US Selumetinib Registry

Study Overview
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Brief Summary
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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.

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

Official Title
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US Selumetinib Registry

Conditions
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Neurofibromatosis Type 1, Plexiform Neurofibromas

Participation Criteria
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Eligibility Criteria
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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: Participants currently participating in any clinical trials at time of enrollment or initiation of selumetinib.

Ages Eligible for Study
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Minimum Age: 2 Years
Maximum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

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

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 | Skeletal manifestations may include changes in the angle of spine curvature. | Up to Month 60 | 
| Number of Participants with Treatment Emergent Medical Events of Interest (MEOI) |  | Up to Month 60 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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NF1, PNs, neurofibromatosis type 1, plexiform neurofibromas