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 Temsirolimus (Torisel®) and Erlotinib (Tarceva®) in Platinum-Refractory/Ineligible, Advanced, Squamous Cell Carcinoma (NCT01009203), a na phase 2 study. The primary outcome is Progression Free Survival (PFS).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01009203 |
| Phase | Phase 2 |
| Condition | Squamous Cell Carcinoma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 13 |
| Arms in the primary outcome | n/a |
| Lead sponsor | New Mexico Cancer Research Alliance |
| Results posted | Yes |
2. Clinical Question
Population
The study concerned people with advanced squamous cell carcinoma of the head and neck who were platinum-refractory or platinum-ineligible. The registry record does not report further details about the primary analysis population.
Intervention
The regimen combined erlotinib, 150 mg by mouth daily, with temsirolimus, 15 mg intravenously weekly. Each cycle was 28 days.
Comparator
There was no concurrent control group. The study was single-group; the registry summary describes historical progression-free survival with erlotinib or cetuximab monotherapy as the reference for its expectation.
Primary question
The stated primary question was whether progression-free survival with the combination would be prolonged compared with historical progression-free survival in people treated with erlotinib or cetuximab alone. The registry record does not report the primary endpoint result.
The study rationale was that blocking mTOR in addition to EGFR might produce synergistic clinical activity. The registry summary says this rationale was consistent with preclinical xenograft data. That rationale is a hypothesis, not a clinical outcome. The trial’s single-group design can describe outcomes among participants receiving the combination, but it does not create a concurrent comparison that could separate treatment effects from differences in patient characteristics, care, or assessment between the study and historical populations.
3. Trial Design
This was a phase II, single-group study sponsored by the New Mexico Cancer Research Alliance. The registry lists enrollment of 13 and identifies the study status as terminated. The listed start was 2009-12, and primary completion was 2012-09. The record identifies no allocation scheme and no masking.
The treatment arm was named Erlotinib and Temsirolimus. The regimen description specifies erlotinib at 150 mg by mouth daily and temsirolimus at 15 mg intravenously weekly, with each cycle lasting 28 days. The registry describes the regimen as being given at previously established maximal tolerated doses; details of that prior dose-establishment process are not reported in the registry record.
The primary outcome was median progression-free survival, measured in months, with full range as the stated dispersion. Its timeframe was 3 years. The registry lists toxicity profile, overall response rate, and overall survival as secondary outcomes. It lists four outcome measures in total, but the primary progression-free survival result itself is not reported in the registry record. The record lists zero analyses.
Because this was not a randomized comparison, the historical reference described in the study summary is not equivalent to a control arm enrolled and followed under the same study conditions. The registry record does not report a concurrent comparator, a detailed statistical analysis plan, or a sample-size justification. Any planning illustration based on an observed effect should therefore be kept distinct from the study’s actual design and from observed trial evidence.
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 | Erlotinib and Temsirolimus |
|---|---|
| STARTED | 13 |
| Received Treatment | 12 |
| COMPLETED | 6 |
| NOT COMPLETED | 7 |
Reasons for not completing this period:
| Reason | Erlotinib and Temsirolimus |
|---|---|
| Withdrawal by Subject | 1 |
| Adverse Event | 5 |
| Treatment-unrelated death | 1 |
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 | Erlotinib and Temsirolimus |
|---|---|---|
| Participants analysed | 13 | |
| Age, Continuous | years | 61.5 (45 to 87) |
| Sex: Female, Male: Female | Participants | 1 |
| Sex: Female, Male: Male | Participants | 12 |
6. Primary Endpoint
The registry lists the primary outcome as Progression Free Survival (PFS), measured over 3 years.
| Element | Registry entry |
|---|---|
| Result type | Median |
| Measure of spread | Full Range |
| Unit | Months |
| Endpoint classification | Time-to-Event Analysis |
Progression-free survival is the time from treatment initiation until disease progression or death from any cause. The registry states that progression was assessed using modified Response Evaluation Criteria in Solid Tumors, version 1.0. Target lesions were assessed by CT or MRI. The listed progression criteria include a 20% increase in the sum of the longest diameters of target lesions, unequivocal progression of an existing non-target lesion, or the appearance of new lesions. The description also includes death due to disease without prior objective documentation of progression and global deterioration attributable to disease that requires a change in therapy without objective evidence of progression.
A time-to-event endpoint incorporates both whether an event occurs and how long it takes to occur. It can therefore characterize the timing of progression or death rather than only counting responses at a fixed assessment. The median is the point in the observed time distribution at which half the observations are on either side, subject to the methods used to handle censored follow-up. The registry specifies median and full range but does not report the median, the range, or details about censoring and estimation.
Progression-free survival was selected as the primary signal of efficacy in the study summary, which anticipated longer PFS than historical experience with erlotinib or cetuximab monotherapy. The endpoint definition and rationale are reported; the observed endpoint result is not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record establishes the study question and design but does not provide the primary progression-free survival estimate. It therefore does not support a numerical description of the observed median, its range, or the precision of that estimate. No sponsor-reported hazard ratio or confidence interval is included in the record. Without those results, the stated hypothesis cannot be assessed from the registry information available.
Even if a single-group study reports a progression-free survival estimate, that estimate describes outcomes among participants enrolled and treated in that study. A comparison with historical experience is indirect: the historical patients may differ in eligibility, disease characteristics, clinical care, follow-up, or outcome assessment. The registry record does not report the details needed to evaluate comparability with the historical reference. Thus the historical expectation in the study summary is context for the question, not a randomized control condition.
The record lists enrollment of 13, but enrollment alone does not establish how many participants received treatment, contributed to each endpoint analysis, or had complete follow-up. Those analysis counts and participant-level disposition details are not reported in the registry record. This limits interpretation of how representative any possible endpoint estimate would be, quite apart from the absence of the estimate itself.
The record identifies the study as terminated. That status is relevant context, but the reason for termination and its relationship, if any, to accrual, safety, or efficacy are not reported in the registry record. Termination should not be treated as proof of benefit, lack of benefit, or a particular safety concern.
In general, an estimate’s precision depends on the amount and distribution of observed information, not merely on the nominal enrollment. For a time-to-event outcome, the number and timing of events and the handling of censored observations matter. These details are not reported for the primary endpoint. Accordingly, the defensible interpretation is limited: the registry specifies a phase II combination study and a PFS hypothesis, but it does not report the primary result needed to judge the observed outcome or its precision.
9. Statistical Methods Explained
Statistical methods should be interpreted in light of what the registry actually reports. This record identifies a median time-to-event primary outcome, describes its definition and timeframe, and lists no analyses. It does not report the primary estimate, a confidence interval, or a sponsor-reported hazard ratio. The method concepts below explain how such quantities are generally interpreted; they do not fill in missing trial results.
What does a median time-to-event estimate describe?
For a time-to-event outcome, each participant contributes information about the time until the event or the end of known follow-up. The median is a summary of the event-time distribution: it marks the point at which the estimated distribution reaches its midpoint. It is not an average, and it does not describe every participant’s experience. The registry specifies median progression-free survival and full range as the summary format, but does not report either value. It also does not describe the estimation method or the number of participants contributing to the analysis.
Why does censoring matter?
In general, a participant may leave observation or reach the end of follow-up before progression or death is recorded. Such information is commonly treated as censored: it contributes follow-up up to the last known time without establishing when an event might later occur. Time-to-event methods account for this incomplete event-time information under assumptions about follow-up and censoring. The registry record does not report censoring counts, reasons for censoring, follow-up distribution, or the method used to estimate the endpoint. As a result, those aspects cannot be evaluated for this study from the record.
What does a confidence interval add?
In general, a confidence interval expresses uncertainty around an estimated quantity under the method used to construct it. A narrower interval indicates greater statistical precision than a wider interval, while neither width nor location alone resolves the design limitations of a historical comparison. No confidence interval for the primary progression-free survival result is reported in the registry record, so the precision of that result cannot be assessed.
How should a hazard ratio be read?
In general, a hazard ratio compares event rates over time between groups under a specified model. Its interpretation depends on the groups and analysis, and it is not interchangeable with a ratio of medians. A randomized trial can estimate a concurrent between-group contrast; a single-group trial cannot produce that randomized comparison from its treatment arm alone. The registry record says that only sponsor-reported hazard ratios are used for time-to-event analyses, but it reports no hazard ratio for this study. No such estimate should be inferred from the endpoint description.
Why do distribution and over-dispersion matter?
General statistical methods rely on assumptions about how data vary. When observed variation exceeds what a model assumes, standard errors and uncertainty estimates may be too optimistic if the extra variation is ignored; this is one reason over-dispersion matters. That concept is not evidence that over-dispersion occurred in this trial. The registry record does not report a fitted model, a dispersion assessment, or a diagnostic analysis for the PFS outcome.
How should planning calculations be separated from trial results?
Planning illustrations answer hypothetical questions about sample sizes under stated assumptions. The record’s planning description refers to a two-sided significance level of 0.05, equal allocation between two hypothetical arms, a normal approximation, and effects defined as fractions of an observed effect. It also distinguishes total events for time-to-event calculations from participants per arm for other outcomes. Those assumptions do not change this study’s actual single-group design. A planning illustration is not the number enrolled, an observed treatment contrast, or observed power. The record does not report the study’s own sample-size justification or the observed effect needed to interpret such illustrations as trial-specific planning evidence.
Because the record lists zero analyses and does not report the primary endpoint result, no conclusion about statistical significance or the magnitude of an effect can be drawn from it. General method descriptions clarify what kinds of evidence would be needed; they cannot replace the missing estimate, uncertainty, analysis population, or analytic details.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists progression-free survival as the primary outcome and toxicity profile, overall response rate, and overall survival as secondary outcomes. Considering several outcomes creates a multiplicity issue in general: the more statistical tests or outcome comparisons undertaken, the greater the chance that at least one result appears notable by chance if the analyses are treated as independent confirmations. A prespecified hierarchy, adjustment procedure, or clear distinction between primary and exploratory analyses can help readers interpret multiple results. Such procedures are not reported in the registry record.
The record lists zero analyses and does not report outcome estimates for the primary endpoint. It also does not report whether there were interim analyses, whether analysis decisions changed during the study, or what multiplicity strategy was used. These details should not be inferred from the phase or design description.
Missing-data handling is also not reported in the registry record. For progression-free survival, incomplete follow-up may be represented through censoring, but the registry does not state how censoring was handled, how participants without evaluable follow-up were treated, or whether sensitivity analyses were performed. It does not report an analysis population definition, such as which enrolled participants were included in each outcome analysis. Enrollment of 13 therefore cannot be read as the denominator for every endpoint.
These omissions limit assessment of how the analysis was conducted and how robust any estimate might be. They do not establish that a particular method was or was not used; they identify information not reported in the registry record.
11. Safety Results
The registry reports serious adverse events as affected participants divided by participants at risk.
| Arm | Participants with serious adverse events | Participants at risk | Percentage |
|---|---|---|---|
| Erlotinib and Temsirolimus | 4 | 12 | 33.3% |
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 |
|---|---|
| Erlotinib and Temsirolimus | 12 of 12 |
Most frequent serious adverse event terms
| Event term | Organ system | Erlotinib and Temsirolimus |
|---|---|---|
| Device related infection | Infections and infestations | 1 of 12 |
| Cerebrovascular Ischemia | Nervous system disorders | 1 of 12 |
| Adult respiratory distress syndrome | Respiratory, thoracic and mediastinal disorders | 1 of 12 |
| Dyspnea (Shortness of breath) | Respiratory, thoracic and mediastinal disorders | 1 of 12 |
| Death | General disorders | 1 of 12 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Erlotinib and Temsirolimus |
|---|---|---|
| Fatigue | General disorders | 10 of 12 |
| Rash | Skin and subcutaneous tissue disorders | 7 of 12 |
| Diarrhea | Gastrointestinal disorders | 6 of 12 |
| Ear, nose and throat examination abnormal | General disorders | 6 of 12 |
| Nausea | Gastrointestinal disorders | 5 of 12 |
| Edema: head and neck | General disorders | 4 of 12 |
| Hypokalemia (Low potassium levels) | Investigations | 4 of 12 |
| Platelets decreased | Blood and lymphatic system disorders | 3 of 12 |
| Constipation | Gastrointestinal disorders | 3 of 12 |
| Gingival infection (Gum infection) | Infections and infestations | 3 of 12 |
Serious events with the highest arm-level rate: device related infection (8.3%), cerebrovascular ischemia (8.3%), adult respiratory distress syndrome (8.3%), dyspnea (shortness of breath) (8.3%), death (8.3%).
The registry lists 12 participants at risk for serious adverse events and 4 participants affected by serious adverse events in the Erlotinib and Temsirolimus group. It separately lists 12 participants at risk for other adverse events and 12 affected by other adverse events. These are registry counts for the stated categories; they should not be combined into a single count because the categories may overlap and refer to different event groupings.
The listed serious-event terms each have a reported frequency of 8.3%: device related infection, cerebrovascular ischemia, adult respiratory distress syndrome, dyspnea (shortness of breath), and death. The record does not provide event-level detail, timing, severity grades, or an assessment of whether individual events were caused by treatment. The listed term “Death” should not be read as an estimate of treatment-related mortality. The deaths field is not populated, which is not equivalent to a reported count of zero.
These figures describe the safety information recorded in the registry. With no concurrent control group, they do not establish whether the frequency of an event differs from another treatment or from background risk. The record does not report additional details needed to interpret exposure or the completeness of safety follow-up.
12. What the Result Does, and Does Not, Mean
The record documents a phase II investigation of temsirolimus plus erlotinib in advanced squamous cell carcinoma of the head and neck among people described as platinum-refractory or platinum-ineligible. Its stated primary question concerned progression-free survival compared with historical experience on erlotinib or cetuximab monotherapy. The registry also records the regimen, secondary outcomes, enrollment, study status, and selected serious adverse-event information.
What it does not establish is the observed primary outcome. The registry record does not report the median PFS, its full range, a confidence interval, or a sponsor-reported hazard ratio. Without these, the outcome’s magnitude and statistical precision cannot be described. The absence of a reported result is not evidence that the result was favorable, unfavorable, or null.
The single-group design also limits what a treatment comparison could establish. Historical outcomes can provide context, but they do not ensure that the study participants and historical patients were comparable. No concurrent randomized control group is listed. Consequently, the design cannot by itself isolate the contribution of the combination from differences in populations, care, or outcome assessment.
The listed serious adverse-event counts and terms are descriptive. The registry does not supply the causal assessments or comparative information needed to attribute events to the regimen or compare event rates with another treatment. Likewise, the terminated status does not explain why the study ended.
The appropriate reading is therefore bounded: the record describes the clinical hypothesis and study framework, but leaves the primary efficacy result, key analysis details, and reason for termination unreported. It can support an account of what the investigators set out to study, not a conclusion about whether the regimen changed PFS or how its outcomes compare with an alternative.
13. Why This Trial Matters Statistically
This trial illustrates why design and reporting details are essential to statistical interpretation. A single-group study can characterize outcomes observed among participants receiving a regimen, but it does not provide a concurrent randomized estimate of how those outcomes compare with another treatment. When the stated benchmark is historical, differences between the current and historical populations can affect the apparent contrast.
The primary endpoint is a time-to-event measure, so its interpretation depends on more than a label such as median PFS. The event definition, follow-up, censoring, number of events, and uncertainty around the estimate all matter. The registry specifies an endpoint definition and a 3 years timeframe, but does not report the endpoint result or the analysis information needed to assess its precision.
The record also distinguishes the study’s actual design from hypothetical planning calculations. An equal-allocation planning assumption does not turn a single-group trial into a two-arm comparison, and a sample-size illustration is not evidence about treatment effect or observed power. This distinction is broadly important when statistical planning material appears alongside results.
Finally, registry completeness affects what readers can responsibly conclude. Here the termination status and safety counts add context, but the reason for termination, primary efficacy estimate, and several analysis details are not reported in the registry record. Recognizing these gaps prevents a study rationale or an outcome label from being mistaken for clinical evidence.
14. Limitations
- The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
- Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.
The main limitation for interpreting this trial from the registry is that the primary PFS result is not reported. The registry supplies the outcome definition, summary measure, dispersion type, and timeframe, but not the median or full range. It also does not report a confidence interval or sponsor-reported hazard ratio. Thus neither the observed outcome nor its statistical precision can be evaluated from the record.
The single-group design has no concurrent control arm. The study summary invokes historical PFS with erlotinib or cetuximab monotherapy, but details of the historical populations and their comparability are not reported in the registry record. Differences in patient selection, care, follow-up, and assessment can complicate historical comparisons.
Enrollment is listed as 13, but the record does not state how many participants were treated, evaluable for each endpoint, or included in an analysis. It does not report participant disposition, censoring, missing-data procedures, or the analysis population. It also lists zero analyses, with no detailed statistical analysis plan or multiplicity approach reported.
The study is marked terminated, but the reason for termination is not reported in the registry record. The safety entries include counts and selected serious-event terms, but do not describe event timing, attribution, severity detail, or the completeness of safety follow-up. These omissions constrain interpretation and should not be replaced with assumptions about what occurred.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Temsirolimus (Torisel®) and Erlotinib (Tarceva®) in Platinum-Refractory/Ineligible, Advanced, Squamous Cell Carcinoma — NCT01009203.
- Registry record last updated 2015-08-10; status last verified 2015-07.
- Results first posted 2015-08-10.
- 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 describes a phase II, single-group study sponsored by the New Mexico Cancer Research Alliance, with enrollment of 13. It lists the study as terminated, with a start of 2009-12 and primary completion of 2012-09. The regimen combined erlotinib at 150 mg by mouth daily with temsirolimus at 15 mg intravenously weekly in 28-day cycles.
Median progression-free survival was the primary outcome, with a 3 years timeframe and full range specified as the dispersion. Toxicity profile, overall response rate, and overall survival were listed as secondary outcomes. The primary PFS result, reason for termination, and detailed analysis and missing-data procedures are not reported in the registry record. The record lists 4 participants affected by serious adverse events among 12 at risk and identifies several serious-event terms, each at 8.3%.