A Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone Injection in Patients With Prader-Willi Syndrome

Overview

To evaluate the effectiveness of rhGH (Recombinant human growth hormone) injection for improving motor development in patients with PWS.

Full Title of Study: “A Single Arm, Multicenter Phase III Clinical Trial to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone Injection in Patients With Prader-Willi Syndrome”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: N/A
    • Intervention Model: Single Group Assignment
    • Primary Purpose: Treatment
    • Masking: None (Open Label)
  • Study Primary Completion Date: January 2020

Interventions

  • Drug: Recombinant Human Growth Hormone (rhGH) Injection
    • Drug: Recombinant Human Growth Hormone Injection /Jintropin AQ, 30IU/10 mg/3ml/kit, 0.5 mg/m2/d for the first 4 weeks, then 1.0 mg/m2/d for subsequent 48 weeks; by subcutaneous injection, once per day for total 52 weeks.

Arms, Groups and Cohorts

  • Experimental: rhGH injection/Jintropin AQ
    • Drug: Recombinant Human Growth Hormone Injection /Jintropin AQ, 30IU/10 mg/3ml/kit, 0.5 mg/m2/d for the first 4 weeks, then 1.0 mg/m2/d for subsequent 48 weeks; by subcutaneous injection, once per day for total 52 weeks.No control.

Clinical Trial Outcome Measures

Primary Measures

  • The change of the total motor quotient calculated by Peabody Developmental Motor Scale before and after treatment
    • Time Frame: Baseline, 26 weeks, 52 weeks

Secondary Measures

  • Gross motor quotient and fine motor quotient calculated by Peabody Developmental Motor Scale before and after treatment
    • Time Frame: Baseline, 26 weeks, 52 weeks
  • Change of height standard deviation score (SDS) by chronological age before and after treatment
    • Time Frame: Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks
  • Body weight change before and after treatment
    • Time Frame: Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks
  • Change of BMI (Body mass index) standard deviation score before and after treatment
    • Time Frame: Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks
  • Global quotient, locomotor quotient, personal-social development quotient, language quotient, eye and hand coordination quotient, performance quotient and practical reasoning change calculated by Griffiths Mental Development Scale
    • Time Frame: Baseline, 26 weeks, 52 weeks
  • Bone maturation ( bone age/ chronological age: BA/CA)
    • Time Frame: Baseline, 52 weeks
  • Change of IGF-1(Insulin-like growth factor 1) SDS
    • Time Frame: Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks
  • IGF-1/IGFBP-3 molar ratio
    • Time Frame: Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks

Participating in This Clinical Trial

Inclusion Criteria

  • 1.Signed informed consent from legal guardian of the subjects; – Subjects are willing and able to cooperate to complete scheduled visits, treatment plans and laboratory tests and other procedures; – Diagnosed as PWS by gene test; – Age: 1 month (30 days after birth) – 5 years of age; – Male or female; – Calculated by Peabody Developmental Motor Scale, total motor quotient, gross motor quotient or fine motor quotient is less than 90 points; – Thyroid function is within the normal reference range or remained within the normal reference range by substitution therapy; – No history of rhGH therapy before. Exclusion Criteria:

  • Subjects with abnormal liver or kidney function; – Subjects with obvious central sleep apnea and/or moderate or severe obstructive sleep apnea, acute lung infection; – Subjects with chronic diseases that have long-term effects on bone metabolism and body composition; – Subjects with congenital skeletal dysplasia, or spine scoliosis with moderate and above degree requiring treatment or lameness; – Subjects with history of congenital heart disease, or an echocardiogram showing that the structural abnormalities require surgery or interventional therapy or that the left ventricular ejection fraction is <40%, or the abnormal electrocardiogram requiring intervention; – Subjects with history of convulsions or epilepsy; – Subjects with other systemic chronic diseases; – Subjects with diagnosed tumors; – Subjects with family history of cancers, a previous history of cancer, or considered to be a high risk of cancer combinating other information; – Subjects with mental disease; – Subjects with diabetes, or abnormal fasting glucose and researchers believe that may affect the safety of the subject; – Subjects with severe obesity; – Subjects with highly allergic constitution or allergy to proteins or investigational product or its excipient; – Subjects who took part in other clinical trials within 3 months ; – Subjects who received drug treatment that may interfere with GH secretion or GH action within 3 months; – Other conditions in which the investigator preclude enrollment into the study

Gender Eligibility: All

Minimum Age: 1 Month

Maximum Age: 5 Years

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Changchun GeneScience Pharmaceutical Co., Ltd.
  • Collaborator
    • Children’s Hospital of Fudan University
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • Feihong Luo, Doctor, Principal Investigator, Children’s Hospital of Fudan University
  • Overall Contact(s)
    • Xiaohua Feng, 0431-85170552, fengxiaohua@gensci-china.com

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