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 INCB024360 Study for Patients With Myelodysplastic Syndromes (MDS) (NCT01822691), a na phase 2 study. The primary outcome is Overall Response Rate (ORR).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01822691 |
| Phase | Phase 2 |
| Condition | Myelodysplastic Syndromes |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 15 |
| Arms in the primary outcome | n/a |
| Lead sponsor | H. Lee Moffitt Cancer Center and Research Institute |
| Results posted | Yes |
2. Clinical Question
Population
Participants with myelodysplastic syndromes (MDS) were the study population. The primary outcome description identifies the population as all participants. The registry lists enrollment of 15, but does not report a more detailed description of participant characteristics in the information available for this analysis.
Intervention
The study evaluated INCB024360, given orally at 600 mg twice a day for 16 weeks, unless clear evidence of disease progression or toxicity was evident. The primary purpose was to assess whether MDS responded favorably. Long-term disease outcomes after participants finished taking INCB024360 were also part of the study’s stated purpose.
Comparator
There was no control group or randomized comparison. The registry identifies the study as single-group, with allocation listed as not applicable and masking as none. Its results therefore describe outcomes among participants treated in the study; they do not estimate a difference between INCB024360 and another treatment or usual care.
Primary question
The primary endpoint was Overall Response Rate (ORR), measured by the number of participants meeting the registry’s response criteria for MDS, with a timeframe of up to 12 months. The registry describes criteria including Complete Remission, Partial Remission, Marrow CR, and Hematological Improvement, and also defines Stable Disease. It does not report the observed number meeting the primary endpoint or an ORR estimate.
3. Trial Design
This was a Phase II, single-group study of INCB024360 in participants with myelodysplastic syndromes. The registry lists enrollment of 15. The study was sponsored by H. Lee Moffitt Cancer Center and Research Institute and is marked completed. Its listed start was 2013-07 and its primary completion was 2015-01.
Participants in the treatment group received INCB024360 orally at 600 mg twice a day for 16 weeks, unless clear evidence of disease progression or toxicity was evident. The primary outcome timeframe was up to 12 months. The study’s stated purpose was to assess response of MDS to treatment and to evaluate longer-term disease outcomes after participants had finished taking the drug.
The design has no concurrent comparator: allocation is listed as not applicable, masking as none, and the model as single group. This matters when interpreting the results. A response count in a treated group can describe what happened to participants during the study, but it cannot by itself distinguish an intervention effect from other explanations, such as the course of the disease or differences between this study population and another population.
The listed secondary outcomes include mean time to acute myeloid leukemia progression, median overall survival, counts of participants with study-related serious adverse events, and counts of participants with study-treatment-related adverse events. The registry record does not report results for those outcomes in the information available for this analysis. It also does not report a detailed protocol or statistical analysis plan. The design is therefore identifiable, but important details about conduct and analysis cannot be established from 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 | INCB024360 Treatment |
|---|---|
| STARTED | 15 |
| COMPLETED | 13 |
| NOT COMPLETED | 2 |
Reasons for not completing this period:
| Reason | INCB024360 Treatment |
|---|---|
| Withdrawal by Subject | 2 |
Analysis population for the primary outcome, as defined in the registry: All participants
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 participants
| Characteristic | Unit | INCB024360 Treatment |
|---|---|---|
| Participants analysed | 15 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 2 |
| Age, Categorical: >=65 years | Participants | 13 |
| Age, Continuous | years | 72 (62 to 85) |
| Sex: Female, Male: Female | Participants | 3 |
| Sex: Female, Male: Male | Participants | 12 |
| Region of Enrollment: United States | participants | 15 |
6. Primary Endpoint
The registry lists the primary outcome as Overall Response Rate (ORR), measured over Up to 12 months.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is Overall Response Rate (ORR), expressed in the registry as a number of participants. It is intended to summarize how many participants met response criteria for MDS during a timeframe of up to 12 months. The registry cites the Response Criteria for Patients with Myelodysplastic Syndrome and the International Working Group 2006 criteria. It lists Complete Remission, Partial Remission, Marrow CR, and Hematological Improvement in any cell line, and defines Stable Disease as failure to achieve at least Partial Remission without evidence of progression for > 8 weeks.
Response endpoints are used in clinical studies to describe whether participants show specified changes in disease status under prespecified criteria. They can provide a clinically interpretable outcome within a defined follow-up period. A count, however, is not by itself an estimate of how response compares with what would have happened without treatment. That comparison usually requires a suitable control group or other design and analysis assumptions.
The registry record identifies the endpoint and its measurement framework, but does not report the observed number of participants meeting the endpoint, an ORR estimate, or uncertainty around an estimate. The endpoint definition also does not state in the available record exactly how the listed categories were combined into the reported primary count. The response result and that operational detail are not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The available results do not include an observed primary response count or an ORR estimate. Although the registry identifies ORR as the primary outcome and gives a timeframe of up to 12 months, it does not report how many participants met the response definition. The treatment’s response rate, and the precision of any response estimate, therefore cannot be assessed from the registry record.
The registry lists enrollment of 15 and identifies all participants as the primary outcome population. Enrollment is not itself a response result. It should not be read as a count of responders, nor does the record establish from this information how many participants contributed evaluable data to each outcome. The analysis population and handling of participants without an outcome assessment are not reported in the registry record.
The study is single-group and unmasked, with no randomized comparator. Any response observations from this design would describe outcomes among participants in the study, rather than a randomized estimate of the difference attributable to INCB024360. Without a comparison group, the result cannot separate treatment-associated changes from other influences on disease status. This is a design limitation, not evidence for or against a treatment effect.
Safety information lists 3 participants with serious adverse events and 14 with other adverse events in the INCB024360 Treatment group; each of the listed top serious event terms is reported as 6.7%. These counts should be read as registry-reported participant counts, not as proof that treatment caused the events. The registry’s secondary outcomes refer to study-related serious events and treatment-related adverse events, but the available information does not provide further event-level detail or explain the attribution process. Deaths are not reported in the adverse-event information.
No effect estimate, confidence interval, hypothesis test, or sponsor analysis for the primary endpoint is reported in the registry record. Consequently, statistical significance, uncertainty, and the magnitude of any response cannot be evaluated. General statistical interpretation would require the observed response count and a clearly specified analysis population, along with the analysis method and any uncertainty measure. Those details are not reported in the registry record.
The study is marked completed, but that status should not be confused with complete public reporting of outcomes. The registry names several secondary endpoints, yet their results are not reported in the information available for this analysis. The posted information supports a description of the design, primary endpoint definition, and reported adverse-event counts; it does not support a conclusion about efficacy or a comparative safety assessment.
9. Statistical Methods Explained
Statistical methods are difficult to evaluate when the registry record identifies outcomes but does not report their results or the analysis plan. The study design and outcome definitions still provide context for understanding what the intended measurements mean and what conclusions would require additional information.
What does a single-group design estimate?
In a single-group study, outcomes are observed among participants receiving the study intervention. A response count can describe the number meeting the endpoint, and a response proportion can describe that count relative to a defined analysis population. This study’s registry describes the primary endpoint as a number of participants, but does not report the observed count or an ORR estimate. A single-group result would not, on its own, estimate the difference between treatment and a control condition.
Random allocation to concurrent groups is one way to make a treatment comparison less vulnerable to differences between participants and other sources of bias. This study has no randomized comparison. The design therefore cannot offer the same direct comparison as a randomized controlled trial. General explanation: the absence of a control group does not make the observed outcomes meaningless, but it limits what can be attributed to the intervention.
What does a confidence interval communicate?
General explanation: a confidence interval gives a range of values compatible with the observed data under a specified statistical procedure and its assumptions. It communicates uncertainty around an estimate; it does not repair design limitations or establish causation. No confidence interval for ORR is reported in the registry record, and the missing response count means that the precision of the primary result cannot be assessed from this information.
Why does the endpoint definition matter?
The registry names ORR and cites MDS response criteria. It lists Complete Remission, Partial Remission, Marrow CR, and Hematological Improvement in any cell line, and separately describes Stable Disease as failure to achieve at least Partial Remission without evidence of progression for > 8 weeks. A reproducible analysis needs a clear rule for which categories count toward the primary response outcome, how participants with incomplete assessments are handled, and which assessment timepoints are used. The available record does not report all of these operational details. The listed timeframe is up to 12 months, but the exact assessment schedule is not reported in the registry record.
How should the safety counts be understood?
The registry reports participant counts for serious and other adverse events in the treatment group, along with percentages for the listed top serious event terms. Counts describe how many participants were recorded in those categories. They do not by themselves convey the number of separate event episodes, timing, duration, severity beyond the serious classification, or causal attribution. General explanation: safety interpretation depends on definitions, follow-up, exposure, and whether events are judged related to treatment. Details about those processes are not reported in the registry record.
What can planning illustrations tell readers?
General explanation: a sample-size calculation can illustrate how many participants might be needed for a future study under stated assumptions about the effect to detect, significance level, allocation, and statistical model. Such a calculation is a planning aid, not an observed result and not observed power. The assumptions described for planning illustrations in this analysis include a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; the effect to detect is specified as a fraction of the observed effect. Those are illustration assumptions, not evidence that this single-group trial used a two-arm design or that those were its own design assumptions. No sample-size justification for the trial is reported in the registry record. Because an observed primary effect is not reported, a planning illustration should not be treated as a trial result.
What analysis details are absent?
The registry record does not report a statistical analysis plan, the method used to estimate ORR, a hypothesis test, an uncertainty interval, adjustment for covariates, or an interim-analysis plan. The record also does not explain how missing outcome assessments or participants who stopped treatment were handled. These details matter because different analysis choices can change the estimate and its uncertainty. In their absence, interpretation should remain descriptive and limited to the information actually reported.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists a primary endpoint and several secondary outcomes, including time to acute myeloid leukemia progression, overall survival, and adverse-event outcomes. The available record does not report a multiplicity strategy, such as a hierarchy among outcomes or an adjustment to account for multiple statistical tests. It also does not report a statistical analysis plan or inferential test results. Because no primary response estimate or hypothesis test is reported, the effect of multiplicity on interpretation cannot be assessed.
Missing-data handling is also not reported in the registry record. It does not describe how participants without an outcome assessment, participants who discontinued treatment, or assessments outside the planned timeframe were handled in the primary analysis. The registry identifies the primary outcome population as all participants, but does not provide enough detail to determine the analysis population actually used or the completeness of endpoint data.
General explanation: missing outcomes can affect both an estimate and its precision, especially if the likelihood of missingness is related to disease status, treatment, or tolerability. The direction and size of any effect cannot be inferred without information about the amount, timing, and reasons for missing data and the analysis approach. Those details are not reported in the registry record.
The study is single-group, so there is no randomized control-group analysis to interpret. No adjusted or unadjusted primary analysis is reported, and there is no reported interim-analysis plan. These omissions leave uncertainty about both the planned inferential framework and the data used for the final outcome summaries.
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 |
|---|---|---|---|
| INCB024360 Treatment | 3 | 15 | 20.0% |
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 |
|---|---|
| INCB024360 Treatment | 14 of 15 |
Most frequent serious adverse event terms
| Event term | Organ system | INCB024360 Treatment |
|---|---|---|
| Allergic reaction | Immune system disorders | 1 of 15 |
| Lung infection | Infections and infestations | 1 of 15 |
| Lymph gland infection | Infections and infestations | 1 of 15 |
| Lymphedema | Vascular disorders | 1 of 15 |
Most frequent non-serious adverse event terms
| Event term | Organ system | INCB024360 Treatment |
|---|---|---|
| Anorexia | Metabolism and nutrition disorders | 7 of 15 |
| Nausea | Gastrointestinal disorders | 5 of 15 |
| Platelet count decreased | Investigations | 4 of 15 |
| Weight loss | Investigations | 3 of 15 |
| Constipation | Gastrointestinal disorders | 2 of 15 |
| Diarrhea | Gastrointestinal disorders | 2 of 15 |
| Vomiting | Gastrointestinal disorders | 2 of 15 |
| Hyperglycemia | Metabolism and nutrition disorders | 2 of 15 |
| Rash maculo-papular | Skin and subcutaneous tissue disorders | 2 of 15 |
| Chills | General disorders | 2 of 15 |
Serious events with the highest arm-level rate: allergic reaction (6.7%), lung infection (6.7%), lymph gland infection (6.7%), lymphedema (6.7%).
The registry reports 3 participants with serious adverse events and 14 participants with other adverse events in the INCB024360 Treatment group. It also lists allergic reaction, lung infection, lymph gland infection, and lymphedema as top serious event terms, each reported as 6.7%. The listed percentages should be read as the registry reports them; no additional calculation or comparison is needed to describe them.
These counts do not establish that INCB024360 caused the events. The registry’s secondary outcomes distinguish study-related serious adverse events from study-treatment-related adverse events, but the available safety information does not explain how relatedness was assessed or provide event narratives, timing, duration, or severity detail beyond the serious classification. The registry record does not report deaths in this safety information.
General explanation: adverse-event counts are descriptive. They can summarize what was recorded during study follow-up, but interpretation depends on how events were defined, how long participants were observed, the completeness of reporting, and whether events were attributed to treatment. The record does not provide enough detail to judge those factors or compare safety with another treatment. The single-group design also means these counts are not a comparative estimate of risk.
12. What the Result Does, and Does Not, Mean
The registry record establishes that a Phase II, single-group study evaluated INCB024360 in participants with MDS and identifies ORR as its primary endpoint. It describes the response criteria and reports adverse-event counts for the treatment group. It does not report the observed primary response count, an ORR estimate, or uncertainty around the primary endpoint. The main efficacy question therefore remains unanswered by the results described in the available record.
The absence of a randomized comparator also limits what this study could establish about treatment effects. Even if a response estimate were reported, a single-group outcome would describe what happened among treated participants rather than provide a randomized estimate of how outcomes differed from a control group. This does not show that treatment is effective or ineffective; it defines the scope of inference supported by the design.
The safety counts are descriptive and should not be interpreted as proof of causation. They do not provide a complete account of event timing, participant exposure, attribution, or comparison with another group. Those details are not reported in the registry record. The listed serious-event terms and their reported percentages add detail to the safety record, but they do not replace a fuller safety analysis.
The study is marked completed, and the registry lists longer-term outcomes among its aims and secondary outcomes. Completion status does not mean all outcomes are publicly reported in the information available. Results for the named survival and progression outcomes are not reported in the registry record. Nor is there a reported analysis plan, missing-data approach, or account of the population used for each analysis.
In practical statistical terms, the available information supports a careful description of the trial design, endpoint definition, enrollment, and reported adverse-event counts. It does not support a conclusion about the magnitude, precision, or comparative effect of INCB024360 on MDS response, survival, or safety.
13. Why This Trial Matters Statistically
This study is statistically informative as an example of how a clearly named clinical endpoint and a completed trial status do not necessarily provide enough information to interpret a treatment result. The registry identifies ORR as the primary endpoint and describes the response framework, but does not report the response count or estimate. Without that result, readers cannot assess the observed response level or its precision.
The single-group structure is also central to interpretation. A response outcome in a treated group can be described, but it cannot serve as a randomized comparison with an untreated or differently treated group. Readers need to distinguish the question a study can answer from a broader question about the treatment’s causal effect. This design limits comparative inference regardless of the size of any observed response count.
The registry also illustrates the difference between an outcome list and a reported analysis. Several secondary outcomes are named, but their results and the statistical methods used to analyze them are not reported in the available record. This leaves uncertainty about how outcomes were defined in practice, how missing assessments were handled, and whether any multiplicity approach was planned.
Finally, safety counts require careful framing. Participant counts and listed serious event terms are useful descriptive information, but they do not by themselves establish causation or comparative risk. Clear reporting of event definitions, attribution, follow-up, and analysis populations would be needed to support a fuller interpretation. The central statistical lesson is to treat endpoint definitions, study design, and result reporting as separate pieces of evidence, each with its own limits.
14. Limitations
- The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
- Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.
The most important limitation for interpreting efficacy is that the registry record does not report the primary response result. It provides the endpoint name, criteria, and timeframe, but not the number meeting the endpoint, an ORR estimate, or a measure of uncertainty. The registry also does not state in the available information exactly how the response categories were combined for the primary count.
The single-group design has no control group, and masking is listed as none. As a result, the study does not provide a randomized comparison. Any outcomes observed in the treatment group cannot, from this design alone, distinguish an intervention effect from other explanations. The registry record does not provide additional information that would support a comparative analysis.
The registry lists enrollment of 15, but does not report the analysis population used for each outcome, the completeness of outcome assessments, or how missing data and discontinuations were handled. Follow-up and assessment details beyond the primary timeframe of up to 12 months are not reported. Results for the listed secondary survival and progression outcomes are also not reported in the available record.
Safety information is limited to participant counts and listed serious event terms with reported percentages. The record does not provide detailed event timing, duration, exposure, causal attribution methods, or deaths in this safety information. A fuller evaluation of safety and efficacy would require these missing results and methods. These limitations concern what can be concluded from the registry record; they are not evidence that unreported outcomes were favorable or unfavorable.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Phase II INCB024360 Study for Patients With Myelodysplastic Syndromes (MDS) — NCT01822691.
- Registry record last updated 2016-01-18; status last verified 2015-11.
- Results first posted 2016-01-18.
- 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 II, single-group study of INCB024360 in participants with myelodysplastic syndromes. Enrollment is listed as 15. Treatment was 600 mg orally twice a day for 16 weeks, unless clear evidence of disease progression or toxicity was evident. The primary endpoint was Overall Response Rate, measured as a number of participants by criteria cited from the International Working Group 2006, with a timeframe of up to 12 months.
No randomized comparator was used. The registry lists secondary outcomes for AML progression, overall survival, and adverse events. It reports 3 participants with serious adverse events and 14 with other adverse events; each listed top serious event term is reported as 6.7%. The primary response result, results for the named secondary outcomes, and detailed statistical methods are not reported in the registry record.