Clinical trial NCT03290235: Extension Study of Pegylated Somatropin to Treat Growth Retardation Caused by Endogenous Growth Hormone Deficiency in Children
Official title: The Extension Study of Phase IV Clinical Trial of Pegylated Somatropin (PEG Somatropin) to Treat Growth Retardation Caused by Endogenous Growth Hormone Deficiency in Children
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE4
Conditions: Growth Retardation
Lead sponsor: Changchun GeneScience Pharmaceutical Co., Ltd.
Sponsor class: INDUSTRY
First posted: 2017-09-21
Enrollment: 1500
Sex: ALL
Minimum age (years): 42
Maximum age (years): 15
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 1. To further evaluate the safety and efficacy of PEG-Somatropin in the treatment of children with growth hormone deficiency for a relatively long period
2. To explore the factors influencing the efficacy of PEG-Somatropin and to establish the height prediction model based on Chinese children with short stature, and to provide the basis and guidance for standard and reasonable long-term clinical application of PEG-Somatropin.


Eligibility criteria: Inclusion Criteria:

* Children has completed all visits and therapy in previous phase IV study;
* Investigators evaluate subjects could continue growth hormone therapy;
* Subjects is willing and able to cooperate to complete scheduled visits, treatment plans and laboratory tests and other procedures, to sign informed consent.

Exclusion Criteria:

* Children with epiphyseal closure;
* Children is near the adule final height, that is, growth rate≤ 2 cm / year or bone age ≥ 14 years old for girls, bone age ≥ 16 years old for boys;
* Dysfunction of liver and kidney (ALT\> 2 times the upper limit of normal, Cr\> upper limit of normal);
* Patients with known hypersensitivity to PEG-Somatropin or Somatropin or any other components of the study product;
* Patients with severe cardiopulmonary or hematological diseases, a current or past history of malignant tumors, immunodeficiency diseases, or mental diseases;
* Patients with diabetics;
* Patients with congenital bone dysplasia or scoliosis;
* Patients took drugs that would influence the efficacy and safety of PEG-Somatropin after phase IV study and before screening for this extension study;
* Other conditions in which the investigator preclude enrollment into the study.

Primary outcomes:
measure: Ht SDSca (Height standard deviation score for chronological age)
timeFrame: Baseline, every 13 weeks until 130 weeks
Secondary outcomes:
measure: Ht SDSBA (Height standard deviation score for bone age)
timeFrame: Baseline, every 13 weeks until 130 weeks
measure: Yearly growth velocity
timeFrame: Baseline, every 13 weeks until 130 weeks
measure: IGF-1 SDS (Standard deviation score of insulin-like growth factor-1)
timeFrame: Baseline, every 13 weeks until 130 weeks
measure: Bone age
timeFrame: Baseline, 26 weeks, 52 weeks, 78 weeks, 104 weeks, 130 weeks
measure: Near final height for some subjects
timeFrame: Baseline, every 13 weeks until 130 weeks
Other outcomes:
Locations:
city: Wuhan
country: China
facility: TongJi hospital affiliated to TongJi medical college of HuaZhong university of Science & Teconology
state: Hubei
Study officials:
References: