Neurofibromatosis 1 (NF1)

Psychological interventions have been shown to be effective for managing cognitive and mental health challenges in different patient populations. The investigators are testing whether such a problem-solving skills program is effective and beneficial for children and teens with NF1 when implemented in a real-world setting. Unstuck and On Target! (UOT) is a comprehensive manualized intervention program that targets executive functioning (EF), self-determination, autonomy, quality of life, and mental health, as well as impact on parental stress. It has demonstrated effectiveness at improving EF in youth with autism. The investigators are assessing the effectiveness of the program in youth with NF1 and adapting the program for NF1, as needed, based on participant feedback.

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).

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