search
Back to results

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

Primary Purpose

Prader-Willi Syndrome

Status
Unknown status
Phase
Phase 3
Locations
China
Study Type
Interventional
Intervention
Recombinant Human Growth Hormone (rhGH) Injection
Sponsored by
Changchun GeneScience Pharmaceutical Co., Ltd.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Prader-Willi Syndrome

Eligibility Criteria

1 Month - 5 Years (Child)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Tongji medical college huazhong university of science & technologyRecruiting
  • The Children's Hospital,Zhejiang University School of MedicineRecruiting
  • Beijing Children's Hospital, Capital Medical UniversityRecruiting
  • Peking union medical college hospitalRecruiting
  • Peking university first hospitalRecruiting
  • Children's Hospital of Fudan UniversityRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

rhGH injection/Jintropin AQ

Arm Description

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.

Outcomes

Primary Outcome Measures

The change of the total motor quotient calculated by Peabody Developmental Motor Scale before and after treatment

Secondary Outcome Measures

Gross motor quotient and fine motor quotient calculated by Peabody Developmental Motor Scale before and after treatment
Change of height standard deviation score (SDS) by chronological age before and after treatment
Body weight change before and after treatment
Change of BMI (Body mass index) standard deviation score before and after treatment
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
Bone maturation ( bone age/ chronological age: BA/CA)
Change of IGF-1(Insulin-like growth factor 1) SDS
IGF-1/IGFBP-3 molar ratio

Full Information

First Posted
May 30, 2018
Last Updated
May 30, 2018
Sponsor
Changchun GeneScience Pharmaceutical Co., Ltd.
Collaborators
Children's Hospital of Fudan University, Tongji medical college huazhong university of science & technology, Beijing Children's Hospital, Peking University First Hospital, Peking Union Medical College Hospital, The Children's Hospital of Zhejiang University School of Medicine
search

1. Study Identification

Unique Protocol Identification Number
NCT03554031
Brief Title
A Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone Injection in Patients With Prader-Willi Syndrome
Official Title
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
Interventional

2. Study Status

Record Verification Date
May 2018
Overall Recruitment Status
Unknown status
Study Start Date
April 14, 2018 (Actual)
Primary Completion Date
January 2020 (Anticipated)
Study Completion Date
January 1, 2020 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Changchun GeneScience Pharmaceutical Co., Ltd.
Collaborators
Children's Hospital of Fudan University, Tongji medical college huazhong university of science & technology, Beijing Children's Hospital, Peking University First Hospital, Peking Union Medical College Hospital, The Children's Hospital of Zhejiang University School of Medicine

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Prader-Willi Syndrome

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
30 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
rhGH injection/Jintropin AQ
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
Recombinant Human Growth Hormone (rhGH) Injection
Intervention Description
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.
Primary Outcome Measure Information:
Title
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 Outcome Measure Information:
Title
Gross motor quotient and fine motor quotient calculated by Peabody Developmental Motor Scale before and after treatment
Time Frame
Baseline, 26 weeks, 52 weeks
Title
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
Title
Body weight change before and after treatment
Time Frame
Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks
Title
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
Title
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
Title
Bone maturation ( bone age/ chronological age: BA/CA)
Time Frame
Baseline, 52 weeks
Title
Change of IGF-1(Insulin-like growth factor 1) SDS
Time Frame
Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks
Title
IGF-1/IGFBP-3 molar ratio
Time Frame
Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
1 Month
Maximum Age & Unit of Time
5 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Xiaohua Feng
Phone
0431-85170552
Email
fengxiaohua@gensci-china.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Feihong Luo, Doctor
Organizational Affiliation
Children's Hospital of Fudan University
Official's Role
Principal Investigator
Facility Information:
Facility Name
Tongji medical college huazhong university of science & technology
City
Wuhan
State/Province
Hubei
Country
China
Individual Site Status
Recruiting
Facility Name
The Children's Hospital,Zhejiang University School of Medicine
City
Hangzhou
State/Province
Zhejiang
Country
China
Individual Site Status
Recruiting
Facility Name
Beijing Children's Hospital, Capital Medical University
City
Beijing
Country
China
Individual Site Status
Recruiting
Facility Name
Peking union medical college hospital
City
Beijing
Country
China
Individual Site Status
Recruiting
Facility Name
Peking university first hospital
City
Beijing
Country
China
Individual Site Status
Recruiting
Facility Name
Children's Hospital of Fudan University
City
Shanghai
Country
China
Individual Site Status
Recruiting

12. IPD Sharing Statement

Learn more about this trial

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

We'll reach out to this number within 24 hrs