Back to resultsEfficacy and Safety of rhGH (Jintropin®) in Pediatric Participants With ISS
Primary Purpose
Dwarfism
Status
Unknown status
Phase
Phase 3
Locations
China
Study Type
Interventional
Intervention
Negative control
Sponsored by
About this trial
This is an interventional treatment trial for Dwarfism
Eligibility Criteria
Inclusion Criteria:
- Boys are between 4 and 10 years old age and girls are between 4 and 9 years old age;
- Height <-2.25 SD (Standard deviation) for chronological age;
- GH (Growth hormone) peak concentration ≥10.0 ng/mL in GH stimulation tests;
- The bone age (BA) ≤chronological age (CA)+6 months;
- Prepubertal Status (Tanner Stage I);
- Birth weight within the normal range;
- Growth hormone treatment-naive;
- Participants are willing and able to cooperate to complete scheduled visits, treatment plans and laboratory tests and other procedures, to sign informed consent.
Exclusion Criteria:
- Participants with abnormal liver and kidney functions (ALT > upper limit 1.5 times of normal value; Cr > upper limit of normal value);
- Participants are positive for anti-HBc, HbsAg or HbeAg in Hepatitis B virus tests;
- Participants with known highly allergic constitution or allergy to investigational product or its excipient;
- Participants with systemic chronic disease and immune deficiency;
- Participants diagnosed with tumor, or with potential high tumor risks such as tumor markers exceed normal range and some other relative information may be excluded from the treatment;
- Participants with mental disease;
Participants 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 participants, for both male and female participants);
- Laron syndrome (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).
- Participants with impaired glucose regulation (IGR) (including impaired fasting glucose (IFG) and/or impaired glucose tolerance (IGT) ) or diabetes;
- BMI (Body mass index) ≥22kg/m²;
- Congenital skeletal abnormalities or scoliosis, claudication;
- Participants who took part in other clinical trials within 3 months;
- Participants who received medications which may interfere GH secretion or GH function, or other hormones within 3 months (such as sex steroids, glucocorticoids, etc.);
- Other conditions which is inappropriate for this study in the opinion of the investigator.
Sites / Locations
- The First Affiliated Hospital, Sun Yat-sen UniversityRecruiting
- Henan Children's HospitalRecruiting
- Jiangsu Province HospitalRecruiting
- The First Hospital of Jilin UniversityRecruiting
- Children's Hospital of ShanxiRecruiting
- The Second Affiliated Hospital of Xi'an Jiaotong UniversityRecruiting
- The Children's Hospital, Zhejiang University School of MedicineRecruiting
- Children's Hospital of Fudan UniversityRecruiting
- Children's Hospital of ShanghaiRecruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
rhGH/Jintropin AQ
Arm Description
Jintropin AQ, injection, 30IU/10mg/3ml/cartridge, 0.05mg /kg/d in phase 1 and 0.05-0.07mg/kg/d in phase 2.
Outcomes
Primary Outcome Measures
ΔHtSDSca (The change of height standard deviation score of chronological age before and after treatment)
ΔHtSDSca was the difference of HtSDSca before and after treatment, and HtSDSca was calculated by dividing the difference between the actual height of a patient and the mean height of the population for that chronological age by the standard deviation (SD) of the height of the population for that chronological age.
Secondary Outcome Measures
ΔHT (Change of height)
△BA/CA (bone age change / chronological age)
Yearly growth velocity
ΔIGF-1 SDS (Change of insulin-like growth factor 1 standard deviation score)
Full Information
NCT ID
NCT03635580
First Posted
August 15, 2018
Last Updated
August 15, 2018
Sponsor
Changchun GeneScience Pharmaceutical Co., Ltd.
Collaborators
The Children's Hospital of Zhejiang University School of Medicine, First Affiliated Hospital, Sun Yat-Sen University, Second Affiliated Hospital of Xi'an Jiaotong University, Henan Provincial People's Hospital, Children's Hospital of Fudan University, The First Affiliated Hospital with Nanjing Medical University, Shanxi Provincial Maternity and Children's Hospital, The First Hospital of Jilin University, Shanghai Children's Hospital
1. Study Identification
Unique Protocol Identification Number
NCT03635580
Brief Title
Efficacy and Safety of rhGH (Jintropin®) in Pediatric Participants With ISS
Official Title
Phase 2 Study of Efficacy and Safety of rhGH (Jintropin®) in Pediatric Participants With ISS (Idiopathic Short Stature): a 52-week Multicenter, Open-label, Randomized, Negative Controlled Study Followed by a 52-week Open-label Period
Study Type
Interventional
2. Study Status
Record Verification Date
August 2018
Overall Recruitment Status
Unknown status
Study Start Date
June 11, 2018 (Actual)
Primary Completion Date
October 2021 (Anticipated)
Study Completion Date
October 30, 2021 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Changchun GeneScience Pharmaceutical Co., Ltd.
Collaborators
The Children's Hospital of Zhejiang University School of Medicine, First Affiliated Hospital, Sun Yat-Sen University, Second Affiliated Hospital of Xi'an Jiaotong University, Henan Provincial People's Hospital, Children's Hospital of Fudan University, The First Affiliated Hospital with Nanjing Medical University, Shanxi Provincial Maternity and Children's Hospital, The First Hospital of Jilin University, Shanghai Children's Hospital
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
5. Study Description
Brief Summary
Phase 1: To evaluate the safety and efficacy of 0.05mg/kg/d of rhGH (Jintropin®) in the treatment of children with idiopathic short stature (ISS) in 52 weeks.
Phase 2: To evaluate the safety and efficacy of rhGH (Jintropin®) in the treatment of children with ISS in 2 years
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 3
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
480 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
rhGH/Jintropin AQ
Arm Type
Experimental
Arm Description
Jintropin AQ, injection, 30IU/10mg/3ml/cartridge, 0.05mg /kg/d in phase 1 and 0.05-0.07mg/kg/d in phase 2.
Intervention Type
Other
Intervention Name(s)
Negative control
Intervention Description
Untreated-control group
Primary Outcome Measure Information:
Title
ΔHtSDSca (The change of height standard deviation score of chronological age before and after treatment)
Description
ΔHtSDSca was the difference of HtSDSca before and after treatment, and HtSDSca was calculated by dividing the difference between the actual height of a patient and the mean height of the population for that chronological age by the standard deviation (SD) of the height of the population for that chronological age.
Time Frame
Baseline, 4,13,26,39,52,65,78,91,104 weeks after initiating treatment, 52 weeks in phase 1; 2 years in phase 2.
Secondary Outcome Measure Information:
Title
ΔHT (Change of height)
Time Frame
Baseline, 4,13,26,39,52,65,78,91,104 weeks after initiating treatment, 52 weeks in phase 1; 2 years in phase 2.
Title
△BA/CA (bone age change / chronological age)
Time Frame
Baseline, 4,13,26,39,52,65,78,91,104 weeks after initiating treatment, 52 weeks in phase 1; 2 years in phase 2.
Title
Yearly growth velocity
Time Frame
Baseline, 4,13,26,39,52,65,78,91,104 weeks after initiating treatment, 52 weeks in phase 1; 2 years in phase 2.
Title
ΔIGF-1 SDS (Change of insulin-like growth factor 1 standard deviation score)
Time Frame
Baseline, 4,13,26,39,52,65,78,91,104 weeks after initiating treatment, 52 weeks in phase 1; 2 years in phase 2.
10. Eligibility
Sex
All
Minimum Age & Unit of Time
4 Years
Maximum Age & Unit of Time
10 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Boys are between 4 and 10 years old age and girls are between 4 and 9 years old age;
Height <-2.25 SD (Standard deviation) for chronological age;
GH (Growth hormone) peak concentration ≥10.0 ng/mL in GH stimulation tests;
The bone age (BA) ≤chronological age (CA)+6 months;
Prepubertal Status (Tanner Stage I);
Birth weight within the normal range;
Growth hormone treatment-naive;
Participants are willing and able to cooperate to complete scheduled visits, treatment plans and laboratory tests and other procedures, to sign informed consent.
Exclusion Criteria:
Participants with abnormal liver and kidney functions (ALT > upper limit 1.5 times of normal value; Cr > upper limit of normal value);
Participants are positive for anti-HBc, HbsAg or HbeAg in Hepatitis B virus tests;
Participants with known highly allergic constitution or allergy to investigational product or its excipient;
Participants with systemic chronic disease and immune deficiency;
Participants diagnosed with tumor, or with potential high tumor risks such as tumor markers exceed normal range and some other relative information may be excluded from the treatment;
Participants with mental disease;
Participants 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 participants, for both male and female participants);
Laron syndrome (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).
Participants with impaired glucose regulation (IGR) (including impaired fasting glucose (IFG) and/or impaired glucose tolerance (IGT) ) or diabetes;
BMI (Body mass index) ≥22kg/m²;
Congenital skeletal abnormalities or scoliosis, claudication;
Participants who took part in other clinical trials within 3 months;
Participants who received medications which may interfere GH secretion or GH function, or other hormones within 3 months (such as sex steroids, glucocorticoids, etc.);
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
Xiaohua Feng
Phone
0431-85170552
Email
fengxiaohua@gensci-china.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Junfen Fu, Doctor
Organizational Affiliation
The Children's Hospital of Zhejiang University School of Medicine
Official's Role
Principal Investigator
Facility Information:
Facility Name
The First Affiliated Hospital, Sun Yat-sen University
City
Guangzhou
State/Province
Guangdong
Country
China
Individual Site Status
Recruiting
Facility Name
Henan Children's Hospital
City
Zhengzhou
State/Province
Henan
Country
China
Individual Site Status
Recruiting
Facility Name
Jiangsu Province Hospital
City
Nanjing
State/Province
Jiangsu
Country
China
Individual Site Status
Recruiting
Facility Name
The First Hospital of Jilin University
City
Changchun
State/Province
Jilin
Country
China
Individual Site Status
Recruiting
Facility Name
Children's Hospital of Shanxi
City
Taiyuan
State/Province
Shanxi
Country
China
Individual Site Status
Recruiting
Facility Name
The Second Affiliated Hospital of Xi'an Jiaotong University
City
Xi'an
State/Province
Shanxi
Country
China
Individual Site Status
Recruiting
Facility Name
The Children's Hospital, Zhejiang University School of Medicine
City
Hangzhou
State/Province
Zhejiang
Country
China
Individual Site Status
Recruiting
Facility Name
Children's Hospital of Fudan University
City
Shanghai
Country
China
Individual Site Status
Recruiting
Facility Name
Children's Hospital of Shanghai
City
Shanghai
Country
China
Individual Site Status
Recruiting
12. IPD Sharing Statement
Citations:
PubMed Identifier
35573994
Citation
Yuan J, Fu J, Wei H, Zhang G, Xiao Y, Du H, Gu W, Li Y, Chen L, Luo F, Zhong Y, Gong H. A Randomized Controlled Phase 3 Study on the Efficacy and Safety of Recombinant Human Growth Hormone in Children With Idiopathic Short Stature. Front Endocrinol (Lausanne). 2022 Apr 29;13:864908. doi: 10.3389/fendo.2022.864908. eCollection 2022.
Results Reference
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Efficacy and Safety of rhGH (Jintropin®) in Pediatric Participants With ISS
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