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 Dasatinib in Patients With Isocitrate Dehydrogenase (IDH)-Mutant Advanced Intrahepatic Cholangiocarcinoma (NCT02428855), a na phase 2 study. The primary outcome is Objective Response Rate (ORR).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02428855 |
| Phase | Phase 2 |
| Condition | Cholangiocarcinoma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 8 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Massachusetts General Hospital |
| Results posted | Yes |
2. Clinical Question
Population
Patients with advanced intrahepatic cholangiocarcinoma whose tumors had an IDH1 or IDH2 mutation and who had received at least one prior platinum-containing regimen. The registry lists cholangiocarcinoma as the condition and records enrollment of 8 participants.
Intervention
Dasatinib, taken orally each day at a predetermined dosage per cycle. Radiologic response assessment was scheduled every 2 cycles.
Comparator
No concurrent comparator group is described. The registry identifies the study model as single-group and reports allocation as not applicable.
Primary question
How many participants achieved a complete or partial response, assessed using RECIST 1.1 at the 8 Weeks timeframe?
The clinical question is therefore about observed tumor response among a selected group receiving dasatinib, not about a randomized comparison with another treatment or with no treatment. The primary endpoint is a count of participants meeting either response category. The registry does not report that count or an objective response rate, so the primary question cannot be answered from the outcome information in the registry record.
Progression-free survival, overall survival, and the number of participants with adverse events are listed as secondary outcomes. Their listing identifies outcomes of interest but does not itself establish what was observed. Results for those secondary outcomes are not reported in the registry record.
3. Trial Design
This was a Phase II, single-group study of dasatinib in patients with advanced intrahepatic cholangiocarcinoma and an IDH1 or IDH2 mutation. Eligibility also required receipt of at least one prior platinum-containing regimen. The registry lists 8 participants as enrolled and the study status as completed.
The intervention was oral dasatinib, taken daily at a predetermined dosage per cycle. The registry states that radiologic response assessment occurred every 2 cycles. The exact dosage, cycle duration, treatment duration, and assessment procedures beyond that description are not reported in the registry record.
The registry describes no random allocation and no masking. Its allocation field is recorded as not applicable, and its model is single-group. Consequently, the design does not provide a concurrent control group for estimating how outcomes would have differed under another treatment or under no study treatment. Outcomes observed in this group can be described, but treatment effects relative to a control cannot be isolated from the information reported.
The study start is recorded as 2015-04, and primary completion as 2018-02. The registry lists objective response rate as the primary outcome, with a timeframe of 8 Weeks, and names progression-free survival, overall survival, and the number of participants with adverse events as secondary outcomes. Although the record lists 4 outcome measures, it does not report numerical results for the primary or secondary outcomes. The dates and completed status describe the trial record; they do not supply missing outcome estimates or details of the statistical analysis.
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 | Dasatinib |
|---|---|
| STARTED | 8 |
| COMPLETED | 6 |
| NOT COMPLETED | 2 |
Reasons for not completing this period:
| Reason | Dasatinib |
|---|---|
| Withdrawal by Subject | 2 |
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 | Dasatinib |
|---|---|---|
| Participants analysed | 8 | |
| Age, Continuous | years | 59 (45 to 73) |
| Sex: Female, Male: Female | Participants | 6 |
| Sex: Female, Male: Male | Participants | 2 |
| Race and Ethnicity Not Collected | Participants | — |
| Region of Enrollment: United States | participants | 8 |
| Tumor location: Intrahepatic | Participants | 7 |
| Tumor location: Hilar | Participants | 1 |
| IDH Mutation: IDH1 | Participants | 7 |
| IDH Mutation: IDH2 | Participants | 1 |
6. Primary Endpoint
The registry lists the primary outcome as Objective Response Rate (ORR), measured over 8 Weeks.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the number of participants who achieved either a complete response or a partial response, assessed using Response Criteria in Solid Tumors (RECIST 1.1). Its stated timeframe is 8 Weeks. A complete response is defined as disappearance of all target lesions, with any pathological lymph nodes reduced in short axis to <10 mm. A partial response requires at least a 30% decrease in the sum of the diameters of target lesions, using the baseline sum of diameters as the reference.
Objective response is used in oncology trials to describe measurable tumor shrinkage according to prespecified criteria. It provides a categorical endpoint: participants either meet the stated complete- or partial-response definition or do not. Reporting the count can make the observed response experience clear for the study group, while an estimated response proportion and its uncertainty would help convey precision. The registry record does not report a response count, a response proportion, or an uncertainty interval, so these cannot be assessed from the listed endpoint definition alone.
The registry's endpoint is a count of participants, not a measure of how long response lasted or whether participants lived longer. Progression-free survival and overall survival appear as separate secondary outcomes. Results for those outcomes are not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry describes a completed Phase II study with a single-group model and enrollment of 8 participants. Its primary endpoint is the count of participants with a complete or partial response by RECIST 1.1 at 8 Weeks. However, the registry record does not report the observed response count or an objective response rate. It therefore does not establish how many participants met the response definition, nor how precise any response estimate would be.
The design matters when interpreting any outcomes. A single-group study can describe what happened among participants who received dasatinib, but it does not offer a concurrent comparator for separating treatment-associated outcomes from the natural course of disease, prior treatment, participant characteristics, or other influences. The registry reports no randomized comparison. As a result, a response observation, if reported elsewhere, would not by itself establish a comparative treatment effect.
Three secondary outcomes are named: median progression-free survival, overall survival, and the number of participants with adverse events. Their results are not reported in the registry record. No survival estimate, follow-up summary, or adverse-event outcome beyond the adverse-event figures described separately can be inferred from their inclusion as outcome titles. In particular, the registry does not provide enough information to assess the timing or completeness of survival follow-up.
The safety record lists serious adverse events for 8 of 8 participants in the Dasatinib group, and lists 0 of 8 participants in the other adverse-event category. It also records 7 deaths. These are descriptive registry counts. The record does not establish a causal relationship between dasatinib and the reported events or deaths, and does not provide the timing, circumstances, or attribution needed for such interpretation. Serious-event terms and their listed percentages describe reported categories, not an overall measure of risk that can be compared with another group.
Overall, the posted information supports description of the study's design, endpoint definition, and selected safety entries, but not an estimate of the primary response outcome or a comparative conclusion. The statistical interpretation is limited by the absence of reported efficacy results, analysis details, and comparator data. A null or positive treatment effect should not be inferred from the absence of a reported response result.
9. Statistical Methods Explained
The registry identifies objective response rate as the primary outcome, but describes its measured quantity as a count of participants achieving a complete or partial response. That distinction is useful: the endpoint definition explains what is counted, whereas a response rate would express the count relative to an analysis population. The registry record does not report the response count, the analysis population for this endpoint, or a response estimate. No recalculated response estimate can therefore be interpreted from the information reported.
What does the response definition mean?
RECIST 1.1 supplies the criteria used to classify tumor response. In this record, complete response requires disappearance of all target lesions and reduction of pathological lymph nodes to <10 mm in short axis. Partial response requires at least a 30% decrease in the sum of target-lesion diameters compared with the baseline sum. These criteria make the endpoint more reproducible than an informal judgment of improvement, because they specify what change qualifies. The registry does not give additional details about imaging review, independent review, handling of unevaluable assessments, or confirmation of response.
How should uncertainty be understood?
In general, a confidence interval describes a range of values compatible with the observed data under a statistical model. It conveys how imprecisely an estimate may be measured; it does not represent a guarantee that the true value lies in the interval. For a small single-group study, an estimate based on few observations may be particularly uncertain. This is a general statistical consideration, not a reported finding about the response estimate in this trial. The registry record does not report a response estimate or confidence interval to interpret.
Likewise, a p-value addresses compatibility between data and a specified statistical hypothesis under stated assumptions. It is not a measure of clinical importance and does not provide the probability that a treatment works. No primary-outcome p-value or inferential analysis is reported in the registry record. The record lists no analysis entries, so the analysis model, testing procedure, and any adjustment for covariates are not reported.
What does a single-group design allow?
In general, a single-group study can estimate outcomes observed in its participants, subject to the way the outcome and analysis population are defined. It cannot directly estimate a randomized between-group contrast because no concurrent comparator group is described. Any comparison with an external benchmark would require details about that benchmark and assumptions about comparability; those are not reported in the registry record. Thus, the design supports descriptive interpretation more directly than causal or comparative interpretation.
The registry's sample-size assumptions describe planning illustrations based on an observed effect, rather than the trial's own design. They use a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; the effect to detect is a stated fraction of the observed effect. For time-to-event outcomes, the illustration concerns the total number of events using the Schoenfeld method; other illustrations concern participants per arm. These assumptions are not a description of the actual single-group trial, and the resulting planning values are not observed power. The registry record does not report a protocol sample-size justification or the study's own planned statistical design.
In general, over-dispersion arises when observed variability exceeds what a statistical model assumes. If present and not addressed, uncertainty estimates can be too narrow. The registry does not report a model for counts, dispersion assessment, or any analysis for over-dispersion, and the binary response endpoint has no outcome result presented for such assessment. This is a general methodological point, not evidence that over-dispersion occurred in this study.
For time-to-event outcomes, methods generally need to account for censoring: a participant's event time may be unknown beyond the last time they were observed. The registry lists progression-free survival and overall survival but does not report definitions of their event and censoring rules, follow-up completeness, or analysis methods. A median or other survival result cannot be reconstructed from the outcome titles alone.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists objective response rate as the primary outcome and identifies progression-free survival, overall survival, and the number of participants with adverse events as secondary outcomes. It also records 4 outcome measures. Multiple outcomes can create multiplicity concerns when several statistical tests are used to support claims: the chance of at least one apparently noteworthy finding can increase as more tests are considered. In general, an analysis plan may address this through prespecified priorities, adjustments, or careful interpretation. The registry does not report whether any multiplicity strategy was planned or used, and the record lists no analysis entries.
Missing-data handling is not reported in the registry record. The record does not state whether participants without an evaluable scan were included in an analysis, how missing assessments were treated, or whether any sensitivity analysis was performed. The analysis population for the primary endpoint is also not reported. These details matter because a response count can depend on which participants are considered evaluable and how incomplete outcome information is handled.
The registry describes the response assessment timeframe as 8 Weeks, but does not report assessment completion, timing deviations, or rules for participants who discontinued or died before assessment. It is therefore not possible to determine from the registry record whether missingness could have affected the endpoint estimate.
These are reporting gaps, not evidence that a particular method was or was not used in the trial. The absence of a registered analysis description also means that prespecification, statistical model, interim analyses, and analysis-population definitions are not reported in the registry record. Without those details and the outcome results, readers cannot evaluate whether the reported analyses followed a prespecified plan.
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 |
|---|---|---|---|
| Dasatinib | 8 | 8 | 100.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Dasatinib | 7 of 8 |
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 |
|---|---|
| Dasatinib | 0 of 8 |
Most frequent serious adverse event terms
| Event term | Organ system | Dasatinib |
|---|---|---|
| Lymphopenia | Blood and lymphatic system disorders | 4 of 8 |
| Anemia | Blood and lymphatic system disorders | 1 of 8 |
| Hyponatremia | Metabolism and nutrition disorders | 1 of 8 |
| Hypophosphatemia | Metabolism and nutrition disorders | 1 of 8 |
| Aspartate aminotransferase increased | Investigations | 1 of 8 |
| Menorrhagia | Reproductive system and breast disorders | 1 of 8 |
Serious events with the highest arm-level rate: lymphopenia (50.0%), anemia (12.5%), hyponatremia (12.5%), hypophosphatemia (12.5%), aspartate aminotransferase increased (12.5%).
The registry records serious adverse events for 8 of 8 participants in the Dasatinib group. For the other adverse-event category, it records 0 of 8 participants. It also records 7 deaths. These entries are counts in the registry; the record does not report the timing of events, individual event histories, or whether events were judged related to dasatinib. The deaths should therefore not be interpreted as treatment-caused from these figures alone.
The serious adverse-event terms listed with frequencies are lymphopenia, 50.0%; anemia, 12.5%; hyponatremia, 12.5%; hypophosphatemia, 12.5%; aspartate aminotransferase increased, 12.5%; and menorrhagia, 12.5%. The percentages describe the listed terms in the registry record. They do not establish severity beyond the serious-event classification, duration, clinical course, or causal attribution. The record does not explain whether an individual could contribute to more than one term or provide definitions for the other adverse-event category.
These data are descriptive and come from a single-group study. Without a concurrent comparator and more detailed event information, they cannot quantify excess risk attributable to dasatinib or support a comparative safety assessment. The registry record does not report additional context needed to interpret the deaths or adverse-event timing.
12. What the Result Does, and Does Not, Mean
The trial record identifies a focused clinical question: whether participants with advanced intrahepatic cholangiocarcinoma and IDH1 or IDH2 mutations who had received prior platinum-containing treatment achieved a RECIST 1.1 complete or partial response while receiving dasatinib. It documents a Phase II, single-group design and records enrollment of 8 participants. These registry entries describe the setting and design, but they are not evidence of the size or precision of an observed response.
The primary outcome is defined, but its result is not reported in the registry record. Without the response count, a response rate, confidence interval, or statistical test cannot be interpreted. The named secondary outcomes likewise do not supply results for progression-free survival or overall survival. A completed study status does not fill these outcome-reporting gaps.
The adverse-event entries provide descriptive counts and listed serious-event terms. They do not establish that dasatinib caused any particular event or death. The single-group design also lacks a concurrent comparison that could help distinguish outcomes associated with treatment from other influences.
In general, trial evidence is interpreted by considering the endpoint definition, analysis population, uncertainty, study design, missing data, and potential biases together. Here, several of those elements—including the primary outcome result and analysis details—are not reported in the registry record. The appropriate conclusion is consequently limited: the registry documents the study and selected safety information, but does not permit a statistical assessment of the primary efficacy result or a comparative estimate of treatment effect. No conclusion about whether the intervention does or does not work follows from the missing result.
13. Why This Trial Matters Statistically
This study illustrates why an endpoint definition and an endpoint result are different kinds of information. The registry specifies how complete and partial responses were classified, including measurable criteria, but does not report how many participants met them. A carefully defined outcome helps make assessment interpretable; it cannot substitute for the observed outcome data or their uncertainty.
The single-group design is also central to statistical interpretation. It can describe outcomes among participants receiving dasatinib, but provides no concurrent randomized comparison. Even if a response result were available, the design would not by itself identify what would have occurred in a comparable group under a different treatment. This distinction helps separate descriptive evidence from comparative or causal claims.
The record also demonstrates the value of complete reporting. Outcome titles, a completed status, and safety counts do not disclose the analysis population, missing-data handling, statistical model, or inferential results. These details help readers judge how robust an estimate is and whether its uncertainty is adequately represented. Their absence limits interpretation independently of the trial's clinical topic.
Finally, the planning illustrations are not the trial's design or evidence of statistical power. The registry's assumptions describe hypothetical two-arm planning calculations, whereas the study itself is recorded as single-group. Keeping planning scenarios separate from observed results and from the actual design prevents readers from treating a sample-size illustration as evidence that the study had a particular ability to detect an effect.
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 of the registry record for interpreting efficacy is that it does not report the primary response result. Although the endpoint definition and timeframe are specified, there is no response count, response rate, or uncertainty interval. The record therefore cannot establish the observed objective response outcome.
The single-group design has no concurrent comparator. It can describe outcomes in the enrolled study group but cannot support a randomized comparison. The registry's allocation field is not applicable, and masking is listed as none. The record does not report additional design safeguards, eligibility details beyond the stated mutation and prior-treatment criteria, or the exact dosage and treatment duration.
Statistical methods are also insufficiently described for assessing analysis choices. The registry lists no analysis entries and does not report the analysis population, missing-data rules, response-assessment completeness, or a multiplicity strategy. It does not report methods for the listed survival outcomes, including event definitions and censoring rules. These absences limit assessment of bias and precision; they do not demonstrate that a particular practice was absent from the trial.
The safety figures are limited to registry counts and listed serious-event terms. Event timing, attribution, clinical course, and context for the deaths are not reported. As the study has no concurrent comparator, the entries cannot provide a comparative safety estimate.
Enrollment was 8 participants, so findings from this record would apply to a narrowly defined study group and should not be assumed to represent other populations. More importantly, no efficacy result is reported to generalize. The registry's completed status does not resolve these limitations or supply results not included in its record.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Phase II Trial of Dasatinib in Patients With Isocitrate Dehydrogenase (IDH)-Mutant Advanced Intrahepatic Cholangiocarcinoma — NCT02428855.
- Registry record last updated 2020-07-31; status last verified 2020-07.
- Results first posted 2020-07-31.
- 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
The registry describes a completed Phase II, single-group study of oral dasatinib in patients with advanced intrahepatic cholangiocarcinoma, an IDH1 or IDH2 mutation, and at least one prior platinum-containing regimen. Enrollment was 8 participants. The study start is recorded as 2015-04 and primary completion as 2018-02.
The primary outcome is the count of participants with complete or partial response by RECIST 1.1 at 8 Weeks. Progression-free survival, overall survival, and the number of participants with adverse events are listed as secondary outcomes. The primary response result and the listed survival results are not reported in the registry record.
The safety record lists serious adverse events for 8 of 8 participants, 0 of 8 in the other adverse-event category, and 7 deaths. The record does not report event attribution or timing. Its single-group design provides no concurrent comparator, and analysis methods and missing-data handling are not reported in the registry record.