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

Turner Syndrome Clinical Trial

Official title:

A Phase 2 Study of Pegylated Recombinant Human Growth Hormone Injection to Treat Children of Turner Syndrome

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03189160
• Other study ID #: GenSci 032 CT
• Status: Recruiting
• Phase: Phase 2
• First received: June 13, 2017
• Last updated: December 10, 2017
• Start date: March 2016

Study information

• Verified date: June 2017
• Source: GeneScience Pharmaceuticals Co., Ltd.
• Contact: Xiaohua Feng
• Phone: 0431-85170552
• Email: fengxiaohua[@]gensci-china.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 180
• Est. primary completion date: March 2018
• Accepts healthy volunteers: No
• Gender: Female
• Age group: 2 Years to 18 Years

Eligibility

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.

Related Conditions & MeSH terms

• Gonadal Dysgenesis
• Primary Ovarian Insufficiency
• Syndrome
• Turner Syndrome

Intervention

Biological:
• PEG-rhGH low dose
PEG-rhGH Injection 0.1 mg/kg/w by subcutaneous injection for 52 weeks.
• PEG-rhGH high dose
PEG-rhGH Injection 0.2 mg/kg/w by subcutaneous injection for 52 weeks.

Other:
• Non-treatment control group

Locations

Country	Name	City	State
China	Beijing Children's Hospital,Capital Medical University	Beijing
China	First Hospital of Jilin University	Changchun	Jilin
China	The Children's Hospital of Zhejiang University School of Medicine	Hangzhou	Zhejiang
China	Jiangxi Provincial Children's Hospital	Nanchang	Jiangxi
China	The First Affiliated Hospital with Nanjing Medical University	Nanjing	Jiangsu
China	Children's Hospital of Fudan University	Shanghai
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

Sponsors (10)

Lead Sponsor

Collaborator

GeneScience Pharmaceuticals Co., Ltd.

Affiliated Hospital of Jiangnan University, Beijing Children's Hospital, Children's Hospital of Fudan University, First Hospital of Jilin University, Jiangxi Province Children's Hospital, Shanghai Children's Hospital, The Children's Hospital of Zhejiang University School of Medicine, The First Affiliated Hospital with Nanjing Medical University, Xinhua Hospital, Shanghai Jiao Tong University School of Medicine

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change of height standard deviation score before and after treatment (?HT SDS)	The change of height standard deviation score of chronological age before and after treatment	52 weeks
Secondary	Height Velocity	Annual growth rate at the end of treatment	52 weeks
Secondary	?BA/?CA	Bone maturation (changes of bone age/ chang of chronological age)	52 weeks
Secondary	IGF-1(Insulin-like growth factor 1) SDS		52 weeks