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

Launched by VERSARTIS INC. · Apr 8, 2015

Keywords

ClinConnect Summary

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.

Gender

ALL

Eligibility criteria

• 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