Recruiting

Observational Study

Sponsor:

National Institute of Neurological Disorders and Stroke (NINDS)

Code:

NCT01730131

Conditions

Progressive Multifocal Leukoencephalopathy

Eligibility Criteria

Sex: All

Age: 2 - 70+

Healthy Volunteers: Accepted

Study Details

Brief 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.

Conditions

Progressive Multifocal Leukoencephalopathy

Study ID

NCT01730131

Start date

Nov 8, 2012

Status verified date

Aug 4, 2026

Primary completion date

Jan 1, 2052

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 2 - 70+

Healthy Volunteers: Accepted

  • PML Patients

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 legally authorized representative (LAR)
5. Unwillingness to consent for collection of biological samples or their cryopreservation

Control Patients at Risk for PML

INCLUSION CRITERIA:

1. Able to participate in the studies and follow-up required by the protocol
2. At least 2 years old
3. 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)

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 legally authorized representative (LAR)
5. Unwillingness to consent for collection of biological samples or their cryopreservation

Healthy Volunteers

INCLUSION CRITERIA:

1. Able to participate in the studies and follow-up required by the protocol
2. At least 18 years old

EXCLUSION CRITERIA:

1. Impaired immune function (i.e.-history of hematological malignancy, chronic inflammatory disease, history of treatment with immune suppressive or immunomodulatory therapies, primary or acquired immunodeficiency syndromes)
2. Significant condition, which in the judgment of the principal investigator, would make participation in the diagnostic and research parts of evaluation impossible or risky
3. 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)
4. Pregnancy
5. Inability to provide informed consent, either directly or via legally authorized representative (LAR)
6. Unwillingness to consent for collection of biological samples or their cryopreservation

Study Design

Enrollment

700 participants

Anticipated

Interventions and Outcome Measures

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

Primary outcome measure

  • To characterize the baseline presentation of patients with JCV infection and/or PML with regard to clinical, radiological, immunological, genetic and viral features. [ Time Frame: one year following last enrollment ]

Central Contacts and Locations

Central contacts

Mauricio A Campillay

(301) 496-3825mauricio.campillay@nih.gov

Irene CM Cortese, M.D.

(301) 496-1801corteseir@ninds.nih.gov

Locations

National Institutes of Health Clinical Center

Recruiting

Bethesda, Maryland, United States, 20892

Contacts

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

800-411-1222ccopr@nih.gov

More Information

Sponsor

National Institute of Neurological Disorders and Stroke (NINDS)

Last update posted

Aug 31, 2026

Last verified

Aug 4, 2026

Keywords

  • Encephalitis
  • Immune Reconstitution Syndrome
  • Human Immunodeficiency Virus
  • Multiple Sclerosis
  • Natural History

Trial information was received from ClinicalTrials.gov and was last updated on 2026-10-23. This information was provided to ClinicalTrials.gov by National Institute of Neurological Disorders and Stroke (NINDS) on 2026-08-31. Recruitment status is synced daily from ClinicalTrials.gov and may not reflect the sponsor's current status. Confirm during your call.