Selumetinib in Japanese pediatric patients with neurofibromatosis type 1 and symptomatic, inoperable plexiform neurofibromas: An open-label, phase I study.

ABSTRACT:

Background

Plexiform neurofibromas (PN) are a manifestation of neurofibromatosis type 1 (NF1) that may cause morbidity and impact health-related quality of life (HRQoL). Selumetinib (ARRY-142886, AZD6244) is an orally available, selective, mitogen-activated protein kinase kinase 1/2 inhibitor approved for children with NF1 and symptomatic, inoperable PN in regions including the USA (aged ≥2 years), EU (≥3 years), and Japan (≥3 years). This open-label, single-arm, phase I study evaluated selumetinib in Japanese children with NF1 and symptomatic, inoperable PN.

Methods

Eligible patients (aged 3-18 years) received oral selumetinib (25 mg/m² twice daily) continuously in 28-day cycles in a fasted state. Primary objectives were safety and tolerability. Secondary objecti

SUBMITTER: Suenobu S 

PROVIDER: S-EPMC10243862 | biostudies-literature | 2023 Jan-Dec

REPOSITORIES: biostudies-literature