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 Multicenter Study of Pharmacokinetic-Guided Docetaxel in Breast Cancer Patients Receiving Docetaxel and Cyclophosphamide (NCT02502864), a na phase 4 study. The primary outcome is Rate of Achieving Targeted Area Under the Curve (AUC).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02502864 |
| Phase | Phase 4 |
| Condition | Breast Cancer, Breast Cancer Female |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 9 |
| Arms in the primary outcome | n/a |
| Lead sponsor | H. Lee Moffitt Cancer Center and Research Institute |
| Results posted | Yes |
2. Clinical Question
Population
The study concerned patients older than 65 years with breast cancer who were receiving docetaxel and cyclophosphamide chemotherapy. The primary endpoint description refers to participants evaluable at the time of analysis. The registry record lists enrollment of 9 participants. It does not report the number who were evaluable for the primary endpoint or provide further eligibility details.
Intervention
The study evaluated pharmacokinetic-guided docetaxel dosing alongside cyclophosphamide. The purpose described in the registry record was to adjust the docetaxel amount based on measured docetaxel levels in blood. Participants also received Function Assessment of Cancer Therapy surveys. Specific dose-adjustment rules and the full dosing description are not reported in the registry record.
Comparator
This is a single-group study, not a randomized comparison. The primary endpoint description sets the target against historical non-PK-guided therapy among patients receiving a similar regimen. The registry record does not describe a concurrent control group or provide results for a historical comparison group, so the record does not permit a like-for-like comparison between observed groups.
Primary question
The primary question was whether PK-guided docetaxel dosing could achieve a targeted area under the curve (AUC) of 2.5-3.7 mg*hr/L within 4 cycles of therapy. The endpoint description frames this in comparison with historical non-PK-guided therapy. The registry record does not report the number or proportion of participants who reached the target, nor a result for the historical comparison.
3. Trial Design
The registry record describes a Phase 4, completed, single-group study at H. Lee Moffitt Cancer Center and Research Institute. Enrollment was 9. Allocation is recorded as not applicable, and masking as none. These design features mean that the study did not assign participants between randomized treatment groups and was not masked. Its listed intervention group was Standard of Care + Surveys.
Participants received docetaxel and cyclophosphamide, described as the TC regimen, together with Function Assessment of Cancer Therapy surveys. The arm description says that participants received TC for cycle 1, with subsequent cycles repeated every 3 weeks for a total of 4 cycles. That description is truncated while explaining initial dosing, so the complete dosing schedule and the subsequent dose-adjustment procedure are not reported in the registry record.
The study’s stated purpose was to adjust docetaxel dosing according to the level measured in blood. Its primary endpoint concerns reaching a targeted AUC by cycle 4, with a historical non-PK-guided group mentioned as the comparison in the endpoint description. There is no separate concurrent comparison arm in the listed design. The record does not explain how historical participants were selected, whether their outcomes were available for analysis, or how comparability was assessed.
The study started on 2016-02-09, reached primary completion on 2017-09-29, and is marked completed. These dates describe the registry record; they do not establish when analyses were conducted or when results became available. The record lists secondary outcomes relating to Grade 3 and 4 neutropenia and febrile neutropenia, and associations between scores on three named assessment instruments. Further details of the instruments’ analysis and the corresponding results 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 | Standard of Care + Surveys |
|---|---|
| STARTED | 9 |
| COMPLETED | 8 |
| NOT COMPLETED | 1 |
Reasons for not completing this period:
| Reason | Standard of Care + Surveys |
|---|---|
| Withdrawal by Subject | 1 |
Analysis population for the primary outcome, as defined in the registry: All participants evaluable at time of 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.
Population: All participants.
| Characteristic | Unit | Standard of Care + Surveys |
|---|---|---|
| Participants analysed | 9 | |
| Age, Continuous | years | 72 (66 to 85) |
| Sex: Female, Male: Female | Participants | 9 |
| Sex: Female, Male: Male | Participants | 0 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 1 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 8 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 |
| Race (NIH/OMB): White | Participants | 9 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Region of Enrollment: United States | participants | 9 |
6. Primary Endpoint
The registry lists the primary outcome as Rate of Achieving Targeted Area Under the Curve (AUC), measured over Cycle 4 - Up to 6 months.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the rate of achieving a targeted docetaxel AUC, expressed as a count of participants. AUC represents exposure over time: as a general statistical and pharmacokinetic concept, it combines the measured drug concentration with the duration over which that concentration is observed. The target stated in the registry record is 2.5-3.7 mg*hr/L. The endpoint is assessed at cycle 4, with the timeframe described as up to 6 months.
The stated rationale is to assess whether PK-guided dose adjustment improves the ability to reach the target within 4 cycles, compared with historical non-PK-guided therapy in patients receiving a similar regimen. In general, a target-exposure endpoint can describe whether dosing achieves a prespecified concentration-time range. It is distinct from a direct measure of tumor response, survival, or symptoms; those are not the primary endpoint named in this record.
The record does not report the number of participants who achieved the target, the number evaluable, or any estimate of the endpoint’s uncertainty. It also does not report the historical comparator’s results. Therefore, the endpoint’s intended meaning can be explained, but its observed value and comparative result cannot be interpreted from the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central statistical limitation is that the registry record names the primary endpoint but does not report its observed result. It states that the outcome is a count of participants achieving a target AUC of 2.5-3.7 mg*hr/L by cycle 4, but gives no count, rate, interval estimate, or test result for that outcome. The record also does not state how many participants were evaluable at the time of analysis. Consequently, the degree to which participants reached the target cannot be described from the posted results.
The endpoint description refers to historical non-PK-guided therapy as the comparison. This does not amount to a concurrent control arm in the listed single-group design. The record does not report a historical group’s sample size, outcome data, selection process, or methods for making the groups comparable. Without those details and results, there is no basis in the registry record for estimating a difference between PK-guided and non-PK-guided dosing.
General statistical principle: an estimate should be interpreted together with its uncertainty. A confidence interval describes a range of values reasonably compatible with the observed data under the method used; a narrow interval indicates greater precision than a wide interval. No confidence interval for the primary endpoint is reported in the registry record, so precision cannot be assessed. No hypothesis-test result is reported either. This absence is not evidence that the target was or was not achieved.
The enrollment of 9 gives context for the scale of the study, but enrollment alone is not an outcome result. It also does not establish how many participants completed the relevant cycle or contributed to the primary analysis. A small study can produce an estimate with substantial uncertainty, but the actual uncertainty cannot be quantified because the endpoint count and analysis population are not reported.
The record identifies secondary outcomes, including neutropenia and febrile neutropenia, and associations involving assessment scores. It does not report corresponding outcome estimates or analyses. Thus, the listed outcome titles indicate what the study intended to examine, not what it found. The record indicates 6 outcome measures and 0 analyses listed, which leaves the results and inferential approach unavailable in this registry entry.
Overall, the posted record supports a description of the study’s question, design, and selected safety counts. It does not support a conclusion about the primary target-attainment result or comparative effectiveness. This distinction matters: a proposed endpoint and a study rationale are not substitutes for an observed result, and an unreported estimate cannot be treated as either favorable, unfavorable, or null.
9. Statistical Methods Explained
General statistical concepts help clarify what the design could address, while the registry record’s missing analysis details limit what can be said about the actual methods. The design is single-group and unmasked, with allocation recorded as not applicable. In a single-group study, outcomes are observed under the study regimen without a concurrently assigned control group. That can describe the experience of participants enrolled in the study, but by itself it does not isolate the regimen’s effect from other explanations for an observed outcome.
What does the primary endpoint count?
The endpoint is a count of participants achieving the targeted AUC by cycle 4. A count is different from a continuous measurement of AUC for every participant: it classifies participants according to whether their measured exposure reached the specified target. General statistical principle: a binary outcome can be summarized by the number experiencing the event and the number assessed. A rate or proportion requires a defined denominator, usually the participants included in the relevant analysis. The registry record does not report the achieved count or the evaluable denominator, so no observed rate can be interpreted.
The target range is 2.5-3.7 mg*hr/L. The record does not provide the operational details needed to reconstruct classification, such as measurement timing, handling of measurements outside the target, or treatment of participants without a cycle 4 assessment. Those details are not reported in the registry record. They matter because outcome definitions and missing measurements can affect who is counted as having achieved the target.
Why does the comparator matter?
The primary endpoint description invokes historical non-PK-guided therapy. General statistical principle: a concurrent control group can help distinguish differences associated with an intervention from differences in patient characteristics, care practices, or measurement conditions. Historical comparisons may be informative, but the interpretation depends on the comparability of the historical and current participants and on consistent outcome measurement. The record does not give the historical group’s size, results, selection criteria, or analysis, so those features cannot be assessed.
Because there is no randomized allocation between groups, this study should not be read as a randomized trial comparison. The registry record does not report adjustment methods for differences between the study participants and any historical patients. It also does not report whether the historical comparison was actually analyzed. The stated comparator therefore describes the intended context for the endpoint, rather than a result that can be quantified from the available record.
How should uncertainty be understood?
General statistical principle: an estimate has sampling uncertainty because the participants observed are one possible sample from a broader population. Confidence intervals communicate that uncertainty for an estimate, subject to the assumptions of the method. A p-value, when used, summarizes how compatible the data are with a specified null hypothesis under the statistical model; it does not measure the size or clinical importance of an effect. The registry record reports no primary endpoint estimate, confidence interval, or p-value. No particular inferential method should be attributed to the study.
Over-dispersion is a general concern when count data vary more than a simple model expects. It can make standard errors too small if the model does not account for the extra variation. This concept does not establish that over-dispersion occurred in this study: the primary measure is a participant count, and the registry record does not report a fitted model or an over-dispersion assessment.
What do the planning illustrations mean?
General statistical principle: sample-size planning asks how many participants may be needed to detect a chosen effect under specified assumptions. The planning illustration associated with this analysis is described as based on the observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It considers effects expressed as a stated fraction of the observed effect. These are planning assumptions, not evidence that the trial had those design features: the actual study is single-group, and no observed primary effect is reported in the registry record. The registry record does not report its own sample-size justification. Any sample-size illustration is a planning exercise, not observed power, and cannot supply the missing endpoint result.
The study’s endpoint timeframe is cycle 4, up to 6 months, and the treatment description specifies a total of 4 cycles repeated every 3 weeks after cycle 1. The record does not report rules for censoring, missing cycle assessments, or analysis populations beyond the description that the primary population consists of participants evaluable at the time of analysis. Without those details, the analysis cannot be reproduced from the registry record.
10. Multiplicity, Missing Data and Analysis Populations
The registry record identifies a primary endpoint and secondary outcomes that include Grade 3 and 4 neutropenia and febrile neutropenia, plus associations involving scores on the Chemotherapy Risk Assessment Scale for High-Age Patients, Instrumental Activities of Daily Living, and Cumulative Illness Rating Scale for Geriatrics. It indicates 6 outcome measures and lists 0 analyses. The record does not describe a testing hierarchy, a multiplicity adjustment, or how confirmatory and exploratory analyses were distinguished. Those aspects are not reported in the registry record.
General statistical principle: when several outcomes or hypotheses are examined, the chance of at least one apparently notable result can increase if each is tested separately without an adjustment or prespecified hierarchy. Whether and how to address multiplicity depends on the study’s aims and analysis plan. Because the analysis plan and outcome results are not reported, no conclusion can be drawn about how multiplicity was handled or its effect on interpretation.
The primary analysis population is described as all participants evaluable at the time of analysis, but the registry record does not define evaluability or report the number analyzed. It does not state how missing outcome measurements were handled, whether all enrolled participants were included in any analysis, or whether sensitivity analyses were conducted. General statistical principle: missing outcomes can affect an estimate if the participants with observed data differ systematically from those without observed data. The extent and direction of any such effect cannot be assessed without information about missingness and the analysis approach.
The available record therefore does not establish a complete analysis population, missing-data strategy, or multiplicity procedure. These omissions constrain assessment of both precision and the reliability of any unreported secondary findings.
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 |
|---|---|---|---|
| Standard of Care + Surveys | 3 | 9 | 33.3% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Standard of Care + Surveys | 0 of 9 |
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 |
|---|---|
| Standard of Care + Surveys | 9 of 9 |
Most frequent serious adverse event terms
| Event term | Organ system | Standard of Care + Surveys |
|---|---|---|
| Fatigue | General disorders | 1 of 9 |
| Non-cardiac chest pain | General disorders | 1 of 9 |
| Sepsis | Infections and infestations | 1 of 9 |
| Urinary tract infection | Infections and infestations | 1 of 9 |
| Syncope | Nervous system disorders | 1 of 9 |
| Anxiety | Psychiatric disorders | 1 of 9 |
| Thromboembolic event | Vascular disorders | 1 of 9 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Standard of Care + Surveys |
|---|---|---|
| Fatigue | General disorders | 9 of 9 |
| Constipation | Gastrointestinal disorders | 6 of 9 |
| Anemia | Blood and lymphatic system disorders | 6 of 9 |
| Alopecia | Skin and subcutaneous tissue disorders | 6 of 9 |
| Diarrhea | Gastrointestinal disorders | 5 of 9 |
| Bone pain | Musculoskeletal and connective tissue disorders | 5 of 9 |
| Nausea | Gastrointestinal disorders | 4 of 9 |
| Neutrophil count decreased | Investigations | 4 of 9 |
| White blood cell decreased | Investigations | 4 of 9 |
| Dry mouth | Gastrointestinal disorders | 3 of 9 |
Serious events with the highest arm-level rate: fatigue (11.1%), non-cardiac chest pain (11.1%), sepsis (11.1%), urinary tract infection (11.1%), syncope (11.1%).
The safety summary lists 9 participants at risk for serious adverse events, with 3 affected, and 9 at risk for other adverse events, with 9 affected. It lists 0 deaths. These are participant counts in the registry record; they do not by themselves identify whether events were judged related to docetaxel, cyclophosphamide, or another cause. The record does not report event-level timing, duration, outcome, or attribution.
The serious-event terms listed with percentages are Fatigue, Non-cardiac chest pain, Sepsis, Urinary tract infection, Syncope, and Anxiety, each recorded as 11.1%. The percentages are presented as event-term summaries, not as a comparison between treatment groups. Because this is a single-group study, there is no concurrent control group in the listed design to provide a like-for-like safety comparison.
General statistical principle: an adverse-event count describes how many participants were recorded with events under the reporting definitions; it does not establish causation. Multiple terms may occur in one participant, and the record does not specify whether the listed terms are mutually exclusive. A zero count for deaths in this enrolled group is a description of the posted safety summary, not proof that the risk of death is absent. The record does not give further detail needed to assess completeness of safety follow-up.
12. What the Result Does, and Does Not, Mean
This study asks a focused pharmacokinetic question: can docetaxel dosing guided by measured blood levels achieve a target AUC within the stated treatment period? The registry record specifies the target range and intended assessment by cycle 4. It also describes a historical non-PK-guided reference. However, the record does not report the primary endpoint count or rate, the number evaluable, or the historical comparison result. The main question therefore remains unanswered by the posted results.
The study design also defines what can and cannot be inferred. A completed single-group study can document observations among its participants, but without a concurrent control group it cannot make a randomized comparison. A historical comparator would require outcome data and methods to assess comparability; those details are not reported in the registry record. The design description alone does not demonstrate that PK-guided dosing increased target attainment.
The enrollment of 9 is the recorded study size, not the number known to have completed the primary assessment. The registry record does not indicate how many completed cycle 4 or were included in the primary analysis. It also does not provide estimates of uncertainty. Consequently, neither the magnitude nor the precision of target attainment can be judged.
The safety counts are descriptive: the record lists 3 participants affected by serious adverse events, 9 affected by other adverse events, and 0 deaths. These counts do not establish that the study regimen caused the events, and they do not allow a comparison of risk against another treatment group. The serious-event terms and their recorded percentages add descriptive detail, but do not resolve attribution or comparative safety.
In general, a study rationale describes why a question is worth asking; an endpoint states how a question is to be measured; and an observed result provides the evidence needed to answer it. Here, the registry record contains the rationale and endpoint definition, but not the primary outcome result. It should not be read as evidence that the target was achieved or not achieved, or that one dosing strategy is better than another. Any conclusion beyond the study’s stated design and posted counts would require results and analysis details that are not reported in the registry record.
13. Why This Trial Matters Statistically
This study is statistically notable because its primary question concerns a measured exposure target rather than a direct clinical outcome. AUC provides a way to describe drug exposure over time, and the registry record defines a target range for docetaxel. The intended endpoint is the number of participants who reach that range by cycle 4. This kind of endpoint can help make a dosing strategy measurable, but interpretation depends on the measurement procedure, outcome definition, and analysis population.
The design also illustrates the difference between a single-group study and a controlled comparison. The study intended to set target attainment against historical non-PK-guided therapy, but its listed design has one group and no randomized allocation. Historical comparison requires information about the reference participants and their outcome measurement. Those details and the comparative results are not reported in the registry record, so the intended comparison cannot be evaluated statistically.
The record’s incomplete reporting is itself important for statistical reading. It names an endpoint and reports enrollment, but does not report the primary endpoint result, evaluable count, or uncertainty. Without these, readers cannot assess the estimate’s precision or determine whether the target was reached. The listed secondary outcomes likewise do not have reported analyses in this registry entry.
General statistical principle: transparent reporting connects a question, an outcome definition, a population, an analysis method, and an estimate with uncertainty. Each link supports interpretation and reproducibility. When the result and analysis are absent, the study can still be described accurately, but its principal statistical finding cannot be assessed. This makes the distinction between a planned question and an observed answer especially consequential in this record.
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 has several trial-specific limitations for interpretation. The primary endpoint result is not reported, despite the record identifying target AUC attainment as the primary measure. The number of participants evaluable at cycle 4 is also not reported, so the denominator and completeness of the primary assessment are unknown.
The study is listed as single-group, with allocation not applicable and no masking. The endpoint description references historical non-PK-guided therapy, but no concurrent control arm is listed and no historical group results or selection details are reported. This prevents assessment of the proposed comparison and of whether the groups would be comparable.
The arm description ends during the explanation of initial dosing. The complete dosing protocol and specific PK-guided adjustment rules are not reported in the registry record. The record also does not describe measurement timing, adherence, missing cycle assessments, or how participants without an evaluable AUC were handled. These omissions make it difficult to determine how consistently the target was assessed.
Although the record lists secondary outcomes and a safety summary, it does not provide analyses for the secondary measures or detailed event-level information. It does not report methods for multiplicity, missing data, or the choice of analysis populations beyond the statement that the primary population comprises participants evaluable at analysis. The registry record also does not report its own sample-size justification. These limitations restrict reproducibility and prevent conclusions about the primary result, its precision, or comparative benefit and risk.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Multicenter Study of Pharmacokinetic-Guided Docetaxel in Breast Cancer Patients Receiving Docetaxel and Cyclophosphamide — NCT02502864.
- Registry record last updated 2020-09-11; status last verified 2020-09.
- Results first posted 2018-10-18.
- 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
NCT02502864 is a completed Phase 4, single-group study of pharmacokinetic-guided docetaxel with cyclophosphamide in senior adult breast cancer patients. Enrollment was 9. The study started on 2016-02-09, with primary completion on 2017-09-29. The listed group received standard-of-care TC chemotherapy and Function Assessment of Cancer Therapy surveys.
The primary endpoint was the count of participants achieving a targeted docetaxel AUC of 2.5-3.7 mg*hr/L by cycle 4, with historical non-PK-guided therapy named as the reference. The record does not report the primary outcome result, the evaluable count, or a historical comparison result. It lists 6 outcome measures and 0 analyses.
The safety summary reports 3 participants with serious adverse events, 9 with other adverse events, and 0 deaths. These counts are descriptive and do not establish causation or comparative safety. Important dosing, analysis, and missing-data details are not reported in the registry record.