Feasibility of Once/Daily Administered GLP/1 Receptoragonist (Lixisenatide) in Combination With Basal Insulin: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Feasibility of Once/Daily Administered GLP/1 Receptoragonist (Lixisenatide) in Combination With Basal Insulin (NCT02168491), a na phase 3 study. The primary outcome is Change in HbA1c From Baseline to End.

2. Clinical Question

The clinical question concerns the feasibility of using lixisenatide in combination with basal insulin after a change from premixed insulin. The registry’s background describes the rationale as an approach to treatment intensification, but the posted study design does not compare this regimen with continued premixed insulin or another treatment. It therefore cannot establish what would have happened to the same patients under an alternative regimen.

The primary endpoint is the change in HbA1c from baseline to the end of the study, with a timeframe of 12 weeks. The listed secondary outcomes are change in fasting plasma glucose, described as a mean over 2 weeks, and change in body weight from baseline to the end of the study. The record does not give numerical results for these outcomes. This leaves the central descriptive question—the size and direction of the observed changes—unanswered by the registry record.

In general, a single-group study can describe outcomes observed after an intervention and can help identify practical questions for later research. Without a concurrent comparator, however, changes cannot be separated from time trends, other care, measurement variation, or changes that might have occurred without the intervention. This distinction matters when interpreting the study’s primary question.

3. Trial Design

The registry record identifies this as a completed Phase 3 study sponsored by the Medical University of Vienna. It lists a single-group model, no masking, and allocation as NA. The recorded enrollment is 10. The study start is listed as 2014-11, and primary completion as 2015-07.

The intervention group description says that patients with type 2 diabetes were to be switched from premixed insulin to insulin glargine and lixisenatide. Both drugs are listed as interventions. There is no separate comparator arm in the recorded design. Consequently, the study is not a randomized comparison, and its baseline measurements should not be treated as if they came from an independent control group.

The primary outcome is change in HbA1c from baseline to the end of the study, reported as a mean with standard deviation and a timeframe of 12 weeks. The secondary outcomes concern fasting plasma glucose and body weight. The registry lists three outcome measures and three analyses, but does not report the analytical details associated with those analyses.

The record does not report the recruitment setting, participant eligibility criteria beyond the description of type 2 diabetes and inadequate therapeutic targets with premixed insulin, treatment doses, adherence procedures, or visit schedule. It also does not report a sample-size justification, a protocol-level statistical analysis plan, or details about outcome assessment. Those omissions limit how fully the conduct and intended interpretation of the study can be reconstructed from the registry record.

Because the design is single-group and unmasked, interpretation is descriptive rather than comparative. The recorded completion status indicates that the study is marked completed; it does not itself supply evidence about the size, precision, or direction of any clinical outcome.

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: 2 patients declined to participate because of time restraints, thus study medication was administered in 9 patients

Overall Study

Reasons for not completing this 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.

6. Primary Endpoint

The registry lists the primary outcome as Change in HbA1c From Baseline to End, measured over 12 weeks.

The primary endpoint is change in HbA1c from baseline to the end of the study, with a stated timeframe of 12 weeks. HbA1c is a continuous measure, and the registry specifies that the result is characterized by a mean and standard deviation. The record’s description says that a change between two time points is reported.

In general, HbA1c is used to assess longer-term glycemic status, so measuring change can describe whether the average value at follow-up differs from baseline. A mean summarizes the average change across participants, while a standard deviation describes how much individual changes vary around that mean. These are different quantities: the standard deviation is not, by itself, a confidence interval or a measure of uncertainty in the estimated mean.

The numerical mean change, its standard deviation, and any confidence interval or hypothesis-test result are not reported in the registry record. The direction and magnitude of the observed endpoint therefore cannot be characterized from the posted results described in the record. Nor can the precision of the estimated change be assessed without the numerical result and an uncertainty measure.

The endpoint is a before-and-after measure in a single-group study. In general, an observed change can describe what happened over the study period, but it does not isolate the effect of the intervention from other influences. A concurrent control group would be needed for a direct comparative estimate; none is recorded for this study.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What does a mean change describe?

In general, a mean change is the average of participants’ differences between a follow-up measurement and baseline. It gives a concise description of the group’s average direction and magnitude of change. The registry specifies the primary outcome as a mean change in HbA1c from baseline to the end of the study, with a standard deviation, over 12 weeks. It does not report the numerical mean or standard deviation.

A standard deviation describes variation among individual observations around the mean. It is not interchangeable with a standard error or a confidence interval. A confidence interval is, in general, a range calculated using an estimation method to express uncertainty about a population quantity. No confidence interval for the primary outcome is reported in the registry record, so the uncertainty of the mean estimate cannot be judged from the posted results.

What can a single-group design estimate?

In general, a single-group before-and-after study can describe the outcomes observed in its participants over time. It does not directly estimate a contrast between treatment strategies because there is no separate concurrent control group. A participant’s baseline value is useful as a reference for measuring change, but it is not a substitute for an independent control condition. The registry lists this study as single-group, with allocation marked NA, and no masking.

Without a comparison group, the change could reflect the intervention together with other influences. These may include changes in care, events during follow-up, measurement variation, or the underlying course of the condition. The design alone cannot distinguish these explanations. Thus, the primary endpoint would be a within-group description, not a randomized estimate of the treatment effect relative to another approach.

Why does the analysis plan matter?

In general, an analysis plan states how outcomes are defined, which participants are included, how missing observations are handled, and which statistical procedures are used. Prespecification helps readers understand whether analyses followed planned decisions or were selected after results were known. The registry record lists three outcome measures and three analyses, but the detailed statistical analysis plan is not reported in the registry record. The treatment of missing measurements, analysis populations, and any adjustments is also not reported in the registry record.

The record specifies a continuous outcome summarized as a mean with standard deviation. It does not state whether the primary analysis used paired observations, how distributional assumptions were assessed, or whether an alternative method was planned if those assumptions were unsuitable. These details matter because different methods can yield different estimates and uncertainty measures, especially in a small study.

What is over-dispersion, in general?

For count outcomes, over-dispersion means that observed variability is greater than a model’s assumed variability. In general, failing to account for it can make standard errors too small and confidence intervals too narrow. This concept is not a reported feature of this study’s continuous primary endpoint, and the registry record does not describe a count-outcome model. It is a general statistical consideration rather than a finding about this study.

How should planning illustrations be read?

The planning assumptions associated with the study describe an illustration based on an observed effect, using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. The effect to detect is specified as a fraction of the observed effect. These are assumptions for an illustration, not the reported design of this single-group study. The registry record does not report that these assumptions were used to plan the study.

In general, a sample-size calculation asks how many participants would be needed under stated assumptions to detect a specified effect with a chosen probability. Such a calculation is conditional on its assumptions and is not evidence that a study had that probability of detecting an effect after it was completed. An illustration using an observed effect is especially dependent on that estimate and should not be described as observed power. No numerical planning results are stated in the registry record.

Because this study has no two-arm allocation, the equal-allocation assumption in the planning illustration does not match its recorded design. The illustration therefore should not be interpreted as the study’s own sample-size justification or as a retrospective assessment of its ability to detect a treatment difference.

What can be concluded from a non-significant or unreported result?

In general, a p-value describes how compatible data are with a specified statistical model and null hypothesis; it is not the probability that a treatment works, nor does it measure the size or importance of an effect. The registry record does not report a p-value for the primary outcome. Since the numerical result itself is not reported, no statistical conclusion about the endpoint can be drawn from the registry information.

Even when a p-value is available, interpretation should consider the estimate and its uncertainty, the design, outcome definition, and analysis choices. In this study, the absence of a concurrent comparator is central: a test of within-group change would not establish a comparative effect. The missing numerical outcome and missing analysis details further restrict interpretation.

10. Confidence Intervals for Each Arm

No two-arm comparison is shown for this trial, but each arm's mean can be given with a t-based 95% confidence interval computed from the posted mean, spread and number of participants.

R code and output

arm <- c("Lixisenatide With Basal Insulin (LixiBIT)")
n   <- c(9)
m   <- c(-0.54)
sp  <- c(0.52)
sd  <- sp      # posted standard deviations
for (i in seq_along(n)) {
  se <- sd[i] / sqrt(n[i]); h <- qt(0.975, n[i] - 1) * se
  cat(sprintf("CARM|%s|%d|%.3f|%.3f|%.3f|%.3f\n", arm[i], n[i], m[i], sd[i], m[i] - h, m[i] + h))
}

CARM  Lixisenatide With Basal Insulin (LixiBIT)  9  -0.540  0.520  -0.940  -0.140

11. Multiplicity, Missing Data and Analysis Populations

The registry record lists three outcome measures and three analyses. The primary outcome is HbA1c change from baseline to the end of the study. The listed secondary outcomes are change in fasting plasma glucose, described as a mean over 2 weeks, and change in body weight from baseline to the end of the study. The detailed analysis plan and the numerical results are not reported in the registry record.

In general, multiplicity arises when several outcomes, time points, subgroups, or statistical tests are examined. Conducting multiple tests can increase the chance of finding at least one apparently unusual result by chance. Prespecified primary and secondary outcomes, alongside a stated adjustment or interpretation strategy, help readers assess this issue. The registry record does not report whether multiplicity adjustments were planned or how the three analyses relate to the listed outcomes. No adjustment method should therefore be assumed.

The record does not report how missing outcome measurements were handled, whether participants were analyzed according to a defined analysis population, or how departures from the intended intervention were addressed. It also does not describe an imputation method or a sensitivity analysis for missing data. These details are important because missing observations can affect both the estimate and its uncertainty, particularly if missingness is related to participants’ outcomes or study experience.

The registry records enrollment of 10, but the primary outcome population is not stated. The safety summary has 9 participants at risk in each of its listed categories; the relationship between those denominators and the primary outcome analysis is not described. The registry record therefore does not establish whether every enrolled participant contributed to each endpoint or analysis.

12. 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: elective ent surgery (11.1%).

The registry’s serious adverse-event summary lists 9 participants at risk and 1 affected in the group titled “Lixisenatide With Basal Insulin (LixiBIT).” The other-adverse-event summary lists 9 participants at risk and 3 affected. These are separate registry categories; the record does not explain whether the participants in them overlap, or how events were counted if an individual experienced more than one event.

The serious adverse-event term listed is elective ENT surgery, reported as 11.1%. That entry identifies a reported term, but the registry record does not provide further detail about timing, severity, clinical circumstances, or the investigator’s assessment of relatedness to treatment. It should not be interpreted as evidence that the intervention caused the event.

Deaths are not reported in the registry record. The record also does not provide a narrative describing how adverse events were collected, the observation period for each participant, or the definitions used for seriousness and other-event categories. These omissions limit comparisons and make it difficult to judge how comprehensively the summary reflects the study’s safety experience.

In general, adverse-event counts describe events observed and recorded during a study; they do not by themselves establish causality. A small, uncontrolled study also cannot provide a reliable comparative estimate of event risk against another treatment. The listed counts should therefore be read as descriptive registry information, not as a treatment comparison.

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

The study record describes a completed investigation of switching patients with type 2 diabetes from premixed insulin to insulin glargine and lixisenatide. Its primary endpoint is HbA1c change from baseline over 12 weeks. It also lists fasting plasma glucose and body weight outcomes. These registry entries describe the question and planned measurements, but the numerical results for those outcomes are not reported in the registry record.

That means the posted record does not establish the direction, size, or precision of the HbA1c change. It also does not allow readers to assess whether the observed changes in fasting plasma glucose or body weight were large or small. The absence of numerical outcomes is not evidence that there was no change; it means the registry record does not report the result needed to characterize one.

Even if the within-group changes were available, the design would remain non-comparative. There is no separate control group, and allocation is listed as NA. In general, measurements taken before and after an intervention can describe change over time, but cannot by themselves show what would have happened under an alternative treatment. The record therefore cannot establish a causal treatment difference.

The recorded enrollment is 10, and the study is unmasked. These features are relevant to how precisely and securely any estimates could be interpreted. The numerical endpoint estimates and their uncertainty are not reported, and the registry does not describe the analysis population or handling of missing data. Readers cannot use the record to assess the precision of the primary endpoint or determine how many participants contributed to each analysis.

The adverse-event information is also descriptive. The record lists 1 participant affected by a serious adverse event among 9 at risk and 3 affected by other adverse events among 9 at risk; the serious term reported is elective ENT surgery at 11.1%. The registry does not report deaths or provide details sufficient to assess event attribution. These counts do not establish whether events were caused by treatment or how the observed safety experience compares with another regimen.

In short, the registry record identifies a small, single-group study and its outcomes, but does not report the numerical primary or secondary outcome results. It cannot support a comparative efficacy conclusion, and the safety summary should not be read as a causal assessment.

14. Why This Trial Matters Statistically

This study is statistically notable because its central endpoint is a change over time in a single group, rather than a contrast between randomized groups. That distinction determines what the design can answer. A within-group change can describe the participants’ observed course, while a treatment comparison requires a credible estimate of what would have happened under another strategy. The registry lists no concurrent control group.

The study also makes the distinction between an endpoint definition and an endpoint result especially important. The record specifies HbA1c change, a mean and standard deviation, and a 12-week timeframe. It does not report the numerical estimate. Naming an outcome does not supply its result, and without an estimate or uncertainty interval, readers cannot assess its direction, magnitude, or precision.

The recorded enrollment of 10 also makes transparent reporting of individual outcome availability and uncertainty particularly important in general. A small group may yield estimates that are sensitive to individual observations. The registry does not state the primary analysis population, missing-data approach, or detailed statistical plan, so the extent to which these issues were addressed cannot be determined.

Finally, the planning assumptions described in the record use a two-arm equal-allocation framework, while the study itself is recorded as single-group. That difference is a useful reminder that planning illustrations are not the same as the trial’s actual design or its own sample-size justification. The registry does not report a sample-size justification.

These features matter beyond this particular study: statistical interpretation depends not only on the outcome named, but also on the comparison structure, analysis choices, missingness, and the reporting of estimates with uncertainty. Here, several of those details are absent, so the record supports only a limited descriptive account.

15. 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 limitation for interpreting outcomes is that the registry record does not report the numerical HbA1c change, its standard deviation, or a confidence interval. Numerical results for the listed secondary outcomes are also not reported. This prevents assessment of outcome direction, magnitude, and precision.

The study is single-group, with allocation marked NA and no masking. There is no concurrent control condition for separating changes over time from differences attributable to the intervention. Baseline measurements do not provide the same protection against confounding as a randomized comparison. The design therefore limits causal interpretation even if numerical changes were available.

The recorded enrollment is 10. The registry record does not report a sample-size justification, and the planning assumptions described elsewhere do not establish the study’s own design rationale. It also does not report detailed eligibility criteria, intervention doses, adherence, or the schedule and procedures for outcome collection. These gaps limit assessment of how representative the participants were and how consistently the intervention and measurements were implemented.

The analysis details are not reported in the registry record. The intended analysis population, handling of missing values, methods for the continuous endpoint, and any sensitivity analyses are unspecified. The record lists three outcome measures and three analyses but does not describe multiplicity handling. These omissions restrict reproducibility and complicate interpretation of any results that might be available elsewhere.

Safety reporting is limited to summary counts and a serious-event term. Deaths are not reported in the registry record, and event timing, attribution, overlap between categories, and collection methods are not described. With no comparator, these counts cannot provide a comparative safety estimate. Together, the missing outcomes and methodological detail mean the registry record offers an incomplete basis for evaluating this study’s results.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Feasibility of Once/Daily Administered GLP/1 Receptoragonist (Lixisenatide) in Combination With Basal Insulin — NCT02168491.
• Registry record last updated 2017-05-09; status last verified 2017-03.
• Results first posted 2017-04-04.
• 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.

19. Record Summary

This completed Phase 3 study, sponsored by the Medical University of Vienna, is recorded as a single-group, unmasked study with allocation listed as NA. Enrollment is 10. It concerns patients with type 2 diabetes mellitus who were not achieving therapeutic targets with premixed insulin and describes a switch to insulin glargine with lixisenatide.

The primary outcome is mean change in HbA1c from baseline to the end of the study, with standard deviation and a timeframe of 12 weeks. The listed secondary outcomes are change in fasting plasma glucose and change in body weight. Numerical results for these outcomes are not reported in the registry record.

The safety summary lists 9 at risk and 1 affected for serious adverse events, and 9 at risk and 3 affected for other adverse events. Elective ENT surgery is listed as a serious adverse-event term at 11.1%. Deaths are not reported in the registry record. The record does not describe a comparator group, detailed statistical plan, missing-data methods, or sample-size justification.