The purpose of the study is to evaluate the safety of Lutathera alone in patients with recurrent glioblastoma and the combination of Lutathera with radiotherapy with temozolomide (TMZ) in patients with newly diagnosed glioblastoma (GB) and to establish the appropriate dose of this combination. Lutathera is a radioligand therapy, which is a targeted…
The purpose of this study is to find the best dose of a drug called MTX110 and a contrast agent called gadolinium that can be given safely in children with newly diagnosed diffuse midline gliomas, which is a kind of brain tumor. All patients enrolled in the study will receive infusion of MTX110 and Gadolinium delivered with a pump directly into the tumor…
The objective of the LIFE-BTK RCT is to evaluate the safety and efficacy of the ESPRIT BTK device (which is a bioresorbable polymeric scaffold with the everolimus drug and a bioresorbable polymeric coating mounted on a balloon dilatation catheter) compared to Percutaneous Transluminal Angioplasty (PTA), which is is a procedure that can open up a blocked…
The purpose of this study is to evaluate the efficacy and safety of ION-682884 given for 24 months in patients with hereditary transthyretin-mediated amyloid polyneuropathy (hATTR-PN) also known as Familial Amyloid Polyneuropathy (FAP), and determine if it can help people with mild or moderate polyneuropathy. To be in this study you must have FAP and be…
HELIOS-A is a global Phase 3 randomized, open-label study designed to evaluate the safety and efficacy of ALN-TTRSC02 in adult patients with hATTR amyloidosis experiencing neurologic symptoms brought on by the disease. The study will also evaluate any changes in quality of life experienced by study participants. ALN-TTRSC02 utilizes the mechanism of RNA…