This page provides an independent statistical analysis and educational interpretation of publicly reported results. ClinicalTrials.gov provides the official trial registry record. Numerical trial results on this page are restricted to the results posted in the ClinicalTrials.gov record; calculations added by Clinical Biostats are labelled as such.
1. Trial at a Glance
This page covers the registry results for A Phase II Study of Carfilzomib in the Treatment of Relapsed/Refractory Mantle Cell Lymphoma (NCT02042950), a na phase 2 study. The primary outcome is Overall Response Rate of Carfilzomib.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02042950 |
| Phase | Phase 2 |
| Condition | Lymphoma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 6 |
| Arms in the primary outcome | n/a |
| Lead sponsor | M.D. Anderson Cancer Center |
| Results posted | Yes |
2. Clinical Question
Population
Patients with relapsed or refractory mantle cell lymphoma (MCL). The condition is listed as lymphoma, and the primary outcome description specifies relapsed/refractory MCL.
Intervention
Single-agent carfilzomib. The recorded schedule starts with 20 mg/m² by vein on Days 1 and 2 in Cycle 1, followed by 56 mg/m² for subsequent doses. Doses are scheduled on days 1 and 2, 8 and 9, 15 and 16 of a 28-day cycle; after cycle 12, dosing is on days 1 and 2 and 15 and 16 only.
Comparator
There is no separate comparator group. The study is recorded as single-group, with no randomized allocation and no masking.
Primary question
The primary question is whether single-agent carfilzomib is associated with responses in patients with relapsed/refractory MCL, measured by overall response rate over a timeframe of 21 months. The registry record says a final analysis could not be completed because enrollment requirements were not met.
3. Trial Design
This is a Phase II, single-group study of carfilzomib in relapsed or refractory mantle cell lymphoma. The registry record lists M.D. Anderson Cancer Center as the sponsor, an enrollment of 6, and a status of terminated. The recorded start date is 2014-07-14, and the primary completion date is 2017-12-20.
Because the study has one treatment group, its design does not include a concurrent control arm. Allocation is recorded as NA, and masking is recorded as none. The registry record therefore does not describe a randomized, blinded comparison of carfilzomib with another treatment or with no treatment. The posted observations can describe the treated group, but they cannot provide a direct between-group estimate of a treatment effect.
The intervention is carfilzomib, given on a repeated schedule within 28-day cycles. The recorded dose starts at 20 mg/m² by vein on Days 1 and 2 in Cycle 1, followed by 56 mg/m² for subsequent doses. The schedule includes days 1 and 2, 8 and 9, 15 and 16; following cycle 12, doses are scheduled on days 1 and 2 and 15 and 16 only. The registry record does not give additional treatment-delivery details such as dose modifications or adherence.
The study's stated aim is to evaluate efficacy through response rate and to study safety. The record reports that enrollment requirements were not met and that a final analysis could not be completed. The specific enrollment target, the requirements that were unmet, and the reasons for termination beyond that statement are not reported in the registry record.
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: Out of 6 enrolled participants 2 failed screening.
Overall Study
| Stage | Carfilzomib |
|---|---|
| STARTED | 4 |
| COMPLETED | 0 |
| NOT COMPLETED | 4 |
Reasons for not completing this period:
| Reason | Carfilzomib |
|---|---|
| Progressive Disease | 4 |
Analysis population for the primary outcome, as defined in the registry: Unable to complete final analysis due to not meeting enrollment requirements.
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 | Carfilzomib |
|---|---|---|
| Participants analysed | 4 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 1 |
| Age, Categorical: >=65 years | Participants | 3 |
| Sex: Female, Male: Female | Participants | 1 |
| Sex: Female, Male: Male | Participants | 3 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 1 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 3 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Region of Enrollment: United States | participants | 4 |
6. Primary Endpoint
The registry lists the primary outcome as Overall Response Rate of Carfilzomib, measured over 21 months.
| Element | Registry entry |
|---|---|
| Result type | N/A |
| Measure of spread | not applicable |
| Unit | not stated |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is overall response rate, with a timeframe of 21 months. In general, response rate is the proportion of participants who meet prespecified criteria for a response to treatment. It is used to describe tumor response in a defined study population, rather than the length of time a response lasts. The response criteria and the exact assessment schedule are not reported in the registry record.
For this study, the primary outcome description says response rate is intended to evaluate the efficacy of single-agent carfilzomib in patients with relapsed/refractory MCL. However, the record states that a final analysis could not be completed because enrollment requirements were not met. It does not provide a final response-rate estimate, the number of participants included in an endpoint analysis, or uncertainty estimates such as a confidence interval. Consequently, the stated endpoint identifies the question the study was designed to address, but the record does not present a completed answer to that question.
Response rate can be informative in a single-group study because it records whether responses were observed among participants receiving the intervention. Without a comparator group, though, it cannot by itself distinguish the intervention's contribution from other explanations for observed responses. That is a general limitation of interpreting response outcomes from uncontrolled studies, not a finding about this particular treatment.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies overall response rate as the primary outcome and gives a timeframe of 21 months. It does not report a completed primary analysis. Its outcome description explicitly states that a final analysis could not be completed because enrollment requirements were not met. The enrollment listed is 6, but the record does not say how many participants had evaluable response data or whether every enrolled participant was included in any endpoint assessment.
No primary response-rate estimate, confidence interval, or p-value is reported in the registry record. Without an estimate and its denominator, the magnitude and precision of any response finding cannot be assessed from the primary outcome information. The absence of a reported estimate is not evidence that the response rate was zero, nor does it establish that responses were or were not observed. The registry record simply does not provide a final analysis of that endpoint.
The design also constrains interpretation. This is a single-group study with no randomized allocation, no masking, and no separate comparator. In general, an outcome observed in a single group does not provide a direct estimate of how outcomes would differ under another treatment or under a control condition. The record therefore cannot support a controlled comparison based on the design information reported.
Enrollment of 6 is important context for the incomplete analysis, but it should not be treated as an endpoint denominator. The registry record does not specify the planned enrollment, the eligibility or enrollment requirements that were unmet, or the number of participants with outcome assessments. Those details are not reported in the registry record, so the reasons the primary analysis could not be completed cannot be elaborated beyond the record's statement.
The safety information is also limited in scope. The registry reports serious adverse event figures for a group titled Carfilzomib, alongside other adverse-event figures and serious-event terms. Those counts describe the recorded safety observations; they do not provide a comparative safety estimate because the study has no control group. The record does not establish from those figures whether an event was caused by carfilzomib.
Overall, the posted information documents a Phase II study, its intervention, enrollment, and selected safety observations, but it does not provide a final efficacy analysis. No conclusion about the size, precision, or comparative meaning of an efficacy effect can be drawn from a response-rate result that is not reported.
9. Statistical Methods Explained
The registry record describes a Phase II study with a single treatment group. It does not report an inferential statistical method for the primary endpoint. The primary outcome is overall response rate, but the record provides no response-rate estimate, confidence interval, p-value, or analysis population. The number of analyses reported is 0. The statistical analysis plan, including any prespecified model or testing procedure, is not reported in the registry record.
What does a response-rate estimate represent?
In general, a response-rate estimate is a proportion: the number of participants meeting a response definition relative to the number evaluated under that definition. Its interpretation depends on a clear response definition, a specified assessment period, and a defined analysis population. Those details matter because different rules for response classification or for handling participants without an assessment can change what the proportion represents. The response criteria, denominator, and missing-assessment rules are not reported in the registry record.
In general, a confidence interval describes the uncertainty around an estimated quantity under the assumptions of the method used. For a proportion, uncertainty depends in part on how many participants contribute outcome information and how the data are analyzed. A small information base can make an estimate imprecise, so the point estimate alone would not tell the whole story. In this study, however, no final response-rate estimate or confidence interval is reported. The record therefore does not permit an assessment of the primary endpoint's statistical precision.
Why does the single-group design matter?
In general, a single-group study can describe outcomes observed among people who received the intervention. It does not directly estimate the contrast between that intervention and a concurrent control condition. Differences in participant characteristics, disease course, assessment practices, or other factors can affect outcomes, and a single-group design cannot separate those influences through random allocation to a comparison group. This is a general design limitation; it is not evidence that any particular alternative explanation occurred in this study.
No allocation comparison is available in the registry record, and masking is recorded as none. The record does not state whether the endpoint assessments used independent review or any other procedure to limit assessment bias. Such procedures should not be assumed. The specific methods for classifying responses are also not reported.
What can be inferred from the safety counts?
Safety counts and percentages are descriptive summaries of the recorded events and participants at risk. A percentage needs its denominator to be interpreted, and denominators can differ across safety outcomes or analysis populations. The registry lists 4 participants at risk for serious adverse events and 2 affected, and 4 at risk for other adverse events and 4 affected. It also lists 0 deaths. These are the posted counts, not a comparative test of safety. The record does not report a statistical comparison or an adjusted analysis of adverse events.
The listed serious-event terms, Upper Respiratory Infection and Hypotension, each have a reported value of 25.0%. These values should be read as registry-reported percentages. The record does not describe the event-level counting rules, whether a participant could contribute more than one event, or whether events were assessed as treatment-related. No causal attribution should be inferred from the percentages alone.
How should planning calculations be understood?
General sample-size planning calculations depend on choices such as the target effect, significance level, allocation, and statistical approximation. The planning assumptions associated with this record describe a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the effect to detect expressed as a stated fraction of an observed effect. These assumptions describe planning illustrations, not the design of this single-group trial. They do not establish that the trial had two arms, and they are not evidence of observed power. The record does not report that these assumptions were the study's own design justification.
Any sample-size illustration should be kept separate from the study's actual enrollment and from its incomplete primary analysis. Planning values indicate what might be required under stated assumptions; they do not show what happened in the trial or resolve the missing endpoint result. In this record, the stated enrollment is 6, while the reason given for the incomplete final analysis is that enrollment requirements were not met. The target enrollment and the detailed planning rationale are not reported in the registry record.
Overall, the available record supports a descriptive account of the design and selected outcome information, but not a reconstruction of the primary statistical analysis. The analysis population, missing-data approach, response criteria, precision estimates, and inferential methods are not reported in the registry record. General statistical principles can clarify why those details matter, but cannot substitute for the absent study-specific analysis.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists 3 outcome measures and 0 analyses. It identifies overall response rate as the primary outcome, but does not report the other outcome measures' details in the information available for this account. The statistical analysis plan and any strategy for handling multiple outcomes or multiple statistical tests are not reported in the registry record.
In general, multiplicity arises when several outcomes, comparisons, time points, or statistical tests are examined. If multiple tests are treated as independent opportunities to find a result, the chance of at least one apparently notable result can increase. Prespecified priorities and an appropriate analysis plan help readers understand how multiple results should be interpreted. No such plan is reported, and no analyses are reported, so it is not possible to determine whether multiplicity was addressed or whether it affected any findings.
The record says the final analysis could not be completed because enrollment requirements were not met. It does not report how missing outcome information was handled, what counted as an evaluable participant, or whether an intention-to-treat or another analysis population was specified. The number enrolled is 6, but that number should not be assumed to be the denominator for the primary endpoint or for every safety summary.
In general, missing-data rules can influence an endpoint estimate, especially when the outcome is not observed for every participant. A complete interpretation would require the outcome assessment status and prespecified handling rules. Those details are not reported in the registry record. The available information therefore does not permit an assessment of missing-data bias, analysis-population selection, or multiplicity adjustments.
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 | 2 | 4 | 50.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Carfilzomib | 0 of 4 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 0% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Carfilzomib | 4 of 4 |
Most frequent serious adverse event terms
| Event term | Organ system | Carfilzomib |
|---|---|---|
| Upper Respiratory Infection | Infections and infestations | 1 of 4 |
| Hypotension | Vascular disorders | 1 of 4 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Carfilzomib |
|---|---|---|
| Fatigue | General disorders | 4 of 4 |
| Platelet Count Decrease | Investigations | 4 of 4 |
| Anemia | Blood and lymphatic system disorders | 3 of 4 |
| Elevated BUN | Metabolism and nutrition disorders | 3 of 4 |
| Hyperglycemia | Metabolism and nutrition disorders | 3 of 4 |
| White Blood Cell Decrease | Investigations | 3 of 4 |
| Blurred Vision | Eye disorders | 2 of 4 |
| Cough | Respiratory, thoracic and mediastinal disorders | 2 of 4 |
| Creatinine Increase | Investigations | 2 of 4 |
| Dehydration | Metabolism and nutrition disorders | 2 of 4 |
Serious events with the highest arm-level rate: upper respiratory infection (25.0%), hypotension (25.0%).
The registry record lists safety figures for the Carfilzomib group. For serious adverse events, 4 participants are listed as at risk and 2 as affected. For other adverse events, 4 are listed as at risk and 4 as affected. The record also lists 0 deaths. These are descriptive counts; they are not estimates from a comparison between treatment groups.
Two serious adverse event terms are listed: Upper Respiratory Infection and Hypotension, each with a reported value of 25.0%. The percentages are presented as recorded. The registry does not give further detail about event-level counting, timing, severity beyond the serious-event grouping, or treatment attribution. The listed percentages should not be used to infer those details.
The serious-event and other-event risk groups both have 4 participants listed, but the record does not explain the relationship between these safety denominators and enrollment of 6. It is therefore not possible to determine from the registry information why the safety group counts differ from total enrollment. With no comparator group, these figures cannot establish whether the observed events were more or less frequent than they would have been under another treatment or condition.
12. What the Result Does, and Does Not, Mean
The record establishes that a Phase II, single-group study of carfilzomib was conducted in relapsed or refractory MCL, with enrollment listed as 6. It also identifies overall response rate over 21 months as the primary endpoint and gives the carfilzomib schedule. The safety record includes counts for serious and other adverse events, 0 deaths, and two serious-event terms with reported percentages.
What the record does not establish is a final estimate of response rate. It states that a final analysis could not be completed because enrollment requirements were not met, and it reports no response estimate, confidence interval, or p-value. The absence of a reported estimate cannot be interpreted as no response, and the available information cannot establish the size or precision of an efficacy finding.
The single-group design also means the study record does not give a randomized comparison with another treatment or a control condition. In general, outcomes recorded in one group are descriptive and do not by themselves identify what would have happened to comparable participants under a different condition. No treatment-effect contrast can be read from the design information reported.
The safety counts are likewise descriptive. They do not by themselves show that carfilzomib caused an event, nor do they quantify how safety compares with another option. The registry does not report event attribution or a comparator group.
Thus, the record can be used to understand the study's intent, design, enrollment, and selected safety observations. It cannot be used to state a completed primary efficacy result, to judge its statistical precision, or to make a comparative treatment claim. Those limits follow from the incomplete final analysis and the single-group design, rather than from any conclusion about the treatment itself.
13. Why This Trial Matters Statistically
This study is statistically notable because its stated primary question and its available results do not align: the registry identifies overall response rate as the primary endpoint, but says a final analysis could not be completed because enrollment requirements were not met. Enrollment is listed as 6. Without a reported endpoint estimate and analysis population, readers cannot evaluate the primary result's magnitude or precision.
The case illustrates the distinction between a planned outcome and an analyzed outcome. Naming a primary endpoint clarifies what a study intends to measure, but it does not supply an estimate or show how that estimate was obtained. Response criteria, denominator rules, missing-data handling, and an analysis method are important to interpreting a response-rate result. Those details are not reported in the registry record.
The single-group design provides another important statistical lesson. It can describe outcomes among participants receiving carfilzomib, but it lacks a randomized comparator for estimating a between-group difference. Consequently, even a completed response-rate summary would have answered a descriptive question about the group rather than supplying a controlled treatment contrast.
The record also separates safety description from comparative safety inference. Adverse-event counts and percentages can communicate what was recorded in the treated group, but without a control group they do not indicate how event frequency compares under another condition. Interpretation is further limited by the lack of details about event attribution and safety denominators.
Finally, planning assumptions should not be mistaken for actual study design or evidence of statistical power. The registry's planning illustration uses assumptions that include equal allocation between two arms, while the study design is recorded as single-group. That contrast underscores why planning examples must be interpreted as illustrations rather than as trial characteristics or observed 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.
The principal limitation is the lack of a completed primary analysis. The registry record states that a final analysis could not be completed because enrollment requirements were not met. Although enrollment is listed as 6, the record does not identify the intended enrollment target, the specific requirements that were unmet, or the number of participants with evaluable primary outcome data.
The primary endpoint is named, but key interpretation details are not reported in the registry record: response criteria, assessment procedures, the endpoint denominator, the analysis population, and methods for handling missing outcomes. No response-rate estimate or uncertainty interval is reported. As a result, neither the outcome magnitude nor its precision can be assessed from the primary endpoint information.
The single-group design has no separate comparator, and masking is recorded as none. The record does not describe procedures that might otherwise address assessment concerns, nor does it report a statistical method for the primary endpoint. The statistical analysis plan and the rationale for the study's design are not reported in the registry record.
The safety summaries have limitations as well. The record lists different adverse-event categories and their risk groups, but does not explain why those groups differ from total enrollment. Event attribution, event-level counting rules, and a comparative safety analysis are not reported. These data therefore support description of recorded events only.
The registry status is terminated, and the recorded primary completion date is 2017-12-20. The record does not provide further detail about the termination or its operational circumstances. These limitations prevent a fuller account of why enrollment requirements were not met and how that affected the planned analyses.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: A Phase II Study of Carfilzomib in the Treatment of Relapsed/Refractory Mantle Cell Lymphoma — NCT02042950.
- Registry record last updated 2019-05-01; status last verified 2019-04.
- Results first posted 2019-05-01.
- 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 NCT02042950, a Phase II, single-group study of carfilzomib in relapsed or refractory mantle cell lymphoma. M.D. Anderson Cancer Center is listed as the sponsor. Enrollment is 6; status is terminated. The recorded start date is 2014-07-14, and the primary completion date is 2017-12-20.
The primary endpoint is overall response rate over 21 months. The registry states that a final analysis could not be completed because enrollment requirements were not met; no final response-rate estimate is reported. The safety record lists 2 of 4 participants at risk as affected by serious adverse events, 4 of 4 at risk as affected by other adverse events, and 0 deaths. Upper Respiratory Infection and Hypotension are each listed at 25.0% as serious adverse event terms.