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 A Study to Determine the Management of Palmar-plantar Erythrodysesthesia (PPE) in Patients With Metastatic Ovarian or Breast Cancer Treated With Caelyx (Study P05020) (NCT00746694), a registered phase not stated study. The primary outcome is Number of Participants Who Received Concomitant Treatment Strategies to Manage Palmar-Plantar Erythrodysesthesia (PPE).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00746694 |
| Phase | Phase not stated |
| Condition | Ovarian Neoplasms, Breast Neoplasms, Paresthesia |
| Allocation | Not Stated |
| Design model | Not Stated |
| Masking | Not Stated |
| Enrollment | 154 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Merck Sharp & Dohme LLC |
| Results posted | Yes |
2. Clinical Question
Population
Participants with metastatic ovarian or breast cancer treated with Caelyx. The registry record lists enrollment of 154 participants. The primary outcome concerns participants treated with Caelyx who developed PPE, but the number who developed PPE is not reported in the registry record.
Intervention
Caelyx was given as part of standard treatment and according to approved indications in the data sheet. The study also recorded strategies used to manage PPE, including prescribed treatments and recommendations about skin hydration, sweating and physical activity, clothing, and local cooling.
Comparator
No comparison group is described. The registry record does not state an allocation method, and the listed material does not support a like-for-like comparison between treatment arms. The study therefore addresses management in the Caelyx-treated group rather than a comparative treatment effect.
Primary question
Among participants treated with Caelyx who developed PPE, what concomitant strategies were used to manage symptoms? For four behavioral recommendations, participants were counted if they reported always or sometimes following the strategy. The registry objective also includes the frequency of pharmacological treatment and adherence to educational recommendations, but the corresponding primary counts are not reported in the registry record.
3. Trial Design
NCT00746694 is a completed epidemiological study of PPE management in participants with metastatic ovarian or breast cancer treated with Caelyx. The registry lists enrollment of 154. Caelyx was used as part of standard treatment and according to data-sheet-approved indications. The study was not described as a randomized comparison, and the allocation method is not stated. No like-for-like control comparison is described.
The primary outcome is the number of participants who received concomitant strategies to manage PPE. The outcome description focuses on participants who developed PPE and on categories of strategies prescribed to manage symptoms. For the recommendations to keep skin hydrated, avoid sweating and physical activity, avoid tight-fitting clothing, and use local cooling of hands and feet, the record says that participants were counted if they followed the recommendation always or sometimes.
The outcome timeframe describes a single visit for participants, with cases collected during a period of 12 months. The registry lists a start date of 2007-10 and primary completion in 2009-06, and records the study as completed. These dates do not establish the duration of an individual participant’s treatment or follow-up beyond the stated outcome timeframe.
The registry objective includes describing the frequency of preventive or therapeutic pharmacological treatment for PPE and compliance with educational recommendations. The registry record does not report the primary outcome counts, the number who developed PPE, or detailed results for each management strategy. It lists 2 outcome measures and 0 analyses, so the available record does not supply a statistical comparison or a complete numerical account of management.
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 | Caelyx |
|---|---|
| STARTED | 154 |
| COMPLETED | 154 |
| 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 | Caelyx |
|---|---|---|
| Participants analysed | 154 | |
| Age, Continuous | years | 62.4 (SD 10.1) |
| Sex: Female, Male: Female | Participants | 154 |
| Sex: Female, Male: Male | Participants | 0 |
| Region of Enrollment: Canada | participants | 154 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Participants Who Received Concomitant Treatment Strategies to Manage Palmar-Plantar Erythrodysesthesia (PPE), measured over Participants will do a single visit, but cases will be collected during a period of 12 months..
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is a count of participants who received concomitant strategies to manage PPE. It is intended to describe the management of symptoms among participants treated with Caelyx who developed PPE. The listed strategies span prescribed treatments and behavioral recommendations; the outcome is therefore about reported management practices, not a direct measurement of symptom severity or treatment efficacy.
For the recommendations to keep skin hydrated, avoid sweating and physical activity, avoid tight-fitting clothing, and use local cooling of hands and feet, a participant was counted if they followed the strategy always or sometimes. That definition combines those response categories for the count. It does not show how often each strategy was followed separately, or distinguish always from sometimes, unless those results are reported elsewhere. Those breakdowns are not reported in the registry record.
A count endpoint can describe how many participants met a defined condition, but it needs a clear denominator to convey the frequency of that condition. The record gives no primary endpoint counts and does not report the number who developed PPE. Consequently, the endpoint’s observed frequency and precision cannot be assessed from the registry record. The endpoint is useful for characterizing care practices, but the available description alone cannot establish whether a strategy changed PPE outcomes.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry describes a study intended to characterize how PPE was managed among participants with metastatic ovarian or breast cancer treated with Caelyx. Its primary outcome is a participant count for concomitant management strategies, including behavioral recommendations. However, the primary counts are not reported in the registry record. The number of participants who developed PPE is also not reported, so a rate or proportion of affected participants receiving any particular strategy cannot be interpreted from the available information.
The record lists enrollment of 154 and reports 154 at risk in the Caelyx group for the adverse-event summaries. These figures do not supply the missing denominator for the primary PPE-management endpoint: the endpoint description focuses on participants who developed PPE, and their number is not given. The registry does not report a primary endpoint estimate, confidence interval, or inferential analysis. It lists 0 analyses. Thus, there is no reported statistical estimate from which to judge uncertainty, statistical significance, or differences between strategies.
The adverse-event material is separate from the primary management endpoint. It records 3 serious adverse events among 154 at risk and 122 other adverse events among 154 at risk. The serious-event terms listed include neutropenia at 1.3%, anaemia at 0.6%, and febrile neutropenia at 0.6%. These figures describe the registry’s safety entries; they do not quantify PPE-management practices or establish that Caelyx caused an event. The record does not provide additional detail needed to interpret event timing, attribution, or overlap between categories.
No control group is described, and allocation is not stated. The study therefore does not provide a like-for-like comparison of management strategies or a basis for attributing observed outcomes to one strategy rather than another. Its stated aim is descriptive: to study management and the use of pharmacological treatment and educational recommendations.
Any sample-size planning illustrations based on observed effects should be read as hypothetical planning calculations, not as results from this study or evidence of observed power. The assumptions describe a two-arm design with equal allocation, a two-sided significance level of 0.05, a normal approximation, and an effect defined as a stated fraction of an observed effect. Those assumptions do not turn this single-group record into a comparative trial. The primary result cannot be evaluated numerically without the missing endpoint counts and analysis details.
9. Statistical Methods Explained
What does a participant count tell the reader?
The primary endpoint is a count: it describes the number of participants meeting the endpoint definition. In general, a count becomes easier to interpret when its denominator is also clear. A proportion relates the count to the population eligible for that outcome, while a confidence interval expresses the statistical uncertainty around an estimate. Neither an endpoint count, a denominator for participants who developed PPE, nor an interval estimate is reported in the registry record. It is therefore not possible to assess the observed frequency or its precision from the available primary outcome information.
The endpoint definition also matters. The registry specifies that participants reporting they always or sometimes followed four named recommendations were counted under those strategies. Combining response categories can be a practical way to summarize adherence, but it does not retain the distinction between those responses. Separate counts by recommendation and response category would answer more detailed questions; those results are not reported in the registry record.
Why does the design affect interpretation?
In general, a randomized comparison can help distinguish treatment effects from differences that existed before treatment, because assignment to groups is controlled by the study. This registry does not describe a randomized comparison or a control group, and its allocation method is not stated. Its stated purpose is to document management among people treated with Caelyx. That descriptive design can characterize practice, but it does not by itself establish that a particular management strategy caused a change in PPE or another outcome.
The timeframe says participants had a single visit and that cases were collected during a period of 12 months. A single visit is not the same as a repeated-measures design. Without further details, the record does not establish when each strategy was used relative to PPE onset, how long participants were observed, or whether management changed over time. Those details are not reported in the registry record.
How should uncertainty and significance be read?
In general, a confidence interval gives a range of values compatible with the data and the specified statistical method. Its width reflects precision: wider intervals indicate greater uncertainty. A p-value, when used, summarizes how incompatible the observed data are with a specified null hypothesis under the analysis assumptions; it does not measure the size or clinical importance of an effect. Neither confidence intervals nor p-values for the primary endpoint are reported in the registry record. No conclusion about statistical significance or precision is therefore supported by the primary outcome information.
For a binary outcome, a model may estimate the probability of an event or compare probabilities between groups. A count of participants can be converted into such an estimate only when the relevant denominator and outcome definition are available. Here, the primary endpoint counts and the number of participants who developed PPE are not reported, and no comparative groups are described. No model-based effect estimate can be interpreted from the registry record.
What do planning assumptions mean?
General sample-size planning asks how much information a future study would need to detect a chosen effect under stated assumptions. The registry’s planning description specifies a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; it also defines the target effect as a stated fraction of the observed effect. Such calculations are illustrations, not evidence that the completed study used those design choices. The record does not report a protocol-based sample-size justification, and the described two-arm assumptions do not match a like-for-like comparison documented in this study.
Planning values are not observed power. Power is a design concept based on assumptions about an effect and variability; calculating it after observing data does not show the probability that the study correctly detected a real effect. No primary endpoint analysis is reported, so planning assumptions cannot substitute for an estimate and its uncertainty. If an illustration uses an effect derived from observed results, it remains conditional on that effect and the stated model assumptions.
Why might over-dispersion matter in general?
Over-dispersion occurs when observed variability is greater than a statistical model expects. It can arise when outcomes are more heterogeneous or correlated than the model assumes. For a simple binary outcome, an appropriate analysis depends on the sampling structure, the denominator, and whether observations are independent. If participants are clustered or measured repeatedly, methods that account for that structure may be needed. The registry record does not report clustering, repeated measurements, or an analysis model, so it is not possible to determine whether over-dispersion or dependence was assessed.
These general concepts explain what would be needed to assess a quantitative result; they do not imply that any particular model was used in this study. The registry lists 0 analyses and does not report the primary endpoint counts, uncertainty estimates, or model specifications. The statistical interpretation must therefore remain descriptive and limited to the recorded design and safety figures.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 2 outcome measures and 0 analyses. It does not report how many hypotheses were tested, whether outcomes were ranked for inferential testing, or whether any adjustment for multiple comparisons was planned or used. Multiplicity methods are not reported in the registry record. Without analysis results and a prespecified testing strategy, it is not possible to assess how the study handled the chance of false-positive findings across multiple outcomes or strategy categories.
Missing-data procedures are not reported in the registry record. The record does not describe whether participants with incomplete responses were excluded, whether missing responses were imputed, or how missing endpoint information was handled. The number of participants with missing primary outcome data is not reported. As a result, the completeness of the endpoint data and the potential for missingness to affect the observed counts cannot be assessed.
The analysis population is also not reported in the registry record. Although enrollment is listed as 154, the record does not specify which participants contributed to each primary outcome analysis, nor does it give the number who developed PPE. The primary endpoint is defined for participants treated with Caelyx who developed PPE, but the corresponding denominator and analysis set are not stated.
These omissions limit the interpretation of any apparent frequency or pattern. They do not establish that the study failed to address multiplicity or missing data in its full protocol; those details are simply not reported in the registry record.
11. Safety Results
The registry reports serious adverse events as affected participants divided by participants at risk.
| Arm | Participants with serious adverse events | Participants at risk | Percentage |
|---|---|---|---|
| Caelyx | 3 | 154 | 1.9% |
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 |
|---|---|
| Caelyx | 122 of 154 |
Most frequent serious adverse event terms
| Event term | Organ system | Caelyx |
|---|---|---|
| Neutropenia | Blood and lymphatic system disorders | 2 of 154 |
| Anaemia | Blood and lymphatic system disorders | 1 of 154 |
| Febrile neutropenia | Blood and lymphatic system disorders | 1 of 154 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Caelyx |
|---|---|---|
| Stomatitis | Gastrointestinal disorders | 68 of 154 |
| Asthenia | General disorders | 61 of 154 |
| Nausea | Gastrointestinal disorders | 51 of 154 |
| Alopecia | Skin and subcutaneous tissue disorders | 44 of 154 |
| Anorexia | Metabolism and nutrition disorders | 27 of 154 |
| Constipation | Gastrointestinal disorders | 21 of 154 |
| Diarrhoea | Gastrointestinal disorders | 21 of 154 |
| Neutropenia | Blood and lymphatic system disorders | 18 of 154 |
| Vomiting | Gastrointestinal disorders | 15 of 154 |
| Abdominal pain | Gastrointestinal disorders | 11 of 154 |
Serious events with the highest arm-level rate: neutropenia (1.3%), anaemia (0.6%), febrile neutropenia (0.6%).
The safety summary lists 154 participants at risk in the Caelyx group, with 3 serious adverse events and 122 other adverse events. These are separate registry categories. The figures should not be added together to estimate a total, because the record does not describe whether categories overlap or how events were counted. The registry does not provide enough detail to determine whether each figure represents participants, event episodes, or another counting convention beyond its listed adverse-event fields.
The serious adverse-event terms reported include neutropenia at 1.3%, anaemia at 0.6%, and febrile neutropenia at 0.6%. These percentages are registry entries for the listed terms; the record does not give term-specific counts in the material. They should not be read as a complete account of all possible adverse events, nor do they establish attribution to Caelyx. The registry record does not report further details about timing, severity criteria, event overlap, or the assessment of causality.
Safety summaries describe events recorded in a study population; they do not provide a controlled comparison when no like-for-like control group is described. These figures should also be kept distinct from the primary PPE-management endpoint. The latter’s counts are not reported in the registry record.
12. What the Result Does, and Does Not, Mean
The study’s stated purpose is to describe how PPE was managed in participants with metastatic ovarian or breast cancer treated with Caelyx. It focuses on the use of preventive or therapeutic pharmacological treatment and adherence to educational recommendations. The endpoint definition identifies several recommendations and counts participants who reported always or sometimes following them. This is a description of recorded management practices, not a direct comparison of treatments.
The primary endpoint counts are not reported in the registry record. Neither is the number of participants who developed PPE. Therefore, the available record does not show how common any management strategy was among the participants eligible for that endpoint, how strategies differed in frequency, or how precisely those frequencies were estimated. It also does not show whether following a recommendation was associated with a change in PPE symptoms.
The registry does not describe a control group or a randomized allocation method. Consequently, the record does not support a causal conclusion about Caelyx, a particular PPE strategy, or the comparative effects of different management approaches. It cannot establish whether a strategy prevented PPE, reduced its severity, or improved another clinical outcome. Those questions require outcome data and an appropriate comparative analysis, neither of which is reported for the primary endpoint.
The adverse-event summary provides separate recorded safety figures, but it does not fill the gap in the primary management results. The listed counts and percentages should be interpreted as registry entries, not as a comparison or a causal assessment. Overall, the record identifies the study question and broad design, but lacks the primary numerical results needed to judge the observed management patterns.
13. Why This Trial Matters Statistically
This study addresses a practical statistical question: how to describe management strategies in a clinical population when the goal is to document care rather than compare assigned treatments. Its endpoint combines participant counts across several strategy categories, including both prescribed treatment and behavioral recommendations. Clear definitions are important because a count depends on who qualifies for the endpoint and how responses are classified. The record specifies an always-or-sometimes rule for four recommendations, which helps define those categories.
The study also illustrates why denominators matter. The primary outcome concerns participants who developed PPE, but the number who developed it is not reported. Without that denominator, the primary counts cannot be translated into interpretable frequencies for the affected group. Without uncertainty estimates, the stability and precision of any frequency cannot be assessed.
Design language matters as well. The registry describes an epidemiological study and does not document a randomized comparison or a control group. A descriptive study can inform understanding of recorded practice, but it cannot isolate the effect of a management strategy in the way a suitable comparative design may. The distinction between describing care and evaluating causal effects is central to reading clinical registry results responsibly.
Finally, the record separates its management objective from safety reporting. Serious and other adverse-event figures provide a different kind of information and should not be treated as evidence about the primary endpoint. The missing primary counts and analyses mean the registry supports a description of the research question and design, but not a numerical conclusion about management frequency or comparative effectiveness.
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 principal limitation for interpreting this trial record is that the primary endpoint counts are not reported in the registry record. The number of participants who developed PPE, the denominator for management frequencies, and strategy-specific results are also not reported. This prevents assessment of how common the recorded management approaches were and how precisely their frequencies were estimated.
The record describes a single visit for participants, with cases collected during a period of 12 months, but does not provide further detail about the timing of PPE onset, the timing of management, or changes in strategies. It does not report how complete the endpoint data were, how missing responses were handled, or which analysis population contributed to each outcome. These omissions make it difficult to assess the representativeness of the recorded responses.
No control group or randomized allocation method is described. The study therefore does not support a like-for-like comparison of management approaches. The registry also lists 0 analyses and does not report confidence intervals, p-values, model details, multiplicity procedures, or a protocol-based sample-size justification. Those methods are not reported in the registry record, so it is not possible to evaluate the precision or inferential basis of a primary result.
The safety summary gives event figures and selected serious-event terms, but does not report further detail about attribution, timing, overlap, or counting conventions. The limitations concern what can be concluded from the registry record; they do not establish what analyses or procedures may have been described in other study documents.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: A Study to Determine the Management of Palmar-plantar Erythrodysesthesia (PPE) in Patients With Metastatic Ovarian or Breast Cancer Treated With Caelyx (Study P05020) — NCT00746694.
- Registry record last updated 2015-08-27; status last verified 2015-08.
- Results first posted 2011-01-17.
- 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
NCT00746694 is a completed epidemiological study of PPE management among participants with metastatic ovarian or breast cancer treated with Caelyx. The registry lists enrollment of 154, a single visit for participants, and collection of cases during a period of 12 months. The primary endpoint is the number of participants receiving concomitant strategies to manage PPE, including specified behavioral recommendations.
The primary endpoint counts and the number of participants who developed PPE are not reported in the registry record. The record lists 2 outcome measures and 0 analyses, and does not describe a like-for-like control comparison. Safety entries list 3 serious adverse events and 122 other adverse events among 154 at risk; selected serious-event terms are also reported as percentages. The available record defines the study question and broad design, but does not provide the primary numerical results or inferential analyses needed to quantify management patterns.