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 of 5-azacitidine in Combination With Vorinostat in Patients With Relapsed or Refractory Diffuse Large b Cell Lymphoma (DLBCL) (NCT01120834), a na phase 1, phase 2 study. The primary outcome is Overall Response Rate (ORR).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01120834 |
| Phase | Phase 1, Phase 2 |
| Condition | Lymphoma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 17 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Weill Medical College of Cornell University |
| Results posted | Yes |
2. Clinical Question
Population
Patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL). The registry record lists lymphoma as the condition and records enrollment of 17.
Intervention
The study used the combination of azacitidine, also referred to in the study title as 5-azacitidine, and vorinostat. Both are listed as drug interventions.
Comparator
No separate comparator group is listed. The study model is single group, and allocation is recorded as NA. The record therefore does not describe a like-for-like control comparison.
Primary question
The listed primary outcome is overall response rate (ORR), measured as a count of participants at 2 cycles. The registry record does not report the response count, the denominator used for that outcome, or an ORR estimate.
The clinical question is whether an objective response was observed among patients receiving the combination, as assessed at the stated time point. A single-group outcome can describe what happened among participants receiving the regimen, but it cannot by itself distinguish a treatment effect from other explanations. Without a comparator, the record cannot establish how the outcome would differ under another treatment or under no treatment.
The study summary also describes hypotheses about safety, effectiveness, and possible epigenetic signatures associated with objective response. These are stated as hypotheses, not as findings. The registry record does not report biomarker results or a gene classifier, and it does not provide the primary ORR result needed to assess the stated response question.
3. Trial Design
This is a phase I/II study of azacitidine in combination with vorinostat in patients with relapsed or refractory DLBCL. The registry lists the study status as completed, enrollment as 17, and a single-group model. Allocation is recorded as NA and masking as none. The sponsor is Weill Medical College of Cornell University.
The registry record lists a start date of 2010-09 and a primary completion date of 2013-04. These dates describe the record’s study timeline; they do not supply results. Although the design is labeled phase I/II, the record does not give separate phase-specific enrollment, treatment schedules, dose levels, escalation rules, or phase-specific outcome analyses. Those details are not reported in the registry record.
A single-group design means the listed outcome concerns participants in the study group receiving the combination, rather than a comparison between randomized treatment groups. The absence of a comparator is important for interpretation: observed responses or adverse events can be described, but differences attributable to treatment cannot be isolated through a concurrent control group. The study is unmasked, so masking is not part of the recorded design.
The primary outcome is Overall Response Rate (ORR), with the measure expressed as a count of participants and a timeframe of 2 cycles. The registry record identifies one outcome measure, but gives no response count, analysis population, response criteria, or statistical analysis method. It also contains no analysis entries. Details of how participants entered the study, whether all enrolled participants received treatment, how treatment exposure was defined, and how the phase I and phase II components were organized 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.
Overall Study
| Stage | All Subjects |
|---|---|
| STARTED | 18 |
| COMPLETED | 18 |
| NOT COMPLETED | 0 |
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 | All Subjects |
|---|---|---|
| Participants analysed | 18 | |
| Age, Continuous | years | 66.5 (27 to 83) |
| Sex: Female, Male: Female | Participants | 7 |
| Sex: Female, Male: Male | Participants | 11 |
6. Primary Endpoint
The registry lists the primary outcome as Overall Response Rate (ORR), measured over 2 cycles.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
Overall response rate is intended to describe the proportion of participants who meet a prespecified definition of objective response during an assessment period. In this record, the primary measure is expressed as a count of participants, and the stated timeframe is 2 cycles. The registry names ORR but does not report the response count or the denominator used to calculate a rate. It also does not specify the response criteria or how assessments were conducted.
As a general statistical concept, a response endpoint offers a direct way to summarize whether participants experienced an outcome classified as response under the study’s criteria. Expressing the measure as a count is not, by itself, the same as reporting a rate: a rate requires a denominator and a clearly defined analysis population. Without those details, an ORR estimate cannot be reconstructed from the registry record.
ORR is used in clinical studies because it focuses on objective response rather than a broader judgment of benefit. However, it does not by itself describe how long a response lasts, whether symptoms changed, or how outcomes compare with another treatment. Those questions require additional outcomes or a suitable comparative design. The record’s stated endpoint and timeframe identify the intended measurement, but the missing result and methods prevent assessment of its magnitude and precision.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies ORR at 2 cycles as the primary outcome, but does not report the outcome result. No response count, response rate, denominator, confidence interval, or p-value is listed. As a result, the posted information does not permit a quantitative assessment of the primary endpoint’s magnitude or precision. The title, study summary, and hypotheses describe the study’s rationale and aims; they are not evidence of an observed response result.
Enrollment is recorded as 17, but that number should not be treated as the ORR denominator. The record does not state how many enrolled participants were evaluable for response, how missing assessments were handled, or whether the analysis included every enrolled participant. The safety information also lists 18 participants at risk, a count that differs from the recorded enrollment. The registry record does not explain this discrepancy, and the two counts should not be reconciled by assumption.
The study’s single-group design supports descriptive statements about the participants included in its analyses, if those results are reported. It does not supply a concurrent comparison group. Therefore, even an observed response rate would not establish how the outcome compares with another treatment or what would have happened in the absence of the combination. The record does not report a randomized allocation or a controlled comparison.
Precision matters as well as the estimated response rate. In general, a confidence interval communicates the range of values reasonably compatible with an estimate under the chosen statistical model. A count from a small study can be particularly uncertain, and a point estimate alone would not show that uncertainty. Here, no estimate or interval is reported, so its precision cannot be evaluated.
The study summary proposes that response may be associated with specific epigenetic signatures and that a biomarker or gene classifier might identify patients likely to respond. These statements are hypotheses. No biomarker analysis, classifier performance, or related result is reported in the registry record. Similarly, the record does not provide the trial’s statistical plan, prespecified analysis population, or any analysis entries. The most defensible reading is therefore limited: the registry describes a completed single-group phase I/II study and names its intended primary outcome, but does not report the outcome evidence needed to judge response or its uncertainty.
9. Statistical Methods Explained
What does a single-group design estimate?
As a general statistical concept, a single-group study can estimate the frequency of an outcome in the group analyzed, provided the outcome definition, denominator, and analysis population are clear. It does not directly estimate a difference between treatment strategies because there is no separate comparator group. In this study, the registry records a single-group model and allocation as NA. No randomized comparison is described.
This distinction matters because descriptive frequency and treatment effect are not interchangeable. An observed outcome may reflect the intervention, participant characteristics, the natural course of disease, other care, or the way participants were selected and assessed. A concurrent comparator can help address some of these alternative explanations; a single-group result cannot resolve them on its own.
How should a response count be interpreted?
The primary outcome is named Overall Response Rate (ORR), expressed as a count of participants at 2 cycles. In general, a count is the number of participants classified as having the event of interest. To interpret it as a rate, readers also need the relevant denominator and a clear account of who was included. The registry record does not report the outcome count, denominator, response criteria, or analysis population. Thus, neither a rate nor a comparison with another group can be recovered from the listed endpoint description.
For a binary outcome such as response or no response, a common general approach is to estimate the response proportion and quantify uncertainty with a confidence interval. The interval is useful because it indicates the range of values compatible with the observed data under the chosen model; it is not a guarantee that the true value lies within that range. No interval or response estimate is reported for this study, and no method for calculating one is listed.
Why does the analysis population matter?
Analysis population rules determine which participants contribute to an estimate. For example, a protocol might define an intention-to-treat population, an evaluable population, or another group, but no such rule is reported in the registry record. It is therefore not possible to tell whether the intended ORR denominator was all enrolled participants, those who started treatment, or those with a response assessment. These choices can change the meaning of the estimate, especially when participants do not complete assessments.
Missing outcome data can also affect interpretation. As a general matter, excluding participants without an assessment may produce a different estimate from counting them as nonresponders or using another prespecified method. A sound interpretation needs to know how missing assessments were identified and handled. The registry record does not report missing-data methods or the number of participants with missing response information.
What role does statistical significance play?
A p-value, when used, summarizes how compatible observed data are with a specified null model and its assumptions. It does not measure the size or clinical importance of an effect. A confidence interval and the underlying estimate are also needed to assess magnitude and precision. The registry record reports no p-value, confidence interval, or inferential analysis for ORR. It also contains no analysis entries, so the statistical methods actually used are not reported.
There is no randomized control comparison in the recorded design to which a conventional between-group test could be applied. This does not prevent descriptive estimation, but it limits the claims supported by the design. Any hypothetical comparison with outcomes from another setting would require careful consideration of differences in populations, outcome definitions, follow-up, and care; no such comparison is reported in the registry record.
What can planning calculations tell readers?
In general, sample-size planning explores how many participants might be needed to detect a specified effect under stated assumptions about variability, significance level, allocation, and statistical method. Such calculations are prospective design illustrations, not observed evidence and not a substitute for the trial’s results. A planned power level is also not the probability that the study’s conclusion is correct.
The registry record does not report a trial-specific sample-size justification, statistical plan, or interim analysis plan. It therefore does not establish which assumptions informed the study’s design or whether any planning target was met. The observed enrollment alone does not reveal the intended power, and no power claim can be drawn from the enrollment count.
Why can over-dispersion matter?
As a general concept, over-dispersion occurs when data vary more than a statistical model assumes. If a model underestimates that variability, its uncertainty estimates may be too narrow and inferential results may be misleading. Whether over-dispersion is relevant depends on the outcome and model. The registry record does not describe a model for ORR or any dispersion assessment, so there is no basis for saying that an over-dispersion adjustment was used or needed in this analysis.
Overall, the registry identifies the design and intended primary measure but omits the result and analysis details required to reproduce or evaluate an estimate. Statistical interpretation must therefore remain descriptive of what the record reports, rather than inferential about efficacy.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists Overall Response Rate (ORR) as the primary outcome and records one outcome measure. It does not provide a statistical analysis plan, a hierarchy of outcomes, or details about planned testing. No analysis entries are listed. The record therefore does not report whether any multiplicity adjustment was planned or used.
In general, multiplicity arises when several hypotheses or outcomes are tested. Testing many questions can increase the chance of obtaining at least one apparently notable result by chance. A prespecified primary outcome and an analysis hierarchy can help readers understand which findings were confirmatory and how other analyses were treated. The record’s designation of ORR as primary identifies the stated endpoint, but does not provide enough information to assess a testing strategy.
Missing-data handling and the analysis population are also not reported. The registry does not say how participants without an assessment at 2 cycles were treated, whether participants who discontinued treatment remained in the analysis, or what denominator was used. These details are essential for interpreting a response count or rate.
The study summary mentions possible epigenetic signatures and a biomarker or gene classifier as hypotheses. The registry record does not report whether these analyses were planned, how many markers were considered, or how any findings would be validated. Without those details, the risk of selective analysis or chance findings cannot be assessed. These omissions do not prove that a particular method was or was not used; they mean the methods are not reported in the registry record.
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 |
|---|---|---|---|
| All Subjects | 0 | 18 | 0.0% |
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 |
|---|---|
| All Subjects | 18 of 18 |
Most frequent non-serious adverse event terms
| Event term | Organ system | All Subjects |
|---|---|---|
| anemia | Blood and lymphatic system disorders | 14 of 18 |
| nausea | General disorders | 12 of 18 |
| hypoglycemia | Blood and lymphatic system disorders | 7 of 18 |
| renal impairment | Renal and urinary disorders | 7 of 18 |
| thrombocytopenia | Blood and lymphatic system disorders | 6 of 18 |
| diarrhea | Gastrointestinal disorders | 6 of 18 |
| vomiting | General disorders | 6 of 18 |
| leucopenia | Blood and lymphatic system disorders | 5 of 18 |
| raised ALP | Blood and lymphatic system disorders | 5 of 18 |
| hyperglycemia | Blood and lymphatic system disorders | 5 of 18 |
The safety record lists an All Subjects group with 18 participants at risk for serious adverse events and 0 participants affected. It also lists 18 participants at risk for other adverse events and 18 affected. The death count is not reported in the registry record. The record does not give event terms among the top serious adverse events.
These counts describe what is entered in the registry, but the record does not define the event collection period, severity criteria, attribution rules, or whether the counts refer to participants with at least one event. The category labeled other adverse events should not be interpreted as a specific event type beyond the wording of the registry entry.
The safety group’s at-risk count of 18 differs from the recorded enrollment of 17. The registry record does not explain the difference, so it is not possible to determine from the record whether the counts use different populations or reflect a data-entry issue. They should not be combined or treated as interchangeable.
A zero count in the serious adverse event field means that no affected participants are listed for that field in this record; it does not establish that serious events were impossible or that all safety outcomes were captured. The entry for other adverse events indicates affected participants were recorded, but the absence of event details limits interpretation.
12. What the Result Does, and Does Not, Mean
The record establishes that a completed phase I/II study was registered for azacitidine combined with vorinostat in patients with relapsed or refractory DLBCL. It identifies ORR at 2 cycles as the primary outcome and describes a single-group design. It also records enrollment of 17 and provides limited safety counts. These are features of the study record, not evidence that the primary outcome was achieved.
The primary ORR result is not reported in the registry record. Without a response count, denominator, response criteria, or analysis population, readers cannot determine the observed response rate. Without an estimate and measure of uncertainty, they also cannot assess how precise such a result would be. The registry record does not report a treatment comparison, so it cannot show whether outcomes differ from another treatment or control condition.
The study summary states hypotheses about the combination’s safety and effectiveness and about epigenetic signatures associated with response. Those statements describe the rationale and questions under study; they do not establish findings. No biomarker result or gene-classifier performance is reported. Likewise, the serious adverse event entry and other adverse event counts are descriptive registry entries, with important details and an explanation of the safety-population count not reported.
In general, a completed study can still have incomplete public results in its registry record. Completion status does not substitute for endpoint estimates, analysis methods, or uncertainty measures. The record supports a description of the intended study and its listed data fields, but not a conclusion about the magnitude or reliability of an ORR result, a causal treatment effect, or biomarker prediction.
13. Why This Trial Matters Statistically
This study illustrates why design and result reporting must be read together. The registry describes a phase I/II study and names a primary endpoint, but the endpoint result is not reported. A stated outcome is not the same as an observed estimate, and completion status does not fill that gap. Clear outcome counts, denominators, analysis populations, and uncertainty measures are needed for readers to evaluate what the study found.
The single-group design also makes the distinction between describing outcomes and estimating comparative effects especially important. A single-group study can characterize outcomes in the analyzed participants, but it does not provide a randomized treatment contrast. This limits causal interpretation even if an outcome estimate is available. Here, the absence of the ORR result further restricts interpretation.
The registry record additionally highlights the importance of coherent participant accounting. Enrollment is recorded as 17, while the safety information lists 18 at risk. Since the record does not explain the difference, readers cannot determine how the safety population relates to enrollment. Reporting population definitions alongside outcome counts would make such figures easier to interpret.
Finally, the record presents biomarker and gene-classifier ideas as hypotheses without reporting results or methods for evaluating them. For statistical interpretation, a hypothesis must be distinguished from a validated finding. The record’s limitations demonstrate why transparent reporting of prespecified analyses, missing-data rules, outcome definitions, and results is necessary for evaluating both clinical outcomes and exploratory biological questions.
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 main limitation for interpreting this trial is that the registry record does not report the primary ORR result. It also omits the response denominator, response criteria, analysis population, and assessment procedures. This prevents evaluation of the endpoint’s magnitude, precision, and reproducibility.
The single-group design has no listed comparator, so the record cannot support a controlled estimate of differences between the combination and another treatment strategy. The record does not report randomization or a masked assessment process; masking is listed as none. It does not describe how the phase I and phase II components were organized, how treatment was administered, or how participants were selected for response assessment. These details are not reported in the registry record.
The safety information has a population-count discrepancy: enrollment is recorded as 17, while the safety group lists 18 at risk. No explanation is reported. The record also does not provide event definitions, collection period, attribution, or detailed serious adverse event terms. The death count is not reported in the registry record.
Further limitations include the absence of a reported statistical analysis plan, sample-size justification, interim analysis information, missing-data approach, and multiplicity strategy. The proposed biomarker and gene-classifier questions are not accompanied by reported results or validation details. These gaps prevent a full assessment of bias, uncertainty, and the robustness of any findings, and they should not be filled by assumptions.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Study of 5-azacitidine in Combination With Vorinostat in Patients With Relapsed or Refractory Diffuse Large b Cell Lymphoma (DLBCL) — NCT01120834.
- Registry record last updated 2017-04-10; status last verified 2017-02.
- Results first posted 2017-04-10.
- 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 a completed phase I/II, single-group study of azacitidine with vorinostat in patients with relapsed or refractory DLBCL. It lists enrollment of 17, no masking, and Overall Response Rate at 2 cycles as the primary outcome, measured as a count of participants.
The primary outcome result, its denominator, and its analysis methods are not reported in the registry record. The safety entries list 18 at risk for serious adverse events, with 0 affected, and 18 at risk for other adverse events, with 18 affected. The safety count differs from the enrollment count, and the record does not explain the discrepancy. A death count is not reported in the registry record.
The record also states hypotheses about response and potential epigenetic biomarkers, but does not report related findings. The study design and aims can be described, while efficacy, precision, comparative effects, and biomarker performance cannot be assessed from the registry entries reported.