Pediatric Growth Hormone DeficiencyPhase 1/2VERTICALNCT01718041
Versartis Trial in Children to Assess Long-Acting Growth Hormone
A Phase 1/2 study testing Experimental: VRS-317 in people with Pediatric Growth Hormone Deficiency, run by Versartis Inc. at 23 sites in United States.
Trial summary
This is a Phase 1b/2a study of VRS-317 (long-acting growth hormone) in pediatric patients with growth hormone deficiency. During Phase 1b, pediatric patients each will receive a single subcutaneous injection of VRS-317. Read more about this study
- Who is it for?
- Ages 3 to 11 years
- Conditions the study is for
- Pediatric Growth Hormone Deficiency
- What is being tested?
- Experimental: VRS-317
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 64 participants
- Where
- 23 sitesUnited States
- Study dates
- Oct 2012to Jul 2014
- Phase
- Phase 1/2
- 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 11 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Chronological Age ≥ 3.0 years and ≤ 11.0
- Diagnosis of GHD as documented by GH stimulation test
- Below average height SDS at screening
- Appropriate weight for Stature
- Decreased IGF-I SDS at screening
- Delayed bone age
- Normal thyroid function test results at screening visit
- Legally authorized representative informed consent.
Who cannot join
- Prior treatment with any growth promoting agent
- Documented history of, or current, significant disease
- Chromosomal aneuploidy, significant gene mutations
- Diagnosis of Attention Deficit Hyperactivity Disorder
- Daily use of anti-inflammatory doses of glucocorticoid
- Prior history of leukemia, lymphoma, sarcoma or cancer
Show all 9Show fewer
- Known allergy to constituents of the study drug formulation
- Abnormal ocular findings at screening
- Significant abnormality in screening laboratory studies
Trial locations and contacts
23 sites in 1 country.
23 sites in 1 country
United States23 sites
Study site
Atlanta, Georgia
Study site
Baltimore, Maryland
Study site
Buffalo, New York
Study site
Cleveland, Ohio
Study site
Clovis, California
Study site
Columbus, Ohio
Study site
Greenwood Village, Colorado
Study site
Indianapolis, Indiana
Study site
Kansas City, Missouri
Study site
Livingston, New Jersey
Study site
Los Angeles, California
Study site
Mineola, New York
Study site
Minneapolis, Minnesota
Study site
Morristown, New Jersey
Study site
Orlando, Florida
Study site
San Antonio, Texas
Study site
San Diego, California
Study site
San Francisco, California
Study site
Seattle, Washington
Study site
Sleepy Hollow, New York
Study site
Springfield, Massachusetts
Study site
Tallahassee, Florida
Study site
Tampa, Florida
About this study
Official title: A Long-acting Human Growth Hormone (VRS-317) in Pre-pubertal Children With Growth Hormone Deficiency: A Randomized, Open-label, Multi-center, Phase 1b/2a Study of Safety, Tolerability, Pharmacokinetics (PK), Pharmacodynamics (PD) and Efficacy After Subcutaneous Administration for 6 Months
This is a Phase 1b/2a study of VRS-317 (long-acting growth hormone) in pediatric patients with growth hormone deficiency. During Phase 1b, pediatric patients each will receive a single subcutaneous injection of VRS-317. During the Phase 2a stage, patients will receive 6 months of VRS-317 treatment at dose levels selected from the Phase 1b stage. The primary endpoints for the study are to determine the safety and efficacy of repeat dose VRS-317.
Read the detailed description
In Phase 1b, separate cohorts of patients will be tested in a single ascending dose format. Safety review committee meetings will take place prior to escalating to each increasing dose level. Enrolled patients will be monitored for 60 days for safety with PK/PD and safety labs collected.
Two dose levels will be selected after completion of Phase 1b to be tested during Phase 2a (6 months of continuous VRS-317 treatment). Safety and PK assessments will be made during the Phase 2a stage. Patient heights will be measured by stadiometer.
- Conditions
- Pediatric Growth Hormone Deficiency
- Being tested
- Experimental: VRS-317
- Keywords
- Growth hormone, Growth hormone deficiency, Growth, Pediatric growth hormone deficiency, Long acting growth hormone, GHD, PGHD, VRS 317, Exten, Versartis
- Medical subject headings
- Dwarfism, Pituitary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Experimental: VRS-317Drug
Active treatment arm
| Study group | Role | What they receive |
|---|---|---|
| VRS-317 | Experimental | Active treatment arm Receives: Drug: Experimental: VRS-317 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Phase 1b and 2a: Evaluate the safety and tolerability of multiple dosing levels of subcutaneous (SC) VRS-317 in pediatric growth hormone deficient (GHD) patients. Phase 2a: Evaluate the efficacy of multiple dose levels of VRS-317
Safety observations will include the number of patients with adverse events, concomitant medications, safety labs, vital signs, physical exams, single dose immunogenicity (Phase 1b), and repeat dose immunogenicity (Phase 2a).
The primary efficacy outcome measure is the height velocity over 6 months as measured by standing height taken with stadiometer at baseline and after 6 Months of VRS-317 dosing in Phase 2a.
Measured: 8 Months
Secondary outcomes (1)
Evaluate pharmacokinetic measures of VRS-317.
Secondary outcome measures include evaluation of pharmacokinetics (PK) of VRS-317 including Cmax, Tmax and AUC. PK measures will be analysed at multiple timepoints after single dosing of VRS-317 during Phase 1b and after repeat dosing of VRS-317 during Phase 2a.
Measured: 8 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Oct 2012 (estimated)
- Study startOct 2012
- Primary completionJul 2014
- Study completionJul 2014
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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 64 participants (actual)
Sponsor and investigators
- Lead sponsor
- Versartis Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01718041
- Sponsor's protocol ID
- VRS-317 Protocol 12VR2
- Acronym
- VERTICAL
- Status last verified
- Jul 2022
- First submitted
- 19 Oct 2012
- Last updated
- 26 Jul 2022
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Jul 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Long-acting Human Growth Hormone (VRS-317) in Pre-pubertal Children With Growth Hormone Deficiency: A Randomized, Open-label, Multi-center, Phase 1b/2a Study of Safety, Tolerability, Pharmacokinetics (PK), Pharmacodynamics (PD) and Efficacy After Subcutaneous Administration for 6 Months
- ClinicalTrials.gov ID
- NCT01718041
- Acronym / sponsor ID
- VERTICAL / VRS-317 Protocol 12VR2
- Conditions
- Pediatric Growth Hormone Deficiency
- Keywords
- Growth hormone, Growth hormone deficiency, Growth, Pediatric growth hormone deficiency, Long acting growth hormone, GHD, PGHD, VRS 317
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 64 participants (actual)
- Lead sponsor
- Versartis Inc.Industry (pharma, biotech, devices)
- Study start
- Oct 2012
- Primary completion
- Jul 2014 (actual)
- Study completion
- Jul 2014 (actual)
- First posted
- 31 Oct 2012 (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.
This is a Phase 1b/2a study of VRS-317 (long-acting growth hormone) in pediatric patients with growth hormone deficiency. During Phase 1b, pediatric patients each will receive a single subcutaneous injection of VRS-317. During the Phase 2a stage, patients will receive 6 months of VRS-317 treatment at dose levels selected from the Phase 1b stage. The primary endpoints for the study are to determine the safety and efficacy of repeat dose VRS-317.
Detailed description
In Phase 1b, separate cohorts of patients will be tested in a single ascending dose format. Safety review committee meetings will take place prior to escalating to each increasing dose level. Enrolled patients will be monitored for 60 days for safety with PK/PD and safety labs collected.
Two dose levels will be selected after completion of Phase 1b to be tested during Phase 2a (6 months of continuous VRS-317 treatment). Safety and PK assessments will be made during the Phase 2a stage. Patient heights will be measured by stadiometer.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| VRS-317 | Experimental | Drug: Experimental: VRS-317Active treatment arm |
Who may qualify
- Ages
- 3 to 11 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (8 listed)
- Chronological Age ≥ 3.0 years and ≤ 11.0
- Diagnosis of GHD as documented by GH stimulation test
- Below average height SDS at screening
- Appropriate weight for Stature
- Decreased IGF-I SDS at screening
Key exclusion criteria (9 listed)
- Prior treatment with any growth promoting agent
- Documented history of, or current, significant disease
- Chromosomal aneuploidy, significant gene mutations
- 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 |
|---|---|---|
| Experimental: VRS-317Active treatment arm | Drug | VRS-317 |
Primary outcome
Phase 1b and 2a: Evaluate the safety and tolerability of multiple dosing levels of subcutaneous (SC) VRS-317 in pediatric growth hormone deficient (GHD) patients. Phase 2a: Evaluate the efficacy of multiple dose levels of VRS-317
Time frame: 8 Months
Safety observations will include the number of patients with adverse events, concomitant medications, safety labs, vital signs, physical exams, single dose immunogenicity (Phase 1b), and repeat dose immunogenicity (Phase 2a). The primary efficacy outcome measure is the height velocity over 6 months as measured by standing height taken with stadiometer at baseline and after 6 Months of VRS-317 dosing in Phase 2a.
1 secondary outcome
Evaluate pharmacokinetic measures of VRS-317.
Time frame: 8 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Jul 2022.
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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.
The record lists no contact. Use the official record to reach the sponsor.
23 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Experimental: VRS-317 in people with Pediatric Growth Hormone Deficiency, run by Versartis Inc. at 23 sites 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 9 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 26 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 23 sites 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Phase 1b and 2a: Evaluate the safety and tolerability of multiple dosing levels of subcutaneous (SC) VRS-317 in pediatric growth hormone deficient (GHD) patients. Phase 2a: Evaluate the efficacy of multiple dose levels of VRS-317 (time frame: 8 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 26 Jul 2022.