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 A Study of the Safety, Tolerability, and Efficacy of Topical SM04554 Solution in Male Subjects With Androgenetic Alopecia (AGA) (NCT02275351), a randomized phase 2 study. The primary outcome is Change From Baseline in Non-vellus Hair Count.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02275351 |
| Phase | Phase 2 |
| Condition | Androgenetic Alopecia |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | Double |
| Enrollment | 302 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Biosplice Therapeutics, Inc. |
| Results posted | Yes |
2. Clinical Question
Population
Male subjects with androgenetic alopecia (AGA). The registry record describes the primary analysis population as subjects who received at least one dose of study treatment; it does not give the analyzed number for each primary-outcome group.
Intervention
Topical SM04554 solution at 0.15% or 0.25%, applied to the scalp once a day for 90 days.
Comparator
Topical vehicle solution, also applied once a day for 90 days.
Primary question
How did non-vellus hair count change from baseline over time in the treatment groups? The listed assessment days are Day 45, Day 90, and Day 135.
The clinical question is whether the change in non-vellus hair count differed among the two SM04554 concentrations and vehicle during the recorded assessment period. The trial also set out to characterize safety and tolerability. The registry record lists secondary outcomes concerning investigator-assessed hair growth, quality of life impact, and total sum of hair width.
The outcome description specifies a measurement process based on standardized macrophotography of a target area. That makes the primary question about a count measured from images, rather than a general judgment of appearance. The record says image assessments were conducted by a vendor using blinded assessment. It does not report the actual group results, the size of any treatment differences, or uncertainty intervals. Therefore, the page can describe the question and how the outcome was collected, but cannot establish from this record whether the groups differed.
3. Trial Design
This was a Phase 2, randomized, double-blind, parallel-group study of topical SM04554 in male subjects with androgenetic alopecia. The registry record describes three groups: SM04554 0.15%, SM04554 0.25%, and vehicle. Each solution was applied once a day for 90 days. Overall enrollment was 302. In the safety information, the groups at risk are listed as 102, 102, and 98, respectively; these are safety-group counts and should not automatically be treated as the primary-outcome analysis counts.
The study is marked completed. Its recorded start was 2014-11 and its primary completion was 2015-11. The registry record gives no more detailed account of recruitment, site-level conduct, or the timing of the final study visit beyond the outcome time points listed. The primary outcome was assessed at Day 45, Day 90, and Day 135, so the measurement schedule includes assessments during treatment and after the 90-day application period.
Randomization and double masking are stated, but the method used to generate or conceal assignments, the masking procedures, and whether masking was assessed are not reported in the registry record. Nor is the allocation ratio reported as a design specification. The 102, 102, and 98 safety counts do not replace that missing detail. The record identifies the study as multicenter in its official title, but it does not provide the number or locations of sites.
The brief summary describes the study purpose as characterizing safety, tolerability, and efficacy. The registry record does not report a protocol-level sample-size justification, planned primary contrast, or full statistical analysis plan. Those omissions limit how much can be inferred about the intended confirmatory role of the comparisons.
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 | SM04554 0.15% | SM04554 0.25% | Vehicle |
|---|---|---|---|
| STARTED | 102 | 102 | 98 |
| COMPLETED | 74 | 77 | 76 |
| NOT COMPLETED | 28 | 25 | 22 |
Reasons for not completing this period:
| Reason | SM04554 0.15% | SM04554 0.25% | Vehicle |
|---|---|---|---|
| Adverse Event | 11 | 13 | 12 |
| Lost to Follow-up | 4 | 4 | 1 |
| Protocol Violation | 10 | 3 | 3 |
| Withdrawal by Subject | 2 | 5 | 6 |
| Medication Dispensing Error | 1 | 0 | 0 |
Analysis population for the primary outcome, as defined in the registry: The overall number of participants analyzed includes all subjects who received at least one dose of study treatment.
5. Baseline Characteristics
The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.
Population: All subjects who received at least one dose of study treatment
| Characteristic | Unit | SM04554 0.15% | SM04554 0.25% | Vehicle | Total |
|---|---|---|---|---|---|
| Participants analysed | 102 | 102 | 98 | 302 | |
| Age, Continuous | years | 44.2 (SD 8.21) | 44.7 (SD 8.83) | 45.0 (SD 8.60) | 44.6 (SD 8.53) |
| Sex: Female, Male: Female | Participants | 0 | 0 | 0 | 0 |
| Sex: Female, Male: Male | Participants | 102 | 102 | 98 | 302 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 14 | 22 | 18 | 54 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 88 | 80 | 80 | 248 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 1 | 1 |
| Race (NIH/OMB): Asian | Participants | 1 | 1 | 0 | 2 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 1 | 2 | 0 | 3 |
| Race (NIH/OMB): Black or African American | Participants | 10 | 10 | 6 | 26 |
| Race (NIH/OMB): White | Participants | 89 | 88 | 90 | 267 |
| Race (NIH/OMB): More than one race | Participants | 1 | 1 | 1 | 3 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 | 0 |
| Region of Enrollment: United States | participants | 102 | 102 | 98 | 302 |
| Weight [kg] | kg | 95.90 (SD 17.334) | 94.15 (SD 18.320) | 92.50 (SD 19.535) | 94.21 (SD 18.394) |
| Height [cm] | cm | 178.64 (SD 7.802) | 177.58 (SD 7.731) | 177.46 (SD 7.246) | 177.90 (SD 7.595) |
| Norwood-Hamilton Classification Score: 4 | Participants | 29 | 36 | 35 | 100 |
| Norwood-Hamilton Classification Score: 5 | Participants | 9 | 14 | 17 | 40 |
| Norwood-Hamilton Classification Score: 5A | Participants | 18 | 11 | 22 | 51 |
| Norwood-Hamilton Classification Score: 5V | Participants | 26 | 22 | 14 | 62 |
| Norwood-Hamilton Classification Score: 6 | Participants | 20 | 19 | 10 | 49 |
6. Primary Endpoint
The registry lists the primary outcome as Change From Baseline in Non-vellus Hair Count, measured over Day 45, Day 90, and Day 135.
| Element | Registry entry |
|---|---|
| Result type | Mean |
| Measure of spread | Standard Deviation |
| Unit | Non-vellus hair count |
| Endpoint classification | Continuous Endpoint |
The primary endpoint is change from baseline in non-vellus hair count, measured at Day 45, Day 90, and Day 135. In general, a change-from-baseline endpoint compares a later measurement with the participant’s starting measurement. It can describe how the measured count evolves over time, although the registry record does not give the numerical changes or say how the change was modeled statistically.
The record says non-vellus hair count was determined using macrophotography. Standardized color photographs were taken of a treatment area. Before imaging, a circular area 1.9 cm in diameter was clipped to approximately 1 mm in length. A scalp tattoo approximately 1 mm in size was placed in the center of the target area at the baseline visit to anchor the photography. Images were uploaded from sites to a vendor for blinded assessment.
In general, a standardized image-based method can make a repeated count more consistent by defining the area being assessed and using a common capture procedure. Blinded assessment can reduce the risk that knowledge of treatment assignment influences image evaluation. These are general methodological considerations, not evidence that measurement error was absent in this study. The registry record does not report repeatability data, image-quality exclusions, or the exact counting rules.
The endpoint is relevant to the study’s stated question because it quantifies a feature of hair growth in a defined treatment area. It does not by itself summarize every aspect of hair appearance, quality of life, or safety. Those areas appear separately in the listed secondary outcomes and safety information.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies non-vellus hair count change as the primary outcome and lists assessments at Day 45, Day 90, and Day 135. It does not provide the outcome values by group, an estimated difference between groups, a confidence interval, or a p-value. As a result, the posted information does not allow a statistical estimate of the treatment contrasts or a judgment about their precision.
This distinction matters: a trial’s design and measurement procedures explain what was intended to be compared, but they do not show the observed efficacy result. Enrollment of 302 describes the overall number enrolled, not necessarily the number contributing a measurement at every visit. The primary analysis population is described as subjects who received at least one dose, but the registry record does not state the number analyzed by group or at each time point.
In general, a confidence interval expresses the range of effect estimates reasonably compatible with the data and the specified statistical model. A narrow interval indicates greater precision than a wide interval, while neither width nor statistical significance alone establishes clinical importance. No interval for the primary outcome is reported in the registry record, so precision cannot be assessed.
The record lists 66 analyses and five outcome measures. It does not describe which analyses correspond to which outcome, time point, or comparison, or whether a multiplicity adjustment was applied. When several outcomes, visits, or contrasts are examined, the chance of at least one apparently unusual result can increase if the analysis plan does not account for the collection of tests. This is a general statistical concern; the registry record does not say how it was handled in this trial.
The safety record lists serious adverse events in the vehicle group and none in either SM04554 group, alongside other adverse events in all three groups. Those counts describe events recorded under the registry’s categories; they are not a substitute for comparative efficacy estimates and do not establish causation. The record also lists no deaths. The sections below explain the design and these limits without making a treatment recommendation.
9. Statistical Methods Explained
The record identifies the primary measure as a mean, with standard deviation as the dispersion measure, for change from baseline in non-vellus hair count. It does not provide the group means or standard deviations in the information. The endpoint is assessed at three listed time points, so a complete interpretation would ordinarily consider both the values at each visit and how the measurements relate across visits. The registry record does not state the model or test used for that purpose.
What does a mean describe?
In general, the mean summarizes the central value of a set of observations. For a change-from-baseline measure, a group mean would summarize the average observed change among participants included in the relevant analysis. A mean can be useful for comparing groups, but by itself it does not show whether individual participants had similar changes or widely different ones. It is also sensitive to unusually large or small observations.
What does the standard deviation add?
In general, the standard deviation describes the spread of individual measurements around their mean. It is not the same as a confidence interval for the mean and is not, by itself, a measure of uncertainty in a treatment contrast. To evaluate a difference between groups, readers also need information about the analysis population, the relevant group estimates, and the method used to estimate uncertainty. Those primary-outcome results and details are not reported in the registry record.
Why are the repeated time points important?
Repeated measurements can show whether an outcome’s pattern changes over time. In general, observations from the same participant are correlated: a participant’s measurements at different visits are not independent in the way measurements from unrelated participants might be. Statistical methods for repeated measures can account for this relationship and use available observations under specified assumptions. The registry record does not identify whether such a method was used, whether each visit was analyzed separately, or whether one visit was designated as the main comparison.
Day 45 and Day 90 fall within the stated 90-day application period, while Day 135 is also listed as an assessment. This schedule makes it possible to describe measurements across the recorded period, but it does not establish what happened between visits or after the last listed assessment. The record does not state whether the Day 135 result was a primary time point, a follow-up measure, or part of a joint statistical model.
What does randomization contribute?
In general, random allocation aims to make treatment groups comparable at the start of a trial, on average, with respect to both measured and unmeasured characteristics. This supports a fairer comparison than treatment selection based on participant or investigator preference. Randomization does not guarantee identical groups, and it does not prevent bias caused by missing measurements, deviations from assigned treatment, or analysis decisions made after seeing results. The registry record says the study was randomized but does not describe the allocation procedure or the analysis strategy in enough detail to evaluate those issues.
What does double masking contribute?
In general, masking can reduce the influence of treatment knowledge on participants’ reports, investigator assessments, and study conduct. It is especially pertinent when an outcome involves image assessment or subjective judgments. The primary endpoint’s images were assessed by a vendor under blinded assessment, according to the record. The record does not explain who else was masked, how the solutions were made indistinguishable, or whether masking was maintained throughout the study.
How should the sample-size material be read?
The page includes a calculated sample-size section. Such values are planning illustrations based on assumptions, not observed trial outcomes and not the trial’s original sample-size justification. In general, a planning calculation asks how many participants per arm might be needed to detect a specified effect under assumptions about significance level, allocation, and the statistical model. The record’s assumptions describe a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and an effect expressed as a fraction of an observed effect. However, the primary outcome has several categories or time points and is not reduced to one comparison. The registry record does not report the trial’s own sample-size justification. Planning illustrations should therefore not be read as observed power, proof of adequate power, or evidence that a clinical effect was present.
In general, over-dispersion means that observed variability is greater than a statistical model expects. It can make uncertainty estimates too small if the model does not allow for the extra variation. This is a general issue for count outcomes, but the registry record does not state that over-dispersion was assessed or that a count model was used in this study. It would be inappropriate to infer a particular model from the outcome being a count.
Finally, the record lists 66 analyses, but does not identify their statistical procedures or explain how they were selected. The methods needed to interpret a reported estimate include the analysis population, handling of missing data, model specification, contrast definition, and uncertainty calculation. Those details are not reported in the registry record. Consequently, the endpoint description supports an account of measurement, but not a reconstruction of the trial’s inferential analysis.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists three secondary outcome topics: investigator-assessed change in hair growth, impact of AGA on quality of life, and change from baseline in total sum of hair width. It also lists five outcome measures and 66 analyses. The record does not map each analysis to an outcome, visit, treatment contrast, or analysis population. It does not say whether the primary endpoint was tested at each listed visit separately or through one overall model.
When several statistical tests are conducted, multiplicity is a general concern. Without an adjustment or a clearly prioritized testing sequence, the chance of a false-positive result across a family of tests may be higher than the chance attached to any single test. This does not mean that any particular result is false; it means that the interpretation depends on knowing what was planned and how the set of analyses was handled. The registry record does not report a multiplicity strategy.
Missing data also affect interpretation, especially for a repeated outcome. The record describes the primary analysis population as all subjects who received at least one dose of study treatment, but does not report the number with measurements at each visit, reasons for missing measurements, or methods for handling missingness. It does not state whether analyses followed assigned treatment, treatment received, or another rule. These analysis-population and missing-data details are not reported in the registry record.
In general, an analysis that uses only participants with observed measurements can be biased if the reasons for missingness are related to the outcome or treatment. Statistical methods can address some missing-data patterns under assumptions, but no particular method or assumption should be attributed to this trial without documentation. The absence of these details means readers cannot evaluate how sensitive the primary result might be to missing observations.
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 |
|---|---|---|---|
| SM04554 0.15% | 0 | 102 | 0.0% |
| SM04554 0.25% | 0 | 102 | 0.0% |
| Vehicle | 1 | 98 | 1.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| SM04554 0.15% | 0 of 102 |
| SM04554 0.25% | 0 of 102 |
| Vehicle | 0 of 98 |
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 |
|---|---|
| SM04554 0.15% | 45 of 102 |
| SM04554 0.25% | 46 of 102 |
| Vehicle | 47 of 98 |
Most frequent serious adverse event terms
| Event term | Organ system | SM04554 0.15% | SM04554 0.25% | Vehicle |
|---|---|---|---|---|
| Small intestinal obstruction | Gastrointestinal disorders | 0 of 102 | 0 of 102 | 1 of 98 |
Most frequent non-serious adverse event terms
| Event term | Organ system | SM04554 0.15% | SM04554 0.25% | Vehicle |
|---|---|---|---|---|
| Application site erythema | General disorders and administration site conditions | 5 of 102 | 7 of 102 | 5 of 98 |
| Application site pruritus | General disorders and administration site conditions | 6 of 102 | 4 of 102 | 6 of 98 |
| Application site paraesthesia | General disorders and administration site conditions | 6 of 102 | 3 of 102 | 6 of 98 |
| Skin exfoliation | Skin and subcutaneous tissue disorders | 2 of 102 | 3 of 102 | 6 of 98 |
| Nausea | Gastrointestinal disorders | 1 of 102 | 3 of 102 | 2 of 98 |
| Pruritus | Skin and subcutaneous tissue disorders | 1 of 102 | 4 of 102 | 1 of 98 |
| Headache | Nervous system disorders | 1 of 102 | 1 of 102 | 3 of 98 |
| Diarrhoea | Gastrointestinal disorders | 2 of 102 | 1 of 102 | 2 of 98 |
| Nasopharyngitis | Infections and infestations | 2 of 102 | 1 of 102 | 2 of 98 |
| Aspartate aminotransferase increased | Investigations | 2 of 102 | 2 of 102 | 1 of 98 |
Serious events with the highest arm-level rate: small intestinal obstruction (1.0%).
The safety figures list 102 subjects at risk in the SM04554 0.15% group, 102 in the SM04554 0.25% group, and 98 in the vehicle group. Serious adverse events affected 0, 0, and 1 subjects, respectively. The registry record lists no deaths in any group. The serious event term shown is Small intestinal obstruction, with 1.0% displayed.
The figures also list other adverse events affecting 45 subjects in the 0.15% group, 46 in the 0.25% group, and 47 in the vehicle group. These are counts of affected subjects, not event rates calculated. The record’s figures describe what was recorded in the safety categories; they do not establish that a treatment caused an event or that groups are equivalent in safety.
In general, safety comparisons depend on the duration of observation, event definitions, completeness of reporting, and the number of participants exposed. The registry record does not provide detailed event narratives, exposure-adjusted analyses, or a full breakdown of the other adverse-event terms. The small number of serious events also limits what can be inferred about uncommon harms. These data should be read as a registry summary, not as a complete safety evaluation.
12. What the Result Does, and Does Not, Mean
This record establishes that a randomized, double-blind Phase 2 study compared two concentrations of topical SM04554 with vehicle in male subjects with androgenetic alopecia. It specifies once-daily application for 90 days and a primary outcome based on change from baseline in non-vellus hair count at Day 45, Day 90, and Day 135. It also describes a standardized macrophotography procedure and blinded vendor assessment.
Those details explain the trial question and its measurement approach. They do not show the answer to the primary question. The registry record does not report group-level primary-outcome estimates, treatment contrasts, confidence intervals, or p-values in the registry record. Without them, there is no basis to characterize the direction, size, or precision of an efficacy difference.
In general, statistical evidence is best interpreted by considering the estimated effect and its uncertainty, not by treating a single threshold as the whole result. Clinical importance is a separate question from statistical evidence and depends on context that is not described in this record. Neither can be determined from the design description alone.
The safety counts provide a limited descriptive picture: the registry lists one serious adverse event in the vehicle group, none in either SM04554 group, and no deaths. Other adverse events are also listed for each group. These entries do not establish causality or rule out risks that were not observed or not fully reported.
Therefore, this page can clarify how the study was structured and what its registry record documents. It cannot conclude that either concentration did or did not change hair count, nor can it provide a comprehensive benefit-risk judgment. A fuller interpretation would require the posted primary outcome results, uncertainty estimates, analysis methods, missing-data information, and detailed safety reporting.
13. Why This Trial Matters Statistically
This study raises several useful statistical issues for clinical researchers. First, its primary endpoint is a repeated count measured using standardized imaging. That combination makes measurement procedures, assessment blinding, repeated-measures analysis, and the definition of the analysis population important to interpretation. The registry record describes elements of image acquisition and blinded assessment, while leaving the inferential model and analysis details unspecified.
Second, the trial has two active concentrations and a vehicle comparator, with several outcome topics and three listed primary-outcome time points. In general, a study with multiple potential comparisons needs a clear hierarchy or a stated approach to multiplicity so readers can distinguish planned primary evidence from exploratory findings. The record lists 66 analyses but does not explain how they relate to the outcomes or how multiplicity was addressed.
Third, the registry highlights the difference between enrollment and analysis. Enrollment was 302, and safety-group counts are reported, but the primary-outcome analyzed counts at each visit are not. For longitudinal outcomes, incomplete follow-up can affect both precision and the validity of estimates, depending on why measurements are missing and how the analysis handles them.
Finally, planning calculations are not trial results. Sample-size illustrations can help explain the assumptions behind future studies, but they cannot replace the original design justification or an estimate with uncertainty from the observed data. These distinctions matter when reading registry entries that contain a detailed design but limited reporting of efficacy results.
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 this trial from the registry record is that the primary outcome results are not reported in the registry record. There are no group means, standard deviations, treatment contrasts, confidence intervals, or p-values for change in non-vellus hair count. The record therefore supports description of the endpoint and schedule, but not a quantitative comparison of efficacy.
The registry record does not provide the number of participants included in the primary analysis by group or visit. Although it states that the overall analyzed population includes subjects who received at least one dose, it does not give a visit-level accounting or explain how participants with missing measurements were handled. The consequences of attrition or incomplete image assessments cannot be evaluated.
Important design and analysis details are also not reported in the registry record. These include the randomization and allocation-concealment procedures, the detailed masking arrangements, the statistical model, the prespecified primary contrast, the handling of multiple analyses, and the trial’s sample-size justification. The number and locations of sites are not given despite the multicenter description in the official title.
The outcome measurement description is informative but does not include details such as image-quality criteria, counting rules, repeatability, or how unusable images were treated. Safety reporting is also limited to the summarized affected-subject counts and the listed serious event term; detailed narratives and broader event breakdowns are not included. These omissions constrain interpretation and should not be filled with assumptions about the protocol or conduct.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: A Study of the Safety, Tolerability, and Efficacy of Topical SM04554 Solution in Male Subjects With Androgenetic Alopecia (AGA) — NCT02275351.
- Registry record last updated 2026-10-21; status last verified 2026-09.
- Results first posted 2026-10-01.
- 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.
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18. Record Summary
The registry record describes a completed, randomized, double-blind, parallel-group Phase 2 study of topical SM04554 in male subjects with androgenetic alopecia. Overall enrollment was 302. The three groups received SM04554 0.15%, SM04554 0.25%, or vehicle once a day for 90 days.
The primary outcome was change from baseline in non-vellus hair count, assessed at Day 45, Day 90, and Day 135 using macrophotography and blinded vendor assessment. Listed secondary outcomes concern investigator-assessed hair growth, AGA quality-of-life impact, and total sum of hair width.
The registry safety summary lists no deaths, one serious adverse event in the vehicle group, and other adverse events in all groups. Primary-outcome estimates and key analysis details are not reported in the registry record, so the efficacy comparison and its precision cannot be evaluated from this record alone.