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 Androgen Excess as a Cause for Adipogenic Dysfunction in PCOS Women (NCT01889199), a randomized phase 2 study. The primary outcome is Lipid Content of PCOS Subcutaneous (SC) Abdominal Adipocytes Matured in Vitro..
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01889199 |
| Phase | Phase 2 |
| Condition | Polycystic Ovary Syndrome (PCOS) |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | Triple |
| Enrollment | 23 |
| Arms in the primary outcome | n/a |
| Lead sponsor | University of California, Los Angeles |
| Results posted | Yes |
2. Clinical Question
Population
The primary endpoint concerns participants with polycystic ovary syndrome (PCOS). The study also included age- and body mass index-matched controls without PCOS for specimen collection and other assessments. The registry record lists total enrollment as 23; it does not report the number enrolled in each study population beyond the group counts in its safety record.
Intervention
Participants with PCOS were randomized to flutamide, described as 125 mg orally daily for six 28-day cycles, or placebo. The record describes specimen collection and laboratory assessment of subcutaneous abdominal fat, including adipocyte maturation in vitro.
Comparator
The randomized comparison was flutamide versus placebo among participants with PCOS. The controls without PCOS are a separate group, not the randomized placebo comparator. Their role was to contribute comparison data about women without PCOS.
Primary question
The primary endpoint is lipid content in PCOS subcutaneous abdominal stem cells matured into adipocytes in vitro, measured at baseline and after 6 months of treatment. The clinical question is whether the posted results show a difference in this laboratory measure between the randomized treatment groups over that period. Numerical endpoint results and the primary statistical comparison are not reported in the registry record, so the question cannot be answered from the record alone.
3. Trial Design
This completed phase 2 study is described as randomized, triple-masked, and parallel. Its stated enrollment is 23. The brief description anticipated participation by 32 women, divided between women without PCOS and women with PCOS; that anticipated number is not the same as the recorded enrollment. The registry record does not explain the difference between the anticipated and recorded totals.
Women with PCOS were randomized to flutamide or placebo. The listed flutamide regimen was 125 mg orally daily for six 28-day cycles. Women without PCOS were included as age- and body mass index-matched controls, but were not described as randomized to the treatment arms. Thus, the treatment comparison is flutamide versus placebo among women with PCOS, while the control group serves a different descriptive and research role.
The study description outlines physical examination, blood testing, ovarian ultrasound, subcutaneous abdominal fat sampling, regional body-fat measurement, and a modified frequently-sampled intravenous glucose tolerance test. It also describes urine collection and monthly visits for the PCOS participants taking flutamide or placebo. These details describe planned study procedures; the registry record does not report completion or adherence for each procedure.
The primary outcome timeframe is baseline and 6 months. The record describes a continuous laboratory measure, reported as a mean with standard deviation, based on fluorescence units per cell number. The specific randomization method, identities of the masked parties, allocation concealment procedures, and details of the 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 | Sugar Pill | Flutamide | Age- and Body Mass Index-matched Controls |
|---|---|---|---|
| STARTED | 6 | 5 | 12 |
| Baseline | 6 | 5 | 12 |
| 6-month Assessment on Intervention Arms | 6 | 5 | 0 |
| COMPLETED | 6 | 5 | 0 |
| NOT COMPLETED | 0 | 0 | 12 |
Analysis population for the primary outcome, as defined in the registry: Assessed for participants with PCOS
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 | Flutamide | Age- and Body Mass Index-matched Controls | Total |
|---|---|---|---|---|---|
| Participants analysed | 6 | 5 | 12 | 23 | |
| Age, Categorical: <=18 years | Participants | 0 | 0 | 0 | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 6 | 5 | 12 | 23 |
| Age, Categorical: >=65 years | Participants | 0 | 0 | 0 | 0 |
| Sex: Female, Male: Female | Participants | 6 | 5 | 12 | 23 |
| Sex: Female, Male: Male | Participants | 0 | 0 | 0 | 0 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 | 0 | 0 | 0 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 6 | 4 | 4 | 14 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 | 1 | 8 | 9 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 0 | 0 | 1 | 1 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): White | Participants | 6 | 4 | 3 | 13 |
| Race (NIH/OMB): More than one race | Participants | 0 | 1 | 0 | 1 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 8 | 8 |
| Region of Enrollment: United States | participants | 6 | 5 | 12 | 23 |
6. Primary Endpoint
The registry lists the primary outcome as Lipid Content of PCOS Subcutaneous (SC) Abdominal Adipocytes Matured in Vitro., measured over Baseline, 6 months.
| Element | Registry entry |
|---|---|
| Result type | Mean |
| Measure of spread | Standard Deviation |
| Unit | fluorescence units/cell number |
| Endpoint classification | Continuous Endpoint |
The primary endpoint is lipid content in subcutaneous abdominal stem cells from participants with PCOS after the cells have matured into adipocytes in vitro. The registry describes lipid staining with Oil-Red-O and nuclear staining, followed by immunofluorescence quantification. It states that representative fluorescent-cell images were captured and that fluorescence units per cell number were quantified. The endpoint is therefore a laboratory measurement of lipid content normalized to cell number, rather than a direct measure of whole-body fat or a clinical symptom.
Such a measure can be used to investigate cellular features of adipocyte development and lipid storage. In this study, measuring the endpoint at baseline and after 6 months provides a way to describe the laboratory measure over the treatment period and compare the randomized groups, if results are available. This is a general explanation of the role of a continuous laboratory endpoint; it does not establish that a change occurred or that any observed change would have clinical importance.
The record specifies the population, unit, summary form, and time points, but does not report the group means, standard deviations, changes from baseline, between-group estimate, confidence interval, or test result. The magnitude, direction, and precision of the primary comparison are therefore not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record establishes the trial question and design but does not provide numerical results for the primary endpoint. It identifies lipid content in PCOS subcutaneous abdominal adipocytes matured in vitro as the primary outcome, measured at baseline and 6 months, with mean and standard deviation as the stated summary. Without those values or a treatment-group comparison, the size and direction of any difference cannot be described. No conclusion about a treatment effect can be drawn from the endpoint description alone.
The record lists 23 participants enrolled and marks the study completed. Completion status does not itself indicate that every participant completed treatment, provided every specimen, or contributed data at both measurement times. Participant flow, endpoint-specific numbers analyzed, missing measurements, and reasons for missingness are not reported in the registry record. The brief description's anticipated enrollment of 32 is a planning statement and should not be interpreted as the actual enrollment.
The record reports four analyses, but does not describe the analyses or identify which correspond to the primary endpoint. It also lists seven outcome measures and names secondary outcomes including fasting glucose, depression assessed by the Beck Depression Inventory, percent android fat mass, fasting serum lipoprotein levels, and fasting serum total cholesterol. Numerical results for those measures are not reported in the registry record. The presence of multiple outcomes makes it important to know which comparisons were prespecified and how multiplicity was addressed; those details are also not reported.
The safety record lists zero participants with serious adverse events in each of the three groups shown: sugar pill, flutamide, and age- and body mass index-matched controls. It also lists zero participants with other adverse events in each group, and zero deaths. These counts should be read alongside the numbers at risk recorded for each group, and not as proof that no events of any kind occurred outside the registry's reported categories or observation period. The record does not supply detailed event definitions, follow-up completeness, or event-specific descriptions.
Overall, the posted information supports a description of the study design, the endpoint, and the recorded safety counts. It does not support an estimate of the primary treatment contrast or its uncertainty. A confidence interval, if reported, would describe the range of effect estimates compatible with the data and analysis under its assumptions; no such interval is reported for the primary outcome in the registry record.
9. Statistical Methods Explained
What does a continuous endpoint mean?
The primary endpoint is a continuous laboratory measurement: fluorescence units per cell number. Continuous measurements can take a range of values rather than placing each participant into a category. The registry specifies that results are summarized as a mean and standard deviation. A mean describes the center of a set of measurements, while a standard deviation describes their spread around that center. These summaries would help readers understand both the typical measured value and how variable values were among participants.
The record does not state whether the primary comparison uses endpoint values at 6 months, changes from baseline, or another formulation. Although the timeframe names baseline and 6 months, the statistical contrast and model are not reported in the registry record. This matters because different analysis choices answer related but not identical questions. A baseline-adjusted comparison, for example, has a different specification from a comparison of raw changes. No particular approach should be assumed.
What does randomization contribute?
Random assignment of participants with PCOS to flutamide or placebo is intended to make the treatment groups comparable on average, including with respect to characteristics not measured by the investigators. In a randomized comparison, the principal treatment contrast is between the assigned groups. Randomization does not guarantee that small groups will be identical in every characteristic, nor does it prevent missing measurements or deviations from assigned treatment.
The record says the study was triple-masked, but does not state which people or roles were masked, how masking was maintained, or whether masking was evaluated. Masking is generally intended to reduce the influence of knowledge about treatment assignment on participant behavior, care, measurement, or analysis. The precise protections used in this study are not reported in the registry record.
How should an effect estimate and confidence interval be read?
For a continuous outcome, an effect estimate would quantify the treatment-group contrast using the chosen analysis scale. A confidence interval expresses the precision of that estimate: narrower intervals indicate greater precision, while wider intervals indicate more uncertainty. The interval also reflects sampling variability under the statistical model and does not account automatically for every possible source of bias, such as missing data or unblinded assessment.
No primary effect estimate, confidence interval, or p-value is reported in the registry record. Therefore, neither the magnitude nor statistical uncertainty of the treatment comparison can be assessed from the posted information. Statistical significance, if a test result were available, would not by itself describe the size, precision, or clinical relevance of a difference.
Why can small studies produce imprecise estimates?
In general, the amount of information available to estimate a treatment contrast depends on the number of participants with usable outcome data, the variability of the measurement, and the analysis design. When the number of observations is limited or the outcome is variable, estimates can be imprecise. The registry lists total enrollment as 23, but the number contributing primary outcome data is not reported in the registry record. The endpoint's observed variability is also not available because group summaries are absent.
The record includes planning illustrations based on an observed effect and assumptions of a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. Such illustrations are not the trial's original sample-size justification, and their assumptions do not establish that the study had a particular probability of detecting an effect. The registry record does not report the trial's sample-size justification. A sample-size calculation is a planning exercise, not observed power, and cannot replace the actual effect estimate and its uncertainty.
What is over-dispersion, in general?
Over-dispersion is a concern for some types of outcomes when the observed variability is greater than a statistical model expects. It can make standard errors too small if the model does not account for that extra variability. The primary measure is described as continuous and summarized by a mean and standard deviation; no model or evidence of over-dispersion is reported. This general concept should not be taken as a claim that over-dispersion occurred in this study.
What statistical plan is available?
The record identifies four analyses but does not describe their methods, populations, covariates, handling of repeated measurements, or decision rules. Interim analyses, stopping rules, missing-data methods, and the analysis population are not reported in the registry record. The reported analysis count alone cannot establish how the primary outcome was tested or whether the analysis followed a prespecified plan. Those details are needed to evaluate the assumptions behind any treatment estimate, but no estimate is posted for the primary outcome.
10. Multiplicity, Missing Data and Analysis Populations
The record lists seven outcome measures and four analyses. It names five secondary outcomes: fasting glucose levels, depression as assessed by the Beck Depression Inventory, percent android fat mass, fasting serum lipoprotein levels, and fasting serum total cholesterol. The primary adipocyte lipid measure is also specified. How the remaining outcome measure is defined, how the four analyses map to the outcomes, and which tests were designated as primary or secondary are not reported in the registry record.
When a study examines multiple outcomes or comparisons, the chance of at least one apparently unusual result can increase if each is assessed separately without accounting for multiplicity. This is a general statistical consideration, not a claim about the results of this study. Whether any adjustment, hierarchy, or other multiplicity strategy was planned or used is not reported in the registry record. The record also does not report interim analyses or stopping rules.
Missing-data handling is another important part of interpreting a longitudinal endpoint. The record identifies baseline and 6-month measurement times but does not report endpoint-specific completion, reasons for missing measurements, imputation procedures, or sensitivity analyses. The analysis population—such as whether all randomized participants were included in the primary comparison—is not reported in the registry record. Nor are exclusions or deviations from assigned treatment described for the primary analysis.
Without these details, readers cannot determine how incomplete observations or multiple comparisons may affect the precision and interpretation of a treatment estimate. The record does not report a primary result with which to assess those issues directly.
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 | 6 | 0.0% |
| Flutamide | 0 | 5 | 0.0% |
| Age- and Body Mass Index-matched Controls | 0 | 12 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Sugar Pill | 0 of 6 |
| Flutamide | 0 of 5 |
| Age- and Body Mass Index-matched Controls | 0 of 12 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 0% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Sugar Pill | 0 of 6 |
| Flutamide | 0 of 5 |
| Age- and Body Mass Index-matched Controls | 0 of 12 |
The registry safety record shows zero participants with serious adverse events among the 6 at risk in the sugar pill group, the 5 at risk in the flutamide group, and the 12 at risk in the age- and body mass index-matched control group. It also records zero participants with other adverse events in each of those groups and zero deaths in each group.
These are counts for the categories reported in the registry, not evidence that every participant had complete safety follow-up or that no unrecorded symptoms or events occurred. The numbers at risk differ across the groups, and the safety record does not give the duration or completeness of safety observation. It also does not provide event-level descriptions, definitions, severity grading beyond the serious-event category, or the methods used to collect and classify events. These details are not reported in the registry record.
With no reported events in these groups, the registry counts do not establish that the treatments have no safety risks. In general, a zero count in a study of limited size may still be compatible with uncertainty about how often an event could occur in a broader population. The posted counts should therefore be read as recorded observations within this study, rather than as a general safety guarantee or a comparison demonstrating that the groups had identical risk.
12. What the Result Does, and Does Not, Mean
The record describes a study designed to investigate whether flutamide treatment is associated with a difference in a laboratory measure of lipid content in adipocytes derived from subcutaneous abdominal stem cells in women with PCOS. The randomized comparison is flutamide versus placebo, with the endpoint specified at baseline and 6 months. The study also included age- and body mass index-matched controls without PCOS, who are not the randomized treatment comparator.
What the posted record does not show is the primary endpoint result. No group means, standard deviations, treatment contrast, confidence interval, or p-value for that outcome are reported. Therefore, the direction, magnitude, and precision of any difference cannot be determined from the registry record. Study completion and the existence of a defined endpoint do not supply evidence about the endpoint's observed values.
Even if a laboratory difference were reported, interpretation would require attention to uncertainty, missing measurements, analysis choices, and how the cellular measure relates to outcomes beyond the laboratory assay. The endpoint is a measure of lipid content in cells matured in vitro; it is not itself a direct measurement of whole-body fat, a symptom, or a clinical outcome. General statistical interpretation should distinguish a measured association or randomized-group contrast from broader claims about health.
The registry lists zero serious adverse events and zero other adverse events in the recorded groups, but those counts alone cannot establish absence of risk. The study's recorded enrollment is 23, and the record does not provide the primary endpoint analysis or detailed follow-up information. The appropriate reading is therefore limited: the registry records a completed randomized study and its planned laboratory question, but does not report enough primary outcome data to judge the treatment contrast or its precision.
13. Why This Trial Matters Statistically
This study is statistically notable because it combines a randomized treatment comparison with a laboratory endpoint collected from participants with PCOS. The design distinguishes the flutamide-versus-placebo comparison from the separate group of age- and body mass index-matched controls without PCOS. Keeping those roles distinct is important: the controls can provide context about the study population, but they do not replace the randomized comparator for estimating a treatment contrast.
The endpoint is a continuous measurement of lipid content in adipocytes matured in vitro, expressed as fluorescence units per cell number. Such an outcome requires attention to the scale and variability of the assay, the number of participants contributing specimens, and the way baseline and follow-up data are compared. The record describes image-based measurement procedures, but does not report assay variability, reproducibility, or the statistical method used for the primary comparison.
The registry also illustrates why a completed status is not a substitute for posted results. The record identifies the study's question and describes its procedures, but numerical results for the primary endpoint are not reported. Without an estimate and an uncertainty measure, readers cannot assess whether the observed data are consistent with a small or large difference, or how precisely any difference was estimated.
Finally, the record lists multiple outcomes and analyses, while not reporting how multiplicity, missingness, or analysis populations were handled. These are central considerations for interpreting a study with repeated measurements and several outcomes. The statistical importance of this trial record lies in identifying those design and reporting needs, rather than in supporting a conclusion about the treatment effect.
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 principal limitation for interpreting the primary question is that numerical results for the primary endpoint are not reported in the registry record. There is no group-level summary, treatment contrast, confidence interval, or test result from which to assess direction, magnitude, or precision. The analysis approach for baseline and 6-month measurements is also not reported.
The record lists total enrollment as 23, while the brief description anticipated 32 women. It does not explain the difference, provide a participant flow, or state how many participants were randomized, treated, and analyzed for the primary endpoint. The safety counts provide group-specific numbers at risk, but do not resolve endpoint-specific sample sizes or missing follow-up.
Further limitations include the absence of reported details on allocation procedures, the roles covered by triple masking, adherence, treatment discontinuation, and protocol deviations. The statistical plan, sample-size justification, interim analyses, missing-data methods, and analysis populations are not reported in the registry record. The record lists multiple outcome measures and analyses without explaining how they were related or how multiplicity was handled.
The endpoint itself is a laboratory measure of lipid content in cells matured in vitro. Its relationship to outcomes beyond this assay is not established in the registry record. No assay reliability information or details about specimen availability and measurement completeness are reported. These gaps limit assessment of both the treatment comparison and the broader meaning of any potential laboratory difference.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Androgen Excess as a Cause for Adipogenic Dysfunction in PCOS Women — NCT01889199.
- Registry record last updated 2025-01-10; status last verified 2024-12.
- Results first posted 2025-01-10.
- 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
NCT01889199 is a completed phase 2 randomized, triple-masked, parallel study sponsored by the University of California, Los Angeles. The condition listed is PCOS, and recorded enrollment is 23. Women with PCOS were randomized to flutamide, 125 mg orally daily for six 28-day cycles, or placebo. The study also included age- and body mass index-matched controls without PCOS.
The primary outcome is lipid content in PCOS subcutaneous abdominal adipocytes matured in vitro, measured at baseline and 6 months and described as a mean with standard deviation. The registry record does not report numerical primary outcome results or the primary statistical comparison. It records zero serious adverse events, zero other adverse events, and zero deaths in each of the three listed groups. Details on missing data, analysis populations, multiplicity, and the statistical plan are not reported in the registry record.