MEK Inhibitor Mirdametinib (PD-0325901) in Patients With Neurofibromatosis Type 1 Associated Plexiform Neurofibromas (RENEU)
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
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Clinical Trial Information
Trial Contact: Spinelli, Jennifer; El-Shami, Jessica; Leffin, Melissa; Cevallos, Claudia
Trial Phone: 321.841.5357 ; 321.841.3837 ; 321.841.2008 ; 321-841-7561
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IRB No: 19.252.09
Protocol Abbrev: MEK-NF-201
Principal Investigator: Kalpana Vikram Patel, MD
Phase: Drug: Phase II
Age Group: Pediatric
Secondary Protocol No: MEK-NF-201
Treatment: Drug: PD-0325901 oral capsule
Therapies Involved: Medication
ClinicalTrials.gov ID: NCT03962543
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Objective
To evaluate the confirmed complete and
partial response (PR) rate of PD-0325901
using volumetric magnetic resonance imaging
(MRI) analysis in participants with an
inoperable NF1-associated PN that is
progressing or causing significant morbidity. -
Key Eligibility
• 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.
• Participant can swallow capsules whole.
