Clinical Trials

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 PD-0325901 in the treatment of symptomatic inoperable neurofibromatosis type-1 (NF1)-associated plexiform neurofibromas (PNs). All participants will receive PD-0325901.

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 registry is to collect clinical, diagnostic, and therapeutic data specific for primary spinal column tumor patients, and evaluate it to understand the predictive factors which influence survival, local recurrence, adverse events, perioperative morbidity, and health related quality of life.

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.