NCT05388370Active, not recruiting
PASS of Paediatric Patients Initiating Selumetinib
Post-Authorisation Safety Study of Paediatric Patients Initiating Selumetinib: A Multiple-Country Prospective Cohort Study.
Source: ClinicalTrials.gov · Last updated 24 Aug 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Active, not recruiting
- Study type
- Observational
- Enrollment
- 124 (actual)
- First submitted
- 12 Apr 2022
- Start date
- 23 May 2022
- Primary completion date
- 23 May 2028
- Lead sponsor
- AstraZeneca
- NCT number
- NCT05388370
What you commit to
- Study type: Observational
- Participants enrolled: 124
- From 23 May 2022 to the primary completion date, 23 May 2028
- 46 sites in 10 countries
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Have received treatment with a mitogen-activated protein kinase inhibitor before the index date
Other
- Are participating in an interventional study at index date
Who the study is looking for
- 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
Age in the registry: 3 years to 17 years · Sex: All · Does not accept healthy volunteers
Sites (46)
Sites in registry order. Pick your city on the disease page to sort them by distance.
This page is information, not medical advice. Discuss any trial with your doctor.