Clinical Trials

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 objective of this study is to collect blood samples from living patients with neurofibromatosis type 1 (NF1) for future genomic, proteomic, metabolomic, and other research studies.

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 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 ...

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.