Ongoing Clinical Trials for Progressive Multifocal Leukoencephalopathy
There is currently one clinical trial investigating treatments for Progressive Multifocal Leukoencephalopathy (PML), a rare and serious brain infection that affects people with weakened immune systems. This trial is testing whether an immune-boosting medication called Pembrolizumab can help reduce virus levels in the brain and improve outcomes for patients.
Clinical trial locations
Study of Pembrolizumab for Progressive Multifocal Leukoencephalopathy in Immunocompromised Patients Without HIV
This clinical trial is taking place in France and focuses on treating Progressive Multifocal Leukoencephalopathy in patients whose immune systems are weakened, but who do not have HIV and are not taking certain medications for chronic inflammatory conditions.
Who can join this trial:
- Adults who are 18 years old or older
- Patients who have been diagnosed with PML within the last 2 months, confirmed according to American Academy of Neurology guidelines
- Patients who have the JC virus (JCV) present in their cerebrospinal fluid, which is the fluid surrounding the brain and spinal cord
- Patients who have signed informed consent, either themselves or through a representative if they are unable to do so
- Women of childbearing age must have a negative pregnancy test and agree to use highly effective birth control methods during the trial and for 8 months after treatment ends
Who cannot join this trial:
- Patients whose weakened immune system is caused by a condition that cannot be easily reversed
- This trial specifically excludes patients with HIV or those taking certain medications (biologics) for long-term inflammatory diseases
What the trial is studying:
The main goal of this study is to find out whether Pembrolizumab can help reduce the amount of JC virus in the cerebrospinal fluid of patients with PML. Researchers want to see if this medication can help the immune system fight the virus more effectively and improve patient outcomes.
How the trial works:
Participants will receive Pembrolizumab through an intravenous infusion, meaning the medication is given directly into a vein. Throughout the study, patients will have regular check-ups that include lumbar punctures to measure virus levels in the cerebrospinal fluid at the start of the study and at 1, 2, and 3 months. Doctors will also assess neurological health using specialized scales that evaluate how well patients can perform daily activities and their overall brain function. These assessments continue at 6 and 12 months as well.
The investigational drug:
Pembrolizumab is a type of medication called an immune checkpoint inhibitor. It works as a protein that helps boost the immune system’s ability to fight diseases. In this trial, researchers are testing whether it can help the body fight against the JC virus that causes PML. The medication is administered in a clinical setting under medical supervision, and patient safety is closely monitored throughout the treatment period.
The study is expected to continue until September 2027, giving researchers time to collect detailed information about how well Pembrolizumab works for this condition and whether it causes any side effects.
Summary
Currently, there is one active clinical trial for Progressive Multifocal Leukoencephalopathy, located in France. This trial represents an important research effort for this rare and serious brain infection, which has limited treatment options. The study focuses on using Pembrolizumab, an immune-boosting medication, in a specific group of patients who have weakened immune systems but do not have HIV. The trial aims to determine whether this approach can help reduce virus levels in the brain and improve patient outcomes. Patients interested in participating should discuss their eligibility with their healthcare provider, as the trial has specific requirements regarding diagnosis timing, virus presence, and immune system status.
