A Phase III Study of Selumetinib in Adults with Inoperable Plexiform Neurofibromas Causing Symptoms


Full Title

A Phase III, Multicentre, International Study with a Parallel, Randomised, Double-blind, Placebo-controlled, 2 Arm Design to Assess the Efficacy and Safety of Selumetinib in Adult Participants with NF1 who have Symptomatic, Inoperable Plexiform Neurofibromas (KOMET) (WIRB)


The purpose of this study is to assess the effectiveness of the drug selumetinib to treat adults with neurofibromatosis 1 (NF1) who have inoperable plexiform neurofibromas (tumors that grow along a nerve) that are causing symptoms such as pain. Selumetinib is used in children with NF1 and painful plexiform neurofibromas, and doctors want to know if this medication is also useful in adult patients.

Selumetinib works by blocking a protein that lets tumor cells grow without stopping. It is taken orally (by mouth). Participants in this study will first be randomly assigned to receive selumetinib or a placebo (inactive drug). Patients in the placebo group will then have an opportunity to take the study drug.

Who Can Join

To be eligible for this study, patients must meet several requirements, including:

  • Participants must have NF1 and inoperable plexiform neurofibroma that is causing pain. 
  • Patients must be physically well enough that they are able to be mobile, take care of themselves, and engage in all but physically strenuous activities. For example, they must be well enough that they could carry out office work or light housework.
  • This study is for people age 18 and older.



For more information and to ask about eligibility for this study, please contact the office of Dr. Anna Piotrowski at 212-639-6767.