A Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Short Stature Children Due to Chronic Kidney Disease Before Transplantation

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

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.

Study Overview

• Status: Unknown
• Conditions: Dwarfism
• Intervention / Treatment: Drug: Recombinant Human Growth Hormone Injection (rhGH)

• Study Type: Interventional
• Enrollment (Anticipated): 68
• Phase: Phase 3

Contacts and Locations

Study Locations

• China
  • Beijing, China
    • Recruiting
    • Peking University First Hospital
    • Contact: Huijie Xiao
  • Beijing, China
    • Recruiting
    • Beijing Children's Hospital, Capital Medical University
    • Contact: Ying Shen
  • Beijing, China
    • Recruiting
    • Children's Hospital Capital Institute of Pediatrics
    • Contact: Chaoying Chen
  • Chongqing, China
    • Recruiting
    • Children's Hospital of Chongqing Medical University
    • Contact: Mo Wang
  • Shanghai, China
    • Recruiting
    • Children's Hospital of Fudan University
    • Contact: Hong Xu
  • Shanghai, China
    • Recruiting
    • Children's Hospital of Shanghai
    • Contact: Wenyan Huang
  • Fujian
    • Fuzhou, Fujian, China
      • Recruiting
      • Fuzhou General hospital of Nanjing Military Command
      • Contact: Zihua Yu
  • Henan
    • Zhengzhou, Henan, China
      • Recruiting
      • The First Affiliated Hospital of Zhengzhou University
      • Contact: Jianjiang Zhang
  • Hubei
    • Wuhan, Hubei, China
      • Recruiting
      • Tongji Medical College Huazhong University of Science & Technology
      • Contact: Jianhua Zhou
  • Liaoning
    • Shenyang, Liaoning, China
      • Recruiting
      • Shengjing hospital of chian medical university
      • Contact: Yubin Wu
  • Shanghai
    • Shanghai, Shanghai, China
      • Recruiting
      • Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
      • Contact: Yufeng Li
  • Zhejiang
    • Hangzhou, Zhejiang, China
      • Recruiting
      • The Children's Hospital ,Zhejiang University School of Medicine
      • Contact: Jianhua Mao

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 7 months to 12 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: rhGH Injection; rhGH 0.05mg/kg/d by subcutaneous injection	Drug: Recombinant Human Growth Hormone Injection (rhGH); rhGH 0.05mg/kg/d by subcutaneous injection
No Intervention: Non-treatment control group; Only follow-up without treatment

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
The change of height standard deviation score of chronological age before and after treatment (ΔHT SDS)	52 weeks，104 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Growth velocity	365.25*（height at the time of assessment -height at baseline）/duration of treatment(days)	12 months, 24 months
Bone maturation ( bone age/ chronological age: BA/CA)		12 months, 24 months
IGF-1 (Insulin-like growth factor 1) SDS		12 months, 24 months
IGF-1/IGFBP-3 molar ratio		12 months, 24 months

Collaborators and Investigators

• Sponsor: GeneScience Pharmaceuticals Co., Ltd.
• Collaborators: Peking University First Hospital; Shengjing Hospital; The First Affiliated Hospital of Zhengzhou University; Children's Hospital of Fudan University; Shanghai Children's Hospital; Beijing Children's Hospital; Fuzhou General Hospital; The Children's Hospital of Zhejiang University School of Medicine; Children's Hospital of Chongqing Medical University; Children's Hospital of The Capital Institute of Pediatrics; Xin Hua Hospital Affiliated to Shanghai Jiao Tong University School of Medicine; Tongji medical college huazhong university of science & technology
• Investigators: Principal Investigator: Hong Xu, PhD, Children's Hospital of Fudan University

Study record dates

Study Major Dates

• Study Start (Actual): June 25, 2018
• Primary Completion (Anticipated): September 1, 2020
• Study Completion (Anticipated): September 1, 2021

Study Registration Dates

• First Submitted: April 28, 2018
• First Submitted That Met QC Criteria: May 22, 2018
• First Posted (Actual): May 24, 2018

Study Record Updates

• Last Update Posted (Actual): November 23, 2018
• Last Update Submitted That Met QC Criteria: November 21, 2018
• Last Verified: November 1, 2018

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Urologic Diseases; Endocrine System Diseases; Renal Insufficiency; Genetic Diseases, Inborn; Musculoskeletal Diseases; Bone Diseases; Bone Diseases, Developmental; Kidney Diseases; Renal Insufficiency, Chronic; Dwarfism; Physiological Effects of Drugs; Hormones, Hormone Substitutes, and Hormone Antagonists; Hormones
• Other Study ID Numbers: GenSci GH AQ CT-CKD

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No