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 Phase I/II, Multi-Center, Open-Label, Dose-Escalation, Safety and Efficacy Study of PHY906 Plus Capecitabine in Patients With Advanced Pancreatic Carcinoma (NCT00411762), a na phase 1, phase 2 study. The primary outcome is Median Progression Free Survival.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00411762 |
| Phase | Phase 1, Phase 2 |
| Condition | Pancreatic Cancer |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 25 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Yale University |
| Results posted | Yes |
2. Clinical Question
Population
The study concerned patients with advanced pancreatic carcinoma. The registry record reports enrollment of 25 participants. For the response-evaluation population, it specifies 20 subjects who received 2 cycles or more and were evaluated for response. These descriptions identify the study population and the group named for response evaluation; the record does not explain how the two population descriptions relate for every analysis.
Intervention
The intervention combined PHY906 with capecitabine. The listed regimen describes PHY906 800mg orally twice a day for days 1-4, together with capecitabine 1500mg/m^2 on days 1-7 of a 14-day cycle. The study was Phase I/II and included dose escalation, with safety and efficacy among its stated aims. The record does not report the dose-escalation outcomes or describe which dose levels were administered.
Comparator
There was no separate control group: the registry classifies the study as single group, with allocation listed as NA and masking as NONE. Thus, the design does not provide a randomized, like-for-like comparison against another treatment or against no study treatment. Any outcome description is about the participants treated in this study, not a contrast between assigned groups.
Primary question
The registered primary outcome was median progression-free survival, measured in weeks, with a timeframe of up to 100 weeks. Progression was defined using RECIST v1.0 criteria: a 20% increase in the sum of the longest diameter of target lesions, a measurable increase in a non-target lesion, or the appearance of new lesions. The registry record does not report the median value or its full range. The study also named median overall survival as a secondary outcome, but its numerical result is not reported in the registry record.
3. Trial Design
This was a completed Phase I/II, multicenter, open-label study sponsored by Yale University. The registry lists a start date of 2006-12 and primary completion in 2009-12. Its title and stated aims describe a dose-escalation investigation of PHY906 plus capecitabine in patients with advanced pancreatic carcinoma, with attention to safety and efficacy.
The study is classified as single group. Allocation is listed as NA and masking as NONE, so the design does not include a randomized comparison or blinded assignment. The listed intervention arm describes PHY906 800mg orally twice a day for days 1-4 and capecitabine 1500mg/m^2 on days 1-7 of a 14-day cycle. The registry does not report which dose levels were tested, how escalation proceeded, or whether every participant received the listed regimen.
Enrollment is recorded as 25. The primary-outcome population description separately identifies 20 subjects who received 2 cycles or more and were evaluated for response. The record does not give a flow of participants through treatment and follow-up or explain the relationship between these population descriptions for each outcome.
A single-group study can describe outcomes among treated participants, but it cannot by itself distinguish treatment-associated changes from the course of disease or other influences. This is especially important when reading efficacy outcomes: without a concurrent comparison group, differences from another treatment cannot be estimated within this design. The registry record does not report a statistical analysis plan, a sample-size justification, or details of dose-escalation decisions. The study status is COMPLETED, but completion status alone does not supply numerical outcome results.
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 | PHY906 Administration |
|---|---|
| STARTED | 25 |
| COMPLETED | 20 |
| NOT COMPLETED | 5 |
Reasons for not completing this period:
| Reason | PHY906 Administration |
|---|---|
| Withdrawal by Subject | 5 |
Analysis population for the primary outcome, as defined in the registry: 20 subjects received 2 cycles or more and were evaluated for response
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 | PHY906 Administration |
|---|---|---|
| Participants analysed | 25 | |
| Age, Continuous | years | 64 (45 to 84) |
| Sex: Female, Male: Female | Participants | 10 |
| Sex: Female, Male: Male | Participants | 15 |
6. Primary Endpoint
The registry lists the primary outcome as Median Progression Free Survival, measured over Up to 100 weeks.
| Element | Registry entry |
|---|---|
| Result type | Median |
| Measure of spread | Full Range |
| Unit | weeks |
| Endpoint classification | Time-to-Event Analysis |
The primary endpoint is median progression-free survival, measured in weeks. Progression-free survival is a time-to-event measure: it concerns the time until disease progression or another event defined for the analysis. The registry describes progression using RECIST v1.0: a 20% increase in the sum of the longest diameter of target lesions, a measurable increase in a non-target lesion, or the appearance of new lesions. The stated timeframe is up to 100 weeks.
A median is the point at which half of the observed time-to-event distribution lies on either side, under the relevant analysis definition. In general, this summary can be useful because it describes the middle of a time-to-event distribution without requiring that the distribution be symmetric. Follow-up may differ between participants, so time-to-event analyses generally need to account for participants whose event status is not observed through the full follow-up period. The registry record does not report how censoring or incomplete follow-up was handled.
The endpoint is relevant to the study's stated interest in efficacy, but the record does not report the median progression-free survival or its full range. The registered dispersion measure is full range, yet the range values are not reported in the registry record. No confidence interval or other estimate of precision for the median is reported there either.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record describes a completed, single-group study of PHY906 plus capecitabine, with median progression-free survival as its primary outcome. It does not report the numerical median, the full range, or a confidence interval for that endpoint. Consequently, the record does not allow a reader to quantify the observed progression-free survival or its precision. It also does not report a numerical median overall survival, which is named as a secondary outcome.
The design matters for interpretation. Because there was no randomized comparator, the outcomes cannot be read as an estimate of how this combination compares with another treatment. Outcomes observed in one treated group do not isolate the effect of treatment from other influences on disease course. The registry's status of COMPLETED indicates study status, not that a particular efficacy result was achieved.
The primary-outcome description identifies 20 subjects who received 2 cycles or more and were evaluated for response, while the enrollment field reports 25. These are different descriptions in the record. It does not provide a participant flow or explain how outcomes were analyzed for all enrolled participants, so the difference should not be interpreted as a stated reason for non-inclusion or as a specific missing-data pattern.
The safety fields report 25 participants at risk for serious adverse events and 0 recorded as affected. They also report 25 at risk for other adverse events and 10 recorded as affected. No serious adverse-event terms are listed, and deaths are not reported in the registry record. These figures describe registry entries; they do not provide event-level detail, timing, severity breakdowns, or a causal assessment.
The record contains no sponsor-reported hazard ratio or numerical time-to-event analysis result. The number of analyses is recorded as 0. Without reported effect estimates and uncertainty measures, efficacy cannot be assessed quantitatively from this record. The available details support describing the trial's design, endpoint definition, stated analysis population, and recorded safety counts, but not drawing a numerical conclusion about treatment effects.
9. Statistical Methods Explained
In general, a clinical endpoint is a defined outcome used to assess what happens to participants during a study. This trial's primary endpoint is median progression-free survival, a time-to-event outcome. Its interpretation depends on how progression and follow-up are defined. The registry uses RECIST v1.0 criteria for progression: a 20% increase in the sum of the longest diameter of target lesions, a measurable increase in a non-target lesion, or the appearance of new lesions. The record states a timeframe of up to 100 weeks but does not provide the observed endpoint result.
In general, time-to-event data differ from outcomes measured at a single visit. Participants may have different lengths of observation, and some may not have a recorded event before follow-up ends. Statistical methods for such outcomes commonly retain information about follow-up up to the last known observation while accounting for event status. The registry record does not state how censoring, missed assessments, withdrawals, or other incomplete observations were handled. It also does not report a Kaplan-Meier estimate, a confidence interval, or another numerical summary beyond naming the median and full range as the endpoint parameter and dispersion.
A median is a location summary, while a full range describes the smallest and largest observed values under the analysis definition. In general, neither by itself describes every feature of a time-to-event distribution. A confidence interval, when reported, conveys uncertainty around an estimate under the model and analysis assumptions; it is not a guarantee that the true value falls within that interval. No confidence interval is reported in the registry record, so the precision of the primary estimate cannot be assessed from the available entry.
The study is identified as single group, with no masking and allocation listed as NA. In general, random assignment to a concurrent control can help balance measured and unmeasured factors between groups, while masking can reduce some assessment or reporting influences. Neither feature is listed for this study. A single-group result therefore describes outcomes within the treated study population and does not provide a randomized estimate of a difference between treatments. This does not invalidate descriptive reporting; it limits the causal comparisons that the design can support.
The study is Phase I/II and its title describes dose escalation. In general, early-phase dose-escalation studies may examine safety while evaluating dose choices, alongside other objectives. The registry's summary states an aim of determining how much capecitabine could be given safely with PHY906 on a novel schedule and assessing effects of the combination. However, the record does not provide the dose-escalation method, decision rules, dose-limiting toxicity definition, or actual dose-level results. Those details must not be inferred from the title or the listed regimen.
The registry describes an analysis population of 20 subjects who received 2 cycles or more and were evaluated for response. In general, specifying an analysis population matters because estimates depend on who is included and how participants with incomplete follow-up are treated. The record does not explain whether this population applies to the primary time-to-event endpoint, nor does it give a separate analysis population for overall survival or safety. The enrollment field reports 25, but the record does not provide a full participant disposition, so the reason for the difference between these stated population figures is not reported in the registry record.
The posted planning assumptions describe illustrations using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the effect to detect expressed as a fraction of the observed effect. In general, such planning scenarios ask what study size might be needed under stated assumptions; they are not measures of power observed in the completed study. They should not be mistaken for the design actually used: the registry classifies this trial as single group, not as a two-arm randomized comparison. The assumptions also specify that time-to-event planning uses total event counts, while other planning uses participants per arm. No numerical planning estimates or observed effect size are reported in the registry record.
In general, multiplicity arises when several outcomes or statistical comparisons are evaluated, because repeated testing can increase the chance of a chance finding. The record names median progression-free survival as primary and median overall survival as secondary, and lists 2 outcome measures. It does not state whether any multiplicity adjustment was planned or applied. The number of analyses is recorded as 0, and the registry does not report a detailed statistical plan, sponsor-reported hazard ratios, or numerical test results. As a result, there is no basis in the record for reconstructing inferential analyses.
10. Multiplicity, Missing Data and Analysis Populations
The registry names median progression-free survival as the primary outcome and median overall survival as a secondary outcome. It lists 2 outcome measures, but the multiplicity strategy is not reported in the registry record. In general, when more than one outcome or comparison is examined, a prespecified approach can clarify how findings are interpreted and whether adjustment is used. The record does not say whether any adjustment was planned or performed.
The number of analyses is recorded as 0. No detailed statistical analysis plan, outcome-specific test results, or sponsor-reported hazard ratios are reported. Accordingly, the record does not establish which inferential procedures were intended, how any model assumptions were assessed, or whether analyses were changed after data collection.
The primary-outcome population is described as 20 subjects who received 2 cycles or more and were evaluated for response. Enrollment is reported as 25. The registry does not give participant-flow details or say how the primary time-to-event analysis treated people outside the stated response-evaluation population. It also does not report how missing assessments, withdrawal, loss to follow-up, or censoring were handled. These details are important because incomplete follow-up can affect the amount of information available for a time-to-event estimate.
The record does not identify a separate analysis population for the secondary outcome or safety summaries. It also does not report imputation methods or sensitivity analyses. Therefore, the available entry is insufficient to determine whether missing observations could have influenced any outcome estimate.
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 |
|---|---|---|---|
| PHY906 Administration | 0 | 25 | 0.0% |
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 |
|---|---|
| PHY906 Administration | 10 of 25 |
Most frequent non-serious adverse event terms
| Event term | Organ system | PHY906 Administration |
|---|---|---|
| Anorexia | Gastrointestinal disorders | 8 of 25 |
| Fatigue | General disorders | 7 of 25 |
| Hand-Foot Syndrome | General disorders | 5 of 25 |
| Mucositis | Skin and subcutaneous tissue disorders | 4 of 25 |
| Diarrhea | Gastrointestinal disorders | 4 of 25 |
| Anemia | Blood and lymphatic system disorders | 3 of 25 |
| Neutropenia | Blood and lymphatic system disorders | 3 of 25 |
| Thrombocytopenia | Blood and lymphatic system disorders | 3 of 25 |
| Nausea/vomiting | Gastrointestinal disorders | 3 of 25 |
| Constipation | Gastrointestinal disorders | 3 of 25 |
The registry records 25 participants at risk for serious adverse events and 0 recorded as affected. It lists no serious adverse-event terms. For other adverse events, it records 25 participants at risk and 10 affected. These figures are counts of participants recorded in the safety fields; the record does not give event-level descriptions, timing, severity categories, or duration.
The death field is null, so deaths are not reported in the registry record. That should not be read as a report of zero deaths. Likewise, a recorded value of 0 for participants affected by serious adverse events describes the registry entry, not a guarantee that every possible event was captured or that there were no safety concerns of other kinds. The record does not report an attribution assessment linking events to study treatment.
In general, safety counts are interpreted in light of how events were defined, collected, and coded, and the number of participants observed. The registry does not provide those collection details or a breakdown by event type. The safety figures therefore support only a limited description of what is recorded, not a comprehensive characterization of the combination's safety.
12. What the Result Does, and Does Not, Mean
This registry record documents a Phase I/II, multicenter, open-label, single-group study of PHY906 plus capecitabine in advanced pancreatic carcinoma. It specifies the primary endpoint and its RECIST-based progression definition, gives the named response-evaluation population, and records selected safety counts. These details describe the study question and how some outcomes were framed.
The record does not report the numerical median progression-free survival, its full range, a confidence interval, or a numerical median overall survival. It also does not report hazard ratios or other efficacy estimates. Without those results, the magnitude and precision of the outcomes cannot be characterized from the registry entry. Completion status is not itself an outcome finding.
Even if numerical outcomes were available, the single-group design would not establish a randomized comparison against another treatment. It can describe what was observed among participants in the study, but it cannot by itself determine what would have happened to a comparable group under an alternative treatment. The absence of masking and a concurrent comparator are features of the registered design, not evidence for or against a particular result.
The safety entries record 0 participants affected by serious adverse events among 25 at risk, and 10 affected by other adverse events among 25 at risk. These entries should be read within the limits of the registry's reporting: serious-event terms are not listed, event details are absent, and deaths are not reported in the registry record. The counts do not establish treatment causality or describe every aspect of safety.
Overall, the registry supports a careful account of the study's purpose, design, endpoint definition, population descriptions, and recorded safety fields. It does not provide enough outcome statistics to quantify efficacy or assess precision, and it does not support a comparative treatment-effect conclusion.
13. Why This Trial Matters Statistically
This trial is statistically informative as an example of how design determines the questions an outcome can answer. Its primary outcome is a time-to-event measure, median progression-free survival, defined using explicit RECIST v1.0 progression criteria. That specificity matters: an endpoint is interpretable only when the event definition and relevant follow-up framework are clear.
The study is also classified as single group, with no masking. This makes the distinction between descriptive evidence and comparative evidence central. An outcome among treated participants can be summarized, but without a concurrent randomized comparator the study cannot estimate a randomized contrast between treatment strategies. The design should shape the claims drawn from any results.
The entry illustrates why numerical estimates and measures of uncertainty are both important. It names a median and full range but does not report their values, and it does not provide a confidence interval. As a result, readers cannot assess the observed outcome level or its precision from the registry record. A completed status does not replace those statistics.
The participant descriptions also show why analysis populations and follow-up accounting matter. The record reports enrollment of 25 and identifies 20 subjects who received 2 cycles or more and were evaluated for response, but does not give participant flow or explain how the population descriptions relate across outcomes. Without details on censoring and missing data, the analysis basis remains unclear.
Finally, planning assumptions are not the same as observed evidence. A planning illustration based on an assumed effect and allocation cannot be treated as power observed in this single-group study. Keeping design, analysis, uncertainty, and reporting separate is essential to a sound statistical reading.
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 provides limited numerical outcome information. Although median progression-free survival is identified as primary, its value and full range are not reported in the registry record. The median overall survival is named as secondary, but its result is also not reported. No confidence intervals, sponsor-reported hazard ratios, or outcome test statistics are reported, preventing assessment of effect size and precision.
The single-group, open-label design has no randomized comparator and no masking. Consequently, the study does not support a like-for-like treatment comparison, and any observed outcome would need to be interpreted as a description of the study group rather than a controlled estimate of treatment difference.
The registry reports enrollment of 25 and a primary-outcome description involving 20 subjects who received 2 cycles or more and were evaluated for response, but does not give a participant flow or explain how those populations were used for each endpoint. It does not report handling of missing data, censoring, withdrawals, or incomplete assessments. The relation between response evaluation and the time-to-event analysis is therefore not clear from the entry.
For the dose-escalation component, the record does not report escalation rules, tested dose levels, or dose-level safety results. For safety, serious-event terms are absent, event details are not given, and deaths are not reported in the registry record. The statistical analysis plan and multiplicity approach are also not reported. These omissions limit interpretation of both the efficacy and safety information recorded.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: A Phase I/II, Multi-Center, Open-Label, Dose-Escalation, Safety and Efficacy Study of PHY906 Plus Capecitabine in Patients With Advanced Pancreatic Carcinoma — NCT00411762.
- Registry record last updated 2015-03-27; status last verified 2015-03.
- Results first posted 2015-03-27.
- 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
NCT00411762 is a completed Phase I/II, multicenter, open-label, dose-escalation study sponsored by Yale University. It enrolled 25 participants and studied PHY906 plus capecitabine in advanced pancreatic carcinoma. The listed regimen describes PHY906 800mg orally twice a day for days 1-4 and capecitabine 1500mg/m^2 on days 1-7 of a 14-day cycle.
The primary outcome was median progression-free survival, measured in weeks, with progression defined by RECIST v1.0 and follow-up stated as up to 100 weeks. Median overall survival was named as a secondary outcome. The record identifies 20 subjects who received 2 cycles or more and were evaluated for response. Numerical efficacy results are not reported in the registry record.
The study was single group, with allocation listed as NA and masking as NONE. Safety fields record 0 participants affected by serious adverse events and 10 affected by other adverse events, each among 25 at risk. Serious-event terms are not listed, and deaths are not reported in the registry record.