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

Part of paid clinical trials in Bethesda, Maryland.

Sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
Study ID
NCT07511049
Phase
PHASE2
Status
Recruiting
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Conditions

  • Progressive Multifocal Leukoencephalopathy

Eligibility Criteria

Sex
ALL
Age
18 Years - 99 Years
Healthy Volunteers
Not accepted

Interventions

  • Brincidofovir — DRUG
    intravenous administraion of anti-viral agent

Study Details

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.

Key Dates

Start date
Jun 5, 2026
Status verified
Jun 2026
Primary completion
Dec 31, 2029
Completion
Dec 31, 2029

Study Design

Enrollment
24 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Brincidofovir treatment arm
    experimental treatment arm

Primary Outcome Measure

Incidence of treatment-related adverse events (AEs) of Grade 3 severity or higher as determined by Common Terminology Criteria for Adverse Events (CTCAE) [ Time Frame: Over duration of trial participation ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
National Institutes of Health Clinical CenterBethesdaMaryland20892
Neuroimmunology Clinic
[email protected]
301-496-3825
NIH Clinical Center Office of Patient Recruitment (OPR)
[email protected]
(800) 411-1222

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By research site
National Institutes of Health Clinical Center· Bethesda, MD

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