Growth Hormone DeficiencyPhase 3NCT04326374
Safety, Tolerability and Efficacy of TransCon hGH Weekly Versus Daily hGH in Chinese Pediatric Growth Hormone Deficiency
A Phase 3 study testing TransCon hGH and daily hGH in people with Growth Hormone Deficiency and Endocrine System Diseases, run by Visen Pharmaceuticals (Shanghai) Co., Ltd. at 1 site in China.
Trial summary
This study is conducted in China only. The purpose is to demonstrate the efficacy and safety of once weekly dosing of TransCon hGH, a long-acting growth hormone product, compare to once-daily dosing of human growth hormone (hGH) after 52 weeks of treatment in prepubertal children with growth hormone deficiency (GHD). Read more about this study
- Who is it for?
- Ages 3 to 17 years
- Conditions the study is for
- Growth Hormone Deficiency, Endocrine System Diseases, Hormones
- What is being tested?
- TransCon hGH and daily hGH
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 150 participants
- Where
- 1 siteChina
- Study dates
- 30 Dec 2019to Apr 2022
- 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 3 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Prepubertal children with GHD in Tanner stage 1, aged of 3 years and below 17 years;
- Impaired HT defined as at least 2.0 standard deviations (SD) below the mean height for chronological age and sex (HT SDS≤-2.0) according to the Chinese 2005 standard；
- Diagnosis of GHD confirmed by 2 different GH stimulation tests, defined as a peak GH level of ≤10 ng/mL, determined with a validated assay. Bone age (BA) at least 6 months less than the chronological age；
- Baseline IGF-1 level of at least 1.0 SD below the mean IGF-1 level standardized for age and sex (IGF-1 SDS ≤-1.0)；
- 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
- Children with a body weight below 12 kg；
- Prior exposure to recombinant hGH or IGF-1 therapy；
- Children with past or present intracranial tumor growth as confirmed by a sellar MRI scan (with contrast dye recommended) at Screening
- Children born SGA (birth weight ≤10th percentile for gestational age according to the Chinese reference)；
- Children with psychosocial dwarfism；
- Children with idiopathic short stature；
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- Other causes of short stature such as coeliac disease, hypothyroidism, or rickets；
- History or presence of malignant disease; any evidence of present tumor growth;
- Subjects with diabetes mellitus；
- Closed epiphyses；
- Major medical conditions and/or presence of contraindication to hGH treatment；
- Participation in any other trial of an investigational agent within 3 months prior to Screening.
Trial locations and contacts
1 site in 1 country.
xiangling Wang, MD contact
China1 site, 1 recruiting
- Recruiting
Department of Pediatrics, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology,
Wuhan, Hubei, 430030
Xiaoping Luo, MD
About this study
Official title: The Efficacy, Safety and Tolerability of TransCon hGH Administered Weekly Versus Daily hGH in Prepubertal Children With Growth Hormone Deficiency: a Randomized, Open-lable, Active-controlled, Parallel-group Study in China
This study is conducted in China only. The purpose is to demonstrate the efficacy and safety of once weekly dosing of TransCon hGH, a long-acting growth hormone product, compare to once-daily dosing of human growth hormone (hGH) after 52 weeks of treatment in prepubertal children with growth hormone deficiency (GHD).
Read the detailed description
Not Provided
- Conditions
- Growth Hormone Deficiency, Endocrine System Diseases, Hormones, Pituitary Diseases, Pituitary Disease, Anterior
- Being tested
- TransCon hGH, daily hGH
- Keywords
- Growth Hormone Deficiency, TransCon hGH, human growth hormone, hGH
- Medical subject headings
- Dwarfism, Pituitary, Endocrine System Diseases, Pituitary Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TransCon hGHDrug
Also called ACP-011
Once weekly subcutaneous injection
daily hGHDrug
Also called somatropin (rDNA orgin) for injection
Once daily subcutaneous injection
| Study group | Role | What they receive |
|---|---|---|
| TransCon hGH | Experimental | TransCon hGH will be self-administered or injected by parents once weekly. The dose will be adjusted based on subject's weight.
The treatment will continue 52 weeks. Receives: Drug: TransCon hGH |
| Daily hGH | Active comparator | Daily hGH will be self-administered or injected by parents once daily. The dose will be adjusted based on subject's weight.
The treatment will continue 52 weeks. Receives: Drug: daily hGH |
What is being measured
The primary outcome is the main question the study is designed to answer.
Annualized height velocity(AHV) at Week 52
Annualized height velocity at Week 52 for weekly TransCon hGH treatment and the daily hGH treatment groups
Measured: 52 Weeks
Secondary outcomes (6)
AHV and the change from baseline over 52 weeks at each visit
AHV and the change from baseline for the TransCon hGH and the daily hGH treatment group over 52 weeks at each visit
Measured: 52 Weeks
Height SDS and the change from baseline over 52 weeks
Height SDS and the change from baseline for the TransCon hGH and the daily hGH treatment group over 52 weeks
Measured: 52 weeks
Serum IGF-1 and IGFBP-3 levels, and IGF-1 SDS, IGFBP-3 SDS, and the change from baseline over 52 weeks at each visit
Serum IGF-1 and IGFBP-3 levels, and IGF-1 SDS, IGFBP-3 SDS, and the change from baseline for the TransCon hGH and the daily hGH treatment group over 52 weeks at each visit
Measured: 52 weeks
The percentage of participants achieving IGF-1 SDS 0 to +2.0
The normalization of IGF-1 SDS (percentage of participants achieving IGF-1 SDS 0 to +2.0 over 52 weeks for the TransCon hGH and the daily hGH treatment group
Measured: 52 weeks
Incidence of Treatment-Emergent Adverse Events
Measured: 52 weeks
The plasma concentration
pharmacokinetics measure of hGH over 52 weeks in TransCon hGH group
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 posted30 Mar 2020
- Study start30 Dec 2019
- Primary completionApr 2022 (estimated)
- Study completionApr 2022 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Growth Hormone DeficiencyStudies of the same condition, open and under way.
- 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
- Model description
- Eligible subjects will be randomized to 1 of the 2 treatment groups: either weekly TransCon hGH or daily hGH, in a 2:1 ratio
- Masking
- None (open label)
- Enrolment
- 150 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Visen Pharmaceuticals (Shanghai) Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Ascendis Pharma A/S
- Responsible party
- Sponsor
- Investigators
- Xiaoping Luo, MD, Principal investigator, Tongji Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04326374
- Sponsor's protocol ID
- CT301-CN
- Other IDs
- CTR20200399
- Status last verified
- Mar 2020
- First submitted
- 26 Mar 2020
- Last updated
- 30 Mar 2020
- Results posted
- No
- Data monitoring committee
- Yes
- 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 30 Mar 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Efficacy, Safety and Tolerability of TransCon hGH Administered Weekly Versus Daily hGH in Prepubertal Children With Growth Hormone Deficiency: a Randomized, Open-lable, Active-controlled, Parallel-group Study in China
- ClinicalTrials.gov ID
- NCT04326374
- Acronym / sponsor ID
- CT301-CN
- Conditions
- Growth Hormone Deficiency, Endocrine System Diseases, Hormones, Pituitary Diseases, Pituitary Disease, Anterior
- Keywords
- Growth Hormone Deficiency, TransCon hGH, human growth hormone, hGH
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 150 participants (estimated)
- Lead sponsor
- Visen Pharmaceuticals (Shanghai) Co., Ltd.Industry (pharma, biotech, devices)
- Collaborators
- Ascendis Pharma A/S
- Study start
- 30 Dec 2019 (actual)
- Primary completion
- Apr 2022 (estimated)
- Study completion
- Apr 2022 (estimated)
- First posted
- 30 Mar 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 study is conducted in China only. The purpose is to demonstrate the efficacy and safety of once weekly dosing of TransCon hGH, a long-acting growth hormone product, compare to once-daily dosing of human growth hormone (hGH) after 52 weeks of treatment in prepubertal children with growth hormone deficiency (GHD).
Detailed description
Not Provided
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- Eligible subjects will be randomized to 1 of the 2 treatment groups: either weekly TransCon hGH or daily hGH, in a 2:1 ratio
| Arm | Type | Receives |
|---|---|---|
| TransCon hGH | Experimental | Drug: TransCon hGHTransCon hGH will be self-administered or injected by parents once weekly. The dose will be adjusted based on subject's weight. The treatment will continue 52 weeks. |
| Daily hGH | Active comparator | Drug: daily hGHDaily hGH will be self-administered or injected by parents once daily. The dose will be adjusted based on subject's weight. The treatment will continue 52 weeks. |
Who may qualify
- Ages
- 3 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- Prepubertal children with GHD in Tanner stage 1, aged of 3 years and below 17 years;
- Impaired HT defined as at least 2.0 standard deviations (SD) below the mean height for chronological age and sex (HT SDS≤-2.0) according to the Chinese 2005 standard；
- Diagnosis of GHD confirmed by 2 different GH stimulation tests, defined as a peak GH level of ≤10 ng/mL, determined with a validated assay. Bone age (BA) at least 6 months less than the chronological age；
- Baseline IGF-1 level of at least 1.0 SD below the mean IGF-1 level standardized for age and sex (IGF-1 SDS ≤-1.0)；
- 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 (12 listed)
- Children with a body weight below 12 kg；
- Prior exposure to recombinant hGH or IGF-1 therapy；
- Children with past or present intracranial tumor growth as confirmed by a sellar MRI scan (with contrast dye recommended) at Screening
- Children born SGA (birth weight ≤10th percentile for gestational age according to the Chinese reference)；
- Children with psychosocial dwarfism；
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 |
|---|---|---|
| TransCon hGHAlso: ACP-011Once weekly subcutaneous injection | Drug | TransCon hGH |
| daily hGHAlso: somatropin (rDNA orgin) for injectionOnce daily subcutaneous injection | Drug | Daily hGH |
Primary outcome
Annualized height velocity(AHV) at Week 52
Time frame: 52 Weeks
Annualized height velocity at Week 52 for weekly TransCon hGH treatment and the daily hGH treatment groups
6 secondary outcomes
AHV and the change from baseline over 52 weeks at each visit
Time frame: 52 Weeks
Height SDS and the change from baseline over 52 weeks
Time frame: 52 weeks
Serum IGF-1 and IGFBP-3 levels, and IGF-1 SDS, IGFBP-3 SDS, and the change from baseline over 52 weeks at each visit
Time frame: 52 weeks
The percentage of participants achieving IGF-1 SDS 0 to +2.0
Time frame: 52 weeks
Incidence of Treatment-Emergent Adverse Events
Time frame: 52 weeks
The plasma concentration
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 30 Mar 2020.
- 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. This study is not recruiting at the moment.
xiangling Wang, MD contact
Xiaoping Luo, MD 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 3 study testing TransCon hGH and daily hGH in people with Growth Hormone Deficiency and Endocrine System Diseases, run by Visen Pharmaceuticals (Shanghai) 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 12 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 30 Mar 2020. 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 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 Lisa Chen, MD (+86-02152999605, lisa.chen@visenpharma.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Annualized height velocity(AHV) at Week 52 (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 30 Mar 2020.