PASS of Paediatric Patients Initiating Selumetinib

Launched by ASTRAZENECA · May 23, 2022

Keywords

ClinConnect Summary

The PASS of Paediatric Patients Initiating Selumetinib trial is studying the safety of a medication called selumetinib in children and teenagers with Neurofibromatosis Type 1 (NF1), particularly those who have inoperable tumors known as plexiform neurofibromas. This trial is important because it aims to gather more information about how selumetinib affects young patients over time, especially regarding any long-term side effects. The study will include participants aged 3 to under 18 years old who have started treatment with selumetinib within the last six months.

To join the trial, participants must have a diagnosis of NF1 with symptomatic, inoperable plexiform neurofibromas, and their parents or guardians need to provide consent. The trial is currently recruiting and will involve two groups: one for all eligible patients aged 3 to under 18, and a second group for those aged 8 to under 18 who have not yet reached a certain stage of physical development. Participants can expect regular check-ups and monitoring as part of the study, which will help doctors better understand the safety of selumetinib in real-life settings across different countries.

Gender

ALL

Eligibility 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