This page provides an independent statistical analysis and educational interpretation of publicly reported results. ClinicalTrials.gov provides the official trial registry record. Numerical trial results on this page are restricted to the results posted in the ClinicalTrials.gov record; calculations added by Clinical Biostats are labelled as such.
1. Trial at a Glance
This page covers the registry results for Eplerenone for Subclinical Cardiomyopathy in Duchenne Muscular Dystrophy (NCT01521546), a randomized na study. The primary outcome is 12-month Change in Myocardial Strain.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01521546 |
| Phase | NA |
| Condition | Duchenne Muscular Dystrophy |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | Quadruple |
| Enrollment | 42 |
| Arms in the primary outcome | 2 |
| Lead sponsor | Subha Raman |
| Results posted | Yes |
2. Clinical Question
Population
The trial enrolled people with Duchenne muscular dystrophy. The registry describes the concern as early heart muscle changes that may not yet be detected as clinically significant heart disease. The posted primary outcome record does not specify the age range, cardiac imaging eligibility criteria, or other characteristics of the analyzed participants; these details are not reported in the registry record.
Intervention
The intervention was eplerenone, described in the arm information as the active study drug. The registry summary frames the study as a test of early aldosterone antagonism in DMD-related cardiomyopathy. Dose, schedule, treatment duration beyond the outcome’s 12-month timeframe, and permitted concomitant treatments are not reported in the registry record.
Comparator
The comparator was placebo, described in the arm information as a sugar pill. The trial used a randomized, quadruple-masked, parallel design. The registry record does not specify which roles were masked or how the masking was implemented.
Primary question
The primary question was whether the groups differed in 12-month change in myocardial strain, a cardiac MRI measure described as a sensitive measurement of heart function. Change was defined as the 12-month value minus baseline. The posted outcome gives the median change as 1.0 for eplerenone and 2.2 for placebo, with inter-quartile bounds of 0.3 to 2.2 and 1.3 to 3.1, respectively. The record does not provide a reported test or p-value for this comparison.
The clinical motivation in the registry summary is that DMD can affect cardiac muscle, while recognized heart disease may appear later than early myocardial changes. The trial therefore addresses a question about an imaging measure in a population with DMD, rather than a posted measure of symptoms, survival, or a broad clinical outcome. The registry record does not report a threshold for a clinically important strain change, so the clinical meaning of the observed medians cannot be determined from the endpoint values alone.
3. Trial Design
NCT01521546 is titled “Eplerenone for Subclinical Cardiomyopathy in Duchenne Muscular Dystrophy”; its official title is “Early Treatment With Aldosterone Antagonism Attenuates Cardiomyopathy in Duchenne Muscular Dystrophy.” The record lists the study as completed, with a start date of 2012-02 and primary completion in 2016-06. The recorded phase is NA. Enrollment is listed as 42, and Duchenne muscular dystrophy is the condition.
The design is recorded as randomized, quadruple-masked, and parallel, with eplerenone and placebo arms. Randomization means assignment is intended to use a chance-based process; as a general statistical principle, random allocation can reduce systematic differences between groups at baseline. The registry record does not describe the sequence-generation method, allocation concealment, stratification, or the roles included in quadruple masking. The word “parallel” indicates that the arms are compared as concurrent groups rather than as treatment periods within the same participant; the record does not give further operational details.
The posted primary outcome is 12-month change in myocardial strain, defined as the 12-month measure minus baseline and expressed as percent change in heart dimension. The outcome record reports a median of 2.2 for placebo and 1.0 for eplerenone. Each outcome arm lists 20.0 participants. These outcome counts and the overall enrollment are distinct registry entries; the record does not explain their relationship, so no inference about completion, exclusion, or missing observations should be drawn from them.
The registry lists one outcome measure and zero secondary outcomes posted. It records zero analysis entries. The statistical analysis plan, eligibility criteria, study locations, treatment dosing, follow-up procedures, and handling of withdrawals are not reported in the registry record. Those omissions limit reconstruction of how the trial was conducted and how the posted result was generated.
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.
Before assignment: Enrollment was required prior to assignment.
Overall Study
| Stage | Placebo | Eplerenone |
|---|---|---|
| STARTED | 22 | 20 |
| COMPLETED | 20 | 20 |
| NOT COMPLETED | 2 | 0 |
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: Males with Duchenne Muscular Dystrophy
| Characteristic | Unit | Eplerenone | Placebo | Total |
|---|---|---|---|---|
| Participants analysed | 20 | 22 | 42 | |
| Age, Continuous: Age | years | 14.5 (12.0 to 18.5) | 15.0 (11.0 to 19.0) | 15.0 (11.75 to 19.0) |
| Sex: Female, Male: Female | Participants | 0 | 0 | 0 |
| Sex: Female, Male: Male | Participants | 20 | 22 | 42 |
6. Primary Endpoint
The registry lists the primary outcome as 12-month Change in Myocardial Strain, measured over baseline and 12 months.
| Element | Registry entry |
|---|---|
| Result type | Median |
| Measure of spread | Inter-Quartile Range |
| Unit | percent change in heart dimension |
| Endpoint classification | not reduced to a single comparison |
Myocardial strain is described in the registry as a sensitive measurement of heart function assessed using cardiac MRI. The primary endpoint is the change from baseline to 12 months, calculated as the 12-month measurement minus the baseline measurement. Its unit is stated as percent change in heart dimension. A change score expresses how a measurement moved over the stated follow-up rather than reporting only the final measurement. In general, cardiac imaging measures can provide information about organ function that may not be captured by symptoms alone; that general rationale does not establish how well this endpoint predicts later clinical outcomes in this trial.
The outcome is summarized by median and inter-quartile range. The posted median is 2.2 for placebo, with bounds of 1.3 to 3.1, and 1.0 for eplerenone, with bounds of 0.3 to 2.2. The record labels the dispersion as inter-quartile range but does not separately label the posted bounds in the outcome fields. These values describe the reported distributions; they do not by themselves quantify the uncertainty around the difference between group medians.
The registry does not report a threshold that defines a meaningful change in myocardial strain, nor does it state how the imaging measure relates to clinical outcomes for the participants in this trial. The endpoint can therefore be interpreted as a cardiac MRI measure of change over the specified period, but its clinical importance cannot be established from the posted endpoint description alone.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The posted primary outcome is a median change in myocardial strain from baseline to 12 months. The placebo median is 2.2, with reported bounds of 1.3 to 3.1; the eplerenone median is 1.0, with reported bounds of 0.3 to 2.2. The outcome is defined as 12-month value minus baseline and is expressed as percent change in heart dimension. Descriptively, the posted median is lower in the eplerenone arm. The registry record does not provide a comparison estimate, a test statistic, a p-value, or an interval for the between-arm difference, so the precision of that group contrast cannot be assessed from the reported medians alone.
The posted bounds overlap. Because the record describes the summaries as medians with inter-quartile range, these bounds characterize the spread of participant values, not a confidence interval for the treatment contrast. As a general statistical distinction, a confidence interval for a group contrast addresses uncertainty in an estimated difference, while an inter-quartile range describes the middle portion of an observed distribution. The registry does not report a confidence interval for the contrast or the individual-level data needed to reconstruct one reliably.
The outcome record lists 20.0 participants in each arm. Overall enrollment is listed as 42, but the registry does not explain why the posted outcome arm counts differ from the enrollment entry. It would be inappropriate to infer attrition, exclusions, or the analysis population from these entries alone. The record also does not state whether the median summaries are based on all randomized participants, participants with available MRI measurements, or another population.
These are descriptive outcome data, not a complete account of the sponsor’s statistical comparison. The record contains zero analysis entries and does not report the test, adjustment, or model used for the primary outcome. As a result, the displayed medians can be described, but a formal conclusion about the strength of evidence for a group difference cannot be drawn from the registry record alone.
The registry summary gives a biological and clinical rationale for evaluating early treatment, but rationale is not evidence that the posted endpoint changed in a clinically important way. The trial’s primary outcome concerns a cardiac MRI measure over 12 months. The record does not report a threshold for clinical relevance, longer-term outcomes, or whether changes in this measure corresponded to later cardiac events. The endpoint findings should therefore be interpreted within their stated measurement and follow-up, not as proof of an effect on disease progression or clinical outcomes.
The safety entries are also descriptive. The registry lists serious adverse events affecting 1 participant in the placebo group and 0 in the eplerenone group, with other adverse events affecting 2 and 1, respectively. These counts do not establish comparative safety: the record does not provide event definitions, exposure-adjusted rates, attribution, or a complete account of how events were collected. The serious-event term “death due to fat embolism” is listed with 4.5%, but its group attribution and denominator are not specified in the record.
9. Statistical Methods Explained
What does the design contribute?
The study is recorded as randomized, quadruple-masked, and parallel. In general, randomization aims to make treatment assignment independent of participant characteristics, which can support a fair comparison when the process is implemented and analyzed as planned. Masking can reduce the influence of treatment knowledge on care, measurement, or reporting. The registry does not describe the randomization procedure or the people covered by quadruple masking, so those features cannot be assessed in detail. A parallel design compares groups assigned to different arms; the posted outcome is summarized separately for placebo and eplerenone.
These design labels do not replace information about the analysis. For example, a randomized trial still needs a defined analysis population, a method for handling missing outcomes, and a prespecified approach to estimating the group contrast. Those details are not reported in the registry record. The record also does not provide baseline characteristics with which to assess the groups’ observed comparability.
How should the endpoint summary be read?
The outcome is a change score: the measurement at 12 months minus the baseline measurement. It is expressed as percent change in heart dimension and is described as a cardiac MRI measure of heart function. Change scores focus attention on movement from baseline, but interpretation depends on the measurement’s direction, variability, and clinical meaning. The record does not state a threshold for meaningful change or provide details of the measurement procedure.
The summary statistic is the median, and dispersion is identified as the inter-quartile range. In general, the median is the central value after observations are ordered, while the inter-quartile range describes the spread between the central portions of the distribution. These summaries can be useful when values are skewed or when extreme observations would unduly influence a mean. No distributional plot or participant-level values are described in the registry record, so the shape of the observed data cannot be assessed from the registry text alone.
The outcome lists a placebo median of 2.2, with bounds of 1.3 to 3.1, and an eplerenone median of 1.0, with bounds of 0.3 to 2.2. These are summaries within each arm. A formal comparison would need to quantify the difference between arms and its uncertainty using an appropriate method. That comparison method and its uncertainty estimate are not reported in the registry record. The inter-quartile bounds should not be mistaken for a confidence interval around an arm difference.
What statistical test was used?
The registry has zero analysis entries. It does not report whether the primary outcome comparison used a rank-based test, a model, an adjusted analysis, or another method. No p-value for the primary comparison is reported. Without the analysis details and a contrast estimate, the posted medians support a descriptive reading but not a reconstruction of the sponsor’s inferential analysis.
As a general principle, a p-value addresses how compatible data are with a specified statistical model and null hypothesis; it does not measure the size or clinical importance of an effect. A confidence interval for a contrast can communicate both its estimated magnitude and the precision of that estimate. Neither is reported for this primary outcome in the registry record. The record does not state whether any planned interim analyses occurred, or whether stopping rules were used.
What does the planning illustration mean?
The planning assumptions describe an illustration computed from the observed effect, not the trial’s own design. They specify a two-sided significance level of 0.05, equal allocation between arms, and a normal approximation; the effect considered is a stated fraction of the observed effect. The record says that non-time-to-event planning results are expressed as participants per arm. The sample-size values themselves are not included in the registry entries considered for this explanation, so no planning count can be stated.
As a general concept, a planning calculation asks how many participants per arm would be needed under chosen assumptions to detect a target effect with a specified probability, if those assumptions hold. It is not a measure of the probability that the completed trial detected an effect, and it is not observed power. Calculations based on an observed effect are particularly sensitive to uncertainty in that effect estimate; smaller target fractions imply a different planning target, but the registry does not report the numerical fractions or resulting counts. These illustrations should not be treated as the trial’s original sample-size justification, which is not reported in the registry record.
What information is needed to reproduce the analysis?
Reproduction would require details not present in the registry record, including the statistical model or test, the analysis population, the method for handling missing MRI measurements, any baseline adjustment, and the handling of protocol deviations. It would also require a clear definition of the posted bounds and a reported group contrast with its uncertainty. The trial’s record provides outcome summaries and arm counts, but not enough detail to reconstruct the inferential analysis or independently verify its assumptions.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists one outcome measure and zero secondary outcomes posted. It also records zero analysis entries. This means the registry record does not provide a family of reported secondary outcome tests from which to assess multiplicity, but it does not establish what outcomes may have been planned in the protocol or analyzed outside the registry entry. The statistical plan, including any multiplicity strategy, is not reported in the registry record. No claim about adjustment or absence of adjustment can therefore be made.
Missing-data handling is also not reported. The record does not state how missing baseline or 12-month MRI measurements were treated, whether participants were excluded from the outcome summary, or whether any imputation method was used. Although the outcome arm entries list 20.0 participants for each group and enrollment is listed as 42, these entries do not explain the analysis population. They should not be used to infer the number who completed follow-up or the reason for any difference in counts.
The registry does not identify an intention-to-treat, per-protocol, or other analysis population. In general, the analysis population matters because different inclusion rules can lead to different estimates and affect how closely the analysis reflects randomized assignment. The record also does not describe withdrawals, protocol deviations, or sensitivity analyses. Consequently, the extent to which missing outcomes or analysis choices could affect the primary result cannot be evaluated from the registry record.
11. Safety Results
The registry reports serious adverse events as affected participants divided by participants at risk.
| Arm | Participants with serious adverse events | Participants at risk | Percentage |
|---|---|---|---|
| Placebo | 1 | 22 | 4.5% |
| Eplerenone | 0 | 20 | 0.0% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 1% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Placebo | 2 of 22 |
| Eplerenone | 1 of 20 |
Most frequent serious adverse event terms
| Event term | Organ system | Placebo | Eplerenone |
|---|---|---|---|
| death due to fat embolism | Vascular disorders | 1 of 22 | 0 of 20 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Placebo | Eplerenone |
|---|---|---|---|
| flushing | Skin and subcutaneous tissue disorders | 1 of 22 | 0 of 20 |
| anxiety | Psychiatric disorders | 1 of 22 | 0 of 20 |
| headache coincident with seasonal allergies | Nervous system disorders | 0 of 22 | 1 of 20 |
Serious events with the highest arm-level rate: death due to fat embolism (4.5%).
The registry lists serious adverse events affecting 1 participant in the placebo group and 0 in the eplerenone group. It lists other adverse events affecting 2 participants in the placebo group and 1 in the eplerenone group. The at-risk counts shown for the serious and other adverse event entries are 22 for placebo and 20 for eplerenone. These safety counts are reported separately from the primary outcome arm counts, and the registry does not explain the relationship between those entries.
The record also lists “death due to fat embolism” among serious adverse event terms, with 4.5%. It does not identify the group attribution or denominator for that percentage, so it should not be assigned to an arm based on the information recorded. Death counts are not reported in the adverse event fields.
As a general statistical point, small event counts provide limited information about the frequency of uncommon harms. A count alone does not establish whether an event was caused by treatment, and a comparison of counts is not equivalent to a complete risk analysis. The registry does not report event-level timing, severity criteria, attribution, person-time, confidence intervals, or a full description of adverse-event collection. The posted figures should therefore be read as recorded counts, not as a definitive comparison of safety between treatments.
12. What the Result Does, and Does Not, Mean
The registry record documents a randomized comparison of eplerenone with placebo and reports a primary cardiac MRI outcome at 12 months. The median change is 1.0 in the eplerenone arm and 2.2 in the placebo arm. The record describes the measure as percent change in heart dimension and defines change as the 12-month value minus baseline. Those numbers establish what was posted for the arm summaries, but they do not by themselves establish the size or precision of a treatment contrast.
The record does not report a p-value, confidence interval, or other inferential comparison for the primary endpoint. It also does not state a threshold for clinically meaningful change. The posted medians therefore cannot determine whether the difference is statistically persuasive or clinically important. Their inter-quartile bounds describe the distributions as recorded and are not an uncertainty interval for the difference between arms.
The endpoint is a cardiac MRI measure, not a direct report of symptoms, cardiac events, survival, or long-term disease progression. The registry summary explains why early heart muscle changes matter in DMD, but the primary outcome result does not establish that a change in strain leads to a change in those clinical outcomes. The record does not report such outcomes in the posted primary endpoint entry.
The trial’s status is completed, but details needed to interpret the result fully are absent from the registry record: the statistical analysis plan, analysis population, missing-data approach, and group-comparison method. The outcome arm counts and overall enrollment are listed separately, without an explanation of their relationship. These limitations mean that the posted values support a bounded descriptive interpretation rather than a complete assessment of efficacy or safety.
In general, a randomized design can support causal comparison when assignment, follow-up, and analysis are adequately implemented. The registry design labels alone do not show whether every relevant assumption was met. The result should be understood as a reported imaging outcome from this trial, not as advice about treatment or proof of a broader clinical effect.
13. Why This Trial Matters Statistically
This trial is statistically notable for focusing on an imaging measure of cardiac function in Duchenne muscular dystrophy, a setting where the registry summary describes early myocardial changes and later recognition of clinically significant heart disease. The primary outcome is change in myocardial strain assessed by cardiac MRI from baseline to 12 months. That choice makes the measurement target explicit: a change in a physiological imaging measure rather than a direct clinical event.
The record also illustrates the distinction between describing each randomized group and estimating a treatment contrast. It reports medians and inter-quartile bounds for each arm, but does not report a contrast estimate or its uncertainty. Readers can identify the observed arm summaries, yet cannot assess from the registry entry alone how precisely the groups differ or what inferential method was used.
Another statistical issue is transparency about analysis populations and missing observations. The record gives an overall enrollment entry and separate outcome-arm counts, but does not explain how those entries relate. Without a stated population definition and missing-data plan, it is not possible to determine from the registry whether the outcome summaries include all randomized participants or only those with available measurements.
Finally, the planning assumptions are explicitly described as an illustration based on the observed effect, not as the trial’s design rationale. This distinction matters: a retrospective planning calculation does not establish the study’s original target or provide observed power. The registry record is most informative when the design labels, outcome summaries, and omissions are read together, with descriptive evidence kept separate from claims that require an inferential analysis or clinical threshold.
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.
The registry entry does not report the primary comparison’s statistical test, model, effect estimate, p-value, or confidence interval. As a result, the arm-level medians cannot be translated into a formal estimate of the between-group difference or its precision. The record labels the summaries as medians with inter-quartile range, but does not separately identify the posted bounds in the outcome fields.
The outcome counts and enrollment entry are not reconciled. Enrollment is listed as 42, while each arm’s primary outcome entry lists 20.0 participants. The record does not say whether this reflects missing measurements, exclusions, a particular analysis population, or another registry convention. No explanation should be inferred from the counts alone.
Important design and conduct details are not reported in the registry record, including eligibility criteria, participant characteristics, dosing, treatment adherence, randomization procedures, masking roles, and follow-up procedures. The record does not give a missing-data method, an analysis population, or information about protocol deviations. These omissions limit assessment of bias and reproducibility.
The clinical interpretation is also constrained: no threshold for meaningful change in myocardial strain is reported, and the primary outcome entry does not connect strain changes to clinical events or longer-term outcomes. Safety reporting is limited to event counts and a serious-event term with a percentage whose denominator and group attribution are not specified. The record does not provide enough detail to estimate comparative safety precisely.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Eplerenone for Subclinical Cardiomyopathy in Duchenne Muscular Dystrophy — NCT01521546.
- Registry record last updated 2016-11-08; status last verified 2016-10.
- Results first posted 2015-05-27.
- 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.
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18. Record Summary
NCT01521546 is a completed randomized, quadruple-masked, parallel trial of eplerenone versus placebo in Duchenne muscular dystrophy. Enrollment is listed as 42. The primary endpoint is 12-month change in myocardial strain, defined as the 12-month measure minus baseline and summarized by median and inter-quartile range.
The posted median is 2.2 for placebo, with bounds of 1.3 to 3.1, and 1.0 for eplerenone, with bounds of 0.3 to 2.2. Each outcome arm lists 20.0 participants. The registry record does not report a formal between-arm analysis, p-value, confidence interval, analysis population, or missing-data method. Its safety entries list serious and other adverse-event counts by group, but do not provide enough detail for a complete comparative safety assessment.