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 participants with neurofibromatosis type 1 (NF1) and plexiform neurofibromas (PNs) who were aged 2 to 18 years at the time selumetinib was started 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 who were or will be aged 2 to 18 years at the time of selumetinib initiation on or after 10 April 2020 including participants who:
- Discontinued selumetinib before enrollment; or
- Initiated treatment before enrollment and are currently on selumetinib; or
- Intend to initiate selumetinib within approximately 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 - 18
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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