A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I): Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I) (NCT03783923), a na phase 3 study. The primary outcome is Change From Baseline in Time to Climb 4 Stairs After 26 Weeks of Treatment With Deflazacort.

2. Clinical Question

3. Trial Design

The registry describes a Phase 3, multicenter, open-label, single-group study of deflazacort in participants with LGMD2I. It records allocation as not applicable and masking as none. With no randomized comparison recorded, the study design does not support a direct treatment-versus-control estimate from the information available in the registry. The enrollment was 11, and the study status is terminated. The reason for termination is not reported in the registry record.

The planned regimen was oral deflazacort at 0.6 milligrams per kilogram per day, with the possibility of dose reduction for tolerability issues. The brief summary describes a screening visit and additional visits after 1, 13, and 26 weeks of treatment for most participants. The primary endpoint timeframe is baseline and Week 26. The record does not state whether every participant completed those visits or received treatment for the full planned period.

The registry lists a start date of 2019-10-31 and a primary completion date of 2021-01-01. These dates describe the trial record; they do not establish the timing of any particular participant's follow-up. The registry lists 15 outcome measures, including the primary stair-climbing measure and secondary measures of lung function, timed movement, muscle strength, and muscle imaging. It records 0 analyses. Consequently, the available trial description supplies design and outcome definitions, but not a reported estimate of the primary treatment-period change.

The arm description contains a reference to participants assigned to placebo before a protocol amendment and an option to consent to an unspecified arrangement. The text is incomplete, and the overall design is recorded as single-group. The registry record does not report enough detail to reconstruct the amendment's effects on participant allocation or to treat this study as a randomized comparison.

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: Participants enrolled under protocol v3.0 were 1:1 randomized to get placebo or deflazacort. After protocol v4.0, the study became an open-label study and all participants received deflazacort. Per planned analysis, participants who took at least 1 dose of deflazacort were pooled in the safety and efficacy summaries.

Placebo-Controlled Period (26 Weeks)

Reasons for not completing this period:

Open-Label Extension (26 Weeks)

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: Safety population included all enrolled participants who received at least 1 dose of deflazacort. Here, 'Number analyzed' = participants evaluable at specified timepoint.

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: Enrolled population included all informed-consented participants who met all inclusion and exclusion criteria.

6. Primary Endpoint

The registry lists the primary outcome as Change From Baseline in Time to Climb 4 Stairs After 26 Weeks of Treatment With Deflazacort, measured over Baseline, Week 26.

The primary endpoint is change from baseline in time to climb 4 stairs after 26 weeks of deflazacort treatment. It is measured in seconds, with the registry specifying a mean and standard deviation and giving baseline and Week 26 as the measurement timepoints. In general, a timed task records how long a participant takes to complete a defined activity; a change measure compares follow-up with baseline. The registry record does not define the sign convention for change, so a positive or negative value cannot be interpreted from the endpoint description alone.

In general, stair-climbing time can be used as a functional performance measure because it captures the time needed to complete a specified movement task. Repeated measurement allows researchers to describe whether the recorded time differs from baseline during the study period. This endpoint is one component of the planned assessment, rather than a complete description of all aspects of LGMD2I or participant experience.

The endpoint definition does not by itself indicate what happened during the study. The registry record reports no primary-outcome analysis, so no mean change, standard deviation, confidence interval, or test result is available in the record. It also does not report how many participants contributed endpoint values at baseline or Week 26. The primary population description says that the number analyzed means participants evaluable at the specified timepoint, but gives no timepoint-specific counts.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The primary endpoint is continuous: it is a time measured in seconds, and the registry specifies a mean and standard deviation as the summary form. In general, a mean describes the central value of a set of measurements, while a standard deviation describes how dispersed individual measurements are around that mean. These summaries can help characterize a group, but they do not replace the individual-level data or explain why participants' measurements differ.

What does a change-from-baseline measure represent?

In general, a change score compares a participant's measurement at follow-up with that participant's baseline measurement. A group summary can then describe the distribution or average of those changes. The registry specifies baseline and Week 26 for the primary outcome, but does not state the arithmetic convention used to define change. Without that convention and the actual estimate, the direction of a hypothetical value should not be assigned a clinical meaning. The record also does not report whether the analysis used a particular model or adjusted for any baseline factors.

How should mean and standard deviation be read?

In general, the mean gives a summary of the location of measurements, and the standard deviation describes their spread in the measurement's units. For a timed endpoint, the units remain seconds. A standard deviation is not the same as a standard error or confidence interval: the standard deviation concerns variation among observations, whereas a standard error concerns uncertainty in an estimated quantity under a statistical model. The registry's endpoint description names the mean and standard deviation but reports neither numerical value for the primary result.

A confidence interval is a general statistical tool for expressing uncertainty around an estimate. Its width depends on factors such as the amount of information available and the variability of the measured outcome, as well as the analysis method. A narrow interval usually indicates greater precision than a wide interval under the same framework. No confidence interval for the primary endpoint is reported in the registry record, so precision cannot be evaluated from that source.

What does the single-group design imply?

In general, a single-group study can describe outcomes observed among participants receiving the intervention, but it does not provide the same direct contrast as a concurrent control group. An observed change between baseline and follow-up can reflect multiple influences, and a within-group comparison cannot by itself identify which influence produced the change. This is a design principle, not a statement that any particular influence occurred in this trial. The registry records a single-group model, allocation as not applicable, and no masking.

Open-label status means masking was not used, as reflected by the registry's masking entry. In general, knowledge of treatment assignment may influence some aspects of study conduct or measurement, depending on the endpoint and procedures. The registry record does not describe measures intended to limit such influences, so no further claim about safeguards can be made. The primary endpoint is a timed task, but the record does not specify who administered or assessed it.

What can planning assumptions tell a reader?

The registry describes a sample-size planning illustration based on the observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It says the effect to detect is a stated fraction of the observed effect. These are planning assumptions, not evidence that the trial used those assumptions in its own design. The record does not report a sample-size justification for the trial. It also does not provide numerical planning outputs in the registry entries, so no required sample size can be stated.

In general, a sample-size calculation estimates the amount of information a future or planned study may need under specified assumptions. Its result depends on the target effect, variability, design, and chosen error and power criteria. Such a calculation is not an observed trial outcome and is not a substitute for the results. A planning illustration based on an observed effect also depends on having a relevant effect estimate; the registry record does not report the primary effect estimate needed to assess that basis.

Why does over-dispersion matter in general?

For outcomes whose variability is greater than assumed by a statistical model, standard errors can be too small if that extra variability is ignored. This is often described generally as over-dispersion. It can make confidence intervals too narrow and significance tests too optimistic. The primary endpoint in this study is a continuous timed measure, and the registry does not report an analysis model or evidence of over-dispersion. This general concept therefore cannot be used to infer anything about the trial's unreported analysis.

More broadly, the registry lists 15 outcome measures and 0 analyses. The design and outcome definitions clarify what investigators intended to evaluate, while the absence of reported analyses prevents assessment of estimates, uncertainty, or statistical significance. No particular inferential method, covariate adjustment, or model specification is reported in the registry record.

10. Multiplicity, Missing Data and Analysis Populations

The registry lists 15 outcome measures, including the primary stair-climbing endpoint and secondary measures covering respiratory function, mobility, timed tasks, muscle strength, and muscle imaging. When a study evaluates multiple outcomes, general statistical reasoning recognizes that the chance of at least one apparently notable result can increase if many tests are conducted and interpreted without an appropriate plan. Methods such as prespecifying a primary endpoint or adjusting for multiple comparisons may address particular multiplicity concerns. The registry record does not report a multiplicity procedure, so it is not possible to say whether or how the listed outcomes were handled in an analysis.

The endpoint description identifies the primary measure and its timeframe, but the record does not report the analysis plan for that endpoint. It does not report a method for handling missing measurements, a rule for defining evaluability beyond the stated population description, or an imputation method. These details are important in general because incomplete follow-up can affect which participants contribute to an estimate and the assumptions underlying an analysis. No missing-data method should be inferred from the visit schedule.

The primary population description says that the safety population included enrolled participants who received at least 1 dose of deflazacort. It also states that “Number analyzed” means participants evaluable at the specified timepoint. The registry does not provide timepoint-specific participant counts for the primary endpoint. Enrollment of 11 does not establish how many contributed baseline or Week 26 data.

The registry records 0 analyses. It therefore does not report a primary endpoint estimate, secondary endpoint estimates, or how any analysis population was applied. Whether an intention-to-treat population, a per-protocol population, or another efficacy population was used is not reported in the registry record. The treatment of participants who discontinued, had a dose reduction, or lacked a measurement is also not reported.

11. 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 5% reporting threshold.

Most frequent non-serious adverse event terms

The adverse-event summary lists 7 participants at risk in the deflazacort group for serious adverse events and 0 affected. For other adverse events, it lists 7 at risk and 5 affected. The death count is 0. These are the recorded figures for the group; they should be read as counts in the registry summary, not as a comparison with another treatment group. The record lists no top serious-event terms.

In general, an at-risk count identifies the group included in a particular adverse-event summary, while an affected count records participants meeting that summary's event criterion. The figures do not, by themselves, describe event severity beyond the category label, timing, duration, recurrence, or relationship to treatment. The record does not provide individual event narratives or a breakdown of the other adverse events.

The adverse-event group size is 7, while total enrollment is 11. The available record does not explain the difference, and it should not be assumed that the adverse-event group represents every enrolled participant. The summary is descriptive and does not establish a comparative safety effect. The reason for termination is not reported in the registry record and should not be inferred from these counts.

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

The registry record establishes that a Phase 3, open-label, single-group study of deflazacort in participants with LGMD2I was registered, with enrollment of 11 and a primary endpoint defined as change from baseline in time to climb 4 stairs after 26 weeks. It also records that the trial was terminated. These registry entries describe the study's status and planned measurement, not the direction or magnitude of an outcome.

The record does not include a primary endpoint result or any analysis results. As a result, it does not show whether the mean stair-climbing time changed, how variable the measurements were, how many participants were evaluable at Week 26, or how precise an estimate might be. It also does not provide a statistical test or confidence interval for the primary measure. No conclusion about the observed efficacy outcome can be drawn from the endpoint definition alone.

The design is single-group and open-label, with allocation recorded as not applicable and masking as none. In general, a single-group study can document outcomes during treatment, but it cannot make a direct randomized comparison with a control group when none is recorded. This design context would matter when interpreting any change, even if an estimate were available. It does not establish that an unreported change occurred or explain its cause.

The safety summary records 0 participants affected by serious adverse events among 7 at risk, 5 affected by other adverse events among 7 at risk, and 0 deaths. These are descriptive counts in the registry record. They do not, on their own, establish comparative safety or provide detail about the nature and timing of events. The record does not explain the difference between the enrollment count and the adverse-event group size.

Accordingly, the statistical meaning of the available record is limited: it documents the design, intended outcomes, and selected safety counts, but supplies no reported analysis of the primary endpoint. It does not establish that deflazacort did or did not change stair-climbing time, nor does it support a treatment-versus-control conclusion.

13. Why This Trial Matters Statistically

This study is statistically notable for the contrast between a defined primary endpoint and the absence of a reported primary analysis in the registry record. The endpoint is specific: change from baseline in time to climb 4 stairs after 26 weeks, measured in seconds and summarized as a mean with standard deviation. Yet the record lists 0 analyses. A clearly specified measure cannot be interpreted as a result unless an estimate and its supporting information are reported.

The design also makes the comparison structure important. The trial is recorded as open-label and single-group, with allocation not applicable and masking none. A within-group outcome can describe what was measured over time, but does not supply the same comparative evidence as a concurrently randomized control group. That distinction is useful for readers assessing what a study design can establish, separate from any judgment about the intervention.

The record describes 15 outcome measures. Multiple outcomes can offer information across different domains, but they also make the statistical plan for prioritizing and interpreting findings relevant. The registry record does not report how multiplicity was addressed, and it reports no analyses. Readers therefore cannot determine whether any secondary outcome was analyzed or how any findings were interpreted relative to the primary measure.

The case also illustrates why enrollment, evaluability, and safety denominators should be kept distinct. Enrollment is 11, the primary population definition depends on receiving at least 1 dose and being evaluable at a specified timepoint, and the adverse-event summary lists 7 at risk. The record does not supply timepoint-specific endpoint counts or explain the denominator difference. These distinctions matter because an outcome estimate depends on which participants contribute data and how incomplete measurements are handled.

Finally, a planning illustration is not a substitute for trial results or a documented design rationale. The record describes assumptions for a planning calculation but does not report the trial's own sample-size justification. This separation helps prevent a planning exercise from being read as observed evidence or as proof that the study was adequately sized for its endpoint.

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 most direct limitation for interpreting this trial record is that it contains no reported analysis results. Although the primary outcome and its measurement timeframe are specified, no estimate, variability measure, interval, or test result is reported. The registry also lists 0 analyses, so conclusions about the observed primary change cannot be drawn from this record.

The study is described as single-group and open-label, with no randomized comparison recorded. Thus, the design information does not provide a like-for-like control group for estimating a between-group treatment contrast. The registry mentions placebo in an incomplete arm description concerning participants before an amendment, but does not provide sufficient detail to reconstruct a comparator or its role in any analysis. The study should not be described as a randomized comparison on that basis.

The registry reports enrollment of 11 but does not give the number evaluable for the primary endpoint at baseline or Week 26. It defines the analysis count as participants evaluable at the specified timepoint, but does not report those counts or how missing values were handled. It also does not report the number who completed treatment, whether doses were reduced, or the reason for termination.

Other trial-specific details are also absent from the record: baseline characteristics, full eligibility criteria, a detailed statistical analysis plan, the rationale for sample size, and the reason for termination. The registry lists 15 outcomes but does not report a multiplicity procedure or analyses for the secondary measures. These omissions limit assessment of representativeness, precision, missing-data assumptions, and the relationship between planned outcomes and reported evidence.

The safety summary includes 7 participants at risk, which differs from the enrollment count of 11; the registry record does not explain that difference. The adverse-event figures are descriptive and do not provide event timing, duration, individual terms, or a control comparison. These limitations concern what can be established from the registry record and should not be mistaken for findings about unreported data.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I) — NCT03783923.
• Registry record last updated 2022-06-27; status last verified 2022-05.
• Results first posted 2022-06-27.
• 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.

18. Record Summary

The registry describes NCT03783923 as a Phase 3, multicenter, open-label, single-group study of deflazacort in participants with LGMD2I. Enrollment was 11, and the study status is terminated. The planned oral dose was 0.6 milligrams per kilogram per day, with possible reduction for tolerability issues. The primary endpoint was change from baseline in time to climb 4 stairs after 26 weeks, measured in seconds.

The record lists 15 outcome measures and 0 analyses. It therefore supplies no reported primary endpoint estimate or statistical uncertainty. The safety summary records 0 serious adverse events among 7 at risk, 5 affected by other adverse events among 7 at risk, and 0 deaths. The reason for termination, timepoint-specific endpoint counts, and the trial's own sample-size justification are not reported in the registry record.