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 Study of Dovitinib and Biomarkers in Advanced Non-Small Cell Lung Cancer or Advanced Colorectal Cancer (NCT01676714), a na phase 2 study. The primary outcome is Overall Response Rate.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01676714 |
| Phase | Phase 2 |
| Condition | Non-Small Cell Lung Cancer, Colorectal 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 | University of California, Davis |
| Results posted | Yes |
2. Clinical Question
Population
Participants had advanced non-small cell lung cancer or advanced colorectal cancer and had previously received anti-vascular endothelial growth factor therapy. The registry record describes a total enrollment of 10 participants. It does not report a more detailed primary analysis population, such as how many participants had each cancer type or how many were included in the overall response analysis.
Intervention
The intervention was dovitinib, given as 500 mg, or 5 capsules, once a day for 5 continuous days, followed by a 2-day break. The regimen was to continue until progression or unacceptable toxicity developed. The registry record does not report how many participants completed treatment or how long participants received it.
Comparator
There was no concurrent comparator arm. The registry describes a single-group study, with allocation and masking both listed as none. Outcomes therefore describe participants receiving dovitinib in this study; they do not provide a randomized comparison with another treatment or with no treatment.
Primary question
The primary outcome was overall response rate, defined using RECIST v1.1 categories of complete response and partial response. The question was whether tumor assessments recorded a complete or partial response among participants receiving dovitinib. The registry record does not report the number of participants with either response, the overall response rate, or an uncertainty interval for that result.
3. Trial Design
This was a phase II, single-group study sponsored by the University of California, Davis. The registry lists an enrollment of 10 and describes participants with advanced non-small cell lung cancer or advanced colorectal cancer who had previously received anti-VEGF therapy. The study is marked completed. Its recorded start was 2013-02 and its primary completion was 2016-01.
Participants received dovitinib according to a repeated dosing schedule: 500 mg, or 5 capsules, once a day for 5 continuous days, followed by a 2-day break. Treatment was described as continuing until progression or unacceptable toxicity. The registry lists no masking and no allocation, consistent with its single-group model. No control group is described, so the study cannot directly estimate a treatment difference against a concurrent comparator.
The stated primary outcome was overall response rate. The registry describes complete and partial response according to RECIST v1.1 and says target lesions were assessed by CT or MRI. Its stated timeframe is up to 100 months. Secondary outcomes listed are disease control rate, progression-free survival, and the number of patients who experienced treatment-related toxicities. The registry lists four outcome measures in total, but it does not provide results for the primary endpoint or the listed secondary outcomes in the record described.
The record does not report a detailed protocol, a statistical analysis plan, a sample-size justification for this single-group design, or a planned comparison with another treatment. The planning assumptions recorded for sample-size illustrations describe equal allocation between two arms, which is not the design registered for this study. Those assumptions should not be read as evidence that the trial itself had two arms or as a description of its treatment allocation.
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 | Dovitinib |
|---|---|
| STARTED | 10 |
| COMPLETED | 10 |
| 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 | Dovitinib |
|---|---|---|
| Participants analysed | 10 | |
| Age, Continuous | years | 63 (48 to 77) |
| Sex: Female, Male: Female | Participants | 5 |
| Sex: Female, Male: Male | Participants | 5 |
| Region of Enrollment: United States | Participants | 10 |
6. Primary Endpoint
The registry lists the primary outcome as Overall Response Rate, measured over Up to 100 months.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
Overall response rate is a binary outcome: a participant is classified according to whether an overall response was recorded or not. In this study, overall response was defined as complete response plus partial response under RECIST v1.1. The registry describes complete response as disappearance of all target lesions and partial response as a decrease of >=30% in the sum of the longest diameter of target lesions. Target lesions were assessed by CT or MRI.
As a general statistical concept, a response endpoint can summarize whether tumor measurements meet prespecified response categories during a stated assessment period. It is distinct from an outcome such as progression-free survival, which concerns time until an event, and from disease control rate, which is a separately listed secondary outcome. The endpoint’s meaning depends on the response criteria and on which participants are included in its denominator.
The registry names overall response rate as the primary outcome and gives its timeframe as up to 100 months. It does not report the number of complete responses, partial responses, or participants evaluated for response. The response rate and its precision therefore cannot be read from the registry record. It also does not report the rules for handling participants without an evaluable assessment or the analysis population used for this endpoint.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central interpretive limitation is that the registry record names overall response rate as the primary outcome but does not report its result. There is no reported count of complete responses or partial responses, no denominator for an analyzed response population, and no reported estimate of overall response rate. Consequently, the record does not establish how frequently responses were observed in this study or how precisely that frequency was estimated.
The study enrolled 10 participants and used a single-group design. Those details describe the study’s size and structure, but they do not substitute for outcome results. Without a reported response count and denominator, a numerical response estimate cannot be reconstructed. Without a reported uncertainty interval, the precision of any response estimate cannot be assessed. The registry also does not report inferential analyses; it lists 0 analyses.
The design matters to interpretation as well. Because there was no concurrent comparator, any outcomes recorded for participants receiving dovitinib would be descriptive of this group. They would not, by themselves, estimate the difference between dovitinib and another treatment. Differences in prognosis, prior treatment, disease type, and other factors cannot be separated through a randomized contrast that this design does not contain.
The safety information is more concrete but still limited. The registry reports serious adverse events affecting 3 of 10 participants at risk, other adverse events affecting 10 of 10 participants at risk, and 1 death. It also lists Thrombolic Event, Esophageal pain, and Sepsis among the top serious terms, each with a reported value of 10.0%. These counts describe what is recorded for this group; the record does not supply enough detail in this summary to attribute individual events to treatment, determine their timing, or compare their frequency with another group.
The registry lists disease control rate, progression-free survival, and treatment-related toxicities as secondary outcomes, but does not report their outcome results in the record described. It also does not report how missing assessments, withdrawals, or participants with unevaluable scans were handled. The evidence available in the registry is therefore a description of the intended endpoint and treatment, together with limited safety counts, rather than a complete account of efficacy results or their uncertainty.
9. Statistical Methods Explained
What kind of endpoint is overall response rate?
As a general statistical explanation, an outcome that records whether each participant did or did not meet a response definition is binary. A response rate is interpreted as the count meeting the definition in relation to the participants included in the analysis. Both parts matter: the count alone does not establish the rate, and the denominator depends on the stated analysis population and rules for evaluability.
In this study, the registry defines overall response as complete response or partial response under RECIST v1.1. The stated criteria use changes in target lesions, with assessment by CT or MRI. Such criteria make the outcome depend on tumor measurements and response classification, rather than on a participant’s subjective impression alone. The registry record does not report the response counts, denominator, or analysis rules needed to evaluate the resulting estimate.
What does the single-group design allow?
As a general concept, a single-group study can describe outcomes among people receiving the study intervention. It does not create a concurrent counterfactual group showing what would have happened under another treatment. In a randomized comparison, allocation can support an estimate of a contrast between groups; that kind of treatment contrast is not available in this design.
The registry lists allocation as none, masking as none, and the model as single group. These design features should guide interpretation of any outcomes: they are observations in the dovitinib group, not a randomized comparison. The registry does not report a control group, allocation procedure, or masking procedure beyond those entries.
How should precision be considered?
As a general statistical explanation, a confidence interval describes a range of values compatible with the data under a specified statistical method. A narrow interval indicates greater precision than a wide interval, while an estimate without an interval gives less information about its uncertainty. For a binary response outcome, the amount of information depends in part on the number of participants included and the response count.
The enrollment was 10, but the registry does not report the analyzed response count or a confidence interval. Precision cannot therefore be assessed from the stated enrollment alone. It would also be inappropriate to infer a response rate or uncertainty interval from safety counts, because those are different outcomes.
What do the planning assumptions mean?
The registry describes sample-size calculations as planning illustrations based on an observed effect, not as the trial’s own design. The listed assumptions are a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and an effect to detect expressed as a stated fraction of the observed effect. For time-to-event outcomes, the planning tables are described as giving total events; for other outcomes, they are described as giving participants per arm.
These assumptions do not match the registered single-group design: the registry lists no allocation and no comparator arm. The planning assumptions therefore should not be interpreted as the actual study design or as the trial’s sample-size justification. The record does not report a protocol-based sample-size justification. Nor should any planning illustration be described as observed power. Such calculations are prospective illustrations under specified assumptions, not a measure of the probability that a completed study detected an effect.
There is an additional interpretive constraint: the primary response result that would define an observed effect is not reported in the registry record. Without that result, the effect magnitude underlying an illustration cannot be independently assessed from the record. The illustration’s assumptions can be explained, but they do not supply missing outcome findings.
What can be concluded from the registered analyses?
The registry lists 0 analyses. This means that no inferential analysis results are reported in the record described. It does not establish which methods may have been used outside the registry, and it does not reveal whether any analysis was planned or conducted in other documents. A model, adjustment strategy, or statistical test should not be inferred when it is not reported.
Over-dispersion is a general concern for certain count outcomes when variability exceeds what a simple model assumes. It is not a reported feature of the primary outcome here, which is defined as a participant count for a binary response measure, and no model results are reported from which over-dispersion could be assessed. In general, model choice depends on the outcome structure and analysis plan; the registry does not specify such a plan.
10. Exact Estimates for Each Arm
No two-arm comparison is shown for this trial, but each arm's result can still be summarized with exact interval estimates computed from the posted counts. The Clopper-Pearson interval is exact and conservative; the Wilson interval is a common closer-to-nominal alternative; the Bayesian credible interval uses a uniform Beta(1, 1) prior. For small groups the three can differ noticeably.
| Arm | Participants | Events | Observed % | Clopper-Pearson 95% CI | Wilson 95% CI | Bayesian 95% credible interval |
|---|---|---|---|---|---|---|
| Dovitinib | 7 | 1 | 14.3% | 0.4% to 57.9% | 2.6% to 51.3% | 3.2% to 52.7% |
R code and output
arm <- c("Dovitinib")
n <- c(7)
events <- c(1)
for (i in seq_along(n)) {
ci <- binom.test(events[i], n[i])$conf.int # Clopper-Pearson exact interval
cr <- qbeta(c(0.025, 0.975), 1 + events[i], 1 + n[i] - events[i]) # Bayesian, uniform Beta(1,1) prior
wi <- (function(x, n) { p <- x / n; z <- qnorm(0.975); d <- 1 + z^2 / n
c((p + z^2 / (2 * n) - z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d, (p + z^2 / (2 * n) + z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d) })(events[i], n[i])
cat(sprintf("ARM|%s|%d|%d|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f\n", arm[i], n[i], events[i], 100 * events[i] / n[i],
100 * ci[1], 100 * ci[2], 100 * wi[1], 100 * wi[2], 100 * cr[1], 100 * cr[2]))
}
ARM Dovitinib 7 1 14.3 0.4 57.9 2.6 51.3 3.2 52.711. Multiplicity, Missing Data and Analysis Populations
The registry names overall response rate as the primary outcome and lists disease control rate, progression-free survival, and the number of patients who experienced treatment-related toxicities as secondary outcomes. It lists four outcome measures in total. When a study evaluates several outcomes, multiplicity is a general statistical consideration: repeated testing can increase the chance of at least one apparently notable result if no adjustment or hierarchy is specified. The registry record does not report a multiplicity adjustment, testing hierarchy, or other procedure for addressing this issue.
The record lists 0 analyses and does not report inferential results for the primary or secondary outcomes. It also does not describe interim analyses. The analysis plan, including any planned handling of multiple outcomes, is not reported in the registry record; the absence of a described procedure should not be taken to mean that a particular procedure was or was not used outside the record.
Missing-data handling is also not reported. For overall response, relevant details would include how participants without an evaluable scan were treated, how withdrawals were handled, and which participants formed the denominator. The registry does not state these rules or provide the number of participants with evaluable response assessments. It similarly does not report analysis populations for disease control or progression-free survival.
These omissions limit interpretation because outcome estimates depend on both the observed data and the rules used to include participants and handle missing information. No assumptions about missingness, imputation, censoring, or analysis populations can be attributed to this study from the registry record.
12. 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 |
|---|---|---|---|
| Dovitinib | 3 | 10 | 30.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Dovitinib | 1 of 10 |
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 |
|---|---|
| Dovitinib | 10 of 10 |
Most frequent serious adverse event terms
| Event term | Organ system | Dovitinib |
|---|---|---|
| Thrombolic Event | Vascular disorders | 1 of 10 |
| Esophageal pain | Gastrointestinal disorders | 1 of 10 |
| Sepsis | Infections and infestations | 1 of 10 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Dovitinib |
|---|---|---|
| Fatigue | General disorders | 9 of 10 |
| Alkaline phosphatase increased | Investigations | 8 of 10 |
| Anorexia | Metabolism and nutrition disorders | 8 of 10 |
| Nausea | Gastrointestinal disorders | 6 of 10 |
| GCT Increased | Investigations | 6 of 10 |
| Hypoalbuminemia | Metabolism and nutrition disorders | 6 of 10 |
| ALT Increased | Investigations | 5 of 10 |
| AST increased | Investigations | 5 of 10 |
| Blood Bilirubin Increased | Investigations | 5 of 10 |
| Weight Loss | Investigations | 5 of 10 |
Serious events with the highest arm-level rate: thrombolic event (10.0%), esophageal pain (10.0%), sepsis (10.0%).
The registry reports 3 participants with serious adverse events among 10 participants at risk in the dovitinib group. It separately reports other adverse events affecting 10 participants among 10 at risk, and 1 death. These are counts for the group, not a comparison with an untreated or alternative-treatment group.
The top serious terms listed are Thrombolic Event, Esophageal pain, and Sepsis, each with a reported value of 10.0%. The record does not establish from these entries whether the terms refer to distinct participants or whether events overlapped, nor does it report event timing or duration. The term Thrombolic Event is retained as written in the registry.
These safety figures should be read as recorded event information, not as proof that dovitinib caused an event or that an event was unrelated to treatment. Although treatment-related toxicities are listed as a secondary outcome, the registry record does not report that outcome’s results or the attribution rules used. With no comparator group, the figures also cannot quantify a difference in safety relative to another treatment.
13. What the Result Does, and Does Not, Mean
The registry establishes that a phase II, single-group study enrolled 10 participants with advanced non-small cell lung cancer or advanced colorectal cancer previously treated with anti-VEGF therapy. It records a dovitinib regimen and defines overall response according to RECIST v1.1. It also provides some adverse-event counts for the treated group.
It does not report the primary overall response result. There is no response count, response rate, confidence interval, or reported analysis result in the record described. The registry therefore does not support a numerical conclusion about the frequency of complete or partial responses in this study. It also does not report results for the listed secondary outcomes of disease control rate, progression-free survival, and treatment-related toxicities.
Even if response results were available, a single-group design would describe outcomes among participants receiving dovitinib rather than estimate a randomized treatment effect. The absence of a concurrent comparator means that outcomes cannot be directly contrasted with another treatment group within this study. The registry does not report adjustment methods or other analyses that might address differences between groups, and it does not report such a comparison.
The safety counts are informative as recorded observations but do not, by themselves, establish causation or comparative risk. The record also omits key details about outcome evaluability, missing data, and analysis populations. Those gaps limit judgments about how representative the recorded results are of all enrolled participants. Overall, the registry supports a description of the study design, endpoint definitions, and certain safety counts, but it does not provide the primary efficacy result or the statistical detail needed for a fuller assessment of efficacy and precision.
14. Why This Trial Matters Statistically
This study is statistically notable for the gap between a clearly named primary endpoint and the absence of its result in the registry record. Overall response rate is defined using RECIST v1.1, including target-lesion measurement and imaging assessment by CT or MRI. Yet the record does not report the response count, rate, denominator, or uncertainty. This illustrates why an outcome definition alone is not an outcome finding.
The single-group design is also important. It can describe recorded outcomes among participants receiving dovitinib, but it does not provide a concurrent randomized contrast. The design therefore determines which statistical questions can be answered: a within-group response description is different from an estimate of how outcomes compare with another treatment.
The enrollment of 10 makes clear why a transparent account of denominators and precision would matter, but the registry does not supply those details for response. Without response counts and an uncertainty interval, readers cannot assess the stability of an estimate from the primary outcome. Safety information is reported as counts, but without a comparator or detailed attribution and event information, it remains descriptive.
The record also lists several secondary outcomes while reporting 0 analyses and no multiplicity approach. This highlights the importance of distinguishing registered outcomes from analyzed results. A list of planned or named outcomes does not establish that results were collected, analyzed, or reported. Statistical interpretation depends on transparent outcome reporting, analysis populations, missing-data rules, and methods, none of which can be reconstructed when they are not reported.
15. 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.
Several limitations specific to this registry record constrain interpretation. The total enrollment was 10, and the study was single-group, with no allocation and no masking listed. There is no concurrent comparator, so the design does not support a randomized estimate of a difference between dovitinib and another treatment.
The primary outcome is defined, but its result is not reported. The record gives no response counts, response rate, or confidence interval, and it does not identify the denominator used for response analysis. The registry also does not report outcomes for the listed secondary measures. Although it states a timeframe of up to 100 months for overall response, it does not provide assessment schedules or detailed follow-up information.
The record does not report analysis populations, missing-data rules, treatment exposure, or the number of participants with evaluable imaging. It lists 0 analyses and does not report a statistical analysis plan, interim analyses, or a sample-size justification for the registered single-group design. The planning assumptions described for sample-size illustrations concern equal allocation between two arms, so they should not be mistaken for the design or justification of this study.
Safety reporting is also limited in detail. Counts are available for serious and other adverse events and for deaths, but the record does not give event timing, duration, attribution procedures, or enough information to determine whether listed serious terms overlap among participants. These limitations mean that the available information is descriptive and incomplete, particularly for judging response and its precision.
16. Related Tutorials
17. Related Statistical Calculators
18. Sources
- ClinicalTrials.gov: Study of Dovitinib and Biomarkers in Advanced Non-Small Cell Lung Cancer or Advanced Colorectal Cancer — NCT01676714.
- Registry record last updated 2018-01-10; status last verified 2018-01.
- Results first posted 2017-05-15.
- 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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19. Record Summary
This completed phase II study, sponsored by the University of California, Davis, enrolled 10 participants with advanced non-small cell lung cancer or advanced colorectal cancer previously treated with anti-VEGF therapy. It used a single-group design with dovitinib and listed no masking or allocation. The regimen was 500 mg, or 5 capsules, once a day for 5 continuous days followed by a 2-day break, continuing until progression or unacceptable toxicity.
The primary outcome was overall response rate under RECIST v1.1, assessed by CT or MRI, with a stated timeframe of up to 100 months. The primary result and listed secondary outcome results are not reported in the registry record. Safety information includes 3 participants with serious adverse events among 10 at risk, 10 with other adverse events among 10 at risk, and 1 death. The record lists 0 analyses and does not report the statistical analysis plan, missing-data handling, or analysis populations.