New drug shows promise for shrinking painful NF1 tumors

NCT ID NCT03962543

Ongoing Disease control Sponsor: SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jul 10, 2026 · Updated 1 time

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 removed by surgery and cause serious problems. The drug works by blocking a protein that helps tumors grow. The main goal is to see if the tumors shrink by at least 20% on MRI scans. Participants take the drug for up to 24 cycles and may continue in a long-term follow-up phase.

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Conditions

The condition(s) this trial relates to.

neurofibroma neurofibromatosis neurofibromatosis type 1 plexiform neurofibroma

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

NEUROFIBROMATOSIS TYPE 1 (NF1) PLEXIFORM NEUROFIBROMA

Contacts and locations

Locations

Albany Medical Center

Albany, New York, 12208, United States
Arkansas Children's Hospital

Little Rock, Arkansas, 72202, United States
Arnold Palmer Hospital for Children

Orlando, Florida, 32806, United States
Children's Hospital Colorado

Aurora, Colorado, 80045, United States
Children's Hospital Los Angeles

Los Angeles, California, 90027, United States
Children's Hospital at Montefiore

The Bronx, New York, 10467, United States
Children's Hospital of Orange County

Orange, California, 92868, United States
Children's Hospital of Pittsburgh UPMC

Pittsburgh, Pennsylvania, 15219-2739, United States
Children's Hospital of The King's Daughters

Norfolk, Virginia, 23510, United States
Children's National Hospital

Washington D.C., District of Columbia, 20010, United States
Cook Children's Medical Center

Fort Worth, Texas, 76104, United States
Duke University Medical Center

Durham, North Carolina, 27710, United States
Florida Hospital Cancer Institute

Orlando, Florida, 32804, United States
Henry Ford Hospital

Detroit, Michigan, 48202, United States
Indiana University

Indianapolis, Indiana, 46202, United States
Johns Hopkins All Children's Hospital

St. Petersburg, Florida, 33701, United States
Lucile Packard Children's Hospital Stanford

Palo Alto, California, 94304, United States
MD Anderson Cancer Center

Houston, Texas, 77030, United States
Mayo Clinic

Rochester, Minnesota, 55905, United States
Mayo Clinic Arizona

Phoenix, Arizona, 98195, United States
Moffitt

Tampa, Florida, 33612, United States
Nationwide Children's Hospital

Columbus, Ohio, 43205, United States
Nemours A. I. duPont Hospital for Children

Wilmington, Delaware, 19803, United States
Nicklaus Children's Hospital

Miami, Florida, 33155, United States
Oregon Health and Science University

Portland, Oregon, 97239, United States
Saint Joseph's Regional Medical Center

Paterson, New Jersey, 07503, United States
The University of Utah Health Sciences Center

Salt Lake City, Utah, 84108, United States
UC Health

Cincinnati, Ohio, 45219, United States
UNC Medical Center

Chapel Hill, North Carolina, 27599, United States
University of Alabama at Birmingham/Children's of Alabama

Birmingham, Alabama, 35233, United States
University of California - Davis

Sacramento, California, 95817, United States
University of California - Irvine Health

Orange, California, 92868, United States
University of California, Los Angeles (UCLA)

Los Angeles, California, 90095, United States
University of Florida Clinical Research Center

Gainesville, Florida, 32610, United States
University of Illinois Hospital and Health Systems

Chicago, Illinois, 60612, United States
University of Iowa Health Care

Iowa City, Iowa, 52242, United States
University of Michigan Comprehensive Cancer Center

Ann Arbor, Michigan, 48109, United States
University of Minnesota/Masonic Cancer Center

Minneapolis, Minnesota, 55455, United States
University of Oklahoma - Health Sciences Center

Oklahoma City, Oklahoma, 73104, United States
University of Texas Southwestern

Dallas, Texas, 75244, United States
University of Virginia - Emily Couric Cancer Center

Charlottesville, Virginia, 22903, United States
Vanderbilt Children's Hospital

Nashville, Tennessee, 37232, United States
Washington University

St Louis, Missouri, 63110, United States

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