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Experimental drug targets deadly brain virus in small trial

NCT ID NCT07511049

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 24 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jun 2026

Expected to finish

Dec 2029

An estimate. End dates often move.

Lead sponsor

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

18 to 99 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

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.

  1. The places running it

    1 site. The list below names each one and where it is.

  2. 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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  3. A doctor treating you

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Contacts and locations

Locations

  • National Institutes of Health Clinical Center

    RECRUITING

    Bethesda, Maryland, 20892, United States

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