Cardiomyopathy in DMD: Lisinopril vs. Losartan: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Cardiomyopathy in DMD: Lisinopril vs. Losartan (NCT01982695), a randomized na study. The primary outcome is Cardiac Ejection Fraction as Measured by Echocardiogram.

2. Clinical Question

The trial asks whether the mean cardiac ejection fraction measured at the 12 month study visit differs between people with Duchenne muscular dystrophy and cardiomyopathy assigned to lisinopril or losartan. The registry identifies ejection fraction as the primary outcome and describes the outcome population as patients who received a 12 month echocardiogram evaluation.

This is an active-treatment comparison: both arms received a named medication, and the central statistical question is comparative rather than whether either treatment differs from no treatment. The posted endpoint summaries give an arm mean and standard deviation for each group, but the registry states that values are missing or not estimable for a two-arm comparison of this continuous endpoint. As a result, the record does not provide an inferential estimate of the between-group difference or its uncertainty.

3. Trial Design

The registry describes NCT01982695 as a completed, randomized, parallel-group trial comparing lisinopril with losartan for cardiomyopathy in Duchenne muscular dystrophy. Enrollment is listed as 23. The trial is classified as quadruple-masked; the brief summary also describes it as double-blind. The registry does not explain which people or roles were included in the masking classification, or how masking was maintained.

The recorded start is 2009-03, and primary completion is listed as 2012-08. Nationwide Children's Hospital is the sponsor. The phase entry is NA. These details describe the registration record and do not establish how participants were recruited, how allocation was concealed, or how treatment was implemented. Those details are not reported in the registry record.

The two interventions are lisinopril and losartan. Arm descriptions are empty, so details such as dose, titration, treatment schedule, adherence, and concomitant care are not reported in the registry record. The record identifies one primary outcome measure: cardiac ejection fraction measured by echocardiogram at the 12 month visit. It reports group summaries for participants with that evaluation, with n 7 in the lisinopril group and n 9 in the losartan group. The registry enrollment count and the endpoint-specific group counts refer to different parts of the record; participant flow and the relationship between them are not explained.

Random allocation can support a fair comparison by reducing systematic differences between groups at the point of assignment, while masking may reduce some influences on care or outcome assessment. These are general design principles, not confirmation of how successfully those features operated in this trial. The record does not report allocation procedures, masking details, protocol deviations, or a participant-flow account.

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: All patients that received a 12 month echocardiogram evaluation

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 Cardiac Ejection Fraction as Measured by Echocardiogram, measured over 12 month visit.

The primary endpoint is cardiac ejection fraction measured by echocardiogram at the 12 month study visit. The registry describes it as the mean percentage of blood leaving the heart and says measurements used the biplane Simpson's rule with images obtained from apical 4 chamber views. The recorded summary measure is a mean, with standard deviation as the measure of spread. The outcome population is described as all patients who received a 12 month echocardiogram evaluation.

Ejection fraction is a continuous measurement: it can take a range of values, rather than being limited to categories such as event or no event. Reporting a group mean gives a measure of the group's average value, while the standard deviation describes how dispersed individual measurements are around that average. Together, they offer a basic description of the endpoint in each arm. They do not by themselves establish whether the groups differ beyond what could plausibly arise from sampling variation.

In this record, the lisinopril group mean is 54.6 with standard deviation 5.19, and the losartan group mean is 55.2 with standard deviation 7.19. These are descriptive summaries among participants with the recorded 12 month evaluation. The registry does not provide an estimable two-arm comparison, a confidence interval, or a p-value for this endpoint. The summaries therefore describe the observed groups but do not quantify the precision of a treatment contrast.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry reports a continuous primary outcome summarized by group means and standard deviations. It does not identify a statistical test or model for the comparison. The concepts below explain how such summaries and common inferential methods are generally interpreted; they do not describe an analysis confirmed to have been used in this trial.

What does a mean describe?

A mean is the arithmetic average of the recorded measurements in a group. It gives a compact description of the group's central value, but it does not show every participant's measurement or the shape of the distribution. Different sets of observations can have the same mean while differing in their spread or distribution. For that reason, the mean is most informative when read alongside a measure of variability and the number of observations contributing to it.

The registry reports a mean of 54.6 for lisinopril and 55.2 for losartan. These are summaries for the outcome population recorded in the endpoint description, not adjusted estimates. The registry does not state whether any covariates were used in an analysis or whether the reported summaries were adjusted for baseline measurements.

What does the standard deviation describe?

A standard deviation describes the spread of individual values around a group's mean. A larger standard deviation indicates more variability among the recorded observations, in the same units as the measured outcome. It does not directly measure the uncertainty in the mean or in a difference between groups. The standard deviations reported are 5.19 for lisinopril and 7.19 for losartan. The registry does not provide individual-level measurements or distribution plots, so readers cannot assess the observations' shape, unusual values, or whether a model's assumptions would be plausible.

How does a group comparison differ from descriptive summaries?

A treatment comparison generally targets a contrast between groups, such as the difference in their mean outcomes. An inferential analysis combines that contrast with information about variability and sample size to express uncertainty. A confidence interval is one general way to show that uncertainty: it gives a range of values compatible with the observed data under the analysis assumptions. A hypothesis test and p-value address a related but different question, evaluating data against a specified null hypothesis. Neither a confidence interval nor a p-value is reported for the primary comparison in this record.

The registry's explicit statement that a two-arm comparison is not estimable is important. The displayed arm summaries alone do not permit the missing inferential result to be treated as if it had been posted. A recalculation from summary data would require appropriate inputs and assumptions, and it would not replace a sponsor analysis if the registry indicated a discrepancy. The record does not flag a discrepancy, but it also does not give a recalculated comparison. No treatment contrast should be invented from the group summaries.

Why do analysis assumptions matter?

Many common methods for comparing means rely on assumptions about the observations, such as independence between participants and a suitable model for the distribution of outcomes or the sampling distribution of the mean. In general, departures from assumptions can make standard errors, confidence intervals, or tests less reliable. With limited observations, individual values may have substantial influence on a mean, and it can be difficult to assess distributional assumptions from group summaries alone. These general considerations are relevant to continuous outcomes, but the registry does not say which method or assumptions were used for this trial's comparison.

Randomization is intended to make assigned groups comparable on average at baseline. It does not guarantee that every characteristic is balanced in a particular trial, especially when the analyzed outcome groups are small. Masking can reduce some forms of measurement or treatment-related bias, but the registry's masking label does not identify which roles were masked or document whether masking was maintained. These design features do not resolve uncertainty created by unavailable outcome data or a non-estimable comparison.

What do the planning assumptions mean?

The registry record describes planning illustrations based on the observed effect, using a two-sided significance level of 0.05, equal allocation between arms, and a normal approximation. The target effect in those illustrations is a stated fraction of the observed effect. Such calculations are planning scenarios, not evidence about the trial's observed ability to detect an effect. A sample-size illustration is not observed power and does not repair the absence of an estimable comparison. The record does not report the trial's original sample-size justification or confirm that these illustration assumptions were part of the trial's design.

The record's stated planning convention distinguishes participants per arm for non-time-to-event outcomes from total events for time-to-event outcomes. The primary outcome is continuous, not time-to-event. The planning details do not supply a primary comparison estimate, a confidence interval, or a p-value, and none can be inferred from the assumptions alone.

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("Lisinopril", "Losartan")
n   <- c(7, 9)
m   <- c(54.6, 55.2)
sp  <- c(5.19, 7.19)
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  Lisinopril  7  54.600  5.190  49.800  59.400
CARM  Losartan  9  55.200  7.190  49.673  60.727

11. Multiplicity, Missing Data and Analysis Populations

The registry lists one outcome measure, the primary cardiac ejection fraction endpoint, and reports zero posted secondary outcomes. It records zero analyses. These entries indicate what is posted in the registry record; they do not establish that no additional outcomes or analyses were considered outside the record. The statistical plan for selecting, adjusting, or interpreting multiple analyses is not reported in the registry record.

Missing-data handling is also not reported in the registry record. The endpoint population is described as all patients who received a 12 month echocardiogram evaluation, and the group summaries list n 7 for lisinopril and n 9 for losartan. The record does not explain why other enrolled participants are not represented in those summaries, whether an assessment was missed, or how missing measurements were handled. It does not report an intention-to-treat analysis, a per-protocol analysis, imputation, or sensitivity analyses.

These omissions matter because the observed-evaluation group may not represent everyone assigned to treatment if availability of a 12 month measurement is related to participant characteristics or outcome. That is a general concern, not a claim that such a relationship occurred in this trial. Without participant flow and a stated analysis population beyond the evaluation-based description, readers cannot determine how the primary summaries relate to all randomized participants.

Multiplicity procedures are not reported in the registry record. Since no secondary outcomes or analyses are posted, the record does not allow an assessment of how multiple testing may have been handled, or whether unposted analyses influenced interpretation. The absence of those details should not be mistaken for evidence that no multiplicity issue existed.

12. 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 5% reporting threshold.

Most frequent non-serious adverse event terms

The adverse-event record lists serious events in the group at risk as 0 among 12 for lisinopril and 0 among 10 for losartan. For the separate category of other adverse events, it lists 0 among 12 for lisinopril and 2 among 10 for losartan. These are the counts recorded for the adverse-event groups; they are not the same group counts as those listed for the primary echocardiogram endpoint.

Counts are descriptive. They do not by themselves establish whether an event was caused by a treatment, or how precisely the underlying event frequency is estimated. The record does not provide event descriptions for the other adverse events, their severity, timing, relationship to treatment, or the period over which events were collected. Deaths are listed as null, so death information is not reported in the registry record.

The adverse-event denominators differ from the endpoint-specific counts, and the record does not explain participant flow or the relationship between these populations. The listed counts should therefore be read within their own adverse-event groups, not as a complete safety comparison for everyone enrolled. No broader safety conclusion follows from these counts alone.

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

The posted results provide descriptive information about mean cardiac ejection fraction among participants with a recorded 12 month echocardiogram evaluation. The reported means are 54.6 for lisinopril and 55.2 for losartan, with standard deviations of 5.19 and 7.19. The registry does not provide an estimable two-arm comparison for this continuous endpoint. It therefore does not show an inferential estimate of the treatment contrast or how precisely that contrast is known.

These results do not establish that one treatment is superior, that the treatments are equivalent, or that either treatment changes ejection fraction. Equivalence is a specific statistical conclusion requiring an appropriate design, a prespecified margin, and an analysis; none of these is reported for this comparison. Similarly, the absence of a reported test or confidence interval is not evidence that there is no difference.

The endpoint summaries also do not answer questions about outcomes outside the recorded 12 month evaluation, or about skeletal muscle effects, functional outcomes, or other measures not posted as trial outcomes. The registry record does not report secondary outcome results. The adverse-event counts offer limited descriptive information and do not establish causality or provide a complete safety assessment.

The result should be understood as a limited registry record: it documents arm-specific outcome summaries and states that a two-arm comparison is not estimable. The reason for that status, the missing-data process, participant flow, and the planned analysis are not reported in the registry record. Those gaps limit what can be concluded about the comparative result. The numerical summaries should not be read as a treatment recommendation or as evidence for decisions about an individual.

14. Why This Trial Matters Statistically

This trial is statistically notable because it is a randomized comparison of two active treatments, yet the registry's primary outcome entry does not provide an estimable two-arm comparison. It illustrates the difference between having descriptive summaries for each arm and having an inferential result that directly addresses the comparative question. Means and standard deviations characterize the recorded outcome groups; they do not alone establish the size or precision of a treatment contrast.

The endpoint counts also draw attention to the analysis population. The trial enrollment entry is 23, while the primary outcome summaries report n 7 and n 9 in the respective arms. Those values should not be combined or treated as a complete participant-flow account. The registry does not explain why the endpoint summaries include these participants, nor how they relate to all enrolled or randomized participants. Clear accounting of who contributed to an outcome analysis is essential to interpreting its relevance.

Another statistical lesson is that a completed randomized trial can still have an incomplete public analysis record. The registry posts one outcome measure, no secondary outcomes, and no analyses, while leaving the statistical comparison and missing-data approach unreported. These details constrain independent assessment and make it difficult to distinguish an unavailable analysis from an analysis that was never planned or performed.

Finally, planning illustrations should be distinguished from trial results. The registry describes calculations based on an observed effect and stated assumptions, but such illustrations are prospective planning scenarios, not observed power and not a substitute for the missing comparative estimate. This distinction helps prevent a planning calculation from being mistaken for evidence about what the trial found.

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 most direct limitation is that the registry states the primary continuous endpoint has no values that are estimable for a two-arm comparison. The arm means and standard deviations are posted, but no treatment contrast, confidence interval, or p-value is given. This leaves the main comparative question unresolved in the registry record.

The primary outcome summaries are based on the evaluation-defined population, with n 7 for lisinopril and n 9 for losartan. The registry enrollment is 23, but it does not report participant flow or explain the relationship between enrollment and the endpoint counts. Reasons for unavailable evaluations, exclusions, withdrawals, and missing-data methods are not reported in the registry record. The possibility of selection related to outcome availability cannot be assessed.

Other trial details that would help interpret the results are also absent: dose and treatment schedule, adherence, allocation concealment, the roles included in masking, baseline endpoint values, and the statistical analysis plan are not reported in the registry record. The registry does not report whether the primary comparison was adjusted for baseline measures or other characteristics. Arm descriptions are empty.

The record posts no secondary outcome results and no analyses. This limits assessment of outcomes beyond ejection fraction and of any prespecified multiplicity strategy. Safety descriptions are also brief: the other adverse events are not named, deaths are not reported, and the adverse-event collection period and event attribution are not specified. These limitations apply to what the registry record documents and should not be taken as proof that unreported procedures or analyses did not occur.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Cardiomyopathy in DMD: Lisinopril vs. Losartan — NCT01982695.
• Registry record last updated 2017-03-21; status last verified 2017-01.
• Results first posted 2017-03-21.
• 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

NCT01982695 is a completed randomized, parallel-group comparison of lisinopril and losartan for cardiomyopathy in Duchenne muscular dystrophy. The registry lists enrollment of 23, quadruple masking, Nationwide Children's Hospital as sponsor, a start of 2009-03, and primary completion in 2012-08. The phase entry is NA.

The primary outcome is mean cardiac ejection fraction by echocardiogram at the 12 month visit. The posted summaries are 54.6 with standard deviation 5.19 for lisinopril, n 7, and 55.2 with standard deviation 7.19 for losartan, n 9. The registry states that a two-arm comparison is not estimable. No secondary outcomes or analyses are posted. Participant flow, missing-data handling, and the planned statistical analysis are not reported in the registry record.