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Brief Title: MEK Inhibitor Mirdametinib (PD-0325901) in Patients With Neurofibromatosis Type 1 Associated Plexiform Neurofibromas
Official Title: A Phase 2b Trial of the MEK 1/2 Inhibitor (MEKi) PD-0325901 in Adult and Pediatric Patients With Neurofibromatosis Type 1 (NF1)-Associated Inoperable Plexiform Neurofibromas (PNs) That Are Causing Significant Morbidity
Study ID: NCT03962543
Brief Summary: This study evaluates mirdametinib (PD-0325901) in the treatment of symptomatic inoperable neurofibromatosis type-1 (NF1)-associated plexiform neurofibromas (PNs). All participants will receive mirdametinib (PD-0325901). Eligible participants may continue in a long-term follow-up phase.
Detailed Description: Neurofibromas are benign peripheral nerve sheath tumors, which are classified as plexiform neurofibromas (PNs) if they extend longitudinally along a nerve and involve multiple fascicles. PNs are a major cause of morbidity and disfigurement in individuals with NF1, and as the tumor growth progresses, can cause a multitude of clinical deficits including pain and impaired physical function. PNs have the potential to undergo malignant transformation to Malignant Peripheral Nerve Sheet Tumors (MPNST). Mirdametinib (PD-0325901) is an orally delivered, highly selective small-molecule inhibitor of the dual specificity kinases, MEK1 and MEK2 (MAPK/ERK Kinase) which prevents the phosphorylation and subsequent activation of mitogen-activated protein kinase (MAPK). Previous studies of mirdametinib (PD-0325901) demonstrated PN shrinkage and sustained inhibition of pERK. Reduced tumor volume indicated that cell proliferation or cell death may be altered in PNs with administration of mirdametinib (PD-0325901).
Minimum Age: 2 Years
Eligible Ages: CHILD, ADULT, OLDER_ADULT
Sex: ALL
Healthy Volunteers: No
University of Alabama at Birmingham/Children's of Alabama, Birmingham, Alabama, United States
Mayo Clinic Hospital, Phoenix, Arizona, United States
Arkansas Children's Hospital, Little Rock, Arkansas, United States
Children's Hospital Los Angeles, Los Angeles, California, United States
UCLA Oncology Center, Los Angeles, California, United States
University of California - Irvine Health, Orange, California, United States
Children's Hospital of Orange County, Orange, California, United States
Lucile Packard Children's Hospital Stanford, Palo Alto, California, United States
University of California - Davis Comprehensive Cancer Center, Sacramento, California, United States
Children's Hospital Colorado, Aurora, Colorado, United States
Nemours A. I. duPont Hospital for Children, Wilmington, Delaware, United States
Children's National Medical Center, Washington, District of Columbia, United States
University of Florida Clinical Research Center, Gainesville, Florida, United States
Nicklaus Children's Hospital, Miami, Florida, United States
AdventHealth Pediatric Oncology Hematology at Orlando, Orlando, Florida, United States
Orlando Health, Inc., Orlando, Florida, United States
Johns Hopkins All Children's Hospital, Saint Petersburg, Florida, United States
Moffitt Cancer Center, Tampa, Florida, United States
Children's Healthcare of Atlanta - Center for Advanced Pediatrics, Atlanta, Georgia, United States
University of Illinois Hospital and Health Systems, Chicago, Illinois, United States
University of Chicago Medical Centers, Chicago, Illinois, United States
IU Health Brain Tumor Infusion Clinic, Indianapolis, Indiana, United States
University of Iowa Hospitals & Clinics, Iowa City, Iowa, United States
Kosair Charities Pediatric Clinical Research Unit, Louisville, Kentucky, United States
University of Michigan CS Mott Children's Hospital, Ann Arbor, Michigan, United States
Henry Ford Hospital, Detroit, Michigan, United States
University of Minnesota/Masonic Cancer Center, Minneapolis, Minnesota, United States
Mayo Clinic, Rochester, Minnesota, United States
Washington University School of Medicine-Siteman Cancer Center, Saint Louis, Missouri, United States
St. Joseph's Univeristy Medical Center, Paterson, New Jersey, United States
Albany Medical Center, Albany, New York, United States
Children's Hospital at Montefiore, Bronx, New York, United States
UNC Medical Center, Chapel Hill, North Carolina, United States
Duke University Medical Center, Durham, North Carolina, United States
University of Cincinnati Medical Center, Cincinnati, Ohio, United States
Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, United States
Cleveland Clinic Foundation, Cleveland, Ohio, United States
Nationwide Children's Hospital, Columbus, Ohio, United States
University of Oklahoma Health Sciences Center, Jimmy Everest Center for Cancer and Blood Disorders in Children, Oklahoma City, Oklahoma, United States
Oregon Health and Science University, Portland, Oregon, United States
Children's Hospital of Pittsburgh UPMC, Pittsburgh, Pennsylvania, United States
Henry-Joyce Cancer Clinic, Nashville, Tennessee, United States
Children's Medical Center, Dallas, Texas, United States
Cook Children's Medical Center, Fort Worth, Texas, United States
The University of Texas MD Anderson Cancer Center, Houston, Texas, United States
University of Utah, Center for Clinical and Translational Sciences, Salt Lake City, Utah, United States
UVA Health, Division of Neuro-Oncology, Charlottesville, Virginia, United States
Children's Hospital of The King's Daughters, Norfolk, Virginia, United States
Swedish Medical Center - Cherry Hill Campus, Seattle, Washington, United States
MACC Fund Research Center, Milwaukee, Wisconsin, United States
Name: Christopher L Moertel, MD
Affiliation: University of Minnesota
Role: PRINCIPAL_INVESTIGATOR