Selumetinib Post-Authorization Safety Study (PASS)

The investigational drug selumetinib, a MEK inhibitor, has been developed for the treatment of plexiform neurofibromas (PN) in neurofibromatosis Type 1 (NF1). 

This study is a specific obligation in the context of a conditional marketing authorisation for selumetinib (ie, Category 2 PASS). Study results will contribute to updating the safety profile of selumetinib in a relatively large population of patients with different personal characteristics across multiple health care systems and patterns of real-world clinical practice in the European Union (EU) and in the UK.

The planned non-interventional PASS will address gaps in knowledge identified by the Risk Management Plan, including the important identified risk and some of the potential risks and missing information on long-term developmental toxicity in children, by characterising the safety profile associated with selumetinib use among paediatric patients (ages > 8 to < 18 years old) with a diagnosis of NF1 with symptomatic, inoperable PN.

The study will enrol 2 cohorts:

1. The Base Cohort includes all enrolled patients aged 3 to < 18 years.
2. The Nested Prospective Cohort will include the subset of Base Cohort patients aged 8 to < 18 years who have not reached Tanner Stage V on the index date.