Versartis Trial in Children to Assess Long-Acting Growth Hormone

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.

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