Trial · NCT00746941
Study to Explore the Effect of Mefloquine in Participants With Progressive Multifocal Leukoencephalopathy (PML)
NCT00746941, Phase 1, Phase 2, Terminated, Biogen; results first posted 2014-07-31
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study to Explore the Effect of Mefloquine in Participants With Progressive Multifocal Leukoencephalopathy (PML). Trials, record NCT00746941. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00746941
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study to Explore the Effect of Mefloquine in Participants With Progressive Multifocal Leukoencephalopathy (PML)
- NCT number
- NCT00746941
- Status
- Terminated
- Phases
- Phase 1, Phase 2
- Enrollment
- 37 participants, actual
- Lead sponsor
- Biogen
- Conditions
- Progressive Multifocal Leukoencephalopathy
- Interventions
- mefloquine
- First posted
- 2008-09-04
- Last updated
- 2014-07-31
- Results first posted
- 2014-07-31
- Limitations and caveats
From ongoing analysis, it became clear, based on conditional power calculations, that the study would not reach its primary endpoint, a decrease of JC viral load in CSF with mefloquine treatment; therefore, the study was terminated early.
Source ClinicalTrials.gov
Registered outcomes10
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline to Week 4 in JC Virus (JCV) Load in Cerebrospinal Fluid (CSF) | 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 |
| Primary | Change From Baseline to Week 8 in JC Virus (JCV) Load in Cerebrospinal Fluid (CSF) | 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 |
| Secondary | Change From Baseline to Week 4 and Week 8 in the Expanded Disability Status Scale (EDSS) Score | Day 0 (baseline), Week 4 and 8 | EDSS assesses disability in 8 functional systems. An overall score ranging from 0 (normal) to 10 (death) was calculated. Negative change scores indicate improvement. |
| Secondary | Change From Baseline to Week 4 and Week 8 in Karnofsky Performance Status (KPS) Index Score | Day 0 (baseline), Week 4, Week 8 | The KPS Index classifies participants' functional impairment. KPS can be used to compare effectiveness of different therapies and to assess the prognosis in individual participants. KPS was recorded on an 11-point scale (0, 10, 20, 30, 40, 50, 60, 70, 80, 90, and 100.) where '0=Dead' and '100=Normal, no complaints, no evidence of disease'. The lower the KPS score, the worse the survival for most serious illnesses. The KPS index is subdivided into 3 categories: incapacitated (0 to 40), self-care (50 to 70), and normal activity (80 to 100). Negative change from baseline scores indicate improved prognosis. |
| Secondary | Change From Baseline to Week 4 and Week 8 in Symbol Digit Modalities Test (SDMT) | Day 0 (baseline), Week 4, Week 8 | The SDMT is a simple substitution task. The test gives participants 90 seconds to pair specific numbers with given geometric figures as a measure for screening cognitive impairment. The total score is the total number of correctly completed boxes in the time allowed. The test score range is from 0 (worst outcome) to 110 (best outcome). Negative change from baseline scores indicates a worsening outcome. |
| Secondary | 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 rate their neurological function on a scale of 100 mm line, where the 0 end of the scale indicates poor neurological function and 100 indicates excellent neurological function. VAS was not required for participants who had physical or cognitive impairments that limited their ability to perform the assessment. Negative change from baseline scores indicates a worsening outcome. |
| Secondary | 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 | |
| Secondary | 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 | |
| Secondary | 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 | |
| Secondary | Participants Who Died Within 6 Months | Day 1 up to 6 months | The death event is counted under the treatment arm relative to adding mefloquine to the treatment regimen. |
Source ClinicalTrials.gov
Outcomes10
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change From Baseline to Week 4 in JC Virus (JCV) Load in Cerebrospinal Fluid (CSF) | Day 0 (baseline), Week 4 | Local standard of care participants who added mefloquine at Week 4 or Week 8 were counted as being dosed under both treatment arms.~Participants with undetectable CSF JCV load at baseline were not included in the efficacy analysis. All other enrolled participants were included in the efficacy analysis if values for Week 4 were available. | log10 copies/mL | MEAN |
| PRIMARY | Change From Baseline to Week 8 in JC Virus (JCV) Load in Cerebrospinal Fluid (CSF) | Day 0 (baseline), Week 8 | Local standard of care participants who added mefloquine at Week 4 or Week 8 were counted as being dosed under both treatment arms.~Participants with undetectable CSF JCV load at baseline were not included in the efficacy analysis. All other enrolled participants were included in the efficacy analysis if values for Week 8 were available. | log10 copies/mL | MEAN |
| SECONDARY | Change From Baseline to Week 4 and Week 8 in the Expanded Disability Status Scale (EDSS) Score | Day 0 (baseline), Week 4 and 8 | Participants with values at the time frames being measured. EDSS was not required for participants who had physical or cognitive impairments that limited their ability to perform the assessment.~Local standard of care participants who added mefloquine at Week 4 or Week 8 were counted as being dosed under both treatment arms. | units on a scale | MEAN |
| SECONDARY | Change From Baseline to Week 4 and Week 8 in Karnofsky Performance Status (KPS) Index Score | Day 0 (baseline), Week 4, Week 8 | Participants with values at the time frames being measured. Participants with undetectable CSF JCV load at baseline by the central laboratory were not included in the efficacy analysis.~Local standard of care participants who added mefloquine at Week 4 or Week 8 were counted as being dosed under both treatment arms. | units on a scale | MEAN |
| SECONDARY | Change From Baseline to Week 4 and Week 8 in Symbol Digit Modalities Test (SDMT) | Day 0 (baseline), Week 4, Week 8 | Participants with values at the time frames being measured. SDMT was not required for participants who had physical or cognitive impairments that limited their ability to perform the assessment.~Local standard of care participants who added mefloquine at Week 4 or Week 8 were counted as being dosed under both treatment arms. | units on a scale | MEAN |
| SECONDARY | 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 values at the time frames being measured. VAS was not required for participants who had physical or cognitive impairments that limited their ability to perform the assessment.~Local standard of care participants who added mefloquine at Week 4 or Week 8 were counted as being dosed under both treatment arms. | units on a scale | MEAN |
| SECONDARY | 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 | Participants with values at the time frames being measured. Participants with undetectable CSF JCV load at baseline by the central laboratory were not included in the efficacy analysis.~Local standard of care participants who added mefloquine at Week 4 or Week 8 were counted as being dosed under both treatment arms. | participants | NUMBER |
| SECONDARY | 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 | Participants with values at the time frames being measured. Participants with undetectable CSF JCV load at baseline by the central laboratory were not included in the efficacy analysis.~Local standard of care participants who added mefloquine at Week 4 or Week 8 were counted as being dosed under both treatment arms. | log10 mm^3 | MEAN |
| SECONDARY | 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 with values at the time frames being measured. Participants with undetectable CSF JCV load at baseline by the central laboratory were not included in the efficacy analysis.~Local standard of care participants who added mefloquine at Week 4 or Week 8 were counted as being dosed under both treatment arms. | log10 mm^3 | MEAN |
| SECONDARY | Participants Who Died Within 6 Months | Day 1 up to 6 months | Safety population: All participants who enrolled in the study and were dosed, and who have at least 1 post-baseline safety assessment.~The death event is counted under the treatment arm relative to adding mefloquine to the treatment regimen. | participants | NUMBER |
Source ClinicalTrials.gov
Results by group36
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Baseline to Week 4 in JC Virus (JCV) Load in Cerebrospinal Fluid (CSF) | Local Standard of Care | -0.2424 | Standard Deviation 0.83556 | log10 copies/mL | |
| Change From Baseline to Week 4 in JC Virus (JCV) Load in Cerebrospinal Fluid (CSF) | Local Standard of Care Plus Mefloquine 250 mg | -0.0675 | Standard Deviation 1.52685 | log10 copies/mL | |
| Change From Baseline to Week 8 in JC Virus (JCV) Load in Cerebrospinal Fluid (CSF) | Local Standard of Care | -0.2843 | Standard Deviation 0.68666 | log10 copies/mL | |
| Change From Baseline to Week 8 in JC Virus (JCV) Load in Cerebrospinal Fluid (CSF) | Local Standard of Care Plus Mefloquine 250 mg | -0.3455 | Standard Deviation 0.93960 | log10 copies/mL | |
| Change From Baseline to Week 4 and Week 8 in the Expanded Disability Status Scale (EDSS) Score | Local Standard of Care | Week 4 (n=12, 21) | 0.79 | Standard Deviation 1.054 | units on a scale |
| Change From Baseline to Week 4 and Week 8 in the Expanded Disability Status Scale (EDSS) Score | Local Standard of Care Plus Mefloquine 250 mg | Week 4 (n=12, 21) | 0.50 | Standard Deviation 1.129 | units on a scale |
| Change From Baseline to Week 4 and Week 8 in the Expanded Disability Status Scale (EDSS) Score | Local Standard of Care | Week 8 (n=7, 16) | 0.71 | Standard Deviation 1.868 | units on a scale |
| Change From Baseline to Week 4 and Week 8 in the Expanded Disability Status Scale (EDSS) Score | Local Standard of Care Plus Mefloquine 250 mg | Week 8 (n=7, 16) | 0.88 | Standard Deviation 1.360 | units on a scale |
| Change From Baseline to Week 4 and Week 8 in Karnofsky Performance Status (KPS) Index Score | Local Standard of Care | Week 4 (n=12, 21) | 0.00 | Standard Deviation 14.771 | units on a scale |
| Change From Baseline to Week 4 and Week 8 in Karnofsky Performance Status (KPS) Index Score | Local Standard of Care Plus Mefloquine 250 mg | Week 4 (n=12, 21) | -8.10 | Standard Deviation 12.891 | units on a scale |
1 to 10 of 36Source ClinicalTrials.gov
See all 36 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Local Standard of Care | 7 | |
| Overall Study | STARTED | Local Standard of Care; Mefloquine at Week 4 | 5 | |
| Overall Study | STARTED | Local Standard of Care; Mefloquine at Week 8 | 5 | |
| Overall Study | STARTED | Local Standard of Care Plus Mefloquine 250 mg | 20 | |
| Overall Study | COMPLETED | Local Standard of Care | 0 | |
| Overall Study | COMPLETED | Local Standard of Care; Mefloquine at Week 4 | 1 | |
| Overall Study | COMPLETED | Local Standard of Care; Mefloquine at Week 8 | 3 | |
| Overall Study | COMPLETED | Local Standard of Care Plus Mefloquine 250 mg | 7 | |
| Overall Study | NOT COMPLETED | Local Standard of Care | 7 | |
| Overall Study | NOT COMPLETED | Local Standard of Care; Mefloquine at Week 4 | 4 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events1,020
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Headache | Nervous system disorders | Local Standard of Care | 0 | 17 | |
| other | Headache | Nervous system disorders | Local Standard of Care; Mefloquine at Week 4 | 2 | 5 | |
| other | Headache | Nervous system disorders | Local Standard of Care; Mefloquine at Week 8 | 1 | 5 | |
| other | Headache | Nervous system disorders | Local Standard of Care Plus Mefloquine 250 mg | 4 | 20 | |
| other | Ataxia | Nervous system disorders | Local Standard of Care | 0 | 17 | |
| other | Ataxia | Nervous system disorders | Local Standard of Care; Mefloquine at Week 4 | 0 | 5 | |
| other | Ataxia | Nervous system disorders | Local Standard of Care; Mefloquine at Week 8 | 0 | 5 | |
| other | Ataxia | Nervous system disorders | Local Standard of Care Plus Mefloquine 250 mg | 2 | 20 | |
| other | Epilepsy | Nervous system disorders | Local Standard of Care | 0 | 17 | |
| other | Epilepsy | Nervous system disorders | Local Standard of Care; Mefloquine at Week 4 | 0 | 5 |
1 to 10 of 1,020Source ClinicalTrials.gov
See all 1,020 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Local Standard of Care | ||
| Total, all-cause mortality | Local Standard of Care; Mefloquine at Week 4 | ||
| Total, all-cause mortality | Local Standard of Care; Mefloquine at Week 8 | ||
| Total, all-cause mortality | Local Standard of Care Plus Mefloquine 250 mg | ||
| Total, other adverse events | Local Standard of Care | 12 | 17 |
| Total, other adverse events | Local Standard of Care; Mefloquine at Week 4 | 4 | 5 |
| Total, other adverse events | Local Standard of Care; Mefloquine at Week 8 | 5 | 5 |
| Total, other adverse events | Local Standard of Care Plus Mefloquine 250 mg | 19 | 20 |
| Total, serious adverse events | Local Standard of Care | 5 | 17 |
| Total, serious adverse events | Local Standard of Care; Mefloquine at Week 4 | 1 | 5 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 23733308 | RESULT |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons1
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison |
|---|
| Local Standard of Care Plus Mefloquine 250 mg vs Local Standard of Care: Participants Who Died Within 6 Months |
Source Trial comparisons
Tests1
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
| Test | Named as |
|---|---|
| Symbol Coding | Secondary outcome |
Source ConductCognition
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT00746941
GET https://conductscience.org/api/trials/v1/NCT00746941/results?page=2