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

May 30, 2018 updated by: GeneScience Pharmaceuticals Co., Ltd.

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

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

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Unknown

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Anticipated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
30

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • China
      • Beijing, China
        • Recruiting
        • Peking Union Medical College Hospital
      • Beijing, China
        • Recruiting
        • Peking University First Hospital
      • Beijing, China
        • Recruiting
        • Beijing Children's Hospital, Capital Medical University
      • Shanghai, China
        • Recruiting
        • Children's Hospital of Fudan University
    • Hubei
      • Wuhan, Hubei, China
        • Recruiting
        • Tongji Medical College Huazhong University of Science & Technology
    • Zhejiang
      • Hangzhou, Zhejiang, China
        • Recruiting
        • The Children's Hospital,Zhejiang University School of Medicine

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

1 month to 5 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
The change of the total motor quotient calculated by Peabody Developmental Motor Scale before and after treatment
Time Frame: Baseline, 26 weeks, 52 weeks
Baseline, 26 weeks, 52 weeks

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Time Frame
Gross motor quotient and fine motor quotient calculated by Peabody Developmental Motor Scale before and after treatment
Time Frame: Baseline, 26 weeks, 52 weeks
Baseline, 26 weeks, 52 weeks
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
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
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
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
Baseline, 26 weeks, 52 weeks
Bone maturation ( bone age/ chronological age: BA/CA)
Time Frame: Baseline, 52 weeks
Baseline, 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
Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks
IGF-1/IGFBP-3 molar ratio
Time Frame: Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks
Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
GeneScience Pharmaceuticals Co., Ltd.

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
Peking University First Hospital
Peking Union Medical College Hospital
Children's Hospital of Fudan University
Beijing Children's Hospital
The Children's Hospital of Zhejiang University School of Medicine
Tongji medical college huazhong university of science & technology

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
April 14, 2018

Primary Completion (Anticipated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
January 1, 2020

Study Completion (Anticipated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
January 1, 2020

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
May 30, 2018

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
May 30, 2018

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
June 12, 2018

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
June 12, 2018

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
May 30, 2018

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
May 1, 2018

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • GenSci PWS CT

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Cookie Settings

We use cookies to ensure that we give you the best experience on our website. For more details carefully read our Privacy and Cookies Policy. ...>>>You can change your preferences or withdraw your consent at any time by deleting the cookies from your website or computer as described in the policy. Please accept it and choose your preferences by ticking the corresponding boxes in the "Manage Settings" section below. Please carefully read our Terms of Service before you proceed with using any part of this website.
«Manage Settings