Noonan Syndrome
Conditions
Interventions
intervention group:Polyethylene glycol recombinant human growth hormone (Kinserzin)
control group:Recombinant human growth hormone (Cezin) therapy
Sponsors
Shallghai Children's Medical Center
Eligibility
Sex/Gender
All
Age
1 Years to 9 Years
Inclusion criteria
Inclusion criteria: 1. Girls aged 1-8 years old and boys aged 1-9 years old. Physical examination is within the range of pre-puberty (Tanner Stage 1); 2. Children in line with NS clinical diagnosis; 3. Meet the genetic diagnosis; 4. Meet the conditions for the use of growth hormone.
Exclusion criteria
Exclusion criteria: 1. IGF-1 exceeding the normal value of +2.5 SDS for the same age and sex; 2. Combined with other clear causes of growth disorder and short stature, such as GHD, hypothyroidism, multiple pituitary hormone deficiency, SGA, Turner syndrome, short stature associated with other serious chronic diseases (such as malnutrition, heart insufficiency, liver and kidney insufficiency, chronic inflammatory bowel disease); 3. Patients who plan to undergo orthopaedic surgery within one year, or patients whose fractures are not healed; Significant spinal abnormalities; 4. Severe abnormal liver function: ALT or AST exceeding 3 times the upper limit of normal value; Or have hepatitis B "Dasanyang" or other types of viral hepatitis patients; Patients with a history of using alkylating agents; 5. Severe renal dysfunction: that is, CKD-EPI formula was used to estimate the glomerular filtration rate eGFR the upper limit of normal value; 6. Patients with serious cardiovascular diseases: including serious heart disease (such as heart function grade III or above, severe arrhythmia, hypertrophic cardiomyopathy, etc.), and poorly controlled hypertension (such as systolic blood pressure > 140mmHg or diastolic blood pressure > 90mmHg under treatment); 7. Patients with severe brain disease: patients who were considered at risk for growth hormone replacement therapy if head MRI/CT showed intracranial occupying abnormalities at screening/baseline; Or have a history of epilepsy; 8. Patients with abnormal glucose regulation (including impaired fasting blood glucose and abnormal glucose tolerance) or diabetes mellitus; A family history of diabetes; 9. Patients with other serious medical conditions affecting the completion of the trial protocol, such as severe obesity, uncontrolled severe obstructive sleep apnea, active psychosis, severe infection, immunocompromised patients, etc.; 10. Use of other drugs within the last 3 months that may affect the efficacy determination of the study endpoint: These include other growth hormone drugs, aromatase inhibitors (including but not limited to letrozole and anastrozole), gonadotropin-releasing hormone analogues (including but not limited to triprelline and leprelline), sex hormones (including but not limited to any type of estrogen, progesterone and androgen), protein assimilation drugs (including but not limited to oxyandroxone, danazole and stanzolol) ), somatostatin and somatostatin analogues (including but not limited to sitanine and octreotide); Superphysiological dose of glucocorticoids; Drugs that inhibit PTH (such as xenacase, etc.); Drugs that affect calcium and phosphorus metabolism (such as diuretics, etc.); 11. Patients with contraindication for growth hormone therapy: acute hyperplastic or severe non-hyperplastic diabetic retinopathy, prior or present history of malignant tumor, family history of tumor (two or more of the immediate family members within three generations have prior or present history of tumor), tumor markers exceeding the upper limit of the normal range for two consecutive times; 12. Those who are highly allergic, or have a history of allergy to protein drugs, or the researcher suspects that the subject may be allergic to the tested drugs and excipients of this study; 13. Have participated in clinical trials of any drug or medical device within the 3 months prior to screening (except those who have signed ICF only and have not received any drug or device intervention)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Safety assessment;height; | — |
Countries
China
Contacts
Public ContactXiumin Wang
Shanghai Children's Medical Center
Outcome results
None listed