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 Dovitinib (TKI258) and Abiraterone Acetate in Metastatic Castrate-Resistant Prostate Cancer (mCRPC) (NCT01994590), a na phase 2 study. The primary outcome is Safety and Tolerability.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01994590 |
| Phase | Phase 2 |
| Condition | Prostate Cancer |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 4 |
| Arms in the primary outcome | n/a |
| Lead sponsor | M.D. Anderson Cancer Center |
| Results posted | Yes |
2. Clinical Question
Population
Patients with metastatic castrate-resistant prostate cancer (mCRPC). The registry record lists the condition as prostate cancer and describes the study population as patients with metastatic castrate-resistant prostate cancer.
Intervention
Dovitinib combined with abiraterone acetate and prednisone. The study description identifies this combination as the regimen being evaluated.
Comparator
There was no separate comparator group. The registry record describes a single-group study, with allocation listed as NA and no masking.
Primary question
The listed primary outcome measures the number of participants with adverse events, under the heading Safety and Tolerability. The study summary also states an interest in whether adding dovitinib may help control mCRPC, but the recorded primary endpoint is a safety count rather than a comparative disease-control measure.
Statistically, the central question that can be addressed by the posted primary result is descriptive: how many participants in the treated group were reported to have adverse events? With no comparator group, the data cannot estimate a difference in adverse-event risk between the combination and another regimen. They also cannot separate events associated with one component from events associated with the combination or underlying illness.
The official title refers to evaluating markers of FGF and AR signaling in bone marrow and plasma. However, the registry record lists one outcome measure, the safety and tolerability endpoint. Additional marker outcomes or their results are not reported in the registry record. The posted result therefore answers a narrower question than the broad biological aims suggested by the title.
3. Trial Design
This was a Phase II, single-group study sponsored by M.D. Anderson Cancer Center. The registry record lists 4 participants enrolled, allocation as NA, and masking as none. There was no randomized allocation or like-for-like control group, so the study is not a comparative trial of the combination against another treatment.
The treatment group is described as receiving dovitinib with abiraterone acetate and prednisone. Dovitinib was scheduled orally on Days 1-5, 8-12, 15-19, and 22-26 of each 28 day cycle, with a starting dose of 400 mg daily. Abiraterone acetate is described as four tablets of 250 mg each taken orally daily. The arm description begins a prednisone instruction but is truncated in the registry record, so its full dosing description is not reported in the registry record.
The study began on 2014-05-19, and its primary completion date is listed as 2017-06-05. Its status is terminated. The reason for termination is not reported in the registry record. The primary outcome timeframe states that participants were followed while actively taking study drug and for at least 30 days after the last dose.
These design features matter for interpretation. A single-group study can describe outcomes among participants who received the regimen, but it does not provide a concurrent comparison for attributing outcomes to treatment. The small enrollment also means that each participant's experience has substantial influence on the group-level count. The registry record does not report a sample-size justification or a statistical analysis plan for this trial.
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 | All Patients |
|---|---|
| STARTED | 4 |
| COMPLETED | 2 |
| NOT COMPLETED | 2 |
Reasons for not completing this period:
| Reason | All Patients |
|---|---|
| Adverse Event | 1 |
| Trial Terminated | 1 |
5. Baseline Characteristics
The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.
| Characteristic | Unit | All Patients |
|---|---|---|
| Participants analysed | 4 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 0 |
| Age, Categorical: >=65 years | Participants | 4 |
| Age, Continuous | years | 71.25 (66 to 73) |
| Sex: Female, Male: Female | Participants | 0 |
| Sex: Female, Male: Male | Participants | 4 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 0 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 4 |
| Region of Enrollment: United States | Participants | 4 |
6. Primary Endpoint
The registry lists the primary outcome as Safety and Tolerability, measured over Participants are followed while actively taking study drug and for at least 30 days post last dose..
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is titled Safety and Tolerability and is defined as the number of participants with adverse events. It is a count of participants with an event, not a measure of the number of separate event episodes, event duration, or event severity across all participants. The timeframe covers the period while participants were actively taking study drug and at least 30 days after the last dose.
A participant-level adverse-event endpoint is used to describe how often a safety event was reported in the treated group. It can provide a basic account of observed safety experience during the specified follow-up. It does not, on its own, establish whether an event was caused by treatment, nor does it compare safety with a different regimen. Attribution, event definitions, severity grading, and analysis details beyond the listed endpoint are not reported in the registry record.
The primary result reports 4 participants at risk for other adverse events and 4 with such events; for serious adverse events, it reports 4 at risk and 1 affected. These are descriptive counts from the registry record. With the small group and no comparator, the endpoint should be read as a record of what was observed among these participants, not as a precise estimate of the safety profile in a wider population.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The posted primary result is a participant-level safety summary. The record reports that 1 of 4 participants had a serious adverse event, while 4 of 4 had other adverse events. The serious-event terms listed are Thromboembolic Event at 25.0% and Skin Infection at 25.0%. No deaths are reported in the group. These figures describe the recorded experience in the study group; they do not establish that the regimen caused the events.
The enrollment of 4 is central to understanding precision. In a group this small, an individual participant changes the observed count substantially. The counts therefore provide limited information about how frequently similar events might occur in a broader patient population. The registry record does not give confidence intervals or other uncertainty estimates for these safety results. Without such estimates, the reader cannot use the posted counts to assess a range of plausible event rates with a stated level of statistical confidence.
There is no concurrent control group. Consequently, the results cannot show whether the observed event counts are more or less frequent than they would be under another treatment or without the study combination. They also cannot distinguish effects of dovitinib from effects of abiraterone acetate, prednisone, disease characteristics, or other factors. No causal conclusion follows from these descriptive counts.
The study summary mentions a goal of learning whether adding dovitinib may help control mCRPC, as well as studying safety. But the recorded primary outcome is a safety endpoint. No disease-control estimate or biomarker result is included in the listed outcome record. Such efficacy or biomarker results are not reported in the registry record, so they cannot be inferred from the safety figures.
The status is terminated, but the reason is not reported in the registry record. Termination alone does not explain why enrollment ended, whether the stated goals were met, or whether the results were considered favorable or unfavorable. The available record supports a narrow descriptive account of adverse events in this single treated group, with substantial uncertainty and no comparative estimate.
9. Statistical Methods Explained
What does a participant count represent?
A participant count records how many people in the analyzed group met an endpoint definition. For this study, the primary endpoint is the number of participants with adverse events. This differs from an event count: one participant may experience more than one event, but a participant-level count still counts that person once for the relevant outcome. The registry record does not provide additional detail about how repeat events were handled or how adverse events were classified beyond the endpoint description.
The posted safety data report 4 participants at risk for serious adverse events, with 1 affected, and 4 at risk for other adverse events, with 4 affected. The serious adverse-event terms listed include Thromboembolic Event and Skin Infection, each reported at 25.0%. These summaries do not include a comparative treatment effect. They are observations within the one group.
Why does the absence of a comparator matter?
A comparative effect requires outcomes under at least two treatment conditions, such as an intervention group and a control group. In a randomized comparison, random assignment can help make groups comparable on average, although uncertainty remains. Here, the design is single-group, allocation is listed as NA, and masking is none. There is no control-group result against which to estimate a difference. A within-group event count can describe what happened during follow-up, but cannot show what would have happened to those same participants under another regimen.
Without a comparison, observed events cannot be attributed uniquely to the study combination. The regimen includes dovitinib, abiraterone acetate, and prednisone; the registry record does not permit the separate contribution of each component to be estimated. Events may also be related to the disease or other circumstances, and causal attribution details are not reported in the registry record.
What does uncertainty mean for these counts?
General statistical explanation: an estimate from a sample is uncertain because another sample from a similar population could produce different observations. A confidence interval is one way to express that uncertainty by giving a range of values compatible with the data under a specified method. A narrow interval generally indicates greater precision, while a wide interval indicates less precision. The registry record does not report confidence intervals for the primary safety counts.
With only 4 participants enrolled, the observed group count has limited precision as an estimate of event frequency beyond this study group. The record lists counts and percentages for selected serious adverse-event terms, but those values should not be treated as stable population rates. The actual frequency in a larger or different patient group could differ. No additional precision measure is reported in the registry record.
Why can event counts be more informative than a single percentage?
General statistical explanation: percentages are easier to compare across groups of different sizes, but the underlying counts show how much information supports each percentage. A percentage based on few observations can appear numerically precise while remaining highly uncertain. For that reason, the count and the group size should be read together. In this study, the registry reports the counts alongside the at-risk group size, allowing the reader to see the scale of the observed data without treating the percentage as a precise estimate.
What is not available about the analysis?
The registry record lists no analyses and does not report an analysis method, confidence intervals, handling of missing outcomes, or a sample-size justification. A planning illustration is not part of the observed clinical result and cannot substitute for a prespecified analysis. No recalculated estimate is reported in the registry record. It is therefore not possible to assess whether the posted result follows a more detailed statistical plan or whether any adjustments were made.
General statistical explanation: for binary outcomes, common approaches estimate a proportion or compare proportions across groups. The choice of method depends on the design, number of observations, and analysis objective. With no comparator group, a treatment contrast is unavailable. Methods for comparing groups, adjusting for baseline characteristics, or accounting for repeated measurements cannot be assessed from the listed primary safety result. This is a limitation of the available record, not evidence that a particular method was or was not used outside what is documented.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists one outcome measure, Safety and Tolerability, defined as the number of participants with adverse events. It does not report a multiplicity strategy, such as how interpretation would be adjusted if several outcomes or statistical tests were evaluated. Although the official title refers to markers of FGF and AR signaling in bone marrow and plasma, their endpoints and results are not reported in the registry record. The record also lists no analyses.
Missing-data methods are not reported in the registry record. The posted safety counts include an at-risk group of 4 for serious adverse events and an at-risk group of 4 for other adverse events, but the record does not describe how incomplete follow-up, unavailable event assessments, or early discontinuation were handled. It would be inappropriate to assume that no data were missing or that all participants were assessed identically.
The analysis population is not described beyond the listed group of All Patients. The primary endpoint's population field is blank, and the registry record does not specify whether the safety summaries include everyone enrolled, everyone who began treatment, or another defined analysis set. The record does not report rules for inclusion, exclusions, or treatment exposure requirements.
These omissions limit reproducibility and interpretation. Without the analysis population, missing-data approach, and multiplicity plan, readers cannot fully evaluate which observations contributed to the endpoint or how the study handled multiple possible findings. The available counts remain descriptive summaries, but the record does not provide enough information to reconstruct a broader statistical analysis.
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 |
|---|---|---|---|
| All Patients | 1 | 4 | 25.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| All Patients | 0 of 4 |
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 |
|---|---|
| All Patients | 4 of 4 |
Most frequent serious adverse event terms
| Event term | Organ system | All Patients |
|---|---|---|
| Thromboembolic Event | Blood and lymphatic system disorders | 1 of 4 |
| Skin Infection | Skin and subcutaneous tissue disorders | 1 of 4 |
Most frequent non-serious adverse event terms
| Event term | Organ system | All Patients |
|---|---|---|
| Fatigue | Metabolism and nutrition disorders | 4 of 4 |
| Aspartate aminotransferase increase | Hepatobiliary disorders | 3 of 4 |
| Platelet count decreased | Blood and lymphatic system disorders | 3 of 4 |
| Alanine aminotransferase increased | Hepatobiliary disorders | 2 of 4 |
| Alkaline phophatase increased | Hepatobiliary disorders | 2 of 4 |
| Anemia | Blood and lymphatic system disorders | 2 of 4 |
| Diarrhea | Gastrointestinal disorders | 2 of 4 |
| Hypoalbuminemia | Metabolism and nutrition disorders | 2 of 4 |
| Hypomagnesium | Metabolism and nutrition disorders | 2 of 4 |
| Upper respiratory infection | Respiratory, thoracic and mediastinal disorders | 2 of 4 |
Serious events with the highest arm-level rate: thromboembolic event (25.0%), skin infection (25.0%).
The serious adverse-event summary reports 4 participants at risk and 1 affected. The record lists Thromboembolic Event and Skin Infection among the top serious terms, each at 25.0%. It also reports 4 participants at risk and 4 affected for other adverse events. The death count is 0. These figures are presented for the All Patients group.
The serious-event count and the terms are different kinds of summaries: the count describes participants affected by serious adverse events, while the terms identify reported event categories with percentages. The registry record does not provide further detail about event severity grades, duration, timing, recurrence, or attribution. These details should not be inferred from the terms alone.
The data are descriptive and come from a single-group study with enrollment of 4. They cannot establish that the combination caused an event or show how event frequency compares with another treatment. The small group also limits precision: the observed counts may not represent the frequency in a broader population. No confidence intervals are reported in the registry record.
The absence of reported deaths in this group is a statement about the posted count, not proof that a treatment is free of serious risk. The registry status is terminated, but the reason for termination is not reported in the registry record.
12. What the Result Does, and Does Not, Mean
The posted result shows that the registry recorded adverse events among participants treated in this single-group Phase II study. It reports 1 of 4 participants with serious adverse events, 4 of 4 with other adverse events, and no deaths. The listed serious terms are Thromboembolic Event and Skin Infection, each reported at 25.0%. This is the descriptive safety information available in the primary outcome record.
The result does not establish that the combination caused the reported events. There is no control group, and the regimen contains three drugs. The study cannot isolate the contribution of one drug or compare the combination with a different treatment. It also cannot determine from these safety data whether the regimen controls metastatic castrate-resistant prostate cancer. Although disease control is mentioned in the study summary, an efficacy result is not reported in the registry record.
The counts are based on a very small enrolled group and do not precisely characterize event frequency in a wider population. No confidence intervals or other uncertainty estimates are reported. The figures should not be interpreted as stable estimates of how often events would occur in other patients or settings.
The title refers to evaluating FGF and AR signaling markers, but marker results are not reported in the registry record. The record also does not explain why the study was terminated. Those unanswered questions cannot be resolved from the primary safety summary.
In practical statistical terms, the result is a limited description of observed safety events in this particular study group. It is not a comparative safety estimate, a causal analysis, or evidence of disease-control benefit. Further conclusions would require information not reported in the registry record.
13. Why This Trial Matters Statistically
This study illustrates the difference between describing outcomes and estimating a treatment effect. A single-group trial can document adverse events among participants who received a regimen. Without a comparator, it cannot estimate whether those outcomes differ from what would occur under another treatment. That distinction is important whenever a registry result is read as evidence about a treatment.
The enrollment of 4 also makes the scale of evidence especially important. A small number of observations can be useful for recording what happened in a study, but provides limited precision for estimating event frequency beyond the participants observed. The serious adverse-event count, other adverse-event count, and listed terms should therefore be read as a compact description of the recorded experience, not as definitive rates for a wider population.
The record also demonstrates why the endpoint and the study's stated aims should be distinguished. The summary describes interest in disease control and safety, while the primary endpoint listed is a count of participants with adverse events. The title mentions biological markers, but marker outcomes are not reported in the registry record. Statistical interpretation must stay tied to the results actually documented.
Finally, the terminated status does not explain the study's course or its findings. The reason for termination is not reported in the registry record. Clear reporting of the analysis population, missing-data methods, uncertainty estimates, and the status of intended outcomes would help readers evaluate how much can be learned. In their absence, the narrow descriptive interpretation is the most defensible one.
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 is the single-group design. With allocation listed as NA and no masking, there is no randomized or masked comparison. The registry record itself notes that the arms are not a like-for-like control comparison. In this study, no separate comparator group is listed, so the posted safety outcomes cannot support a between-treatment contrast.
The enrolled group contains 4 participants. This severely limits the precision and generalizability of the descriptive safety counts. The record does not report confidence intervals or other uncertainty estimates that would help characterize this limitation quantitatively.
The trial status is terminated, but the reason is not reported in the registry record. The record does not state whether termination was related to safety, recruitment, operational considerations, or another cause, so none should be presumed. The primary completion date is listed, but it does not resolve the reason for termination.
The endpoint is a participant count for adverse events, while detail about event definitions, severity grading, attribution, timing, and recurrence is not reported in the registry record. The analysis population is also unclear: the population field is blank, and the record does not describe inclusion rules for the safety analysis. Handling of missing safety data is not reported.
Finally, only one outcome measure is listed, and no analyses are listed. The title refers to signaling markers and the study summary refers to disease control, but results for those aims are not reported in the registry record. These gaps prevent a full assessment of the broader scientific questions raised by the trial.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Dovitinib (TKI258) and Abiraterone Acetate in Metastatic Castrate-Resistant Prostate Cancer (mCRPC) — NCT01994590.
- Registry record last updated 2019-03-19; status last verified 2019-02.
- Results first posted 2019-03-19.
- 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
NCT01994590 is a Phase II, single-group study of dovitinib combined with abiraterone acetate and prednisone in metastatic castrate-resistant prostate cancer. The registry record lists M.D. Anderson Cancer Center as sponsor, enrollment of 4, no masking, and status as terminated. It lists one primary outcome: Safety and Tolerability, measured as the number of participants with adverse events during treatment and for at least 30 days after the last dose.
The posted safety summary reports 1 of 4 participants with serious adverse events, 4 of 4 with other adverse events, and 0 deaths. Thromboembolic Event and Skin Infection are each listed at 25.0% among the top serious terms. These are descriptive findings from a small, uncontrolled group, not a comparative or causal estimate.
The reason for termination, statistical analysis plan, missing-data methods, and results for disease control or signaling markers are not reported in the registry record.