This page provides an independent statistical analysis and educational interpretation of publicly reported results. ClinicalTrials.gov provides the official trial registry record. Numerical trial results on this page are restricted to the results posted in the ClinicalTrials.gov record; calculations added by Clinical Biostats are labelled as such.
1. Trial at a Glance
This page covers the registry results for Study of Genistein in Reducing Side Effects of Superficial Bladder Cancer Treatment (NCT01489813), a randomized phase 2 study. The primary outcome is Change in Severity of Urinary Symptoms as Determined by the The International Prostate Symptom Score (IPSS) Questionnaire Score..
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01489813 |
| Phase | Phase 2 |
| Condition | Bladder Cancer |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | Single |
| Enrollment | 36 |
| Arms in the primary outcome | 2 |
| Lead sponsor | Emory University |
| Results posted | Yes |
2. Clinical Question
Population
The registry describes patients with non-invasive bladder cancer receiving intravesical therapy. The clinical motivation is that urinary symptoms associated with intravesical treatment may limit the dose patients can receive. The record does not report detailed eligibility criteria or further characteristics of the enrolled participants, so the findings cannot be assigned to a more narrowly defined patient group from the registry information alone.
Intervention
Patients in the genistein arm were assigned a 30 mg genistein supplement by mouth three times daily for 10 weeks. The study description frames genistein as a possible way to address treatment-related lower urinary tract symptoms while patients receive intravesical therapy.
Comparator
The comparison group received placebo pills for 10 weeks. The registry describes the placebo as a sugar pill that looks like the genistein pill. The trial was randomized and single-masked; which people involved in the trial were masked is not reported in the registry record.
Primary question
The primary question was whether urinary symptom severity changed differently with genistein than with placebo. Severity was measured using the International Prostate Symptom Score (IPSS), comparing baseline with the assessment after 6 weeks of treatment. The registry states that lower values indicate a better outcome. Cancer recurrence determined at biopsy at 10 weeks was a secondary question, rather than the primary endpoint.
3. Trial Design
This was a randomized, placebo-controlled, phase II trial with a parallel-group design. The registry lists an enrollment of 36 and marks the study as completed. The study start date is 2017-05-19, and the primary completion date is 2023-08-31. Those dates describe the registry record; they do not provide further detail about the timing of individual participants’ follow-up.
The two arms were genistein supplement and sugar pill. The genistein regimen was 30 mg by mouth three times daily for 10 weeks; the placebo arm received pills for 10 weeks. The record identifies the study as single-masked, but does not state whether participants, clinicians, outcome assessors, or another group were masked. The precise method used to generate the random allocation sequence and the method used to conceal assignments are not reported in the registry record.
The primary outcome was change in IPSS from baseline to after 6 weeks of treatment. The reported summaries are medians and full ranges. The registry reports a median change of -2, with a full range from -3 to 2, in the sugar pill arm, and a median change of -3, with a full range from -4 to 2, in the genistein arm. The primary outcome summaries use 13 participants in the sugar pill arm and 17 in the genistein arm.
The secondary outcome was the number of participants with cancer recurrence determined at a 10-week biopsy. Its posted arm counts use 14 participants in the sugar pill group and 18 in the genistein group. Thus, the reported participant counts differ between the primary and secondary outcome summaries. Reasons for the differences, and details about the analysis populations, are not reported in the registry record. The record does not describe a sample-size justification or an interim-analysis plan.
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
| Stage | Sugar Pill | Genistein Supplement |
|---|---|---|
| STARTED | 16 | 20 |
| COMPLETED | 13 | 17 |
| NOT COMPLETED | 3 | 3 |
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.
| Characteristic | Unit | Sugar Pill | Genistein Supplement | Total |
|---|---|---|---|---|
| Participants analysed | 16 | 20 | 36 | |
| Age, Continuous | years | 73.2 (SD 8.3) | 71.2 (SD 8.7) | 71.5 (SD 8.4) |
| Sex: Female, Male: Female | Participants | 1 | 3 | 4 |
| Sex: Female, Male: Male | Participants | 15 | 17 | 32 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 3 | 7 | 10 |
| Race (NIH/OMB): White | Participants | 12 | 11 | 23 |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 1 | 2 | 3 |
| Region of Enrollment: United States | participants | 16 | 20 | 36 |
| Number of Participants with Insurance | Participants | 13 | 15 | 28 |
6. Primary Endpoint
The registry lists the primary outcome as Change in Severity of Urinary Symptoms as Determined by the The International Prostate Symptom Score (IPSS) Questionnaire Score., measured over At baseline and after 6 weeks of treatment.
| Element | Registry entry |
|---|---|
| Result type | Median |
| Measure of spread | Full Range |
| Unit | score on a scale |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint measures change in urinary symptom severity using the International Prostate Symptom Score (IPSS) questionnaire. The registry specifies a comparison of scores at baseline with scores after 6 weeks of treatment, and reports change summaries as medians with full ranges. It also states that lower values indicate a better outcome. The posted medians are -2 for sugar pill and -3 for genistein; the corresponding full ranges are -3 to 2 and -4 to 2.
The IPSS is used to quantify urinary symptoms rather than relying only on a general impression of whether symptoms improved. The registry describes score categories as mild symptoms from 0 to 7 points, moderate symptoms from 8 to 19 points, and severe symptoms from 20 to 35 points. The posted primary result, however, summarizes changes in score, not the distribution of participants across those symptom categories.
These summaries alone do not establish how precisely the arm outcomes differ. The registry record does not report a comparative effect estimate, confidence interval, or p value for the primary endpoint. It also does not give participant-level scores or explain how missing questionnaire data, if any, were handled. The medians and ranges describe the recorded changes, but do not provide a formal estimate of the uncertainty around the difference between treatment groups.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The primary endpoint summaries show a median change of -2 in the sugar pill arm and -3 in the genistein arm. The full range was -3 to 2 for sugar pill and -4 to 2 for genistein. Because the registry states that lower values indicate a better outcome, the genistein arm’s median change is numerically lower. The ranges overlap, and both extend to positive values. A median is a summary of the middle of an outcome distribution; it does not show how many participants improved, stayed the same, or worsened. The ranges describe the observed minimum and maximum, not the precision of an arm comparison.
The registry record does not report a formal between-arm estimate or statistical test for the primary outcome. It is therefore not possible to quantify the uncertainty of the primary comparison from the posted summaries, or to determine whether a prespecified inferential criterion was met. The reported medians should be read as descriptive statistics, not as proof that the groups differ. Likewise, the numerical ordering of the medians alone does not establish a treatment effect.
For the secondary recurrence outcome, the registry reports 7 participants with recurrence among 14 in the sugar pill arm and 10 among 18 in the genistein arm. The registry’s risk-difference estimate is 0.056, with a confidence interval from -0.293 to 0.404. This interval includes zero and also spans values in either direction. In general, a confidence interval indicates a range of effect estimates compatible with the observed data under the statistical method used; its width conveys uncertainty. Here, the interval is broad, so the posted comparison is compatible with appreciably different underlying risk differences.
The unadjusted p value for this recurrence comparison is 0.755. A p value measures how unusual data at least as extreme as those observed would be under a specified null model; it is not the probability that the null hypothesis is true, nor does it measure clinical importance. This unadjusted result does not provide evidence of a clear separation between the groups, but it also does not demonstrate that the groups have identical recurrence outcomes. The risk-ratio and odds-ratio estimates likewise have broad intervals: the posted risk ratio is 1.111, with an interval from 0.570 to 2.165, and the posted odds ratio is 1.25, with an interval from 0.308 to 5.072.
These endpoints address different questions and are assessed at different times: the primary symptom outcome is measured after 6 weeks, while recurrence is determined at a 10-week biopsy. The recurrence findings cannot substitute for the unreported inferential comparison of the primary symptom outcome. Taken together, the posted results provide descriptive primary summaries and an imprecise secondary comparison. They do not establish a definitive difference, or a definitive absence of difference, between the treatment groups.
9. Statistical Methods Explained
The trial is identified as randomized, single-masked, and parallel-group. Randomization is intended to make treatment assignment independent of measured and unmeasured participant characteristics on average. It does not guarantee that groups will be identical, especially in a small trial, and it does not by itself address missing outcomes or analysis choices. A parallel design assigns participants to one arm for the treatment comparison rather than having each participant receive both interventions. The details of random sequence generation and allocation concealment are not reported in the registry record.
What do the primary summaries describe?
The primary outcome is a change in IPSS score between baseline and the assessment after 6 weeks of treatment. The registry reports a median and full range for each arm. The median is the middle value when observations are ordered, so it is less influenced by extreme observations than the mean. A full range gives the lowest and highest observed values, but does not show how values are distributed between those endpoints. Neither summary alone quantifies the uncertainty in a difference between arms.
The outcome is a change score, and the registry states that lower values indicate a better outcome. The arm medians are -2 for sugar pill and -3 for genistein. The posted ranges are -3 to 2 and -4 to 2, respectively. These summaries should not be interpreted as a formal test of treatment effect: no comparative confidence interval or p value for this endpoint is reported in the registry record. The record also does not identify a particular statistical test or model used for the primary endpoint.
How should the recurrence comparison be read?
Recurrence is recorded as a binary outcome at the 10-week biopsy: a participant either is or is not counted as having recurrence for this endpoint. The registry posts counts of 7 among 14 for sugar pill and 10 among 18 for genistein. It also posts an unadjusted risk difference of 0.056, with a confidence interval from -0.293 to 0.404, and an unadjusted p value of 0.755. The risk difference expresses the comparison on an absolute probability scale. A value near zero represents little difference on that scale, while positive and negative values represent opposite directions according to the comparison’s arm ordering.
A confidence interval conveys the statistical uncertainty around an estimate under the method used. When an interval is broad, a range of possible underlying effects remains compatible with the data. The interval for this risk difference crosses zero, so the posted estimate does not precisely distinguish no difference from effects in either direction. The record also reports a risk ratio of 1.111, with a confidence interval from 0.570 to 2.165, and an odds ratio of 1.25, with a confidence interval from 0.308 to 5.072. Risk ratios compare probabilities; odds ratios compare odds. The numerical scales differ, so an estimate on one scale should not be read as interchangeable with an estimate on another.
The p value is unadjusted. In general, it is calculated under a null model and describes how compatible the observed result is with that model, considering results at least as extreme. It does not give the probability that the treatment has no effect, and it is not a measure of how large or important an effect is. Since the registry does not report adjusted analyses for this endpoint, the posted p value should be read as an unadjusted result.
What do the planning figures mean?
The registry includes sample-size planning illustrations based on the observed recurrence effect. It states assumptions of a two-sided significance level of 0.05, equal allocation between arms, and a normal approximation. The figures are planning illustrations, not a description of the trial’s actual design rationale and not observed power. The record does not report that these assumptions were used to set the trial’s enrollment target.
For planning for 100% of the observed effect, the listed requirement is 1,267 participants per arm for 80% power and 1,695 participants per arm for 90% power. For planning for 75% of the observed effect, the corresponding figures are 2,256 and 3,019 participants per arm. For planning for 50% of the observed effect, they are 5,081 and 6,802 participants per arm. These are hypothetical sample-size illustrations, not estimates of the probability that this completed trial detected an effect. Planning from an observed effect can also be sensitive to the uncertainty in that effect estimate, which is particularly relevant when the estimate comes from a small dataset.
In general, over-dispersion means that observed outcomes vary more than a model assumes. If a statistical model does not account for this extra variability, its uncertainty estimates can be too small. The registry record does not report a model for over-dispersion, nor does the binary recurrence outcome itself establish that such a model was used. No additional statistical model details should be inferred from the posted estimates.
What analytical details are unavailable?
The registry lists no reported analyses for the secondary outcome and no reported analysis count for the posted results. It does not describe a statistical analysis plan, covariate adjustment, handling of repeated measurements, or a method for missing outcomes. The available information also does not state whether all randomized participants were analyzed in their assigned arms, whether a per-protocol population was used, or whether any participants were excluded from the reported summaries. Such details can affect the interpretation of estimates, but cannot be reconstructed from arm summaries alone.
10. Multiplicity, Missing Data and Analysis Populations
Multiplicity arises when several outcomes, time points, subgroups, or statistical tests are examined. In general, the more opportunities there are to find an apparently unusual result, the greater the chance that at least one result will appear noteworthy by chance alone. Statistical plans may address this through a prespecified hierarchy, adjusted significance thresholds, or other approaches. The registry record does not report whether any such strategy was planned or used.
The registry identifies a primary urinary symptom endpoint and a secondary recurrence endpoint. It lists no reported analyses for recurrence and no analysis count for the posted results. The record does not state whether additional outcomes, subgroup analyses, or alternative models were considered, nor does it describe a prespecified order for interpreting endpoints. The unadjusted recurrence p value should therefore not be treated as though it were known to have been protected against multiplicity. Conversely, the absence of a reported adjustment does not establish that no adjustment was conducted outside the registry record.
The primary outcome summaries include 13 participants in the sugar pill arm and 17 in the genistein arm, while the recurrence summaries include 14 and 18. The registry does not explain the difference in these reported counts. It does not report whether the distinction reflects eligibility for a particular assessment, missing measurements, exclusions, or another reason. It also does not specify an intention-to-treat, per-protocol, or other analysis population. These details matter because the population included in an analysis defines what comparison the reported estimate represents.
Missing-data methods are not reported in the registry record. There is no description of the amount or pattern of missing IPSS or biopsy data, any imputation method, or sensitivity analyses for missing outcomes. The registry also does not report how participants without an observed biopsy result were handled in the recurrence analysis. Without this information, it is not possible to assess whether missingness could have affected the results or to determine how robust the estimates are to alternative assumptions.
The record does not report interim analyses, stopping rules, or a sample-size justification. Those omissions do not establish that such procedures were absent; they mean that they cannot be evaluated from the registry information. The posted outcomes and their analysis populations should therefore be interpreted with these uncertainties about multiplicity, missingness, and analysis strategy in mind.
11. Safety Results
The registry reports serious adverse events as affected participants divided by participants at risk.
| Arm | Participants with serious adverse events | Participants at risk | Percentage |
|---|---|---|---|
| Sugar Pill | 0 | 16 | 0.0% |
| Genistein Supplement | 0 | 20 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Sugar Pill | 0 of 16 |
| Genistein Supplement | 0 of 20 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 5% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Sugar Pill | 3 of 16 |
| Genistein Supplement | 3 of 20 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Sugar Pill | Genistein Supplement |
|---|---|---|---|
| Hospitalization | Infections and infestations | 3 of 16 | 3 of 20 |
| UTI | Renal and urinary disorders | 1 of 16 | 1 of 20 |
| Kidney infection | Renal and urinary disorders | 0 of 16 | 1 of 20 |
| infection after urological surgical procedure | Surgical and medical procedures | 1 of 16 | 0 of 20 |
| coronovirus | Infections and infestations | 0 of 16 | 1 of 20 |
The registry reports no serious adverse events among 16 participants at risk in the sugar pill group and none among 20 participants at risk in the genistein group. It reports 3 participants with other, non-serious adverse events in each group, with 16 and 20 at risk, respectively. No deaths are reported in either group. These are counts, not evidence that an adverse event could not occur.
Safety counts require attention to the number of participants at risk and to how events were collected and classified. The registry record does not give further descriptions of the non-serious events, their timing, their relationship to study treatment, or whether a participant could have experienced more than one event. It also does not report event-level detail or follow-up completeness for safety outcomes. The figures therefore describe what is posted, but do not establish that the groups had equivalent safety profiles or that the study could detect uncommon harms.
The adverse-event counts concern a different question from the primary IPSS endpoint and the secondary recurrence endpoint. They should be read as descriptive safety information from this trial, rather than as a definitive comparison of harms.
12. What the Result Does, and Does Not, Mean
The posted primary results indicate that the median change in urinary symptom score was -2 in the sugar pill arm and -3 in the genistein arm. The registry states that lower values indicate a better outcome. The full ranges overlap, and the registry record does not report a comparative estimate or inferential test for this endpoint. The data therefore describe the observed arm summaries but do not establish how precisely the groups differed.
The secondary recurrence result is also uncertain. The reported risk-difference interval, from -0.293 to 0.404, includes zero and spans effects in both directions. The unadjusted p value is 0.755. These statistics do not demonstrate that the groups are identical, nor do they establish a clear difference. They show that the posted recurrence data do not precisely determine the direction or size of the underlying contrast.
These findings should not be generalized beyond what the registry reports. The study is a phase II randomized placebo-controlled trial, and the registry lists enrollment as 36. The primary summaries and recurrence counts use different participant numbers, and the reasons are not reported. Detailed eligibility criteria, participant characteristics, analysis populations, and missing-data procedures are also not reported in the registry record. Those gaps limit assessment of how broadly the results apply and whether the observed summaries represent all randomized participants.
The record’s sample-size illustrations are planning calculations based on the observed recurrence effect. They are not observed power, do not show that the completed trial had a particular probability of detecting an effect, and do not supply a design justification. Likewise, the adverse-event counts are descriptive and do not rule out uncommon events or establish comparable safety.
Overall, the results are best understood as limited descriptive evidence with substantial uncertainty, particularly for the recurrence comparison and for any formal claim about the primary endpoint. They neither establish a treatment benefit nor demonstrate that no effect exists. A reader should distinguish the observed summaries from conclusions that require a reported inferential plan, sufficient precision, and clearly defined analysis populations.
13. Why This Trial Matters Statistically
This trial raises a useful statistical question about how to interpret a small randomized study when the posted outcome summaries are incomplete for formal comparison. The study addresses urinary symptoms during intravesical treatment and also records recurrence at biopsy. These outcomes are clinically distinct and measured at different times, so a single result cannot stand in for the other.
The primary summaries illustrate why effect direction and uncertainty need to be considered separately. The genistein arm has a numerically lower median change, and the registry states that lower values indicate a better outcome. But the full ranges overlap, and no comparative estimate or interval is reported for the primary outcome. A difference between two descriptive summaries is not, by itself, a quantified treatment effect.
The recurrence result illustrates the importance of precision. Its confidence interval includes zero and allows a wide range of possible differences. A nonsignificant unadjusted p value should not be translated into proof of equivalence; equally, an estimate that points in one direction should not be treated as proof of benefit. The central question is not simply whether a threshold is crossed, but how much uncertainty remains around the effect estimate.
The record also shows why analysis populations and missing-data reporting matter. The posted participant counts differ between the symptom and recurrence outcomes, but the reasons are not described. Without that context, readers cannot determine how the analyzed groups relate to the randomized groups or how missing outcomes may affect interpretation. The registry does not report multiplicity handling or an analysis plan, which limits evaluation of how the outcomes were selected and interpreted.
Finally, planning illustrations based on an observed effect are not a substitute for a prospective sample-size rationale. They are useful only as conditional planning exercises under stated assumptions. Keeping planning estimates separate from trial design and observed results prevents a retrospective calculation from being mistaken for evidence that the trial was powered for a particular finding.
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 registry record leaves important trial details unspecified. It identifies randomization and single masking, but does not describe the sequence-generation process, allocation concealment, or which study personnel were masked. Detailed eligibility criteria and participant characteristics are not reported, limiting assessment of how representative the enrolled group may have been.
The main interpretive limitation is that the primary endpoint is presented descriptively. The record gives arm medians and full ranges but no formal comparative estimate, confidence interval, or p value. The sample counts for the primary outcome differ from those for recurrence, and the registry does not explain why. Without an account of missing assessments or analysis populations, it is difficult to assess the completeness of either endpoint.
For recurrence, the estimate is imprecise and the p value is unadjusted. The record does not report a multiplicity strategy, an analysis plan, or whether additional comparisons were considered. It also does not describe how missing biopsy results were handled. These gaps constrain interpretation of the secondary result beyond the uncertainty already conveyed by its interval.
The registry does not report the sample-size justification, interim analyses, stopping rules, or detailed safety-event definitions. It provides counts for serious and other adverse events but not event descriptions, attribution, timing, or completeness of follow-up. Therefore, the posted safety information is limited to the recorded counts and cannot characterize uncommon harms or compare event profiles comprehensively.
These limitations do not establish that unreported procedures were not performed. They indicate that the registry record does not provide enough detail to evaluate them. The results should consequently be read as registry-reported summaries rather than a complete account of every trial procedure or analysis.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Study of Genistein in Reducing Side Effects of Superficial Bladder Cancer Treatment — NCT01489813.
- Registry record last updated 2025-02-05; status last verified 2025-01.
- Results first posted 2024-07-25.
- 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.
Continue through the Clinical Biostats statistical pathway
Use this trial's endpoint as a starting point for deeper study of effect measures, confidence intervals, and sample-size planning.
18. Record Summary
The registry describes a completed, randomized, single-masked, parallel-group phase II trial sponsored by Emory University. The listed enrollment is 36. The study start date is 2017-05-19, and the primary completion date is 2023-08-31.
The primary endpoint is change in IPSS from baseline to after 6 weeks of treatment, reported by arm as a median and full range. The registry posts a secondary outcome of cancer recurrence determined at a 10-week biopsy. It also reports adverse-event counts. A formal comparative analysis for the primary endpoint, details of missing-data handling, and an analysis plan are not reported in the registry record.
The primary outcome summaries are descriptive, while the recurrence comparison is imprecise and unadjusted. The record includes hypothetical sample-size planning illustrations based on the observed recurrence effect; these are not observed power or the trial’s reported design justification. Safety counts are likewise descriptive. Overall, the registry record supports cautious interpretation and does not establish a definitive between-group conclusion.