US Selumetinib Registry
About the study
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.
Who can take part
You may be eligible to participate in the study if you meet the following criteria:
INCLUSION CRITERIA
Inclusion Criteria:
- Participants diagnosed with NF1 and PN
- Started selumetinib between the ages of 2 to 18 years inclusive, and initiated selumetinib treatment on or after 10 April 2020, including patients who:
- Discontinued selumetinib before enrollment; or
- Initiated treatment before enrollment and are currently on selumetinib; or
- Initiated selumetinib on the day of enrollment or intend to initiate selumetinib within 3 months after enrollment
- Able to give written informed consent. Participant or participant's parent/legal guardian must be willing and able to give written informed consent and the participant (if minor) must be willing to give written informed assent.
EXCLUSION CRITERIA
Exclusion Criteria:
* Participants currently participating in any clinical trials at time of enrollment or initiation of selumetinib.
Study Locations
Enter your ZIP code/Postal code/PIN code to locate study sites near you:
How to Apply
Contact the study center to learn if this study is a good match for you.
Study Details
Contition
Neurofibromatosis Type 1,Plexiform Neurofibromas
Age
2 - 22
Participants Needed
200
Est. Completion Date
Jun 21, 2028
Treatment Type
OBSERVATIONAL
Sponsor
Alexion Pharmaceuticals, Inc.
ClinicalTrials.gov NCT Identifier
NCT05683678
Study Number
ALX-NF1-501
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