This page provides an independent statistical analysis and educational interpretation of publicly reported results. ClinicalTrials.gov provides the official trial registry record. Numerical trial results on this page are restricted to the results posted in the ClinicalTrials.gov record; calculations added by Clinical Biostats are labelled as such.
1. Trial at a Glance
This page covers the registry results for Effects of Perampanel on Neurophysiology Test Perimeters (NCT03653741), a na phase 4 study. The primary outcome is Change in Baseline Visual Evoked Potential (P100) Amplitude.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT03653741 |
| Phase | Phase 4 |
| Condition | Healthy |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 12 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Weill Medical College of Cornell University |
| Results posted | Yes |
2. Clinical Question
The clinical question is whether neurophysiology test results differ in healthy males after a single oral dose of perampanel. The registry describes testing before dosing and again after the drug, including visual evoked potential (VEP), electroencephalogram (EEG), somatosensory evoked potential (SEP), and brainstem auditory evoked potential (BAEP). A blood draw for serum perampanel level is also described. The study was motivated by the possibility that a drug might affect test measurements used in neurophysiology, rather than by a comparison of treatment strategies for epilepsy.
Population
Healthy males. The planned enrollment was 12 participants, and the registry lists 12 at risk in the safety record.
Intervention
A single oral dose of perampanel 6 MG, followed by repeat neurophysiology tests and a blood draw as described in the study summary.
Comparator
Each participant's pre-dose test results serve as the within-person reference for the post-dose measurements. There was no placebo, randomization, or separate control group.
Primary question
What is the change in baseline visual evoked potential (P100) amplitude after perampanel? The registry record says the data for this primary outcome were not collected, so it reports no estimate of that change.
This is a before-and-after question in a single group, not a randomized comparison of perampanel with placebo or another treatment. The primary endpoint focuses on VEP P100 amplitude, while the study summary also describes EEG, SEP, and BAEP testing. The registry lists seven outcome measures, but the details of the other outcomes are not reported in the registry record. Consequently, the stated scientific question is broader than the primary result available in the record: the protocol description covers several tests, but the primary outcome has no posted data for evaluating a pre-dose to post-dose difference.
3. Trial Design
The registry identifies this as a phase 4, single-group study with no masking. Allocation is listed as NA, and the study summary explicitly says there was no placebo and no randomization. The planned enrollment was 12 healthy male participants. The record lists the study as completed, with a start date of 2018-10-01 and a primary completion date of 2021-11-29. Completion status describes the record's study status; it does not mean that results for the primary endpoint are available.
The study summary describes a sequence in which participants undergo neurophysiology testing before taking perampanel, receive a single oral dose of 6mg, and then repeat the tests. A blood draw for serum perampanel level is described after dosing. The summary says post-dose testing occurs 1 hour after ingestion. The primary outcome timeframe, however, is recorded as pre-dose and post-dose of drug intake, 7 hours. These descriptions do not align, and the registry record does not clarify the actual timing used for the primary endpoint. The recorded timeframe should therefore be read as an unresolved detail rather than reconciled by assumption.
In a single-group before-and-after design, each participant can act as their own reference: the post-dose measurement is compared with that participant's baseline. This can account for stable differences between participants, but it does not create a concurrent control group. Without randomization or a placebo group, changes observed over time cannot be separated as cleanly from other time-related influences, measurement variation, or effects of the testing sequence. This is a general design consideration, not a claim that any particular source of bias occurred in this study.
The registry record does not report a sample-size justification, a detailed statistical analysis plan, or the process used to select the 12 planned participants. It lists no analysis entries. Thus, the record describes the intended study sequence and the primary outcome, but does not document a completed primary analysis.
4. Participant Flow and Accounting
The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.
Overall Study
| Stage | Healthy Males |
|---|---|
| STARTED | 12 |
| COMPLETED | 12 |
| NOT COMPLETED | 0 |
Analysis population for the primary outcome, as defined in the registry: Data wasn't collected
5. Baseline Characteristics
The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.
| Characteristic | Unit | Healthy Males |
|---|---|---|
| Participants analysed | 12 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 12 |
| Age, Categorical: >=65 years | Participants | 0 |
| Sex: Female, Male: Female | Participants | 0 |
| Sex: Female, Male: Male | Participants | 12 |
| Race and Ethnicity Not Collected | Participants | — |
| Region of Enrollment: United States | participants | 12 |
6. Primary Endpoint
The registry lists the primary outcome as Change in Baseline Visual Evoked Potential (P100) Amplitude, measured over pre-dose and post-dose of drug intake, 7 hours.
| Element | Registry entry |
|---|---|
| Result type | N/A |
| Measure of spread | not applicable |
| Unit | not stated |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is titled “Change in Baseline Visual Evoked Potential (P100) Amplitude.” A visual evoked potential records electrical activity associated with visual stimulation. P100 is the named component of interest, and amplitude describes the size of the recorded response. In general, a change in amplitude is a way to describe whether the measured response differs between assessments; it is not, by itself, a direct measure of a participant's clinical experience or a diagnosis.
The before-and-after structure makes the endpoint a within-participant change: a post-dose amplitude would be compared with the same participant's pre-dose amplitude. In general, paired measurements can be useful because each participant's baseline provides a personal reference. A valid summary would still need the observed measurements and a stated method for combining the individual changes. The registry record does not report those data, a summary estimate, or an uncertainty interval.
The primary outcome population is described in the registry as “Data wasn't collected.” This means the posted record cannot establish the size or direction of a change in P100 amplitude. The timeframe is listed as pre-dose and post-dose of drug intake, 7 hours, while the study summary describes tests 1 hour after dosing. The record does not explain the difference. The remaining outcome titles and measurement details are not reported in the registry record, so the specific endpoint definitions beyond the named primary measure cannot be assessed from this record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central interpretive fact is that the registry record says the primary P100 amplitude data were not collected. There is therefore no reported primary endpoint estimate to interpret: no observed change, no standard error, no confidence interval, and no primary-outcome test result are reported. It is not possible to describe the magnitude, direction, or precision of a primary outcome difference from the registry record. The study's completed status does not fill this gap.
The record identifies a single group of healthy males and describes assessments before and after a single dose of perampanel 6 MG. This design indicates the intended comparison, but an intended comparison is not a result. The registry does not report participant-level values or aggregate results for P100 amplitude. It also lists seven outcome measures and zero analyses, so the record does not furnish a set of analysis results from which a broader account of test changes could be drawn. Details for the other outcome measures are not reported in the registry record.
The timing information also limits interpretation. The study summary describes neurophysiology tests 1 hour after ingestion, whereas the primary outcome timeframe is listed as pre-dose and post-dose, 7 hours. The registry does not resolve whether these refer to different study procedures or whether one description is inaccurate. No timing should be selected as the actual primary assessment time on the basis of this record alone.
In general, a confidence interval describes the range of effect estimates that are compatible with the data and the statistical method used, with its width conveying precision. No such interval is reported for the primary endpoint because the registry says its data were not collected. A missing estimate is not evidence that the change was zero, and it does not establish that measurements were unchanged. It means the posted record cannot answer the primary quantitative question.
Likewise, the record cannot support conclusions about whether any change in the neurophysiology tests was large or small, consistent or variable, or attributable to perampanel. In general, before-and-after measurements without a concurrent control group are subject to alternative explanations for change over time. The record does not report enough outcome data to assess even the observed within-group pattern, much less to distinguish possible explanations. The posted information is useful for understanding the intended design and the absence of the primary data, but not for estimating the primary effect.
9. Statistical Methods Explained
The registry describes a single-group before-and-after design. In general, this design measures participants at baseline and again after an exposure, then summarizes the within-participant differences. The pairing matters: a participant's post-dose result is naturally linked to that participant's own baseline, rather than treated as an independent observation from a different person. A paired analysis can account for stable participant-level differences, but it does not automatically account for changes over time or factors that coincide with the intervention.
For this study, the intended exposure was a single oral dose of perampanel 6 MG. The baseline and post-dose test battery described in the summary included VEP, EEG, SEP, and BAEP. In general, the analysis for each test would depend on its measurement scale and the prespecified endpoint definition. The registry names P100 amplitude as the primary endpoint but does not report a statistical analysis plan or the methods used to summarize that endpoint. The endpoint data themselves are marked as not collected.
A conventional estimate for a paired outcome would be based on the individual pre-dose to post-dose differences, with an accompanying measure of uncertainty. In general, uncertainty can be represented by a standard error or confidence interval, and a test statistic or p-value may be reported if an inferential test was planned. These quantities depend on the observed data and the selected analysis. Since the registry provides no primary outcome measurements or analysis results, none can be interpreted for P100 amplitude in this study.
The design has no randomized allocation, placebo, or separate comparator arm. This is important when considering what a paired comparison can establish. In general, repeated measurement may reduce the influence of stable differences between people, but a before-and-after change alone cannot rule out time effects, measurement variability, or other events occurring between assessments. Randomization and a concurrent control group are common design tools for addressing some of these concerns; their absence is a feature of this study as described, not evidence that a particular bias occurred.
The registry lists seven outcome measures and zero analyses. It does not provide the titles and definitions of the other outcomes in the record summarized for this study, nor does it report whether all outcomes were intended to be analyzed in the same way. In general, multiple outcomes create a multiplicity issue: when many statistical tests are conducted, the chance of at least one apparently unusual result can increase. Researchers may address this through a prespecified hierarchy, an adjustment, or a clearly exploratory interpretation. The registry record does not report a multiplicity strategy, so none should be attributed to the study.
Sample-size planning and analysis should also be distinguished. The registry record does not report the trial's own sample-size justification. A planning illustration is described using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the effect to detect set as a stated fraction of the observed effect. These are assumptions for an illustration, not the design used in this single-group study. Equal allocation between two arms does not describe the actual enrollment structure. Planning calculations are not observed power and do not show that the study achieved a particular probability of detecting an effect. No primary effect estimate is available in the registry record to interpret alongside any such planning assumptions.
Over-dispersion is a general issue when observed variability is greater than a statistical model assumes. In count or other outcome models, ignoring over-dispersion can make uncertainty appear too small and tests appear more decisive than warranted. No distributional model or dispersion assessment is reported in the registry record, and the primary endpoint is not reported as collected. Therefore, over-dispersion cannot be evaluated as a feature of the posted primary analysis; it is relevant only as a general modeling consideration.
Similarly, in general, missing outcome information may require an explicit account of which participants were measured, why observations are absent, and how any missingness was handled. The record's statement that primary data were not collected is not accompanied by participant-level information about missingness or an analysis population. No imputation or other missing-data approach is reported in the registry record. It would be inappropriate to assume that a particular missing-data method was used.
The primary endpoint timeframe is recorded as pre-dose and post-dose, 7 hours, while the study summary says testing was conducted 1 hour after dosing. The analysis time point is therefore not unambiguous in the record. In general, timing needs to be specified because a measurement at one time after dosing may not answer the same question as a measurement at another time. The registry does not clarify which timing governed the primary endpoint. Taken together, the available methods information describes the broad design and planned measurements, but not the actual statistical procedures or an analyzable primary result.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists seven outcome measures but no analysis entries. It identifies P100 amplitude as the primary outcome, yet the primary outcome population is marked “Data wasn't collected.” The registry record does not report results for that endpoint or give enough information to determine whether the other listed outcomes were collected or analyzed. Their titles, definitions, and result summaries are not reported in the registry record.
Multiplicity is a concern in general when investigators examine several outcomes or conduct several tests: more opportunities to test can increase the chance of a nominally unusual finding. A prespecified primary endpoint can help distinguish the main question from secondary or exploratory questions, while an adjustment or a clearly stated interpretation strategy may address multiple testing. The registry record does not report a multiplicity plan, adjustment method, or hierarchy beyond identifying the primary endpoint. No such procedure should be presumed.
The missing primary data are distinct from a nonsignificant result. No test result is reported, and the record does not say that an analysis found no difference. It also does not report the number of participants with complete primary measurements, reasons data were not collected, or methods for handling missing observations. The planned enrollment was 12, but the record does not identify a primary analysis population or state how many participants contributed primary endpoint measurements.
In general, an analysis population definition explains which participants or observations are included in a particular analysis. The registry record does not report such definitions or a missing-data strategy. Because the primary outcome data are explicitly marked as not collected, neither the extent nor the pattern of endpoint missingness can be evaluated. The registry record also does not report interim analyses or other details of the statistical analysis plan.
11. Safety Results
The registry reports serious adverse events as affected participants divided by participants at risk.
| Arm | Participants with serious adverse events | Participants at risk | Percentage |
|---|---|---|---|
| Healthy Males | 0 | 12 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Healthy Males | 0 of 12 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 0% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Healthy Males | 10 of 12 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Healthy Males |
|---|---|---|
| vertigo | Nervous system disorders | 4 of 12 |
| drowsiness | General disorders | 3 of 12 |
| headache | Nervous system disorders | 3 of 12 |
| dizziness | General disorders | 3 of 12 |
| nausea | General disorders | 2 of 12 |
| fatigue | General disorders | 1 of 12 |
| nystagmus | Eye disorders | 1 of 12 |
The registry's safety figures list 12 healthy males at risk for the serious-event category and 0 affected. For the other category, it lists 12 at risk and 10 affected. Deaths are listed as 0, and no top serious-event terms are listed. These are the figures recorded for the group; the registry does not provide further event-level descriptions in the record.
These categories should be read separately. The serious-event figure is not the same as the “other” affected figure, and the record does not describe the 10 affected participants as having serious events. The meaning and event definitions for the “other” category are not reported in the registry record, so the figure should not be relabeled or interpreted as a particular type of event. The record also does not provide details such as event names, timing, duration, severity, or whether an event was considered related to perampanel.
In general, a safety count describes what was recorded in a group over the observed period; it does not by itself establish causation or characterize risk beyond that study context. The registry describes a small study of healthy males and does not provide a comparator group for safety. The listed counts therefore do not support a comparative safety estimate. They are a record of the reported categories, not a complete account of how participants felt or of the safety profile in other populations.
12. What the Result Does, and Does Not, Mean
The record documents a completed phase 4, single-group study intended to examine neurophysiology tests before and after a single dose of perampanel 6 MG in healthy males. It names change in VEP P100 amplitude as the primary endpoint and describes other tests, including EEG, SEP, and BAEP. The most consequential result for interpretation is that the primary outcome data were not collected, and the registry lists no analyses.
Accordingly, the record does not report whether P100 amplitude increased, decreased, or remained similar after dosing. It supplies no effect estimate or measure of precision for that endpoint. This absence should not be interpreted as evidence of no change, and it does not establish that the study found no effect. It means the primary quantitative question cannot be answered from the posted registry results.
The single-group before-and-after design also has limits for causal interpretation. In general, observing a difference between baseline and follow-up would describe a change over time within the group, but without randomization or a placebo group it would not isolate the intervention from all other possible explanations. Here, the absence of a primary estimate means even that within-group change cannot be described from the record.
The safety record lists 0 serious affected among 12 at risk, 10 affected in the other category among 12 at risk, and 0 deaths. These counts provide limited descriptive information; event details and a comparator are not reported. The record is specific to the studied healthy male group and does not establish what might be observed in a different population or setting.
The timing description is unresolved: the study summary says testing was performed 1 hour after ingestion, while the primary endpoint timeframe says 7 hours. The record does not explain the discrepancy. Overall, the posted information establishes the intended design, named primary endpoint, and absence of collected primary data, but it does not establish a direction, size, or precision of change in neurophysiology measurements.
13. Why This Trial Matters Statistically
This study is statistically informative as an example of the distinction between a study's stated question and the results available for answering it. The registry names a measurable primary endpoint and describes a before-and-after design, but marks the primary data as not collected. Without observations, the analysis cannot yield an effect estimate or quantify its uncertainty. Study completion alone does not substitute for endpoint results.
The design also makes the comparison structure important. The intended reference is each participant's pre-dose measurement, not a randomized control group. In general, within-person comparisons can be useful for repeated measurements, but they do not provide the same protection against time-related influences as a concurrent randomized comparison. Recognizing that distinction prevents an intended pre/post assessment from being read as a controlled treatment effect.
The record highlights the importance of clear endpoint timing. The summary's 1-hour description and the primary endpoint timeframe of 7 hours are inconsistent, and the record does not resolve them. Precise timing matters to interpretation because an endpoint is defined not only by what is measured, but also by when it is measured.
Finally, the record lists seven outcomes but no analyses and does not report a multiplicity plan or sample-size justification. These omissions make it impossible to assess how the study intended to handle multiple questions or how its planned enrollment related to the precision it sought. General statistical planning can clarify these choices, but planning assumptions cannot replace observed results. The main statistical lesson is that design descriptions, outcome data, and analysis results are separate parts of the evidence record; only the design description is substantially available for the primary question in this case.
14. Limitations
- The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
- Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.
The primary limitation is explicit: the registry says data were not collected for the primary P100 amplitude outcome. No primary result, uncertainty estimate, or analysis is reported. The record does not explain why the data were not collected or whether any measurements were obtained but not entered, so the reason cannot be inferred.
The study has no placebo, randomization, masking, or separate control group according to the registry. It is described as a single-group study. In general, this limits the ability of a before-and-after design to distinguish a change associated with the intervention from other changes over time. This is a design limitation, not evidence that a particular alternative explanation occurred.
The population is described as healthy males, with planned enrollment of 12. The record does not support generalizing the intended measurement question to other populations. The safety categories are also sparse in detail: the registry provides counts but does not list the events in the other category or describe severity, timing, or attribution. No comparator is available for interpreting those counts comparatively.
The timing descriptions are inconsistent. The study summary says testing took place 1 hour after ingestion, while the primary endpoint timeframe is recorded as pre-dose and post-dose, 7 hours. The registry record does not clarify which time point applies to the primary endpoint. In addition, although seven outcome measures are listed, the registry does not report their full details in this record, nor does it give an analysis plan, multiplicity strategy, analysis population, missing-data method, or sample-size justification. These gaps prevent a thorough appraisal of the intended statistical analysis.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Effects of Perampanel on Neurophysiology Test Perimeters — NCT03653741.
- Registry record last updated 2023-07-03; status last verified 2023-06.
- Results first posted 2023-07-03.
- Data retrieved from ClinicalTrials.gov on 2026-11-09.
- Group sizes, means, event counts and sponsor-reported p-values are reproduced as posted. Effect estimates, confidence intervals and p-values not attributed to the sponsor are independently calculated from the posted summary statistics.
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18. Record Summary
The registry describes a completed phase 4, single-group study of perampanel in healthy males, with planned enrollment of 12. Participants were intended to undergo neurophysiology tests before and after a single oral dose of perampanel 6 MG; the summary also describes a blood draw. There was no placebo or randomization, and masking is listed as none.
The primary endpoint is change in VEP P100 amplitude. The registry states that its data were not collected and lists zero analyses. It identifies seven outcome measures, but other outcome details are not reported in the registry record. The safety record lists 0 serious affected among 12 at risk, 10 affected in the other category among 12 at risk, and 0 deaths.
The study summary describes post-dose tests at 1 hour, while the primary outcome timeframe is recorded as 7 hours; the discrepancy is not explained. The registry record does not report a sample-size justification or detailed statistical analysis plan.