Turner Syndrome
Conditions
Brief summary
This study aims to explore the optimal dose of pegylated recombinant human growth hormone (PEG-rhGH) injection to treat children of Turner syndrome (TS), preliminarily evaluate its safety and efficacy and provide scientific and reliable evidence for the medication dosage in Phase 3 clinical trial.
Interventions
BIOLOGICALPEG-rhGH low dose
PEG-rhGH Injection 0.1 mg/kg/w by subcutaneous injection for 52 weeks.
BIOLOGICALPEG-rhGH high dose
PEG-rhGH Injection 0.2 mg/kg/w by subcutaneous injection for 52 weeks.
OTHERNon-treatment control group
Sponsors
Beijing Children's Hospital
The First Affiliated Hospital with Nanjing Medical University
Shanghai Children's Hospital
Children's Hospital of Fudan University
Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
The First Hospital of Jilin University
Jiangxi Province Children's Hospital
Affiliated Hospital of Jiangnan University
The Children's Hospital of Zhejiang University School of Medicine
Changchun GeneScience Pharmaceutical Co., Ltd.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
FEMALE
Age
2 Years to 18 Years
Healthy volunteers
No
Inclusion criteria
* Female * Bone age \<12 years * Karyotype: 45, X; 45, X / 46, XXqi; 45, X / 46, XXr; 45, X / 46, XX; 46, XXqi; 46, XXpi; 45, X / 47, XXX; 46, XXp-; 45, X / 46, XXp-; 46, XXq-; 45X / 46, XXq-; 45, X / 46, XX / 47, XXX, etc. (count 50 or more cells); * Facial appearance and abnormalities: Patients with at least one of the following signs, which include but are not limited to facial pigmented nevus, short neck, webbed neck, low posterior hairline, low-set ears, micrognathia, high-voulted arch, shield-like chest, cubitus valgus, genu valgum, short 4th and 5th metacarpals, nail dysplasia, scoliosis, ptosis and strabismus, cardiovascular abnormalities (such as aortic stenosis, bicuspid aortic valve and hypertension), reproductive abnormalities (such as primary gonadal dysfunction), renal abnormalities, thyroid hypofunction, middle ear lesion, etc. * Short stature: height below -2.5SD of the mean height of the same age and gender. * Pre-pubertal (Tanner Stage I ) patients * No history of growth hormone treatment * The subject and his/her guardian sign the informed consent (if the subject is incapable to sign the informed consent, his/her legal guardian shall sign the name of the subject instead)
Exclusion criteria
* Subjects with abnormal liver and kidney functions (ALT \> upper limit of normal value; Cr \> upper limit of normal value) * Subjects positive for anti-HBc, HbsAg or HbeAg in Hepatitis B virus tests; * Subjects with highly allergic constitution or allergy to proteins or investigational product or its excipient * Subjects with systemic chronic disease and immune deficiency * Patients diagnosed with tumor * For patients whose tumor markers exceeding normal range in combination with other information, considering as potential high risks of tumor, they may be excluded from the treatment. * Patients with mental disease * Subjects with impaired glucose regulation (IGR) (including impaired fasting glucose (IFG) and/or impaired glucose tolerance (IGT) ) or diabetes * Subjects who took part in other clinical trials within 3 months * Subjects who received medicines which may interfere GH secretion or GH function, or other hormones within 3 months (such as sex steroids, glucocorticoids, etc.) * Other conditions which are unsuitable for this study in the opinion of the investigator.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change of height standard deviation score before and after treatment (ΔHT SDS) | 52 weeks | The change of height standard deviation score of chronological age before and after treatment |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Height Velocity | 52 weeks | Annual growth rate at the end of treatment |
| ΔBA/ΔCA | 52 weeks | Bone maturation (changes of bone age/ chang of chronological age) |
| IGF-1(Insulin-like growth factor 1) SDS | 52 weeks | — |
Countries
China
Contacts
Primary ContactXiaohua Feng
Outcome results
None listed