Versartis Trial in Children to Assess Long-Acting Growth Hormone

Study Overview
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Brief Summary
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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
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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.

Official Title
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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

Conditions
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Pediatric Growth Hormone Deficiency

Intervention / Treatment
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* Drug: Experimental: VRS-317


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 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. Exclusion Criteria: 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 Known allergy to constituents of the study drug formulation Abnormal ocular findings at screening Significant abnormality in screening laboratory studies

Ages Eligible for Study
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Minimum Age: 3 Years
Maximum Age: 11 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: VRS-317<br>Active treatment arm | Drug: Experimental: VRS-317<br>* Active treatment arm<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. | 8 Months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. | 8 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Growth, Growth hormone, Growth hormone deficiency, Pediatric growth hormone deficiency, Long acting growth hormone, GHD, PGHD, VRS 317, Exten, Versartis