Progressive Multifocal Leukoencephalopathy Clinical Trial
Official title:
Natural History Study of Progressive Multifocal Leukoencephalopathy (PML)
NCT number | NCT01730131 |
Other study ID # | 130017 |
Secondary ID | 13-N-0017 |
Status | Recruiting |
Phase | |
First received | |
Last updated | |
Start date | November 8, 2012 |
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.
Status | Recruiting |
Enrollment | 700 |
Est. completion date | |
Est. primary completion date | January 1, 2027 |
Accepts healthy volunteers | Accepts Healthy Volunteers |
Gender | All |
Age group | 2 Years to 120 Years |
Eligibility | - 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 |
Country | Name | City | State |
---|---|---|---|
United States | National Institutes of Health Clinical Center | Bethesda | Maryland |
Lead Sponsor | Collaborator |
---|---|
National Institute of Neurological Disorders and Stroke (NINDS) |
United States,
Cinque P, Koralnik IJ, Gerevini S, Miro JM, Price RW. Progressive multifocal leukoencephalopathy in HIV-1 infection. Lancet Infect Dis. 2009 Oct;9(10):625-36. doi: 10.1016/S1473-3099(09)70226-9. — View Citation
Gheuens S, Pierone G, Peeters P, Koralnik IJ. Progressive multifocal leukoencephalopathy in individuals with minimal or occult immunosuppression. J Neurol Neurosurg Psychiatry. 2010 Mar;81(3):247-54. doi: 10.1136/jnnp.2009.187666. Epub 2009 Oct 14. — View Citation
Padgett BL, Walker DL, ZuRhein GM, Eckroade RJ, Dessel BH. Cultivation of papova-like virus from human brain with progressive multifocal leucoencephalopathy. Lancet. 1971 Jun 19;1(7712):1257-60. doi: 10.1016/s0140-6736(71)91777-6. No abstract available. — View Citation
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | To characterize the baseline features, of patients with PML with regards to clinical features imaging studies, immunological markers, and viral studies. | 1. Characterization of distinctive imaging features to differentiate between actively progressing PML, and PML-IRIS and inactive PML 2. Characterization of distinctive clinical features to differentiate between actively progressing PML, PML- and IRIS, and inactive PML 3. Characterization of immune and virological features to differentiate between actively progressing PML, and PML- IRIS, and inactive PML 4. Characterization of immune and virological differences across PML patient populations with different underlying disease, subjects at risk for developing PML, and healthy individuals 5. Characterization of genetic susceptibility for development of PML | one year following last enrollment | |
Secondary | To longitudinally follow patients with 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 measures 2. To identify biomarkers that can predict long-term outcome and response to treatment interventions 3. To develop a clinically relevant assessment scale for PML that identifies milestones of disease progression 4. To develop a repository of cryopreserved biological samples that will be used for validation of candidate biomarkers in future studies 5. To characterize the immune profile in blood and CSF of patients with PML 6. To determine the eligibility of PML patients for participation in other studies 7. To screen and evaluate healthy subjects as donors for manufacture of virus-specific T cells (to be administered to patients under separate, dedicated protocol) | one year following last enrollment |
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