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

Primary Purpose

Dwarfism

Status
Unknown status
Phase
Phase 2
Locations
China
Study Type
Interventional
Intervention
Jintrolong® low dose group
Jintrolong® high dose 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 Dwarfism

Eligibility Criteria

4 Years - 9 Years (Child)All SexesDoes not accept 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.

Sites / Locations

  • Tongji Hospital of Tongji Medical College of Huazhong University of Science and TechnologyRecruiting
  • The First Affiated Hospital of Nanjing Medical UniversityRecruiting
  • Affiliated Hospital of Jiangnan UniversityRecruiting
  • The First Hospital of Jilin UniversityRecruiting
  • The Children's Hospital of Zhejiang University School of MedicineRecruiting
  • Shanghai Children's Hospital of Fudan UniversityRecruiting
  • Shanghai Children's HospitalRecruiting

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

No Intervention

Arm Label

Jintrolong® low dose group

Jintrolong® high dose group

Negative 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.

Untreated Control Group

Outcomes

Primary Outcome Measures

Change in Height Standard Deviation Score for Chronological Age (ΔHtSDSCA)
Change in Height Standard Deviation Score for Chronological Age (ΔHtSDSCA) from Baseline to 52 weeks；ΔHtSDSCA=(height Yx - reference mean for CA Yx) / reference SD for CA Yx (Yx refers to the height value at particular timepoint x)

Secondary Outcome Measures

Change in Annualized Height Velocity
Annualized Height Velocity=12×(Height Yx - Height at Baseline)/(Date of Yx - Date of Baseline) (Yx refers to the height value at particular timepoint x)
Change in Bone Maturation
Bone Maturation=(BA Yx-BA at Baseline)/(Date of Yx - Date of Baseline) (Yx refers to the BA value at particular timepoint x)
Change in IGF-1 Standard Deviation Score (IGF-1 SDS)
GF-1 SDS=(IGF-1 Yx - reference mean for CA Yx) / reference SD for CA Yx (Yx refers to the IGF-1 value at particular timepoint x)
IGF-1/IGFBP-3 molar ratio at 52 weeks
IGF-1/IGFBP-3 molar ration=[IGF-1(ng/ml)/7.6]/[IGFBP-3 (ng/ml)/25.75]

Full Information

First Posted
July 14, 2017
Last Updated
July 17, 2017
Sponsor
Changchun GeneScience Pharmaceutical Co., Ltd.
Collaborators
Tongji Hospital, The First Hospital of Jilin University, Affiliated Hospital of Jiangnan University, The First Affiliated Hospital with Nanjing Medical University, Shanghai Children's Hospital, The Children's Hospital of Zhejiang University School of Medicine, Children's Hospital of Fudan University
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1. Study Identification

Unique Protocol Identification Number
NCT03221088
Brief Title
A Study of PEG-somatropin in the Treatment of Children With Idiopathic Short Stature
Official Title
Pegylated Somatropin (PEG Somatropin) in the Treatment of Children With Idiopathic Short Stature: A Controlled, Prospective, Randomized, Multicenter Phase-II Study With An Untreated Control Group.
Study Type
Interventional

2. Study Status

Record Verification Date
July 2017
Overall Recruitment Status
Unknown status
Study Start Date
June 2015 (Actual)
Primary Completion Date
December 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
Tongji Hospital, The First Hospital of Jilin University, Affiliated Hospital of Jiangnan University, The First Affiliated Hospital with Nanjing Medical University, Shanghai Children's Hospital, The Children's Hospital of Zhejiang University School of Medicine, Children's Hospital of Fudan University

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device 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 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.

6. Conditions and Keywords

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

7. Study Design

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

8. Arms, Groups, and Interventions

Arm Title
Jintrolong® low dose group
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
Jintrolong® high dose group
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
Negative control group
Arm Type
No Intervention
Arm Description
Untreated Control Group
Intervention Type
Drug
Intervention Name(s)
Jintrolong® low dose group
Other Intervention Name(s)
PEG-somatropin
Intervention Description
PEG-somatropin 0.1mg/kg/wk by weekly subcutaneous injection for 52 weeks.
Intervention Type
Drug
Intervention Name(s)
Jintrolong® high dose group
Other Intervention Name(s)
PEG-somatropin
Intervention Description
PEG-somatropin 0.2 mg/kg/wk by weekly subcutaneous injection for 52 weeks.
Primary Outcome Measure Information:
Title
Change in Height Standard Deviation Score for Chronological Age (ΔHtSDSCA)
Description
Change in Height Standard Deviation Score for Chronological Age (ΔHtSDSCA) from Baseline to 52 weeks；ΔHtSDSCA=(height Yx - reference mean for CA Yx) / reference SD for CA Yx (Yx refers to the height value at particular timepoint x)
Time Frame
Baseline，52 weeks
Secondary Outcome Measure Information:
Title
Change in Annualized Height Velocity
Description
Annualized Height Velocity=12×(Height Yx - Height at Baseline)/(Date of Yx - Date of Baseline) (Yx refers to the height value at particular timepoint x)
Time Frame
Baseline，52 weeks
Title
Change in Bone Maturation
Description
Bone Maturation=(BA Yx-BA at Baseline)/(Date of Yx - Date of Baseline) (Yx refers to the BA value at particular timepoint x)
Time Frame
Baseline，52 weeks
Title
Change in IGF-1 Standard Deviation Score (IGF-1 SDS)
Description
GF-1 SDS=(IGF-1 Yx - reference mean for CA Yx) / reference SD for CA Yx (Yx refers to the IGF-1 value at particular timepoint x)
Time Frame
Baseline，52 weeks
Title
IGF-1/IGFBP-3 molar ratio at 52 weeks
Description
IGF-1/IGFBP-3 molar ration=[IGF-1(ng/ml)/7.6]/[IGFBP-3 (ng/ml)/25.75]
Time Frame
Baseline，52 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
4 Years
Maximum Age & Unit of Time
9 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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. Growth hormone deficiency (GHD) (confirmed by GH stimulation test); Turner syndrome (confirmed by karyotype test of girls); 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); Laron sydrome (confirmed by IGF-1 generation test); 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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Yanlin Chen
Phone
+86-60871786-8197
Email
chenyanlin@gensci-china.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Xiaoping Luo
Organizational Affiliation
Department of Pediatrics of Tongji Hospital of Tongji Medical College, Huazhong University of Science and Technology
Official's Role
Principal Investigator
Facility Information:
Facility Name
Tongji Hospital of Tongji Medical College of Huazhong University of Science and Technology
City
Wuhan
State/Province
Hubei
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Xiaoping Luo, PhD
Facility Name
The First Affiated Hospital of 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, MD
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, MD
Facility Name
The 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, MD
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
Junfen Fu, MD
Facility Name
Shanghai 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, MD
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, MD

12. IPD Sharing Statement

Learn more about this trial

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

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