A Study of Teduglutide in Japanese Children With Short Bowel Syndrome Aged 4 Months or Older: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for A Study of Teduglutide in Japanese Children With Short Bowel Syndrome Aged 4 Months or Older (NCT05027308), a na phase 3 study. The primary outcome is Number of Participants With Treatment-emergent Adverse Events (TEAEs).

2. Clinical Question

The study is listed as phase 3 and completed, with enrollment of 3. Its stated main aims were to check for side effects from teduglutide. The primary endpoint is a participant count, not a comparison of outcomes between treatment groups. The registry record does not report a numerical result for that primary endpoint.

The record also lists secondary outcomes concerning parenteral-support volume and use, reductions in parenteral-support volume, and enteral autonomy. These measures describe aspects of support needs and nutritional autonomy, but their numerical results are not reported in the registry record. The available information therefore describes the questions the study set out to examine, rather than establishing how those outcomes changed during treatment.

3. Trial Design

This was a phase 3, open-label, single-group study of teduglutide in Japanese pediatric patients with short bowel syndrome. The registry lists no masking and an allocation value of NA. Enrollment was 3, and the study status is completed. With no separate control group, the design cannot provide a randomized, like-for-like treatment comparison.

The planned treatment description specifies a daily subcutaneous injection of teduglutide at 0.05 mg/kg, with 0.025 mg/kg for participants with moderate or greater renal impairment. The arm description calls for a 28-week treatment cycle: a 24-week treatment period followed by a 4-week period without treatment. The brief summary says participants may be able to repeat treatment if they meet specific criteria. It also describes follow-up for another 4 weeks after the treatment period and continued checks for side effects until teduglutide becomes commercially available. The maximum treatment duration is described as approximately 51.3 weeks.

The primary safety observation timeframe runs from the first dose until a follow-up visit, 4 weeks after the end of treatment or termination, with the registry giving a range of up to 47.3-51.3 weeks. This is an observation window, not evidence that every participant received treatment for the same duration.

The registry record does not report the reason for the enrollment size, a sample-size justification, or a detailed statistical analysis plan. It also does not report interim analyses. The available design details support a descriptive reading of safety observations in the enrolled group; they do not support treating this study as a controlled estimate of comparative effects.

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: Pediatric participants with a diagnosis of short bowel syndrome (SBS) dependent on parenteral support (PS) were enrolled in the study based on the eligibility criteria to receive teduglutide.

Overall Study

Analysis population for the primary outcome, as defined in the registry: Safety Analysis Set included all participants who received at least 1 dose of study teduglutide.

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: Safety Analysis Set included all participants who received at least 1 dose of study teduglutide.

6. Primary Endpoint

The registry lists the primary outcome as Number of Participants With Treatment-emergent Adverse Events (TEAEs), measured over From first dose of study drug until follow-up visit (4 weeks after end of treatment [EOT]/end of termination [ET] {up to 47.3-51.3 weeks}).

The primary endpoint is the number of participants with treatment-emergent adverse events (TEAEs). It counts participants, rather than the total number of event episodes. The registry defines an adverse event as an untoward medical occurrence in a participant administered a drug and notes that an event need not be causally related to the treatment. A TEAE is an event whose onset occurred, severity worsened, or intensity increased after the participant received the investigational product.

This endpoint is used to describe safety events during exposure and the defined follow-up period. Counting participants with events gives a participant-level view of how many people experienced at least one qualifying event, but it does not by itself describe the number of episodes, their duration, or whether the study drug caused them. Nor does the endpoint alone convey event severity or clinical consequences.

The safety analysis set is defined as participants who received at least one dose of study teduglutide. Although the registry gives the endpoint definition and observation timeframe, it does not report a numerical result for the primary TEAE endpoint. The endpoint should therefore be understood as the study’s stated safety measure, not as a reported estimate of the frequency of TEAEs in this record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

Statistical methods are useful only when they match the study question and the available data. In this study, the main stated aim was safety, and the primary endpoint is a participant count. The design is single-group, with no masking and no separate comparator. The registry record does not report a detailed statistical analysis plan, so the methods below explain general statistical concepts relevant to reading a small safety study; they are not claims about analyses performed by the investigators.

What a participant count represents

A count of participants with TEAEs records how many people experienced at least one event meeting the registry definition. It is different from a count of all event episodes: one participant may experience more than one event, but a participant-level endpoint counts that person once for the endpoint. The record does not report a numerical result for this primary endpoint, so no participant-level TEAE count can be interpreted from it.

A percentage expresses a count relative to a group size. In a small group, a change in the experience of a single participant can noticeably change the percentage. This is why descriptive percentages from a small study should not be treated as stable estimates for a broader patient population. The serious-event terms are listed as percentages, but the registry record does not report confidence intervals for those terms.

What uncertainty means

General statistical explanation: a confidence interval is a range of values compatible with the data under a specified statistical method. It conveys uncertainty around an estimate; it does not guarantee that the true value lies within the interval. In small studies, uncertainty is often substantial, and interval estimates can help make that imprecision visible. The registry record does not report confidence intervals for the listed serious-event terms or a numerical primary TEAE estimate.

Precision and statistical significance are not interchangeable. Precision concerns how narrowly an estimate is determined; statistical significance is assessed using a specified hypothesis-testing framework. The record does not report a primary endpoint analysis or its statistical test, so it does not support a significance claim for that endpoint.

Why a control group matters

General statistical explanation: a control group can provide a reference for what happened under a different condition, helping distinguish changes associated with the study intervention from events or changes that might occur otherwise. Random allocation can make groups more comparable on average, while masking can reduce some influences on outcome assessment. This study is listed as single-group, with allocation NA and no masking. It therefore does not provide a randomized treatment comparison. The registry does not describe a separate control condition.

For safety events, observing an event after treatment starts establishes timing under the TEAE definition, not causation. The registry’s definition states that an adverse event need not have a causal relationship with treatment. Without a control group, the posted figures cannot determine how many listed events would have occurred without teduglutide.

Planning calculations are not observed evidence

General statistical explanation: sample-size planning uses assumptions about the outcome, the effect to detect, and the statistical method to estimate how many participants may be needed for a planned study. A planning calculation is not an observed result and does not establish that a study had a particular probability of detecting an effect after completion. The registry describes sample-size illustrations as planning based on a stated fraction of an observed effect, using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It also notes that these illustrations are not the trial’s own design. Such planning assumptions do not change the fact that this study was single-group. No numerical planning results are reported in the registry record.

Over-dispersion and event data

General statistical explanation: over-dispersion occurs when count data vary more than a chosen statistical model expects. If present, standard errors from an unsuitable model may be too small, giving an overly narrow impression of uncertainty. This study’s primary endpoint is a participant count, and the record does not report a count model or an over-dispersion assessment. There is therefore no basis to say that over-dispersion was tested or that a particular model was used.

For this record, the clearest statistical reading is descriptive: identify the study population and design, distinguish the stated endpoint from the numerical outcomes actually reported, and avoid causal or comparative conclusions that the design and posted results do not support.

10. Exact Estimates for Each Arm

No two-arm comparison is shown for this trial, but each arm's result can still be summarized with exact interval estimates computed from the posted counts. The Clopper-Pearson interval is exact and conservative; the Wilson interval is a common closer-to-nominal alternative; the Bayesian credible interval uses a uniform Beta(1, 1) prior. For small groups the three can differ noticeably.

R code and output

arm    <- c("Teduglutide")
n      <- c(3)
events <- c(3)
for (i in seq_along(n)) {
  ci <- binom.test(events[i], n[i])$conf.int          # Clopper-Pearson exact interval
  cr <- qbeta(c(0.025, 0.975), 1 + events[i], 1 + n[i] - events[i])   # Bayesian, uniform Beta(1,1) prior
  wi <- (function(x, n) { p <- x / n; z <- qnorm(0.975); d <- 1 + z^2 / n
    c((p + z^2 / (2 * n) - z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d, (p + z^2 / (2 * n) + z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d) })(events[i], n[i])
  cat(sprintf("ARM|%s|%d|%d|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f\n", arm[i], n[i], events[i], 100 * events[i] / n[i],
              100 * ci[1], 100 * ci[2], 100 * wi[1], 100 * wi[2], 100 * cr[1], 100 * cr[2]))
}

ARM  Teduglutide  3  3  100.0  29.2  100.0  43.9  100.0  39.8  99.4

11. Multiplicity, Missing Data and Analysis Populations

The registry lists 16 outcome measures, including the primary TEAE endpoint and secondary measures concerning parenteral-support volume, parenteral-support use, reductions in support volume, and enteral autonomy. When a study assesses multiple outcomes, general statistical practice may consider how the number and relationship of tests affect the interpretation of chance findings. The registry record does not report a multiplicity strategy, such as an adjustment procedure or a hierarchy for interpreting outcomes. It also does not report which outcomes, if any, were included in a prespecified testing sequence.

The record lists 0 analyses. Numerical results for the primary TEAE endpoint and the listed secondary outcomes are not reported in the registry record. It is therefore not possible to assess missing outcome data, the handling of incomplete observations, or whether any sensitivity analyses were conducted. The record does not report imputation methods or reasons for missing measurements.

The safety analysis set includes participants who received at least one dose of study teduglutide. This definition identifies the intended population for the safety endpoint, but the registry record does not give a numerical primary TEAE result or provide participant-level analysis details. The recorded enrollment is 3; the registry does not report further information about the number contributing to each secondary outcome.

These absences limit assessment of how outcomes were selected, how missing observations were handled, and how any multiple comparisons were interpreted. They should not be filled in by assumption. The statistical analysis plan, missing-data procedures, and multiplicity handling are not reported in the registry record.

12. 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 serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: device related infection (66.7%), bacteraemia (33.3%), catheter site pruritus (33.3%), device breakage (33.3%), vascular device occlusion (33.3%).

The serious-adverse-event listing gives 3 participants at risk and 3 affected in the teduglutide group. It also names Device related infection at 66.7%, and Bacteraemia, Catheter site pruritus, Device breakage, and Vascular device occlusion at 33.3% each. These are reported serious-event terms and percentages, not estimates of causal risk attributable to teduglutide.

The primary endpoint is a separate measure: the number of participants with treatment-emergent adverse events. Its result is not reported in the registry record. The serious-event listing should not be substituted for that missing primary endpoint result. The registry defines adverse events broadly and notes that an event does not necessarily have a causal relationship with treatment.

Because enrollment was 3 and the study had no control group, the listed figures are descriptive observations from a very small group. They do not establish how often such events would occur in a larger population or whether they would have occurred without treatment. The registry does not report confidence intervals or further event-level details for these listings.

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

This record documents a completed phase 3, open-label, single-group study of teduglutide in Japanese children with short bowel syndrome who depended on parenteral support. It describes the dosing schedule and identifies treatment-emergent adverse events as the primary safety endpoint. It also lists serious-event terms and percentages for the teduglutide group.

The record does not report a numerical result for the primary TEAE endpoint or numerical results for the listed secondary outcomes. It therefore does not establish a reported change in parenteral-support needs, the number achieving enteral autonomy, or a numerical estimate for the primary safety endpoint. The serious-adverse-event figures remain descriptive and do not establish that treatment caused the listed events.

The study’s single-group design does not provide a randomized comparator. Its small enrollment also means that the listed observations cannot be treated as precise estimates for a broader population. These limitations do not erase the information that the registry does report, but they restrict what can be concluded from it.

General statistical interpretation distinguishes an observed event from a causal effect and a descriptive estimate from a comparative result. Here, the record supports description of the study design and the listed safety observations. It does not support claims about comparative benefit, treatment efficacy, or the likelihood of events outside the enrolled group. The registry record does not report the detailed analysis plan, missing-data methods, or a numerical analysis of the primary endpoint.

14. Why This Trial Matters Statistically

This study is statistically notable because its stated focus is safety in a specific pediatric population, while its design is single-group and its enrollment is 3. Safety studies can describe events observed during treatment and follow-up, but a study without a control group cannot directly estimate how those observations differ from an untreated or alternative-treatment condition.

The primary endpoint is defined at the participant level, which is an interpretable way to describe how many people experienced qualifying events. However, the numerical result for that endpoint is not reported in the registry record. The serious-event listing provides some descriptive information, but its small group size and lack of a comparator limit generalization and causal interpretation.

The registry also lists secondary outcomes related to parenteral support and enteral autonomy. Their presence shows that the study considered outcomes beyond safety, but their numerical results are not reported in the registry record. Distinguishing listed outcomes from reported results is important: an outcome’s presence in a protocol or registry entry does not itself establish what happened.

More broadly, the record illustrates why design, endpoint definitions, and result reporting need to be read together. A completed study status does not substitute for outcome estimates, and a safety event following exposure is not automatically caused by the treatment. The available information allows a careful account of the study’s structure and listed safety observations, while leaving important questions about numerical outcomes and analysis methods unanswered.

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 main limitation for interpreting results is that the registry record does not report a numerical result for the primary TEAE endpoint. It lists 16 outcome measures and 0 analyses, but does not give numerical results for the listed secondary outcomes. This prevents assessment of the magnitude or precision of the primary outcome and of changes in parenteral-support measures or enteral autonomy.

Enrollment was 3, and the study was single-group, open-label, and had no masking. There is no randomized control group for comparison. The serious-event figures are therefore descriptive and cannot establish a treatment-related risk or distinguish events occurring during exposure from those that might have occurred otherwise.

The record does not report a sample-size justification, detailed statistical analysis plan, interim analyses, multiplicity strategy, or missing-data methods. It also does not provide confidence intervals or participant-level details for the listed serious-event terms. These omissions limit the ability to evaluate uncertainty, outcome selection, and the handling of incomplete measurements.

The treatment description allows possible repeat treatment under specific criteria, and the maximum duration is described as approximately 51.3 weeks. The registry does not report individual treatment durations or the number of participants who repeated treatment. The listed observation timeframe should not be assumed to represent identical exposure or follow-up for every participant.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: A Study of Teduglutide in Japanese Children With Short Bowel Syndrome Aged 4 Months or Older — NCT05027308.
• Registry record last updated 2024-05-16; status last verified 2024-05.
• Results first posted 2024-05-16.
• 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.

19. Record Summary

NCT05027308 is a completed phase 3, open-label, single-group study of teduglutide in Japanese pediatric patients with short bowel syndrome who depended on parenteral support. The registry lists enrollment of 3 and no masking. The intervention was daily subcutaneous teduglutide, with the described dose adjusted for participants with moderate or greater renal impairment.

The primary endpoint was the number of participants with treatment-emergent adverse events during the stated safety observation period. Its numerical result is not reported in the registry record. Serious-event figures list 3 participants at risk and 3 affected, with five serious-event terms reported at percentages ranging from 33.3% to 66.7%.

Secondary outcomes included measures of parenteral-support volume and use, and enteral autonomy. Their numerical results, the detailed statistical analysis plan, and missing-data methods are not reported in the registry record.