Clinical trial NCT03554031: 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
Overall status: UNKNOWN
Phase: PHASE3
Conditions: Prader-Willi Syndrome
Lead sponsor: Changchun GeneScience Pharmaceutical Co., Ltd.
Sponsor class: INDUSTRY
Collaborators: name: Children's Hospital of Fudan University
name: Tongji medical college huazhong university of science & technology
name: Beijing Children's Hospital
name: Peking University First Hospital
name: Peking Union Medical College Hospital
name: The Children's Hospital of Zhejiang University School of Medicine
First posted: 2018-06-12
Enrollment: 30
Sex: ALL
Minimum age (years): 1
Maximum age (years): 5
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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


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

Primary outcomes:
measure: The change of the total motor quotient calculated by Peabody Developmental Motor Scale before and after treatment
timeFrame: Baseline, 26 weeks, 52 weeks
Secondary outcomes:
measure: Gross motor quotient and fine motor quotient calculated by Peabody Developmental Motor Scale before and after treatment
timeFrame: Baseline, 26 weeks, 52 weeks
measure: Change of height standard deviation score (SDS) by chronological age before and after treatment
timeFrame: Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks
measure: Body weight change before and after treatment
timeFrame: Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks
measure: Change of BMI (Body mass index) standard deviation score before and after treatment
timeFrame: Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks
measure: 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
timeFrame: Baseline, 26 weeks, 52 weeks
measure: Bone maturation ( bone age/ chronological age: BA/CA)
timeFrame: Baseline, 52 weeks
measure: Change of IGF-1(Insulin-like growth factor 1) SDS
timeFrame: Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks
measure: IGF-1/IGFBP-3 molar ratio
timeFrame: Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks
Other outcomes:
Locations:
city: Wuhan
country: China
facility: Tongji medical college huazhong university of science & technology
state: Hubei
city: Hangzhou
country: China
facility: The Children's Hospital,Zhejiang University School of Medicine
state: Zhejiang
city: Beijing
country: China
facility: Beijing Children's Hospital, Capital Medical University
city: Beijing
country: China
facility: Peking union medical college hospital
city: Beijing
country: China
facility: Peking university first hospital
city: Shanghai
country: China
facility: Children's Hospital of Fudan University
Study officials:
affiliation: Children's Hospital of Fudan University
name: Feihong Luo, Doctor
role: PRINCIPAL_INVESTIGATOR
References: