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 Extension Study of Patidegib Topical Gel, 2% in Subjects With Gorlin Syndrome (Basal Cell Nevus Syndrome) (NCT04308395), a na phase 3 study. The primary outcome is Incidence of Treatment-emergent Adverse Events (TEAEs).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT04308395 |
| Phase | Phase 3 |
| Condition | Basal Cell Nevus Syndrome |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 108 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Sol-Gel Technologies, Ltd. |
| Results posted | Yes |
2. Clinical Question
Population
Adults with Gorlin syndrome, also called basal cell nevus syndrome. The registry describes the study population as adult subjects with Gorlin syndrome and records Basal Cell Nevus Syndrome as the condition.
Intervention
Patidegib Topical Gel, 2%, applied topically to the face twice daily. The registry describes the study as an extension study evaluating safety.
Comparator
There is no comparator group in the registry design. The study is listed as single group, with allocation marked NA and masking listed as none.
Primary question
What number of subjects experienced treatment-emergent adverse events during the 12 months specified for the primary outcome? The record's primary outcome is the incidence of TEAEs, measured as number of subjects.
This is a safety question, not a randomized treatment comparison. The registry describes an open-label extension study and names patidegib topical gel as the intervention. Because there is no control group, the design does not directly compare adverse-event experience under patidegib with experience under another treatment or with no treatment.
The primary outcome is framed around whether subjects experienced treatment-emergent adverse events. That makes the subject, rather than the total number of individual event episodes, the unit of the stated outcome. The registry's description also explains that a unique adverse event of a preferred term is counted only once within each subject. It defines a related adverse event as one judged definitely, probably or possibly related.
The registry record reports selected adverse-event counts, including serious events and other adverse events, but does not report an overall result for the primary outcome. Nor does it report efficacy findings for the listed secondary outcomes. Therefore, the clinical question that can be assessed from this record is limited to the safety information it records; it cannot establish a comparative treatment effect.
3. Trial Design
This is a phase 3, multicenter, open-label extension study of Patidegib Topical Gel, 2%, in subjects with Gorlin syndrome. The registry lists a single-group model, allocation as NA, and masking as none. Its brief summary describes topical application to the face twice daily, and the primary outcome has a 12-month timeframe.
The registry records enrollment of 108. The study start date is 2020-06-03, and the primary completion date is 2021-07-14. Its status is terminated. The reason for termination is not reported in the registry record, so the status alone cannot explain why the study ended or whether that reason was related to safety, recruitment, operations, or another issue.
An extension study follows an earlier study or treatment period, but details of the preceding study and the eligibility route into this extension are not reported in the registry record. The available description identifies the intended population and intervention but does not specify the prior-treatment requirements, the extension's detailed visit schedule, or how long individual subjects were observed beyond the primary outcome timeframe.
With no randomized allocation, masking, or control arm recorded, this design is not a like-for-like comparative trial. The open-label structure means treatment assignment was not masked in the registry design; the record does not describe any blinding of outcome assessors. This matters especially when interpreting outcomes that can be influenced by assessment or reporting, though the primary safety endpoint is defined as a count of subjects with events.
The registry lists seven outcome measures, including the primary TEAE outcome and secondary measures concerning facial BCCs removed by surgery, new lesions suspicious for BCC, aBCCdex lesion score, DLQI, and EQ 5D 5L. These measures cover safety, disease-related observations, and quality-of-life instruments, but the registry record does not supply results for each outcome. The planned sample size, recruitment rationale, and detailed analysis plan are not reported 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.
Overall Study
| Stage | Patidegib Topical Gel, 2% |
|---|---|
| STARTED | 108 |
| COMPLETED | 3 |
| NOT COMPLETED | 105 |
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.
| Characteristic | Unit | Patidegib Topical Gel, 2%, |
|---|---|---|
| Participants analysed | 105 | |
| Age, Continuous | years | 53.9 (SD 13.9) |
| Sex: Female, Male: Female | Participants | 59 |
| Sex: Female, Male: Male | Participants | 46 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 6 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 84 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 15 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 8 |
| Race (NIH/OMB): Black or African American | Participants | 0 |
| Race (NIH/OMB): White | Participants | 87 |
| Race (NIH/OMB): More than one race | Participants | 2 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 8 |
| Height | cm | 175.9 (SD 10.0) |
| Weight | kg | 93.1 (SD 21.4) |
| BMI | kg/m2 | 29.9 (SD 6.2) |
6. Primary Endpoint
The registry lists the primary outcome as Incidence of Treatment-emergent Adverse Events (TEAEs), measured over 12 months.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | Number of subjects |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the incidence of treatment-emergent adverse events, expressed as the number of subjects. Its timeframe is 12 months. In general, a treatment-emergent event is an adverse event arising or worsening after treatment begins; the registry record names the endpoint but does not provide a fuller operational definition of how onset or worsening was determined.
Counting subjects with an event answers how many people experienced an adverse event, rather than how many event episodes occurred. This distinction is useful because repeated events in the same subject do not necessarily represent additional affected subjects. The registry's endpoint description further says that an adverse event of a given preferred term is counted only once within each subject. This rule concerns repeated events of the same term within an individual; it does not establish how events with different terms were combined for the overall endpoint.
Safety endpoints are used to characterize unwanted medical events observed during treatment and follow-up. They can describe the frequency of affected subjects in the study group, but without a comparator they cannot by themselves show how much of that experience was caused by treatment rather than by the underlying condition, background health, or other factors.
The registry lists the primary measure as a subject count, but an overall TEAE count or proportion is not reported in the registry record. The adverse-event summary gives separate counts for serious and other events. Those categories are relevant safety information, but they should not be treated as an overall primary-endpoint result or combined without information about overlap and endpoint rules.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record describes a single-group safety study and reports an adverse-event summary for Patidegib Topical Gel, 2%. In that summary, 105 subjects are listed as at risk for serious adverse events, and 3 are listed as affected. For other adverse events, 105 are listed as at risk, and 46 are listed as affected. These are separate category summaries; the record does not report whether the subjects in the categories overlap. It therefore does not support adding the affected counts or treating them as mutually exclusive groups.
The serious adverse-event terms listed are atrial fibrillation, craniocerebral injury, and breast cancer, each recorded at 1.0%. These term-level figures describe what the registry lists; the record does not give further detail about event timing, severity beyond the serious classification, treatment, outcome, or relationship to the investigational medicinal product for each listed term. The registry reports deaths as 0 in the adverse-event summary. That figure should be read as the recorded count, not as evidence that the study's treatment eliminates mortality risk.
The primary endpoint is the number of subjects with treatment-emergent adverse events over 12 months. The summary counts for serious and other events do not state the total number of subjects with any TEAE. The overall primary-endpoint result is not reported in the registry record. In addition, no uncertainty interval or inferential analysis for these counts is reported in the registry record, so their precision cannot be judged from the available summary.
Even a complete count of events in this group would describe observed experience, not establish causation. An open-label, single-group design has no concurrent comparison to distinguish treatment-associated events from events that might occur in this population without the study intervention. The registry does not report an adjudication method or an analysis comparing related and unrelated events. It defines related events as those assessed as definitely, probably, or possibly related, but no related-event count is given in the record.
The study status is terminated, but the reason is not reported in the registry record. That status does not itself establish that treatment was stopped for a safety concern, that follow-up was incomplete, or that the stated outcomes were not collected. Those explanations would be speculation. The most defensible reading is therefore descriptive: the record lists the specified safety counts and terms, while leaving the overall primary-endpoint result, uncertainty, and study termination reason unreported.
9. Statistical Methods Explained
The registry identifies the study as single group, open label, and without masking. That design determines what statistical questions can be answered. A single-group summary can describe how many subjects in the study experienced an outcome, but it cannot estimate a direct contrast with an untreated or alternative-treatment group. Randomization is not listed, and allocation is marked NA. Consequently, the results should be read as observations within this study population rather than as an estimate from a randomized comparison.
What does a subject count represent?
The primary outcome is measured as number of subjects. This is a participant-level summary: it concerns subjects who experienced the outcome, not the total number of event episodes. In general, counts of affected subjects are easier to interpret as the number of people with an event, while event counts can include repeat episodes in an individual. The registry's endpoint description says that a unique adverse event of a certain preferred term is counted only once within each subject. It does not report a full analysis specification for how all event terms contribute to the overall primary endpoint.
The adverse-event summary lists 105 at risk for each of its serious and other categories. It reports 3 affected for serious events and 46 affected for other events. A denominator describes the subjects considered at risk for that category; an affected count describes subjects recorded with an event in that category. These summaries do not identify overlap between categories, and should not be added together. They also do not supply the overall number with any treatment-emergent adverse event.
How should event relatedness be understood?
The registry defines a related adverse event as one with a relationship assessed as definitely, probably, or possibly related. This is a categorization of the assessed relationship, not a statistical test establishing that the intervention caused an event. The registry record does not report the number of related events or describe who made those assessments, what evidence they considered, or whether assessment was independent. The term-specific serious-event percentages therefore should not be read as causal estimates.
In general, a confidence interval communicates the range of values reasonably compatible with an estimate under a statistical model and reflects uncertainty from the available data. No confidence interval for the adverse-event counts is reported in the registry record. The posted counts can be described, but the precision of the corresponding event rates cannot be assessed from the information recorded. No significance test or comparative effect estimate is reported either.
What do the planning assumptions mean?
The registry's sample-size assumptions describe a planning illustration based on an observed effect, not the original trial design. The illustration uses a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; the effect to detect is expressed as a stated fraction of the observed effect. For non-time-to-event outcomes, the assumptions define the output as participants per arm. These hypothetical two-arm planning assumptions do not change the actual study design, which is recorded as single group with allocation NA. They are not evidence that the trial was planned with those assumptions, and sample-size illustrations are not observed power.
In general, a sample-size calculation estimates how much information a future study might need to detect a specified effect under stated assumptions. It does not show the probability that the completed study's findings are true, nor does it compensate for the absence of a concurrent comparator. The detailed planning inputs and any trial-specific sample-size justification are not reported in the registry record. The observed effect used in the illustration and numerical planning outputs are not stated in the registry record details available for interpretation.
Why do design and event definitions matter?
Open-label studies can be vulnerable to differences in event reporting and assessment because treatment status is known. This general concern does not prove that reporting bias occurred in this study. It indicates why details such as standardized event collection, independent review, and consistent follow-up are useful. Those procedures are not reported in the registry record. The extension format also makes prior study and treatment history potentially relevant context, but those details are not reported in the registry record.
The registry lists the primary endpoint timeframe as 12 months, but does not report individual follow-up duration, censoring rules, or how missing observations were handled. In general, unequal observation time can affect the opportunity to record events, while missing follow-up can make event counts incomplete. Without the analysis rules and follow-up information, these potential issues cannot be quantified or resolved from the record.
Lastly, the registry reports zero outcome analyses. That does not erase the adverse-event counts in the record, but it means no registered analytical results such as adjusted comparisons or model-based estimates are available to accompany them. A reader should distinguish descriptive safety counts from inferential analyses and avoid reading statistical conclusions into values that the record does not report.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists a primary safety outcome and secondary outcomes concerning facial BCCs removed by surgery, new lesions suspicious for BCC, aBCCdex lesion score, DLQI, and EQ 5D 5L. When a study assesses multiple outcomes, testing each one can increase the chance of at least one apparently notable result arising by chance. In general, multiplicity procedures can control that risk, or investigators can designate outcomes as descriptive. The registry record does not report a multiplicity strategy, hierarchy among secondary outcomes, or statistical testing plan.
The record reports zero outcome analyses. It does not give analysis results for the listed secondary outcomes or specify whether any planned analyses were conducted. A lack of registered analyses should not be interpreted as proof that no analyses occurred outside the registry; it means those analyses are not reported in the registry record.
Missing-data handling is not reported in the registry record. There is no reported method for handling missing endpoint assessments, incomplete follow-up, withdrawals, or subjects with no recorded event information. In general, these choices matter because the observed data may not represent all enrolled subjects if follow-up is incomplete. The record does not provide enough detail to assess the extent or direction of any such issue.
The analysis population is also not reported in the registry record. Although enrollment is recorded as 108 and the adverse-event summary lists 105 at risk for each category, the record does not explain how the at-risk groups relate to enrollment or identify the rules used to include subjects in the primary analysis. Those counts should therefore be reported as recorded, without assuming a particular exclusion, completion, or analysis-population rule.
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 |
|---|---|---|---|
| Patidegib Topical Gel, 2% | 3 | 105 | 2.9% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Patidegib Topical Gel, 2% | 0 of 105 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 5% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Patidegib Topical Gel, 2% | 46 of 105 |
Most frequent serious adverse event terms
| Event term | Organ system | Patidegib Topical Gel, 2% |
|---|---|---|
| Atrial fibrillation | Cardiac disorders | 1 of 105 |
| Craniocerebral injury | Injury, poisoning and procedural complications | 1 of 105 |
| Breast cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 1 of 105 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Patidegib Topical Gel, 2% |
|---|---|---|
| No adverse event occured in 5% or more of the patients | General disorders | 46 of 105 |
Serious events with the highest arm-level rate: atrial fibrillation (1.0%), craniocerebral injury (1.0%), breast cancer (1.0%).
The adverse-event summary lists 105 subjects at risk for serious events and 3 affected. It also lists 105 at risk for other adverse events and 46 affected. The record does not state whether subjects appear in both categories, so the affected counts must remain separate and should not be combined to create a total. The overall number of subjects with any treatment-emergent adverse event is not reported in the registry record.
The serious-event terms are atrial fibrillation, craniocerebral injury, and breast cancer, each listed at 1.0%. These figures are term-specific entries in the registry summary. The record does not describe whether any subject had more than one listed term, provide event narratives, or report the relationship assessment for those events. The related-event definition includes events assessed as definitely, probably, or possibly related, but the summary does not provide a related-event count.
The reported death count is 0. This is a descriptive registry entry and does not establish the absence of risk beyond the study record. Serious adverse events, other adverse events, and deaths are distinct summaries; their definitions and denominators should not be assumed interchangeable. The record also does not provide uncertainty intervals for these figures, so their statistical precision cannot be assessed from the available information.
12. What the Result Does, and Does Not, Mean
The record supports a limited descriptive statement: in the patidegib group, it lists 3 subjects affected by serious adverse events among 105 at risk and 46 affected by other adverse events among 105 at risk. It also lists three serious-event terms, each at 1.0%, and records deaths as 0. These values are safety summaries, not a comparison between treatments.
They do not establish that patidegib caused the reported events or prevented them. In general, a single-group study cannot separate events related to treatment from events that might have occurred because of the underlying condition, background health, or other circumstances. The registry defines relatedness categories, but does not provide a related-event result or supporting assessment details. No controlled comparison is available in the recorded design.
The primary endpoint is the incidence of treatment-emergent adverse events measured as number of subjects over 12 months. However, the record does not report the overall primary-endpoint result. The serious and other adverse-event counts should not be substituted for that missing total because the record does not establish how the categories overlap or how they map to the overall endpoint.
The record also does not report results for its listed secondary outcomes. Therefore, it does not support a conclusion about changes in BCC surgery, new suspicious lesions, aBCCdex score, or quality-of-life measures. The study's terminated status is recorded, but its reason is not reported in the registry record. Nothing in the status alone explains whether termination was related to safety, feasibility, or another consideration.
In general, a treatment-effect conclusion requires a clearly defined comparison and an analysis that accounts for uncertainty and study conduct. The available information is instead a partial safety description from an open-label, single-group extension study. It can inform what the registry records for this group, but not a causal or comparative claim about treatment effects.
13. Why This Trial Matters Statistically
This study illustrates why the design behind a safety result matters as much as the event counts. A single-group extension can document adverse events during treatment and follow-up in a defined clinical population. It can also add safety observations in an open-label setting. But it does not provide the concurrent comparison needed to distinguish the observed event experience from what might have happened without the intervention or under another treatment.
The endpoint is specified as a number of subjects with treatment-emergent adverse events, which highlights the importance of defining the unit of analysis. A person-level count differs from a total of event episodes, and the registry's rule for counting a unique preferred term once within each subject is relevant to interpreting such summaries. Even with that rule, the record does not provide an overall primary-endpoint result or enough detail to reconstruct how different event terms were combined.
The recorded adverse-event information demonstrates another common interpretive challenge: serious and other events are presented as separate categories, but their overlap is not described. Reporting the category counts separately preserves what the record says without implying a combined total or a mutually exclusive classification. Term-specific percentages likewise need their assessment and denominator context to support stronger interpretation.
The record also underscores the distinction between a study's actual design and a planning illustration. The stated sample-size assumptions describe a hypothetical two-arm calculation, while the registered study is single group. Keeping those purposes separate prevents planning assumptions from being mistaken for trial design or observed statistical power.
Finally, registry completeness affects what can be concluded. Analysis populations, missing-data handling, multiplicity procedures, uncertainty estimates, and the reason for termination are not reported in the registry record. These omissions do not establish that particular problems occurred, but they constrain statistical assessment. A careful reading recognizes both the safety information that is recorded and the inferential questions that remain unanswered.
14. Limitations
- The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
- Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.
The central limitation is the single-group, open-label design. There is no comparator, and the registry records allocation as NA and masking as none. As a result, the adverse-event summaries describe observations in the patidegib group but do not estimate a comparative safety effect. The study design also cannot, by itself, establish that events were caused by the intervention.
The registry record does not report the overall number of subjects with treatment-emergent adverse events, despite identifying that measure as the primary endpoint. It gives serious and other adverse-event summaries instead, without stating whether the affected groups overlap or how those summaries map onto the overall endpoint. It also does not report confidence intervals, statistical tests, or model-based results for the safety counts.
Further details needed to assess completeness and precision are not reported in the registry record. These include individual follow-up duration, missing-data rules, the analysis population, event collection and adjudication procedures, and the number of related adverse events. The enrollment entry and adverse-event at-risk counts are both recorded, but the rules connecting these populations are not described.
The registry lists secondary outcomes but does not report results for them. It also reports zero outcome analyses, and does not specify a multiplicity plan or analysis hierarchy. The reason for the study's terminated status is not reported in the registry record. Without these details, it is not possible to determine from the registry whether results were unavailable, uncollected, or analyzed but not entered.
These are limitations of what can be concluded from the registry record, not evidence that an unreported procedure was absent or that an unreported event occurred. The findings should therefore remain descriptive and confined to the recorded safety information.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Extension Study of Patidegib Topical Gel, 2% in Subjects With Gorlin Syndrome (Basal Cell Nevus Syndrome) — NCT04308395.
- Registry record last updated 2024-09-24; status last verified 2024-07.
- Results first posted 2024-09-24.
- 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 NCT04308395, a phase 3, multicenter, open-label extension study of Patidegib Topical Gel, 2%, in subjects with Gorlin syndrome. It records a single-group design, allocation as NA, no masking, enrollment of 108, and terminated status. The study started on 2020-06-03, with primary completion on 2021-07-14.
The primary outcome is incidence of treatment-emergent adverse events, measured as number of subjects over 12 months. The adverse-event summary lists 105 at risk and 3 affected for serious events, and 105 at risk and 46 affected for other events. Three serious-event terms are listed at 1.0% each; the reported death count is 0.
The overall primary-endpoint result, secondary-outcome results, statistical analysis plan, analysis population, missing-data methods, and reason for termination are not reported in the registry record. The single-group design supports descriptive safety reporting, not a controlled treatment comparison.