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

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ClinicalTrials.gov Identifier: NCT03554031
Recruitment Status : Recruiting
First Posted : June 12, 2018
Last Update Posted : June 12, 2018
Sponsor:
Collaborators:
Children's Hospital of Fudan University
Tongji medical college huazhong university of science & technology
Beijing Children's Hospital
Peking University First Hospital
Peking Union Medical College Hospital
The Children's Hospital of Zhejiang University School of Medicine
Information provided by (Responsible Party):
GeneScience Pharmaceuticals Co., Ltd.

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

Condition or disease Intervention/treatment Phase
Prader-Willi Syndrome Drug: Recombinant Human Growth Hormone (rhGH) Injection Phase 3

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : April 14, 2018
Estimated Primary Completion Date : January 2020
Estimated Study Completion Date : January 1, 2020


Arm Intervention/treatment
Experimental: rhGH injection/Jintropin AQ
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
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.




Primary Outcome Measures :
  1. 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 Measures :
  1. Gross motor quotient and fine motor quotient calculated by Peabody Developmental Motor Scale before and after treatment [ Time Frame: Baseline, 26 weeks, 52 weeks ]
  2. 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 ]
  3. Body weight change before and after treatment [ Time Frame: Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks ]
  4. 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 ]
  5. 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 ]
  6. Bone maturation ( bone age/ chronological age: BA/CA) [ Time Frame: Baseline, 52 weeks ]
  7. Change of IGF-1(Insulin-like growth factor 1) SDS [ Time Frame: Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks ]
  8. IGF-1/IGFBP-3 molar ratio [ Time Frame: Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks ]


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Ages Eligible for Study:   1 Month to 5 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03554031


Contacts
Contact: Xiaohua Feng 0431-85170552 fengxiaohua@gensci-china.com

Locations
China, Hubei
Tongji medical college huazhong university of science & technology Recruiting
Wuhan, Hubei, China
China, Zhejiang
The Children's Hospital,Zhejiang University School of Medicine Recruiting
Hangzhou, Zhejiang, China
China
Beijing Children's Hospital, Capital Medical University Recruiting
Beijing, China
Peking union medical college hospital Recruiting
Beijing, China
Peking university first hospital Recruiting
Beijing, China
Children's Hospital of Fudan University Recruiting
Shanghai, China
Sponsors and Collaborators
GeneScience Pharmaceuticals Co., Ltd.
Children's Hospital of Fudan University
Tongji medical college huazhong university of science & technology
Beijing Children's Hospital
Peking University First Hospital
Peking Union Medical College Hospital
The Children's Hospital of Zhejiang University School of Medicine
Investigators
Principal Investigator: Feihong Luo, Doctor Children's Hospital of Fudan University

Responsible Party: GeneScience Pharmaceuticals Co., Ltd.
ClinicalTrials.gov Identifier: NCT03554031     History of Changes
Other Study ID Numbers: GenSci PWS CT
First Posted: June 12, 2018    Key Record Dates
Last Update Posted: June 12, 2018
Last Verified: May 2018

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Prader-Willi Syndrome
Syndrome
Disease
Pathologic Processes
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Abnormalities, Multiple
Congenital Abnormalities
Chromosome Disorders
Genetic Diseases, Inborn
Obesity
Overnutrition
Nutrition Disorders
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs