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. (part of AstraZeneca) · 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.
176 studies on the registry are indexed under Neurofibromatosis 1; 43 are open to participants now.
This study's enrollment of 37 is below the median of 188 across 58 observational studies indexed under Neurofibromatosis 1.
Browse Neurofibromatosis 1 studies →Alexion Pharmaceuticals, Inc. is the lead sponsor of 255 studies on the registry; 23 are open to participants now.
Of its 101 completed or terminated interventional studies of FDA-regulated products, 75 (74%) have results posted.
First submitted after Alexion Pharmaceuticals, Inc. became part of AstraZeneca.
Counted across the registry records on this site, refreshed daily.
Approximately 200 participants who meet the eligibility criteria will be enrolled from 20-25 sites.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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, Statistical analysis plan, Clinical study report
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.
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