Neurofibromatosis Type 1, Plexiform Neurofibromas
Conditions
Keywords
neurofibromatosis type 1, plexiform neurofibromas, NF1, PNs
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 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.
Detailed description
This is a US observational registry of pediatric NF1 PN participants treated with selumetinib.
Interventions
None listed
Sponsors
Alexion Pharmaceuticals, Inc.
Study design
Observational model
CASE_ONLY
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
2 Years to 18 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants with Treatment Emergent Medical Events of Interest (MEOI) | Up to Month 60 | — |
| 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. |
Secondary
| Measure | Time frame |
|---|---|
| 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 |
| 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 |
Countries
United States
Outcome results
None listed