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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03554031
Recruitment Status : Unknown
Verified May 2018 by GeneScience Pharmaceuticals Co., Ltd..
Recruitment status was:  Recruiting
First Posted : June 12, 2018
Last Update Posted : June 12, 2018
Sponsor:
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
Information provided by (Responsible Party):
GeneScience Pharmaceuticals Co., Ltd.

Tracking Information
First Submitted Date  ICMJE May 30, 2018
First Posted Date  ICMJE June 12, 2018
Last Update Posted Date June 12, 2018
Actual Study Start Date  ICMJE April 14, 2018
Estimated Primary Completion Date January 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 30, 2018)
The change of the total motor quotient calculated by Peabody Developmental Motor Scale before and after treatment [ Time Frame: Baseline, 26 weeks, 52 weeks ]
Original Primary Outcome Measures  ICMJE Same as current
Change History No Changes Posted
Current Secondary Outcome Measures  ICMJE
 (submitted: May 30, 2018)
  • 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 ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone Injection in Patients With Prader-Willi Syndrome
Official Title  ICMJE 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
Brief Summary To evaluate the effectiveness of rhGH (Recombinant human growth hormone) injection for improving motor development in patients with PWS.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Prader-Willi Syndrome
Intervention  ICMJE 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.
Study Arms  ICMJE 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.
Intervention: Drug: Recombinant Human Growth Hormone (rhGH) Injection
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Unknown status
Estimated Enrollment  ICMJE
 (submitted: May 30, 2018)
30
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE January 1, 2020
Estimated Primary Completion Date January 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 1 Month to 5 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE China
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03554031
Other Study ID Numbers  ICMJE GenSci PWS CT
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party GeneScience Pharmaceuticals Co., Ltd.
Study Sponsor  ICMJE GeneScience Pharmaceuticals Co., Ltd.
Collaborators  ICMJE
  • 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
Investigators  ICMJE
Principal Investigator: Feihong Luo, Doctor Children's Hospital of Fudan University
PRS Account GeneScience Pharmaceuticals Co., Ltd.
Verification Date May 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP