Efficacy and Safety of rhGH (Jintropin®) in Pediatric Participants With ISS

Dwarfism Clinical Trial

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03635580
• Other study ID #: GenSci 046 CT
• Status: Recruiting
• Phase: Phase 3
• Start date: June 11, 2018
• Est. completion date: October 30, 2021

Study information

• Verified date: August 2018
• 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

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

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 480
• Est. completion date: October 30, 2021
• Est. primary completion date: October 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 4 Years to 10 Years

Eligibility

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;

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 participants, for both male and female participants);

4. Laron syndrome (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);

6. 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.

Related Conditions & MeSH terms

• Dwarfism

Intervention

Other:
• Negative control
Untreated-control group

Locations

Country	Name	City	State
China	The First Hospital of Jilin University	Changchun	Jilin
China	The First Affiliated Hospital, Sun Yat-sen University	Guangzhou	Guangdong
China	The Children's Hospital, Zhejiang University School of Medicine	Hangzhou	Zhejiang
China	Jiangsu Province Hospital	Nanjing	Jiangsu
China	Children's Hospital of Fudan University	Shanghai
China	Children's Hospital of Shanghai	Shanghai
China	Children's Hospital of Shanxi	Taiyuan	Shanxi
China	The Second Affiliated Hospital of Xi'an Jiaotong University	Xi'an	Shanxi
China	Henan Children's Hospital	Zhengzhou	Henan

Sponsors (10)

Lead Sponsor

Collaborator

GeneScience Pharmaceuticals Co., Ltd.

Children's Hospital of Fudan University, Children's Hospital of Shanghai, Children’s hospital of Shanxi, First Affiliated Hospital, Sun Yat-Sen University, First Hospital of Jilin University, Henan Provincial Hospital, Second Affiliated Hospital of Xi'an Jiaotong University, The Children's Hospital of Zhejiang University School of Medicine, The First Affiliated Hospital with Nanjing Medical University

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	?HtSDSca (The change of height standard deviation score of chronological age before and after treatment)	?HtSDSca 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.	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	?HT (Change of height)		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	?BA/CA (bone age change / chronological age)		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	Yearly growth velocity		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	?IGF-1 SDS (Change of insulin-like growth factor 1 standard deviation score)		Baseline, 4,13,26,39,52,65,78,91,104 weeks after initiating treatment, 52 weeks in phase 1; 2 years in phase 2.