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 Feasibility of "At-home" Titration of Solifenacin (NCT00759577), a na phase 4 study. The primary outcome is Improvement in Patient Perception of Bladder Condition Score (PPBC).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00759577 |
| Phase | Phase 4 |
| Condition | Overactive Bladder |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 36 |
| Arms in the primary outcome | n/a |
| Lead sponsor | NYU Langone Health |
| Results posted | Yes |
2. Clinical Question
Population
People with overactive bladder in a study described as reflecting typical male and female patients presenting for treatment. The registry lists enrollment of 36; further eligibility details are not reported in the registry record.
Intervention
At-home self-titration of solifenacin, with patients given the drug to adjust between 5 mg and 10 mg.
Comparator
There was no concurrent comparator group. The study is listed as single-group, with no allocation and no masking.
Primary question
Whether patient-perceived bladder condition changed from the start of medication to the end of the 12-week trial, as measured by the Patient Perception of Bladder Condition score.
The intended question was about the feasibility and effects of a self-titration approach in a treatment-seeking overactive bladder population. The outcome was a patient-reported global assessment of current urinary problems. Participants were asked to rate their perceived bladder condition on a scale ranging from 1, “no problem,” to 6, “many severe problems.” The registry defines change as the baseline score subtracted from the end-of-study score, with negative values representing improvement.
This design addresses change over time in the participants who take part, rather than a difference between treatment groups. Without a concurrent control group, any recorded change could not by itself distinguish the effect of medication or titration from other influences over time. In this record, the intended primary outcome cannot answer even that within-group change question: no questionnaire was available for analysis. The registry attributes stopping the study to poor enrollment and says the planned effort to collect questionnaires earlier was not carried out. The clinical question therefore remains unanswered by posted primary-outcome data.
3. Trial Design
The registry describes a Phase 4, open-label, single-center, single-group trial of at-home solifenacin titration for overactive bladder. Its stated focus was the feasibility of self-titration with solifenacin 5 mg and 10 mg, along with symptom bother and quality of life in a typical overactive bladder population. The arm description says patients were given the drug to self-titrate. The record lists enrollment of 36 and identifies NYU Langone Health as the sponsor.
There was no random allocation, masking, or like-for-like control group. The registry’s allocation entry is “NA,” its masking entry is “NONE,” and its model is “SINGLE GROUP.” An open-label, single-group structure can describe how participants’ outcomes change during a study, but it does not provide a randomized comparison that separates treatment effects from other explanations for change. The registry does not report a protocol for how participants selected or changed their dose, or additional details about visit schedules beyond the primary outcome timeframe.
The record lists a start in 2008-09 and primary completion in 2010-05, and the status is “TERMINATED.” It states that poor enrollment led to stopping the study and that efforts to collect questionnaires at an earlier time point were not carried out. The registry does not report a reason for termination beyond poor enrollment, nor does it provide further operational details about recruitment, adherence, or the number who completed follow-up.
Although the record lists one primary outcome measure, it lists no analyses. It also states that there was no questionnaire available for analysis. Consequently, the design and planned measurement can be described, but an observed primary-outcome estimate cannot be interpreted from the registry record. Details such as a formal sample-size justification, interim analyses, and a prespecified statistical analysis plan 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
| Stage | At Home Titration |
|---|---|
| STARTED | 36 |
| COMPLETED | 36 |
| NOT COMPLETED | 0 |
Analysis population for the primary outcome, as defined in the registry: There was no questionnaire available for analysis. Secondary to poor enrollment the study was stopped and efforts to collect questionnaires at an earlier time point were not carried out.
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 | At Home Titration |
|---|---|---|
| Participants analysed | 0 | |
| Age, Categorical: <=18 years | Participants | — |
| Age, Categorical: Between 18 and 65 years | Participants | — |
| Age, Categorical: >=65 years | Participants | — |
| Sex: Female, Male: Female | Participants | — |
| Sex: Female, Male: Male | Participants | — |
6. Primary Endpoint
The registry lists the primary outcome as Improvement in Patient Perception of Bladder Condition Score (PPBC), measured over 12 weeks.
| Element | Registry entry |
|---|---|
| Result type | N/A |
| Measure of spread | not applicable |
| Unit | not stated |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint was change in the Patient Perception of Bladder Condition score from the start of medication to the end of the 12-week trial. This single-item patient-reported global question asks participants to rate their subjective impression of their current urinary problems on a six-point scale, from 1 (“no problem”) to 6 (“many severe problems”). The registry defines the change by subtracting the baseline value from the end-of-study value and states that negative values represent improvement.
A patient-reported global measure can capture a participant’s overall impression rather than relying only on a clinician’s assessment or a count of specific symptoms. In general, this kind of measure may be useful when the research question concerns how a health condition is experienced by the participant. A change score also makes the intended comparison explicit: each participant’s end-of-study rating is considered relative to their own baseline rating.
Those features describe what the endpoint was intended to measure, not what the trial found. The registry says no questionnaire was available for analysis. It therefore contains no analyzed baseline or end-of-study scores and no primary outcome estimate. The endpoint’s definition and timeframe are reported, but its result is not.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central statistical fact is that the registry record lists no analyses and reports that no questionnaire was available for analysis. The primary endpoint is defined, but there is no posted estimate of change in Patient Perception of Bladder Condition score, no measure of uncertainty around such an estimate, and no test result. The record therefore does not establish the direction or size of any change in the primary outcome.
The registry lists enrollment of 36, but that figure does not establish how many participants contributed primary-outcome data. The record attributes termination to poor enrollment and says attempts to obtain questionnaires at an earlier time point were not carried out. The number of participants with complete or partial outcome data is not reported in the registry record. Enrollment and analysis are different stages: a person can be counted in enrollment without having an available endpoint measurement. The missing questionnaire means the intended primary analysis could not be carried out as described in the record.
The planned measure would have been a within-person change over 12 weeks. Even if such a change estimate had been available, the single-group design would not provide a randomized comparison with an untreated or alternative-treatment group. In general, change over time in one group can reflect several influences, including treatment, natural variation, concurrent care, or changes in how participants assess their symptoms. Without a control group, those explanations cannot be separated by a treatment-versus-control contrast. In this trial record, the absence of analyzed outcome data is an additional and decisive limitation: there is no posted change estimate to characterize in the first place.
There is no basis in the registry record for describing statistical precision. A confidence interval would ordinarily express the uncertainty around an estimated effect, but no primary effect estimate or confidence interval is reported. No p-value or analysis details are reported either. The record does not report whether any missing-data method was used; it says the questionnaire was unavailable and the earlier collection effort was not pursued. Thus, there is no completed primary analysis whose assumptions or handling of missingness can be evaluated.
The adverse-event record lists zero participants at risk and zero affected in the group, with no serious adverse-event terms listed. Because the record also lists zero at risk, those entries do not provide an interpretable estimate of adverse events among participants. They should not be read as evidence that no participant experienced an adverse event. The number of participants assessed for safety and the completeness of safety reporting are not reported in the registry record.
Any planning illustrations based on an observed effect should be read as hypothetical planning exercises, not as trial results or observed power. The primary outcome data needed to interpret an observed effect are absent from the record. The sample-size justification for the trial itself is not reported in the registry record. Overall, the posted information establishes the intended design and measurement, but it does not provide evidence about the magnitude, direction, or precision of a primary-outcome result.
9. Statistical Methods Explained
Statistical methods help translate observations into estimates and describe how much uncertainty surrounds those estimates. In this trial, the registry identifies a planned patient-reported outcome and its scoring direction, but it lists no analyses and says no questionnaire was available for analysis. The methods discussion must therefore distinguish the intended measurement from analyses that were actually reported. A statistical method cannot recover an outcome result that is not present in the record.
What does the change score represent?
The endpoint is defined as the baseline Patient Perception of Bladder Condition score subtracted from the end-of-study score. The scale runs from 1, “no problem,” to 6, “many severe problems,” and the timeframe is 12 weeks. Under the registry’s stated sign convention, a negative change represents improvement. This is a within-person comparison: each participant’s later rating is related to their own starting rating. In general, analyzing change scores can provide a direct description of how measurements differ over time for the people with measurements at both time points.
A change score does not, on its own, establish why the measurement changed. In a single-group study, there is no concurrent randomized group to show what would have happened over the same period under another condition. General influences on a before-and-after measurement can include natural changes, other care, and variation in participants’ responses. The registry does not report an analysis that adjusts for such influences, and its design does not include a control-group contrast.
What does a confidence interval communicate?
In general, a confidence interval gives a range of values compatible with an estimate under the assumptions of the statistical method. A narrower interval indicates greater statistical precision than a wider interval, while neither width nor location alone resolves limitations in study design or missing data. A confidence interval for a treatment contrast would also require a clearly defined contrast. No confidence interval or primary effect estimate is reported in this registry record, so there is no interval to interpret for the PPBC outcome.
What can a p-value tell readers?
In general, a p-value evaluates how compatible the observed data are with a specified null model, assuming the analysis model and other assumptions hold. It does not measure the probability that a treatment works, and it does not indicate the practical importance of an estimate. A p-value is meaningful only alongside a defined analysis and the data to which it was applied. The registry lists no analyses and reports no primary-outcome p-value. It does not report a statistical analysis plan or the significance threshold for an outcome analysis.
Why does missing outcome data matter?
Missing outcome measurements reduce the information available to estimate change. In general, analyses based only on participants with observed outcomes describe that observed subset, and the result may not represent all enrolled participants if the availability of outcomes is related to their condition or experience. Other approaches can sometimes address missingness, but they depend on assumptions and on having data suitable for the chosen method. The record does not report a missing-data method, the number of missing questionnaires, or participant-level outcome data. It states that no questionnaire was available for analysis and that earlier collection efforts were not carried out.
The registry says poor enrollment led to termination. This helps explain why the planned assessment was not completed, but it does not establish how the missing outcome information would have looked. The number enrolled is not a substitute for the number analyzed, and the latter is not reported. The record also does not report an analysis population definition, such as which participants would have been included in a primary analysis.
What does a planning illustration mean?
In general, a sample-size calculation explores how many participants might be needed to detect a specified effect under stated assumptions. Such a calculation is a planning exercise, not a summary of what happened in a trial. The registry’s planning assumptions describe a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the target effect expressed relative to an observed effect. The record does not report the trial’s own sample-size justification, and the trial itself is single-group rather than a two-arm randomized comparison. Any such planning illustration should therefore not be confused with the actual design or with observed power. Observed power is not a substitute for an effect estimate and its uncertainty.
The record gives no information about interim analyses, multiplicity procedures, or a prespecified statistical analysis plan. It also gives no estimates that would permit checking model assumptions or comparing alternative analyses. General statistical concepts can clarify what a complete analysis might communicate, but they cannot supply absent measurements. For this trial, the most defensible statistical conclusion is that the primary outcome was not analyzed in the registry record.
10. Multiplicity, Missing Data and Analysis Populations
Multiplicity concerns arise when a study evaluates several outcomes, time points, subgroups, or statistical tests. In general, examining multiple results can increase the chance that at least one appears notable by chance, so a prespecified analysis plan may explain which outcome is primary and how multiple comparisons are handled. The registry lists one primary outcome measure, improvement in Patient Perception of Bladder Condition score, with a 12-week timeframe. It does not report a multiplicity adjustment, a broader testing hierarchy, or a statistical analysis plan. The number of secondary outcomes and their analyses is not reported in the registry record.
Missing data are central to interpreting this record. It states that no questionnaire was available for analysis. It also says that, because of poor enrollment, the study was stopped and efforts to collect questionnaires earlier were not carried out. The record does not report the number of missing questionnaires, the number of participants with observed endpoint data, reasons for missingness by participant, or a method for handling missing outcomes. Without those details and the outcome measurements, neither a complete-case analysis nor a missing-data adjustment can be evaluated.
The analysis population is also not reported in the registry record. Enrollment of 36 does not indicate how many participants would have been included in an endpoint analysis. No distinction among enrolled, treated, followed, or analyzed participants is available in the record. The registry lists zero analyses, so no analyzed population or primary-outcome result can be identified. These gaps limit both reproducibility and assessment of potential bias; they do not establish the value that an analysis would have produced.
11. What the Result Does, and Does Not, Mean
The record establishes that investigators planned an open-label, single-group study of at-home solifenacin self-titration in people with overactive bladder. The intended primary measure was a patient-reported rating of bladder condition at the start of medication and at 12 weeks. The registry also reports that the study was terminated after poor enrollment and that no questionnaire was available for analysis. These registry entries describe the study’s purpose, design, and lack of primary-outcome data.
The record does not establish whether participants’ perceived bladder condition improved, worsened, or stayed the same. There is no reported primary estimate, confidence interval, or p-value. It also does not establish whether the self-titration approach was feasible in practice: although feasibility was part of the study’s stated focus, a feasibility result is not reported. No questionnaire results are available to quantify symptom bother or quality of life.
Even if the registry had included a before-and-after score, the absence of a control group would limit causal interpretation. A within-group change can describe what happened among observed participants, but it cannot by itself show what would have happened without the study intervention. Here, the further absence of analyzed outcome data means that no such change can be described from the posted primary endpoint.
The adverse-event entries list no participants at risk or affected, and no serious adverse-event terms. Since the risk set is recorded as zero, these entries cannot establish that there were no adverse events among participants. The record does not report safety follow-up completeness or an evaluable participant count for those outcomes.
In short, the registry supports conclusions about the planned study and its incomplete outcome reporting, not about treatment benefit, lack of benefit, or safety. The primary clinical question remains unanswered in the posted results.
12. Why This Trial Matters Statistically
This trial illustrates an important distinction between a clinical question and evidence capable of answering it. The registry describes a clearly identifiable intended question: whether a patient-reported measure of bladder condition changed during 12 weeks of at-home self-titration. It also specifies the score scale and the direction of change. Yet a well-defined endpoint is not itself an outcome result. Because no questionnaire was available for analysis, the record cannot provide an estimate of the change it set out to measure.
The study also shows why enrollment and analyzable data should not be treated as interchangeable. The registry lists enrollment of 36, but it does not report the number contributing outcome measurements. Its explanation of termination links poor enrollment to the decision to stop and notes that earlier questionnaire collection was not undertaken. For statistical interpretation, the availability and completeness of outcome data matter directly: without observations, there is no primary effect estimate, and without a reported analysis population, readers cannot determine who was represented in an analysis.
The single-group structure is another useful design lesson. A within-person assessment can track change, but a concurrent comparison is generally needed to distinguish a treatment-related difference from other changes over time. This design limitation would remain even if endpoint measurements were present. In this record, missing outcome data and the lack of a control group are separate issues, each limiting what can be inferred.
Finally, planning calculations and safety entries require context. Planning illustrations do not become evidence of an observed effect, while zero participants listed as at risk do not establish an absence of adverse events. Careful statistical interpretation depends on what was measured, who contributed data, and what comparison the design permits. This record is instructive precisely because it makes the gap between a planned analysis and a reported result visible.
13. Limitations
- The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
- Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.
The most specific limitation is that the planned primary outcome could not be analyzed. The registry explicitly states that no questionnaire was available for analysis. It also reports that poor enrollment led to stopping the study and that efforts to collect questionnaires at an earlier time point were not carried out. Consequently, there is no posted result for the primary endpoint, despite its detailed definition and 12-week timeframe.
The registry lists enrollment of 36 but does not report the number who received medication, completed follow-up, or had outcome measurements. It does not report the extent or pattern of missing data, nor does it describe any method for addressing missingness. These omissions prevent assessment of how well any analyzed participants might represent those enrolled. Since the record lists no analyses, no analysis population can be identified.
The single-group, open-label design has no concurrent comparator and no masking. As a general matter, this limits separation of changes associated with the intervention from other influences, and subjective ratings may be affected by participants knowing their treatment. The record does not report details of the titration process, adherence, concomitant care, or protocol deviations. It also does not report a statistical analysis plan, sample-size justification, or interim-analysis plan.
Safety reporting is difficult to interpret because the adverse-event group lists zero participants at risk and zero affected. The record does not provide a safety-evaluable population or details about safety follow-up. Those entries therefore cannot be taken as a complete account of adverse events. These limitations are specific to the information and design documented in the registry record; they do not determine what unreported participant-level data might have shown.
14. Related Tutorials
15. Related Statistical Calculators
16. Sources
- ClinicalTrials.gov: Feasibility of "At-home" Titration of Solifenacin — NCT00759577.
- Registry record last updated 2015-10-26; status last verified 2015-10.
- Results first posted 2012-05-30.
- 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.
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17. Record Summary
The registry record describes NCT00759577, “Feasibility of ‘At-home’ Titration of Solifenacin,” as a Phase 4, open-label, single-center, single-group study of at-home solifenacin self-titration for overactive bladder. The listed enrollment is 36, and the sponsor is NYU Langone Health. The record lists a start in 2008-09, primary completion in 2010-05, and status “TERMINATED.”
The primary outcome was change in Patient Perception of Bladder Condition score from the start of medication to the end of the 12-week trial. The registry says no questionnaire was available for analysis and lists no analyses. It attributes termination to poor enrollment and says earlier questionnaire collection was not carried out. No primary-outcome result or analysis population is reported in the registry record.