A randomized safety and efficacy study of somavaratan (VRS-317), a long-acting rhGH, in pediatric growth hormone deficiency

Wayne V. Moore, Huong Jil Nguyen, Gad B. Kletter, Bradley S. Miller, Douglas Rogers, David Ng, Jerome A. Moore, Eric Humphriss, Jeffrey L. Cleland, George M. Bright

Research output: Contribution to journalArticlepeer-review

23 Scopus citations

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 weekly, 2.5 mg/kg twice monthly, or 5.0 mg/kg monthly for 6 months. Main Outcome Measures: Safety, pharmacokinetics, pharmacodynamics, 6-month height velocity (HV). Results: Somavaratan pharmacokinetics was linearly proportional to dose; dose-dependent increases in the magnitude and duration of IGF-1 responses enabled weekly, twice-monthly or monthly dosing. A single dose of somavaratan sustained IGF-1 responses for up to 1 month. No somavaratan or IGF-1 accumulation occurred with repeat dosing. Mean annualized HVs for somavaratan administered monthly, twice monthly, or weekly (7.86±2.5, 8.61±2.7, and 7.58±2.5 cm/y, respectively) were similar between groups. Adverse events were mostly mild and transient. Conclusions: Somavaratan demonstrated clinically meaningful improvements in HV and IGF-1 in prepubertal children with GHD, with no significant differences between monthly, twice-monthly, or weekly dosing.

Original languageEnglish (US)
Pages (from-to)1091-1097
Number of pages7
JournalJournal of Clinical Endocrinology and Metabolism
Volume101
Issue number3
DOIs
StatePublished - Mar 2016

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