This page provides an independent statistical analysis and educational interpretation of publicly reported results. ClinicalTrials.gov provides the official trial registry record. Numerical trial results on this page are restricted to the results posted in the ClinicalTrials.gov record; calculations added by Clinical Biostats are labelled as such.
1. Trial at a Glance
This page covers the registry results for Study to Investigate the Pharmacokinetics (PK) and Pharmacodynamics (PD) of Idarucizumab in Chinese Healthy Male and Female Volunteers Who Had Taken Dabigatran Etexilate and Whose Plasma Concentrations of Dabigatran Were at or Close to Steady State (NCT03086356), a na phase 1 study. The primary outcome is Maximum Measured Concentration of Idarucizumab in Plasma (Cmax).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT03086356 |
| Phase | Phase 1 |
| Condition | Healthy Volunteers |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 12 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Boehringer Ingelheim |
| Results posted | Yes |
2. Clinical Question
The trial was designed to investigate the pharmacokinetics and pharmacodynamics of idarucizumab in healthy Chinese male and female volunteers whose dabigatran concentrations were at or close to steady state. It also aimed to explore how idarucizumab affected pharmacokinetic and pharmacodynamic parameters of dabigatran. These objectives describe what the study set out to investigate; the registry record does not report numerical results for those questions.
Population
Healthy Chinese male and female volunteers who had taken dabigatran etexilate. The record lists an enrollment of 12 and describes the pharmacokinetic population as treated subjects with at least one pharmacokinetic parameter analysed.
Intervention
Idarucizumab administered intravenously during dabigatran treatment. The brief summary describes administration of idarucizumab followed by another idarucizumab administration with a 15-minute interval when dabigatran was at or close to steady state.
Comparator
There was no randomized control group. The record describes a single-group study in which participants took dabigatran etexilate alone during specified study days and with idarucizumab on day 11.
Primary question
What was the maximum measured concentration of idarucizumab in plasma, expressed as Cmax, and how variable was that measure in the pharmacokinetic set? Numerical estimates are not reported in the registry record.
This is a pharmacology-focused question rather than a comparison of clinical outcomes between treatments. The primary endpoint describes drug concentration in plasma, while the stated secondary objectives address dabigatran amounts excreted in urine and dabigatran concentrations in plasma. The registry record does not give numerical estimates for those measures or report whether observed changes met a prespecified criterion.
3. Trial Design
This was a completed, open-label Phase I trial with a single-group design. The registry record lists an enrollment of 12 and describes the participants as healthy Chinese male and female volunteers. Allocation is listed as not applicable, and masking is listed as none. Accordingly, the record does not describe random assignment to treatment groups or a blinded comparison.
The regimen was organized around dabigatran etexilate exposure. The arm description states that participants received dabigatran etexilate alone on days 1–4 and days 8–10, and with idarucizumab on day 11. The brief summary describes intravenous idarucizumab administration, including a further idarucizumab administration after a 15-minute interval, at or close to dabigatran steady state. Details such as the doses and full administration schedule are not reported in the registry record.
The primary outcome was maximum measured idarucizumab concentration in plasma, Cmax, in nanomoles (nmol) per litre (L). The listed observation times run from −0.017 to 72.083 hours and include multiple sampling times. The endpoint is characterized by a geometric mean and geometric coefficient of variation. The record identifies the pharmacokinetic set as the analysis population for this outcome: it included treated subjects who had at least one pharmacokinetic parameter analysed.
The record lists six outcome measures and no analysis entries. It does not report a randomized comparison, a control arm, or numerical outcome estimates. The dates and status identify the study as completed, but completion alone does not supply the missing endpoint values or explain how the results were analysed.
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: Healthy male and female volunteers, aged ≥18 and ≤45 years, body weight ≥50 kilograms (kg) with a Body Mass Index (BMI) in the range from ≥19 to \<24 kilogram per square meter (kg/m2) were included in the trial.
Overall Study
| Stage | Dabigatran Etexilate+Idarucizumab |
|---|---|
| STARTED | 12 |
| COMPLETED | 12 |
| NOT COMPLETED | 0 |
Analysis population for the primary outcome, as defined in the registry: Pharmacokinetic set (PKS): The PKS included all subjects of the Treated set (TS) who had at least one Pharmacokinetic (PK) parameter analysed.
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: Treated set (TS): The TS included all subjects who received at least 1 infusion of idarucizumab.
| Characteristic | Unit | Dabigatran Etexilate+Idarucizumab |
|---|---|---|
| Participants analysed | 12 | |
| Age, Continuous | years | 35.6 (SD 5.8) |
| Sex: Female, Male: Female | Participants | 6 |
| Sex: Female, Male: Male | Participants | 6 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 12 |
| 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 |
6. Primary Endpoint
The registry lists the primary outcome as Maximum Measured Concentration of Idarucizumab in Plasma (Cmax), measured over -0.017, 0.083, 0.167, 0.317, 0.417, 0.45, 0.583, 0.917, 1.417, 2.083, 3.083, 4.083, 6.083, 10.083, 12.083, 24.083, 48.083, 72.083 hours (h).
| Element | Registry entry |
|---|---|
| Result type | Geometric Mean |
| Measure of spread | Geometric Coefficient of Variation |
| Unit | nanomoles (nmol) per litre (L) |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is the maximum measured concentration of idarucizumab in plasma, designated Cmax. It is a pharmacokinetic measure: it describes the highest concentration recorded across the specified blood-sampling times, rather than a clinical outcome or a direct measure of how a participant feels or functions. The unit is nanomoles (nmol) per litre (L).
Measuring a maximum concentration helps characterize the concentration profile after drug administration. In general, pharmacokinetic concentration measures can help describe exposure and support comparisons across dosing schedules or populations when comparable data and suitable analyses are available. In this trial, the endpoint is framed as a geometric mean, with geometric coefficient of variation describing dispersion. General statistical interpretation: a geometric mean is often used for positive measurements that are right-skewed or that vary multiplicatively; the geometric coefficient of variation expresses variability on a relative, multiplicative scale.
The registry record lists sampling times from −0.017 through 72.083 hours, but it does not report the resulting Cmax estimate, its geometric coefficient of variation, a confidence interval, or a p-value. Thus, the endpoint definition and measurement schedule are available, while the numerical findings and their precision are not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record establishes the study question and design but does not provide a numerical result for the primary endpoint. It names maximum measured plasma concentration of idarucizumab as the primary outcome, specifies a geometric mean and geometric coefficient of variation, and identifies the pharmacokinetic set. Without the observed estimate and its dispersion, the magnitude and precision of the primary pharmacokinetic finding cannot be assessed from this record.
The stated study objectives also included pharmacodynamic investigation and exploration of idarucizumab’s effect on dabigatran pharmacokinetic and pharmacodynamic parameters. The record lists secondary measures involving the amount of sum dabigatran excreted in urine over the specified interval on day 4 and day 11, and the area under the concentration-time curve for unbound sum dabigatran over the stated interval. Numerical estimates for these outcomes are not reported in the registry record. Their inclusion as outcomes does not establish the direction or size of any observed change.
The single-group structure matters when interpreting the questions. Dabigatran exposure was described before and during idarucizumab administration, but there was no randomized control group. An observed within-participant change, if reported, would not by itself separate the effect of idarucizumab from time-related changes, measurement variation, or other influences. No causal comparison should be inferred from the design description alone.
The registry lists 12 enrolled participants. This is the enrollment figure, not a reported endpoint-specific denominator: the pharmacokinetic set has its own eligibility description, and the number analysed for Cmax is not stated. The record also does not supply individual observations, confidence intervals, or a detailed statistical analysis plan. Precision and robustness therefore cannot be judged from the available information.
Safety information is also limited. The adverse-event record lists 12 participants at risk for other adverse events and 12 affected, while it lists 12 at risk for serious adverse events and 0 affected. These are registry counts, not a substitute for detailed event descriptions, severity, timing, or an assessment of causality. No serious adverse event terms are listed among the top serious terms.
Overall, the available record supports a description of the trial’s setting, regimen, and intended measurements. It does not support a quantitative conclusion about idarucizumab concentrations, pharmacodynamic response, dabigatran exposure, or the precision of any estimated effect. The absence of posted numerical results should not be read as evidence that an effect was absent or that the endpoint was not measured.
9. Statistical Methods Explained
The registry identifies the primary parameter as a geometric mean and its dispersion as a geometric coefficient of variation. It does not report the numerical outcome estimates, confidence intervals, or analysis details. The explanations below describe the general statistical ideas relevant to those choices; they are not a claim about additional methods used in this trial.
What does Cmax measure?
Cmax is the maximum measured concentration of a drug in plasma across the scheduled sampling observations. It is a summary of the observed concentration profile, not necessarily the exact biological peak between samples. The sampling schedule therefore matters: a peak occurring between measurements may not be captured as an observed maximum. The record lists multiple time points, from −0.017 to 72.083 hours, but does not provide the concentration observations or explain how the maximum was selected when measurements were missing or unusable.
As a pharmacokinetic endpoint, Cmax describes concentration rather than a clinical benefit or harm. Its interpretation depends on the treatment schedule, the population, the assay and measurement procedures, and the timing of samples. The registry record gives the unit and sampling times but does not report assay details or a numerical concentration estimate.
Why use a geometric mean?
General statistical explanation: concentrations are positive values and may be unevenly distributed, with some observations much larger than others. For positive data that vary multiplicatively, analysis on a logarithmic scale can be useful. A geometric mean summarizes the central tendency by averaging on that transformed scale and converting the summary back to the original scale. It is not the same as the arithmetic average.
The registry specifies a geometric mean for Cmax, but it does not state the transformation or calculation procedure used, nor provide the estimate. The choice of summary alone cannot establish whether the data followed a particular distribution. The geometric coefficient of variation is a measure of relative spread appropriate to this multiplicative framing; a numerical value is not reported.
What does a confidence interval add?
General statistical explanation: a confidence interval describes uncertainty around an estimated quantity under the assumptions of the statistical procedure. A narrow interval indicates greater precision than a wide interval for the same type of estimate. It does not show that the estimate is free of bias, and it does not replace examination of the study design and data quality. No confidence interval for the primary endpoint is reported in the registry record, so the precision of the estimate cannot be evaluated from the posted information.
How does the single-group design shape interpretation?
The participants received dabigatran etexilate alone during specified days and with idarucizumab on day 11. This arrangement can support characterization of pharmacokinetic measurements over the study schedule. It is not a randomized comparison between separate treatment groups. General statistical explanation: randomization can help balance measured and unmeasured factors between groups, while a within-participant sequence can reduce some between-person variation. A sequential design can still be affected by time, order, carryover, or changes in measurement conditions. The registry record does not describe a statistical method for addressing these issues, so no such adjustment should be assumed.
The study is open-label, with masking listed as none. For an objective concentration assay, knowledge of treatment may be less directly influential on the measured laboratory value than on a subjective assessment, but the record does not provide enough information to evaluate possible sources of measurement or analysis bias. This general distinction does not establish that bias occurred or that it was absent.
What can be inferred from the listed analysis population?
The primary outcome population is described as the pharmacokinetic set. It includes treated subjects who had at least one pharmacokinetic parameter analysed. This definition indicates that enrollment and inclusion in an endpoint analysis are not necessarily identical. The registry does not report how many participants contributed to Cmax, whether any were excluded, or how missing concentration measurements were handled.
General statistical explanation: analysis-population definitions affect the interpretation of an estimate because they determine which observations contribute. Excluding participants or measurements can introduce bias if the reasons for missingness are related to treatment or outcome. The appropriate assessment depends on the missing-data pattern and analysis plan, neither of which is described in the registry record.
What does the record say about statistical testing?
The record lists no analysis entries. It does not report a hypothesis test for the primary endpoint, a prespecified effect threshold, a confidence interval, or a model. The study objectives are investigative, but that label does not reveal the statistical procedures used. Statistical significance should not be inferred from a geometric mean or from the fact that an endpoint was designated primary.
The registry also contains generic planning assumptions for illustrations based on an observed effect. Such illustrations are not observations from this trial, do not establish the trial’s original design rationale, and are not observed power. No calculated planning figures are reported in the record details considered for this interpretation. Sample-size planning in general depends on assumptions about the target effect, variability, significance level, allocation, and analysis; these are distinct from the data observed in a completed study.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists six outcome measures, including the primary Cmax measure and secondary measures concerning dabigatran in urine and plasma. It does not provide a multiplicity procedure, such as an adjustment to control the chance of false-positive findings across several statistical tests. It also lists no analysis entries. Therefore, the record does not establish which outcomes were formally tested, whether any were considered confirmatory, or how multiple comparisons were handled.
General statistical explanation: when several outcomes or comparisons are tested, the chance of at least one apparently unusual result can increase if each test is considered separately. Prespecified outcome hierarchies or multiplicity adjustments can address this issue, but their use cannot be assumed. Because no numerical results or analysis details are reported, there is no basis for assessing whether a particular finding would remain persuasive after accounting for multiple testing.
Missing-data methods are not described in the registry record. The pharmacokinetic set includes treated participants with at least one pharmacokinetic parameter analysed, but the number contributing to each outcome and the handling of missing or unusable measurements are not stated. The record does not report whether values below assay limits occurred, how partial concentration profiles were treated, or whether any imputation was used. These are unknowns, not evidence that data were missing or that a particular method was applied.
The record identifies a population for the primary endpoint but does not give a complete analysis plan, participant flow, or outcome-specific denominators. As a result, reproducibility and the possible influence of missing measurements cannot be assessed. The registry record also does not report interim analyses or a sample-size justification. Neither should be inferred from the Phase I designation or the enrollment figure.
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 |
|---|---|---|---|
| Dabigatran Etexilate+Idarucizumab | 0 | 12 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Dabigatran Etexilate+Idarucizumab | 0 of 12 |
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 |
|---|---|
| Dabigatran Etexilate+Idarucizumab | 12 of 12 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Dabigatran Etexilate+Idarucizumab |
|---|---|---|
| Albumin urine present | Investigations | 12 of 12 |
| Alpha 1 microglobulin increased | Investigations | 12 of 12 |
| Protein urine present | Investigations | 11 of 12 |
| Activated partial thromboplastin time prolonged | Investigations | 10 of 12 |
| Urine electrophoresis abnormal | Investigations | 10 of 12 |
| Prothrombin level decreased | Investigations | 4 of 12 |
| Haematoma | Vascular disorders | 1 of 12 |
| Diarrhoea | Gastrointestinal disorders | 1 of 12 |
| Blood triglycerides increased | Investigations | 1 of 12 |
The adverse-event record lists the Dabigatran Etexilate+Idarucizumab group with 12 participants at risk for serious adverse events and 0 affected. For other adverse events, it lists 12 participants at risk and 12 affected. No top serious adverse-event terms are listed.
These entries are counts recorded for the study group. They do not describe the event types, severity, timing, duration, or whether events were considered related to a study drug. The record does not provide a fuller narrative or participant-level information, so the counts cannot answer those questions. In particular, a count of participants affected does not state how many individual events occurred, because a participant may experience more than one event.
General statistical explanation: safety counts are descriptive and depend on the group at risk, the period of observation, the definitions used, and how events were collected and coded. They should be interpreted within the study’s size and design, rather than treated as a controlled comparison. There was no randomized comparator group in this single-group study, so these figures do not estimate a relative safety difference between treatment strategies. The registry counts alone are not a complete account of the safety profile.
12. What the Result Does, and Does Not, Mean
This trial’s stated purpose was to investigate idarucizumab pharmacokinetics and pharmacodynamics in healthy Chinese volunteers taking dabigatran etexilate, and to explore the effect of idarucizumab on dabigatran parameters. Its primary endpoint was maximum measured idarucizumab plasma concentration. The record provides the endpoint definition, units, sampling schedule, and a description of the analysis population.
What the record does not provide is the numerical Cmax estimate, the geometric coefficient of variation, or a confidence interval. It also does not provide numerical results for the listed secondary measures. Consequently, the size and precision of the measured concentrations, and the extent of any change in dabigatran measures, cannot be determined from the registry record. Missing numerical results should not be interpreted as a null result.
The study’s single-group, open-label design is appropriate to describe as a pharmacology investigation, but it does not furnish a randomized comparison with a control group. Any within-study pattern would need to be interpreted in light of the sequence of dabigatran exposure and idarucizumab administration, as well as possible time-related and measurement influences. The registry does not provide an analysis that separates those influences.
The findings, if available elsewhere, would concern the enrolled healthy-volunteer setting. The record does not establish that the same measurements or patterns apply to people with a medical condition, different characteristics, or a different treatment context. Nor does a pharmacokinetic endpoint by itself establish a clinical outcome. The trial’s stated objective regarding reversal of anticoagulant activity is not, on its own, a reported result.
The adverse-event counts describe recorded participants affected and at risk, but limited details are available. They do not establish comparative safety or provide enough information to characterize specific events. Overall, the registry record supports understanding what was studied and how the primary endpoint was defined, but not a conclusion about the magnitude, precision, or clinical implications of the results.
13. Why This Trial Matters Statistically
This study illustrates the role of early-phase pharmacology research in characterizing drug concentrations and pharmacodynamic measures under a defined exposure sequence. The primary endpoint, Cmax, focuses on the highest measured idarucizumab plasma concentration, while the secondary objectives address dabigatran excretion and plasma exposure. These measures ask how drug levels behave in the study setting, rather than directly comparing clinical outcomes across treatment groups.
Statistically, the record highlights the difference between an endpoint definition and an endpoint result. It identifies a geometric mean and geometric coefficient of variation, but without their values or an uncertainty interval, readers cannot judge either the observed concentration level or its precision. Reporting the estimator, variability, denominator, and uncertainty is important for interpreting pharmacokinetic results and for understanding how much the data support conclusions.
The design also illustrates why study structure must be considered alongside endpoint summaries. A single-group sequence can describe measurements during treatment, but it lacks a randomized control group for separating treatment-related changes from other changes over time. That limitation does not make the measurements uninformative; it defines the kinds of questions they can answer and the strength of any causal interpretation.
Finally, the record distinguishes enrollment from the defined pharmacokinetic analysis population. A participant must be treated and have at least one pharmacokinetic parameter analysed to meet that population description, while the outcome-specific denominator is not reported. Transparent reporting of participant flow, missing measurements, analysis procedures, and numerical results would allow readers to assess representativeness and precision more fully. These statistical considerations matter because they determine whether a result is interpretable, not merely whether a study has a completed status.
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 most important limitation of interpretation is the absence of numerical outcome results in the registry record. Although the primary endpoint and its summary statistics are named, the Cmax estimate and geometric coefficient of variation are not reported. Numerical results for the secondary measures are also not reported. This prevents assessment of effect magnitude, variability, and precision.
The study has a single-group, open-label design, with no randomized control group. The sequence described in the record allows pharmacokinetic measurements during dabigatran treatment and after idarucizumab administration, but it does not provide a concurrent randomized comparison. Potential time, order, or measurement influences cannot be assessed from the available description.
The record lists 12 enrolled participants, but does not state the number contributing to each outcome. The pharmacokinetic set definition requires treatment and at least one pharmacokinetic parameter analysed; it does not give the Cmax-specific denominator. Missing-data patterns, exclusions, and procedures for incomplete profiles are not reported.
Other methodological details are also unavailable, including doses, assay procedures, a detailed statistical analysis plan, confidence intervals, hypothesis tests, and any approach to multiple outcomes. The record does not report a sample-size justification or interim analyses. Their absence from the registry record does not establish that these activities did or did not occur.
Finally, the study population consisted of healthy Chinese male and female volunteers. Results from that setting, if available, would not automatically generalize to other populations or clinical settings. The registry entry describes the intended investigation, but it does not provide enough outcome information to evaluate the size or clinical relevance of any observed pharmacological response.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Study to Investigate the Pharmacokinetics (PK) and Pharmacodynamics (PD) of Idarucizumab in Chinese Healthy Male and Female Volunteers Who Had Taken Dabigatran Etexilate and Whose Plasma Concentrations of Dabigatran Were at or Close to Steady State — NCT03086356.
- Registry record last updated 2019-03-08; status last verified 2019-03.
- Results first posted 2019-03-08.
- 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
NCT03086356 is a completed, open-label Phase I, single-group study sponsored by Boehringer Ingelheim. It enrolled 12 healthy Chinese male and female volunteers and investigated idarucizumab pharmacokinetics and pharmacodynamics during dabigatran etexilate treatment. The primary outcome was maximum measured idarucizumab concentration in plasma, summarized as a geometric mean with geometric coefficient of variation.
The record describes dabigatran etexilate alone on days 1–4 and days 8–10, and with idarucizumab on day 11. It lists six outcome measures and no analysis entries. The primary endpoint’s numerical estimate, its variability, and confidence intervals are not reported in the registry record. The adverse-event entry lists 0 affected by serious adverse events and 12 affected by other adverse events, with 12 at risk in each category.