Pilot Study Testing Intravenous Brincidofovir for Adults with Progressive Multifocal Leukoencephalopathy

What this Trial Is About

Progressive multifocal leukoencephalopathy PML is a rare and often fatal brain infection caused by the JC virus, which is common among adults but usually does not cause illness except in people with weakened immune systems. Researchers are evaluating intravenous brincidofovir BCV, an antiviral drug approved for smallpox, to see if it can help treat people with PML. This pilot study focuses on safety, tolerability, and the biological and clinical effects of BCV in adults with PML. Participants will receive BCV intravenously twice a week for a 4-week treatment cycle. If the drug shows benefit, they may receive up to three cycles over 12 weeks. Before and after each treatment cycle, participants will have brain imaging scans with contrast dye, lumbar punctures to collect spinal fluid, and other tests. After treatment ends, participants will be monitored for up to 12 months with six follow-up visits including repeated scans, spinal taps, and assessments. During the study, participants will undergo physical exams, blood tests, brain imaging, and spinal fluid collection to track their condition and response to treatment. Researchers will measure treatment-related adverse events, changes in JC virus levels in spinal fluid, brain lesion burden, disability scores, and survival over 12 months. This comprehensive monitoring aims to evaluate the safety and potential antiviral effects of BCV in managing PML.

Intravenous Brincidofovir as an Antiviral for Treatment of Progressive Multifocal Leukoencephalopathy: A Pilot Study