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

GeneScience Pharmaceuticals (GenSci)

Status and phase: Unknown; Phase 3

Conditions: Prader-Willi Syndrome

Treatments: Drug: Recombinant Human Growth Hormone (rhGH) Injection

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT03554031

GenSci PWS CT

Details and patient eligibility

About

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

Enrollment: 30 estimated patients

Sex: All

Ages: 1 month to 5 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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30 participants in 1 patient group

rhGH injection/Jintropin AQ

Experimental group

Description:

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.

Treatment:

Drug: Recombinant Human Growth Hormone (rhGH) Injection