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New drug shows promise for shrinking painful NF1 tumors

NCT ID NCT03962543

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

114 people

The number who actually took part.

Started

Sep 2019

Expected to finish

Dec 2028

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

2 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Key Inclusion Criteria: * Participant has documented NF1 mutation or a diagnosis of neurofibromatosis type 1 (NF1) using National Institute of Health (NIH) Consensus Conference criteria inclusive of the presence of a plexiform neurofibroma (PN). * Participant has a PN that is causing significant morbidity. * Participant has a PN that cannot be completely surgically removed. * Participant has a target tumor that is amenable to volumetric MRI analysis. * Participant is willing to undergo a tumor biopsy pre and post treatment if ≥ 18 years of age. * Participant has adequate organ and bone marrow function. Key Exclusion Criteria: * Participant has abnormal liver function or history of liver disease. * Participant has lymphoma, leukemia or any malignancy within the past 5 years (except for resected basal/squamous skin carcinomas without metastases within 3 years). * Participant has breast cancer within 10 years. * Participant has active optic glioma or other low-grade glioma requiring treatment. * Participant has abnormal QT interval corrected or other heart disease within 6 months. * Participant has a history of retinal pathology, risk factors for retinal vein occlusion or has a history of glaucoma. * Participant has known malabsorption syndrome or gastrointestinal conditions that would impair absorption of mirdametinib (PD-0325901). * Participant has received NF1 PN-targeted therapy within 45 days. * Participant previously received or is currently receiving therapy with mirdametinib (PD-0325901) or any other MEK1/2 inhibitor. * Participant has received radiation therapy within 6 months or has received radiation to the orbit at any time. * Participant is unable to undergo or tolerate MRI. * Participant has active bacterial, fungal or viral infection. * Participant has experienced other severe acute or chronic medical or psychiatric conditions within 1 year.

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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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