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Active, not recruiting Phase 2 Interventional Results available

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

ClinicalTrials.gov ID: NCT03962543

Public ClinicalTrials.gov record NCT03962543. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

ClinicalTrials.gov public records Last synced Oct 26, 2026, 1:12 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Official title

A Phase 2b Trial of the MEK 1/2 Inhibitor (MEKi) PD-0325901 in Adult and Pediatric Patients With Neurofibromatosis Type 1 (NF1)-Associated Inoperable Plexiform Neurofibromas (PNs) That Are Causing Significant Morbidity

Brief summary

Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.

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.

Study identification

NCT ID
NCT03962543
Recruitment status
Active, not recruiting
Study type
Interventional
Phase
Phase 2
Enrollment
114 participants

Conditions and interventions

Eligibility (public fields only)

Age range
2 Years and older
Sex
All
Healthy volunteers
Healthy volunteers not accepted

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date
Sep 29, 2019 (Actual)
Primary completion
Sep 20, 2023 (Actual)
Completion
Dec 22, 2028 (Estimated)
Last update posted
Oct 6, 2026

2019 – 2028

United States locations

U.S. sites
43
U.S. states
25
U.S. cities
40
Facility City State ZIP Site status
University of Alabama at Birmingham (UAB) - Children's of Alabama Birmingham Alabama 35233 —
Mayo Clinic Arizona Phoenix Arizona 85054 —
Arkansas Children's Hospital - University of Arkansas Little Rock Arkansas 72202 —
Children's Hospital Los Angeles Los Angeles California 90027 —
University of California, Los Angeles (UCLA) Los Angeles California 90095 —
Children's Hospital of Orange County Orange California 92868 —
University of California - Irvine Health Orange California 92868 —
Lucile Packard Children's Hospital Stanford University Palo Alto California 94304 —
University of California - Davis Sacramento California 95817 —
Children's Hospital Colorado Aurora Colorado 80045 —
Nemours A. I. duPont Hospital for Children Wilmington Delaware 19803 —
Children's National Hospital Washington D.C. District of Columbia 20010 —
University of Florida (UF) - Gainesville Gainesville Florida 32610 —
Nicklaus Children's Hospital Miami Florida 33155 —
Florida Hospital Cancer Institute Orlando Florida 32804 —
Arnold Palmer Hospital for Children Orlando Florida 32806 —
Johns Hopkins All Children's Hospital St. Petersburg Florida 33701 —
Moffitt Tampa Florida 33612 —
University of Illinois Hospital Chicago Illinois 60612 —
Indiana University Indianapolis Indiana 46202 —
University of Iowa Health Care Iowa City Iowa 52242 —
University of Michigan Comprehensive Cancer Center Ann Arbor Michigan 48109 —
Henry Ford Hospital Detroit Michigan 48202 —
University of Minnesota Masonic Cancer Center Minneapolis Minnesota 55455 —
Mayo Clinic Rochester Minnesota 55905 —
Washington University St Louis Missouri 63110 —
Saint Joseph's Regional Medical Center Paterson New Jersey 07503 —
Albany Medical Center Albany New York 12208 —
Children's Hospital at Montefiore The Bronx New York 10467 —
University of North Carolina (UNC) Hospitals Chapel Hill North Carolina 27599 —
Duke University Medical Center Durham North Carolina 27710 —
UC Health Cincinnati Ohio 45229 —
Nationwide Children's Hospital Columbus Ohio 43205 —
University of Oklahoma - Health Sciences Center Oklahoma City Oklahoma 73104 —
Oregon Health and Science University Portland Oregon 97239 —
UPMC Children's Hospital of Pittsburgh Pittsburgh Pennsylvania 15219-2739 —
Vanderbilt Children's Hospital Nashville Tennessee 37232 —
University of Texas Southwestern Dallas Texas 75244 —
Cook Children's Medical Center Fort Worth Texas 76104 —
MD Anderson Cancer Center Houston Texas 77030 —
The University of Utah Health Sciences Center Salt Lake City Utah 84108 —
University of Virginia - Emily Couric Cancer Center Charlottesville Virginia 22903 —
Children's Hospital of The King's Daughters Norfolk Virginia 23510 —

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

About this trial record page

What this page shows
Public field values for ClinicalTrials.gov record NCT03962543, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do
No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from
Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh
Last update posted Oct 6, 2026 · Synced Oct 26, 2026

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Open the official record

The complete public registry record, listed eligibility criteria, and available contact information for NCT03962543 live on ClinicalTrials.gov.

View official ClinicalTrials.gov record →