A Randomized Safety and Efficacy Study of Somavaratan (VRS-317), a Long-Acting rhGH, in Pediatric Growth Hormone Deficiency.

ABSTRACT:

Context

Somavaratan (VRS-317) is a long-acting form of recombinant human GH under development for children and adults with GH deficiency (GHD).

Objectives

To determine the optimal somavaratan dose regimen to normalize IGF-1 in pediatric GHD and to evaluate safety and efficacy of somavaratan over 6 months.

Design

Open-label, multicenter, single ascending dose study followed by 6-month randomized comparison of 3 dosing regimens.

Setting

Twenty-five United States pediatric endocrinology centers.

Patients

Naive-to-treatment, prepubertal children with GHD (n = 68).

Intervention(s)

Patients received single sc doses of somavaratan (0.8, 1.2, 1.8, 2.7, 4.0, or 6.0 mg/kg) during the 30-day dose-finding phase, then were randomized to somavaratan 1.15 mg/kg

SUBMITTER: Moore WV 

PROVIDER: S-EPMC4803167 | biostudies-literature | 2016 Mar

REPOSITORIES: biostudies-literature