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Clinical Trials in the USA / NCT03962543
Active, not recruiting Phase 2

MEK Inhibitor Mirdametinib (PD-0325901) in Patients With Neurofibromatosis Type 1 Associated Plexiform Neurofibromas

NCT03962543 · tracked via the Priya Life Science USA tracker
Sponsor
SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany
Phase
Phase 2
Started
2019-09-29
Last updated
2026-07-10

Condition(s) studied

Plexiform NeurofibromaNeurofibromatosis Type 1 (NF1)

Investigational drug(s) / intervention(s)

Mirdametinib (PD-0325901) oral capsule or dispersible tablet

Mirdametinib (PD-0325901) oral capsule or dispersible tablet: Mirdametinib (PD-0325901) capsule or dispersible tablet

Study 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.

Eligibility

Sex
ALL
Min age
2 Years
Max age
Healthy volunteers
No
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.

Primary outcome measure(s)

Trial sites (43)

FacilityCityRegionStatus
University of Alabama at Birmingham/Children's of Alabama Birmingham Alabama
Mayo Clinic Arizona Phoenix Arizona
Arkansas Children's Hospital Little Rock Arkansas
Children's Hospital Los Angeles Los Angeles California
University of California, Los Angeles (UCLA) Los Angeles California
Children's Hospital of Orange County Orange California
University of California - Irvine Health Orange California
Lucile Packard Children's Hospital Stanford Palo Alto California
University of California - Davis Sacramento California
Children's Hospital Colorado Aurora Colorado
Nemours A. I. duPont Hospital for Children Wilmington Delaware
Children's National Hospital Washington D.C. District of Columbia
University of Florida Clinical Research Center Gainesville Florida
Nicklaus Children's Hospital Miami Florida
Florida Hospital Cancer Institute Orlando Florida
Arnold Palmer Hospital for Children Orlando Florida
Johns Hopkins All Children's Hospital St. Petersburg Florida
Moffitt Tampa Florida
University of Illinois Hospital and Health Systems Chicago Illinois
Indiana University Indianapolis Indiana
University of Iowa Health Care Iowa City Iowa
University of Michigan Comprehensive Cancer Center Ann Arbor Michigan
Henry Ford Hospital Detroit Michigan
University of Minnesota/Masonic Cancer Center Minneapolis Minnesota
Mayo Clinic Rochester Minnesota
Washington University St Louis Missouri
Saint Joseph's Regional Medical Center Paterson New Jersey
Albany Medical Center Albany New York
Children's Hospital at Montefiore The Bronx New York
UNC Medical Center Chapel Hill North Carolina
Duke University Medical Center Durham North Carolina
UC Health Cincinnati Ohio
Nationwide Children's Hospital Columbus Ohio
University of Oklahoma - Health Sciences Center Oklahoma City Oklahoma
Oregon Health and Science University Portland Oregon
Children's Hospital of Pittsburgh UPMC Pittsburgh Pennsylvania
Vanderbilt Children's Hospital Nashville Tennessee
University of Texas Southwestern Dallas Texas
Cook Children's Medical Center Fort Worth Texas
MD Anderson Cancer Center Houston Texas

+ 3 more sites — see the full list on the official registry below.

More SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany trials in the USA

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT03962543 on ClinicalTrials.gov ↗ ← All trials in the USA