Natural History Study of Progressive Multifocal Leukoencephalopathy (PML) (NCT01730131) | Clinical Trial Compass
RecruitingNot Applicable
Natural History Study of Progressive Multifocal Leukoencephalopathy (PML)
United States700 participantsStarted 2012-11-08
Plain-language summary
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.
Who can participate
Age range
2 Years – 120 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Suspected or confirmed PML
. MRI compatible with PML
. Able to participate in the studies and follow-up required by the protocol
. At least 2 years old
Exclusion criteria
. 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)
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Since this is a natural history study — meaning it's observational and not testing a treatment — what would my actual participation involve, and would joining this study delay or interfere with any active treatment I might need for PML?
2This study is collecting clinical, imaging, immune system, genetic, and virus-related data about people with JC virus infection and PML — would the detailed testing and monitoring involved give me access to information about my own condition that I might not otherwise get through standard care?
3Because PML is a serious and fast-moving disease, how does my doctor weigh the time and energy required to participate in a research study like this against focusing entirely on my immediate treatment and recovery?
4This trial is still recruiting, which means researchers are still gathering baseline data — does my doctor think there's enough evidence from this or similar studies already to guide my treatment decisions, or are there still big unknowns about how PML progresses that make contributing to this kind of research particularly valuable right now?
5Since the study is tracking genetic and immunological features alongside the virus itself, would any of the genetic or immune testing done as part of this study give my care team useful information about why I developed PML or how my immune system might respond going forward?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
To characterize the baseline presentation of patients with JCV infection and/or PML with regard to clinical, radiological, immunological, genetic and viral features.
Timeframe: one year following last enrollment
Trial details
NCT IDNCT01730131
SponsorNational Institute of Neurological Disorders and Stroke (NINDS)
. Inability to provide informed consent, either directly or via legally authorized representative (LAR)
. Unwillingness to consent for collection of biological samples or their cryopreservation
. Able to participate in the studies and follow-up required by the protocol
. At least 2 years old
. Impaired immune function from any cause and considered at risk for PML (i.e.-history of hematological malignancy, chronic inflammatory disease, history of treatment with immune suppressive or immunomodulatory therapies, primary or acquired immunodeficiency syndromes)