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 Dabrafenib and Trametinib in Treating Patients With BRAF Mutated Ameloblastoma (NCT02367859), a na phase 2 study. The primary outcome is Tumor Response.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02367859 |
| Phase | Phase 2 |
| Condition | Ameloblastoma, BRAF Gene Mutation |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 1 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Stanford University |
| Results posted | Yes |
2. Clinical Question
Population
Patients with ameloblastoma and a BRAF gene mutation were the intended population. The registry record lists an enrollment of 1. It does not give further eligibility details or describe the characteristics of the enrolled participant, so the fit between the enrolled participant and a broader clinical population cannot be assessed from this record.
Intervention
The study evaluated dabrafenib together with trametinib. The arm description states that dabrafenib was taken by mouth twice daily, every 12 hours, and trametinib by mouth daily for 6 weeks, in the absence of disease progression or unacceptable toxicity. It also describes continued treatment for patients judged not amenable to resection, but the registry text is incomplete at that point.
Comparator
This was a single-group study, with no randomized control group identified in the record. Allocation is listed as not applicable, and masking as none. The study therefore does not offer a like-for-like comparison against another treatment or a control condition.
Primary question
The primary outcome was tumor response at 6 weeks, assessed using RECIST v1.1. It was defined as the number of participants achieving a complete response or partial response. The registry record does not report the response count, so the primary outcome cannot be described as an observed result from this record.
The clinical question is whether tumor response was observed during treatment in this small, single-group study of patients with BRAF-mutated ameloblastoma. Without a reported response count, the registry record does not establish what response category was observed. Without a comparator, any response that might have occurred could not be attributed to treatment by a randomized between-group contrast. The trial is best understood as a pilot investigation, not as a comparative estimate of treatment effect.
3. Trial Design
The registry classifies this as a Phase 2, single-group trial. It is marked completed, with a start date of 2017-07-17 and a primary completion date of 2018-11-05. Stanford University is listed as the sponsor. The record gives an enrollment of 1 and describes the condition as ameloblastoma with a BRAF gene mutation.
The treatment arm combined dabrafenib and trametinib. The arm description specifies oral dabrafenib twice daily, every 12 hours, with oral trametinib daily for 6 weeks unless disease progression or unacceptable toxicity occurred. It further states that treatment could continue for patients judged not amenable to resection, but the continuation description ends mid-sentence in the registry record. Details beyond the text that is recorded should not be inferred.
There was no randomized allocation and no masking. With a single group and no control comparison, the design can describe what was recorded among participants receiving the regimen, but it cannot separate a treatment-associated change from other explanations through a randomized contrast. The record does not report a protocol-level statistical plan, a sample-size justification, or details about how eligibility and outcome assessments were implemented.
The primary outcome was tumor response at 6 weeks. Secondary outcomes named in the record were percent tumor necrosis, change in proliferation, and phosphorylation of the tumor markers MEK and ERK. The record identifies four outcome measures overall, but it does not give the primary response count or results for those secondary outcomes in the registry record for this analysis. There are no analysis records listed. Thus the design and outcome definitions can be described, while the outcome findings and inferential analyses cannot be reconstructed from 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 | Treatment (Dabrafenib) |
|---|---|
| STARTED | 1 |
| COMPLETED | 1 |
| NOT COMPLETED | 0 |
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 | Treatment (Dabrafenib) |
|---|---|---|
| Participants analysed | 1 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 1 |
| Age, Categorical: >=65 years | Participants | 0 |
| Sex: Female, Male: Female | Participants | 0 |
| Sex: Female, Male: Male | Participants | 1 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 1 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 1 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 |
| Race (NIH/OMB): White | Participants | 0 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Region of Enrollment: United States | participants | 1 |
6. Primary Endpoint
The registry lists the primary outcome as Tumor Response, measured over 6 weeks.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint was tumor response at 6 weeks, expressed as the number of participants in each response category under RECIST v1.1. The stated overall-response measure counts participants with either a complete response or a partial response. A complete response means disappearance of all target lesions. A partial response requires a decrease of at least 30% in the sum of the longest diameters of target lesions. The record also defines progressive disease using an increase of 20% in that sum and/or the appearance of one or more new lesions; stable disease describes changes that do not meet the other criteria.
RECIST provides a structured way to classify changes in measured tumors, which can make response reporting more consistent than an informal description. The endpoint is a participant count, not a continuous measurement of tumor size, and its meaning depends on applying the stated criteria at the specified assessment time. A count alone does not show how precisely a response rate has been estimated; that requires the relevant denominator and an uncertainty measure.
The registry record does not report the number of participants achieving complete response, partial response, or overall response. It therefore supplies an endpoint definition but not an observed primary endpoint result. The response count, denominator used for analysis, handling of unavailable assessments, and any uncertainty interval are not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The record supports a description of the study design and its intended primary outcome, but not a conclusion about the observed tumor-response result. Although enrollment is listed as 1, no primary response count is reported. It is therefore not possible to tell from this record whether a complete response, partial response, stable disease, or progressive disease was recorded at the 6-week assessment. The endpoint definition should not be mistaken for a result.
The study was single-group, with allocation listed as not applicable and masking listed as none. There was no randomized comparator. Consequently, even if a response result were available, this design would not provide a randomized estimate of how outcomes differed from a control group. Changes in an individual participant over time can be clinically and scientifically informative, but a before-and-after observation does not by itself establish why the change occurred.
Precision is also a central concern. A confidence interval is a range that communicates the uncertainty around an estimate under a statistical model. The registry record does not report an interval for the primary endpoint, and the response count itself is absent. There is therefore no reported numerical estimate whose precision can be evaluated. With the listed enrollment of 1, a single participant's outcome would represent the entire recorded enrollment, which makes any group-level interpretation especially dependent on that individual's result and on the analysis definition.
The safety fields record 0 serious adverse events and 1 event in the other adverse-event category for the treatment group, with 1 at risk in each category. These are descriptive registry counts; they do not establish whether an event was caused by treatment, how it was defined, or whether all safety outcomes were captured. The record lists 0 deaths.
No analyses are listed in the registry record. It does not report a statistical comparison, a confidence interval, a p-value, or adjustment for covariates. Without reported primary outcome findings and an analysis, the evidence available in this record is insufficient to quantify a treatment effect or its uncertainty. The appropriate reading is limited to the planned outcome, design, listed enrollment, and recorded safety counts.
9. Statistical Methods Explained
Statistical interpretation begins by distinguishing the study design from its outcome definitions and from the results actually reported. This trial is classified as single-group and Phase 2. Its primary endpoint is a count of participants meeting a response definition at 6 weeks. These registry entries describe what the study set out to assess. They do not supply the count of responders or a statistical estimate of response.
What does a participant count tell us?
A count records how many participants fall into a defined category. For a response endpoint, the category is based on the RECIST v1.1 criteria in the registry record. Counts are straightforward to interpret when the analysis population and denominator are clear. To interpret a response frequency as a proportion, a reader also needs to know which participants were included in the denominator and how participants without an evaluable assessment were treated. Those details are not reported in the registry record.
A count and a proportion are not interchangeable. A count gives the number of observed participants in a category; a proportion relates that count to the participants assessed under a defined analysis rule. The registry describes the primary outcome as a participant count and says it is reported without dispersion. It does not report a response count, a denominator for response, or an uncertainty interval. No response proportion should therefore be inferred from the endpoint description alone.
Why does uncertainty matter?
General statistical explanation: an estimate based on a small amount of information can be highly sensitive to the observation of an individual participant. A confidence interval communicates the uncertainty associated with an estimate under its model assumptions. It does not guarantee that the true value lies in the interval, and it does not resolve limitations of design or measurement. The registry record does not report a confidence interval for the primary endpoint.
A p-value, when reported, addresses how compatible observed data are with a specified statistical model and null hypothesis. It is not a direct measure of clinical importance, the probability that a hypothesis is true, or the probability that a result will reproduce. No p-value or inferential analysis is listed in this record. This absence should be read as missing reporting, not as evidence that a test found no effect.
What can a single-group design establish?
General statistical explanation: a randomized comparison can help distinguish the effect of an intervention from other causes of outcome differences by creating comparable groups in expectation. A single-group study does not provide that between-group contrast. Descriptive observations can document what happened to participants receiving the regimen, but interpretation may also depend on the course of disease, measurement variation, selection, and other factors. The registry identifies no randomized comparator, and the record cannot support a comparative treatment-effect estimate.
The study has no masking listed. Masking can reduce some opportunities for expectations to influence treatment, assessment, or reporting, depending on the design and outcome. Its absence is a feature of this record, but the registry does not give enough detail to determine how response assessments were conducted or whether any safeguards were used.
How should the response criteria be read?
RECIST v1.1 converts measurements of target lesions and the appearance of new lesions into response categories. The record defines complete response as disappearance of all target lesions and partial response as a decrease of at least 30% in the sum of the longest diameters. It defines progressive disease by an increase of 20% in that sum and/or one or more new lesions. Stable disease applies when changes do not meet the listed criteria for the other categories. These rules make the endpoint operational, but the record does not include the measurements or category assignments needed to verify an outcome.
For a binary summary such as overall response, complete and partial response are grouped together. Combining categories can make an endpoint easier to summarize, but it also hides distinctions between them. Here, the primary endpoint description specifies the combined count, while the actual category counts are not reported. The relative contribution of complete and partial responses therefore cannot be evaluated.
What does the record say about analyses?
The registry lists no analysis records. It does not report an inferential model, an adjustment strategy, an analysis population, or a method for handling missing outcome assessments. It also does not report an interim-analysis plan or sample-size justification. These omissions mean that readers cannot assess whether a particular analysis followed a prespecified plan or how sensitive the findings might be to analysis choices.
General statistical explanation: over-dispersion matters when observed variability exceeds what a chosen model expects, and may lead to uncertainty estimates that are too narrow if ignored. Its relevance depends on the outcome and model. No model or analysis is reported for this endpoint, so it is not possible to assess whether over-dispersion was considered or whether it would have been relevant to the analysis conducted.
The registry's listed enrollment and safety counts are descriptive record entries. They do not substitute for the missing primary outcome result or an analysis of that result. Any planning illustration based on an observed effect is distinct from observed power and from evidence of a comparative effect. The record does not provide a numerical primary result in the registry record for interpretation, so such an effect cannot be independently assessed.
10. Multiplicity, Missing Data and Analysis Populations
The registry names a primary outcome and three secondary outcomes: percent tumor necrosis, change in proliferation, and phosphorylation of MEK and ERK. It also lists four outcome measures overall. When a study assesses multiple outcomes, the chance of at least one apparently unusual result can depend on how many tests are conducted and how they are interpreted. A multiplicity plan can specify which outcomes are confirmatory and whether statistical error rates are adjusted. No multiplicity strategy is reported in the registry record, and no analyses are listed.
The record does not report an analysis population for the primary or secondary outcomes. In particular, it does not say whether analyses were intended to include all enrolled participants, only those with an evaluable assessment, or another defined group. This matters because the denominator determines how a participant count is interpreted.
Missing outcome data and the handling of unavailable assessments are not reported in the registry record. The record does not state whether missing values were imputed, excluded, treated as nonresponse, or handled by another method. No particular approach should be assumed. Likewise, an interim-analysis plan and stopping rules are not reported in the registry record.
These are reporting gaps, not evidence that a particular analysis or adjustment was or was not performed outside the registry. Without a stated analysis population, missing-data approach, and multiplicity plan, readers cannot evaluate how those choices might affect the interpretation of the primary and secondary outcomes. Most importantly, the registry record does not provide the primary response count needed to interpret the stated endpoint.
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 |
|---|---|---|---|
| Treatment (Dabrafenib) | 0 | 1 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Treatment (Dabrafenib) | 0 of 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 |
|---|---|
| Treatment (Dabrafenib) | 1 of 1 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Treatment (Dabrafenib) |
|---|---|---|
| Flu like symptoms | General disorders | 1 of 1 |
| Headache | Nervous system disorders | 1 of 1 |
| Fatigue | General disorders | 1 of 1 |
| Myalgia | Musculoskeletal and connective tissue disorders | 1 of 1 |
| Rash acneform | Skin and subcutaneous tissue disorders | 1 of 1 |
| Weight loss | Investigations | 1 of 1 |
| Anorexia | Metabolism and nutrition disorders | 1 of 1 |
| Dermatologic syndrome | Skin and subcutaneous tissue disorders | 1 of 1 |
For the treatment group, the registry records 0 serious adverse events among 1 at risk in that category. It records 1 event in the other adverse-event category among 1 at risk in that category, and 0 deaths. These are counts as recorded in the registry, not comparative estimates of risk.
The record does not provide the specific term for the other adverse event; no serious adverse event terms are listed. It also does not report event timing, duration, severity beyond the serious-versus-other classification, or whether the event was judged related to treatment. A recorded event should not be assumed to have been caused by the regimen, and the absence of a recorded serious event is not proof that no such event could occur.
The safety counts are limited by the enrollment listed in the record and by the absence of detail about safety collection and reporting. They cannot characterize the frequency or range of adverse events in a broader population. The registry record does not describe a comparison group, so these counts cannot be contrasted with an untreated or alternative-treatment group.
12. What the Result Does, and Does Not, Mean
The registry record describes a pilot Phase 2 study of dabrafenib and trametinib for patients with BRAF-mutated ameloblastoma. It establishes the stated treatment schedule, the single-group design, the RECIST v1.1 primary endpoint, and the enrollment listed as 1. It also records safety counts for the treatment group. These are useful pieces of information about what the trial was intended to study and what the registry reports.
The record does not report the primary tumor-response count. As a result, it does not establish which response category was observed at 6 weeks or how many participants met the overall-response definition. The endpoint's criteria are not evidence that a response occurred. The record also does not provide a confidence interval, p-value, or reported statistical analysis for that outcome.
Even a reported response in a single-group study would be a descriptive observation, not a randomized estimate of the effect of treatment compared with a control. The absence of a comparator limits causal interpretation, while the listed enrollment and missing response result prevent assessment of the primary outcome from this record. The record cannot establish how representative the enrolled participant was of people with ameloblastoma and a BRAF mutation.
The safety entries should also be read narrowly: they describe events recorded in the registry, not a complete characterization of safety or proof of causation. The broader implication is that the available registry information identifies a research question and design, but leaves the main efficacy result and important analytic details unreported. Neither a positive nor a negative treatment conclusion can be drawn from the primary outcome information in this record.
13. Why This Trial Matters Statistically
This study is statistically notable for the gap between a clearly defined primary endpoint and the absence of a reported primary result. The registry specifies tumor response at 6 weeks under RECIST v1.1 and defines the categories used to construct overall response. Yet it does not report the number achieving complete response or partial response. Readers can understand the intended measurement, but cannot assess the observed result or its precision.
The single-group design is important when interpreting any observations. Without randomized allocation to a comparator, outcomes cannot be contrasted in a way that isolates a treatment effect from other possible influences. This distinction is especially important in a pilot study, where the study's structure and scale may make descriptive learning more relevant than a definitive comparative estimate. The registry does not report a protocol-level statistical plan or sample-size justification, so the intended inferential role of the study is not fully documented.
The record also illustrates why the denominator, analysis population, missing-data rules, and outcome reporting matter. A count has limited interpretive value unless readers know how assessments and participants were handled. Those details are not reported. The safety fields are similarly descriptive: they record event counts, but not attribution or detailed event characterization.
These limitations do not make the study question unimportant. They define what can responsibly be learned from this registry record: the intervention, design, endpoint definition, listed enrollment, and recorded safety counts are available, while the primary finding and key analysis details are not. Clear separation of design details from outcome evidence is essential when drawing conclusions from a small pilot record.
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 most direct limitation is that the registry record does not report the primary tumor-response count. The primary endpoint is defined, but no complete-response, partial-response, or overall-response result is given. The record therefore does not support a numerical efficacy interpretation or an assessment of precision.
The listed enrollment is 1, and the study has a single group with no randomized comparator. The record cannot provide a randomized comparison or establish that any observed change was caused by treatment. It also does not report participant characteristics beyond the intended population of patients with ameloblastoma and a BRAF gene mutation, limiting assessment of how broadly the record might apply.
The registry text describing continued treatment for patients judged not amenable to resection is incomplete. The details of treatment after the stated 6-week period therefore cannot be fully characterized from the record. The record also does not report the analysis population, missing-data handling, outcome-assessment completion, or a statistical plan. It gives no reported analysis of the primary endpoint.
For safety, the record includes counts but no specific term for the other adverse event, no serious-event terms, and no details about attribution or timing. These omissions limit interpretation of the safety entries. The record's completed status does not fill gaps in outcome reporting or analytical detail.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Dabrafenib and Trametinib in Treating Patients With BRAF Mutated Ameloblastoma — NCT02367859.
- Registry record last updated 2024-01-30; status last verified 2024-01.
- Results first posted 2020-01-21.
- 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
NCT02367859 is a completed, Phase 2, single-group study sponsored by Stanford University. The registry lists an enrollment of 1 and evaluates dabrafenib with trametinib in patients with ameloblastoma and a BRAF gene mutation. The primary outcome is tumor response at 6 weeks under RECIST v1.1.
The primary response count is not reported in the registry record. No analysis records are listed. The record names secondary outcomes for tumor necrosis, proliferation, and phosphorylation of MEK and ERK, but does not report their results in the information available for interpretation. Safety entries include 0 serious adverse events, 1 event in the other adverse-event category, and 0 deaths.
The single-group design does not provide a randomized control comparison. The registry record describes the question and endpoint but does not establish a treatment effect or report the primary outcome result needed to assess response.