An observational study in Neurofibromatosis Type 1 and Plexiform Neurofibromas, sponsored by Alexion Pharmaceuticals, Inc.. Terminated at 10 sites in United States. Open to participants aged 2 Years to 18 Years. Per ClinicalTrials.gov, last updated 2025-04-04.
Sponsored by Alexion Pharmaceuticals, Inc. · Observational
The purpose of this study is to understand treatment patterns and assess long-term effectiveness and safety outcomes associated with selumetinib treatment as well as to explore clinical and non-clinical factors affecting those outcomes in patients with neurofibromatosis type 1 (NF1) and plexiform neurofibromas (PNs) who started selumetinib between the ages of 2 to 18 years inclusive, and initiated selumetinib treatment on or after 10 April 2020 in a real-world setting.
This is a US observational registry of pediatric NF1 PN participants treated with selumetinib.
Approximately 200 participants who meet the eligibility criteria will be enrolled from 20-25 sites.
Exclusion Criteria:
Participants who started selumetinib and discontinued selumetinib before enrollment (the "discontinued" cohort).
Participants who started selumetinib before enrollment and are continuing to receive selumetinib at the time of enrollment (the "continuing" cohort).
Participants who started selumetinib on the day of enrollment or intend to initiate selumetinib within 3 months after enrollment (the "initiating" cohort).
Number of Participants with Improvement in Clinically Significant Plexiform Neurofibromas (PNs) as Assessed by the Treating Physician
Time frame: Up to Month 60
Number of Participants with Improvement in Disease Status as Assessed by the Treating Physician
Time frame: Up to Month 60
Number of Participants with Improvement in Neurofibromatosis type 1 (NF1) Skeletal Manifestations
Skeletal manifestations may include changes in the angle of spine curvature.
Time frame: Up to Month 60
Number of Participants with Treatment Emergent Medical Events of Interest (MEOI)
Time frame: Up to Month 60
Change From Baseline in the Numerical Rating Scale (NRS-11) Score at Month 60
Time frame: Baseline, Month 60
Change From Baseline in the Pain Interference Index (PII) Score at Month 60
Time frame: Baseline, Month 60
Change From Baseline in the Patient Global Impression of Change (GIC) Scale at Month 60
Time frame: Baseline, Month 60
Change From Baseline in the Pediatric Quality of Life Inventory (PedsQL) at Month 60
Time frame: Baseline, Month 60
Plan to share: Yes — Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.
Supporting information: Study protocol, Sap, Csr
No publications or documents are linked to this record.
This study is terminated, as verified in Mar 2025. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Alexion Pharmaceuticals, Inc.