Clinical trial NCT05683678: US Selumetinib Registry
Official title: US Selumetinib Registry
Study type: OBSERVATIONAL
Overall status: TERMINATED
Conditions: Neurofibromatosis Type 1
Plexiform Neurofibromas
Keywords: neurofibromatosis type 1
plexiform neurofibromas
NF1
PNs
Lead sponsor: Alexion Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2023-01-13
Enrollment: 37
Sex: ALL
Minimum age (years): 2
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Approximately 200 participants who meet the eligibility criteria will be enrolled from 20-25 sites.

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.

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.

Primary outcomes:
measure: Number of Participants with Improvement in Clinically Significant Plexiform Neurofibromas (PNs) as Assessed by the Treating Physician
timeFrame: Up to Month 60
measure: Number of Participants with Improvement in Disease Status as Assessed by the Treating Physician
timeFrame: Up to Month 60
measure: Number of Participants with Improvement in Neurofibromatosis type 1 (NF1) Skeletal Manifestations
timeFrame: Up to Month 60
measure: Number of Participants with Treatment Emergent Medical Events of Interest (MEOI)
timeFrame: Up to Month 60
Secondary outcomes:
measure: Change From Baseline in the Numerical Rating Scale (NRS-11) Score at Month 60
timeFrame: Baseline, Month 60
measure: Change From Baseline in the Pain Interference Index (PII) Score at Month 60
timeFrame: Baseline, Month 60
measure: Change From Baseline in the Patient Global Impression of Change (GIC) Scale at Month 60
timeFrame: Baseline, Month 60
measure: Change From Baseline in the Pediatric Quality of Life Inventory (PedsQL) at Month 60
timeFrame: Baseline, Month 60
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Phoenix Children's Hospital
state: Arizona
city: Los Angeles
country: United States
facility: Children's Hospital Los Angeles
state: California
city: Miami
country: United States
facility: Nicklaus Children's Hospital
state: Florida
city: Orlando
country: United States
facility: Arnold Palmer Hospital For Children
state: Florida
city: New Orleans
country: United States
facility: Ochsner Medical Center
state: Louisiana
city: Rochester
country: United States
facility: Mayo Clinic
state: Minnesota
city: Albany
country: United States
facility: Albany Medical Center
state: New York
city: Bronx
country: United States
facility: The Children's Hospital at Montefiore (CHAM)
state: New York
city: Syracuse
country: United States
facility: Waters Center for Childrens Cancer and Blood Disorders
state: New York
city: Fort Worth
country: United States
facility: Cook Children's Medical Center
state: Texas
Study officials:
References: