US Selumetinib Registry

Alexion Pharmaceuticals

Status: Terminated

Conditions: Neurofibromatosis Type 1; Plexiform Neurofibromas

Study type: Observational

Funder types: Industry

Identifiers

NCT05683678

ALX-NF1-501

Details and patient eligibility

About

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.

Full description

This is a US observational registry of pediatric NF1 PN participants treated with selumetinib.

Enrollment: 37 patients

Sex: All

Ages: 2 to 18 years old

Volunteers: No Healthy Volunteers

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.

Trial design

37 participants in 3 patient groups

Cohort 1

Description:

Participants who started selumetinib and discontinued selumetinib before enrollment (the "discontinued" cohort).

Cohort 2

Description:

Participants who started selumetinib before enrollment and are continuing to receive selumetinib at the time of enrollment (the "continuing" cohort).

Cohort 3

Description:

Participants who started selumetinib on the day of enrollment or intend to initiate selumetinib within 3 months after enrollment (the "initiating" cohort).