Experimental drug targets deadly brain virus in small trial

NCT ID NCT07511049

Recruiting now Disease control Sponsor: National Institute of Neurological Disorders and Stroke (NINDS) Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jul 23, 2026 · Updated 6 times

Summary

This phase 2 trial tests the safety of brincidofovir, an antiviral drug, in 24 adults with progressive multifocal leukoencephalopathy (PML), a rare brain infection caused by the JC virus. Participants receive the drug intravenously twice a week for up to 12 weeks, with follow-up for one year. The goal is to see if the drug is safe and can reduce the virus in spinal fluid.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: brincidofovir (an antiviral drug given intravenously)
What this could lead to: If it works, this could point toward a treatment for PML, a rare and often fatal brain infection with no approved therapies.
What could go wrong: This is a small, early-phase pilot study with only 24 participants, so results may not apply broadly. The drug may cause serious side effects or fail to control the virus.

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Conditions

The condition(s) this trial relates to.

progressive multifocal leukoencephalopathy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

PROGRESSIVE MULTIFOCAL LEUKOENCEPHALOPATHY

Contacts and locations

Locations

National Institutes of Health Clinical Center
RECRUITING

Bethesda, Maryland, 20892, United States

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