- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03554031
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
Conditions
Intervention / Treatment
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
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Beijing, China
- Recruiting
- Peking Union Medical College Hospital
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Beijing, China
- Recruiting
- Peking University First Hospital
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Beijing, China
- Recruiting
- Beijing Children's Hospital, Capital Medical University
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Shanghai, China
- Recruiting
- Children's Hospital of Fudan University
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Hubei
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Wuhan, Hubei, China
- Recruiting
- Tongji Medical College Huazhong University of Science & Technology
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Zhejiang
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Hangzhou, Zhejiang, China
- Recruiting
- The Children's Hospital,Zhejiang University School of Medicine
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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.
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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
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Baseline, 26 weeks, 52 weeks
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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
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Baseline, 26 weeks, 52 weeks
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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
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Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks
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Body weight change before and after treatment
Time Frame: Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks
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Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks
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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
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Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks
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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
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Baseline, 26 weeks, 52 weeks
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Bone maturation ( bone age/ chronological age: BA/CA)
Time Frame: Baseline, 52 weeks
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Baseline, 52 weeks
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Change of IGF-1(Insulin-like growth factor 1) SDS
Time Frame: Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks
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Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks
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IGF-1/IGFBP-3 molar ratio
Time Frame: Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks
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Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks
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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
Terms related to this study
Additional Relevant MeSH Terms
- Pathologic Processes
- Nervous System Diseases
- Neurologic Manifestations
- Neurobehavioral Manifestations
- Disease
- Congenital Abnormalities
- Overnutrition
- Nutrition Disorders
- Genetic Diseases, Inborn
- Intellectual Disability
- Abnormalities, Multiple
- Chromosome Disorders
- Obesity
- Syndrome
- Prader-Willi Syndrome
- Physiological Effects of Drugs
- Hormones, Hormone Substitutes, and Hormone Antagonists
- Hormones
Other Study ID Numbers
- GenSci PWS CT
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
No
Studies a U.S. FDA-regulated device product
No
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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