NCT07511049 Intravenous Brincidofovir as an Antiviral for Treatment of Progressive Multifocal Leukoencephalopathy: A Pilot Study
| NCT ID | NCT07511049 |
| Status | Recruiting |
| Phase | Phase 2 |
| Sponsor | National Institute of Neurological Disorders and Stroke (NINDS) |
| Condition | Progressive Multifocal Leukoencephalopathy |
| Study Type | INTERVENTIONAL |
| Enrollment | 24 participants |
| Start Date | 2026-06-05 |
| Primary Completion | 2029-12-31 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
In Phase 2, researchers evaluate early signs of effectiveness. You may be randomized to receive the active treatment or a comparator. Monitoring continues closely.
This trial targets 24 participants in total. It began in 2026-06-05 with a primary completion date of 2029-12-31.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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.
Eligibility Criteria
* INCLUSION CRITERIA: * Able to provide informed 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
Contact & Investigator
Irene CM Cortese, M.D.
PRINCIPAL INVESTIGATOR
National Institute of Neurological Disorders and Stroke (NINDS)
Frequently Asked Questions
Who can join the NCT07511049 clinical trial?
This trial is open to participants of all sexes, aged 18 Years or older, up to 99 Years, studying Progressive Multifocal Leukoencephalopathy. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT07511049 trial and what does that mean for participants?
Phase 2 trials evaluate whether the treatment shows signs of effectiveness while continuing to monitor safety. More participants are enrolled than in Phase 1 to help refine the treatment protocol.
Is NCT07511049 currently recruiting?
Yes, NCT07511049 is actively recruiting participants. Contact the research team at corteseir@ninds.nih.gov for enrollment information.
Where is the NCT07511049 trial being conducted?
This trial is being conducted at Bethesda, United States.
Who is sponsoring the NCT07511049 clinical trial?
NCT07511049 is sponsored by National Institute of Neurological Disorders and Stroke (NINDS). The principal investigator is Irene CM Cortese, M.D. at National Institute of Neurological Disorders and Stroke (NINDS). The trial plans to enroll 24 participants.