IU/kg. The results of this trial will provide evidence on whether hGH Injection represents an effective and safe treatment option for improving height in children with ISS.
Interventions
This product is human growth hormone (hGH) injection (Sinotropin AQ). Usage and dosage: 0.15 IU/kg/day (0.05 mg/kg/day), once daily, administered by subcutaneous injection.
Sponsors
Zhongshan Sansure Hygene Biomedicine Co., Ltd.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
3 Years to 11 Years
Healthy volunteers
No
Inclusion criteria
* 1\. Tanner stage I (prepubertal) at informed consent: males aged 3 to \<11 years ; females aged 3 to \<10 years ; * 2\. At screening, height \< -2 standard deviations (SD) for age and sex (height reference per Appendix 1); * 3\. Peak GH ≥10.0 ng/mL in any growth hormone (GH) stimulation test; * 4\. Bone age minus chronological age ≤ 1 year.
Exclusion criteria
* 1.Closed epiphyses (skeletally mature)； * 2.Severe allergic diathesis, or known hypersensitivity to growth hormone or any of its excipients； * 3.History of malignancy or current active malignancy； * 4.Systemic chronic diseases, including but not limited to moderate-to-severe anemia, hypothyroidism, chronic kidney disease, cardiovascular disease (e.g., dilated cardiomyopathy), psychiatric disorders, or congenital anomalies that require clinical intervention per the investigator's judgment. * 5.Patients with a prior diagnosis of intracranial hypertension; * 6.Patients with congenital skeletal dysplasia, or scoliosis ≥15° (or moderate or greater), limping (gait disturbance), and patients with a prior diagnosis of slipped capital femoral epiphysis ; * 7.Other types of growth and developmental disorders, including confirmed or highly suspected growth hormone deficiency (GHD), Noonan syndrome, Prader-Willi syndrome, Russell-Silver syndrome, Turner syndrome, small for gestational age (SGA), short stature due to SHOX gene defects, and short stature of other identified causes; * 8\. Patients with a confirmed diagnosis of diabetes mellitus, or fasting blood glucose ≥6.1 mmol/L on two consecutive measurements.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| The value of Annual Height velocity | At the end of 52 weeks of treatment | The value of Annual Height velocity (HV, cm/year) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| The change of ΔHT SDS value | At all visit points from baseline to week 52 | The change from baseline in height SDS (ΔHT SDS) |
| The change of IGF-1 SDS value | At all visit points from baseline to Week 52 | The change of insulin-like growth factor-1 standard deviation score value |
| The change of height velocity value (ΔHV) | At all visit points from baseline to Week 52 | The change from baseline in height velocity(ΔHV) |
| The change of bone age relative to change of chronological age(ΔBA/ΔCA) value | At all visit points from basline to week 52 | The change of bone age (BA) relative to the change in chronological age (CA) from basline to end of 52-week treatment |
| The safety of human growth hormone injection | Baseline, Week 13, Week 26, Week 39, and Week 52 | Evaluate all TEAEs, SAEs, and clinically significant abnormalities in laboratory tests, vital signs, and physical examination. |
| Immunogenicity of Human Growth Hormone Injection | At all visit points from baseline to week 52 | Assessment of anti-GH and neutralizing antibody incidence |
Countries
China
Contacts
CONTACTJian Qi
Outcome results
None listed