Natural History Study of Progressive Multifocal Leukoencephalopathy (PML)
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age2-120
SponsorNational Institute of Neurological Disorders and Stroke (NINDS)
About this trial
Background:
\- Progressive multifocal leukoencephalopathy (PML) is a severe viral infection of the brain. It is caused by JC virus. Many people have this virus in their bodies all their life, but it is usually kept in check by their immune system. If the immune system does not work right because of a disease or medication, the virus becomes active and can damage cells in the brain. Not much is known about PML or how it affects the immune system. Researchers want to study people with PML to better understand the natural history of the disease.
Objectives:
\- To study the natural history of PML.
Eligibility:
\- Individuals at least 2 years of age who have PML.
Design:
* Participants will be screened with a physical exam, medical history, and imaging studies. * Participants will have several visits to the National Institutes of Health Clinical Center. There will be an initial visit, monthly visits for the next 6 months, a 12-month visit, and possible visits afterward. * At the initial visit, participants will give blood, urine, and spinal fluid samples. They will also have neurological tests and imaging studies of the brain. * For the next five visits, participants will give blood and urine samples. They will also have neurological tests and imaging studies of the brain. * The 6-month and 12-month visits will repeat the tests from the initial visit. * Other optional procedures include bone marrow samples and skin biopsies. Additional blood tests and imaging studies may be performed. * Treatment will not be provided as part of this study.
Eligibility criteria
This trial accepts healthy volunteersQualifiers
PML Patients
Suspected or confirmed PML
MRI compatible with PML
Able to participate in the studies and follow-up required by the protocol
Disqualifiers
Significant condition, which in the judgment of the principal investigator, would make participation in the diagnostic and research parts of evaluation impossible or risky
Medical contraindication to MRI (i.e., devices such as a cardiac pacemaker or infusion pump, other metallic implants, metallic foreign objects, body piercings that cannot be removed)
Pregnancy
Inability to provide informed consent, either directly or via legally authorized representative (LAR)
Trial population
Primary clinical for participants that have PML or at risk for PML and community sample/healthy relatives for healthy volunteers.
Trial design
Design model
Cohort
Time perspective
Prospective
Treatments tested in this trial
Not listed
Trial groups
700 Participants
are grouped into 3 trial groupsGroup A: Control Patients at Risk for PML
Group B: Healthy Volunteers
Group C: PML Patients
Trial outcomes
Primary outcomes
1
To characterize the baseline presentation of patients with JCV infection and/or PML with regard to clinical, radiological, immunological, genetic and viral features.
1\. Characterization of distinctive imaging features to differentiate between actively progressing PML, PML-IRIS/inflammatory PML and inactive PML2. Characterization of distinctive clinical features to differentiate between actively progressing PML, PML-IRIS/inflammatory PML, and inactive PML3. Characterization of immune and virological features to differentiate between actively progressing PML, PML-IRIS/inflammatory PML, and inactive PML4. Characterization of immune and virological differences across PML patient populations with different underlying disease, subjects at risk for developing PML, and healthy individuals5. Characterization of genetic susceptibility for development of PML
Time frame
one year following last enrollmentSecondary outcomes
1
To longitudinally follow patients with JCV infection and/or PML with thorough characterization of their clinical course, imaging correlates, immunological markers, and viral studies
1\. Temporal correlation between clinical course and imaging and/or laboratory measures2. To identify biomarkers that can predict long-term outcome and response to treatment interventions3. To develop a clinically relevant assessment scale for PML that identifies milestones of disease progression4. To develop a repository of cryopreserved biological samples that will be used for validation of candidate biomarkers in future studies5. To characterize the immune profile in blood and CSF of patients with PML6. To determine the eligibility of PML patients for participation in other studies7. To screen and evaluate healthy subjects as donors for manufacture of virus-specific T cells (to be administered to patients under separate, dedicated protocol); to collect PBMC for the purpose of manufacture of virus-specific T cells, which will be stored in a bank and can be used for any appropriately HLA-matched recipient
Time frame
one year following last enrollmentSponsors and contacts
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Source ClinicalTrials.gov