Long-Acting Growth Hormone in Children Compared to Daily rhGH

Comparison of Somavaratan (VRS-317), a Long-acting Human Growth Hormone, to Daily rhGH in a Phase 3, Randomized, One-year, Open-label, Multi-center, Non-inferiority Trial in Pre-pubertal Children With Growth Hormone Deficiency

The trial will compare a twice-monthly somavaratan dosing regimen for non-inferiority of treatment effect against daily injections of rhGH.

This study is designed as a pivotal study to compare the safety and efficacy of a selected dose regimen of somavaratan to daily rhGH. The study is a randomized, multi-center, open label study of 12 months duration. The primary endpoint is height velocity at 12 months.

Inclusion Criteria:

• Chronological Age ≥ 3.0 years and ≤ 10.0 (girls) and ≤ 11.0 (boys).
• Pre-pubertal status: Absent breast development in girls, testicular volume < 4.0 mL in boys.
• Diagnosis of growth hormone deficiency (GHD) as documented by two or more growth hormone (GH) stimulation test results ≤ 10.0 ng/mL.
• Height standard deviation score (SDS) ≤ -2.0 at screening.
• Weight for Stature ≥ 10th percentile.
• Insulin-like growth factor-I (IGF-I) SDS ≤ -1.0 at screening.
• Delayed bone age (≥ 6 months).

Exclusion Criteria:

• Prior treatment with any growth promoting agent
• History of, or concurrent significant disease (for example, diabetes, cystic fibrosis, renal insufficiency).
• Chromosomal aneuploidy, significant gene mutations (other than those that cause GHD) or confirmed diagnosis of a named syndrome.
• A diagnosis of Attention Deficit Hyperactivity Disorder.
• Daily use of anti-inflammatory doses of glucocorticoid.
• Prior history of leukemia, lymphoma, sarcoma or cancer.
• Treatment with an investigational drug in the 30 days prior to screening.
• Known allergy to constituents of the study drug formulation.
• 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