Pharmacokinetics, Safety and Efficacy of the Selumetinib Granule Formulation in Children Aged ≥1 to <7 Years With NF1-related Symptomatic, Inoperable PN

Summary

This study is designed to define a dosing regimen and assess the pharmacokinetics(PK) and safety of the granule formulation; the study will also include descriptive analyses of exploratory efficacy endpoints. The study will inform the benefit risk profile of the granule formulation in children aged ≥ 1 to \< 7 years with NF1 related symptomatic, inoperable PN.

Timeline

Start

2022-01-21

Primary completion

2027-11-30

Completion

2028-04-28

Publications

• Hernaiz Driever P, Kordes UR, Brecht IB, Saletti V, Fisher MJ, Doughton G, Arefayene M, Rigazio A, Lluch N, Llorente N, Diede SJ, Salvador H. Pharmacokinetics and Safety of Selumetinib Granule Formulation in Children With Symptomatic, Inoperable Neurofibromatosis Type 1-Related Plexiform Neurofibromas (SPRINKLE; phase I/II). J Clin Oncol. 2026 Apr;44(10):849-860. doi: 10.1200/JCO-25-01447. Epub 2026 Jan 27.

Drugs

Indications