Turner SyndromePhase 2NCT03189160
A Study of PEG-somatropin Injection to Treat Children of Turner Syndrome
A Phase 2 study testing PEG-rhGH low dose and PEG-rhGH high dose in people with Turner Syndrome, run by Changchun GeneScience Pharmaceutical Co., Ltd. at 9 sites in China.
Trial summary
This study aims to explore the optimal dose of pegylated recombinant human growth hormone (PEG-rhGH) injection to treat children of Turner syndrome (TS), preliminarily evaluate its safety and efficacy and provide scientific and reliable evidence for the medication dosage in Phase 3 clinical trial. Read more about this study
- Who is it for?
- Ages 2 to 18 years, women only
- Conditions the study is for
- Turner Syndrome
- What is being tested?
- PEG-rhGH low dose and PEG-rhGH high dose
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 180 participants
- Where
- 9 sitesChina
- Study dates
- Mar 2016to Mar 2018
- Phase
- Phase 2
- 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 2 to 18 years
- Women only
- No healthy volunteers
- Child, Adult
Who may be able to join
- Female
- Bone age <12 years
- Karyotype: 45, X; 45, X / 46, XXqi; 45, X / 46, XXr; 45, X / 46, XX; 46, XXqi; 46, XXpi; 45, X / 47, XXX; 46, XXp-; 45, X / 46, XXp-; 46, XXq-; 45X / 46, XXq-; 45, X / 46, XX / 47, XXX, etc. (count 50 or more cells);
- Facial appearance and abnormalities: Patients with at least one of the following signs, which include but are not limited to facial pigmented nevus, short neck, webbed neck, low posterior hairline, low-set ears, micrognathia, high-voulted arch, shield-like chest, cubitus valgus, genu valgum, short 4th and 5th metacarpals, nail dysplasia, scoliosis, ptosis and strabismus, cardiovascular abnormalities (such as aortic stenosis, bicuspid aortic valve and hypertension), reproductive abnormalities (such as primary gonadal dysfunction), renal abnormalities, thyroid hypofunction, middle ear lesion, etc.
- Short stature: height below -2.5SD of the mean height of the same age and gender.
- Pre-pubertal (Tanner Stage I ) patients
- No history of growth hormone treatment
- The subject and his/her guardian sign the informed consent (if the subject is incapable to sign the informed consent, his/her legal guardian shall sign the name of the subject instead)
Who cannot join
- Subjects with abnormal liver and kidney functions (ALT > upper limit of normal value; Cr > upper limit of normal value)
- Subjects positive for anti-HBc, HbsAg or HbeAg in Hepatitis B virus tests;
- Subjects with highly allergic constitution or allergy to proteins or investigational product or its excipient
- Subjects with systemic chronic disease and immune deficiency
- Patients diagnosed with tumor
- For patients whose tumor markers exceeding normal range in combination with other information, considering as potential high risks of tumor, they may be excluded from the treatment.
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- Patients with mental disease
- Subjects with impaired glucose regulation (IGR) (including impaired fasting glucose (IFG) and/or impaired glucose tolerance (IGT) ) or diabetes
- Subjects who took part in other clinical trials within 3 months
- Subjects who received medicines which may interfere GH secretion or GH function, or other hormones within 3 months (such as sex steroids, glucocorticoids, etc.)
- Other conditions which are unsuitable for this study in the opinion of the investigator.
Trial locations and contacts
9 sites in 1 country.
9 sites in 1 country
China9 sites, 9 recruiting
- Recruiting
Beijing Children's Hospital,Capital Medical University
Beijing
Chunxiu Gong, PhD
- Recruiting
First Hospital of Jilin University
Changchun, Jilin
Hongwei Du
- Recruiting
The Children's Hospital of Zhejiang University School of Medicine
Hangzhou, Zhejiang
Junfeng Fu
- Recruiting
Jiangxi Provincial Children's Hospital
Nanchang, Jiangxi
Yu Yang
- Recruiting
The First Affiliated Hospital with Nanjing Medical University
Nanjing, Jiangsu
Yuhua Hu
- Recruiting
Children's Hospital of Fudan University
Shanghai
Feihong Luo
- Recruiting
Shanghai Children's Hospital
Shanghai
Pin Li
- Recruiting
Xinhua Hospital of Shanghai Jiao Tong University School of Medicine
Shanghai
Jun Ye
- Recruiting
Affiliated Hospital of Jiangnan University
Wuxi, Jiangsu
Zhuangjian Xu
About this study
Official title: A Phase 2 Study of Pegylated Recombinant Human Growth Hormone Injection to Treat Children of Turner Syndrome
This study aims to explore the optimal dose of pegylated recombinant human growth hormone (PEG-rhGH) injection to treat children of Turner syndrome (TS), preliminarily evaluate its safety and efficacy and provide scientific and reliable evidence for the medication dosage in Phase 3 clinical trial.
- Conditions
- Turner Syndrome
- Being tested
- PEG-rhGH low dose, PEG-rhGH high dose, Non-treatment control group
- Medical subject headings
- Turner Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PEG-rhGH low doseBiological
Also called Polyethylene Glycol Recombinant Human Somatropin Injection
PEG-rhGH Injection 0.1 mg/kg/w by subcutaneous injection for 52 weeks.
PEG-rhGH high doseBiological
Also called Polyethylene Glycol Recombinant Human Somatropin Injection
PEG-rhGH Injection 0.2 mg/kg/w by subcutaneous injection for 52 weeks.
Non-treatment control groupOther
| Study group | Role | What they receive |
|---|---|---|
| PEG-rhGH low dose | Experimental | PEG-rhGH Injection (27IU/4.5mg/0.5ml/bottle) 0.1 mg/kg/w by subcutaneous injection for 52 weeks. Receives: Biological: PEG-rhGH low dose; Biological: PEG-rhGH high dose; Other: Non-treatment control group |
| PEG-rhGH high dose | Experimental | PEG-rhGH Injection (27IU/4.5mg/0.5ml/bottle) 0.2 mg/kg/w by subcutaneous injection for 52 weeks. Receives: Biological: PEG-rhGH low dose; Biological: PEG-rhGH high dose; Other: Non-treatment control group |
| Non-treatment control group | No intervention | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change of height standard deviation score before and after treatment (ΔHT SDS)
The change of height standard deviation score of chronological age before and after treatment
Measured: 52 weeks
Secondary outcomes (3)
Height Velocity
Annual growth rate at the end of treatment
Measured: 52 weeks
ΔBA/ΔCA
Bone maturation (changes of bone age/ chang of chronological age)
Measured: 52 weeks
IGF-1(Insulin-like growth factor 1) SDS
Measured: 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Jun 2017
- Study startMar 2016
- Primary completionMar 2018 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Turner 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 180 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Changchun GeneScience Pharmaceutical Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Beijing Children's Hospital, The First Affiliated Hospital with Nanjing Medical University, Shanghai Children's Hospital, Children's Hospital of Fudan University, Xinhua Hospital, Shanghai Jiao Tong University School of Medicine, The First Hospital of Jilin University, Jiangxi Province Children's Hospital, Affiliated Hospital of Jiangnan University, The Children's Hospital of Zhejiang University School of Medicine
- Responsible party
- Sponsor
- Investigators
- Chunxiu Gong, PhD, Principal investigator, Beijing Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03189160
- Sponsor's protocol ID
- GenSci 032 CT
- Status last verified
- Jun 2017
- First submitted
- 13 Jun 2017
- Last updated
- 12 Dec 2017
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Dec 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Study of Pegylated Recombinant Human Growth Hormone Injection to Treat Children of Turner Syndrome
- ClinicalTrials.gov ID
- NCT03189160
- Acronym / sponsor ID
- GenSci 032 CT
- Conditions
- Turner Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 180 participants (estimated)
- Lead sponsor
- Changchun GeneScience Pharmaceutical Co., Ltd.Industry (pharma, biotech, devices)
- Collaborators
- Beijing Children's Hospital, The First Affiliated Hospital with Nanjing Medical University, Shanghai Children's Hospital, Children's Hospital of Fudan University, Xinhua Hospital, Shanghai Jiao Tong University School of Medicine, The First Hospital of Jilin University, Jiangxi Province Children's Hospital, Affiliated Hospital of Jiangnan University, The Children's Hospital of Zhejiang University School of Medicine
- Study start
- Mar 2016
- Primary completion
- Mar 2018 (estimated)
- First posted
- 16 Jun 2017 (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.
This study aims to explore the optimal dose of pegylated recombinant human growth hormone (PEG-rhGH) injection to treat children of Turner syndrome (TS), preliminarily evaluate its safety and efficacy and provide scientific and reliable evidence for the medication dosage in Phase 3 clinical trial.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| PEG-rhGH low dose | Experimental | Biological: PEG-rhGH low dose, Biological: PEG-rhGH high dose, Other: Non-treatment control groupPEG-rhGH Injection (27IU/4.5mg/0.5ml/bottle) 0.1 mg/kg/w by subcutaneous injection for 52 weeks. |
| PEG-rhGH high dose | Experimental | Biological: PEG-rhGH low dose, Biological: PEG-rhGH high dose, Other: Non-treatment control groupPEG-rhGH Injection (27IU/4.5mg/0.5ml/bottle) 0.2 mg/kg/w by subcutaneous injection for 52 weeks. |
| Non-treatment control group | No intervention | – |
Who may qualify
- Ages
- 2 to 18 years
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (8 listed)
- Female
- Bone age <12 years
- Karyotype: 45, X; 45, X / 46, XXqi; 45, X / 46, XXr; 45, X / 46, XX; 46, XXqi; 46, XXpi; 45, X / 47, XXX; 46, XXp-; 45, X / 46, XXp-; 46, XXq-; 45X / 46, XXq-; 45, X / 46, XX / 47, XXX, etc. (count 50 or more cells);
- Facial appearance and abnormalities: Patients with at least one of the following signs, which include but are not limited to facial pigmented nevus, short neck, webbed neck, low posterior hairline, low-set ears, micrognathia, high-voulted arch, shield-like chest, cubitus valgus, genu valgum, short 4th and 5th metacarpals, nail dysplasia, scoliosis, ptosis and strabismus, cardiovascular abnormalities (such as aortic stenosis, bicuspid aortic valve and hypertension), reproductive abnormalities (such as primary gonadal dysfunction), renal abnormalities, thyroid hypofunction, middle ear lesion, etc.
- Short stature: height below -2.5SD of the mean height of the same age and gender.
Key exclusion criteria (11 listed)
- Subjects with abnormal liver and kidney functions (ALT > upper limit of normal value; Cr > upper limit of normal value)
- Subjects positive for anti-HBc, HbsAg or HbeAg in Hepatitis B virus tests;
- Subjects with highly allergic constitution or allergy to proteins or investigational product or its excipient
- Subjects with systemic chronic disease and immune deficiency
- Patients diagnosed with tumor
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 |
|---|---|---|
| PEG-rhGH low doseAlso: Polyethylene Glycol Recombinant Human Somatropin InjectionPEG-rhGH Injection 0.1 mg/kg/w by subcutaneous injection for 52 weeks. | Biological | PEG-rhGH high dose, PEG-rhGH low dose |
| PEG-rhGH high doseAlso: Polyethylene Glycol Recombinant Human Somatropin InjectionPEG-rhGH Injection 0.2 mg/kg/w by subcutaneous injection for 52 weeks. | Biological | PEG-rhGH high dose, PEG-rhGH low dose |
| Non-treatment control group | Other | PEG-rhGH high dose, PEG-rhGH low dose |
Primary outcome
Change of height standard deviation score before and after treatment (ΔHT SDS)
Time frame: 52 weeks
The change of height standard deviation score of chronological age before and after treatment
3 secondary outcomes
Height Velocity
Time frame: 52 weeks
ΔBA/ΔCA
Time frame: 52 weeks
IGF-1(Insulin-like growth factor 1) SDS
Time frame: 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 Dec 2017.
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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.
Chunxiu Gong, PhD principal investigator
Beijing Children's Hospital
9 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing PEG-rhGH low dose and PEG-rhGH high dose in people with Turner Syndrome, run by Changchun GeneScience Pharmaceutical Co., Ltd. at 9 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 11 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 Dec 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
Change of height standard deviation score before and after treatment (ΔHT SDS) (time frame: 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 Dec 2017.