Mirdametinib and Vorinostat for NF1-Associated MPNST
This study is testing a short course of two drugs, mirdametinib and vorinostat, for newly diagnosed malignant peripheral nerve sheath tumors (MPNSTs) in people with Neurofibromatosis type 1 (NF1). MPNSTs are a type of cancer that affects nerves. This treatment is given before standard care like radiation and surgery. The study wants to see how safe the drug combination is, how it affects the tumor, and if imaging can show its effects. You may be able to join if you are 12 years or older, have NF1, and your tumor shows a specific biomarker (H3K27 trimethylation deficient). The study plans to enroll 8 patients.
- Study design
- This is a single-center study, meaning it's conducted at one location. It is a Phase 0 trial, which is an early stage of research, and plans to enroll 8 participants.
- What's involved
- After giving consent, you would have MRI and PET scans of your tumor, and a needle biopsy to collect a tumor sample.
- Compensation
- Not stated in the trial record.
- Follow-up
- The study will assess outcomes like toxicity, therapeutic target response, and radiographic response over 48 months.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
A Window of Opportunity Trial of Mirdametinib Plus Vorinostat for NF1 Associated, H3K27 Trimethylation Deficient Malignant Peripheral Nerve Sheath Tumor [MPNST]
At a glance
Conditions
NCT06693284
Where you'd take part
This study runs at 1 site. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.
University of Minnesota
Minneapolis, Minnesotastudy coordinator listed
Recruiting
Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.
Study leadership
- Robert Galvin, MD · PRINCIPAL_INVESTIGATOR · University of Minnesota
Who to contact
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Inclusion
Exclusion
What this trial measures
- Assess the toxicity of the combination of mirdametinib and vorinostat in patients with NF1-associated high grade MPNSTs.48 months
As a result of the vaccine, both unique CNS toxicity and edema have been previously observed as soon as 10 days from the initiation of therapy. Neurotoxicity ≤ Grade 3 is an expected risk of this treatment. This will be measured as the number of participants experiencing this adverse event.
- Determine the response of therapeutic targets48 months
measuring phospho-ERK and histone H3K27 acetylation in post-therapy tumor tissue as compared to pre-treatment tumor tissue.
- Evaluate the utility of radiographic response to this regimen48 months
using MRI and PET. Each patient will undergo MRI of the primary lesion and CT/PET scan for staging and assessment of FDG uptake in the tumor at diagnosis and after completing 28 days of therapy with mirdametinib and vorinostat. Each patient will have a diagnostic tumor biopsy performed as a condition of entry into the study and to measure specific molecular tumor markers. A second biopsy will be performed at the completion of protocol therapy to assess tumor response to the therapy and to see if the specific tumor markers have changed as a result of the therapy given.