Valchlor in the Treatment of Lichen Planopilaris: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Valchlor in the Treatment of Lichen Planopilaris (NCT03417141), a na phase 2 study. The primary outcome is Change in Lichen Planopilaris Activity Index (LLPAI).

2. Clinical Question

This is an exploratory question about change over time in a treated group. Without a comparator, observed changes cannot isolate the treatment’s contribution from other influences, including the natural course of disease, measurement variability, or expectations in an unmasked study. The registry identifies the study as Phase 2 and records Mayo Clinic as sponsor. Its listed secondary outcomes include changes in Dermatology Quality of Life Index, follicular units, and mean follicular density.

3. Trial Design

The registry describes Valchlor in the treatment of lichen planopilaris as a Phase 2, single-arm, open-label, exploratory study. Allocation is listed as not applicable, masking as none, and the model as single group. The intervention was mechlorethamine 0.016% topical gel, applied once daily. The record lists enrollment of 12 and identifies Mayo Clinic as sponsor.

The study start date is 2018-04-01, primary completion is recorded as 2019-08-30, and status is completed. These record dates describe the study timeline; they do not establish when each participant began or completed treatment. Individual treatment duration and participant-level follow-up are not reported in the registry record.

The primary outcome is described as change in LPPAI, with a timeframe of baseline to up to 24 weeks. The study summary instead describes measurement before and after 6 months of therapy. These descriptions indicate an intended before-and-after assessment, but the registry does not explain the difference in timeframe wording. The outcome title uses “LLPAI,” while its description uses “LPPAI.” The record does not clarify this naming difference.

Secondary outcomes listed are change in Dermatology Quality of Life Index (DQLI), change in follicular units, and change in mean follicular density. There is no concurrent control group, so comparisons are within the treated group over time rather than between randomized groups. The registry record does not report details of visit procedures, adherence assessment, concomitant care, eligibility criteria, or a statistical analysis 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

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: One subject withdrew and data was not analyzed for that subject

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 washed out prior treatments for 4 weeks before starting Valchor topically.

6. Primary Endpoint

The registry lists the primary outcome as Change in Lichen Planopilaris Activity Index (LLPAI), measured over baseline, up to 24 weeks.

The primary outcome is the percentage of change from baseline in the Lichen Planopilaris Activity Index, described in the registry as a standardized, validated quantitative measure of disease activity. The stated timeframe is baseline to up to 24 weeks. The study summary describes assessing the index before and after 6 months of treatment, while the primary-outcome record uses the wording “up to 24 weeks.” How these time descriptions correspond is not reported in the registry record.

The index combines symptoms and clinical signs. The description includes pruritus, pain, burning, scalp erythema, perifollicular erythema, perifollicular scale, the pull test, and disease spread. Symptoms and signs are scored on a 4-point scale from absent to severe; progression and a positive hair-pull test are recorded as yes or no. The registry gives a score range of 0-10 and specifies how these components are combined.

A standardized index can make assessment more consistent and provide a structured way to track disease activity over time. A percentage change expresses change relative to baseline rather than only the follow-up score. That interpretation depends on having comparable baseline and follow-up measurements and a clear analysis definition. The registry does not report the observed baseline or follow-up scores, the percentage-change result, or its variability. Therefore, the record does not allow readers to judge the magnitude or precision of change in this endpoint.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What does a median with full range describe?

The registry specifies the median and full range for the primary outcome. In general, the median is the middle value after observations are ordered. It describes the center without requiring unusually high or low observations to have the same influence they would have on an arithmetic mean. The full range reports the smallest and largest observed values, so it indicates the span of the data but not how observations are distributed between those endpoints.

These summaries can be useful when data are skewed or when a small number of observations might strongly affect a mean. However, a median and range do not by themselves quantify uncertainty about the median or show how much individual values vary around it. A confidence interval, when reported, gives a range of values compatible with the data under the method’s assumptions; it is not a guarantee that the true value lies within that interval. No confidence interval for the primary outcome is reported in the registry record.

How should percentage change be understood?

The primary outcome is defined as percentage change from baseline. In general, a relative change expresses a difference with reference to the starting value, whereas an absolute change describes the change on the measurement scale. Relative changes can be difficult to interpret without the underlying baseline and follow-up values, particularly when baseline values vary across participants. The registry describes the endpoint but does not report its numerical result or the baseline values used in the calculation.

The description gives the LPPAI components and scoring approach, but the registry does not state how individual participant changes were combined into the reported group summary beyond identifying the summary as a median with full range. It also does not provide a statistical test or an uncertainty estimate. The record’s outcome label uses “LLPAI,” while the endpoint description uses “LPPAI”; this naming difference is not resolved in the registry record.

What does a single-group comparison permit?

In general, measuring participants before and after treatment can describe change within the observed group. It does not provide the same protection against bias as a concurrent randomized comparison. Without a control group, the before-and-after contrast combines any treatment-associated change with other changes that occur over time. Open-label assessment can also be susceptible to expectation effects, particularly for outcomes involving symptoms or clinical judgment. These points describe general design considerations, not findings about what occurred in this study.

The registry lists the study as single-group and open-label, with no masking. It does not report a comparator, randomization procedure, or methods for reducing observer or participant expectation effects. Thus, a reported change would be an observation in the study group, not an estimate of a randomized treatment contrast.

What do the planning assumptions mean?

The registry describes sample-size illustrations as planning calculations based on the observed effect, using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. The stated effect to detect is a fraction of the observed effect. These are planning assumptions for a hypothetical comparison, not a description of the single-group study’s design. The registry does not report that the study itself used this design or these assumptions for its enrollment decision.

Such calculations are not observed power. In general, a planning calculation asks how many participants per arm would be needed under specified assumptions to detect a chosen effect with a target power. It depends on the assumed effect and statistical model. The registry record does not include the numerical planning results or the observed primary-outcome estimate needed to assess the illustration’s inputs. It also states that time-to-event illustrations use total events, whereas other illustrations use participants per arm. No time-to-event primary outcome is identified for this study.

What can be said about uncertainty?

Uncertainty concerns how much the estimated result might vary across repeated samples or under the analysis model. In general, an estimate’s precision is conveyed with an uncertainty measure such as a confidence interval or standard error. A range of observed values is not interchangeable with a confidence interval: the former describes the observed data’s spread, while the latter addresses uncertainty in an estimate. The registry identifies full range as the dispersion measure but reports no numerical range or confidence interval for the primary outcome.

Because the primary numerical result is not reported in the registry record, the direction and scale of the finding cannot be assessed. Statistical methods can clarify how a result would be interpreted, but they cannot recover an unreported estimate. No recalculated estimate is presented in the record, and no sponsor analysis result is stated for comparison.

10. Multiplicity, Missing Data and Analysis Populations

The registry lists four outcome measures and five analyses. The primary outcome is change in LPPAI; the listed secondary outcomes are changes in DQLI, follicular units, and mean follicular density. The record does not identify the five analyses in enough detail to determine how they relate to the four outcomes, whether any outcome was analyzed more than once, or which analysis was designated primary beyond the primary-outcome label.

When a study examines multiple outcomes or performs multiple analyses, the chance of at least one apparently notable result can increase. In general, a prespecified analysis plan and an approach to multiplicity help readers distinguish confirmatory findings from exploratory ones. The statistical analysis plan, prespecification details, and any multiplicity adjustment are not reported in the registry record. The study is characterized as exploratory, but that label does not specify how results should be interpreted statistically.

The primary-outcome population note states that one subject withdrew and data were not analyzed for that subject. The reason, timing, and handling of the missing outcome are not reported in the registry record. The record does not state whether an intention-to-treat, per-protocol, or other analysis population was used, nor does it describe imputation, sensitivity analyses, or other missing-data methods. The available information therefore does not show how robust the reported analysis would be to assumptions about the unobserved outcome.

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 safety record lists 12 participants at risk for serious adverse events and records zero serious events. It also records zero deaths. For other adverse events, 12 were listed as at risk and 11 as affected. These are registry counts, not rates calculated. The record does not provide event terms for the serious-event category.

These counts should be read descriptively. The registry does not say whether reported events were considered related to treatment, how severe they were, when they occurred, or how events were collected. The “other” adverse-event count does not specify the number or type of individual events in the information available in the record. It also does not establish that every affected participant had only one event.

The absence of recorded serious events in this study is not evidence that serious events cannot occur. A small, uncontrolled study can describe what was recorded among its participants, but it cannot by itself characterize uncommon risks or compare safety against another group. The safety counts do not establish causation or provide medical advice.

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

The registry record establishes that this was a completed, open-label, single-group exploratory study of once-daily mechlorethamine 0.016% topical gel for lichen planopilaris. It identifies LPPAI change as the primary outcome and lists changes in DQLI, follicular units, and mean follicular density as secondary outcomes. It also records the number at risk and affected for serious and other adverse events.

The record does not report the numerical primary-outcome result or numerical results for the secondary outcomes. It therefore does not show how much LPPAI changed, whether any observed change was consistent across participants, or how precise the estimate was. Without those values, the result cannot be characterized as large, small, positive, or negative on the basis of this record. The outcome definition alone is not an outcome finding.

Even if a before-and-after change were reported, the single-group design would not establish that treatment caused the change. A concurrent comparison is absent, and masking was not used. In general, observed change in this design can reflect treatment and other influences over time; the registry record does not quantify those contributions.

The safety counts describe events recorded in this study, not a comparative safety estimate. They do not show whether events were treatment-related or establish the absence of risk. Taken together, the record is useful for identifying the study’s exploratory question and design, but the missing outcome estimates and limited design details prevent a firm assessment of efficacy or safety.

13. Why This Trial Matters Statistically

This study is statistically informative as an example of the distinction between a clearly stated research question and a result that can be evaluated quantitatively. The registry describes a standardized disease-activity measure, a before-and-after assessment, and secondary clinical and quality-of-life measures. Yet the numerical outcome findings are not reported in the registry record. Without estimates and measures of uncertainty, readers cannot assess the observed magnitude, consistency, or precision of change.

The single-group, open-label design also makes the target of inference important. A within-group change is not the same as a controlled treatment effect. General influences such as the course of disease, changes in other care, and measurement expectations cannot be separated from treatment effects using a single group alone. This matters when interpreting exploratory studies: descriptive observations can motivate further evaluation, but they do not answer the same causal question as a controlled comparison.

The record also illustrates why endpoint definitions, analysis populations, and missing-data methods matter. The primary outcome is described in detail, but its numerical result is absent, and the record notes that one subject withdrew and was not analyzed for that outcome. The relationship between the stated timeframe of up to 24 weeks and the summary’s 6-month assessment is not clarified. These details affect how readers understand what was measured and which observations contributed.

Finally, the adverse-event counts are meaningful as a description of what the registry records, but not as proof of causation or a complete characterization of risk. Clear reporting of outcome estimates, uncertainty, analysis methods, and event details would be needed for a fuller statistical interpretation.

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.

Several trial-specific details needed to interpret the findings are not reported in the registry record. The numerical primary and secondary outcome results are absent, as are uncertainty measures and the statistical tests used. The primary outcome is described as a median with full range, but no median or range values are stated. The record also does not clarify the difference between the primary timeframe of baseline to up to 24 weeks and the study summary’s before-and-after assessment at 6 months.

The design has no concurrent control group and is open-label. Consequently, a within-group change cannot separate treatment-associated change from other influences over time, and unmasked assessments may be influenced by expectations. These are design limitations, not evidence that any particular bias occurred.

The primary-outcome note says one subject withdrew and was not analyzed. The reason and timing of withdrawal, the missing-data approach, and the analysis population are not reported in the registry record. The record does not describe eligibility criteria, adherence, concomitant treatments, or participant-level follow-up, so these features cannot be incorporated into interpretation.

Safety information is also limited: the registry records serious and other adverse-event counts but does not give event terms for serious events, timing, severity, relatedness, or collection procedures. Finally, the endpoint is referred to as “LLPAI” in its title and “LPPAI” in its description, without clarification. These reporting gaps limit reproducibility and prevent a detailed appraisal of the outcome analyses.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Valchlor in the Treatment of Lichen Planopilaris — NCT03417141.
• Registry record last updated 2021-01-28; status last verified 2021-01.
• Results first posted 2020-07-02.
• 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

NCT03417141, “Valchlor in the Treatment of Lichen Planopilaris,” is recorded as a completed Phase 2, single-group, open-label exploratory study sponsored by Mayo Clinic. The intervention was mechlorethamine 0.016% topical gel, applied once daily. Enrollment was 12; the study start date was 2018-04-01 and primary completion was 2019-08-30.

The primary outcome was percentage change from baseline in LPPAI, with the registry listing a median and full range and a timeframe of baseline to up to 24 weeks. One subject withdrew and was not analyzed for that outcome. Numerical outcome results are not reported in the registry record. Secondary outcomes included changes in DQLI, follicular units, and mean follicular density. The safety record lists zero serious adverse events and zero deaths, with other adverse events recorded for 11 of 12 at risk.