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

This study has been terminated.
Elan Pharmaceuticals
Information provided by:
Biogen Idec Identifier:
First received: September 3, 2008
Last updated: June 7, 2012
Last verified: December 2011

The purpose of the study is to explore if mefloquine works to slow or stop the worsening of PML and to better understand PML. We will measure if mefloquine is working by determining if it reduces the level of JC virus in spinal fluid, as well as, clinical and MRI measures.

Condition Intervention Phase
Progressive Multifocal Leukoencephalopathy
Drug: mefloquine
Phase 1
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Single Blind (Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Randomized, Rater-Blinded Study to Explore the Effect of Mefloquine in Subjects With Progressive Multifocal Leukoencephalopathy(PML)

Resource links provided by NLM:

Further study details as provided by Biogen Idec:

Primary Outcome Measures:
  • Change in JCV DNA levels in CSF from baseline [ Time Frame: 4 weeks ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Change in Karnofsky Performance Scale Index (KPSI) from baseline [ Time Frame: 8 weeks ] [ Designated as safety issue: No ]
  • Change in Expanded Disability Status Scale (EDSS) from baseline [ Time Frame: 8 weeks ] [ Designated as safety issue: No ]
  • Change in Symbol Digit Modalities Test (SDMT) from baseline [ Time Frame: 8 weeks ] [ Designated as safety issue: No ]
  • Change in Visual Analogue Scale (VAS) from baseline [ Time Frame: 8 weeks ] [ Designated as safety issue: No ]
  • Number of gadolinium-enhancing lesions [ Time Frame: 8 weeks ] [ Designated as safety issue: No ]
  • Change in volume of T1 hypointense lesions from baseline [ Time Frame: 8 weeks ] [ Designated as safety issue: No ]
  • Change in volume of T2 hyperintense lesions from baseline [ Time Frame: 8 weeks ] [ Designated as safety issue: No ]
  • Assessment of mortality [ Time Frame: 6 months ] [ Designated as safety issue: No ]

Enrollment: 37
Study Start Date: September 2008
Primary Completion Date: November 2010 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
No Intervention: A
Standard of Care
Active Comparator: B
Standard of Care plus Mefloquine
Drug: mefloquine
250 mg orally each day for 3 days and then weekly up to 6 months.
Other Name: Lariam®


Ages Eligible for Study:   18 Years to 75 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Diagnosis of PML confirmed by detection of JC viral DNA in spinal fluid.
  • Onset of PML symptoms within 6 months prior to study.

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.

Other protocol-defined criteria may also apply.

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00746941

  Hide Study Locations
United States, California
Research Site
Los Angeles, California, United States, 90059
Research Site
San Diego, California, United States, 92103
Research Site
San Francisco, California, United States, 94110
United States, Colorado
Research Site
Denver, Colorado, United States, 80262
United States, District of Columbia
Research Site
Washington, District of Columbia, United States, 20007
United States, Illinois
Research Site
Chicago, Illinois, United States, 60611
United States, Kentucky
Research Site
Lexington, Kentucky, United States, 40536
United States, Maryland
Research Site
Baltimore, Maryland, United States, 21205
United States, Massachusetts
Research Site
Boston, Massachusetts, United States, 2215
United States, Missouri
Research Site
St. Louis, Missouri, United States, 63110
United States, New York
Research Site
New York, New York, United States, 10029
Australia, New South Wales
Research Site
Sydney, New South Wales, Australia, 2010
Australia, Victoria
Research Site
Melbourne, Victoria, Australia, 3181
Research Site
Rio, Brazil, 21040-900
Research Site
Sao Paulo, Brazil, 01246-900
Research Site
Le Kremlin-Bicêtre, France, 94275
Research Site
Dusseldorf, North Rhine-Westphalia, Germany, 40225
Research Site
Muenster, North Rhine-Westphalia, Germany, 48129
Research Site
Berlin, Germany, 12157
Research Site
Hamburg, Germany, 22807
Research Site
Milano, Italy, 20132
Research Site
Rome, Italy, 00168
Puerto Rico
Research Site
San Juan, Puerto Rico, 936
Research Site
Barcelona, Spain, 8036
Research Site
Madrid, Spain, 28007
United Kingdom
Research Site
London, United Kingdom, W21NY
Research Site
London, United Kingdom, E12AT
Research Site
Manchester, United Kingdom, M85RB
Sponsors and Collaborators
Biogen Idec
Elan Pharmaceuticals
  More Information

No publications provided

Responsible Party: Biogen Idec Identifier: NCT00746941     History of Changes
Other Study ID Numbers: 111JC101
Study First Received: September 3, 2008
Last Updated: June 7, 2012
Health Authority: Brazil: National Health Surveillance Agency
United States: Institutional Review Board
Australia: Department of Health and Ageing Therapeutic Goods Administration
United Kingdom: Medicines and Healthcare Products Regulatory Agency
Italy: Ministry of Health
Spain: Spanish Agency of Medicines
Germany: Federal Institute for Drugs and Medical Devices
United States: Food and Drug Administration

Keywords provided by Biogen Idec:
Human Polyomavirus JC
Central Nervous System Disease
JC Virus

Additional relevant MeSH terms:
Leukoencephalopathy, Progressive Multifocal
Encephalitis, Viral
Central Nervous System Viral Diseases
Virus Diseases
Polyomavirus Infections
DNA Virus Infections
Slow Virus Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Central Nervous System Infections
Demyelinating Diseases
Antiprotozoal Agents
Antiparasitic Agents
Anti-Infective Agents
Therapeutic Uses
Pharmacologic Actions processed this record on July 22, 2014