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

Phase 3

• Conditions: Dwarfism
• Interventions: Other: Negative control

• Registration Number: NCT03635580
• Lead Sponsor: Changchun GeneScience Pharmaceutical Co., Ltd.

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

Detailed Description

Not available

Study Timeline

• Study Start: 2018-06-11
• Status Verified: 2018-08
• Study First Submitted: 2018-08-15
• Study First Submitted QC: 2018-08-15
• Last Update Submitted: 2018-08-15
• Study First Posted: 2018-08-17
• Last Update Posted: 2018-08-17
• Primary Completion: 2021-10
• Study Completion: 2021-10-30

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 480

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
rhGH/Jintropin AQ	Negative control	Jintropin AQ, injection, 30IU/10mg/3ml/cartridge, 0.05mg /kg/d in phase 1 and 0.05-0.07mg/kg/d in phase 2.

Primary Outcome Measures

Name	Time	Method
ΔHtSDSca (The change of height standard deviation score of chronological age before and after treatment)	Baseline, 4,13,26,39,52,65,78,91,104 weeks after initiating treatment, 52 weeks in phase 1; 2 years in phase 2.	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

Name	Time	Method
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.
ΔIGF-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.
ΔHT (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.
△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.

Trial Locations

Locations (9)

Henan Children's Hospital; 🇨🇳 Zhengzhou, Henan, China

Jiangsu Province Hospital; 🇨🇳 Nanjing, Jiangsu, China

The First Affiliated Hospital, Sun Yat-sen University; 🇨🇳 Guangzhou, Guangdong, China

Children's Hospital of Shanghai; 🇨🇳 Shanghai, China

The Children's Hospital, Zhejiang University School of Medicine; 🇨🇳 Hangzhou, Zhejiang, China

Children's Hospital of Shanxi; 🇨🇳 Taiyuan, Shanxi, China

Children's Hospital of Fudan University; 🇨🇳 Shanghai, China

The Second Affiliated Hospital of Xi'an Jiaotong University; 🇨🇳 Xi'an, Shanxi, China

The First Hospital of Jilin University; 🇨🇳 Changchun, Jilin, China