A Safety and Efficacy Study of BBI-4000 in Subjects With Axillary Hyperhidrosis: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for A Safety and Efficacy Study of BBI-4000 in Subjects With Axillary Hyperhidrosis (NCT02336503), a randomized phase 2 study. The primary outcome is Proportion of Subjects Achieving at Least 2-point Decrease in Hyperhidrosis Disease Severity Scale (HDSS) From Baseline to Week 4.

2. Clinical Question

The question concerns a threshold-defined change in reported sweating severity, rather than a comparison of average HDSS scores. The trial also lists a secondary HDSS responder outcome using a different improvement threshold, along with absolute and percent change in gravimetrically measured sweat production. These outcomes address related but not identical aspects of symptoms and sweat production. Their inclusion does not establish what the results were or which comparisons were statistically tested.

The listed study population is subjects with axillary hyperhidrosis. The registry record does not report further eligibility details in the available information. The primary endpoint description specifies that participants needed reported scores at baseline and Day 29 for inclusion in its described population; the record does not provide the number meeting that condition in each group.

3. Trial Design

This completed phase 2 study is described as multicenter, randomized, double blind, vehicle-controlled, and parallel. The registry record labels masking as quadruple, but does not specify which people or roles were masked. Randomization assigns participants to treatment groups by chance; a parallel design means participants receive their assigned group’s treatment rather than switching among treatments as part of the design. These are general descriptions of the design terms, not additional details about how this trial was conducted.

The four treatment arms were BBI-4000 Gel at 5%, 10%, and 15%, and vehicle. The study summary describes application once a day for 4 weeks. The primary endpoint timeframe is baseline through Week 4 (Day 29). Enrollment is recorded as 189. That enrollment figure does not provide arm-specific numbers for the primary analysis, and it should not be used to infer how many participants contributed endpoint data.

The registry lists a start date of 2015-03-10 and a primary completion date of 2015-11-05, and the overall status is completed. These dates describe the record’s study timeline; they do not indicate when results were analyzed or posted.

Randomization and masking can reduce some sources of bias, while a vehicle group provides a comparison against the study formulation without the listed active concentrations. Those are general design considerations. The registry record does not report details such as the randomization procedure, the allocation concealment process, the masking procedures, adherence, or the duration of follow-up beyond the stated endpoint timeframe. It also does not report a prespecified primary comparison among the four groups.

4. Participant Flow and Accounting

The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.

Overall Study

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: Randomized subjects with reported scores on Baseline and Day 29 (week 4), Count of participants includes number of participants who did not report at-least a 2-point decrease in score (NO) and number of participants who did report at-least a 2-point decrease in score (YES); proportions were calculated as percentages = (# of participants/total # of participants that provided baseline and day 29 HDSS scores) x 100%

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: Population (N=189) was defined as all randomized subjects

6. Primary Endpoint

The registry lists the primary outcome as Proportion of Subjects Achieving at Least 2-point Decrease in Hyperhidrosis Disease Severity Scale (HDSS) From Baseline to Week 4, measured over Baseline through Week 4 (Day 29).

The primary endpoint is the proportion of participants achieving at least a 2-point decrease in Hyperhidrosis Disease Severity Scale (HDSS) score from baseline through Week 4 (Day 29). The HDSS is described as a severity scale with scores from 1 to 4: a score of 1 represents sweating that is never noticeable and never interferes with daily activities, while a score of 4 represents sweating that is intolerable and always interferes with daily activities. The endpoint turns change on that scale into a yes-or-no responder classification using the stated improvement threshold.

As a general statistical approach, a responder endpoint can make the result easier to interpret as the share of participants reaching a chosen degree of improvement. It also discards information: participants just short of the threshold and those with little or no change may be grouped together as nonresponders, even if their changes differ. A threshold-based proportion therefore answers a different question from the average change in score, and its interpretation depends on the threshold and on how missing scores are handled.

The registry description says the endpoint population consists of randomized subjects with reported scores at baseline and Day 29. It describes counting both participants who did and did not reach the threshold, but does not report the arm-level counts, proportions, confidence intervals, or test results. The record also lists a secondary HDSS outcome using at least a 1-point improvement; that is a separate responder definition, not a substitute for the primary endpoint result.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The design and endpoints permit several useful statistical concepts to be explained, but the registry record does not report the trial’s detailed analysis plan or an analysis of the primary endpoint. The explanations below are general; they should not be read as a description of methods actually used for this study unless explicitly identified in the record.

What does randomization contribute?

In general, random assignment aims to make treatment groups comparable with respect to both measured and unmeasured participant characteristics, on average. This supports interpreting a between-group contrast as an effect associated with assigned treatment, provided the trial is conducted and analyzed appropriately. Randomization does not guarantee exact balance in a particular trial, nor does it resolve missing outcome data or problems with adherence. The record identifies the allocation as randomized but does not describe the sequence generation, concealment, stratification, or implementation procedures.

What does the responder endpoint estimate?

The primary outcome classifies each eligible participant according to whether the HDSS score decreased by at least 2 points from baseline through Week 4 (Day 29). A group proportion would describe the share of analyzed participants meeting that criterion. A comparison between groups could be expressed as a difference in proportions, a ratio of proportions, or another measure, depending on the prespecified analysis. These are general possibilities, not reported methods or results for this trial. The registry record does not state which treatment contrast or effect measure was intended.

A binary responder outcome can be analyzed using methods designed for categorical data. The method should match the estimand, the group structure, and the data available. For example, an unadjusted comparison and a model adjusted for baseline variables do not necessarily produce the same estimate. The registry record does not report whether any adjustment was planned or performed, which covariates might have been used, or how results were to be presented.

Why do precision and uncertainty matter?

General statistical interpretation considers both the estimated effect and its uncertainty. A confidence interval indicates a range of values compatible with the data under the specified method and assumptions. A narrow interval usually indicates greater precision than a wide interval, but neither width nor direction can be assessed without a reported estimate and interval. Statistical significance is not a measure of clinical importance, and a non-significant result does not by itself establish equivalence or absence of an effect. For this study, the record does not report primary endpoint confidence intervals, p-values, or a prespecified threshold for interpretation.

What do the planning assumptions mean?

The record describes planning illustrations 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 the effect to detect is a stated fraction of the observed effect. These are planning assumptions, not evidence that the trial had a particular probability of detecting an effect. Sample-size planning values are hypothetical illustrations under assumptions; they are not observed power and do not show what the actual analysis found. No resulting planning values are reported in the registry record, and the observed effect needed to anchor such illustrations is not reported either.

The record’s equal-allocation assumption concerns a two-arm planning illustration. It should not be confused with the listed trial structure, which has three BBI-4000 concentrations and a vehicle group. The record does not state how the planning illustration relates to a particular pairwise contrast, or whether the trial’s original design used the same assumptions.

How should multiple groups and outcomes be considered?

In general, testing several treatment contrasts or outcomes can increase the chance of at least one apparently positive result if each test is judged separately without accounting for multiplicity. Methods such as prespecifying a primary contrast, adjusting significance thresholds, or using a hierarchical testing strategy can address this concern, but the registry record does not report whether any such approach was planned or applied. The presence of multiple groups and listed outcomes alone does not identify the analysis strategy.

The record lists four outcome measures and zero analyses. This means the registry material identifies outcome measures but does not include reported statistical analyses in the available information. It does not establish whether analyses were conducted elsewhere, whether results were withheld, or what methods the investigators used. Those details are not reported in the registry record.

10. Multiplicity, Missing Data and Analysis Populations

The study has four treatment arms and lists a primary HDSS responder outcome alongside secondary outcomes for a different HDSS response threshold and gravimetrically measured sweat production. General statistical practice treats multiple outcomes and multiple treatment comparisons as a potential multiplicity issue: if many tests are performed and interpreted separately, the chance of at least one misleading positive finding can increase. A prespecified comparison strategy or multiplicity adjustment can help manage that issue. The registry record does not report whether the trial used an adjustment, a testing hierarchy, or another multiplicity strategy.

The primary endpoint description defines its population as randomized subjects with reported scores at baseline and Day 29. This indicates that both measurements are relevant to the endpoint classification. However, the record does not give the number of participants with data at both visits by group, the number or reasons for missing scores, or the approach to missing observations. It does not report imputation, sensitivity analyses, or how participants lacking one or both scores were treated.

The information therefore does not establish whether the primary results, if analyzed, would represent all randomized participants or only participants meeting the endpoint’s reporting requirement. The analysis populations for the secondary outcomes are also not reported in the registry record. These omissions limit assessment of potential attrition bias and the reproducibility of an analysis. They should not be resolved by assuming a particular missing-data method or analysis set.

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.

Most frequent non-serious adverse event terms

The registry safety record lists serious adverse events separately from other adverse events and deaths. For serious adverse events, the affected count is 0 in the BBI-4000 Gel, 5%, 10%, and 15% groups and 0 in the vehicle group. The record lists 47, 48, 48, and 46 participants at risk, respectively. Deaths are recorded as 0 in every group.

For other adverse events, the number of participants affected is listed as 8 for BBI-4000 Gel, 5%, 12 for BBI-4000 Gel, 10%, 14 for BBI-4000 Gel, 15%, and 6 for vehicle. The corresponding at-risk counts are 47, 48, 48, and 46. These are counts of participants affected, not a measure of event severity or a statistical comparison. The record does not give event-level descriptions in the available safety information; the listed serious-event terms are empty.

A zero serious-event count means no serious events are recorded for that group in this safety record. It does not establish that the risk is zero, and counts by themselves do not quantify uncertainty or establish a difference between groups. The record does not report comparative safety estimates, confidence intervals, or a formal safety analysis.

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

The record describes a randomized, masked, vehicle-controlled phase 2 study of three topical BBI-4000 concentrations in subjects with axillary hyperhidrosis. Its primary question is whether participants achieved a threshold-defined improvement in HDSS score from baseline through Week 4 (Day 29). That design and endpoint are useful context for understanding what the study set out to assess.

The available registry information does not report the primary endpoint counts or proportions by group, a treatment effect estimate, a confidence interval, or a statistical test. As a result, it does not show whether the groups differed on the stated endpoint or how precise any estimated difference was. It also does not support a conclusion about the relative results of the three concentrations and vehicle. A completed study status is not a substitute for endpoint results.

The safety information records no serious adverse events or deaths in the groups and provides counts of participants affected by other adverse events. These observations describe the posted safety counts only. They do not establish that there were no safety concerns of any kind, and they do not determine whether safety differed between groups.

In general, an endpoint result should be interpreted with its denominator, effect estimate, uncertainty, analysis population, and missing-data handling in view. Those details are not reported for the primary outcome in the registry record. The record also does not report the statistical analysis plan or how multiple outcomes and comparisons were handled. Without those elements, the posted information supports a description of study design and recorded safety counts, but not a quantitative interpretation of treatment efficacy.

These limits concern what can be concluded from the registry record; they do not establish that analyses were never conducted or that results were not reported elsewhere. Such information is not reported in the registry record.

13. Why This Trial Matters Statistically

This trial is statistically informative as an example of the distinction between a registered research question and a reported result. The record specifies a randomized, parallel, vehicle-controlled study with three active concentrations and a threshold-based HDSS endpoint. Yet the endpoint counts and comparative analyses are not reported in the available registry material. Knowing the design does not reveal the observed treatment contrast.

The responder definition also illustrates a general feature of clinical outcome analysis. Converting a scale into a threshold-based yes-or-no outcome can make a result understandable as the proportion meeting a specified criterion. At the same time, it removes information about the range of score changes and makes the result dependent on the chosen cutoff. The listed secondary HDSS outcome uses a different improvement threshold, reinforcing that outcome definitions matter when interpreting or comparing analyses.

Multiple concentrations, a vehicle group, and several listed outcomes make it important to know which comparisons were prespecified and how multiplicity was handled. The registry record does not report those methods. This matters because readers cannot infer the statistical plan solely from the number of arms or outcomes.

The study also highlights why endpoint denominators and missing-data procedures are essential. The primary description requires baseline and Day 29 scores, but group-specific numbers meeting that requirement are not reported. Without those details, readers cannot assess the relationship between enrollment and the analyzed population or judge possible effects of missing measurements.

These are general lessons in reading clinical trial records: distinguish the question from its answer, the count from a comparison, and design features from evidence about an outcome. Here, the record permits a clear account of the trial structure and selected safety counts, while leaving the primary statistical result unreported.

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 central limitation is that the registry record identifies the primary endpoint but does not report its arm-level results. There are no reported yes-or-no responder counts, proportions, treatment-effect estimates, confidence intervals, or p-values. The primary endpoint description names a population requiring reported baseline and Day 29 scores, but gives no group-specific denominator for that population.

The record lists secondary outcomes but does not report their estimates or analyses. It also does not report the prespecified treatment contrasts, statistical model, adjustment variables, multiplicity strategy, or analysis populations. The record’s planning assumptions describe illustrations based on an observed effect, but the observed effect and resulting planning values are not reported. The trial’s original sample-size justification is not reported in the registry record.

Information about missing scores, participant flow by group, and the handling of missing outcomes is not reported. The masking designation is recorded as quadruple, but the masked roles and procedures are not described. Additional eligibility and treatment-adherence details are not reported in the available information.

For safety, the record includes serious-event, other-event, and death counts, but does not provide comparative statistical estimates or event-level descriptions in the available information. These limitations prevent a complete assessment of endpoint precision, possible attrition effects, the intended analysis strategy, or comparative safety. They describe gaps in the registry record and should not be interpreted as proof that the information does not exist elsewhere.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: A Safety and Efficacy Study of BBI-4000 in Subjects With Axillary Hyperhidrosis — NCT02336503.
• Registry record last updated 2023-03-15; status last verified 2022-12.
• Results first posted 2023-03-15.
• 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

NCT02336503 is a completed phase 2, multicenter, randomized, double-blind, vehicle-controlled, parallel study of topical BBI-4000 in subjects with axillary hyperhidrosis. The four arms were BBI-4000 Gel at 5%, 10%, and 15%, and vehicle. The study summary describes once-daily application for 4 weeks; enrollment is recorded as 189.

The primary endpoint is the proportion achieving at least a 2-point HDSS decrease from baseline through Week 4 (Day 29). Secondary outcomes include a different HDSS responder threshold and absolute and percent change in gravimetrically measured sweat production. The registry record does not report arm-level outcome results or statistical analyses.

Serious adverse events and deaths are recorded as 0 in each group. Other adverse-event counts are reported by group. The registry record does not report the detailed analysis plan, multiplicity handling, or missing-data methods.