search
Back to results

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

Primary Purpose

Turner Syndrome

Status
Unknown status
Phase
Phase 2
Locations
China
Study Type
Interventional
Intervention
PEG-rhGH low dose
PEG-rhGH high dose
Non-treatment control group
Sponsored by
Changchun GeneScience Pharmaceutical Co., Ltd.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Turner Syndrome

Eligibility Criteria

2 Years - 18 Years (Child, Adult)FemaleDoes not accept 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.

Sites / Locations

  • The First Affiliated Hospital with Nanjing Medical UniversityRecruiting
  • Affiliated Hospital of Jiangnan UniversityRecruiting
  • Jiangxi Provincial Children's HospitalRecruiting
  • First Hospital of Jilin UniversityRecruiting
  • The Children's Hospital of Zhejiang University School of MedicineRecruiting
  • Beijing Children's Hospital,Capital Medical UniversityRecruiting
  • Children's Hospital of Fudan UniversityRecruiting
  • Shanghai Children's HospitalRecruiting
  • Xinhua Hospital of Shanghai Jiao Tong University School of MedicineRecruiting

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

No Intervention

Arm Label

PEG-rhGH low dose

PEG-rhGH high dose

Non-treatment control group

Arm Description

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

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

Outcomes

Primary Outcome Measures

Change of height standard deviation score before and after treatment (ΔHT SDS)
The change of height standard deviation score of chronological age before and after treatment

Secondary Outcome Measures

Height Velocity
Annual growth rate at the end of treatment
ΔBA/ΔCA
Bone maturation (changes of bone age/ chang of chronological age)
IGF-1(Insulin-like growth factor 1) SDS

Full Information

First Posted
June 13, 2017
Last Updated
December 10, 2017
Sponsor
Changchun GeneScience Pharmaceutical Co., Ltd.
Collaborators
Beijing Children's Hospital, The First Affiliated Hospital with Nanjing Medical University, Shanghai Children's Hospital, Children's Hospital of Fudan University, Xinhua Hospital, Shanghai Jiao Tong University School of Medicine, The First Hospital of Jilin University, Jiangxi Province Children's Hospital, Affiliated Hospital of Jiangnan University, The Children's Hospital of Zhejiang University School of Medicine
search

1. Study Identification

Unique Protocol Identification Number
NCT03189160
Brief Title
A Study of PEG-somatropin Injection to Treat Children of Turner Syndrome
Official Title
A Phase 2 Study of Pegylated Recombinant Human Growth Hormone Injection to Treat Children of Turner Syndrome
Study Type
Interventional

2. Study Status

Record Verification Date
June 2017
Overall Recruitment Status
Unknown status
Study Start Date
March 2016 (undefined)
Primary Completion Date
March 2018 (Anticipated)
Study Completion Date
undefined (undefined)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Changchun GeneScience Pharmaceutical Co., Ltd.
Collaborators
Beijing Children's Hospital, The First Affiliated Hospital with Nanjing Medical University, Shanghai Children's Hospital, Children's Hospital of Fudan University, Xinhua Hospital, Shanghai Jiao Tong University School of Medicine, The First Hospital of Jilin University, Jiangxi Province Children's Hospital, Affiliated Hospital of Jiangnan University, The Children's Hospital of Zhejiang University School of Medicine

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Turner Syndrome

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
180 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
PEG-rhGH low dose
Arm Type
Experimental
Arm Description
PEG-rhGH Injection (27IU/4.5mg/0.5ml/bottle) 0.1 mg/kg/w by subcutaneous injection for 52 weeks.
Arm Title
PEG-rhGH high dose
Arm Type
Experimental
Arm Description
PEG-rhGH Injection (27IU/4.5mg/0.5ml/bottle) 0.2 mg/kg/w by subcutaneous injection for 52 weeks.
Arm Title
Non-treatment control group
Arm Type
No Intervention
Intervention Type
Biological
Intervention Name(s)
PEG-rhGH low dose
Other Intervention Name(s)
Polyethylene Glycol Recombinant Human Somatropin Injection
Intervention Description
PEG-rhGH Injection 0.1 mg/kg/w by subcutaneous injection for 52 weeks.
Intervention Type
Biological
Intervention Name(s)
PEG-rhGH high dose
Other Intervention Name(s)
Polyethylene Glycol Recombinant Human Somatropin Injection
Intervention Description
PEG-rhGH Injection 0.2 mg/kg/w by subcutaneous injection for 52 weeks.
Intervention Type
Other
Intervention Name(s)
Non-treatment control group
Primary Outcome Measure Information:
Title
Change of height standard deviation score before and after treatment (ΔHT SDS)
Description
The change of height standard deviation score of chronological age before and after treatment
Time Frame
52 weeks
Secondary Outcome Measure Information:
Title
Height Velocity
Description
Annual growth rate at the end of treatment
Time Frame
52 weeks
Title
ΔBA/ΔCA
Description
Bone maturation (changes of bone age/ chang of chronological age)
Time Frame
52 weeks
Title
IGF-1(Insulin-like growth factor 1) SDS
Time Frame
52 weeks

10. Eligibility

Sex
Female
Minimum Age & Unit of Time
2 Years
Maximum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Xiaohua Feng
Phone
0431-85170552
Email
fengxiaohua@gensci-china.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Chunxiu Gong, PhD
Organizational Affiliation
Beijing Children's Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
The First Affiliated Hospital with Nanjing Medical University
City
Nanjing
State/Province
Jiangsu
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Yuhua Hu
Facility Name
Affiliated Hospital of Jiangnan University
City
Wuxi
State/Province
Jiangsu
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Zhuangjian Xu
Facility Name
Jiangxi Provincial Children's Hospital
City
Nanchang
State/Province
Jiangxi
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Yu Yang
Facility Name
First Hospital of Jilin University
City
Changchun
State/Province
Jilin
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Hongwei Du
Facility Name
The Children's Hospital of Zhejiang University School of Medicine
City
Hangzhou
State/Province
Zhejiang
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Junfeng Fu
Facility Name
Beijing Children's Hospital,Capital Medical University
City
Beijing
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Chunxiu Gong, PhD
Facility Name
Children's Hospital of Fudan University
City
Shanghai
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Feihong Luo
Facility Name
Shanghai Children's Hospital
City
Shanghai
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Pin Li
Facility Name
Xinhua Hospital of Shanghai Jiao Tong University School of Medicine
City
Shanghai
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Jun Ye

12. IPD Sharing Statement

Learn more about this trial

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

We'll reach out to this number within 24 hrs