Combining mirdametinib and vorinostat for treating specific nerve tumors in NF1 patients
A Window of Opportunity Trial of Mirdametinib Plus Vorinostat for NF1 Associated, H3K27 Trimethylation Deficient Malignant Peripheral Nerve Sheath Tumor [MPNST]
This study is testing a new combination of two medications to see if they can help patients with a specific type of nerve tumor related to Neurofibromatosis type 1 before starting regular treatments.
Quick facts
| Phase | Early Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 8 (estimated) |
| Ages | 12 Years and up |
| Sex | All |
| Sponsor | University of Minnesota Academic / other |
| Drugs / interventions | mirdametinib, chemotherapy, Radiation |
| Locations | 1 site (Minneapolis, Minnesota) |
| Trial ID | NCT06693284 on ClinicalTrials.gov |
What this trial studies
This trial investigates the effectiveness of a short course of mirdametinib and vorinostat in patients with newly diagnosed malignant peripheral nerve sheath tumors (MPNSTs) associated with Neurofibromatosis type 1 (NF1) and specific histone deficiencies. The study aims to enroll 4 to 8 patients aged 12 years or older who meet the biomarker criteria. Participants will undergo imaging and a biopsy before receiving the treatment, followed by a repeat MRI to assess the tumor's response. This early-phase trial serves as a 'window of opportunity' before standard treatment options are initiated.
Who should consider this trial
Good fit: Ideal candidates are individuals aged 12 years or older with NF1 and histone H3K27 trimethylation deficient MPNST.
Not a fit: Patients without NF1 or those with other types of tumors not meeting the histone deficiency criteria may not benefit from this study.
Why it matters
Potential benefit: If successful, this approach could provide a new treatment option for patients with aggressive nerve tumors, potentially improving outcomes.
How similar studies have performed: While this specific combination is novel, similar approaches targeting tumor biology in NF1-related tumors have shown promise in other studies.
Eligibility criteria
Show full inclusion / exclusion 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.
Where this trial is running
Minneapolis, Minnesota
- University of Minnesota — Minneapolis, Minnesota, United States (Recruiting)
Study contacts
- Principal investigator: Robert Galvin, MD — University of Minnesota
- Study coordinator: Kris Beatrez
- Email: kbeatrez@umn.edu
- Phone: 612.624.1928
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.