Post-Authorisation Safety Study (PASS) of Pediatric Patients Initiating Selumetinib: A Multiple-Country Prospective Cohort Study
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:
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