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 Sorafenib for Patients With Metastatic or Recurrent Esophageal and Gastroesophageal Junction Cancer (NCT00917462), a na phase 2 study. The primary outcome is 2-month Progression-free Survival (PFS) of Sorafenib in Patients With Metastatic or Recurrent Esophageal and Gastroesophageal (GE) Junction Cancer..
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00917462 |
| Phase | Phase 2 |
| Condition | Esophageal Cancer, Gastroesophageal Junction Cancer |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 35 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Memorial Sloan Kettering Cancer Center |
| Results posted | Yes |
2. Clinical Question
Population
Patients with metastatic or recurrent esophageal cancer or gastroesophageal junction cancer. The registry record gives an enrollment of 35.
Intervention
Sorafenib, administered orally. CT/MRI is also listed as a study procedure.
Comparator
There is no separate comparator group. The study is described as single-group, with allocation listed as not applicable.
Primary question
How many participants were progression-free at the 2-month assessment while receiving sorafenib? The registry record names 2-month progression-free survival as the primary outcome but does not report the outcome count or estimate.
The study was intended to examine the effects of sorafenib in advanced esophageal and gastroesophageal junction cancer. The registry summary describes questions about tumor shrinkage or slowing of tumor growth, as well as side effects. The listed primary outcome focuses more narrowly on progression-free survival at a stated assessment time.
Because the design is single-group, the central descriptive question is what happened among the enrolled participants during the study. There is no randomized control group in the registry record for estimating how outcomes would have differed under another treatment or under no study treatment. The registry also lists overall response rate, adverse events, exploratory response differences between squamous cell carcinoma and adenocarcinoma, and tumor expression of phosphorylated extracellular signal-regulated kinase among the secondary outcomes.
The clinical question can therefore be framed as an assessment of outcomes and safety observations in a specific enrolled group, rather than a randomized comparison of treatments. The registry record does not report the primary result or enough analysis details to establish how the endpoint was defined or assessed in practice.
3. Trial Design
This is a completed Phase II study with a single-group model. The registry record lists an enrollment of 35 and identifies Memorial Sloan Kettering Cancer Center as the sponsor. The study began in 2009-06, reached primary completion in 2018-08, and has completed status.
The sole listed treatment arm is Sorafenib, described as sorafenib for patients with metastatic or recurrent esophageal and gastroesophageal junction cancer. The drug intervention is oral sorafenib. CT/MRI is listed separately as a procedure. Masking is listed as none, and allocation is not applicable. These details indicate that the registry does not describe a masked, randomized comparison between treatment groups.
The primary outcome is 2-month progression-free survival, expressed as a count of participants. The listed secondary outcomes cover overall response rate, adverse events, exploratory response differences between squamous cell carcinoma and adenocarcinoma, and the percentage of tumors with high phosphorylated extracellular signal-regulated kinase expression. The registry lists five outcome measures in total.
A single-group Phase II design can describe outcomes observed in the enrolled group and may provide information for further study. It cannot, by itself, separate treatment effects from the natural course of disease, participant characteristics, or other influences. No randomized control arm is listed, so a like-for-like treatment contrast is not available from this design.
The registry record does not report the protocol’s sample-size justification, the planned statistical analysis, or details such as eligibility criteria, dosing schedule, or follow-up procedures. Those details should not be inferred from the study title or the listed outcomes.
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 | Sorafenib |
|---|---|
| STARTED | 35 |
| COMPLETED | 34 |
| NOT COMPLETED | 1 |
Reasons for not completing this period:
| Reason | Sorafenib |
|---|---|
| Clinical Deterioration | 1 |
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 | Sorafenib |
|---|---|---|
| Participants analysed | 35 | |
| Age, Continuous | years | 62 (42 to 77) |
| Sex: Female, Male: Female | Participants | 4 |
| Sex: Female, Male: Male | Participants | 31 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 1 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 34 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 3 |
| 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 | 31 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 1 |
| Region of Enrollment: United States | Participants | 35 |
6. Primary Endpoint
The registry lists the primary outcome as 2-month Progression-free Survival (PFS) of Sorafenib in Patients With Metastatic or Recurrent Esophageal and Gastroesophageal (GE) Junction Cancer., measured over 2 months.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is titled 2-month progression-free survival and is recorded as a count of participants. In general, progression-free survival concerns the time a participant remains alive without disease progression. A fixed-time assessment asks how many participants meet the endpoint at the specified time. The registry record identifies the timeframe as 2 months, but it does not report the resulting count or proportion.
Progression-free survival is used in cancer studies because it captures disease progression as well as death, rather than focusing only on tumor response. It can therefore describe whether participants remain free of a defined progression event over a stated period. This is a general description of the endpoint concept, not a statement about the study’s specific operational rules.
The registry record does not report the progression criteria, the assessment schedule, how death was handled, or how participants without an assessment at the specified time were treated in the analysis. It also does not give a statistical estimate or confidence interval for the primary outcome. Without those details and the outcome result, the endpoint’s observed level and precision cannot be evaluated from the registry record.
The count format is distinct from a time-to-event summary that incorporates the timing of events throughout follow-up. The record’s stated primary outcome is a participant count at 2 months; it does not provide a fuller account of the analysis method used to obtain that count.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry identifies a completed, single-group Phase II study with an enrollment of 35. Its primary outcome is 2-month progression-free survival, recorded as a count of participants. However, the registry record does not report the primary outcome result. There is therefore no posted estimate in this record from which to describe the observed number of participants who were progression-free at the specified time or the precision of that result.
The design also limits what can be concluded from any within-group outcome. With no separate comparator arm, an observed outcome would describe participants in the sorafenib group but would not establish how the same participants would have fared under another treatment. Allocation is listed as not applicable, masking as none, and the model as single-group. The registry explicitly notes that this is not a randomized comparison and that arms are not a like-for-like control comparison.
The registry lists secondary outcomes including overall response rate, adverse events, exploratory response differences by histology, and tumor expression of phosphorylated extracellular signal-regulated kinase. Their inclusion indicates areas the study intended to assess, but the record does not provide the outcome findings or analysis details for these questions. The registry lists no analyses. Statistical significance, effect estimates, uncertainty intervals, and adjustment for multiple outcomes are not reported in the registry record.
The adverse-event summary contains participant counts and several serious-event terms with percentages. These are safety observations within the study group, not comparative estimates of excess risk. The record does not establish whether individual events were attributed to sorafenib, how long participants were followed for safety, or how event severity and relatedness were classified. The listed deaths should not be interpreted as evidence of cause or timing without additional information.
A planning illustration may appear alongside the registry information, but it should not be read as observed trial evidence or as the trial’s original sample-size rationale. The stated assumptions describe a planning calculation based on an observed effect; the registry record does not report the study’s own design justification. Such calculations cannot replace a randomized comparison or supply a missing primary outcome.
Overall, the registry supports a description of the study’s design, enrolled group, endpoints, and some adverse-event counts. It does not support a quantitative conclusion about the primary outcome, comparative efficacy, or the precision of an efficacy estimate. The absence of posted results is a limit on interpretation, not evidence that the endpoint did or did not favor the intervention.
9. Statistical Methods Explained
Statistical interpretation depends on both the design and the outcome definition. This study is listed as single-group, with no masking and no applicable allocation. That design can describe outcomes among participants who received the study intervention, but it does not provide a randomized comparison with another group. Any statistical method applied to a single group would still be constrained by the absence of a concurrent control.
What does a participant count tell the reader?
The primary endpoint is recorded as a count of participants with 2-month progression-free survival. A count is a direct summary of how many people met the outcome definition in the analyzed group. To interpret that count, readers would also need to know the analysis population, the number assessed, and how missing endpoint status was handled. Those details are not reported in the registry record. The record also does not give the outcome count itself.
In general, a count alone does not express uncertainty as fully as an estimate accompanied by an interval. A confidence interval describes a range of values compatible with the data and the method used, subject to its assumptions. It can help show how precise an estimate is. No confidence interval for the primary endpoint is reported in the registry record, so no precision statement can be made for that outcome.
Why does the single-group design matter?
In a randomized comparison, random allocation is intended to make treatment groups comparable, on average, for factors that affect outcomes. Here, the registry describes a single group and no randomized comparator. An outcome observed during the study could reflect the intervention, the underlying disease, participant characteristics, other care, or a combination of influences. Because there is no concurrent comparison group, the registry record cannot isolate the intervention’s contribution from those influences.
Blinding can also affect how outcomes are assessed or reported, depending on the endpoint. Masking is listed as none. The record does not specify whether the primary endpoint involved independent review or other safeguards against assessment bias. The absence of masking does not by itself establish that an assessment was biased, but it is relevant context when considering subjective judgments and reporting.
What can be learned from the planned sample-size illustration?
The stated sample-size assumptions describe a planning illustration based on the 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 described as a stated fraction of the observed effect. Those assumptions are not a description of this trial’s single-group design. The registry record does not report the trial’s own sample-size justification, nor does it provide the endpoint result needed to assess the observed effect. Any illustrative sample-size figures should be understood as planning scenarios, not as observed power or evidence that the study could detect a particular effect.
In general, power is a design property tied to specified assumptions about the effect, variability, sample size, and analysis. It is not a retrospective measure of whether a result is true. A calculation based on an observed effect can be especially dependent on that estimate and should not be interpreted as proof that a study had adequate or inadequate power. The registry record does not provide a planned power calculation for this trial.
Why can over-dispersion matter?
General statistical explanation: over-dispersion occurs when count data vary more than a model assumes. If a model underestimates that variability, its uncertainty estimates can be too narrow and tests can appear more decisive than warranted. This issue is relevant to some count outcomes, but the primary endpoint is recorded as a participant count and the registry record does not report a count model, a dispersion estimate, or an analysis method. It would therefore be inappropriate to assert that over-dispersion was present or that a particular correction was used.
What analysis details are missing?
The registry record does not report the planned statistical model, the analysis population, the handling of missing observations, censoring rules, or whether any interim analyses were planned or conducted. It lists no analyses. The record also does not provide a primary endpoint estimate, a confidence interval, or a p-value. These omissions prevent assessment of the analysis choices and their influence on the reported evidence.
General principle: statistical methods should match the outcome type and study design, and results should be presented with enough information to assess their uncertainty and assumptions. For a single-group endpoint, a clear definition of who was included and how incomplete follow-up was handled is particularly important. Without those details, the registry information supports a description of the intended endpoint and design, but not a reconstruction or independent evaluation of the primary analysis.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists a primary outcome and several secondary outcomes. The secondary outcomes include overall response rate, adverse events, exploratory response differences between squamous cell carcinoma and adenocarcinoma, and the percentage of tumors with high phosphorylated extracellular signal-regulated kinase expression. The record lists five outcome measures in total. Considering multiple outcomes can raise the chance of finding an apparently notable result by chance, particularly when many comparisons are tested. This is a general statistical concern, not evidence that a particular finding in this study is false.
The registry record does not report a multiplicity strategy, such as a prespecified hierarchy among outcomes or an adjustment to account for multiple testing. It lists no analyses and provides no outcome estimates or p-values. It is therefore not possible to determine how statistical testing was conducted or whether any findings were adjusted for the number of outcomes examined.
Missing-data handling is also not reported in the registry record. The record does not state how participants without an assessment at 2 months were treated, whether any participants were excluded from the primary analysis, or what analysis population was used. For a progression-free survival endpoint, the rules for event assessment and incomplete follow-up can affect interpretation; those rules are not available in the registry information.
The registry record does not report whether interim analyses were planned or conducted. No interim-analysis procedure or stopping rule should be inferred. In the absence of the statistical analysis plan and reported results, the primary and secondary outcomes can be described as registered questions, but their analysis, missingness, and multiplicity cannot be independently evaluated.
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 |
|---|---|---|---|
| Sorafenib | 15 | 35 | 42.9% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Sorafenib | 35 of 35 |
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 |
|---|---|
| Sorafenib | 34 of 35 |
Most frequent serious adverse event terms
| Event term | Organ system | Sorafenib |
|---|---|---|
| Dehydration | Metabolism and nutrition disorders | 4 of 35 |
| Confusion | Psychiatric disorders | 2 of 35 |
| Fatigue | General disorders | 2 of 35 |
| Fistula - GI, Esophagus | Gastrointestinal disorders | 2 of 35 |
| Motor neuropathy | Nervous system disorders | 2 of 35 |
| Hyperbilirubinemia | Investigations | 1 of 35 |
| Hypercalcemia | Metabolism and nutrition disorders | 1 of 35 |
| Constipation | Gastrointestinal disorders | 1 of 35 |
| Constitutional Symptoms, Other | General disorders | 1 of 35 |
| Cough | Respiratory, thoracic and mediastinal disorders | 1 of 35 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Sorafenib |
|---|---|---|
| Fatigue | General disorders | 30 of 35 |
| Diarrhea | Gastrointestinal disorders | 18 of 35 |
| Nausea | Gastrointestinal disorders | 12 of 35 |
| Dermatology/Skin - other | Skin and subcutaneous tissue disorders | 10 of 35 |
| Albumin, low | Metabolism and nutrition disorders | 9 of 35 |
| Anorexia | Metabolism and nutrition disorders | 9 of 35 |
| Hemoglobin/Anemia | Blood and lymphatic system disorders | 9 of 35 |
| Dry Skin | Skin and subcutaneous tissue disorders | 8 of 35 |
| Rash: hand-foot skin reaction | Skin and subcutaneous tissue disorders | 8 of 35 |
| Hyperglycemia | Metabolism and nutrition disorders | 7 of 35 |
Serious events with the highest arm-level rate: dehydration (11.4%), confusion (5.7%), fatigue (5.7%), fistula - gi, esophagus (5.7%), motor neuropathy (5.7%).
The registry adverse-event summary lists 35 participants at risk in the Sorafenib group. It records 15 participants with serious adverse events and 34 participants with other adverse events. It also records 35 deaths among 35 at risk. These are the posted counts; the record does not describe the timing or cause of the deaths. The counts should not be taken to show that sorafenib caused them.
The listed serious-event terms and percentages are dehydration, 11.4%; confusion, 5.7%; fatigue, 5.7%; fistula - GI, Esophagus, 5.7%; motor neuropathy, 5.7%; and hyperbilirubinemia, 2.9%. These are the terms identified in the registry’s serious-event summary. The record does not give participant counts for each term, clarify whether participants could have more than one event, or describe the event definitions and observation period.
Adverse-event counts are descriptive safety observations. Without a comparator group, they do not estimate how event frequency differs from another treatment or from background illness. The registry record also does not report event relatedness, grading details, or the criteria used to classify seriousness. The percentages should therefore be read as posted summaries, not as causal or comparative risk estimates.
12. What the Result Does, and Does Not, Mean
The registry record describes a completed Phase II, single-group study of oral sorafenib in patients with metastatic or recurrent esophageal and gastroesophageal junction cancer. Its primary question concerns 2-month progression-free survival. The record does not report the primary outcome result, so it cannot establish how many participants met that endpoint or how precise an estimate might be.
The study’s design is important to the meaning of its observations. With no randomized comparator group, outcomes in the sorafenib group cannot be directly contrasted with outcomes under another treatment. Even if a within-group outcome had been posted, the design would not by itself determine whether the intervention caused that outcome. The registry record also does not supply the planned analysis details needed to evaluate endpoint definitions, missing data, or uncertainty.
The adverse-event summary documents reported safety observations, including serious-event counts, named serious-event terms, and deaths. Those figures are not comparative estimates and do not establish that the intervention caused any particular event. The registry information does not provide event attribution, timing, or the full context needed to interpret individual safety outcomes.
The record lists several secondary questions, including response and exploratory tumor-characteristic analyses. Their presence does not establish that results were observed or reported for those questions. The registry record lists no analyses and does not provide estimates or statistical tests for these outcomes.
These limitations do not imply that the intervention was ineffective or that it was effective. They define what can be concluded from the posted registry information: the study design and intended outcomes can be described, and certain adverse-event summaries can be quoted, but the primary efficacy result and comparative treatment effect are not reported in the registry record.
13. Why This Trial Matters Statistically
This study illustrates why statistical interpretation begins with design rather than with an outcome label. The registry calls the primary endpoint 2-month progression-free survival, but the model is single-group and there is no randomized comparator. A registered endpoint can define the question a study intends to address; it does not, on its own, provide an answer or create a treatment contrast.
The distinction between a count and an effect estimate is also important. The primary outcome is expressed as a participant count, yet the registry record does not report the count or an uncertainty interval. Without those, readers cannot assess the observed endpoint level or its precision. Even a fully reported within-group count would describe the enrolled group, not the difference attributable to treatment.
The study also demonstrates the importance of analysis transparency. The registry lists multiple outcome areas, but does not report analyses, a multiplicity approach, missing-data rules, or the primary endpoint result. Such information is needed to understand how registered questions were translated into statistical findings and how much confidence to place in those findings.
Finally, the safety summary requires a different interpretation from an efficacy comparison. Counts and percentages can identify events recorded among participants, but without a control group and details about event attribution, they do not establish comparative harm or causation. The study is statistically informative as an example of how design, endpoint reporting, and analysis transparency constrain interpretation; the posted registry information is not enough to quantify the primary clinical outcome.
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 is that the registry record does not report the primary outcome result. Although the endpoint and timeframe are named, no count, estimate, or uncertainty interval is available. This prevents assessment of the observed 2-month progression-free survival outcome.
The single-group design offers no randomized control group for a like-for-like comparison. Consequently, outcomes within the sorafenib group cannot isolate the effect of the intervention from other influences. The record lists no allocation method beyond not applicable and no masking.
Important endpoint and analysis details are also not reported in the registry record. These include the operational definition of progression, assessment procedures, analysis population, missing-data handling, and any rules for incomplete follow-up. The record does not report a statistical analysis plan, sample-size justification, interim-analysis plan, or multiplicity procedure. These omissions limit reproducibility and appraisal of the intended and actual analyses.
The safety information is limited to summary counts and selected serious-event terms with percentages. The registry record does not provide the event timing, attribution, grading details, or full context needed to interpret causality. The recorded deaths cannot be characterized by cause or timing from the information reported.
Finally, the registry lists secondary outcomes but no analyses or corresponding results. Findings for response, histology-related differences, and tumor expression therefore cannot be evaluated from this record. These limitations concern the available registry information and design; they do not establish a direction of effect.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Sorafenib for Patients With Metastatic or Recurrent Esophageal and Gastroesophageal Junction Cancer — NCT00917462.
- Registry record last updated 2019-03-27; status last verified 2018-08.
- Results first posted 2019-03-13.
- 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
The registry record describes NCT00917462, a completed Phase II, single-group study sponsored by Memorial Sloan Kettering Cancer Center. It enrolled 35 participants with metastatic or recurrent esophageal or gastroesophageal junction cancer. Oral sorafenib was the listed drug intervention, and CT/MRI was listed as a procedure. The study began in 2009-06 and reached primary completion in 2018-08.
The primary outcome was 2-month progression-free survival, recorded as a participant count. Secondary outcome areas included overall response rate, adverse events, exploratory response differences by histology, and tumor expression. The registry record does not report the primary endpoint result or statistical analyses. Its adverse-event summary records serious adverse events in 15 participants, other adverse events in 34 participants, and 35 deaths among 35 at risk; these observations are not comparative or causal estimates.