Versartis Trial in Pre-pubertal Japanese Children With Growth Hormone Deficiency (GHD) to Assess Long-Acting Growth Hormone (Somavaratan, VRS-317)

Study Overview
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Brief Summary
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The trial will consist of three stages: 1) a 30 day Phase 2 PK and PD evaluation of somavaratan, 2) an optional Phase 2 Extension and 3) a 12 month Phase 3 safety and efficacy stage. Upon completion of the PK/PD stage, the PK/PD profiles for the GHD children in this study will be compared to the PK/PD profiles for the GHD children treated in the Western study Phase 1b/2a study (Protocol 12VR2) and identify the somavaratan dose to be used in the Phase 3 stage in Japan. The Phase 3 stage will continue dosing for 12 months to obtain safety and efficacy data on 48 subjects.

Detailed Description
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The trial will consist of three stages: 1) a 30 day Phase 2 PK and PD evaluation of somavaratan, 2) an optional Phase 2 Extension and 3) a 12 month Phase 3 safety and efficacy stage. The study is a randomized, multi-center, open label study. The primary endpoint is height velocity at 12 months.

Official Title
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A Randomized, Phase 2/3, Open-Label, Multi-center Study of the Pharmacokinetics, Pharmacodynamics, Safety and Efficacy of A Long-acting Human Growth Hormone (Somavaratan, VRS-317) in Pre-pubertal Japanese Children With Growth Hormone Deficiency (GHD)

Conditions
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Pediatric Growth Hormone Deficiency, Growth Disorders

Intervention / Treatment
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* Drug: Somavaratan (VRS-317)


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Chronological Age ≥ 3.0 years and ≤ 9.0 years (girls) or ≤ 10.0 years (boys) Pre-pubertal status Diagnosis of GHD as documented by two or more GH stimulation test results Height SD score ≤ -2.0 at screening Weight for Stature ≥ 10th percentile IGF-I SD score ≤ -1.0 at screening Delayed bone age Exclusion Criteria: Prior treatment with any growth promoting agent History of, or current, significant disease Chromosomal aneuploidy, significant gene mutations (other than those that cause GHD) or confirmed diagnosis of a named syndrome Birth weight and/or birth length less than 5th percentile for gestational age A diagnosis of Attention Deficit Hyperactivity Disorder Daily use of anti-inflammatory doses of glucocorticoid Prior history of leukemia, lymphoma, sarcoma or cancer Ocular findings suggestive of increased intracranial pressure and/or retinopathy at screening Significant spinal abnormalities including scoliosis, kyphosis and spina bifida variants Significant abnormality in screening laboratory studies

Ages Eligible for Study
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Minimum Age: 3 Years
Maximum Age: 10 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
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Phase 2: Somavaratan (VRS-317)<br>Active treatment arm | Drug: Somavaratan (VRS-317)<br>* Long acting recombinant human growth hormone<br>* Other names: Long acting recombinant human growth hormone;|
| Experimental: Phase 3: Somavaratan (VRS-317)<br>Somavaratan long acting recombinant human growth hormone administered subcutaneously twice-monthly | Drug: Somavaratan (VRS-317)<br>* Long acting recombinant human growth hormone<br>* Other names: Long acting recombinant human growth hormone;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Efficacy (Annual Height velocity) | Annual Height velocity. | 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Pharmacodynamics (IGF-I responses to study drug administration) | IGF-I responses to study drug administration. | 12 months | 
| Pharmacodynamics (IGFBP-3 responses to study drug administration) | IGFBP-3 responses to study drug administration. | 12 months | 
| Safety (Number of subjects with adverse events ) | Number of subjects with adverse events (including repeat dose immunogenicity). | 12 months | 
| Safety (Concomitant medications) | Concomitant medications | 12 months | 
| Safety (Safety labs) | Safety labs | 12 months | 
| Safety (Vital signs) | Vital signs | 12 months | 
| Safety (Physical Exams) | Physical Exams | 12 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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GHD