US Selumetinib Registry

Sponsor

Alexion Pharmaceuticals (Industry)

Overall Status

Not yet recruiting

CT.gov ID

NCT05683678

Collaborator

(none)

200

Enrollment

64.5

Anticipated Duration (Months)

Study Details

Study Description

Brief 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 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.

Condition or Disease	Intervention/Treatment	Phase
Neurofibromatosis Type 1 Plexiform Neurofibromas

Detailed Description

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

Study Design

Study Type:

Observational

Anticipated Enrollment :

200 participants

Observational Model:

Case-Only

Time Perspective:

Prospective

Official Title:

US Selumetinib Registry

Anticipated Study Start Date :

Feb 5, 2023

Anticipated Primary Completion Date :

Feb 6, 2028

Anticipated Study Completion Date :

Jun 21, 2028

Arms and Interventions

Arm	Intervention/Treatment
Cohort 1 Participants who started selumetinib and discontinued selumetinib before enrollment (the "discontinued" cohort).
Cohort 2 Participants who started selumetinib before enrollment and are continuing to receive selumetinib at the time of enrollment (the "continuing" cohort).
Cohort 3 Participants who intend to initiate selumetinib within approximately 3 months after enrollment (the "initiating" cohort).

Outcome Measures

Primary Outcome Measures

Number of Participants with Improvement in Clinically Significant Plexiform Neurofibromas (PNs) as Assessed by the Treating Physician [Up to Month 60]
Number of Participants with Improvement in Disease Status as Assessed by the Treating Physician [Up to Month 60]
Number of Participants with Improvement in Neurofibromatosis type 1 (NF1) Skeletal Manifestations [Up to Month 60]
Skeletal manifestations may include changes in the angle of spine curvature.
Number of Participants with Treatment Emergent Medical Events of Interest (MEOI) [Up to Month 60]

Secondary Outcome Measures

Change From Baseline in the Numerical Rating Scale (NRS-11) Score at Month 60 [Baseline, Month 60]
Change From Baseline in the Pain Interference Index (PII) Score at Month 60 [Baseline, Month 60]
Change From Baseline in the Patient Global Impression of Change (GIC) Scale at Month 60 [Baseline, Month 60]
Change From Baseline in the Pediatric Quality of Life Inventory (PedsQL) at Month 60 [Baseline, Month 60]

Eligibility Criteria

Criteria

Ages Eligible for Study:

2 Years to 25 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Patients 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 patients 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. Patient or patient's parent/legal guardian must be willing and able to give written informed consent and the patient (if minor) must be willing to give written informed assent.