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

May 30, 2018 updated by: GeneScience Pharmaceuticals Co., Ltd.

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

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

Study Overview

Status

Unknown

Study Type

Interventional

Enrollment (Anticipated)

30

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

      • Beijing, China
        • Recruiting
        • Peking Union Medical College Hospital
      • Beijing, China
        • Recruiting
        • Peking University First Hospital
      • Beijing, China
        • Recruiting
        • Beijing Children's Hospital, Capital Medical University
      • Shanghai, China
        • Recruiting
        • Children's Hospital of Fudan University
    • Hubei
      • Wuhan, Hubei, China
        • Recruiting
        • Tongji Medical College Huazhong University of Science & Technology
    • Zhejiang
      • Hangzhou, Zhejiang, China
        • Recruiting
        • The Children's Hospital,Zhejiang University School of Medicine

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 month to 5 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
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.
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.

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

Outcome Measure
Time Frame
Gross motor quotient and fine motor quotient calculated by Peabody Developmental Motor Scale before and after treatment
Time Frame: Baseline, 26 weeks, 52 weeks
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
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
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
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
Baseline, 26 weeks, 52 weeks
Bone maturation ( bone age/ chronological age: BA/CA)
Time Frame: Baseline, 52 weeks
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
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
Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

April 14, 2018

Primary Completion (Anticipated)

January 1, 2020

Study Completion (Anticipated)

January 1, 2020

Study Registration Dates

First Submitted

May 30, 2018

First Submitted That Met QC Criteria

May 30, 2018

First Posted (Actual)

June 12, 2018

Study Record Updates

Last Update Posted (Actual)

June 12, 2018

Last Update Submitted That Met QC Criteria

May 30, 2018

Last Verified

May 1, 2018

More Information

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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