A Placebo Controlled Safety and Efficacy Study of MSDC-0160 in Type 2 Diabetes With Active Comparator: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for A Placebo Controlled Safety and Efficacy Study of MSDC-0160 in Type 2 Diabetes With Active Comparator (NCT00760578), a randomized phase 2 study. The primary outcome is Change From Baseline in Averaged Postprandial Glucose in Response to a MMT Test.

2. Clinical Question

The study was intended to evaluate safety, tolerability and efficacy in Type 2 diabetes. Its design included both placebo and an active comparator, but the posted comparative estimates described for the primary endpoint are each against placebo. Results for pioglitazone therefore provide context within the randomized study rather than a direct comparison with either MSDC-0160 dose in the posted estimates.

3. Trial Design

The registry record describes a Phase 2A, randomized, double-blind, comparator- and placebo-controlled, multiple-dose, parallel study. Masking is listed as quadruple. These labels indicate a randomized comparison with several treatment arms, but the registry record does not identify the particular roles included in the masking designation or give further detail on allocation procedures.

Enrollment was 86. The four arms were placebo, pioglitazone 45 mg, MSDC-0160 90 mg, and MSDC-0160 220 mg, each taken once daily. The placebo was microcrystalline cellulose. The study began in 2008-09, primary completion was recorded in 2009-01, and the study status is completed. The listed primary endpoint timeframe is 28 days.

The registry’s primary endpoint population consisted of patients compliant during treatment who had Day 8 and Day 43 averaged mixed-meal tolerance tests and laboratory assessments. Compliance was determined by the sponsor through manual review of pill counts, including review of comments; the text describing those comments is incomplete in the registry record. The number enrolled and the arm-specific endpoint counts are not identical concepts: the primary endpoint results list 20 patients for MSDC-0160 90 mg, 19 for MSDC-0160 220 mg, 16 for pioglitazone, and 21 for placebo.

The registry record lists seven outcome measures, including the primary endpoint and secondary measures of insulin response to a mixed-meal tolerance test, free fatty acids, triglycerides, and HDL. Four secondary outcomes are posted in the record. The registry record does not report the original sample-size justification, the planned allocation ratio, details of randomization, or the full statistical analysis plan. Those omissions prevent a complete reconstruction of the design rationale or planned inferential procedures.

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: This was a Ph 2A, double-blind, randomized, comparator- \& placebo-controlled study of Mitoglitazone in otherwise healthy adult patients with type 2 diabetes. The study consisted of a maximum 21-day screening period, a 2 week, single-blind, placebo lead-in period, a 4 week, double-blind treatment period and a 1 week posttreatment follow-up period.

Overall Study

Analysis population for the primary outcome, as defined in the registry: All patients who were compliant during the treatment period and who had a Day 8 and Day 43 averaged mixed-meal tolerance test and laboratory assessments. Compliance during the treatment period was determined by the sponsor through a manual review of pill counts, including a review of comments from the study sites.

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 From Baseline in Averaged Postprandial Glucose in Response to a MMT Test, measured over 28 days.

The primary endpoint is change from baseline in averaged postprandial glucose in response to a mixed-meal tolerance test. A mixed-meal test measures the body’s response after a meal; averaging post-meal glucose readings produces a continuous summary of that response. The endpoint is expressed in mmol/L, with change from baseline represented by a least-squares mean. A negative change denotes a lower value relative to baseline, while a positive change denotes a higher value.

Postprandial glucose is used as a short-term measure of glucose handling following a meal challenge. Unlike an outcome such as a clinical event, it is a laboratory measure that can be compared across randomized arms over the study timeframe. The registry record lists the timeframe as 28 days and describes the endpoint population as compliant patients with Day 8 and Day 43 mixed-meal assessments and laboratory assessments.

The endpoint’s reported standard errors describe uncertainty around each arm’s estimated mean change. The reported comparisons express the difference between each treatment arm and placebo. These measures can indicate the direction and precision of the observed changes, but they do not by themselves establish the clinical importance of a difference. The registry record does not report a threshold for a clinically important change in postprandial glucose.

7. Results Posted in the Registry

• Mitoglitazone 90 mg: mean 0.16 versus 0.51 on control; difference -0.35 (95% CI -1.30 to 0.60), standardized effect d = -0.22; z-test p = 0.473.
• Mitoglitazone 220 mg: mean -0.44 versus 0.51 on control; difference -0.95 (95% CI -1.92 to 0.02), standardized effect d = -0.61; z-test p = 0.054.
• Pioglitazone 45 mg: mean -1.10 versus 0.51 on control; difference -1.61 (95% CI -2.62 to -0.60), standardized effect d = -1.03; z-test p = 0.002.

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.

Change From Baseline in Averaged Insulin Levels in Response to a Mixed-meal Tolerance Test

• 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 errors.
• Result:
  • Mitoglitazone 90 mg: mean 0.74 versus -1.60 on control; difference 2.34 (95% CI -10.43 to 15.11), standardized effect d = 0.11; z-test p = 0.720.
  • Mitoglitazone 220 mg: mean -9.77 versus -1.60 on control; difference -8.17 (95% CI -21.11 to 4.77), standardized effect d = -0.39; z-test p = 0.216.
• Interpretation:
  • The 95% confidence interval includes no difference, so these data do not show a statistically significant difference between Mitoglitazone 90 mg and the control. The interval gives the range of effects that remain compatible with the data.
  • The 95% confidence interval includes no difference, so these data do not show a statistically significant difference between Mitoglitazone 220 mg and the control. The interval gives the range of effects that remain compatible with the data.

Change From Baseline in FFAs

• 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 errors.
• Result:
  • Mitoglitazone 90 mg: mean -0.05 versus -0.01 on control; difference -0.04 (95% CI -0.18 to 0.10), standardized effect d = -0.17; z-test p = 0.575.
  • Mitoglitazone 220 mg: mean -0.15 versus -0.01 on control; difference -0.14 (95% CI -0.28 to 0.00), standardized effect d = -0.61; z-test p = 0.052.
• Interpretation:
  • The 95% confidence interval includes no difference, so these data do not show a statistically significant difference between Mitoglitazone 90 mg and the control. The interval gives the range of effects that remain compatible with the data.
  • The 95% confidence interval includes no difference, so these data do not show a statistically significant difference between Mitoglitazone 220 mg and the control. The interval gives the range of effects that remain compatible with the data.

Change From Baseline in Triglycerides

• 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 errors.
• Result:
  • MSDC-0160 90 mg: mean 0.12 versus 0.15 on control; difference -0.03 (95% CI -0.47 to 0.41), standardized effect d = -0.04; z-test p = 0.893.
  • MSDC-0160 220 mg: mean -0.21 versus 0.15 on control; difference -0.36 (95% CI -0.80 to 0.08), standardized effect d = -0.50; z-test p = 0.112.
• Interpretation:
  • The 95% confidence interval includes no difference, so these data do not show a statistically significant difference between MSDC-0160 90 mg and the control. The interval gives the range of effects that remain compatible with the data.
  • The 95% confidence interval includes no difference, so these data do not show a statistically significant difference between MSDC-0160 220 mg and the control. The interval gives the range of effects that remain compatible with the data.

The secondary measures broaden the description of short-term metabolic responses. The registry record posts outcomes for insulin response to a mixed-meal tolerance test, free fatty acids, and triglycerides, and lists HDL among the secondary outcomes. Their arm-level estimates do not all move in the same pattern, and the precision differs across outcomes. This variability is important: secondary measures are related aspects of physiology, but they are not interchangeable measures of the primary glucose endpoint.

At a broad level, the secondary results do not provide a single, consistent pattern that can independently settle the primary question. They offer additional descriptive context about changes during active therapy, while the primary endpoint remains the prespecified central outcome identified in the record. No sponsor-reported analyses are listed for the posted secondary comparisons; the recalculated comparisons are unadjusted and should not be elevated to confirmatory findings.

When several outcomes and treatment contrasts are examined, some estimates can appear more notable by chance. A multiplicity procedure can control the interpretation across a family of tests, but the planned procedure is not reported in the registry record. Without knowing which outcomes were prioritized, how testing was ordered, or whether adjustment was planned, the secondary p-values have less evidential weight than a clearly prespecified and multiplicity-aware primary analysis.

Secondary findings can be useful for generating hypotheses and understanding whether different measured processes show compatible or divergent patterns. They cannot establish a mechanism or clinical consequence on their own. Their interpretation is also constrained by the short study timeframe, the small arm counts, and the absence of details on missing-data handling and analysis populations. They should be read alongside the primary endpoint as exploratory supporting information, not as a substitute for it.

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

• Change From Baseline in Averaged Insulin Levels in Response to a Mixed-meal Tolerance Test
• Change From Baseline in FFAs
• Change From Baseline in Triglycerides
• Change From Baseline in HDL

9. Statistical Interpretation

10. Statistical Methods Explained

What does a least-squares mean represent?

A mean is a summary of the central value of a continuous measure. The primary results are labelled least-squares means, a term commonly used for means estimated from a statistical model. Such estimates can account for variables included in that model, but the registry record does not report the model specification or the covariates used. The displayed values should therefore be described as the reported least-squares mean changes, without assuming which adjustments produced them.

The standard error describes the estimated uncertainty of a mean. It is not the spread of individual observations. For the primary endpoint, the standard errors were 0.349 mmol/L for MSDC-0160 90 mg, 0.358 mmol/L for MSDC-0160 220 mg, 0.39 mmol/L for pioglitazone, and 0.34 mmol/L for placebo. A standard error is useful for understanding how precisely an arm’s mean is estimated, but it does not alone convey the full uncertainty in a contrast between two arms.

How should a treatment-versus-placebo contrast be read?

A mean difference compares the reported mean change in one arm with the mean change in placebo. A negative estimate means that the treatment arm’s change was lower than placebo’s change on the reported scale. For example, the recalculated unadjusted difference for MSDC-0160 220 mg was -0.95 mmol/L. This is an estimate of the contrast; it is not a count of participants or a measure of the range of individual responses.

The comparisons labelled recalculated are simple unadjusted recalculations from the posted arm-level summaries. The pooled standard deviations used in standardized comparisons are approximate because the posted arm-level dispersion is a standard error. These recalculations do not substitute for the sponsor’s analysis. The discrepancy indicator is false, but the registry record still does not report the sponsor’s complete analysis output or methods.

What do confidence intervals and p-values contribute?

A confidence interval gives a range of values compatible with the data under the calculation’s assumptions. Wider intervals represent less precision; intervals that include zero are compatible with no mean difference as well as with values on either side of zero. A p-value measures how unusual results at least as inconsistent with a null hypothesis would be under that hypothesis and the statistical assumptions. It is not the probability that the null hypothesis is true, nor does it show the probability that a finding will replicate.

The reported recalculated p-values are unadjusted. Multiple comparisons can increase the chance of at least one apparently notable result when many outcomes or contrasts are examined. The record lists seven outcome measures and includes several arms and secondary endpoints, but the planned multiplicity strategy is not reported in the registry record. The unadjusted p-values should therefore be treated as descriptive evidence rather than as a family-wise conclusion.

Why does over-dispersion matter?

Over-dispersion means that observed variation is greater than a model’s assumed variation. When that occurs, model-based standard errors can be too small if the extra variation is not accounted for, making confidence intervals too narrow and p-values too small. The posted primary endpoint is continuous, and no over-dispersion assessment is reported in the registry record. This is a general statistical consideration, not a claim that over-dispersion occurred in this trial.

What do standardized effects add?

A standardized effect expresses a mean difference in units of a pooled standard deviation, allowing outcomes measured on different scales to be considered in a common framework. The posted standardized effects for the primary comparisons are -0.224 for MSDC-0160 90 mg, -0.609 for MSDC-0160 220 mg, and -1.033 for pioglitazone. These are approximate recalculations using pooled variability inferred from the arm-level standard errors. Standardization does not make a result clinically important by itself, and it can be sensitive to how variability is estimated.

Overall, the methods support a cautious reading: arm means describe the observed summaries, contrasts describe differences from placebo, confidence intervals describe precision, and unadjusted p-values describe compatibility with a null contrast under calculation assumptions. Because the registry record does not report the full analysis plan, adjustment model, missing-data approach, or multiplicity procedure, the recalculated quantities are best treated as transparent exploratory summaries of the posted results.

11. Confidence Intervals and Precision

The confidence intervals around the primary comparisons show how uncertain the estimated differences remain. For MSDC-0160 90 mg versus placebo, the recalculated unadjusted difference was -0.35 mmol/L, with a 95% confidence interval from -1.305 to 0.605. The interval spans zero and includes values on both sides, so the estimate is not precise enough to rule out no difference under this calculation.

For MSDC-0160 220 mg versus placebo, the estimate was -0.95 mmol/L and the 95% confidence interval ran from -1.918 to 0.018. This interval is also compatible with zero, though its values are predominantly negative. The pioglitazone comparison was -1.61 mmol/L, with a 95% confidence interval from -2.624 to -0.596, which lies below zero in the recalculation. These intervals express uncertainty in the contrasts; they do not describe the range of outcomes for individual participants.

The confidence intervals are based on recalculated unadjusted comparisons from summary statistics, and the pooled variation is approximate. They should not be interpreted as the sponsor’s reported model-based intervals. The registry record does not report the sponsor’s analysis methods in enough detail to establish whether its original uncertainty estimates would differ.

Arm sizes for the primary endpoint range from 16 to 21 participants. Small groups generally yield less precise estimates than larger groups when other conditions are similar. Precision is also affected by variability in the outcome, and the reported standard errors indicate appreciable uncertainty relative to the observed mean changes. The registry record does not report a clinically important difference threshold, so the confidence intervals cannot be translated into a judgment about clinical importance. Their central message is that the treatment-versus-placebo estimates are uncertain, with the 90 mg and 220 mg intervals including zero.

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 sizes, not the trial’s original design calculations. For these continuous outcomes, the entries are participants per arm, rather than total events. They describe hypothetical future planning under the stated assumptions: a two-sided significance level of 0.05, equal allocation between the two arms, and a normal approximation. The assumed effect to detect is a stated fraction of the observed effect.

The 80% power column gives the planned number of participants per arm for a design with an 80% chance of detecting the assumed effect under those assumptions, if the assumptions and effect are correct. The 90% power column gives the corresponding per-arm planning number for a design with a 90% chance. These are design probabilities for hypothetical studies; they are not measures of the probability that the current result is true.

Each planning scenario uses a different fraction of the observed effect. Planning for 75% of the observed effect means targeting a smaller difference than the full observed estimate; planning for 50% means targeting a still smaller difference. Smaller effects are harder to distinguish from background variability, so they require more participants per arm to maintain the same target power. The planning numbers can become very large when the observed contrast is small relative to the estimated variability.

These figures are not observed power. They should not be used to reinterpret the trial’s results as if the study had a known probability of success after observing its estimates. The registry record does not report the trial’s original sample-size justification, so the planning illustrations do not explain why the original enrollment was chosen.

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 = -0.350; pooled standard deviation σ = 1.559.
2. n = 2 × (1.96 + 0.842)² × 1.559² / -0.350² = 311.6, rounded up to 312 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 <- -0.35                      # observed difference in means
sigma <- 1.55939156445136                     # 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(760578)
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 = 312 (80% power), 418 (90% power)
 75% of observed effect: n per arm = 554 (80% power), 742 (90% power)
 50% of observed effect: n per arm = 1,247 (80% power), 1,669 (90% power)
power.t.test: 312.6 
Simulated power at n = 312 per arm: 0.798 (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. Multiplicity, Missing Data and Analysis Populations

The registry record lists seven outcome measures and posts several treatment arms and secondary endpoints. Testing multiple outcomes and contrasts creates a multiplicity issue: even when each individual test uses a conventional threshold, the chance of at least one apparently unusual result across a collection of tests can increase. Statistical plans often address this by defining a primary comparison, a testing hierarchy, or an adjustment procedure. The registry record does not report whether such a procedure was planned or used.

The primary endpoint is identified, but the posted comparison calculations are unadjusted recalculations from summary statistics. They should not be treated as a substitute for the sponsor’s complete analysis. The record lists zero analyses in its analysis listing and does not report sponsor-reported analyses for the secondary outcomes. The discrepancy indicator is false, but this does not supply the missing details of the sponsor’s statistical methods.

Missing-data handling is also not reported in the registry record. The endpoint population is described as compliant patients with Day 8 and Day 43 assessments and laboratory assessments, which implies a defined analysis population based on compliance and observed assessments. The number of patients excluded from that population, reasons for missing assessments, and methods used to address missing values are not reported. The record also does not describe whether all randomized patients were included in any analysis or whether the endpoint population was defined before results were examined.

These omissions affect how broadly the estimates can be generalized to the randomized participants. A compliant-participant population with specified assessments may differ from all enrolled patients, particularly if compliance or completion is related to outcomes. That possibility is a general concern, not evidence that such differences occurred in this study. Without a reported participant flow, missing-data strategy, multiplicity plan, and full analysis specification, readers cannot determine how sensitive the reported results are to these choices.

15. 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 registry record lists serious adverse events affecting zero participants in each of the four groups: MSDC-0160 90 mg, placebo, pioglitazone, and MSDC-0160 220 mg. Each group’s serious-event risk count is also listed, with 22, 22, 21, and 21 participants, respectively. These figures describe the registry’s serious adverse event summaries and should not be taken to mean that no adverse experiences of any kind occurred.

Other adverse events are listed as affecting 4 participants in the MSDC-0160 90 mg group, 3 in the placebo group, 2 in the pioglitazone group, and 2 in the MSDC-0160 220 mg group. The record does not provide serious adverse event terms, and no top serious terms are listed. Death counts are not reported in the registry record.

These group counts are descriptive. They do not establish whether differences between groups are meaningful, and the small group sizes limit how much can be inferred about uncommon events. The record’s safety figures should be interpreted within the study’s short 28-day primary timeframe and alongside the limited detail available on event definitions, timing, severity, and relatedness.

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

The posted primary results describe short-term changes in averaged postprandial glucose during a mixed-meal tolerance test. The treatment-versus-placebo estimates suggest different observed mean changes across the two MSDC-0160 doses, but the recalculated unadjusted confidence intervals for both comparisons include zero. This means the posted summaries do not provide a precise estimate that excludes no difference under those calculations. The pioglitazone comparison is more separated from zero in the recalculation, but that does not turn the MSDC-0160 estimates into confirmatory findings.

These results do not establish whether MSDC-0160 is effective or ineffective. They describe an endpoint over 28 days in a small Phase 2A study, using the endpoint population defined by compliance and availability of assessments. They do not establish long-term effects, effects on clinical outcomes, or the results that might be observed in a different patient population or study design. The registry record does not report a threshold for a clinically meaningful difference in the primary measure.

The comparator structure is also important. The posted recalculated contrasts compare each treatment arm with placebo. They are not direct estimates of the difference between MSDC-0160 and pioglitazone. Although pioglitazone was included as an active comparator, a numerical contrast against placebo should not be presented as a head-to-head comparison between the active treatments.

The safety summaries record zero participants with serious adverse events in each listed group, alongside some participants with other adverse events. These counts are not enough to characterize uncommon risks, and the registry record does not report deaths. More broadly, interpretation is limited by the absence of the original sample-size rationale, complete analysis methods, multiplicity strategy, and missing-data details. The most defensible reading is therefore descriptive: the record reports short-term mean changes and uncertain unadjusted contrasts, rather than a complete basis for a definitive efficacy or safety conclusion.

17. Why This Trial Matters Statistically

This trial is statistically informative because it illustrates the difference between an observed estimate and a precise estimate. The primary mean changes vary across arms, but the recalculated treatment-versus-placebo intervals for the two MSDC-0160 doses include zero. Looking only at the point estimates would conceal the uncertainty; considering the intervals makes clear that a range of effects remains compatible with the posted summaries.

The study also illustrates how a multiple-arm design supplies context without automatically answering every comparison. Placebo and pioglitazone were included alongside two MSDC-0160 doses, but the posted recalculations are each against placebo. Direct comparisons between the active treatments are not reported. Keeping each contrast distinct prevents an indirect comparison from being mistaken for a randomized head-to-head estimate.

Another statistical lesson is the importance of separating the registry’s reported outcomes from recalculated summaries. The arm-level least-squares means and standard errors are posted, while the comparison estimates and p-values described in this analysis are unadjusted recalculations. Because the full sponsor analysis and multiplicity plan are not reported, the calculations provide a transparent way to inspect the summaries but do not reproduce a complete prespecified analysis.

Finally, the sample-size illustrations show why planning depends strongly on the effect a future study aims to detect. Smaller target effects require more participants per arm under the stated assumptions. Such planning is distinct from post hoc claims about power in the completed study. The record’s missing original sample-size rationale means the enrollment cannot be linked confidently to a target effect or precision goal.

18. 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 trial’s primary endpoint is a short-term laboratory measure, with a listed timeframe of 28 days. The registry record does not report a clinically important difference threshold, and it does not establish how a measured change relates to longer-term outcomes. Consequently, statistical differences on this measure cannot be translated directly into clinical importance.

Primary endpoint counts range from 16 to 21 per arm, and the endpoint population is defined by treatment compliance and completion of specified assessments. The registry record does not report participant flow, the number excluded from the endpoint population, reasons for missing data, or how missing values were handled. These details matter because the analyzed subset may not represent all enrolled participants.

The posted comparison calculations are unadjusted and use approximate pooled variability inferred from standard errors. The registry record does not report the sponsor’s complete model, covariate adjustment, or inferential results. Although the discrepancy indicator is false, the missing analysis details still limit comparison between the recalculations and the sponsor’s intended analysis. The multiplicity strategy is also not reported despite several outcomes and treatment contrasts.

The registry lists seven outcome measures, while four secondary outcomes are posted. The relationship between all listed measures and the analyses conducted is not fully described. The original sample-size justification, interim-analysis plans, and detailed randomization procedures are not reported in the registry record. These limitations constrain conclusions about the design’s intended precision, the role of secondary findings, and the robustness of the results.

19. Related Tutorials

20. Related Statistical Calculators

21. Sources

• ClinicalTrials.gov: A Placebo Controlled Safety and Efficacy Study of MSDC-0160 in Type 2 Diabetes With Active Comparator — NCT00760578.
• Registry record last updated 2013-05-03; status last verified 2013-03.
• Results first posted 2013-05-03.
• 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.

22. Record Summary

This completed randomized Phase 2A parallel study enrolled 86 people with Type 2 diabetes and compared placebo, pioglitazone 45 mg, and MSDC-0160 at 90 mg and 220 mg once daily. The primary endpoint was change from baseline in averaged postprandial glucose during a mixed-meal tolerance test over 28 days.

The recalculated unadjusted comparisons of both MSDC-0160 doses with placebo have confidence intervals that include zero. The posted results provide short-term estimates, but the registry record does not report the complete sponsor analysis, original sample-size rationale, multiplicity plan, or missing-data methods. Serious adverse events were listed for zero participants in each group; other adverse events affected participants in all groups, and deaths are not reported in the registry record.