Development of Ivermectin for Alcohol Use Disorders: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Development of Ivermectin for Alcohol Use Disorders (NCT02046200), a randomized phase 1, phase 2 study. The primary outcome is Heart Rate.

2. Clinical Question

The trial examined whether heart rate changed after ivermectin compared with placebo, including while participants received an alcohol infusion. The registry describes the heart-rate monitoring as a way to assess the safety of combining ivermectin with moderate doses of alcohol. The infusion targeted BrAC levels from 0.00 to 0.08 g/dl, and measurements were planned across post-medication time points extending from 0 to 48 hours. The registry record does not report numerical heart-rate results, so it does not establish the size or precision of any difference between conditions.

This is a question about a measured physiological response in a small, early-phase trial, not a direct assessment of long-term treatment outcomes. The registry lists phase 1 and phase 2 and describes the study as randomized, triple-masked, and crossover. Details such as the order of conditions, timing between treatment periods, and the precise comparison used for the primary analysis are not reported in the registry record. Those omissions matter when interpreting a crossover study because measurements may depend on treatment sequence, period, and the way repeated observations are analyzed.

3. Trial Design

The registry identifies this study as a randomized, triple-masked crossover trial in phase 1 and phase 2. It was sponsored by the University of California, Los Angeles, began in 2014-02, reached primary completion in 2015-03, and is listed as completed. Enrollment was 11. The condition was Alcohol Use Disorder.

The intervention descriptions list a single 30 mg dose of ivermectin and a single dose of matched placebo. Alcohol is also listed among the interventions, and the primary-outcome description refers to an alcohol infusion. In this setting, ivermectin and placebo are the medication conditions, while alcohol infusion is a challenge during which heart rate was monitored. The record does not describe the sequence or timing of these conditions in enough detail to reconstruct the full participant-level schedule.

In a crossover design, participants receive more than one study condition at different times. General statistical context: comparing conditions within the same participants can account for some stable differences between people, but it also makes the analysis dependent on period, sequence, and possible carryover effects. Whether and how the analysis addressed these factors is not reported in the registry record. The label triple-masked indicates masking at three levels, but the identities of those masked are not reported in the registry record.

The primary population is described as all randomized subjects included. The record does not specify the precise analysis model, the handling of incomplete observations, or whether any participants were excluded from a particular analysis. Although the registry lists 17 outcome measures and 11 analyses, the numerical primary heart-rate results and the detailed analysis output are not reported in the registry record. The study’s early-phase designation and enrollment describe its scale and stage; they do not, by themselves, establish what the study could reliably detect.

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.

First Intervention (1 Day)

Second Intervention (1 Day)

Analysis population for the primary outcome, as defined in the registry: All randomized subjects included.

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.

6. Primary Endpoint

The registry lists the primary outcome as Heart Rate, measured over Post-medication administration (hours): 0, 0.5, 1, 2, 4, 6, 8, 12, 16, 24, 48; During alcohol infusion at BrAC = 0.00, 0.02, 0.04, 0.06, 0.08 g/dl.

The primary endpoint is heart rate, measured in beats per minute. Heart rate is a continuous physiological measure: it can take a range of values rather than placing participants into categories. The registry specifies reporting the mean and standard deviation. The mean describes the average observed heart rate, while the standard deviation describes how spread out individual measurements are around that average.

The registry describes monitoring heart rate to determine the safety of combining ivermectin at 30 mg with moderate doses of alcohol, up to a BrAC of 0.08 g/dl. Measurements were planned after medication administration at 0, 0.5, 1, 2, 4, 6, 8, 12, 16, 24, and 48 hours. During infusion, measurements were tied to BrAC levels of 0.00, 0.02, 0.04, 0.06, and 0.08 g/dl. The record notes that the collection times during infusion varied according to how long participants took to reach the targeted BrAC levels.

Repeated heart-rate measurements can describe how a physiological response changes over time and across alcohol exposure levels. General statistical context: repeated observations from the same participant are related, so they should not automatically be treated as independent measurements. The registry record does not report the numerical heart-rate results or the statistical approach used to account for these repeated observations. It therefore does not permit a quantitative assessment of the size, variability, or precision of the primary-outcome comparisons.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry describes a continuous primary outcome summarized by a mean and standard deviation. It also specifies repeated measurements after medication and during alcohol infusion. The interpretation of such data depends not only on the measured values but also on how the analysis handles repeated observations, the crossover structure, and the several assessment points. The numerical primary results and detailed statistical analysis method are not reported in the registry record.

What does the mean describe?

The mean is the arithmetic average of a set of measurements. For heart rate, it represents the average beats per minute for the observations included in a particular summary. A mean can make a set of continuous measurements easier to summarize, but it does not show the full distribution. Two groups or conditions can have similar means while differing in how variable their measurements are, or the same mean can conceal different patterns across people or time.

The standard deviation describes the spread of measurements around the mean. It is a measure of variability among the observations, not a measure of uncertainty in the mean itself. General statistical context: the standard error and a confidence interval are used to express uncertainty about an estimated mean or contrast. The registry identifies standard deviation as the dispersion measure for the primary outcome but does not report its values or the corresponding uncertainty estimates.

Why do repeated measurements matter?

Heart rate was scheduled for measurement at multiple post-medication times and at multiple BrAC levels during infusion. Measurements from the same participant are generally related because they come from one person. General statistical context: treating correlated observations as though they were independent can misstate uncertainty. A suitable analysis typically needs to reflect the repeated-measures structure, but the particular model used for this trial is not reported in the registry record.

The infusion measurement times could vary according to how long it took participants to reach the targeted BrAC levels. This means the measurement schedule during infusion was linked to reaching those levels rather than necessarily following identical clock times for all participants. How the analysis accounted for that feature is not reported in the registry record. The registry identifies the target exposure levels and the time-varying collection schedule but does not provide participant-level timing or outcome values.

What does crossover add to the analysis?

In a crossover study, participants receive different conditions in successive periods, allowing comparison within the same participant. General statistical context: within-participant comparisons can reduce the influence of stable differences between people, but they do not remove every potential source of bias. The treatment sequence, period effects, and carryover from an earlier condition may affect results. The registry labels the design crossover, but the sequence, washout details, and statistical handling of period or carryover effects are not reported in the registry record.

The record describes the study as randomized and triple-masked. Randomization is intended to reduce systematic differences in allocation, while masking is intended to limit influences from knowledge of treatment assignment. These design features do not replace the need to report the analysis, outcome estimates, and uncertainty. The identities of the masked parties and the method of randomization are not reported in the registry record.

How should uncertainty and significance be read?

A confidence interval gives a range of values compatible with an estimate under the assumptions of the analysis. Its width indicates precision: narrower intervals represent more precise estimates, while wider intervals indicate more uncertainty. A p-value summarizes how unusual data would be under a specified null hypothesis and analysis model. It does not measure the probability that a treatment effect is real, and it does not describe the magnitude of an effect.

No confidence intervals, p-values, or numerical primary-outcome contrasts are reported in the registry record. It is therefore not possible to determine whether a difference was estimated precisely, whether it was compatible with no difference, or how the results varied across assessment conditions. General statistical explanations cannot substitute for these missing results.

What is the role of planning illustrations?

The statistical materials describe planning illustrations using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. They frame the effect to detect as a stated fraction of the observed effect. General statistical context: such calculations illustrate the number of participants per arm that might be needed under specified assumptions; they are not observed power and do not show what the completed trial established. The calculations are not the trial’s own design justification unless the registry says so, and the registry record does not report that justification.

Because numerical primary-outcome results are not reported in the registry record, planning illustrations cannot establish the observed effect or its precision. They should not be interpreted as evidence that the trial was adequately powered or that its results support a particular conclusion. The trial’s enrollment was 11, but that fact alone does not determine the reliability of an estimate; the outcome variability, analysis, and uncertainty are also relevant.

Why does multiplicity matter?

The record lists multiple outcome measures and analyses, and the primary measure itself includes multiple assessment times and BrAC levels. General statistical context: when many comparisons are made, the chance of finding an apparently notable result by chance can increase, depending on the analysis and decision rules. Approaches to multiplicity can include prespecifying a primary contrast, controlling an error rate, or interpreting results as exploratory. The registry record does not report a multiplicity strategy or identify a single primary contrast among the repeated assessments. No particular adjustment or interpretation should therefore be attributed to the study.

10. Multiplicity, Missing Data and Analysis Populations

The primary endpoint is not a single measurement at one time. It includes heart-rate assessments after medication at multiple time points and during alcohol infusion at several BrAC levels. The registry record explicitly notes that the primary outcome has several categories or time points and is not reduced to one comparison. This structure creates several potential comparisons and makes it important to know which contrasts were prespecified and how results were interpreted.

The registry lists 17 outcome measures and 11 analyses. It does not report a multiplicity strategy for the heart-rate assessments, such as a hierarchy of comparisons or an adjustment for multiple testing. General statistical context: evaluating many contrasts can increase the chance of a nominally notable result occurring by chance. A prespecified primary contrast or a stated adjustment can help guide interpretation, but neither is reported in the registry record.

Missing-data handling is not reported in the registry record. The record does not state how missed heart-rate measurements, incomplete infusion assessments, or participants without measurements at a particular time were handled. It also does not describe imputation, exclusions, or sensitivity analyses. The primary population is described as all randomized subjects included, but that wording does not establish that every randomized participant contributed data to every assessment.

The registry does not report a detailed analysis population beyond that description, nor does it specify whether the analysis followed an intention-to-treat principle or another approach. In a crossover design, availability of measurements can differ by treatment period, and the method for handling such patterns can affect estimates. Without the analysis details and numerical results, the consequences of missing data and multiplicity cannot be assessed.

11. Safety Results

The registry reports serious adverse events as affected participants divided by participants at risk.

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 0% reporting threshold.

The adverse-event record lists an ivermectin group and a placebo group. For each listed group, 11 were at risk for serious adverse events and none were recorded as affected. The same at-risk figures and no affected participants are listed for other adverse events. The death field is null for both groups; mortality figures are not reported in the registry record.

These figures describe what was recorded in the registry for the listed groups. They should not be read as proof that no adverse events could occur, or as a precise estimate of the frequency of uncommon harms. General statistical context: when an event is not observed in a limited study, uncertainty about its possible frequency remains. The record does not provide event-level descriptions because no serious adverse-event terms are listed.

The design is crossover, so participants may contribute to more than one condition at different times. The group-specific at-risk figures should therefore not be added together to infer a count of distinct participants. The registry does not give further detail on the timing of adverse-event monitoring, event attribution, or how events were assigned across treatment periods. These limitations restrict what can be inferred from the recorded safety counts.

12. What the Result Does, and Does Not, Mean

The registry record establishes that a randomized, triple-masked crossover study evaluated single-dose ivermectin and placebo in people with Alcohol Use Disorder, with heart rate monitored after medication and during alcohol infusion. It also records enrollment, the study’s phase and status, the outcome definition, and the planned measurement schedule. These details clarify the question the study set out to address.

The numerical heart-rate results are not reported in the registry record. As a result, the record does not show whether heart rate was higher, lower, or similar under the medication conditions, whether any pattern changed over time or across BrAC levels, or how precisely any comparison was estimated. It cannot support a claim about the magnitude or statistical significance of a treatment contrast.

The registry describes a safety-monitoring objective, but the absence of recorded adverse events in the listed groups is not a guarantee of safety. This was an early-phase study with enrollment of 11, and the adverse-event figures alone cannot establish the likelihood of rare events or effects beyond the study conditions. The record also does not report long-term clinical outcomes or evidence about treatment of Alcohol Use Disorder.

General statistical context: a result from a small study may be uncertain, and the ability to detect a difference depends on variability, design, analysis, and the size of the effect. The registry does not provide enough numerical outcome information to assess these elements for heart rate. Nor does it report the trial’s own sample-size justification or full statistical plan.

The appropriate interpretation is therefore limited to the study’s design and registered aims, together with the posted safety counts. The record does not establish that ivermectin reduces alcohol use, improves Alcohol Use Disorder, or has a particular heart-rate effect during alcohol exposure. Those conclusions would require results and evidence not reported in the registry record.

13. Why This Trial Matters Statistically

This trial is statistically notable because its primary measure was collected repeatedly in a crossover setting. Heart rate was tracked over a sequence of post-medication times and during an alcohol infusion at several target BrAC levels. That design produces a richer response profile than a single measurement, but it also raises analytic questions about within-person correlation, treatment sequence, period effects, and the interpretation of multiple contrasts.

The crossover structure can be useful when participants serve as their own comparison, because stable differences between people may be less influential in a within-person contrast. General statistical context: this advantage depends on appropriate design and analysis. The registry record does not report sequence details, washout information, or the model used to handle repeated observations, so the extent to which those features were addressed cannot be judged.

The registry lists 17 outcome measures and 11 analyses, while the primary endpoint itself spans several assessment points. This illustrates why a statistical result is not interpreted solely by looking for a single p-value. Prespecified contrasts, multiplicity handling, estimates, and uncertainty all matter. Those elements are not reported for the primary heart-rate outcome in the registry record.

The record also makes the distinction between study aims and demonstrated results important. It describes a rationale for evaluating ivermectin and identifies heart rate as a safety-monitoring measure, but it does not include numerical primary-outcome results. The registry’s adverse-event counts are descriptive and do not by themselves characterize the precision of safety estimates.

For biostatisticians and clinical researchers, the central lesson is that a design label and endpoint definition are not substitutes for transparent analysis reporting. Without outcome estimates, uncertainty measures, and analysis details, readers can understand what was intended but cannot evaluate the strength or precision of the evidence from the primary endpoint.

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.

Several trial-specific limitations restrict interpretation of the registry record. First, numerical results for the primary heart-rate outcome are not reported in the registry record. Without means, standard deviations, contrasts, or uncertainty estimates, the direction and size of any response cannot be evaluated.

Second, the primary endpoint spans multiple post-medication times and alcohol infusion levels. The record notes that it is not reduced to one comparison, but it does not identify a primary contrast or describe how multiplicity was handled. It also does not state how correlations among repeated measurements were modeled.

Third, key details of the crossover implementation are not reported in the registry record. The treatment sequence, timing between periods, and any consideration of period or carryover effects are not described. The identities of the three masked parties and the randomization method are also not reported.

Fourth, the registry does not describe missing-data procedures, exclusions, sensitivity analyses, or the exact analysis population beyond stating that all randomized subjects were included. This leaves uncertainty about whether all participants contributed measurements at every time point and how incomplete observations were treated.

Finally, enrollment was 11, and the registry does not report its own sample-size justification or full statistical plan. The recorded absence of adverse events in the listed groups should be interpreted descriptively, not as assurance about uncommon risks. These limitations do not establish that the study methods were inadequate; they mark information that is not reported in the registry record and that is needed for a fuller assessment.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Development of Ivermectin for Alcohol Use Disorders — NCT02046200.
• Registry record last updated 2018-08-08; status last verified 2018-07.
• Results first posted 2017-04-13.
• 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.

18. Record Summary

NCT02046200, titled Development of Ivermectin for Alcohol Use Disorders, is listed as completed. The study was sponsored by the University of California, Los Angeles, and is classified as phase 1 and phase 2, randomized, triple-masked, and crossover. It enrolled 11 participants with Alcohol Use Disorder.

The medication conditions were a single 30 mg dose of ivermectin and a single matched placebo dose. Alcohol infusion was used as a challenge condition. The primary outcome was mean heart rate in beats per minute, with measurements planned after medication and during infusion at specified BrAC levels. The registry describes all randomized subjects as the primary-outcome population.

The registry record does not report numerical primary heart-rate results or the detailed statistical plan. It lists no serious or other adverse events in either recorded group; mortality figures are not reported in the registry record. These records describe the study and its posted information, but do not provide a quantitative estimate of the primary heart-rate response.