Prader-Willi SyndromePhase 3NCT03554031
A Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone Injection in Patients With Prader-Willi Syndrome
A Phase 3 study testing Recombinant Human Growth Hormone (rhGH) Injection in people with Prader-Willi Syndrome, run by Changchun GeneScience Pharmaceutical Co., Ltd. at 6 sites in China.
Trial summary
To evaluate the effectiveness of rhGH (Recombinant human growth hormone) injection for improving motor development in patients with PWS. Read more about this study
- Who is it for?
- Ages 1 month to 5 years
- Conditions the study is for
- Prader-Willi Syndrome
- What is being tested?
- Recombinant Human Growth Hormone (rhGH) Injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 6 sitesChina
- Study dates
- 14 Apr 2018to 1 Jan 2020
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 month to 5 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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;
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- 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 locations and contacts
6 sites in 1 country.
6 sites in 1 country
China6 sites, 6 recruiting
- Recruiting
Beijing Children's Hospital, Capital Medical University
Beijing
- Recruiting
Peking union medical college hospital
Beijing
- Recruiting
Peking university first hospital
Beijing
- Recruiting
The Children's Hospital,Zhejiang University School of Medicine
Hangzhou, Zhejiang
- Recruiting
Children's Hospital of Fudan University
Shanghai
- Recruiting
Tongji medical college huazhong university of science & technology
Wuhan, Hubei
About this study
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
To evaluate the effectiveness of rhGH (Recombinant human growth hormone) injection for improving motor development in patients with PWS.
- Conditions
- Prader-Willi Syndrome
- Being tested
- Recombinant Human Growth Hormone (rhGH) Injection
- Medical subject headings
- Prader-Willi Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Recombinant Human Growth Hormone (rhGH) InjectionDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Recombinant Human Growth Hormone (rhGH) Injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
The change of the total motor quotient calculated by Peabody Developmental Motor Scale before and after treatment
Measured: 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
Measured: Baseline, 26 weeks, 52 weeks
Change of height standard deviation score (SDS) by chronological age before and after treatment
Measured: Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks
Body weight change before and after treatment
Measured: Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks
Change of BMI (Body mass index) standard deviation score before and after treatment
Measured: 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
Measured: Baseline, 26 weeks, 52 weeks
Bone maturation ( bone age/ chronological age: BA/CA)
Measured: Baseline, 52 weeks
Change of IGF-1(Insulin-like growth factor 1) SDS
Measured: Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks
IGF-1/IGFBP-3 molar ratio
Measured: Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Jun 2018
- Study start14 Apr 2018
- Primary completionJan 2020 (estimated)
- Study completion1 Jan 2020 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Prader-Willi SyndromeStudies of the same condition, open and under way.
- More trials run by Changchun GeneScience Pharmaceutical Co., Ltd.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Changchun GeneScience Pharmaceutical Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Children's Hospital of Fudan University, Huazhong University of Science and Technology, Beijing Children's Hospital, Peking University First Hospital, Peking Union Medical College Hospital, The Children's Hospital of Zhejiang University School of Medicine
- Responsible party
- Sponsor
- Investigators
- Feihong Luo, Doctor, Principal investigator, Children's Hospital of Fudan University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03554031
- Sponsor's protocol ID
- GenSci PWS CT
- Status last verified
- May 2018
- First submitted
- 30 May 2018
- Last updated
- 12 Jun 2018
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Jun 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03554031
- Acronym / sponsor ID
- GenSci PWS CT
- Conditions
- Prader-Willi Syndrome
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Changchun GeneScience Pharmaceutical Co., Ltd.Industry (pharma, biotech, devices)
- Collaborators
- Children's Hospital of Fudan University, Huazhong University of Science and Technology, Beijing Children's Hospital, Peking University First Hospital, Peking Union Medical College Hospital, The Children's Hospital of Zhejiang University School of Medicine
- Study start
- 14 Apr 2018 (actual)
- Primary completion
- Jan 2020 (estimated)
- Study completion
- 1 Jan 2020 (estimated)
- First posted
- 12 Jun 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
To evaluate the effectiveness of rhGH (Recombinant human growth hormone) injection for improving motor development in patients with PWS.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| rhGH injection/Jintropin AQ | Experimental | Drug: Recombinant Human Growth Hormone (rhGH) InjectionDrug: 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. |
Who may qualify
- Ages
- 1 month to 5 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (8 listed)
- 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;
Key exclusion criteria (16 listed)
- 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;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Recombinant Human Growth Hormone (rhGH) InjectionDrug: 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. | Drug | rhGH injection/Jintropin AQ |
Primary outcome
The change of the total motor quotient calculated by Peabody Developmental Motor Scale before and after treatment
Time frame: Baseline, 26 weeks, 52 weeks
8 secondary outcomes
Gross motor quotient and fine motor quotient calculated by Peabody Developmental Motor Scale before and after treatment
Time frame: 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
Body weight change before and after treatment
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
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
Bone maturation ( bone age/ chronological age: BA/CA)
Time frame: 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
IGF-1/IGFBP-3 molar ratio
Time frame: Baseline, 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Jun 2018.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Feihong Luo, Doctor principal investigator
Children's Hospital of Fudan University
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Recombinant Human Growth Hormone (rhGH) Injection in people with Prader-Willi Syndrome, run by Changchun GeneScience Pharmaceutical Co., Ltd. at 6 sites in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 12 Jun 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Xiaohua Feng (0431-85170552, fengxiaohua@gensci-china.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The change of the total motor quotient calculated by Peabody Developmental Motor Scale before and after treatment (time frame: Baseline, 26 weeks, 52 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Jun 2018.