A Window of Opportunity Trial of Mirdametinib Plus Vorinostat for NF1 Associated, H3K27 Trimethylation Deficient Malignant Peripheral Nerve Sheath Tumor [MPNST]

Conditions

• Primary Malignant Peripheral Nerve Sheath Tumors

Eligibility Criteria

Sex

ALL

Age

12 Years - N/A

Healthy Volunteers

Not accepted

Interventions

• mirdametinib and vorinostat — DRUG
  short course of the combination of mirdametinib and vorinostat prior to the most appropriate standard of care treatment for the patient's specific tumor (typically localized radiation followed by surgical resection).

Study Details

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.

Key Dates

Start date

Nov 20, 2025

Status verified

May 2026

Primary completion

Jan 1, 2027

Completion

Jan 1, 2027

Study Design

Enrollment

8 participants (estimated)

Allocation

NA

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Arms

• Experimental: Experimental group
  newly diagnosed, PRC2 deficient, primary malignant peripheral nerve sheath tumors (MPNSTs)

Primary Outcome Measure

Assess the toxicity of the combination of mirdametinib and vorinostat in patients with NF1-associated high grade MPNSTs. [ Time Frame: 48 months ]

Central Contacts

• Allison Fullenkamp
  612-625-6125
  [email protected]

Locations (1)

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