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 Study of the Safety, Tolerability and Efficacy of BTX 1204 in Patients With Moderate Atopic Dermatitis (NCT03824405), a randomized phase 2 study. The primary outcome is Investigator's Global Assessment (IGA).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT03824405 |
| Phase | Phase 2 |
| Condition | Atopic Dermatitis |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | Quadruple |
| Enrollment | 200 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Botanix Pharmaceuticals |
| Results posted | Yes |
2. Clinical Question
Population
Participants with moderate atopic dermatitis were enrolled. The registry record lists an enrollment of 200, with 100 randomized to each arm. The primary outcome description refers to an intent-to-treat summary and notes exclusions from that population.
Intervention
BTX 1204 was administered twice daily. The record identifies the intervention as BTX 1204 4% BID in the description of randomized subjects.
Comparator
The comparator was vehicle, administered twice daily. The study is described as vehicle-controlled.
Primary question
The primary outcome was Investigator's Global Assessment success at Day 85: an IGA score of 'Clear' (0) or 'Almost Clear' (1), together with at least a 2-grade improvement. The registry record does not report the number of participants meeting this definition in either arm or a comparison between arms.
In statistical terms, the intended question is whether the participant-level success outcome at Day 85 differed between the randomized BTX 1204 and vehicle arms. The outcome combines an absolute status at the assessment with improvement from an earlier score; success therefore requires meeting both parts of the stated definition. The reported material establishes the outcome definition, but not the observed success counts or an estimated treatment contrast. Without those results, the direction and size of any difference, and its statistical precision, cannot be assessed from the registry record.
The study was randomized and masked, which describes how the comparison was structured. Those design features do not substitute for outcome data: interpretation of the primary question still depends on the observed endpoint results and the analysis used to compare the arms. The registry record does not report those details.
3. Trial Design
This was a randomized, parallel-group Phase 2 study of BTX 1204 and vehicle in participants with moderate atopic dermatitis. The official title describes it as double-blind and vehicle-controlled; the registry record separately lists masking as quadruple. Both arms used their assigned treatment twice daily. The sponsor was Botanix Pharmaceuticals.
The recorded enrollment was 200, with 100 randomized subjects in each arm according to the primary outcome population description. The study status is completed. Its recorded start date is 2019-02-04, and its primary completion date is 2020-01-27. These are registry dates and do not, by themselves, describe how long any individual participant was followed.
The primary outcome was assessed at Day 85 and concerned IGA success. Its definition required an IGA score of 'Clear' or 'Almost Clear' and at least a 2-grade improvement. The record also reports that some randomized subjects were excluded from the ITT population because they did not have at least one post-baseline assessment. It does not give the outcome counts for either randomized arm.
Random allocation is intended to create groups that can be compared, while masking is intended to limit the influence of knowledge about assigned treatment on conduct or assessment. These are general features of trial design, not evidence about the observed endpoint. The registry record does not explain the specific allocation procedure, how masking was maintained, or the roles covered by the quadruple-masking label. Those details are not reported in the registry record.
The record identifies one primary outcome measure and no analysis entries. It does not report the statistical analysis plan, planned sample-size justification, or any interim-analysis arrangements. Accordingly, design features can be described, but it is not possible to determine from the registry record whether the posted analyses followed a prespecified plan.
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 | BTX 1204 | Vehicle |
|---|---|---|
| STARTED | 100 | 100 |
| COMPLETED | 65 | 55 |
| NOT COMPLETED | 35 | 45 |
Analysis population for the primary outcome, as defined in the registry: ITT Summary of Subjects Excluded From Analyses (Randomized Subjects) BTX 1204 4% BID (N=100) Vehicle BID (N=100) Number of Subjects Excluded from the ITT Population, n (%) BTX 1204 4% BID 1 (1.0) Vehicle BID 5 (5.0) Reason for Exclusion from the ITT Population, n (%) Not At Least One Post-Baseline Efficacy Assessment BTX 1204 4% BID 1 (1.0) Vehicle BID 5 (5.0)
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: Intent-to-treat Population
| Characteristic | Unit | BTX 1204 | Vehicle | Total |
|---|---|---|---|---|
| Participants analysed | 99 | 95 | 194 | |
| Age, Categorical: <=18 years | Participants | 20 | 15 | 35 |
| Age, Categorical: Between 18 and 65 years | Participants | 77 | 76 | 153 |
| Age, Categorical: >=65 years | Participants | 2 | 4 | 6 |
| Sex: Female, Male: Female | Participants | 62 | 54 | 116 |
| Sex: Female, Male: Male | Participants | 37 | 41 | 78 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 9 | 11 | 20 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 90 | 84 | 174 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 1 | 0 | 1 |
| Race (NIH/OMB): Asian | Participants | 18 | 17 | 35 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 2 | 2 | 4 |
| Race (NIH/OMB): Black or African American | Participants | 14 | 18 | 32 |
| Race (NIH/OMB): White | Participants | 53 | 55 | 108 |
| Race (NIH/OMB): More than one race | Participants | 11 | 3 | 14 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 |
| Investigator Global Assessment: 0 - Clear | Participants | 0 | 0 | 0 |
| Investigator Global Assessment: 1 - Almost Clear | Participants | 0 | 0 | 0 |
| Investigator Global Assessment: 2 - Mild | Participants | 0 | 0 | 0 |
| Investigator Global Assessment: 3 - Moderate | Participants | 99 | 95 | 194 |
| Investigator Global Assessment: 4 - Severe | Participants | 0 | 0 | 0 |
6. Primary Endpoint
The registry lists the primary outcome as Investigator's Global Assessment (IGA), measured over Day 85.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint was Investigator's Global Assessment success at Day 85. Success was defined as an IGA score of 'Clear' (0) or 'Almost Clear' (1) with at least a 2-grade improvement. The outcome is binary at the participant level: each participant either meets the complete success definition or does not. It is not enough to satisfy only the score threshold or only the improvement requirement.
IGA is an investigator assessment, and the registry describes a defined score threshold and change requirement to identify participants meeting the endpoint. In general, a prespecified categorical endpoint can make the target outcome explicit and permit a comparison of the proportions of participants meeting the definition in each arm. That is a general statistical explanation; the registry record does not report the observed proportions for this trial.
The endpoint's time point matters because the stated assessment is at Day 85. The record does not provide additional primary outcome results at other time points, nor does it report a treatment-effect estimate, confidence interval, or p-value. The endpoint definition is therefore available, but the registry record does not establish how many participants achieved success or how precisely the arms differed.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record describes the primary outcome clearly enough to identify what was intended to count as success, but it does not report primary outcome results. In particular, it gives no Day 85 success counts for BTX 1204 or vehicle and no estimate comparing the arms. As a result, the posted information does not establish the direction or magnitude of the primary outcome difference.
No confidence interval or p-value for the primary outcome is reported in the registry record. A confidence interval is a statistical range that expresses uncertainty around an estimated effect under the analysis assumptions; its width helps communicate precision. A p-value describes how compatible the observed data are with a specified null model under the test assumptions. These are general explanations. Neither statistic can be interpreted for this primary comparison without the corresponding estimate and analysis results.
The record lists 100 randomized subjects per arm, while the ITT exclusion summary states that 1 participant in the BTX 1204 arm and 5 in the vehicle arm were excluded from the ITT population. The stated reason was not having at least one post-baseline assessment. Those numbers concern analysis-population exclusions; they do not reveal the endpoint outcomes among excluded participants or how those data were handled.
Safety information is reported separately. Serious adverse events affected 2 participants among 99 at risk in the BTX 1204 group and 0 among 98 at risk in the vehicle group. Other adverse events affected 37 participants among 99 at risk and 25 among 98 at risk, respectively. The registry reports 0 deaths in both groups. These are descriptive counts; the record does not provide an inferential comparison or enough detail to determine the timing, duration, or full clinical context of each event.
For the serious-event terms listed in the record, 'Post-procedural complication' and 'Pneumonia' are each reported as 1.0%. The record does not identify the arm for each listed term in the available information. Those entries should not be treated as a substitute for the overall serious-event counts or as an estimate of the primary endpoint.
The study's randomized design is relevant to how its comparison was intended to be made, but the absence of posted efficacy results prevents an assessment of the primary comparison. No conclusion about an effect, its precision, or statistical significance follows from the design description alone.
9. Statistical Methods Explained
The statistical methods that can be described are limited to the design and the information recorded about the endpoint and planning assumptions. The registry record lists a randomized, parallel-group study, one primary outcome measure, and no analysis entries. It does not report the primary outcome analysis method, model, test, or estimated contrast. The explanations below distinguish those documented details from general statistical concepts.
What does a binary endpoint represent?
General: A binary endpoint classifies each participant into one of two categories, such as meeting or not meeting a prespecified success definition. For this trial, the endpoint definition combines an IGA score of 'Clear' (0) or 'Almost Clear' (1) with at least a 2-grade improvement at Day 85. A participant must satisfy both requirements to count as a success. The registry record does not report how many participants met that definition in either arm.
For a randomized comparison, a common descriptive summary for a binary outcome is the number and proportion of participants meeting the endpoint in each arm. A treatment contrast can then be expressed using a chosen measure of difference or association. These are general approaches; the registry record does not state which measure or test was planned or used. No additional numerical contrast can be reconstructed from the available endpoint information.
What do randomization and masking contribute?
General: Randomization assigns participants to study arms using a chance-based process, with the aim of making the groups comparable on average. Masking can reduce the influence of treatment knowledge on participant experience, study conduct, and outcome assessment. The registry record labels the study randomized, describes it as double-blind, and lists quadruple masking. It does not describe the allocation mechanism, concealment procedures, or who was masked. The design labels alone do not show whether those procedures were implemented as intended.
How should the analysis population be understood?
The record describes an ITT summary of randomized subjects excluded from the ITT population. It reports 1 exclusion in the BTX 1204 arm and 5 in the vehicle arm, with the reason stated as not having at least one post-baseline assessment. General: An intent-to-treat approach ordinarily aims to analyze participants according to their randomized assignment, regardless of treatment adherence. The registry record's exclusion summary indicates that not all randomized subjects were included in the named ITT population, but it does not explain the full population definition, whether other analysis populations were used, or how missing outcome data were handled.
What do the planning assumptions mean?
The planning note describes an illustration based on the observed effect, using a two-sided significance level of 0.05, equal allocation between arms, and a normal approximation. It states that the effect to detect is a stated fraction of the observed effect. The note distinguishes participants per arm for outcomes other than time-to-event outcomes, for which the total number of events is used. These are planning assumptions, not evidence that the trial was designed using those assumptions. The registry record does not report the trial's own sample-size justification or planned effect size. It also does not report the numerical planning outputs or the observed primary effect needed to interpret such an illustration.
General: Sample-size calculations estimate how many participants may be needed to detect a specified effect under stated assumptions. A planning calculation is not an observed result and does not show the power achieved after the trial. In particular, a calculation based on an observed effect is a retrospective illustration, not a substitute for a prospective design justification. No sample-size result should be read as observed power.
Why do confidence intervals and multiplicity matter?
General: A confidence interval communicates uncertainty around an estimated effect, while a statistical test evaluates data against a specified null hypothesis. The registry record reports neither a primary effect estimate nor its confidence interval or test result. Multiplicity arises when multiple hypotheses, outcomes, or time points are tested; without an analysis plan, it is not possible to determine what control, if any, was planned for multiple testing. The record does not report a multiplicity procedure or interim analyses.
Why can over-dispersion matter?
General: Over-dispersion occurs when variability in count data exceeds what a statistical model assumes. It can affect standard errors and uncertainty estimates in analyses of counts. The primary endpoint in this record is a participant-level binary outcome, not a reported event-rate model, and no over-dispersion assessment or count model is described. It would therefore be inappropriate to claim that over-dispersion was assessed or relevant to the analysis of this primary endpoint.
10. Multiplicity, Missing Data and Analysis Populations
The registry record identifies one primary outcome measure: IGA success at Day 85. It does not report the primary comparison results, analysis method, or a statistical analysis plan. Although the record lists one primary outcome measure, it does not establish whether additional outcomes, time points, or hypotheses were analyzed outside the information recorded.
Multiplicity refers, in general, to the increased chance of a false-positive result when several hypotheses or comparisons are tested. Prespecified procedures can address that issue, but the registry record does not report a multiplicity adjustment or explain how multiple testing was handled. It also does not report whether interim analyses were planned or conducted. No assumption about such procedures can be made from the available record.
The record states that 1 randomized participant in the BTX 1204 arm and 5 in the vehicle arm were excluded from the ITT population because they did not have at least one post-baseline assessment. It does not describe additional analysis populations, an imputation method, or a sensitivity analysis for missing data. General: Missing outcome data can affect estimates if the participants without measurements differ from those with measurements in outcome-related ways. The registry record does not provide enough information to assess that possibility or determine how missing Day 85 outcomes were treated.
Because the primary outcome counts and analysis are not reported, it is not possible to evaluate whether the analysis population used for the endpoint matched a prespecified plan, or whether the conclusion was robust to alternative missing-data assumptions. Those details are not reported in the registry record.
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 |
|---|---|---|---|
| BTX 1204 | 2 | 99 | 2.0% |
| Vehicle | 0 | 98 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| BTX 1204 | 0 of 99 |
| Vehicle | 0 of 98 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 2% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| BTX 1204 | 37 of 99 |
| Vehicle | 25 of 98 |
Most frequent serious adverse event terms
| Event term | Organ system | BTX 1204 | Vehicle |
|---|---|---|---|
| Post-procedural complication | General disorders | 1 of 99 | 0 of 98 |
| Pneumonia | Respiratory, thoracic and mediastinal disorders | 1 of 99 | 0 of 98 |
Most frequent non-serious adverse event terms
| Event term | Organ system | BTX 1204 | Vehicle |
|---|---|---|---|
| Dermatitis atopic | Skin and subcutaneous tissue disorders | 11 of 99 | 15 of 98 |
| Erythema | Skin and subcutaneous tissue disorders | 7 of 99 | 1 of 98 |
| Nasopharyngitis | Skin and subcutaneous tissue disorders | 6 of 99 | 1 of 98 |
| Application Site Pain | Skin and subcutaneous tissue disorders | 4 of 99 | 2 of 98 |
| Pruritis | Skin and subcutaneous tissue disorders | 3 of 99 | 3 of 98 |
| Application Site Reaction | Skin and subcutaneous tissue disorders | 3 of 99 | 2 of 98 |
| Dry Skin | Skin and subcutaneous tissue disorders | 3 of 99 | 1 of 98 |
Serious events with the highest arm-level rate: post-procedural complication (1.0%), pneumonia (1.0%).
The registry record reports serious adverse events for participants at risk in each group. In the BTX 1204 group, 2 participants were affected among 99 at risk; in the vehicle group, 0 participants were affected among 98 at risk. The record lists 0 deaths in each group. These counts describe the recorded safety data and are not a statistical comparison.
For other adverse events, 37 participants were affected among 99 at risk in the BTX 1204 group, and 25 were affected among 98 at risk in the vehicle group. These figures refer to participants affected, not necessarily the total number of event episodes. The registry record does not give the event-level details, timing, severity distribution, or duration needed to describe those events further.
The listed serious-event terms are 'Post-procedural complication' and 'Pneumonia', each reported as 1.0%. The record does not specify which group each term belongs to in the available information. The serious-event counts and listed terms should be interpreted as descriptive registry entries; no adjusted comparison, uncertainty interval, or causal assessment is reported.
12. What the Result Does, and Does Not, Mean
The trial record documents a randomized, parallel-group comparison of twice-daily BTX 1204 and twice-daily vehicle in participants with moderate atopic dermatitis. It defines a primary outcome at Day 85 and provides some participant-level safety counts. These details describe the study question and selected results that are recorded.
The primary outcome results themselves are not reported in the registry record. There are no arm-specific counts of participants meeting the IGA success definition, no estimated contrast, and no confidence interval or p-value. Therefore, the record does not allow readers to determine how the groups compared on the primary endpoint or how precisely any difference was estimated.
General: A randomized design supports a planned comparison between assigned groups, but interpretation still requires the actual outcome data, the analysis population, and the method used to estimate uncertainty. A study's completion status and enrollment count do not indicate whether a particular endpoint difference was observed or whether it was statistically precise.
The safety information includes serious and other adverse-event counts and reports no deaths in either group. These descriptive data do not establish whether an event was caused by treatment, and the registry record does not report an inferential safety comparison. The listed serious-event terms are not linked to a specific arm in the available information.
Accordingly, the record supports a description of the design, endpoint definition, ITT exclusions, and reported safety counts. It does not support a conclusion about the primary efficacy comparison, the effect's size or certainty, or the study's overall benefit-risk balance. Those are not reported in the registry record.
13. Why This Trial Matters Statistically
This trial illustrates why a clearly defined endpoint and a reported result are separate requirements for statistical interpretation. The registry record specifies IGA success at Day 85, including both the target score and the required improvement. That definition identifies what the primary outcome was intended to measure, but without success counts or an estimated comparison, the central statistical question remains unanswered by the posted results.
The record also distinguishes randomized enrollment from the ITT population: it reports 100 randomized subjects per arm and exclusions from the ITT population. That distinction matters because the population included in an analysis can affect how a treatment comparison is interpreted. The stated reason for exclusion was absence of at least one post-baseline assessment, but details on missing outcome handling are not reported in the registry record.
Safety counts provide a separate example of the difference between descriptive information and inferential evidence. The record gives counts of participants affected by serious and other adverse events, but it does not give uncertainty estimates or an adjusted comparison. Counts can be informative, yet their interpretation depends on definitions, follow-up, and analysis details that are not reported in the registry record.
Finally, the planning note distinguishes a sample-size illustration from the trial's design justification. General: A planning exercise depends on assumptions about the target effect, error rate, allocation, and analysis. Such an illustration should not be mistaken for an observed result or a measure of achieved power. In this record, the numerical primary effect and planning outputs are not reported, reinforcing the importance of separating design assumptions from outcome evidence.
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 main limitation for statistical interpretation is that the registry record does not report primary outcome results. It states the Day 85 IGA success definition but gives no success counts by arm, estimated difference, confidence interval, or p-value. The primary comparison therefore cannot be evaluated for direction, magnitude, or precision.
The analysis population information is incomplete. The record reports exclusions from the ITT population because participants did not have at least one post-baseline assessment, but does not explain how Day 85 missingness was handled or whether sensitivity analyses were conducted. It also does not describe other analysis populations or the final analysis set.
The statistical plan is not reported in the registry record. The analysis method, prespecified hypotheses, multiplicity procedures, interim analyses, and trial-specific sample-size justification are not available. The planning note describes assumptions for an illustration, but does not provide the observed primary effect or numerical outputs in the information recorded. Thus it cannot supply the missing primary comparison.
Safety reporting is also limited to the counts and terms recorded. The registry does not provide detailed event timing, duration, severity distribution, or an inferential comparison. It does not identify the treatment arm associated with each listed serious-event term in the available information. These limitations restrict interpretation to descriptive reporting rather than a full assessment of between-arm safety differences.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Study of the Safety, Tolerability and Efficacy of BTX 1204 in Patients With Moderate Atopic Dermatitis — NCT03824405.
- Registry record last updated 2022-10-25; status last verified 2022-09.
- Results first posted 2022-09-27.
- 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, vehicle-controlled Phase 2 study of BTX 1204 in participants with moderate atopic dermatitis. The study used a parallel-group design, and both BTX 1204 and vehicle were administered twice daily. Enrollment was 200, with 100 randomized subjects in each arm according to the primary outcome population description.
The primary outcome was IGA success at Day 85, defined as 'Clear' (0) or 'Almost Clear' (1) with at least a 2-grade improvement. The record reports ITT exclusions of 1 participant in the BTX 1204 arm and 5 in the vehicle arm due to not having at least one post-baseline assessment, but does not report the primary outcome comparison.
Serious adverse events affected 2 participants among 99 at risk in the BTX 1204 group and 0 among 98 at risk in the vehicle group; 0 deaths were reported in each group. The primary analysis, uncertainty estimates, and trial-specific statistical plan are not reported in the registry record.