NCT05388370

PASS of Paediatric Patients Initiating Selumetinib

Active, enrolled Last updated 12 March 2026

What this trial tests

trial in Neurofibromatosis Type 1 in 124 participants. Participants enrolled and being followed up; not accepting new ones.

Timeline

23 May 2022

Primary endpoint
23 May 2028

23 May 2028

Quick facts

Lead sponsor	AstraZeneca
Status	Active, enrolled
Study type	OBSERVATIONAL
Enrollment	124
Start date	23 May 2022
Primary completion	23 May 2028
Estimated completion	23 May 2028
Sites	46 locations across France, Italy, Netherlands, Austria, United Kingdom, Israel, Germany, Switzerland

Conditions studied

• Neurofibromatosis Type 1 — all drugs for Neurofibromatosis Type 1 →

Sponsor

AstraZeneca — full company profile →

Who can join

Adults 3 to 17, any sex, with Neurofibromatosis Type 1. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

Neurofibromatosis type 1 (NF1) is a rare, autosomal dominant genetic disorder that is caused by germline mutations in the NF1 tumour suppressor gene, which encodes the tumour suppressor protein neurofibromin 1. Plexiform neurofibromas (PN) are histologically benign nerve sheath tumours, which typically grow along large nerves and plexi.

On 5 March 2020, a centralised Marketing Authorisation Application was submitted to the European Medicines Agency (EMA), Marketing Authorisation in EU was granted on 17 Jun 2021.

As part of the approval process, a Risk Management Plan (RMP) was developed and submitted to the EMA to summarise the safety concerns emerging from the clinical development program. The RMP included additional pharmacovigilance plans for a noninterventional Post-authorisation Safety Study (PASS) to further characterise the safety of selumetinib in paediatric patients with NF1-related PN in routine clinical practice.

The planned non-interventional PASS will address gaps in knowledge identified by the RMP, 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 (age d 8 to \< 18 years old) with a diagnosis of NF1 with symptomatic, inoperable PN.

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 European countries and Israel.

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.

Publications & conference data

No peer-reviewed publications indexed yet for this trial.