MEK Inhibitor Mirdametinib (PD-0325901) in Patients With Neurofibromatosis Type 1 Associated Plexiform Neurofibromas
NCT ID: NCT03962543
Last Updated: 2025-08-07
Study Results
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View full resultsBasic Information
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ACTIVE_NOT_RECRUITING
PHASE2
114 participants
INTERVENTIONAL
2019-09-29
2028-12-22
Brief Summary
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Detailed Description
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Mirdametinib (PD-0325901) is an orally delivered, highly selective small-molecule inhibitor of the dual specificity kinases, MEK1 and MEK2 (MAPK/ERK Kinase) which prevents the phosphorylation and subsequent activation of mitogen-activated protein kinase (MAPK).
Previous studies of mirdametinib (PD-0325901) demonstrated PN shrinkage and sustained inhibition of pERK. Reduced tumor volume indicated that cell proliferation or cell death may be altered in PNs with administration of mirdametinib (PD-0325901).
Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Mirdametinib (PD-0325901)
Mirdametinib (PD-0325901) capsule or dispersible tablet 2 mg/m\^2 (maximum dose of 4 mg) by mouth twice daily
Mirdametinib (PD-0325901) oral capsule or dispersible tablet
Mirdametinib (PD-0325901) capsule or dispersible tablet
Interventions
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Mirdametinib (PD-0325901) oral capsule or dispersible tablet
Mirdametinib (PD-0325901) capsule or dispersible tablet
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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.
Exclusion Criteria
* 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.
2 Years
ALL
No
Sponsors
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SpringWorks Therapeutics, Inc.
INDUSTRY
Responsible Party
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Principal Investigators
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Christopher L Moertel, MD
Role: PRINCIPAL_INVESTIGATOR
University of Minnesota
Locations
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University of Alabama at Birmingham/Children's of Alabama
Birmingham, Alabama, United States
Mayo Clinic Hospital
Phoenix, Arizona, United States
Arkansas Children's Hospital
Little Rock, Arkansas, United States
Children's Hospital Los Angeles
Los Angeles, California, United States
UCLA Oncology Center
Los Angeles, California, United States
University of California - Irvine Health
Orange, California, United States
Children's Hospital of Orange County
Orange, California, United States
Lucile Packard Children's Hospital Stanford
Palo Alto, California, United States
University of California - Davis Comprehensive Cancer Center
Sacramento, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
Nemours A. I. duPont Hospital for Children
Wilmington, Delaware, United States
Children's National Medical Center
Washington D.C., District of Columbia, United States
University of Florida Clinical Research Center
Gainesville, Florida, United States
Nicklaus Children's Hospital
Miami, Florida, United States
AdventHealth Pediatric Oncology Hematology at Orlando
Orlando, Florida, United States
Orlando Health, Inc.
Orlando, Florida, United States
Johns Hopkins All Children's Hospital
St. Petersburg, Florida, United States
Moffitt Cancer Center
Tampa, Florida, United States
Children's Healthcare of Atlanta - Center for Advanced Pediatrics
Atlanta, Georgia, United States
University of Illinois Hospital and Health Systems
Chicago, Illinois, United States
University of Chicago Medical Centers
Chicago, Illinois, United States
IU Health Brain Tumor Infusion Clinic
Indianapolis, Indiana, United States
University of Iowa Hospitals & Clinics
Iowa City, Iowa, United States
Kosair Charities Pediatric Clinical Research Unit
Louisville, Kentucky, United States
University of Michigan CS Mott Children's Hospital
Ann Arbor, Michigan, United States
Henry Ford Hospital
Detroit, Michigan, United States
University of Minnesota/Masonic Cancer Center
Minneapolis, Minnesota, United States
Mayo Clinic
Rochester, Minnesota, United States
Washington University School of Medicine-Siteman Cancer Center
St Louis, Missouri, United States
St. Joseph's Univeristy Medical Center
Paterson, New Jersey, United States
Albany Medical Center
Albany, New York, United States
Children's Hospital at Montefiore
The Bronx, New York, United States
UNC Medical Center
Chapel Hill, North Carolina, United States
Duke University Medical Center
Durham, North Carolina, United States
University of Cincinnati Medical Center
Cincinnati, Ohio, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Cleveland Clinic Foundation
Cleveland, Ohio, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
University of Oklahoma Health Sciences Center, Jimmy Everest Center for Cancer and Blood Disorders in Children
Oklahoma City, Oklahoma, United States
Oregon Health and Science University
Portland, Oregon, United States
Children's Hospital of Pittsburgh UPMC
Pittsburgh, Pennsylvania, United States
Henry-Joyce Cancer Clinic
Nashville, Tennessee, United States
Children's Medical Center
Dallas, Texas, United States
Cook Children's Medical Center
Fort Worth, Texas, United States
The University of Texas MD Anderson Cancer Center
Houston, Texas, United States
University of Utah, Center for Clinical and Translational Sciences
Salt Lake City, Utah, United States
UVA Health, Division of Neuro-Oncology
Charlottesville, Virginia, United States
Children's Hospital of The King's Daughters
Norfolk, Virginia, United States
Swedish Medical Center - Cherry Hill Campus
Seattle, Washington, United States
MACC Fund Research Center
Milwaukee, Wisconsin, United States
Countries
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References
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Moertel CL, Hirbe AC, Shuhaiber HH, Bielamowicz K, Sidhu A, Viskochil D, Weber MD, Lokku A, Smith LM, Foreman NK, Hajjar FM, McNall-Knapp RY, Weintraub L, Antony R, Franson AT, Meade J, Schiff D, Walbert T, Ambady P, Bota DA, Campen CJ, Kaur G, Klesse LJ, Maraka S, Moots PL, Nevel K, Bornhorst M, Aguilar-Bonilla A, Chagnon S, Dalvi N, Gupta P, Khatib Z, Metrock LK, Nghiemphu PL, Roberts RD, Robison NJ, Sadighi Z, Stapleton S, Babovic-Vuksanovic D, Gershon TR; ReNeu Trial Investigators; ReNeu Study Investigators. ReNeu: A Pivotal, Phase IIb Trial of Mirdametinib in Adults and Children With Symptomatic Neurofibromatosis Type 1-Associated Plexiform Neurofibroma. J Clin Oncol. 2025 Feb 20;43(6):716-729. doi: 10.1200/JCO.24.01034. Epub 2024 Nov 8.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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MEK-NF-201
Identifier Type: -
Identifier Source: org_study_id
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