A Randomized Safety and Efficacy Study of Somavaratan (VRS-317), a Long-Acting rhGH, in Pediatric Growth Hormone Deficiency.
J Clin Endocrinol Metab
; 101(3): 1091-7, 2016 Mar.
Article
en En
| MEDLINE
| ID: mdl-26672637
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 OUTCOMEMEASURES:
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.
Texto completo:
1
Colección:
01-internacional
Banco de datos:
MEDLINE
Asunto principal:
Hormona de Crecimiento Humana
/
Trastornos del Crecimiento
Tipo de estudio:
Clinical_trials
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Etiology_studies
Límite:
Child
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Child, preschool
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Female
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Humans
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Male
Idioma:
En
Revista:
J Clin Endocrinol Metab
Año:
2016
Tipo del documento:
Article