Clinical trial NCT03249480: Pegylated Somatropin (PEG Somatropin) in the Treatment of Children With Growth Hormone Deficiency
Official title: Pegylated Somatropin (PEG Somatropin) in the Treatment of Children With Growth Hormone Deficiency: A Multicenter, Open-label, Phase IV Clinical Trial With Different Administration Dosage of PEG Somatropin
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE4
Conditions: Growth Hormone Deficiency
Lead sponsor: Changchun GeneScience Pharmaceutical Co., Ltd.
Sponsor class: INDUSTRY
Collaborators: name: Beijing Children's Hospital
name: Tianjin Medical University General Hospital
name: Peking University Third Hospital
name: China-Japan Friendship Hospital
name: Dalian Children's Hospital
name: Xian Children's Hospital
name: Wuhan Children's Hospital
name: Fuzhou Children s Hospital
name: Anhui Provincial Children's Hospital
name: Hunan Children's Hospital
name: Shenzhen Children's Hospital
name: Children's Hospital of Hebei Province
name: Second Affiliated Hospital of Xi'an Jiaotong University
name: Zhengzhou Children's Hospital, China
name: Children's Hospital of Soochow University
name: Maternal and Child Health Hospital of Guangxi Zhuang Autonomous Region
name: First Affiliated Hospital of Kunming Medical University
name: Kunming Children's Hospital
name: The First People's Hospital of Yunnan
name: Xuzhou Children's Hopspital
name: Henan Provincial People's Hospital
name: The First Affiliated Hospital of Henan University of Traditional Chinese Medicine
name: First Affiliated Hospital of Harbin Medical University
name: The Second Hospital of Hebei Medical University
name: Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University
name: Union Hospital of Tongji Medical College of HUST
name: Wuxi Women's & Children's Hospital
name: The Second Affiliated Hospital of Kunming Medical University
name: Nanjing Children's Hospital
First posted: 2017-08-15
Enrollment: 900
Sex: ALL
Minimum age (years): 3
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: To Evaluate the safety and efficacy of PEG Somatropin in the treatment of children with growth hormone deficiency, as well as to study the dosage of PEG Somatropin.


Eligibility criteria: Inclusion Criteria:

* Diagnosed as GHD before starting treatment, according to medical history, clinical symptoms and signs, GH provocation tests and imaging and other examinations:

  1. According to statistical height data of physical development of Chinese children in nine cities in 2005, the height is lower than 3rd percentile growth curve of the same age, same sex normal children;
  2. Height velocity (HV) ≤5.0 cm / yr;
  3. GH provocative tests (with two drugs of different mechanism of action) confirmed plasma GH peak \<10.0 ng / ml;
  4. bone age for girl≤9 years old, for Boy≤10 years old, bone age is one year or more later than the actual age, that is the actual age - bone age ≥ 1 year;
* Before puberty (Tanner I stage), age≥3 years old, male or female;
* Have not received hormone therapy within 6 months;
* 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:

* Dysfunction of liver and kidney (ALT\> 2 times the upper limit of normal, Cr\> upper limit of normal);
* Patients positive for hepatitis B c-antibody (HBcAb), hepatitis B surface antigen (HBsAg) or hepatitis B e antigen (HBeAg);
* 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;
* Potential cancer patients (family history);
* Patients with diabetics;
* Patients with other growth disorders, such as Turner's syndrome, sexual physical delayed puberty, Laron syndrome, growth hormone receptor deficiency, girls with slowly growing who did not rule out chromosomal abnormalities;
* Patients with congenital bone dysplasia or scoliosis;
* Subjects took part in other clinical trial study during 3 months;
* 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
measure: Change of yearly growth velocity from baseline to 26 weeks
timeFrame: Baseline, 26 weeks after initiating treatment
measure: Ht SDSca
timeFrame: 26 weeks after initiating treatment
Secondary outcomes:
measure: Ht SDSBA (Height Standard Deviation Score for Bone Age)
timeFrame: Baseline,26 weeks after initiating treatment
measure: IGF-1 SDS (IGF-1 Standard Deviation Score)
timeFrame: Baseline,26 weeks after initiating treatment
measure: IGFBP-3 SDS (IGFBP-3 Standard Deviation Score)
timeFrame: Baseline,26 weeks after initiating treatment
measure: Skeletal maturity
timeFrame: Baseline,26 weeks after initiating treatment
Other outcomes:
Locations:
city: Hefei
country: China
facility: Anhui Provincial Children's Hospital
state: Anhui
city: Fuzhou
country: China
facility: Fuzhou Children s Hospital
state: Fujian
city: Guangzhou
country: China
facility: Sun Yat-Sen Memorial Hospital
state: Guangdong
city: Shijiangzhuang
country: China
facility: The Second Hospital of Hebei Medical University
state: Hebei
city: Shijiazhuang
country: China
facility: Children's Hospital of Hebei Province
state: Hebei
city: Harbin
country: China
facility: The First Affiliated Hospital of Harbin Medical University
state: Heilongjiang
city: Zhengzhou
country: China
facility: Children's Hospital of Zhengzhou
state: Henan
city: Zhengzhou
country: China
facility: Henan Provincal People's Hospital
state: Henan
city: Zhengzhou
country: China
facility: The First Affiliated Hospital of Henan University of TCM
state: Henan
city: Wuhan
country: China
facility: Union Hospital, Tongji Medical College, HUST
state: Hubei
city: Wuhan
country: China
facility: Wuhan Children's Hospital
state: Hubei
city: Changsha
country: China
facility: Hunan Children's Hospital
state: Hunan
city: Nanjing
country: China
facility: Maternal & Child Health Hospital of Guangxi Zhuang Autonomous Region
state: Jiangsu
city: Nanjing
country: China
facility: Nanjing Children's Hospital
state: Jiangsu
city: Suzhou
country: China
facility: Children's Hospital of Soochow University
state: Jiangsu
city: Wuxi
country: China
facility: Wuxi Children's Hospital
state: Jiangsu
city: Xuzhou
country: China
facility: Xuzhou Children's Hpspital
state: Jiangsu
city: Dalian
country: China
facility: Dalian Children's Hospital
state: Liaoning
city: Xi'an
country: China
facility: The Second Affiliated Hospital of Xi'an Jiaotong University
state: Shanxi
city: Xi'an
country: China
facility: Xi'an Children's Hospital
state: Shanxi
city: Kunming
country: China
facility: First Affiliated Hospital of Kunming Medical University
state: Yunnan
city: Kunming
country: China
facility: Kunming Children's Hospital
state: Yunnan
city: Kunming
country: China
facility: Second Affiliated Hospital of Kunming Medical University
state: Yunnan
city: Kunming
country: China
facility: The First People's Hospital of Yunnan Province
state: Yunnan
city: Beijing
country: China
facility: China-Japan Friendship Hospital
city: Beijing
country: China
facility: Peking University Third Hospital
city: Shenzhen
country: China
facility: Shenzhen Children's Hospital
city: Tianjin
country: China
facility: Tianjin Medical University General Hospital
Study officials:
affiliation: Beijing Children's Hospital
name: Chunxiu Gong
role: PRINCIPAL_INVESTIGATOR
References:
citation: Chen J, Zhong Y, Wei H, Chen S, Su Z, Liu L, Liang L, Lu P, Chen L, Chen R, Ni S, Wang X, Li L, Wang Y, Xu X, Xiao Y, Yao H, Liu G, Jin R, Cao B, Wu D, Su C, Li W, Qin M, Li X, Luo X, Gong C. Polyethylene glycol recombinant human growth hormone in Chinese prepubertal slow-growing short children: doses reported in a multicenter real-world study. BMC Endocr Disord. 2022 Aug 9;22(1):201. doi: 10.1186/s12902-022-01101-8.