Ospemifene vs. Conjugated Estrogens in the Treatment of Postmenopausal Sexual Dysfunction: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Ospemifene vs. Conjugated Estrogens in the Treatment of Postmenopausal Sexual Dysfunction (NCT03018106), a randomized phase 4 study. The primary outcome is Female Sexual Function Index Score.

2. Clinical Question

The registry describes a planned comparison of improvements in sexual health and VVA symptom severity. Its background notes that VVA can affect postmenopausal women and that dyspareunia is a common symptom. It also states that vaginal estrogen is commonly used for VVA, while some women and clinicians avoid exogenous estrogen because of concerns about its use during menopause. Those statements explain the clinical motivation for studying a non-estrogen alternative; they are not findings from this trial.

The clinical question is comparative: whether the outcomes of the two treatment strategies differ in the study population. A change observed within one arm alone would not establish that one strategy performs better than the other. A direct comparison requires interpretable outcome data from both groups, along with an analysis that accounts for the trial design. The registry record does not report such results, and the participant lost before Week 12 could not contribute a completed primary endpoint assessment.

3. Trial Design

This was a Phase 4, randomized, parallel-group trial sponsored by Emory University. The registry lists the study as terminated. The recorded start date is 2017-06-30, and the primary completion date is 2017-09-29. The enrollment field records 1 participant. The study description, in contrast, states that 104 women would be randomized; that figure describes the plan in the summary, not the enrollment recorded for the trial.

Participants were intended to be assigned to one of two drug regimens: 60mg oral ospemifene daily or 0.5mg vaginal conjugated estrogens twice per week, with each regimen planned for 12 weeks. The parallel design means the planned comparison was between separate groups receiving different interventions, rather than a crossover in which the same participant received both. The record describes allocation as randomized. Masking is listed as none, so the study was open-label according to the registry.

The primary outcome was the FSFI score, assessed at baseline and Week 12. The record says the single participant was lost to follow-up before completing the Week 12 assessment. It therefore does not report a completed primary endpoint measurement for that participant. The record lists five outcome measures, but detailed information available for the primary endpoint concerns the FSFI. Further outcome definitions and their analysis roles are not reported in the registry record.

The combination of a recorded enrollment of 1, termination status, and no completed Week 12 assessment is important when interpreting the study. The planned randomized comparison and the observed record are not equivalent: the design describes what investigators intended to do, while the recorded participant and outcome information describes what is available for interpretation. Reasons for termination and details about whether randomization occurred 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.

Before assignment: One individual gave informed consent to participate in the study and was randomized to the Estrogen arm.

Overall Study

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: The single participant was lost to follow up prior to completing the Week 12 assessment.

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.

Population: All participants consenting to take part in the trial are included in the baseline analysis.

6. Primary Endpoint

The registry lists the primary outcome as Female Sexual Function Index Score, measured over Baseline, Week 12.

The primary endpoint was the Female Sexual Function Index (FSFI) score, reported in units on a scale and assessed at baseline and Week 12. The FSFI is a 19 item questionnaire about sexual function during the prior four weeks. It was developed to assess areas including sexual arousal, orgasm, satisfaction, and pain related to sexual functioning. Respondents select from five to six question-specific options, with each option assigned points. The responses are combined into a total score ranging from 2 to 36. The registry states that scores equal to or less than 26.55 indicate female sexual dysfunction.

A questionnaire score can provide a structured way to describe participants’ reported experiences. Repeating the measure at baseline and follow-up is intended to make it possible to examine change over time, while a comparison between randomized groups can address whether the observed changes differ between treatment assignments. The threshold described in the record gives context for interpreting an individual score; it does not by itself show whether a treatment caused a change or whether a difference between groups is meaningful.

The registry does not report numerical FSFI results, dispersion, or a treatment comparison. It states that the single participant was lost to follow-up before the Week 12 assessment. Consequently, the record does not provide a completed Week 12 primary endpoint value or an estimate of change from baseline. It also does not report a clinically meaningful change threshold for comparing treatment groups.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

Statistical methods help turn measurements from a trial into an estimate of the difference between assigned treatments, while also describing uncertainty and the assumptions behind the estimate. In this trial, the registry record identifies randomized allocation, a parallel design, no masking, and the FSFI as the primary outcome. It does not report a statistical analysis plan or completed outcome analyses. The explanations below describe general principles; they should not be read as methods confirmed to have been used in this study.

What does randomization contribute?

In general, randomization assigns participants to study groups using a chance-based process. Its purpose is to make treatment assignment less dependent on participant characteristics and to support a fair comparison. Randomization does not guarantee that groups are similar in every respect, especially when the amount of observed information is limited. It also cannot address problems that arise after assignment, such as missing outcome measurements, unless the analysis handles those problems appropriately. The registry says allocation was randomized but does not report the sequence-generation or allocation-concealment procedures.

The parallel design means each participant was intended to receive one assigned regimen during the trial. A comparative analysis would ordinarily focus on outcomes between groups, rather than treating a change in one group as evidence of a difference between treatments. The registry does not report whether the intended analysis was based on assigned treatment, treatment received, or another population definition. Such an analysis population and its implementation are not reported in the registry record.

What does an estimate and its confidence interval mean?

In general, an estimated treatment contrast summarizes the observed difference between groups on a chosen scale. A confidence interval expresses the uncertainty around that estimate under the assumptions of the statistical procedure. A narrow interval is more precise than a wide one, but precision does not by itself establish clinical importance. The interval’s location relative to a null value can inform statistical interpretation, but conclusions also depend on the analysis plan, endpoint, and context.

No treatment contrast or confidence interval is reported for the FSFI in this record. Baseline and Week 12 scores are not posted, and the single participant was lost before the Week 12 assessment. Thus there is no estimate whose magnitude or precision can be interpreted. A p value is also not reported. In general, a p value describes how compatible the observed data are with a specified statistical model and null hypothesis; it is not the probability that the null hypothesis is true, and it does not convey the size or importance of an effect by itself.

How does the endpoint affect analysis?

The FSFI is a questionnaire total score, not a count of events. General choices for analyzing such a score may include modeling the follow-up value while accounting for baseline, or analyzing change from baseline. These approaches can have different assumptions and can yield different estimates. Which approach is appropriate depends on the design and prespecified analysis plan. The registry record does not report which approach was intended or used, nor does it report any score distribution or variability that could support an independent analysis.

The record gives the FSFI’s total-score range and a threshold associated with female sexual dysfunction. That threshold can describe the status of a score, but it is not the same as a between-group treatment effect. A comparison of whether participants cross a threshold would require a defined analysis and the relevant observed values. Neither is reported. Similarly, the use of a questionnaire does not eliminate measurement uncertainty: responses reflect reported experience over the stated recall period, and the registry’s open-label design means participants were not masked to assigned treatment.

What do the planning assumptions say?

The registry describes a sample-size planning illustration based on the observed effect, with a two-sided significance level of 0.05, equal allocation, and a normal approximation. It says the effect to detect is a stated fraction of the observed effect. These are assumptions for a planning calculation, not evidence that the trial reached its planned enrollment or produced the effect used in the illustration. The record’s enrollment field is 1, while the study summary describes a plan to randomize 104 women. No calculated participant requirements are reported in the registry record for this analysis.

Planning calculations ask how much information a future study might need under specified assumptions. They do not measure the probability that the completed trial found a true effect. Sample sizes generated from an observed effect can also be sensitive to how that effect was estimated; an unstable or imprecise estimate can lead to an unreliable planning target. In general, power is a design property under assumed conditions, not a retrospective description of whether a particular trial succeeded. No observed-power conclusion is supported by this record.

What is missing from the analysis record?

The registry does not report the model, covariates, treatment-effect scale, handling of missing data, or analysis population. It does not give a protocol-defined approach to multiplicity or describe interim analyses. Those details are not reported in the registry record and should not be inferred from the randomized design alone. The absence of a reported analysis means there is no basis for reconstructing an adjusted or unadjusted estimate from the posted primary outcome information.

In general, methods should be interpreted together with the data they analyze and the assumptions they require. Here, the essential primary endpoint values and comparative analysis are absent, so statistical concepts can explain what would ordinarily be examined but cannot fill in the missing result. Any recalculation would require posted summary statistics that are not present in this record.

10. Multiplicity, Missing Data and Analysis Populations

The registry records five outcome measures overall and identifies the FSFI as the primary outcome. The other outcome definitions and their analysis roles are not reported in the registry record. It also does not report a multiplicity strategy, such as whether or how inferential testing across multiple outcomes was to be controlled.

In general, multiplicity arises when a study makes several statistical comparisons or tests. If each test is interpreted separately, the chance of at least one apparently notable result can increase. Prespecifying a primary endpoint and analysis can help clarify which comparison carries the main confirmatory interpretation. Those general principles do not establish what procedures this trial planned: a multiplicity adjustment or hierarchy is not reported in the registry record.

Missing data are central to this record because the single participant was lost to follow-up before completing the Week 12 assessment. The record does not say why the participant was lost, whether a baseline FSFI value was available, or whether any other endpoint information was collected. It does not report imputation, a missing-data model, sensitivity analyses, or another approach for handling the absent Week 12 measure. No method should be assumed.

The analysis population is also not reported. For example, the record does not specify whether the intended analysis would include all randomized participants, participants who completed follow-up, or participants defined by another rule. Since no analyses are posted, it is not possible to determine how participant flow, missing outcomes, or treatment assignment were reflected in an analysis. The registry’s stated enrollment and planned enrollment are not substitutes for an analysis population definition.

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 reported adverse-event figures list the Estrogen group, with one participant at risk. For that group, the record reports no serious adverse events, no other adverse events, and no deaths. No serious adverse event terms are listed. These are the registry’s recorded counts for the group and should be read as descriptive information, not as a statistical comparison.

In general, an adverse-event count describes what was recorded during a defined period among participants represented in the safety data. A zero count does not establish that an event could not occur, and a small amount of safety information cannot characterize uncommon risks reliably. Interpretation also depends on how events were defined, collected, and followed. The registry record does not report those details beyond the figures listed.

The record does not give corresponding adverse-event figures for the ospemifene group. It therefore does not permit a comparison of serious adverse events, other adverse events, or deaths between assigned treatments. Nor does the Estrogen-group entry establish that no adverse events occurred in the study as a whole. The participant was lost to follow-up before the Week 12 assessment, and the record does not state how that loss affected safety follow-up. Reasons for loss and further safety ascertainment details are not reported in the registry record.

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

The registry record documents a randomized trial intended to compare ospemifene with vaginal conjugated estrogens for postmenopausal sexual dysfunction. It identifies the FSFI as the primary endpoint, specifies baseline and Week 12 assessments, and describes the treatment schedules. It also records that the trial was terminated, that enrollment was 1, and that the single participant was lost before the Week 12 assessment.

What the record does not establish is the comparative effect of the treatments. No primary endpoint values, changes, between-group estimate, confidence interval, or p value are reported. As a result, the available record cannot show whether FSFI scores differed between groups, what direction a difference might have taken, or how precisely an effect was estimated. It is not valid to interpret the absence of a result as evidence of no difference.

The study summary describes a plan to randomize 104 women. That planned figure should not be read as the number enrolled or analyzed. The enrollment field records 1, and the record does not report completed Week 12 outcome data. Reasons for termination and details about whether the intended recruitment or randomization procedures proceeded are not reported in the registry record.

The safety entry is similarly limited: it gives figures for the Estrogen group only. It does not provide a comparative safety assessment and cannot establish the absence of adverse events in the other group. The sparse record also does not support conclusions about how representative participants would have been of the wider population described by the trial.

Accordingly, the trial record is informative about the planned research question and the lack of a reported primary outcome analysis, but it does not provide evidence to determine which treatment had better measured outcomes. No treatment recommendation follows from these registry entries.

13. Why This Trial Matters Statistically

This trial matters statistically because it illustrates the difference between a clearly stated research question and an interpretable treatment-effect estimate. The registry describes a randomized comparison, two defined treatment schedules, and a patient-reported primary endpoint. Those design elements identify what investigators intended to compare. A statistical conclusion, however, also depends on collected outcome data and a reported analysis.

The contrast between the planned enrollment of 104 women in the study summary and the enrollment field of 1 makes it especially important to distinguish design plans from the evidence available in the record. The primary endpoint required a Week 12 assessment, but the record says the single participant was lost before completing it. No comparative FSFI results are posted. Consequently, the key issue is not whether a reported estimate is large or small, or whether its interval is wide or narrow: there is no reported estimate to interpret.

The case also demonstrates why a registry’s endpoint definition and follow-up schedule are not results. A questionnaire score can be clinically relevant and carefully defined, but without reported observations its treatment contrast cannot be estimated. Similarly, a planned randomization does not alone establish that a comparative analysis was completed.

Finally, sparse safety reporting should not be confused with a broad safety comparison. The listed adverse-event figures cover the Estrogen group only. Together, these features make the record a useful example of how trial status, enrollment, follow-up, endpoint availability, and analysis reporting shape what readers can conclude. The most informative statistical interpretation may be to state clearly what is absent rather than infer an effect from the intended design.

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 primary limitation is that the registry does not report a completed Week 12 FSFI assessment or a comparative analysis. The single participant was lost to follow-up before that assessment. Baseline values, participant-level data, summary statistics, treatment contrasts, and uncertainty estimates are not reported in the registry record. This prevents evaluation of the primary question and precludes an independent recalculation.

The record also presents a substantial difference between the intended and recorded enrollment information: the study summary says 104 women would be randomized, while the enrollment field records 1. The record does not explain this difference, document recruitment progress, or report whether randomization was carried out. The study is listed as terminated, but the reason and timing of termination are not reported in the registry record.

Other design and analysis details are unavailable. Although the record lists randomized allocation and no masking, it does not report sequence generation, allocation concealment, detailed eligibility criteria, baseline characteristics, the statistical model, analysis population, missing-data approach, or multiplicity procedures. The lack of masking may be relevant to a self-reported endpoint, but the record does not demonstrate that it affected responses.

Safety information is incomplete for comparison because only the Estrogen group is listed. The record does not provide corresponding ospemifene figures or detailed ascertainment procedures. The limited posted counts cannot establish comparative safety or characterize events beyond those recorded. These limitations are specific to the information available in the registry record; they do not show what may or may not exist outside that record.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Ospemifene vs. Conjugated Estrogens in the Treatment of Postmenopausal Sexual Dysfunction — NCT03018106.
• Registry record last updated 2018-01-23; status last verified 2017-11.
• Results first posted 2017-12-20.
• 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

NCT03018106 is a Phase 4 randomized parallel-group trial sponsored by Emory University and listed as terminated. It was designed to compare 60mg oral ospemifene daily with 0.5mg vaginal conjugated estrogens twice per week, each for 12 weeks, in the treatment of postmenopausal sexual dysfunction. The study summary describes planned enrollment of 104 women, while the enrollment field records 1.

The primary endpoint was the FSFI score at baseline and Week 12. The registry states that the single participant was lost to follow-up before completing the Week 12 assessment. No primary outcome analysis or treatment comparison is reported. Safety figures are listed for the Estrogen group only: one at risk, no serious adverse events, no other adverse events, and no deaths. The registry record does not report a comparative efficacy or safety result.