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

GeneScience Pharmaceuticals (GenSci)

Status and phase: Unknown; Phase 2

Conditions: Dwarfism

Treatments: Drug: Jintrolong® high dose group; Drug: Jintrolong® low dose group

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT03221088

GenSci 033 CT-one year

Details and patient eligibility

About

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.

Enrollment: 360 estimated patients

Sex: All

Ages: 4 to 9 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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360 participants in 3 patient groups

Jintrolong® low dose group

Experimental group

Description:

PEG-rhGH Injection (27IU/4.5mg/0.5ml/bottle) 0.1 mg/kg/w by subcutaneous injection for 52 weeks.

Treatment:

Drug: Jintrolong® low dose group

Drug: Jintrolong® high dose group

Jintrolong® high dose group

Experimental group

Description:

PEG-rhGH Injection (27IU/4.5mg/0.5ml/bottle) 0.2 mg/kg/w by subcutaneous injection for 52 weeks.

Treatment:

Drug: Jintrolong® low dose group

Drug: Jintrolong® high dose group

Negative control group

No Intervention group

Description:

Untreated Control Group