Mefloquine in Subjects with PML Version 2 dated 27 June 2008

• Research type

  Research Study

• Full title

  A Randomized, Rater-Blinded Study to Explore the Effect of Mefloquine in Subjects with Progressive Multifocal Leukoencephalopathy (PML)

• IRAS ID

  10558

• Sponsor organisation

  Biogen Idec

• Eudract number

  2008-001314-24

• ISRCTN Number

  NCT00746941

• Research summary

  Progressive multifocal leukoencephalopathy (PML) is a progressive, demyelinating (condition that results in damage to the protective covering that surrounds nerves in the brain and spinal cord) disease of the central nervous system (CNS) caused by infection with the JC virus (JCV), a human polyomavirus (virus). PML is a rare disease usually associated with immunosuppression. It occurs predominantly in patients who are immunocompromised, including those immunocompromised due to human immunodeficiency virus (HIV), hematological malignancies, organ transplants, anti-neoplastic, or immunosuppressive therapies. Currently, there is no approved or proven effective treatment for PML. Various anti-viral and anti-neoplastic agents have been used to treat PML without clear evidence of efficacy.Mefloquine is being used for prophylaxis and treatment of malaria. The use of mefloquine for the treatment of PML has not been evaluated in humans. This study will establish proof of concept for the effectiveness of mefloquine to reduce JCV load in CSF (Cerebral Spinaflud) in subjects diagnosed with PML. Effectiveness of the treatment will be evaluated by observing the levels of JCV DNA in CSF over time.

• REC name

  London - Fulham Research Ethics Committee

• REC reference

  08/H0712/124

• Date of REC Opinion

  2 Jan 2009

• REC opinion

  Further Information Favourable Opinion