Growth DisordersPhase 3VELOCITYNCT02339090
Long-Acting Growth Hormone in Children Compared to Daily rhGH
A Phase 3 study testing Somavaratan and rhGH in people with Growth Disorders, run by Versartis Inc..
Trial summary
The trial will compare a twice-monthly somavaratan dosing regimen for non-inferiority of treatment effect against daily injections of rhGH. Read more about this study
- Who is it for?
- Ages 3 to 11 years
- Conditions the study is for
- Growth Disorders
- What is being tested?
- Somavaratan and rhGH
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 138 participants
- Where
- No sites listed yet
- Study dates
- 26 Aug 2015to 23 Aug 2017
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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
- Chronological Age ≥ 3.0 years and ≤ 10.0 (girls) and ≤ 11.0 (boys).
- Pre-pubertal status: Absent breast development in girls, testicular volume < 4.0 mL in boys.
- Diagnosis of growth hormone deficiency (GHD) as documented by two or more growth hormone (GH) stimulation test results ≤ 10.0 ng/mL.
- Height standard deviation score (SDS) ≤ -2.0 at screening.
- Weight for Stature ≥ 10th percentile.
- Insulin-like growth factor-I (IGF-I) SDS ≤ -1.0 at screening.
- Delayed bone age (≥ 6 months).
Who cannot join
- Prior treatment with any growth promoting agent
- History of, or concurrent significant disease (for example, diabetes, cystic fibrosis, renal insufficiency).
- Chromosomal aneuploidy, significant gene mutations (other than those that cause GHD) or confirmed diagnosis of a named syndrome.
- A diagnosis of Attention Deficit Hyperactivity Disorder.
- Daily use of anti-inflammatory doses of glucocorticoid.
- Prior history of leukemia, lymphoma, sarcoma or cancer.
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- Treatment with an investigational drug in the 30 days prior to screening.
- Known allergy to constituents of the study drug formulation.
- 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
About this study
Official title: Comparison of Somavaratan (VRS-317), a Long-acting Human Growth Hormone, to Daily rhGH in a Phase 3, Randomized, One-year, Open-label, Multi-center, Non-inferiority Trial in Pre-pubertal Children With Growth Hormone Deficiency
The trial will compare a twice-monthly somavaratan dosing regimen for non-inferiority of treatment effect against daily injections of rhGH.
Read the detailed description
This study is designed as a pivotal study to compare the safety and efficacy of a selected dose regimen of somavaratan to daily rhGH. The study is a randomized, multi-center, open label study of 12 months duration. The primary endpoint is height velocity at 12 months.
- Conditions
- Growth Disorders
- Being tested
- Somavaratan, rhGH
- Keywords
- VRS-317, Growth Hormone Deficiency, Long Acting Recombinant Growth Hormone, Long Acting Growth Hormone, Pediatric Growth Hormone Deficiency, Growth Hormone Replacement Therapy, Versartis, Xten, Human Growth Hormone, somavaratan, growth failure
- Medical subject headings
- Growth Disorders, Dwarfism, Pituitary, Failure to Thrive
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SomavaratanDrug
Also called Long-acting recombinant human growth hormone, VRS-317
Somavaratan will be administered per dose and schedule specified in the arm description.
rhGHDrug
Also called daily growth hormone, recombinant growth hormone therapy
rhGH will be administered per dose and schedule specified in the arm description.
| Study group | Role | What they receive |
|---|---|---|
| Somavaratan | Experimental | Participants will receive somavaratan 3.5 milligrams (mg)/kilogram (kg) subcutaneous (SC) bolus injection twice monthly for 12 months. Receives: Drug: Somavaratan |
| rhGH | Active comparator | Participants will receive commercially available rhGH (genotropin) 34 micrograms (μg)/kg once daily SC bolus injection for 12 months. Receives: Drug: rhGH |
What is being measured
The primary outcome is the main question the study is designed to answer.
Annual Height Velocity
Height measured without shoes in triplicate by stadiometer. Annual height velocity was calculated as (height at Month 12 - height at Baseline)/(Month 12 Date - Baseline Date) * 365.25, where height was expressed as centimeters (cm) so that height velocity is expressed as centimeters per year (cm/yr). Annual height velocity after 12 months continuous treatment with either somavaratan or daily rhGH has been reported. Missing data was imputed using last observation carried forward. Least square (LS) mean was calculated using analysis of covariance (ANCOVA) model.
Measured: 12 months
Secondary outcomes (7)
Change From Baseline in Height Standard Deviation Score (SDS) at Month 12
Height SDS was determined using the Center for Disease Control (CDC) Clinical Growth Charts; 2000. The SD score was calculated as the participant's height value minus the mean divided by the standard deviation (SD). The mean and the SD vary depending on the age and sex of the participant. Mean change from baseline in height SDS at Month 12 is presented.
Measured: Baseline, Month 12
Change From Baseline in Bone Age Relative to Chronological Age at Month 12, as Assessed by Central Reader
Bone age was assessed from a radiograph of the left hand and wrist by central reader.
Measured: Baseline, Month 12
Change From Baseline in Body Mass Index (BMI) at Month 12
The BMI is a person's weight in kilograms (kg) divided by the square of height in meters.
Measured: Baseline, Month 12
Change From Baseline in Body Weight at Month 12
Body weight measured in light clothing and without shoes.
Measured: Baseline, Month 12
Change From Baseline in Insulin-like Growth Factor 1 (IGF-I) SDS at Month 12
The SD score was calculated as the actual value of IGF-I minus mean reference value of IGF-1 divided by reference standard deviation of IGF-I. The mean and the SD vary depending on the age and sex of the participant. Change in IGF-I level (SD score) at Month 12 from Baseline was assessed. A higher score reflects a better outcome.
Measured: Baseline, Month 12
Change From Baseline in Insulin-like Growth Factor Binding Protein 3 (IGFBP-3) at Month 12
Measured: Baseline, Month 12
Number of Participants With Adverse Events (AEs)
An AE was defined as any untoward medical occurrence that develops or worsens in severity during the conduct of a clinical study and does not necessarily have a causal relationship to the study drug. SAEs included death, a life-threatening adverse event, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, a congenital anomaly or birth defect, or an important medical event that jeopardized the participant and required medical intervention to prevent 1 of the outcomes listed in this definition. A summary of serious and non-serious AEs regardless of causality is located in 'Reported Adverse Events module'.
Measured: Baseline up to Month 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Jan 2015 (estimated)
- Study start26 Aug 2015
- Primary completion23 Aug 2017
- Study completion23 Aug 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Growth DisordersStudies 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
- Masking
- None (open label)
- Enrolment
- 138 participants (actual)
Sponsor and investigators
- Lead sponsor
- Versartis Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Will Charlton, MD, Study director, Sponsor GmbH
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02339090
- Sponsor's protocol ID
- 14VR4
- Acronym
- VELOCITY
- Status last verified
- Nov 2022
- First submitted
- 7 Jan 2015
- Last updated
- 30 Dec 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Dec 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Comparison of Somavaratan (VRS-317), a Long-acting Human Growth Hormone, to Daily rhGH in a Phase 3, Randomized, One-year, Open-label, Multi-center, Non-inferiority Trial in Pre-pubertal Children With Growth Hormone Deficiency
- ClinicalTrials.gov ID
- NCT02339090
- Acronym / sponsor ID
- VELOCITY / 14VR4
- Conditions
- Growth Disorders
- Keywords
- VRS-317, Growth Hormone Deficiency, Long Acting Recombinant Growth Hormone, Long Acting Growth Hormone, Pediatric Growth Hormone Deficiency, Growth Hormone Replacement Therapy, Versartis, Xten
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 138 participants (actual)
- Lead sponsor
- Versartis Inc.Industry (pharma, biotech, devices)
- Study start
- 26 Aug 2015 (actual)
- Primary completion
- 23 Aug 2017 (actual)
- Study completion
- 23 Aug 2017 (actual)
- First posted
- 15 Jan 2015 (estimated)
- Results
- Posted 30 Dec 2022
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 compare a twice-monthly somavaratan dosing regimen for non-inferiority of treatment effect against daily injections of rhGH.
Detailed description
This study is designed as a pivotal study to compare the safety and efficacy of a selected dose regimen of somavaratan to daily rhGH. The study is a randomized, multi-center, open label study of 12 months duration. 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 |
|---|---|---|
| Somavaratan | Experimental | Drug: SomavaratanParticipants will receive somavaratan 3.5 milligrams (mg)/kilogram (kg) subcutaneous (SC) bolus injection twice monthly for 12 months. |
| rhGH | Active comparator | Drug: rhGHParticipants will receive commercially available rhGH (genotropin) 34 micrograms (μg)/kg once daily SC bolus injection for 12 months. |
Who may qualify
- Ages
- 3 to 11 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (7 listed)
- Chronological Age ≥ 3.0 years and ≤ 10.0 (girls) and ≤ 11.0 (boys).
- Pre-pubertal status: Absent breast development in girls, testicular volume < 4.0 mL in boys.
- Diagnosis of growth hormone deficiency (GHD) as documented by two or more growth hormone (GH) stimulation test results ≤ 10.0 ng/mL.
- Height standard deviation score (SDS) ≤ -2.0 at screening.
- Weight for Stature ≥ 10th percentile.
Key exclusion criteria (11 listed)
- Prior treatment with any growth promoting agent
- History of, or concurrent significant disease (for example, diabetes, cystic fibrosis, renal insufficiency).
- Chromosomal aneuploidy, significant gene mutations (other than those that cause GHD) or confirmed diagnosis of a named syndrome.
- A diagnosis of Attention Deficit Hyperactivity Disorder.
- Daily use of anti-inflammatory doses of glucocorticoid.
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 |
|---|---|---|
| SomavaratanAlso: Long-acting recombinant human growth hormone, VRS-317Somavaratan will be administered per dose and schedule specified in the arm description. | Drug | Somavaratan |
| rhGHAlso: daily growth hormone, recombinant growth hormone therapyrhGH will be administered per dose and schedule specified in the arm description. | Drug | rhGH |
Primary outcome
Annual Height Velocity
Time frame: 12 months
Height measured without shoes in triplicate by stadiometer. Annual height velocity was calculated as (height at Month 12 - height at Baseline)/(Month 12 Date - Baseline Date) * 365.25, where height was expressed as centimeters (cm) so that height velocity is expressed as centimeters per year (cm/yr). Annual height velocity after 12 months continuous treatment with either somavaratan or daily rhGH has been reported. Missing data was imputed using last observation carried forward. Least square (LS) mean was calculated using analysis of covariance (ANCOVA) model.
7 secondary outcomes
Change From Baseline in Height Standard Deviation Score (SDS) at Month 12
Time frame: Baseline, Month 12
Change From Baseline in Bone Age Relative to Chronological Age at Month 12, as Assessed by Central Reader
Time frame: Baseline, Month 12
Change From Baseline in Body Mass Index (BMI) at Month 12
Time frame: Baseline, Month 12
Change From Baseline in Body Weight at Month 12
Time frame: Baseline, Month 12
Change From Baseline in Insulin-like Growth Factor 1 (IGF-I) SDS at Month 12
Time frame: Baseline, Month 12
Change From Baseline in Insulin-like Growth Factor Binding Protein 3 (IGFBP-3) at Month 12
Time frame: Baseline, Month 12
Number of Participants With Adverse Events (AEs)
Time frame: Baseline up to Month 12
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Dec 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- 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
Sponsor GmbH
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Somavaratan and rhGH in people with Growth Disorders, run by Versartis Inc..
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 11 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 30 Dec 2022. Status can change between updates, so confirm with the study contact before making plans.
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?
Yes. Results are posted in the registry (first posted 30 Dec 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
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 30 Dec 2022.