Fenofibrate Treatment in SCI: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Fenofibrate Treatment in SCI (NCT02455336), a randomized phase 2, phase 3 study. The primary outcome is Triglyceride Concentration (Percent Change From Baseline).

2. Clinical Question

The clinical rationale in the registry record focuses on dyslipidemia in chronic spinal cord injury, including low HDL-C and elevated triglycerides. The stated aim is to assess short-term fenofibrate safety and efficacy in this context. The posted primary results describe group means and standard deviations for percent change from baseline. They do not include an inferential comparison between the groups, so the recorded summaries answer the descriptive part of the question but do not establish the precision or statistical significance of a treatment contrast.

3. Trial Design

This completed phase 2 and phase 3 study is titled “Fenofibrate Treatment in SCI.” The official title describes an open-label safety and efficacy trial in persons with spinal cord injury. The registry record identifies randomized allocation and a parallel-group model, with no masking. The sponsor is the VA Office of Research and Development. The recorded start date is 2015-05-18, and the primary completion date is 2018-08-01. Enrollment is listed as 23.

Participants assigned to fenofibrate were described as receiving 145 mg once daily for 4 months. Participants assigned to no intervention were described as receiving no therapy for 4 months. The eligibility descriptions refer to adverse triglyceride concentrations, with different thresholds for paraplegia and tetraplegia. The registry record does not report additional details about the randomization procedure, allocation concealment, or how eligibility thresholds were applied.

The primary endpoint is triglyceride concentration percent change from baseline at two months from initiating drug treatment. The posted arm summaries list 10 participants for fenofibrate and 8 for no intervention. Those endpoint-specific counts differ from the overall enrollment listing; the registry record does not explain the difference. It also does not report why the recorded treatment period is 4 months while the primary endpoint timeframe is two months. These details matter when interpreting who contributed to the endpoint and how the assessment relates to the full treatment period.

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

Reasons for not completing this period:

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: Only those participants who completed the 4 month trial are included in the analysis. Three participants who experienced adverse events that discontinued the trial do not appear in the results. Two participants who did not respond to 2 months of treatment do not appear in the results.

6. Primary Endpoint

The registry lists the primary outcome as Triglyceride Concentration (Percent Change From Baseline), measured over two months from initiating drug treatment.

The primary endpoint is triglyceride concentration expressed as percent change from baseline, assessed two months after initiating drug treatment. It is a continuous measure: each participant’s follow-up concentration is represented relative to that participant’s own baseline concentration. The registry record describes the group statistic as a mean and the measure of spread as a standard deviation.

The endpoint is relevant to the trial’s stated aim because the clinical rationale identifies elevated triglycerides as part of the dyslipidemia of spinal cord injury, and the study description says fenofibrate’s primary action is to lower triglycerides and raise HDL-C. The endpoint description defines a successful response as a 25% reduction in serum triglyceride concentration at two months. That criterion is part of the stated endpoint description; the posted results do not report the number or proportion of participants meeting it.

A mean percent change gives a group-level account of the direction and average size of change, while the standard deviation describes how much individual observations vary around that mean. Neither statistic alone shows the range of individual responses or gives a confidence interval for the group mean. The registry record does not report baseline or follow-up triglyceride concentrations, participant-level changes, or an analysis estimating the treatment contrast with uncertainty.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry record reports a continuous outcome summarized by a mean and standard deviation. The primary endpoint is percent change in triglyceride concentration from baseline, and the arm summaries are given at two months. No inferential comparison for the endpoint is posted. The statistical concepts below are general explanations of how such summaries and analyses are commonly interpreted; they are not claims about unreported procedures in this trial.

What does a mean describe?

General: The mean is the arithmetic average of participant-level observations in a group. For a change-from-baseline endpoint, it summarizes the average direction and size of change across participants with an observed value included in that summary. A mean can be useful for communicating a group pattern, but it does not show whether the individual responses are tightly clustered or widely dispersed. It can also be influenced by unusually large or small observations.

In this trial, the posted means are -40.0 for fenofibrate and -2.0 for no intervention. Because the endpoint is percent change from baseline, a negative value denotes a reduction relative to baseline. These means should not be mistaken for the percent of participants who improved, nor for a treatment-effect estimate with uncertainty. The record does not report participant-level values or the median and range, which could add information about the shape and spread of the observed changes.

What does the standard deviation describe?

General: A standard deviation describes the spread of observations around their group mean, in the same units as the measured outcome. A larger standard deviation indicates greater variability around the mean, but does not specify the shape of the distribution or the precision of the mean. Precision depends on both the spread and the number of observations. A standard deviation is not a confidence interval and should not be read as one.

The reported standard deviations are 12.0 in the fenofibrate group and 16.0 in the no-intervention group. These values describe within-group variation in the percent-change measure. They do not quantify uncertainty in the difference between groups. The registry record does not report confidence intervals for either mean or for a between-group contrast.

Why is a direct comparison important?

General: In a randomized parallel-group study, the central comparative question is usually whether the outcomes differ between assigned groups, and how precisely that difference is estimated. A direct analysis can account for the design and may use baseline information or other prespecified covariates when appropriate. A test result alone would not describe the size or precision of an effect; an estimate with a confidence interval is generally more informative for those purposes.

Here, the registry record states that values are missing or not estimable for a two-arm comparison of the continuous endpoint. It does not report a p-value, confidence interval, adjusted estimate, or unadjusted estimate for that comparison. Therefore, the group summaries can be described, but the inferential question about the between-group contrast cannot be answered from the posted analysis. No particular statistical model or test can be attributed to the trial based on the registry record.

What does a confidence interval convey?

General: A confidence interval is a range produced by a statistical procedure to express uncertainty about an estimated quantity, such as a mean or a difference between means. Its width is influenced by the variability in the data and the amount of information available. A narrow interval indicates greater precision than a wide interval, under the assumptions of the method used. A confidence interval is not a guarantee that the true value lies in that particular interval, and it does not replace assessment of the clinical meaning of the estimate.

No confidence interval is posted for this endpoint. Consequently, the degree of statistical precision around the observed group means and any possible between-group effect is not reported. This is distinct from the standard deviations, which describe variation among observations rather than uncertainty in an estimated mean or contrast.

What is over-dispersion, and is it relevant?

General: Over-dispersion refers to variability in data that is greater than a statistical model expects. It can make standard errors too small and confidence intervals too narrow if a model does not accommodate the extra variability. It is especially discussed for count outcomes and some other non-normal data structures. The primary endpoint in this trial is continuous and is summarized with means and standard deviations; the registry record does not report a model, a distributional assessment, or an over-dispersion analysis. There is no basis to claim that over-dispersion was present or assessed.

How should planning illustrations be understood?

The record describes planning illustrations based on the observed effect, with a two-sided significance level of 0.05, equal allocation, and a normal approximation. Such calculations are hypothetical planning tools: they estimate how many participants per arm might be needed to detect an effect under the stated assumptions. They are not observed power, do not show the probability that the trial’s finding is true, and do not replace an analysis of the collected data. The stated planning method does not establish that the original trial was designed using those assumptions. The registry record does not report the trial’s own sample-size justification.

Planning estimates that use an observed effect depend on that estimate, which may be imprecise in a small study. If an observed effect is used as the target, the illustration describes a hypothetical future design under its stated assumptions rather than proving that the current study had adequate power. The registry record does not report interim analyses, a statistical analysis plan, or the assumptions used in the original design.

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("Fenofibrate", "No Intervention")
n   <- c(10, 8)
m   <- c(-40, -2)
sp  <- c(12, 16)
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  Fenofibrate  10  -40.000  12.000  -48.584  -31.416
CARM  No Intervention  8  -2.000  16.000  -15.376  11.376

11. Multiplicity, Missing Data and Analysis Populations

The registry record lists one posted secondary outcome, triglyceride concentration percent change from baseline, and three outcome measures. It reports no analyses. The primary endpoint is also described as triglyceride percent change from baseline, so the record does not make clear how the secondary-outcome listing relates to the primary endpoint or to the other outcome measures. The registry record does not report a multiplicity adjustment, a hierarchy of hypotheses, or a plan for interpreting multiple outcomes. It is therefore not possible to assess whether multiplicity was addressed in the trial’s analysis.

Missing-data methods are not reported in the registry record. There is no description of how absent measurements were handled, whether participants were included according to randomized assignment, or whether any imputation or sensitivity analysis was conducted. The posted endpoint summaries have sample sizes of 10 and 8, while overall enrollment is 23; the record does not explain this difference. It also does not identify an intention-to-treat, per-protocol, or other analysis population.

These omissions limit interpretation of the endpoint summaries. If participants with missing measurements differ from those with observed measurements, the available-case summaries may not represent the full randomized groups. That is a general possibility, not evidence that such a pattern occurred in this trial. Without the analysis population and missing-data details, the registry record does not permit assessment of how robust the reported group means are to missing observations.

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 2% reporting threshold.

Most frequent non-serious adverse event terms

Serious adverse events are reported for 0 of 15 participants at risk in the fenofibrate group and 0 of 8 at risk in the no-intervention group. Deaths are reported as 0 in both groups. These figures describe the registry’s recorded counts; they do not show that no other safety events occurred.

Other adverse events are listed for 2 of 15 participants at risk in the fenofibrate group and 0 of 8 at risk in the no-intervention group. The registry record does not provide event terms for these reports in the posted serious-event listing, and it does not describe their severity, timing, duration, or relationship to treatment. The at-risk counts used for safety reporting are not necessarily the same as the endpoint sample sizes: the primary-outcome summaries list 10 and 8 participants. The record does not explain the difference in the fenofibrate counts.

Zero recorded serious events in these groups should be interpreted in the context of the reported at-risk counts and the limited information about event ascertainment. These counts are descriptive, not a statistical comparison of safety between groups. The registry record does not report an inferential safety analysis, and these figures alone do not establish that a treatment is safe or unsafe.

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

The posted results establish that the registry record contains group-level summaries for triglyceride percent change at two months: a mean of -40.0 with standard deviation 12.0 in the fenofibrate group, and a mean of -2.0 with standard deviation 16.0 in the no-intervention group. The endpoint description sets a 25% reduction as its definition of a successful response. The reported summaries do not show how many participants met that definition, nor do they describe each participant’s response.

The record does not report a statistical comparison of the continuous endpoint. It therefore does not provide an estimate of the between-group effect with a confidence interval or a p-value. The observed group means alone cannot establish whether the groups differed beyond what might arise from sampling variability. They also cannot show whether baseline values or other factors influenced the observed changes.

The findings are from a randomized, open-label, parallel-group trial with enrollment listed as 23. The endpoint summaries use smaller listed group counts, and the record does not explain the difference. The lack of a reported analysis population and missing-data method makes it difficult to determine how the endpoint summaries relate to all enrolled participants.

The safety counts report no serious adverse events and no deaths in either group, with other adverse events recorded for 2 participants at risk in the fenofibrate group and none in the no-intervention group. Those counts are not a substitute for detailed event descriptions or a comparative safety analysis. Overall, the registry record offers descriptive outcome and safety information, but leaves important aspects of statistical uncertainty and analysis conduct unreported. It does not, on its own, support a definitive conclusion about treatment efficacy or safety.

14. Why This Trial Matters Statistically

This trial is statistically informative as an example of the distinction between a numerical pattern and an inferential result. The group means differ in their observed direction and size, but a comparative estimate and its uncertainty are not reported. Readers should not treat the visual or numerical separation between two means as a substitute for a confidence interval or a formal comparison.

The endpoint is continuous and defined as percent change from baseline, with a stated response criterion. Yet the posted results provide group means and standard deviations rather than responder counts. Those summaries answer a question about average change, not the proportion of participants achieving the stated criterion. Keeping these estimands distinct prevents a group average from being interpreted as an individual-level success rate.

The record also highlights the value of reporting analysis populations and missing-data methods. Enrollment is listed as 23, whereas endpoint sample sizes are 10 and 8. Without an explanation, readers cannot tell how the summaries relate to the enrolled or randomized groups. A clear account of participant flow, outcome availability, and analysis choices would help readers judge the applicability and robustness of the estimates.

Finally, the serious-event counts are sparse and descriptive. They provide useful registry-level reporting but cannot establish comparative safety or rule out uncommon events. In small clinical studies, precise reporting of denominators, event definitions, outcome timing, and uncertainty is essential to prevent stronger conclusions than the 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 trial-specific limitations follow from what the registry record reports and omits. The study is open label and has no masking. General: when treatment assignment is known, expectations or behavior can influence some aspects of care, measurement, or reporting; whether that affected this endpoint is not reported. The primary outcome is a laboratory measure, but the record does not report details of measurement procedures or quality control.

The primary endpoint is assessed two months after initiating treatment, while the arm descriptions specify a 4-month treatment period. The registry record does not explain how the two-month endpoint relates to the remainder of treatment or whether later outcomes were assessed. Although the record lists three outcome measures and one posted secondary outcome, it does not give enough information to reconstruct the full outcome plan or determine how outcomes were selected for reporting.

The endpoint sample sizes are 10 for fenofibrate and 8 for no intervention, while enrollment is listed as 23. Reasons for the difference are not reported. Participant flow, baseline characteristics, missing-data handling, and the analysis population are also not reported in the registry record. Without these details, the representativeness of the endpoint summaries and potential effects of missing observations cannot be assessed.

The registry record does not report a statistical comparison for the primary continuous endpoint, a confidence interval, or a p-value. It also does not report the original sample-size justification, interim analyses, or a statistical analysis plan. These gaps prevent assessment of the prespecified inferential approach and the precision of a treatment contrast. The small endpoint group counts make clear reporting of these details especially important, but no claims about the trial’s power or validity can be established from the record alone.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Fenofibrate Treatment in SCI — NCT02455336.
• Registry record last updated 2019-06-26; status last verified 2019-05.
• Results first posted 2019-06-26.
• 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

NCT02455336 is a completed randomized, open-label, parallel-group phase 2 and phase 3 trial sponsored by the VA Office of Research and Development. Enrollment is listed as 23. The trial compared once-daily fenofibrate at 145 mg with no intervention, with both arm descriptions specifying 4 months of treatment or no therapy.

The primary endpoint was triglyceride concentration percent change from baseline at two months from initiating drug treatment. The posted mean was -40.0, standard deviation 12.0, and sample size 10 for fenofibrate; the corresponding no-intervention values were -2.0, 16.0, and 8. No two-arm comparison is reported for the continuous endpoint. Serious adverse events and deaths are recorded as 0 in both groups; other adverse events are listed for 2 participants at risk in the fenofibrate group and 0 in the no-intervention group.

The registry record does not report the analysis population, missing-data methods, or a statistical plan. The endpoint summaries are descriptive and do not include a confidence interval or p-value for a treatment contrast.