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 High-Dose Weekly Carfilzomib Plus Cyclophosphamide and Dexamethasone in the Treatment of Relapsed Multiple Myeloma (NCT02597062), a na phase 2 study. The primary outcome is Overall Response Rate After 4 Cycles.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02597062 |
| Phase | Phase 2 |
| Condition | Multiple Myeloma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 76 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Canadian Cancer Trials Group |
| Results posted | Yes |
2. Clinical Question
Population
People with relapsed multiple myeloma after 1-3 prior therapies were the intended population. The primary endpoint description refers to eligible patients who received treatment. The registry lists an enrollment of 76, but does not report how many participants contributed to the primary endpoint analysis.
Intervention
The study evaluated a combination of carfilzomib, cyclophosphamide and dexamethasone. The regimen description specifies carfilzomib at 20 mg/m2 on day 1 of the first cycle, escalating to 70 mg/m2 for subsequent doses on days 1, 8 and 15 of a 28 day cycle. Cyclophosphamide was given weekly, and dexamethasone was given weekly with doses described by age.
Comparator
There was no separate comparator group. This was a single-group study with no masking, so the results cannot provide a randomized, like-for-like comparison against another treatment or a control condition.
Primary question
The primary question concerned the number of participants meeting the registry’s response criteria after 4 cycles, with a listed timeframe of 4 months. The registry identifies the endpoint but does not report the response count or response rate.
This is a question about activity observed under a particular combination regimen in a selected group, not a direct estimate of comparative benefit. The response definition includes stringent complete response, complete response, very good partial response, partial response and minimal response. Those categories reflect different degrees of change in disease measurements, as specified in the endpoint description.
Progression-free survival and overall survival are listed as secondary outcomes. Their outcome results are not reported in the registry record. The registry also does not describe a formal comparison, an analysis population beyond the primary endpoint’s brief description, or an adjustment for differences among participants. Consequently, the central interpretive issue is not whether the regimen outperformed another option, but what evidence the registry makes available about the response endpoint and safety observations in this single group.
3. Trial Design
This was a phase II, single-group study sponsored by the Canadian Cancer Trials Group. The registry marks the study completed and lists an enrollment of 76. It gives a start date of 2016-07-05 and a primary completion date of 2019-06-19. These dates describe the registered study timeline; they do not by themselves establish when each participant was enrolled, treated or followed.
The study evaluated carfilzomib with cyclophosphamide and dexamethasone in relapsed multiple myeloma after 1-3 prior therapies. The regimen description says that carfilzomib began at 20 mg/m2 on day 1 of the first cycle and escalated to 70 mg/m2 for subsequent doses, administered on days 1, 8 and 15 of a 28 day cycle. Cyclophosphamide was 300 mg/m2, capped at 500 mg, and dexamethasone was weekly, with the registry describing 40 mg for participants younger than 70 years and 20mg for those aged 70 years or older.
Allocation is listed as not applicable, masking as none, and the model as single group. There is therefore no concurrent control arm in the registry design. The results cannot be read as a randomized treatment contrast, and the observed course of disease cannot be separated from other influences using an internal comparison group.
The primary outcome is titled “Overall Response Rate After 4 Cycles,” measured as a count of participants and assigned a timeframe of 4 months. Progression-free survival and overall survival are listed as secondary outcomes. Although the registry lists three outcome measures, it reports no analyses. The protocol’s sample-size justification, planned statistical analysis, interim analyses and detailed methods for handling follow-up are not reported in the registry record. The trial’s completed status should not be mistaken for complete public reporting of these results.
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 | Carfilzomib Plus Cyclophosphamide Plus Dexamethasone |
|---|---|
| STARTED | 76 |
| COMPLETED | 75 |
| NOT COMPLETED | 1 |
Reasons for not completing this period:
| Reason | Carfilzomib Plus Cyclophosphamide Plus Dexamethasone |
|---|---|
| Ineligible | 1 |
Analysis population for the primary outcome, as defined in the registry: All eligible patients how have received treatment.
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: All eligible and treated patients
| Characteristic | Unit | Carfilzomib Plus Cyclophosphamide Plus Dexamethasone |
|---|---|---|
| Participants analysed | 75 | |
| Age, Continuous | years | 66.0 (44.0 to 82.0) |
| Sex: Female, Male: Female | Participants | 27 |
| Sex: Female, Male: Male | Participants | 48 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 3 |
| Race (NIH/OMB): Asian | Participants | 1 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 2 |
| Race (NIH/OMB): White | Participants | 68 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 1 |
| Region of Enrollment: Canada | participants | 75 |
6. Primary Endpoint
The registry lists the primary outcome as Overall Response Rate After 4 Cycles, measured over 4 months.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the count of participants who meet the registry’s response definition after 4 cycles, with a stated timeframe of 4 months. The definition combines stringent complete response, complete response, very good partial response, partial response and minimal response. These categories use findings such as serum and urine M-protein, bone marrow plasma cells, soft tissue plasmacytomas and, for stringent complete response, absence of clonal cells in bone marrow.
In general, a response endpoint is used to describe whether disease measurements have improved according to prespecified criteria over a defined assessment period. Combining several response categories can capture degrees of improvement rather than only the most complete response. The precise criteria matter because a participant’s classification depends on the thresholds and assessments specified in the definition.
The registry identifies the endpoint as a participant count, but does not give the observed count, a response rate, uncertainty around a response estimate, or the handling of participants without an assessment. It also does not report the primary endpoint analysis population beyond describing eligible patients who received treatment. Therefore, the endpoint’s definition can be described, but the posted record does not allow readers to assess the observed response result or its precision.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The available record establishes the study design and identifies the question the primary endpoint was intended to address. It does not report the primary response count or rate. The title “Overall Response Rate After 4 Cycles” and the listed timeframe of 4 months describe the endpoint; they are not themselves evidence of the observed response. No conclusion about the size or precision of the response can be drawn from this record.
The trial’s single-group design is central to interpretation. Any observed response in a treated group would describe outcomes during the regimen, but without a concurrent comparator it would not establish how outcomes would have differed under another treatment or no study regimen. Differences in participant characteristics, disease course, assessment timing and other care can complicate comparisons with external groups. Such comparisons are not reported.
The record lists an enrollment of 76, but the safety information uses 75 as the number at risk for the serious-event and other-event summaries. The record does not explain the difference between these figures or say whether the enrolled participants all received treatment or contributed to each endpoint. It is therefore important not to treat the enrollment figure as the denominator for every analysis.
Safety information includes 39 participants with serious events and 28 with other events in the listed group, with 75 at risk for each summary. The registry also lists deaths, but does not describe their causes or whether they were attributed to treatment. The listed serious-event terms include lung infection at 18.7%, fever at 5.3%, sepsis at 5.3%, febrile neutropenia at 4.0%, upper respiratory infection at 4.0% and alanine aminotransferase increased at 4.0%. These figures provide a description of reported events, not a comparison of risk against another group.
Progression-free survival and overall survival are named as secondary outcomes, but their results are not reported in the registry record. There are no analyses listed. Confidence intervals, hypothesis tests and adjusted estimates are also not reported. In general, confidence intervals convey the range of values compatible with data under a statistical model; without an estimate and its interval, the precision of the primary result cannot be evaluated.
The safest statistical reading is therefore limited: the registry documents a completed phase II, single-group study, its treatment regimen, its endpoint definition and selected safety counts. It does not provide enough outcome reporting to judge the magnitude or precision of efficacy results, nor does it support a controlled treatment comparison.
9. Statistical Methods Explained
What does the single-group design permit?
A single-group study observes participants receiving the study regimen without a concurrent control group. In general, it can describe outcomes among the treated group and can provide information about the frequency of recorded events. It cannot directly estimate a randomized difference between the regimen and another treatment. Any comparison with a separate study would be indirect and could reflect differences in eligibility, care, follow-up or outcome measurement.
Allocation is marked not applicable, masking is listed as none, and the model is single group. These design features mean that the record does not describe random assignment or masked assessment. The registry does not report additional procedures to reduce observer influence or to make an external comparison equivalent to a randomized one.
What does a count endpoint mean?
The primary outcome is a count of participants meeting a response definition after 4 cycles. A count is a number of people classified as having the event or outcome of interest. In general, a response rate uses a count together with an analysis denominator. A count alone does not establish a rate, and neither is interpretable without knowing who was eligible for assessment and how missing assessments were handled. The registry does not report the response count, rate or analysis denominator.
The endpoint’s response categories rely on clinical and laboratory criteria. A participant can meet one category based on the specified evidence, and classification depends on applying the definitions consistently. The record describes the criteria but does not report the assessment schedule in detail, independent review procedures, or how disagreement or unavailable measurements were handled.
What can uncertainty measures tell readers?
In general, a confidence interval expresses uncertainty around an estimate under the assumptions of the statistical method. A narrow interval indicates greater precision than a wide interval, while an interval that spans a broad range allows more uncertainty about the underlying value. A p-value, when used, measures how compatible the observed data are with a specified null hypothesis and model; it is not a measure of clinical importance or the probability that a treatment works.
No confidence interval, p-value or other statistical estimate for the primary endpoint is reported in the registry record. The registry lists zero analyses. As a result, readers cannot evaluate the precision of a response estimate or assess any formal statistical test from this record.
How should planning illustrations be distinguished from trial findings?
A sample-size illustration based on an observed effect is a planning exercise, not a report of observed power. In general, such an illustration asks how many participants per arm might be needed under stated assumptions to detect a chosen fraction of an effect. The registry’s illustration assumptions describe a two-sided significance level of 0.05, equal allocation between two arms and a normal approximation. The targeted effect is described as a stated fraction of the observed effect.
Those assumptions do not describe this single-group trial’s design. They should not be read as the trial’s own sample-size calculation, its prespecified analysis plan or evidence that the study had adequate power. The trial’s sample-size justification and statistical plan are not reported in the registry record. No planning estimate should be presented as an observed result or as proof that a result would be detected.
Why does over-dispersion matter in general?
Over-dispersion occurs when the variability in data is greater than a statistical model expects. In general, ignoring it can make standard errors too small and confidence intervals too narrow, giving a misleading impression of precision. The registry does not report a dispersion measure for the primary endpoint, and the endpoint is a participant count rather than a reported rate model. No claim about over-dispersion or a model adjustment can be made for this trial.
What can the safety summaries establish?
The registry gives counts of participants with serious events and other events, alongside numbers at risk. Such summaries are descriptive: they indicate events recorded in the group, not a causal treatment effect. In general, participants may experience more than one type of event, so event categories should not automatically be treated as mutually exclusive. The registry does not specify whether listed term percentages use the same event-counting rules or provide counts for each term. It also does not explain the deaths’ relationship to adverse events or treatment.
Overall, the statistical information is incomplete for inferential evaluation. The endpoint and design can be explained, and selected event summaries can be described, but the absent outcome estimates and analysis details prevent an assessment of effect size, uncertainty or model validity.
10. Multiplicity, Missing Data and Analysis Populations
Multiplicity concerns arise when a study evaluates several outcomes, response categories, time points or statistical tests. In general, testing multiple hypotheses can increase the chance of finding a nominally notable result by chance, so a statistical plan may identify a primary test or specify an adjustment. The registry lists an overall response endpoint and the secondary outcomes progression-free survival and overall survival. It does not report whether these outcomes were tested inferentially, how tests were prioritized, or whether multiplicity was addressed.
The registry lists three outcome measures but zero analyses. It does not provide the primary response result or results for the secondary outcomes. The statistical analysis plan, any interim analysis plan and any multiplicity adjustment are not reported in the registry record. No assumption about these procedures is justified.
Missing-data handling is also not reported. The record does not state how participants without an endpoint assessment were treated, whether assessments could occur outside the stated timeframe, or how incomplete follow-up affected survival outcomes. The primary endpoint description refers to eligible patients who received treatment, but does not give an analysis denominator or explain exclusions.
The enrollment is listed as 76, while safety summaries list 75 at risk. The registry does not explain this difference. It also does not specify analysis populations for the safety outcomes or survival endpoints. These omissions limit the ability to reproduce an analysis or determine whether the reported groups align across outcomes. The endpoint definition and safety counts remain descriptive, but the missing statistical and population details constrain interpretation.
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 |
|---|---|---|---|
| Carfilzomib Plus Cyclophosphamide Plus Dexamethasone | 39 | 75 | 52.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Carfilzomib Plus Cyclophosphamide Plus Dexamethasone | 31 of 75 |
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 |
|---|---|
| Carfilzomib Plus Cyclophosphamide Plus Dexamethasone | 28 of 75 |
Most frequent serious adverse event terms
| Event term | Organ system | Carfilzomib Plus Cyclophosphamide Plus Dexamethasone |
|---|---|---|
| Lung infection | Infections and infestations | 14 of 75 |
| Fever | General disorders | 4 of 75 |
| Sepsis | Infections and infestations | 4 of 75 |
| Febrile neutropenia | Blood and lymphatic system disorders | 3 of 75 |
| Upper respiratory infection | Infections and infestations | 3 of 75 |
| Alanine aminotransferase increased | Investigations | 3 of 75 |
| Aspartate aminotransferase increased | Investigations | 3 of 75 |
| Thrombotic thrombocytopenic purpura | Blood and lymphatic system disorders | 2 of 75 |
| Heart failure | Cardiac disorders | 2 of 75 |
| Non-cardiac chest pain | General disorders | 2 of 75 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Carfilzomib Plus Cyclophosphamide Plus Dexamethasone |
|---|---|---|
| Lung infection | Infections and infestations | 15 of 75 |
| Fatigue | General disorders | 12 of 75 |
| Hypertension | Vascular disorders | 7 of 75 |
| Sepsis | Infections and infestations | 5 of 75 |
| Dyspnea | Respiratory, thoracic and mediastinal disorders | 5 of 75 |
Serious events with the highest arm-level rate: lung infection (18.7%), fever (5.3%), sepsis (5.3%), febrile neutropenia (4.0%), upper respiratory infection (4.0%).
The safety summary lists 75 at risk for both the serious-event and other-event categories. It reports 39 participants with serious events and 28 with other events in the carfilzomib plus cyclophosphamide plus dexamethasone group. The registry also lists 31 deaths. It does not state the causes of these deaths, whether they were considered adverse events, or whether they were judged related to treatment.
The serious-event terms listed include lung infection at 18.7%, fever at 5.3%, sepsis at 5.3%, febrile neutropenia at 4.0%, upper respiratory infection at 4.0% and alanine aminotransferase increased at 4.0%. These percentages describe the registry’s listed term frequencies; individual term counts and further details are not reported. The record does not specify whether participants could contribute to more than one term, so the percentages should not be added or treated as mutually exclusive.
In general, event summaries describe what was recorded in a study group. Without a comparator, they do not establish whether an event was more or less frequent than it would have been under another treatment. They also do not, by themselves, establish causation. The difference between the enrollment figure and the safety at-risk figure is not explained in the registry record.
12. What the Result Does, and Does Not, Mean
The registry record identifies a completed phase II study of a three-drug regimen in relapsed multiple myeloma and defines a primary response endpoint after 4 cycles. It also lists selected safety summaries. These details help readers understand what the study set out to assess and what event information was recorded.
However, the record does not report the primary response count or rate. It therefore does not establish the observed magnitude of response, and no measure of uncertainty is available to show how precise such an estimate would be. The listed secondary outcomes, progression-free survival and overall survival, have no results reported in the registry record. The record lists no analyses.
The design is single group, with no separate comparator. Even if an outcome estimate were available, results from this design would describe the treated group and would not alone establish how outcomes compare with another treatment. A causal claim or comparative conclusion cannot be drawn from the study design information in the registry.
The safety information indicates recorded serious and other events and lists deaths, but it does not supply details about event attribution or causes of death. The event term percentages do not establish a comparative risk. A lack of comparator information is especially important when interpreting safety because event occurrence can also reflect the underlying illness and other factors.
These limitations do not erase the value of a clearly defined endpoint or a record of observed safety events. They do define what the available evidence can support. The registry provides a description of the design, regimen, intended response criteria and selected event data; it does not provide a complete efficacy analysis, a comparative estimate, or enough information to judge statistical precision.
13. Why This Trial Matters Statistically
This trial is statistically instructive because its design and reporting need to be kept distinct. The study is a completed phase II evaluation of a combination regimen, but it has a single group and no concurrent control. That means the design addresses outcomes observed under the regimen rather than a randomized difference between treatment options.
The primary outcome is clearly named and its response criteria are described, yet the result itself is absent from the registry record. This contrast illustrates why endpoint definitions and outcome estimates serve different purposes. A detailed definition supports understanding of what would count as response; only reported outcome data can show how many participants met that definition and how precisely the result is estimated.
The safety summary also illustrates the importance of denominators and event definitions. The registry gives an enrollment figure and separate numbers at risk for safety summaries, without explaining the difference. It lists event counts and term percentages, but does not provide term counts or clarify overlap. Careful reading avoids treating the summaries as directly comparable risks or combining categories without justification.
Finally, planning assumptions should not be confused with a trial’s prespecified statistical design. The registry describes a sample-size illustration based on an observed effect, while the actual study is single group. Such an illustration is not observed power, not an efficacy result and not evidence of a randomized comparison. The record’s missing analysis details make that distinction particularly important.
The trial therefore matters as a case in reading clinical research with attention to design, endpoint definition, denominators and reporting completeness. Statistical interpretation depends not only on the question a study intended to answer, but also on the results and methods actually documented.
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 consequential limitation for interpreting efficacy is that the registry identifies the primary response endpoint but does not report its result. There is no response count, response rate, confidence interval or analysis result. The secondary outcomes progression-free survival and overall survival are listed without results. The registry lists zero analyses.
The single-group design has no concurrent comparator, so it cannot directly estimate a treatment contrast. The record also does not report the protocol’s statistical plan, sample-size justification, interim analyses, multiplicity procedures or details of endpoint assessment. Missing-data handling and analysis populations are not described in sufficient detail to establish who contributed to each outcome.
For safety, the registry gives 75 at risk for both event summaries, while total enrollment is listed as 76; this difference is unexplained. Deaths are listed, but their causes and relationship to treatment are not reported. The registry lists percentages for selected serious-event terms without term counts or details on whether participants could contribute to more than one category.
The population is described as having relapsed multiple myeloma after 1-3 prior therapies, but the record does not provide further participant characteristics in the information summarized. This limits assessment of how representative the group may be of other clinical populations. These reporting and design constraints make the record useful for understanding the study’s stated question and selected safety information, but insufficient for a precise efficacy assessment or comparative conclusion.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: High-Dose Weekly Carfilzomib Plus Cyclophosphamide and Dexamethasone in the Treatment of Relapsed Multiple Myeloma — NCT02597062.
- Registry record last updated 2023-08-28; status last verified 2022-02.
- Results first posted 2020-03-03.
- 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
The registry describes a completed phase II, single-group study of carfilzomib, cyclophosphamide and dexamethasone for relapsed multiple myeloma after 1-3 prior therapies. Enrollment is listed as 76. The primary endpoint is the participant count meeting response criteria after 4 cycles, with a timeframe of 4 months. Progression-free survival and overall survival are listed as secondary outcomes.
The primary response result and secondary outcome results are not reported in the registry record. The registry lists no analyses. Safety summaries list 75 at risk, 39 participants with serious events and 28 with other events, as well as 31 deaths; the record does not describe causes of death or treatment attribution. The single-group design does not provide a concurrent control comparison.