This page provides an independent statistical analysis and educational interpretation of publicly reported results. ClinicalTrials.gov provides the official trial registry record. Numerical trial results on this page are restricted to the results posted in the ClinicalTrials.gov record; calculations added by Clinical Biostats are labelled as such.
1. Trial at a Glance
This page covers the registry results for A Screening Study to Detect BRAF V600 Mutation-Positive Patients For Enrollment Into Clinical Research Studies of Zelboraf (Vemurafenib) (NCT01804140), a registered phase not stated study. The primary outcome is Percentage of Participants With BRAF V600 Mutation Positivity in Tumor Samples by Cancer Type.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01804140 |
| Phase | Phase not stated |
| Condition | Multiple Myeloma, Neoplasms |
| Allocation | Not Stated |
| Design model | Not Stated |
| Masking | Not Stated |
| Enrollment | 662 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Genentech, Inc. |
| Results posted | Yes |
2. Clinical Question
Population
Eligible participants had solid tumors other than metastatic melanoma or papillary thyroid cancer, or had multiple myeloma. The primary analysis population was participants who were enrolled and evaluable for BRAF V600 mutations. The record defines the number for each cancer type as the number of participants with that cancer in the total population.
Intervention
This was a screening procedure, not a treatment comparison. Tumor samples were collected and sent to a central laboratory to test for activating BRAF V600 mutations. Samples could come from archived tissue or fresh biopsies performed according to local standards.
Comparator
No comparator group is described. The registry record does not identify a randomized allocation or a control arm, so the study is not a like-for-like comparison of treatments or testing strategies.
Primary question
Among evaluable participants, what percentage had a tumor sample with a BRAF V600 mutation, considered by cancer type? The intended use was to identify mutation-positive patients for possible enrollment in separate clinical research studies of vemurafenib. The record does not report the observed percentage or the number of positive participants by cancer type.
3. Trial Design
This was a screening study intended to detect BRAF V600 mutation-positive patients for potential enrollment in clinical research studies of vemurafenib. The registry lists an enrollment of 662 and a completed status. The study began in 2012-12, and primary completion was recorded as 2013-09. These are registry milestones; they do not supply the missing primary endpoint results.
Eligible participants had solid tumors other than metastatic melanoma or papillary thyroid cancer, or had multiple myeloma. The study collected tumor material rather than assigning participants to a therapeutic intervention. Samples were sent to a central laboratory for mutation testing using bidirectional direct Sanger sequencing. Archived tumor sections or fresh biopsies performed according to local standards could be used.
The record describes one cohort, but its arm description is blank. Allocation is not stated, and there is no described randomized comparison. Therefore, the design does not support a direct comparison of treatment outcomes between groups. The primary endpoint is a percentage of participants with mutation-positive samples, considered by cancer type.
The study summary states that institutions with patients identified under the screening protocol could have potential access to the separate vemurafenib protocol MO28072. That possibility is not evidence that participants entered the separate protocol or received vemurafenib. Details about participation in that separate study are 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 | Participants With Confirmed Solid Tumors or Multiple Myeloma |
|---|---|
| STARTED | 662 |
| COMPLETED | 548 |
| NOT COMPLETED | 114 |
Reasons for not completing this period:
| Reason | Participants With Confirmed Solid Tumors or Multiple Myeloma |
|---|---|
| No sampling or biopsy procedures done | 114 |
Analysis population for the primary outcome, as defined in the registry: All the participants who were enrolled and were evaluable for the BRAF V600 mutations were included in the analysis. n is the number of participants with that type of cancer in the total population.
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: All the participants who were enrolled and were evaluable for the BRAF V600 mutations were included in the analysis.
| Characteristic | Unit | Participants With Confirmed Solid Tumors or Multiple Myeloma |
|---|---|---|
| Participants analysed | 548 | |
| Age, Continuous | years | 63.3 (SD 12.38) |
| Gender: Female | Participants | 326 |
| Gender: Male | Participants | 222 |
6. Primary Endpoint
The registry lists the primary outcome as Percentage of Participants With BRAF V600 Mutation Positivity in Tumor Samples by Cancer Type, measured over Up to 1 year.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | 95% Confidence Interval |
| Unit | percentage of participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the percentage of participants with BRAF V600 mutation positivity in tumor samples, assessed by cancer type. The registry describes testing formalin-fixed paraffin-embedded tumor samples using bidirectional direct Sanger sequencing in a central laboratory. Samples could be archived material from the initial cancer diagnosis or fresh biopsies obtained according to local standards.
The analysis population is described as enrolled participants evaluable for BRAF V600 mutations. For each cancer type, the record defines the denominator as the number of participants with that cancer in the total population. The endpoint timeframe is up to 1 year, and the endpoint description specifies a 95% confidence interval.
As a screening endpoint, mutation positivity describes how often the targeted mutation was detected in the tested samples within the study population. It is relevant to identifying potential candidates for separate research studies. It does not measure response to vemurafenib, compare treatments, or establish a clinical benefit. The registry record does not report the cancer-type-specific counts, percentages, or confidence intervals, so the observed prevalence cannot be read from the recorded endpoint details.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The primary question concerns the proportion of evaluable participants whose tumor samples tested positive for BRAF V600 mutations, with results considered by cancer type. The registry record describes the endpoint and laboratory approach, but it does not report the outcome values. In particular, the record does not give the number of positive samples, the denominators for individual cancer types, the percentages, or the confidence intervals. The registry lists two outcome measures, but the other measure’s details are not reported in the registry record.
Without those endpoint results, the positivity rate in any cancer type cannot be characterized from this record. The overall enrollment of 662 is not a substitute for the endpoint denominator: the analysis population is described as enrolled participants evaluable for mutation testing, and cancer-type denominators may differ. The number who were not evaluable is not reported in the registry record.
The study is not a randomized treatment comparison. Its screening endpoint can describe mutation detection in the tested samples, but it cannot show whether vemurafenib changes outcomes. Nor does a positive test result, by itself, establish that a participant enrolled in or benefited from a separate treatment study. Potential access to protocol MO28072 is described, but participation and results from that protocol are not reported in the registry record.
The registry’s safety summary lists 548 participants at risk for serious adverse events and 0 affected, and the same at-risk count and affected count for other adverse events. These are distinct safety summaries and do not fill the gap in the primary mutation endpoint. The deaths field is not populated with a numeric result, and no serious adverse-event terms are listed.
Consequently, the central statistical limitation for interpreting the screening question is not a difficult comparison between groups; it is the absence of the reported primary endpoint estimates. No conclusion about the frequency of BRAF V600 positivity by cancer type can be drawn from the available endpoint description alone.
9. Statistical Methods Explained
The registry describes a binary outcome: each evaluable participant’s tumor sample is classified according to whether BRAF V600 mutation positivity was detected. The primary endpoint expresses the number positive as a percentage of participants, with results considered by cancer type. The record does not provide the cancer-specific counts or percentages, so no observed positivity estimate can be interpreted from it.
What does a percentage describe?
General: A percentage for a binary outcome summarizes the share of the defined analysis population with the event of interest. Interpretation depends on the denominator as well as the number of events. In this study, the record says the analysis includes enrolled participants evaluable for mutation testing, and defines the number for a cancer type as participants with that cancer in the total population. If some enrolled participants were not evaluable, they would not be represented in that endpoint analysis under the stated definition. How many were not evaluable is not reported in the registry record.
The endpoint is based on a laboratory classification of tumor samples, not a clinical response. The record states that samples were sent to a central laboratory and tested for activating BRAF V600 mutations using bidirectional direct Sanger sequencing. Central testing describes where the analysis occurred; the registry does not report additional information about assay performance, quality-control procedures, or how uncertain test results were handled.
How should a confidence interval be read?
General: A confidence interval communicates statistical uncertainty around an estimate. A 95% confidence interval is intended to show a range of values compatible with the observed data under the method used to construct it. It does not mean that 95% of participants fall within the interval, and it does not guarantee that the interval contains the true value. In general, interval width depends on the amount of information in the data and the method used.
The endpoint description specifies a 95% confidence interval, but the interval estimates themselves and the method used to calculate them are not reported in the registry record. Without the estimate, interval limits, and denominator, neither the size nor the precision of the observed positivity rate can be assessed. The method should not be assumed from the confidence level alone.
Why does the analysis population matter?
General: A result applies most directly to the participants included in its analysis. If evaluability is related to participant or sample characteristics, excluding non-evaluable cases can affect how representative the analyzed group is. This is a general concern for studies using stored or newly collected tissue. The record permits archived sections or fresh biopsies and specifies an evaluable population, but it does not report how many samples came from each source, how many failed evaluation, or why any samples were unevaluable.
The cancer-type breakdown also matters because a pooled percentage across different cancers could obscure variation between types. The registry defines the endpoint by cancer type but does not report the cancer-specific results. No cross-cancer comparison or overall pooled estimate should be inferred from the endpoint title alone.
What can planning calculations establish?
General: A sample-size planning illustration describes the number of participants that might be needed under stated assumptions. It is not a measure of the study’s observed statistical power after data collection, and it does not turn an estimate into evidence of a treatment effect. The record’s planning description refers to a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and an effect defined as a stated fraction of the observed effect. It also distinguishes participant counts per arm from total event counts for time-to-event planning.
Those planning assumptions do not match the described screening design, which has no stated randomized allocation or like-for-like arms. Numerical planning estimates and the fractions used are not reported in the registry record. They therefore cannot establish that the screening study was designed for a particular precision or comparison. In general, planning figures should be read as conditional illustrations based on their assumptions, not as observed findings.
Finally, no inferential analysis results are listed in the registry record. It reports zero analyses. This leaves no reported statistical test, adjusted estimate, or model result to interpret for the primary endpoint. The stated endpoint and laboratory procedure explain what the study sought to measure, while the missing numerical outcome results prevent assessment of the actual estimate and its precision.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists two outcome measures, but only the primary endpoint’s title and description are available in the record. The other outcome measure’s definition, timing, and analysis are not reported in the registry record. No analyses are listed, and no statistical analysis plan or multiplicity procedure is reported. It is therefore not possible to determine whether multiple outcomes or cancer-type-specific estimates were treated as a family of tests, or whether any adjustment was planned or used. No adjustment should be assumed.
The primary endpoint population is described as enrolled participants evaluable for BRAF V600 mutations. The record does not report the number who were unevaluable, reasons for missing or unusable samples, or how those cases were handled. It does not describe imputation or another method for addressing missing mutation results. The enrollment of 662 should not be treated as the denominator for every cancer type because the analysis definition refers to evaluable participants and cancer-specific counts.
The record also does not report a prespecified analysis population beyond the stated evaluable group, nor does it give details about exclusions after enrollment. Without the outcome counts and these missing-data details, the impact of incomplete testing on the reported percentages cannot be assessed. The endpoint results and associated uncertainty are not reported in the registry record.
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 |
|---|---|---|---|
| Participants With Confirmed Solid Tumors or Multiple Myeloma | 0 | 548 | 0.0% |
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 |
|---|---|
| Participants With Confirmed Solid Tumors or Multiple Myeloma | 0 of 548 |
The registry safety summary names a group of participants with confirmed solid tumors or multiple myeloma. It lists 548 participants at risk for serious adverse events and 0 affected. For other adverse events, it also lists 548 at risk and 0 affected. These are counts in the registry summary; they are not results for the primary mutation endpoint.
Serious adverse events and other adverse events are shown as separate categories. The registry record does not provide event terms in its list of top serious terms. The deaths entry is not a numeric count, so deaths are not reported in the registry record. No conclusions about how events were defined, collected, or followed should be added beyond what the record states.
A recorded count of 0 affected means no affected participants are listed in that particular summary. It does not establish that no events occurred outside the scope or reporting represented by the registry entry. The at-risk count of 548 is the safety denominator recorded for each category; it should not be replaced with the overall enrollment figure.
12. What the Result Does, and Does Not, Mean
The study’s statistical purpose was to screen tumor samples for BRAF V600 mutation positivity and describe the percentage positive by cancer type. The registry record identifies the testing method and the intended analysis population, but it does not report the observed positive counts, percentages, or confidence intervals. As a result, the central screening result cannot be quantified from the record.
Even if a mutation-positive result were reported, its meaning would be limited to detection in the tested samples and the population represented by the analysis. A screening result is not a measure of treatment response or survival, and it does not compare vemurafenib with another treatment. The study summary describes potential access to a separate vemurafenib research protocol, but does not report whether participants entered that protocol or what happened to them there.
The completed status and enrollment figure describe the study’s administrative record, not the missing endpoint findings. Similarly, the safety counts are separate from mutation detection and cannot answer how often the mutation was found. No treatment effect, predictive value, or benefit can be inferred from the endpoint description alone.
In practical statistical terms, the record identifies the question and a method for measuring it but leaves out the numerical results needed to answer it. The percentages and their precision by cancer type are not reported in the registry record. Interpretation should therefore remain descriptive of the study’s objective and design rather than asserting a result that the registry does not contain.
13. Why This Trial Matters Statistically
This study is statistically distinct from a conventional efficacy trial. Its primary aim was to identify participants with a tumor mutation for possible enrollment in separate clinical research studies. The central quantity of interest is therefore the proportion of evaluable participants with positive tumor samples, considered by cancer type, rather than a difference in clinical outcomes between treatment groups.
That distinction matters because the interpretation depends on the tested population, evaluability, and cancer-specific denominators. The record defines an evaluable population and describes central laboratory testing, but does not report the positive counts, denominators by cancer type, or confidence intervals. These missing values prevent readers from assessing either the observed mutation frequency or its statistical precision.
The study also illustrates why a screening result should not be conflated with a treatment result. Identifying a mutation may support selection for further research, but the screening endpoint does not measure whether a therapy changes an outcome. The record’s mention of potential access to a separate protocol does not supply results from that research.
Finally, the absence of a stated comparator and randomization is appropriate to recognize when reading the record. The listed safety summary and completed status do not create a treatment comparison. Clear separation of screening, treatment evaluation, and safety reporting helps prevent claims that go beyond the outcomes actually documented.
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 statistical interpretation is that the registry record does not report the primary endpoint values. No positive counts, cancer-specific denominators, percentages, or confidence intervals are given. The record lists two outcome measures but does not report the second measure’s details. It also lists no analyses, leaving no test results or modeled estimates to evaluate.
The analysis is described as including participants enrolled and evaluable for mutation testing, but the number of non-evaluable participants and the reasons for non-evaluability are not reported in the registry record. The handling of missing or unusable samples is also not described. These omissions limit assessment of how complete the testing was and whether the analyzed participants represent everyone enrolled.
The study has no stated randomization or comparator, so it cannot support a comparative treatment-effect interpretation. The registry describes one cohort with a blank arm description. The sample-size planning assumptions refer to equal allocation between two arms, but the numerical planning estimates are not reported in the registry record and do not establish the design of this screening study.
Additional details about assay performance, quality control, and the handling of uncertain laboratory findings are not reported in the registry record. The record also does not establish whether screened participants proceeded to the separate vemurafenib protocol or provide outcomes from it. These limitations leave the objective and testing approach clear, while limiting conclusions about results, representativeness, and downstream clinical outcomes.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: A Screening Study to Detect BRAF V600 Mutation-Positive Patients For Enrollment Into Clinical Research Studies of Zelboraf (Vemurafenib) — NCT01804140.
- Registry record last updated 2016-11-02; status last verified 2016-11.
- Results first posted 2015-07-31.
- 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
NCT01804140 is a completed screening study sponsored by Genentech, Inc., with enrollment of 662. The study began in 2012-12, and primary completion was recorded as 2013-09. It sought to detect BRAF V600 mutations in tumor samples from eligible participants with certain solid tumors or multiple myeloma.
Samples were tested at a central laboratory using bidirectional direct Sanger sequencing. The primary endpoint was the percentage of evaluable participants with mutation-positive samples by cancer type, with a 95% confidence interval and a timeframe of up to 1 year. The registry record does not report the endpoint results or the second outcome measure’s details. It describes potential access to a separate vemurafenib protocol but does not report participation or results from that protocol.
The safety summary lists 548 participants at risk and 0 affected for both serious and other adverse events. The deaths count is not reported in the registry record.