Growth Hormone DeficiencyPhase 2/3NCT04513171
Safety and Efficacy of Y-shape Pegylated Somatropin in Growth Hormone Deficiency Children
A Phase 2/3 study testing Y-shape pegylated somatropin, Norditropin® and Norditropin in people with Growth Hormone Deficiency, run by Xiamen Amoytop Biotech Co., Ltd. at 1 site in China.
Trial summary
This is a multicenter, randomized, open-labeled, positive controlled phase 2\&3 combined study to evaluate the safety and efficacy of weekly Y-shape pegylated somatropin, compared to daily somatropin (Norditropin®), in prepubertal, treatment-naive children with growth hormone deficiency. Read more about this study
- Who is it for?
- Ages 3 to 11 years
- Conditions the study is for
- Growth Hormone Deficiency
- What is being tested?
- Y-shape pegylated somatropin, Norditropin® and Norditropin
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 434 participants
- Where
- 1 siteChina
- Study dates
- 26 Dec 2018to 10 Jul 2023
- Phase
- Phase 2/3
- 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 3 to 11 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Diagnosis of GHD confirmed by two different GH stimulation tests, defined as a peak of GH level of <10.0 ng/ml, determined with a validated assay. Bone age (BA) at least 2 years less than the chronological age. Growth velocity less than 5.0 cm/year. Impaired HT defined as at least 2.0 standard deviations (SD) below of the mean height for chronological age and sex (HT SDS<-2.0).
- Prepubertal (Tanner Ⅰ) males and females by physical examination, aged older than 3 years and younger than10 years for girls and 11 years for boys.
- Short stature with normal intelligence.
- Baseline IGF-1 level below the median IGF-1 level standardized for age and sex.
- Written, signed informed consent of the parent(s) or legal guardian(s) of the subject and written assent of the subject (if the subject is 8 years old or above).
Who cannot join
- Prior exposure to growth promotion treatment, such as recombinant human growth hormone or gonadal hormones, for more than 1 month.
- Known hypersensitivity to somatropin or excipients, such as mannitol, lysine, sodium chloride.
- Children with closed epiphyses.
- Short stature etiologies other than GHD, such as idiopathic short stature, Turner syndrome, Prader-Willi syndrome, Russell-Silver syndrome, born small for gestational age regardless of GH status.
- Other causes of short stature such as hypothyroidism, adrenocortical hormone deficiency, antidiuretic hormone deficiency.
- Any medical conditions and/or presence that may affect growth velocity such as liver dysfunction, kidney dysfunction, malnutrition, diabetes mellitus, severe dysfunction in major organ such as heart, sever systemic infections, severe immune dysfunction, mental disorders, and other congenital malformations.
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- Suffering from chronic infectious diseases such as chronic hepatitis B, AIDS or tuberculosis.
- Receiving non-physiological adrenal corticosteroids.
- Confirmed pituitary and/or hypothalamic malignance by MRI within one year prior to screening. History or presence of any other malignance disease, any evidence of present tumor growth.
- Evidence of congenital intracranial hypertension.
- Evidence of slipped capital femoral epiphysis.
- Evidence of scoliosis over 15°.
- Participation in any other trial of an investigational agent within 3 months prior to screening.
- Any other conditions which in the opinion of the investigator precluded enrollment into the study.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Multi-center, Randomized, Positive-control, Phase 2&3 Combined Study of Y-shape Pegylated Somatropin in Prepubertal Children With Growth Hormone Deficiency.
This is a multicenter, randomized, open-labeled, positive controlled phase 2\&3 combined study to evaluate the safety and efficacy of weekly Y-shape pegylated somatropin, compared to daily somatropin (Norditropin®), in prepubertal, treatment-naive children with growth hormone deficiency.
Read the detailed description
This multicenter, randomized, open-labeled, positive controlled study is divided into two stages. The first one is aimed to exploit the optimal dose of Y-shape pegylated somatropin, while the second one is aimed to confirm the efficacy and safety of the study drug. A total of 400 prepubertal children with growth hormone deficiency were expected to enrolled. Subjects will firstly undergo a 52 weeks treatment, and then followed for 5 weeks.
- Conditions
- Growth Hormone Deficiency
- Being tested
- Y-shape pegylated somatropin, Norditropin®, Norditropin
- Keywords
- Growth Hormone Deficiency, somatropin, recombinated human growth hormone, Growth Hormone
- Medical subject headings
- Dwarfism, Pituitary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Y-shape pegylated somatropinDrug
Y-shape pegylated somatropin 100μg/kg, subcutaneous injection, Once weekly.
Y-shape pegylated somatropinDrug
Y-shape pegylated somatropin 120μg/kg, subcutaneous injection, Once weekly.
Y-shape pegylated somatropinDrug
Y-shape pegylated somatropin 140μg/kg, subcutaneous injection, Once weekly.
Norditropin®Drug
Norditropin 245μg/kg/week, subcutaneous injection, Once daily.
Y-shape pegylated somatropinDrug
Y-shape pegylated somatropin 140μg/kg, subcutaneous injection, Once weekly.
NorditropinDrug
Norditropin 245μg/kg/week, subcutaneous injection, Once daily.
| Study group | Role | What they receive |
|---|---|---|
| Y-shape pegylated somatropin low dose | Experimental | Not described Receives: Drug: Y-shape pegylated somatropin |
| Y-shape pegylated somatropin middle dose | Experimental | Not described Receives: Drug: Y-shape pegylated somatropin |
| Y-shape pegylated somatropin high dose | Experimental | Not described Receives: Drug: Y-shape pegylated somatropin |
| Norditropin-1 | Active comparator | Not described Receives: Drug: Norditropin® |
| Y-shape pegylated somatropin optimal dose | Experimental | Not described Receives: Drug: Y-shape pegylated somatropin |
| Norditropin-2 | Active comparator | Not described Receives: Drug: Norditropin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Phase 2: Change of areas under the curve of IGF-1 concentration from baseline (ΔIGF-1 AUC).
Measured: 12 weeks
Phase 3: Height velocity.
Measured: 52 weeks
Secondary outcomes (4)
Change of height standard deviation score according to chronological age.
Measured: 52 weeks
Change of height standard deviation score according to bone age.
Measured: 52 weeks
Serum IGF-l level
Measured: change from baseline to 52 weeks
Serum IGFBP-3 level
Measured: change from baseline to 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Aug 2020
- Study start26 Dec 2018
- Primary completion7 Jun 2023
- Study completion10 Jul 2023
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 Growth Hormone DeficiencyStudies 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 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 434 participants (actual)
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, MD, Ph.D, Principal investigator, Tongji Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04513171
- Sponsor's protocol ID
- TB1805GH
- Status last verified
- Jan 2024
- First submitted
- 7 Aug 2020
- Last updated
- 24 Jan 2024
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Liang Y, Zhang C, Wei H, Du H, Zhang G, Yang Y, Zhang H, Gong H, Li P, Song F, Xu Z, He R, Zhou W, Zheng H, Sun L, Luo X. The pharmacokinetic and pharmacodynamic properties and short-term outcome of a novel once-weekly PEGylated recombinant human growth hormone for children with growth hormone deficiency. Front Endocrinol (Lausanne). 2022 Aug 11;13:922304. doi: 10.3389/fendo.2022.922304. eCollection 2022.
PubMed 36034448
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 Multi-center, Randomized, Positive-control, Phase 2&3 Combined Study of Y-shape Pegylated Somatropin in Prepubertal Children With Growth Hormone Deficiency.
- ClinicalTrials.gov ID
- NCT04513171
- Acronym / sponsor ID
- TB1805GH
- Conditions
- Growth Hormone Deficiency
- Keywords
- Growth Hormone Deficiency, somatropin, recombinated human growth hormone, Growth Hormone
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 434 participants (actual)
- Lead sponsor
- Xiamen Amoytop Biotech Co., Ltd.Industry (pharma, biotech, devices)
- Collaborators
- Tongji Hospital
- Study start
- 26 Dec 2018 (actual)
- Primary completion
- 7 Jun 2023 (actual)
- Study completion
- 10 Jul 2023 (actual)
- First posted
- 14 Aug 2020 (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-labeled, positive controlled phase 2\&3 combined study to evaluate the safety and efficacy of weekly Y-shape pegylated somatropin, compared to daily somatropin (Norditropin®), in prepubertal, treatment-naive children with growth hormone deficiency.
Detailed description
This multicenter, randomized, open-labeled, positive controlled study is divided into two stages. The first one is aimed to exploit the optimal dose of Y-shape pegylated somatropin, while the second one is aimed to confirm the efficacy and safety of the study drug. A total of 400 prepubertal children with growth hormone deficiency were expected to enrolled. Subjects will firstly undergo a 52 weeks treatment, and then followed for 5 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 6
| Arm | Type | Receives |
|---|---|---|
| Y-shape pegylated somatropin low dose | Experimental | Drug: Y-shape pegylated somatropin |
| Y-shape pegylated somatropin middle dose | Experimental | Drug: Y-shape pegylated somatropin |
| Y-shape pegylated somatropin high dose | Experimental | Drug: Y-shape pegylated somatropin |
| Norditropin-1 | Active comparator | Drug: Norditropin® |
| Y-shape pegylated somatropin optimal dose | Experimental | Drug: Y-shape pegylated somatropin |
| Norditropin-2 | Active comparator | Drug: Norditropin |
Who may qualify
- Ages
- 3 to 11 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- Diagnosis of GHD confirmed by two different GH stimulation tests, defined as a peak of GH level of <10.0 ng/ml, determined with a validated assay. Bone age (BA) at least 2 years less than the chronological age. Growth velocity less than 5.0 cm/year. Impaired HT defined as at least 2.0 standard deviations (SD) below of the mean height for chronological age and sex (HT SDS<-2.0).
- Prepubertal (Tanner Ⅰ) males and females by physical examination, aged older than 3 years and younger than10 years for girls and 11 years for boys.
- Short stature with normal intelligence.
- Baseline IGF-1 level below the median IGF-1 level standardized for age and sex.
- Written, signed informed consent of the parent(s) or legal guardian(s) of the subject and written assent of the subject (if the subject is 8 years old or above).
Key exclusion criteria (14 listed)
- Prior exposure to growth promotion treatment, such as recombinant human growth hormone or gonadal hormones, for more than 1 month.
- Known hypersensitivity to somatropin or excipients, such as mannitol, lysine, sodium chloride.
- Children with closed epiphyses.
- Short stature etiologies other than GHD, such as idiopathic short stature, Turner syndrome, Prader-Willi syndrome, Russell-Silver syndrome, born small for gestational age regardless of GH status.
- Other causes of short stature such as hypothyroidism, adrenocortical hormone deficiency, antidiuretic hormone deficiency.
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 |
|---|---|---|
| Y-shape pegylated somatropinY-shape pegylated somatropin 100μg/kg, subcutaneous injection, Once weekly. | Drug | Y-shape pegylated somatropin low dose |
| Y-shape pegylated somatropinY-shape pegylated somatropin 120μg/kg, subcutaneous injection, Once weekly. | Drug | Y-shape pegylated somatropin middle dose |
| Y-shape pegylated somatropinY-shape pegylated somatropin 140μg/kg, subcutaneous injection, Once weekly. | Drug | Y-shape pegylated somatropin high dose |
| Norditropin®Norditropin 245μg/kg/week, subcutaneous injection, Once daily. | Drug | Norditropin-1 |
| Y-shape pegylated somatropinY-shape pegylated somatropin 140μg/kg, subcutaneous injection, Once weekly. | Drug | Y-shape pegylated somatropin optimal dose |
| NorditropinNorditropin 245μg/kg/week, subcutaneous injection, Once daily. | Drug | Norditropin-2 |
Primary outcome
Phase 2: Change of areas under the curve of IGF-1 concentration from baseline (ΔIGF-1 AUC).
Time frame: 12 weeks
Phase 3: Height velocity.
Time frame: 52 weeks
4 secondary outcomes
Change of height standard deviation score according to chronological age.
Time frame: 52 weeks
Change of height standard deviation score according to bone age.
Time frame: 52 weeks
Serum IGF-l level
Time frame: change from baseline to 52 weeks
Serum IGFBP-3 level
Time frame: change from baseline to 52 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.
- Derived from this study: PubMed 36034448Liang Y, Zhang C, Wei H, Du H, Zhang G, Yang Y, Zhang H, Gong H, Li P, Song F, Xu Z, He R, Zhou W, Zheng H, Sun L, Luo X. The pharmacokinetic and pharmacodynamic properties and short-term outcome of a novel once-weekly PEGylated recombinant human growth hormone for children with growth hormone deficiency. Front Endocrinol (Lausanne). 2022 Aug 11;13:922304. doi: 10.3389/fendo.2022.922304. 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.
Xiaoping Luo, MD, Ph.D principal investigator
Tongji Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Y-shape pegylated somatropin, Norditropin® and Norditropin in people with Growth Hormone Deficiency, run by Xiamen Amoytop Biotech Co., Ltd. at 1 site 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 5 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: 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 1 site 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 combined 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.
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?
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?
Phase 2: Change of areas under the curve of IGF-1 concentration from baseline (ΔIGF-1 AUC). (time frame: 12 weeks). The study lists 2 primary outcomes in all. 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 NCT04513171, 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 24 Jan 2024.