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Natural History Study of Progressive Multifocal Leukoencephalopathy (PML)

Study Purpose

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.

Recruitment Criteria

Accepts Healthy Volunteers Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms	Yes
Study Type An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes. An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes. Searching Both is inclusive of interventional and observational studies.	Observational
Eligible Ages	2 Years and Over
Gender	All

More Inclusion & Exclusion Criteria

-
INCLUSION CRITERIA:
1.

Suspected or confirmed PML. 2. MRI compatible with PML. 3. Able to participate in the studies and follow-up required by the protocol. 4. At least 2 years old.

EXCLUSION CRITERIA:

1. Significant condition, which in the judgment of the principal investigator, would make participation in the diagnostic and research parts of evaluation impossible or risky. 2. 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) 3. Pregnancy. 4. Inability to provide informed consent, either directly or via appointed power of attorney. 5. Unwillingness to consent for collection of biological samples or their cryopreservation

Trial Details

Trial ID: This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.	NCT01730131
Phase Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans. Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data. Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs. Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.
Lead Sponsor The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.	National Institute of Neurological Disorders and Stroke (NINDS)
Principal Investigator The person who is responsible for the scientific and technical direction of the entire clinical study.	Irene CM Cortese, M.D.
Principal Investigator Affiliation	National Institute of Neurological Disorders and Stroke (NINDS)
Agency Class Category of organization(s) involved as sponsor (and collaborator) supporting the trial.	NIH
Overall Status	Recruiting
Countries	United States
Conditions The disease, disorder, syndrome, illness, or injury that is being studied.	Progressive Multifocal Leukoencephalopathy
Study Website:	View Trial Website

Additional Details

The objective of this study is to examine the risk factors and natural course of JCV infection and progressive multifocal leukoencephalopathy (PML). PML is a devastating, demyelinating neurological disease affecting the brain of patients with a compromised immune system. It is caused by reactivation of JC virus (JCV), a small DNA virus that infects the majority of the population without clinical significance. There are currently no treatments available for PML. We plan to study patients with suspected or confirmed PML with different underlying conditions including patients on immune-modulatory therapies for multiple sclerosis (MS), rheumatologic diseases or other autoimmune diseases, as well as patients with HIV infection or other conditions leading to a compromised immune system. Patients will be seen at defined time points during their disease course and detailed assessments will be performed to collect clinical and imaging data. Blood, cerebrospinal fluid (CSF) and urine will also be collected at these time points to evaluate the behavior and biology of the JCV and the patients immune responses to the infection. These tests will lead to a better understanding of the pathophysiology of PML and the course of this disease in different patient groups. Additionally, we will recruit a patient control cohort represented by patients with impaired immune function for any cause and considered at risk for development of PML, and a healthy volunteer cohort. The purpose of these additional cohorts is to explore and validate biomarkers for risk of development of PML and early diagnosis. Additionally, the healthy volunteer cohort may be screened and evaluated as possible donors of peripheral blood mononuclear cells (PBMC) for manufacture of virus-specific T cells. The detailed characterization described above will be used to help identify: 1. Clinical imaging and/or laboratory features pathognomonic of JCV infection and disease course that may aid in earlier diagnosis and appropriate intervention. 2. Clinical imaging and/or laboratory features of the disease course that is predictive of clinical outcomes. This information will be integrated to develop a clinically relevant, disease-specific assessment scale of PML, which is currently not available. Such a scale would be a useful tool for the clinical management of patients (i.e., for development of standards of care), as well as for clinical trial design and interpretation. The long-term objectives of this study are to improve the understanding of the disease course and underlying pathophysiology, to identify subgroups with different prognosis and/or susceptibility to interventions, and to help identify therapeutic targets and/or intervention strategies. Equally important, these efforts will allow development of a repository of cryopreserved biological samples that will be used for validation of candidate biomarkers in future studies; this data and biological bank will be made available to outside laboratories.

Arms & Interventions

Arms

: Control Patients at Risk for PML

Participants with impaired immune function from any cause and considered at risk for PML

: Healthy Volunteers

Healthy volunteers without impaired immune function

: PML Patients

Participants with PML

Interventions

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

Bethesda, Maryland

Status

Recruiting

Address

National Institutes of Health Clinical Center

Bethesda, Maryland, 20892

Site Contact

For more information at the NIH Clinical Center contact Office of Patient Recruitment (OPR)

[email protected]

800-411-1222 #TTY dial 711

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