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.
Brief Title
US Selumetinib Registry
Detailed Description
This is a US observational registry of pediatric NF1 PN participants treated with selumetinib.
Categories
Completion Date
Completion Date Type
Actual
Conditions
Neurofibromatosis Type 1
Plexiform Neurofibromas
Eligibility 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.
* 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.
Inclusion 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.
* 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.
Gender
All
Gender Based
false
Keywords
neurofibromatosis type 1
plexiform neurofibromas
NF1
PNs
Healthy Volunteers
No
Last Update Post Date
Last Update Post Date Type
Actual
Last Update Submit Date
Maximum Age
18 Years
Minimum Age
2 Years
NCT Id
NCT05683678
Org Class
Industry
Org Full Name
Alexion Pharmaceuticals, Inc.
Org Study Id
ALX-NF1-501
Overall Status
Terminated
Primary Completion Date
Primary Completion Date Type
Actual
Official Title
US Selumetinib Registry
Primary Outcomes
Outcome Measure
Number of Participants with Improvement in Clinically Significant Plexiform Neurofibromas (PNs) as Assessed by the Treating Physician
Outcome Time Frame
Up to Month 60
Outcome Measure
Number of Participants with Improvement in Disease Status as Assessed by the Treating Physician
Outcome Time Frame
Up to Month 60
Outcome Description
Skeletal manifestations may include changes in the angle of spine curvature.
Outcome Measure
Number of Participants with Improvement in Neurofibromatosis type 1 (NF1) Skeletal Manifestations
Outcome Time Frame
Up to Month 60
Outcome Measure
Number of Participants with Treatment Emergent Medical Events of Interest (MEOI)
Outcome Time Frame
Up to Month 60
Secondary Outcomes
Outcome Time Frame
Baseline, Month 60
Outcome Measure
Change From Baseline in the Numerical Rating Scale (NRS-11) Score at Month 60
Outcome Time Frame
Baseline, Month 60
Outcome Measure
Change From Baseline in the Pain Interference Index (PII) Score at Month 60
Outcome Time Frame
Baseline, Month 60
Outcome Measure
Change From Baseline in the Patient Global Impression of Change (GIC) Scale at Month 60
Outcome Time Frame
Baseline, Month 60
Outcome Measure
Change From Baseline in the Pediatric Quality of Life Inventory (PedsQL) at Month 60
Start Date
Start Date Type
Actual
Status Verified Date
First Post Date
First Post Date Type
Actual
First Submit Date
First Submit QC Date
Study Population
Approximately 200 participants who meet the eligibility criteria will be enrolled from 20-25 sites.
Std Ages
Child
Adult
Maximum Age Number (converted to Years and rounded down)
18
Minimum Age Number (converted to Years and rounded down)
2
Investigators
Investigator Type
Principal Investigator
Investigator Name
Nagma Dalvi
Investigator Email
ndalvi@montefiore.org