Clinical Trials

The purpose of this study in adolescent participants is to evaluate the effect of a low fat meal on steady state selumetinib exposure; to assess the effect on GI toxicity when selumetinib is dosed under fed and fasted conditions; and potentially, to confirm an appropriate dosing recommendation of selumetinib with a low fat meal that maintains efficacy with acceptable safety and tolerability. 

The purpose of this study will determine whether participants with asymptomatic PN in high-risk locations can potentially benefit from early treatment with selumetinib.

The purpose of this study is to evaluate children ≥ 1 year of age and adults with neurofibromatosis type 1 (NF1) and plexiform neurofibromas treated with the MEK inhibitor, binimetinib. The primary objective is to determine if there is an adequate level of disease responsiveness to binimetinib in children and adults with NF1 and inoperable plexiform neurofibromas. The objective response to binimetinib is defined as ≥ 20% decrease in tumor volume reduction by 12 courses.

The purpose of this study is to evaluate PD-0325901 in the treatment of symptomatic inoperable neurofibromatosis type-1 (NF1)-associated plexiform neurofibromas (PNs). All participants will receive PD-0325901.

The pupose of this study is to evaluate whether or not selumetinib works just as well as the standard treatment with carboplatine/vincristine (CV) for subjects with NF1-associated low grade glioma (LGG), and to see if selumetinib is better than CV in improving vision in subjects with LGG of the optic pathway (vision nerves). Selumetinib is a drug that works by blocking some enzymes that low grade glioma tumor cells need for their growth. This results in killing tumor cells. Drugs used as chemotherapy, such as carboplatin and vincristine, work in different ways to stop the growth of tumor cells, either by ...

This phase II trial studies how well vismodegib and focal adhesion kinase (FAK) inhibitor GSK2256098 work in treating patients with meningiomas that may have gotten bigger or grew back after treatment. Vismodegib and FAK inhibitor GSK2256098 may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

The purpose of this research study is to understand the natural history of vision in patients with OPG and determine if there are factors (e.g. age at diagnosis, male/female, tumor location, features of the MRI exam, etc) that predict future vision loss or change in tumor size.

Another purpose of the study is to collect and store blood and tissue samples to use for future research to evaluate if there are certain variations in DNA, RNA, or proteins that predict the likelihood of an OPG to grow or cause vision loss.

The purpose of this study is to investigate the effectiveness and safety of REC-2282 in patients with progressive NF2 mutated meningiomas who have either NF2 disease-related meningioma or sporadic meningiomas that have NF2 mutations.

The purpose of this study is to test multiple experimental therapies simultaneously in patients with neurofibromatosis type 2 (NF2) with associated progressive tumors of vestibular schwannomas (VS), non-vestibular schwannomas (non-VS), meningiomas, and ependymomas. This Master Study is being conducted as a "basket" study that may allow people with multiple tumor types associated with NF2 to receive new drugs throughout this study. Embedded within the Master Study are individual drug substudies. - Investigational Drug Sub-study A: Brigatinib.