Pediatric Growth Hormone DeficiencyPhase 2/3VISTANCT02068521
Versartis Long-Term Safety Study of Somavaratan
A Phase 2/3 study testing somavaratan in people with Pediatric Growth Hormone Deficiency, run by Versartis Inc..
Trial summary
Protocol 13VR3 is is a multi-center, open-label study assessing long-term somavaratan administration. Patients will be monitored for safety throughout their participation in the study. Read more about this study
- Who is it for?
- Ages 3 years and older
- Conditions the study is for
- Pediatric Growth Hormone Deficiency
- What is being tested?
- somavaratan
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 385 participants
- Where
- No sites listed yet
- Study dates
- 3 Mar 2014to 17 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 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Chronological Age ≥ 3.0 years.
- Pre-pubertal status.
- Diagnosis of GHD as documented by two or more GH stimulation test results ≤ 10.0 ng/mL.
- Normal thyroid function at Screening Visit in subjects not being treated for hypothyroidism.
- Normal adrenal function at Screening Visit or within 6 months of the Screening Visit, in subjects not being treated for adrenal insufficiency. Subjects with adrenal insufficiency must receive glucocorticoid treatment for a minimum of 4 weeks before study drug administration.
- Pathology relating to cause of GHD must be stable for at least 6 months prior to screening.
- Legally authorized representatives must be willing and able to give informed consent.
Who cannot join
- Prior/concomitant treatment with any growth promoting agent.
- Current, significant disease.
- Chromosomal aneuploidy, significant gene mutations or confirmed diagnosis of a named syndrome.
- Birth weight and/or birth length less than 5th percentile for gestational age.
- Prolonged daily use of anti-inflammatory doses of oral glucocorticoids.
- Prior history of malignancy.
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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, Chiari malformation, and spina bifida variants.
- Significant abnormality in screening studies.
- History of pancreatitis or undiagnosed chronic abdominal pain.
- History of spinal or total body irradiation.
- Other pituitary hormone deficiencies that are not properly treated.
About this study
Official title: An Open-Label, Long-Term Safety Study of A Long-acting Human Growth Hormone Somavaratan (VRS-317) in Children With Growth Hormone Deficiency
Protocol 13VR3 is is a multi-center, open-label study assessing long-term somavaratan administration.
Patients will be monitored for safety throughout their participation in the study. Safety will be monitored by physical examination, inspection of injection sites, vital signs, clinical laboratory determinations (including fasting glucose, insulin, and lipids), 12-lead ECGs (for new treatment naïve subjects and subjects not previously exposed to somavaratan), PK/PD assessments, and immunogenicity assessments.
Adverse events (AEs) and concomitant medications will be captured. AEs will be coded using CTCAE v 4.0. AEs will be coded using the MedDRA dictionary and CMs using the WHO Drug dictionary.
Read the detailed description
Protocol 13VR3 is a multi-center, open-label study assessing long-term somavaratan administration. It is open to subjects completing a somavaratan study in children with growth hormone deficiency (GHD), as well as up to 100 new treatment naïve subjects with GHD. All subjects will receive somavaratan 3.5 mg/kg twice monthly. The study will be conducted at approximately 70 Pediatric Endocrinology centers in the United States, Canada, and Europe.
- Conditions
- Pediatric Growth Hormone Deficiency
- Being tested
- somavaratan
- Keywords
- Growth Hormone Deficiency, Pediatric Growth Hormone Deficiency, GHD, PGHD, VRS-317, Versartis, Growth disorder, Long acting growth hormone, Growth hormone, Weekly growth hormone dosing, Semi-monthly growth hormone dosing, Monthly growth hormone dosing, IGF-I, Short stature, Height velocity, Annual height velocity, Growth rate, Somavaratan
- Medical subject headings
- Dwarfism, Pituitary, Growth Disorders, Dwarfism
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
somavaratanDrug
Also called VRS-317
Subcutaneous injection
| Study group | Role | What they receive |
|---|---|---|
| Treatment naive subjects with GHD | Experimental | Up to 100 new treatment naïve subjects with GHD will receive somavaratan 3.5mg/kg twice monthly. Receives: Drug: somavaratan |
| Subjects who have completed a somavaratan study | Experimental | All subjects after participation in (12VR2) or participation in the 14VR4 protocols have the option to receive somavaratan 3.5mg/kg twice monthly. Receives: Drug: somavaratan |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety
Safety assessments include recording of adverse events and concomitant medications, monitoring of injection sites, vital signs and clinical laboratory determinations.
Measured: Up to 4 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Feb 2014 (estimated)
- Study start3 Mar 2014
- Primary completion17 Nov 2017
- Study completion17 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
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 385 participants (actual)
Sponsor and investigators
- Lead sponsor
- Versartis Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Will Charlton, MD, Study director, Versartis Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02068521
- Sponsor's protocol ID
- 13VR3
- Acronym
- VISTA
- Status last verified
- Mar 2018
- First submitted
- 6 Feb 2014
- Last updated
- 9 Mar 2018
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 9 Mar 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label, Long-Term Safety Study of A Long-acting Human Growth Hormone Somavaratan (VRS-317) in Children With Growth Hormone Deficiency
- ClinicalTrials.gov ID
- NCT02068521
- Acronym / sponsor ID
- VISTA / 13VR3
- Conditions
- Pediatric Growth Hormone Deficiency
- Keywords
- Growth Hormone Deficiency, Pediatric Growth Hormone Deficiency, GHD, PGHD, VRS-317, Versartis, Growth disorder, Long acting growth hormone
- 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
- 385 participants (actual)
- Lead sponsor
- Versartis Inc.Industry (pharma, biotech, devices)
- Study start
- 3 Mar 2014 (actual)
- Primary completion
- 17 Nov 2017 (actual)
- Study completion
- 17 Nov 2017 (actual)
- First posted
- 21 Feb 2014 (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.
Protocol 13VR3 is is a multi-center, open-label study assessing long-term somavaratan administration.
Patients will be monitored for safety throughout their participation in the study. Safety will be monitored by physical examination, inspection of injection sites, vital signs, clinical laboratory determinations (including fasting glucose, insulin, and lipids), 12-lead ECGs (for new treatment naïve subjects and subjects not previously exposed to somavaratan), PK/PD assessments, and immunogenicity assessments.
Adverse events (AEs) and concomitant medications will be captured. AEs will be coded using CTCAE v 4.0. AEs will be coded using the MedDRA dictionary and CMs using the WHO Drug dictionary.
Detailed description
Protocol 13VR3 is a multi-center, open-label study assessing long-term somavaratan administration. It is open to subjects completing a somavaratan study in children with growth hormone deficiency (GHD), as well as up to 100 new treatment naïve subjects with GHD. All subjects will receive somavaratan 3.5 mg/kg twice monthly. The study will be conducted at approximately 70 Pediatric Endocrinology centers in the United States, Canada, and Europe.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Treatment naive subjects with GHD | Experimental | Drug: somavaratanUp to 100 new treatment naïve subjects with GHD will receive somavaratan 3.5mg/kg twice monthly. |
| Subjects who have completed a somavaratan study | Experimental | Drug: somavaratanAll subjects after participation in (12VR2) or participation in the 14VR4 protocols have the option to receive somavaratan 3.5mg/kg twice monthly. |
Who may qualify
- Ages
- 3 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- Chronological Age ≥ 3.0 years.
- Pre-pubertal status.
- Diagnosis of GHD as documented by two or more GH stimulation test results ≤ 10.0 ng/mL.
- Normal thyroid function at Screening Visit in subjects not being treated for hypothyroidism.
- Normal adrenal function at Screening Visit or within 6 months of the Screening Visit, in subjects not being treated for adrenal insufficiency. Subjects with adrenal insufficiency must receive glucocorticoid treatment for a minimum of 4 weeks before study drug administration.
Key exclusion criteria (14 listed)
- Prior/concomitant treatment with any growth promoting agent.
- Current, significant disease.
- Chromosomal aneuploidy, significant gene mutations or confirmed diagnosis of a named syndrome.
- Birth weight and/or birth length less than 5th percentile for gestational age.
- Prolonged daily use of anti-inflammatory doses of oral glucocorticoids.
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: VRS-317Subcutaneous injection | Drug | Subjects who have completed a somavaratan study, Treatment naive subjects with GHD |
Primary outcome
Safety
Time frame: Up to 4 years
Safety assessments include recording of adverse events and concomitant medications, monitoring of injection sites, vital signs and clinical laboratory determinations.
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 9 Mar 2018.
- Versartis websiteLink provided by the sponsor.
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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
Versartis Inc.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing somavaratan in people with Pediatric Growth Hormone Deficiency, 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 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: stopped early, as of the sponsor's last update on 9 Mar 2018. 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 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?
Safety (time frame: Up to 4 years). 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 9 Mar 2018.