MEK and MDM2 inhibitors for NF1-related tumors
Treatments studied
Part of Brain & nervous system, Cancer, Genetic & congenital clinical trials.
This trial tests two targeted drugs (selumetinib and milademetan) for people with neurofibromatosis type 1 (NF1) who have either a malignant peripheral nerve sheath tumor (MPNST) or an atypical neurofibromatous neoplasm with uncertain biologic potential (ANNUBP). The goal is to see if blocking certain proteins can shrink or control these tumors.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have NF1 and a tumor type called MPNST (that has spread or cannot be removed) or ANNUBP (planned for surgery).
- You are at least 12 years old (18 or older for Part C).
- You have measurable disease on a CT scan or MRI within the last 4 weeks.
- For MPNST, you have had at least one prior chemotherapy or you and your doctor agree the trial is a better option first.
- You are healthy enough to have a performance status of 50% or more (able to get out of bed more than half the day).
- Your blood counts, liver and kidney function are within safe limits, and your heart is working well.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
Similar studies
Other trials that look related to this one.
This trial tests a drug called mirdametinib, which targets a protein linked to tumor growth, given alone or with radiation. It is for people with certain brain or spinal cord tumors, especially those with a condition called NF1, to see if it can help control the cancer.
This trial studies whether the medicine abemaciclib can shrink or slow the growth of “atypical neurofibromas” in people with NF1. Participants take pills and undergo regular check-ups, including MRI scans to measure the tumor.
This trial tests two oral targeted drugs, avutometinib and defactinib, for malignant peripheral nerve sheath tumor (MPNST) that has come back or spread. The hope is that these drugs can shrink or slow the tumor where chemotherapy hasn't worked.
This trial tests two targeted drugs, mirdametinib and vorinostat, to see if they can shrink MPNST tumors in people with NF1 before surgery. It is for patients whose tumors lack a specific protein marker (H3K27me3).
This trial tests a new pill (smeitinib) to shrink tumors caused by neurofibromatosis type 1 (NF1). It is for adults with NF1 who have tumors that cannot be removed by surgery, including those that press on the brain or spine.
This trial tests a capsule medication (smetinib) for people with type 1 neurofibromatosis (NF1) who have tumors that can be surgically removed but have not spread to vital areas like the brain or spine. The goal is to see if the drug can help shrink or control these tumors.
Hear when a new Malignant Peripheral Nerve Sheath Tumor (MPNST) trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.