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 A Retrospective Observational Study of Patients With Early-stage HER2-positive Breast Cancer, Treated With Neratinib (NCT05599334), a registered phase not stated study. The primary outcome is Description of Age.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT05599334 |
| Phase | Phase not stated |
| Condition | Breast Cancer, Adjuvant Therapy, HER2-positive Breast Cancer |
| Allocation | Not Stated |
| Design model | Not Stated |
| Masking | Not Stated |
| Enrollment | 111 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Pierre Fabre Medicament |
| Results posted | Yes |
2. Clinical Question
Population
Adults with early-stage HER2-positive breast cancer treated with neratinib in the context of the European Early Access Program. The registry lists 111 enrolled participants. The stated population for the primary measure is FAS, but the registry record does not report how that population is defined.
Intervention
Neratinib used as extended adjuvant therapy. The study describes treatment received in the European Early Access Program; it is retrospective and observational rather than a randomized assignment of treatment.
Comparator
No like-for-like control comparison is reported. The registry states that allocation was not stated, and the listed Neratinib arm should not be interpreted as a randomized comparison against another treatment or an untreated group.
Primary question
The primary outcome is a description of age at baseline, expressed in years and summarized using the mean and standard deviation. The record does not report the resulting mean or standard deviation, nor does it report an analysis comparing age between groups.
The study’s stated purpose is to describe demographic and clinical profiles among patients with early-stage HER2-positive breast cancer treated with neratinib as extended adjuvant therapy in the European Early Access Program. Its question is therefore descriptive: what characteristics were recorded for the treated population? It is not framed in the registry as a randomized test of neratinib against a comparator.
Age is the designated primary measure. The registry defines its timing as baseline and its calculation as the neratinib initiation date minus the date of birth, with the result expressed in years. This indicates what age measure was intended, but does not supply the observed age distribution. The registry record does not report an age mean, standard deviation, or other summary values.
The study is listed as completed, with 111 enrolled participants. Those details describe the registry record and do not establish how many had age data available for the primary measure. The record does not report the number with an observed age value or the handling of missing age data. The central statistical question that can be answered from the record is thus limited to what was intended to be described, not the actual age profile or any treatment effect.
3. Trial Design
This is a retrospective observational study of adults with early-stage HER2-positive breast cancer treated with neratinib as extended adjuvant therapy in the context of the European Early Access Program in Europe. The registry describes the purpose as characterizing demographic and clinical profiles. It lists Pierre Fabre Medicament as sponsor and reports the study status as completed.
The registry lists 111 enrolled participants. The recorded study start date is 2022-07-05, and the primary completion date is 2023-01-30. These dates and enrollment describe the registry entry; they do not give the number of records available for every outcome. The registry does not report the data collection process, the source records used, or the timing and completeness of follow-up for outcomes other than the primary measure’s baseline designation.
The record does not describe randomized assignment. Allocation is not stated, and the registry cautions that the arms are not a like-for-like control comparison. The listed Neratinib arm is therefore not evidence of a randomized contrast. No comparator group suitable for estimating a relative treatment effect is reported. Any description of outcomes in the treated group should be read as observational rather than as a causal comparison.
The primary measure is age at baseline. The registry specifies a mean and standard deviation and names FAS as the population. The record does not define FAS or report how this population relates to enrollment. It lists 11 outcome measures and 0 analyses. The protocol, detailed statistical analysis plan, eligibility criteria, and approach to confounding are not reported in the registry record. The status “completed” does not by itself establish that every planned outcome was analyzed or that results were posted.
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 | Neratinib Arm |
|---|---|
| STARTED | 111 |
| COMPLETED | 108 |
| NOT COMPLETED | 3 |
Analysis population for the primary outcome, as defined in the registry: FAS
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.
Population: The baseline characteristics have been analysed in 108 patients corresponding to the number of patients analysed in the Full Analysis Set
| Characteristic | Unit | Neratinib Arm |
|---|---|---|
| Participants analysed | 111 | |
| Age, Continuous | years | 49.2 (SD 8.83) |
| Sex: Female, Male: Female | Participants | 107 |
| Sex: Female, Male: Male | Participants | 1 |
| 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 | 0 |
| Race (NIH/OMB): White | Participants | 0 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Region of Enrollment: Croatia | participants | 35 |
| Region of Enrollment: Spain | participants | 24 |
| Region of Enrollment: Italy | participants | 21 |
| Region of Enrollment: Belgium | participants | 16 |
| Region of Enrollment: France | participants | 15 |
6. Primary Endpoint
The registry lists the primary outcome as Description of Age, measured over At baseline.
| Element | Registry entry |
|---|---|
| Result type | Mean |
| Measure of spread | Standard Deviation |
| Unit | years |
| Endpoint classification | Continuous Endpoint |
The primary measure is titled “Description of Age.” It is a continuous measure expressed in years, with the mean as the stated parameter and the standard deviation as the stated measure of dispersion. Its timeframe is baseline. The registry defines age as the neratinib initiation date minus the date of birth, and identifies FAS as the population for this measure.
Age is commonly described in clinical research because it helps characterize who was included in a study. A mean gives a summary of the center of the observed values, while a standard deviation describes how spread out those values are around the mean. These are descriptive statistics: they summarize a distribution and do not, by themselves, establish a treatment effect or explain why participants have different outcomes.
The registry’s description identifies the intended measurement and summary, but it does not report the observed mean or standard deviation. It also does not report the number of participants with an available age value, the age distribution, or any confidence interval. The stated primary measure therefore cannot be interpreted as an observed result from the information in the registry record. It is also not a measure of efficacy or a comparison of neratinib with another treatment.
The record does not explain whether the baseline age calculation was adjusted for any other factors. It does not report the handling of missing or invalid dates. Those details would matter when assessing how representative the reported age summary is, if such a summary were available.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record supports a description of the study’s setting, population of interest, and intended primary measure, but it does not provide an observed age result. Although the primary outcome is defined as mean age with standard deviation at baseline, neither statistic is reported. There is consequently no numerical age estimate to interpret, and no precision estimate such as a confidence interval is reported for that measure.
The study is observational and retrospective. Its purpose is to describe the profiles of adults with early-stage HER2-positive breast cancer treated with neratinib as extended adjuvant therapy in a European Early Access Program. This design can describe characteristics recorded among treated participants, but the registry does not report a randomized comparator or an analysis that estimates what would have happened under a different treatment. The record explicitly cautions that the arms are not a like-for-like control comparison. It therefore does not support interpreting differences between any listed groups as causal treatment effects.
The registry reports 111 enrolled participants, while the adverse-event record lists 108 at risk for the Neratinib arm. These figures refer to different registry contexts, and the record does not explain the relationship between them. They should not be treated as interchangeable denominators or used to infer outcome completeness. The registry does not report how many participants contributed data to the primary age measure.
The record lists 11 outcome measures and 0 analyses. This is important for interpretation: the existence of an outcome definition does not mean that an observed estimate or inferential test was posted. No p-value, confidence interval, adjusted estimate, or statistical comparison for the primary measure is reported. The record also does not report a multiplicity strategy or a missing-data method. Absence of those details is not evidence that a particular method was or was not used outside the registry entry.
The safety figures are descriptive counts in the listed Neratinib arm. They provide neither a concurrent control rate nor an estimate of excess risk attributable to treatment. The serious adverse event terms and percentages recorded by the registry should be read as reported, without assuming that events were caused by neratinib or that the percentages are mutually exclusive. The record does not provide enough information to assess event timing, overlap, or causal attribution.
Overall, the available record describes a completed retrospective study with a stated descriptive objective and a defined age measure. It does not report the primary outcome result or an inferential analysis. The appropriate interpretation is therefore about what the study set out to characterize and what adverse-event counts were entered, not about comparative effectiveness or a causal safety conclusion.
9. Statistical Methods Explained
Statistical interpretation begins with the design. This study is described as retrospective and observational, with treatment received as extended adjuvant neratinib in the context of a European Early Access Program. In general, an observational study records exposures and outcomes without randomized treatment assignment. Such data can be useful for describing a treated population, but differences between groups can reflect differences in who received each treatment as well as treatment itself. Here, the registry says allocation was not stated and warns that the arms are not a like-for-like control comparison.
What does the primary measure summarize?
Age is a continuous variable measured in years. The stated summary is the mean, accompanied by the standard deviation. In general, the mean is the arithmetic center of observed values, while the standard deviation describes their spread around that center. A standard deviation is not the same as a confidence interval: it describes variation among observations, whereas a confidence interval describes uncertainty around an estimate under a statistical model. The registry record does not report either the observed mean or the standard deviation, and it does not report a confidence interval for age.
The endpoint is specified at baseline and defined using the neratinib initiation date and date of birth. The population is identified as FAS, but that term is not defined in the registry record. The number of participants with usable dates and the handling of missing dates are also not reported. These omissions prevent assessment of whether the age summary, if calculated, would represent all enrolled participants or a subset with available information.
What can the adverse-event denominators tell a reader?
The serious adverse event record lists 108 at risk and 12 affected in the Neratinib arm. The other adverse event record lists 108 at risk and 10 affected. These are descriptive registry counts. The record also lists 1 death. The entries do not establish a comparison with a control group, and they do not, by themselves, show that an event was caused by treatment.
In general, a count needs its denominator and a clear definition to be interpreted. A denominator indicates the recorded population at risk for that outcome, but it does not explain whether every participant was followed for the same period or whether all events were captured. The registry does not report those details. It also does not explain why the adverse-event risk denominator differs from the enrollment figure, so no explanation should be inferred from the counts alone.
Seriousness is a classification of an adverse event and is not interchangeable with intensity or severity. The record lists the serious terms Hypokalaemia and Hyperkalaemia at 1.9% each, and Subarachnoid haemorrhage, Infected seroma, Diarrhea, and Death at 0.9% each. The registry does not say whether a participant could appear under more than one term, provide event-level timing, or report attribution assessments. Accordingly, these entries should be treated as reported safety descriptions, not as independent, mutually exclusive categories or causal estimates.
How should planning illustrations be understood?
The stated sample-size assumptions describe planning illustrations based on an observed effect, using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. The stated effect to detect is a fraction of the observed effect. These are planning assumptions, not evidence that the retrospective study was designed with those features. The registry does not report a randomized two-arm design, and its own note says the arms are not a like-for-like control comparison.
In general, a planning illustration asks how much information a hypothetical study might need under specified assumptions. It is not “observed power,” and it cannot establish that a result is reliable or clinically important. The registry does not report the study’s own sample-size justification, so the planning assumptions should not be mistaken for the protocol’s rationale or analysis plan.
The registry lists 11 outcome measures and 0 analyses. It does not report a statistical analysis plan, inferential tests, adjustment methods, or a multiplicity strategy. Without those details and without a posted age estimate, the primary endpoint can be explained methodologically but not evaluated as a numerical result. General statistical concepts help clarify what a mean, standard deviation, denominator, or planning illustration means; they do not fill in the missing study-specific estimates.
10. Confidence Intervals for Each Arm
No two-arm comparison is shown for this trial, but each arm's mean can be given with a t-based 95% confidence interval computed from the posted mean, spread and number of participants.
| Arm | Participants | Mean | Standard deviation | 95% CI for the mean |
|---|---|---|---|---|
| Neratinib Arm | 108 | 49.200 | 8.830 | 47.516 to 50.884 |
R code and output
arm <- c("Neratinib Arm")
n <- c(108)
m <- c(49.2)
sp <- c(8.83)
sd <- sp # posted standard deviations
for (i in seq_along(n)) {
se <- sd[i] / sqrt(n[i]); h <- qt(0.975, n[i] - 1) * se
cat(sprintf("CARM|%s|%d|%.3f|%.3f|%.3f|%.3f\n", arm[i], n[i], m[i], sd[i], m[i] - h, m[i] + h))
}
CARM Neratinib Arm 108 49.200 8.830 47.516 50.88411. Multiplicity, Missing Data and Analysis Populations
The registry lists 11 outcome measures and 0 analyses. The primary measure is specified as baseline age, summarized by a mean and standard deviation, but no observed result for that measure is reported. The record does not identify which of the listed outcomes were analyzed or provide inferential results for them.
When a study examines multiple outcomes, multiplicity can matter because repeated statistical tests may increase the chance of a chance-positive finding. In general, investigators may prespecify a primary outcome or use an adjustment strategy, but the registry record does not report a multiplicity plan or say whether any adjustment was used. No conclusion about the presence or absence of such procedures outside the registry entry is warranted.
The record names FAS as the population for the age measure but does not define the acronym or explain who is included. It reports 111 enrolled participants, but does not report the number with available age data or the number analyzed for the primary measure. The adverse-event risk denominator is reported separately; its relationship to enrollment is not explained.
The approach to missing data is not reported in the registry record. In general, missing values can affect a descriptive mean if the participants with recorded values differ from those without them. The record does not state whether missing age values were excluded, imputed, or handled in another way. It also does not report the extent or reasons for missing data. Without these details, the representativeness and precision of any unposted estimate cannot be assessed.
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 |
|---|---|---|---|
| Neratinib Arm | 12 | 108 | 11.1% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Neratinib Arm | 1 of 108 |
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 |
|---|---|
| Neratinib Arm | 10 of 108 |
Most frequent serious adverse event terms
| Event term | Organ system | Neratinib Arm |
|---|---|---|
| Hypokalaemia | Metabolism and nutrition disorders | 2 of 108 |
| Hyperkalaemia | Metabolism and nutrition disorders | 2 of 108 |
| Subarachnoid haemorrhage | Nervous system disorders | 1 of 108 |
| Infected seroma | Infections and infestations | 1 of 108 |
| Diarrhea | Gastrointestinal disorders | 1 of 108 |
| Death | General disorders | 1 of 108 |
| Pyrexia | General disorders | 1 of 108 |
| Chest pain | General disorders | 1 of 108 |
| Hepatic enzymze increased | Investigations | 1 of 108 |
| Hypercalcaemia | Metabolism and nutrition disorders | 1 of 108 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Neratinib Arm |
|---|---|---|
| Diarrhea | Gastrointestinal disorders | 10 of 108 |
| Fatigue | General disorders | 5 of 108 |
| Nausea | Gastrointestinal disorders | 4 of 108 |
| Abdominal pain upper | Gastrointestinal disorders | 3 of 108 |
| Vomiting | Gastrointestinal disorders | 3 of 108 |
| Decreased apetite | Metabolism and nutrition disorders | 2 of 108 |
| Dizziness | Nervous system disorders | 2 of 108 |
| Hyperlipidaemia | Metabolism and nutrition disorders | 2 of 108 |
| Muscle spasm | Musculoskeletal and connective tissue disorders | 2 of 108 |
| rash | Skin and subcutaneous tissue disorders | 2 of 108 |
Serious events with the highest arm-level rate: hypokalaemia (1.9%), hyperkalaemia (1.9%), subarachnoid haemorrhage (0.9%), infected seroma (0.9%), diarrhea (0.9%).
The registry’s Neratinib arm safety record lists 108 at risk for serious adverse events and 12 affected. It separately lists 108 at risk for other adverse events and 10 affected. The record lists 1 death. These are descriptive entries, not a comparison against a control group, and they do not establish that neratinib caused any event.
The listed serious adverse event terms include Hypokalaemia and Hyperkalaemia, each reported as 1.9%, and Subarachnoid haemorrhage, Infected seroma, Diarrhea, and Death, each reported as 0.9%. These registry percentages should be read as recorded; the entry does not provide further information on event timing, overlap among terms, or how attribution was assessed. The record does not explain the difference between the enrollment figure and the safety denominator, so that difference should not be assigned a cause.
In general, “serious” is a reporting classification and does not simply mean that an event was more intense than another event. A count and denominator give context, but interpretation also depends on follow-up duration, event definitions, and completeness of reporting. Those details are not reported in the registry record. The safety entries therefore describe what was recorded, without supporting a comparative risk estimate or a causal conclusion.
13. What the Result Does, and Does Not, Mean
The record establishes that a retrospective observational study was conducted to describe demographic and clinical profiles of adults with early-stage HER2-positive breast cancer treated with neratinib as extended adjuvant therapy in a European Early Access Program. It identifies baseline age as the primary measure and specifies that age was to be summarized using a mean and standard deviation. It also lists safety counts and serious adverse event terms for the Neratinib arm.
Those details do not provide the observed age mean or standard deviation. The registry record does not report the primary measure’s numerical result, the number with available age data, or a confidence interval. It also lists 11 outcome measures and 0 analyses. Therefore, the record cannot support a numerical conclusion about the age profile or an inferential conclusion about the primary measure.
The record does not establish that neratinib caused or prevented any outcome. The study is observational, allocation is not stated, and the registry warns that the arms are not a like-for-like control comparison. No randomized comparator or causal analysis is reported. The adverse-event entries are descriptive counts and terms; without a suitable comparison, they do not quantify a treatment-attributable increase or decrease in risk.
The enrollment figure and safety denominators describe different parts of the record, and the reason for their difference is not reported. Neither should be used to infer that all participants had complete outcome data. Missing-data methods, follow-up completeness, and details of the analysis population are not reported. The stated population label FAS is not defined in the registry entry.
In general, a descriptive observational study can add information about the characteristics and recorded events of a treated population. The strength of any such interpretation depends on how participants were selected, how outcomes were captured, and how complete the records were. Those details are not all. The appropriate conclusion is limited to the study’s stated descriptive purpose and the specific registry entries, without extending them to comparative efficacy or causal safety claims.
14. Why This Trial Matters Statistically
This study is statistically notable for the distinction between an outcome definition and an outcome result. The registry clearly specifies baseline age as the primary measure, including the unit, summary statistic, dispersion measure, and stated population. Yet it lists 0 analyses and does not report the age mean or standard deviation. This illustrates why a well-defined endpoint does not, on its own, permit interpretation of an observed result.
The retrospective observational setting is also central to statistical interpretation. The stated purpose is to characterize people treated with neratinib in an early access context, not to report a randomized efficacy comparison. The registry does not state allocation and cautions that the arms are not a like-for-like control comparison. That distinction limits the questions the available data can answer: descriptive features can be reported, but a causal contrast requires a valid comparison and appropriate methods that are not described in this record.
The safety record provides counts, denominators, and selected serious adverse event terms. These entries can help readers understand what the registry recorded, while also showing why denominators and event definitions matter. The record does not report follow-up duration, event overlap, or causal attribution, so counts should not be converted into stronger claims than they support.
Finally, the registry lists 11 outcome measures but no analyses. This makes transparency about analysis plans, missing data, and multiplicity especially relevant. The record does not report these methods, and it does not provide a sample-size justification for the study’s own design. Any planning illustrations are not observed power and should not be mistaken for evidence that the study was planned as a randomized comparison. Together, these features make the record useful for understanding the stated study question and its reporting limits, while leaving the primary descriptive result unavailable.
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.
The most direct limitation is that the primary age result is not reported. The registry specifies a mean and standard deviation at baseline but provides neither value, nor a confidence interval or the number of participants with an available age measure. As a result, the stated primary endpoint cannot be interpreted numerically.
The design is retrospective and observational, and no like-for-like control comparison is reported. Allocation is not stated. This limits causal interpretation of outcomes and means the listed Neratinib arm should not be treated as a randomized treatment group. The record does not describe how treatment selection, clinical differences, or potential confounding were addressed.
The registry lists 111 enrolled participants, whereas the adverse-event entries report 108 at risk. The record does not explain this difference or show how many participants contributed to each outcome. It does not report the completeness of follow-up, missing-data methods, or the definition of FAS. These gaps limit assessment of representativeness and outcome completeness.
Although 11 outcome measures are listed, the registry reports 0 analyses. It does not provide a statistical analysis plan, multiplicity approach, or inferential results. The serious adverse event terms and percentages are reported without event-level timing, overlap information, or attribution detail. Consequently, they support description of recorded entries but not comparative or causal safety conclusions.
The registry reports the study as completed, but completion status does not fill in these reporting gaps. The study’s own sample-size justification and detailed data collection methods are not reported in the registry record. These limitations are specific to what is recorded for this study and should frame interpretation of both the endpoint and safety entries.
16. Related Tutorials
17. Related Statistical Calculators
18. Sources
- ClinicalTrials.gov: A Retrospective Observational Study of Patients With Early-stage HER2-positive Breast Cancer, Treated With Neratinib — NCT05599334.
- Registry record last updated 2025-10-06; status last verified 2025-09.
- Results first posted 2025-10-06.
- 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.
Continue through the Clinical Biostats statistical pathway
Use this trial's endpoint as a starting point for deeper study of effect measures, confidence intervals, and sample-size planning.
19. Record Summary
The registry record describes a completed retrospective observational study of adults with early-stage HER2-positive breast cancer treated with neratinib as extended adjuvant therapy in the context of a European Early Access Program. Pierre Fabre Medicament is listed as sponsor. The recorded start date is 2022-07-05, the primary completion date is 2023-01-30, and enrollment is listed as 111.
The primary measure is baseline age in years, summarized by mean and standard deviation, with FAS named as the population. The record lists 11 outcome measures and 0 analyses; it does not report the observed age summary or define FAS. The safety record lists 108 at risk and 12 affected for serious adverse events, 108 at risk and 10 affected for other adverse events, and 1 death. It also lists selected serious adverse event terms and percentages.
No randomized comparator is reported, allocation is not stated, and the registry cautions that the arms are not a like-for-like control comparison. Missing-data methods, multiplicity procedures, and the study’s own sample-size justification are not reported in the registry record. The available information supports a description of the stated study and its recorded entries, but not a comparative or causal treatment conclusion.