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 Vemurafenib and Sorafenib in Pancreatic Cancer (NCT05068752), a na phase 2 study. The primary outcome is Disease Control Rate (DCR).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT05068752 |
| Phase | Phase 2 |
| Condition | Pancreas Cancer |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 10 |
| Arms in the primary outcome | n/a |
| Lead sponsor | HonorHealth Research Institute |
| Results posted | Yes |
2. Clinical Question
Population
People with advanced KRAS-mutated pancreatic cancer who had progressed on standard chemotherapy were the intended population. The registry describes the condition as pancreas cancer. The enrollment recorded for the trial was 10. Further eligibility details and a fuller description of the analyzed population are not reported in the registry record.
Intervention
The study evaluated the combination of the oral chemotherapy drugs vemurafenib and sorafenib. The registry lists both drugs as interventions and describes one combination group. The available record does not provide details about dose, schedule, treatment duration, or modifications to treatment.
Comparator
There was no separate comparator group in the registry design. The trial is recorded as single-group, with allocation listed as NA and masking as none. Consequently, the study does not provide a randomized, like-for-like comparison against another treatment or a control group.
Primary question
The primary endpoint asks what proportion of participants had complete response, partial response, or stable disease at 16 weeks after initiation of study treatment. Responses were defined using RECIST v1.1 imaging criteria. The registry record does not report the observed disease control rate or an analysis of this endpoint.
In practical statistical terms, the question is whether disease control was observed in this enrolled group during the stated assessment period. The endpoint combines three response categories, so it is broader than a measure limited to tumor shrinkage: stable disease is included alongside complete and partial response. The registry describes the intended population as people with advanced KRAS-mutated pancreatic cancer whose disease had progressed on standard chemotherapy, but the record does not give additional eligibility criteria or details of the participants actually assessed for the primary endpoint.
Because the design has one group, any observed response pattern would describe participants receiving the combination; it would not by itself establish how that pattern compares with what would have occurred under another treatment or without the combination. Such a comparison is not part of the registry design. Further information on the primary endpoint result, its precision, and the analysis population is not reported in the registry record.
3. Trial Design
This is a phase II, single-group trial of vemurafenib in combination with sorafenib for advanced KRAS-mutated pancreatic cancer. The registry lists HonorHealth Research Institute as the sponsor and records the trial as completed. Enrollment is recorded as 10. The recorded start date is 2021-10-28, and the primary completion date is 2024-11-08.
The registry lists no masking and identifies allocation as NA. Together with the single-group model, this means the registered design is not a randomized comparison and does not include a like-for-like control arm. The arm description identifies the combination of vemurafenib and sorafenib; no separate treatment group is listed. The registry record does not describe dose levels, treatment schedules, stopping rules, or how treatment exposure was managed.
The primary outcome is disease control rate, defined as the number of participants with complete response, partial response, or stable disease at 16 weeks divided by the total number of participants. The timeframe is stated as initiation of study treatment until 16 weeks. Response is based on RECIST v1.1 imaging criteria. The registry also lists secondary outcomes concerning adverse events possibly or definitely related to the combination, progression-free survival, overall survival, and percent changes in plasma phospho-ERK and phospho-AKT levels from baseline. Six outcome measures are recorded in total, but the result values and analysis details are not reported in the registry record.
The design should therefore be read as a small, single-group investigation intended to characterize outcomes in the treated group, not as evidence from a randomized treatment comparison. The registry does not report the protocol, a sample-size justification, planned statistical methods, analysis populations, or whether an interim analysis was planned. Those details cannot be inferred from the phase designation or enrollment count.
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 | Vemurafenib in Combination With Sorafenib |
|---|---|
| STARTED | 10 |
| COMPLETED | 9 |
| NOT COMPLETED | 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 | Vemurafenib in Combination With Sorafenib |
|---|---|---|
| Participants analysed | 9 | |
| Age, Continuous | years | 62.8 (48.0 to 85.8) |
| Sex: Female, Male: Female | Participants | 4 |
| Sex: Female, Male: Male | Participants | 5 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 2 |
| Race (NIH/OMB): White | Participants | 7 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 1 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 7 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 1 |
| Region of Enrollment: United States | Participants | 9 |
| Eastern Cooperative Oncology Group (ECOG) Score 0 - 1 | Participants | 9 |
| Cancer Antigen 19-9 (CA 19-9): < 35 U/mL | Participants | 3 |
| Cancer Antigen 19-9 (CA 19-9): ≥ 35 U/mL | Participants | 5 |
| Primary Tumor Location: Head | Participants | 6 |
| Primary Tumor Location: Body | Participants | 2 |
| Primary Tumor Location: Tail | Participants | 1 |
| Site of Metastasis: Liver | Participants | 4 |
| Site of Metastasis: Lung | Participants | 1 |
| Site of Metastasis: Other | Participants | 1 |
| Site of Metastasis: Multiple sites | Participants | 3 |
| Number of Prior Lines of Therapy: 2 prior lines of therapy | Participants | 1 |
| Number of Prior Lines of Therapy: 3 prior lines of therapy | Participants | 5 |
| Number of Prior Lines of Therapy: 4 prior lines of therapy | Participants | 3 |
6. Primary Endpoint
The registry lists the primary outcome as Disease Control Rate (DCR), measured over Initiation of study treatment until 16 weeks..
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | DCR, % of participants with CR/PR/SD |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is disease control rate at 16 weeks. It combines participants with complete response, partial response, or stable disease, using response categories defined by RECIST v1.1 imaging criteria. The registry defines the rate as the number of participants in any of those three categories divided by the total number of participants. Its timeframe begins at initiation of study treatment and extends until 16 weeks.
As a general statistical concept, a disease control endpoint summarizes the proportion of a group reaching a prespecified set of response states by a defined assessment period. Including stable disease captures participants whose disease does not meet the registry's complete or partial response categories but is classified as stable at the specified assessment. The endpoint is therefore not identical to a response rate restricted to complete or partial response.
A proportion is most informative when its numerator, denominator, assessment timing, and handling of participants without an evaluable assessment are clear. The registry provides the endpoint definition and timeframe, but the observed disease control rate, denominator used in the analysis, and handling of missing or unevaluable assessments are not reported in the registry record. No confidence interval or other measure of precision for the primary endpoint is reported either. Without those results, the endpoint definition alone does not indicate what disease control was observed in this trial.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies a completed phase II investigation of vemurafenib plus sorafenib in a single group of people with advanced KRAS-mutated pancreatic cancer. It records enrollment of 10 and defines disease control rate at 16 weeks as the primary endpoint. However, it does not report the primary endpoint value, the number of participants in each response category, or a statistical analysis of disease control. The record therefore does not support a numerical description of the observed primary outcome.
The single-group structure is central to interpretation. If a disease control rate were reported, it would describe the enrolled group under the study treatment and assessment procedures. It would not establish a treatment effect relative to a control, because no separate comparator group is listed and allocation is recorded as NA. Differences from outcomes in other settings cannot be attributed to the combination using this design alone; the registry record supplies no controlled comparison.
Precision also matters. A proportion from a small group can be sensitive to the classification of individual participants, and an interval estimate would help communicate uncertainty around a reported rate. No primary endpoint estimate or confidence interval is reported in the registry record. The enrollment count should not be mistaken for an analyzed denominator: the number included in the primary endpoint analysis is not reported.
The registry lists six outcome measures, including safety, survival, and biomarker outcomes, but provides no analysis entries. It does list safety-group counts: 9 participants at risk for serious adverse events, 4 with serious events, 9 at risk for other adverse events, 9 with other adverse events, and deaths of 9. These are registry safety figures, not a comparison between treatment groups. The record does not establish that the listed deaths were caused by treatment, nor does it provide enough context to interpret their timing or causes.
Accordingly, the posted information describes the study question, design, endpoint definition, and selected safety counts, but not a complete account of clinical outcomes. Lack of a reported result in the registry is not evidence that an outcome did or did not occur. It means that the outcome cannot be characterized from this record. Any conclusion about the size, direction, or precision of the primary endpoint result would go beyond what the registry reports.
9. Statistical Methods Explained
What does a single-group design tell us?
In general, a single-group study describes outcomes among participants receiving the study intervention. It can document what was observed under the treatment and study procedures, but it does not estimate a randomized between-group contrast when no control group is present. Here, the registry classifies the model as single group, allocation as NA, and masking as none. Those design details mean that a treatment-versus-control effect cannot be read from this record.
Randomization is generally used to make comparison groups more alike, on average, with respect to measured and unmeasured characteristics. Without a randomized comparator, differences between the study group and an external group could reflect differences in who was enrolled, how outcomes were assessed, or other circumstances, rather than the intervention. This is a general design principle; the registry does not report an external comparison for this trial.
How should a disease control proportion be understood?
In general, a proportion has a numerator and a denominator. For the primary endpoint, the registry defines the numerator as participants classified as having complete response, partial response, or stable disease at 16 weeks, and defines the denominator as the total number of participants. It specifies RECIST v1.1 imaging criteria and a timeframe from initiation of treatment until 16 weeks. The actual numerator, analysis denominator, and resulting proportion are not reported in the registry record.
A confidence interval is a statistical way to express uncertainty around an estimated quantity. For a proportion, it helps show the range of values compatible with the observed data under the interval's assumptions. It does not remove the limitations of the design, and it does not provide a control comparison. No confidence interval for the primary endpoint is reported in the registry record.
What do the adverse-event counts represent?
In general, safety summaries distinguish the number of participants assessed or at risk from the number who experienced an event. The registry's safety group lists 9 participants at risk for serious adverse events and 4 affected; it separately lists 9 at risk for other adverse events and 9 affected. These are counts in the combination group, not comparative estimates. Serious-event terms listed include Sepsis at 22.2%, Abdominal pain at 11.1%, and Urinary tract infection at 11.1%. The registry does not provide further detail about event timing, recurrence, severity grades, or attribution for each listed term.
The safety summary also records deaths of 9. A count of deaths, by itself, does not show cause, timing, or relationship to treatment. Those details, as well as a fuller account of follow-up and exposure, are not reported in the registry record. Safety counts should therefore be read as descriptive registry entries rather than estimates of comparative risk.
What can be said about statistical planning?
In general, a statistical analysis plan specifies how outcomes will be summarized or compared, which participants will be included, how missing observations will be handled, and how uncertainty will be quantified. The registry lists no analysis entries. A statistical plan, protocol details, and any sample-size justification are not reported in the registry record, so they cannot be reconstructed from the trial's phase or enrollment.
Planning sample-size illustrations are distinct from the trial's actual design and from observed statistical power. The registry's description of such illustrations specifies 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 stated fraction of the observed effect. Those are assumptions for planning illustrations, not evidence that this trial randomized participants between two arms. The record identifies the study as single-group. A sample-size illustration is not an observed result, and it does not establish what power the completed trial had.
General statistical methods can help explain how results would ordinarily be interpreted, but they cannot fill in missing analyses. The registry record does not report the primary endpoint estimate, confidence interval, statistical test, analysis population, treatment of unevaluable responses, or any adjustment for other outcomes. Consequently, no inferential result for disease control can be reproduced from the information recorded.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists six outcome measures, including the primary disease control endpoint and secondary outcomes covering adverse events, progression-free survival, overall survival, and plasma phospho-ERK and phospho-AKT changes. The record contains no analysis entries. It does not report which outcomes were formally tested, whether tests were planned, or whether any adjustment for multiple comparisons was specified.
In general, when several outcomes or statistical tests are examined, the chance of at least one apparently notable result can increase. Multiplicity methods are sometimes used to control that risk, or investigators may identify one outcome as primary and treat other results as exploratory. The registry identifies disease control rate as primary, but its statistical plan and any approach to multiplicity are not reported in the registry record. No assumptions about adjustment or testing hierarchy should be made.
Missing-data procedures are also important for a response proportion assessed at a fixed time. Participants may lack an evaluable scan or may not have an assessment at the specified time; an analysis plan can define how such cases are handled. The registry provides the response definition and timeframe but does not report the number assessed, the analysis denominator, or rules for missing and unevaluable observations.
Likewise, the analysis populations are not described. The record does not say which participants were included in each outcome analysis, how survival follow-up was handled, or whether the safety summaries include everyone who began treatment. The enrollment count and safety counts should not be assumed to refer to identical populations. These omissions limit reproducibility and make it difficult to assess whether the reported outcomes, if available elsewhere, followed prespecified methods.
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 |
|---|---|---|---|
| Vemurafenib in Combination With Sorafenib | 4 | 9 | 44.4% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Vemurafenib in Combination With Sorafenib | 9 of 9 |
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 |
|---|---|
| Vemurafenib in Combination With Sorafenib | 9 of 9 |
Most frequent serious adverse event terms
| Event term | Organ system | Vemurafenib in Combination With Sorafenib |
|---|---|---|
| Sepsis | Infections and infestations | 2 of 9 |
| Abdominal pain | Gastrointestinal disorders | 1 of 9 |
| Urinary tract infection | Infections and infestations | 1 of 9 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Vemurafenib in Combination With Sorafenib |
|---|---|---|
| Nausea | Gastrointestinal disorders | 5 of 9 |
| Vomiting | Gastrointestinal disorders | 5 of 9 |
| Hypokalemia | Metabolism and nutrition disorders | 5 of 9 |
| Rash maculo-papular | Skin and subcutaneous tissue disorders | 5 of 9 |
| Hypophosphatemia | Metabolism and nutrition disorders | 4 of 9 |
| Abdominal pain | Gastrointestinal disorders | 3 of 9 |
| Fatigue | General disorders | 3 of 9 |
| Pruritus | Skin and subcutaneous tissue disorders | 3 of 9 |
| Hypertension | Vascular disorders | 3 of 9 |
| Gastroparesis | Gastrointestinal disorders | 2 of 9 |
Serious events with the highest arm-level rate: sepsis (22.2%), abdominal pain (11.1%), urinary tract infection (11.1%).
The registry lists one safety group, Vemurafenib in Combination With Sorafenib. For serious adverse events, 9 participants are recorded as at risk and 4 as affected. For other adverse events, 9 are recorded as at risk and 9 as affected. The listed serious-event terms are Sepsis at 22.2%, Abdominal pain at 11.1%, and Urinary tract infection at 11.1%. The record also lists deaths of 9.
These entries are descriptive counts and percentages for the group, not comparisons with another treatment. The serious-event terms and percentages should be read as registry safety entries; further details about their definitions, timing, recurrence, severity, and attribution are not reported in the registry record. The record's phrase concerning events possibly or definitely related to the combination appears among the secondary outcomes, but the specific attribution method and results are not reported.
The deaths count does not establish that the deaths were caused by the combination. The registry does not report their causes, timing, or relationship to treatment. Nor should the enrollment count of 10 automatically be used as the denominator for the safety entries, which record 9 at risk. A complete interpretation would require further information about treatment exposure, follow-up, and the safety analysis population, which is not reported in the registry record.
12. What the Result Does, and Does Not, Mean
The record establishes that this was a completed phase II, single-group study of vemurafenib with sorafenib, with enrollment recorded as 10. It defines a primary endpoint: the proportion of participants with complete response, partial response, or stable disease at 16 weeks, based on RECIST v1.1 imaging criteria. It also lists secondary outcomes and selected safety summaries. These details describe the study's question and the information captured in the registry.
The record does not report the observed disease control rate, response-category counts, or a confidence interval for the primary endpoint. It also does not include analysis entries. Therefore, the registry information cannot establish the amount or precision of disease control observed in the trial. The absence of an outcome result in this record should not be interpreted as evidence of either a favorable or unfavorable result.
The design also limits what any single-group outcome can establish. Without a comparator group, the registry cannot support a randomized estimate of how outcomes differ from another treatment or from no study treatment. Even if an outcome value were available, it would describe this group under the study procedures and would not by itself identify the effect attributable to the combination.
The listed safety counts warrant similarly careful reading. The record includes serious adverse event and other adverse event counts, serious-event terms, and a death count. It does not provide the context needed to determine causes, timing, or treatment attribution for deaths, nor does it report a comparative safety analysis. The counts should not be converted into a causal claim.
In short, the registry record documents the study design, target population, endpoint definition, and some safety information. It does not provide the outcome analyses needed to characterize efficacy, precision, or comparative effects. Further details about the protocol, analysis plan, missing data, and analysis populations are not reported in the registry record.
13. Why This Trial Matters Statistically
This trial is statistically notable because it asks a focused response question in a single-group phase II design. The primary endpoint uses a defined assessment period and combines complete response, partial response, and stable disease under RECIST v1.1. A clear endpoint definition helps make the intended outcome interpretable, but it is only one part of an evaluable result: the observed count, denominator, and uncertainty are also needed. Those primary endpoint results are not reported in the registry record.
The trial also illustrates why design and analysis must be considered together. A single group can describe outcomes among those enrolled and treated, but it does not supply a randomized control comparison. This distinction matters when interpreting a reported rate: a descriptive proportion is not the same as an estimate of benefit relative to an alternative. The registry's designation of a phase II study does not substitute for details of the statistical plan or sample-size justification, neither of which is reported.
Safety reporting likewise demonstrates the importance of denominators and context. The record lists participants at risk and affected for serious and other adverse events, along with selected serious-event terms and a death count. These entries are useful descriptive information, but they do not establish causation or comparative risk. Additional detail about follow-up, event attribution, and analysis populations is not reported.
Finally, the registry identifies multiple outcome areas but no analysis entries. Without reported estimates and methods, readers cannot assess precision, the handling of missing assessments, or how findings across outcomes were interpreted. The statistical value of the record lies in clarifying what was intended and what was registered, while also making clear that the outcome analyses needed for stronger conclusions are not reported.
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 trial-specific limitation in the registry record is the absence of a reported primary endpoint result. Although disease control rate is defined at 16 weeks, the record does not give the response counts, analyzed denominator, estimated rate, or confidence interval. The endpoint definition therefore cannot be used to infer the observed result.
The design is single group, with allocation listed as NA and masking as none. No like-for-like comparator is registered, so the study does not estimate a randomized difference between the combination and another treatment. The enrollment recorded as 10 is small, but the number analyzed for each outcome is not reported; enrollment should not be assumed to equal the endpoint or safety denominator.
The registry does not report dose and schedule details, the protocol, a sample-size justification, the statistical analysis plan, analysis populations, missing-data rules, or interim analyses. These details are important to evaluating how the endpoint was assessed and how the results, if reported elsewhere, were produced. The registry also does not report outcome analysis entries for the listed secondary measures.
Safety interpretation is limited by the descriptive nature of the listed counts. The record does not provide detail on event timing, recurrence, grading, treatment attribution, or the causes and timing of deaths. A causal relationship between the combination and the listed deaths cannot be determined from the counts. These limitations reflect what is not reported in the registry record and do not establish that the information is absent from all other sources.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Phase II Trial of Vemurafenib and Sorafenib in Pancreatic Cancer — NCT05068752.
- Registry record last updated 2026-03-30; status last verified 2026-02.
- Results first posted 2026-03-30.
- 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 records NCT05068752 as a completed phase II, single-group trial sponsored by HonorHealth Research Institute. It evaluates vemurafenib in combination with sorafenib in advanced KRAS-mutated pancreatic cancer. Enrollment is recorded as 10; allocation is NA and masking is none.
The primary endpoint is disease control rate, defined as complete response, partial response, or stable disease at 16 weeks using RECIST v1.1 imaging criteria. Secondary outcomes include safety, progression-free survival, overall survival, and plasma phospho-ERK and phospho-AKT changes. The registry lists safety counts and selected serious-event terms, but no analysis entries. The primary endpoint result, analysis methods, and further interpretation details are not reported in the registry record.