Growth disorder caused by insufficient growth hormone secretion in children
Conditions
Interventions
Test group:Study drug GB06
Control group:Norditropin? FlexPro?
Sponsors
Chengdu Women's and Children's Central Hospital, School of Medicine, University of Electronic Science and Technology of China
Eligibility
Sex/Gender
All
Age
3 Years to 11 Years
Inclusion criteria
Inclusion criteria: 1. The guardian understands and signs the written informed consent form (ICF); participants aged 8 years or older also need to sign the ICF, and for those under 8 years who can express consent, their opinions should be explicitly recorded; 2. Age at signing the informed consent form >= 3 years, with boys <= 11 years and girls <= 10 years; 3. Height at screening is below 2 standard deviations (SD) of the average height for healthy children of the same age and sex (see Attachment 1); 4. Annualized height velocity (AHV) within 6 to 18 months prior to screening < 5 cm/year (referencing the 2008 Guidelines for Diagnosis and Treatment of Short Stature in Children by the Pediatrics Endocrinology and Genetics Group of the Chinese Medical Association, and Recommendations for Clinical Application of Recombinant Human Growth Hormone in Pediatrics); 5. Body mass index (BMI) within ±2 SD of the average for healthy children of the same age and sex (see Attachment 2); 6. Proportionate short stature with normal intelligence; 7. Development at Tanner stage I (testicular volume < 4 ml for boys, no palpable breast tissue for girls, see Attachment 3); 8. IGF-1 levels below the reference value corresponding to -1 SDS for children of the same age and sex (see Appendix 4 for details); 9. Bone age lagging behind chronological age; 10. Diagnosis of growth hormone deficiency (GHD) confirmed by two different drug combined GH stimulation tests within 12 months prior to screening, with a GH peak level <=10.0 ng/ml (refer to the National Medical Products Administration Center for Drug Evaluation "Technical Guidelines for Clinical Trials of Growth Hormone Preparations for Growth Hormone Deficiency"); 11. Girls with normal karyotype 46, XX (any previous chromosome test results are acceptable);
Exclusion criteria
Exclusion criteria: 1. Known allergy to components of the investigational drug; 2. Previously received recombinant human growth hormone (rhGH) or IGF-1 treatment or combined use of other treatments that may affect growth, including but not limited to sex hormones and anabolic steroids, excluding hormone replacement therapy (thyroid hormone, hydrocortisone); 3. Received any investigational drug within 3 months prior to screening or participated in another clinical trial before randomization; 4. Small for gestational age (according to the 2023 publication "Diagnosis and Clinical Management of Small for Gestational Age," birth weight below the 10th percentile of the average weight for the same sex and gestational age); 5. Epiphyseal closure; 6. Congenital intracranial hypertension; 7. Slipped capital femoral epiphysis; 8. Growth hormone deficiency as part of other pituitary hormone deficiencies; 9. History of or current malignant tumor (including intracranial tumor); intracranial tumors must be confirmed by MRI or CT. If medical evaluation and conclusion are available, images or scans obtained within 12 months before screening can be used as screening data; 10. History of fundus lesions (optic disc edema); 11. Diagnosed diabetes, or fasting blood glucose >= 7.0 mmol/L or HbA1c >= 6.5%; 12. Received systemic corticosteroid treatment for more than 2 consecutive weeks within 3 months prior to screening; 13. Children expected to require inhaled budesonide > 400 µg/day or equivalent dosage of inhaled glucocorticoid continuously for more than 4 weeks during the trial; 14. Other growth abnormalities or conditions that may affect height, including but not limited to: chromosomal aneuploidy, Turner syndrome, Laron syndrome, Noonan syndrome, Prader-Willi syndrome, SHOX-1 gene abnormalities, GH receptor deficiency, and other medical syndromes with short stature caused by significant gene mutations; significant spinal abnormalities, including but not limited to scoliosis, kyphosis, and spinal bifida variants; congenital abnormalities (leading to skeletal abnormalities), including but not limited to Russell-Silver syndrome and skeletal dysplasia; family history of skeletal dysplasia; 15. Other clinically significant abnormalities that may affect growth or the assessment of growth capacity, including but not limited to liver and kidney dysfunction [e.g., alanine aminotransferase (ALT) > 1.5 times the upper limit of normal, creatinine (Cr) > upper limit of normal], malnutrition, severe cardiovascular, pulmonary, or hematologic diseases, systemic infections, immunodeficiency, psychiatric abnormalities, and coexisting other congenital malformations; 16. Infectious diseases, such as hepatitis B, hepatitis C, HIV/AIDS, syphilis, or tuberculosis. Participants positive for HBV surface antigen must undergo further testing for hepatitis B virus DNA (HBV DNA); those positive for hepatitis C virus antibody must undergo further testing for hepatitis C virus RNA (HCV RNA). Participants are excluded if HBV DNA or HCV RNA is above the quantitative lower limit; 17. Concurrent use of other treatments that may affect growth, including but not limited to the use of methylphenidate for attention deficit hyperactivity disorder (ADHD); 18. History of substance, drug, or alcohol abuse; 19. Children with hypothyroidism and/or adrenal insufficiency who have not received sufficiently stable replacement therapy for at least 90 days prior to randomization; 20. Other diseases that the investigator bel
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| AHV; | — |
Secondary
| Measure | Time frame |
|---|---|
| Chronological age (CA) height SDS (Ht SDS CA) and its change from baseline;Bone age (BA) and its change from baseline;All observed adverse events, including abnormal clinical symptoms and vital signs, administration site examinations, laboratory and imaging examinations (including but not limited to fasting blood glu;Tanner staging and its change from baseline;Anti?drug antibody (ADA) and neutralizing antibody (NAb);BA/CA and its change from baseline;IGF-1 and IGF-1 SDS, along with their changes from baseline.;Height SDS by bone age (Ht SDS BA) and its change from baseline.;IGFBP-3 and IGFBP-3 SDS, and their changes from baseline.;Annualized Height Velocity (AHV) standard deviation score (SDS) and its change from baseline.; | — |
Countries
China
Contacts
Public ContactCheng Xinran
Chengdu Women's and Children's Central Hospital, School of Medicine, University of Electronic Science and Technology of China
Outcome results
None listed