Clinical Trial of Efficacy and Safety of Kolofort in Functional Dyspepsia Patients: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Clinical Trial of Efficacy and Safety of Kolofort in Functional Dyspepsia Patients (NCT03119766), a randomized phase 4 study. The primary outcome is Changes in Severity of Functional Dyspepsia Symptoms.

2. Clinical Question

3. Trial Design

The registry record describes a Phase 4, randomized, parallel-group clinical trial comparing Kolofort with placebo. Its official title identifies it as multicenter, double-blind, and placebo-controlled; the record’s masking classification is quadruple. The record does not specify which participant, clinician, or study roles were included in that masking classification, or how masking was maintained.

Enrollment is recorded as 370. The listed study start date is 2017-06-28, and the primary completion date is 2020-01-11. The study status is completed. Materia Medica Holding is identified as the sponsor. These registry entries describe the trial’s recorded design and administration, but do not establish how many participants contributed data to each endpoint analysis.

Both groups received tablets for oral use on the same schedule: 2 tablets twice daily, held in the mouth until completely dissolved and taken without a meal. The intervention descriptions differ by whether the tablets were Kolofort or placebo. The record does not describe additional treatment, adherence procedures, or permitted co-interventions.

The primary outcome concerns change in functional dyspepsia symptom severity, assessed using GIS at baseline and after 4 and 8 weeks of treatment. Secondary outcomes include symptom severity, the Nepean Dyspepsia Index, SF-36 quality of life, early study termination, and therapeutic and side-effect indicators on the CGI-EI scale. The record lists six outcome measures and 14 analyses. It does not report the allocation sequence method, concealment procedures, or a detailed analysis plan.

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 370 patients were enrolled in the study and signed informed consent. After undergoing screening procedures, 61 patients were excluded by the investigators because they did not meet the inclusion criteria, or they had non-inclusion criteria. 309 patients were randomized into two groups - 151 into Kolofort group and 158 into Placebo group.

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.

Population: The study will enroll the subjects of either gender aged 18-45 years old with verified diagnosis of "functional dyspepsia" according to Rome-IV criteria, intensity of dyspeptic symptoms ≥ 6 according to GIS and negative H. pylori test.

6. Primary Endpoint

The registry lists the primary outcome as Changes in Severity of Functional Dyspepsia Symptoms, measured over On baseline, after 4 and 8 weeks of the treatment.

The primary endpoint is change in the severity of functional dyspepsia symptoms measured with the Gastrointestinal Symptom Score (GIS). The recorded assessment schedule includes baseline and assessments after 4 and 8 weeks of treatment; the primary outcome description focuses on the week-8 score from the start of study therapy.

GIS combines ratings across 10 symptom subscales. Each symptom is rated from 0 to 4 points: 0 represents absence of the symptom and 4 represents the most severe rating. The summed score ranges from 0 to 40. On this scoring description, a lower score corresponds to less symptom severity. The record does not report how changes were calculated for analysis, such as whether the reported change was a difference from baseline or another summary.

In general, a symptom score can make patient-reported symptom burden measurable in a consistent way, and repeated assessment can describe how that burden changes during follow-up. A score is still a summary: it combines symptom ratings and does not, by itself, show which symptoms changed. The registry record does not report the week-8 group means, standard deviations, treatment contrast, confidence interval, or p-value, so the magnitude and precision of the comparative endpoint cannot be assessed from these details.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The trial is recorded as randomized, parallel-group, and quadruple-masked. In general, random allocation is intended to make treatment assignment independent of participants’ measured and unmeasured characteristics, while masking can reduce the influence of treatment expectations on care, reporting, or assessment. These design features do not guarantee that groups will be identical or that bias is absent. The registry record does not describe the randomization procedure, allocation concealment, or which roles were masked.

What does a mean and standard deviation describe?

The primary outcome is classified as a continuous measure summarized by a mean and standard deviation. In general, a mean describes the arithmetic average of observed scores, while a standard deviation describes how spread out individual scores are around that average. Both are useful for describing a group, but neither alone expresses the uncertainty in a comparison between groups. The registry record does not report the GIS means or standard deviations for the treatment groups.

A treatment comparison requires more than knowing that the outcome is a mean. It needs a defined contrast and an estimate based on the observed data. For a continuous outcome, the analysis might compare follow-up scores or changes from baseline, but the registry record does not say which approach was used. It does not report an adjusted or unadjusted comparison, or describe any covariates used in an analysis.

How should uncertainty be understood?

In general, a confidence interval describes a range of values compatible with the data and the assumptions of the analysis. Its width conveys precision: a wider interval indicates less precision than a narrower interval. A p-value addresses compatibility between data and a specified null hypothesis under the analysis assumptions; it does not measure the size or clinical importance of an effect. Neither a confidence interval nor a p-value is reported for the primary GIS comparison in the registry record. It is therefore not possible to assess statistical uncertainty or infer a significance result from the outcome description alone.

What does the assessment schedule imply?

GIS was assessed at baseline and after 4 and 8 weeks of treatment, while the primary outcome description focuses on week 8. Repeated assessments can describe the course of symptoms over time. In general, analyzing repeated measurements requires a method that accounts for the relationship between measurements from the same participant. The registry record does not report whether such a method was used, whether each time point was analyzed separately, or how the primary comparison was selected. It also does not provide results for the week-4 assessment.

The registry note says that the primary outcome has several categories or time points and was not reduced to one comparison. This means the posted information does not establish a single reported primary-outcome contrast for statistical interpretation. The record does not explain the analysis choice or give estimates for the listed time points.

What can the sample-size illustration say?

In general, a sample-size calculation is a planning exercise that estimates the number of participants needed to detect a specified effect under stated assumptions. The recorded illustration assumptions include a two-sided significance level of 0.05, equal allocation between the two arms, and a normal approximation; the target effect is described as a fraction of the observed effect. This illustration is not the trial’s own design justification. The record does not report the protocol’s sample-size justification, and the values for any planning illustrations are not reported in the registry record. Such an illustration is not observed power and cannot establish whether the trial detected an effect.

For continuous outcomes, the amount of variability matters because more spread in scores can make a group contrast less precise. In general, a normal approximation is a modeling assumption for calculating a planning quantity; it is not proof that the observed score distribution follows a normal distribution. The registry record does not describe diagnostic checks or alternative modeling choices.

How do multiple outcomes affect interpretation?

The registry record lists six outcome measures and 14 analyses, alongside several secondary outcomes. In general, examining multiple outcomes or comparisons creates a multiplicity issue: the chance of obtaining at least one apparently notable result can rise when many tests are considered. Statistical methods can address multiplicity, but the choice depends on the planned family of comparisons and the analysis strategy. A multiplicity adjustment or prespecified testing hierarchy is not reported in the registry record. Without that information and the underlying estimates, the number of analyses alone does not establish which results should be treated as confirmatory.

Overall, the record supplies design and measurement descriptions but not the primary outcome analysis needed to interpret the treatment contrast. The unreported analysis population, missing-data approach, model, and multiplicity strategy also limit assessment of how the results were generated.

10. Multiplicity, Missing Data and Analysis Populations

The registry record lists six outcome measures and 14 analyses. The secondary outcomes include symptom improvement, the Nepean Dyspepsia Index, SF-36 quality of life, early study termination, and therapeutic and side-effect indicators on the CGI-EI scale. The primary GIS outcome is scheduled across baseline and follow-up assessments. Multiple outcomes, time points, and analyses raise questions about which comparisons were designated as primary and whether inferential testing accounted for multiplicity.

In general, multiplicity can increase the chance that at least one result appears statistically notable when many comparisons are made. A prespecified testing hierarchy or adjustment method can help control that issue. The registry record does not report a multiplicity strategy, a detailed statistical analysis plan, or which outcome comparisons were confirmatory. It also does not report p-values for the primary outcome.

The analysis population is not reported in the registry record. It does not specify whether the primary analysis included all randomized participants, participants who took treatment, or a different subset. The record also does not describe missing outcome data, withdrawals, imputation, or sensitivity analyses for missingness. These omissions matter because the amount and handling of missing data can affect the estimated group comparison.

The enrollment figure and the adverse-event risk counts do not establish the number of participants included in the primary outcome analysis. The registry record does not provide a participant flow or endpoint-specific denominators for the GIS assessments. Consequently, the completeness and comparability of the primary outcome data cannot be evaluated from the recorded details.

11. Safety Results

The registry reports serious adverse events as affected participants divided by participants at risk.

Deaths

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 0% reporting threshold.

Most frequent non-serious adverse event terms

The adverse-event record lists 151 participants at risk for serious adverse events in the Kolofort group and 158 in the placebo group. Serious adverse events affected 0 participants in each group, and deaths were 0 in each group. The record lists 13 participants with other adverse events in the Kolofort group and 12 in the placebo group, with 151 and 158 participants at risk, respectively.

These are counts, not estimates of a treatment effect. The record does not provide event descriptions for the other adverse events, severity, timing, attribution, or information on whether a participant could contribute more than one event. No serious-event terms are listed. The denominators shown for safety should not be assumed to be the denominators for the primary GIS outcome; endpoint-specific analysis counts are not reported.

In general, observing no serious events in a study group does not prove that the risk is absent. Interpretation depends on the amount of exposure, the event definitions, follow-up, and the precision of a comparative estimate. The registry record does not report comparative safety estimates or confidence intervals. These counts therefore describe what was recorded, but do not establish equivalence or rule out uncommon harms.

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

The registry record establishes that a randomized, quadruple-masked trial compared Kolofort with placebo in patients with functional dyspepsia and specified a symptom-severity endpoint based on GIS. It describes how the scale is scored and identifies week 8 as the primary outcome assessment. Those details clarify the research question and what the endpoint was intended to measure.

The record does not report the group-level primary outcome results or the comparative estimate. It therefore does not show whether GIS scores changed, how large any difference between groups was, or how precise such a difference might be. It also does not report a primary-outcome confidence interval or p-value. No conclusion about the comparative symptom result can be drawn from the scale description or trial design alone.

The reported safety counts describe zero serious adverse events and zero deaths in both groups, alongside counts for other adverse events. They do not establish that risks were equal, that no adverse effects occurred, or that uncommon harms are absent. The registry record lacks event-level details and comparative safety estimates.

In general, a trial result must be interpreted in light of its estimate, uncertainty, analysis population, missing-data handling, and planned comparisons. The relevant details are not reported for the primary GIS analysis. The enrollment figure of 370 is a design or registry characteristic, not a substitute for the number analyzed or for an outcome result.

Thus, the record can be used to understand the study question, design labels, endpoint scale, and recorded adverse-event counts. It cannot support a conclusion about whether the groups differed on the primary symptom outcome or about the magnitude and precision of that difference.

13. Why This Trial Matters Statistically

This trial is statistically informative as an example of why a clear outcome definition and a reported comparison are both needed. The record defines GIS as a composite symptom score built from 10 symptom subscales, each rated from 0 to 4, and gives a total range from 0 to 40. That makes the measurement framework understandable, but does not reveal the observed group scores or the difference between groups.

The design also illustrates the distinction between trial structure and trial results. Randomization, masking, and a placebo comparator describe how the study was organized. They do not supply an estimate of the primary outcome or show how much uncertainty surrounds it. For clinical researchers and statisticians, this distinction is central: a well-described endpoint and design are necessary context, while interpretation still depends on complete outcome reporting.

The record lists multiple outcomes and analyses, with symptom assessments at baseline and after 4 and 8 weeks. That makes prespecification and multiplicity handling relevant statistical issues. The registry record does not report a testing hierarchy or adjustment strategy, so readers cannot determine how the analyses were prioritized or how multiplicity was addressed.

The safety record offers a limited additional perspective through counts of serious adverse events, deaths, and other adverse events. It does not report the event detail or uncertainty needed for broader comparisons. Together, these features show why transparent reporting should include endpoint-specific denominators, analysis methods, estimates, and uncertainty—not only a study design and outcome definition.

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.

The main limitation for statistical interpretation is that comparative results for the primary GIS outcome are not reported in the registry record. Arm-level scores, changes, the treatment contrast, uncertainty intervals, and p-values are absent. Although the endpoint description names week 8 and the assessment schedule includes baseline and week 4, the record does not give the corresponding outcome data.

The record does not specify how the primary endpoint was analyzed, including whether the comparison used follow-up scores or change from baseline, whether any adjustment was made, or how repeated assessments were handled. It notes that the primary outcome has several categories or time points and was not reduced to one comparison, but does not explain the analysis decision. A statistical analysis plan and a multiplicity strategy are not reported.

The analysis population, participant flow, endpoint-specific denominators, missing-data patterns, and methods for handling missing outcomes are not reported. Enrollment is recorded as 370, but that figure alone does not show how many participants contributed to each analysis. The safety denominators are given, but they cannot be used to infer the GIS analysis population.

Further details about randomization and masking procedures are not reported, including allocation concealment and which roles were masked. The registry record also does not provide detailed eligibility criteria or information about adherence and other treatments. For safety, event descriptions and comparative estimates are absent; the record lists counts only. These limitations concern the information available in the registry record and do not establish that the unreported procedures or analyses were not performed.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Clinical Trial of Efficacy and Safety of Kolofort in Functional Dyspepsia Patients — NCT03119766.
• Registry record last updated 2022-06-07; status last verified 2019-06.
• Results first posted 2022-06-07.
• 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

The registry record describes a completed Phase 4 randomized, parallel-group, quadruple-masked trial of Kolofort versus placebo in functional dyspepsia. Enrollment is recorded as 370; the study start date is 2017-06-28, and primary completion is dated 2020-01-11. Materia Medica Holding is listed as sponsor.

The primary outcome is functional dyspepsia symptom severity measured with GIS, with the primary description focused on week 8. The scale combines 10 symptom ratings, each scored from 0 to 4, into a total score from 0 to 40. The record does not report the primary comparative results or analysis details.

Safety counts list no serious adverse events and no deaths in either group; other adverse events are reported for 13 Kolofort participants and 12 placebo participants. The record also lists secondary outcomes and 14 analyses, but does not report a multiplicity strategy, analysis population, or missing-data methods.