Experimental drug targets deadly brain virus in small trial
NCT ID NCT07511049
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.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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About 24 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Jun 2026
- Expected to finish
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Dec 2029
An estimate. End dates often move.
- Lead sponsor
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A government research agency
The lead sponsor is the US National Institutes of Health.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 to 99 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
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Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
* INCLUSION CRITERIA: * Able to provide informed consent or have a designated legally authorized representative (LAR) to provide consent * Stated willingness to comply with study procedures and to participate for the duration of the study including follow-up * Actively progressing, clinically definite or probable PML (2013 AAN Consensus Diagnostic Criteria) * Positive PCR for JCPyV in CSF * Age 18 or older * Medically stable and able to tolerate travel to NIH * Participants of childbearing or child-fathering potential must be willing to use a medically acceptable form of birth control, which includes abstinence, while being treated on this study EXCLUSION CRITERIA: * ALT or AST \> 5 x the ULN, total bilirubin \> 3 mg/dL (SI: \>51 micromol/L), or spontaneous prothrombin time-international normalized ratio (PT-INR) \> 2 x ULN within 7 days prior to Day 1 * An estimated glomerular filtration rate of \< 30 mL/min within 7 days prior to Day 1 * Hypersensitivity to CDV or to BCV or its formulation excipients, or prior intolerance to these agents that, in the opinion of the investigator, would pose an unacceptable safety risk. * Active CNS disease other than PML that, in the opinion of the investigator, would confound study assessments or pose an unacceptable safety risk. * Contraindication to MRI (including cardiac pacemakers and some infusion pumps, other metallic implants, metallic foreign objects) * Medical contraindication to LP * Positive pregnancy test or nursing
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
1 site. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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National Institutes of Health Clinical Center
RECRUITINGBethesda, Maryland, 20892, United States
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