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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03554031
• Date of registration: 30/05/2018
• Prospective Registration: No
• Primary sponsor: GeneScience Pharmaceuticals Co., Ltd.
• Public title: A Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone Injection in Patients With Prader-Willi Syndrome
• Scientific 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
• Date of first enrolment: April 14, 2018
• Target sample size: 30
• Recruitment status: Unknown status
• URL: https://clinicaltrials.gov/show/NCT03554031
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

China

Contacts

• Name: Xiaohua Feng

• Name: Feihong Luo, Doctor
• Affiliation: Children's Hospital of Fudan University

Key inclusion & exclusion 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



Age minimum: 1 Month
Age maximum: 5 Years
Gender: All

Health Condition(s) or Problem(s) studied

Prader-Willi Syndrome

Intervention(s)

Drug: Recombinant Human Growth Hormone (rhGH) Injection

Primary Outcome(s)

The change of the total motor quotient calculated by Peabody Developmental Motor Scale before and after treatment [Time Frame: Baseline, 26 weeks, 52 weeks]

Secondary Outcome(s)

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]

Change of IGF-1(Insulin-like growth factor 1) SDS [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]

Bone maturation ( bone age/ chronological age: BA/CA) [Time Frame: Baseline, 52 weeks]

Gross motor quotient and fine motor quotient calculated by Peabody Developmental Motor Scale before and after treatment [Time Frame: Baseline, 26 weeks, 52 weeks]

IGF-1/IGFBP-3 molar ratio [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]

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]

Secondary ID(s)

GenSci PWS CT

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Beijing Children's Hospital

Peking University First Hospital

Children's Hospital of Fudan University

The Children's Hospital of Zhejiang University School of Medicine

Tongji medical college huazhong university of science & technology

Peking Union Medical College Hospital