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

Phase 2

• Conditions: Turner Syndrome
• Interventions: Biological: PEG-rhGH low dose; Biological: PEG-rhGH high dose; Other: Non-treatment control group

• Registration Number: NCT03189160
• Lead Sponsor: Changchun GeneScience Pharmaceutical Co., Ltd.

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2016-03
• Status Verified: 2017-06
• Study First Submitted: 2017-06-13
• Study First Submitted QC: 2017-06-14
• Study First Posted: 2017-06-16
• Last Update Submitted: 2017-12-10
• Last Update Posted: 2017-12-12
• Primary Completion: 2018-03

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: Female
• Target Recruitment: 180

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
PEG-rhGH high dose	Non-treatment control group	PEG-rhGH Injection (27IU/4.5mg/0.5ml/bottle) 0.2 mg/kg/w by subcutaneous injection for 52 weeks.
PEG-rhGH low dose	Non-treatment control group	PEG-rhGH Injection (27IU/4.5mg/0.5ml/bottle) 0.1 mg/kg/w by subcutaneous injection for 52 weeks.
PEG-rhGH low dose	PEG-rhGH low dose	PEG-rhGH Injection (27IU/4.5mg/0.5ml/bottle) 0.1 mg/kg/w by subcutaneous injection for 52 weeks.
PEG-rhGH low dose	PEG-rhGH high dose	PEG-rhGH Injection (27IU/4.5mg/0.5ml/bottle) 0.1 mg/kg/w by subcutaneous injection for 52 weeks.
PEG-rhGH high dose	PEG-rhGH high dose	PEG-rhGH Injection (27IU/4.5mg/0.5ml/bottle) 0.2 mg/kg/w by subcutaneous injection for 52 weeks.
PEG-rhGH high dose	PEG-rhGH low dose	PEG-rhGH Injection (27IU/4.5mg/0.5ml/bottle) 0.2 mg/kg/w by subcutaneous injection for 52 weeks.

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
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

Trial Locations

Locations (9)

The First Affiliated Hospital with Nanjing Medical University; 🇨🇳 Nanjing, Jiangsu, China

Jiangxi Provincial Children's Hospital; 🇨🇳 Nanchang, Jiangxi, China

Beijing Children's Hospital,Capital Medical University; 🇨🇳 Beijing, China

Shanghai Children's Hospital; 🇨🇳 Shanghai, China

Xinhua Hospital of Shanghai Jiao Tong University School of Medicine; 🇨🇳 Shanghai, China

Affiliated Hospital of Jiangnan University; 🇨🇳 Wuxi, Jiangsu, China

First Hospital of Jilin University; 🇨🇳 Changchun, Jilin, China

Children's Hospital of Fudan University; 🇨🇳 Shanghai, China

The Children's Hospital of Zhejiang University School of Medicine; 🇨🇳 Hangzhou, Zhejiang, China