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

Biogen

Status and phase: Terminated; Phase 2; Phase 1

Conditions: Progressive Multifocal Leukoencephalopathy

Treatments: Drug: mefloquine

Study type: Interventional

Funder types: Industry

Identifiers

NCT00746941

111JC101

Details and patient eligibility

About

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.

Enrollment: 37 patients

Sex: All

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Single Blind

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37 participants in 2 patient groups

Local standard of care

No Intervention group

Description:

All participants received local standard of care, which may have included any treatment or procedure that the Investigator would normally use in the treatment of a PML patient at their study site or hospital. Participants in this treatment arm had the option of adding 250 mg mefloquine by mouth at Week 4 (Day 28) or Week 8 (Day 56) daily for 3 days, and then weekly through Week 24.

Local standard of care plus mefloquine 250 mg

Experimental group

Description:

All participants received local standard of care, which may have included any treatment or procedure that the Investigator would normally use in the treatment of a PML patient at their study site or hospital. Participants received 250 mg mefloquine by mouth on Days 0, 1, and 2 and then weekly through Week 24.

Treatment:

Drug: mefloquine