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Observing the safety of selumetinib in children with neurofibromatosis type 1

Post-Authorisation Safety Study of Paediatric Patients Initiating Selumetinib: A Multiple-Country Prospective Cohort Study.

Observational AstraZeneca · NCT05388370

This study is testing how safe the medication selumetinib is for children aged 3 to 17 with neurofibromatosis type 1 who have certain tumors that can't be removed by surgery.

Quick facts

Study typeObservational
Enrollment125 (estimated)
Ages3 Years to 17 Years
SexAll
SponsorAstraZeneca Industry-sponsored
Drugs / interventionsselumetinib
Locations43 sites (Wien and 42 other locations)
Trial IDNCT05388370 on ClinicalTrials.gov

What this trial studies

This study aims to monitor the safety of selumetinib in pediatric patients diagnosed with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas. It is a non-interventional post-authorisation safety study that will collect data on the long-term effects and safety concerns associated with selumetinib treatment in routine clinical practice. The study will include children aged 3 to 17 years who have started treatment with selumetinib within the last six months. The findings will help address gaps in knowledge regarding the safety profile of this medication in a pediatric population.

Who should consider this trial

Good fit: Ideal candidates for this study are children aged 3 to 17 years diagnosed with NF1 and symptomatic, inoperable plexiform neurofibromas who have recently started treatment with selumetinib.

Not a fit: Patients who have previously received treatment with a mitogen-activated protein kinase inhibitor or are currently participating in an interventional study may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide valuable insights into the safety of selumetinib, potentially improving treatment protocols for children with NF1.

How similar studies have performed: Other studies have shown success in monitoring the safety of treatments for NF1, but this specific approach to selumetinib in a pediatric population is relatively novel.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

* Have been diagnosed with NF1 with symptomatic, inoperable PN
* Have initial treatment with selumetinib up to 6 months (i.e.182 days)prior to enrolment into the study (i.e. signature of the ICF)
* Are aged 3 years and above, and are \< 18 years of age on the index date
* Parent or legal guardian, as required by country-specific regulation, have provided informed consent (unless a country-specific waiver is obtained) Additional Criteria for Nested Prospective Cohort
* Are at least 8 years old and
* Are prior to attainment of Tanner Stage V on the index date

Exclusion Criteria:

* Have received treatment with a mitogen-activated protein kinase inhibitor before the index date
* Are participating in an interventional study at index date

Where this trial is running

Wien and 42 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Neurofibromatosis Type 1
Last reviewed 2026-06-10 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.