Actively Recruiting
Intravenous Brincidofovir as an Antiviral for Treatment of Progressive Multifocal Leukoencephalopathy: A Pilot Study
Led by National Institute of Neurological Disorders and Stroke (NINDS) · Updated on 2026-05-14
24
Participants Needed
1
Research Sites
188 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Background: Progressive multifocal leukoencephalopathy (PML) is a rare and often fatal brain infection caused by the JC virus. The JC virus is common. More than half of adults have been exposed to it. Most people do not get sick from the JC virus, but in people with weakened immune systems, it can cause PML. Brincidofovir (BCV) is an antiviral drug approved to treat smallpox. Researchers want to know if it can help people with PML. Objective: To test BCV in people with PML. Eligibility: People aged 18 years or older with PML. Design: Participants will be screened. They will have a physical exam with blood tests. They will have an imaging scan of the brain with contrast dye. They will have a lumbar puncture (spinal tap): A thin needle will be inserted into their lower back to draw out a sample of the fluid around their spinal cord. BCV will be given through a tube attached to a needle inserted into a vein. Participants will receive the drug 2 times a week for 4 weeks (this is 1 cycle). If the drug is helping them, they may have up to 3 drug cycles (12 weeks). Imaging scans, spinal taps, and other tests will be repeated after every 4 weeks of treatment. Participants will have 6 follow-up visits in 1 year after treatment ends. The imaging scan, spinal tap, and other tests will be repeated at each visit.
CONDITIONS
Official Title
Intravenous Brincidofovir as an Antiviral for Treatment of Progressive Multifocal Leukoencephalopathy: A Pilot Study
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Able to provide informed consent
- Willing to comply with study procedures and participate for the entire study including follow-up
- Have actively progressing, clinically definite or probable PML
- Positive PCR test for JC virus in cerebrospinal fluid
- Age 18 years or older
- Medically stable and able to travel to the study site
- Willing to use medically acceptable birth control if able to father or bear children during treatment
You will not qualify if you...
- ALT or AST liver enzymes more than 5 times normal limit, total bilirubin over 3 mg/dL, or prothrombin time-INR more than twice normal within 7 days before study start
- Kidney function with estimated glomerular filtration rate less than 30 mL/min within 7 days before study start
- Hypersensitivity or prior intolerance to cidofovir or brincidofovir or their ingredients posing safety risk
- Active central nervous system disease other than PML that could affect study or safety
- Contraindications to MRI such as pacemakers or metallic implants
- Medical reasons preventing lumbar puncture
- Positive pregnancy test or currently nursing
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 1 location
1
National Institutes of Health Clinical Center
Bethesda, Maryland, United States, 20892
Actively Recruiting
Research Team
I
Irene CM Cortese, M.D.
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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