Study of Teduglutide in Japanese Participants With Short Bowel Syndrome: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Study of Teduglutide in Japanese Participants With Short Bowel Syndrome (NCT03663582), a na phase 3 study. The primary outcome is Change From Baseline in Weekly Parenteral Support (PS) Volume at End of Treatment/Early Termination (EOT/ET).

2. Clinical Question

The primary endpoint focuses on the volume of parenteral support participants received each week. It is a continuous measure, reported as a mean with standard deviation, and compares each participant’s baseline value with the value at end of treatment or early termination. The registry gives the endpoint definition and timeframe, but its numerical mean change and standard deviation are not reported in the registry record.

Because this was a single-group study, any recorded change describes participants’ measurements over time during the study; it is not a randomized estimate of the difference between teduglutide and a control treatment. The registry record does not report a control group against which to distinguish treatment-associated change from other explanations, such as natural variation or changes in care. The study enrolled 7 participants, so the scope for drawing precise conclusions from the group-level data would be limited even if the endpoint result were available.

3. Trial Design

This completed phase 3 study evaluated teduglutide in Japanese participants with short bowel syndrome who depended on parenteral nutrition or intravenous support. The study objectives included safety, efficacy, and pharmacokinetics over a 24-week treatment period. The record reports a single-group model and no masking. Allocation is listed as NA, and there is no separate control arm described.

Participants in the teduglutide group were assigned the regimen described in the record: 0.05 mg/kg by subcutaneous injection once daily for 24 weeks. The injection could be given into one of the four abdominal quadrants or into either thigh or arm. The named study interventions also include a syringe, needle, and vial adapter for device.

The study enrolled 7 participants. Its primary endpoint was change from baseline in weekly parenteral support volume at end of treatment or early termination. The endpoint timeframe is stated as baseline and end of treatment or early termination, up to Week 28. The ITT population included participants deemed eligible for teduglutide treatment at the baseline visit.

The registry record lists 29 outcome measures but 0 analyses. It does not report a numerical result for the primary endpoint, a confidence interval, a p-value, or an inferential analysis. It also does not report the protocol’s sample-size justification, any interim analyses, or a detailed statistical analysis plan. Those omissions mean the design and endpoint can be described, but the planned inferential framework and the observed primary endpoint estimate cannot be reconstructed from this record.

With no randomized comparison, the study’s structure supports description of the enrolled group and its recorded outcomes, rather than a like-for-like comparison between treatment groups. The small enrollment and the absence of posted primary endpoint results should remain central when interpreting what the registry record can establish.

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 7 participants were enrolled and received the treatment. Out of them, 6 participants completed the study.

Overall Study

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: ITT population included all participants who were deemed eligible for teduglutide treatment at the baseline visit.

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 population included all participants in the intent-to-treat (ITT) population who received at least 1 dose of study treatment.

6. Primary Endpoint

The registry lists the primary outcome as Change From Baseline in Weekly Parenteral Support (PS) Volume at End of Treatment/Early Termination (EOT/ET), measured over Baseline, EOT/ET (up to Week 28).

The primary endpoint is the change from baseline in weekly parenteral support volume at end of treatment or early termination. The unit is liters per week, and the endpoint is defined as a mean with standard deviation. The assessment timeframe includes baseline and end of treatment or early termination, up to Week 28. The ITT population is defined as participants deemed eligible for teduglutide treatment at the baseline visit.

Weekly parenteral support volume is a measure of the amount of support received over a week. Tracking change from baseline describes how that volume differed at the later assessment from each participant’s starting measurement. This type of endpoint is relevant to the study’s stated focus on participants dependent on parenteral support: it quantifies a feature of their support requirements rather than relying only on a broad judgment of change.

As a general statistical point, a mean summarizes the group’s average change, while the standard deviation describes variation among participant-level measurements around that mean. Both are useful for understanding the group result, but neither replaces the individual data or establishes why a change occurred. In this record, the endpoint definition is present, but the numerical mean change and standard deviation are not reported in the registry record. Consequently, the direction, size, and variability of the primary endpoint result cannot be described from the posted information.

The endpoint is also not a treatment-versus-control contrast. In a single-group study, change over time is a within-group description; without a comparator, it does not by itself estimate what would have happened over the same period without teduglutide.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What does a mean change describe?

The primary endpoint is a continuous measure: change from baseline in weekly parenteral support volume, expressed in liters per week. A mean change summarizes the average participant-level change in the analysis population. It is a compact description of the group, not a guarantee that every participant changed by that amount. The standard deviation describes the spread of participant-level values around the mean and helps show whether observations were relatively clustered or varied widely.

For this endpoint, the intended comparison is each participant’s baseline measurement with the measurement at end of treatment or early termination. As a general statistical explanation, analyzing change scores can make the comparison to baseline explicit. The resulting group mean still needs to be interpreted alongside its variability, the number of participants contributing data, and the analysis population. The registry record gives the endpoint definition and population description, but it does not report the primary endpoint’s mean change or standard deviation.

What does a single-group design allow?

The study is listed as single-group, with no separate comparator and no masking. The endpoint can therefore be understood as a within-group change over time, rather than as a difference between a treatment group and a control group. As a general statistical point, a concurrent control group can help distinguish a treatment-associated difference from changes that might also occur over time for other reasons. Without one, changes observed during follow-up cannot by themselves establish what would have happened in the absence of the study treatment.

The registry lists allocation as NA. It does not report random assignment to different groups. Accordingly, there is no randomized treatment contrast to interpret. The study’s phase designation and stated objectives do not supply that missing comparison or a numerical endpoint result.

How should uncertainty be assessed?

General statistical explanation: an estimate is more informative when its uncertainty is also reported. A confidence interval gives a range of values compatible with the observed data under a specified statistical method. A standard error may also describe the sampling uncertainty of an estimate, while the standard deviation describes variability among observations. Those quantities answer different questions.

The registry record reports neither a primary endpoint confidence interval nor a p-value or other inferential analysis. The primary endpoint’s standard deviation is part of the stated measure, but its numerical value is not reported in the registry record. As a result, neither the magnitude of the group’s average change nor its precision can be assessed from the posted primary result. No statistical significance claim can be made from an analysis that is not reported.

What can sample-size planning illustrations mean?

General statistical explanation: sample-size planning estimates how many participants a future study might need under specified assumptions about the effect to detect, the significance level, allocation, and statistical model. Such calculations are planning illustrations, not observed results, and they do not establish the probability that a completed study produced a true finding. A calculation based on an observed effect can be especially uncertain when the estimate comes from a small group.

The registry record does not report its own sample-size justification or detailed statistical plan. It describes planning assumptions for an illustration based on the observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. Those are assumptions for a planning illustration, not evidence that this single-group study had two arms or used that design for its primary analysis. No numerical planning estimates are stated in the record information for interpretation. Such hypothetical planning quantities should not be confused with an observed sample size beyond the 7 participants enrolled.

Why does over-dispersion matter in general?

General statistical explanation: over-dispersion occurs when observed variability is greater than a statistical model assumes. If a model understates variability, its uncertainty estimates can be too small, making results appear more precise than the data warrant. The relevance of over-dispersion depends on the outcome and model; the primary endpoint in this study is a continuous measure summarized by a mean and standard deviation. The registry record does not report a model-based analysis or a diagnostic assessment of over-dispersion. It would therefore be inappropriate to claim that over-dispersion was present, absent, or addressed.

The registry lists 29 outcome measures but 0 analyses. This distinction matters: an outcome measure describes what was assessed, whereas an analysis specifies how the resulting data were modeled or compared. Without posted analysis details, the statistical handling of secondary outcomes, covariates, repeated measurements, or any departures from planned methods is not reported in the registry record.

10. Confidence Intervals for Each Arm

No two-arm comparison is shown for this trial, but each arm's mean can be given with a t-based 95% confidence interval computed from the posted mean, spread and number of participants.

R code and output

arm <- c("Teduglutide")
n   <- c(7)
m   <- c(-2.92)
sp  <- c(3.463)
sd  <- sp      # posted standard deviations
for (i in seq_along(n)) {
  se <- sd[i] / sqrt(n[i]); h <- qt(0.975, n[i] - 1) * se
  cat(sprintf("CARM|%s|%d|%.3f|%.3f|%.3f|%.3f\n", arm[i], n[i], m[i], sd[i], m[i] - h, m[i] + h))
}

CARM  Teduglutide  7  -2.920  3.463  -6.123  0.283

11. Multiplicity, Missing Data and Analysis Populations

Multiplicity concerns the interpretation of results when several outcomes, time points, or comparisons are examined. As a general statistical principle, making many statistical tests can increase the chance of at least one apparently notable result arising by chance unless the analysis plan addresses that issue. The registry lists 29 outcome measures, but 0 analyses. It does not report how multiplicity was handled, whether any outcomes were designated as secondary or exploratory for inferential testing, or whether any adjustment procedure was planned. The registry record does identify a primary endpoint, but it does not provide an analysis plan that would clarify the role of the other measures.

Missing-data methods are also not reported in the registry record. The primary endpoint allows an end-of-treatment or early-termination assessment, and its timeframe is baseline to end of treatment or early termination, up to Week 28. The record does not state how participants without an endpoint measurement were handled, whether measurements after treatment discontinuation were included, or what assumptions were used for any missing values. It does not report the amount or pattern of missing primary endpoint data.

The ITT population is defined as all participants deemed eligible for teduglutide treatment at the baseline visit. That definition identifies the intended population for the endpoint, but the record does not give the number contributing primary endpoint data or detail any exclusions from the analysis. Enrollment was 7; the analyzed endpoint denominator is not reported in the registry record.

These gaps prevent assessment of whether missingness or multiple testing affected the reported estimates. Since no primary endpoint estimate or analyses are posted, no sensitivity analysis or multiplicity-adjusted inference can be evaluated from 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 5% 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 (14.3%), medical device site infection (14.3%), spinal compression fracture (14.3%), epilepsy (14.3%).

For the teduglutide group, the registry lists 7 participants at risk for serious adverse events and 3 affected. For other adverse events, it lists 7 at risk and 7 affected. The deaths entry is 0. These are participant counts in the posted safety record; they are not the same as event counts, and the categories should not be added together to create a total because an individual may contribute to more than one category.

The four serious adverse event terms listed most prominently are device related infection, medical device site infection, spinal compression fracture, and epilepsy. Each term is reported as 14.3%. These percentages are registry entries for the terms, not a measure of treatment effect or evidence about cause. The record does not report additional detail about timing, seriousness criteria, recurrence, duration, or whether participants had more than one listed event.

Safety counts in a group of 7 are descriptive. They can identify what the record reports, but they do not establish whether an event was caused by teduglutide or a device, nor do they precisely characterize how often events might occur in a broader population. The registry record does not provide a comparator group for safety-rate comparisons.

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

The record describes a completed phase 3, single-group study of teduglutide in Japanese participants with short bowel syndrome dependent on parenteral support. The regimen was 0.05 mg/kg by subcutaneous injection once daily for 24 weeks. The primary endpoint was change from baseline in weekly parenteral support volume at end of treatment or early termination. Those details establish the study question and the outcome it was designed to assess.

The record does not report the numerical primary endpoint result. It therefore does not show the mean change, its standard deviation, or an interval expressing uncertainty. Without those results, the direction and size of any change cannot be stated. The registry also lists 0 analyses, so no inferential estimate or test result is available to interpret.

Even if a within-group change were reported, a single-group design would not establish the difference between teduglutide and a control treatment. Change over time can reflect multiple influences, and the record does not include a randomized comparator to support a causal contrast. The enrollment of 7 participants further means that the group’s descriptive findings would need cautious interpretation and could not alone characterize the range of experiences in a wider population.

The safety figures report affected participants and several serious-event terms, but they do not establish causation. The registry record does not provide a comparative safety analysis or enough event-level detail to draw conclusions about timing and clinical context.

Accordingly, this record does not establish that teduglutide improves or fails to improve the primary endpoint, nor does it establish a treatment-related safety profile. It documents the study design, planned endpoint, and some safety counts, while leaving the principal numerical efficacy result and analysis details unreported. The appropriate interpretation is limited to those recorded details, without filling gaps with assumptions.

14. Why This Trial Matters Statistically

This study is statistically notable because it combines a clinically focused continuous endpoint with a single-group design and a small enrollment. Weekly parenteral support volume is measured in liters per week, and change from baseline offers a direct description of how the endpoint was assessed over time. The endpoint’s mean and standard deviation would be useful for understanding both average change and participant variability, but their numerical results are not reported in the registry record.

The design shapes what an analysis could answer. A single group can describe outcomes among participants receiving the study regimen, but it cannot provide a randomized comparison with an alternative or control. This is not merely a matter of statistical technique: without a comparator, the study structure does not isolate a between-treatment difference. The registry record lists allocation as NA and no masking, consistent with its single-group description.

The enrollment was 7. In a group of this size, individual observations can have substantial influence on a mean, and uncertainty can be difficult to characterize precisely. This is a general statistical consideration, not a claim about the unreported endpoint result. It reinforces why the underlying numerical estimate, variability, analysis population, and missing-data handling matter when interpreting a small study.

The registry lists 29 outcome measures but 0 analyses. That contrast underscores the difference between recording an outcome and reporting a statistical result. Since the primary endpoint estimate and inferential details are not reported, the record can support a description of study intent and structure, but not a quantitative assessment of the endpoint. This makes clear reporting of effect estimates and their uncertainty especially important for readers assessing evidence from small, uncontrolled studies.

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 most direct limitation for interpretation is that the registry record does not report a numerical result for the primary endpoint. Although the measure, unit, timeframe, and ITT population are defined, the mean change and standard deviation are absent. The endpoint’s direction, magnitude, and variability therefore cannot be assessed.

The study is single-group and has no separate comparator. Its design does not provide a randomized estimate of the difference between teduglutide and another treatment or control. The registry lists allocation as NA and masking as none. These features should be considered when interpreting any within-group changes, independent of whether such changes are numerically reported.

Enrollment was 7. The registry does not report how many participants contributed data to the primary endpoint analysis, the amount of missing endpoint data, or the handling of missing observations. The ITT population is defined, but a definition alone does not establish the final analysis denominator or reveal whether all eligible participants had endpoint measurements.

The record lists 29 outcome measures and 0 analyses. It does not report the statistical analysis plan, multiplicity procedures, interim analyses, or the rationale for the planned sample size. The endpoint result also lacks a reported confidence interval or p-value. Consequently, precision and inferential evidence cannot be evaluated from the registry record.

The safety entries are limited: they report counts, deaths, and selected serious-event terms, but do not provide further event-level context or a comparator. These limitations are specific to the information recorded and do not establish what may have been included in other study documents.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Study of Teduglutide in Japanese Participants With Short Bowel Syndrome — NCT03663582.
• Registry record last updated 2020-08-04; status last verified 2020-07.
• Results first posted 2020-08-04.
• 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

The registry record describes a completed phase 3, single-group study of teduglutide in Japanese participants with short bowel syndrome dependent on parenteral support. The study enrolled 7 participants. The regimen was teduglutide 0.05 mg/kg by subcutaneous injection once daily for 24 weeks.

The primary endpoint was change from baseline in weekly parenteral support volume at end of treatment or early termination, up to Week 28. It was defined as a mean with standard deviation and assessed in the ITT population, described as participants deemed eligible for teduglutide treatment at baseline. The numerical primary endpoint result is not reported in the registry record.

The registry lists 29 outcome measures and 0 analyses. It reports 3 of 7 participants with serious adverse events, 7 of 7 with other adverse events, and 0 deaths. Four serious adverse event terms are each reported at 14.3%. A comparator group, inferential endpoint result, and detailed statistical analysis plan are not reported in the registry record.