Ongoing Clinical Trials for NF-Related Issues

The following clinical trials and other NF-related trials can be found at ClinicalTrials.gov by searching “neurofibromatosis”. While some of the information is very technical, the basic information on the purpose of the trial is usually straightforward. There is detailed information on who is eligible and at what locations the trial is open.

-Phase II Trial of the MEK1/2 Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) in Adults With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas

The connection between neurofibromatosis, tumors, and cancer treatment is strong, and this has made clinical trials for NF-related tumors a major part of ongoing research in NF. New medications that block the growth of cells that lack the NF1 protein or the NF2 protein have been discovered and are useful in some cancers that are not part of NF. There is a particularly promising set of medications that are being tested for progressive plexiform neurofibromas of NF1. Dr. Brigitte Widemann at the National Cancer Institute and collaborators at many NF Clinics are currently testing “MEK inhibitors” for progressing plexiform neurofibromas.

-Acceptance and Commitment Training for Adolescents and Young Adults With Neurofibromatosis Type 1, Plexiform Neurofibromas, and Chronic Pain

Clinical trials that address other features of NF are also very important. This trial, sponsored by the National Cancer Institute, is a good example that involves dealing with pain management for NF patients with tumors.