NCT01730131

Natural History Study of Progressive Multifocal Leukoencephalopathy (PML)

Recruiting now Last updated 16 April 2026

What this trial tests

trial in Progressive Multifocal Leukoencephalopathy in 700 participants. Currently enrolling.

Timeline

8 November 2012

Primary endpoint
1 January 2052

Quick facts

Lead sponsor	National Institute of Neurological Disorders and Stroke (NINDS)
Status	Recruiting now
Study type	OBSERVATIONAL
Enrollment	700
Start date	8 November 2012
Primary completion	1 January 2052
Sites	1 location across United States

Conditions studied

• Progressive Multifocal Leukoencephalopathy — all drugs for Progressive Multifocal Leukoencephalopathy →

Sponsor

National Institute of Neurological Disorders and Stroke (NINDS)

Who can join

Adults 2 to 120, any sex, with Progressive Multifocal Leukoencephalopathy. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

6 peer-reviewed publications reference this trial (live from Europe PMC):

1. Progressive Multifocal Leukoencephalopathy: Pathogenesis, Diagnostic Tools, and Potential Biomarkers of Response to Therapy.
   Schweitzer F, Laurent S, Cortese I, Fink GR, et al · · 2023 · cited 32× · PMID 37487750 · DOI 10.1212/wnl.0000000000207622
2. Characteristics of Progressive Multifocal Leukoencephalopathy Associated With Sarcoidosis Without Therapeutic Immune Suppression.
   McEntire CRS, Fletcher A, Toledano M, Epstein S, et al · · 2023 · cited 11× · PMID 37093609 · DOI 10.1001/jamaneurol.2023.0841
3. Biomarkers for progressive multifocal leukoencephalopathy: emerging data for use of JC virus DNA copy number in clinical trials.
   Cortese I, Norato G, Harrington PR, Usher T, et al · · 2024 · cited 10× · PMID 38631769 · DOI 10.1016/s1474-4422(24)00099-1
4. Progressive multifocal leukoencephalopathy lesion and brain parenchymal segmentation from MRI using serial deep convolutional neural networks.
   Al-Louzi O, Roy S, Osuorah I, Parvathaneni P, et al · · 2020 · cited 9× · PMID 33395989 · DOI 10.1016/j.nicl.2020.102499
5. Portable ultra-low-field MRI for progressive multifocal leukoencephalopathy: Case studies, sensitivity, and potential applications.
   Okar SV, Kawatra KD, Thommana AA, Vultorius DC, et al · · 2025 · cited 4× · PMID 39932567 · DOI 10.1007/s00415-025-12938-z
6. Cortical lesions are common in a longitudinal progressive multifocal leukoencephalopathy cohort.
   Walvekar S, Nistri R, Al-Louzi O, Norato G, et al · · 2025 · PMID 41089375 · DOI 10.1093/braincomms/fcaf373