Pediatric Growth Hormone DeficiencyPhase 2/3NCT02413138
Versartis Trial in Pre-pubertal Japanese Children With Growth Hormone Deficiency (GHD) to Assess Long-Acting Growth Hormone (Somavaratan, VRS-317)
A Phase 2/3 study testing Somavaratan (VRS-317) in people with Pediatric Growth Hormone Deficiency and Growth Disorders, run by Versartis Inc. at 1 site in Japan.
Trial summary
The trial will consist of three stages: 1) a 30 day Phase 2 PK and PD evaluation of somavaratan, 2) an optional Phase 2 Extension and 3) a 12 month Phase 3 safety and efficacy stage. Read more about this study
- Who is it for?
- Ages 3 to 10 years
- Conditions the study is for
- Pediatric Growth Hormone Deficiency, Growth Disorders
- What is being tested?
- Somavaratan (VRS-317)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 41 participants
- Where
- 1 siteJapan
- Study dates
- 8 Aug 2015to 30 Nov 2017
- 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 10 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Chronological Age ≥ 3.0 years and ≤ 9.0 years (girls) or ≤ 10.0 years (boys)
- Pre-pubertal status
- Diagnosis of GHD as documented by two or more GH stimulation test results
- Height SD score ≤ -2.0 at screening
- Weight for Stature ≥ 10th percentile
- IGF-I SD score ≤ -1.0 at screening
- Delayed bone age
Who cannot join
- Prior treatment with any growth promoting agent
- History of, or current, significant disease
- Chromosomal aneuploidy, significant gene mutations (other than those that cause GHD) or confirmed diagnosis of a named syndrome
- Birth weight and/or birth length less than 5th percentile for gestational age
- A diagnosis of Attention Deficit Hyperactivity Disorder
- Daily use of anti-inflammatory doses of glucocorticoid
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- Prior history of leukemia, lymphoma, sarcoma or cancer
- Ocular findings suggestive of increased intracranial pressure and/or retinopathy at screening
- Significant spinal abnormalities including scoliosis, kyphosis and spina bifida variants
- Significant abnormality in screening laboratory studies
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Randomized, Phase 2/3, Open-Label, Multi-center Study of the Pharmacokinetics, Pharmacodynamics, Safety and Efficacy of A Long-acting Human Growth Hormone (Somavaratan, VRS-317) in Pre-pubertal Japanese Children With Growth Hormone Deficiency (GHD)
The trial will consist of three stages: 1) a 30 day Phase 2 PK and PD evaluation of somavaratan, 2) an optional Phase 2 Extension and 3) a 12 month Phase 3 safety and efficacy stage. Upon completion of the PK/PD stage, the PK/PD profiles for the GHD children in this study will be compared to the PK/PD profiles for the GHD children treated in the Western study Phase 1b/2a study (Protocol 12VR2) and identify the somavaratan dose to be used in the Phase 3 stage in Japan. The Phase 3 stage will continue dosing for 12 months to obtain safety and efficacy data on 48 subjects.
Read the detailed description
The trial will consist of three stages: 1) a 30 day Phase 2 PK and PD evaluation of somavaratan, 2) an optional Phase 2 Extension and 3) a 12 month Phase 3 safety and efficacy stage. The study is a randomized, multi-center, open label study. The primary endpoint is height velocity at 12 months.
- Being tested
- Somavaratan (VRS-317)
- Keywords
- GHD
- Medical subject headings
- Growth Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Somavaratan (VRS-317)Drug
Also called Long acting recombinant human growth hormone
Long acting recombinant human growth hormone
| Study group | Role | What they receive |
|---|---|---|
| Phase 2: Somavaratan (VRS-317) | Experimental | Active treatment arm Receives: Drug: Somavaratan (VRS-317) |
| Phase 3: Somavaratan (VRS-317) | Experimental | Somavaratan long acting recombinant human growth hormone administered subcutaneously twice-monthly Receives: Drug: Somavaratan (VRS-317) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Efficacy (Annual Height velocity)
Annual Height velocity.
Measured: 12 months
Secondary outcomes (7)
Pharmacodynamics (IGF-I responses to study drug administration)
IGF-I responses to study drug administration.
Measured: 12 months
Pharmacodynamics (IGFBP-3 responses to study drug administration)
IGFBP-3 responses to study drug administration.
Measured: 12 months
Safety (Number of subjects with adverse events )
Number of subjects with adverse events (including repeat dose immunogenicity).
Measured: 12 months
Safety (Concomitant medications)
Concomitant medications
Measured: 12 months
Safety (Safety labs)
Safety labs
Measured: 12 months
Safety (Vital signs)
Vital signs
Measured: 12 months
Safety (Physical Exams)
Physical Exams
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Apr 2015 (estimated)
- Study start8 Aug 2015
- Primary completion30 Nov 2017
- Study completion30 Nov 2017
Why it stopped: The primary endpoint of non-inferiority to daily therapy in the pediatric Phase 3 study was not achieved
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pediatric 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 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 41 participants (actual)
Sponsor and investigators
- Lead sponsor
- Versartis Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Will Charlton, MD, Study director, Vesrartis
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02413138
- Sponsor's protocol ID
- J14VR5
- Status last verified
- Jul 2022
- First submitted
- 23 Mar 2015
- Last updated
- 25 Jul 2022
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Jul 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Phase 2/3, Open-Label, Multi-center Study of the Pharmacokinetics, Pharmacodynamics, Safety and Efficacy of A Long-acting Human Growth Hormone (Somavaratan, VRS-317) in Pre-pubertal Japanese Children With Growth Hormone Deficiency (GHD)
- ClinicalTrials.gov ID
- NCT02413138
- Acronym / sponsor ID
- J14VR5
- Keywords
- GHD
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: The primary endpoint of non-inferiority to daily therapy in the pediatric Phase 3 study was not achieved
- Enrolment
- 41 participants (actual)
- Lead sponsor
- Versartis Inc.Industry (pharma, biotech, devices)
- Study start
- 8 Aug 2015 (actual)
- Primary completion
- 30 Nov 2017 (actual)
- Study completion
- 30 Nov 2017 (actual)
- First posted
- 9 Apr 2015 (estimated)
- 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.
The trial will consist of three stages: 1) a 30 day Phase 2 PK and PD evaluation of somavaratan, 2) an optional Phase 2 Extension and 3) a 12 month Phase 3 safety and efficacy stage. Upon completion of the PK/PD stage, the PK/PD profiles for the GHD children in this study will be compared to the PK/PD profiles for the GHD children treated in the Western study Phase 1b/2a study (Protocol 12VR2) and identify the somavaratan dose to be used in the Phase 3 stage in Japan. The Phase 3 stage will continue dosing for 12 months to obtain safety and efficacy data on 48 subjects.
Detailed description
The trial will consist of three stages: 1) a 30 day Phase 2 PK and PD evaluation of somavaratan, 2) an optional Phase 2 Extension and 3) a 12 month Phase 3 safety and efficacy stage. The study is a randomized, multi-center, open label study. The primary endpoint is height velocity at 12 months.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Phase 2: Somavaratan (VRS-317) | Experimental | Drug: Somavaratan (VRS-317)Active treatment arm |
| Phase 3: Somavaratan (VRS-317) | Experimental | Drug: Somavaratan (VRS-317)Somavaratan long acting recombinant human growth hormone administered subcutaneously twice-monthly |
Who may qualify
- Ages
- 3 to 10 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (7 listed)
- Chronological Age ≥ 3.0 years and ≤ 9.0 years (girls) or ≤ 10.0 years (boys)
- Pre-pubertal status
- Diagnosis of GHD as documented by two or more GH stimulation test results
- Height SD score ≤ -2.0 at screening
- Weight for Stature ≥ 10th percentile
Key exclusion criteria (10 listed)
- Prior treatment with any growth promoting agent
- History of, or current, significant disease
- Chromosomal aneuploidy, significant gene mutations (other than those that cause GHD) or confirmed diagnosis of a named syndrome
- Birth weight and/or birth length less than 5th percentile for gestational age
- A diagnosis of Attention Deficit Hyperactivity Disorder
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 |
|---|---|---|
| Somavaratan (VRS-317)Also: Long acting recombinant human growth hormoneLong acting recombinant human growth hormone | Drug | Phase 2: Somavaratan (VRS-317), Phase 3: Somavaratan (VRS-317) |
Primary outcome
Efficacy (Annual Height velocity)
Time frame: 12 months
Annual Height velocity.
7 secondary outcomes
Pharmacodynamics (IGF-I responses to study drug administration)
Time frame: 12 months
Pharmacodynamics (IGFBP-3 responses to study drug administration)
Time frame: 12 months
Safety (Number of subjects with adverse events )
Time frame: 12 months
Safety (Concomitant medications)
Time frame: 12 months
Safety (Safety labs)
Time frame: 12 months
Safety (Vital signs)
Time frame: 12 months
Safety (Physical Exams)
Time frame: 12 months
Results and safety
The study was stopped early (reason given: The primary endpoint of non-inferiority to daily therapy in the pediatric Phase 3 study was not achieved). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Jul 2022.
- 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.
Will Charlton, MD study director
Vesrartis
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 Somavaratan (VRS-317) in people with Pediatric Growth Hormone Deficiency and Growth Disorders, run by Versartis Inc. at 1 site in Japan.
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 10 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: stopped early, as of the sponsor's last update on 25 Jul 2022. 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 Japan. 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 was stopped early (reason given: The primary endpoint of non-inferiority to daily therapy in the pediatric Phase 3 study was not achieved). Results are not available in the source record.
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?
Efficacy (Annual Height velocity) (time frame: 12 months). 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 25 Jul 2022.