This page provides an independent statistical analysis and educational interpretation of publicly reported results. ClinicalTrials.gov provides the official trial registry record. Numerical trial results on this page are restricted to the results posted in the ClinicalTrials.gov record; calculations added by Clinical Biostats are labelled as such.
1. Trial at a Glance
This page covers the registry results for Open Label Safety Study of Enteric-Coated Spheroid Suspension in Infants Aged Less Than 12 Months With Presumed Gastroesophageal Reflux Disease (GERD) (NCT00868296), a non-randomized phase 3 study. The primary outcome is Number of Patients With Laboratory Test Values of Potential Clinical Importance During Treatment Period.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00868296 |
| Phase | Phase 3 |
| Condition | Gastroesophageal Reflux |
| Allocation | Non-Randomized |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 58 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Wyeth is now a wholly owned subsidiary of Pfizer |
| Results posted | Yes |
2. Clinical Question
Population
Infants aged less than 12 months with presumed gastroesophageal reflux disease were eligible if they had successfully completed the 3001B3-331 study (NCT00362609) or the 3001B3-333 study (NCT00259012). The primary endpoint population is described as patients who received ≥1 dose of pantoprazole and had laboratory test results. The number tested varied by laboratory test.
Intervention
The intervention was pantoprazole sodium enteric-coated spheroid suspension. The study evaluated low-dose and high-dose pantoprazole groups. The stated purpose was to provide additional information on safety and tolerability after multiple doses.
Comparator
There was no randomized control group. The record identifies a non-randomized, open-label study and lists low-dose and high-dose groups. These dose groups are not a like-for-like control comparison, so contrasts between them should be interpreted descriptively rather than as randomized treatment effects.
Primary question
During the 6-week treatment period, how many patients had laboratory test values meeting pre-defined criteria for potential clinical importance? The endpoint counts patients meeting thresholds across specified laboratory tests. The registry record does not report the primary endpoint counts, so the frequency of these findings cannot be determined from the posted information.
3. Trial Design
The registry identifies this as a phase 3, multicenter, open-label safety study of 2 doses of pantoprazole sodium enteric-coated spheroid suspension in infants aged less than 12 months with presumed GERD. It was completed, with enrollment reported as 58. The start is listed as 2006-03 and primary completion as 2008-03. Allocation was non-randomized, masking was absent, and the study model is classified as single-group.
The registry also lists low-dose and high-dose pantoprazole groups. Because assignment was not randomized and there was no blinded control group, the group labels do not create a like-for-like comparison. Differences in who entered or remained in each group, or in who had a particular laboratory test, could affect a descriptive contrast. The record does not report how dose assignment was determined.
Eligibility was restricted to patients who successfully completed one of the named prior studies. This describes the study population as selected from patients with prior study participation, rather than all infants with presumed GERD. Details about the prior studies’ treatment histories or how that eligibility requirement influenced the population are not reported in the registry record.
The stated objective was additional safety and tolerability information after multiple doses of pantoprazole. The primary outcome was the number of patients with laboratory values of potential clinical importance during treatment, assessed over 6 weeks. The registry lists 2 outcome measures and 9 analyses, but further details of the statistical plan, including analysis populations and any planned comparisons, 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: Patients were eligible for enrollment into the study after completion of either study 3001B3-331 (NCT00362609) or study 3001B3-333 (NCT00259012).
Overall Study
| Stage | Low Dose Pantoprazole | High Dose Pantoprazole |
|---|---|---|
| STARTED | 12 | 46 |
| COMPLETED | 12 | 43 |
| NOT COMPLETED | 0 | 3 |
Reasons for not completing this period:
| Reason | Low Dose Pantoprazole | High Dose Pantoprazole |
|---|---|---|
| Adverse Event | 0 | 2 |
| Withdrawal by Subject | 0 | 1 |
Analysis population for the primary outcome, as defined in the registry: Patients who received ≥1 dose of pantoprazole and had laboratory test results. The number of patients (n) tested varied by test, variations shown (low dose, high dose): Carbon dioxide (n=7,26), CPK (n=12,42), Gastrin (n=9,25), Neutrophils (n=12,44), Platelet count (n=12,41), Urine protein albumin (n=10,38), Urine leukocyte esterase (n=10,38).
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.
| Characteristic | Unit | Low Dose Pantoprazole | High Dose Pantoprazole | Total |
|---|---|---|---|---|
| Participants analysed | 12 | 46 | 58 | |
| Age Continuous | months | 7.34 (SD 3.06) | 5.55 (SD 3.44) | 5.92 (SD 3.42) |
| Sex: Female, Male: Female | Participants | 5 | 21 | 26 |
| Sex: Female, Male: Male | Participants | 7 | 25 | 32 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Patients With Laboratory Test Values of Potential Clinical Importance During Treatment Period, measured over 6 weeks.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | patients |
| Endpoint classification | Binary Endpoint |
The primary endpoint counts patients with laboratory test values meeting pre-defined criteria for potential clinical importance during the 6-week treatment period. The registry describes a patient-level count, not a continuous summary of how far a laboratory value was from a reference value. The tests and thresholds include potassium ≤ 3.0 mEq/L or ≥ 6.2 mEq/L; carbon dioxide < 12 mEq/L or > 35 mEq/L; total bilirubin > 1.5xULN; CPK > 3xULN; gastrin ≥ 600 pg/mL; neutrophils < 10% or > 80%; platelet count < 100 x10 to the third power/ul or > 600 x10 to the third power/ul; and specified urine protein albumin and urine leukocyte esterase findings.
Pre-defined thresholds make it possible to flag laboratory findings according to criteria set for the endpoint. This is useful for describing the frequency of values considered potentially important under those criteria. It does not, by itself, establish that a finding caused symptoms, was caused by treatment, or represented a clinically consequential event for an individual patient. Those conclusions require context beyond a threshold count.
The endpoint population includes patients who received ≥1 dose and had laboratory results, with the number tested varying by test. The registry gives tested counts for some measures, but the primary endpoint counts meeting criteria are not reported in the registry record. Therefore, the endpoint definition is available, while its observed frequency cannot be assessed from this record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record provides the study design and endpoint definition, but it does not report the primary endpoint counts. It is therefore not possible to describe how many patients met the laboratory criteria, compare those counts across dose groups, or assess the precision of such estimates. A defined outcome is not the same as a reported outcome result.
For the laboratory population, the number tested varied across measures. The record lists carbon dioxide results for low dose n=7 and high dose n=26; CPK for n=12 and n=42; gastrin for n=9 and n=25; neutrophils for n=12 and n=44; and platelet count for n=12 and n=41. These are test-specific populations, not a common denominator across all laboratory outcomes. The tested counts for other listed tests are not fully reported in the registry record.
The adverse-event summary lists 1 serious-event-affected patient in the low-dose group and 2 in the high-dose group. It also lists other adverse-event-affected counts of 8 and 30, respectively. The corresponding numbers at risk are not reported in the registry record. Without denominators, these counts do not provide event rates, and their difference should not be interpreted as evidence that one dose has greater or lesser risk. The registry also lists serious-event terms and percentages: gastroenteritis, 8.3%; otitis media, 8.3%; respiratory disorder, 2.2%; and respiratory failure, 2.2%. The record does not provide enough accompanying denominator information to independently assess those percentages.
These data are descriptive. Non-randomized allocation, open-label conduct, differing test-specific populations, and missing denominators limit comparisons between dose groups. There is no randomized control group against which to distinguish treatment-associated findings from findings that could occur in the study population for other reasons. The posted information does not establish causation, comparative safety, or the absence of laboratory or adverse events.
The registry does not report confidence intervals, p-values, adjusted analyses, or a statistical plan for the primary endpoint. The magnitude and precision of any treatment-group contrast therefore cannot be evaluated from the record. The absence of a reported result is not evidence that no events occurred; it means the primary endpoint result is not reported in the registry record.
9. Statistical Methods Explained
Statistical interpretation depends on the outcome type, the population included in each analysis, and the design that generated the data. The primary endpoint is a count of patients whose laboratory results met specified criteria. Because it counts patients with an event rather than measuring a continuous laboratory value, it is a binary outcome at the patient level: each patient either meets the endpoint definition for a test or does not, provided a result is available. The registry record does not give the observed endpoint counts.
What does a patient count describe?
A count conveys how many patients had a finding, but the count alone does not describe its frequency in the group. Interpreting frequency requires the relevant denominator: the number of patients assessed for that outcome. A denominator must match the outcome and analysis population. This is especially important because laboratory testing populations varied by test. Counts for carbon dioxide, CPK, gastrin, neutrophils, and platelet count are not interchangeable denominators for other laboratory outcomes.
In general, a proportion summarizes an event count relative to the number assessed. A confidence interval describes uncertainty around an estimated quantity, such as an event proportion, and helps distinguish a precise estimate from one with substantial uncertainty. The registry record does not report the primary endpoint counts or confidence intervals, so neither event proportions nor their precision can be assessed from the record. No new rates or intervals can be inferred from the reported tested counts alone.
Why does non-randomized allocation matter?
Random allocation is used in comparative trials to make treatment groups more comparable, on average, with respect to factors known and unknown at enrollment. In a non-randomized study, group membership can be associated with patient characteristics or other circumstances. A difference in observed counts between dose groups may therefore reflect differences in the groups, in addition to any dose-related effect. Open-label conduct also means masking was absent; the registry does not describe additional procedures to limit the influence of that design.
The study is classified as single-group, while the registry also lists low-dose and high-dose groups. The record says allocation was non-randomized and explicitly cautions that the arms are not a like-for-like control comparison. The listed dose groups should thus be treated as descriptive categories, not as evidence from a randomized head-to-head trial. The basis for dose assignment is not reported in the registry record.
Why do varying test populations matter?
Laboratory outcomes can have different numbers assessed because not every patient necessarily contributes a result to every test. The registry specifies a population of patients who received ≥1 dose and had test results, and gives differing tested counts for several laboratory measures. This means each test’s result pertains to its own available population. Comparing counts without aligning the populations and denominators can be misleading.
In general, missing measurements can affect both the size and composition of the population analyzed. If availability of a test result is related to patient status or treatment experience, the observed patients may not represent everyone who entered the study. The registry record does not explain why the test populations differed, how missing laboratory results were handled, or whether any imputation or sensitivity analysis was used.
What can a threshold-based endpoint establish?
Pre-defined thresholds turn laboratory measurements into flags for values considered potentially important under the study’s criteria. This can support a safety description, but it discards some information about the magnitude and pattern of the underlying values. A patient just across a threshold and a patient far beyond it both meet the criterion. The endpoint also does not itself establish symptoms, clinical consequences, or a causal relationship with treatment.
The general rationale for pre-defining thresholds is to make the classification rule explicit rather than selecting a cutoff after seeing the results. The record supplies criteria for multiple laboratory tests, but does not report details of how a patient with more than one qualifying test was counted in any combined summary. It also does not describe whether analyses were adjusted for multiple testing.
How should planning calculations be read?
Sample-size calculations are planning illustrations, not results observed in the trial. The registry’s stated illustration assumptions include a two-sided significance level of 0.05, equal allocation between arms, a normal approximation, and effects defined as fractions of an observed effect. Such assumptions describe a hypothetical planning exercise; they do not establish the trial’s actual power or show that the trial was designed using those calculations. The registry does not report a sample-size justification, and no planning values or underlying observed effect are reported in the registry record.
More generally, a p-value addresses how compatible data are with a specified statistical model and null hypothesis; it does not measure the probability that a treatment works or the size of an effect. A confidence interval conveys uncertainty about an estimate, while neither measure resolves confounding in a non-randomized comparison. The registry record does not report p-values or confidence intervals for the primary endpoint. Statistical significance, if reported for another analysis, would not by itself establish clinical importance or causation.
10. Multiplicity, Missing Data and Analysis Populations
The primary outcome involves multiple laboratory tests, each with pre-defined criteria, and the registry lists 2 outcome measures and 9 analyses. When multiple outcomes or analyses are examined, the chance of finding an apparently unusual result can increase. Statistical approaches to multiplicity can control or describe that issue, but the registry record does not report whether any adjustment was planned or applied. It also does not describe a hierarchy of outcomes or identify which analyses were confirmatory.
Missing-data handling is not reported in the registry record. The reported tested counts vary by laboratory measure: carbon dioxide has low-dose n=7 and high-dose n=26; CPK n=12 and n=42; gastrin n=9 and n=25; neutrophils n=12 and n=44; and platelet count n=12 and n=41. These differences show that the test-specific analysis populations were not the same size. The record does not explain why measurements were unavailable, whether patients without a result were excluded, or how missing results affected the endpoint summaries.
The primary endpoint population is described as patients who received ≥1 dose and had laboratory results. The registry does not report further analysis-population definitions, such as whether all eligible patients were included in each safety analysis or how discontinuations were treated. It also does not report interim analyses or a statistical analysis plan. Without these details and the endpoint counts, the risk of selection effects and the robustness of the findings cannot be evaluated from the record.
11. What the Result Does, and Does Not, Mean
The study addresses a safety and tolerability question in a selected population of infants with presumed GERD who had completed one of the named prior studies. It describes use of low-dose and high-dose pantoprazole and defines laboratory thresholds intended to flag values of potential clinical importance during treatment. Those design and endpoint details clarify what the study sought to observe, but they do not supply the missing primary result.
The registry lists adverse-event-affected counts in both dose groups and percentages for several serious-event terms. These are descriptive entries. The numbers at risk are not reported in the registry record, and the group assignment was non-randomized. The counts therefore cannot establish comparative event rates or show that any observed event was caused by pantoprazole. They also cannot establish that an event did not occur when no count is displayed for a particular outcome.
Because the primary laboratory endpoint counts are not reported, the registry does not permit a conclusion about the frequency of patients meeting those thresholds. Varying test populations further mean that each laboratory finding would need to be interpreted with its own denominator. The posted information also does not report confidence intervals or other measures of precision for the primary outcome.
These limitations do not make the endpoint definition meaningless; the pre-defined thresholds describe how potential laboratory findings were classified. They do limit what can be concluded from the available record. The study’s non-randomized, open-label design and lack of a like-for-like control comparison mean that the record cannot establish a causal effect or comparative safety between doses. No medical conclusion about an individual infant follows from these group-level entries.
12. Why This Trial Matters Statistically
This study illustrates why safety endpoints need to be interpreted alongside their denominators and analysis populations. The primary measure is a count of patients crossing specified laboratory thresholds, but the record does not report those counts. For several tests, the number assessed also varies by dose group. A reader can identify the classification criteria and some test-specific populations, but cannot calculate or verify the primary endpoint frequency from the registry record.
The design adds another important statistical constraint. Although low-dose and high-dose groups are listed, allocation was non-randomized, the study was open-label, and the registry cautions that the arms are not a like-for-like control comparison. Descriptive contrasts between such groups do not have the same interpretation as randomized comparisons. Group differences may reflect how patients came to be in each dose group, as well as treatment or chance.
The record also demonstrates the distinction between safety signals and causal evidence. Listed counts and event terms can help characterize what was recorded, but absent denominators and the primary endpoint results, they do not establish comparative risk or show that treatment caused an event. Pre-defined laboratory thresholds offer a clear operational rule, yet a threshold flag is not equivalent to a clinical outcome or a causal attribution.
For biostatisticians and clinical researchers, the key lesson is that reporting the endpoint definition is not enough for interpretation. Event counts, denominators, missing-data handling, analysis-population definitions, and the statistical plan are also needed to judge frequency and precision. Several of those details are not reported in the registry record. The design can be described, but the available results support only limited descriptive interpretation.
13. 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 principal limitation for interpreting the primary outcome is that its observed counts are not reported in the registry record. Without those counts and the corresponding denominators, the frequency of laboratory values meeting criteria and the precision of any estimate cannot be assessed. Test-specific populations vary, and the record does not explain why results were unavailable for some patients or how missing measurements were handled.
The study was non-randomized and open-label, with no randomized control group. Although low-dose and high-dose groups are listed, the registry cautions that these are not a like-for-like control comparison. The reason for dose assignment and the characteristics of each group are not reported in the registry record. Consequently, descriptive differences between groups cannot be attributed to dose alone.
The eligible population was restricted to patients who successfully completed one of two named prior studies. The registry does not report how this selection may affect applicability to infants outside that population. It also does not give the statistical analysis plan, missing-data procedures, multiplicity approach, interim-analysis information, or a sample-size justification.
For adverse events, affected counts are listed without the numbers at risk. The record also lists serious-event terms with percentages but does not provide enough accompanying denominator information to independently evaluate those percentages. These gaps limit rate comparisons and precision assessments. The registry’s completion status and enrollment do not resolve these limitations in the reported outcome information.
14. Related Tutorials
15. Related Statistical Calculators
16. Sources
- ClinicalTrials.gov: Open Label Safety Study of Enteric-Coated Spheroid Suspension in Infants Aged Less Than 12 Months With Presumed Gastroesophageal Reflux Disease (GERD) — NCT00868296.
- Registry record last updated 2010-05-04; status last verified 2010-04.
- Results first posted 2010-01-07.
- 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.
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17. Record Summary
This completed phase 3 study evaluated low-dose and high-dose pantoprazole sodium enteric-coated spheroid suspension in infants aged less than 12 months with presumed GERD. It was multicenter, open-label, non-randomized, and classified as single-group. Enrollment is reported as 58; the study start is 2006-03 and primary completion is 2008-03.
Eligibility was limited to patients who successfully completed NCT00362609 or NCT00259012. The stated objective was additional safety and tolerability information after multiple doses. The primary endpoint was the number of patients with laboratory test values meeting pre-defined criteria for potential clinical importance during 6 weeks.
The registry provides test-specific counts for some laboratory populations and adverse-event summaries, but the primary endpoint counts, numbers at risk for adverse events, and several analysis details are not reported in the registry record. Non-randomized allocation and differing test populations constrain comparisons between dose groups.