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 Cemiplimab-rwlc for Unresectable Locally Recurrent and/or Metastatic CSCC (NCT04242173), a na phase 2 study. The primary outcome is Overall Response Rate.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT04242173 |
| Phase | Phase 2 |
| Condition | Cutaneous Squamous Cell Carcinoma, Cutaneous Squamous Cell Carcinoma of the Head and Neck |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 3 |
| Arms in the primary outcome | n/a |
| Lead sponsor | H. Lee Moffitt Cancer Center and Research Institute |
| Results posted | Yes |
2. Clinical Question
Population
Immunocompromised patients with unresectable locally recurrent and/or metastatic cutaneous squamous cell carcinoma (CSCC) were the intended population. The registry record also lists cutaneous squamous cell carcinoma of the head and neck among the conditions studied. It does not report further eligibility details or the characteristics of enrolled participants.
Intervention
The intervention was cemiplimab-rwlc. The treatment description states that immunocompromised patients would receive it every 3 weeks. The registry record does not report the dose or treatment duration.
Comparator
There was no comparison group. This was a single-group study with no masking and no randomized allocation. As a result, the record cannot provide a direct comparison of cemiplimab-rwlc with another treatment or with a control group.
Primary question
The primary question was whether patients would have an overall response, assessed by RECIST 1.1 using radiologic scans, with some patients assessed using composite efficacy criteria that could include clinical photographs. The primary outcome timeframe was up to 12 months. The registry record states that response data were not collected because the number of patients accrued was low, so it does not answer the response question with an observed trial estimate.
3. Trial Design
This was a phase II, single-group study sponsored by H. Lee Moffitt Cancer Center and Research Institute. The registry record lists an enrollment of 3 and a status of terminated. The listed study start date is 2020-06-25, and the primary completion date is 2022-05-26. These dates describe the record’s study timeline; they do not establish why the study ended.
All participants were assigned to the cemiplimab-rwlc treatment group, described as receiving treatment every 3 weeks. Allocation is listed as not applicable, and masking is listed as none. The design therefore has no randomized comparator and no blinding. Any outcome observed in the treatment group would be a description of that group, not an estimate of a treatment contrast against a concurrent control.
The primary outcome was overall response rate (ORR), assessed through RECIST 1.1 radiologic scans, with composite efficacy criteria for some patients. Progression-free survival and overall survival are listed as secondary outcomes. The registry record does not report outcome analyses for those endpoints. It also states that primary response data were not collected because of low accrual. Thus, the small enrollment is not simply a source of imprecision around a reported response estimate: the response estimate itself is absent.
The registry record does not report the reason for termination, a planned enrollment target, a sample-size justification, or details of the statistical analysis plan. Those omissions limit what can be concluded about the intended operating characteristics of the study and the circumstances leading to its termination.
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 | Cemiplimab-rwlc Treatment |
|---|---|
| STARTED | 3 |
| COMPLETED | 3 |
| NOT COMPLETED | 0 |
Analysis population for the primary outcome, as defined in the registry: Data was not collected due to low number of patients accrued to study
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: Study terminated early due to lack of interest
| Characteristic | Unit | Cemiplimab-rwlc Treatment |
|---|---|---|
| Participants analysed | 3 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 1 |
| Age, Categorical: >=65 years | Participants | 2 |
| Sex: Female, Male: Female | Participants | 0 |
| Sex: Female, Male: Male | Participants | 3 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 3 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| 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 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 1 |
| Race (NIH/OMB): White | Participants | 2 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Region of Enrollment: United States | participants | 3 |
6. Primary Endpoint
The registry lists the primary outcome as Overall Response Rate, measured over Up to 12 months.
| Element | Registry entry |
|---|---|
| Result type | N/A |
| Measure of spread | not applicable |
| Unit | not stated |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint was overall response rate, a measure of the proportion of evaluated patients who meet predefined criteria for tumor response. In this study, response was to be assessed using RECIST 1.1 analysis of radiologic scans. For some patients, assessment could also use clinical photographs and radiologic scans under composite efficacy criteria.
The endpoint description defines which superficial clinical lesions could be measured: examples include skin nodules and palpable lymph nodes, and qualifying lesions had to be at least 10 mm (at least 1 cm) in diameter when assessed using calipers. It also states that patients deemed not evaluable by RECIST 1.1 or the composite criteria would be considered not to have reached ORR. This rule makes evaluability part of how the response outcome is defined, rather than treating an unevaluable assessment as a response.
ORR is used in oncology trials to summarize observed tumor shrinkage according to specified criteria. It is an endpoint about response classification, not by itself a complete account of duration of response, progression, survival, or comparative benefit. Those distinctions matter particularly in a single-group study, where no control group is available for a treatment contrast.
The planned timeframe was up to 12 months. However, the registry record states that response data were not collected because of low accrual. It therefore contains no observed ORR estimate to interpret.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The main statistical finding is the absence of a primary response result. The registry record states that data were not collected because of low accrual, and it lists no analyses. Consequently, there is no posted ORR estimate, confidence interval, or hypothesis-test result from which to assess the observed response rate or its precision. This is not equivalent to observing no responses, and it does not establish that the response rate was zero.
The enrollment was 3, but that count must not be mistaken for a response analysis population. The record explicitly says primary response data were not collected. It therefore does not support a calculation of the proportion responding, nor an estimate of uncertainty around that proportion. Even where a small sample has been measured, estimates can be imprecise; in this record, the more basic limitation is that the primary outcome result is missing altogether.
The study’s single-group design also limits interpretation. Had an ORR estimate been reported, it would have described outcomes among participants in the cemiplimab-rwlc group. Without a concurrent control, that estimate could not by itself quantify a difference relative to another treatment or separate treatment effects from other influences on outcomes. The registry record offers no direct randomized comparison.
Progression-free survival and overall survival are listed as secondary outcomes, but their analyses and results are not reported in the registry record. Their inclusion as planned outcomes is not evidence that either endpoint was measured or that a result was obtained.
The adverse-event summary is separate from the missing efficacy result. It records 3 participants at risk for serious adverse events, 0 affected, and 0 deaths. For other adverse events, 3 were at risk and 1 was affected. These counts are descriptive for the reported group and do not establish a comparative safety profile. In particular, the small enrollment and lack of a control group mean the counts should be read as limited registry observations, not as a precise estimate of event risk.
Overall, the registry record establishes the intended question and gives limited enrollment and safety counts, but does not provide the primary efficacy data needed to estimate ORR. The appropriate statistical conclusion is that the treatment response question remains unanswered by the posted trial results, not that the treatment did or did not produce responses.
9. Statistical Methods Explained
Statistical methods help distinguish the quantity a study intended to measure from the evidence it actually reports. In this record, the primary quantity was ORR under RECIST 1.1 or, for some patients, composite efficacy criteria. The record specifies assessment rules and a timeframe up to 12 months, but also states that the response data were not collected because of low accrual. No analysis is listed. Because the outcome was not reported, the usual inferential summaries cannot be interpreted for this study.
What would an ORR estimate represent?
In general, an ORR estimate is a proportion: the number of participants classified as having a response divided by the number included in the response analysis. The analysis denominator matters. Eligibility, assessment availability, and the handling of participants who cannot be evaluated can affect the definition of that denominator. Here, the endpoint description states that participants deemed not evaluable by the specified criteria would be considered not to have reached ORR. But the record does not report an analyzed set of participants or response classifications, so there is no proportion to calculate or interpret.
What does a confidence interval add?
In general, a confidence interval expresses uncertainty around an estimate under a statistical model and procedure. For an ORR, it would communicate how imprecisely the response proportion is estimated, rather than merely presenting a point estimate. A narrow interval would indicate greater precision under the method’s assumptions, while a wide interval would indicate greater uncertainty. No ORR estimate or interval is reported in the registry record. It is therefore not possible to assess the precision of a treatment response estimate from these results.
What would a hypothesis test answer?
In general, a hypothesis test evaluates data relative to a specified null hypothesis and statistical model. A p-value describes how unusual the observed data, or data more extreme, would be under that null model; it is not the probability that a treatment works, nor a measure of the size or clinical importance of an effect. The registry record lists no analyses and reports no primary endpoint test. It also does not report a null response rate or a planned testing procedure. Accordingly, no significance conclusion can be drawn.
Why does the design matter?
This is a single-group study, not a randomized comparison. General methods for estimating a response proportion can describe outcomes within one group, but they do not create a control group or establish a treatment contrast. When there is no concurrent comparator, other explanations for observed outcomes cannot be evaluated through randomization between study groups. The registry record does not report an external control analysis or an adjusted comparison.
The study’s enrollment of 3 and the statement that response data were not collected should be considered together. A small sample can make an estimate imprecise, but the record does not present an estimate whose precision could be evaluated. It would be inappropriate to substitute a planning calculation for an observed result. The planning assumptions associated with sample-size illustrations describe a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; time-to-event illustrations use a total event count. Those are general planning assumptions, not evidence that this single-group trial had a two-arm design or achieved a specified power. The registry record does not report a sample-size justification for the trial.
For a binary response endpoint, general planning calculations may use an assumed response difference, a chosen significance level, and a desired power to illustrate the sample size that would be needed under those assumptions. Such calculations are conditional on the assumed effect and model. They are not observed power, and they do not show what this trial found. Numeric planning estimates and a trial-specific justification are not reported in the registry record. In addition, the record supplies no primary response estimate from which the observed effect could be assessed.
Over-dispersion is a general concern when observed variability exceeds what a simple statistical model expects. For example, it can affect standard errors and the reliability of model-based intervals or tests. The registry record does not report an outcome model, variance estimate, or evidence of over-dispersion. It would therefore be speculative to claim that over-dispersion was present or that a particular correction was used.
For time-to-event endpoints such as progression-free survival and overall survival, general analyses may use event times and account for censoring, when participants’ event status is not observed through the full follow-up period. Such analyses depend on event definitions, follow-up, and censoring information. Although these outcomes are listed as secondary endpoints, their analysis methods and results are not reported in the registry record. No survival estimate or event count should be inferred from their listing.
10. Multiplicity, Missing Data and Analysis Populations
Multiplicity concerns arise when several endpoints or statistical tests are evaluated. In general, the more hypotheses tested, the greater the chance that at least one result may appear statistically notable by chance unless the analysis plan addresses that issue. This study lists ORR as its primary outcome and progression-free survival and overall survival as secondary outcomes. The registry record does not report a multiplicity adjustment, a testing hierarchy, or a statistical analysis plan specifying how multiple outcomes would be interpreted. No analyses are listed, so there are no reported test results for which a multiplicity procedure can be assessed.
Missing outcome data are distinct from a negative outcome. The registry record states that primary response data were not collected because of the low number of patients accrued. It does not give participant-level assessment status or explain whether any scans were performed. Therefore, the absence of an ORR result should not be reclassified as a set of nonresponses or as a zero response rate.
The endpoint definition does state that patients deemed not evaluable under RECIST 1.1 or the composite efficacy criteria would be considered not to have reached ORR. That rule describes how an unevaluable participant would be handled in the endpoint definition. It does not establish that any participants were in fact deemed unevaluable, and it does not replace the separate registry statement that response data were not collected.
The analysis populations are not described in the registry record. It does not report an intention-to-treat population, an evaluable population, or the denominator used for efficacy analysis. The number enrolled alone cannot fill in those missing details. It is also not possible to assess whether missingness could have affected an estimated response, since no response analysis is reported.
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 |
|---|---|---|---|
| Cemiplimab-rwlc Treatment | 0 | 3 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Cemiplimab-rwlc Treatment | 0 of 3 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 0% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Cemiplimab-rwlc Treatment | 1 of 3 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Cemiplimab-rwlc Treatment |
|---|---|---|
| Leukocytosis | Blood and lymphatic system disorders | 1 of 3 |
| Hyperkalemia | Metabolism and nutrition disorders | 1 of 3 |
The safety summary reports counts for the cemiplimab-rwlc treatment group. For serious adverse events, 3 participants were at risk and 0 were affected; deaths were 0. For other adverse events, 3 were at risk and 1 was affected. These are participant counts in the registry summary, not rates estimated from a comparative analysis.
No serious adverse-event terms are listed. The record therefore does not identify a specific serious event in the posted summary. The count of 0 affected among 3 at risk describes what was recorded for this group; it does not prove that serious events are impossible or establish how the group compares with patients receiving another treatment. There was no control group.
The count of 1 affected by other adverse events does not, by itself, describe the event’s type, severity, timing, duration, or relationship to treatment. Those details are not reported in the registry record. The summary should be read as a limited descriptive safety account alongside the small enrollment, not as a complete estimate of safety or a basis for comparative conclusions.
12. What the Result Does, and Does Not, Mean
The registry record documents a phase II, single-group study intended to assess cemiplimab-rwlc in immunocompromised patients with unresectable locally recurrent and/or metastatic CSCC. It identifies ORR as the primary outcome and describes assessment using RECIST 1.1, with composite criteria for some patients. It also records enrollment of 3 and a terminated status.
The central limitation for efficacy interpretation is that the registry states response data were not collected because of low accrual. There is no reported response proportion, no confidence interval, and no primary endpoint analysis. The result therefore cannot be interpreted as evidence of a high or low response rate. Lack of a reported estimate is not the same as evidence of no responses.
The design also cannot answer a comparative question. Because the study had a single group and no randomized comparator, it does not estimate how outcomes differed from those under another treatment or control condition. Even a reported response proportion would have been descriptive of the treated group rather than a randomized treatment effect.
The record lists progression-free survival and overall survival as secondary outcomes, but their results are not reported. Safety information is limited to the adverse-event counts in the record and cannot establish comparative risk. Neither those counts nor the listed outcomes resolve the missing primary efficacy result.
Thus, the registry record contributes information about the intended study question, design, enrollment, and limited safety reporting. It does not provide sufficient outcome data to estimate response or to make a statistical comparison. The response question remains unanswered in the posted results.
13. Why This Trial Matters Statistically
This trial is statistically informative in part because it illustrates the difference between a planned endpoint and an analyzable result. ORR was specified as the primary outcome, with criteria describing how lesions and unevaluable assessments would be handled. Yet the registry record states that response data were not collected because of low accrual. An endpoint definition alone cannot provide an estimate: the outcome classifications and analysis denominator are also needed.
The record also shows why design and inference must be considered together. A single-group study can describe outcomes among participants who receive the intervention, but it does not provide a randomized control group for estimating a treatment contrast. This distinction remains important even when an endpoint is clearly defined. A descriptive result and a comparative effect answer different questions.
Low accrual affects the information available, but the statistical issue is not only sample size. With no primary outcome result reported, precision cannot be quantified and response cannot be estimated. No inferential result should be created from enrollment alone. The adverse-event counts are similarly descriptive and should not be mistaken for a comprehensive or comparative safety analysis.
Finally, the record distinguishes outcomes listed for study from outcomes with posted analyses. Progression-free survival and overall survival appear as secondary outcomes, but no results are reported. Recognizing that distinction helps prevent an outcome’s presence in a registry listing from being interpreted as evidence that the endpoint was successfully analyzed. The record is therefore useful for understanding what was intended and what was reported, while leaving the principal response question unresolved.
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 trial-specific limitation is that primary response data were not collected because of low accrual. As a result, there is no reported ORR estimate or uncertainty interval. The enrollment of 3 does not provide a substitute for an outcome analysis, and no conclusion about observed response can be drawn.
The single-group design offers no concurrent randomized comparison. Any reported within-group outcome would not, on its own, establish a difference relative to another treatment. The absence of masking is also recorded, but the registry does not describe any procedures to limit assessment bias. It is not possible to determine from the record whether or how such procedures were used.
The registry record does not report the reason for termination, the planned sample size, a sample-size justification, or a complete statistical analysis plan. It also does not report the populations used for analysis or participant-level outcome availability. These omissions prevent assessment of whether the planned methods were followed and how missing assessments would have affected an efficacy estimate.
Progression-free survival and overall survival are listed as secondary outcomes, but their analyses and results are not reported. The safety summary gives limited counts without detailed event descriptions or a comparison group. Together, these constraints leave both efficacy and safety interpretation descriptive and incomplete.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Cemiplimab-rwlc for Unresectable Locally Recurrent and/or Metastatic CSCC — NCT04242173.
- Registry record last updated 2022-11-04; status last verified 2022-11.
- Results first posted 2022-07-14.
- 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 record describes a phase II, single-group study of cemiplimab-rwlc in immunocompromised patients with unresectable locally recurrent and/or metastatic CSCC. The treatment description specifies administration every 3 weeks. Enrollment was 3, and the study status is terminated.
ORR, assessed using RECIST 1.1 and composite criteria for some patients, was the primary outcome with a timeframe up to 12 months. The registry states that response data were not collected because of low accrual. Progression-free survival and overall survival are listed as secondary outcomes, with no analyses reported.
The safety summary records 0 serious adverse events among 3 at risk, 1 affected by other adverse events among 3 at risk, and 0 deaths. The single-group design and missing efficacy data do not support a comparative treatment estimate.