Study Evaluating SOM230 in Patients With Metastatic Carcinoid Tumors: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Study Evaluating SOM230 in Patients With Metastatic Carcinoid Tumors (NCT00088595), a non-randomized phase 2 study. The primary outcome is Symptom Control (Diarrhea/Flushing) Using a Patient Symptom Diary.

2. Clinical Question

This question focuses on symptom control rather than a comparison of treatment strategies. Complete control requires a specified pattern of bowel movements and no flushing episodes; partial control allows a different symptom pattern. The definitions also identify treatment failure as not obtaining either kind of success during a consecutive 15-day period at a constant dose level.

The registry describes a phase II study in patients with carcinoid tumors and identifies the metastatic population in the study title. It does not report a control group against which to estimate how symptoms would have changed without pasireotide or under another intervention. Consequently, the central statistical question is descriptive: what symptom-control outcomes were recorded among the assessed participants? The posted results do not give the counts needed to answer that question.

3. Trial Design

This was an open-label, multicenter, phase II study evaluating twice daily dosing of SOM230, also known as pasireotide, in patients with metastatic carcinoid tumors. The registry lists a single-group model, nonrandomized allocation, and no masking. Enrollment is recorded as 45. The study status is completed; the recorded start is 2004-01 and primary completion is 2008-07.

These design features matter to interpretation. A single-group study can describe outcomes among participants receiving the intervention, but it does not provide a randomized concurrent control group for estimating a treatment contrast. Nonrandomized allocation also means that dose classes should not be treated as if assignment to them were randomized. The registry does not describe a comparator arm, and the posted information does not support a controlled estimate of what would have happened under another treatment or without treatment.

Open-label means masking is listed as none. The record therefore does not describe masking of participants or study personnel. It does not report whether outcome assessment was independently blinded. Because the primary endpoint uses a patient symptom diary, the absence of stated masking is relevant context when considering symptom reports, but it does not establish the direction or size of any possible reporting influence.

The efficacy analysis population consisted of 44 patients who had received at least one dose and had at least one efficacy assessment available after treatment began. The registry says that one patient without a post-baseline efficacy assessment was excluded from this population. The record does not explain further how participants were assigned to dose levels, whether doses changed over time, or how each dose class related to the primary endpoint analysis. Those details are not reported in the registry record.

The registry identifies five outcome measures, including symptom control as the primary outcome and secondary outcomes concerning duration of symptom control by dose class, overall tumor response, and safety and tolerability. It lists no analysis entries. The detailed statistical plan, rationale for the enrollment target, and any interim analyses 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.

Overall Study

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: The efficacy analysis population (EAP) consisted of 44 patients all of whom had at least one efficacy assessment available after receiving at least one dose of study drug, but excluded 1 patient who had no post-baseline efficacy assessments.

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 Symptom Control (Diarrhea/Flushing) Using a Patient Symptom Diary, measured over 15 days.

The primary endpoint is titled symptom control for diarrhea and flushing using a patient symptom diary. It is recorded as a number of participants, making the measure a count of people meeting an outcome definition rather than a continuous summary of symptom scores. The assessment timeframe is 15 days.

Complete symptom control is defined as an average of ≤ 3 bowel movements per day for at least 15 consecutive days, with no more than 3 episodes on any given day, and no flushing episodes during the interval studied. Partial symptom control is defined as an average of < 4 bowel movements per day for at least 15 consecutive days, with no more than 6 episodes on a given day, and an average of fewer than 2 daily flushing episodes over the same interval. Treatment failure is failure to obtain partial or complete treatment success over a consecutive 15-day period at a constant dose level.

Using explicit criteria can make a symptom endpoint easier to interpret than an undefined judgment of improvement. A diary can capture recurring symptoms over time, while the consecutive-day requirement distinguishes sustained control from a brief favorable interval. These are general measurement advantages; they do not establish that the diary was completed consistently or that the endpoint was assessed without bias in this study.

The registry gives the endpoint definition and analysis population but does not report how many participants achieved complete control, partial control, or treatment failure. It also does not report an estimate of uncertainty around those counts.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What kind of outcome is being counted?

General statistical context: A binary outcome classifies each assessed participant into a category, such as meeting or not meeting a prespecified criterion. A count outcome then reports how many participants fall into the category. The registry describes the primary endpoint as a number of participants and identifies the outcome type as participants with an event. This is distinct from reporting an average symptom score or a rate per unit of follow-up.

For this endpoint, classification depends on the diary-based definitions of complete control, partial control, or treatment failure. Those categories have operational thresholds for bowel movements, flushing, and the duration of the observed interval. Clear thresholds allow readers to understand what the outcome is intended to represent. They do not, by themselves, show how often the outcome occurred. The registry record does not report the primary counts.

What does a confidence interval tell a reader?

General statistical context: A confidence interval describes the uncertainty around an estimate under the assumptions of the statistical method used. For a proportion or count-based outcome, an interval can help distinguish a precisely estimated result from one with substantial uncertainty. It does not correct for a biased design, incomplete outcome measurement, or an unsuitable definition.

The registry does not report a confidence interval for the primary endpoint. It also does not report a primary effect estimate or a test result. Accordingly, no claim about the precision or statistical significance of symptom control can be made from the posted primary outcome information. The absence of an interval is especially important for a small phase II study because a point estimate, even if present, would need to be considered alongside its uncertainty; no such estimate is given.

Why does the study structure matter?

General statistical context: Randomization helps balance measured and unmeasured factors between treatment groups on average, supporting a causal interpretation of a group contrast. A single-group study does not offer that concurrent randomized comparison. Outcomes observed after treatment can be described, but temporal changes alone cannot isolate the effect of the intervention from other explanations.

This study is listed as nonrandomized and single-group, with no masking. The registry also gives safety information across dose classes, but does not report randomized allocation to those classes. Any differences between dose-class observations could reflect factors other than dose, including how participants entered or remained in a class. The record does not describe these assignment processes, so dose-class findings should remain descriptive.

How should planning calculations be understood?

General statistical context: A sample-size calculation asks how many observations a future study might require under specified assumptions. It is not a measure of the probability that the completed study detected an effect, and it is not observed power. Calculations based on a fraction of an observed effect are planning illustrations: they show how a future design could be sized if that assumed effect were of interest.

The planning assumptions described for this analysis use a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. The effect to detect is defined as a stated fraction of an observed effect. For non-time-to-event outcomes, the quantities are participants per arm for 80% power or participants per arm for 90% power; time-to-event illustrations instead concern total events. These are planning conventions and do not describe the actual design of this single-group study. The registry does not report its own sample-size justification, and no planning value should be mistaken for an observed trial result.

What statistical details are absent?

General statistical context: A complete analysis description would usually identify the analysis population, outcome coding, method for estimating results, handling of missing observations, and any planned adjustments or subgroup analyses. The analysis choices affect how results should be interpreted. In particular, missing symptom diaries can change which participants contribute to an endpoint, while adjustment choices can alter estimates when comparisons are made.

The registry describes the efficacy analysis population and says it excluded one patient with no post-baseline efficacy assessment. It lists no analysis entries. A model, confidence-interval method, hypothesis test, covariate adjustment strategy, and statistical analysis plan are not reported in the registry record. Nor does the record describe how diary incompleteness or dose changes were handled. These absences prevent reconstruction of an inferential analysis from the registry information.

Over-dispersion is a general issue for count outcomes when observed variability exceeds what a simple count model assumes. It can make standard errors too small if a model ignores the extra variability. That concept is not directly the primary endpoint’s structure: the registry describes a count of participants meeting a binary criterion, not a count of repeated symptom events as the primary result. No model for over-dispersion is reported, and the primary outcome results are not available to assess a distributional model.

10. Multiplicity, Missing Data and Analysis Populations

The registry identifies symptom control as the primary outcome and lists five outcome measures. Named secondary outcomes concern duration of complete symptom control by dose class, duration of partial symptom control by dose class, overall tumor response, and safety and tolerability. Considering multiple outcomes can create multiplicity: when many hypotheses are tested, the chance of at least one apparently unusual result can increase. General approaches include prespecifying a primary comparison, adjusting inference across tests, or clearly treating secondary analyses as exploratory.

The registry record does not report a multiplicity procedure, a hierarchy among secondary outcomes, or a detailed statistical analysis plan. It lists no analysis entries. It is therefore not possible to determine whether any adjustment was planned or performed. The posted information does not include p-values for the primary or secondary outcomes, and it does not report the primary symptom-control counts.

The efficacy analysis population included 44 patients with at least one efficacy assessment after receiving at least one dose. The registry states that one patient with no post-baseline efficacy assessment was excluded. This identifies a population rule, but it does not explain how missing data among assessed participants were handled, whether incomplete diaries occurred, or whether any imputation was used. Those methods are not reported in the registry record.

General statistical context: excluding participants without post-baseline measurements can make the analyzed population differ from the enrolled population. The consequences depend on why measurements are missing and how the excluded participants might have fared; the registry does not report enough information to assess those possibilities. The named efficacy population should therefore be kept distinct from the overall enrollment figure, and neither should be used to infer unreported outcome values.

11. Safety Results

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

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 5% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: hepatic artery embolism (6.5%), erosive duodenitis (3.2%), gastritis (3.2%), nausea (3.2%), rectal haemorrhage (3.2%).

The registry reports serious and other adverse-event counts separately for three pasireotide dose classes. Serious adverse events are listed as 3 of 45 at risk for Pasireotide 300 ≤ 900 μg, 3 of 43 at risk for Pasireotide > 900 ≤ 1500 μg, and 10 of 31 at risk for Pasireotide > 1500 ≤ 2400 μg. The other adverse-event counts are 24 of 45, 20 of 43, and 21 of 31, respectively. These are counts within dose-class records, not randomized comparisons.

The listed serious-event terms are hepatic artery embolism at 6.5%, and erosive duodenitis, gastritis, nausea, rectal haemorrhage, and vomiting at 3.2% each. The registry does not explain whether participants could contribute more than one term, so the percentages should not be added or interpreted as mutually exclusive categories. Death counts are not reported in the safety entries.

General statistical context: adverse-event counts describe events recorded under the registry’s reporting definitions. They do not establish causation, and comparisons across groups require attention to assignment, time at risk, and ascertainment. Because this study is nonrandomized and single-group, these dose-class figures are descriptive. The record does not provide a like-for-like randomized comparison or details sufficient to interpret differences as dose effects.

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

The record establishes that this was a completed, open-label, nonrandomized phase II study of pasireotide in patients with metastatic carcinoid tumors. It defines a diary-based primary endpoint for sustained control of diarrhea and flushing, gives the timeframe and criteria, and describes the efficacy analysis population. These details allow a reader to understand what the investigators intended to measure and which population was identified for efficacy assessment.

The record does not report the primary endpoint results. It gives no count of participants with complete symptom control, partial symptom control, or treatment failure, and no estimate of uncertainty for those outcomes. As a result, the posted information cannot quantify symptom-control outcomes or establish how precisely they were estimated. Secondary outcome results are also not reported in the information described in the registry record.

The study design does not include a randomized comparator. Therefore, the registry information cannot support a randomized estimate of the difference between pasireotide and another treatment or control condition. Dose-class safety counts are also not randomized comparisons and cannot establish that any observed difference arose because of dose. These limits concern interpretation of the design; they do not determine what an unreported analysis might have found.

The adverse-event entries provide descriptive counts and named serious-event terms, but they do not establish that the intervention caused those events. The record does not report deaths in those entries, and it does not provide enough detail to infer whether the listed terms overlap. No medical decision should be based on these registry entries alone.

In short, the registry record is informative about the study’s structure, population definition, endpoint rules, and selected safety reporting. It does not provide the efficacy results needed to judge the observed symptom-control outcomes. Distinguishing what the study measured from what the record actually reports is essential: endpoint definitions are not outcome findings, and planning illustrations are not observed trial results.

13. Why This Trial Matters Statistically

This trial is statistically instructive because its primary endpoint is a participant count built from explicit symptom criteria. Readers can see how the investigators translated diarrhea and flushing into a sustained outcome over a consecutive 15-day period. That makes the target construct concrete, while also highlighting that a result depends on diary completeness and consistent application of the classification rules.

The single-group, nonrandomized design illustrates the distinction between describing outcomes and estimating comparative effects. A study can document what was observed among participants who received an intervention without supplying a randomized counterfactual. The registry’s dose-class safety summaries provide another useful example: group-specific counts can be reported, but they do not become causal dose comparisons merely because they are displayed separately.

The difference between enrollment and the stated efficacy population also matters. The record identifies 45 enrolled and an efficacy analysis population of 44, with one patient excluded because no post-baseline efficacy assessment was available. This is a transparent population detail, but it does not reveal how other missing measurements were handled or whether the available assessments represent all enrolled participants equally.

Finally, this record demonstrates why trial registration details and outcome findings should be read separately. The definitions, dates, design descriptors, and safety listings provide context, but the primary efficacy counts and statistical uncertainty are not reported. Statistical interpretation depends not only on the method a study intended to use, but also on whether the results and analysis details needed to evaluate it are available. The registry record is therefore useful for understanding the measurement and design while leaving the principal efficacy question unanswered.

14. Limitations

• The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
• Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.

The main limitation for interpreting efficacy is that the registry record does not report the primary outcome results. It specifies complete and partial symptom-control criteria but gives no participant counts for those categories, no treatment-failure count, and no estimate of uncertainty. The listed outcome definition cannot substitute for the missing result.

The design is nonrandomized, open-label, and single-group. There is no randomized comparator described, so the record cannot support a controlled treatment contrast. Masking is listed as none; the registry does not say whether any outcome assessment was independently blinded. Because the endpoint relies on a patient symptom diary, the absence of reported assessment procedures leaves uncertainty about how diary information was collected and adjudicated.

The efficacy population is defined as 44 patients with at least one post-baseline efficacy assessment after at least one dose, excluding one patient without such an assessment. The record does not report other missing-data patterns, diary completion, imputation, or sensitivity analyses. It also does not describe how participants entered dose classes or how dose changes related to outcome classification.

The statistical analysis plan, sample-size justification, model choices, confidence-interval methods, hypothesis tests, multiplicity handling, and interim analyses are not reported in the registry record. The record lists no analysis entries. Safety counts are descriptive and grouped by dose class; they should not be treated as randomized comparisons. The registry also does not report deaths in the listed safety entries or clarify whether serious-event terms overlap.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Study Evaluating SOM230 in Patients With Metastatic Carcinoid Tumors — NCT00088595.
• Registry record last updated 2012-06-04; status last verified 2012-05.
• Results first posted 2011-05-06.
• 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

NCT00088595 is a completed, open-label, multicenter, phase II study evaluating twice daily pasireotide (SOM230) in patients with metastatic carcinoid tumors. It is listed as nonrandomized and single-group, with enrollment of 45. The recorded start is 2004-01 and primary completion is 2008-07.

The primary outcome is the number of participants meeting diary-based definitions of complete or partial symptom control for diarrhea and flushing over a consecutive 15-day period. The efficacy analysis population consisted of 44 patients with at least one efficacy assessment after at least one dose; one patient without a post-baseline efficacy assessment was excluded.

The primary outcome counts, their statistical uncertainty, and detailed analysis methods are not reported in the registry record. Safety counts are listed by dose class, but they are descriptive rather than randomized comparisons.