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

Dwarfism Clinical Trial

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.

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03221088
• Other study ID #: GenSci 033 CT-one year
• Status: Recruiting
• Phase: Phase 2
• First received: July 14, 2017
• Last updated: July 17, 2017
• Start date: June 2015

Study information

• Verified date: July 2017
• Source: GeneScience Pharmaceuticals Co., Ltd.
• Contact: Yanlin Chen
• Phone: +86-60871786-8197
• Email: chenyanlin[@]gensci-china.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 360
• Est. primary completion date: December 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 4 Years to 9 Years

Eligibility

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.

Related Conditions & MeSH terms

• Dwarfism

Intervention

Drug:
• Jintrolong® low dose group
PEG-somatropin 0.1mg/kg/wk by weekly subcutaneous injection for 52 weeks.
• Jintrolong® high dose group
PEG-somatropin 0.2 mg/kg/wk by weekly subcutaneous injection for 52 weeks.

Locations

Country	Name	City	State
China	The First Hospital of Jilin University	Changchun	Jilin
China	The Children's Hospital of Zhejiang University School of Medicine	Hangzhou	Zhejiang
China	The First Affiated Hospital of Nanjing Medical University	Nanjing	Jiangsu
China	Shanghai Children's Hospital	Shanghai
China	Shanghai Children's Hospital of Fudan University	Shanghai
China	Tongji Hospital of Tongji Medical College of Huazhong University of Science and Technology	Wuhan	Hubei
China	Affiliated Hospital of Jiangnan University	Wuxi	Jiangsu

Sponsors (8)

Lead Sponsor	Collaborator
GeneScience Pharmaceuticals Co., Ltd.	Affiliated Hospital of Jiangnan University, Children's Hospital of Fudan University, First Hospital of Jilin University, Shanghai Children's Hospital, The Children's Hospital of Zhejiang University School of Medicine, The First Affiliated Hospital with Nanjing Medical University, Tongji Hospital

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in Height Standard Deviation Score for Chronological Age (?HtSDSCA)	Change in Height Standard Deviation Score for Chronological Age (?HtSDSCA) from Baseline to 52 weeks;?HtSDSCA=(height Yx - reference mean for CA Yx) / reference SD for CA Yx (Yx refers to the height value at particular timepoint x)	Baseline,52 weeks
Secondary	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)	Baseline,52 weeks
Secondary	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)	Baseline,52 weeks
Secondary	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)	Baseline,52 weeks
Secondary	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]	Baseline,52 weeks