New drug shows promise for shrinking painful NF1 tumors
NCT ID NCT03962543
First seen Mar 27, 2026 · Last updated May 16, 2026 · Updated 8 times
Summary
This study tests a drug called mirdametinib in 114 adults and children with a genetic condition called NF1 that causes nerve tumors (plexiform neurofibromas) that cannot be surgically removed and cause serious problems. The goal is to see if the drug can shrink these tumors by at least 20% and improve symptoms. All participants receive the drug, and those who benefit may continue long-term treatment.
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Locations
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AdventHealth Pediatric Oncology Hematology at Orlando
Orlando, Florida, 32804, United States
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Albany Medical Center
Albany, New York, 12208, United States
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Arkansas Children's Hospital
Little Rock, Arkansas, 72202, United States
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Children's Healthcare of Atlanta - Center for Advanced Pediatrics
Atlanta, Georgia, 30329, United States
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Children's Hospital Colorado
Aurora, Colorado, 80045, United States
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Children's Hospital Los Angeles
Los Angeles, California, 90027, United States
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Children's Hospital at Montefiore
The Bronx, New York, 10467, United States
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Children's Hospital of Orange County
Orange, California, 92868, United States
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Children's Hospital of Pittsburgh UPMC
Pittsburgh, Pennsylvania, 15224, United States
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Children's Hospital of The King's Daughters
Norfolk, Virginia, 23510, United States
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Children's Medical Center
Dallas, Texas, 75235, United States
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Children's National Medical Center
Washington D.C., District of Columbia, 20010, United States
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Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
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Cleveland Clinic Foundation
Cleveland, Ohio, 44195, United States
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Cook Children's Medical Center
Fort Worth, Texas, 76104, United States
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Duke University Medical Center
Durham, North Carolina, 27710, United States
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Henry Ford Hospital
Detroit, Michigan, 48202, United States
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Henry-Joyce Cancer Clinic
Nashville, Tennessee, 37232, United States
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IU Health Brain Tumor Infusion Clinic
Indianapolis, Indiana, 46202, United States
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Johns Hopkins All Children's Hospital
St. Petersburg, Florida, 33701, United States
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Kosair Charities Pediatric Clinical Research Unit
Louisville, Kentucky, 40202, United States
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Lucile Packard Children's Hospital Stanford
Palo Alto, California, 94304, United States
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MACC Fund Research Center
Milwaukee, Wisconsin, 53226, United States
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Mayo Clinic
Rochester, Minnesota, 55905, United States
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Mayo Clinic Hospital
Phoenix, Arizona, 85054, United States
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Moffitt Cancer Center
Tampa, Florida, 33612, United States
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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
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Nemours A. I. duPont Hospital for Children
Wilmington, Delaware, 19803, United States
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Nicklaus Children's Hospital
Miami, Florida, 33155, United States
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Oregon Health and Science University
Portland, Oregon, 97239, United States
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Orlando Health, Inc.
Orlando, Florida, 32806, United States
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St. Joseph's Univeristy Medical Center
Paterson, New Jersey, 07503, United States
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Swedish Medical Center - Cherry Hill Campus
Seattle, Washington, 98122, United States
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The University of Texas MD Anderson Cancer Center
Houston, Texas, 77030, United States
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UCLA Oncology Center
Los Angeles, California, 90095, United States
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UNC Medical Center
Chapel Hill, North Carolina, 27514, United States
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UVA Health, Division of Neuro-Oncology
Charlottesville, Virginia, 22903, United States
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University of Alabama at Birmingham/Children's of Alabama
Birmingham, Alabama, 35233, United States
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University of California - Davis Comprehensive Cancer Center
Sacramento, California, 95817, United States
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University of California - Irvine Health
Orange, California, 92868-3201, United States
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University of Chicago Medical Centers
Chicago, Illinois, 60637, United States
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University of Cincinnati Medical Center
Cincinnati, Ohio, 45219, United States
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University of Florida Clinical Research Center
Gainesville, Florida, 32610, United States
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University of Illinois Hospital and Health Systems
Chicago, Illinois, 60612, United States
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University of Iowa Hospitals & Clinics
Iowa City, Iowa, 52242, United States
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University of Michigan CS Mott Children's Hospital
Ann Arbor, Michigan, 48109, United States
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University of Minnesota/Masonic Cancer Center
Minneapolis, Minnesota, 55455, United States
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University of Oklahoma Health Sciences Center, Jimmy Everest Center for Cancer and Blood Disorders in Children
Oklahoma City, Oklahoma, 73104, United States
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University of Utah, Center for Clinical and Translational Sciences
Salt Lake City, Utah, 84108, United States
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Washington University School of Medicine-Siteman Cancer Center
St Louis, Missouri, 63110, United States
Conditions
Explore the condition pages connected to this study.