US Selumetinib Registry
Reason given: Sponsor decision.
Key facts
- Where
- Albany, Fort Worth, Los Angeles, Miami, New Orleans, Orlando, Phoenix, Rochester, Syracuse, and The Bronx, United StatesSee the 10 sites
Where in United States
- Albany Medical CenterAlbany, United StatesNo longer taking part
- Cook Children's Medical CenterFort Worth, United StatesNo longer taking part
- Children's Hospital Los AngelesLos Angeles, United StatesNo longer taking part
- Nicklaus Children's HospitalMiami, United StatesNo longer taking part
- Ochsner Medical CenterNew Orleans, United StatesNo longer taking part
- Arnold Palmer Hospital for ChildrenOrlando, United StatesNo longer taking part
- Phoenix Children s HospitalPhoenix, United StatesNo longer taking part
- Mayo ClinicRochester, United StatesNo longer taking part
- Waters Center for Childrens Cancer and Blood DisordersSyracuse, United StatesNo longer taking part
- The Children's Hospital at Montefiore (CHAM)The Bronx, United StatesNo longer taking part
Trial details
- Who runs it
- Alexion Pharmaceuticals, Inc.
- Trial code
- NCT05683678
- Record updated
- 4 April 2025
- Original description
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.Read full original descriptionShow less
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.
Inclusion: 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: Participants currently participating in any clinical trials at time of enrollment or initiation of selumetinib.