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

Summary

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.

Timeline

Start

2009-01

Primary completion

2010-11

Completion

2010-11

Outcome

Missed primary endpoint

Stopped: “Primary endpoint not achieved”

registry analysis (superiority or other test); Local Standard of Care vs Local Standard of Care Plus Mefloquine 250 mg; p = 0.7132; Student's t-test NCT00746941 ↗

registry analysis (superiority or other test); Local Standard of Care vs Local Standard of Care Plus Mefloquine 250 mg; p = 0.9086; Student's t-test NCT00746941 ↗

paper There was no significant difference between groups in CSF JCV DNA loads or clinical/MRI findings. PMID 23733308 ↗

Publications

• Results Clifford DB, Nath A, Cinque P, Brew BJ, Zivadinov R, Gorelik L, Zhao Z, Duda P. A study of mefloquine treatment for progressive multifocal leukoencephalopathy: results and exploration of predictors of PML outcomes. J Neurovirol. 2013 Aug;19(4):351-8. doi: 10.1007/s13365-013-0173-y. Epub 2013 Jun 4.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Mefloquine	Unknown	250 mg	Oral

Indications