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 Phase II Trial of Sorafenib (Nexavar) in Patients With Advanced Thyroid Cancer (NCT00654238), a na phase 2 study. The primary outcome is To Determine the Efficacy (Best Response) of BAY 43-9006 (Objective Response Rate and Stable Disease) in Patients With Metastatic Thyroid Carcinoma..
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00654238 |
| Phase | Phase 2 |
| Condition | Metastatic Differentiated Thyroid Cancer, Metastatic Poorly Differentiated Thyroid Cancer, Metastatic Anaplastic Thyroid Cancer |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 59 |
| Arms in the primary outcome | n/a |
| Lead sponsor | University of Pennsylvania |
| Results posted | Yes |
2. Clinical Question
The study asks whether sorafenib has activity in people with advanced thyroid cancer that is metastatic or recurrent. Its primary outcome concerns best response, including objective response and stable disease, among participants with metastatic thyroid carcinoma. The registry describes response assessment using RECIST v1.0 criteria for target lesions assessed by MRI.
Population
Participants with advanced thyroid cancer, including metastatic differentiated, poorly differentiated, anaplastic, and medullary thyroid cancer. The registry records enrollment of 59 participants. It does not report how enrollment was distributed among the histologic subtypes.
Intervention
Sorafenib, also identified in the study title by the brand name Nexavar and in the official title by the development code BAY 43-9006.
Comparator
There is no comparison group in the registry record. The study is described as a single-arm study, with allocation listed as not applicable and masking listed as none.
Primary question
What best response was recorded with sorafenib, including objective response and stable disease, among participants with metastatic thyroid carcinoma, assessed by the registry’s stated response criteria?
This is a single-group question, not a randomized comparison of sorafenib with another treatment. Consequently, the study can describe outcomes observed in its enrolled participants, but its design does not by itself estimate how those outcomes compare with outcomes under a control treatment. Numerical results for the primary response outcome are not reported in the registry record.
3. Trial Design
This is a phase II, single-group study sponsored by the University of Pennsylvania. The registry lists an enrollment of 59 and describes sorafenib as the intervention. The trial status is completed. Its recorded start is 2006-02, and its primary completion is 2010-12.
The record lists metastatic differentiated, poorly differentiated, anaplastic, and medullary thyroid cancer as conditions. It does not report the number enrolled within each histologic subtype. The arm description identifies a single arm. Allocation is listed as not applicable, and masking is listed as none. These features mean the design is not a masked, randomized comparison between treatment groups.
The primary outcome is best response, described as objective response rate and stable disease. A secondary outcome is median progression-free survival among participants receiving sorafenib. The primary outcome’s timeframe is stated as beginning at randomization and continuing until documented progression or death from any cause, whichever came first, assessed up to 60 months. Because the design is described as single-group and allocation is not applicable, the reference to randomization does not fit the recorded design; how the time origin was implemented is not reported in the registry record.
The registry gives no protocol-level explanation for the sample size or the reasons for choosing this design. It does not report a comparator arm, an allocation procedure, or masking beyond the entries noted above. Numerical primary or secondary outcome results are not reported in the registry record, so the design description cannot be used to infer the size or precision of any treatment effect.
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 | 59 |
| COMPLETED | 55 |
| NOT COMPLETED | 4 |
Reasons for not completing this period:
| Reason | Sorafenib |
|---|---|
| Screen Failures | 4 |
Analysis population for the primary outcome, as defined in the registry: Responses were calculated by total enrolled to each group by histologic subtype.
5. Baseline Characteristics
The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.
Population: Four patients were deemed ineligible as a result of inadequate CBC and the establishment of an alternative diagnosis resulting from secondary pathology review.
| Characteristic | Unit | Sorafenib |
|---|---|---|
| Participants analysed | 55 | |
| Age, Continuous | years | 63 (30 to 86) |
| Sex: Female, Male: Female | Participants | 27 |
| Sex: Female, Male: Male | Participants | 28 |
| Race and Ethnicity Not Collected | Participants | — |
| Region of Enrollment: United States | participants | 55 |
| Histology subtype: DIfferentiated | Participants | 44 |
| Histology subtype: Poorly Differentiated | Participants | 6 |
| Histology subtype: Medullary | Participants | 3 |
| Histology subtype: Anaplastic | Participants | 2 |
| ECOG Performance Status at Baseline: ECOG Performance Status 0 | Participants | 29 |
| ECOG Performance Status at Baseline: ECOG Performance Status 1 | Participants | 26 |
6. Primary Endpoint
The registry lists the primary outcome as To Determine the Efficacy (Best Response) of BAY 43-9006 (Objective Response Rate and Stable Disease) in Patients With Metastatic Thyroid Carcinoma., measured over From date of randomization until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 60 months.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary outcome concerns best response to sorafenib in participants with metastatic thyroid carcinoma. The registry names objective response rate and stable disease and describes complete response, partial response, and overall response under RECIST v1.0. Complete response is defined as disappearance of all target lesions. Partial response is defined as a >=30% decrease in the sum of the longest diameter of target lesions. Overall response is defined as complete response plus partial response. Assessment is described as being performed by MRI.
Response categories are useful in oncology trials because they provide a structured way to describe changes in measurable target lesions. An objective response measure distinguishes participants meeting the stated complete or partial response criteria from those who do not meet them. Stable disease is also named in the endpoint title, but its operational definition is not reported in the registry record. The record says responses were calculated by total enrolled to each group by histologic subtype; the actual subtype-specific counts and response values are not reported.
The timeframe is stated as from randomization until documented progression or death from any cause, whichever came first, assessed up to 60 months. The randomization wording is not consistent with the recorded single-group design, and clarification of the actual time origin is not reported. Without numerical endpoint results, the record does not establish how many participants had each response category or how precisely the response outcome was estimated.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies a completed phase II study of sorafenib in advanced thyroid cancer, with a single treatment group and enrollment of 59. It gives definitions for the primary response endpoint but does not report the observed primary endpoint results. Therefore, the record does not establish the number or proportion of participants with complete response, partial response, overall response, or stable disease. It also does not provide an estimate of response precision, such as a confidence interval.
The study’s single-group design matters for interpretation. Outcomes observed among participants receiving sorafenib can be described, but there is no concurrent control group in the registry record against which to compare those outcomes. Without such a comparison, observed response or disease course cannot be separated from the influences of participant selection, cancer subtype, prior clinical course, assessment timing, or other factors. These are general considerations for interpreting single-group clinical studies, not claims about what occurred among these participants.
The record names metastatic differentiated, poorly differentiated, anaplastic, and medullary thyroid cancer. These are distinct histologic categories, but subtype-specific enrollment and outcome figures are not reported in the registry record. A pooled response result, even if available, would not show whether outcomes were similar across the listed subtypes unless subtype-specific data were also reported. No pooled primary outcome value is available in the record.
A secondary outcome is median progression-free survival. Its numerical result, the method used to estimate it, and any uncertainty interval are not reported in the registry record. The stated primary outcome timeframe extends up to 60 months, but that assessment window is not itself a result and does not indicate how many participants were followed for that duration.
The safety entries list 55 at risk for serious adverse events and 4 affected, alongside 55 at risk for other adverse events and 43 affected; deaths are listed as 36. The record also names deep vein thrombosis and cerebral vascular accident/stroke, each with a listed value of 3.6%. These figures should be read as registry safety entries, not as a comparison of treatment against a control. The record does not explain the relationship of the deaths to treatment or provide the detailed timing, severity, or attribution needed to interpret them further.
Overall, the posted results do not permit a quantitative conclusion about the primary response outcome or its precision. The design and endpoint definitions are described, but the outcome data necessary to assess the observed response are not reported in the registry record.
9. Statistical Methods Explained
Interpreting a clinical trial requires separating its design, outcome definition, analysis, and reported results. In this study, the design is recorded as phase II and single group. The primary endpoint is a count-based response outcome, and the registry supplies a clinical definition of response. The primary numerical findings and the statistical analysis details are not reported in the registry record.
What does a single-group design estimate?
In general, a single-group study records outcomes among participants who receive the study intervention. It can describe the frequency of observed outcomes in that group. It does not provide a concurrent treatment comparison, so it cannot estimate a randomized between-group difference. A comparison with an external benchmark would require a clearly specified benchmark and methods for addressing differences in populations and care. Such a comparison is not reported in the registry record.
Allocation is listed as not applicable, the model is single group, and the arm description says the study has a single arm. Masking is listed as none. These entries describe the recorded design; they do not disclose how outcome assessors handled knowledge of treatment or how potential assessment bias was addressed. Additional operational details are not reported in the registry record.
How should a response endpoint be understood?
In general, a binary outcome identifies whether a participant meets a defined criterion or does not. The registry describes complete response and partial response according to RECIST v1.0, with overall response defined as the combination of those categories. It also names stable disease in the primary outcome title. A count of participants meeting a response category is not, by itself, a rate: calculating a rate requires a defined denominator and the reported outcome count. The registry says responses were calculated by total enrolled to each group by histologic subtype, but it does not report the response counts or subtype-specific denominators.
For a binary outcome, uncertainty can be described with a confidence interval. In general, a confidence interval expresses the range of values reasonably compatible with the observed data under the method’s assumptions. Its width depends in part on the amount of information available and the observed outcome frequency. Since the registry does not report the primary response counts or an interval, neither the estimated response frequency nor its precision can be assessed from this record.
What can be inferred from the timeframe?
The registry’s primary outcome timeframe is stated as extending from randomization until documented progression or death from any cause, whichever came first, with assessment up to 60 months. That wording is different from the recorded single-group design, where randomization is not applicable. The record does not explain the time origin used in practice. It also does not report follow-up distributions, censoring, or the number of participants assessed at particular times.
The secondary endpoint is median progression-free survival. In general, time-to-event outcomes account for both the time until an event and the fact that some participants may not have had an event by the end of observation. Methods for handling censoring and estimating a median are important to interpretation. The registry record does not give the analysis method, event count, estimate, confidence interval, or details of censoring for this secondary outcome.
What do the planning assumptions mean?
In general, sample-size planning describes how many participants a future study may need under stated assumptions; it is not an observed outcome and is not observed power. The planning assumptions associated with this record describe a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and an effect to detect expressed as a stated fraction of the observed effect. They are described as a planning illustration, not as the trial’s own design. This is distinct from the recorded single-group study. The numerical planning outputs and the original protocol’s sample-size justification are not reported in the registry record.
For a binary endpoint, planning generally depends on the assumed event frequency in each comparison group and the effect the study is intended to detect. For a time-to-event endpoint, planning depends on event information and assumptions about the event process. These are general methodological considerations. The registry does not report the assumptions used for the trial’s original design or a statistical analysis plan that would show how its planned analyses were to be conducted.
What analysis details are absent?
The registry records 0 analyses. This means that analysis results are not presented in the record; it does not establish that no analyses were conducted outside the registry. No test statistic, p-value, confidence interval, adjusted estimate, or analysis population for the primary outcome is reported. The record also does not state whether the endpoint was analyzed by intention-to-treat, by participants who received treatment, or under another population definition.
In general, adjustment may be used to account for prespecified participant characteristics or design features, and multiplicity procedures may address the increased chance of false-positive findings when many outcomes or comparisons are tested. Whether either approach was used is not reported in the registry record. With no numerical primary endpoint result and no analysis description, an independent recalculation from summary statistics is not possible.
10. Multiplicity, Missing Data and Analysis Populations
The registry identifies a primary response outcome and a secondary median progression-free survival outcome. It also records 0 analyses. Numerical results for either outcome and a statistical analysis plan are not reported in the registry record. The record does not describe how the primary and secondary outcomes were prioritized in statistical testing, whether any adjustment for multiple outcomes was planned, or whether subgroup analyses were prespecified.
Multiplicity is a general statistical concern when several hypotheses are tested: the chance of at least one false-positive finding can increase as more tests are considered. A prespecified testing strategy can define how findings should be interpreted. No such strategy is reported for this study. The mere listing of outcome measures does not show that formal hypothesis tests were performed.
Missing-data methods are also not reported in the registry record. There is no description of how participants with unavailable response assessments, incomplete follow-up, or unknown outcomes were handled. The record says responses were calculated by total enrolled to each group by histologic subtype, but it does not provide the underlying subtype denominators, response counts, or participant-level analysis population. It therefore is not possible to determine whether the reported endpoint was based on all enrolled participants or a subset.
The stated response timeframe refers to progression or death and assessment up to 60 months. How incomplete observation and censoring were handled is not reported. The time origin also requires clarification because the timeframe refers to randomization while the trial is recorded as single group with allocation not applicable. These omissions limit assessment of both the endpoint definition in practice and the completeness of follow-up.
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 | 4 | 55 | 7.3% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Sorafenib | 36 of 55 |
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 | 43 of 55 |
Most frequent serious adverse event terms
| Event term | Organ system | Sorafenib |
|---|---|---|
| Deep Vein Thhrombosis | Blood and lymphatic system disorders | 2 of 55 |
| Cerebral Vascular Accident/Stroke | Nervous system disorders | 2 of 55 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Sorafenib |
|---|---|---|
| Hand Foot Skin Reaction | Skin and subcutaneous tissue disorders | 43 of 55 |
| Rash | Skin and subcutaneous tissue disorders | 41 of 55 |
| Diarrhea | Gastrointestinal disorders | 29 of 55 |
| Alopecia | Skin and subcutaneous tissue disorders | 25 of 55 |
| Stomatitis/Mucositis | Gastrointestinal disorders | 18 of 55 |
| Fatigue | General disorders | 15 of 55 |
| Myalgia | Musculoskeletal and connective tissue disorders | 11 of 55 |
| GI Discomfort | Gastrointestinal disorders | 9 of 55 |
| Hypertension | Vascular disorders | 8 of 55 |
| Squamous Cell Carcinoma of the Skin | Skin and subcutaneous tissue disorders | 8 of 55 |
Serious events with the highest arm-level rate: deep vein thhrombosis (3.6%), cerebral vascular accident/stroke (3.6%).
The registry lists a Sorafenib safety group with 55 at risk for serious adverse events and 4 affected. For other adverse events, it lists 55 at risk and 43 affected. It lists deaths as 36. These are counts recorded in the registry, not comparative effects: the study has no control group in the recorded design. The registry does not explain whether deaths were considered treatment-related, how they were adjudicated, or the timeframe over which they were counted.
The listed serious terms are Deep Vein Thhrombosis and Cerebral Vascular Accident/Stroke, each reported as 3.6%. The record does not provide the event counts underlying these percentages, details of recurrence, timing, severity grading, or attribution. The term spelling is reproduced as recorded for the first event. These entries should not be interpreted as a complete account of all safety outcomes or as evidence of a causal relationship.
In general, adverse-event counts and percentages depend on their definitions, observation period, and denominator. A count of participants affected is different from a count of event episodes, since a participant may experience more than one event. The registry entries do not provide enough detail to determine those distinctions for each listed term. Without a control group, these safety figures describe reports within the sorafenib group but cannot quantify excess risk compared with another treatment.
12. What the Result Does, and Does Not, Mean
This record establishes that a phase II, single-group study of sorafenib in advanced thyroid cancer was completed and that the registry defined a primary response endpoint. It does not report the numerical primary endpoint results. As a result, it does not show the observed number of participants with objective response or stable disease, nor does it provide an estimate of uncertainty around those outcomes.
The endpoint definition gives a framework for classifying radiographic response, but definitions alone are not evidence that a response occurred. The primary outcome description includes complete and partial response criteria and states that overall response combines those categories. The registry does not report category counts, response rates, or subtype-specific results. The secondary progression-free survival outcome is named, but its median and uncertainty are also not reported.
The single-group design supports description of outcomes among those enrolled but does not provide a randomized estimate of comparative benefit or harm. It also cannot, on its own, distinguish an observed outcome from differences in participant characteristics, disease subtype, or other influences. These are general limits of this design and are not assertions about the cause of any particular outcome in this study.
The safety record reports serious and other adverse-event counts, deaths, and two serious event terms with listed percentages. These entries are useful as registry-reported safety information, but missing attribution and event-detail information constrain interpretation. They should not be treated as comparative risk estimates.
Accordingly, the registry record supports statements about the study’s design, enrolled population description, endpoint definitions, and listed safety entries. It does not support a quantitative conclusion about the primary response result, comparative effectiveness, or precision of the response estimate. Those conclusions require outcome and analysis information not reported in the registry record.
13. Why This Trial Matters Statistically
This trial is statistically informative as an example of the difference between a clearly named endpoint and a reported endpoint result. The registry specifies response categories and assessment criteria, yet does not report the numerical findings needed to estimate the observed response or its uncertainty. A detailed outcome definition is important, but it cannot substitute for observed counts and a description of how those counts were analyzed.
The study also illustrates the interpretive limits of single-group evidence. Without a concurrent comparator, the outcome observed in participants receiving sorafenib is descriptive. A randomized comparison would address a different question: how outcomes differ between assigned treatment groups under the design and analysis plan. The registry does not provide such a comparison, so comparative claims cannot be derived from the recorded design.
Histologic subtype is especially relevant because the eligible conditions include differentiated, poorly differentiated, anaplastic, and medullary thyroid cancer. The record says response calculations were organized by histologic subtype, but does not give subtype-specific enrollment or results. Without those data, the degree to which findings varied across the listed disease categories cannot be assessed.
The record further highlights the value of consistent design and endpoint language. Its single-group description and allocation entry sit alongside a primary outcome timeframe referring to randomization. The actual time origin and the handling of follow-up are not reported. Clarifying these details would help readers understand precisely how response and progression-free survival were measured.
Finally, registry safety counts can be read descriptively, but their interpretation depends on definitions, timing, and attribution. Those details are limited in this record. For statistical readers, the central lesson is to distinguish what the registry defines, what it counts, and what it actually reports as an analyzed 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 main limitation for interpretation is that numerical results for the primary endpoint are not reported in the registry record. The record therefore does not allow readers to verify response counts, calculate a response rate, or assess precision. It also does not report numerical results for the named secondary endpoint, median progression-free survival.
The single-group design has no control arm, and allocation is listed as not applicable. Consequently, there is no within-trial randomized contrast. The record does not report the distribution of participants across the listed histologic subtypes, making it impossible to assess the composition of the enrolled group or any subtype-specific response pattern.
The endpoint timeframe refers to randomization despite the single-group design. The record does not clarify the time origin, assessment schedule, completeness of follow-up, censoring, or handling of participants with missing response assessments. The operational definition of stable disease is not reported. Analysis populations and methods for the primary and secondary outcomes are also not reported.
For safety, the registry lists affected counts, deaths, and serious event terms but does not describe their relationship to treatment, observation periods, event grading, or detailed denominators for the listed percentages. These limits prevent a fuller account of the timing and attribution of reported events.
The record does not provide the protocol’s sample-size justification, a statistical analysis plan, or details of multiplicity and missing-data procedures. The associated planning assumptions are described as an illustration rather than the trial’s own design. These missing details limit independent assessment of the intended analysis and its assumptions.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Phase II Trial of Sorafenib (Nexavar) in Patients With Advanced Thyroid Cancer — NCT00654238.
- Registry record last updated 2019-11-18; status last verified 2019-11.
- Results first posted 2019-11-18.
- Data retrieved from ClinicalTrials.gov on 2026-11-09.
- Group sizes, means, event counts and sponsor-reported p-values are reproduced as posted. Effect estimates, confidence intervals and p-values not attributed to the sponsor are independently calculated from the posted summary statistics.
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18. Record Summary
The registry record describes NCT00654238, a completed phase II single-group study of sorafenib in advanced metastatic thyroid cancer. The sponsor is the University of Pennsylvania. Enrollment is listed as 59; the recorded start is 2006-02 and primary completion is 2010-12. Masking is listed as none, and allocation is not applicable.
The primary outcome is best response, described in terms of objective response and stable disease using RECIST v1.0 criteria for target lesions assessed by MRI. Median progression-free survival is listed as a secondary outcome. The registry does not report numerical results for either outcome or the analyses used to estimate them.
Safety entries list 55 at risk and 4 affected for serious adverse events, 55 at risk and 43 affected for other adverse events, and 36 deaths. Deep vein thrombosis and cerebral vascular accident/stroke are each listed at 3.6%. The record does not provide further detail on attribution or the underlying event counts for those terms.