2. Study to Evaluate the Efficacy/Safety of Bremelanotide in Premenopausal Women With Hypoactive Sexual Desire Disorder: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for 2. Study to Evaluate the Efficacy/Safety of Bremelanotide in Premenopausal Women With Hypoactive Sexual Desire Disorder (NCT02338960), a randomized phase 3 study. The primary outcome is Efficacy of a Fixed Dose of Bremelanotide as Measured by FSFI (Question Q1 and Q2), 28-day Recall..

2. Clinical Question

The registry identifies the FSFI desire domain as a co-primary endpoint. The score ranges from 1.2 to 6, with higher scores representing increased sexual desire and a better outcome. The endpoint description specifies change from baseline to end of study, while the listed timeframe describes an 8-week baseline plus a 24-week main study and a 52-week open-label extension. The endpoint result and the comparison between the randomized groups are not reported in the registry record.

The clinical question therefore has a clear population, intervention, comparator, and measure, but the posted information does not establish the size or precision of any between-group difference. The registry describes an optional extension, but the main randomized comparison is the bremelanotide-versus-placebo contrast during the main study. The extension is open-label, so it is distinct from the blinded placebo-controlled comparison. The record does not report enough endpoint results to assess whether the groups changed differently, how uncertain an estimated difference might be, or how the primary endpoint was analyzed.

3. Trial Design

This was a Phase 3, randomized, triple-masked, parallel-group, placebo-controlled study sponsored by Palatin Technologies, Inc. The registry lists enrollment of 714 and a completed status. The recorded start was 2015-01, and primary completion was 2016-08. These design details describe the trial structure and registration history; they do not provide evidence about the endpoint effect.

In the main study, participants were assigned to bremelanotide or placebo. The bremelanotide arm used a fixed 1.75 mg subcutaneous dose on an as-needed basis, with no more than 1 dose every 24 hours, for 24 weeks. The placebo arm received subcutaneous placebo on an as-desired basis for 24 weeks. The registry labels the study triple-masked but does not report which specific people or roles were masked, or how masking was maintained.

An optional open-label extension followed the core study. The record states that 321 of the 392 subjects who completed the Core Study Phase continued into the optional OLE Study Phase, reported as 81.9%. The extension description includes bremelanotide administration, and the placebo-to-bremelanotide arm description specifies bremelanotide in the extension. Because the extension was open-label, it should not be read as another blinded placebo-controlled comparison. The registry does not report extension outcome results in the information described.

The registered intervention descriptions, allocation type, masking, parallel model, and enrollment help identify the intended comparison. They do not establish the number included in each endpoint analysis, the allocation ratio actually achieved, adherence, treatment discontinuation, or the handling of participants with missing measurements. Those details 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: Core Study: 703 participants enrolled, 89 run-in failures 614 participants were randomized. OLE Study (optional): Of the 392 completers of the Core study, 321 participants enrolled in optional OLE study. The OLE Study was not reported.

Core Study

Open Label Extension

Analysis population for the primary outcome, as defined in the registry: A total 321 of the 392 (81.9%) subjects who completed the Core Study Phase continued into the optional OLE Study Phase.

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: Randomized population = 614, of which 10 were not dosed, leaving 604 participants. A total 321 of the 392 (81.9%) subjects who completed the Core Study Phase continued into the optional OLE Study Phase, including 130 in the BMT group (BMT-BMT) and 191 in the PBO group (PBO-BMT)

6. Primary Endpoint

The registry lists the primary outcome as Efficacy of a Fixed Dose of Bremelanotide as Measured by FSFI (Question Q1 and Q2), 28-day Recall., measured over 8 weeks baseline plus 24 weeks (Main Study), 52 Weeks (OLE).

The primary endpoint is change from baseline in the desire domain of the Female Sexual Function Index (FSFI), using Questions Q1 and Q2 and a 28-day recall period. The score is described as ranging from 1.2 to 6; a higher score represents increased sexual desire and is identified as a better outcome. The endpoint is a continuous measure, and the registry identifies the parameter as a mean with standard deviation as its dispersion measure.

A change score expresses how a participant's measured score at the end of the study differs from the baseline score. Comparing changes between randomized groups can help distinguish a difference associated with assignment to the study intervention from changes that may also occur in the placebo group. This is a general explanation of the role of a baseline-to-endpoint comparison, not a statement about the result of this trial.

The endpoint is relevant to the registered condition because it focuses on sexual desire, and the stated direction of the scale makes interpretation of score changes possible. The registry identifies it as a co-primary endpoint. It describes an 8-week baseline plus 24 weeks for the main study and 52 weeks for the open-label extension, but does not report a numerical primary endpoint estimate, a between-group contrast, or a confidence interval. The registry also does not clarify in the posted information how the co-primary designation was incorporated into the analysis.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry describes a continuous primary measure analyzed in terms of a mean and standard deviation. It does not report the actual statistical model, the primary comparison estimate, or a test result. General statistical explanations below describe what these concepts mean; they do not establish which procedures were used in this trial.

What does a mean describe?

For a continuous outcome, a mean is a summary of the observed scores in a group. When the outcome is change from baseline, the mean change summarizes participants' score changes according to the analysis used. A comparison of mean changes between randomized groups can quantify the average difference in change. The registry names a mean parameter, but does not report group means or an estimated between-group difference.

The standard deviation describes variation among individual observations around a group's mean. It is not the same as the uncertainty in an estimated mean or in a between-group difference. A confidence interval, when reported, gives a range of values compatible with the data and the stated statistical procedure. No confidence interval for the primary endpoint is reported in the registry record.

Why does randomization matter?

In general, random assignment is intended to make study groups comparable with respect to factors that could affect outcomes, including factors not measured by investigators. A parallel-group design compares groups assigned to different study conditions over the study period. A placebo control provides a comparator under the study's masking arrangement. These design features support interpretation of a between-group contrast, but do not guarantee that all enrolled participants contribute outcome data or that the analysis is free from bias.

The record labels the study randomized, triple-masked, and parallel. It does not report the randomization procedure, allocation concealment, the roles covered by triple masking, or details of the primary analysis. Those particulars cannot be inferred from the design labels alone.

How should the change score be understood?

A baseline-to-endpoint change is a way to describe how a measured score shifts over time. The primary endpoint is identified as change from baseline in the FSFI desire domain based on Questions Q1 and Q2. Comparing change between assigned groups is different from describing change within one group: the randomized comparison asks whether the pattern of change differs between the bremelanotide and placebo groups. The record does not provide the numerical estimates needed to make that comparison.

General considerations for continuous-outcome analysis include how baseline scores are incorporated, which endpoint visit is selected, and how repeated or missing measurements are handled. The registry information does not report those methods. It also does not identify a covariate-adjustment strategy, model assumptions, or the analysis population for the primary endpoint.

What do the planning assumptions mean?

The registry describes a sample-size planning illustration based on the observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It says that the effect to detect is a stated fraction of the observed effect. Such calculations are planning illustrations, not observed power and not trial results. The registry does not report the numerical sample-size illustration or the observed effect used in it in the information available for this analysis. No participant count for a particular power target can therefore be stated.

More generally, a sample-size calculation depends on assumptions about the outcome variability, target effect, significance level, allocation, and analysis. A planning estimate is conditional on those assumptions; it does not show that a study had a particular probability of finding a result after the data were collected. The registry's description alone does not supply a protocol-specific sample-size justification, and that justification is not reported in the registry record.

What can a significance test establish?

In general, a significance test evaluates how compatible data are with a specified null hypothesis under the test's assumptions. A p-value is not the probability that the null hypothesis is true, nor does it measure the size or importance of an effect. Interpretation is stronger when the estimate and its confidence interval are considered alongside any test result. The primary endpoint's test statistic and p-value are not reported in the registry record, so no test-based conclusion can be drawn from the posted information.

The registry lists 15 outcome measures and 0 analyses. The list of outcomes does not reveal which analyses were conducted or reported. The registered primary outcome is also flagged as having several categories or time points and not reduced to one comparison. Without analysis details and numerical results, it is not possible to determine how the primary endpoint was operationalized across visits or how its co-primary status was handled.

10. Multiplicity, Missing Data and Analysis Populations

The registry identifies the FSFI desire-domain outcome as a co-primary endpoint and lists multiple secondary outcomes, including satisfying sexual events, desire and satisfaction ratings, sexual distress, sexual function, arousal, and concern about difficulty with arousal. Testing multiple endpoints or time points can increase the chance of obtaining at least one apparently notable result by chance. General methods for addressing multiplicity include adjusting significance thresholds or defining a testing sequence in advance. The registry information does not report a multiplicity-control strategy, a hierarchy for co-primary endpoints, or the results of any such strategy.

The record lists 15 outcome measures and 0 analyses. It also flags the primary outcome as having several categories or time points and not being reduced to one comparison. The specific analysis population, selected time point, statistical model, and handling of multiple comparisons are not reported in the registry record. It is therefore not possible to determine which comparisons were conducted or how their results should be interpreted together.

Missing outcome data can affect a comparison if availability of measurements is related to treatment assignment or outcome. General approaches may use assumptions about the missingness process and may include sensitivity analyses to assess how conclusions change under different assumptions. The registry does not report the amount or pattern of missing primary endpoint data, the method used to handle missingness, or any sensitivity analyses. No missing-data method should be inferred from the endpoint description.

The record reports that 321 of the 392 subjects who completed the Core Study Phase continued into the optional OLE Study Phase, reported as 81.9%. This describes extension participation among core-study completers; it does not identify the main-study primary analysis population or explain why other participants did not continue. The analysis populations and participant disposition for the primary comparison are not reported in the registry record.

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 2% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: cholecystitis (0.8%), colon cancer (0.3%), anemia (0.3%), pregnancy (0.3%), abdominal pain (0.3%).

The registry reports serious adverse events and other adverse events separately for the main study and the open-label extension. In the main study, the bremelanotide group had 3 participants with serious adverse events among 303 at risk, and the placebo group had 2 among 301 at risk. The record reports 224 participants with other adverse events among 303 at risk in the bremelanotide group, and 137 among 301 at risk in the placebo group.

For the OLE groups, the record reports 1 participant with a serious adverse event among 130 at risk in the bremelanotide group, and 0 among 191 at risk in the placebo group. Other adverse events were reported for 93 participants among 130 at risk in the bremelanotide OLE group and 142 among 191 at risk in the placebo OLE group. No deaths are reported in any of the listed groups.

These counts describe the registry's event summaries; they are not a randomized efficacy comparison. The extension was open-label, and the record does not provide enough detail to establish whether exposure duration, event collection, or group composition was comparable across its groups. The serious-event terms listed include cholecystitis at 0.8%, and Colon cancer, Anemia, Pregnancy, Abdominal Pain, and Pneumonia at 0.3% each. The registry information does not specify the denominators or group attribution for these listed percentages. Event counts alone do not establish causation or comparative risk.

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

The registry establishes that a Phase 3 randomized, placebo-controlled trial was conducted to evaluate bremelanotide in premenopausal women with hypoactive sexual desire disorder. It identifies the main-study intervention, placebo comparator, masking and parallel-group design, and the FSFI desire-domain change as a co-primary endpoint. It also records an optional open-label extension and provides some adverse-event counts.

Those details describe the study question and design, but they do not answer whether the primary endpoint differed between groups. The numerical primary endpoint results, estimated treatment contrast, confidence interval, and test result are not reported in the registry record. It is therefore not possible to state the direction or magnitude of the primary comparison, or how precisely it was estimated. The scale's stated meaning does not replace outcome data.

Likewise, the enrollment count does not establish how many participants contributed to the primary analysis. The record does not report the analysis population, missing-data methods, or multiplicity strategy. The extension participation figures describe continuation among core-study completers, not the result of the randomized primary comparison. Open-label extension observations should be kept distinct from the blinded placebo-controlled phase.

The adverse-event counts provide descriptive safety information as registered, but do not show that an event was caused by a study treatment or establish a comparative safety effect. They also cannot be used to infer efficacy. A complete interpretation of the trial would require the endpoint estimates and their uncertainty, along with the analysis methods and participant flow; those details are not reported in the registry record.

Thus, the record supports a description of what was planned and which measures were registered, alongside the reported safety counts. It does not support a conclusion about the efficacy result, its clinical importance, or the robustness of an endpoint comparison. This distinction is important: the presence of a completed trial record is not itself a report of the trial's primary statistical findings.

13. Why This Trial Matters Statistically

This trial is statistically notable for the contrast between a clearly described randomized design and the absence of a reported primary endpoint estimate in the registry information. The record specifies the population, a placebo-controlled parallel-group structure, a continuous outcome, its direction, and the planned study periods. Those elements help define what a valid comparison would need to address, even though they do not reveal its result.

The endpoint uses a score with an explicit range and interpretation: higher FSFI desire-domain scores represent increased sexual desire and are defined as better. That makes the outcome's direction readable, but a scale definition is not an effect estimate. Statistical interpretation still depends on the observed group changes, between-group difference, and uncertainty, which are not reported in the registry record.

The registry also identifies the primary outcome as co-primary and signals that it involves several categories or time points. Such features make the analysis plan relevant to interpretation: readers need to know how the endpoint and its time points were selected and how multiplicity was addressed. Those methods and the numerical analyses are not reported in the registry record. The status listing of 0 analyses further limits what can be concluded from the available record details.

Finally, the optional open-label extension illustrates why phases with different masking and comparator structures should not be treated as interchangeable. The extension participation count and safety summaries provide context, but do not replace the main randomized comparison. Statistically, the case underscores the difference between having an identifiable design and having sufficient posted results to evaluate an 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 of the registry information is that it does not report the numerical primary endpoint results. There is no group-specific change, between-group estimate, confidence interval, or significance test with which to describe the primary comparison. The scale direction is stated, but the size and precision of any observed difference cannot be assessed.

The analysis plan is also incompletely described. The record identifies a co-primary endpoint and notes several categories or time points, but does not report the exact primary analysis, endpoint visit selection, model, multiplicity procedure, or analysis population. It does not report missing-data methods, the amount or pattern of missing measurements, or sensitivity analyses. These omissions prevent assessment of how robust a primary estimate might be even if one were available.

The overall enrollment count should not be mistaken for the primary endpoint analysis sample. The record gives extension continuation among core-study completers, but does not describe participant flow or reasons for non-continuation in the information presented. It also does not report adherence, treatment exposure, or the extent to which participants followed the as-needed dosing instructions.

Safety information is limited to the registered counts and listed serious-event terms. The record does not provide the detailed event definitions, exposure-adjusted summaries, or sufficient attribution information for the listed percentages. The open-label extension is a different design context from the blinded main study, so its group summaries should not be interpreted as if they came from the same randomized comparison. These limitations arise from the registry information available and do not establish whether additional results exist elsewhere.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: 2. Study to Evaluate the Efficacy/Safety of Bremelanotide in Premenopausal Women With Hypoactive Sexual Desire Disorder — NCT02338960.
• Registry record last updated 2021-01-28; status last verified 2020-12.
• Results first posted 2021-01-28.
• 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 NCT02338960 as a completed Phase 3 randomized, triple-masked, parallel-group, placebo-controlled trial sponsored by Palatin Technologies, Inc. Enrollment was 714. The main study compared as-needed subcutaneous bremelanotide with placebo over 24 weeks, with an optional open-label extension.

The registered co-primary endpoint was change from baseline in the FSFI desire domain based on Questions Q1 and Q2, using a 28-day recall. Higher scores on the stated scale represent increased sexual desire and a better outcome. The primary endpoint result and its statistical comparison are not reported in the registry record. The record lists 15 outcome measures and 0 analyses.

Serious and other adverse-event counts are reported for the main study and extension groups, with no deaths listed. The registry also reports that 321 of 392 core-study completers continued into the extension, or 81.9%. These descriptive details do not provide the missing primary efficacy estimate or its precision.