NCT06693284 A Window of Opportunity Trial of Mirdametinib Plus Vorinostat for NF1 Associated, H3K27 Trimethylation Deficient Malignant Peripheral Nerve Sheath Tumor [MPNST]
| NCT ID | NCT06693284 |
| Status | Recruiting |
| Phase | EARLY_Phase 1 |
| Sponsor | University of Minnesota |
| Condition | Primary Malignant Peripheral Nerve Sheath Tumors |
| Study Type | INTERVENTIONAL |
| Enrollment | 8 participants |
| Start Date | 2025-11-20 |
| Primary Completion | 2027-01-01 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 8 participants in total. It began in 2025-11-20 with a primary completion date of 2027-01-01.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
This is a single center Phase 0 "window of opportunity" trial for the treatment of newly diagnosed, PRC2 deficient, primary malignant peripheral nerve sheath tumors (MPNSTs) with a short course of the combination of mirdametinib and vorinostat prior to the most appropriate standard of care treatment for their specific tumor (typically localized radiation followed by surgical resection). Four to eight patients, 12 years of age or older and meeting the study's biomarker inclusion criteria, would be enrolled onto this trial. After voluntary written consent (assent with parent consent for minors) the patient undergoes MRI and PET imaging of the tumor and a needle biopsy to collect tumor is performed. Patients with histone H3K27 trimethylation deficient MPNST, as confirmed by immunohistochemistry, receive a single 28-day course of mirdametinib and vorinostat at standard oral dosing for each. At day 26, 27, or 28 the patient returns to clinic for a research visit repeating the baseline MRI and PET imaging and the needle biopsy for tumor tissue. This ends direct study participation. The patient goes on to the most appropriate standard of care treatment for their MPNST. Information about the subsequent standard of care treatment is collected for the purposes of this study.
Eligibility Criteria
Inclusion Criteria: * Known Neurofibromatosis type 1 (NF-1) syndrome based on current diagnostic criteria. * Diagnosis of suspected MPNST by PET or MRI imaging * Confirmation of histone H3 lysine 27 trimethylation-negative MPSNT by immunohistochemistry * Twelve years of age or older * Complete blood count (CBC), platelet, liver and kidney function within institutional normal limits performed within 14 days of 1 st dose of study drug. * Must be able to swallow capsules. * Females of childbearing potential must use highly effective contraception (see inclusion criteria section) from the time of study enrollment through 6 months after the last dose of vorinostat and mirdametinib. Males with partners of childbearing potential must use highly effective contraception from the time of study enrollment through 3 months after the last dose of vorinostat. * Provides voluntary written consent prior to any study related activities, with parental/guardian consent and assent for those 12 to 17 years of age at enrollment. Exclusion Criteria: * Pregnant or breastfeeding - females of childbearing potential must have a negative pregnancy test (serum or urine) within 7 days prior to the 1 st dose of the study drugs. * Significant cardiac disease * Significant eye disease * Radiation therapy or chemotherapy in the past year.
Contact & Investigator
Robert Galvin, MD
PRINCIPAL INVESTIGATOR
University of Minnesota
Frequently Asked Questions
Who can join the NCT06693284 clinical trial?
This trial is open to participants of all sexes, aged 12 Years or older, studying Primary Malignant Peripheral Nerve Sheath Tumors. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT06693284 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT06693284 currently recruiting?
Yes, NCT06693284 is actively recruiting participants. Contact the research team at fulle631@umn.edu for enrollment information.
Where is the NCT06693284 trial being conducted?
This trial is being conducted at Minneapolis, United States.
Who is sponsoring the NCT06693284 clinical trial?
NCT06693284 is sponsored by University of Minnesota. The principal investigator is Robert Galvin, MD at University of Minnesota. The trial plans to enroll 8 participants.