Cardiometabolic Effects of Eplerenone in HIV Infection: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Cardiometabolic Effects of Eplerenone in HIV Infection (NCT02629094), a na phase 2 study. The primary outcome is Improvement of Cardiac Steatosis: Mean Change in Intraventricular Septum Percentage of Lipid by MR Spectroscopy..

2. Clinical Question

The study title identifies cardiometabolic effects of eplerenone in HIV infection, and the listed conditions include cardiac steatosis and hepatic steatosis. The brief summary describes the rationale as examining whether a blood pressure drug that blocks a hormone could reduce fat stored in the heart muscle and liver in people with HIV and increased visceral fat. The primary endpoint focuses specifically on lipid percentage in the intraventricular septum; it is not a direct measure of every cardiometabolic outcome named in the title or summary.

The registry record lists enrollment of 5 and a terminated status. It does not report how many participants completed the required treatment period or contributed to the primary endpoint analysis. Consequently, the stated target question can be described, but the posted record does not allow a quantitative answer about the observed change or its precision.

3. Trial Design

This was a phase 2, single-group study of eplerenone. The record describes no masking and no randomized allocation; allocation is listed as not applicable. There is no concurrent comparator arm, so the study is not a randomized comparison and does not support a direct estimate of how outcomes would have differed under another treatment or no treatment.

The registry lists enrollment of 5 and status as terminated. The recorded start date is 2015-12-02, and the primary completion date is 2017-09-11. These dates and status describe the registry record; they do not establish why the study was terminated or whether every planned procedure was completed. The reason for termination is not reported in the registry record.

The intervention description states that eplerenone was administered for 6 months, beginning at 25 mg once daily for 1 week and then continuing at 50 mg once daily for the remainder of the study. The primary endpoint has a timeframe of 24 weeks and is defined for participants who completed 24 weeks on study drug. The record does not explain the relationship between the 6-month treatment description and the 24-week endpoint beyond specifying both.

The brief summary describes screening procedures that included medical assessment, blood tests, body measurements, MRI, electrocardiography, transient elastography and an optional liver sample. It also describes baseline assessments, including a DEXA scan and resting energy expenditure measurement. These procedures provide context for the broad cardiometabolic research question, but they do not change the definition of the primary endpoint.

The registry lists 2 outcome measures and no analysis entries. A detailed statistical analysis plan, the reason for the single-group design, the planned recruitment target and the rationale for the enrollment recorded are not reported in the registry record. The posted record also does not state whether any interim review was planned or conducted.

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

Analysis population for the primary outcome, as defined in the registry: Analyses included participants who completed 24 weeks on study drug eplerenone.

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.

Population: All screened participants were evaluated for baseline data, of which 5 were eligible to start.

6. Primary Endpoint

The registry lists the primary outcome as Improvement of Cardiac Steatosis: Mean Change in Intraventricular Septum Percentage of Lipid by MR Spectroscopy., measured over 24 weeks.

The primary endpoint is mean change in intraventricular septum percentage of lipid measured by MR spectroscopy. The registry defines the change as the week 24 lipid-percentage value minus the baseline value. Under that definition, a negative change would indicate a lower measured percentage at week 24 than at baseline, while a positive change would indicate a higher measured percentage. This direction describes the arithmetic of the endpoint, not a clinical benefit or harm.

The endpoint is used to quantify change in a cardiac tissue measure over the treatment period. MR spectroscopy is the measurement method specified in the registry; the endpoint is not a symptom score, a clinical event, or a measurement of liver fat. The record’s brief summary mentions both heart and liver fat, but the stated primary endpoint is limited to lipid percentage in the intraventricular septum.

The registry reports that the endpoint analysis included participants who completed 24 weeks on study drug eplerenone. It does not report the mean change, standard deviation, number of participants included in this analysis, or a confidence interval. The numerical result and its precision are therefore not reported in the registry record. Without those values, the size and direction of the observed endpoint change cannot be described from the posted information.

As a general statistical point, a mean change summarizes the average within-person difference in the measured endpoint among the analyzed participants. It does not show how similar or variable individual changes were; a dispersion measure such as the standard deviation helps describe that variation. The registry names standard deviation as the dispersion measure, but gives no numerical value for it.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

Statistical interpretation begins with the estimand: the quantity a study intends to describe or compare. In this record, the primary endpoint is the mean within-person change in intraventricular septum lipid percentage from baseline to week 24. Its analysis population is defined by completion of 24 weeks on study drug. This is a description of change among completers, not automatically an estimate for every person enrolled or for all people with HIV and increased visceral fat.

What does a mean change represent?

For each participant with the required measurements, change is defined as the week 24 value minus the baseline value. The mean summarizes those participant-level changes. It is not the same as the average week 24 measurement alone, and it does not describe the spread of individual responses. The registry identifies standard deviation as the dispersion measure, which would help show how varied the observed changes were. Neither the mean nor the standard deviation is reported in the registry record.

As a general statistical explanation, a confidence interval describes a range of values compatible with the data and the method used to calculate it. A narrow interval indicates greater statistical precision than a wide interval, while neither width nor location alone establishes clinical importance. The registry record does not report a confidence interval for the primary endpoint, so its uncertainty cannot be evaluated from the posted results.

Why does the single-group design matter?

The design is single-group, without a concurrent control arm. A within-person comparison uses each participant’s baseline measurement as a reference, but baseline is not a substitute for a randomized control group. General sources of change over time, including measurement variability and events occurring during follow-up, cannot be separated from an intervention-associated change using a single-group comparison alone. This is a limitation of the design’s ability to support causal interpretation; it does not identify which, if any, such influences occurred in this study.

The record lists allocation as not applicable and masking as none. It does not describe blinded measurement or an independent outcome assessment process. These details matter because knowledge of treatment or study status can sometimes affect behavior, assessment, or reporting. Whether such effects occurred is not reported in the registry record.

How should the planning calculations be understood?

The stated planning illustration uses a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It describes an effect to detect as a stated fraction of the observed effect. These are assumptions for a hypothetical planning exercise, not evidence that this single-group study enrolled two randomized arms or used those assumptions for its own design. The record specifically identifies the calculations as planning illustrations rather than the trial’s own design.

In general, a sample-size calculation asks how much information might be needed to detect a specified effect under assumed variability, allocation and error rates. Changing the assumed effect or other inputs can change the calculated requirement. Such planning figures should be interpreted as conditional illustrations, not as the number of participants actually analyzed, not as proof that the study had adequate precision, and never as observed power. The numerical planning outputs are not stated in the registry entries described for this analysis.

A two-sided significance level concerns the probability threshold used by a specified testing procedure under its assumptions. It does not describe the probability that a result is true. A normal approximation is a mathematical method used in calculations; its appropriateness depends on the statistical problem and available data. Equal allocation between two arms is an assumption of the planning illustration, not a feature of the recorded single-group trial. These general explanations do not establish that the study used a hypothesis test or that its outcome met any threshold.

What can dispersion and over-dispersion tell readers?

Standard deviation describes the spread of participant-level measurements around their mean. It is different from the standard error, which describes uncertainty in an estimated mean and depends on the amount of information available. A confidence interval is commonly built using an estimate and an uncertainty measure, but its exact construction depends on the analysis method. The registry gives no numerical standard deviation, standard error, or interval for the primary endpoint.

As a general concept, over-dispersion occurs when observed variability is greater than a statistical model assumes. If a method underestimates variability, inferential uncertainty can appear too small. The primary endpoint is continuous and its stated dispersion is standard deviation; the record does not report an over-dispersion assessment or identify a model that would permit one. There is therefore no basis for claiming over-dispersion was present or addressed.

What does a recalculation mean?

A recalculation labeled as such is a simple, unadjusted calculation from posted summary statistics. It is not the sponsor’s analysis and may not reproduce an analysis that uses individual-level data, covariate adjustment, a different population, or a prespecified model. If a discrepancy is identified, the sponsor’s own analysis takes precedence. For this endpoint, the summary statistics needed for an unadjusted recalculation are not reported in the registry record, so no recalculated endpoint estimate can be described.

More broadly, statistical methods answer different questions: a point estimate describes magnitude, a dispersion measure describes variability, and inferential measures describe uncertainty under specified assumptions. None can replace the others. Here, the endpoint definition and the design are recorded, but the primary numerical estimate and its uncertainty are not. Statistical interpretation should not fill those gaps by inferring an effect from the study title, intended objective, or safety information.

10. Confidence Intervals for Each Arm

No two-arm comparison is shown for this trial, but each arm's mean can be given with a t-based 95% confidence interval computed from the posted mean, spread and number of participants.

R code and output

arm <- c("Eplerenone")
n   <- c(5)
m   <- c(-0.33)
sp  <- c(4.3)
sd  <- sp      # posted standard deviations
for (i in seq_along(n)) {
  se <- sd[i] / sqrt(n[i]); h <- qt(0.975, n[i] - 1) * se
  cat(sprintf("CARM|%s|%d|%.3f|%.3f|%.3f|%.3f\n", arm[i], n[i], m[i], sd[i], m[i] - h, m[i] + h))
}

CARM  Eplerenone  5  -0.330  4.300  -5.669  5.009

11. Multiplicity, Missing Data and Analysis Populations

The registry lists 2 outcome measures and no analysis entries. It does not report a multiplicity strategy, such as whether one outcome was designated as the sole basis for confirmatory inference or whether any adjustment was planned across outcomes. The primary endpoint is identified, but the status of other endpoint analyses and their role in interpretation are not reported in the registry record.

As a general statistical concept, multiplicity arises when several hypotheses or outcomes are assessed. Without an appropriate plan, repeated testing can increase the chance of at least one apparently unusual result under conditions where no true effect exists. Adjustment procedures or a clearly prioritized testing plan can address that concern, but the record does not state whether either was used. This general explanation should not be read as evidence that multiplicity affected the study’s findings.

The primary endpoint population is defined by completing 24 weeks on study drug. The number of participants who met that definition is not reported in the registry record, nor are the reasons for any missing baseline or week 24 measurements. The record does not describe an approach to missing data, such as imputation, a model using incomplete observations, or a sensitivity analysis. It is therefore not possible to assess whether missingness could have influenced the reported endpoint estimate; the estimate itself is also not reported.

As a general matter, excluding participants without a complete endpoint can be reasonable for a defined completer analysis, but the result describes that selected analysis population. If completion is related to outcomes or participant characteristics, a completer estimate may differ from an estimate for everyone enrolled. Whether completion was related to outcomes in this study is not reported in the registry record.

The record does not report a detailed analysis population beyond the statement that the primary analysis included participants who completed 24 weeks on drug. It also does not report prespecified handling of protocol deviations, treatment discontinuation, or measurements taken outside the stated timeframe. These omissions prevent a fuller assessment of how the analyzed population and missing observations were defined.

12. Safety Results

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

Deaths

Other (non-serious) adverse events

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

Most frequent non-serious adverse event terms

The registry safety figures list 5 participants at risk for serious adverse events and 0 participants affected by serious adverse events in the Eplerenone group. The listed death count is 0. The record does not list serious adverse event terms among the top serious terms. These figures describe the posted safety record and should not be read as proof that no safety events of any kind occurred.

The same record lists 5 participants at risk for other adverse events and 5 affected by other adverse events. The distinction matters: the serious adverse event figures and other adverse event figures refer to different categories. The record does not give the specific other adverse event terms, their severity, timing, duration, or whether they were considered related to treatment. The timeframe for these safety figures is not reported in the registry record.

As a general statistical consideration, absence of a reported serious event in a small study does not establish that a treatment is free of serious risks. Safety counts from a single-group study also lack a concurrent comparator for estimating differences in event frequency. These observations describe the limits of interpreting the posted counts; they are not medical advice and do not establish a causal relationship between eplerenone and any event.

The registry lists enrollment of 5, but the safety figures separately report the number at risk. They should be read as stated rather than combined with other counts or treated as a measure of exposure time. The duration of individual exposure and the method used to collect adverse event information are not reported in the registry record.

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

The record identifies a focused research question: change in intraventricular septum lipid percentage from baseline to week 24 among participants who completed 24 weeks on eplerenone. It also documents a phase 2, single-group design and an enrollment of 5. These details establish what the study set out to assess and the broad setting in which it was conducted.

What the posted record does not establish is the size or direction of the primary endpoint change. The mean change, its standard deviation, the endpoint analysis denominator and a confidence interval are not reported in the registry record. Consequently, the quantitative result and its statistical precision cannot be evaluated. The treatment objective or endpoint title should not be substituted for a measured outcome.

Even if a before-and-after change were reported, the single-group design would not by itself separate a change associated with treatment from other changes over time. In general, a concurrent control group can help estimate what might have happened over the same period without the intervention, although design and implementation still matter. This study has no like-for-like control comparison in the registry record.

The primary endpoint is a tissue lipid measurement, not a clinical event or a measure of every possible health outcome. The brief summary also describes an interest in liver fat, but the stated primary endpoint concerns the intraventricular septum. Results for that cardiac measure, even if reported, would not automatically answer questions about liver outcomes or broader cardiometabolic effects.

The terminated status should be reported as a feature of the record, not treated as an explanation or a result. The reason for termination and its relationship to data collection are not reported in the registry record. The safety figures indicate no participants affected by serious adverse events in the recorded Eplerenone group, while other adverse events are listed for all participants at risk; these counts do not establish a full safety profile or a comparative risk.

Overall, the record supports a description of the study question, design and posted safety counts, but not a numerical conclusion about the primary endpoint. It does not establish that eplerenone did or did not change the measured cardiac lipid percentage. Any interpretation beyond the documented design and counts would go beyond the results reported in the registry record.

14. Why This Trial Matters Statistically

This study raises several statistical issues that are important in early clinical research. First, the design is single-group, while the primary endpoint is a change over time. Such a design can describe the trajectory observed among participants, but without a concurrent comparator it cannot isolate the intervention’s contribution from other possible influences. That distinction between observed change and a comparative treatment effect is central to interpreting uncontrolled studies.

Second, the endpoint population is defined by completion of 24 weeks on study drug. That definition makes the analysis population consequential: the estimate would describe participants who completed the specified period, and not necessarily everyone enrolled. The registry does not report the number contributing to the endpoint or how missing measurements were handled, limiting assessment of both representativeness and precision.

Third, the outcome is a continuous measurement reported as a mean change with standard deviation. A point estimate alone would not show how variable participant-level changes were, and a mean without an uncertainty measure would not communicate precision. The registry record does not report the numerical estimate or its dispersion, so the principal statistical question cannot be answered from the posted results.

The record also lists 2 outcome measures but no analysis entries. This makes transparency about outcome prioritization, multiplicity and analysis decisions especially relevant. The absence of those details in the registry record does not show that the study lacked such decisions; it means readers cannot verify them from the posted information.

Finally, planning illustrations should be distinguished from observed evidence. Calculations based on a stated fraction of an observed effect and specified assumptions can inform future study planning, but they are not observed power and do not supply missing outcome results. Keeping these distinctions clear helps prevent a small early-phase study, an incomplete statistical record, or an illustrative calculation from being interpreted as stronger evidence than the reported data support.

15. 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 limitations specific to this study constrain interpretation. The registry describes a single-group design with no masking and no like-for-like control comparison. This means the primary endpoint is framed as a within-person change, not a randomized estimate of the difference between eplerenone and a comparator. A change over time cannot by itself distinguish treatment-associated change from other potential influences.

The registry lists enrollment of 5, while the primary analysis is described as including participants who completed 24 weeks on study drug. The number of completers and the number with evaluable baseline and week 24 measurements are not reported in the registry record. Because the endpoint analysis denominator is unavailable, the amount of information contributing to the mean change cannot be assessed.

The record does not report the primary mean change, standard deviation, confidence interval or p-value. It therefore does not permit an assessment of the observed direction, magnitude, variability or precision. The status is terminated, but the reason and its implications for endpoint collection are not reported in the registry record.

Although the brief summary describes interest in both heart and liver fat, the primary endpoint is specifically intraventricular septum lipid percentage by MR spectroscopy. It should not be taken as a measure of liver fat or of broader clinical outcomes. The record also does not provide enough information to assess how missing data, protocol deviations, multiplicity or additional endpoint analyses were handled.

The safety figures are limited to counts of participants at risk and affected, without event terms for other adverse events, timing, duration, exposure information or a comparator group. The available counts therefore cannot characterize comparative safety or establish the absence of risk. These limitations concern what the registry record makes available; they do not establish that additional data or analyses do not exist elsewhere.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Cardiometabolic Effects of Eplerenone in HIV Infection — NCT02629094.
• Registry record last updated 2018-07-17; status last verified 2017-09.
• Results first posted 2018-07-17.
• 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.

19. Record Summary

The registry record describes NCT02629094, titled “Cardiometabolic Effects of Eplerenone in HIV Infection.” It is listed as a phase 2, single-group study with no masking, enrollment of 5, and terminated status. The recorded start date is 2015-12-02, and the primary completion date is 2017-09-11. The sponsor is the National Institute of Allergy and Infectious Diseases (NIAID).

The intervention is eplerenone, described as 25 mg once daily for 1 week followed by 50 mg once daily for the remainder of a 6-month study. The primary endpoint is mean change in intraventricular septum percentage of lipid by MR spectroscopy at 24 weeks, among participants who completed 24 weeks on study drug. The numerical endpoint result and its standard deviation are not reported in the registry record.

The safety record lists 0 participants affected by serious adverse events and 0 deaths in the Eplerenone group, and 5 participants affected by other adverse events. The registry lists 2 outcome measures and no analysis entries. The reason for termination, endpoint analysis denominator, missing-data methods and multiplicity strategy are not reported in the registry record.