Study Testing Benefits of Ursolic Acid (UA) as a Countermeasure To Myopenia and Insulin Resistance in Chronic Spinal Cord Injury (SCI): Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Study Testing Benefits of Ursolic Acid (UA) as a Countermeasure To Myopenia and Insulin Resistance in Chronic Spinal Cord Injury (SCI) (NCT05776862), a randomized phase 2 study. The primary outcome is Change in Muscle Mass Using Dual X-ray Absorptiometry (DXA).

2. Clinical Question

3. Trial Design

This is a completed, randomized, parallel-group Phase 2 pilot study sponsored by the University of Miami. The registry record lists an enrollment of 10. The study started on 2023-03-27, and the primary completion date was 2025-01-29. Masking is listed as none.

The intervention descriptions identify two groups: participants with paraplegia taking UA while participating in exercise, and participants with tetraplegia taking UA. Both descriptions specify four capsules twice daily for 12 weeks; the paraplegia group is additionally described as exercising three times per week. The registry record does not describe a placebo or a group that did not receive UA.

Although allocation is recorded as randomized, the method of randomization and the process used to assign participants are not reported in the registry record. Nor does the record explain how random allocation relates to the distinct paralysis types and exercise participation in the two posted groups. Accordingly, the group results should not be read as a straightforward randomized comparison of UA against another treatment. They describe outcomes in groups that differ in more than the intervention exposure being considered.

The primary completion date indicates when the primary outcome period was recorded as complete; it does not by itself establish when results were analyzed or submitted. The registry record lists four outcome measures, but no secondary results are posted. No analysis is listed in the registry record.

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.

6. Primary Endpoint

The registry lists the primary outcome as Change in Muscle Mass Using Dual X-ray Absorptiometry (DXA), measured over Baseline, 12 weeks.

The primary endpoint is change in muscle mass measured by dual X-ray absorptiometry, or DXA, and reported in grams. The specified measurement points are baseline and 12 weeks. DXA estimates body composition from measurements of how tissues absorb X-rays; in this study, the chosen measure is intended to quantify muscle mass rather than directly measure strength or physical function.

Change from baseline focuses on how the measured value differs between the start and end of the intervention period. Reporting the mean summarizes the average change among the participants included in each group’s posted result. The standard deviation (SD) describes how much individual changes varied around that group mean. A large SD relative to a mean can indicate substantial variation, although the SD alone does not establish why participants differed.

Muscle mass is relevant to the study’s stated focus on muscle loss in chronic spinal cord injury. It is nevertheless a surrogate measurement: a change in DXA-measured muscle mass is not itself a measurement of insulin resistance, blood sugar control, strength, or everyday function. Those outcomes require their own measures and results. The primary endpoint results therefore address the recorded change in muscle mass only, and should not be extended to other outcomes.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What does the mean change represent?

For a continuous measurement such as muscle mass in grams, a mean change summarizes the average difference between measurements at the specified time points among participants included in a group’s result. Here the registry specifies baseline and 12 weeks. The posted mean is -183.0 grams for UA with exercise in the paraplegia group and 1434.0 grams for UA in the tetraplegia group. The negative sign indicates a mean change in the decreasing direction; the positive value indicates a mean change in the increasing direction. These descriptions concern the recorded measure, not a clinical judgment about benefit.

General statistical context: a mean is a useful summary when the question concerns an average, but it does not show the range or shape of individual outcomes. Participants may have changes above or below the mean, and the mean alone does not reveal how many changed in either direction. Individual outcome data and their distribution are not reported in the registry record.

How should the standard deviation be read?

The reported SD is a measure of dispersion: it summarizes the spread of individual observations around the group mean. The SD is 2643.0 grams in the UA and exercise paraplegia group and 1885.0 grams in the UA tetraplegia group. It is not the standard error, a confidence interval, or an estimate of the treatment effect. An SD can be large even when the mean is near zero, and it does not by itself indicate whether a mean difference is statistically distinguishable from chance.

General statistical context: when outcomes are highly variable, a group mean can provide an incomplete picture, especially with few observations. A confidence interval can express uncertainty around an estimated mean or contrast, but no confidence interval for this endpoint is reported in the registry record. The posted SDs should not be treated as substitutes for such intervals.

What comparison is supported by these results?

The primary results are descriptive means for two groups. Both groups received UA, while one group is described as exercising and the other is not. The groups also represent different paralysis types. Thus, a direct subtraction of the means would not create a clean estimate of UA’s effect: there is no posted group without UA, and group membership bundles differences in paralysis type and exercise participation. No adjusted effect estimate or statistical test is reported in the registry record.

The registry classifies allocation as randomized, but the randomization method and its relationship to the two differently defined groups are not reported in the registry record. Randomization can, in general, help balance participant characteristics between treatment groups when it assigns participants from a common eligible population to different interventions. The registry description does not establish that this is the contrast represented by the two posted groups. Therefore, the word randomized alone is not enough to interpret the reported means as a randomized UA treatment effect.

Why does over-dispersion matter?

General statistical context: over-dispersion refers to variability greater than a model would expect under its assumptions. For continuous outcomes, the broader issue is that observed changes can be much more variable than a simple average suggests. When variability is high relative to the number of observations, estimates may be unstable and uncertainty can be substantial. The reported SDs communicate observed spread, but the registry does not report a model, a variance assessment, or an inferential method for the endpoint. It is not possible to determine from the registry whether a formal over-dispersion assessment was made.

A statistical model is only as informative as its assumptions and the design it represents. For example, an analysis comparing group means would need to define the intended contrast and address the groups’ distinct characteristics. The registry record does not report such a model or its assumptions. No model-based conclusion should therefore be inferred from the descriptive outcome values alone.

What can the planning illustrations say?

General statistical context: sample-size planning illustrations can show how the number of participants required for a future study depends on a target effect, variability, significance level, and allocation assumptions. They are not estimates of what a completed trial achieved, and they are not observed power. The planning assumptions described for the page use the observed effect as a basis, equal allocation, a two-sided significance level of 0.05, and a normal approximation, with the target effect expressed as a fraction of the observed effect.

Such illustrations are hypothetical planning aids, not the trial’s design rationale. The registry record does not report a sample-size justification or its own planning assumptions. No sample-size calculation should be read as evidence that the completed study had a particular power or that its results establish an effect.

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("UA and Exercise in Paraplegia Group", "UA in Tetraplegia Group")
n   <- c(4, 4)
m   <- c(-183, 1434)
sp  <- c(2642.65, 1885.21)
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  UA and Exercise in Paraplegia Group  4  -183.000  2642.650  -4388.046  4022.046
CARM  UA in Tetraplegia Group  4  1434.000  1885.210  -1565.790  4433.790

11. Multiplicity, Missing Data and Analysis Populations

The registry record lists four outcome measures, but it posts no secondary outcome results. No analysis is listed in the registry record. The analysis plan, any hierarchy among outcomes, and any correction for multiple testing are not reported in the registry record. Multiplicity arises when several outcomes or comparisons are tested: conducting multiple tests can increase the chance of at least one apparently notable result by chance. Whether this concern affected the study’s analysis cannot be determined from the posted information.

Missing-data methods, the number of participants with available primary endpoint measurements, and the reasons for any missing measurements are not reported in the registry record. The primary outcome lists four participants in each group, while total enrollment is 10. The record does not explain how the endpoint counts relate to all enrolled participants, or whether any observations were excluded or unavailable.

The registry record also does not report an analysis population, such as whether results include all randomized participants, only participants who completed the intervention, or another defined group. The handling of adherence, withdrawals, and protocol deviations is not reported in the registry record. These details affect how an estimate should be interpreted, because analysis of a subset can differ from an analysis that preserves the randomized groups.

Without a posted statistical analysis plan, missing-data description, or analysis population, it is not possible to assess whether the primary endpoint analysis followed prespecified procedures or how missing observations were handled. These are reporting gaps, not proof that the study did not address these issues outside the registry.

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 serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: urinary tract infection (20.0%).

Serious adverse event figures are counts among participants listed as at risk, not estimates of treatment efficacy. The registry reports five participants at risk for serious adverse events in each group. There were zero serious adverse events in the UA and exercise paraplegia group and one in the UA tetraplegia group. Deaths were zero in both groups.

Urinary tract infection is listed among the serious adverse event terms at 20.0%. The registry record does not clarify the term’s group attribution in the information. The listed at-risk counts and event counts should not be used to infer comparative risk beyond the posted figures.

The registry also reports other, non-serious adverse events: three among five participants at risk in the UA and exercise paraplegia group, and two among five at risk in the UA tetraplegia group. The primary endpoint group counts are four per group, whereas the safety figures list five at risk in each group. The registry record does not explain the difference in denominators or describe event severity beyond the serious and other categories.

General statistical context: safety counts in a small study can be useful for describing what was reported, but they do not precisely characterize uncommon events or establish a comparative safety profile. The figures should be read as registry reporting for this study, not as a complete account of all possible risks.

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

The posted results describe mean changes in DXA-measured muscle mass from baseline to 12 weeks among participants represented in each group’s outcome data. The mean change was -183.0 grams in the UA and exercise paraplegia group and 1434.0 grams in the UA tetraplegia group. The reported SDs, 2643.0 grams and 1885.0 grams respectively, indicate variation in participant changes around those means. The registry does not post a confidence interval or a statistical test for this endpoint.

These values do not establish what would have happened to similar participants without UA. Both groups received UA, and the groups differ in paralysis type as well as in the described exercise participation. The observed results therefore cannot separate the effects of UA, exercise, and group characteristics. The registry record also does not report an adjusted comparison that would address those differences.

The endpoint is muscle mass, not blood sugar control or insulin resistance. Although the study’s stated purpose includes blood sugar control, results for that outcome are not posted in the registry record. The listed muscle-mass changes should not be taken as evidence about glucose-related outcomes, strength, function, or other unreported measures.

The results also do not prove that no effect exists or that a particular effect is present. The small endpoint group counts, sizeable observed spread, and lack of reported inferential results limit what can be concluded about precision. These are descriptive registry results from a pilot study, and they do not by themselves establish a causal treatment effect or support a broader claim about outcomes in people with spinal cord injury.

14. Why This Trial Matters Statistically

This study illustrates why a reported group mean needs to be interpreted alongside its design, outcome definition, and variability. The registry gives an average change in muscle mass for each group, but the groups are defined by different paralysis types and different exercise participation. That makes the contrast difficult to interpret as an isolated treatment comparison, even though allocation is listed as randomized. The randomization procedure and its relation to the group definitions are not reported in the registry record.

The endpoint also shows why descriptive statistics and inferential statistics answer different questions. A mean describes the average observed change, and an SD describes variation among observations. Neither alone supplies the uncertainty around a group estimate or establishes whether groups differ. The registry record posts no confidence interval, test, or analysis for the primary outcome, so the precision of any comparison cannot be assessed using reported inferential results.

The difference between enrollment of 10 and four endpoint participants reported in each group makes transparent accounting important. The registry does not explain the relationship between these counts, whether all enrolled participants contributed endpoint measurements, or how missing outcomes were handled. Such details help readers evaluate the population represented by an analysis and the potential for bias from unavailable data.

Finally, the study’s stated interest includes muscle loss and blood sugar control, but the posted primary results concern DXA-measured muscle mass only. Keeping the endpoint distinct from the broader research motivation prevents conclusions from extending beyond the outcome with posted results. These are important considerations when reading pilot studies, where clear description of the estimand, analysis population, and uncertainty is particularly valuable.

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.

The primary endpoint has four participants reported in each group, despite total enrollment of 10. The registry record does not explain the endpoint counts, the number of participants who completed follow-up, or the reasons why other enrolled participants are not represented in the outcome counts. This limits assessment of missingness and of how closely the posted results reflect the enrolled group.

The intervention groups also differ in paralysis type and exercise participation. Both received UA, and the registry does not post a UA-free comparison group. The randomized allocation label is not accompanied by a randomization method or an explanation of how allocation produced these groups. As a result, the posted data do not isolate the effect of UA or exercise.

The primary outcome is reported as a mean and SD without a confidence interval, test, or adjusted analysis. Individual observations and their distributions are not reported in the registry record. The relatively broad observed variability, together with the small endpoint group counts, makes it difficult to characterize the precision or stability of the means.

The registry record does not report a sample-size justification, statistical analysis plan, missing-data method, or analysis population. It lists four outcome measures but posts no secondary outcome results, so the scope of available findings is limited. Safety figures also use five participants at risk per group, while the primary outcome reports four participants per group; the registry does not explain the differing counts.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Study Testing Benefits of Ursolic Acid (UA) as a Countermeasure To Myopenia and Insulin Resistance in Chronic Spinal Cord Injury (SCI) — NCT05776862.
• Registry record last updated 2025-05-21; status last verified 2025-05.
• Results first posted 2025-05-21.
• 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

NCT05776862 is a completed randomized, unmasked, parallel-group Phase 2 pilot study sponsored by the University of Miami. It enrolled 10 participants and studied UA in groups described as paraplegia with exercise and tetraplegia without the described exercise program. Both UA regimens were listed as four capsules twice daily for 12 weeks.

The primary outcome was change in DXA-measured muscle mass from baseline to 12 weeks. The posted mean changes were -183.0 grams (SD 2643.0) in the UA and exercise paraplegia group and 1434.0 grams (SD 1885.0) in the UA tetraplegia group, with four participants reported in each group. No analysis is listed in the registry record, and no secondary results are posted. The registry reports zero serious adverse events in the first group and one in the second, with zero deaths in both groups.

The posted outcome values are descriptive and do not isolate a UA effect because both groups received UA and differ in paralysis type and exercise participation. The registry record does not report a confidence interval, statistical test, missing-data method, analysis population, or sample-size justification.