Oleogel-S10 in Wound Healing of Inherited Epidermolysis Bullosa (BEB-10): Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Oleogel-S10 in Wound Healing of Inherited Epidermolysis Bullosa (BEB-10) (NCT01294241), a non-randomized phase 2 study. The primary outcome is Intra-individual Difference in Reepithelialization of Wound (Halves) at Day 14 in 'Recent Wounds' or Day 28 in 'Chronic Wounds'.

2. Clinical Question

The trial’s central question is about the relative progress of wound epithelialization within participants, rather than a comparison between independently randomized groups. The planned comparison paired an Oleogel-S10-treated wound or wound half with a dressing-only wound or wound half. The endpoint was assessed at Day 14 for recent wounds or Day 28 for chronic wounds.

The registry describes eligible wounds as either one wound at least 10 cm2 and no more than 200 cm2, divided into two equal halves, or two comparable wounds at least 5 cm2 each. The stated purpose was to compare reepithelialization in inherited epidermolysis bullosa. The primary outcome description says assessors reviewed chronological photo series and classified which side reepithelialized faster, or whether there was no difference. The actual counts of outcomes in each category are not reported in the registry record, so the record does not establish which treatment condition was judged faster.

3. Trial Design

This was a Phase 2, completed, non-randomized study sponsored by Birken AG. The registry lists an enrollment of 10 participants, a single-group model, and single masking. The start was recorded as 2010-11 and primary completion as 2011-06. Although the official title calls the study open, the primary outcome description specifically states that two independent experts were blind to treatment when they assessed the photographs. The registry does not identify who the single masking designation applies to.

The comparison was intra-individual: each participant contributed treatment and comparator wound areas, rather than being assigned to one of two randomized participant groups. Oleogel-S10 was applied topically to one eligible wound or half and covered with Mepilex®. The comparator wound or half was covered with Mepilex® alone. Dressings were changed about every 24 to 48 hours. The treatment description refers to an end of treatment at Day 14 for recent wounds or Day 28 for chronic wounds, or discharge from hospital.

The primary outcome description reports that 12 wounds in 10 participants were evaluated, including two cycles of treatment in two participants. It also states that four wounds were excluded from the primary analysis because they were either evaluated controversially or judged equal. The number remaining in the primary analysis is not reported in the registry record. The record does not report a sample-size justification, a detailed statistical analysis plan, or any interim analysis plan. The design is not a randomized comparison, and the registry explicitly cautions that the treatment conditions should not be read as like-for-like control arms. The within-participant approach can account for some participant-level differences, but it does not randomize which wound receives which treatment according to the information in the registry record.

4. Participant Flow and Accounting

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

Before assignment: 12 wounds in 10 participants (2 cycles of treatment in 2 participants) were treated with study medication. The reepithelialization was compared intra-individually in either 2 halves of an Epidermolysis bullosa (EB) wound ≥10 cm2 and ≤200 cm2 in size or in 2 EB wounds ≥5 cm2 in size, i.e. the total number of participants was 10 in the overall study.

Overall Study

Analysis population for the primary outcome, as defined in the registry: 12 wounds in 10 participants (intra-individual comparison, 2 cycles of treatment in 2 participants) were evaluated by assessors that were blind to treatment. 'Undecided' wounds that were either evaluated controversially (n=2) or as being equal (n=2) were excluded from the analysis of the primary efficacy variable.

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: As 2 participants received 2 cycles of treatment each, 12 wounds were treated with study medication.

6. Primary Endpoint

The registry lists the primary outcome as Intra-individual Difference in Reepithelialization of Wound (Halves) at Day 14 in 'Recent Wounds' or Day 28 in 'Chronic Wounds', measured over 14 days for 'recent wounds', 28 days for 'chronic wounds'.

The primary endpoint classifies the relative reepithelialization of paired wounds or wound halves at the end of treatment: Day 14 for recent wounds or Day 28 for chronic wounds. Two independent experts, blind to treatment, reviewed chronological series of cropped and coded photographs taken before treatment, during dressing changes, and at the end of treatment. They decided whether one wound or half reepithelialized faster than the other, or whether there was no difference.

This endpoint is used to turn a visual comparison of wound healing progress into a categorical within-participant assessment. It focuses on which of the paired areas appeared to reepithelialize faster, rather than reporting a numerical amount of change in wound area in the primary outcome description. Blinding the assessors can reduce the influence of knowledge about treatment on their judgments, although the registry does not report how disagreements between assessors were resolved beyond noting that some evaluations were controversial.

The registry says that wounds judged controversially or equal were excluded from the primary analysis. The counts of treatment-favoring outcomes and the final primary analysis result are not reported in the registry record. Therefore, the endpoint definition can be described, but the comparative finding cannot be interpreted from the posted results.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry identifies a categorical primary outcome and describes a within-participant comparison. It does not report the statistical test used, an analysis model, an effect estimate, or an uncertainty interval. The concepts below explain how this kind of endpoint is generally interpreted; they do not imply that a particular unreported method was used in this trial.

What does a paired comparison mean?

In general, a paired design compares two observations linked to the same participant. Here, the linked observations were wound areas treated under different conditions. Such a design can account for participant characteristics shared by both observations, because each participant serves as their own comparison. It does not automatically make the wound areas equivalent: wounds can differ within a participant, and the record does not say that treatment allocation between wounds or halves was randomized.

Paired analyses need to preserve that linkage. An analysis that treated each wound as wholly independent could fail to reflect the fact that some wounds came from the same participant. The record states that 12 wounds in 10 participants were evaluated and that two participants had two treatment cycles. It does not report the statistical handling of repeated observations or clustering by participant. Therefore, the registry record does not establish whether the analysis accounted for these dependencies.

How should the winner-or-tie outcome be read?

The primary outcome was a categorical assessment of relative healing progress: one wound or half was judged faster, or there was no difference. In general, a categorical outcome reports the number of observations in each category rather than the size of a continuous change. The trial description does not report a numerical scale for the amount of epithelialization in this primary endpoint. The registry also states that equal assessments were excluded from the primary analysis, along with controversial assessments. Excluding such observations changes the set of comparisons represented by the analyzed result. The number of observations in each final category is not reported in the registry record.

The record describes two independent experts who were blind to treatment and reviewed chronological photographic series. In general, blinding can limit the influence of treatment knowledge on subjective assessment. Independent assessment does not, by itself, show how reproducible the judgments were. Agreement statistics, a resolution procedure for disagreements, and a full account of the assessment process are not reported in the registry record.

What do confidence intervals and p-values usually convey?

In general, a confidence interval gives a range of values compatible with the observed data under the assumptions of the analysis. A narrow interval indicates greater precision than a wide interval, while an interval spanning values on different sides of no difference may leave the direction uncertain. A p-value, when reported, describes how unusual data at least as incompatible with a stated null hypothesis would be under that hypothesis and the analysis assumptions. Neither measure gives the probability that a treatment works, and neither can substitute for understanding the design and endpoint.

The registry record does not report a confidence interval or p-value for the primary outcome, nor does it report an effect estimate. As a result, the precision and inferential strength of the primary comparison cannot be evaluated. No inferential method should be attributed to the trial based on the endpoint description alone.

Why does the analysis population matter?

In general, an analysis population defines which participants or observations contribute to a particular result. The registry reports 12 evaluated wounds from 10 participants, but says that two controversial assessments and two assessments judged equal were excluded from primary analysis. It does not state the final number of observations analyzed or give a fuller account of analysis populations. The safety population is described separately as 10 participants at risk for each of the listed event categories. These population descriptions should not be conflated: wound-level efficacy assessment and participant-level safety reporting have different units of analysis.

What can planning illustrations tell readers?

In general, sample-size planning relates a target effect, a significance level, and assumptions about variability or event frequency to the number of participants needed for a chosen power. The registry’s stated planning assumptions describe illustrations based on a fraction of the observed effect, a two-sided significance level of 0.05, equal allocation, and a normal approximation; the illustrations are not the trial’s own design justification. They should be read as planning scenarios, not as observed power or as trial results. The sample-size justification used for the actual study is not reported in the registry record. In this trial, the observed primary effect itself is not reported, so readers cannot independently assess the basis or precision of such illustrations from the registry record.

Multiplicity refers, in general, to the increased chance of a false-positive finding when many outcomes or comparisons are tested. The registry lists one primary outcome and two secondary outcomes, but does not report whether an adjustment for multiple testing was planned or applied. The statistical analysis plan, including any prespecified test or multiplicity strategy, is not reported in the registry record.

10. Multiplicity, Missing Data and Analysis Populations

The record identifies one primary endpoint and two secondary outcomes: percentage of wound epithelialization at Day 7±1 and Day 14±1. It does not report a statistical analysis plan, the statistical tests used, or whether the analyses included any adjustment for multiple outcomes. In general, when several outcomes are tested, multiplicity procedures can help control the chance of false-positive findings across those tests. Whether such procedures were appropriate or used in this trial is not reported in the registry record.

The primary outcome description states that two controversial assessments and two assessments judged equal were excluded from primary analysis. It does not explain how the exclusion rules were prespecified, how many wound comparisons remained for analysis, or how those exclusions affected the reported results. The number of participants or wounds contributing to the secondary outcome analyses is also not reported in the registry record. Missing-data methods, reasons for missing outcome measurements, and any sensitivity analyses are not reported.

The registry describes a safety population of 10 participants and reports 10 at risk in each of the listed event categories. The efficacy outcome is described at the wound level, with 12 wounds in 10 participants evaluated. These are distinct analysis units. The record does not report a complete account of efficacy analysis populations or how repeated cycles within participants were handled. It also does not report whether all enrolled participants contributed to each outcome. These omissions limit assessment of outcome completeness and the robustness of the analysis.

11. Safety Results

The registry reports serious adverse events as affected participants divided by participants at risk.

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 lists a safety population of 10 participants. In the serious adverse event category, 10 were at risk and zero were recorded as affected. In the other adverse event category, 10 were at risk and six were recorded as affected. These are counts in the registry’s safety summary; they do not describe the severity, timing, duration, or relationship of individual events to treatment.

No serious adverse event terms are listed among the top serious terms. The death count is not reported in the registry record. The record does not provide additional detail on the six participants recorded as affected in the other adverse event category, so the types and clinical context of those events cannot be characterized from this information.

These counts should be read as descriptive safety reporting, not as a randomized comparison of event rates. The study is listed as non-randomized and single-group, and its wound-level efficacy comparison is intra-individual. The safety summary alone cannot establish whether treatment caused or prevented an event, or whether the event counts differ meaningfully between treatment conditions. The registry record does not report further comparative safety analyses.

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

The registry record establishes that this study was designed to compare Oleogel-S10 plus Mepilex® with Mepilex® alone within participants with inherited epidermolysis bullosa. It describes a photographic assessment of relative reepithelialization at the end of treatment, with separate timing for recent and chronic wounds. It also describes blinded review by two independent experts. These features explain the question the study set out to address.

The registry record does not report the primary outcome counts or an estimate of the treatment contrast. It therefore does not show whether assessors judged either treatment condition to be faster, or how consistent or precise any observed difference was. The secondary outcome results are also not reported. No conclusion about statistical significance or the magnitude of an effect can be drawn from the outcome definitions alone.

The design is non-randomized and uses paired wounds or wound halves rather than randomized participant groups. Pairing can address some differences between participants, but wounds within the same person may not be identical, and treatment allocation to wound areas is not described as randomized. The endpoint also excludes controversial and equal assessments from the primary analysis, with no reported final analysis count or detailed account of the exclusion process.

The safety summary records no participants affected in the serious adverse event category and six affected in the other adverse event category, among 10 at risk in each category. The record does not provide event details or a randomized safety comparison. These observations are descriptive and do not establish causation. Overall, the record documents a small exploratory design and its outcome assessment, but the posted results needed to judge the efficacy findings and their uncertainty are not reported in the registry record.

13. Why This Trial Matters Statistically

This trial is statistically notable for its within-participant structure. Rather than comparing separate randomized groups, it compares treatment conditions across wounds or wound halves from the same participant. That design makes the pairing central to interpretation: the unit of assessment is a wound comparison, while participant characteristics are shared across the paired areas. The registry reports 12 wounds evaluated in 10 participants, including two cycles in two participants, making clear that wound counts and participant counts are not interchangeable.

The outcome also illustrates the distinction between a defined endpoint and an available result. The endpoint is described as a blinded judgment of which area reepithelialized faster, or whether there was no difference. Yet the registry record does not report the category counts or an effect estimate. Readers can understand the intended measurement without being able to evaluate its direction, magnitude, or precision.

Assessment exclusions are another important statistical feature. The record says controversial and equal assessments were left out of the primary analysis, but does not report the final analysis count or the impact of those exclusions. Because the outcome is based on judgments, reporting how disagreements and ties are treated is important for understanding what the analyzed comparisons represent.

Finally, the safety figures and the wound-level efficacy assessment use different reporting units, and the record supplies only limited details for either. The study therefore illustrates why interpretation depends not only on a stated endpoint or an event count, but also on the analysis population, handling of paired observations, missingness, and complete results. Those details are not fully reported in the registry record.

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 most consequential limitation for interpretation is that the registry record does not report the primary or secondary outcome results. It provides no treatment-favoring outcome counts, effect estimate, confidence interval, or p-value. The stated endpoint and its assessment procedure therefore cannot be connected to a reported comparative finding.

The study is non-randomized, and the record does not report how treatment was assigned between wound halves or separate wounds. In general, non-random allocation can leave treatment comparisons vulnerable to systematic differences between treated and comparator areas. Although the design compares areas within participants, it does not remove possible differences between wounds, and the record does not provide wound-level baseline characteristics or adjustment methods.

The primary outcome analysis excluded controversial and equal assessments. The number included in the final analysis, the handling of assessor disagreement, and the effect of excluding these comparisons are not reported in the registry record. The record also does not describe how two treatment cycles in two participants were handled statistically, or whether dependence among multiple wounds from a participant was accounted for.

The single masking designation is not explained, although the outcome description states that the experts were blind to treatment. The record does not report assessor agreement, missing-data procedures, a full analysis plan, or multiplicity handling. The sample-size justification is also not reported. The enrollment of 10 participants and the evaluated 12 wounds provide limited scale for assessing variability, but the absent results prevent direct evaluation of precision. Safety reporting is brief: event details are not listed for the six participants in the other adverse event category, and deaths are not reported in the registry record.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Oleogel-S10 in Wound Healing of Inherited Epidermolysis Bullosa (BEB-10) — NCT01294241.
• Registry record last updated 2018-01-23; status last verified 2017-05.
• Results first posted 2018-01-23.
• 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

NCT01294241 is a completed Phase 2, non-randomized, single-group study sponsored by Birken AG. It enrolled 10 participants with inherited epidermolysis bullosa and compared Oleogel-S10 plus Mepilex® dressing with Mepilex® dressing alone in an intra-individual wound assessment.

The primary endpoint was a blinded assessment of which wound or wound half reepithelialized faster by Day 14 for recent wounds or Day 28 for chronic wounds, or whether there was no difference. The record describes 12 wounds evaluated in 10 participants and notes exclusions of controversial and equal assessments from the primary analysis.

Primary and secondary outcome results are not reported in the registry record. The safety summary lists zero participants affected by serious adverse events and six affected in the other adverse event category, with 10 at risk in each category. The death count is not reported in the registry record.