Condition: Neurofibromatosis 1 · Plexiform Neurofibroma · Sponsor: University of Alabama at Birmingham
Plexiform neurofibromas (PN) are known to cause significant morbidity in children with NF1. The recent FDA approval for selumetinib in children 2 years and older with inoperable symptomatic PN was based on the finding that selumetinib shrinks the majority of PN in children with NF1 and results in clinically meaningful benefit such as improvement in pain or range of motion. However, many morbidities, such as blindness or nerve damage, cannot be fully reversed with PN shrinkage. Therefore, there remains a critical need in this patient population to determine if young participants with PN in high-risk locations may benefit from early medical intervention prior to the development of clinical problems. This study will determine whether participants with asymptomatic PN in high-risk locations can potentially benefit from early treatment with selumetinib.
This description comes directly from the study's public registry record.
Karen Cole-Plourde, BA · 2055141317 · kplourde@uab.edu
Juliette Southworth, BS · 2055298967 · jsouthworth@uab.edu
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| Childrens of Alabama | Birmingham, Alabama, United States | Recruiting |
| Children's Hospital of Los Angeles | Los Angeles, California, United States | Recruiting |
| Stanford University | Palo Alto, California, United States | Recruiting |
| Children's National Hospital | Washington D.C., District of Columbia, United States | Recruiting |
| Lurie Children's Hospital of Chicago | Chicago, Illinois, United States | Recruiting |
| University of Chicago | Chicago, Illinois, United States | Recruiting |
| Riley Hospital for Children/Indiana University | Indianapolis, Indiana, United States | Recruiting |
| Johns Hopkins University | Baltimore, Maryland, United States | Recruiting |
| National Cancer Institute/ National Institutes of Health | Bethesda, Maryland, United States | Recruiting |
| Boston Children's Hospital | Boston, Massachusetts, United States | Recruiting |
| Mayo Clinic | Rochester, Minnesota, United States | Recruiting |
| Washington University - St. Louis | St Louis, Missouri, United States | Recruiting |
+ 3 more locations — full list on the registry record.
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Source record: clinicaltrials.gov/study/NCT06188741