Versartis Trial in Children to Assess Long-Acting Growth Hormone (VERTICAL)
|Pediatric Growth Hormone Deficiency||Drug: Experimental: VRS-317||Phase 1 Phase 2|
|Study Design:||Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
|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|
- 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.
- Evaluate pharmacokinetic measures of VRS-317. [ Time Frame: 8 months ]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.
- Evaluate secondary efficacy measures after 6 months of VRS-317 dosing. [ Time Frame: 6 Months ]Secondary efficacy measures during Phase 2a include change in height SDS and change in bone age after 6 months VRS-317 treatment.
- Evaluate pharmacodynamic measures [ Time Frame: 8 Months ]Secondary outcome measures include evaluation of pharmacodynamics (PD) as measured by IGF-I and IGFBP-3 responses to single dose VRS-317 during Phase 1b and as measured by IGF-I and IGFBP-3 responses to multiple doses of VRS-317 after repeat dosing during Phase 2a. PD measures will be obtained at multiple timepoints during Phase 1b and Phase 2a.
|Study Start Date:||October 2012|
|Study Completion Date:||July 2014|
|Primary Completion Date:||July 2014 (Final data collection date for primary outcome measure)|
Active treatment arm
Drug: Experimental: VRS-317
Active treatment arm
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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01718041
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