Efficacy of Pantoprazole in Patients Older Than 12 Years Who Have Symptoms of Non-Erosive Reflux Disease (NERD) or Erosive Gastroesophageal Reflux Disease (eGERD): Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Efficacy of Pantoprazole in Patients Older Than 12 Years Who Have Symptoms of Non-Erosive Reflux Disease (NERD) or Erosive Gastroesophageal Reflux Disease (eGERD) (NCT00561730), a registered phase not stated study. The primary outcome is Patient's Assessment of General Well-being During the Last 24 Hours (Diaries; ReQuest™ in Practice).

2. Clinical Question

The study’s stated purpose was to assess symptoms during a short course of pantoprazole in patients with non-erosive or erosive reflux disease in ordinary clinical practice. Its secondary outcomes include physician assessments of heartburn, acid eructation, and painful swallowing, as well as final-visit assessments of efficacy and tolerability. Those registered outcomes describe what the study intended to assess; they do not, by themselves, establish that numerical findings were reported.

The central statistical question is thus limited: what can be learned from the registered outcome definition and available record, and what remains uncertain because outcome estimates and a comparator are not reported? The registry record identifies the measurement framework, but it does not provide the endpoint values needed to estimate symptom levels or changes over time.

3. Trial Design

The registry identifies a completed study sponsored by Nycomed, with a recorded start date of 2007-10 and enrollment of 1995. The condition is gastroesophageal reflux disease. The title refers to patients older than 12 years with non-erosive reflux disease or erosive gastroesophageal reflux disease, while the brief study description says the aim was to evaluate pantoprazole 40 mg and 20 mg over 7 days in clinical practice.

The intervention is recorded as the drug pantoprazole. The arm description is “All patients enrolled,” rather than a description of separate dose groups or a control group. Allocation is not stated. The registry record therefore does not establish that participants were randomized, how the two doses were assigned, or whether there was any untreated or active comparator. The study should not be interpreted as a randomized head-to-head comparison based on the arm information available.

Patients were to complete a short, self-assessed reflux questionnaire, ReQuest™ in Practice. The primary outcome is a diary assessment of general well-being during the last 24 hours, measured over 7 days. Physician assessments and final-visit efficacy and tolerability assessments are listed as secondary outcomes. The record lists 11 outcome measures overall.

The primary-outcome population is described as all patients included and treated, intention to treat, with missing values not imputed and results considered “as observed.” This is the registry’s stated population description; operational details about eligibility after enrollment, treatment adherence, and any exclusions are not reported in the registry record. The record also lists zero analyses, so it does not present the statistical-analysis results needed to compare outcome values or quantify uncertainty.

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

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: All patients included and treated, intention to treat, missing values not imputed ('as observed'). Number of valid cases: * Day 0 = 1769 * Day 1 = 1760 * Day 2 = 1761 * Day 3 = 1751 * Day 4 = 1749 * Day 5 = 1742 * Day 6 = 1747

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 Patient's Assessment of General Well-being During the Last 24 Hours (Diaries; ReQuest™ in Practice), measured over 7 days.

The primary endpoint is the patient’s assessment of general well-being during the last 24 hours, recorded in diaries with ReQuest™ in Practice. It is a continuous measure reported in units on a scale from 0=Excellent to 10=Extremely bad. The registered timeframe is 7 days. The outcome description specifies a mean and standard deviation, which would respectively describe the average reported score and the spread of individual scores.

A patient-reported measure can capture the symptom experience directly, rather than relying only on a clinician’s assessment. Repeated daily diary entries can also describe how reported well-being varies across the treatment period. These are general reasons such outcomes may be used; they do not establish that the measure changed in this study.

The registry lists valid cases for each day, but does not give the corresponding mean or standard deviation values. It also does not report a baseline-to-follow-up contrast, a model-based estimate, a confidence interval, or a p-value for the primary endpoint. Consequently, the direction, magnitude, and precision of the recorded well-being scores cannot be assessed from the posted results. The endpoint definition tells readers what was measured, not what the measurements showed.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The primary outcome is continuous: it is recorded on a scale and summarized in the registry as a mean with a standard deviation. General explanation: a mean represents the average observed score, while a standard deviation describes how dispersed individual scores are around that average. The standard deviation is not a confidence interval and does not, on its own, express the precision of the estimated mean. A confidence interval, when reported, would communicate a range of values compatible with the data and the analysis assumptions. No confidence interval for the primary endpoint is reported in the registry record.

The outcome is recorded repeatedly in diaries over 7 days. General explanation: repeated observations from one person tend to be related, so a statistical analysis of daily values may need to account for within-person dependence. Treating every daily entry as if it came from a different person can understate uncertainty. The registry record does not identify a repeated-measures model or another method for handling that dependence. It also does not state whether the primary inference was based on a particular day, an average across days, or a change from an initial assessment.

What do the scale values represent?

The scale is defined from 0=Excellent to 10=Extremely bad, with higher values therefore corresponding to worse ratings according to the endpoint description. That direction helps readers interpret a reported mean if one is available. It does not make the scale an objective clinical measure, nor does it establish what size of difference patients would consider important. A threshold for clinical importance is not reported in the registry record.

For a continuous outcome, an estimated difference or change would usually be interpreted alongside its uncertainty and the scale’s meaning. General explanation: a narrow interval indicates greater precision than a wide interval, while an interval that includes values on both sides of no difference may leave the direction uncertain. This study’s record does not report such an estimate or interval, so those principles cannot be applied to a numerical treatment effect. No treatment-effect value should be inferred from the outcome definition or the valid-case counts.

How should missing diary entries be understood?

The registry describes the primary-outcome population as all patients included and treated, with an intention-to-treat label, and explicitly says missing values were not imputed, using an “as observed” approach. General explanation: an as-observed summary uses the measurements that are available rather than filling in absent values. This can be straightforward to describe, but the resulting average represents the observed entries; it may not represent all enrolled participants if the presence of an entry is related to the outcome.

The registry gives valid-case counts for day 0 through day 6. Those counts show that the number of valid observations is not identical on each day, but they do not explain why entries are absent. General explanation: missing-data bias depends not only on how many observations are missing but also on the process that made them missing. The record does not report reasons for missing diaries, comparisons of participants with and without entries, imputation, or sensitivity analyses. Therefore, the robustness of an as-observed result cannot be evaluated.

What does the planning illustration mean?

General explanation: a sample-size calculation asks how many participants would be needed under specified assumptions to detect a chosen effect with a chosen level of statistical power. The planning illustration described for this study uses a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; its target effect is expressed as a stated fraction of an observed effect. Such assumptions make the illustration a hypothetical planning exercise, not evidence about how this trial was designed or analyzed.

Because the registry lists a single arm comprising all enrolled patients and does not state allocation, a two-arm planning setup should not be mistaken for the actual trial design. General explanation: if a planning illustration is based on an observed effect, changing the effect size assumed for planning changes the required number of participants. A calculation based on an observed effect is not “observed power,” and it cannot establish that the study was adequately powered for its registered endpoint. The registry record does not report the trial’s own sample-size justification, target power, or design assumptions.

For continuous outcomes, assumptions about variability and the effect to detect matter to a sample-size calculation. General explanation: larger variability makes it harder to distinguish a mean difference from random variation, all else equal. Since the registry record does not provide the primary-outcome means or standard deviations, readers cannot independently judge the observed effect or its variability from the posted results. A planning calculation cannot substitute for a reported estimate and its uncertainty.

Why are multiple outcomes relevant?

The registry lists 11 outcome measures, including the primary diary measure and secondary physician assessments and final-visit assessments. General explanation: when many outcomes or repeated comparisons are examined, the chance of at least one apparently notable result arising by chance can increase. Statistical procedures can address multiplicity, but they need to be described to interpret findings across outcomes. The registry record does not report whether any adjustment, prespecified hierarchy, or other multiplicity strategy was used. With zero analyses listed, it also provides no analysis results on which to assess the issue.

10. Multiplicity, Missing Data and Analysis Populations

The registry lists 11 outcome measures. The primary measure is the patient’s diary assessment of general well-being, while the secondary outcomes include physician assessments of heartburn, acid eructation, and painful swallowing, plus assessments of efficacy and tolerability at the final visit. General explanation: analyzing several outcomes creates opportunities for apparently notable findings by chance, so a prespecified hierarchy or multiplicity adjustment can help clarify which findings are confirmatory and how to interpret the rest. Whether such a strategy was used is not reported in the registry record.

The record lists zero analyses. It does not provide a statistical-analysis plan, interim-analysis information, or a description of any adjustment across outcomes or diary days. The existence or absence of these procedures cannot be inferred from the outcome list alone.

For the primary outcome, the registry specifies all patients included and treated, labels the population intention to treat, and says missing values were not imputed, with results considered “as observed.” It reports valid-case counts for each diary day, but does not explain the missing entries or describe sensitivity analyses. General explanation: when observations are analyzed as available, the results can be influenced if the chance of a missing entry is related to the patient’s experience. The record does not establish whether missingness was random or whether the available diary entries represent all enrolled patients.

The registry’s population label and missing-data statement are useful, but they do not provide a complete account of the analysis population. Eligibility after enrollment, handling of participants with no diary data, and any deviations from the stated approach are not reported in the registry record. These gaps limit assessment of both potential bias and the reproducibility of the analysis.

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 0.01% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: nervousness (0.1%), diarrhoea (0.1%).

The safety record lists 1995 participants at risk for serious adverse events and one participant affected. For other adverse events, it lists 1995 at risk and 10 affected. The serious-event terms shown are nervousness and diarrhoea, each reported as 0.1%. These are descriptive registry figures; they are not comparative estimates of risk.

Without a control group and a reported allocation method, the counts cannot establish whether event frequency differed from what would have occurred under another treatment or no treatment. The record does not give additional detail about event timing, severity beyond the serious-event classification, relatedness to treatment, or the method used to collect events. Those details are not reported in the registry record.

The death field has no numerical value, so deaths are not reported in the registry record. A blank or unreported value should not be interpreted as a confirmed count of zero. The listed serious adverse event and other adverse events should also be kept distinct: they are separate categories in the record and are not a measure of the primary symptom outcome.

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

The record establishes that the study was completed, enrolled 1995 patients, and aimed to evaluate pantoprazole 20 mg and 40 mg for symptoms of non-erosive or erosive reflux disease in clinical practice. It specifies a patient-reported diary measure of general well-being on a defined scale over 7 days. It also lists secondary physician and final-visit assessments and provides descriptive adverse-event counts.

It does not report the numerical primary endpoint results. There is no reported mean, standard deviation, treatment-related change, confidence interval, or p-value for the diary outcome. As a result, the record does not establish the average symptom rating, the amount of any change, or the precision of an estimated effect. The listed valid-case counts alone cannot answer those questions.

The available arm description is not a comparison between pantoprazole and a control treatment: it describes all enrolled patients as belonging to the pantoprazole arm, and allocation is not stated. Any before-and-after pattern, if reported elsewhere, would not by itself show that treatment caused the pattern. A causal comparison requires a design and analysis that address alternative explanations, and those details are not reported.

The record also does not permit assessment of whether a measured change would be important to patients. The endpoint scale is defined, but a threshold for meaningful change is not reported. Nor does the registry describe a multiplicity plan, a sample-size justification, or missing-data sensitivity analyses. These are limitations on what can be concluded from the recorded information, not proof that no additional work was conducted.

The appropriate reading is therefore descriptive and cautious: the record documents a short clinical-practice study and its planned measurement framework, alongside limited safety counts, but does not provide the primary outcome estimates needed to judge the observed symptom data. It cannot support a claim about benefit or lack of benefit, nor a comparative safety conclusion.

13. Why This Trial Matters Statistically

This trial illustrates why a clearly named endpoint is not the same as a reportable result. The registry identifies a patient-centered measure, explains its scale direction, and specifies diary collection over 7 days. Yet without the endpoint values and their uncertainty, readers cannot determine the average ratings, the amount of change, or how precisely any effect was estimated. Statistical interpretation depends on estimates, not only on the outcome definition.

The record also highlights the distinction between a clinical-practice evaluation and a controlled comparison. The study aim refers to two pantoprazole doses, but the registered arm description includes all enrolled patients and allocation is not stated. That gap matters because an observed change over time is not automatically a treatment effect. A comparator and a clearly described allocation process help distinguish treatment-related change from other explanations.

Repeated symptom diaries can capture patients’ experiences over time, but they raise analysis questions about within-person correlation and incomplete records. The record states that missing values were not imputed and gives valid-case counts by day, yet does not describe why entries were missing or how daily observations were analyzed. These details are important for judging whether an average is representative and whether its uncertainty is correctly expressed.

Finally, the registry lists multiple outcomes but no analyses. That makes it impossible to assess how the investigators handled multiple comparisons or which findings, if any, were considered primary in the final analysis. The study is statistically instructive because its registration specifies what was intended to be measured while leaving key result and analysis information unreported.

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 is the absence of numerical primary-outcome results in the registry record. Although the endpoint is defined and the valid-case counts are listed, no mean or standard deviation values are reported. There is no estimate of change, confidence interval, or significance test. Consequently, neither the magnitude nor the precision of the primary outcome can be evaluated.

The registry describes one pantoprazole arm containing all enrolled patients and does not state allocation. It does not describe a control group or explain how the two doses named in the study aim were handled. Thus the record does not support a dose comparison or a controlled estimate of treatment effect. The title and aim should not be taken as evidence of a randomized design.

The record does not report the statistical-analysis plan, the rationale for the sample size, interim analyses, repeated-measures methods, or any multiplicity procedure. It lists 11 outcomes and zero analyses, leaving no reported estimates for the primary or secondary measures. Whether additional analyses were performed outside the registry is not reported in the registry record.

Missing primary-outcome observations were not imputed, according to the registry description, and the results were to be treated as observed. Valid-case counts vary across diary days, but reasons for missingness and sensitivity analyses are not reported. The extent to which missing observations could affect an estimate therefore cannot be assessed.

Safety information is also limited. The record gives affected counts and two serious-event terms, but does not report event timing, treatment relatedness, or deaths. The available figures are not comparative and should not be used to establish relative safety.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Efficacy of Pantoprazole in Patients Older Than 12 Years Who Have Symptoms of Non-Erosive Reflux Disease (NERD) or Erosive Gastroesophageal Reflux Disease (eGERD) — NCT00561730.
• Registry record last updated 2012-05-08; status last verified 2012-05.
• Results first posted 2010-09-16.
• 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, Nycomed-sponsored clinical-practice study with enrollment of 1995 and a recorded start date of 2007-10. Its aim was to evaluate pantoprazole 20 mg and 40 mg for symptoms of non-erosive or erosive gastroesophageal reflux disease. The listed arm is pantoprazole and is described as including all enrolled patients; allocation is not stated.

The primary outcome is patient-reported general well-being during the last 24 hours, recorded in diaries on a scale from 0=Excellent to 10=Extremely bad over 7 days. The record specifies an as-observed approach without imputation, but does not report numerical primary-outcome results or analyses. It lists 11 outcome measures and zero analyses.

Safety entries include one participant affected by a serious adverse event and 10 affected by other adverse events, each among 1995 at risk. Nervousness and diarrhoea are listed as serious-event terms at 0.1% each. Deaths are not reported in the registry record. The available information describes the study and its measures but does not permit an assessment of the size or precision of a treatment effect.