Pasireotide LAR in Severe Polycystic Liver Disease: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Pasireotide LAR in Severe Polycystic Liver Disease (NCT01670110), a randomized phase 2 study. The primary outcome is Change in Liver Volume.

2. Clinical Question

3. Trial Design

This was a Phase 2 randomized, double-masked, placebo-controlled trial with a crossover model. Mayo Clinic was the sponsor. The registry lists enrollment of 48 and a completed study. The recorded start was 2012-08, and primary completion was 2018-09. These registry dates describe the study record; they do not establish how long each participant received either intervention.

The interventions were pasireotide LAR (SOM230) and placebo injection. For the primary outcome, the posted arm summaries include 29 participants for pasireotide LAR and 12 for placebo. These outcome-specific counts differ from the enrollment figure, and the reason for that difference is not reported in the registry record. The analysis population and the handling of missing primary-outcome observations are also not reported in the registry record.

The primary outcome timeframe is baseline to 12 month. The registry reports an ANCOVA comparison for the primary outcome, with p = 0.001, but does not give the adjusted estimate, its confidence interval, or details of the model. It records t-tests, 2 sided, for the posted eGFR, serum creatinine, and blood-glucose secondary outcomes. The treatment sequence and crossover-specific analytic details are not reported in the registry record, so it is not possible to determine from the record how the crossover structure was incorporated into each analysis.

The registry lists 8 outcome measures and 15 analyses. It also lists secondary outcomes concerning eGFR, serum creatinine, blood glucose, hemoglobin A1C, heart rate, and quality of life. Although six secondary outcome titles are listed, numerical summaries are available in the record for only three of them.

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

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 Change in Liver Volume, measured over baseline , 12 month.

The primary endpoint is change in liver volume, measured as percentage of change from baseline to 12 month. The registry describes the endpoint as a mean and reports standard deviations for the two arms. Its stated calculation uses the baseline and 12-month liver-volume values. The registry’s wording of the calculation is unusual, so the precise implementation cannot be clarified beyond the definition recorded.

Liver volume is the direct measurement named in the primary outcome, while percentage change expresses the change relative to the baseline measurement. This form can help describe change on a scale that accounts for the starting value, although it does not by itself establish whether a difference is clinically important. That judgment would require context about patients, measurement variability, and meaningful thresholds, none of which is reported in the registry record.

The primary endpoint is appropriate for asking whether the two randomized groups had different average changes in the measured quantity over the stated timeframe. It is a continuous outcome, so its interpretation depends on both the difference in group means and the spread of participant values. The posted group standard deviations describe that spread. The registry does not report the distribution of individual changes, a clinically meaningful threshold, or fuller details of how liver volume was measured.

7. Results Posted in the Registry

• Pasireotide LAR (SOM230): mean -3.36 versus 6.29 on control; difference -9.65 (95% CI -14.41 to -4.89), standardized effect d = -1.33; z-test p = <0.001.

8. Secondary Efficacy Endpoints

The registry also posts results for secondary outcomes. For each outcome below, the endpoint type, the test applied to the posted summary statistics, the result and its interpretation are given in turn. Secondary endpoints are usually not adjusted for multiple testing, so they support rather than confirm the primary finding.

Percentage Change in Estimated Glomerular Filtration Rate (eGFR)

• Endpoint type: Continuous: a mean value per participant, compared as a difference in means.
• Test: Unadjusted two-sample z-test for the difference in means, using the posted standard deviations.
• Result:
  • Pasireotide LAR (SOM230): mean -0.30 versus -2.20 on control; difference 1.90 (95% CI -9.34 to 13.14), standardized effect d = 0.13; z-test p = 0.740.
• Interpretation:
  • The 95% confidence interval includes no difference, so these data do not show a statistically significant difference between Pasireotide LAR (SOM230) and the control. The interval gives the range of effects that remain compatible with the data.

Percentage Change in Serum Creatinine

• Endpoint type: Continuous: a mean value per participant, compared as a difference in means.
• Test: Unadjusted two-sample z-test for the difference in means, using the posted standard deviations.
• Result:
  • Pasireotide LAR (SOM230): mean 1.80 versus 3.40 on control; difference -1.60 (95% CI -11.45 to 8.25), standardized effect d = -0.12; z-test p = 0.750.
• Interpretation:
  • The 95% confidence interval includes no difference, so these data do not show a statistically significant difference between Pasireotide LAR (SOM230) and the control. The interval gives the range of effects that remain compatible with the data.

Percent Change in Blood Glucose

• Endpoint type: Continuous: a mean value per participant, compared as a difference in means.
• Test: Unadjusted two-sample z-test for the difference in means, using the posted standard deviations.
• Result:
  • Pasireotide LAR (SOM230): mean 39.00 versus 2.20 on control; difference 36.80 (95% CI 22.90 to 50.70), standardized effect d = 1.38; z-test p = <0.001.
• Interpretation:
  • The 95% confidence interval excludes no difference, so the mean is statistically higher for Pasireotide LAR (SOM230) than for the control. This is an unadjusted, secondary comparison, so it is suggestive rather than confirmatory.

The numerical secondary results available in the registry cover eGFR, serum creatinine, and blood glucose. Across these outcomes, the pattern is not uniform: the recalculated mean differences and their intervals differ in direction and in how much uncertainty they express. The posted results therefore do not support treating all secondary measures as if they showed the same pattern as the primary liver-volume comparison.

The eGFR and serum-creatinine comparisons have intervals that include zero, whereas the blood-glucose comparison’s reported recalculated interval does not. These are unadjusted comparisons and should be interpreted as secondary findings, not as a replacement for the primary endpoint. The registry lists hemoglobin A1C, heart rate, and quality of life as additional secondary outcomes, but numerical summaries for those outcomes are not reported in the registry record. Their results cannot be inferred from the available outcomes.

Secondary findings generally carry less evidential weight when many outcomes or analyses are examined without a reported multiplicity strategy. The record lists 15 analyses and does not report whether adjustment for multiple comparisons was planned or performed. Without such information, isolated small p-values among secondary analyses may reflect chance as well as real differences. This concern does not erase the reported estimates; it changes how confidently they should be treated as confirmatory.

The secondary measures also address different aspects of the study, including kidney-function markers, glucose-related measures, heart rate, and quality of life. Their relation to the primary liver-volume question is therefore supportive or contextual rather than a direct replication of it. The registry does not report a hierarchy among the secondary outcomes or a prespecified strategy for deciding which would be interpreted as key evidence.

Secondary outcomes registered for this trial, for reference (only those above were recalculated):

• Percentage Change in Estimated Glomerular Filtration Rate (eGFR)
• Percentage Change in Serum Creatinine
• Percent Change in Blood Glucose
• Percentage Change in Hemoglobin A1C
• Percentage Change in Heart Rate
• Change in Quality of Life

9. Statistical Interpretation

10. Statistical Methods Explained

The primary endpoint is continuous: each participant’s outcome is represented by a numerical change in liver volume, and the registry summarizes each arm with a mean and standard deviation. A mean describes the arithmetic center of the observations. A standard deviation describes how dispersed individual observations are around that mean. These summaries are useful for describing group results, but they do not show the distribution’s shape or individual participant outcomes.

What does the mean difference represent?

The recalculated comparison subtracts the placebo mean from the pasireotide LAR mean. The resulting mean difference is -9.65 percentage points as a difference between the two reported percentage-change means. Its negative sign indicates that the pasireotide LAR group’s posted mean is lower. The estimate is unadjusted: it is derived from the arm-level summaries and does not account for covariates or other model terms. It should not be mistaken for the sponsor’s ANCOVA estimate, which is not reported.

The standard deviations describe variation within each arm, whereas the mean difference describes separation between the arm means. Neither statistic alone gives a complete account of the data. For example, the same mean difference can be estimated with different precision depending on variability and the amount of information available. The registry reports 29 participants for the pasireotide LAR primary summary and 12 for placebo. It does not report why these outcome counts differ from one another or from enrollment.

What do the p-values say?

A p-value measures how compatible the observed data are with a specified null hypothesis under the assumptions of the analysis. A small p-value is not the probability that the null hypothesis is true, and it does not quantify clinical importance. The unadjusted recalculation reports p < 0.001. The sponsor’s reported primary ANCOVA gives p = 0.001. Since the sponsor’s model and the recalculation are not the same procedure, their p-values answer related but distinct analysis questions.

The registry reports t-tests, 2 sided, for the three secondary outcomes with numerical summaries. A two-sided test assesses evidence for a difference in either direction, rather than restricting the comparison to one direction. The exact analysis details beyond the named tests are not reported in the registry record. The crossover model makes the unreported handling of within-participant and treatment-sequence information especially relevant to understanding the analyses; the registry does not describe that handling.

Why does the crossover model matter?

In general, crossover designs allow participants to receive more than one intervention in different periods, which can support within-participant comparisons. Such designs require attention to treatment sequence, period effects, and possible carryover from an earlier treatment period. These are general statistical considerations, not claims about the conduct of this trial. The registry identifies the model as crossover but does not report sequences, period structure, washout, or a crossover-specific analysis. The posted arm summaries alone do not show whether the reported group-level calculations use paired information.

The sponsor’s primary method is listed as ANCOVA. In general, analysis of covariance compares outcomes while accounting for selected covariates. The registry does not specify which covariates were included, whether baseline liver volume was included, or what other model assumptions were used. Because the adjusted estimate and model details are absent, the sponsor’s result can be described by its reported method and p-value, but not reconstructed from the record.

What is the role of the recalculation?

A recalculated estimate is an unadjusted analysis of posted summary statistics. It provides a transparent comparison of the reported group means, but it cannot reproduce an analysis that uses participant-level values, covariate adjustment, paired crossover data, or a different missing-data strategy. The recalculation is therefore a complementary statistical reading, not a replacement for the sponsor’s analysis. The registry does not report a discrepancy between the sponsor result and the recalculation.

Standardized effect measures can express a mean difference relative to a measure of variability. The posted recalculation gives a pooled standard deviation of 7.23 and a standardized effect of -1.335 for the primary comparison. Such a standardized measure can help compare effects measured on different scales, but it is less directly interpretable than the difference in the endpoint’s original units. Its value also depends on the choice of standard deviation and does not establish clinical importance.

Confidence intervals provide an estimate and a range of uncertainty, subject to the analysis assumptions. The registry does not report the confidence level for the recalculated intervals. A narrow interval generally indicates greater precision than a wide interval, while the location of the interval indicates which values remain compatible with the analysis. An interval does not account for unreported sources of bias or uncertainty, such as unreported missing-data handling or the details of a crossover analysis.

Finally, repeated testing across multiple outcomes raises a multiplicity concern. Testing many outcomes can increase the chance that at least one result appears noteworthy by chance if each is interpreted without adjustment. The record lists 15 analyses, but a multiplicity strategy is not reported in the registry record. The secondary p-values should therefore be read as unadjusted results, not as definitive evidence across the full set of analyses.

11. Confidence Intervals and Precision

The recalculated primary mean difference is -9.65, with a confidence interval from -14.41 to -4.889. This interval describes uncertainty around the unadjusted comparison, conditional on the calculation and its assumptions. The interval’s confidence level is not reported in the registry record, so it should not be labeled with a confidence percentage that the record does not state. The sponsor’s ANCOVA result has no reported estimate or interval, limiting direct assessment of its precision.

In general, a confidence interval gives a range of values that are reasonably compatible with the observed data under a specified method. Its width reflects statistical precision: narrower intervals indicate less sampling uncertainty than wider intervals, all else equal. The interval does not quantify every uncertainty. In particular, it cannot correct for missing data, bias, model misspecification, or incomplete information about the crossover analysis.

The reported primary interval lies below zero, while its endpoints remain separated from zero. This describes the recalculated statistical comparison, not the clinical importance of the effect. A meaningful-change threshold is not reported in the registry record. Nor does the interval tell readers how consistently individual participants changed; the arm standard deviations indicate appreciable variation around the means, and individual outcome distributions are not reported.

The differing primary endpoint counts also matter when judging precision. The registry gives n = 29 for pasireotide LAR and n = 12 for placebo, against enrollment of 48, but does not explain the analysis-population difference. Without that explanation, it is not possible to determine whether the available summaries reflect all eligible measurements or how the excluded or missing observations may affect the estimate. The confidence interval captures sampling uncertainty under the recalculation, not this unresolved reporting uncertainty.

12. Planning a Similar Trial: Sample Size

This is a planning illustration, not a re-evaluation of the original trial. It asks: if a new study wanted to detect a difference like the one observed, or a smaller one, how large would it need to be? It is deliberately not “observed power” of the finished trial, which is not a meaningful quantity.

The planning figures are illustrative calculations based on the observed effect, not the trial’s original design assumptions. For the continuous outcomes in this record, the figures are participants per arm, not a total across both arms or a count of events. The planning assumptions listed are a two-sided significance level of 0.05, equal allocation between the two arms, and a normal approximation. Each scenario uses the stated fraction of the observed effect as the effect to detect. The original trial’s own sample-size assumptions are not reported in the registry record.

The 80% power column indicates the planned participants per arm under the stated assumptions for a design with 80% power to detect the chosen effect. The 90% power column gives the corresponding per-arm count for 90% power. Power is a design property calculated under specified assumptions; it is not a measure of whether the observed trial result is true or important. These figures are planning illustrations, not observed power.

Rows that plan for a smaller fraction of the observed effect require more participants per arm than rows planning for the full observed effect. This is because smaller differences are generally harder to distinguish from random variation under the same assumptions. The figures also depend on the planning assumptions and should not be treated as sample-size recommendations for a future study without considering its own endpoint, design, variability, and analysis plan.

The planning calculations use the observed effect as their reference, so they are sensitive to the uncertainty in that observed estimate. In a small or imbalanced dataset, an observed effect may not be a stable estimate of the effect a future study should target. These calculations are best read as scenario illustrations of how required sample size changes with the target effect and desired power, rather than as evidence that the trial was adequately or inadequately designed.

13. How the Sample Size Is Derived

Power is the probability that a study of a given size rejects the null hypothesis when a specific true effect exists. It depends on four things: the effect to detect, the variability of the outcome, the significance level (here a two-sided α of 0.05, so z_1−α/2 = 1.960) and the desired power (z_1−β = 0.842 for 80% and 1.282 for 90%).

Formula

n is the number of participants in each arm, Δ is the difference in means to detect and σ is the common standard deviation.

Worked calculation for this trial

1. Observed difference Δ = -9.650; pooled standard deviation σ = 7.230.
2. n = 2 × (1.96 + 0.842)² × 7.230² / -9.650² = 8.8, rounded up to 9 participants per arm for 80% power.

The smaller-effect rows in the table above repeat the same calculation with the effect multiplied by 0.75 and 0.5 (for ratios, applied to the logarithm of the ratio).

R code

alpha <- 0.05
z_a   <- qnorm(1 - alpha / 2)          # two-sided
delta <- -9.65                      # observed difference in means
sigma <- 7.23027661988115                     # pooled standard deviation
n_per_arm <- function(power, frac = 1) {
  ceiling(round(2 * (z_a + qnorm(power))^2 * sigma^2 / (delta * frac)^2, 6))
}
for (f in c(1, 0.75, 0.5))
  cat(sprintf("%3.0f%% of observed effect: n per arm = %s (80%% power), %s (90%% power)\n", 100 * f,
              format(n_per_arm(0.80, f), big.mark = ","), format(n_per_arm(0.90, f), big.mark = ",")))

# Check against R's built-in function (t-based, so very slightly larger)
cat("power.t.test:", round(power.t.test(delta = delta, sd = sigma, power = 0.80)$n, 1), "\n")

# Simulation: exact two-sample t-test using simulated means and variances
set.seed(1670110)
n    <- n_per_arm(0.80)
reps <- 20000
m_diff <- rnorm(reps, delta, sigma * sqrt(2 / n))             # difference in sample means
s2     <- sigma^2 * rchisq(reps, 2 * (n - 1)) / (2 * (n - 1))   # pooled sample variance
tstat  <- m_diff / sqrt(s2 * 2 / n)
cat(sprintf("Simulated power at n = %s per arm: %.3f (%d replicates)\n", format(n, big.mark = ","), mean(abs(tstat) > qt(0.975, 2 * (n - 1))), reps))

Output from running the code

100% of observed effect: n per arm = 9 (80% power), 12 (90% power)
 75% of observed effect: n per arm = 16 (80% power), 21 (90% power)
 50% of observed effect: n per arm = 36 (80% power), 48 (90% power)
power.t.test: 9.9 
Simulated power at n = 9 per arm: 0.755 (20000 replicates)

The simulation draws many sets of group means and variances at the planned sample size and counts how often the two-sample t-test rejects the null. It should land close to the target power; at very small required sample sizes it can fall below it, because the normal approximation is rough there.

14. Analyses Reported by the Sponsor

The registry reports that the sponsor analyzed the primary comparison using ANCOVA and gives p = 0.001. It does not report the sponsor’s adjusted mean difference, confidence interval, covariates, or fuller model specification. As a result, the magnitude and precision of the sponsor’s adjusted estimate cannot be compared directly with the recalculated unadjusted estimate.

The recalculation uses the posted arm means, standard deviations, and participant counts. It gives a mean difference of -9.65, a confidence interval from -14.41 to -4.889, and p < 0.001. This is a simple unadjusted comparison. It does not reproduce ANCOVA or incorporate participant-level information that may be relevant to a crossover design. The two analyses should therefore be described separately rather than treated as interchangeable.

The reported p-values are close in their broad statistical implication, but they are reported with different precision and come from different methods. The registry does not identify a discrepancy between them. In the absence of the sponsor’s adjusted estimate and model details, the posted ANCOVA result is evidence about the sponsor’s specified analysis as recorded, while the recalculation provides a check based on the published summaries. Neither resolves the unreported analysis-population, missing-data, or crossover-method details.

15. Multiplicity, Missing Data and Analysis Populations

The record lists 8 outcome measures and 15 analyses, including the primary liver-volume outcome and multiple secondary outcomes. When multiple outcomes are tested, the chance of at least one apparently unusual result can increase if each test is evaluated separately at its nominal threshold. This is the general statistical issue of multiplicity. Whether and how it should be addressed depends on the analysis plan and the role assigned to each outcome.

A multiplicity adjustment or prespecified testing hierarchy is not reported in the registry record. The posted p-values should therefore be read as reported for their individual analyses, without assuming that they were adjusted across outcomes. This is especially relevant when interpreting the secondary results. The absence of a reported multiplicity procedure does not prove that none was used, but the record does not document one.

The registry provides unequal primary outcome counts by arm and a total enrollment figure, but does not state why the counts differ or describe the analysis population. The approach to missing data, the number of participants with missing measurements, and any sensitivity analyses are not reported in the registry record. Those omissions limit assessment of whether missingness could have influenced the group comparison.

The record identifies a crossover model but does not report treatment sequences, period structure, washout, or how participant-level pairing was handled in the primary analysis. It also does not report the ANCOVA covariates or other model details. These items matter for interpreting a crossover comparison because treatment, period, and sequence information can affect the analysis. Their absence should be treated as uncertainty about the reported methods, not as proof that the design was mishandled.

The primary endpoint’s confidence level and clinically meaningful threshold are also not reported. Together, these gaps mean that the statistical evidence can be described using the recorded summaries and analysis label, but a full evaluation of prespecification, missing-data robustness, and multiplicity control is not possible from the registry record.

16. 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: abdominal hernia repair (6.7%), ruptured liver cyst (3.0%), elevated alkaline phosphatase (3.0%), prolonged qt>480msec (3.0%), right upper quadrant pain (3.0%).

The registry’s adverse-event summaries list 33 participants at risk for serious adverse events in the pasireotide LAR group, with 4 affected, and 15 at risk in the placebo group, with 1 affected. The record lists 0 deaths in the pasireotide LAR group and 1 in the placebo group. These are counts in the safety summaries; the record does not provide enough context to establish causation or compare risk reliably.

For other adverse events, the registry lists 33 at risk and 33 affected with pasireotide LAR, and 15 at risk and 15 affected with placebo. The record also lists serious-event terms and percentages, including abdominal hernia repair at 6.7%, ruptured liver cyst at 3.0%, elevated alkaline phosphatase at 3.0%, prolonged QT>480msec at 3.0%, right upper quadrant pain at 3.0%, and abdominal pain at 3.0%. The registry does not assign these listed terms to treatment groups in the information summarized.

Safety counts are descriptive, and the arm counts at risk differ. The registry does not report event timing, exposure duration, participant-level overlap among events, or judgments about relatedness. These summaries do not by themselves establish whether an event was caused by treatment or allow a complete comparative safety assessment.

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

The posted primary results describe a difference in mean percentage change in liver volume between the pasireotide LAR and placebo groups over the baseline-to-12-month timeframe. The recalculated unadjusted difference is -9.65, and its reported confidence interval is from -14.41 to -4.889. The sponsor’s ANCOVA analysis is reported with p = 0.001. These results characterize the statistical comparisons recorded; they are not sufficient by themselves to determine whether the difference is meaningful to patients.

The measured endpoint is liver-volume change, not a direct measure of symptoms, daily functioning, complications, or quality of life. The study summary describes quality of life as an aim, and the registry lists it as a secondary outcome, but numerical quality-of-life results are not reported in the registry record. The posted primary finding therefore cannot be used to infer a quality-of-life effect.

Nor can the primary result establish effects on every condition listed in the registry or on outcomes that were not numerically reported. The record names several secondary measures, but results are available for only some of them. The safety summaries provide event counts and selected terms, yet do not include enough information to establish causation or comprehensively compare harms.

The crossover model and the difference between enrollment and primary endpoint counts add uncertainty to interpretation because the registry does not report the relevant sequence, missing-data, or analysis-population details. The result should be understood as a comparison in the posted data, interpreted alongside those reporting limitations. It should not be extended beyond the trial’s recorded population, timeframe, interventions, and measured outcomes.

In statistical terms, a small p-value is evidence against a specified null comparison under an analysis, not a guarantee of reproducibility, clinical importance, or a particular outcome for an individual. The confidence interval expresses uncertainty around the recalculated estimate but does not capture all design and reporting uncertainties. A complete judgment would require the missing analysis details and broader clinical context, neither of which is posted by the registry record.

18. Why This Trial Matters Statistically

This trial is statistically notable for pairing a randomized, double-masked design with a crossover model and a continuous primary outcome. Those features make the analysis choices important: a group-level comparison of means and a crossover analysis using within-participant information may rely on different information. The registry names the crossover model and reports an ANCOVA primary analysis, but does not explain how the crossover structure entered the analysis.

The record also makes clear why estimates, uncertainty, and analysis populations should be considered together. The posted primary means are based on different arm counts, while enrollment is 48. Without an explanation of the difference or a missing-data account, the reported estimate cannot be evaluated fully for robustness. A confidence interval and p-value describe sampling evidence under a calculation; they do not substitute for transparent reporting of who contributed data and how.

The secondary outcomes illustrate the distinction between a prespecified primary question and a broader set of exploratory or supportive analyses. The registry lists multiple outcomes and analyses, but does not report a multiplicity strategy. A reader should not give every nominal p-value the same confirmatory weight when the relationship among outcomes and the testing plan are unclear.

Finally, the available summaries permit a simple unadjusted recalculation alongside the sponsor’s reported ANCOVA p-value. This comparison helps readers understand what can be reconstructed from arm summaries and what remains dependent on unreported model details. The trial is a useful example of why statistical interpretation includes not only a result, but also design structure, uncertainty, and the limits of the registry record.

19. 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 specific to interpreting this trial is that the registry identifies a crossover model but does not report treatment sequences, period structure, washout, or crossover-specific analysis details. These omissions prevent readers from assessing how within-participant comparisons, period effects, or possible carryover were handled. They do not establish that such issues were present, only that the record does not resolve them.

The primary endpoint summaries include 29 participants in the pasireotide LAR arm and 12 in the placebo arm, while enrollment is 48. The reason for the different counts is not reported in the registry record. The analysis population, participant flow for this endpoint, and missing-data method are not reported either. This limits assessment of selection and missing-data effects.

The sponsor’s primary analysis is identified as ANCOVA, but the adjusted estimate, interval, covariates, and model specification are absent. The recalculation is unadjusted and cannot reproduce that analysis. The registry also does not report the confidence level for the recalculated interval or a clinically meaningful threshold for the endpoint.

Only three secondary outcomes have numerical summaries in the record, while six secondary outcome titles are listed. A multiplicity plan is not reported despite the listed analyses. The quality-of-life outcome and other listed measures cannot be interpreted numerically from the registry record. Finally, the safety summaries do not report event timing, exposure duration, relatedness, or participant-level overlap, limiting comparative safety interpretation.

20. Related Tutorials

21. Related Statistical Calculators

22. Sources

• ClinicalTrials.gov: Pasireotide LAR in Severe Polycystic Liver Disease — NCT01670110.
• Registry record last updated 2020-05-01; status last verified 2020-04.
• Results first posted 2020-03-02.
• 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.

23. Record Summary

NCT01670110 is a completed Phase 2 randomized, double-masked, placebo-controlled trial with a crossover model, sponsored by Mayo Clinic. The registry lists enrollment of 48 and a primary outcome of mean change in liver volume from baseline to 12 month, expressed as percentage of change.

The posted primary summaries report mean changes of -3.36 for pasireotide LAR and 6.29 for placebo. The recalculated unadjusted mean difference is -9.65, with a confidence interval from -14.41 to -4.889 and p < 0.001. The sponsor reports an ANCOVA analysis with p = 0.001. The record does not report the adjusted estimate or details of the model.

Important interpretation details—including crossover analysis, the primary analysis population, missing-data handling, and multiplicity procedures—are not reported in the registry record. The secondary and safety summaries are informative but incomplete, and should be read as descriptive registry results rather than a complete account of trial methods or outcomes.