Efficacy and Safety of AQX-1125 in Subjects With Chronic Prostatitis/Chronic Pelvic Pain Syndrome: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Efficacy and Safety of AQX-1125 in Subjects With Chronic Prostatitis/Chronic Pelvic Pain Syndrome (NCT03500159), a randomized phase 2 study. The primary outcome is Change From Baseline to Week 12 in Maximum Daily Pelvic Pain (Mean).

2. Clinical Question

In statistical terms, the intended question was whether the change in the recorded pain score differed between randomized treatment groups over the treatment period. The registry record identifies the outcome and comparison, but it does not report group-level outcome summaries, an estimated contrast, uncertainty around that contrast, or a test of the treatment comparison. As a result, the intended question remains unanswered by the posted primary-outcome data. This is not evidence that the groups had the same outcomes; it means the registry record contains no primary analysis with which to evaluate their outcomes.

3. Trial Design

The registry describes a randomized, parallel-group, placebo-controlled phase 2 trial. Participants were intended to receive either AQX-1125 200 mg or matching placebo. The brief summary says assignment was planned in a 1:1 ratio. The record lists quadruple masking; the official title also describes the trial as double-blind. It does not specify in the material available which particular roles were included in the quadruple masking designation, so those roles are not reported in the registry record.

The planned treatment period was 12 weeks. The brief summary describes a screening period of up to 3 weeks, a 4-week off-drug safety follow-up, an ophthalmic safety follow-up call at 3 months and a visit at 6 months after the last dose, and an overall study duration of about 41 weeks. These are descriptions of the planned study schedule; the registry record does not establish that every participant completed those periods or follow-up procedures.

The trial was planned across approximately 30 centers in the United States and Canada. The registry lists enrollment as 3 and status as terminated. The brief summary describes an intended enrollment of approximately 100 male subjects. The record says the trial was terminated prematurely and that no primary analysis was performed. It does not report why the trial ended, how many participants were randomized to each treatment, or how much treatment and follow-up each participant received.

Random allocation can reduce systematic differences between groups when a trial is conducted and analyzed as planned. Masking can also limit some influences on treatment delivery and outcome assessment. Those design features do not, by themselves, supply evidence about treatment effects. In this record, the absence of a primary analysis prevents the planned randomized comparison from answering the efficacy question.

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: Study was terminated prematurely, no analysis was performed

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 From Baseline to Week 12 in Maximum Daily Pelvic Pain (Mean), measured over 12 Weeks.

The primary endpoint was change from baseline to Week 12 in mean maximum daily pelvic pain. Pain was recorded using a standardized 11-point numeric rating scale through an electronic diary. The registry describes the outcome as a comparison of AQX-1125 200 mg with placebo.

A daily pain score can capture a participant's reported symptom experience over time. Focusing on the maximum daily score asks about the highest pain rating recorded within the day, rather than an average across all ratings. Change from baseline is intended to describe how the measure differs at follow-up from its starting value; comparing that change between randomized groups is the treatment contrast of interest. These are general descriptions of the endpoint's statistical role, not results from this trial.

The registry record does not report the scale's observed baseline or Week 12 values, the amount of change in either group, or a between-group estimate. It also does not provide a confidence interval or a p-value for this outcome. The endpoint's measurement method and timing are specified, but its observed treatment effect and precision cannot be assessed from the posted primary-outcome data.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry record describes a randomized parallel-group trial, but it does not report a statistical analysis of the primary endpoint. General statistical concepts can clarify what such an analysis would ordinarily be intended to accomplish; they should not be mistaken for methods documented as used in this trial.

What does randomization contribute?

In general, randomization assigns participants to treatment groups by chance. Its purpose is to make treatment assignment independent of participant characteristics in expectation, helping support a fair comparison. Randomization does not guarantee that groups will be identical, especially in a small trial. It also does not correct missing outcome data or create an estimate when no analysis is performed. The registry describes random assignment and a planned 1:1 allocation, but does not report the treatment-specific number randomized or the method used to generate or conceal the assignment sequence.

What would a change-score comparison mean?

General explanation: a change score compares a follow-up measurement with a baseline measurement for each participant. A group-level comparison of those changes can describe how the outcome evolved in one assigned group relative to the other. For this trial, the named endpoint was change from baseline to Week 12 in mean maximum daily pelvic pain recorded by electronic diary. The registry does not give the group-level changes or the between-group estimate. It also does not report whether a change-score model, a baseline-adjusted model, or another method was intended or used.

How should uncertainty be expressed?

General explanation: an estimated treatment contrast is more informative when accompanied by an uncertainty measure. A confidence interval indicates the precision of an estimate under the chosen analysis and assumptions. A p-value is a measure tied to a statistical test and its assumptions; it is not the probability that a treatment works or that a conclusion is true. Neither a confidence interval nor a p-value for the primary endpoint is reported in the registry record. Without an estimate and its uncertainty, readers cannot assess the range of effects compatible with the trial's analyzed data.

Why do sample size and precision matter?

General explanation: the amount of information available affects how precisely a treatment contrast can be estimated. Small amounts of outcome information generally leave greater uncertainty, while a planned enrollment is not the same as the number with usable endpoint data. The registry lists enrollment as 3, and its brief summary describes an intended enrollment of approximately 100. It does not report the number randomized to each arm, the amount of endpoint data available, or a completed efficacy analysis. Thus, the planned enrollment cannot be treated as the analyzed sample size.

The record also describes planning illustrations based on an observed effect, with a two-sided significance level of 0.05, equal allocation, and a normal approximation. Such illustrations are not observed power and are not evidence that an effect was detected. Because the primary outcome analysis was not performed, the registry record does not provide the observed treatment effect needed to interpret an effect-based planning illustration as a trial result. The record does not report the trial's own sample-size justification or statistical planning assumptions.

What do masking and the parallel design imply?

General explanation: in a parallel-group design, participants remain assigned to one treatment group for the comparison, rather than crossing between treatments. Masking is intended to limit the influence of knowledge about assignment on participants, investigators, or other roles. The registry lists quadruple masking and the official title calls the study double-blind, but does not clarify the roles covered by the masking label. These design features describe intended safeguards; they do not show that masking was maintained or that a treatment effect was present.

Can the analysis plan be reconstructed?

No. The record reports 0 analyses and says no primary analysis was performed. It does not report the statistical model, covariate adjustments, handling of repeated diary measurements, treatment of protocol deviations, or prespecified estimand. An estimand is the precise treatment effect a study seeks to quantify, including the population, outcome, treatment conditions, and handling of events that affect interpretation. General explanation of these concepts cannot establish what the investigators planned or did. The appropriate reading is that the outcome and comparison are named, but the analysis details and results are not reported in the registry record.

10. Multiplicity, Missing Data and Analysis Populations

The registry lists 15 outcome measures, including a primary outcome and several secondary outcomes. When a study evaluates multiple outcomes or multiple comparisons, the chance of obtaining at least one apparently notable result by chance can increase if each is tested separately without an appropriate plan. General statistical methods for addressing multiplicity can include prioritizing a primary endpoint, prespecifying a testing sequence, or adjusting inference. The registry record does not report a multiplicity strategy, so it is not possible to determine whether or how the trial's planned comparisons would have been handled.

The record lists 0 analyses and says the trial was terminated prematurely with no analysis performed. No primary or secondary outcome estimates, confidence intervals, or p-values are reported. Consequently, there are no posted outcome tests to interpret as adjusted or unadjusted. The intended hierarchy among secondary outcomes, if any, is not reported in the registry record.

Missing-data handling is also not reported. The record does not say how many participants had baseline or Week 12 diary data, whether diary completion was incomplete, or how any missing observations would have been treated. In general, missing outcomes can affect the interpretation of a group comparison if the availability of measurements is related to participants' outcomes or treatment. Methods such as imputation or model-based approaches require assumptions; the record does not identify any such method or assumption.

The analysis population is not reported in the registry record. It does not state whether the planned analysis would include all randomized participants, only those who received treatment, or another population. Without this information, and without an analysis, neither the handling of missing data nor the population represented by a treatment estimate can be assessed.

11. Safety Results

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

Deaths

Other (non-serious) adverse events

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

The registry reports serious adverse event counts for the AQX-1125 and placebo groups. For AQX-1125, 2 were listed as at risk and 0 as affected by a serious adverse event. For placebo, 1 was listed as at risk and 0 as affected. Deaths are listed as 0 in both groups. The registry also lists 2 at risk and 0 affected for other adverse events in the AQX-1125 group, and 1 at risk and 0 affected in the placebo group.

These are event counts in the registry's reported safety fields, not a complete comparison of risk. The record does not explain the precise safety analysis population, the duration of exposure represented by the counts, or whether all participants were followed for the same length of time. It also does not provide event rates, confidence intervals, or a comparative statistical analysis. Those measures should not be inferred from the counts.

Zero reported events in these groups does not establish that no adverse events could occur, and the small at-risk counts limit what can be learned about uncommon events. The record's safety figures should be read as descriptive registry entries, not as proof of equivalent safety between treatments or a definitive assessment of the safety profile.

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

The registry record establishes that the trial was designed to compare AQX-1125 200 mg with matching placebo for CP/CPPS, with change in maximum daily pelvic pain at Week 12 named as the primary endpoint. It also states that the trial was terminated prematurely and that no analysis was performed. These registry entries describe the design and reporting status, not an efficacy result.

The record does not show whether pelvic pain improved, worsened, or changed similarly in the two groups. It provides no treatment-effect estimate and no measure of its precision. Therefore, it cannot support a conclusion that AQX-1125 was more effective than placebo, that the treatments were equivalent, or that they had no difference. A missing analysis is not a null finding: a null finding would require analyzed data and an estimate whose uncertainty could be assessed.

The reported adverse-event counts likewise do not establish a comparative safety conclusion. The counts are descriptive, with limited information about exposure and the safety population. They should not be taken as evidence that either treatment is free of risk or that the groups have the same safety profile.

General explanation: a randomized design can support causal interpretation when allocation, follow-up, outcome measurement, and analysis are sufficiently complete and the analysis aligns with the question. Here, the intended randomized comparison is documented, but a primary analysis is absent. That distinction matters: knowing what a trial planned to measure is not the same as having evidence about what happened to participants under each treatment.

The appropriate conclusion is narrow. The registry documents a prematurely terminated study with no reported primary analysis. It leaves the treatment question unresolved in the posted results. The record does not explain the reason for termination, the amount of outcome information collected, or whether results were analyzed outside the registry entry.

13. Why This Trial Matters Statistically

This trial illustrates the difference between a well-defined research question and an answer supported by analyzed data. The registry identifies the intended population, randomized comparison, treatment period, and primary pain endpoint. Those elements make the question understandable, but they cannot replace a treatment-effect estimate.

The record also makes the study's termination consequential for interpretation. It lists enrollment as 3, whereas the brief summary describes an intended enrollment of approximately 100. The registry does not explain that difference or provide a primary analysis. Readers therefore cannot assess the observed treatment contrast, its precision, or how much information was available at termination. A planned enrollment is not evidence that the planned amount of information was collected.

For clinical researchers, the case emphasizes the importance of distinguishing a terminated trial from a completed trial with an unfavorable or null result. When no analysis is performed, the endpoint cannot be used to claim either benefit or lack of benefit. The same distinction applies to the secondary outcomes: their listing in the registry does not mean that results are available.

The record also shows why safety counts need context. Group-specific counts are posted, but the exposure and analysis population are not explained, and no comparative analysis is reported. Descriptive counts can inform what the registry records, but they do not establish relative risk or rule out uncommon events.

Statistically, this record is a reminder that interpretation depends on the chain from allocation and measurement to follow-up and analysis. When that chain ends without a reported outcome analysis, readers should preserve uncertainty rather than convert the absence of results into a claim about treatment performance.

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 is that no primary outcome analysis was performed after premature termination. The record therefore lacks the outcome values and comparative estimate needed to answer the intended efficacy question. It also does not provide confidence intervals or hypothesis tests for the primary endpoint.

The registry lists enrollment as 3, but the brief summary describes an intended enrollment of approximately 100. The record does not explain this difference, report the number randomized to each group, or state how many participants contributed primary endpoint data. It is not possible to determine the amount of usable outcome information or the precision that a completed analysis might have achieved.

The record does not report why the trial was terminated, whether all participants completed treatment, or how much follow-up occurred. The planned schedule includes a 12-week treatment period and later safety follow-up, but it cannot be assumed that these procedures were completed for every participant. The status and primary outcome description do not resolve these questions.

Analysis details are also absent. The statistical model, analysis population, handling of missing diary observations, treatment of protocol deviations, and multiplicity strategy are not reported in the registry record. The registry identifies 15 outcome measures but reports 0 analyses. Without results and methods, secondary outcomes cannot be used to fill the primary endpoint gap.

Safety interpretation is limited by the small listed at-risk groups and the lack of detail about exposure and population definitions. The posted counts do not provide an estimate of comparative risk. These limitations restrict conclusions about both efficacy and safety; they do not establish that either treatment was ineffective or unsafe.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Efficacy and Safety of AQX-1125 in Subjects With Chronic Prostatitis/Chronic Pelvic Pain Syndrome — NCT03500159.
• Registry record last updated 2019-01-09; status last verified 2018-12.
• Results first posted 2019-01-09.
• 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

The registry record describes NCT03500159, a phase 2 randomized, quadruple-masked, parallel-group comparison of AQX-1125 200 mg and matching placebo in male subjects with chronic prostatitis/chronic pelvic pain syndrome. The planned treatment period was 12 weeks, and the primary endpoint was change from baseline to Week 12 in mean maximum daily pelvic pain recorded by electronic diary.

The record lists enrollment as 3 and status as terminated. The brief summary describes an intended enrollment of approximately 100. The trial was terminated prematurely, and no primary analysis was performed. The record lists 15 outcome measures and 0 analyses; it does not report efficacy estimates or uncertainty for the primary endpoint. Posted safety counts include no serious adverse events or deaths in either listed group, but the exposure and safety population are not fully described. The registry record does not explain the reason for termination or the difference between intended and recorded enrollment.