Lanreotide in the Treatment of Small Bowel Motility Disorders: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Lanreotide in the Treatment of Small Bowel Motility Disorders (NCT03012594), a na phase 2 study. The primary outcome is Effect of Lanreotide on Gastrointestinal Motility as Measured by Smart Pill.

2. Clinical Question

This is a question about change observed during treatment in a small, single-group pilot study, not a randomized comparison of lanreotide against another treatment or against no treatment. Without a concurrent comparator, a change over time cannot by itself distinguish a treatment effect from other explanations, such as natural variation in symptoms or measurements, changes in care, or differences in who contributed data at each assessment. These are general limitations of an uncontrolled design; whether any of them affected this study is not reported in the registry record.

The registry identifies improvement in symptoms assessed by the Patient Assessment of Upper GastroIntestinal Symptom Severity Index as a secondary outcome. It does not report the symptom results or details of the instrument’s scoring in the available record. The primary clinical question therefore concerns a measured transit-time outcome, while the listed secondary question concerns patient-reported symptoms. Neither outcome’s results are reported in the registry record.

3. Trial Design

The registry describes a prospective, non-randomized, single-center pilot study. It lists the phase as Phase 2, the allocation as not applicable, the masking as none, and the model as single group. The lanreotide arm is described as open label, meaning treatment was not masked in the study record. There is no separate control arm, so the study cannot make a like-for-like comparison between participants assigned to lanreotide and participants assigned to another condition.

The registry reports enrollment of 12. The official title, however, describes a study of 20 participants. These are inconsistent descriptions in the record; the enrollment entry reports 12, while the title refers to 20 participants. The record does not explain the difference. The study is marked completed. Its recorded start date is 2017-05-11, and its primary completion date is 2018-10-21.

The intervention is lanreotide. The brief summary describes a long-acting medication given once a month by deep subcutaneous injection, with injection sites alternated on subsequent injections. The record does not report the administered dose, treatment adherence, dose changes, or the exact treatment duration for individual participants.

The primary outcome is described as the effect of lanreotide on gastrointestinal motility measured by Smart Pill, with a timeframe of 3 months. The secondary outcome concerns symptom improvement assessed using the Patient Assessment of Upper GastroIntestinal Symptom Severity Index. The registry lists two outcome measures and one analysis, but the results and analysis details are not reported in the registry record. The record also does not describe the recruitment process, detailed eligibility criteria, the number screened, or the handling of participants who did not complete follow-up.

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: This is an open label non-randomized study. All enrolled participants were checked to see if they meet all the screening requirements to participate. All willing and qualified participants received the study mediation.

Overall Study

5. Baseline Characteristics

The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.

6. Primary Endpoint

The registry lists the primary outcome as Effect of Lanreotide on Gastrointestinal Motility as Measured by Smart Pill, measured over 3 months.

The primary outcome concerns small bowel transit time measured using Smart Pill, with the unit recorded as minutes and the outcome parameter described as a mean. The stated timeframe is 3 months. Transit time is a physiological measure of how long material takes to pass through the small bowel; in general, a measured transit outcome can complement reports of symptoms by describing an aspect of gastrointestinal function.

The endpoint description adds a responder definition: if small bowel transit time, measured by wireless capsule endoscopy, is decreased to < 6hrs, the participant would be considered a responder and lanreotide efficacious. This wording combines a continuous measure, mean transit time, with a threshold-based responder concept. The record does not clarify whether the primary analysis was intended to compare mean change, evaluate the proportion meeting the responder definition, or report both. It also does not give a baseline value or explain the reference point for “decreased.”

No observed mean, standard error, change from baseline, confidence interval, p-value, or responder count is reported in the registry record. The endpoint definition therefore indicates what the study intended to assess, but the posted record does not establish the size or precision of any observed change. The record does not describe measurement procedures, device data quality checks, or how missing Smart Pill measurements were handled.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry metadata describes a continuous primary outcome summarized as a mean, with standard error as the dispersion measure. It does not report the actual estimate or the statistical procedure used to produce it. The explanations below describe general statistical principles relevant to this kind of outcome, not confirmed details of the study’s analysis.

What does a mean represent?

A mean combines measurements across participants into a group-level average. For a transit-time outcome, it can provide a concise description of the group’s measured transit time at an assessment. If measurements are taken at multiple times, an analysis may focus on values at a specified time, changes from baseline, or a contrast between timepoints. Those choices can lead to different estimates and interpretations. The registry record specifies the 3-month timeframe but does not report the analysis definition or say whether the primary measure was an endpoint value or a change from baseline.

A group mean does not show how individual participants’ measurements are distributed. Participants may have similar values or widely varying values while sharing the same mean. A mean can also be influenced by unusually high or low observations. Individual-level data or suitable summaries of the distribution would help readers understand these features, but are not reported in the registry record.

What does the standard error describe?

In general, a standard error describes how precisely a sample statistic, such as a mean, estimates the corresponding quantity in a broader population under the statistical model. A smaller standard error indicates greater precision under that model; it is not a measure of the spread among individual participants. The standard deviation is commonly used to describe individual-level spread, while the standard error concerns uncertainty in an estimated mean. The registry record names standard error as the dispersion measure but gives no value, so precision cannot be judged.

A confidence interval is a range of values compatible with the data under a specified statistical procedure and its assumptions. It conveys uncertainty more directly than a point estimate alone. Interpreting an interval requires knowing the estimate, the interval endpoints, and how the interval was calculated. No confidence interval for the primary outcome is reported in the registry record. A p-value, if reported, would address compatibility with a specified null model; it would not measure effect size, clinical importance, or the probability that a hypothesis is true. No p-value or test specification is reported.

How does the responder definition differ from a mean?

The endpoint description also defines response using a threshold: transit time decreased to < 6hrs. In general, a threshold converts a continuous measurement into a category, such as meeting or not meeting a response criterion. The proportion meeting a threshold can be easy to communicate, but it depends on the chosen cutoff and may discard information about how far measurements lie above or below it. A mean preserves the scale of the measure but does not reveal how many participants crossed a threshold. Reporting both can offer complementary descriptions, provided the analysis plan defines them clearly. The registry record does not say how the responder criterion relates to the stated mean endpoint or report responder results.

Why does the single-group design matter?

In general, a pre-post analysis in one group describes changes observed among participants over time. It does not provide a contemporaneous estimate of what would have happened without the intervention. Symptoms and physiological measurements can vary naturally, and observations can be affected by factors other than treatment. Without random assignment and a control group, those influences cannot be separated from a treatment-related change using a direct between-group comparison. The registry record identifies a single-group, non-randomized design, so any change would need cautious interpretation as an observed pattern rather than a definitive causal effect.

Open-label conduct also means the intervention was not masked, according to the record. In general, knowledge of treatment can affect behavior, reporting, co-interventions, or assessment, depending on the outcome and study procedures. A device-measured endpoint may involve different sources of influence than a self-reported symptom endpoint, but the record does not describe safeguards against measurement or reporting bias.

What do planning calculations mean?

Planning illustrations based on an observed effect are not observed power and do not change the evidence produced by the study. The stated illustration assumptions include a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and effects defined as fractions of the observed effect. These are planning assumptions, not a description of this trial’s design: the registry describes a single-group study, not equal allocation between two arms. The record does not report the trial’s own sample-size justification. Any sample-size illustration should therefore be read as a hypothetical planning exercise, not as evidence that the study had a particular probability of detecting an effect or that its observed results are conclusive.

More generally, sample-size planning depends on the outcome, target effect, variability, analysis method, and other assumptions. If these inputs differ from the study context, planning results may not translate directly. The registry record does not report the basis for a prospective sample-size calculation or whether any calculation informed enrollment. No such details should be inferred from a planning illustration.

10. Multiplicity, Missing Data and Analysis Populations

The registry lists a primary motility outcome and a secondary symptom outcome. It does not report a multiplicity strategy, such as whether testing of the secondary outcome was adjusted for multiple comparisons or whether a hierarchy of outcomes was prespecified. When several outcomes or analyses are examined, the chance of at least one apparently notable result can increase; in general, a prespecified approach helps readers understand how that issue was addressed. The record does not report enough analysis detail to assess multiplicity handling.

The registry lists two outcome measures and one analysis, but does not give the analysis population, the number of participants contributing data to each endpoint, or the statistical methods. It does not report whether all enrolled participants were included in the primary analysis, whether the analysis used participants with complete measurements, or whether another population was defined. These details matter because results can depend on which observations are included and how eligibility for analysis is determined.

Missing-data methods are not reported in the registry record. The record does not say whether measurements were missing, why any data might have been missing, or how missing values were handled. In general, excluding participants with missing outcomes can affect an estimate if missingness is related to outcome values or participant characteristics. Imputation and model-based approaches also rely on assumptions that need to be stated and assessed. Without the missingness pattern and analysis details, the effect of missing data cannot be evaluated.

The outcome description also combines a mean measure with a responder threshold. The registry record does not report whether both were analyzed, which was considered primary for inference, or how any multiple outcome definitions were handled. These omissions limit the ability to reconstruct the analysis or judge whether the reported endpoint definition and the statistical analysis align.

11. Safety Results

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

Deaths

Other (non-serious) adverse events

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

Most frequent non-serious adverse event terms

The registry reports 0 serious adverse events among 9 participants at risk in the lanreotide group, 2 other adverse events among 9 participants at risk, and 0 deaths. These are counts reported in the registry record. The record does not identify the types of the other adverse events, and it lists no top serious adverse event terms.

These figures should be read as the study’s reported counts, not as a complete estimate of safety risk. The record does not describe how adverse events were defined, how they were collected, how long participants were observed for safety, or whether events were judged related to lanreotide. It also does not explain why the safety population at risk differs from the registry enrollment entry. The official title’s participant count also differs from the enrollment entry, and the record does not reconcile those descriptions.

In general, a count of zero serious adverse events in a small study does not establish that serious events cannot occur. Rare or delayed events may not be observed in a limited study, and adverse event counts alone do not establish causation. Conversely, the two other adverse events cannot be interpreted fully without their descriptions and context. The registry record’s safety figures are therefore descriptive, with important information about event type, exposure, follow-up, and attribution not reported.

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

The record establishes that a completed pilot study examined lanreotide in patients with small bowel motility disorders. It describes a prospective, non-randomized, open-label, single-group design and identifies Smart Pill transit time as the primary motility outcome at the 3-month timeframe. It also gives a threshold-based responder definition and lists a patient-reported symptom measure as a secondary outcome. This is information about the research question and design, not evidence of benefit or lack of benefit.

The primary outcome results are not reported in the registry record. There is no posted estimate of mean transit time, change over time, uncertainty interval, test result, or responder count. The symptom outcome results are also not reported. Without those results, the direction and magnitude of any observed changes cannot be determined. Missing results should not be interpreted as showing that the intervention had no effect.

Even a reported improvement in a single-group study would not, by itself, establish that lanreotide caused the change. In general, a control group and random assignment help distinguish intervention effects from other influences. This study’s design does not include a concurrent comparator, and its open-label status may be relevant when interpreting outcomes, particularly those involving participant reports. The record does not provide analysis details that would resolve these design limitations.

The safety entries show the adverse event counts recorded for the lanreotide group, but the record does not provide event descriptions or enough context to characterize safety comprehensively. The counts do not establish that events were or were not caused by treatment. The study’s pilot designation and the discrepancy between the enrollment entry and the official title also warrant care when describing the study’s scale.

In general, a pilot study can help describe feasibility and inform later research, but the registry record does not report feasibility outcomes or a sample-size rationale. The stated planning illustrations are hypothetical calculations under their listed assumptions, not observed power and not a substitute for the study’s outcome results. The appropriate conclusion from the posted record is limited: it documents the study design and selected safety counts, while efficacy estimates and important analysis details are not reported.

13. Why This Trial Matters Statistically

This study raises several statistical issues that matter when interpreting small, uncontrolled clinical research. First, its single-group design means that any change over time would be a within-group observation rather than a randomized treatment comparison. Such observations can be useful for describing patterns, but they do not isolate treatment effects from changes that might occur for other reasons. The design should therefore shape the strength of any conclusions drawn from outcome data.

Second, the primary outcome description mixes a continuous mean measure with a threshold-based responder definition. These summaries answer distinct questions and can convey different aspects of the data. A mean can mask variation between participants, while a responder classification depends on the threshold and reference point. Clear prespecification and reporting are needed to understand which question was primary and how the results should be interpreted. That relationship is not clarified in the registry record.

Third, the posted record does not report the primary or secondary efficacy results, nor the uncertainty around them. A study’s completion status and endpoint definitions do not substitute for outcome estimates. Without estimates and precision measures, readers cannot judge how large or uncertain an observed pattern might have been. The record also omits analysis-population, missing-data, and multiplicity details, which are important for understanding how results were generated.

Finally, the enrollment entry and official title give inconsistent participant counts, and the safety population is described separately. These details illustrate why clear accounting of enrollment, analysis populations, and safety exposure matters. The registry record does not explain the discrepancies. Taken together, the study is statistically informative as an example of how design, endpoint definition, transparency, and uncertainty affect interpretation; the information posted is not sufficient to estimate efficacy.

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.

Several trial-specific limitations remain beyond the absence of posted efficacy estimates. The study is described as non-randomized, single-group, open label, and single center. It therefore has no concurrent control group for separating treatment-associated changes from other influences, and the record does not describe measures to reduce bias in outcome assessment.

The registry enrollment entry reports 12, while the official title describes 20 participants. The record does not explain this inconsistency. It also reports 9 participants at risk for the lanreotide safety counts, without explaining how that safety population relates to enrollment. The number contributing data to the primary and secondary outcomes is not reported in the registry record.

The primary endpoint description specifies a mean Smart Pill transit-time measure and also defines response using a decrease to < 6hrs. It does not clarify the reference measurement for “decreased,” whether the mean or responder definition was primary for analysis, or how the two summaries were related. The record does not report baseline values, results, measurement completion, or device-data handling.

Eligibility criteria, participant characteristics, dose, adherence, treatment exposure, and follow-up details are not reported in the registry record. Neither the statistical model nor the analysis population, missing-data approach, multiplicity handling, or sample-size justification is described. The official title calls the study a pilot, but the record does not state its feasibility objectives or provide their results. These gaps restrict assessment of representativeness, precision, outcome validity, and the robustness of any analysis.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Lanreotide in the Treatment of Small Bowel Motility Disorders — NCT03012594.
• Registry record last updated 2021-01-15; status last verified 2020-12.
• Results first posted 2021-01-15.
• 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

NCT03012594 is a completed Phase 2 study sponsored by Northwell Health, titled “Lanreotide in the Treatment of Small Bowel Motility Disorders.” The registry describes a prospective, non-randomized, single-center, open-label, single-group pilot study. Enrollment is recorded as 12, while the official title refers to 20 participants; the discrepancy is not explained.

The primary outcome is mean Smart Pill small bowel transit time, with a 3-month timeframe. Its description also defines response as a decrease to < 6hrs. A secondary outcome concerns symptoms assessed using the Patient Assessment of Upper GastroIntestinal Symptom Severity Index. Efficacy results and key analysis details are not reported in the registry record.

The safety entries report 0 serious adverse events among 9 participants at risk, 2 other adverse events among 9 participants at risk, and 0 deaths. Event descriptions and additional safety context are not reported.