Idiopathic Short StaturePhase 3NCT06927310
The Efficacy and Safety of Inpegsomatropin Injection in Children With Idiopathic Short Stature
A Phase 3 study testing Inpegsomatropin Injection and Recombinant Human Growth Hormone Injection in people with Idiopathic Short Stature, run by Xiamen Amoytop Biotech Co., Ltd. at 38 sites in China.
Trial summary
This is a multicenter, randomized, open-label, active-controlled Phase III clinical trial to evaluate the efficacy and safety of Inpegsomatropin injection，once a week，compared with recombinant human growth hormone (rhGH) in children with idiopathic short stature (ISS). Read more about this study
- Who is it for?
- Ages 3 to 11 years
- Conditions the study is for
- Idiopathic Short Stature
- What is being tested?
- Inpegsomatropin Injection and Recombinant Human Growth Hormone Injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 300 participants
- Where
- 38 sitesChina
- Study dates
- 28 Apr 2025to Jun 2028
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
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 3 to 11 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Prepubertal children: boys aged ≥3 years and <11 years at screening with testicular volume <4 mL; girls aged ≥3 years and <10 years at screening with breast development at Tanner Stage I, i.e., no palpable breast glandular tissue.
- Compared to chronological age, bone age is advanced by no more than 1 year or delayed by no more than 2 years (i.e., -2 years ≤ bone age - chronological age ≤ 1 year).
- Height at screening is below -2 standard deviations (SD) from the mean for age and sex, with height reference to Appendix 1.
- Body mass index (BMI) is within the 5th to 95th percentile for age and sex of healthy children, with reference to Appendix 2.
- Peak GH level ≥10.0 ng/ml in at least one GH stimulation test.
- No prior systemic pharmacological treatment for growth promotion (continuous use ≥1 month), including but not limited to growth hormone, insulin-like growth factor 1 (IGF-1), etc.
- The legal guardian understands and signs the informed consent form. If the participants is ≥8 years old, they must also sign the informed consent form. For participants under 8 years old who are able to express their consent, their assent should be clearly documented.
Who cannot join
- Individuals with closed epiphyses;
- Other types of growth and developmental abnormalities, including confirmed or highly suspected growth hormone deficiency (GHD), Noonan syndrome, Prader-Willi syndrome, Russell-Silver syndrome, Turner syndrome, small for gestational age, short stature due to SHOX gene abnormalities, growth delay due to malnutrition, growth delay due to hypothyroidism, and other short statures with a clear etiology; genetic testing results must be obtained before randomization to exclude short stature caused by definite genetic abnormalities;
- Individuals who have participated in any other clinical trial within 3 months prior to screening and have received pharmacological or non-pharmacological interventions;
- Individuals who received inhaled corticosteroids for more than 2 consecutive weeks, or oral or intravenous corticosteroids for more than 1 consecutive week, within the 3 months prior to screening;
- Individuals who are currently receiving or require long-term treatment with other therapies that may affect growth, including but not limited to methylphenidate, sex hormones, gonadotropin-releasing hormone analogs, aromatase inhibitors, anabolic agents, insulin, etc.;
- Individuals with abnormal liver or kidney function at screening (ALT > 1.5 times the upper limit of normal, Cr > upper limit of normal);
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- Individuals diagnosed with diabetes mellitus, or with fasting blood glucose ≥6.1 mmol/L on two consecutive occasions;
- Individuals with chronic infectious diseases, such as chronic hepatitis B;
- Individuals with systemic chronic diseases, such as moderate to severe anemia, malnutrition, hypothyroidism, chronic kidney disease, cardiovascular diseases (e.g., dilated cardiomyopathy, etc.), psychiatric and psychological disorders, or those with congenital anomalies requiring clinical intervention as determined by the investigator;
- Individuals with congenital skeletal abnormalities, or those with scoliosis exceeding 15°, limping, or a history of slipped capital femoral epiphysis;
- Individuals with a history of increased intracranial pressure;
- Individuals with a history of malignancy or currently having active malignancy, including intracranial tumors;
- Individuals with severe allergic constitutions, or known allergies to growth hormone or its excipients;
- Individuals with any other disease that the investigator deems may endanger the subject's safety or compromise compliance with the study protocol.
Trial locations and contacts
38 sites in 1 country. Contact the study team or a site near you to ask about taking part.
38 sites in 1 country
China38 sites, 38 recruiting
- Recruiting
Capital Institute of Pediatrics, Beijing (SHOUER)
Beijing, Beijing Municipality
Fuying Song
- Recruiting
Hunan Children's Hospital
Changsha, Hunan
Yan Zhong
- Recruiting
Chengdu Women's and Children's Central Hospital
Chengdu, Sichuan
Xinran Cheng
- Recruiting
West China Second University Hospital, Sichuan University
Chengdu, Sichuan
Fan Yang
- Recruiting
Children's Hospital of Chongqing Medical University
Chongqing, Chongqing Municipality
Min Zhu
- Recruiting
Guangzhou Women and Children's Medical Center
Guangzhou, Guangdong
Wen Zhang
- Recruiting
Sun Yat-sen Memorial Hospital, Sun Yat-sen University
Guangzhou, Guangdong
Liyang Liang
- Recruiting
The Third Affiliated Hospital, Sun Yat-sen University
Guangzhou, Guangdong
Shunye Zhu
- Recruiting
Sanya Central Hospital
Hainan
Hua Zhang
- Recruiting
Hangzhou First People's Hospital
Hangzhou
Chunming Jiang
- Recruiting
Zhejiang Provincial People's Hospital
Hangzhou
Xiaoming Luo
- Recruiting
Anhui Provincial Children's Hospital
Hefei, Anhui
Yuqing Chen
- Recruiting
The Second Affiliated Hospital of Anhui Medical University
Hefei, Anhui
Deyun Liu
- Recruiting
The First Affiliated Hospital of Henan University of Science and Technology
Henan
Dan Lou
- Recruiting
Jiaxing First Hospital
Jiaxing
Shunfeng Mao
- Recruiting
Affiliated Hospital of Jining Medical University
Jining
Meiyun Xin
- Recruiting
Kunming Children's Hospital
Kunming
Na Tao
- Recruiting
Gansu Provincial Maternity and Child Care Hospital
Lanzhou, Gansu
Bo Sun
- Recruiting
Jiangxi Provincial Children's Hospital
Nanchang, Jiangxi
Yu Yang
- Recruiting
Nanjing Children's Hospital
Nanjing
Wei Gu
- Recruiting
The Affiliated Women's and Children's Hospital of Ningbo University
Ningbo, Zhejiang
Shuxia Ding
- Recruiting
Qingdao Women and Children's Hospital
Qingdao
Tang Li
- Recruiting
Children's Hospital of Shanghai
Shanghai, Shanghai Municipality
Pin Li
- Recruiting
Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine
Shanghai
Wenli Lu
- Recruiting
Shanghai Children's Medical Center
Shanghai
Xiumin Wang
Yu Ding
- Recruiting
Xinhua Hospital Affiliated to Shanghai Jiao Tong University School of Medicine
Shanghai
Huiwen Zhang
- Recruiting
The First Hospital of Hebei Medical University
Shijiazhuang
Huijuan Ma
- Recruiting
Children's Hospital Affiliated to Soochow University
Suzhou, Jiangsu
Haiying Wu
- Recruiting
The Second Affiliated Hospital and Yuying Children's Hospital of Wenzhou Medical University
Wenzhou, Zhejiang
Xiaoou Shan
- Recruiting
Wuhan Children's Hospital
Wuhan, Hubei
Hui Yao
- Recruiting
Renmin Hospital of Wuhan University
Wuhan
Jingping Ye
- Recruiting
The Second Affiliated Hospital of Xi'an Jiaotong University
Xi'an
Erdi Xu
- Recruiting
The First Affiliated Hospital of Xiamen University
Xiamen, Fujian
Qun Lian
- Recruiting
Xiangyang Central Hospital
Xiangyang
Rui Pan
- Recruiting
The First Affiliated Hospital of Xinjiang Medical University
Xinjiang
Miregul Mamat
- Recruiting
Yuncheng Central Hospital
Yuncheng
Sufang Chang
- Recruiting
Henan Children's Hospital Zhengzhou Children's Hospital
Zhengzhou, Henan
Haiyan Wei
- Recruiting
Zhuzhou Central Hospital
Zhuzhou
Xianglan Wen
About this study
Official title: A Multicenter, Randomized, Open-Label, Active-Controlled Phase III Clinical Study to Evaluate the Efficacy and Safety of Inpegsomatropin Injection, s.c Once a Week, Compared With Recombinant Human Growth Hormone (rhGH) Injection in Children With Idiopathic Short Stature (ISS).
This is a multicenter, randomized, open-label, active-controlled Phase III clinical trial to evaluate the efficacy and safety of Inpegsomatropin injection，once a week，compared with recombinant human growth hormone (rhGH) in children with idiopathic short stature (ISS). It plans to enroll 300 children with ISS, who will be randomized , stratified by gender and age, and assigned to either the experimental group or the positive controlled group. Each participant will undergo a screening period (up to 12 weeks), a treatment period (52 weeks), and a post-treatment follow-up period (5 weeks). And the safety and efficacy will be evaluated.
- Conditions
- Idiopathic Short Stature
- Being tested
- Inpegsomatropin Injection, Recombinant Human Growth Hormone Injection
- Keywords
- Inpegsomatropin Injection, Recombinant Human Growth Hormone Injection
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Inpegsomatropin InjectionDrug
Inpegsomatropin injection, 280 μg/kg/week, s.c., once weekly, for 52 consecutive weeks.
Recombinant Human Growth Hormone InjectionDrug
Recombinant Human Growth Hormone Injection, 350 μg/kg/week, s.c., divided into 7 doses, i.e., 50 μg/kg/day (0.15 IU/kg/day), once daily for 52 consecutive weeks.
| Study group | Role | What they receive |
|---|---|---|
| Inpegsomatropin group | Experimental | Not described Receives: Drug: Inpegsomatropin Injection |
| rhGH group | Active comparator | Not described Receives: Drug: Recombinant Human Growth Hormone Injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Growth velocity (HV, cm/year).
Measured: Week 52
Secondary outcomes (4)
Change in height standard deviation score from baseline (△HT SDS)
Measured: From baseline to all follow-up time points at Week 52
Change in growth velocity from baseline (△HV)
Measured: From baseline to all follow-up time points at Week 52
Change in insulin-like growth factor 1 standard deviation score from baseline (△IGF-1 SDS)
Measured: From baseline to all follow-up time points at Week 52
The ratio of the change in bone age to the change in chronological age (△BA/△CA)
Measured: From baseline to all follow-up time points at Week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Apr 2025
- Study start28 Apr 2025
- Primary completionMar 2026 (estimated)
- Study completionJun 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Idiopathic Short StatureStudies of the same condition, open and under way.
- More trials run by Xiamen Amoytop Biotech 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
- 300 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Xiamen Amoytop Biotech Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Tongji Hospital
- Responsible party
- Sponsor
- Investigators
- Xiaoping Luo, Ph.D, Principal investigator, Tongji Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06927310
- Sponsor's protocol ID
- PEGPESEN-4-2-001
- Status last verified
- Mar 2026
- First submitted
- 6 Apr 2025
- Last updated
- 11 Mar 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Randomized, Open-Label, Active-Controlled Phase III Clinical Study to Evaluate the Efficacy and Safety of Inpegsomatropin Injection, s.c Once a Week, Compared With Recombinant Human Growth Hormone (rhGH) Injection in Children With Idiopathic Short Stature (ISS).
- ClinicalTrials.gov ID
- NCT06927310
- Acronym / sponsor ID
- PEGPESEN-4-2-001
- Conditions
- Idiopathic Short Stature
- Keywords
- Inpegsomatropin Injection, Recombinant Human Growth Hormone Injection
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 300 participants (estimated)
- Lead sponsor
- Xiamen Amoytop Biotech Co., Ltd.Industry (pharma, biotech, devices)
- Collaborators
- Tongji Hospital
- Study start
- 28 Apr 2025 (actual)
- Primary completion
- Mar 2026 (estimated)
- Study completion
- Jun 2028 (estimated)
- First posted
- 15 Apr 2025 (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 is a multicenter, randomized, open-label, active-controlled Phase III clinical trial to evaluate the efficacy and safety of Inpegsomatropin injection，once a week，compared with recombinant human growth hormone (rhGH) in children with idiopathic short stature (ISS). It plans to enroll 300 children with ISS, who will be randomized , stratified by gender and age, and assigned to either the experimental group or the positive controlled group. Each participant will undergo a screening period (up to 12 weeks), a treatment period (52 weeks), and a post-treatment follow-up period (5 weeks). And the safety and efficacy will be evaluated.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Inpegsomatropin group | Experimental | Drug: Inpegsomatropin Injection |
| rhGH group | Active comparator | Drug: Recombinant Human Growth Hormone Injection |
Who may qualify
- Ages
- 3 to 11 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (7 listed)
- Prepubertal children: boys aged ≥3 years and <11 years at screening with testicular volume <4 mL; girls aged ≥3 years and <10 years at screening with breast development at Tanner Stage I, i.e., no palpable breast glandular tissue.
- Compared to chronological age, bone age is advanced by no more than 1 year or delayed by no more than 2 years (i.e., -2 years ≤ bone age - chronological age ≤ 1 year).
- Height at screening is below -2 standard deviations (SD) from the mean for age and sex, with height reference to Appendix 1.
- Body mass index (BMI) is within the 5th to 95th percentile for age and sex of healthy children, with reference to Appendix 2.
- Peak GH level ≥10.0 ng/ml in at least one GH stimulation test.
Key exclusion criteria (14 listed)
- Individuals with closed epiphyses;
- Other types of growth and developmental abnormalities, including confirmed or highly suspected growth hormone deficiency (GHD), Noonan syndrome, Prader-Willi syndrome, Russell-Silver syndrome, Turner syndrome, small for gestational age, short stature due to SHOX gene abnormalities, growth delay due to malnutrition, growth delay due to hypothyroidism, and other short statures with a clear etiology; genetic testing results must be obtained before randomization to exclude short stature caused by definite genetic abnormalities;
- Individuals who have participated in any other clinical trial within 3 months prior to screening and have received pharmacological or non-pharmacological interventions;
- Individuals who received inhaled corticosteroids for more than 2 consecutive weeks, or oral or intravenous corticosteroids for more than 1 consecutive week, within the 3 months prior to screening;
- Individuals who are currently receiving or require long-term treatment with other therapies that may affect growth, including but not limited to methylphenidate, sex hormones, gonadotropin-releasing hormone analogs, aromatase inhibitors, anabolic agents, insulin, etc.;
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 |
|---|---|---|
| Inpegsomatropin InjectionInpegsomatropin injection, 280 μg/kg/week, s.c., once weekly, for 52 consecutive weeks. | Drug | Inpegsomatropin group |
| Recombinant Human Growth Hormone InjectionRecombinant Human Growth Hormone Injection, 350 μg/kg/week, s.c., divided into 7 doses, i.e., 50 μg/kg/day (0.15 IU/kg/day), once daily for 52 consecutive weeks. | Drug | rhGH group |
Primary outcome
Growth velocity (HV, cm/year).
Time frame: Week 52
4 secondary outcomes
Change in height standard deviation score from baseline (△HT SDS)
Time frame: From baseline to all follow-up time points at Week 52
Change in growth velocity from baseline (△HV)
Time frame: From baseline to all follow-up time points at Week 52
Change in insulin-like growth factor 1 standard deviation score from baseline (△IGF-1 SDS)
Time frame: From baseline to all follow-up time points at Week 52
The ratio of the change in bone age to the change in chronological age (△BA/△CA)
Time frame: From baseline to all follow-up time points at Week 52
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Mar 2026.
- Individual participant dataSharing planned: No
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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.
Xiaoping Luo, Ph.D principal investigator
Tongji Hospital
38 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 Inpegsomatropin Injection and Recombinant Human Growth Hormone Injection in people with Idiopathic Short Stature, run by Xiamen Amoytop Biotech Co., Ltd. at 38 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 7 inclusion and 14 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: looking for participants now, as of the sponsor's last update on 11 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 38 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Xiaoping Luo, Ph.D (15671671188, xpluo@tjh.tjmu.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Growth velocity (HV, cm/year). (time frame: Week 52). 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 11 Mar 2026.