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 Phase II Study to Evaluate Overall Response in Patients With Higher Risk Myelodysplastic Syndromes (MDS) Treated With Azacitidine With or Without Deferasirox. (NCT02159040), a randomized phase 2 study. The primary outcome is Overall Response Rate Per IWG 2006 Criteria.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02159040 |
| Phase | Phase 2 |
| Condition | High Risk MDS |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 1 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Novartis Pharmaceuticals |
| Results posted | Yes |
2. Clinical Question
Population
Patients with higher risk myelodysplastic syndromes (MDS), as described in the registry record. The record lists enrollment as 1.
Intervention
Azacitidine with deferasirox. The arm description lists azacitidine 75mg/m2 for 7 days of a 28 day cycle and deferasirox 10 mg/kg/day.
Comparator
Azacitidine alone, described as 75mg/m2 for 7 days of a 28 day cycle.
Primary question
Does adding deferasirox change the overall response rate over 1 year, using IWG 2006 criteria?
The question concerns overall response in patients with higher risk MDS receiving azacitidine, with the randomized comparison intended to distinguish the azacitidine regimen from the regimen that also includes deferasirox. Overall response includes complete response, partial response and hematologic improvement. The registry description says hematologic improvement must be maintained for at least 8 weeks to count as a response.
This is a planned comparative question, not one that the posted results answer. The registry lists only 1 patient and explicitly states that no analysis will ever be done. It does not provide an overall response rate for either group, nor a comparison between groups. The record also does not establish which treatment that patient received. Consequently, the intended clinical question can be identified, but no treatment contrast can be estimated from the registry information.
3. Trial Design
The record describes a Phase II, randomized, open-label, parallel-group comparative trial of azacitidine with or without deferasirox in patients with higher risk MDS. It lists the study as a 2-year, multi-center trial and describes fixed-dose treatment. Masking is listed as none, so the design was open-label rather than blinded. The trial status is terminated.
The planned treatment groups were azacitidine alone and azacitidine plus deferasirox. The listed azacitidine regimen is 75mg/m2 for 7 days of a 28 day cycle. The combination arm also lists deferasirox 10 mg/kg/day. These are registry arm descriptions; they do not establish the treatment received by the patient recorded in the enrollment field.
The registry lists enrollment as 1. The primary outcome is overall response over 1 year, and the record states that no analysis will ever be done. The analysis count is 0. Thus, although randomization and parallel groups describe the planned design, the posted record does not contain a completed comparative analysis or group-specific response findings. It also does not report the reason for termination, whether any planned procedures were completed, or the allocation received by the patient.
The study began on 2014-09-11, and the primary completion date is 2015-06-26. Those registry dates do not supply an explanation for the termination or evidence that the planned follow-up period was completed. The protocol's sample-size justification, recruitment targets, randomization procedure and any changes to the design are not reported in the registry record. The design should therefore be understood as the planned randomized comparison, while the available outcome record remains insufficient to assess that comparison.
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 | Azacitidine |
|---|---|
| STARTED | 1 |
| COMPLETED | 0 |
| NOT COMPLETED | 1 |
Reasons for not completing this period:
| Reason | Azacitidine |
|---|---|
| Death | 1 |
Analysis population for the primary outcome, as defined in the registry: Only one patient in trial. No analysis will ever be done.
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 | Azacitidine |
|---|---|---|
| Participants analysed | 1 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 0 |
| Age, Categorical: >=65 years | Participants | 1 |
| Sex: Female, Male: Female | Participants | 0 |
| Sex: Female, Male: Male | Participants | 1 |
6. Primary Endpoint
The registry lists the primary outcome as Overall Response Rate Per IWG 2006 Criteria, measured over 1 year.
| 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 (ORR) according to IWG 2006 criteria, assessed over 1 year. The registry description includes complete response, partial response and hematologic improvement. It further specifies that hematologic improvement must be maintained for at least 8 weeks to count as such. The description refers to erythroid, platelet and neutrophil response as components of hematologic improvement.
In general, an overall response endpoint combines specified categories of response into a patient-level outcome. A rate can then describe the proportion of an analyzed group meeting the response definition. This kind of endpoint is used to capture more than one clinically relevant response category under a stated set of criteria. Its interpretation depends on applying the criteria consistently, including the required duration for hematologic improvement.
The registry does not provide endpoint values, response counts, group denominators or an analysis. The primary outcome fields for unit, parameter and dispersion are null, and the population description says that only 1 patient was in the trial and no analysis will ever be done. Accordingly, the endpoint definition is available, but no ORR can be estimated from the posted results and no contrast between treatment groups can be evaluated.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central interpretive fact is that the registry lists enrollment as 1 and states, in the primary outcome description, that no analysis will ever be done. The record contains no primary outcome estimate for either treatment group, no treatment difference and no interval expressing the precision of such a difference. There is therefore no posted efficacy result from which to infer whether the regimens had similar or different response rates.
The study was designed as a randomized comparison, but the intended comparison is not the same as an observed comparison. A randomized design can support a causal comparison when participants are assigned to the study groups and outcomes are analyzed appropriately. Here, the record does not report an analyzed outcome by group or identify the treatment received by the listed patient. The design label alone cannot substitute for those missing results.
Precision cannot be assessed without an estimate and its uncertainty. In general, a confidence interval describes a range of values compatible with the observed data under a specified method; a narrow interval indicates greater precision than a wide one. No confidence interval for ORR or for a difference in ORR is reported. Likewise, no test statistic or p-value is posted for the primary comparison. This is not evidence of no difference; it is an absence of an analyzed comparison.
The registry also does not report the number of responses in each arm, an analysis population, or the handling of missing or unevaluable outcome information. As a result, readers cannot determine whether response assessment occurred, how many assessments were available, or how the endpoint definition was applied to individual outcomes. The stated response criteria and time frame describe what the study intended to measure, not what the posted results found.
The safety record is separate from the unreported efficacy comparison. Its serious-event summary lists Death at 100.0%, and the Azacitidine group fields list 1 at risk and 1 affected for serious events. Those entries do not provide a comparative safety analysis, establish a causal relationship to treatment, or resolve the missing response results. The event summary requires cautious reading because a small record and incomplete reporting context do not support a stable estimate of risk.
Any planning illustration based on an observed effect should not be interpreted as a trial result or as observed power. The registry states that the trial had only 1 patient and that no analysis will be done; it does not report a usable observed between-group effect. The overall interpretation is therefore limited: the registry describes a planned Phase II trial and an endpoint, but supplies no analyzed efficacy comparison and only a highly limited safety summary.
9. Statistical Methods Explained
The registry identifies a randomized, parallel-group, open-label Phase II design. In general, random assignment is intended to make treatment groups comparable in expectation, reducing the risk that baseline differences systematically favor one treatment. Parallel-group designs compare groups receiving different regimens over the same study period. Open-label means masking was not used, as the registry lists masking as none. These design features describe the intended study structure; they do not demonstrate that a reliable comparative estimate was obtained.
The primary outcome is a response rate under IWG 2006 criteria. In general, a rate for a binary outcome is based on the number of participants meeting the response definition relative to the number assessed in an analysis group. A treatment comparison might report the rate in each arm and a measure of the difference, with an interval describing uncertainty. The registry contains neither arm-specific response counts nor a comparative estimate. It lists enrollment as 1 and says no analysis will ever be done, so there is no basis for reconstructing an effect from the posted outcome record.
Confidence intervals are useful because an estimate alone does not show how much uncertainty surrounds it. A confidence interval is calculated using a statistical method and the observed data; its width depends in part on the amount of information available and the variability of the outcome. A p-value, when used, assesses how compatible the observed data are with a specified null hypothesis under a statistical model. Neither measure is reported for the primary outcome. Their absence cannot be interpreted as evidence for or against a treatment effect.
The registry lists 0 analyses. It does not state which statistical method was planned for the primary comparison, whether the endpoint would be adjusted for baseline characteristics, or how the response categories would be combined in an analysis beyond the stated ORR definition. The calculation assumptions associated with planning illustrations specify a two-sided significance level of 0.05, equal allocation between the two arms and a normal approximation. They describe planning illustrations, not a reported analysis plan or evidence that those methods were used in this trial.
The planning description says that the effect to detect is a stated fraction of the observed effect, and that the illustrations are computed from the observed effect rather than from the trial's own design. It also distinguishes between participant counts per arm for ordinary outcomes and total event counts for time-to-event outcomes. These are planning conventions. Any resulting sample-size illustration would not be an observed trial result, and it would not be observed power. Because the record provides no analyzed response effect, such an illustration should not be treated as an estimate of what the study demonstrated or as the original enrollment target.
For count or rate outcomes, over-dispersion is a general concern when variation exceeds what a simple model assumes. If ignored, uncertainty may be understated. That issue does not supply a method for this trial: the registry does not report an over-dispersion assessment or a model for the primary outcome. The stated ORR is a response-rate endpoint, but no participant-level outcome data or analysis are posted from which to assess model fit or variability.
For time-to-event outcomes, general methods account for both event timing and incomplete follow-up, often using methods that accommodate censoring. The listed secondary outcomes include time to response, duration of response, progression-free survival, overall survival and time to AML transformation. The registry does not report time-to-event estimates, event counts, censoring rules or analysis methods for these outcomes. It would be inappropriate to infer that a particular survival method was used merely because those outcomes were listed.
Multiplicity arises when several outcomes or hypotheses are assessed: the chance of at least one apparently notable result can increase if many tests are considered without adjustment. The registry lists multiple secondary outcomes, but it does not report a multiplicity strategy. With 0 analyses and no posted primary comparison, there are no trial test results to interpret in light of multiplicity. Similarly, the analysis population, missing-data procedures, interim analyses and sample-size justification are not reported in the registry record.
10. Multiplicity, Missing Data and Analysis Populations
The record lists 10 outcome measures, including secondary outcomes for time to response, duration of response, progression-free survival, overall survival, time to AML transformation, change in serum ferritin, incidence of adverse events and rate of infection. The primary outcome is ORR. In general, considering several outcomes can raise multiplicity concerns: if many hypotheses are tested, chance findings become more likely unless the analysis plan addresses that issue. The registry does not report a multiplicity adjustment, a hierarchy among outcomes or a strategy for interpreting multiple tests.
No inferential results are posted, and the analysis count is 0. Therefore, multiplicity does not explain or qualify any reported efficacy finding; there is no reported efficacy finding to qualify. The statistical plan for the primary and secondary outcomes is not reported in the registry record.
The record does not describe how missing, incomplete or unevaluable response data would be handled. It does not identify an intention-to-treat, per-protocol or other analysis population. The primary outcome description says that only 1 patient was in the trial and no analysis will ever be done, but it does not explain the status of that patient's outcome assessment. No imputation method, censoring rule, sensitivity analysis or approach to withdrawal is reported in the registry record.
These omissions matter because the population included in an analysis and the handling of missing outcomes can affect a response-rate estimate. They also limit assessment of the listed time-to-event outcomes, for which follow-up and censoring details would be important. The record does not establish whether the planned outcome assessments were completed.
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 |
|---|---|---|---|
| Azacitidine | 1 | 1 | 100.0% |
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 |
|---|---|
| Azacitidine | 1 of 1 |
Most frequent serious adverse event terms
| Event term | Organ system | Azacitidine |
|---|---|---|
| Death | Cardiac disorders | 1 of 1 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Azacitidine |
|---|---|---|
| Muscle spasms | Musculoskeletal and connective tissue disorders | 1 of 1 |
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | 1 of 1 |
| Infusion site | Gastrointestinal disorders | 1 of 1 |
Serious events with the highest arm-level rate: death (100.0%).
The serious-event summary lists Death at 100.0%. The Azacitidine group fields list 1 at risk and 1 affected for serious events, and also 1 at risk and 1 affected for other events. These are the posted safety entries; they do not provide an arm-to-arm comparison. The record lists an adverse-event group for Azacitidine, but does not provide corresponding safety fields for the combination regimen in the posted group information.
A percentage in a safety summary should be read together with its denominator and event definition. The registry record does not make the denominator or coding context for the 100.0% figure clear beyond listing the serious term Death and the group fields. Given that the record lists enrollment as 1, the percentage should not be read as a precise estimate of a general risk. It is not evidence that the treatment caused the event, and the registry does not report a causal assessment.
The summary also does not provide timing, patient-level detail, exposure duration or a comparative safety analysis. It should be treated as a limited report of recorded serious-event information, not as a complete evaluation of safety across the planned treatment groups.
12. What the Result Does, and Does Not, Mean
The registry record establishes that a Phase II randomized comparison of azacitidine with or without deferasirox was planned for patients with higher risk MDS. It defines overall response using IWG 2006 criteria, including complete response, partial response and hematologic improvement maintained for at least 8 weeks. It also lists several secondary outcomes and treatment regimens. These details explain the intended research question.
The record does not establish whether adding deferasirox changed response. It lists enrollment as 1, has 0 analyses, and states that no analysis will ever be done. No group-specific response values, treatment difference, confidence interval or primary-outcome test is reported. This means the posted results cannot support a conclusion that the regimens were equivalent, that one was superior, or that either had a particular response rate.
The absence of a result is not a null result. A null result would require an analyzed estimate that showed no evidence of a difference under a stated method; the registry supplies no such estimate. It is also not proof that no response occurred. The response outcome for the listed patient is not reported in the registry record, nor is the treatment assignment documented in the posted information.
The serious-event listing is similarly limited. Death appears as a serious adverse-event term at 100.0%, but the registry information does not support a causal conclusion or a comparative safety estimate. The safety entries should not be generalized into a risk estimate for the intended patient population.
In general, clinical interpretation requires estimates, uncertainty, outcome definitions and enough information about the analyzed population to understand what was compared. Here, the endpoint definition and planned contrast are documented, but the efficacy analysis is absent. The appropriate reading is that this registry record describes an intended trial question and a limited event summary, not evidence resolving the treatment comparison.
13. Why This Trial Matters Statistically
This trial matters statistically because it illustrates the difference between a planned randomized comparison and an interpretable comparative result. Randomization, parallel groups and a defined endpoint describe the study's intended structure. They do not by themselves yield an estimate of treatment effect. The record lists enrollment as 1 and says that no analysis will ever be done, leaving the primary clinical question unanswered in the posted results.
The endpoint definition is also important. ORR combines complete response, partial response and hematologic improvement under IWG 2006 criteria, with a stated minimum duration for hematologic improvement. Such criteria make clear what the study intended to count. Without outcome data, however, a well-defined endpoint cannot be converted into a response rate or a comparison.
The record also shows why safety percentages need context. A percentage without a clear denominator and comparative analysis can be easy to overinterpret, particularly when the listed enrollment is very limited. The serious-event summary is a record of what was entered, not an estimate of treatment-attributable risk.
Finally, the case distinguishes planning calculations from observed evidence. Planning illustrations are not trial results or observed power. They cannot replace the missing response estimates, and they do not explain the registry's enrollment or termination. Careful statistical reading preserves what the record does document while avoiding conclusions that its absent analyses cannot support.
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 trial-specific limitation is the registry's enrollment of 1 and its statement that no analysis will ever be done. The primary outcome therefore has no posted estimate, and the randomized comparison cannot be evaluated. The registry does not identify the treatment received by the listed patient or provide that patient's response status.
The record is marked terminated, but the reason for termination is not reported in the registry record. It is not possible to determine from the posted information whether the trial stopped for recruitment, operational, safety or other reasons. The planned 1-year outcome timeframe is specified, but completion of follow-up and response assessment is not reported in the registry record.
The registry does not provide the protocol's sample-size justification, an analysis population, a statistical method for the primary endpoint, missing-data handling, multiplicity procedures or interim-analysis details. These cannot be inferred from the randomized Phase II label or from the general assumptions used for planning illustrations. The record also does not provide baseline characteristics that would describe the enrolled patient.
The safety information is limited to the listed adverse-event fields and serious-event term. It does not provide a complete comparative safety assessment, exposure context or causal attribution. The listed 100.0% for Death should therefore be interpreted only as a registry entry, not as a stable estimate of risk or evidence of treatment causation. Collectively, these limitations mean that the record documents the study's intent and some safety reporting, but not an analyzable efficacy comparison.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Phase II Study to Evaluate Overall Response in Patients With Higher Risk Myelodysplastic Syndromes (MDS) Treated With Azacitidine With or Without Deferasirox. — NCT02159040.
- Registry record last updated 2017-04-04; status last verified 2017-03.
- Results first posted 2017-02-06.
- 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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Use this trial's endpoint as a starting point for deeper study of effect measures, confidence intervals, and sample-size planning.
18. Record Summary
The registry describes NCT02159040 as a Phase II, randomized, open-label, parallel-group study of azacitidine with or without deferasirox in patients with higher risk MDS. The primary outcome was ORR under IWG 2006 criteria over 1 year. The record lists enrollment as 1, status as terminated and 0 analyses; it states that no analysis will ever be done. No comparative efficacy estimate is reported. The serious-event summary lists Death at 100.0%, alongside limited group-level safety fields. The reason for termination, treatment assignment and outcome status are not reported in the registry record.