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 in Treating Patients With Locally Advanced or Metastatic Kidney Cancer (NCT00727532), a na na study. The primary outcome is Percentage Change in Difference in Apparent Diffusion Coefficient Between Baseline and Week 5.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00727532 |
| Phase | NA |
| Condition | Hereditary Clear Cell Renal Cell Carcinoma, Kidney Cancer |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 9 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Northwestern University |
| Results posted | Yes |
2. Clinical Question
Population
Patients with locally advanced or metastatic clear cell renal cell carcinoma were the intended population. The registry record also lists hereditary clear cell renal cell carcinoma and kidney cancer as conditions. The enrollment was 9. Further eligibility details are not reported in the registry record.
Intervention
Patients received sorafenib 400mg orally twice daily on days 1-28. They underwent abdominal and pelvic diffusion-weighted MRI before treatment and a second scan after completing 28 days.
Comparator
There was no separate comparator group: the study is recorded as a single-group trial. The allocation field is NA, and the study was not masked. It therefore does not provide a randomized, like-for-like treatment comparison.
Primary question
The primary question was whether the apparent diffusion coefficient, or ADC, changed from baseline to week 5 during neoadjuvant sorafenib treatment. ADC is derived from MRI measurements of water mobility in tissue. The registry record describes the outcome as percent change and says the value was calculated for each participant. A numerical primary result is not reported in the registry record.
The study rationale describes sorafenib as a treatment that may affect tumor growth and blood flow, and proposes treatment before surgery as a way to alter the tumor. Those statements are the rationale, not findings from this trial. The registry record does not report a comparison with another treatment or with an untreated group, so the study question is about change measured within the treated group rather than a comparative treatment effect.
The official title emphasizes correlation of pathologic findings after neoadjuvant sorafenib with diffusion-weighted MRI results. The posted primary outcome, however, is the percentage change in the difference in ADC between baseline and week 5. How the pathology and MRI measures were analyzed together is not reported in the registry record.
3. Trial Design
NCT00727532 is recorded as an interventional study with a single-group model and no masking. The allocation field is NA, and the phase is listed as NA. Enrollment was 9. The sponsor was Northwestern University. The study began in 2008-07, had primary completion in 2011-10, and its status is TERMINATED.
The intervention was sorafenib. The arm description says eligible patients underwent pretreatment diffusion-weighted MRI of the abdomen and pelvis, then received sorafenib 400mg orally twice daily on days 1-28. After completing 28 days of treatment, patients obtained a second diffusion-weighted MRI. The primary outcome timeframe is baseline and week 5. The record does not report the interval between the end of dosing and each participant’s second scan beyond the description of obtaining it after the 28-day course.
Because this is a single-group design, the primary outcome is interpreted as a before-and-after measurement in participants receiving sorafenib. There is no separate control arm against which to compare changes, and the study was not masked. The record does not describe a randomization procedure, and the allocation field is NA. This design can describe observed changes over time in the enrolled group, but by itself it cannot distinguish a treatment-associated change from other explanations for change over time.
The registry record lists three outcome measures and no analyses. It does not provide a statistical analysis plan, a sample-size justification, an interim-analysis plan, or further details on eligibility and follow-up. It also does not state why the study was terminated. These omissions limit what can be concluded about the planned conduct and the reasons for stopping.
The rationale mentions treatment before surgery, but the listed primary outcome is an MRI-derived measure rather than a surgical outcome. A separate analysis relating pathology to MRI findings, as suggested by the official title, is 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 | Sorafenib |
|---|---|
| STARTED | 9 |
| COMPLETED | 7 |
| NOT COMPLETED | 2 |
Reasons for not completing this period:
| Reason | Sorafenib |
|---|---|
| Adverse Event | 1 |
| Withdrawal by Subject | 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 | 9 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 5 |
| Age, Categorical: >=65 years | Participants | 4 |
| Sex: Female, Male: Female | Participants | 4 |
| Sex: Female, Male: Male | Participants | 5 |
| Region of Enrollment: United States | participants | 9 |
| Renal cell carcinoma Clinical Staging: T1>3cm | participants | 0 |
| Renal cell carcinoma Clinical Staging: T2 | participants | 0 |
| Renal cell carcinoma Clinical Staging: T3 | participants | 8 |
| Renal cell carcinoma Clinical Staging: T4 | participants | 1 |
6. Primary Endpoint
The registry lists the primary outcome as Percentage Change in Difference in Apparent Diffusion Coefficient Between Baseline and Week 5, measured over Baseline and week 5.
| Element | Registry entry |
|---|---|
| Result type | Mean |
| Measure of spread | Full Range |
| Unit | Percent change |
| Endpoint classification | not reduced to a single comparison |
The primary outcome is titled percentage change in difference in apparent diffusion coefficient between baseline and week 5. ADC is derived from diffusion-weighted MRI and is described in the registry record as a measure of water mobility. The record says lower ADC values correspond to tumor and higher values are consistent with cysts. It also gives a biological rationale for monitoring ADC: therapy-associated necrosis may increase free water in a lesion, potentially increasing ADC.
For each participant, the record describes calculating the week 5 ADC value minus the baseline ADC value, divided by the baseline ADC value. The outcome is reported as a percent change. The measure is intended to assess whether ADC changes during neoadjuvant sorafenib treatment are detectable in locally advanced or metastatic kidney cancer. It is a tissue-imaging measure, not a direct measure of survival, symptoms, or surgical outcome.
A change in ADC may be useful as an imaging signal of altered tissue water mobility, but interpreting it requires the observed values, their variability, and information about how measurements were obtained. The registry record specifies the measurement concept and timeframe but does not report a numerical primary result, participant-level values, or the precision of the estimated change. It also does not report how any relationship between ADC and pathology was assessed.
The registry record labels the summary parameter as a mean and the dispersion as a full range. Those labels describe the planned summary approach; without posted numerical outcome results, the size and spread of the observed ADC change cannot be assessed.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central interpretive limitation is that the registry record does not report a numerical result for the primary outcome. It describes a mean percent change in ADC between baseline and week 5, with full range as the dispersion measure, but no estimate, range, confidence interval, or p-value is reported. Consequently, the direction, magnitude, and precision of the primary outcome cannot be assessed from the registry record.
The study has enrollment 9 and a single-group design. That context matters when reading any within-group change: a baseline-to-week-5 comparison does not have a concurrent comparison group to indicate what might have happened over the same period without sorafenib or under another treatment. A change in an imaging measure would therefore be descriptive of the observed study group and would not, on its own, establish that sorafenib caused the change.
The primary outcome is a derived measure. It uses ADC at baseline and week 5, with the change expressed relative to baseline. Interpretation of such a measure depends on the underlying ADC measurements and their variability. The registry record does not report those values, details about measurement reliability, or how missing scans or incomplete observations were handled. It is therefore not possible to judge whether a reported group summary, if available elsewhere, would be driven by consistent participant changes or by a small number of observations.
The outcome description supplies a biological rationale for ADC as an imaging signal, but a plausible mechanism is not itself evidence that the measure changed in this study. Nor does an ADC change alone establish a change in pathology, tumor burden, surgical requirements, or patient outcomes. Although the official title refers to correlation with pathologic findings, the registry record does not report an analysis of that relationship.
The study status is TERMINATED. The registry record does not state the reason for termination, whether all planned assessments were completed, or whether the termination affected outcome availability. It also lists three outcome measures but no analyses. Without posted numerical results, readers cannot determine whether the primary outcome was evaluated as planned or whether additional analyses were conducted.
Safety information is also limited to the recorded adverse-event summaries. The serious adverse event information includes a group at risk of 9 and affected of 1, while the listed serious terms are pancreatitis and hand-foot skin reaction, each at 11.1%. These are registry summaries, not evidence that sorafenib caused the events. The record does not provide enough detail to assess timing, severity grades, or individual clinical context.
Overall, the registry record documents the question, treatment schedule, and planned MRI endpoint, but does not provide the primary outcome result needed to describe the observed imaging change. Any conclusion about efficacy, the clinical meaning of an ADC change, or a treatment effect would go beyond the reported results.
9. Statistical Methods Explained
What does a before-and-after endpoint describe?
A before-and-after endpoint compares measurements taken at different times in the same study group. Here, the planned measurements are ADC at baseline and week 5 during the sorafenib course. Such a comparison can describe how the measured quantity changed among participants with observations at both times. It does not by itself provide the counterfactual comparison needed to know what would have happened over that period without the intervention. General statistical interpretation therefore separates observed change over time from a causal treatment effect.
The registry record specifies a mean summary and full-range dispersion. A mean gives a group-level summary of the measured changes, while a range describes the span from the lowest to highest observed value. A range can be strongly influenced by extreme observations and does not show how values are distributed between the endpoints. A confidence interval, if reported, would instead communicate uncertainty about an estimated quantity under the statistical model used; no confidence interval for the primary result is reported in the registry record.
Why does the single-group design matter?
A single-group design has no concurrent comparison arm. In a randomized comparison, random assignment can help make groups comparable on average, allowing a treatment contrast to be estimated with fewer concerns about systematic differences between groups. That structure is absent. A baseline value is not a substitute for a concurrent control because time, measurement conditions, disease course, and other factors may also contribute to a difference between measurements. The registry record does not report a control group, random allocation, or masking.
Within-participant measurements can account for some stable differences between people because each participant’s follow-up measurement is related to their own baseline. They do not remove time-varying influences or measurement error. If some participants lack a follow-up scan, the group represented at week 5 may also differ from the group with baseline measurements. The record does not report missing-scan handling or the analysis population used for the primary outcome.
What does percent change add?
The outcome description states that week 5 ADC minus baseline ADC is divided by baseline ADC for each participant, and the primary outcome is expressed as percent change. This standardizes the change relative to the baseline measurement, but it does not remove the need to examine baseline values or measurement variability. When a change score is calculated separately for each participant and then summarized, the group summary depends on the distribution of those individual changes. The registry record does not report the participant-level values or numerical group summary.
The description uses the phrase “percentage change in difference,” while also specifying the participant-level calculation. That wording does not supply an observed estimate or additional analysis details. The record does not state whether transformations, covariate adjustment, or other modeling approaches were used. Those methods should not be inferred from the endpoint label.
How should uncertainty be assessed?
In general, an estimate is more informative when accompanied by a measure of uncertainty. A confidence interval communicates a range of values compatible with the observed data and the assumptions of the method; it is not a guarantee that the true value lies in that interval. A p-value, when used, describes how unusual data at least as incompatible with a specified null model would be under that model’s assumptions. Neither statistic determines clinical importance by itself. No primary outcome estimate, confidence interval, or p-value is reported in the registry record, so the precision and statistical evidence for a change cannot be evaluated.
The record labels dispersion as full range. That can communicate the observed spread but does not directly quantify uncertainty in the estimated mean. General statistical practice distinguishes variation among observations from uncertainty about a group estimate. The registry record does not report additional distributional summaries or model details.
What does over-dispersion mean in general?
For outcomes such as counts, over-dispersion means that observed variation is greater than a model assumes. If ignored, standard errors can be too small and confidence intervals too narrow. This is a general consideration, not a reported feature of this trial. The primary endpoint is a continuous imaging measure, and the registry record does not report an over-dispersion assessment or a model for the primary outcome.
How do planning illustrations differ from results?
Sample-size calculations are prospective planning illustrations: they describe how many participants might be needed under stated assumptions to target a specified statistical power for an assumed effect. They are not a measure of power observed after a trial has been completed. The registry record’s planning assumptions refer to a two-arm illustration with equal allocation and a normal approximation, and define the effect in relation to a fraction of the observed effect. Those assumptions do not turn this single-group study into a two-arm comparison or establish that they formed the trial’s original design. The trial’s own sample-size justification is not reported in the registry record.
No numerical planning outputs are reported in the registry record. Without an observed primary effect and the relevant assumptions, no planning estimate can establish that this study had adequate precision. In general, planning results depend on the effect size, variability, outcome model, significance threshold, and allocation assumptions. A planning illustration should be read as conditional on those assumptions, not as an observed trial finding.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists three outcome measures and no analyses. It does not report a multiplicity strategy, such as a hierarchy of outcomes or an adjustment for testing multiple hypotheses. When several outcomes or analyses are examined, the chance of at least one apparently unusual finding can increase if each is treated as an independent confirmatory test. This is a general statistical concern; the record does not establish which outcomes were analyzed or whether any adjustment was used.
Missing-data methods and analysis populations are not reported in the registry record. The planned imaging endpoint requires a baseline and week 5 ADC measurement, but the record does not state how participants with a missing scan, incomplete treatment, or unavailable ADC data would be handled. It does not identify whether the primary analysis included all enrolled participants or only those with paired measurements. These details matter because a complete-case summary may describe a different subset from the enrolled group if outcome availability is related to health status, treatment tolerance, or other factors.
The registry record reports enrollment 9, but does not provide participant flow, reasons for missing observations, or the number included in the primary analysis. It also does not report an imputation method, sensitivity analysis, or assumptions about missingness. No interim-analysis plan or stopping rule is reported. The TERMINATED status is recorded, but the reason for termination and its relationship to data completeness are not reported in the registry record.
Without these details, readers cannot determine how robust any outcome estimate would be to missing observations or multiple analyses. This is especially important when interpreting a small single-group study: the information available is limited, and the registry record does not provide the analysis details needed to evaluate that limitation quantitatively.
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 | 1 | 9 | 11.1% |
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 |
|---|---|
| Sorafenib | 9 of 9 |
Most frequent serious adverse event terms
| Event term | Organ system | Sorafenib |
|---|---|---|
| Pancreatitis | Hepatobiliary disorders | 1 of 9 |
| Hand-Foot Skin Reaction | Skin and subcutaneous tissue disorders | 1 of 9 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Sorafenib |
|---|---|---|
| Rash | Skin and subcutaneous tissue disorders | 5 of 9 |
| Hand-Foot Skin reaction | Skin and subcutaneous tissue disorders | 4 of 9 |
| Nausea | Gastrointestinal disorders | 3 of 9 |
| Hyponatremia | Metabolism and nutrition disorders | 3 of 9 |
| Mucositis/Stomatitis (functional symptomatic) - Oral Cavity | Gastrointestinal disorders | 2 of 9 |
| Elevated Transaminitis | Investigations | 2 of 9 |
| Diarrhea | Gastrointestinal disorders | 2 of 9 |
| Fatigue | General disorders | 2 of 9 |
| Elevated creatinine | Investigations | 2 of 9 |
| Fever | General disorders | 2 of 9 |
Serious events with the highest arm-level rate: pancreatitis (11.1%), hand-foot skin reaction (11.1%).
The adverse-event summary lists the Sorafenib group with 9 at risk for serious adverse events and 1 affected. The top serious terms are pancreatitis at 11.1% and hand-foot skin reaction at 11.1%. These entries describe what the registry record reports; they do not establish that sorafenib caused the events. The record does not provide event timing, severity grades, duration, management, or participant-level details.
The record also lists 9 at risk and 9 affected for other adverse events. It does not provide further detail in the adverse-event information about the types or severity of those other events. A count of affected participants is not necessarily the same as a count of event episodes, and the registry record does not clarify event counts beyond the listed summaries.
The deaths field is null. A null entry should not be interpreted as a reported count of zero; deaths are not reported in the registry record. The safety figures are descriptive and do not include a comparator group, so they cannot quantify a comparative risk. They also should not be read as a complete account of harms without fuller event definitions and follow-up information.
12. What the Result Does, and Does Not, Mean
The registry record establishes that a small, single-group study examined a planned MRI measure during neoadjuvant sorafenib treatment. It specifies a baseline and week 5 ADC assessment and a participant-level percent-change calculation. It also records enrollment 9, a sorafenib schedule of 400mg orally twice daily on days 1-28, and a TERMINATED status.
What the record does not establish is the observed primary result. A numerical mean change, its full range, or another measure of uncertainty is not reported in the registry record. Without those results, it is not possible to describe the direction or magnitude of the ADC change, or how consistently it appeared among participants. The record also does not report an analysis linking ADC change to pathologic findings, despite the official title’s reference to that relationship.
Even if a before-and-after ADC change were reported, the single-group design would limit causal interpretation. There is no separate control group to show how ADC might have changed over the same period without sorafenib or under another treatment. The study therefore cannot, on the information recorded, establish that treatment caused an imaging change. Nor does an imaging measure alone establish a change in clinical outcomes, tumor status, or surgical requirements.
The biological explanation in the rationale describes why ADC might be of interest as an imaging signal. It is not a result. A useful biomarker or imaging measure requires evidence about its measurement properties and its relationship to outcomes relevant to the study question. Such evidence is not reported in the registry record.
The safety summaries identify serious-event terms and counts, but lack the detail required for a full assessment of event context or comparative risk. The termination reason, missing-data handling, and analysis populations are also not reported. Accordingly, the record supports a description of the study’s design and intended endpoint, but not a conclusion about the size, precision, causality, or clinical importance of an outcome effect.
13. Why This Trial Matters Statistically
This trial is statistically useful as an example of the distinction between a clearly described endpoint and a reported result. The registry record defines an MRI-derived measure, its baseline and week 5 timeframe, and the participant-level percent-change calculation. Yet it does not provide a numerical primary estimate or uncertainty measure. A well-specified measurement question does not by itself reveal what was observed.
The single-group design also makes the estimand important. A within-group change asks how measurements differed over time among participants in the study. A comparative treatment effect asks how outcomes would differ between treatment strategies. Those are not interchangeable questions. Without a concurrent comparison group, changes in the measured endpoint cannot be separated from other influences over time.
The study’s imaging focus highlights a further distinction between a biological or measurement rationale and evidence of clinical relevance. ADC is described as a measure of water mobility, and the record explains why it might change in a lesion. That explanation motivates measurement, but a numerical change and its relationship to pathology or clinical outcomes would be needed to assess how informative the measure was in this study. Those results are not reported in the registry record.
The registry record also illustrates why outcome availability, analysis populations, and missing-data procedures matter. A baseline-to-follow-up endpoint depends on measurements at both times, yet the record does not explain how incomplete data were handled. In a study with enrollment 9, participant-level availability and variation are important to understanding any group summary, but those details are not reported.
For statistical interpretation, the record therefore offers a study question and design, not evidence sufficient to quantify an effect. Its value lies in making clear what was intended to be measured and what information remains unavailable for assessing the result.
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 does not report a numerical primary outcome, confidence interval, p-value, or participant-level ADC data. As a result, the primary endpoint’s direction, magnitude, variability, and precision cannot be assessed. Although the summary parameter is listed as a mean and dispersion as full range, the numerical summaries themselves are not reported in the registry record.
Enrollment was 9, and the study used a single-group design with no masking. There was no concurrent comparator to support a treatment contrast. Any observed before-and-after change would be vulnerable to explanations other than treatment, including time-related change and measurement variability. The record does not report measurement reliability, scan-reading procedures, or whether assessments were conducted without knowledge of measurement timing.
The record does not explain why the study was terminated, whether all planned assessments were completed, or whether termination affected outcome availability. Participant flow, missing scans, the primary analysis population, and missing-data methods are not reported. These omissions prevent assessment of potential selection effects in the outcome summary.
Although three outcome measures are listed, the record reports no analyses and does not describe a multiplicity approach. It also does not provide the statistical analysis plan, an interim-analysis plan, or a trial-specific sample-size justification. The planning assumptions described for an illustration are not evidence of the original design rationale or of the study’s observed precision.
Finally, the official title refers to pathologic findings, but the registry record does not report a statistical analysis relating pathology to ADC. It also does not provide detailed safety information, the reason for termination, or a comparative safety assessment. These limits leave the study’s imaging and safety findings insufficiently characterized in the registry record.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Sorafenib in Treating Patients With Locally Advanced or Metastatic Kidney Cancer — NCT00727532.
- Registry record last updated 2019-05-03; status last verified 2018-10.
- Results first posted 2019-05-03.
- 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
NCT00727532, titled “Sorafenib in Treating Patients With Locally Advanced or Metastatic Kidney Cancer,” was sponsored by Northwestern University. It is recorded as an interventional, single-group, unmasked study with phase NA and enrollment 9. The study began in 2008-07, reached primary completion in 2011-10, and is listed as TERMINATED.
Participants received sorafenib 400mg orally twice daily on days 1-28 and underwent diffusion-weighted MRI before treatment and after the 28-day course. The primary outcome was percentage change in ADC between baseline and week 5. The registry record lists three outcome measures but no analyses and does not report a numerical primary result, the reason for termination, or the primary analysis population.
The serious adverse-event summary lists pancreatitis and hand-foot skin reaction at 11.1% each; the deaths field is null. The registry record does not report the primary result’s precision, missing-data methods, or a comparison group. Its information supports description of the intended imaging question and study design, but not assessment of a treatment effect.