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 Nintedanib For HER2-Negative Metastatic Inflammatory Breast Cancer (MIBC) (NCT02389764), a na phase 2 study. The primary outcome is Clinical Benefit Rate (Complete Response [CR], Partial Response [PR] or Stable Disease [SD] Date) of BIBF 1120 (Nintedanib) in Patients With HER2-negative Metastatic Inflammatory Breast Cancer (IBC)..
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02389764 |
| Phase | Phase 2 |
| Condition | Breast 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 | M.D. Anderson Cancer Center |
| Results posted | Yes |
2. Clinical Question
Population
Patients with metastatic HER2-negative inflammatory breast cancer were the intended population. The registry record lists breast cancer as the condition and records an enrollment of 10. Eligibility criteria and further details about the enrolled participants are not reported in the registry record.
Intervention
The study evaluated BIBF 1120, also called nintedanib. The listed initial dose was 200 mg orally twice daily for a 28 day cycle. Phone calls by study staff every 3 months for up to 1 year after the end-of-treatment visit were also described.
Comparator
There was no comparison group: the design is recorded as single-group, allocation is listed as not applicable, and masking is listed as none. Consequently, the study does not provide a randomized comparison with another treatment or a control group.
Primary question
The primary question was whether participants achieved clinical benefit, defined as complete response or partial response within 3 months post-treatment, or stable disease for at least three months post-treatment. The endpoint was to be determined using RECIST 1.1. The primary outcome results are not reported in the registry record.
3. Trial Design
This was a Phase II, single-group study sponsored by M.D. Anderson Cancer Center. The study title identifies nintedanib, also known as BIBF1120, for patients with metastatic HER2-negative inflammatory breast cancer. It was unmasked, and allocation was recorded as not applicable. These design features describe a study without a randomized comparator; they do not support a direct treatment-versus-control estimate.
The registry record gives a planned enrollment of up to 44 participants in its study description and an enrollment of 10. The study start date was 2015-06-22, and the primary completion date was 2018-06-08. Its status is recorded as terminated. The reason for termination and any consequences for recruitment or follow-up are not reported in the registry record.
The listed drug regimen began with 200 mg of BIBF 1120 by mouth twice daily for a 28 day cycle. The intervention description also includes phone calls by study staff every 3 months for up to 1 year after the end-of-treatment visit. The record does not describe a concurrent control intervention or a masking procedure.
The primary outcome was a count of participants meeting the clinical-benefit definition, with a timeframe of 2 years. A safety outcome is also listed, concerning the type, frequency, and severity of adverse events according to CTCAE Version 4.0. The study's detailed protocol, recruitment flow, analysis population, and reasons for termination are not reported in the registry record.
4. Participant Flow and Accounting
The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.
Overall Study
| Stage | BIBF 1120 |
|---|---|
| STARTED | 9 |
| COMPLETED | 0 |
| NOT COMPLETED | 9 |
Reasons for not completing this period:
| Reason | BIBF 1120 |
|---|---|
| Lack of Efficacy | 9 |
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 | BIBF 1120 |
|---|---|---|
| Participants analysed | 9 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 8 |
| Age, Categorical: >=65 years | Participants | 1 |
| Sex: Female, Male: Female | Participants | 9 |
| Sex: Female, Male: Male | Participants | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 2 |
| Race (NIH/OMB): White | Participants | 7 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Region of Enrollment: United States | participants | 9 |
6. Primary Endpoint
The registry lists the primary outcome as Clinical Benefit Rate (Complete Response [CR], Partial Response [PR] or Stable Disease [SD] Date) of BIBF 1120 (Nintedanib) in Patients With HER2-negative Metastatic Inflammatory Breast Cancer (IBC)., measured over 2 years.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is titled clinical benefit rate, while its recorded unit and measure are a count of participants. Clinical benefit is defined as complete response or partial response within 3 months post-treatment, or stable disease lasting at least three months post-treatment. The record says the assessment uses RECIST 1.1 and gives a timeframe of 2 years. The primary outcome result itself is not reported in the registry record.
The endpoint combines response and disease stability into one measure. In general, a combined endpoint can capture more than one kind of favorable disease status, but its interpretation depends on the exact definitions, assessment schedule, and population included. The registry description defines partial response as a 30% or greater decrease lasting a minimum of 4 weeks in the measurable lesion, using the product of the lesion's perpendicular diameters. It also states that complete response or partial response must occur within the specified post-treatment period, while stable disease must last at least the stated duration.
The endpoint title uses the word “rate,” but the posted measure is a participant count. A denominator, the number assessed for the endpoint, and a reported rate are not reported in the registry record. Without those details and the outcome count, the endpoint cannot be interpreted as an observed proportion or used to assess the precision of an estimated rate.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record describes a single-group study that enrolled 10 participants and was terminated. It does not report the primary clinical-benefit result. Therefore, the posted information does not establish how many participants met the endpoint definition, how often each response category occurred, or how precisely any clinical-benefit estimate was measured. No efficacy conclusion can be drawn from the enrollment and design details alone.
The enrollment of 10 is below the study description's stated plan to enroll up to 44 participants. This difference identifies a gap between the planned maximum and the recorded enrollment, but does not explain why recruitment stopped or what the study team intended to do with the available data. The reason for termination and any planned sample-size justification are not reported in the registry record.
In general, a single-group study can describe outcomes among participants who receive the intervention, but it cannot separate changes associated with treatment from changes that might have occurred without it. Without a concurrent control group, differences due to participant characteristics, disease course, other care, or assessment timing cannot be ruled out through a randomized comparison. This design limitation remains even if participant outcomes are completely observed.
The registry record lists 2 outcome measures and 0 analyses. The safety information includes serious adverse-event counts for the BIBF 1120 group, but the record does not provide the primary endpoint count or a statistical analysis of that endpoint. A p-value, confidence interval, adjusted estimate, or other inferential result is not reported in the registry record. The absence of a recorded analysis means the posted material does not quantify uncertainty around a treatment-effect estimate.
In general, a confidence interval describes the range of values compatible with an estimate under the method used, and its width conveys precision. It would not establish causation in this single-group design. No confidence interval for clinical benefit is reported in the registry record, so neither the estimated benefit nor its precision can be assessed from the primary outcome information.
The sample-size assumptions associated with planning illustrations describe a hypothetical two-arm framework: two-sided significance level 0.05, equal allocation between arms, and a normal approximation, with the target effect expressed as a fraction of the observed effect. They are explicitly planning illustrations rather than the trial's own design. Since this study is recorded as single-group and no primary effect estimate is reported, those assumptions should not be read as an observed trial comparison or as evidence of observed power. The trial's own sample-size justification is not reported in the registry record.
9. Statistical Methods Explained
The primary outcome is a count of participants meeting a composite clinical-benefit definition. The definition includes complete response or partial response within 3 months post-treatment, or stable disease for at least three months post-treatment. The recorded outcome timeframe is 2 years, and assessment is described as using RECIST 1.1. The registry record does not report the observed count, the number of participants assessed, or a calculated rate.
What does a participant count tell the reader?
A count records how many people met a stated condition. To interpret it as a rate or proportion, readers also need the relevant denominator: for example, how many participants were eligible for assessment and how missing assessments were handled. The endpoint title says “rate,” but the measure is recorded as a count of participants. A denominator and the method for deriving a rate are not reported in the registry record. No rate should therefore be inferred from the enrollment figure alone.
In general, binary outcomes classify each participant according to whether a condition was met. A count of participants with an event is descriptive; an estimated proportion requires a denominator, and an interval estimate requires a stated statistical method. This study's primary result and any corresponding uncertainty measure are not reported in the registry record.
How is clinical benefit defined?
The record combines response and stable disease. It describes partial response as a 30% or greater decrease for a minimum of 4 weeks in the measurable lesion, based on the product of the perpendicular diameters. It says every lesion need not regress for a partial response, but progression of any lesion or appearance of new lesions prevents classification as partial response. The description also states that stable disease is between minor response and progressive disease, and defines progressive disease as an increase of 25% in the size of any measured lesion from baseline or the appearance of new lesions. Mixed responses are classified as progressive disease. These criteria indicate that classifications depend on measured disease and lesion changes, rather than on a participant's subjective report alone.
The record also defines pathological complete response as no evidence of residual invasive tumor, including in the axillary lymph nodes. However, details of how response assessments were conducted, reviewed, or timed are not reported in the registry record. The outcome description's timeframe of 2 years should not be taken to mean that every participant had 2 years of observation; individual follow-up durations are not reported in the registry record.
What do the design and analysis entries imply?
The trial is recorded as single-group, with allocation not applicable and no masking. In general, an unmasked study is one in which treatment assignment is not concealed from participants or relevant study personnel. With no comparator group, the main interpretive task would be describing the observed outcomes in the treated group; a treatment contrast against usual care or another therapy cannot be calculated from this design. The registry record does not report an analysis plan, an analysis population, or methods for handling missing outcome assessments.
The record lists 2 outcome measures and 0 analyses. This means no statistical analysis is represented in the posted analysis count; it does not reveal what analyses may have been considered outside the registry record. There is no reported primary-outcome estimate, confidence interval, p-value, or adjustment for covariates. In general, a confidence interval expresses uncertainty around an estimate under specified assumptions, while a p-value assesses compatibility with a particular null hypothesis under a statistical model. Neither can be interpreted when no corresponding result and method are reported.
How should planning illustrations be understood?
The planning assumptions describe a two-arm calculation using a two-sided significance level of 0.05, equal allocation, and a normal approximation. The target effect is described as a fraction of the observed effect. These are planning assumptions, not the actual design of this single-group study. In general, planning calculations describe sample sizes under specified assumptions; they are not measures of the probability that a completed study has found a true effect. The illustrations are not observed power. Because the primary outcome estimate is not reported, the target based on a fraction of the observed effect cannot be checked against a posted result. The trial's own sample-size justification is not reported in the registry record.
Over-dispersion is a general concern when counts vary more than a chosen probability model expects; it can make model-based uncertainty estimates too small if ignored. The registry record does not report a count model or an over-dispersion assessment for the primary outcome. No claim about such a method can be made from the posted entries.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists 2 outcome measures, including the primary clinical-benefit measure and a safety measure concerning adverse-event type, frequency, and severity under CTCAE Version 4.0. It lists 0 analyses. The statistical analysis plan and any method for handling multiple outcomes or repeated testing are not reported in the registry record. There is consequently no basis for determining whether any multiplicity adjustment was planned or applied.
In general, multiplicity arises when several outcomes or statistical tests are considered. Without a prespecified analysis plan, readers cannot establish which outcome was intended to drive a formal conclusion or how the chance of false-positive findings was managed. No adjusted or unadjusted hypothesis-test results for clinical benefit are reported in the registry record.
Missing outcome data can affect both the numerator and denominator of a clinical-benefit rate. The registry record does not report the number of participants with evaluable response assessments, any missingness, reasons for missing assessments, or an imputation or sensitivity-analysis method. It is also not reported in the registry record whether every enrolled participant was included in an analysis population or whether an intention-to-treat approach was planned.
These omissions matter because a participant count is not enough to reconstruct an outcome rate when the assessed population and missing-data handling are unknown. The record's enrollment of 10 should not be substituted for an unreported endpoint denominator. Further analysis-population details and missing-data procedures are not reported in the registry record.
11. Safety Results
The registry reports serious adverse events as affected participants divided by participants at risk.
| Arm | Participants with serious adverse events | Participants at risk | Percentage |
|---|---|---|---|
| BIBF 1120 | 1 | 9 | 11.1% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| BIBF 1120 | 1 of 9 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 5% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| BIBF 1120 | 9 of 9 |
Most frequent serious adverse event terms
| Event term | Organ system | BIBF 1120 |
|---|---|---|
| Hypoxia | Respiratory, thoracic and mediastinal disorders | 1 of 9 |
| Dyspnea | Respiratory, thoracic and mediastinal disorders | 1 of 9 |
Most frequent non-serious adverse event terms
| Event term | Organ system | BIBF 1120 |
|---|---|---|
| Vomiting | Gastrointestinal disorders | 4 of 9 |
| Diarrhea | Gastrointestinal disorders | 4 of 9 |
| Fatigue | General disorders | 4 of 9 |
| Nausea | Gastrointestinal disorders | 3 of 9 |
| Abdominal pain | Gastrointestinal disorders | 2 of 9 |
| Arthralgia | Musculoskeletal and connective tissue disorders | 2 of 9 |
| Tumor pain | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 2 of 9 |
| Platelet count decreased | Blood and lymphatic system disorders | 2 of 9 |
| Headache | Nervous system disorders | 1 of 9 |
| Chest wall pain | Musculoskeletal and connective tissue disorders | 1 of 9 |
Serious events with the highest arm-level rate: hypoxia (11.1%), dyspnea (11.1%).
The registry safety entries list 9 participants at risk for serious adverse events in the BIBF 1120 group and 1 participant with a serious adverse event. The record lists 9 participants at risk for other adverse events and 9 participants with other adverse events. It also records 1 death. The serious terms listed most often are hypoxia and dyspnea, each reported as 11.1%.
These figures describe events recorded in the study group; they do not by themselves show whether an event was caused by nintedanib. The record does not report a comparison group, an attribution analysis, event timing, or the relationship between the death and any adverse event. The listed participant counts also do not establish the number of distinct events when a participant may have had more than one event.
In general, safety counts should be read with their denominator, event definitions, observation period, and rules for attribution in mind. The registry outcome description refers to CTCAE Version 4.0 for type, frequency, and severity, but further details of safety monitoring and the event-level data are not reported in the registry record. The serious adverse-event figures are not evidence for or against a causal relationship on their own.
12. What the Result Does, and Does Not, Mean
The posted information establishes that a Phase II, single-group study of nintedanib for metastatic HER2-negative inflammatory breast cancer was conducted, that enrollment was recorded as 10, and that the study status is terminated. It specifies a clinical-benefit endpoint based on response or stable disease and lists safety measures. It also records serious adverse-event counts in the BIBF 1120 group.
It does not report the primary endpoint result. Readers therefore cannot determine from the registry record how many participants achieved complete response, partial response, stable disease, or the combined clinical-benefit endpoint. The endpoint denominator and a confidence interval or other estimate of precision are also not reported in the registry record. The enrollment count is not a substitute for any of these outcome details.
Because the study has no control group, even complete outcome data would describe what happened among participants in the study, not what would have happened to comparable participants without the intervention. In general, causal treatment comparisons are stronger when treatment groups are made comparable through random allocation and assessed with an appropriate analysis. This record describes neither random allocation nor a comparator group.
The serious adverse-event and death counts are descriptive. They do not establish that an event was caused by study treatment, and the posted safety entries do not include an attribution analysis or comparative risk estimate. The results also cannot be generalized beyond the recorded study population without additional evidence about participant characteristics and the context of care, which are not reported in the registry record.
Overall, the record documents the design, enrollment, endpoint definitions, and selected safety counts, but not the primary clinical outcome or a statistical analysis. It therefore supports a description of the study and its reporting limits, not a conclusion about treatment effect or the precision of an effect estimate.
13. Why This Trial Matters Statistically
This trial is statistically notable because its primary outcome is labeled a clinical-benefit rate but is recorded as a count of participants. A count is informative only alongside a clear denominator and an account of who was assessed. Neither the primary count nor its denominator is reported in the registry record, so a rate and its precision cannot be evaluated.
The study is also a single-group trial with no masking and no randomized comparator. In general, such a design can describe outcomes in participants receiving the intervention, but it cannot provide a randomized estimate of the difference between treatment and control. That distinction is important when interpreting response categories: a favorable observed outcome does not by itself identify what portion, if any, is attributable to the intervention.
Enrollment was recorded as 10, while the study description said up to 44 participants would be enrolled. The study is marked terminated, but the reason for termination and the planned sample-size justification are not reported in the registry record. Without those details and the primary outcome result, the precision and completeness of the intended assessment cannot be judged.
The safety entries offer some descriptive counts, but the record does not report comparative safety estimates or causal attribution. This illustrates a broader statistical distinction: recorded events can characterize what was observed, while estimating excess risk requires an appropriate comparison and clearly defined follow-up.
Finally, planning assumptions for two arms should not be mistaken for a description of this single-group study. Recognizing the difference between hypothetical planning calculations, observed participant data, and an analysis of treatment effect is central to interpreting the posted information accurately.
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 primary clinical-benefit outcome has no reported result, denominator, or uncertainty estimate. The record therefore does not allow an assessment of the observed clinical-benefit count or rate. Although the enrollment is recorded as 10, the endpoint analysis population is not reported in the registry record.
The single-group design has no randomized control arm, and masking is listed as none. Consequently, the study cannot yield a direct randomized comparison. The reason for the recorded terminated status is not reported in the registry record, so its implications for recruitment, follow-up, or analysis cannot be assessed.
The study description states that up to 44 participants would be enrolled, while the recorded enrollment is 10. The planned sample-size justification, expected effect, and assumptions underlying the original design are not reported in the registry record. The separate planning assumptions describe a hypothetical two-arm framework and should not be taken as the trial's own design or as observed power.
Important outcome details are also unavailable: response-assessment schedules and procedures, the number of participants with evaluable assessments, missing-data handling, analysis populations, and the statistical analysis plan are not reported in the registry record. The endpoint title uses “rate,” but the recorded unit is a participant count, and the denominator is not specified in the posted results.
The safety information is limited to participant counts, one death, and two listed serious adverse-event terms with percentages. Event-level details, timing, treatment attribution, and comparative safety analyses are not reported in the registry record. These limitations restrict both the statistical interpretation of outcomes and the strength of any general conclusion from the posted information.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Nintedanib For HER2-Negative Metastatic Inflammatory Breast Cancer (MIBC) — NCT02389764.
- Registry record last updated 2019-07-17; status last verified 2019-06.
- Results first posted 2019-07-17.
- 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
NCT02389764 is a Phase II, single-group study of nintedanib, also called BIBF 1120, in metastatic HER2-negative inflammatory breast cancer. M.D. Anderson Cancer Center is listed as sponsor. The study started on 2015-06-22, had a primary completion date of 2018-06-08, and is recorded as terminated. Enrollment is 10; the study description stated that up to 44 participants would be enrolled.
The primary endpoint is a count of participants with clinical benefit, defined using complete response, partial response, or stable disease, with a 2-year timeframe and RECIST 1.1 named in the description. Safety measures are also listed. The record reports selected adverse-event counts but no primary outcome result, statistical analysis, or reason for termination. Further details about the analysis plan, outcome denominator, and missing data are not reported in the registry record.