Effects Of Exenatide On Liver Biochemistry, Liver Histology And Lipid Metabolism In Patients With Fatty Liver Disease: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Effects Of Exenatide On Liver Biochemistry, Liver Histology And Lipid Metabolism In Patients With Fatty Liver Disease (NCT00529204), a na phase 2 study. The primary outcome is Reduction in Serum ALT From Baseline to 24 Weeks of Exenatide Therapy.

2. Clinical Question

3. Trial Design

This is a Phase 2, single-group study of exenatide, sponsored by the University of California, Davis. Allocation is recorded as NA and masking as none. The registry lists enrollment as 1 and the study status as terminated. The start is recorded as 2007-10, and primary completion as 2009-07.

The listed treatment schedule begins with exenatide 5 µg BID s.c. daily for 28 days, followed by 10 µg BID s.c. daily from day 29 to week 24. The primary endpoint is reduction in serum ALT from baseline to 24 weeks. Secondary outcomes concern changes in liver histology components—steatosis, inflammation and fibrosis—between baseline and week 24, and safety in patients with NAFLD and type 2 diabetes.

Because the record describes a single group and no allocation to a control, the design does not support a randomized comparison between exenatide and another treatment or control condition. Any change from baseline would be a within-group observation. Such a change cannot, on its own, distinguish an intervention effect from other explanations for change over time. The reason for termination, recruitment history, eligibility criteria, and further protocol details 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.

Overall Study

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 Reduction in Serum ALT From Baseline to 24 Weeks of Exenatide Therapy, measured over 24 weeks.

The primary endpoint is titled “Reduction in Serum ALT From Baseline to 24 Weeks of Exenatide Therapy.” It identifies serum ALT as the measure, a baseline-to-follow-up change as the intended comparison, and 24 weeks as the timeframe. The registered unit is IU, and the endpoint is recorded as a number. The record does not specify a further endpoint definition or a population for the measure.

ALT is a liver enzyme measured in blood. In general, a biochemical endpoint can offer a measurable way to track a change in liver-related laboratory values over a defined period. A baseline measurement can serve as a reference for describing subsequent change within the same group. That description is not equivalent to a controlled estimate of treatment effect: without a comparator, changes may also reflect factors other than the intervention.

The registry record does not report the baseline value, the value at 24 weeks, the amount of change, or an uncertainty estimate. It therefore does not establish the direction or precision of any observed change. It also does not report whether all participants with a baseline measurement contributed a follow-up measurement.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The design determines which statistical questions the data can answer. This record describes a single group and no masking, with allocation recorded as NA. In general, a single-group study can describe measurements before and after an intervention. A concurrent control group is needed to make a direct comparison with what happened under another condition. Without that group, a pre-to-post change does not by itself separate an intervention-related change from other changes over time.

What would a baseline-to-follow-up change describe?

For the primary endpoint, the intended contrast is serum ALT at baseline versus serum ALT at 24 weeks. A change score expresses how the follow-up measurement differs from the baseline measurement for the observed participant or participants. In general, the interpretation depends on the endpoint definition and on having the relevant measurements at both time points. The registry record does not give the endpoint calculation details, individual measurements, or a reported change.

The endpoint is recorded as a number, with unit IU. That description alone does not identify whether the registry intended a particular summary across participants, how missing measurements would be handled, or whether a transformation or adjustment would be used. Those details are not reported in the registry record. With enrollment recorded as 1, a group-level estimate of variability cannot be meaningfully characterized from multiple independent participants in this study record. No uncertainty estimate is posted.

What does a confidence interval add?

In general, a confidence interval gives a range of values compatible with a statistical estimate under the assumptions of the method used. Its width reflects precision: a wider interval indicates less precision than a narrower interval. An interval does not repair limitations in design, and it does not turn a within-group change into a randomized comparison. No confidence interval for the primary outcome is reported in the registry record, so the precision of any outcome estimate cannot be assessed from the posted results.

What can a significance test establish?

In general, a statistical test evaluates how compatible the observed data are with a specified null hypothesis, subject to the test's assumptions. A p-value is not the probability that a treatment works, and it does not describe the size or practical importance of a change. Here, no analysis is listed and no p-value is reported. There is therefore no posted hypothesis-test result to interpret. The registry does not report the statistical analysis plan or the intended test.

How should planning calculations be read?

Any sample-size illustration based on an observed effect is a planning exercise, not evidence that a trial had a given probability of detecting an effect. The stated planning assumptions describe a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the target effect expressed as a fraction of the observed effect. They also distinguish participants per arm for ordinary outcomes from a total event count for time-to-event outcomes. These are illustration assumptions, not a reported design justification for this single-group study. The registry record does not report a sample-size justification or a numerical observed effect on which to base a planning interpretation.

Over-dispersion is a general concern when count data vary more than a model assumes. If present, a model that assumes less variability may give uncertainty estimates that are too small. The primary endpoint is recorded as a number, but the record does not provide an analysis identifying it as a count outcome or report any model for it. No claim about over-dispersion in this study can therefore be made.

What is available for independent interpretation?

The record lists 3 outcome measures and 0 analyses. The primary outcome is named, and two secondary outcome descriptions address liver histology and safety. The record does not report estimates, participant-level data, analysis methods, handling of missing data, or the analysis populations. General statistical principles can clarify what those omissions prevent: without estimates and measures of uncertainty, readers cannot judge either the observed direction of change or its precision; without a stated analysis, readers cannot assess the assumptions behind an inferential result.

10. Multiplicity, Missing Data and Analysis Populations

The registry lists 3 outcome measures and 0 analyses. It names the primary endpoint as reduction in serum ALT from baseline to 24 weeks and describes secondary outcomes concerning liver histology components and safety. The registry record does not report a multiplicity plan, such as whether or how inference across outcomes would be adjusted or prioritized. Without that information and without posted analyses, there is no basis for judging how multiple outcome questions were handled.

Missing-data methods are not reported in the registry record. The record does not state whether measurements were missing, how missing values were treated, or whether all enrolled participants contributed data at each endpoint. Enrollment is listed as 1, but that figure alone does not establish completion of follow-up or availability of outcome measurements.

The analysis populations are also not reported in the registry record. There is no description of an intention-to-treat population, a per-protocol population, or another analysis set. Those terms refer generally to rules defining which participants are included in an analysis; the rules can affect how results are interpreted. No such rule can be inferred from the study's status or enrollment entry.

Because results and analysis details are absent, readers cannot assess whether outcome availability, exclusions, or multiplicity affected the findings. These are reporting gaps, not evidence that a particular method was or was not used outside the registry record.

11. 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 0% reporting threshold.

The adverse-event record lists the Exenatide group with 1 at risk and 0 affected for serious adverse events. It also lists 1 at risk and 0 affected for other adverse events. These are the figures recorded for the group; they do not establish that no adverse events occurred beyond what was reported to the registry.

The death count is not reported in the registry record. No serious adverse-event terms are listed. In general, a zero affected count in a small group is a description of the posted count, not evidence that an event is impossible or that risk has been ruled out. With enrollment recorded as 1, these figures provide very limited information about how frequently adverse events might occur across a broader population.

The registry names safety in patients with NAFLD and type 2 diabetes as a secondary outcome, but it does not post further safety results or analysis. The figures should therefore be read as the limited adverse-event counts in the record, not as a comprehensive estimate of safety.

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

The record documents a Phase 2, single-group study of exenatide with a planned serum ALT endpoint and secondary outcomes involving liver histology and safety. It reports enrollment of 1 and a terminated status. It does not report results for the primary or secondary outcomes, and it lists 0 analyses.

What the record does show is the study's stated question and treatment schedule, together with the fact that no comparator group is listed. It does not show whether serum ALT decreased, increased, or remained unchanged. Nor does it give a magnitude of change or an uncertainty estimate. The title and endpoint wording describe what the study sought to assess, not what it found.

Because the study is single group, a change from baseline, if reported, would not by itself provide a controlled estimate of the treatment effect. Other explanations for change over time could not be separated using a randomized contrast that is absent from the record. The absence of posted results also means that the histology and safety questions cannot be answered from this entry.

The record does not explain why the study was terminated. That status alone does not indicate a reason, an outcome, or a safety finding. The registry background's discussion of preliminary evidence is not evidence generated by this trial. The study record should therefore be understood as documenting an intended investigation, not as establishing a clinical benefit, lack of benefit, or a definitive safety conclusion.

13. Why This Trial Matters Statistically

This study illustrates how the design and completeness of reporting shape what can be learned from a clinical trial record. A baseline-to-follow-up endpoint can define a measurable question, but a single-group design does not provide the same causal contrast as a randomized comparison. Readers need to distinguish a within-group change from a treatment-versus-control effect.

The registry makes the intended primary measure and timeframe clear: serum ALT reduction from baseline to 24 weeks. Yet it does not report the outcome result, an uncertainty measure, or an analysis. This contrast between a defined endpoint and absent results is statistically important. An endpoint name alone cannot show the direction, size, or precision of a finding.

Enrollment is recorded as 1, which also limits the conclusions that can be drawn about variability and generalizability. The safety counts are correspondingly limited: zero affected is the recorded count in a group with one at risk, not a broad estimate of event frequency. The record's terminated status adds context, but its reason is not reported.

Finally, the entry shows why analysis plans, missing-data rules, and multiplicity handling matter. Without those details, readers cannot evaluate how outcomes were analyzed or how results across outcome measures were interpreted. The statistical lesson is not a conclusion about exenatide; it is the importance of matching claims to design, reported results, and precision.

14. Limitations

• The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
• Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.

Several limitations specific to this record restrict interpretation. The design is single group, with allocation recorded as NA and no comparator listed. It therefore does not describe a randomized control contrast. The registry enrollment is 1, limiting the amount of participant-level information available for estimating group variability or describing outcomes across a population.

The study is marked terminated, but the reason is not reported in the registry record. The start and primary completion dates are listed, but the record does not state whether the participant completed the planned treatment and follow-up. It does not report eligibility criteria in detail, baseline characteristics, or the measurement schedule beyond the stated primary timeframe and histology assessment.

Most importantly, the registry does not report results for the primary endpoint or the secondary outcomes. It lists 0 analyses, and provides no analysis method, uncertainty estimate, missing-data approach, or analysis-population definition. The registry also does not report a multiplicity plan or a sample-size justification. These gaps prevent assessment of the precision, robustness, and completeness of outcome reporting.

The adverse-event counts are limited to the recorded figures, and a death count is not reported in the registry record. No reason for termination or fuller safety account is available. These limitations mean the entry can describe the trial's design and stated aims, but it cannot support a detailed assessment of efficacy or safety outcomes.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Effects Of Exenatide On Liver Biochemistry, Liver Histology And Lipid Metabolism In Patients With Fatty Liver Disease — NCT00529204.
• Registry record last updated 2017-06-20; status last verified 2017-05.
• Results first posted 2014-12-22.
• Data retrieved from ClinicalTrials.gov on 2026-11-09.
• Group sizes, means, event counts and sponsor-reported p-values are reproduced as posted. Effect estimates, confidence intervals and p-values not attributed to the sponsor are independently calculated from the posted summary statistics.

18. Record Summary

NCT00529204 is a Phase 2, single-group study sponsored by the University of California, Davis, investigating exenatide in patients with fatty liver disease. The regimen was 5 µg BID s.c. daily for 28 days, followed by 10 µg BID s.c. daily from day 29 to week 24. The primary endpoint was reduction in serum ALT from baseline to 24 weeks. Secondary outcomes concerned liver histology and safety.

The registry records enrollment of 1, no masking, and terminated status. It lists 3 outcome measures and 0 analyses. The primary and secondary outcome results, reason for termination, analysis methods, and missing-data approach are not reported in the registry record. The posted information therefore describes the study question and design but does not provide outcome evidence for interpreting changes in ALT, histology, or safety.