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

Prader-Willi Syndrome Clinical Trial

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03554031
• Other study ID #: GenSci PWS CT
• Status: Recruiting
• Phase: Phase 3
• 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
• 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

Related Conditions & MeSH terms

• Hyperphagia
• Prader-Willi Syndrome
• Syndrome

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