Selumetinib Granule Formulation for NF1 (SPRINKLE)

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)

Phase 1/2 Study of the Oral RET Inhibitor LOXO-292 in Pediatric Patients with Advanced RET-Altered Solid or Primary Central Nervous System Tumors



The investigational drug selumetinib, a MEK inhibitor, has been developed for the therapy of neurofibromatosis Ty1 (NF1). The aim of this study is to determine the efficacy and safety of the investigational medicinal product selumetinib granule formulation.

Selumetinib inhibits the MAP kinases MEK1 and MEK, which are an important part of the Ras/Raf/MAPK signaling pathway. Alterations in the tumor genome can lead to overactive signal transmission by MAP kinases and the associated growth of the tumor cells. The investigational drug selumetinib interrupts this cascade and inhibits the uncontrolled growth of the tumor cells.

Main inclusion criteria

  • Age: ≥ 1 to < 7 years
  • diagnosed with with symptomatic inoperable PN
  • at least one measurable PN
  • Lansky > 70


Astra Zeneca

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