Study to Explore the Effect of Mefloquine in Participants With Progressive Multifocal Leukoencephalopathy (PML)

Phase 2TerminatedNCT00746941

Biogen37 enrolled

Overview

The primary objective of the study was to explore whether mefloquine can delay or stop progression of progressive multifocal leukoencephalopathy (PML) as measured by JC virus (human polyomavirus or JCV) deoxyribonucleic acid (DNA) levels in cerebrospinal fluid (CSF). The secondary objective of the study was to explore whether mefloquine can delay or stop progression of PML based on neurological deterioration, magnetic resonance imaging (MRI) measures of brain lesion evolution or the formation of new lesions, and mortality.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

SINGLE

Enrollment

Conditions

Progressive Multifocal Leukoencephalopathy

Interventions

mefloquineDRUG

250 mg orally each day for 3 days and then weekly up to 6 months.

Eligibility

Sex: ALLMin age: 18 YearsMax age: 75 Years

Medical Language ↔ Plain English

Key Inclusion Criteria: * Diagnosis of PML confirmed by detection of JCV DNA in CSF. * Onset of PML symptoms within 6 months prior to study. Key Exclusion Criteria: * Other opportunistic infection of the central nervous system. * Current severe illness or any other conditions that, in the opinion of the Investigator, would make the subject unsuitable for enrollment. * Active severe mental illness (e.g., depression, anxiety, psychosis, and schizophrenia). * Hypersensitivity to mefloquine, quinine, or quinidine, or to any component of these drugs. * Current treatment with quinine, quinidine, chloroquine, or halofantrine. Note: Other protocol-defined criteria may also apply.

Locations (12)

Research Site

Chicago, Illinois, United States

Research Site

Baltimore, Maryland, United States

Research Site

Boston, Massachusetts, United States

Research Site

Outcomes

Primary Outcomes

Change From Baseline to Week 4 in JC Virus (JCV) Load in Cerebrospinal Fluid (CSF)

Change from baseline to Week 4 in JC viral load in CSF is expressed as log10 copies/mL. Negative values indicate a reduction in viral load. Only participants with measurable baseline values are included. Post-baseline values of 'Below the Limit of Quantification' or 'Below Limit of Detection' or 'Negative' were set to 50. Log10 (50) = 1.699

Time frame: Day 0 (baseline), Week 4

Change From Baseline to Week 8 in JC Virus (JCV) Load in Cerebrospinal Fluid (CSF)

Change from baseline to Week 8 in JC viral load in CSF is expressed as log10 copies/mL. Negative values indicate a reduction in viral load. Only participants with measurable baseline values are included. Post-baseline values of 'Below the Limit of Quantification' or 'Below Limit of Detection' or 'Negative' were set to 50. Log10 (50) = 1.699

Time frame: Day 0 (baseline), Week 8

Secondary Outcomes

Change From Baseline to Week 4 and Week 8 in the Expanded Disability Status Scale (EDSS) Score

EDSS assesses disability in 8 functional systems. An overall score ranging from 0 (normal) to 10 (death) was calculated. Negative change scores indicate improvement.

Time frame: Day 0 (baseline), Week 4 and 8

Change From Baseline to Week 4 and Week 8 in Karnofsky Performance Status (KPS) Index Score

The KPS Index classifies participants' functional impairment. KPS can be used to compare effectiveness of different therapies and to assess the prognosis in individual participants. KPS was recorded on an 11-point scale (0, 10, 20, 30, 40, 50, 60, 70, 80, 90, and 100.) where '0=Dead' and '100=Normal, no complaints, no evidence of disease'. The lower the KPS score, the worse the survival for most serious illnesses. The KPS index is subdivided into 3 categories: incapacitated (0 to 40), self-care (50 to 70), and normal activity (80 to 100). Negative change from baseline scores indicate improved prognosis.

Data from ClinicalTrials.gov

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