Selumetinib Post-Authorization Safety Study (PASS)

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:

  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.

Main inclusion criteria

  • Age: ≥ 3 to < 18 years
  • Diagnosed with NF1 with symptomatic, inoperable PN
  • Have been newly prescribed at least one dose of selumetinib


Astra Zeneca

Trial sites

Keine Einträge vorhanden

Diese Webseite verwendet Cookies. Durch die Nutzung der Webseite stimmen Sie der Verwendung von Cookies zu. Datenschutzinformationen