Health Trials

Neurofibromatosis 1 (NF1)

2

Review clinical trials related to Neurofibromatosis 1 (NF1). Use filters to narrow results by trial status, phase, treatment, biological sex and sponsor.

Condition / disease
Location
Status: Recruiting

Early Phase Study Evaluating MEK and MDM2 Inhibition in Patients With NF1 and MPNST

This is a phase 0/1/2, multi-site study to evaluate the MEK inhibitor Selumetinib with the MDM2 Inhibitor APG-115 in patients with Neurofibromatosis Type 1 and pre-malignant and malignant peripheral nerve sheath tumors

Participants needed: 45
Trial details
Phase: Phase 1, Phase 2Age: 18+Biological sex: AllType: InterventionalSponsor: AeRang KimUpdated: Jul 27, 2026Locations: 2
Eligibility criteria

AGE: Part A and C: ≥ 18 years of age AGE: Part B: ≥12 years (minimum BSA ≥0.55m2... [+28]

A malignancy treated with curative intent and with no known active disease ≥2 ye... [+36]

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Status: Not yet recruiting

Efficacy of Mirdametinib Alone or Combination With Radiotherapy for Germline and Sporadic NF1-Altered High-Grade Glioma

This is a phase 2, open label, parallel multi-arm study of mirdametinib in combination with radiation in participants with recurrent sporadic glioblastoma (GBM) harboring NF1 alterations (Cohort 1); participants with NF1 with a newly diagnosed GBM (Cohort 2); mirdametinib alone in other NF1-associated High-Grade Gliomas (Cohort 3).

Participants needed: 55
Trial details
Phase: Phase 2Age: 12-89Biological sex: AllType: InterventionalSponsor: University of Alabama at BirminghamUpdated: Apr 13, 2026Locations: 1
Eligibility criteria

Cohort 1 (applies to non-NF1 related GBM): GBM, IDH-wildtype grade 4 of brain or... [+32]

Tumor positive for pathogenic IDH-mutation, H3.1 or H3.3 mutation, or BRAF alter... [+27]

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