US Selumetinib Registry

Study Identifier:
ALX-NF1-501
ClinicalTrials.gov Identifier:
NCT05683678
EudraCT Identifier:
N/A
EU CT ID:
N/A
Sponsor:
Alexion Pharmaceuticals, Inc.
Study Contact Information:
N/A
Other

Study Details

Medical Condition
  • Unmapped
Study Drug
    Date
    Jan 2019 - Nov 2024
    Phase 1
    Phase 2
    Phase 3
    Phase 4
    N/A
    Patient Requirements
    Sex: Female & Male
    Age: 2 - 18 Years
    Requirements Information
    Inclusion and Exclusion 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
    • Participants currently participating in any clinical trials at time of enrollment or initiation of selumetinib.
    Sex
    Female & Male
    Age
    2 - 18 Years

    Study Details

    Medical Condition
    • Unmapped
    Study Drug
      Date
      Jan 2019 - Nov 2024
      Phase 1
      Phase 2
      Phase 3
      Phase 4
      N/A
      Patient Requirements
      Sex: Female & Male
      Age: 2 - 18 Years years
      Requirements Information
      Inclusion and Exclusion 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
      • Participants currently participating in any clinical trials at time of enrollment or initiation of selumetinib.

      Protocol Summary

      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.

      Trial Locations

      Location
      Status
      Location
      Phoenix Children's Hospital
      Phoenix, Arizona, United States, 85016
      Status
      N/A
      Location
      Children's Hospital Los Angeles
      Los Angeles, California, United States, 90027-6062
      Status
      N/A
      Location
      Nicklaus Children's Hospital
      Miami, Florida, United States, 33155
      Status
      N/A
      Location
      Arnold Palmer Hospital For Children
      Orlando, Florida, United States, 32806
      Status
      N/A
      Location
      Ochsner Medical Center
      New Orleans, Louisiana, United States, 70121
      Status
      N/A
      Location
      Mayo Clinic
      Rochester, Minnesota, United States, 55905
      Status
      N/A
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