Idiopathic Short StaturePhase 2NCT05838885
A Trial of YPEG-rhGH in Children With Short Stature
A Phase 2 study testing YPEG-rhGH and rhGH in people with Idiopathic Short Stature and Small for Gestational Age, run by Xiamen Amoytop Biotech Co., Ltd. at 19 sites in China.
Trial summary
To explore the dose-response relationship between pharmacokinetics and pharmacodynamics of Y- Shaped Pegylated growth hormone injection (YPEG-GH) in children with short stature (idiopathic short stature (ISS), small for gestational age (SGA), Turner syndrome (TS)). Read more about this study
- Who is it for?
- Ages 4 to 11 years
- Conditions the study is for
- Idiopathic Short Stature, Small for Gestational Age, Turner Syndrome
- What is being tested?
- YPEG-rhGH and rhGH
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 78 participants
- Where
- 19 sitesChina
- Study dates
- 15 Feb 2022to 19 Dec 2023
- 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 4 to 11 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Prepubertal (Tanner I), aged older than 4 years and younger than10 years for girls and 11 years for boys.
- Body weight: 12kg ≤ body weight ≤ 50kg.
- For children with idiopathic short stature: a) Birth length and weight were at the 10th percentile and above of normal reference values for infants of the same gestational age and sex; b) Height at screening was 2.0 standard deviations (SD) below the mean height for chronological age and sex c) Exclude other causes such as systemic diseases, other endocrine diseases, nutritional diseases, chromosomal abnormalities, skeletal dysplasia, psycho-emotional disorders, etc. were excluded d) GH peak ≥10.0ng/ml confirmed by two different drug GH provocation tests; e) Bone age (BA)-chronological age (CA) ≤1 year.
- For children with small for gestational age: a) Birth length and weight were at the 10th percentile and below the normal reference values for infants if the same gestational age and sex; b) Gestational age at birth ≥ 24 weeks; c) Height at screening was below -2 SD of the mean for the same age and sex, and please refer to the protocol annex 1 for height.
- For children with Turner syndrome: a) Chromosome 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-; 45, x/46, XXq-; 45, X/46, XX/47, XXX, etc.; b) Having at least one specific physical characteristic: Including but not limited to low posterior hairline, facial skin nevus, neck flips, short neck, low ear position, small jaw, high palatal arch, shield chest, wide breast spacing, elbow ectropion, knee ectropion, short 4th and 5th metacarpal, nail dysplasia, scoliosis, ptosis, strabismus, cardiovascular system abnormalities such as aortic stenosis, bicuspid aortic valve, hypertension, and reproductive system abnormalities such as primary gonadal insufficiency, renal malformation, hypothyroidism and middle ear disease; c) The height at screening was below the mean -2SD of the same age and gender, and please refer to the protocol annex 1 for height.
- Understands and signs the informed consent form voluntarily by the subject's parent(s) and/or legal guardian(s). And written assent of the subject is required if the subject is 8 years of age or older).
Who cannot join
- For children with small for gestational: confirmed or suspected Bloom syndrome.
- For children with Turner syndrome: containing a Y chromosome or a fragment derived from a Y chromosome.
- Children with closed epiphysis.
- Children who diagnosed or highly suspected growth hormone deficiency (GHD), or other types of growth abnormalities: e.g., Noonan syndrome, Prader-Willi syndrome, Russell-Silver syndrome, etc.
- Children who have previously received systemic growth-promoting therapy, including but not limited to rhGH, aromatase inhibitors, sex hormones, etc., for at least 1 month or longer.
- Children who are now receiving or plan to receive the therapy of glucocorticoids, methylphenidate, and any other drugs that may have an effect on growth.
Show all 18Show fewer
- Children with abnormal values of liver and kidney function (ALT > 1.5 ULN, Cr > 1 ULN).
- Concomitant with chronic hepatitis B, AIDS, tuberculosis, and any other chronic infectious disease.
- Patients with severe allergic constitutions or allergic to growth hormone or its excipients such as mannitol, lysine, sodium chloride and other ingredients.
- Patients with a previous history of malignancy or are currently suffering from active malignancy, including intracranial tumors.
- Patients with abnormal glucose regulation (including abnormal fasting glucose and/or abnormal glucose tolerance) or diabetes.
- Patients who are mentally ill or have a family history of mental illness.
- Patients who are suffering from chronic systemic diseases, such as malnutrition, immunocompromised individuals, asthma, etc.
- Patients with congenital intracranial hypertension.
- Patients with slipped capital femoral epiphysis (SCFE).
- Patients with scoliosis exceeding 15°;
- Patients who have participated in any drug clinical study (as a subject) within 3 months prior to screening and have received a drug intervention
- Patients who the investigators considered unfit for the study.
Trial locations and contacts
19 sites in 1 country.
19 sites in 1 country
China19 sites
Children's Hospital, Capital Institute of Pediatrics
Beijing
Hunan Children's Hospital
Changsha, Hunan
Chengdu Women's and Children's Central Hospital
Chengdu, Sichuan
West China Second University Hospital, Sichuan University
Chengdu, Sichuan
Sun Yat-sen Memorial Hospital, Sun Yat-sen University
Guangzhou, Guangdong
The Third Affiliated Hospital, Sun Yat-sen University
Guangzhou, Guangdong
Zhejiang Provincial People's Hospital
Hangzhou, Zhejiang
The Fourth Affiliated Hospital of Anhui Medical University
Hefei, Anhui
The Second Hospital of Anhui Medical University
Hefei, Anhui
Jiangxi Provincial Children's Hospital
Nanchang, Jiangxi
Children's Hospital of Nanjing Medical University
Nanjing, Jiangsu
Sanya Central Hospital (Hainan Third People's Hospital)
Sanya, Hainan
Children's Hospital of Shanghai
Shanghai
Children's Hospital of Soochow University
Suzhou, Jiangsu
Tongji Hospital, Tongji Medical College of HUST
Wuhan, Hubei
Wuhan Children's Hospital
Wuhan, Hubei
Affiliated Hospital of Jiangnan University
Wuxi, Jiangsu
The First Affiliated Hospital of Xiamen University
Xiamen, Fujian
Henan Children's Hospital Zhengzhou Children's Hospital
Zhengzhou, Henan
About this study
Official title: A Multicenter, Randomized, Open-label, Positive-controlled Phase 2 Study to Explore the Optimal Dose of Y- Shaped Pegylated Recombinant Growth Hormone (YPEG-rhGH) in Children With Short Stature (ISS, SGA, TS)
To explore the dose-response relationship between pharmacokinetics and pharmacodynamics of Y- Shaped Pegylated growth hormone injection (YPEG-GH) in children with short stature (idiopathic short stature (ISS), small for gestational age (SGA), Turner syndrome (TS)).
To evaluate its tolerability, safety and efficacy and to provide evidence for dose selection and titration for future clinical development and clinical application in these population.
- Being tested
- YPEG-rhGH, rhGH
- Keywords
- Growth hormone, Somatotropin, Short stature, Idiopathic short stature, Small for gestational age, Turner syndrome
- Medical subject headings
- Turner Syndrome, Dwarfism
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
YPEG-rhGHDrug
YPEG-rhGH, 140μg/kg, s.c., once a week, for 52 weeks
YPEG-rhGHDrug
YPEG-rhGH, 280μg/kg, s.c., once a week, for 52 weeks
rhGHDrug
rhGH, 245μg/kg/week, divided into 7 subcutaneous injections, once daily, for 52 weeks
rhGHDrug
rhGH, 470μg/kg/week, divided into 7 subcutaneous injections, once daily, for 52 weeks
| Study group | Role | What they receive |
|---|---|---|
| YPEG-GH low dose group | Experimental | Not described Receives: Drug: YPEG-rhGH |
| YPEG-GH high dose group | Experimental | Not described Receives: Drug: YPEG-rhGH |
| rhGH low dose group | Active comparator | Not described Receives: Drug: rhGH |
| rhGH high dose group | Active comparator | Not described Receives: Drug: rhGH |
What is being measured
The primary outcome is the main question the study is designed to answer.
Pharmacokinetic-area under plasma concentration versus time curve
Measured: up to 52 weeks
Pharmacokinetic-maximum serum concentration
Measured: up to 52weeks
Pharmacokinetic-time to reach the maximum plasma concentration
Measured: up to 52 weeks
Pharmacokinetic-terminal disposition phase half-life
Measured: up to 52 weeks
Pharmacokinetic-terminal elimination rate constant
Measured: up to 52 weeks
Pharmacokinetic-apparent clearance after extravascular administration
Measured: up to 52 weeks
Pharmacokinetic-apparent volume of distribution
Measured: up to 52 weeks
Secondary outcomes (6)
Pharmacodynamics-the properties of Insulin-like growth facto1 and Insulin-like growth factor binding receptor 3.
Measured: up to 57 weeks
Height velocity (HV, cm/year)
Measured: At 52 weeks of treatment
Change of height velocity compared to baseline (ΔHV, cm/year)
Measured: At 52 weeks of treatment
Height standard deviation according to chronological age (Ht SDS CA)
Measured: At 52 weeks of treatment
Change in bone age
Measured: At 52 weeks of treatment
Adverse events (including injection site reactions), changes from baseline in vital signs and laboratory tests
Measured: up to 57 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 May 2023
- Study start15 Feb 2022
- Primary completion23 Oct 2023
- Study completion19 Dec 2023
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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 78 participants (actual)
Sponsor and investigators
- Lead sponsor
- Xiamen Amoytop Biotech Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Tongji Hospital
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05838885
- Sponsor's protocol ID
- TB2106GH
- Status last verified
- Jan 2024
- First submitted
- 21 Mar 2023
- Last updated
- 24 Jan 2024
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Jan 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Randomized, Open-label, Positive-controlled Phase 2 Study to Explore the Optimal Dose of Y- Shaped Pegylated Recombinant Growth Hormone (YPEG-rhGH) in Children With Short Stature (ISS, SGA, TS)
- ClinicalTrials.gov ID
- NCT05838885
- Acronym / sponsor ID
- TB2106GH
- Keywords
- Growth hormone, Somatotropin, Short stature, Idiopathic short stature, Small for gestational age, Turner syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 78 participants (actual)
- Lead sponsor
- Xiamen Amoytop Biotech Co., Ltd.Industry (pharma, biotech, devices)
- Collaborators
- Tongji Hospital
- Study start
- 15 Feb 2022 (actual)
- Primary completion
- 23 Oct 2023 (actual)
- Study completion
- 19 Dec 2023 (actual)
- First posted
- 3 May 2023 (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 explore the dose-response relationship between pharmacokinetics and pharmacodynamics of Y- Shaped Pegylated growth hormone injection (YPEG-GH) in children with short stature (idiopathic short stature (ISS), small for gestational age (SGA), Turner syndrome (TS)).
To evaluate its tolerability, safety and efficacy and to provide evidence for dose selection and titration for future clinical development and clinical application in these population.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| YPEG-GH low dose group | Experimental | Drug: YPEG-rhGH |
| YPEG-GH high dose group | Experimental | Drug: YPEG-rhGH |
| rhGH low dose group | Active comparator | Drug: rhGH |
| rhGH high dose group | Active comparator | Drug: rhGH |
Who may qualify
- Ages
- 4 to 11 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- Prepubertal (Tanner I), aged older than 4 years and younger than10 years for girls and 11 years for boys.
- Body weight: 12kg ≤ body weight ≤ 50kg.
- For children with idiopathic short stature: a) Birth length and weight were at the 10th percentile and above of normal reference values for infants of the same gestational age and sex; b) Height at screening was 2.0 standard deviations (SD) below the mean height for chronological age and sex c) Exclude other causes such as systemic diseases, other endocrine diseases, nutritional diseases, chromosomal abnormalities, skeletal dysplasia, psycho-emotional disorders, etc. were excluded d) GH peak ≥10.0ng/ml confirmed by two different drug GH provocation tests; e) Bone age (BA)-chronological age (CA) ≤1 year.
- For children with small for gestational age: a) Birth length and weight were at the 10th percentile and below the normal reference values for infants if the same gestational age and sex; b) Gestational age at birth ≥ 24 weeks; c) Height at screening was below -2 SD of the mean for the same age and sex, and please refer to the protocol annex 1 for height.
- For children with Turner syndrome: a) Chromosome 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-; 45, x/46, XXq-; 45, X/46, XX/47, XXX, etc.; b) Having at least one specific physical characteristic: Including but not limited to low posterior hairline, facial skin nevus, neck flips, short neck, low ear position, small jaw, high palatal arch, shield chest, wide breast spacing, elbow ectropion, knee ectropion, short 4th and 5th metacarpal, nail dysplasia, scoliosis, ptosis, strabismus, cardiovascular system abnormalities such as aortic stenosis, bicuspid aortic valve, hypertension, and reproductive system abnormalities such as primary gonadal insufficiency, renal malformation, hypothyroidism and middle ear disease; c) The height at screening was below the mean -2SD of the same age and gender, and please refer to the protocol annex 1 for height.
Key exclusion criteria (18 listed)
- For children with small for gestational: confirmed or suspected Bloom syndrome.
- For children with Turner syndrome: containing a Y chromosome or a fragment derived from a Y chromosome.
- Children with closed epiphysis.
- Children who diagnosed or highly suspected growth hormone deficiency (GHD), or other types of growth abnormalities: e.g., Noonan syndrome, Prader-Willi syndrome, Russell-Silver syndrome, etc.
- Children who have previously received systemic growth-promoting therapy, including but not limited to rhGH, aromatase inhibitors, sex hormones, etc., for at least 1 month or longer.
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 |
|---|---|---|
| YPEG-rhGHYPEG-rhGH, 140μg/kg, s.c., once a week, for 52 weeks | Drug | YPEG-GH low dose group |
| YPEG-rhGHYPEG-rhGH, 280μg/kg, s.c., once a week, for 52 weeks | Drug | YPEG-GH high dose group |
| rhGHrhGH, 245μg/kg/week, divided into 7 subcutaneous injections, once daily, for 52 weeks | Drug | rhGH low dose group |
| rhGHrhGH, 470μg/kg/week, divided into 7 subcutaneous injections, once daily, for 52 weeks | Drug | rhGH high dose group |
Primary outcome
Pharmacokinetic-area under plasma concentration versus time curve
Time frame: up to 52 weeks
Pharmacokinetic-maximum serum concentration
Time frame: up to 52weeks
Pharmacokinetic-time to reach the maximum plasma concentration
Time frame: up to 52 weeks
Pharmacokinetic-terminal disposition phase half-life
Time frame: up to 52 weeks
Pharmacokinetic-terminal elimination rate constant
Time frame: up to 52 weeks
Pharmacokinetic-apparent clearance after extravascular administration
Time frame: up to 52 weeks
Pharmacokinetic-apparent volume of distribution
Time frame: up to 52 weeks
6 secondary outcomes
Pharmacodynamics-the properties of Insulin-like growth facto1 and Insulin-like growth factor binding receptor 3.
Time frame: up to 57 weeks
Height velocity (HV, cm/year)
Time frame: At 52 weeks of treatment
Change of height velocity compared to baseline (ΔHV, cm/year)
Time frame: At 52 weeks of treatment
Height standard deviation according to chronological age (Ht SDS CA)
Time frame: At 52 weeks of treatment
Change in bone age
Time frame: At 52 weeks of treatment
Adverse events (including injection site reactions), changes from baseline in vital signs and laboratory tests
Time frame: up to 57 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Jan 2024.
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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.
The record lists no contact. Use the official record to reach the sponsor.
19 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 YPEG-rhGH and rhGH in people with Idiopathic Short Stature and Small for Gestational Age, run by Xiamen Amoytop Biotech Co., Ltd. at 19 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 6 inclusion and 18 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: finished, as of the sponsor's last update on 24 Jan 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 19 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Pharmacokinetic-area under plasma concentration versus time curve (time frame: up to 52 weeks). The study lists 7 primary outcomes in all. 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 24 Jan 2024.