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 RAD001, Cisplatin and Concurrent Radiation for Locally Advanced, Inoperable Head and Neck Cancer (NCT01057277), a na phase 1 study. The primary outcome is Response.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01057277 |
| Phase | Phase 1 |
| Condition | Head and Neck Cancer |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 3 |
| Arms in the primary outcome | n/a |
| Lead sponsor | howard safran |
| Results posted | Yes |
2. Clinical Question
Population
Patients with locally advanced, inoperable squamous cell carcinoma of the head and neck. The condition is recorded as head and neck cancer, and the registry summary describes the intended population more specifically as patients with the squamous cell type. The enrollment recorded is 3.
Intervention
RAD001 (Afinitor) was given with cisplatin and concurrent radiation. The summary frames the phase I question around whether RAD001, up to a maximum dose of 10 mg/day, could be administered safely with the other treatments. The arm description places radiation across 47 days, cisplatin on specified treatment days, and RAD001 beginning on day 1 according to assigned group through day 47.
Comparator
No concurrent control group is recorded. The design is listed as single group, allocation is not applicable, and masking is listed as none. Consequently, this record does not describe a randomized comparison or a like-for-like control comparison.
Primary question
The primary outcome is titled “Response,” measured as the number of participants, with a timeframe of 1 year. The registry summary emphasizes safety of the combined regimen, while the outcome record does not define response or report a response count. The registry record therefore does not establish how response was assessed or what response result was observed.
Statistically, the study poses a feasibility and safety question about administering the named combination in the stated population, alongside a registered response outcome. A single-group study can describe what was recorded among participants receiving the regimen, but cannot by itself distinguish an intervention effect from the course that would have occurred without the regimen or under another treatment. The small enrollment recorded and absence of posted primary outcome results make the evidence especially limited for estimating response. The registry record does not report a control condition, a response definition, or a statistical comparison. Its stated question should therefore be read as a description of the study’s purpose, not as a conclusion about safety or response.
3. Trial Design
The registry classifies this as a phase I, single-group study with no masking. Allocation is listed as not applicable. Enrollment is recorded as 3, and the study status is terminated. The record gives a start date of 2009-12 and a primary completion date of 2011-05. It does not report why the study was terminated or whether the planned study activities were completed.
The intervention is named RAD001 (Afinitor). The brief summary describes administering RAD001, to a maximum dose of 10 mg/day, with cisplatin and concurrent radiotherapy for locally advanced, inoperable squamous cell carcinoma of the head and neck. The arm description specifies radiation for 47 days, cisplatin on days 1, 8, 15, 22, 29, 36, and 43, and RAD001 from day 1 through day 47 according to assigned group. Although the description refers to an assigned group, the study model is recorded as single group; further details explaining that wording are not reported in the registry record.
A phase I label and a stated safety purpose indicate the study’s broad clinical context, but the record does not report the dose-escalation method, dose cohorts, dose-limiting toxicity definition, or rules for moving between doses. It also does not report a formal sample-size justification, planned stopping rule, or interim analysis procedure. The stated enrollment should not be treated as evidence that a particular target was met, because the target and its justification are not reported.
Because there is no comparator arm, the study design is not suited to a randomized treatment contrast. The registry record contains one intervention arm and safety counts for that arm. These can describe recorded events in the enrolled group, but they do not provide a comparative estimate of the regimen’s effect or risk relative to another treatment. The record lists no statistical analyses.
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 | RAD001(Afinitor) |
|---|---|
| STARTED | 3 |
| COMPLETED | 3 |
| NOT COMPLETED | 0 |
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 | RAD001(Afinitor) |
|---|---|---|
| Participants analysed | 3 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 2 |
| Age, Categorical: >=65 years | Participants | 1 |
| Age, Continuous | years | 58.6 (49 to 71) |
| Sex: Female, Male: Female | Participants | 1 |
| Sex: Female, Male: Male | Participants | 2 |
| Region of Enrollment: United States | participants | 3 |
6. Primary Endpoint
The registry lists the primary outcome as Response, measured over 1 year.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | participants |
| Endpoint classification | Binary Endpoint |
The primary outcome is titled “Response.” Its measurement is listed as the number of participants, and its timeframe is 1 year. In principle, a participant-count endpoint records how many people meet a specified outcome definition during the stated assessment period. Such an outcome can offer a direct summary of activity in a group, but its meaning depends on a clear definition of response, an assessment method, and rules for handling participants whose response status cannot be determined.
The registry record does not define response, identify assessment criteria, state whether response was assessed by investigators or an independent reviewer, or describe how the one-year timeframe was applied. It does not report a response count or a corresponding analysis. Therefore, the endpoint title alone cannot establish whether response meant tumor reduction, disease control, or another criterion. No more specific interpretation is warranted from the registry record.
A response outcome may be included alongside a phase I safety question to describe clinical activity, but it should not be confused with a complete safety assessment or a comparative efficacy endpoint. This record’s stated purpose is to assess whether RAD001 can be administered with cisplatin and concurrent radiation; the registry outcome entry itself is response. Since the response result and its operational definition are not reported, the posted record does not allow readers to estimate the observed response frequency or its precision. It also does not support conclusions about how response compares with another regimen.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record describes a phase I, single-group study with enrollment recorded as 3 and status listed as terminated. It identifies a primary outcome called “Response,” measured as participants over a 1 year timeframe, but reports no primary response result. It also lists no analyses. These omissions mean the central outcome cannot be quantified or interpreted from the registry record.
For a binary participant outcome, a reported count would ordinarily need to be interpreted alongside the number assessed and the outcome definition. A confidence interval, if reported, would communicate the uncertainty around an estimated response proportion; with limited observations, such an interval would generally be wide. That is a general statistical point, not an interval calculated for this study. The registry record does not provide an observed response count, analysis population, or response definition from which to assess the estimate or its precision.
The single-group design matters for interpretation. Outcomes in a group receiving the regimen can be described, but without a control group they do not establish what would have happened under a different treatment or no treatment. The record does not support a causal comparison. The study is not listed as randomized; its allocation field is not applicable, and masking is listed as none.
The safety record lists 3 participants at risk for serious adverse events, with 2 affected. It also lists 3 at risk for other adverse events, with 3 affected. These counts describe the posted safety entries; the record does not explain the event-collection rules or whether one participant could contribute multiple event terms. Four serious-event terms are listed, each with a frequency of 33.3%: vomitting, nausea, colitis, and acute renal failure. The frequency entries should be read as registry-reported term frequencies, not as comparative risks or evidence about causation. Deaths are not reported in the registry record.
No statistical analysis is listed, and no analysis population, adjustment strategy, or confidence interval is reported. The record also does not report how missing outcome or safety information was handled. The safe interpretation is therefore descriptive and narrow: the registry documents a small enrollment, the stated study design and outcome, and safety counts, but not a primary response result or inferential analysis. Neither the response nor the safety entries establish how the regimen compares with alternatives.
9. Statistical Methods Explained
What does a single-group design estimate?
In a single-group study, participants receive the intervention under study, and observed outcomes are described within that group. A binary outcome such as response is often represented as a count of participants meeting the definition and a denominator of participants whose status is assessed. An estimated proportion can then summarize the observed frequency. That description is not automatically a treatment effect: without a concurrent comparison group, the result cannot separate the intervention’s contribution from other influences on the outcome.
For this study, the registry records a single-group model, no masking, and allocation as not applicable. It does not provide an observed response count or denominator for response assessment. Therefore, no response proportion can be interpreted from the registry record. The primary outcome label and timeframe alone do not provide the data needed for an estimate.
What would a confidence interval convey?
A confidence interval is a range produced by a statistical procedure to represent uncertainty around an estimate. For a binary outcome, it can show how imprecisely an observed proportion is estimated from the participants whose outcome status is known. It does not repair an unclear outcome definition, account automatically for missing outcomes, or turn a single-group observation into a controlled treatment comparison. The registry record does not report a response estimate or interval. Any explanation of interval interpretation is therefore general rather than a result for this study.
Why do definitions and denominators matter?
A count is interpretable only when it is clear what qualifies as an event and which participants are included in the denominator. For response, the record gives the outcome title, unit, and timeframe, but not the response criteria or assessment process. It does not state how many participants had response status assessed. For adverse events, the record gives numbers at risk and affected, but does not describe the observation window, ascertainment procedures, or whether counts refer to participants with at least one event versus event episodes. Those details are important because a participant-level count and an event-level count answer different questions.
What does the study status change?
The study is listed as terminated. This is a status description, not an explanation for termination and not a statistical result. The registry record does not say whether termination was related to safety, recruitment, operational issues, or another reason. It also does not say whether the recorded enrollment represents the full number treated or the number included in any outcome analysis. Such distinctions should not be inferred from status alone.
How should planning calculations be understood?
Planning sample sizes are prospective illustrations of how many participants might be needed under specified assumptions to detect a target effect with a chosen statistical procedure. They are not observed outcomes and do not establish the probability that a completed study found a real effect. In particular, a planning calculation based on an observed effect can be unstable when the observed estimate is imprecise, and a hypothetical two-arm calculation does not alter the actual single-group design of this study.
The stated planning assumptions describe a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and an effect defined as a fraction of the observed effect. The record also says that time-to-event planning uses a total event count and other planning outputs use participants per arm. These are planning conventions, not features of the observed trial design. The registry record lists no analyses and does not report a primary response result, so no observed response effect is available in the posted results for interpreting those assumptions as a trial finding. Planning illustrations must not be described as observed power.
Why can over-dispersion matter?
For count outcomes, a simple model may assume that the variability in counts follows a specified relationship to their average. Over-dispersion means the observed variability is greater than that assumption allows. If it is ignored, standard errors can be too small and uncertainty can appear narrower than warranted. This is a general issue in count-data analysis. The primary outcome is recorded as a number of participants with a binary response, not as a repeated event count, and the record does not report an over-dispersion assessment or a count-model analysis.
What can be inferred from the listed analyses?
The record reports zero analyses. It does not identify a statistical test, estimation method, adjustment for covariates, missing-data method, or confidence interval procedure. No unadjusted or adjusted treatment effect is reported. As a result, the analysis plan and the relationship between the stated primary outcome and any formal statistical conclusion are not available. A statistical method should not be imputed from the phase label, study purpose, or outcome type alone.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists one outcome measure and zero analyses. It does not report a multiplicity plan, such as how it would handle testing multiple outcomes, time points, or dose groups. No multiplicity adjustment or hierarchy of outcomes is described. Without a reported analysis plan, it is not possible to determine whether the response outcome was intended as the sole formal endpoint or how any additional analyses would have been interpreted.
Missing-data methods are not reported in the registry record. The record does not say how participants without an outcome assessment would be handled, whether response status was expected for all enrolled participants, or how incomplete adverse-event information was treated. The enrollment figure does not establish the number included in a response analysis. No analysis population—such as all enrolled participants, all treated participants, or participants with an evaluable response—is specified.
These omissions limit interpretation even when a count is available: the denominator and rules for incomplete follow-up are needed to understand what the count represents. In this case, the primary response count itself is not reported, so neither the handling of missing response information nor the impact of missingness can be assessed. The registry record also does not report whether missingness was related to participants’ outcomes or treatment experience. No assumptions about that relationship are justified.
Multiplicity and missing-data procedures are general components of a statistical plan, but their absence from the registry record should not be filled in by assumptions about common practice. The posted record provides no basis for concluding that a particular adjustment, imputation, or analysis-population rule was used.
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 |
|---|---|---|---|
| RAD001(Afinitor) | 2 | 3 | 66.7% |
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 |
|---|---|
| RAD001(Afinitor) | 3 of 3 |
Most frequent serious adverse event terms
| Event term | Organ system | RAD001(Afinitor) |
|---|---|---|
| vomitting | Investigations | 1 of 3 |
| nausea | Investigations | 1 of 3 |
| colitis | Investigations | 1 of 3 |
| acute renal failure | Investigations | 1 of 3 |
Most frequent non-serious adverse event terms
| Event term | Organ system | RAD001(Afinitor) |
|---|---|---|
| nausea | Investigations | 2 of 3 |
| dehydration | Investigations | 2 of 3 |
| wt loss | Investigations | 2 of 3 |
| fatigue | Investigations | 2 of 3 |
| mucositis | Investigations | 1 of 3 |
| obstructed trach- mucous | Investigations | 1 of 3 |
| vomitting | Investigations | 1 of 3 |
| diarrhea | Investigations | 1 of 3 |
| pain-mouth | Investigations | 1 of 3 |
| edema-face | Investigations | 1 of 3 |
Serious events with the highest arm-level rate: vomitting (33.3%), nausea (33.3%), colitis (33.3%), acute renal failure (33.3%).
The safety record lists 3 participants at risk for serious adverse events and 2 affected. For other adverse events, it lists 3 at risk and 3 affected. These figures are descriptive entries for the RAD001 (Afinitor) group. The registry record does not explain the safety observation period or the rules for counting affected participants.
The four serious adverse-event terms listed are vomitting, nausea, colitis, and acute renal failure, each with a registry-reported frequency of 33.3%. These term frequencies should not be added together or treated as mutually exclusive categories: the record does not state whether a participant could have more than one term. It also does not clarify whether the frequencies refer to participants, events, or another reporting convention beyond the displayed adverse-event entries.
Deaths are not reported in the registry record. The safety figures do not establish that the regimen caused an event, and they cannot show how the event frequency compares with another regimen because there is no control group. With the enrollment recorded as 3, the counts describe a very limited set of observations and do not provide a precise estimate of safety risk in a broader population. The record does not report event severity grading, timing, duration, outcome, or attribution to treatment.
12. What the Result Does, and Does Not, Mean
The registry record establishes that a phase I study was registered to examine RAD001 with cisplatin and concurrent radiation for locally advanced, inoperable squamous cell carcinoma of the head and neck. It records a single-group design, no masking, enrollment of 3, and terminated status. It also records a primary response outcome with a 1 year timeframe and limited adverse-event counts for the intervention group.
It does not report the primary response count, define response, or list an analysis. Therefore, the posted results do not permit estimation of the observed response frequency, its uncertainty, or any response comparison. The record cannot support a conclusion that the regimen produced a particular response outcome. The stated study purpose concerns safe administration, but the available safety entries alone do not establish overall safety or identify the cause of any event.
The single-group design also limits what can be learned about treatment effects. A result observed after treatment would not show how outcomes would differ under another treatment, because no control group is recorded. The registry record does not provide a randomized comparison, and no causal contrast should be inferred from the safety or outcome entries.
The listed adverse-event figures can be read as descriptive counts and term frequencies in the registry. They should not be treated as precise population risks or compared with external values that are not part of the record. The study’s terminated status does not explain why it stopped, and it does not itself establish whether the treatment was safe, unsafe, effective, or ineffective. Such explanations are not reported in the registry record.
In short, the record documents the trial’s intended clinical question, design, enrollment, status, and some safety reporting. It does not provide the primary response result or analysis needed to answer the registered outcome question. That distinction—between a study’s purpose and its reported evidence—is essential when interpreting this entry.
13. Why This Trial Matters Statistically
This trial illustrates why a registry record should be read as a report of design and posted results, not as a guarantee that every planned outcome has been reported. The study’s primary outcome is identified as response, but no response result or analysis is listed. Knowing the endpoint label and timeframe is not enough to reconstruct the outcome or judge its precision.
It also demonstrates the limits of small, single-group safety descriptions. The enrollment recorded is 3, and adverse-event counts are reported for the sole intervention group. Such counts can document what the registry reports, but they do not create a comparison group or establish a treatment-related risk. A small set of observations also leaves substantial uncertainty about how well the recorded experience represents a wider population. That is a general statistical limitation, not a calculated interval for this study.
The difference between an endpoint and an analysis is important as well. A registered response outcome does not reveal its operational definition, analysis population, missing-data rules, or inferential method. The record lists no analyses, so those details cannot be evaluated. Similarly, planning assumptions for hypothetical sample-size calculations are not evidence that the actual trial had a two-arm design, nor do they represent observed power.
Finally, “terminated” is a status, not an explanation. The record does not report why the study ended or whether the termination reflected safety, feasibility, or another circumstance. Keeping those unknowns explicit prevents readers from turning incomplete registry information into a clinical conclusion. Statistically, the most defensible interpretation is narrow: the entry documents a phase I single-group study and some safety counts, while leaving the primary response result and important analysis details 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 primary outcome is not sufficiently specified for independent interpretation. Although it is titled “Response” and has a 1 year timeframe, response criteria, assessment methods, and the analysis denominator are not reported. No response count or estimate is included. This prevents assessment of both the outcome itself and its precision.
Enrollment is recorded as 3, but the registry does not state how many participants received each component, completed treatment, or had outcome assessment. It does not report the sample-size target or its justification. The phase I label does not supply missing details about dose escalation, cohort structure, safety rules, or dose-limiting toxicity definitions. The arm description’s reference to an assigned group is not explained alongside the single-group model.
The record lists the trial as terminated but does not report the reason. It does not provide a statistical analysis plan, analysis population, method for missing data, confidence intervals, or adjustment strategy. It is therefore unclear how the registered response outcome would have been analyzed and whether the posted safety counts reflect all participants or a particular subset.
The safety entries lack reported detail on event timing, duration, severity grading, attribution, and whether terms can overlap. The death field is not reported. With no control group, adverse-event counts cannot be interpreted as comparative risks. These limitations are specific to the information and design recorded; they do not establish why results or details are absent, or what occurred beyond what the registry reports.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: RAD001, Cisplatin and Concurrent Radiation for Locally Advanced, Inoperable Head and Neck Cancer — NCT01057277.
- Registry record last updated 2020-02-17; status last verified 2020-02.
- Results first posted 2015-08-04.
- 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
NCT01057277 is recorded as a phase I, single-group study of RAD001 (Afinitor) with cisplatin and concurrent radiation for locally advanced, inoperable head and neck cancer. The study is listed as terminated, with enrollment of 3, no masking, and allocation not applicable. Its stated purpose concerns administering RAD001, up to a maximum dose of 10 mg/day, with cisplatin and radiation.
The primary outcome is “Response,” measured as participants over 1 year. The registry record does not define response or report a response result. It lists no analyses. Safety entries record serious and other adverse-event counts, and list four serious-event terms, each with a frequency of 33.3%. The reason for termination, missing-data handling, analysis population, and statistical plan are not reported in the registry record.