GLP-1 Receptor Agonist Lixisenatide in Patients With Type 2 Diabetes for Glycemic Control and Safety Evaluation, on Top of Basal Insulin +/- Sulfonylurea (GETGOAL-L-ASIA): Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for GLP-1 Receptor Agonist Lixisenatide in Patients With Type 2 Diabetes for Glycemic Control and Safety Evaluation, on Top of Basal Insulin +/- Sulfonylurea (GETGOAL-L-ASIA) (NCT00866658), a randomized phase 3 study. The primary outcome is Absolute Change From Baseline in Glycosylated Hemoglobin (HbA1c) at Week 24.

2. Clinical Question

3. Trial Design

The registry describes a Phase 3, randomized, double-blind, placebo-controlled, two-arm parallel-group, multicenter study. Its stated treatment period was 24 weeks. The trial is marked completed, and enrollment is recorded as 311. It was sponsored by Sanofi. The start is listed as 2009-03 and primary completion as 2010-06.

The study population was people with type 2 diabetes insufficiently controlled with basal insulin, with or without sulfonylurea. Both groups continued basal insulin, and sulfonylurea could be part of background treatment. One group received lixisenatide and the other received matching placebo. The described lixisenatide and placebo regimens each began at 10 mcg once daily for 1 week, increased to 15 mcg once daily for 1 week, then continued at 20 mcg once daily up to Week 24.

The primary endpoint was change from baseline in HbA1c at Week 24. The primary outcome description specifies that change was calculated as the Week 24 value minus the baseline value. The efficacy period was defined from the first study-drug dose until 3 days after the last dose or introduction of rescue therapy, whichever came first. The record also lists secondary outcomes covering postprandial and fasting plasma glucose, body weight, self-monitored glucose, insulin dose, HbA1c thresholds, and rescue therapy, as well as safety and other objectives.

Randomization and double masking support a controlled comparison, but the registry record does not report the allocation procedure, concealment details, or which roles were masked. The trial’s original sample-size justification and other original design assumptions are not reported in the registry record.

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: A total of 437 patients were screened of which 126 (28.8%) were screen failures; main reason for screen failure was glycosylated hemoglobin (HbA1c) values being out of the defined protocol range (greater than or equal to 7% and less than or equal to 10%). A total of 311 patients were randomized.

Overall Study

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: mITT population:all randomized patients who received at least 1 dose;had baseline,at least 1 post-baseline efficacy assessment, irrespective of compliance with study protocol/procedures. Last observation carried forward used. Number of patients analyzed=patients with baseline and at least 1 post-baseline HbA1c assessment during on treatment period.

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: Safety population included all randomized patients who were exposed to at least 1 dose of study drug, regardless of the amount of treatment administered.

6. Primary Endpoint

The registry lists the primary outcome as Absolute Change From Baseline in Glycosylated Hemoglobin (HbA1c) at Week 24, measured over Baseline, Week 24.

The primary endpoint is absolute change from baseline in glycosylated hemoglobin (HbA1c) at Week 24, measured as a percentage of hemoglobin. The record defines change by subtracting baseline HbA1c from the Week 24 value. A negative change therefore represents a lower Week 24 value than baseline, while a positive change represents a higher value. This describes the direction of the numerical change; it does not by itself establish the importance of that change for an individual.

The registry identifies HbA1c reduction at Week 24 as the primary measure of glycemic control for the study’s main objective. Using a continuous measure retains information about the size of the change across participants, rather than reducing the primary outcome to whether each participant did or did not cross a threshold. HbA1c thresholds were also included among the secondary outcomes.

The reported arm summaries are least-squares means with standard errors. The posted primary analysis reports an adjusted least-squares mean difference between groups. The analysis population was modified intention-to-treat: participants had to be randomized, receive at least one dose, and have baseline and at least one post-baseline efficacy assessment. Last observation carried forward was used. The on-treatment efficacy period ended at the earliest of the specified post-dose limit or the start of rescue therapy.

7. Results Posted in the Registry

• Lixisenatide: mean -0.77 versus 0.11 on control; difference -0.88 (95% CI -1.25 to -0.51), standardized effect d = -0.54; z-test p = <0.001.

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 2-hour Postprandial Plasma Glucose (PPG) at Week 24

• 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:
  • Lixisenatide: mean -7.96 versus -0.14 on control; difference -7.82 (95% CI -9.43 to -6.21), standardized effect d = -1.15; z-test p = <0.001.
• Interpretation:
  • The 95% confidence interval excludes no difference, so the mean is statistically lower for Lixisenatide than for the control. This is an unadjusted, secondary comparison, so it is suggestive rather than confirmatory.

Change From Baseline in Body Weight at Week 24

• 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:
  • Lixisenatide: mean -0.38 versus 0.06 on control; difference -0.44 (95% CI -1.21 to 0.33), standardized effect d = -0.13; z-test p = 0.262.
• Interpretation:
  • The 95% confidence interval includes no difference, so these data do not show a statistically significant difference between Lixisenatide and the control. The interval gives the range of effects that remain compatible with the data.

Change From Baseline in Average 7-Point Self Monitored Plasma Glucose (SMPG) Profile at Week 24

• 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:
  • Lixisenatide: mean -1.91 versus -0.56 on control; difference -1.35 (95% CI -2.10 to -0.60), standardized effect d = -0.42; z-test p = <0.001.
• Interpretation:
  • The 95% confidence interval excludes no difference, so the mean is statistically lower for Lixisenatide than for the control. This is an unadjusted, secondary comparison, so it is suggestive rather than confirmatory.

The posted secondary outcomes cover several related but distinct aspects of glucose control and treatment burden, alongside body weight and rescue therapy. The available continuous summaries show a broadly favorable direction for the glucose measurements in the lixisenatide group compared with placebo, while the body-weight comparison is less conclusive. Those endpoints do not all have the same scale, analysis population, or precision, so their estimates should be read individually rather than treated as interchangeable confirmations of the primary endpoint.

These measures relate to the primary HbA1c result by describing other outcomes over the study period. The secondary glucose measures capture outcomes other than the primary HbA1c change, and the body-weight and insulin-dose measures address different questions. Threshold outcomes and the percentage requiring rescue therapy were among the listed outcomes, but detailed results for all listed measures are not available in the posted secondary summaries. The registry lists 12 outcome measures and reports one primary analysis; the record does not establish that every secondary outcome has a posted analysis.

Unadjusted secondary results generally carry less evidential weight than a prespecified primary result because many outcomes can be examined, increasing the chance that some appear notable by chance. The registry record does not report a multiplicity adjustment or a formal hierarchy for secondary hypotheses. Without those details, secondary findings are best treated as supportive or exploratory evidence rather than as independent confirmatory conclusions. The simple recalculations are also not substitutes for any full adjusted analysis that may have been conducted but is not reported in the registry record.

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

• Change From Baseline in 2-hour Postprandial Plasma Glucose (PPG) at Week 24
• Change From Baseline in Body Weight at Week 24
• Change From Baseline in Average 7-Point Self Monitored Plasma Glucose (SMPG) Profile at Week 24
• Change From Baseline in Fasting Plasma Glucose (FPG) at Week 24
• Change From Screening in Total Insulin Dose at Week 24
• Percentage of Patients With Glycosylated Hemoglobin (HbA1c) Level Less Than 7% at Week 24
• Percentage of Patients With Glycosylated Hemoglobin (HbA1c) Level Less Than or Equal to 6.5% at Week 24
• Percentage of Patients Requiring Rescue Therapy During 24-Week Period

9. Statistical Interpretation

10. Statistical Methods Explained

The registry reports an ANCOVA analysis for the primary comparison, with the result expressed as a least-squares mean difference. ANCOVA is a regression-based approach that compares groups while accounting for covariates included in the model. Least-squares means are model-based group estimates, and their difference is the adjusted contrast between groups. The covariates and full model specification are not reported in the registry record, so the adjustment cannot be reconstructed from the posted summary.

The arm-level primary summaries are least-squares means with standard errors: 0.11 for placebo and -0.77 for lixisenatide. Their respective standard errors are 0.131 and 0.137. A standard error describes uncertainty in an estimated mean; it is not the standard deviation of individual participant outcomes. The registry’s reported comparison is -0.88, with a 95% confidence interval from -1.116 to -0.650 and p < 0.001.

What does the adjusted comparison represent?

The adjusted comparison estimates the difference between treatment groups after accounting for the terms included in the ANCOVA model. In general, adjustment can improve the precision of a comparison when relevant baseline information is included, but the registry does not identify the model terms. The sponsor’s reported adjusted estimate should therefore be distinguished from a calculation made directly from the arm summaries. The two approaches can differ because one is model-based and adjusted while the other is a simple unadjusted calculation.

The recalculation associated with the posted results is explicitly an approximate, unadjusted comparison based on summary statistics. It gives an estimate of -0.88 and p <0.001 for the primary endpoint. It is useful as a check on the direction and broad scale of the reported comparison, but it does not reproduce the ANCOVA model. The registry reports no discrepancy between the recalculation and the sponsor’s analysis.

How should the analysis population be understood?

The primary outcome population is described as modified intention-to-treat. It includes randomized participants who received at least one dose and had baseline and at least one post-baseline efficacy assessment. The record says the analysis used last observation carried forward. The on-treatment period for this efficacy variable ran from the first study-drug dose through 3 days after the last dose, or until rescue therapy began, whichever occurred first. These criteria mean the primary result is not a summary of every randomized participant regardless of available outcome data.

In general, last observation carried forward fills a missing later measurement using an earlier observed value. This makes an analysis possible for participants without a Week 24 value, but it relies on assumptions about how the unobserved outcome would have evolved. The registry record does not report the amount or pattern of missing primary outcome data, sensitivity analyses for missingness, or a detailed justification for this method.

What does the p-value tell the reader?

In general, a p-value measures how incompatible the observed data are with a specified null model, assuming that model and the analysis assumptions hold. It is not the chance that the null hypothesis is true, and it does not measure the size or importance of a treatment effect. The sponsor reports p < 0.001 for the primary comparison; the simple recalculation reports p <0.001. These values come from different analysis procedures and should not be treated as interchangeable estimates of the same adjusted model.

The planning illustrations use a two-sided significance level of 0.05, equal allocation between the two arms, and a normal approximation. They use fractions of the observed effect to illustrate participant counts for stated power targets. These are retrospective planning illustrations, not a description of how the trial’s own size was originally chosen. The trial’s original sample-size justification and original design assumptions are not reported in the registry record.

Why can variation matter?

For continuous outcomes, variability affects how precisely a group mean and a between-group difference can be estimated. In general, greater variation makes it harder to distinguish a group difference of a given size from random variation. The primary arm summaries report standard errors rather than standard deviations. The unadjusted recalculation uses an approximate pooled standard deviation derived from the posted summaries; it is not a participant-level estimate from the analysis dataset. This distinction matters when reading planning illustrations based on the observed effect.

The registry record does not report the complete statistical analysis plan, model diagnostics, assumptions checks, or details of any interim analyses. None should be inferred from the endpoint summaries alone.

11. Confidence Intervals and Precision

The sponsor-reported primary estimate is a least-squares mean difference of -0.88 percentage points, with a 95% confidence interval from -1.116 to -0.650. The interval lies below zero, and the reported p-value is p < 0.001. Together, these statistics indicate that the estimated group difference is negative under the reported ANCOVA analysis and quantify uncertainty around that estimate.

In general, a confidence interval gives a range of values compatible with the data and the method used, at the stated confidence level. A 95% confidence interval is not a range for individual participants, and it does not mean there is a 95% probability that the fixed treatment effect lies inside this particular interval. Its width conveys precision: a narrower interval indicates greater precision than a wider interval under comparable methods and measures.

The endpoint arm summaries have standard errors of 0.131 for placebo and 0.137 for lixisenatide. These describe precision of the reported arm means, not the spread of individual HbA1c changes. The sponsor’s interval reflects the reported adjusted analysis, whereas the simple recalculation is unadjusted and approximate. The recalculated estimate is -0.88; its p-value is p <0.001. The adjusted result and recalculation should therefore be interpreted according to their respective methods.

Precision is specific to this endpoint, population, and analysis. It does not establish that all secondary outcomes are estimated with similar precision. Nor does statistical precision alone determine whether the magnitude is important in clinical practice. The registry does not report a clinical-importance threshold for this comparison.

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 participants per arm, not total events. They are illustrations computed from the observed effect, with the effect to detect set in turn to 100%, 75%, or 50% of that observed effect. The stated assumptions are a two-sided significance level of 0.05, equal allocation between the two arms, and a normal approximation. These are planning assumptions for the illustrations; the trial’s original sample-size assumptions and justification are not reported in the registry record.

The 80% power column gives the illustrative number of participants per arm for an analysis to have an 80% chance of detecting the specified effect under the stated assumptions, if that effect is the true one. The 90% power column gives the corresponding number for a 90% chance. Power is a property of a design under assumptions, not a guarantee about what a particular trial will find. These figures are not observed power.

For the primary endpoint, the illustration for 100% of the observed effect is 55 participants per arm for 80% power and 74 per arm for 90% power. For 75% of the observed effect, the corresponding figures are 97 and 130; for 50%, they are 219 and 293. Smaller effects need more participants to be detected with the same power under the same assumptions. The figures should not be read as the original enrollment target or as a retrospective judgment of whether the trial was adequately designed.

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 = -0.880; pooled standard deviation σ = 1.640.
2. n = 2 × (1.96 + 0.842)² × 1.640² / -0.880² = 54.5, rounded up to 55 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 <- -0.88                      # observed difference in means
sigma <- 1.64019070343182                     # 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(866658)
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 = 55 (80% power), 74 (90% power)
 75% of observed effect: n per arm = 97 (80% power), 130 (90% power)
 50% of observed effect: n per arm = 219 (80% power), 293 (90% power)
power.t.test: 55.5 
Simulated power at n = 55 per arm: 0.800 (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 sponsor’s own primary analysis is an ANCOVA, reported as a least-squares mean difference of -0.88, with a 95% confidence interval from -1.116 to -0.650 and p < 0.001. That is the analysis to use when describing the trial’s reported primary result.

The accompanying recalculation uses posted arm-level summary statistics to make a simple, unadjusted comparison. It also estimates the mean difference as -0.88, with p <0.001. Because this recalculation is approximate and does not reproduce the ANCOVA adjustment, its p-value and uncertainty are not a replacement for the sponsor’s result. The registry does not flag a discrepancy between the two estimates.

Agreement in the direction and rounded estimate is a useful consistency check, but it does not mean that the two analyses use identical assumptions or answer every statistical question in the same way. The model covariates and full ANCOVA specification are not reported in the registry record. In this case, the sponsor’s analysis remains the primary reported analysis, while the recalculation provides a supplementary unadjusted view.

15. Multiplicity, Missing Data and Analysis Populations

The primary endpoint is clearly identified as change from baseline in HbA1c at Week 24, and the registry reports an ANCOVA result for it. The record also lists multiple secondary outcomes, including glucose measures, body weight, insulin dose, HbA1c thresholds, and rescue therapy. Testing many outcomes can increase the chance of observing at least one nominally significant result even when there is no corresponding underlying effect. In general, multiplicity methods address that issue by controlling the error rate across a set of tests or by specifying an ordered testing strategy.

Whether multiplicity was addressed, and if so how, is not reported in the registry record. A hierarchy of secondary tests, adjusted p-values, and a family-wise error strategy are not reported. The secondary results should therefore not be assumed to have confirmatory status based solely on their numerical direction or unadjusted p-values. The primary result is distinguishable from the broader set of outcomes because it is explicitly designated as primary.

The record defines the primary analysis population as randomized participants who received at least one dose and had baseline and at least one post-baseline efficacy assessment. It states that last observation carried forward was used. The efficacy period ended at the earlier of 3 days after the final study-drug dose or the start of rescue therapy. These details affect which measurements contribute to the primary estimate and how later missing observations were handled.

The number and pattern of missing primary outcome measurements, reasons for missingness, and sensitivity analyses are not reported in the registry record. The statistical assumptions behind carrying the last observed value forward are also not described. The full statistical analysis plan, including details of prespecification, model terms, and any interim analyses, is not reported in the registry record. No conclusion about those unreported procedures can be drawn from the posted outcome alone.

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: cerebral infarction (1.3%), herpes zoster oticus (0.6%), upper respiratory tract infection (0.6%), hypertensive crisis (0.6%), colonic polyp (0.6%).

The registry reports serious adverse events in 9 participants among 157 at risk in the placebo group and 10 among 154 at risk in the lixisenatide group. It separately reports other adverse events affecting 71 participants among 157 at risk in the placebo group and 125 among 154 at risk in the lixisenatide group. These are counts of participants reported in the two categories, not estimates of comparative risk adjusted for follow-up or other factors.

The listed serious adverse event terms include cerebral infarction at 1.3%, and herpes zoster oticus, upper respiratory tract infection, hypertensive crisis, colonic polyp, and nausea at 0.6% each. The registry record does not provide further detail in these summaries about event timing, duration, attribution, or clinical course. Death counts are not reported in the registry record.

These figures should be read as descriptive safety reporting for the study groups. They do not establish that an event was caused by a study treatment, and the counts alone do not support a comprehensive comparison of safety. The record’s safety summaries are separate from the primary efficacy analysis and should be considered in the context of the study’s population and treatment period.

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

The primary result describes a lower mean HbA1c change in the lixisenatide group than in the placebo group over the study’s stated treatment period, under the sponsor-reported ANCOVA analysis. The estimated difference is -0.88 percentage points, with a 95% confidence interval from -1.116 to -0.650. This is a group-level result for the analysis population defined in the registry, not a statement that every participant experienced that change.

The result does not show the distribution of individual changes, identify which participants had larger or smaller changes, or establish the mechanism behind the observed group difference. Nor does a p-value establish clinical importance. The registry does not report a threshold for a clinically important difference, and the statistical result alone cannot answer that question.

The study compared lixisenatide with placebo as an addition to basal insulin, with or without sulfonylurea, in a specific population and over 24 weeks. The result should not be extended automatically to different populations, background regimens, treatment durations, or outcomes not assessed in the trial. The registry’s stated secondary outcomes and safety summaries offer additional information, but they do not remove the limits imposed by the study’s design and reporting.

The trial record marks the study completed. Its summary is evidence about the measured outcomes within the reported analysis, not individual medical advice or a guarantee of future outcomes. The registry does not report every detail needed to assess all aspects of the statistical plan, missing data, multiplicity, or longer-term effects.

18. Why This Trial Matters Statistically

This trial illustrates why the primary endpoint and analysis population matter when reading a clinical result. HbA1c change at Week 24 is identified as the primary measure, and the reported comparison is an adjusted least-squares mean difference from ANCOVA. That makes it possible to distinguish the trial’s stated primary analysis from supplementary calculations based only on arm summaries.

It also shows why the estimate and its precision should be read together. The difference of -0.88 is accompanied by a 95% confidence interval from -1.116 to -0.650 and a reported p-value of p < 0.001. The estimate conveys the observed group contrast, while the interval conveys uncertainty under the reported analysis. Neither statistic alone answers whether the difference is clinically important.

The secondary outcomes broaden the statistical picture but create interpretive challenges. They address several outcomes with different units and meanings, and multiplicity procedures are not reported. A pattern across outcomes can add context, but unadjusted secondary findings should not be assigned the same evidential weight as a designated primary analysis.

Finally, the record demonstrates the importance of transparent reporting. It identifies the use of last observation carried forward and defines the modified intention-to-treat population, yet does not report the full model specification, the extent and pattern of missingness, or multiplicity methods. Those details affect how confidently readers can assess robustness. Distinguishing what is reported from what remains undocumented is a central part of careful statistical interpretation.

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 outcome analysis used last observation carried forward, but the registry record does not report how many primary measurements were missing, why they were missing, or whether sensitivity analyses evaluated alternative assumptions. This limits assessment of how missing data may have affected the estimate.

The record reports an ANCOVA result but does not report the model covariates or full specification. The modified intention-to-treat definition also requires receipt of at least one dose and at least one post-baseline efficacy assessment, so the analysis population is narrower than all randomized participants. The registry does not provide enough detail to assess the consequences of those eligibility criteria for the analysis.

Many secondary outcomes are listed, but multiplicity handling and a testing hierarchy are not reported. The record lists 12 outcome measures and one primary analysis; detailed results are not posted for every listed secondary outcome. This constrains interpretation of the totality of secondary findings.

The primary comparison covers a 24-week treatment period. Longer-term results are not reported in the registry record. The safety summaries provide participant counts and selected serious adverse event terms, but do not include the detail needed to assess timing, attribution, or event course. Death counts are not reported in the registry record.

The trial’s original sample-size justification, original design assumptions, full statistical analysis plan, and details of interim analyses are not reported in the registry record. These reporting gaps do not invalidate the posted result, but they limit independent evaluation of the complete design and analysis process.

20. Related Tutorials

21. Related Statistical Calculators

22. Sources

• ClinicalTrials.gov: GLP-1 Receptor Agonist Lixisenatide in Patients With Type 2 Diabetes for Glycemic Control and Safety Evaluation, on Top of Basal Insulin +/- Sulfonylurea (GETGOAL-L-ASIA) — NCT00866658.
• Registry record last updated 2016-10-11; status last verified 2016-08.
• Results first posted 2016-10-11.
• 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

NCT00866658 is a completed, randomized, double-blind, placebo-controlled, two-arm parallel-group, multicenter Phase 3 study sponsored by Sanofi. It enrolled 311 participants with type 2 diabetes insufficiently controlled with basal insulin, with or without sulfonylurea, and compared add-on lixisenatide with matching placebo over 24 weeks.

The primary outcome was change from baseline in HbA1c at Week 24. The sponsor-reported ANCOVA estimate was -0.88 percentage points, with a 95% confidence interval from -1.116 to -0.650 and p < 0.001. The primary analysis used the defined modified intention-to-treat population and last observation carried forward. Secondary outcomes covered additional glucose measures, body weight, insulin dose, thresholds, rescue therapy, and safety. Details of multiplicity handling and several other elements of the statistical plan are not reported in the registry record.