A Study of PEG-somatropin Injection to Treat Children of Turner Syndrome

GeneScience Pharmaceuticals (GenSci)

Status and phase: Unknown; Phase 2

Conditions: Turner Syndrome

Treatments: Other: Non-treatment control group; Biological: PEG-rhGH high dose; Biological: PEG-rhGH low dose

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT03189160

GenSci 032 CT

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 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.

Enrollment: 180 estimated patients

Sex: Female

Ages: 2 to 18 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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180 participants in 3 patient groups

PEG-rhGH low dose

Experimental group

Description:

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

Treatment:

Other: Non-treatment control group

Biological: PEG-rhGH high dose

Biological: PEG-rhGH low dose

PEG-rhGH high dose

Experimental group

Description:

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

Treatment:

Other: Non-treatment control group

Biological: PEG-rhGH high dose

Biological: PEG-rhGH low dose

Non-treatment control group

No Intervention group