Pharmacokinetics and Safety of Selumetinib Granule Formulation in Children With Symptomatic, Inoperable Neurofibromatosis Type 1-Related Plexiform Neurofibromas (SPRINKLE; phase I/II)

Key Points

• Selumetinib granule formulation shows similar pharmacokinetics to the capsule form, indicating effective dosing.
• The study emphasizes a manageable safety profile, making it favorable for young children with specific health conditions.
• The formulation is palatable for children, which is vital for adherence to treatment protocols.
• Analysis focused on children with symptomatic, inoperable plexiform neurofibromas caused by neurofibromatosis type 1.