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 Pilot Study to Evaluate Reparixin With Weekly Paclitaxel in Patients With HER 2 Negative Metastatic Breast Cancer (MBC) (NCT02001974), a non-randomized phase 1 study. The primary outcome is Treatment-Emergent Adverse Events (TEAEs).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02001974 |
| Phase | Phase 1 |
| Condition | Metastatic Breast Cancer |
| Allocation | Non-Randomized |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 33 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Dompé Farmaceutici S.p.A |
| Results posted | Yes |
2. Clinical Question
Population
Patients with HER 2 negative metastatic breast cancer. The study enrolled 33 participants; the safety population was defined as enrolled patients who took at least one dose of reparixin.
Intervention
Oral reparixin combined with intravenous paclitaxel. The described groups used reparixin doses of 400 mg, 800 mg or 1200 mg three times daily, with paclitaxel 80 mg/m2 on Days 1, 8, and 15 of a 28-day cycle.
Comparator
No separate control group is described. The study was non-randomized and used a single-group model; the dose groups are not a like-for-like control comparison.
Primary question
The primary objective was to evaluate safety and define the pharmacokinetic profile of orally administered reparixin in combination with paclitaxel. The primary outcome was treatment-emergent adverse events, monitored through the end or off-treatment visit and up to 28 days following the last dose, with the stated timeframe extending up to 24 months.
The study also had exploratory and secondary objectives. These included assessing cancer stem cell markers, the tumoral microenvironment, cytokine-inflammation markers, circulating tumor cells and epithelial-mesenchymal transition biomarkers. Disease response was assessed as an indication of efficacy, with registry-listed secondary outcomes including response categories at assessment visits, best overall response, clinical benefit rate, 6-month progression-free survival rate and median time to tumor progression.
These objectives describe what investigators intended to evaluate, not the results. Numerical pharmacokinetic findings, biomarker results and response results are not reported in the registry record cited for this analysis. The design therefore supports a description of the planned safety and pharmacokinetic questions, but does not establish a comparative treatment effect.
3. Trial Design
This was a phase I study, titled as a Phase Ib pilot study, of reparixin in combination with weekly paclitaxel in HER 2 negative metastatic breast cancer. The registry record lists a non-randomized allocation, no masking and a single-group model. It records an enrollment of 33 and a completed study. The recorded start date was 2012-02-27, and primary completion was 2014-06-25.
The intervention was a combination of paclitaxel and reparixin. Paclitaxel was described at 80 mg/m2 intravenously on Days 1, 8, and 15 of a 28-day cycle. Reparixin was taken orally three times daily, three weeks on and one week off. Group 1 used 400 mg. Group 2 specified an increase to 800 mg if there was no toxicity in the previous group, and Group 3 specified an increase to 1200 mg if there was no toxicity in the previous group. Each group description states three to six patients.
The stated dose progression and toxicity condition describe the regimen structure, but the registry record does not report the specific dose-escalation algorithm, stopping rules or decision process used to assign participants. The number of participants listed as at risk in the safety figures varies by group; those figures should not be treated as randomized arm sizes or evidence of a controlled comparison.
With no randomization, masking or separate control group, the design is descriptive and exploratory. Outcomes across dose groups may reflect differences other than dose, and the groups are not a like-for-like control comparison. The registry record does not report a protocol-based statistical analysis plan, a sample-size justification or interim-analysis details. It also does not report results for the pharmacokinetic objective in the registry record for this analysis.
4. Participant Flow and Accounting
The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.
Before assignment: Reparixin + paclitaxel were administered in 28-day cycles as long as clinical benefit was observed. Reparixin was administered in oral tablets every six to ten hours during a 3 day run-in period and for 21 consecutive days during each cycle with 7 days off treatment between each cycle. Paclitaxel 80 mg/m2/week was administered in combination with the day's first dose of reparixin as an i.v. infusion on Days 1, 8 and 15 of each 28-day cycle.
Run-in Period
| Stage | Paclitaxel 80 mg/m2 i.v.+Reparixin Oral 400 mg t.i.d. | Paclitaxel 80 mg/m2 i.v.+Reparixin Oral 800 mg t.i.d. | Paclitaxel 80 mg/m2 i.v.+Reparixin Oral 1200 mg t.i.d. |
|---|---|---|---|
| STARTED | 4 | 3 | 26 |
| COMPLETED | 4 | 3 | 26 |
| NOT COMPLETED | 0 | 0 | 0 |
Reparixin + Paclitaxel (28-day Cycles)
| Stage | Paclitaxel 80 mg/m2 i.v.+Reparixin Oral 400 mg t.i.d. | Paclitaxel 80 mg/m2 i.v.+Reparixin Oral 800 mg t.i.d. | Paclitaxel 80 mg/m2 i.v.+Reparixin Oral 1200 mg t.i.d. |
|---|---|---|---|
| STARTED | 4 | 3 | 26 |
| COMPLETED | 0 | 0 | 0 |
| NOT COMPLETED | 4 | 3 | 26 |
Reasons for not completing this period:
| Reason | Paclitaxel 80 mg/m2 i.v.+Reparixin Oral 400 mg t.i.d. | Paclitaxel 80 mg/m2 i.v.+Reparixin Oral 800 mg t.i.d. | Paclitaxel 80 mg/m2 i.v.+Reparixin Oral 1200 mg t.i.d. |
|---|---|---|---|
| Disease progression | 2 | 3 | 14 |
| Adverse Event | 1 | 0 | 4 |
| Withdrawal by Subject | 1 | 0 | 6 |
| Physician Decision | 0 | 0 | 2 |
Analysis population for the primary outcome, as defined in the registry: Safety Population: all enrolled patients who took at least one dose of study drug, reparixin.
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: Safety Population: all enrolled patients who took at least one dose of study drug, reparixin.
| Characteristic | Unit | Group 1 | Group 2 | Group 3 | Total |
|---|---|---|---|---|---|
| Participants analysed | 4 | 3 | 23 | 30 | |
| Age, Categorical: <=18 years | Participants | 0 | 0 | 0 | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 3 | 3 | 16 | 22 |
| Age, Categorical: >=65 years | Participants | 1 | 0 | 7 | 8 |
| Age, Continuous | years | 55.0 (SD 9.13) | 55.3 (SD 7.77) | 56.2 (SD 12.69) | 55.9 (SD 11.59) |
| Sex: Female, Male: Female | Participants | 4 | 3 | 23 | 30 |
| Sex: Female, Male: Male | Participants | 0 | 0 | 0 | 0 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 | 0 | 1 | 1 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 4 | 3 | 22 | 29 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 1 | 0 | 0 | 1 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 | 2 | 3 | 5 |
| Race (NIH/OMB): White | Participants | 3 | 1 | 20 | 24 |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 | 0 |
| Region of Enrollment: United States | participants | 4 | 3 | 23 | 30 |
6. Primary Endpoint
The registry lists the primary outcome as Treatment-Emergent Adverse Events (TEAEs), measured over Up to 28 days following the last dose of study drug (up to 24 months)..
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | adverse events |
| Endpoint classification | not reduced to a single comparison |
The primary outcome is titled treatment-emergent adverse events (TEAEs), with the outcome parameter described as a number of adverse events. The monitoring description says adverse events were followed throughout the study until the end or off-treatment visit. Its timeframe is stated as up to 28 days following the last dose of study drug, with the timeframe listed as up to 24 months. The defined safety population includes all enrolled patients who took at least one dose of reparixin.
Safety is a central endpoint in an early-phase combination study because it characterizes adverse events observed during exposure and can inform how tolerability is assessed alongside dose and pharmacokinetic objectives. General statistical context: an event count and the number of participants experiencing at least one event are different summaries. A participant may experience more than one event, so a count of events should not automatically be read as a count of affected participants. The registry record does not provide a numerical result for the primary TEAE outcome in the registry record, nor does it specify a detailed event-count analysis method.
The posted serious-event figures separately report numbers at risk and numbers affected for each group. Those figures provide some safety information, but they do not replace a complete TEAE result, event-level descriptions or a comparative safety analysis. The registry record does not report a separate control-group estimate.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies safety and pharmacokinetics as the primary objectives, with TEAEs as the primary outcome. However, a numerical result for the primary TEAE outcome and numerical pharmacokinetic findings are not reported in the record details available for this analysis. The results therefore cannot be characterized as showing a particular overall event burden, exposure profile or dose-response pattern.
The serious-event figures are presented by group. Group 1 lists 4 at risk and 2 affected; Group 2 lists 3 at risk and 1 affected; Group 3 lists 23 at risk and 4 affected. Each group lists 0 deaths. The additional adverse-event figures list 4 at risk and 4 affected for Group 1, 3 at risk and 3 affected for Group 2, and 23 at risk and 23 affected for Group 3. These are descriptive registry counts. They do not establish that one dose has greater or lesser risk than another. The design was non-randomized, and the groups are not a like-for-like control comparison.
The record lists serious-event terms with percentages: Dehydration, 50.0%; Pneumothorax, 33.3%; Metastasis to central nervous system, 25.0%; Hyponatraemia, 25.0%; Dyspnoea, 4.3%; and Hypoxia, 4.3%. The denominator and precise basis for these percentages are not reported in the registry record. These values should therefore be read as listed, without inferring a population incidence or comparing them across terms.
General statistical context: safety counts in a small early-phase study are useful for describing events observed during follow-up, but estimates based on few participants are imprecise. A zero count of deaths in the posted group figures means no deaths are listed in those figures; it does not demonstrate that the risk of death is absent. Likewise, events recorded during the study do not by themselves establish that the study drug caused them.
The secondary outcomes include response categories, best overall response, clinical benefit rate, 6-month progression-free survival rate and median time to tumor progression. Their numerical results are not reported in the registry record details used. No conclusion about disease response or comparative efficacy can be drawn from the outcome names alone. The absence of a separate control group also means that any observed changes would not, by themselves, distinguish treatment effects from other explanations.
9. Statistical Methods Explained
The registry record describes the study as non-randomized, unmasked and single-group. Those design features determine what statistical interpretations are reasonable. There is no separately randomized control group against which the combination can be compared. The dose groups describe increasing reparixin doses under stated toxicity conditions, not randomized treatment arms. Differences among them cannot be attributed to dose alone from the design information available.
What does a single-group design allow?
General statistical context: a single-group study can describe outcomes among participants who received the intervention. It can characterize observed adverse events and, when reported, pharmacokinetic measurements or disease-response outcomes. Without a concurrent control group, it cannot isolate the effect of the intervention from the natural course of disease, other care or differences among participants. This limitation is particularly important when reading disease-response outcomes as an indication of efficacy.
The study title and objectives identify reparixin with weekly paclitaxel as the intervention of interest. Because paclitaxel was given as part of the combination, observed events or outcomes cannot be assigned to reparixin alone based on the design. The registry record does not report a method for separating the effects of the two drugs.
How should event counts be interpreted?
The primary outcome is described as a number of TEAEs. General statistical context: event counts, counts of participants with at least one event, and rates over time answer different questions. Repeated events in one participant can make the event count exceed the number of participants affected. A rate also requires a defined time-at-risk denominator. The posted serious-event figures include at-risk and affected counts, but the registry record details do not give a complete numerical TEAE result or a detailed statistical method for the primary outcome. It would not be appropriate to substitute the serious-event counts for that missing primary result.
The posted serious-event terms also include percentages, but their denominator and calculation basis are not reported in the registry record. Without that information, the percentages should not be interpreted as incidence estimates with a known population denominator. Comparisons across listed terms would also require consistent definitions and denominators.
What do the planning assumptions mean?
The record describes sample-size planning illustrations as based on an observed effect, with a two-sided significance level of 0.05, equal allocation between two arms and a normal approximation. It states that the effect to detect is a stated fraction of the observed effect, and that time-to-event illustrations use the total number of events while other illustrations use participants per arm. These are planning assumptions, not evidence that the study was designed with those assumptions. No numerical planning results or observed-effect estimate are reported in the registry entries.
General statistical context: a planning illustration asks what sample size might be needed under specified assumptions. It is not a measure of the probability that a completed study detected an effect, and it should not be described as observed power. If a planning exercise uses an observed effect, it inherits uncertainty and potential bias in that estimate. In this case, the record does not provide numerical planning outputs with which to evaluate the implications.
The listed assumption of equal allocation between two arms does not match the study’s recorded single-group, non-randomized design as a direct trial comparison. It is therefore important to distinguish that hypothetical planning framework from the actual study structure. The registry record does not report that the study itself used equal allocation or a two-arm randomized design.
What can confidence intervals and significance tests contribute?
General statistical context: a confidence interval describes uncertainty around an estimate under a specified statistical model and procedure. Its width reflects both the observed data and assumptions such as the sampling model. A significance test addresses compatibility between data and a specified null hypothesis; it does not measure clinical importance or establish causation. Neither an interval nor a p-value can be interpreted without knowing the endpoint, analysis population and method.
No confidence intervals, p-values or numerical primary endpoint estimates are reported in the registry entries available for this analysis. The analysis method for TEAEs, pharmacokinetic outcomes or disease-response outcomes is also not reported in the registry record. No model, covariate adjustment or handling of repeated observations should be inferred.
Why does over-dispersion matter?
General statistical context: count data can vary more than a simple model assumes, a feature often called over-dispersion. If it is present and ignored, standard errors and intervals may be misleading. Assessing it requires suitable outcome data and a defined model. The registry record details do not describe a count model or an over-dispersion assessment, so neither should be attributed to the study.
Overall, the available information supports a design-based reading and a descriptive account of the posted safety figures. It does not support reconstructing unreported analyses, numerical endpoint results or the statistical plan.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists 32 outcome measures and 72 analyses. It also names multiple secondary outcomes, including response categories at assessment visits, best overall response, clinical benefit rate, 6-month progression-free survival rate and median time to tumor progression. The study objectives include several biomarker and circulating tumor cell assessments. Taken together, these outcomes represent multiple opportunities to examine different endpoints, time points or analyses.
General statistical context: when many hypotheses are tested, the chance of at least one apparently unusual result can increase unless the analysis plan addresses multiplicity. Approaches can include prespecifying a primary comparison, adjusting inference across tests or clearly labeling secondary and exploratory analyses. The registry record does not report a multiplicity adjustment, a hierarchy among the secondary outcomes or a prespecified strategy for interpreting the 72 analyses. No such method should be assumed.
The registry record defines the primary safety population as all enrolled patients who took at least one dose of reparixin. It does not report, in the registry record, the number analyzed for every outcome or whether the analysis populations differed across endpoints. Enrollment was 33, but the registry record’s group-specific at-risk counts should not be used to infer an analysis population for other outcomes.
Missing-data handling is not reported in the registry record. The record does not describe how missing assessments, withdrawals, incomplete follow-up or unavailable biomarker measurements were treated. It also does not report whether any sensitivity analyses were used. These omissions matter because missing information can affect estimates, especially when outcomes are collected over repeated visits or follow-up periods. Without the analysis details, the direction or size of any impact cannot be determined.
These limitations do not show that the analyses were necessarily inappropriate. They mean that the registry entries do not permit assessment of multiplicity control, completeness of analysis populations or robustness to missing data.
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 |
|---|---|---|---|
| Group 1 | 2 | 4 | 50.0% |
| Group 2 | 1 | 3 | 33.3% |
| Group 3 | 4 | 23 | 17.4% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Group 1 | 0 of 4 |
| Group 2 | 0 of 3 |
| Group 3 | 0 of 23 |
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 |
|---|---|
| Group 1 | 4 of 4 |
| Group 2 | 3 of 3 |
| Group 3 | 23 of 23 |
Most frequent serious adverse event terms
| Event term | Organ system | Group 1 | Group 2 | Group 3 |
|---|---|---|---|---|
| Dehydration | Metabolism and nutrition disorders | 2 of 4 | 0 of 3 | 0 of 23 |
| Metastasis to central nervous system | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 1 of 4 | 0 of 3 | 0 of 23 |
| Pneumothorax | Respiratory, thoracic and mediastinal disorders | 0 of 4 | 1 of 3 | 0 of 23 |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | 0 of 4 | 0 of 3 | 1 of 23 |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | 0 of 4 | 0 of 3 | 1 of 23 |
| Intracranial hypotension | Nervous system disorders | 0 of 4 | 0 of 3 | 1 of 23 |
| Abdominal pain | Gastrointestinal disorders | 0 of 4 | 0 of 3 | 1 of 23 |
| Disease progression | General disorders | 0 of 4 | 0 of 3 | 1 of 23 |
| Hyponatraemia | Metabolism and nutrition disorders | 1 of 4 | 0 of 3 | 0 of 23 |
| Oesophageal adenocarcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 0 of 4 | 0 of 3 | 1 of 23 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Group 1 | Group 2 | Group 3 |
|---|---|---|---|---|
| Nausea | Gastrointestinal disorders | 3 of 4 | 3 of 3 | 12 of 23 |
| Constipation | Gastrointestinal disorders | 3 of 4 | 3 of 3 | 10 of 23 |
| Diarrhoea | Gastrointestinal disorders | 2 of 4 | 3 of 3 | 8 of 23 |
| Vomiting | Gastrointestinal disorders | 3 of 4 | 2 of 3 | 6 of 23 |
Serious events with the highest arm-level rate: dehydration (50.0%), pneumothorax (33.3%), metastasis to central nervous system (25.0%), hyponatraemia (25.0%), dyspnoea (4.3%).
The posted serious-event figures list the number at risk and the number affected for each group. Group 1 lists 4 at risk and 2 affected; Group 2 lists 3 at risk and 1 affected; Group 3 lists 23 at risk and 4 affected. The figures list 0 deaths in each group. A separate set of adverse-event figures lists 4 at risk and 4 affected for Group 1, 3 at risk and 3 affected for Group 2, and 23 at risk and 23 affected for Group 3.
These group-level figures are descriptive. The study was non-randomized and did not include a separate control group, so differences between groups do not provide a controlled estimate of the effect of dose. The group sizes and reported figures should not be treated as if participants had been randomly assigned to comparable arms. The registry record does not report a statistical comparison of these safety counts.
Serious-event terms are listed with percentages, including Dehydration at 50.0%, Pneumothorax at 33.3%, Metastasis to central nervous system at 25.0%, Hyponatraemia at 25.0%, Dyspnoea at 4.3% and Hypoxia at 4.3%. The denominator and calculation basis for these percentages are not reported in the registry record. They therefore cannot be read as rates with a known denominator or directly compared as measures of risk.
General statistical context: a count of affected participants is not the same as a count of all events, since an individual may experience multiple events. The posted figures do not establish causality. The registry record also does not provide a complete numerical result for the primary TEAE endpoint in the registry record.
12. What the Result Does, and Does Not, Mean
This study contributes a registry description of a phase I combination regimen and its safety and pharmacokinetic objectives in HER 2 negative metastatic breast cancer. It records 33 enrolled participants and describes three reparixin dose groups given with weekly paclitaxel. The posted safety figures provide group-specific counts for serious events, other events and deaths. These are useful as descriptive information about events recorded in the study.
The record does not report a numerical primary TEAE result or numerical pharmacokinetic findings in the registry record for this analysis. The registry also lists secondary response and time-to-event outcomes, but their numerical results are not reported. The outcome names alone cannot establish what happened to participants, how precisely an estimate was measured or whether outcomes differed from what would have occurred without the combination.
The non-randomized, single-group design does not provide a control-group comparison. The dose groups are not a like-for-like control comparison, and any differences between them could reflect factors beyond reparixin dose. Because paclitaxel was administered in combination, the design information also does not isolate effects attributable to reparixin from effects attributable to paclitaxel or their combination.
General statistical context: an early-phase study can describe observed events and generate information for further investigation, but descriptive observations do not by themselves establish a causal treatment effect. A small or limited set of observations may also leave substantial uncertainty. No confidence intervals or numerical primary outcome estimates are reported in the registry entries, so precision cannot be assessed for those outcomes.
The record does not establish that the combination improves disease outcomes, nor does it establish that it does not. It documents the study’s objectives, design and selected safety counts. Interpretation beyond those details would require outcome results and analysis details that are not reported in the registry record.
13. Why This Trial Matters Statistically
The study is statistically informative as an example of how an early-phase combination study can be structured around safety and pharmacokinetic questions before a comparative design is available. The registry record makes the single-group, non-randomized structure explicit and describes increasing reparixin doses alongside a fixed paclitaxel schedule. That structure matters because it sets the limits on what comparisons can be interpreted.
It also illustrates why safety summaries need clear definitions. The primary outcome is described as a number of treatment-emergent adverse events, while the posted group figures separately distinguish at-risk and affected counts for serious and other events. Those summaries are not interchangeable. The record’s serious-event terms include percentages, but the denominator and calculation basis are not reported, underscoring the importance of transparent denominators when interpreting safety data.
The listed 32 outcome measures and 72 analyses make multiplicity relevant to interpretation. General statistical context: when many outcomes and analyses are considered, readers need to know which questions were primary, how secondary or exploratory findings were treated and whether any adjustment was planned. The registry record does not report those details.
The study also highlights the distinction between a planning illustration and a trial’s actual design. The stated planning assumptions describe hypothetical calculations, including equal allocation between two arms, while the actual study is recorded as single-group and non-randomized. A planning scenario should not be mistaken for a feature of the enrolled trial or for evidence of the study’s statistical power.
Finally, the absence of numerical primary endpoint, pharmacokinetic and secondary outcome results in the registry record constrains the interpretation. The study’s statistical importance lies in its design and the questions it set out to examine; the available record details do not permit a quantitative account of those unreported results.
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.
Several limitations specific to this study affect interpretation. First, the registry describes a non-randomized, single-group study with no masking and no separate control group. Dose groups therefore do not provide a randomized comparison, and observed differences among groups cannot be attributed to dose alone.
Second, this was a combination regimen. Paclitaxel and reparixin were administered together, so the design description does not isolate the contribution of either drug. The registry record does not report a method for separating their effects.
Third, the enrollment was 33, while group-specific safety counts are reported separately. The registry record does not explain in the registry record how participants were assigned among the groups, beyond the dose descriptions and stated toxicity conditions. It does not report the detailed escalation algorithm, stopping rules or decision process.
Fourth, the numerical result for the primary TEAE outcome is not reported in the registry entries available for this analysis. Numerical pharmacokinetic and secondary outcome results are also not reported. Although serious-event counts and terms are available, the denominator and calculation basis for the listed percentages are not reported.
Finally, the registry record does not report the detailed statistical analysis plan, missing-data methods, multiplicity strategy, interim-analysis details or sample-size justification. The planning assumptions described in the record are not a substitute for those study-specific methods. These gaps prevent a full assessment of precision, robustness and the handling of multiple outcomes; they do not establish what the unreported analyses would have shown.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Pilot Study to Evaluate Reparixin With Weekly Paclitaxel in Patients With HER 2 Negative Metastatic Breast Cancer (MBC) — NCT02001974.
- Registry record last updated 2021-09-27; status last verified 2021-09.
- Results first posted 2021-04-19.
- 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
The registry record describes NCT02001974, a completed phase I pilot study of reparixin with weekly paclitaxel in HER 2 negative metastatic breast cancer. It lists enrollment of 33, non-randomized allocation, no masking and a single-group model. The recorded start date was 2012-02-27, with primary completion on 2014-06-25.
The primary objective was safety and pharmacokinetics. The primary outcome was treatment-emergent adverse events in a safety population defined by receipt of at least one reparixin dose. The regimen descriptions include reparixin doses of 400 mg, 800 mg and 1200 mg three times daily with paclitaxel 80 mg/m2 on Days 1, 8, and 15 of a 28-day cycle.
Group-specific serious-event and other-event counts are reported, with 0 deaths listed in each group. Numerical primary TEAE, pharmacokinetic and secondary outcome results are not reported in the registry entries. The non-randomized single-group design does not support a controlled comparison.