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Trial Title:
US Selumetinib Registry
NCT ID:
NCT05683678
Condition:
Neurofibromatosis Type 1
Plexiform Neurofibromas
Conditions: Official terms:
Neurofibromatoses
Neurofibromatosis 1
Neurofibroma
Neurofibroma, Plexiform
Conditions: Keywords:
neurofibromatosis type 1
plexiform neurofibromas
NF1
PNs
Study type:
Observational
Overall status:
Recruiting
Study design:
Time perspective:
Prospective
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.
Criteria for eligibility:
Study pop:
Approximately 200 participants who meet the eligibility criteria will be enrolled from
20-25 sites.
Sampling method:
Non-Probability Sample
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.
Gender:
All
Minimum age:
2 Years
Maximum age:
22 Years
Healthy volunteers:
No
Locations:
Facility:
Name:
Phoenix Children's Hospital
Address:
City:
Phoenix
Zip:
85016
Country:
United States
Status:
Recruiting
Facility:
Name:
Children's Hospital Los Angeles
Address:
City:
Los Angeles
Zip:
90027-6062
Country:
United States
Status:
Recruiting
Facility:
Name:
Nicklaus Children's Hospital
Address:
City:
Miami
Zip:
33155
Country:
United States
Status:
Recruiting
Facility:
Name:
Arnold Palmer Hospital For Children
Address:
City:
Orlando
Zip:
32806
Country:
United States
Status:
Recruiting
Facility:
Name:
Ochsner Medical Center
Address:
City:
New Orleans
Zip:
70121
Country:
United States
Status:
Recruiting
Facility:
Name:
Mayo Clinic
Address:
City:
Rochester
Zip:
55905
Country:
United States
Status:
Recruiting
Facility:
Name:
Albany Medical Center
Address:
City:
Albany
Zip:
12208
Country:
United States
Status:
Recruiting
Facility:
Name:
The Children's Hospital at Montefiore (CHAM)
Address:
City:
Bronx
Zip:
10467
Country:
United States
Status:
Recruiting
Facility:
Name:
Waters Center for Childrens Cancer and Blood Disorders
Address:
City:
Syracuse
Zip:
13210
Country:
United States
Status:
Recruiting
Facility:
Name:
Cook Children's Medical Center
Address:
City:
Fort Worth
Zip:
76104
Country:
United States
Status:
Recruiting
Start date:
July 18, 2023
Completion date:
June 21, 2028
Lead sponsor:
Agency:
Alexion Pharmaceuticals, Inc.
Agency class:
Industry
Source:
Alexion Pharmaceuticals, Inc.
Record processing date:
ClinicalTrials.gov processed this data on November 12, 2024
Source: ClinicalTrials.gov page:
https://clinicaltrials.gov/ct2/show/NCT05683678
