Understanding the natural history of Progressive Multifocal Leukoencephalopathy

Natural History Study of Progressive Multifocal Leukoencephalopathy (PML)

This study looks at how Progressive Multifocal Leukoencephalopathy (PML) affects people with weakened immune systems to better understand the disease and its risk factors.

Quick facts

• Study type: Observational
• Enrollment: 700 (estimated)
• Ages: 2 Years to 120 Years
• Sex: All
• Sponsor: National Institutes of Health Clinical Center (CC) (nih)
• Locations: 1 site (Bethesda, Maryland)
• Trial ID: NCT01730131 on ClinicalTrials.gov

What this trial studies

This observational study aims to investigate the natural history and risk factors associated with Progressive Multifocal Leukoencephalopathy (PML), a severe brain infection caused by the JC virus in individuals with compromised immune systems. Participants, aged 2 years and older, will undergo a series of assessments including physical exams, medical history reviews, and imaging studies at the National Institutes of Health Clinical Center. The study will collect clinical data, blood, cerebrospinal fluid, and urine samples over multiple visits to better understand the disease's progression and the immune response to the JC virus. Additionally, a control cohort will be included to compare findings with those at risk for PML.

Who should consider this trial

Good fit: Ideal candidates include individuals aged 2 years and older with suspected or confirmed PML.

Not a fit: Patients with significant medical conditions that would prevent safe participation or those unable to provide informed consent may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide critical insights into the progression and immune response to PML, potentially guiding future treatment strategies.

How similar studies have performed: While studies on PML exist, this specific observational approach focusing on the natural history of the disease is relatively novel.

Eligibility criteria

* 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

Where this trial is running

Bethesda, Maryland

• National Institutes of Health Clinical Center — Bethesda, Maryland, United States (RECRUITING)

Study contacts

• Principal investigator: Irene CM Cortese, M.D. — National Institute of Neurological Disorders and Stroke (NINDS)
• Study coordinator: Mauricio A Campillay

Conditions: Progressive Multifocal Leukoencephalopathy, Encephalitis, Immune Reconstitution Syndrome, Human Immunodeficiency Virus, Multiple Sclerosis, Natural History