A Phase I/II, Single-Arm, Open label Study to Evaluate the Pharmacokinetics, Safety/Tolerability and Efficacy of the Selumetinib Granule Formulation in Children Aged ≥ 1 to < 7 Years with Neurofibromatosis Type 1 (NF1) Related Symptomatic, Inoperable Plexiform Neurofibromas (PN) (SPRINKLE).

Neurofibromatosis Type 1 (NF1) Related Plexiform Neurofibromas (PN)

Selumetinib AUC0-12 derived after single dose administration. Safety and tolerability will be evaluated in terms of AEs, clinical safety laboratory assessments, physical examination, weight, vital signs, ECG, ECHO, ophthalmologic assessment, MRI/X-ray, and performance status. Assessments related to AEs will include: occurrence/frequency; relationship to study intervention; CTCAE grade; seriousness; death; AEs leading to discontinuation of study intervention; AEs of special interest.

Parent-reported observer palatability assessment. Plasma concentrations and PK parameters of selumetinib including: AUC0-6, AUC0-12, AUC0-24, Cmax, Rac Cmax, Rac AUC0-12, Vss/F, Vz/F, t½λz, AUClast, CL/F, tmax and tlast. Plasma concentrations and PK parameters of N desmethyl selumetinib including: Cmax, AUC0-6, AUC0-12, AUClast, tmax, tlast, Rac Cmax and Rac AUC0-12. Parent-to-metabolite ratio for AUC0-6, AUC0-12 and AUC0-24 and Cmax. Objective Response Rate (ORR). See CSP for more details.

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