Efficacy Study of Raloxifene to Induce Ovulation in Polycystic Ovarian Syndrome: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Efficacy Study of Raloxifene to Induce Ovulation in Polycystic Ovarian Syndrome (NCT01607320), a randomized na study. The primary outcome is Pregnancy.

2. Clinical Question

The clinical question is whether the pregnancy outcome differed between the two treatment groups. That comparison cannot be answered from the registry results described in the record: there are no analysis records, and the primary-outcome population note says that data collection is incomplete. No pregnancy counts, group-specific estimates, uncertainty intervals, or statistical tests are reported.

Ovulation is listed as a secondary outcome, so the record identifies it as another outcome of interest. It does not state an ovulation result or a definition of how ovulation was assessed. The record also does not specify how pregnancy was defined or confirmed. The timeframe description is not a fixed follow-up schedule: it says duration would vary according to the number of cycles needed for pregnancy and that participation would likely be about 4 months if pregnancy did not occur.

These details frame the intended comparison, not a demonstrated difference between treatments. The record says the study was randomized between the treatments, but also says the study was never unblinded, that randomization is unknown, and that data collection is incomplete. Those qualifications leave the status and completeness of the primary-outcome data unclear.

3. Trial Design

The registry record describes a randomized, single-masked, parallel study of raloxifene and clomiphene in women with polycystic ovarian syndrome. The listed enrollment is 3. The study is marked terminated. Its recorded start is 2012-06, and its primary completion is 2013-03. The phase is listed as NA.

The raloxifene regimen is described as 3 cycles of 120mg/day of Evista on days 3 to 7. The clomiphene regimen is described as 3 cycles of 100mg/day of Clomid on days 3 to 7. These are the registry's arm descriptions; the record does not report how many participants were assigned to each arm, how many received treatment, or how many completed treatment.

The design labels need to be read alongside the registry's qualification about the primary-outcome population. Although allocation is recorded as randomized, that note says the study was randomized between the two treatments and was never unblinded, therefore randomization is unknown and data collection is incomplete. The record does not explain this apparent tension or clarify which aspects of allocation or randomization could be verified. It also does not describe how masking was implemented or who was masked.

Termination is a study status, not an outcome. The record does not give a reason for termination or establish whether termination affected treatment delivery or outcome follow-up. Nor does it report a recruitment history, participant flow, protocol deviations, or the timing of any termination decision. Those omissions make it difficult to reconstruct how the planned comparison proceeded in practice.

The listed sponsor is Bruce Lessey. The record identifies pregnancy as the primary outcome and ovulation as a secondary outcome. It does not provide a clinical trial phase beyond the NA designation, a detailed protocol, or a complete account of procedures. The enrollment figure and status describe the registry entry; they do not provide the missing comparative outcome results.

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

Analysis population for the primary outcome, as defined in the registry: The study was randomized between the two treatments and was never unblinded, therefore randomization is unknown and data collection is incomplete.

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 Pregnancy, measured over 4 months.

The primary outcome is titled Pregnancy, with a listed timeframe of 4 months. The description qualifies that timeframe: it says the time will vary depending on how many treatment cycles it takes to get pregnant and that, if no pregnancy occurs, study participation will likely be about 4 months. Thus, the record presents pregnancy as the outcome of interest while acknowledging that observation time could differ according to the participant's course.

In general, a pregnancy endpoint can address whether the outcome of interest occurred during follow-up. Its interpretation depends on the operational definition, ascertainment procedures, and follow-up rules. Those details are not reported in the registry record. The record does not say whether pregnancy required confirmation by a particular test, how pregnancy status was collected, or how participants with incomplete follow-up were handled.

The endpoint is useful for the study's stated question because the registry describes the trial as examining treatments to induce ovulation and lists pregnancy as its primary outcome. Pregnancy and ovulation are not interchangeable outcomes: the record lists ovulation separately as a secondary outcome. No ovulation result is reported, and no pregnancy result or treatment-group comparison is available. The registry therefore documents the intended primary measure and its broad timeframe, but not the evidence needed to estimate the outcome in either group.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What does randomization contribute?

In general, random assignment is intended to make treatment groups comparable in expectation, so that outcome contrasts can be interpreted as treatment comparisons rather than simply as differences between people who selected different treatments. Randomization does not guarantee that groups will be balanced in every realized study, and its value depends on allocation being implemented and documented. Here, the registry records randomized allocation but also states that randomization is unknown in the primary-outcome population note. The record does not explain that qualification or describe the allocation process, so the strength of the randomization documentation cannot be assessed.

What does masking mean?

The study is listed as single-masked. In general, masking is used to limit the influence of knowledge about treatment assignment on care, outcome assessment, or reporting. The label alone does not identify who was masked or how masking was maintained. The record says the study was never unblinded, but it does not define the masked party or describe whether outcome assessment was masked. The single-masking designation should therefore be read as a design label, not as a detailed account of conduct.

How would a treatment comparison usually be reported?

For a binary outcome such as whether pregnancy occurred, a comparative analysis would generally report the outcome in each group and a measure of the contrast between groups. The choice of measure and method depends on the analysis plan and available data. For an outcome involving timing, methods may also account for when an event occurred and for participants whose event status remains unknown at the end of observation. These are general statistical options, not methods confirmed for this trial. The registry record does not state the planned estimand, model, test, adjustment strategy, or handling of incomplete follow-up.

A confidence interval is a way of expressing uncertainty around an estimate under specified statistical assumptions. A narrower interval usually indicates greater precision, while a wider interval indicates less precision. It does not establish that an effect is clinically important, and it cannot repair missing or incomplete outcome data. No confidence interval is reported for pregnancy or ovulation in the registry record.

Why does incomplete follow-up matter?

When outcome collection is incomplete, the recorded outcomes may not represent the outcomes that would have been observed under complete follow-up. The effect of missingness on an estimate depends on why observations are missing and how missing data are handled. In general, analysts may describe missingness, define assumptions, and conduct sensitivity analyses to explore how results might change under alternative assumptions. The registry record says data collection is incomplete but does not report the amount, pattern, or reasons for missingness, a missing-data method, or sensitivity analyses. No direction or magnitude of bias can be inferred from that statement alone.

What do the planning calculations mean?

The stated planning illustration uses the observed effect as its starting point and considers detecting a stated fraction of that effect. It specifies a two-sided significance level of 0.05, equal allocation between arms, and a normal approximation. In general, sample-size planning asks what study size would be needed to detect a target effect under assumptions about variability, allocation, and statistical error. Such calculations are prospective illustrations, not estimates of what the trial actually had a chance to detect after the fact.

In particular, a planning illustration based on an observed effect must not be described as observed power. Power is a design quantity that depends on assumptions about the target effect and analysis; calculating it from an observed effect does not resolve uncertainty in the observed data. The registry record does not report a trial-specific sample-size justification or establish that these planning assumptions were used to design the trial. It also does not provide the effect estimate needed to assess the illustrations as trial evidence. Any sample-size values should therefore be read only as planning scenarios, not as results or a substitute for the absent analysis.

Over-dispersion is a general concern when observed variation is greater than a statistical model assumes. If a model underestimates variation, its uncertainty estimates may be too small and its tests may be poorly calibrated. The registry record does not identify an over-dispersed outcome or a model for which this issue is relevant; it reports no analyses. It would therefore be speculative to claim that over-dispersion affected this study.

What can be concluded from the posted statistical record?

The record lists two outcome measures, identifies pregnancy as primary and ovulation as secondary, and lists zero analyses. There are no reported treatment effect estimates, uncertainty intervals, or tests to interpret. General statistical principles can explain how such results would ordinarily be assessed, but they cannot fill in results that are not reported in the registry record.

10. Multiplicity, Missing Data and Analysis Populations

The registry record identifies pregnancy as the primary outcome and ovulation as a secondary outcome. It does not report a statistical analysis plan or a strategy for addressing multiplicity. In general, evaluating several outcomes or conducting several analyses can increase the chance of a misleading positive finding if the testing strategy is not prespecified or appropriately accounted for. Whether that concern applies to any actual analysis cannot be determined here, because the record lists zero analyses and gives no test results.

Missing outcome data are a central limitation. The primary-outcome population note says data collection is incomplete, but the record does not describe which observations are missing, why they are missing, or whether missingness differs by treatment. It also does not report a method for handling incomplete data, assumptions about missingness, or sensitivity analyses. The absence of these details prevents assessment of how missing outcomes may have affected any estimate.

The record does not define an analysis population, such as a population based on randomized assignment or treatment received. Although allocation is recorded as randomized, the primary-outcome note says randomization is unknown and that the study was never unblinded. The record does not explain how those statements relate to the analysis population or to the available outcome data. It also does not report participant flow, exclusions from analysis, or protocol deviations.

These omissions do not establish that a particular analysis was biased. They mean that the registry record does not support a transparent assessment of multiplicity, missing-data handling, or analysis-population choices.

11. Safety Results

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

Other (non-serious) adverse events

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

Most frequent non-serious adverse event terms

The adverse-event summary lists 3 participants at risk for serious adverse events and 0 affected. For other adverse events, it lists 3 at risk and 2 affected. These are the registry's reported counts; they are not comparative estimates between raloxifene and clomiphene because the summary is presented for all study participants rather than separately by treatment group.

The record lists no serious adverse-event terms. It does not provide descriptions of the other adverse events, their timing, severity, or relationship to treatment. A count of affected participants does not by itself establish whether an event was caused by a study drug. The serious-event count also cannot be interpreted as a complete safety assessment in light of the record's statement that data collection is incomplete.

Deaths are listed as null, which means a death count is not reported in the registry record; it should not be read as a reported count of zero. These safety figures are descriptive registry entries and do not establish comparative safety.

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

The registry record establishes that a study was intended to compare raloxifene with clomiphene for ovulation induction in women with polycystic ovarian syndrome. It describes a randomized, single-masked, parallel design, lists pregnancy as the primary outcome and ovulation as a secondary outcome, and gives treatment regimens and a pregnancy timeframe. It also records enrollment of 3 and a terminated status.

It does not establish whether either treatment led to more pregnancies, whether ovulation differed between groups, or how precise any treatment contrast might be. No outcome estimates or analyses are reported, and the record says data collection is incomplete. The registry's design description is not a result. Neither the absence of an analysis nor the termination status demonstrates that a treatment was beneficial, ineffective, or harmful.

In general, a treatment comparison is informative when its outcomes are collected and analyzed in a way that allows both the estimated effect and its uncertainty to be assessed. The registry record does not include the information required for that assessment. There are no reported group-specific pregnancy counts, no definition of pregnancy ascertainment, no uncertainty intervals, and no documented analysis plan. The result is not merely imprecise evidence in the form of a wide interval; the comparative estimate itself is not reported.

The safety counts are also limited in meaning. They are reported for all study participants, not as a treatment-group comparison, and the record does not provide event descriptions or a reported death count. These data cannot answer the primary efficacy question and should not be treated as a complete account of safety.

The appropriate conclusion is narrow: the registry describes the intended trial and records incomplete outcome collection, but does not report comparative efficacy analyses. Any stronger conclusion about treatment effects would go beyond what the registry record states.

13. Why This Trial Matters Statistically

This trial matters statistically because its registry entry illustrates the difference between a planned comparison and an interpretable result. Randomized allocation, masking, treatment descriptions, and a named primary outcome describe aspects of design. They do not, on their own, establish what happened to participants or what the treatment contrast was. A useful efficacy interpretation also needs outcome data, a defined analysis, and an account of uncertainty.

The record makes the gap especially clear: it identifies pregnancy as the primary outcome, but reports incomplete data collection and lists no analyses. It also marks the study terminated. These details alert readers that the intended comparison cannot be evaluated from the recorded results, while leaving the reasons and consequences of termination unreported. Statistical reporting should distinguish those known details from conjecture about why the evidence is incomplete.

The note that randomization is unknown is also consequential for interpretation. Randomization is a basis for comparing groups, but a registry label is not enough to establish how assignment was implemented or how it relates to the available data. Clear reporting of allocation and analysis populations helps readers assess whether a result represents the randomized comparison.

Finally, the record shows why planning calculations and observed results must be kept separate. A planning illustration based on an observed effect can describe hypothetical study sizes under assumptions, but it does not produce a treatment estimate, recover missing outcomes, or demonstrate observed power. The central statistical lesson is that design information and planning scenarios cannot replace complete outcome reporting and a transparent analysis.

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 contains limited information about how the study was conducted and what outcomes were observed. It lists enrollment as 3, but does not report treatment-group enrollment, participant flow, adherence, or completion of the stated treatment regimens. The study is marked terminated, but the reason for termination and its timing are not reported in the registry record.

The primary outcome is titled Pregnancy and has a timeframe of 4 months, but the description says timing varies according to the number of cycles needed to get pregnant. The record does not report a standardized pregnancy definition, ascertainment method, or handling of differing observation times. Data collection is explicitly described as incomplete, with no account of which outcomes or participants lack data.

Although the design is listed as randomized and single-masked, the primary-outcome population note says randomization is unknown and the study was never unblinded. The record does not explain these statements, identify who was masked, or describe allocation implementation. It also does not provide an analysis plan, statistical method, analysis population, or missing-data strategy.

There are no reported primary or secondary outcome analyses, group-specific efficacy results, or estimates of uncertainty. The safety information is aggregated across all study participants and lacks event descriptions; deaths are not reported. These limitations prevent assessment of comparative efficacy and limit interpretation of the adverse-event summary. They are limitations of the available registry record and do not establish what additional information may exist outside it.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Efficacy Study of Raloxifene to Induce Ovulation in Polycystic Ovarian Syndrome — NCT01607320.
• Registry record last updated 2018-09-20; status last verified 2018-08.
• Results first posted 2015-06-01.
• 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 a randomized, single-masked, parallel comparison of raloxifene and clomiphene in women with polycystic ovarian syndrome. Enrollment is listed as 3, and the study status is terminated. The primary outcome is pregnancy, with a listed timeframe of 4 months; ovulation is listed as a secondary outcome.

The record says data collection is incomplete and that randomization is unknown. It lists zero analyses and reports no comparative efficacy results. The adverse-event summary reports serious events for 0 of 3 at risk and other adverse events for 2 of 3 at risk across all study participants. Deaths are not reported in the registry record.

The entry documents the intended comparison and some design details, but it does not provide the outcome data or analysis needed to estimate a treatment effect.