Clinical trial NCT03535415: A Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Short Stature Children Due to Chronic Kidney Disease Before Transplantation
Official title: A Phase 3 Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Short Stature Children Due to Chronic Kidney Disease Before Transplantation
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE3
Conditions: Dwarfism
Lead sponsor: Changchun GeneScience Pharmaceutical Co., Ltd.
Sponsor class: INDUSTRY
Collaborators: name: Children's Hospital of Fudan University
name: Beijing Children's Hospital
name: Shanghai Children's Hospital
name: Xin Hua Hospital Affiliated to Shanghai Jiao Tong University School of Medicine
name: The Children's Hospital of Zhejiang University School of Medicine
name: Children's Hospital of The Capital Institute of Pediatrics
name: Peking University First Hospital
name: Fuzhou General Hospital
name: The First Affiliated Hospital of Zhengzhou University
name: Shengjing Hospital
name: Children's Hospital of Chongqing Medical University
name: Tongji medical college huazhong university of science & technology
First posted: 2018-05-24
Enrollment: 68
Sex: ALL
Minimum age (years): 2
Maximum age (years): 14
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This study aims to evaluate the efficacy and safety of recombinant human growth hormone injection in short stature children due to chronic kidney disease before transplantation.


Eligibility criteria: Inclusion Criteria:

* Signed informed consent of the subjects or the legal guardian.
* Subjects and guardian are willing and able to cooperate to complete scheduled visits,treatment plans and laboratory tests and other procedures.
* Diagnosed as chronic kidney disease.
* Glomerular filtration rate (GFR) \<75ml/per min/1.73m2.
* After the treatment and adjustment of hypertension, anemia, metabolic acidosis, malnutrition, renal osteopathy, hypothyroidism, etc..
* Chronological age: ≥2years and ≤14years.
* Height Standard Deviation Score(HtSDS) ≤-2SD for chronological age.
* Bone age: girls≤10 years; boys≤11years.
* Pre-pubertal (Tanner Stage I ) patients.
* No history of growth hormone treatment.

Exclusion Criteria:

* Subjects with abnormal liver functions.
* Subjects positive for anti-HBc, HbsAg or HbeAg in Hepatitis B virus tests.
* After adjustment heart function,Cardiac ejection fraction(EF) \<50%.
* Subjects with highly allergic constitution or allergy to proteins or investigational product or its excipient.
* Subjects with systemic chronic disease or general infection or mental disease.
* Subjects with diabetes or impaired fasting glucose.
* Subjects with tumor or potential tumor.
* Subjects who are using glucocorticoid or immunosuppressant.
* Other causes for growth retardation.
* Inability to obtain accurate height measurements.
* Subjects who took part in other clinical trials within 3 months.
* Concomitant administration of other treatment that may have an effect on growth within 3 months.
* Other conditions which are unsuitable for this study in the opinion of the investigator.

Primary outcomes:
measure: The change of height standard deviation score of chronological age before and after treatment (ΔHT SDS)
timeFrame: 52 weeks，104 weeks
Secondary outcomes:
measure: Growth velocity
timeFrame: 12 months, 24 months
measure: Bone maturation ( bone age/ chronological age: BA/CA)
timeFrame: 12 months, 24 months
measure: IGF-1 (Insulin-like growth factor 1) SDS
timeFrame: 12 months, 24 months
measure: IGF-1/IGFBP-3 molar ratio
timeFrame: 12 months, 24 months
Other outcomes:
Locations:
city: Fuzhou
country: China
facility: Fuzhou general hospital of Nanjing military command
state: Fujian
city: Zhengzhou
country: China
facility: The first affiliated hospital of zhengzhou university
state: Henan
city: Wuhan
country: China
facility: Tongji medical college huazhong university of science & technology
state: Hubei
city: Shenyang
country: China
facility: Shengjing hospital of chian medical university
state: Liaoning
city: Shanghai
country: China
facility: Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
state: Shanghai
city: Hangzhou
country: China
facility: The Children's Hospital ,Zhejiang University School of Medicine
state: Zhejiang
city: Beijing
country: China
facility: Beijing Children's Hospital, Capital Medical University
city: Beijing
country: China
facility: Children's hospital capital institute of pediatrics
city: Beijing
country: China
facility: Peking university first hospital
city: Chongqing
country: China
facility: Children's hospital of Chongqing medical university
city: Shanghai
country: China
facility: Children's Hospital of Fudan University
city: Shanghai
country: China
facility: Children's Hospital of Shanghai
Study officials:
affiliation: Children's Hospital of Fudan University
name: Hong Xu, PhD
role: PRINCIPAL_INVESTIGATOR
References: