Trial of RAD001 in Triple Negative Metastatic Breast Cancer: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Trial of RAD001 in Triple Negative Metastatic Breast Cancer (NCT00827567), a na phase 2 study. The primary outcome is Time to Progression(TTP).

2. Clinical Question

The research question was whether RAD001 could shrink or slow the growth of the specified metastatic breast cancer, alongside study of its safety. The registry summary says that physical state, symptoms, tumor-size changes, and laboratory findings obtained during the study would inform the research team’s assessment. These statements describe the study’s aims, not evidence that tumor shrinkage or slowed growth occurred.

The record lists enrollment of 6 and identifies the study as phase II. It does not report an outcome estimate for TTP, a count of patients who progressed by the stated assessment, or a comparison with another treatment or control. Therefore, the registry record does not establish what happened to progression in the enrolled group. Details of the eligibility criteria beyond the disease description, and the rationale for selecting RAD001, are not reported in the registry record.

3. Trial Design

This was a phase II, single-group study sponsored by Milton S. Hershey Medical Center. The registry lists enrollment of 6, allocation as not applicable, and masking as none. The listed intervention was RAD001, described as 10 mg by mouth once every day. The registry record does not identify a concurrent control group, so the design does not support a like-for-like comparison between treatment arms.

The study start is listed as 2009-04 and primary completion as 2011-02. Its status is terminated. The reason for termination, the timing of the decision, and whether the planned enrollment or follow-up was completed are not reported in the registry record. The status alone does not establish why the study ended or what results were observed.

The primary outcome is named time to progression (TTP), with each patient assessed at 8 weeks from the start of study drug. The registry defines progression as a new lesion or a 20% increase over baseline in the sum of target lesions. It does not report the assessment schedule beyond this stated primary timeframe, nor does it explain how an event time was assigned between assessments.

The record does not report the protocol’s statistical analysis plan, sample-size justification, randomization procedures, interim analyses, or planned handling of incomplete observations. No analysis results are listed. These absences mean the design can be described from the registry’s basic entries, but the operational details needed to reproduce or fully evaluate the study cannot be established from this record alone.

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.

Before assignment: Two of the patients that were screen failures had cholesterol levels higher than inclusion criteria as this drug may increase cholesterol levels they were not eligible.

Overall Study

Reasons for not completing this period:

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.

6. Primary Endpoint

The registry lists the primary outcome as Time to Progression(TTP), measured over Each patient assessed at 8 weeks from start of study drug.

The primary endpoint is time to progression (TTP). In general, a time-to-event endpoint measures the time from a defined starting point until a specified event occurs. Here, the registry identifies the start as the beginning of study drug and describes assessment at 8 weeks. Its stated progression criteria are a new lesion or a 20% increase over baseline in the sum of target lesions.

TTP is used in clinical research to describe how long participants remain without the defined progression event. Unlike a simple tumor-size measurement at one moment, a time-to-event outcome can represent both whether and when progression is observed. General statistical analyses of such outcomes may account for participants whose event has not been observed by the end of follow-up, but the registry record does not report whether or how that was done in this study.

The posted results do not include a TTP estimate, event count, distribution of follow-up, or uncertainty interval. The record also does not explain the handling of death, missed assessments, or progression occurring between scheduled assessments. Consequently, the endpoint definition is available, but the observed time-to-progression experience cannot be interpreted from the registry record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What does a time-to-event outcome represent?

In general, a time-to-event outcome combines information about whether a defined event occurred with information about when it occurred. For TTP, the event is progression under the study’s stated definition. A time-to-event analysis can use observations from participants with different follow-up durations, rather than treating every participant as if they had the same observation period.

Such analyses often distinguish an observed event from a participant whose event was not observed during available follow-up. The latter observation may be censored: the exact event time is unknown, although the participant was known to be event-free up to a point. This approach relies on clear definitions of the time origin, event, censoring, and follow-up. The registry identifies the start of study drug and gives an 8-week assessment timeframe, but does not report rules for censoring or for assigning event times between assessments. It also does not report how death was handled in relation to TTP.

How should the progression definition be read?

The registry defines progression as any new lesion or the sum of target lesions increasing by 20% over baseline. This makes the endpoint dependent on both lesion identification and measurement. In general, measurement rules matter because changes in imaging, evaluability, or assessment timing can affect whether and when an event is recorded. The registry calls the criteria “RESIST”; whether additional criteria or confirmation procedures were used is not reported in the registry record.

The stated assessment at 8 weeks gives a planned timeframe, not an observed result. The record does not report whether all participants had an assessment at that time, how unevaluable scans were handled, or whether additional assessments contributed to the endpoint. Without those details and the actual endpoint data, a reader cannot reconstruct an event-time analysis.

What do estimates and confidence intervals usually communicate?

General statistical interpretation separates the estimated effect from its precision. An estimate is a summary derived from observed data; a confidence interval describes a range of values compatible with the data and the method’s assumptions. A narrow interval generally indicates greater precision than a wide interval, while neither a point estimate nor an interval alone establishes causation. The registry record reports no TTP estimate or confidence interval, so there is no posted precision measure to interpret for the primary endpoint.

A p-value, when reported, measures how compatible data are with a specified null model under the analysis assumptions. It is not the probability that the null hypothesis is true and does not convey the size or clinical importance of an effect. No p-value or primary endpoint test is reported in the registry record. The specific inferential method, model assumptions, and any planned threshold for statistical significance are also not reported.

What is over-dispersion, and does it apply to these results?

General explanation: over-dispersion occurs when count data vary more than a statistical model expects. If ignored, it can lead to uncertainty estimates that are too small and tests that appear more conclusive than warranted. Methods that allow extra variation may be appropriate for some count outcomes. The primary endpoint in this record is TTP, not a reported count analysis, and there are no endpoint results with which to assess over-dispersion. The registry record does not report a model or an over-dispersion assessment.

How should planning illustrations be distinguished from trial findings?

General explanation: sample-size planning asks how many participants or events might be needed under specified assumptions to detect a chosen effect with a chosen operating characteristic. Such calculations depend on assumptions about the effect, variability, allocation, and analysis method. They are not measurements of what happened in a completed study and are not observed power.

Any planning illustrations associated with this record are described as calculations from an observed effect, using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; the effect to detect is stated as a fraction of the observed effect. The registry record does not report that these were the study’s own design assumptions. Because this trial is described as single-group, a two-arm planning illustration should not be mistaken for the actual trial design. No calculated planning values are reported in the registry record, and no such illustration can substitute for a reported endpoint analysis.

10. Multiplicity, Missing Data and Analysis Populations

Multiplicity concerns the interpretation of several statistical tests or outcomes considered together. When many hypotheses are tested, the chance of finding at least one apparently notable result can increase. General approaches include defining a primary hypothesis in advance, ordering tests, or adjusting the threshold for multiple comparisons. The registry identifies TTP as the primary outcome, but it does not report a statistical analysis plan, a testing hierarchy, or any multiplicity procedure. It is therefore not possible to determine how multiple outcomes or analyses were intended to be interpreted.

The registry lists one outcome measure and no analyses. That does not establish whether other outcomes were collected or analyzed outside the posted registry results. The record’s summary mentions physical state, symptoms, tumor-size changes, and laboratory findings as information the research team would consider, but numerical results for these measures are not reported in the registry record.

Missing-data methods and analysis populations are also not reported in the registry record. It does not state whether the primary analysis was intended to include all enrolled participants, only participants with an evaluable assessment, or another defined group. Nor does it explain how missed visits, incomplete measurements, or early withdrawal would affect the endpoint analysis.

These omissions matter because results can depend on which participants and observations are included and on how absent information is treated. Without those rules and the analysis results, readers cannot assess the extent of missingness, whether it could influence the endpoint, or whether the analysis followed the intended plan. No assumptions about missing outcomes or population definitions should be inferred from the enrollment count alone.

11. Safety Results

The registry reports serious adverse events as affected participants divided by participants at risk.

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 5% reporting threshold.

Most frequent non-serious adverse event terms

The safety record lists 0 serious adverse events among 4 participants at risk. It separately lists 2 other adverse events among 4 participants at risk. These are the counts and denominators recorded for the RAD 001 group; they should not be combined into a single category because the registry reports serious and other events separately.

The deaths entry is not reported in the registry record, so it should not be interpreted as a count of zero. No serious adverse event terms are listed. The record does not provide descriptions of the other adverse events, their severity, timing, duration, or relationship to study drug. It also does not describe the criteria used to classify events as serious or other.

In general, adverse-event counts summarize recorded events in the monitored group; they do not by themselves establish cause. A small risk group can provide only limited information about how frequently events occur, especially for uncommon events. These entries therefore describe the posted safety counts, but are not a complete safety profile and do not support conclusions beyond those counts.

12. What the Result Does, and Does Not, Mean

The registry record establishes that a phase II, single-group study of RAD001 was conducted with enrollment of 6, and that it was later listed as terminated. It records a daily oral dose of 10 mg and a primary endpoint of TTP, assessed at 8 weeks from the start of study drug. It also gives a progression definition and limited safety counts.

Those details do not establish whether RAD001 changed tumor growth or progression. No primary endpoint estimate, event count, or analysis is reported. The research aim described in the summary is not an outcome finding. In particular, an aim to discover whether a drug can shrink or slow tumor growth should not be read as evidence that either occurred.

The single-group design also lacks a concurrent comparison for separating outcomes associated with treatment from other influences. Even if a group-level outcome had been reported, a before-and-after or single-group description would not on its own provide the same evidence as a controlled comparison. In this record, the primary outcome result is not reported at all.

The safety entries show the recorded serious and other adverse-event counts in the stated group of participants at risk. They do not include enough information to characterize event types, attribution, or the broader range of possible harms. The deaths count is not reported in the registry record and cannot be treated as zero.

The record does not report why the study was terminated, how missing assessments were handled, or what statistical methods were used. Any planning illustrations should be understood as hypothetical calculations under stated assumptions, not as observed power or results from the study. The defensible interpretation is therefore limited: the registry documents a study design, endpoint definition, status, and a small set of safety counts, while leaving the efficacy analysis and many interpretive details unreported.

13. Why This Trial Matters Statistically

This study illustrates why a registry entry must be read in layers. The design description identifies a single-group phase II study and its intervention, while the outcome entry specifies what progression meant and when the primary assessment was planned. Those details are useful for understanding the question being asked. They are distinct from the evidence needed to answer it, such as observed event data, an estimate, and a measure of uncertainty.

The contrast between a clearly named primary endpoint and the absence of a reported analysis is statistically important. A defined endpoint does not guarantee that results are available or interpretable. Readers need to know what was observed, how follow-up and incomplete observations were handled, and what analysis was prespecified. Those details are not reported in the registry record.

The single-group structure also makes the distinction between description and comparison especially relevant. A treatment group’s outcomes, even when fully reported, cannot automatically be attributed to the intervention without considering alternative explanations. Here, no endpoint outcome is posted, so even a descriptive account of TTP cannot be made from the record.

The limited adverse-event counts provide another lesson: denominators and categories shape interpretation. Serious and other adverse events are separately recorded, while deaths are not reported. These entries should not be collapsed into broader claims about safety. Small recorded groups and absent event descriptions constrain what can be learned.

Finally, termination status and planning calculations answer different questions from trial outcomes. Status does not explain why a study ended, and planning illustrations do not show what was observed or establish the study’s original justification. Recognizing these distinctions prevents design details, registry status, and hypothetical calculations from being mistaken for evidence about the primary research question.

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 direct limitation of the registry record is that it contains no primary endpoint analysis. It names TTP and gives an assessment timeframe and progression definition, but does not report event counts, times, an estimate, or uncertainty. The efficacy question therefore cannot be evaluated from the posted results.

Enrollment was 6, and the record describes a single-group study without a comparator. This limits the scope of any potential inference from group outcomes, even if such outcomes were available. The posted safety data have a risk denominator of 4; the record does not explain how that denominator relates to the enrollment count or why it differs. No inference about the reason can be made.

Important design and analysis details are absent. The registry record does not report the rationale for the sample size, the statistical analysis plan, analysis populations, handling of missing assessments, interim analyses, or rules for censoring and event timing. It also does not report further eligibility details, follow-up beyond the stated timeframe, or a reason for termination.

Safety interpretation is constrained by the limited counts and the lack of event descriptions, severity, timing, attribution, and a reported deaths count. The registry’s summary indicates that symptoms, physical state, tumor changes, and laboratory findings were considered, but corresponding results are not reported in the registry record.

These limitations concern what can be established from the registry record; they do not prove that additional information was never collected or analyzed. Without posted results and methodological details, however, readers cannot verify whether further analyses were performed or assess their validity.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Trial of RAD001 in Triple Negative Metastatic Breast Cancer — NCT00827567.
• Registry record last updated 2014-01-13; status last verified 2013-12.
• Results first posted 2012-04-20.
• 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.

18. Record Summary

The record describes NCT00827567, a phase II, single-group study of RAD001 in triple-negative metastatic breast cancer, sponsored by Milton S. Hershey Medical Center. Enrollment is listed as 6. The intervention is described as RAD001, 10 mg by mouth once every day. The study started in 2009-04, has primary completion listed as 2011-02, and is marked terminated.

The primary outcome is TTP, assessed at 8 weeks from starting study drug. Progression is defined as a new lesion or a 20% increase over baseline in the sum of target lesions. The registry lists one outcome measure and no analyses, and reports no TTP result.

Safety entries list 0 serious adverse events among 4 at risk and 2 other adverse events among 4 at risk. Deaths are not reported in the registry record. The reason for termination and detailed statistical methods are also not reported in the registry record.