A Randomized, Rater-Blinded Study to Explore the Effect of Mefloquine in Subjects With Progressive Multifocal Leukoencephalopathy (PML)
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 发起方
- Biogen
- 入组人数
- 37
- 试验地点
- 1
- 主要终点
- Change From Baseline to Week 4 in JC Virus (JCV) Load in Cerebrospinal Fluid (CSF)
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Single (Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of PML confirmed by detection of JCV DNA in CSF.
- •Onset of PML symptoms within 6 months prior to study.
排除标准
- •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.
研究组 & 干预措施
Local standard of care plus mefloquine 250 mg
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.
干预措施: mefloquine (Drug)
结局指标
主要结局
Change From Baseline to Week 4 in JC Virus (JCV) Load in Cerebrospinal Fluid (CSF)
时间窗: Day 0 (baseline), Week 4
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
Change From Baseline to Week 8 in JC Virus (JCV) Load in Cerebrospinal Fluid (CSF)
时间窗: Day 0 (baseline), Week 8
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
次要结局
- Change From Baseline to Week 4 and Week 8 in the Expanded Disability Status Scale (EDSS) Score(Day 0 (baseline), Week 4 and 8)
- Change From Baseline to Week 4 and Week 8 in Karnofsky Performance Status (KPS) Index Score(Day 0 (baseline), Week 4, Week 8)
- Change From Baseline to Week 4 and Week 8 in Symbol Digit Modalities Test (SDMT)(Day 0 (baseline), Week 4, Week 8)
- Change From Baseline to Week 4 and Week 8 in Participants' Neurological Function Using a Visual Analog Scale (VAS)(Day 0 (baseline), Week 4, Week 8)
- Participants With Gadolinium (Gd)-Enhanced Lesions at Baseline, Week 4 and Week 8 as Seen on Magnetic Resonance Imaging (MRI) Scans of Participants' Brains(Day 0 (baseline), Week 4, Week 8)
- Change From Baseline to Week 4 and Week 8 in T1 Lesion Volume as Seen on Magnetic Resonance Imaging (MRI) Scans of Participants' Brains(Day 0 (baseline), Week 4, Week 8)
- Change From Baseline to Week 4 and Week 8 in T2 Lesion Volume as Seen on Magnetic Resonance Imaging (MRI) Scans of Participants' Brains(Day 0 (baseline), Week 4, Week 8)
- Participants Who Died Within 6 Months(Day 1 up to 6 months)