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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT03554031
Other study ID # GenSci PWS CT
Secondary ID
Status Recruiting
Phase Phase 3
First received
Last updated
Start date April 14, 2018
Est. completion date January 1, 2020

Study information

Verified date May 2018
Source GeneScience Pharmaceuticals Co., Ltd.
Contact Xiaohua Feng
Phone 0431-85170552
Email fengxiaohua@gensci-china.com
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

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


Recruitment information / eligibility

Status Recruiting
Enrollment 30
Est. completion date January 1, 2020
Est. primary completion date January 2020
Accepts healthy volunteers No
Gender All
Age group 1 Month to 5 Years
Eligibility 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 Design


Related Conditions & MeSH terms


Intervention

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.

Locations

Country Name City State
China Beijing Children's Hospital, Capital Medical University Beijing
China Peking union medical college hospital Beijing
China Peking university first hospital Beijing
China The Children's Hospital,Zhejiang University School of Medicine Hangzhou Zhejiang
China Children's Hospital of Fudan University Shanghai
China Tongji medical college huazhong university of science & technology Wuhan Hubei

Sponsors (7)

Lead Sponsor Collaborator
GeneScience Pharmaceuticals Co., Ltd. Beijing Children's Hospital, Children's Hospital of Fudan University, gwcmc, Peking University First Hospital, The Children's Hospital, Zhejiang University School Of Medicine, Tongji medical college huazhong university of science & technology

Country where clinical trial is conducted

China, 

Outcome

Type Measure Description Time frame Safety issue
Primary The change of the total motor quotient calculated by Peabody Developmental Motor Scale before and after treatment Baseline, 26 weeks, 52 weeks
Secondary Gross motor quotient and fine motor quotient calculated by Peabody Developmental Motor Scale before and after treatment Baseline, 26 weeks, 52 weeks
Secondary Change of height standard deviation score (SDS) by chronological age before and after treatment Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks
Secondary Body weight change before and after treatment Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks
Secondary Change of BMI (Body mass index) standard deviation score before and after treatment Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks
Secondary 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 Baseline, 26 weeks, 52 weeks
Secondary Bone maturation ( bone age/ chronological age: BA/CA) Baseline, 52 weeks
Secondary Change of IGF-1(Insulin-like growth factor 1) SDS Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks
Secondary IGF-1/IGFBP-3 molar ratio Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks
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