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

NCT03554031 | Unknown Phase 3

• 1 other identifier: GenSci PWS CT
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 6

Brief Summary

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

Conditions

Prader-Willi Syndrome

Outcome Measures

Primary Outcomes (1)

• The change of the total motor quotient calculated by Peabody Developmental Motor Scale before and after treatment

  Baseline, 26 weeks, 52 weeks

Secondary Outcomes (8)

• Gross motor quotient and fine motor quotient calculated by Peabody Developmental Motor Scale before and after treatment

  Baseline, 26 weeks, 52 weeks

• 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

• Body weight change before and after treatment

  Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks

• Change of BMI (Body mass index) standard deviation score before and after treatment

  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

  Baseline, 26 weeks, 52 weeks

Study Arms (1)

rhGH injection/Jintropin AQ

EXPERIMENTAL

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.

Drug: Recombinant Human Growth Hormone (rhGH) Injection

Interventions

Recombinant Human Growth Hormone (rhGH) Injection DRUG

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.

rhGH injection/Jintropin AQ

Eligibility Criteria

• Age: 1 Month - 5 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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;

Sponsors & Collaborators

• Changchun GeneScience Pharmaceutical Co., Ltd.: lead
• Children's Hospital of Fudan University: collaborator
• Tongji medical college huazhong university of science & technology: collaborator
• Beijing Children's Hospital: collaborator
• Peking University First Hospital: collaborator
• Peking Union Medical College Hospital: collaborator
• The Children's Hospital of Zhejiang University School of Medicine: collaborator

Study Sites (6)

Tongji medical college huazhong university of science & technology

Wuhan, Hubei, China

RECRUITING

The Children's Hospital,Zhejiang University School of Medicine

Hangzhou, Zhejiang, China

RECRUITING

Beijing Children's Hospital, Capital Medical University

Beijing, China

RECRUITING

Peking union medical college hospital

Beijing, China

RECRUITING

Peking university first hospital

Beijing, China

RECRUITING

Children's Hospital of Fudan University

Shanghai, China

RECRUITING

MeSH Terms

Conditions

Prader-Willi Syndrome

Interventions

Growth Hormone

Condition Hierarchy (Ancestors)

Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Chromosome Disorders; Genetic Diseases, Inborn; Imprinting Disorders; Obesity; Overweight; Overnutrition; Nutrition Disorders; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Pituitary Hormones, Anterior; Pituitary Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Peptides; Amino Acids, Peptides, and Proteins

Study Officials

• Feihong Luo, Doctor

  Children's Hospital of Fudan University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Xiaohua Feng

CONTACT

0431-85170552; fengxiaohua@gensci-china.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 30, 2018
• First Posted: June 12, 2018
• Study Start: April 14, 2018
• Primary Completion: January 1, 2020
• Study Completion: January 1, 2020
• Last Updated: June 12, 2018

Record last verified: 2018-05

Locations

CN (6)