DwarfismPhase 3NCT03635580
Efficacy and Safety of rhGH (Jintropin®) in Pediatric Participants With ISS
A Phase 3 study of Dwarfism, run by Changchun GeneScience Pharmaceutical Co., Ltd. at 9 sites in China.
Trial summary
Phase 1: To evaluate the safety and efficacy of 0.05mg/kg/d of rhGH (Jintropin®) in the treatment of children with idiopathic short stature (ISS) in 52 weeks. Phase 2: To evaluate the safety and efficacy of rhGH (Jintropin®) in the treatment of children with ISS in 2 years Read more about this study
- Who is it for?
- Ages 4 to 10 years
- Conditions the study is for
- Dwarfism
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 480 participants
- Where
- 9 sitesChina
- Study dates
- 11 Jun 2018to 30 Oct 2021
- 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 4 to 10 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Boys are between 4 and 10 years old age and girls are between 4 and 9 years old age;
- Height <-2.25 SD (Standard deviation) for chronological age;
- GH (Growth hormone) peak concentration ≥10.0 ng/mL in GH stimulation tests;
- The bone age (BA) ≤chronological age (CA)+6 months;
- Prepubertal Status (Tanner Stage I);
- Birth weight within the normal range;
- Growth hormone treatment-naive;
- Participants are willing and able to cooperate to complete scheduled visits, treatment plans and laboratory tests and other procedures, to sign informed consent.
Who cannot join
- Participants with abnormal liver and kidney functions (ALT > upper limit 1.5 times of normal value; Cr > upper limit of normal value);
- Participants are positive for anti-HBc, HbsAg or HbeAg in Hepatitis B virus tests;
- Participants with known highly allergic constitution or allergy to investigational product or its excipient;
- Participants with systemic chronic disease and immune deficiency;
- Participants diagnosed with tumor, or with potential high tumor risks such as tumor markers exceed normal range and some other relative information may be excluded from the treatment;
- Participants with mental disease;
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- Participants with other types of abnormal growth and development;
- Growth hormone deficiency (GHD) (confirmed by GH stimulation test);
- Turner syndrome (confirmed by karyotype test of girls);
- Noonan syndrome (hypertelorism, pectus carinatum, hypophrenia, frequently with skin disease and congenital heart disease, missense mutation of the protein tyrosine phosphatase, non-receptor type 11 (PTPN11) gene on chromosome 12 for half of the participants, for both male and female participants);
- Laron syndrome (confirmed by IGF-1 generation test);
- Small for gestational age ( the birth height or weight is below the tenth percentile or 2 SD, with catch-up growth uncompleted at 2 years old);
- Growth disorders caused by malnutrition or hypothyroidism (thyroid function test).
- Participants with impaired glucose regulation (IGR) (including impaired fasting glucose (IFG) and/or impaired glucose tolerance (IGT) ) or diabetes;
- BMI (Body mass index) ≥22kg/m²;
- Congenital skeletal abnormalities or scoliosis, claudication;
- Participants who took part in other clinical trials within 3 months;
- Participants who received medications which may interfere GH secretion or GH function, or other hormones within 3 months (such as sex steroids, glucocorticoids, etc.);
- Other conditions which is inappropriate 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
The First Hospital of Jilin University
Changchun, Jilin
- Recruiting
The First Affiliated Hospital, Sun Yat-sen University
Guangzhou, Guangdong
- Recruiting
The Children's Hospital, Zhejiang University School of Medicine
Hangzhou, Zhejiang
- Recruiting
Jiangsu Province Hospital
Nanjing, Jiangsu
- Recruiting
Children's Hospital of Fudan University
Shanghai
- Recruiting
Children's Hospital of Shanghai
Shanghai
- Recruiting
Children's Hospital of Shanxi
Taiyuan, Shanxi
- Recruiting
The Second Affiliated Hospital of Xi'an Jiaotong University
Xi’an, Shanxi
- Recruiting
Henan Children's Hospital
Zhengzhou, Henan
About this study
Official title: Phase 2 Study of Efficacy and Safety of rhGH (Jintropin®) in Pediatric Participants With ISS (Idiopathic Short Stature): a 52-week Multicenter, Open-label, Randomized, Negative Controlled Study Followed by a 52-week Open-label Period
Phase 1: To evaluate the safety and efficacy of 0.05mg/kg/d of rhGH (Jintropin®) in the treatment of children with idiopathic short stature (ISS) in 52 weeks.
Phase 2: To evaluate the safety and efficacy of rhGH (Jintropin®) in the treatment of children with ISS in 2 years
- Conditions
- Dwarfism
- Being tested
- Negative control
- Medical subject headings
- Dwarfism
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Negative controlOther
Untreated-control group
| Study group | Role | What they receive |
|---|---|---|
| rhGH/Jintropin AQ | Experimental | Jintropin AQ, injection, 30IU/10mg/3ml/cartridge, 0.05mg /kg/d in phase 1 and 0.05-0.07mg/kg/d in phase 2. Receives: Other: Negative control |
What is being measured
The primary outcome is the main question the study is designed to answer.
ΔHtSDSca (The change of height standard deviation score of chronological age before and after treatment)
ΔHtSDSca was the difference of HtSDSca before and after treatment, and HtSDSca was calculated by dividing the difference between the actual height of a patient and the mean height of the population for that chronological age by the standard deviation (SD) of the height of the population for that chronological age.
Measured: Baseline, 4,13,26,39,52,65,78,91,104 weeks after initiating treatment, 52 weeks in phase 1; 2 years in phase 2.
Secondary outcomes (4)
ΔHT (Change of height)
Measured: Baseline, 4,13,26,39,52,65,78,91,104 weeks after initiating treatment, 52 weeks in phase 1; 2 years in phase 2.
△BA/CA (bone age change / chronological age)
Measured: Baseline, 4,13,26,39,52,65,78,91,104 weeks after initiating treatment, 52 weeks in phase 1; 2 years in phase 2.
Yearly growth velocity
Measured: Baseline, 4,13,26,39,52,65,78,91,104 weeks after initiating treatment, 52 weeks in phase 1; 2 years in phase 2.
ΔIGF-1 SDS (Change of insulin-like growth factor 1 standard deviation score)
Measured: Baseline, 4,13,26,39,52,65,78,91,104 weeks after initiating treatment, 52 weeks in phase 1; 2 years in phase 2.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Aug 2018
- Study start11 Jun 2018
- Primary completionOct 2021 (estimated)
- Study completion30 Oct 2021 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for DwarfismStudies 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 480 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Changchun GeneScience Pharmaceutical Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- The Children's Hospital of Zhejiang University School of Medicine, First Affiliated Hospital, Sun Yat-Sen University, Second Affiliated Hospital of Xi'an Jiaotong University, Henan Provincial People's Hospital, Children's Hospital of Fudan University, The First Affiliated Hospital with Nanjing Medical University, Shanxi Provincial Maternity and Children's Hospital, The First Hospital of Jilin University, Shanghai Children's Hospital
- Responsible party
- Sponsor
- Investigators
- Junfen Fu, Doctor, Principal investigator, The Children's Hospital of Zhejiang University School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03635580
- Sponsor's protocol ID
- GenSci 046 CT
- Status last verified
- Aug 2018
- First submitted
- 15 Aug 2018
- Last updated
- 17 Aug 2018
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Yuan J, Fu J, Wei H, Zhang G, Xiao Y, Du H, Gu W, Li Y, Chen L, Luo F, Zhong Y, Gong H. A Randomized Controlled Phase 3 Study on the Efficacy and Safety of Recombinant Human Growth Hormone in Children With Idiopathic Short Stature. Front Endocrinol (Lausanne). 2022 Apr 29;13:864908. doi: 10.3389/fendo.2022.864908. eCollection 2022.
PubMed 35573994
Study at a glance
From the registry record, last updated by the sponsor on 17 Aug 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 2 Study of Efficacy and Safety of rhGH (Jintropin®) in Pediatric Participants With ISS (Idiopathic Short Stature): a 52-week Multicenter, Open-label, Randomized, Negative Controlled Study Followed by a 52-week Open-label Period
- ClinicalTrials.gov ID
- NCT03635580
- Acronym / sponsor ID
- GenSci 046 CT
- Conditions
- Dwarfism
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 480 participants (estimated)
- Lead sponsor
- Changchun GeneScience Pharmaceutical Co., Ltd.Industry (pharma, biotech, devices)
- Collaborators
- The Children's Hospital of Zhejiang University School of Medicine, First Affiliated Hospital, Sun Yat-Sen University, Second Affiliated Hospital of Xi'an Jiaotong University, Henan Provincial People's Hospital, Children's Hospital of Fudan University, The First Affiliated Hospital with Nanjing Medical University, Shanxi Provincial Maternity and Children's Hospital, The First Hospital of Jilin University, Shanghai Children's Hospital
- Study start
- 11 Jun 2018 (actual)
- Primary completion
- Oct 2021 (estimated)
- Study completion
- 30 Oct 2021 (estimated)
- First posted
- 17 Aug 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.
Phase 1: To evaluate the safety and efficacy of 0.05mg/kg/d of rhGH (Jintropin®) in the treatment of children with idiopathic short stature (ISS) in 52 weeks.
Phase 2: To evaluate the safety and efficacy of rhGH (Jintropin®) in the treatment of children with ISS in 2 years
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| rhGH/Jintropin AQ | Experimental | Other: Negative controlJintropin AQ, injection, 30IU/10mg/3ml/cartridge, 0.05mg /kg/d in phase 1 and 0.05-0.07mg/kg/d in phase 2. |
Who may qualify
- Ages
- 4 to 10 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (8 listed)
- Boys are between 4 and 10 years old age and girls are between 4 and 9 years old age;
- Height <-2.25 SD (Standard deviation) for chronological age;
- GH (Growth hormone) peak concentration ≥10.0 ng/mL in GH stimulation tests;
- The bone age (BA) ≤chronological age (CA)+6 months;
- Prepubertal Status (Tanner Stage I);
Key exclusion criteria (19 listed)
- Participants with abnormal liver and kidney functions (ALT > upper limit 1.5 times of normal value; Cr > upper limit of normal value);
- Participants are positive for anti-HBc, HbsAg or HbeAg in Hepatitis B virus tests;
- Participants with known highly allergic constitution or allergy to investigational product or its excipient;
- Participants with systemic chronic disease and immune deficiency;
- Participants diagnosed with tumor, or with potential high tumor risks such as tumor markers exceed normal range and some other relative information may be excluded from the treatment;
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 |
|---|---|---|
| Negative controlUntreated-control group | Other | rhGH/Jintropin AQ |
Primary outcome
ΔHtSDSca (The change of height standard deviation score of chronological age before and after treatment)
Time frame: Baseline, 4,13,26,39,52,65,78,91,104 weeks after initiating treatment, 52 weeks in phase 1; 2 years in phase 2.
ΔHtSDSca was the difference of HtSDSca before and after treatment, and HtSDSca was calculated by dividing the difference between the actual height of a patient and the mean height of the population for that chronological age by the standard deviation (SD) of the height of the population for that chronological age.
4 secondary outcomes
ΔHT (Change of height)
Time frame: Baseline, 4,13,26,39,52,65,78,91,104 weeks after initiating treatment, 52 weeks in phase 1; 2 years in phase 2.
△BA/CA (bone age change / chronological age)
Time frame: Baseline, 4,13,26,39,52,65,78,91,104 weeks after initiating treatment, 52 weeks in phase 1; 2 years in phase 2.
Yearly growth velocity
Time frame: Baseline, 4,13,26,39,52,65,78,91,104 weeks after initiating treatment, 52 weeks in phase 1; 2 years in phase 2.
ΔIGF-1 SDS (Change of insulin-like growth factor 1 standard deviation score)
Time frame: Baseline, 4,13,26,39,52,65,78,91,104 weeks after initiating treatment, 52 weeks in phase 1; 2 years in phase 2.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Aug 2018.
- Derived from this study: PubMed 35573994Yuan J, Fu J, Wei H, Zhang G, Xiao Y, Du H, Gu W, Li Y, Chen L, Luo F, Zhong Y, Gong H. A Randomized Controlled Phase 3 Study on the Efficacy and Safety of Recombinant Human Growth Hormone in Children With Idiopathic Short Stature. Front Endocrinol (Lausanne). 2022 Apr 29;13:864908. doi: 10.3389/fendo.2022.864908. eCollection 2022.
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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.
Junfen Fu, Doctor principal investigator
The Children's Hospital of Zhejiang University School of Medicine
9 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study of Dwarfism, 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 19 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 17 Aug 2018. 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 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?
ΔHtSDSca (The change of height standard deviation score of chronological age before and after treatment) (time frame: Baseline, 4,13,26,39,52,65,78,91,104 weeks after initiating treatment, 52 weeks in phase 1; 2 years in phase 2.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03635580, which also lists 1 linked publication.
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 17 Aug 2018.