Clinical trial NCT01495468: Phase III Clinical Study of Pegylated Somatropin (PEG Somatropin) to Treat Growth Hormone Deficiency Children
Official title: Pegylated Somatropin in the Treatment of Children With Growth Hormone Deficient：A Multicenter, Randomized, Open-label, Controlled Phase Ⅲ Clinical Trial
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Growth Hormone Deficiency
Keywords: pegylated somatropin
PEG-rhGH
growth hormone
Growth Hormone Deficiency
phase III study
Lead sponsor: Changchun GeneScience Pharmaceutical Co., Ltd.
Sponsor class: INDUSTRY
Collaborators: name: Tongji Hospital
name: First Affiliated Hospital, Sun Yat-Sen University
name: Capital Medical University
name: Children's Hospital of Fudan University
name: The First Hospital of Jilin University
First posted: 2011-12-20
Enrollment: 343
Sex: ALL
Minimum age (years): 8
Maximum age (years): 15
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This was a multicenter, randomized, open-label, parallel controlled phase III study, compared pegylated somatropin with Jintropin AQ (somatropin liquid injection, daily administration formulation).

All the subjects were randomized into two groups, test group (PEG somatorpin) and control group (Jintropin AQ), 200 children were enrolled in test group and 100 children were enrolled in control group. Considering the case loss during the clinical study, 20% of the patients were added in each of the group, that is 240 children were in test group and 120 children were in control group, totally 360 children were enrolled in the phase III clinical study.

Whole treatment were lasted for 6 months, 4 times of follow-up were carried out at the point of baseline, 1 month, 3 month and 6 month after treatment. The evaluation of the primary time point was 3 month and 6 month after treatment, if the treatment was less than 6 months, the evaluation would be made when the treatment is finished.


Eligibility criteria: Inclusion Criteria:

* 1. Subjects have a height less than two standard deviations (SD) below the median height for individuals of he same age or height,the growth velocity (CV≤4.0 cm/yr),GH peak concentration \<7.0ng/ml in two difference provocative tests, Bone age (BA) ≤9 years in girls and ≤10 years in boys, at least 2 year less than his/her chronological age (CA)
* be in preadolescence (Tanner stage 1) and have a CA \> 3 years
* have a height value recorded 3 months before the start of GH treatment to calculate pre-treatment GV.
* receive no prior GH treatment.
* sign informed consent

Exclusion Criteria:

* 1. patients positive for hepatitis B e-antibody (HBeAb), hepatitis B surface antigen (HBsAg) or hepatitis B e antigen (HBeAg)
* 2.Known hypersensitivity to Somatropin or any other components of the study product.
* 3. severe cardiopulmonary or hematological diseases, a current or past history of malignant tumors, immunodeficiency diseases, or mental diseases
* 4. with other growth disorders, such as Turner syndrome, constitutional delay of growth and puberty, and Laron syndrome
* 5. Subjects took part in other clinical trial study during 3 months.
* 6. Other conditions which in the opinion of the investigator preclude enrollment into the study.

Primary outcomes:
measure: yearly growth velocity
timeFrame: 6 months
Secondary outcomes:
measure: Ht SDSca
timeFrame: 6 months
measure: IGF-l
timeFrame: 6 months
measure: IGFBP-3
timeFrame: 6 months
measure: bone maturity
timeFrame: 6 months
Other outcomes:
Locations:
city: Beijing
country: China
city: Changchun
country: China
city: Guangzhou
country: China
city: Hangzhou
country: China
city: Shanghai
country: China
city: Wu Han
country: China
Study officials:
affiliation: Tongji Hospital
name: Xiaoping Luo, Ph. D
role: PRINCIPAL_INVESTIGATOR
References:
citation: Luo X, Hou L, Liang L, Dong G, Shen S, Zhao Z, Gong CX, Li Y, Du ML, Su Z, Du H, Yan C. Long-acting PEGylated recombinant human growth hormone (Jintrolong) for children with growth hormone deficiency: phase II and phase III multicenter, randomized studies. Eur J Endocrinol. 2017 Aug;177(2):195-205. doi: 10.1530/EJE-16-0905. Epub 2017 May 31.