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 Open Label Study To Evaluate The Long-Term Safety Profiles Of Caduet In Japanese Patients (NCT01190007), a non-randomized phase 4 study. The primary outcome is Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01190007 |
| Phase | Phase 4 |
| Condition | Hypertension, Hypercholesterolemia, Angina Pectoris |
| Allocation | Non-Randomized |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 159 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Pfizer's Upjohn has merged with Mylan to form Viatris Inc. |
| Results posted | Yes |
2. Clinical Question
The central question was whether adverse events occurred during treatment with Caduet in Japanese patients who had hypercholesterolemia together with either hypertension or angina pectoris. The registry describes a safety study, not a randomized comparison of treatments. Its primary outcome counted participants with treatment-emergent adverse events and serious adverse events during 52 weeks.
Population
Japanese patients with both hypertension and hypercholesterolemia, or with both angina pectoris and hypercholesterolemia. The safety analysis set included participants who took at least one dose of study drug. The registry reports 159 participants in the Caduet group.
Intervention
Caduet, with the listed amlodipine/atorvastatin dose combinations: 2.5 mg/5 mg, 2.5 mg/10 mg, 5 mg/5 mg or 5 mg/10 mg. The treatment period for the primary safety objective was 52 weeks.
Comparator
There was no separate control group. The study was non-randomized, open label and single group, so the record does not support a like-for-like comparison between Caduet and another treatment or control condition.
Primary question
How many participants experienced treatment-emergent adverse events or serious adverse events? Treatment-emergent events were defined as newly occurring or worsening after the first dose. Investigators assessed whether events were related to study drug.
This framing matters when interpreting the findings. A count of participants with an event describes what was recorded among those treated; by itself it does not estimate what would have happened to similar patients without Caduet. The secondary outcomes concern changes in blood pressure and blood lipid measures, but the registry record excerpt does not include their numerical results. The primary safety question can therefore be discussed from the posted adverse-event counts, while conclusions about changes in blood pressure or lipids cannot be drawn from the outcome titles alone.
3. Trial Design
This was a Phase 4, multi-center, open-label study with non-randomized allocation and a single-group model. The registry identifies 159 participants in the Caduet group and lists the study as completed. Its primary objective was to investigate Caduet safety during a 52-week treatment period in Japanese patients with both hypertension and hypercholesterolemia, or both angina pectoris and hypercholesterolemia.
Caduet combines amlodipine and atorvastatin. The listed dose combinations were 2.5 mg/5 mg, 2.5 mg/10 mg, 5 mg/5 mg and 5 mg/10 mg, expressed as amlodipine/atorvastatin. The registry lists one drug intervention and does not provide further arm-specific description. Details of how doses were selected or adjusted are not reported in the registry record.
Because the study was open label, participants and investigators were not masked to treatment, according to the registry’s masking classification. This is relevant to the ascertainment and classification of adverse events: investigators assessed whether an event was related to the study drug, and the record does not describe blinded adjudication. The design can describe events observed during treatment, but without a randomized control group it cannot separate treatment-associated risk from background events, underlying conditions, or other influences.
The registry lists a start date of 2010-08 and primary completion in 2012-02. The date of final study completion, the number who completed treatment, and participant follow-up beyond the primary treatment period are not reported in the registry record. The enrollment number describes the study enrollment, while the primary outcome population is defined as participants who took at least one dose. The record does not provide additional details about exclusions from the safety analysis set or the number of participants in each underlying condition group.
Secondary outcomes included changes from baseline in trough systolic and diastolic blood pressure for the two condition groups, and percent changes from baseline in LDL-C, total cholesterol, HDL-C and triglycerides. These outcomes indicate that the study also collected cardiovascular risk-factor measures. Their listed presence does not, by itself, provide evidence about the direction or size of any change. Numerical secondary-outcome results are not reported in the registry record considered 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.
Overall Study
| Stage | Caduet |
|---|---|
| STARTED | 159 |
| COMPLETED | 145 |
| NOT COMPLETED | 14 |
Reasons for not completing this period:
| Reason | Caduet |
|---|---|
| Withdrawal by Subject | 4 |
| Adverse Event | 8 |
| Difficulty of visit for complications | 1 |
| Participant was arrested | 1 |
Analysis population for the primary outcome, as defined in the registry: The safety analysis set included all participants who took at least one dose of study drug.
5. Baseline Characteristics
The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.
| Characteristic | Unit | Caduet |
|---|---|---|
| Participants analysed | 159 | |
| Age, Continuous | years | 65.0 (SD 9.5) |
| Sex: Female, Male: Female | Participants | 108 |
| Sex: Female, Male: Male | Participants | 51 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs), measured over 52 weeks.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint counts participants with adverse events and serious adverse events during the 52-week treatment period. The registry defines treatment-emergent adverse events as events that were newly occurring or worsened after the first dose. Investigators assessed relatedness to study drug as yes or no. When a participant had multiple occurrences of an adverse event within a category, that participant was counted once in that category.
Participant counts are useful in safety reporting because they describe how many people experienced at least one event in a category, rather than how many separate episodes occurred. This reduces the influence of repeated events in the same person on the participant-level count. It also means that the count does not convey the total number of event episodes, how long events lasted, or their full clinical course.
Serious adverse events are a distinct safety category and should be read separately from other adverse events. A participant may have more than one type of event, and the record does not establish that the categories are mutually exclusive. The counts therefore should not be added to produce a total number of affected participants. The registry’s safety analysis set included all participants who took at least one dose of study drug; the reported group denominator is 159.
This endpoint is suited to describing observed safety events during treatment. It does not alone establish that Caduet caused an event. Nor does it quantify comparative risk, since there was no control group. The registry record does not report a primary endpoint estimate adjusted for other factors, an uncertainty interval, or a formal hypothesis test.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The posted safety counts describe events recorded among participants in the Caduet group over the study’s 52-week treatment period. The registry reports 11 participants with serious adverse events among 159 at risk, and 101 participants with other adverse events among 159 at risk. These are participant counts, not counts of every event episode. A participant with multiple occurrences within a category was counted once in that category.
The serious adverse event terms listed include atrial fibrillation, atrioventricular block complete, colitis ischaemic, colonic polyp, cholecystitis acute and humerus fracture. Each is listed with 0.6%. These figures describe the registry’s reported terms; they do not give the duration, severity beyond the serious classification, treatment course, or participant-level context for each event. The record does not provide a full narrative for these cases in the information.
These figures are descriptive. In a single-group study, the observed counts cannot be compared with an untreated or alternative-treatment group within the trial. They therefore do not show how many events would have occurred in the absence of Caduet or whether the observed counts are higher or lower than a comparable background rate. The registry does not report a causal estimate or a comparison adjusted for patient characteristics.
The 11 participants with serious adverse events and the 101 participants with other adverse events should not be treated as disjoint groups unless the reporting definition establishes that. The registry definition explains counting within each category, but it does not state that a participant could appear in only one category. Adding those counts would risk double-counting. The listed serious-event percentages also should not be used to infer exact event counts beyond what the record explicitly reports.
Precision is difficult to assess because no confidence intervals or other uncertainty estimates for the event counts are reported in the registry record. In general, a confidence interval communicates the range of values reasonably compatible with data under specified assumptions. Without one, the posted counts remain point descriptions of this study’s participants, rather than precise estimates of event frequencies in a wider population.
The record also does not report subgroup-specific safety counts for patients with hypertension and hypercholesterolemia versus those with angina pectoris and hypercholesterolemia. It is therefore not possible to determine from these figures whether the event experience differed between those clinical groups or across the listed dose combinations. The registry includes secondary blood pressure and lipid outcome titles but no numerical results in the information. The safety counts should not be used to infer those outcomes.
Overall, the results document adverse events recorded during exposure in this study population. They provide a descriptive safety account, not a controlled estimate of treatment effect or a basis for medical advice. The limitations of the design and the absence of reported uncertainty measures are important when considering how precisely the counts characterize safety beyond the study participants.
9. Statistical Methods Explained
The registry’s primary outcome is a participant-level count. It identifies people who experienced an event, rather than assigning each event episode its own count. This distinction matters because repeated episodes in one participant do not increase that participant’s count within the same category. The outcome is therefore best read as the number of participants with at least one recorded event in a category during the stated observation period.
What does a participant count convey?
A count has a direct interpretation: it describes the number of people meeting the event definition in the study group. The corresponding at-risk count gives the recorded group denominator. Together, these figures communicate the observed extent of events in the enrolled safety population. A count alone does not express uncertainty, and it is not a comparative treatment effect. It also does not show event timing, duration, recurrence, or the number of individual episodes.
In general, statistical summaries of binary outcomes may report a proportion, an effect estimate comparing groups, or an interval describing uncertainty. Which summary is most useful depends on the design and question. For this study, there is one treatment group, so a comparative effect against a control group cannot be estimated from the trial design. The posted primary figures are counts and denominators. A confidence interval for the event frequency is not reported in the registry record.
How should the event definition be understood?
The registry defines treatment-emergent events as newly occurring or worsening after the first dose. The timing rule identifies events that arose or changed after treatment began, but temporal sequence alone does not establish causation. Investigators assessed relatedness to study drug as yes or no. That assessment is part of the recorded safety evaluation; because the study was open label and the record does not describe blinded adjudication, the possibility of judgment or ascertainment effects should be kept in mind.
Serious adverse events and other adverse events are separate reported categories. The record specifies that a participant with multiple occurrences within an event category was counted once within that category. It does not say the categories are mutually exclusive. Consequently, counts across categories should not be summed as though each person could appear only once in the overall safety record.
What does the design allow statistically?
A single-group, non-randomized study can describe observed events among participants who received the intervention. Randomization is commonly used to balance measured and unmeasured factors across groups, while a concurrent comparator helps distinguish outcomes under treatment from outcomes under another condition. Neither is part of the design described in this registry. Differences between the observed event burden and an external or expected rate cannot be attributed to treatment without additional assumptions and suitable comparative data.
Open-label status also matters for outcomes that involve reporting or investigator classification. In general, knowledge of treatment can affect which symptoms are reported, how events are assessed, or how relatedness is judged. The registry does not state whether any independent review process was used. That detail is not reported in the registry record.
What analysis details are missing?
The registry record does not report a statistical analysis plan, a sample-size justification, an interim analysis plan, or the methods used for any formal statistical testing. It also does not provide confidence intervals or p-values for the primary safety counts. The sample-size rationale and whether the enrollment was intended to provide a particular precision for safety outcomes are not reported in the registry record. Without those details, the counts should be treated as descriptive results rather than as the outcome of a stated confirmatory test.
General statistical practice distinguishes a design’s planned ability to detect an effect from the effect actually estimated after a study. A planning calculation is not an observed result and does not make a non-randomized, single-group study comparative. The record’s reported primary results do not include an effect estimate against a control condition. No conclusion about statistical power or precision can be drawn from the enrollment count alone.
For event data, over-dispersion is a general concern when event episodes are analyzed as counts, because some participants may experience repeated events and the variability can exceed assumptions of simple count models. This primary endpoint instead reports participants counted once within each category, so it is not a count of all repeated episodes. That does not remove other limits: the record does not report event-level timing or a model for recurrent events.
Missing-data methods also affect interpretation when participants discontinue or have incomplete follow-up. The registry defines the safety analysis set as all participants who took at least one dose, but the number completing the 52-week period, reasons for discontinuation, and approach to incomplete follow-up are not reported in the registry record. It is therefore not possible to assess how much observation time each participant contributed or whether missing follow-up could have influenced the recorded counts.
Overall, the statistical information supports a descriptive reading of adverse events in the treated group. More detailed inference would require definitions, analysis methods and uncertainty estimates not reported in the registry record. The absence of those items does not change the recorded counts, but it limits how far those counts can be generalized or interpreted comparatively.
10. Multiplicity, Missing Data and Analysis Populations
The study lists a primary safety outcome and several secondary outcomes. The secondary outcomes include trough systolic and diastolic blood pressure changes in the two condition groups, and percent changes in LDL-C, total cholesterol, HDL-C and triglycerides. Multiple outcomes create a general multiplicity issue: when many separate hypotheses are tested, the chance of at least one apparently notable result can rise if no adjustment or interpretation strategy is specified. The registry record does not report a multiplicity adjustment or a statistical testing hierarchy.
The registry lists 12 outcome measures, but it reports zero analyses in the information considered for this interpretation. Numerical results for the listed secondary outcomes are not reported in the registry record. The titles alone do not indicate whether any outcome changed, how large a change was, or how uncertain an estimate might be.
Missing-data handling is also not described. Although the safety set is defined as participants who took at least one dose, the record does not report the number completing the treatment period, the completeness of follow-up, reasons for missing measurements, or methods for handling missing values. These omissions matter particularly for repeated measurements planned at each visit, since incomplete observation could affect which participants contribute to a given visit’s result.
The record does not report how participants were handled in analyses of the blood pressure and lipid outcomes, whether any outcome analyses were adjusted, or whether there were prespecified subgroup comparisons. It also does not report a sample-size justification, interim analyses, or a statistical analysis plan. These details are not reported in the registry record, so the listed outcomes should not be assumed to reflect a prespecified inferential strategy or complete follow-up.
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 |
|---|---|---|---|
| Caduet | 11 | 159 | 6.9% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 2% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Caduet | 101 of 159 |
Most frequent serious adverse event terms
| Event term | Organ system | Caduet |
|---|---|---|
| Atrial fibrillation | Cardiac disorders | 1 of 159 |
| Atrioventricular block complete | Cardiac disorders | 1 of 159 |
| Colitis ischaemic | Gastrointestinal disorders | 1 of 159 |
| Colonic polyp | Gastrointestinal disorders | 1 of 159 |
| Cholecystitis acute | Hepatobiliary disorders | 1 of 159 |
| Humerus fracture | Injury, poisoning and procedural complications | 1 of 159 |
| Thoracic vertebral fracture | Injury, poisoning and procedural complications | 1 of 159 |
| Benign soft tissue neoplasm | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 1 of 159 |
| Breast cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 1 of 159 |
| Carcinoid tumour of the gastrointestinal tract | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 1 of 159 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Caduet |
|---|---|---|
| Nasopharyngitis | Infections and infestations | 53 of 159 |
| Pharyngitis | Infections and infestations | 17 of 159 |
| Bronchitis | Infections and infestations | 11 of 159 |
| Fall | Injury, poisoning and procedural complications | 9 of 159 |
| Eczema | Skin and subcutaneous tissue disorders | 9 of 159 |
| Upper respiratory tract inflammation | Respiratory, thoracic and mediastinal disorders | 8 of 159 |
| Cystitis | Infections and infestations | 6 of 159 |
| Arthralgia | Musculoskeletal and connective tissue disorders | 6 of 159 |
| Back pain | Musculoskeletal and connective tissue disorders | 6 of 159 |
| Headache | Nervous system disorders | 6 of 159 |
Serious events with the highest arm-level rate: atrial fibrillation (0.6%), atrioventricular block complete (0.6%), colitis ischaemic (0.6%), colonic polyp (0.6%), cholecystitis acute (0.6%).
The registry reports 11 participants with serious adverse events among 159 at risk in the Caduet group. It also reports 101 participants with other adverse events among 159 at risk. The safety analysis set included participants who took at least one dose. These counts refer to participants, with repeated occurrences within a category counted once for each participant.
The listed serious adverse event terms are atrial fibrillation, atrioventricular block complete, colitis ischaemic, colonic polyp, cholecystitis acute and humerus fracture. Each term is accompanied by 0.6% in the registry. These entries do not provide event narratives or establish that the study drug caused an event. The record says investigators assessed relatedness, but does not provide the relatedness result for each listed term in the information.
Serious adverse events and other adverse events should be interpreted as reported categories, not added together to obtain a count of unique participants. The registry does not state that participants could appear in only one category. The figures are descriptive of this single treated group; there is no trial control group against which to compare them. Deaths are not reported in the registry record.
12. What the Result Does, and Does Not, Mean
The study’s primary safety results describe adverse events recorded among participants who received Caduet. They show that 11 participants were reported with serious adverse events and 101 with other adverse events, with a listed group size of 159. The serious-event terms and their reported percentages provide additional descriptions of the recorded events, but not a causal assessment or a complete account of each case.
These observations do not establish that Caduet caused the events, nor do they show what the event experience would have been under another treatment or without treatment. The non-randomized, single-group design has no concurrent comparator. Conditions included hypertension, hypercholesterolemia and angina pectoris, and events can arise in the context of patients’ underlying health as well as during treatment. The registry counts do not separate those possible influences.
The numbers also do not establish that events were absent among other participants in every relevant sense: they summarize the categories and definitions recorded in the registry, and the record does not provide full event-level detail. Counts of participants do not equal counts of event episodes, and the record does not report uncertainty intervals for these results. The listed percentages for serious terms should be read as the registry’s reported values rather than treated as a basis for reconstructing unreported details.
The outcomes do not answer whether blood pressure or lipid measures changed. Although those outcomes are listed, their numerical results are not reported in the registry record. Nor can the counts identify differences between the two condition groups or between the listed dose combinations, because subgroup-specific safety results are not reported in the information.
The soundest interpretation is therefore limited: these are descriptive safety observations from a 52-week open-label study in Japanese patients, not a controlled estimate of benefit or harm. They contribute a record of what was reported during treatment, while leaving comparative risk, causation, subgroup differences and the precision of the event frequencies unresolved.
13. Why This Trial Matters Statistically
This trial is statistically informative as an example of the distinction between documenting events and estimating comparative effects. A single-group safety study can record what occurred among participants exposed to a treatment. The resulting counts are useful descriptions of that study population, but they cannot by themselves show how those outcomes differ from what would have happened under another condition.
The primary endpoint is participant-based, and the rule that a person is counted once within a category keeps repeated occurrences from inflating that category’s participant count. That makes the figures relatively straightforward to understand, while also limiting what they say: repeated episodes, duration and event timing are not represented by the participant count. The serious and other event categories should not be combined without confirmation that participants cannot appear in both.
The registry also illustrates why interpretation depends on more than the point counts. Precision measures, complete follow-up information and a statistical analysis plan would help readers assess uncertainty and understand how the outcomes were analyzed. Those details are not reported in the registry record. The absence of those details does not invalidate the recorded counts, but constrains inference from them.
Finally, the study’s secondary outcomes show the breadth of questions that can accompany a safety objective. Blood pressure and lipid measures are listed, but numerical results are not reported in the record. Having an outcome listed is not equivalent to having a result available for interpretation. This distinction is important when assessing what a registry entry can establish: the primary safety counts are available, while conclusions about the listed secondary measures remain unsupported by the reported information.
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 is the absence of a control group. With one non-randomized treatment group, the event counts cannot estimate comparative risk or distinguish treatment effects from background events and other influences. Open-label treatment also means participants and investigators were not masked, and investigator-assessed relatedness may involve judgment. The registry does not report independent blinded event adjudication.
The reported event counts do not include confidence intervals or other uncertainty estimates in the registry record. The record also does not provide a statistical analysis plan, a sample-size justification, or an interim analysis plan. As a result, the intended precision and the prespecified approach to statistical inference are not reported.
Follow-up completeness is unclear. Although the primary treatment period was 52 weeks and the safety set included participants who took at least one dose, the number who completed treatment, reasons for discontinuation, and handling of incomplete observation are not reported in the registry record. This limits assessment of how fully events were ascertained over the planned period.
Further limitations include the lack of reported subgroup counts by underlying condition or dose combination, and the absence of numerical results for the listed blood pressure and lipid outcomes. The record does not provide enough detail to assess how safety varied across those groups or whether the secondary measures changed. Deaths are not reported in the registry record. These limits mean the findings are best treated as descriptive safety information for the study group, rather than broad or comparative estimates.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Open Label Study To Evaluate The Long-Term Safety Profiles Of Caduet In Japanese Patients — NCT01190007.
- Registry record last updated 2021-01-28; status last verified 2021-01.
- Results first posted 2012-12-03.
- 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
NCT01190007 is a completed Phase 4, multi-center, open-label, non-randomized, single-group study of Caduet in Japanese patients with hypertension and hypercholesterolemia, or angina pectoris and hypercholesterolemia. The registry lists 159 participants in the Caduet group and a 52-week primary safety period. The listed amlodipine/atorvastatin dose combinations were 2.5 mg/5 mg, 2.5 mg/10 mg, 5 mg/5 mg and 5 mg/10 mg.
The primary outcome counted participants with treatment-emergent adverse events and serious adverse events. The record reports 11 participants with serious adverse events and 101 with other adverse events among 159 at risk. It lists six serious-event terms, each with 0.6%. There is no control group, so these are descriptive observations rather than comparative treatment estimates. Numerical results for the listed secondary blood pressure and lipid outcomes, analysis methods, uncertainty estimates, missing-data handling and sample-size justification are not reported in the registry record.