ECE2017 Guided Posters Developmental & Protein Endocrinology (9 abstracts)
3-year safety and efficacy Update of the VERTICAL & VISTA trials of somavaratan (VRS-317), a long-acting rhGH, in children with Growth Hormone Deficiency (GHD)
Bradley S. Miller 1 , Wayne V. Moore 2, , Patricia Y. Fechner 4, , Quentin L. Van Meter 6 , John S. Fuqua 7 , David Ng 8 , Eric Humphriss 9 , R. William Charlton 9 & George M. Bright 9
1University of Minnesota Masonic Children’s Hospital, Minneapolis, MN, USA; 2Children’s Mercy Hospital, Kansas City, MO, USA; 3University of Missouri-Kansas City, Kansas City, MO, USA; 4Seattle Children’s Hospital, Seattle, WA, USA; 5University of Washington, Seattle, Seattle, WA, USA; 6Van Meter Pediatric Endocrinology, P.C., Atlanta, GA, USA; 7Indiana University School of Medicine, Indianapolis, IN, USA; 8ResearchPoint Global, Inc., Austin, TX, USA; 9Versartis, Inc., Menlo Park, CA, USA.
Requirement for daily rhGH injections is a treatment burden that can compromise adherence and efficacy in patients with GHD. Somavaratan is a novel long-acting rhGH fusion protein in clinical development for pediatric and adult GHD. In a multicenter, randomized Phase 1b/2a study, somavaratan significantly improved height velocity (HV) and IGF-I in pre-pubertal children with GHD. Preliminary efficacy and safety in subjects who have completed 3 years of somavaratan treatment are presented. Of 64 subjects randomized in the Phase 2a VERTICAL study to receive 5.0 mg/kg/month (weekly/twice-monthly/monthly dosing) for 6 months, 60 elected to continue treatment in the long-term safety study, VISTA. Initial IGF-I response supported a dose increase, and all subjects transitioned to somavaratan 3.5 mg/kg twice-monthly by the 2nd treatment year. Data cutoff was December 8, 2016. Of 48 subjects (24 males, 24 females), the mean±SD age was 7.6±2.4 years, and mean IGF-I SDS was −1.6±0.8 at baseline. In Year 3, IGF-I SDS increased to 1.2±1.7 at peak (3–5 days post-injection) and −0.3±1.0 at trough (end of dosing cycle). Mean HV remained consistent during Years 1, 2, and 3, at 8.4±2.0, 8.3±1.6, and 7.8±1.6 cm/year, and height (HT)-SDS continued to increase from −2.6±0.6 at baseline to −2.1±0.6, −1.6±0.7, and −1.2±0.8; delay in bone age (years) was −1.48±0.81 at baseline, −1.34±0.89 (Year 1, n=48), −1.09±1.02 (Year 2, n=47), and −0.66±0.82 (Year 3, n=25). Treatment-related AEs were generally transient and mild. Overall, IGF-I, HV, HT-SDS, and bone age showed continued improvement through 3 years of somavaratan treatment in pre-pubertal children with GHD. Dose increase to somavaratan 3.5 mg/kg twice-monthly resulted in consistent growth rates through 3 years of treatment, and overall Year 3 growth was consistent with daily rhGH from US registries. A Phase 3 study of somavaratan 3.5 mg/kg twice-monthly in treatment-naïve GHD children is ongoing.
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Endocrine Abstracts
ISSN 1470-3947 (print) | ISSN 1479-6848 (online)
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