A Study of PEG-somatropin in the Treatment of Children With Idiopathic Short Stature

• Status: Unknown status
• Trial end date: 1969-12-31
• Target enrollment: 0
• Participant gender: All

Summary

This study aims to explore the optimal dose of pegylated recombinant human growth hormone (PEG-rhGH) injection to treat children with idiopathic short stature (ISS), evaluate its safety and efficacy, and provide scientific and reliable evidence for the medication dosage in Phase III clinical study.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: GeneScience Pharmaceuticals Co., Ltd.
• Collaborators: Affiliated Hospital of Jiangnan University; Children's Hospital of Fudan University; First Hospital of Jilin University; Shanghai Children's Hospital; The Children's Hospital of Zhejiang University School of Medicine; The First Affiliated Hospital with Nanjing Medical University; The First Hospital of Jilin University; Tongji Hospital

Criteria

Inclusion Criteria:

• Boys are between 4 and 9 years of age and girls are between 4 and 8 years of age.

• Height <-2 SD for chronological age.

• Growth velocity<5.0 cm/yr.

• GH peak concentration ≥10.0 ng/mL in two different stimulation tests.

• The difference of bone age (BA) and chronological age (CA) is within -2 to +2.

• IGF-1 concentration is between -2 SDS to +2 SDS.

• Prepubertal Status(Tanner Stage I).

• Birth weight within the normal range.

• Growth hormone treatment-naive.

• Subjects are willing and able to cooperate to complete scheduled visits, treatment plans and laboratory tests and other procedures, to sign informed consent.

Exclusion Criteria:

• Subjects with abnormal liver and kidney functions (ALT > upper limit of normal value; Cr > upper limit of normal value).

• Subjects are positive for anti-HBc, HbsAg or HbeAg in Hepatitis B virus tests.

• Subjects with known highly allergic constitution or allergy to investigational product or its excipient.

• Subjects with systemic chronic disease and immune deficiency.

• Patients diagnosed with tumor.

• Patients with mental disease.

• Patients with other types of abnormal growth and development.

1. Growth hormone deficiency (GHD) (confirmed by GH stimulation test);

2. Turner syndrome (confirmed by karyotype test of girls);

3. Noonan syndrome (hypertelorism, pectus carinatum, hypophrenia, frequently with skin disease and congenital heart disease, missense mutation of the protein tyrosine phosphatase, non-receptor type 11 (PTPN11) gene on chromosome 12 for half of the patients, for both male and female patients);

4. Laron sydrome (confirmed by IGF-1 generation test);

5. Small for gestational age ( the birth height or weight is below the tenth percentile or 2 SD, with catch-up growth uncompleted at 2 years old).

• Growth disorders caused by malnutrition or hypothyroidism (thyroid function test).

• Congenital skeletal abnormalities or scoliosis, claudication.

• Subjects with impaired glucose regulation (IGR) (including impaired fasting glucose (IFG) and/or impaired glucose tolerance (IGT) ) or diabetes).

• Subjects with abnormal electrolyte, blood gas analysis (vein), creatine kinase.

• Subjects who took part in other clinical trials within 3 months.

• Subjects who received medications which may interfere GH secretion or GH function, or other hormones within 3 months (such as sex steroids, glucocorticoids, etc.).

• For patients with potential high tumor risks such as tumor markers exceed normal range and some other relative information, they may be excluded from the treatment.

• Other conditions which is inappropriate for this study in the opinion of the investigator.