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

What this Trial Is About

Progressive multifocal leukoencephalopathy (PML) is a rare and often fatal brain infection caused by the JC virus, which commonly infects adults but usually does not cause illness unless the immune system is weakened. Researchers are studying intravenous Brincidofovir (BCV), an antiviral drug approved for smallpox, to see if it can help people with PML. This pilot study focuses on safety, tolerability, and preliminary effects of BCV in adults diagnosed with PML confirmed by positive JC virus DNA in spinal fluid. Participants will receive BCV through a vein twice a week for 4 weeks per treatment cycle, with up to three cycles totaling 12 weeks if the drug shows benefit. After each cycle, brain imaging scans, spinal taps, and other tests will be repeated to monitor progress. Following treatment, participants will have monthly evaluations for three months to decide whether to continue treatment or monitoring. They will be followed for up to one year after treatment ends with repeated scans, spinal taps, and tests at six follow-up visits to assess changes in disease and safety. During the study, people will undergo physical exams, blood tests, brain MRI with contrast dye, and lumbar punctures to collect spinal fluid. Researchers will monitor safety by recording treatment-related side effects and measure viral loads in spinal fluid to evaluate antiviral effects. They will also assess changes in clinical symptoms, disability scores, brain lesion size, and survival up to 12 months after treatment. Participation includes regular visits over about 15 months from screening through follow-up to track treatment impact and safety.

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