Intravenous brincidofovir treatment for progressive multifocal leukoencephalopathy

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a Phase 2 pilot study testing safety and tolerability of IV brincidofovir (BCV) in adults with confirmed PML and positive JCPyV DNA in cerebrospinal fluid. Eighteen participants will receive BCV 20 mg (or 0.4 mg/kg if <50 kg) twice weekly in 4-week infusion cycles for up to three cycles (12 weeks total), with the first three participants enrolled in a staggered fashion. Baseline evaluations include physical exam, blood tests, contrast brain MRI, and lumbar puncture, and participants are monitored monthly between cycles and for up to 12 months after treatment. Secondary data collection will track changes in JCPyV viral load in CSF and clinical course.

Why it matters

Who can join: the full rules

* 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

Where it's running

• National Institutes of Health Clinical Center
  Bethesda, Maryland, United States Recruiting

Study contacts

• Principal investigator: Irene CM Cortese, M.D. — National Institute of Neurological Disorders and Stroke (NINDS)
• Study coordinator: Irene CM Cortese, M.D.
• Email: corteseir@ninds.nih.gov
• Phone: (301) 496-1801

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• PML risk in people with MS treated with ozanimod

  This study looks at whether people with MS who take ozanimod develop PML and how often it happens.

  Recruiting Bristol-Myers Squibb · Lawrenceville, New Jersey · NCT07732907

• Observational cohort of chronic brain viral infections (including PML)

  This project will try immune checkpoint inhibitors in people with chronic central nervous system viral infections like PML to see if they lead to better long-term outcomes.

  Recruiting Peking Union Medical College Hospital · Beijing, Beijing Municipality · NCT07512518

• Using a radioactive tracer to detect immune cells in the brain for multiple sclerosis and PML

  This study is testing a new radioactive tracer to see if it can help find certain immune cells in the brains of people with multiple sclerosis and PML.

  Recruiting Phase 1 National Institutes of Health Clinical Center (CC) · Bethesda, Maryland · NCT05849467

• Understanding the natural history of Progressive Multifocal Leukoencephalopathy

  This study looks at how Progressive Multifocal Leukoencephalopathy (PML) affects people with weakened immune systems to better understand the disease and its risk factors.

  Recruiting National Institutes of Health Clinical Center (CC) · Bethesda, Maryland · NCT01730131

• T-cell therapy for progressive multifocal leukoencephalopathy (PML).

  This trial will test whether human allogeneic HPyV-2-specific T cells are safe and can improve neurological function, quality of life, and survival in adults with recent PML.

  Recruiting at 3 sites Phase 2 Hannover Medical School · München, Bavaria and 6 other locations · NCT06990087

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.