Clinical trial NCT02976675: 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, Randomized, Open-label, Parallel Phase IV Clinical Trial With Different Administration Frequency 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: Children's Hospital of Fudan University
name: Suzhou Municipal Hospital
name: General Hospital of Ningxia Medical University
name: The First Affiliated Hospital of Henan University of Science and Technology
name: Maternal and Child Health Hospital of Hubei Province
name: Nantong University
First posted: 2016-11-29
Enrollment: 600
Sex: ALL
Minimum age (years): 3
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: To evaluate the safety and efficacy of PEG Somatropin Injection in the treatment of children with growth hormone deficiency, as well as to study the feasibility of extending the dosing intervals (once per two weeks) of PEG Somatropin Injection.


Eligibility criteria: Inclusion Criteria:

* Subjects are 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;
* Subjects 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
timeFrame: Baseline, 4,12,26 weeks after initiating treatment
Secondary outcomes:
measure: Ht SDSBA
timeFrame: Baseline, 4,12,26 weeks after initiating treatment
measure: Yearly growth velocity
timeFrame: Baseline, 4,12,26 weeks after initiating treatment
measure: IGF-1SDS
timeFrame: Baseline, 4,12,26 weeks after initiating treatment
measure: IGF-1/IGFBP-3 molar ratio
timeFrame: Baseline, 4,12,26 weeks after initiating treatment
measure: Bone age
timeFrame: Baseline, 4,12,26 weeks after initiating treatment
Other outcomes:
Locations:
city: Hefei
country: China
facility: Maternal and Child Health Hospital of Anhui province
state: Anhui
city: Lu'an
country: China
facility: First People's Hospital of Lu'an City
state: Anhui
city: Lanzhou
country: China
facility: Second Hospital of Lanzhou University
state: Gansu
city: Guiyangtou
country: China
facility: Maternal and Child Health Hospital of Guiyang City
state: Guizhou
city: Haikou
country: China
facility: Maternal and Child Health Hospital of Hainan province
state: Hainan
city: Shijiazhuang
country: China
facility: Hebei Provincial People's Hospital
state: Hebei
city: Zhengzhou
country: China
facility: First Affiliated Hospital of Henan University of Science and Technology
state: Henan
city: Wuhan
country: China
facility: Maternal and Child Health Hospital of Hubei province
state: Hubei
city: Changzhou
country: China
facility: First People's Hospital of Changzhou City
state: Jiangsu
city: Lianyungang
country: China
facility: First People's Hospital of Lianyungang City
state: Jiangsu
city: Nanjingkou
country: China
facility: Jiangsu Provincial Hospital of Chinese Traditional medicine
state: Jiangsu
city: Nantong
country: China
facility: Affiliated Hospital of Nantong University
state: Jiangsu
city: Suzhou
country: China
facility: Suzhou City Hospital
state: Jiangsu
city: Nanchang
country: China
facility: Second Affiliated Hospital of Nanchang University
state: Jiangxi
city: Tai'an
country: China
facility: Maternal and Child Health Hospital of Tai'an city
state: Shandong
city: Chengdu
country: China
facility: Children's Hospital of Chengdu city
state: Sichuan
city: Cixi
country: China
facility: People's Hospital of Cixi City
state: Zhejiang
city: Hangzhou
country: China
facility: First People's Hospital
state: Zhejiang
city: Jiaxing
country: China
facility: First People's Hospital of Jiaxing City
state: Zhejiang
city: Jinhua
country: China
facility: Jinhua Central Hospital
state: Zhejiang
city: Beijing
country: China
facility: Jishuitan Hospital of Beijing City
city: Inner Mongolia Autonomous Region
country: China
facility: Peoples' hospital of Inner Mongolia Autonomous Region
city: Ningxia Autonomous Region
country: China
facility: General Hospital of Ningxia Medical University
city: Shanghai
country: China
facility: Children's Hospital of Fudan University
Study officials:
affiliation: Children's Hospital of Fudan University
name: Feihong Luo, Doctor
role: PRINCIPAL_INVESTIGATOR
References:
citation: Sun C, Lu B, Liu Y, Zhang Y, Wei H, Hu X, Hu P, Zhao Q, Liu Y, Ye K, Wang K, Gu Z, Liu Z, Ye J, Zhang H, Zhu H, Jiang Z, Liu Y, Wan N, Yan C, Yin J, Ying L, Huang F, Yin Q, Xi L, Luo F, Cheng R. Reduced Effectiveness and Comparable Safety in Biweekly vs. Weekly PEGylated Recombinant Human Growth Hormone for Children With Growth Hormone Deficiency: A Phase IV Non-Inferiority Threshold Targeted Trial. Front Endocrinol (Lausanne). 2021 Nov 25;12:779365. doi: 10.3389/fendo.2021.779365. eCollection 2021.