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

GeneScience Pharmaceuticals (GenSci)

Status and phase

Unknown

Phase 3

Conditions

Dwarfism

Treatments

Other: Negative control

Study type

Interventional

Funder types

Other

Industry

Identifiers

NCT03635580

GenSci 046 CT

Details and patient eligibility

About

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

Enrollment

480 estimated patients

Sex

All

Ages

4 to 10 years old

Volunteers

No Healthy Volunteers

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.