A Phase III Study of Selumetinib versus Carboplatin/Vincristine in Patients with Neurofibromatosis and Low-Grade Glioma


Full Title

A Phase 3 Randomized Study of Selumetinib (IND # 77782) versus Carboplatin/Vincristine in Newly Diagnosed or Previously Untreated Neurofibromatosis Type 1 (NF1) Associated Low-Grade Glioma (LGG) (ACNS1831) (CIRB)


People with neurofibromatosis type 1 and a brain cancer called low-grade glioma (NF1-associated LGG) have a higher chance of getting LGG in their optic pathway or brainstem. This can lead to vision loss in one or both eyes or a difficulty in moving certain parts of the body.

In this study, researchers want to see if a drug called selumetinib works as well as the standard treatment (carboplatin and vincristine or CV) for children, teens, and young adults with NF1-associated LGG. They also want to see if selumetinib is better than CV for improving vision in patients with LGG of the optic pathway.

Selumetinib works by blocking proteins needed for cell growth and killing cancer cells. Patients in this study will be randomly assigned to receive either standard CV therapy for 15 months or selumetinib for 27 months. CV is given intravenously (by vein) and selumetinib is taken orally (by mouth).


To be eligible for this study, patients must meet several criteria, including but not limited to the following:

  • Patients must have NF1-associated LGG.
  • Patients may not have received any prior treatment for NF1-associated LGG other than surgery.
  • Patients must be able to walk and do routine activities for more than half of their normal waking hours.
  • This study is for patients ages 2-21.

For more information about this study and to inquire about eligibility, please contact 1-833-MSK-KIDS.






Farouk Sait, Sameer, MD