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)
Confirmed Objective Response Rate at the End of the Treatment Phase. — Up to 24 months Response will be determined by a blinded centralized review of volumetric MRI. The confirmed objective response rate (complete or partial response) by the end of Treatment Phase (i.e., Cycle 24) is defined as the proportion of participants who have a confirmed ≥ 20% reduction in target tumor volume as compared to baseline as assessed by a BICR, and the response needs to be confirmed by BICR in a consecutive tumor assessment within 2 - 6 months. Partial response is defined as a ≥ 20% reduction in target tumor volume from baseline. Complete response is defined as the complete resolution of the target tumor.
Trial sites (43)
Facility
City
Region
Status
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
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.
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