Growth Hormone DeficiencyPhase 3NCT03145831
A Long-Term Safety Study of Somavaratan in Japanese Children With Growth Hormone Deficiency
A Phase 3 study testing Somavaratan in people with Growth Hormone Deficiency, run by Versartis Inc. at 1 site in United States.
Trial summary
This study is a multi-center, open-label safety study assessing long-term somavaratan administration. Read more about this study
- Who is it for?
- Ages 3 years and older
- Conditions the study is for
- 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
- 21 participants
- Where
- 1 siteUnited States
- Study dates
- 31 Mar 2017to 30 Nov 2017
- 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 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: Absent breast development in girls, testicular volume < 4.0 mL in boys.
- Subjects with GHD (diagnosed according to the current diagnostic guidelines) who are receiving treatment with daily rhGH.
- Normal thyroid function at screening visit in subjects not being treated for hypothyroidism. Subjects requiring thyroxine replacement must be considered adequately treated by the PI and Medical Monitor.
- Normal adrenal function (morning cortisol and/or local stimulation test) 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.
- Willingness to discontinue daily rhGH therapy.
- Legally authorized representatives must be willing and able to give informed consent
Who cannot join
- 1. Prior (in the last 12 months) or concomitant treatment with a growth promoting agent other than rhGH [e.g., IGF-I, GH releasing hormone (GHRH), sex steroids (except when used as primer for GH stimulation test), aromatase inhibitors and/or GnRH agonist].
- 2. Current significant disease (e.g., diabetes, cystic fibrosis, renal insufficiency). In all cases of concurrent disease, screening must be approved in writing by the medical monitor.
- 3. Chromosomal aneuploidy, significant gene mutations (other than those that cause GHD) or confirmed diagnosis of a named syndrome (e.g., Russell Silver, Prader Willi, Turner, etc.).
- 4. Birth weight and/or birth length less than 5th percentile for gestational age using local gestational age growth charts.
- 5. Prolonged daily (> 14 days) use of anti-inflammatory doses of oral glucocorticoids.
- 6. Prior history of malignancy. 7. Treatment with an investigational drug in the 30 days prior to screening. 8. Known allergy to constituents of the study drug formulation. 9. Ocular findings suggestive of increased intracranial pressure and/or retinopathy at screening.
Show all 11Show fewer
- 10. Significant spinal abnormalities including scoliosis, kyphosis, Chiari malformation, and spina bifida variants.
- 11. Significant abnormality in screening laboratory studies (as assessed by PI and medical monitor).
- 12. Current social conditions which would prevent completion of study activities (e.g., planned family move to a distant location).
- 13. History of pancreatitis or undiagnosed chronic abdominal pain. 14. History of spinal or total body irradiation. 15. Presence of other pituitary hormone deficiencies that are not properly treated.
- 16. Unwillingness to provide consent for participation in all trial activities
Trial locations and contacts
1 site in 1 country.
About this study
Official title: An Open-Label, Long-Term Safety Study of Long-acting Human Growth Hormone Somavaratan (VRS-317) in Japanese Children With Growth Hormone Deficiency
This study is a multi-center, open-label safety study assessing long-term somavaratan administration.
Read the detailed description
This study is a multi-center, open-label safety study assessing long-term somavaratan administration. It is open to subjects completing a somavaratan Japanese Phase 2/3 study (Protocol J14VR5) in children with growth hormone deficiency (GHD), as well as approximately 20 new children currently receiving daily rhGH therapy for GHD (switch subjects). For switch subjects, the first dose of somavaratan will be administered approximately 48 hours after the last dose of the daily rhGH. All subjects will receive somavaratan 3.5mg/kg twice-monthly. The study will be conducted at approximately 40 medical institutions in Japan.
- Conditions
- Growth Hormone Deficiency
- Being tested
- Somavaratan
- Keywords
- Pediatrics
- Medical subject headings
- Dwarfism, Pituitary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SomavaratanDrug
Also called VRS 317
All subjects will receive somavaratan 3.5 mg/kg twice monthly (every 15 days ± 2 days). Administered as a subcutaneous bolus injection.
| Study group | Role | What they receive |
|---|---|---|
| Somavaratan | Experimental | fusion protein, subcutaneous bolus injection, 3.5 mg/kg twice monthly Receives: Drug: Somavaratan |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse Events
Incidence and severity of adverse events
Measured: 12 months
Secondary outcomes (3)
Height velocity
Comparison of Height Velocity (HV) and HV-SDS before and after switching therapy
Measured: 12 months
IGF-I expression
Change from Day 1
Measured: 12 months
Immunogenicity
Evaluated by anti-drug antibody response
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 May 2017
- Study start31 Mar 2017
- 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 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 21 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
- NCT03145831
- Sponsor's protocol ID
- J15VR6
- Status last verified
- Mar 2018
- First submitted
- 5 Apr 2017
- Last updated
- 9 Mar 2018
- 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 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 Long-acting Human Growth Hormone Somavaratan (VRS-317) in Japanese Children With Growth Hormone Deficiency
- ClinicalTrials.gov ID
- NCT03145831
- Acronym / sponsor ID
- J15VR6
- Conditions
- Growth Hormone Deficiency
- Keywords
- Pediatrics
- Phase
- Phase 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
- 21 participants (actual)
- Lead sponsor
- Versartis Inc.Industry (pharma, biotech, devices)
- Study start
- 31 Mar 2017 (actual)
- Primary completion
- 30 Nov 2017 (actual)
- Study completion
- 30 Nov 2017 (actual)
- First posted
- 9 May 2017 (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 a multi-center, open-label safety study assessing long-term somavaratan administration.
Detailed description
This study is a multi-center, open-label safety study assessing long-term somavaratan administration. It is open to subjects completing a somavaratan Japanese Phase 2/3 study (Protocol J14VR5) in children with growth hormone deficiency (GHD), as well as approximately 20 new children currently receiving daily rhGH therapy for GHD (switch subjects). For switch subjects, the first dose of somavaratan will be administered approximately 48 hours after the last dose of the daily rhGH. All subjects will receive somavaratan 3.5mg/kg twice-monthly. The study will be conducted at approximately 40 medical institutions in Japan.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Somavaratan | Experimental | Drug: Somavaratanfusion protein, subcutaneous bolus injection, 3.5 mg/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 (8 listed)
- Chronological Age ≥ 3.0 years.
- Pre-pubertal status: Absent breast development in girls, testicular volume < 4.0 mL in boys.
- Subjects with GHD (diagnosed according to the current diagnostic guidelines) who are receiving treatment with daily rhGH.
- Normal thyroid function at screening visit in subjects not being treated for hypothyroidism. Subjects requiring thyroxine replacement must be considered adequately treated by the PI and Medical Monitor.
- Normal adrenal function (morning cortisol and/or local stimulation test) 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 (11 listed)
- 1. Prior (in the last 12 months) or concomitant treatment with a growth promoting agent other than rhGH [e.g., IGF-I, GH releasing hormone (GHRH), sex steroids (except when used as primer for GH stimulation test), aromatase inhibitors and/or GnRH agonist].
- 2. Current significant disease (e.g., diabetes, cystic fibrosis, renal insufficiency). In all cases of concurrent disease, screening must be approved in writing by the medical monitor.
- 3. Chromosomal aneuploidy, significant gene mutations (other than those that cause GHD) or confirmed diagnosis of a named syndrome (e.g., Russell Silver, Prader Willi, Turner, etc.).
- 4. Birth weight and/or birth length less than 5th percentile for gestational age using local gestational age growth charts.
- 5. Prolonged daily (> 14 days) 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 317All subjects will receive somavaratan 3.5 mg/kg twice monthly (every 15 days ± 2 days). Administered as a subcutaneous bolus injection. | Drug | Somavaratan |
Primary outcome
Adverse Events
Time frame: 12 months
Incidence and severity of adverse events
3 secondary outcomes
Height velocity
Time frame: 12 months
IGF-I expression
Time frame: 12 months
Immunogenicity
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 9 Mar 2018.
- Individual participant dataSharing planned: No
Related trials for Growth Hormone Deficiency
RecruitingPhase 3
Trial Investigating the Efficacy and Safety of Weekly Lonapegsomatropin Compared to Daily Somatropin in Children and Adolescents With Short Stature or Growth Failure Due to Growth Hormone Sufficient Disorders
Turner Syndrome, Short Stature Homeobox Gene Mutation
RecruitingPhase 3
Randomized Clinical Trial of Two Different Initial Growth Hormone Doses in Children
Growth Disorders, Growth Failure
RecruitingPhase 3
Phase 3 Study of LUM-201 in Children With Growth Hormone Deficiency
Growth Hormone Deficiency (GHD)
RecruitingPhase 3
Growth Hormone Replacement Therapy in Veterans With Mild Traumatic Brain Injury (mTBI) and Adult Growth Hormone Deficiency (AGHD)
Adult Growth Hormone Deficiency, Mild Traumatic Brain Injury
Enrolling by invitationObservational study
Influence of Adherence to Growth Hormone Therapy (GHT) With Norditropin® on Near Final Height in Patients With Growth Hormone Deficiency (GHD) and Born Small for Gestational Age (SGA)
Growth Hormone Deficiency in Children, Born Small for Gestational Age
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 3 study testing Somavaratan in people with Growth Hormone Deficiency, run by Versartis Inc. at 1 site in United States.
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 8 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: 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.
Where is the study taking place?
The record lists 1 site in United States. 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?
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?
Adverse Events (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 9 Mar 2018.