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 + Weekly Paclitaxel + Carboplatin for Recurrent or Metastatic Head and Neck Squamous Cell Cancer (HNSCC) (NCT01016769), a na phase 1, phase 2 study. The primary outcome is Phase II Recommended Dose for the Combination of Temsirolimus + Weekly Paclitaxel + Carboplatin..
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01016769 |
| Phase | Phase 1, Phase 2 |
| Condition | Squamous Cell Cancer, Head and Neck Cancer |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 48 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Memorial Sloan Kettering Cancer Center |
| Results posted | Yes |
2. Clinical Question
Population
People with recurrent or metastatic head and neck squamous cell cancer are the intended population described by the study title. The listed conditions are squamous cell cancer and head and neck cancer. Further eligibility details, such as specific inclusion or exclusion criteria, are not reported in the registry record.
Intervention
The study evaluates temsirolimus with weekly paclitaxel and carboplatin. The primary endpoint is a dose-finding question: establishing the phase II recommended dose for the combination. The registry describes Phase I dose escalation followed by a Phase II portion. It reports that the phase II recommended temsirolimus dose was established at dose level three, alongside Carboplatin AUC 1.5 and Taxol 80 mg/m2.
Comparator
There is no separate control group in the registry design. The study is listed as single-group, with allocation recorded as NA and masking as none. Dose-level groups are part of the escalation and treatment structure; they should not be treated as randomized, like-for-like comparisons. The record does not support a causal comparison of the combination against another regimen.
Primary question
The primary question is which dose of the combination should be recommended for the Phase II portion, rather than whether the regimen improves an efficacy outcome relative to a control. The primary endpoint is titled “Phase II Recommended Dose for the Combination of Temsirolimus + Weekly Paclitaxel + Carboplatin.” Secondary outcomes include adverse events, median overall survival, and potential molecular markers of resistance to mTOR inhibition. Their results are not reported in the registry record.
3. Trial Design
The registry identifies NCT01016769 as a completed Phase I/II study sponsored by Memorial Sloan Kettering Cancer Center. The recorded start is 2009-11 and primary completion is 2018-06. The study concerns temsirolimus combined with weekly paclitaxel and carboplatin for recurrent or metastatic head and neck squamous cell cancer. Enrollment is listed as 48.
The design is single-group, with allocation recorded as NA and masking recorded as none. The record therefore does not describe random assignment to the regimen and a separate control treatment. Its dose-level groups reflect the dose-finding and treatment portions, not randomized comparison arms. Results between dose levels should not be interpreted as controlled treatment effects.
The Phase I description specifies a standard 3 + 3 dose-escalation design and says up to 3 dose levels are planned. Its stated primary purpose is establishing the recommended dose for Phase II. The record reports that dose level three was established as the phase II recommended dose. The Phase I description also states that 6 participants treated at Phase I dose level 3 and 30 participants treated in the Phase II portion were combined for analysis of the primary result.
The Phase II portion uses the selected dose level, but details of its statistical design, target enrollment by portion, and efficacy analysis are not reported in the registry record. The registry lists five outcome measures. It names adverse events, median overall survival, and potential molecular markers of resistance to mTOR inhibition as secondary outcomes; results for these outcomes 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 | Phase 1, Dose Level 1 | Phase 1, Dose Level 2 | Phase 1, Dose Level 3 | Phase 2, Dose Level 3 |
|---|---|---|---|---|
| STARTED | 4 | 7 | 7 | 30 |
| COMPLETED | 3 | 6 | 6 | 30 |
| NOT COMPLETED | 1 | 1 | 1 | 0 |
Analysis population for the primary outcome, as defined in the registry: The phase II recommended dose of Temsirolimus was established at dose level three (Carboplatin AUC 1.5, Taxol 80 mg/m2, Temsirolimus 25 mg). 6 participants treated on Phase I, dose level 3 and 30 participants treated on the Phase II portion were combined for analysis.
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 | Phase 1, Dose Level 1 | Phase 1, Dose Level 2 | Phase 1, Dose Level 3 | Phase 2, Dose Level 3 | Total |
|---|---|---|---|---|---|---|
| Participants analysed | 4 | 7 | 7 | 30 | 48 | |
| Age, Categorical: <=18 years | Participants | 0 | 0 | 0 | 0 | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 3 | 5 | 6 | 20 | 34 |
| Age, Categorical: >=65 years | Participants | 1 | 2 | 1 | 10 | 14 |
| Sex: Female, Male: Female | Participants | 0 | 3 | 1 | 8 | 12 |
| Sex: Female, Male: Male | Participants | 4 | 4 | 6 | 22 | 36 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 0 | 0 | 1 | 1 | 2 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 | 2 | 0 | 3 | 5 |
| Race (NIH/OMB): White | Participants | 4 | 5 | 6 | 26 | 41 |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 | 0 | 0 |
| Region of Enrollment: United States | Participants | 4 | 7 | 7 | 30 | 48 |
6. Primary Endpoint
The registry lists the primary outcome as Phase II Recommended Dose for the Combination of Temsirolimus + Weekly Paclitaxel + Carboplatin., measured over 2 years.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | mg |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is the phase II recommended dose for temsirolimus with weekly paclitaxel and carboplatin. It measures a dose-selection decision, not tumor response, survival benefit, or a comparison with another treatment. In an early-phase combination study, dose finding is used to identify a regimen dose considered suitable for evaluation in a later treatment portion, while monitoring tolerability. The registry describes the Phase I portion as a 3 + 3 dose-escalation design.
The record states that the phase II recommended dose was established at dose level three: Carboplatin AUC 1.5, Taxol 80 mg/m2, and Temsirolimus 25 mg. For the primary analysis, 6 participants treated on Phase I at dose level 3 and 30 participants treated on the Phase II portion were combined. Those groups therefore contribute to the reported dose-selection result, but the registry does not provide a numerical uncertainty estimate around that decision.
The endpoint is useful for describing the regimen carried forward within this study. It does not, by itself, establish comparative efficacy or quantify the balance of benefit and harm. Details of the decision criteria used to select the dose, including any formal toxicity threshold, are not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central reported result is a dose-selection finding: the registry states that dose level three was established as the phase II recommended dose. The listed regimen at that level is Carboplatin AUC 1.5, Taxol 80 mg/m2, and Temsirolimus 25 mg. This answers the primary endpoint as described in the record. It is not an estimate of treatment effect against a control, because the study is listed as single-group and has no separate comparator.
The primary endpoint description says that 6 participants treated on Phase I at dose level 3 and 30 participants treated in the Phase II portion were combined for analysis. That pooled description is relevant when reading the dose-selection result: it is not a result exclusively from the Phase I dose-escalation participants. The registry does not report a confidence interval or another measure of statistical uncertainty for the dose recommendation, nor does it describe the decision rule that led to selection.
The safety summaries are organized by Phase I dose level and Phase II dose level. For Phase 1, Dose Level 1, the registry reports 4 at risk for serious adverse events and 2 affected; for Phase 1, Dose Level 2, 7 at risk and 4 affected; for Phase 1, Dose Level 3, 7 at risk and 5 affected; and for Phase 2, Dose Level 3, 30 at risk and 21 affected. The corresponding counts listed for other adverse events are 4 and 4, 7 and 7, 7 and 7, and 30 and 30, respectively. These are descriptive group counts. They do not establish that the regimen caused an event or show how the event risk compares with an alternative treatment.
Deaths are also listed by group: 4 for Phase 1, Dose Level 1; 6 for Phase 1, Dose Level 2; 7 for Phase 1, Dose Level 3; and 28 for Phase 2, Dose Level 3. The registry does not establish from these counts whether deaths were treatment-related, and the counts should not be read as a comparative mortality analysis.
The secondary outcomes include median overall survival and potential molecular markers of resistance to mTOR inhibition, but their results are not reported in the registry record. Accordingly, the posted information supports a description of the selected dose and recorded safety counts, not a conclusion about comparative effectiveness, survival, or a treatment effect.
9. Statistical Methods Explained
Several statistical ideas help distinguish the questions this study can address. The registry describes a Phase I/II study, but its main endpoint is a dose recommendation. It is therefore important to separate dose selection from efficacy estimation. A dose-finding result identifies the regimen dose selected for the Phase II portion according to the study’s decision process. It does not estimate what would have happened to the same participants under a different treatment.
What does a single-group design imply?
In a single-group study, outcomes are observed among people receiving the study regimen, without a randomized control group in the listed design. This can describe what happened in the treated groups, including adverse events, and can inform dose selection. It cannot remove the possibility that observed outcomes reflect participant characteristics, the underlying disease, other care, or other influences. Since allocation is recorded as NA and masking as none, the record does not support a randomized, blinded comparison. The dose-level groups are not interchangeable comparator arms: they arise from the dose-escalation structure and differ by dose and phase.
For that reason, comparing the counts across dose levels does not by itself identify a dose-related causal effect. Participants were not assigned to dose levels as part of a reported randomized comparison. The groups may also differ in their place in the study and in the amount of follow-up available. Details of follow-up duration by group and methods for handling it are not reported in the registry record.
How should a 3 + 3 design be understood?
As a general statistical explanation, a 3 + 3 dose-escalation design uses observed tolerability at a dose level to guide whether escalation, continuation, or stopping is appropriate under its protocol rules. The registry identifies this design for Phase I and says up to 3 dose levels were planned. However, the protocol’s specific dose-limiting toxicity definition, observation window, escalation rules, and dose-selection threshold are not reported in the registry record. Those details matter because they determine how observed events translate into a recommendation.
The listed primary result states that dose level three was selected, and describes the regimen as Carboplatin AUC 1.5, Taxol 80 mg/m2, and Temsirolimus 25 mg. The record does not report a formal probability of toxicity at that dose or an interval expressing uncertainty. A dose recommendation is consequently best read as the study’s stated selection, not as a precise estimate that the selected dose is optimal for every patient or setting.
What does statistical uncertainty mean?
As a general concept, a confidence interval describes a range of values compatible with the data under a specified statistical model. It communicates precision as well as the direction or magnitude of an estimate. The registry does not report a confidence interval for the primary dose recommendation. Nor does it give a numerical efficacy estimate with an interval in the listed information. This limits how precisely the result can be characterized beyond the stated dose decision.
For an outcome such as survival, methods must account for the timing of events and differences in follow-up. A median survival estimate is generally a summary of the time-to-event distribution, and its interpretation depends on the analysis population, censoring, and follow-up. Median overall survival is listed as a secondary outcome, but its result and analysis details are not reported in the registry record. No survival estimate should be inferred from the death counts alone.
How do the safety counts relate to statistical evidence?
Counts of participants affected by events are useful descriptive information. They are not equivalent to a treatment-versus-control effect estimate. A comparison of event counts needs clearly defined populations, event definitions, observation periods, and an appropriate method. Although serious and other adverse-event counts are listed across dose-level groups, the registry does not report a comparative statistical analysis for those groups. The listed percentages for serious adverse-event terms also lack a stated denominator in the record, so their precise basis cannot be established from the posted information.
As a general matter, event-count data can be sparse and may vary substantially by chance. A confidence interval can show the uncertainty around an event rate or contrast, while a model can account for relevant design features when justified. Neither an interval nor a modeled comparison should be presumed in the absence of reported methods and results. The adverse-event counts should remain descriptive.
What do planning illustrations represent?
As a general concept, a sample-size planning calculation asks how many participants or events might be needed to detect a specified effect under stated assumptions. Such a calculation is not an observed result and is not “observed power.” The registry’s stated illustration assumptions are a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and an effect to detect defined as a fraction of the observed effect. Time-to-event illustrations are described as total events, while other illustrations are described as participants per arm.
Those assumptions do not describe the actual single-group dose-finding design, which has no randomized two-arm allocation in the registry record. A planning illustration based on an observed effect is not a substitute for a trial-specific design justification. The registry does not report a sample-size justification or the analysis plan for the dose-finding decision. It also does not report an observed effect estimate in the information describing the primary endpoint. Any planning quantities should therefore be interpreted only under their stated assumptions, not as evidence about the precision or power of this study.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists five outcome measures, with the primary endpoint focused on establishing the Phase II recommended dose. Named secondary outcomes include the number of participants who experienced adverse events, median overall survival, and the number of participants with potential molecular markers of resistance to mTOR inhibition. The results for those secondary outcomes are not reported in the registry record.
When a study examines multiple outcomes, statistical multiplicity can arise because repeated testing increases the chance of at least one apparently notable result under a collection of null hypotheses. As a general statistical principle, interpretation depends on whether outcomes were prespecified, which analyses were planned, and whether any adjustment or hierarchy was used. The registry does not report a multiplicity strategy, a detailed statistical analysis plan, or results for the listed secondary outcomes. It is therefore not possible to determine how multiple outcomes were handled.
Missing-data methods and analysis populations are also not reported in the registry record. The primary outcome description identifies participants from the Phase I dose level 3 group and the Phase II portion as combined for analysis, but it does not specify a broader analysis-population definition, eligibility for inclusion, or how missing observations were handled. The adverse-event summary lists group counts, but the record does not explain whether all participants had complete event follow-up or how incomplete follow-up was treated.
These omissions limit assessment of potential selection effects and the robustness of outcome estimates. No missing-data method, imputation approach, interim analysis, or adjustment procedure should be assumed from the posted information.
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 |
|---|---|---|---|
| Phase 1, Dose Level 1 | 2 | 4 | 50.0% |
| Phase 1, Dose Level 2 | 4 | 7 | 57.1% |
| Phase 1, Dose Level 3 | 5 | 7 | 71.4% |
| Phase 2, Dose Level 3 | 21 | 30 | 70.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Phase 1, Dose Level 1 | 4 of 4 |
| Phase 1, Dose Level 2 | 6 of 7 |
| Phase 1, Dose Level 3 | 7 of 7 |
| Phase 2, Dose Level 3 | 28 of 30 |
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 |
|---|---|
| Phase 1, Dose Level 1 | 4 of 4 |
| Phase 1, Dose Level 2 | 7 of 7 |
| Phase 1, Dose Level 3 | 7 of 7 |
| Phase 2, Dose Level 3 | 30 of 30 |
Most frequent serious adverse event terms
| Event term | Organ system | Phase 1, Dose Level 1 | Phase 1, Dose Level 2 | Phase 1, Dose Level 3 | Phase 2, Dose Level 3 |
|---|---|---|---|---|---|
| Dyspnea (shortness of breath) | Respiratory, thoracic and mediastinal disorders | 0 of 4 | 0 of 7 | 2 of 7 | 3 of 30 |
| Hemoglobin | Investigations | 1 of 4 | 1 of 7 | 0 of 7 | 3 of 30 |
| Thrombosis/thrombus/embolism | Vascular disorders | 0 of 4 | 1 of 7 | 0 of 7 | 4 of 30 |
| Febrile neutropenia | Blood and lymphatic system disorders | 0 of 4 | 1 of 7 | 0 of 7 | 3 of 30 |
| Fever (in the absence of neutropenia) | General disorders | 0 of 4 | 0 of 7 | 0 of 7 | 4 of 30 |
| Vomiting | Gastrointestinal disorders | 0 of 4 | 1 of 7 | 2 of 7 | 1 of 30 |
| Cough | Respiratory, thoracic and mediastinal disorders | 0 of 4 | 0 of 7 | 1 of 7 | 2 of 30 |
| Diarrhea | Gastrointestinal disorders | 0 of 4 | 1 of 7 | 0 of 7 | 2 of 30 |
| Dysphagia (Difficulty swallowing) | Gastrointestinal disorders | 0 of 4 | 0 of 7 | 2 of 7 | 1 of 30 |
| Fatigue (asthenia, lethargy, malaise) | General disorders | 0 of 4 | 0 of 7 | 0 of 7 | 3 of 30 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Phase 1, Dose Level 1 | Phase 1, Dose Level 2 | Phase 1, Dose Level 3 | Phase 2, Dose Level 3 |
|---|---|---|---|---|---|
| Hemoglobin | Blood and lymphatic system disorders | 4 of 4 | 7 of 7 | 6 of 7 | 30 of 30 |
| Fatigue (asthenia, lethargy, malaise) | General disorders | 2 of 4 | 6 of 7 | 6 of 7 | 27 of 30 |
| Glucose, high (hyperglycemia) | Metabolism and nutrition disorders | 4 of 4 | 5 of 7 | 7 of 7 | 25 of 30 |
| Leukocytes (total WBC) | Investigations | 4 of 4 | 5 of 7 | 6 of 7 | 26 of 30 |
| Albumin, low (hypoalbuminemia) | Metabolism and nutrition disorders | 3 of 4 | 3 of 7 | 7 of 7 | 22 of 30 |
| Platelets | Investigations | 3 of 4 | 4 of 7 | 5 of 7 | 23 of 30 |
| Cholesterol,high(hypercholestremia) | Investigations | 2 of 4 | 3 of 7 | 4 of 7 | 23 of 30 |
| Lymphopenia | Investigations | 3 of 4 | 6 of 7 | 4 of 7 | 17 of 30 |
| Neuropathy: sensory | Nervous system disorders | 3 of 4 | 4 of 7 | 4 of 7 | 19 of 30 |
| Dysphagia (Difficulty swallowing) | Gastrointestinal disorders | 2 of 4 | 5 of 7 | 5 of 7 | 18 of 30 |
Serious events with the highest arm-level rate: dysphagia (difficulty swallowing) (28.6%), dyspnea (shortness of breath) (28.6%), vomiting (28.6%), atrial fibrillation (25.0%), calcium, low (hypocalcemia) (25.0%).
The registry reports serious adverse-event counts by dose-level group. The at-risk and affected counts are 4 and 2 for Phase 1, Dose Level 1; 7 and 4 for Phase 1, Dose Level 2; 7 and 5 for Phase 1, Dose Level 3; and 30 and 21 for Phase 2, Dose Level 3. For other adverse events, the corresponding at-risk and affected counts are 4 and 4, 7 and 7, 7 and 7, and 30 and 30. The registry also lists deaths by group: 4, 6, 7, and 28 in the same group order.
These are descriptive counts, not comparative estimates. The dose-level groups were not randomized control groups, and differences in counts cannot establish that a dose caused more or fewer events. The record does not specify a comparative safety analysis or establish whether listed deaths were treatment-related. The at-risk counts should be read as the group counts recorded for these summaries, not as evidence that follow-up was identical.
The registry lists serious-event terms with percentages, including Dysphagia (Difficulty swallowing), Dyspnea (shortness of breath), and Vomiting at 28.6% each, and Atrial fibrillation, Calcium, low (hypocalcemia), and Fistula, pulm/upp respir - Trachea at 25.0% each. The denominator and calculation basis for those percentages are not reported in the registry record. These term percentages should therefore be interpreted cautiously and are not estimates of comparative risk.
12. What the Result Does, and Does Not, Mean
The result means that the registry identifies dose level three as the phase II recommended dose for the combination. The listed regimen is Carboplatin AUC 1.5, Taxol 80 mg/m2, and Temsirolimus 25 mg. This is a dose-selection finding within the study’s Phase I/II structure. The primary endpoint description says that 6 participants treated at Phase I dose level 3 and 30 participants treated in Phase II were combined for analysis.
It does not mean that the regimen has been shown to improve outcomes compared with another regimen. The design is single-group, with allocation recorded as NA, and no separate control treatment is listed. The record also does not provide the results of the secondary outcomes for median overall survival or molecular markers of resistance to mTOR inhibition. The listed death counts do not substitute for a survival analysis and do not establish treatment attribution.
The adverse-event summaries show that events were recorded in the listed groups. They can help describe what the registry reports about safety during the study, but they are not a randomized comparison and do not establish that the treatment caused each event. The record does not provide a formal comparative safety estimate, a confidence interval for the primary endpoint, or a detailed account of how the dose-selection criteria were applied.
In general, early-phase dose selection is a step in evaluating a regimen, not proof of efficacy or a guarantee of tolerability in all settings. A dose recommendation applies to the decision described in this study; its interpretation depends on the population, protocol rules, observation period, and completeness of follow-up. Several of those details are not reported in the registry record. The posted information should therefore be read as a selected dose and descriptive event reporting, rather than as a controlled assessment of benefit or harm.
13. Why This Trial Matters Statistically
This study illustrates an important distinction in clinical-trial statistics: a dose-finding endpoint and a treatment-effect endpoint answer different questions. The primary endpoint is the phase II recommended dose for a combination regimen. Its result identifies the dose selected for further evaluation within the study, while it does not quantify an advantage over another treatment. Interpreting the endpoint according to its purpose helps avoid treating a dose recommendation as evidence of efficacy.
The design also highlights why group labels matter. Phase I dose levels and the Phase II group are not randomized comparator arms. They arise from different stages of the study and have different dose-level roles. Descriptive counts across those groups can be informative, but they do not form a controlled dose-response comparison. The registry’s report that Phase I dose level 3 and the Phase II portion were combined for the primary analysis is particularly relevant to understanding what participants contributed to the stated result.
Safety reporting is another useful statistical lesson. Counts of participants affected by serious and other adverse events, alongside deaths, communicate observed events in the study groups. Without a comparator and detailed event-analysis methods, those counts cannot establish comparative risk or treatment causation. The term-specific serious-event percentages also need a stated denominator for their magnitude to be fully interpretable.
Finally, the record separates what is named from what is reported: survival and molecular-marker outcomes are listed, but their results are not reported in the registry record. Recognizing that boundary is essential when assessing what conclusions the study can support. The record offers a dose-selection result and descriptive safety summaries, while leaving important analysis details and secondary results unavailable.
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 registry record does not describe the detailed eligibility criteria, the dose-limiting toxicity definition, the period used to assess dose-limiting toxicity, or the protocol rules for dose escalation and selection. Those details are important for evaluating how the stated 3 + 3 design led to the recommended dose. The record also does not report a statistical analysis plan, sample-size justification, analysis populations, missing-data procedures, or any interim-analysis plan.
The study is single-group and has no randomized comparator in the listed design. Accordingly, event counts across dose levels do not provide a controlled comparison. Changes in group counts cannot be interpreted as treatment effects, and the registry does not report an adjusted comparison or confidence interval for the dose recommendation.
The primary endpoint description states that the Phase I dose level 3 and Phase II participants were combined for analysis, but the record does not explain the analysis method or report a measure of uncertainty. Results for median overall survival and potential molecular markers of resistance to mTOR inhibition are not reported in the registry record. The serious-event term percentages are listed without a reported denominator or calculation basis. These gaps restrict independent assessment of precision, completeness, and the scope of conclusions supported by the posted information.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Temsirolimus + Weekly Paclitaxel + Carboplatin for Recurrent or Metastatic Head and Neck Squamous Cell Cancer (HNSCC) — NCT01016769.
- Registry record last updated 2019-06-26; status last verified 2018-06.
- Results first posted 2019-06-26.
- 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
NCT01016769 is a completed Phase I/II, single-group study sponsored by Memorial Sloan Kettering Cancer Center. It evaluates temsirolimus with weekly paclitaxel and carboplatin for recurrent or metastatic head and neck squamous cell cancer. The registry records enrollment of 48, a start date of 2009-11, and primary completion in 2018-06.
The primary endpoint is establishing the phase II recommended dose. The record states that dose level three was selected, with Carboplatin AUC 1.5, Taxol 80 mg/m2, and Temsirolimus 25 mg. The primary analysis description combines 6 Phase I participants at dose level 3 with 30 participants in the Phase II portion. Safety counts are reported by dose-level group. Results for the listed survival and molecular-marker secondary outcomes, and detailed statistical methods for the primary decision, are not reported in the registry record.