Effectiveness of Stem Cell Treatment for Adults With Ischemic Cardiomyopathy (The FOCUS Study): Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Effectiveness of Stem Cell Treatment for Adults With Ischemic Cardiomyopathy (The FOCUS Study) (NCT00824005), a randomized phase 2 study. The primary outcome is Change in Maximal Oxygen Consumption (VO2max).

2. Clinical Question

3. Trial Design

The FOCUS Study is registered as a randomized, controlled, phase II trial with a parallel-group design. Its official title describes a double-blind study of intramyocardial injection of autologous bone marrow mononuclear cells under electromechanical guidance in people with chronic ischemic heart disease and left ventricular dysfunction. The registry lists quadruple masking. These labels indicate the registered design, but details such as which roles were masked and how the masking was implemented are not reported in the registry record.

The interventions are adult stem cells and placebo. The arm descriptions specify active stem cell injections and placebo injections. The registry’s brief summary frames the study as an assessment of the safety and effectiveness of collecting cells from a participant’s bone marrow and injecting them into the heart. This page describes the posted outcomes; it does not establish the biological mechanism or assess the procedure beyond the reported measures.

The registry reports enrollment of 92. The study is marked completed, with a start date of 2009-03 and primary completion in 2011-11. The outcome-specific analysis groups do not all have the same numbers of participants, and the primary endpoint population is restricted to participants with both baseline and Month 6 VO2max data. The reasons participants may be absent from an outcome analysis are not reported in the registry record.

The record lists 14 outcome measures and 12 analyses, and reports 11 posted secondary outcomes. Secondary measures include imaging, clinical classifications, medication use, exercise, and serum BNP. Their presence gives context to the range of outcomes examined, but does not by itself establish that they were prespecified as confirmatory tests or that a particular multiplicity procedure was used.

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:

Analysis population for the primary outcome, as defined in the registry: Only participants with both baseline and 6 month VO2max data available are included.

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 Maximal Oxygen Consumption (VO2max), measured over Measured at Baseline and Month 6.

The primary endpoint is change in maximal oxygen consumption, or VO2max, measured at baseline and Month 6. VO2max is a continuous measure of exercise-related oxygen consumption, reported in mL/kg/min. The registry says it was assessed using the Naughton treadmill protocol. The primary analysis is based on the mean change in each group and the mean difference between groups.

In general, a continuous endpoint can retain information about the degree of change across participants rather than reducing outcomes to categories. Comparing mean changes between randomized groups can describe whether the groups, on average, changed differently during follow-up. That comparison is not a direct description of every participant’s response, and group means do not show the full distribution of individual outcomes.

The primary endpoint population includes only participants with both baseline and 6 month VO2max data available. Consequently, the posted estimate pertains to this available-data subset. The registry does not report the reasons for missing endpoint measurements, the number of participants who lacked either measurement, or a detailed missing-data method. The result should therefore be read together with this restriction: it is an estimate for the analyzed participants, not automatically an estimate for every person enrolled.

The endpoint provides a functional measure relevant to the trial’s question, but it is one outcome among the clinical and imaging measures listed in the record. The registry does not report a threshold for a clinically important change in VO2max. Statistical interpretation of the mean difference and its confidence interval therefore does not by itself determine the clinical importance of a particular value.

7. Results Posted in the Registry

• Active Stem Cell Injections: mean 0.40 versus -0.60 on control; difference 1.00 (95% CI -0.43 to 2.43), standardized effect d = 0.34; z-test p = 0.170.

8. Secondary Efficacy Endpoints

The registry also posts results for secondary outcomes. For each outcome below, the endpoint type, the test applied to the posted summary statistics, the result and its interpretation are given in turn. Secondary endpoints are usually not adjusted for multiple testing, so they support rather than confirm the primary finding.

Regional Wall Motion by Echocardiography

• Endpoint type: Continuous: a mean value per participant, compared as a difference in means.
• Test: Unadjusted two-sample z-test for the difference in means, using the posted standard deviations.
• Result:
  • Active Stem Cell Injections: mean 0.00 versus 0.00 on control; difference 0.00 (95% CI -0.20 to 0.20), standardized effect d = 0.00; z-test p = 1.000.
• Interpretation:
  • The 95% confidence interval includes no difference, so these data do not show a statistically significant difference between Active Stem Cell Injections and the control. The interval gives the range of effects that remain compatible with the data.

Clinical Improvement in CCS Classification (Angina Pectoris)

• Endpoint type: Continuous: a mean value per participant, compared as a difference in means.
• Test: Unadjusted two-sample z-test for the difference in means, using the posted standard deviations.
• Result:
  • Active Stem Cell Injections: mean -0.50 versus -0.30 on control; difference -0.20 (95% CI -0.58 to 0.18), standardized effect d = -0.26; z-test p = 0.297.
• Interpretation:
  • The 95% confidence interval includes no difference, so these data do not show a statistically significant difference between Active Stem Cell Injections and the control. The interval gives the range of effects that remain compatible with the data.

Clinical Improvement in NYHA Classification

• Endpoint type: Continuous: a mean value per participant, compared as a difference in means.
• Test: Unadjusted two-sample z-test for the difference in means, using the posted standard deviations.
• Result:
  • Active Stem Cell Injections: mean -0.30 versus -0.10 on control; difference -0.20 (95% CI -0.55 to 0.15), standardized effect d = -0.24; z-test p = 0.256.
• Interpretation:
  • The 95% confidence interval includes no difference, so these data do not show a statistically significant difference between Active Stem Cell Injections and the control. The interval gives the range of effects that remain compatible with the data.

The registry posts secondary outcomes spanning cardiac imaging, clinical classification, medication use, exercise, and serum BNP. The continuous secondary comparisons represented in the posted results do not show a consistent, clearly separated pattern between active injections and placebo. Their estimates and intervals should be interpreted outcome by outcome, rather than treated as a single combined measure of benefit or as a substitute for the primary VO2max comparison.

The secondary outcomes add context because they examine different aspects of cardiac function and clinical status. They do not independently resolve the uncertainty in the primary result. A secondary outcome may differ in measurement properties, eligible analysis population, or available follow-up data, so agreement or disagreement across outcomes is not automatically evidence for or against a single underlying effect.

In general, examining many outcomes increases the chance that some results will appear notable by chance, even when there is no underlying difference. This is why unadjusted secondary results usually carry less confirmatory weight than a prespecified primary comparison. The registry record does not report whether a multiplicity adjustment was used, how outcomes were prioritized, or whether all secondary analyses were prespecified. Accordingly, secondary findings should be viewed as supportive or exploratory context rather than decisive evidence.

The posted results also do not establish that all listed secondary outcomes were measured in the same participants or analyzed under the same assumptions. The registry reports differing outcome-specific populations in the results. The reasons for those differences are not reported in the registry record. The overall interpretation should therefore remain centered on the primary endpoint while recognizing that the secondary outcomes broaden the description of what was measured.

Secondary outcomes registered for this trial, for reference (only those above were recalculated):

• Regional Wall Motion by MRI (in Eligible Patients)
• Regional Blood Flow Improvement by MRI (in Eligible Patients)
• Regional Wall Motion by Echocardiography
• Clinical Improvement in CCS Classification (Angina Pectoris)
• Clinical Improvement in NYHA Classification
• Number of Participants With a Decrease in Anti-anginal Medication
• Exercise Time and Level
• Serum BNP Levels in Patients With CHF

9. Statistical Interpretation

10. Statistical Methods Explained

The primary comparison uses mean change, a measure suited to a continuous endpoint such as VO2max. For each group, the posted result is the average change from baseline to Month 6. The between-group mean difference compares those averages. A positive value in the reported direction means the active group’s average change was higher than the placebo group’s average change; it does not mean every participant changed in that direction.

What does a confidence interval describe?

In general, a confidence interval expresses uncertainty around an estimated effect under the statistical model used. A narrower interval indicates greater precision, while a wider interval leaves a broader set of effect values compatible with the data and model. The primary interval includes zero, so the posted result does not clearly distinguish a nonzero average difference from no average difference. The interval should not be read as a range containing every individual participant’s response.

What does the reported p-value tell the reader?

The sponsor reports a two-sided t-test for the primary comparison. In general, a p-value describes how compatible the observed data are with a specified null hypothesis under the test’s assumptions; it is not the probability that the null hypothesis is true, nor does it measure clinical importance. The sponsor reports p = 0.169, while the unadjusted recalculation gives p = 0.17. Those values express limited evidence against a no-difference null in this analysis, but the p-value alone does not establish the size, precision, or practical importance of the difference.

Why does the endpoint analysis population matter?

The primary outcome is restricted to participants with both baseline and 6 month VO2max measurements. A change score requires measurements at both time points, so the reported mean change is calculated among those with the needed data. In general, excluding participants with missing measurements can affect the interpretation if missingness is related to outcomes or treatment. The registry record does not report reasons for missing measurements or the method used to address missing data. It is therefore not possible to determine from the record whether the available-data analysis may be affected by such patterns.

What does randomization contribute?

In general, random assignment is intended to make comparison groups similar, on average, with respect to measured and unmeasured characteristics at the start of a trial. This supports an interpretation of the contrast as a comparison of assigned interventions, subject to follow-up, adherence, missing-data, and analysis issues. The registry identifies the study as randomized, but does not report the sequence-generation or allocation-concealment methods. Those implementation details are not reported in the registry record.

How should masking be understood?

The record labels the trial double-blind and quadruple masked. Masking can reduce the influence of expectations on treatment delivery, participant behavior, and outcome assessment, depending on who is masked and how outcomes are measured. The registry does not identify the roles covered by the masking labels or report how masking was maintained. The labels therefore describe the registered design, while the practical details remain not reported in the registry record.

Why can over-dispersion matter?

In general, over-dispersion means that outcomes vary more than a statistical model expects. If a model assumes less variability than the data contain, uncertainty may be understated and intervals may be too narrow. For a continuous outcome, the spread of participant-level values informs uncertainty about a mean. The registry reports standard deviations for the primary groups, and the recalculation uses those posted summaries. There is no report of an over-dispersion assessment, and the continuous-outcome comparison should not be interpreted as evidence that such an assessment was performed.

What does the recalculation represent?

The recalculated primary comparison is an unadjusted calculation from the posted group means, standard deviations, and analysis counts. It is not an adjusted estimate from participant-level data and does not account for covariates or other modeling details that may have been used in the sponsor’s analysis. The sponsor’s reported test is a two-sided t-test. The registry does not provide enough detail to reconstruct all elements of the sponsor’s analysis beyond the reported result and method label.

Secondary comparisons also use continuous measures in the posted results, but their interpretation depends on the specific outcome, analysis population, and timing. In general, outcome definitions and analysis choices should be prespecified to limit selective emphasis. The registry record does not report a full statistical analysis plan, the handling of multiplicity, interim analyses, or a sample-size justification. Those omissions limit the ability to evaluate the complete analysis strategy.

11. Confidence Intervals and Precision

The sponsor’s primary estimate is a mean difference of 1.0 mL/kg/min, with a 95% confidence interval from -0.42 to 2.34. The unadjusted recalculation gives the same displayed mean difference of 1.0, with a 95% confidence interval from -0.427 to 2.427. Both intervals extend across zero, the value associated with no difference between group means. Thus, the result is not precise enough to exclude no difference, and the interval also allows for effects in either direction.

In general, a confidence interval is useful because it combines the estimated effect with its uncertainty. It is not a guarantee that the true effect lies within its endpoints, and it is not a prediction interval for individual outcomes. Its interpretation depends on the analysis assumptions and the data used. The interval is based on the primary endpoint analysis population, which includes participants with both baseline and Month 6 measurements.

The interval width reflects uncertainty in the estimated group contrast. The analysis counts are unequal, and the group standard deviations indicate variation in VO2max changes. This context helps explain why an estimated difference does not necessarily translate into a tightly estimated effect. The registry does not report a clinically important difference threshold for this endpoint, so the interval cannot be classified against such a threshold from the record alone.

The sponsor and recalculated intervals are not identical, although both include zero and support the same broad interpretation of uncertainty. The sponsor’s reported result is the trial’s own analysis; the recalculated result is a simple unadjusted estimate from posted summary statistics. Differences in rounding or analysis details may contribute, but the specific reason is not reported in the registry record.

12. Planning a Similar Trial: Sample Size

This is a planning illustration, not a re-evaluation of the original trial. It asks: if a new study wanted to detect a difference like the one observed, or a smaller one, how large would it need to be? It is deliberately not “observed power” of the finished trial, which is not a meaningful quantity.

The planning figures are illustrations based on the observed effect, not a report of the trial’s original design calculation. For these continuous-outcome illustrations, the figures are participants per arm, not total events. The planning assumptions are a two-sided significance level of 0.05, equal allocation between the two arms, and a normal approximation. The effect to detect is set to the stated fraction of the observed effect. The trial’s own original design assumptions and sample-size justification are not reported in the registry record.

The 80% power column gives the illustrated number of participants per arm for an analysis with an 80% chance of detecting the specified effect if that effect is the true one and the stated assumptions hold. The 90% power column gives the corresponding planning number for a 90% chance. These are prospective planning concepts, not descriptions of the trial’s observed probability of success. The figures are labeled by the assumed fraction of the observed effect, allowing readers to see how the illustration changes when the target effect is smaller.

Smaller effects are harder to distinguish from random variation, so they generally require more participants to achieve the same planned power under otherwise similar assumptions. The table and curve should therefore be read as sensitivity illustrations: they show how planning needs change when the effect selected for a future study changes. They do not show how many participants this trial should have enrolled under its actual protocol, because the original design assumptions are not reported.

This is not observed power. The planning estimates do not establish that the trial had a particular probability of detecting an effect after its results were known, and they should not be used to reinterpret the primary p-value as a measure of success. They are conditional illustrations based on the observed effect and the stated planning assumptions.

13. How the Sample Size Is Derived

Power is the probability that a study of a given size rejects the null hypothesis when a specific true effect exists. It depends on four things: the effect to detect, the variability of the outcome, the significance level (here a two-sided α of 0.05, so z_1−α/2 = 1.960) and the desired power (z_1−β = 0.842 for 80% and 1.282 for 90%).

Formula

n is the number of participants in each arm, Δ is the difference in means to detect and σ is the common standard deviation.

Worked calculation for this trial

1. Observed difference Δ = 1.000; pooled standard deviation σ = 2.941.
2. n = 2 × (1.96 + 0.842)² × 2.941² / 1.000² = 135.8, rounded up to 136 participants per arm for 80% power.

The smaller-effect rows in the table above repeat the same calculation with the effect multiplied by 0.75 and 0.5 (for ratios, applied to the logarithm of the ratio).

R code

alpha <- 0.05
z_a   <- qnorm(1 - alpha / 2)          # two-sided
delta <- 1                      # observed difference in means
sigma <- 2.94115446897806                     # pooled standard deviation
n_per_arm <- function(power, frac = 1) {
  ceiling(round(2 * (z_a + qnorm(power))^2 * sigma^2 / (delta * frac)^2, 6))
}
for (f in c(1, 0.75, 0.5))
  cat(sprintf("%3.0f%% of observed effect: n per arm = %s (80%% power), %s (90%% power)\n", 100 * f,
              format(n_per_arm(0.80, f), big.mark = ","), format(n_per_arm(0.90, f), big.mark = ",")))

# Check against R's built-in function (t-based, so very slightly larger)
cat("power.t.test:", round(power.t.test(delta = delta, sd = sigma, power = 0.80)$n, 1), "\n")

# Simulation: exact two-sample t-test using simulated means and variances
set.seed(824005)
n    <- n_per_arm(0.80)
reps <- 20000
m_diff <- rnorm(reps, delta, sigma * sqrt(2 / n))             # difference in sample means
s2     <- sigma^2 * rchisq(reps, 2 * (n - 1)) / (2 * (n - 1))   # pooled sample variance
tstat  <- m_diff / sqrt(s2 * 2 / n)
cat(sprintf("Simulated power at n = %s per arm: %.3f (%d replicates)\n", format(n, big.mark = ","), mean(abs(tstat) > qt(0.975, 2 * (n - 1))), reps))

Output from running the code

100% of observed effect: n per arm = 136 (80% power), 182 (90% power)
 75% of observed effect: n per arm = 242 (80% power), 324 (90% power)
 50% of observed effect: n per arm = 544 (80% power), 728 (90% power)
power.t.test: 136.8 
Simulated power at n = 136 per arm: 0.796 (20000 replicates)

The simulation draws many sets of group means and variances at the planned sample size and counts how often the two-sample t-test rejects the null. It should land close to the target power; at very small required sample sizes it can fall below it, because the normal approximation is rough there.

14. Analyses Reported by the Sponsor

The registry reports the sponsor’s primary analysis as a two-sided t-test. It gives a mean difference of 1.0, a 95% confidence interval from -0.42 to 2.34, and p = 0.169. The simple unadjusted recalculation from the posted group summaries also gives a mean difference of 1.0, with a 95% confidence interval from -0.427 to 2.427 and p = 0.17. The estimates are close, but the interval endpoints and displayed p-values are not identical.

The sponsor’s analysis is the trial’s reported result; the recalculation is a separate, unadjusted reconstruction from summary statistics. The record does not explain the differences between the intervals. Both intervals include zero, so the broad interpretation is unchanged: the comparison leaves uncertainty about whether the groups differ in mean change. The discrepancy flag is false, so there is no flagged disagreement requiring the sponsor’s analysis to take precedence on that basis. The sponsor result should nevertheless remain the primary reference for what the trial itself reported.

The recalculation cannot recover participant-level adjustments, data handling, or other analysis details that are not reported. It is best read as a transparent cross-check of the posted summaries, not as a replacement for the trial’s own analysis.

15. Multiplicity, Missing Data and Analysis Populations

The registry lists multiple outcome measures and analyses, including a primary endpoint and a range of secondary outcomes. When several outcomes or comparisons are examined, the probability of at least one apparently notable result arising by chance can increase. In general, multiplicity procedures can control that risk, but the appropriate method depends on the trial’s aims and analysis plan.

The registry record does not report a multiplicity adjustment, a hierarchy for testing outcomes, or a complete statistical analysis plan. It is therefore not possible to determine whether secondary analyses were intended as confirmatory tests, whether their interpretation was adjusted for the number of comparisons, or how the trial planned to handle multiple endpoints. Unadjusted secondary p-values, when reported, should not be treated as if they were isolated tests chosen in advance.

The primary analysis population is defined as participants with both baseline and 6 month VO2max data. The registry does not report the reasons for missing baseline or follow-up values, the missing-data method, or a sensitivity analysis addressing missingness. In general, an available-case comparison can be biased if the likelihood of having measurements is related to treatment or outcome. The record does not permit assessment of that possibility.

The outcome-specific analysis counts vary, and the registry does not explain all differences among the analyzed populations. The record also does not report whether analyses followed an intention-to-treat principle, how adherence or treatment discontinuation was handled, or how protocol deviations were addressed. Interim analyses and stopping rules are not reported in the registry record. These omissions do not show that a particular procedure was or was not used; they limit what can be concluded from the public record about the full analysis plan.

For these reasons, the posted primary result can be interpreted as the reported comparison in the stated endpoint population, while broader claims about the complete prespecified analysis strategy require information not reported in the registry record.

16. 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 serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: heart failure (9.7%), chest pain (6.6%), tachycardia (4.9%).

The registry reports serious adverse events among participants at risk in each group. For placebo injections, 6 participants were affected among 31 at risk; for active stem cell injections, 9 were affected among 61 at risk. The record also reports other adverse events affecting 11 participants among 31 at risk in the placebo group and 19 among 61 at risk in the active group. These are group-specific reported counts, not a statistical comparison of treatment-related risk.

The listed serious adverse event terms include Heart Failure at 9.7%, Chest pain at 6.6%, and Tachycardia at 4.9%. These terms describe the most frequent serious event categories shown in the record; the percentages should be read as reported, not recalculated. The registry does not provide enough information to determine the severity, timing, relatedness, or recurrence of each event.

Deaths are not reported in the registry record. The record does not establish whether an adverse event was caused by an intervention, and the counts alone do not show that one group had a greater or lesser treatment-related risk. Interpretation is limited by the information and follow-up recorded for these safety outcomes.

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

The primary result is an estimate of the difference between groups in mean change in VO2max from baseline to Month 6 among participants with both measurements. The observed mean difference is positive, but the sponsor’s confidence interval includes zero and extends to values on either side of no difference. The result therefore leaves substantial uncertainty about the size and direction of the underlying group difference.

In general, a confidence interval that includes zero does not prove that the groups are identical. It means the data and analysis do not distinguish a range of possible effects from no difference at the stated confidence level. Conversely, a positive point estimate alone does not establish that the active intervention has a clinically important effect. The registry does not report a threshold for clinical importance for VO2max change.

The result does not establish an effect for every participant, because the endpoint comparison concerns group averages. It also does not necessarily describe all enrolled participants, because only those with both baseline and Month 6 measurements were included. The registry does not report the missing-data reasons or analyses needed to assess the impact of that restriction.

The posted secondary measures and adverse event counts add descriptive information, but they do not resolve the primary comparison’s uncertainty. Secondary results have less confirmatory weight when the analysis plan and multiplicity handling are not reported. Safety counts are not causal comparisons, and the event data do not by themselves establish whether events were related to injections.

Overall, the registry record documents a randomized comparison and reports an uncertain primary mean difference. It does not establish a definitive clinical conclusion about the intervention, its mechanism, or its effects beyond the measured outcomes and populations recorded.

18. Why This Trial Matters Statistically

This trial is statistically informative because it illustrates the distinction between an estimated effect and the precision of that estimate. The primary mean difference is positive, yet the confidence interval includes no difference and allows effects in either direction. A point estimate alone would conceal that uncertainty; the interval makes it visible.

The primary endpoint analysis also illustrates why the analysis population matters. The registry defines the endpoint population by availability of both baseline and follow-up VO2max data, while the study enrollment figure describes a broader trial-level count. Readers need to distinguish enrollment from the participants contributing to a specific outcome. The record does not explain the missing measurements, so the representativeness of the analysis population cannot be assessed from the registry alone.

The secondary outcomes reinforce the importance of an analysis plan. Trials may examine several clinical and imaging measures, but unadjusted results across many outcomes are less persuasive than a clearly prespecified primary comparison. The registry does not report multiplicity handling or a full statistical analysis plan, which limits assessment of how the many outcomes were intended to support conclusions.

Finally, the planning illustrations show how assumptions about the effect to detect influence future sample-size requirements. They are not observed power and do not reveal the original rationale for the trial’s enrollment. Distinguishing planned design, observed data, and post hoc planning calculations is essential for reading trial statistics without overstating what the results establish.

19. 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 result is based on participants with both baseline and Month 6 VO2max measurements. The registry does not report why measurements were missing, how missingness was handled, or whether sensitivity analyses were conducted. This limits assessment of how well the available-data estimate represents the randomized groups.

The posted primary comparison is a summary-level result. The registry identifies a two-sided t-test but does not report a full analysis plan, covariate adjustments, allocation-concealment details, or the implementation of masking. The masking labels alone do not identify which trial roles were masked. These details are not reported in the registry record.

The registry lists multiple secondary outcomes, but does not report whether multiplicity was addressed, which secondary outcomes were intended to be confirmatory, or the hierarchy among analyses. This makes secondary findings more difficult to evaluate as a unified evidential set. Outcome-specific analysis counts differ, and reasons for these differences are not reported.

The trial is described as phase II and reports enrollment of 92, but the registry does not report the original sample-size justification or the assumptions used in its design. The planning illustrations based on the observed effect cannot fill that gap. The record also does not report a clinically important threshold for the primary endpoint, limiting interpretation of the estimate’s clinical scale.

Safety information is presented as counts and selected serious event terms. The registry does not report event timing, relatedness, recurrent events, or a formal comparison of adverse event risks. Deaths are not reported in the registry record. These limitations constrain interpretation of both the efficacy and safety information to what is explicitly posted.

20. Related Tutorials

21. Related Statistical Calculators

22. Sources

• ClinicalTrials.gov: Effectiveness of Stem Cell Treatment for Adults With Ischemic Cardiomyopathy (The FOCUS Study) — NCT00824005.
• Registry record last updated 2015-07-01; status last verified 2015-06.
• Results first posted 2013-04-04.
• 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.

23. Record Summary

The FOCUS Study is a completed randomized, controlled, parallel-group, phase II trial of active autologous bone marrow mononuclear cell injections versus placebo injections in chronic ischemic heart disease with left ventricular dysfunction. The registry describes the study as double-blind and quadruple masked. Enrollment is reported as 92.

The primary endpoint is change in VO2max from baseline to Month 6, assessed with the Naughton treadmill protocol. The primary analysis includes participants with both measurements. The sponsor reports a mean difference of 1.0, with a 95% confidence interval from -0.42 to 2.34 and p = 0.169. The interval includes zero, leaving uncertainty about the between-group difference.

The registry also posts secondary outcomes and adverse event counts. Multiplicity handling, missing-data methods, the full statistical analysis plan, and the trial’s original sample-size justification are not reported in the registry record. The posted results should be interpreted within those reporting limits.