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 Vosaroxin for Intermediate 2 or High-risk MDS After Failure With Hypomethylating Agent-based Therapy (NCT01980056), a registered phase 1, phase 2 study. The primary outcome is Dosage Determination for IV-infusion of Vosaroxin in Int-2 or High-risk Mds.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01980056 |
| Phase | Phase 1, Phase 2 |
| Condition | Myelodysplastic Syndrome |
| Allocation | Not Stated |
| Design model | Sequential |
| Masking | None (open label) |
| Enrollment | 10 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Weill Medical College of Cornell University |
| Results posted | Yes |
2. Clinical Question
Population
Adults with pathologically confirmed myelodysplastic syndrome (MDS), classified as intermediate 2 or high-risk after failure of hypomethylating agent-based therapy. The registry description specifies less than 20% blasts in bone marrow, peripheral blood, or both, and an International Scoring System (IPSS) high-risk threshold of at least 1.5.
Intervention
Vosaroxin given by short intravenous infusion. Participants received the dose assigned to their enrolled dose cohort; the study evaluated escalating doses and schedules.
Comparator
No randomized comparator is described. The registry record lists one intervention and says all patients received vosaroxin according to dose cohort. The allocation method is not stated.
Primary question
What maximum tolerated dose of short-infusion vosaroxin could be identified for participants with intermediate 2 or high-risk MDS after failure of hypomethylating agent-based therapy, using dose-limiting toxicity during the first cycle to assess dose levels?
The main statistical task was dose finding, not a randomized estimate of the difference between vosaroxin and another treatment. The primary outcome description defines maximum tolerated dose as the highest dose level at which no more than 33% of patients observed at that level experience a dose-limiting toxicity. The assessed population comprised participants who received at least one dose of vosaroxin, with dose-limiting toxicity assessed in the first cycle of therapy.
The registry also names response and transfusion requirements as secondary outcomes. However, it does not report numerical results for those outcomes, nor does it report a numerical dose-limiting toxicity result or an identified maximum tolerated dose. The clinical question can therefore be described from the planned endpoint and eligibility criteria, but the registered information does not establish what dose was selected or what responses were observed.
3. Trial Design
This was a Phase 1/2, open-label, sequential study sponsored by Weill Medical College of Cornell University. It was marked completed and recorded an enrollment of 10. The study began on 2013-10-25, and its primary completion date was 2015-01-19. No masking was used.
The study enrolled participants with intermediate 2 or high-risk MDS after failure of hypomethylating agent-based therapy. Participants received vosaroxin according to their dose cohort. The adverse-event record lists three dose levels: 50 mg/m2 IV on Days 1 and 4 of 28 Day; 72 mg/m2 IV on Days 1 and 4 of 28 Day; and 50 mg/m2 IV on Days 1, 4, 8 and 11 of. The third dose-level description is incomplete in the registry record, so its full schedule is not reported in the registry record.
The design is described as sequential, and the registry record does not identify a randomized comparison. Dose cohorts are not like-for-like control groups: dose and schedule differ, and the allocation method is not stated. Consequently, contrasts between cohorts would not by themselves provide a controlled estimate of a treatment effect. The record does not report how participants were assigned to dose levels or whether any dose-escalation rules beyond the stated maximum-tolerated-dose definition were used.
The primary outcome was dose determination for IV-infusion vosaroxin, with a timeframe of 1 year. Its description focuses on dose-limiting toxicity during the first cycle. Secondary outcomes include the number of subjects who experience a response and the number of transfusions required during treatment with vosaroxin. Although the registry lists 3 outcome measures, it reports 0 analyses. Numerical primary and secondary outcome results are therefore 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 | Vosaroxin: All Patients Receiving Dose Level 1: Vosaroxin 50 m | Dose Level 2: Vosaroxin 72 mg/m^2 IV on Days 1 and 4 of 28 Day | Dose Level 3: Vosaroxin 50 mg/m^2 IV on Days 1, 4, 8 and 11 of |
|---|---|---|---|
| STARTED | 4 | 4 | 2 |
| COMPLETED | 3 | 3 | 2 |
| NOT COMPLETED | 1 | 1 | 0 |
Reasons for not completing this period:
| Reason | Vosaroxin: All Patients Receiving Dose Level 1: Vosaroxin 50 m | Dose Level 2: Vosaroxin 72 mg/m^2 IV on Days 1 and 4 of 28 Day | Dose Level 3: Vosaroxin 50 mg/m^2 IV on Days 1, 4, 8 and 11 of |
|---|---|---|---|
| Death | 1 | 1 | 0 |
Analysis population for the primary outcome, as defined in the registry: All participants who received at least one dose of Vosaroxin were assessed for DLT in the first cycle of therapy. For this study. MTD is defined as the highest dose level at which no more than 33% of the patients observed at a given dose level experience a DLT.
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 | Dose Level 1: Vosaroxin 50 mg/m^2 IV on Days 1 and 4 of 28 Day | Dose Level 2: Vosaroxin 72 mg/m^2 IV on Days 1 and 4 of 28 Day | Dose Level 3: Vosaroxin 50mg/m^2 IV on Days 1, 4, 8 and 11 of | Total |
|---|---|---|---|---|---|
| Participants analysed | 4 | 4 | 2 | 10 | |
| Age, Categorical: <=18 years | Participants | 0 | 0 | 0 | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 0 | 0 | 0 | 0 |
| Age, Categorical: >=65 years | Participants | 4 | 4 | 2 | 10 |
| Sex: Female, Male: Female | Participants | 1 | 2 | 1 | 4 |
| Sex: Female, Male: Male | Participants | 3 | 2 | 1 | 6 |
| Race/Ethnicity, Customized: White | Participants | 2 | 2 | 1 | 5 |
| Race/Ethnicity, Customized: Unknown or not reported | Participants | 2 | 2 | 1 | 5 |
| Region of Enrollment: United States | participants | 4 | 4 | 2 | 10 |
6. Primary Endpoint
The registry lists the primary outcome as Dosage Determination for IV-infusion of Vosaroxin in Int-2 or High-risk Mds, measured over 1 year.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | mg/m^2 |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint addresses dose determination rather than a direct comparison of clinical outcomes. It is described as the maximum tolerated dose of vosaroxin for short IV infusion in participants with intermediate 2 or high-risk MDS. The registry specifies that participants who received at least one dose were assessed for dose-limiting toxicity in the first cycle of therapy. The maximum tolerated dose is defined as the highest dose level at which no more than 33% of the patients observed at a given level experience a dose-limiting toxicity.
This kind of endpoint is used in dose-escalation research to characterize tolerability across tested dose levels and to guide selection of a dose for further study. That is a general explanation of dose-finding endpoints; it does not establish which dose was selected in this study. A maximum tolerated dose is also not, by itself, evidence that a dose improves disease outcomes. The registry record gives the endpoint definition and timeframe of 1 year, but does not report the observed dose-limiting toxicity counts, the estimated maximum tolerated dose, or a numerical endpoint result.
The two listed secondary outcomes concern response and transfusions during treatment. Those outcomes could describe clinical activity and treatment burden, but their numerical findings are not reported in the registry record. No inference about response frequency or transfusion requirements can be made from their names alone.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry information supports a description of the study’s intent and structure, but not an estimate of its primary endpoint. It identifies a sequential, open-label Phase 1/2 dose-escalation study, an enrollment of 10, three dose-level descriptions, and a primary outcome definition based on dose-limiting toxicity in the first cycle. It does not give the observed dose-limiting toxicity results or identify a maximum tolerated dose. The status is completed, but completion status is not a substitute for outcome data.
The serious adverse-event record contains dose-level counts for participants at risk and participants affected. For Dose Level 1, 4 were at risk and 3 were affected; for Dose Level 2, 4 were at risk and 1 was affected; for Dose Level 3, 2 were at risk and 2 were affected. The record separately lists deaths of 1, 1, and 0 for these levels, respectively. These are descriptive counts in small cohorts, not randomized comparisons. The record does not establish that differences across dose levels are caused by dose, and the allocation method is not stated.
Serious-event terms listed in the registry include Febrile Neutropenia at 75.0%, Disease Progression at 50.0%, Non- Cardiogenic Shock at 25.0%, Sudden Death at 25.0%, and Respiratory Distress at 25.0%. These values are presented as registry term percentages. The registry information does not specify the denominator or counting details for these listed percentages, so they should not be reinterpreted as risks for a particular cohort or as rates over a defined exposure period. The presence of a term in the list also does not establish its cause.
No numerical results are reported for response or transfusion outcomes. Likewise, there are 0 analyses recorded, so no inferential estimate, confidence interval, or p-value for the primary or secondary outcomes is available. Without such results, the size and precision of any treatment-associated change cannot be assessed. This does not mean that the study found no effect; it means the registry record does not report an analysis from which an effect can be evaluated.
The study had no randomized comparator described. Even if cohort-level outcomes were available, comparisons across dose levels would need to be interpreted in light of the sequential design, differing dose schedules, small cohort sizes, and unspecified allocation process. The registry record therefore supports a cautious account of the dose-finding question and the recorded safety counts, but not a causal efficacy conclusion or a reliable between-dose comparison.
9. Statistical Methods Explained
Statistical interpretation begins with identifying the question the design can address. This study is described as sequential and open-label, with participants receiving vosaroxin according to dose cohort. It is not described as a randomized comparison. Accordingly, the central design aim was to evaluate dose levels and dose-limiting toxicity, rather than to estimate a treatment contrast against a concurrent control group. The registry does not report the detailed escalation algorithm, cohort assignment procedure, or statistical analysis plan.
What dose-finding means
In general, dose-finding studies examine tolerability at successive dose levels. A dose-limiting toxicity is an event or condition designated by a protocol as limiting further dose escalation; the exact criteria for defining such toxicity are not reported in the registry record. The listed endpoint defines maximum tolerated dose as the highest observed dose level at which no more than 33% of patients at that level experience a dose-limiting toxicity. This definition describes a decision target, but it does not tell the reader how the study handled incomplete observations, events outside the first cycle, or decisions when the observed data did not align neatly with the threshold. Those details are not reported in the registry record.
The primary endpoint population is specified as participants who received at least one dose of vosaroxin. Dose-limiting toxicity assessment is specified for the first cycle of therapy, while the endpoint timeframe is listed as 1 year. The registry does not clarify how that timeframe relates to the first-cycle assessment or whether additional toxicity observations contributed to dose determination. It also does not report a numerical maximum tolerated dose or the data used to select one.
Reading counts and percentages
Counts are direct descriptions of how many participants were recorded in a category, while a percentage expresses a count relative to a denominator. The adverse-event record lists participants at risk and participants affected for each dose level, along with deaths. These are useful descriptive summaries, but their interpretation depends on the definitions used for “affected,” the observation period, and whether participants can contribute to more than one event category. Such details are not reported in the registry record. The listed serious-event term percentages have no denominator or counting method stated in the record, so they should be read as reported labels rather than recalculated or treated as comparable cohort-specific risks.
Small cohort counts are especially sensitive to individual observations. In general, with few participants, a single person can materially change a descriptive proportion, and estimates tend to be imprecise. That general consideration is relevant to reading these dose-level counts, but the registry record does not provide confidence intervals or other uncertainty estimates for them. Counts across different dose levels should not be treated as a randomized comparison because dose cohort and schedule differ and allocation is not stated.
What an analysis would add
In general, an analysis can add an estimate of an outcome and an account of its uncertainty. A confidence interval describes a range of values reasonably compatible with the data under the analysis assumptions; it is not a guarantee that the true value lies within that range. A p-value, when reported, summarizes how unusual the observed data would be under a specified null model and assumptions. Neither measure replaces the need to assess design, outcome definitions, and data completeness. The registry lists 0 analyses, and no inferential results are reported for the primary endpoint or named secondary outcomes. Therefore, no confidence interval or p-value can be interpreted for those outcomes from the registry record.
The record also includes planning assumptions for sample-size illustrations: a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and an effect to detect expressed as a stated fraction of the observed effect. It says time-to-event illustrations give the total number of events, using the Schoenfeld method, while other illustrations give participants per arm. These are planning assumptions, not observed study results or evidence of observed power. The registry does not report a randomized two-arm comparison for this study, and the listed information does not provide a numerical illustration to interpret. Sample-size planning under hypothetical assumptions cannot supply the missing outcome estimates or establish precision for this study’s dose-finding results.
Over-dispersion and count outcomes
As a general statistical concept, over-dispersion occurs when count data vary more than an assumed model expects. If it is ignored, standard errors can be too small and uncertainty can appear lower than it is. The registry lists response counts and transfusion requirements as secondary outcomes, but it does not report their observed values, distributions, or analysis methods. There is therefore no basis in the registry record to determine whether over-dispersion was relevant or how it was addressed.
More broadly, methods such as regression adjustment, time-to-event analysis, or missing-data modeling should not be attributed to this study unless documented. The registry record does not report such methods, an interim analysis, a sample-size justification for the trial, or a detailed statistical plan. The appropriate interpretation is limited to the design description, outcome definitions, and posted descriptive safety information.
10. Multiplicity, Missing Data and Analysis Populations
The registry names multiple outcomes: a primary dose-determination measure, a response outcome, and a transfusion outcome. It does not report an analysis for these outcomes, and the record lists 0 analyses. A multiplicity strategy—how the chance of false-positive findings across multiple statistical tests would be handled—is not reported in the registry record. No adjustment, testing hierarchy, or prespecified order of secondary outcome tests can be inferred.
Missing-data methods are also not reported in the registry record. The primary endpoint population is described as participants who received at least one dose, with dose-limiting toxicity assessed in the first cycle. The record does not state how participants with incomplete first-cycle assessments, early discontinuation, or missing outcome information were handled. It does not specify whether any analyses used imputation, complete cases, or another approach.
The analysis populations are not described beyond the primary endpoint population definition. It is not reported whether any separate safety, response, or transfusion analysis populations were defined, or how participants were included in the listed adverse-event summaries. The registry does not report an interim-analysis plan, a prespecified statistical analysis plan, or a sample-size justification for the study. These absences limit assessment of how outcome selection, incomplete data, and analysis choices may have affected results. They should not be filled in by assuming conventional practice.
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 |
|---|---|---|---|
| Dose Level 1: Vosaroxin 50 mg/m2 IV on Days 1 and 4 of 28 Day | 3 | 4 | 75.0% |
| Dose Level 2: Vosaroxin 72 mg/m2 IV on Days 1 and 4 of 28 Day | 1 | 4 | 25.0% |
| Dose Level 3: Vosaroxin 50 mg/m2 IV on Days 1, 4, 8 and 11 of | 2 | 2 | 100.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Dose Level 1: Vosaroxin 50 mg/m2 IV on Days 1 and 4 of 28 Day | 1 of 4 |
| Dose Level 2: Vosaroxin 72 mg/m2 IV on Days 1 and 4 of 28 Day | 1 of 4 |
| Dose Level 3: Vosaroxin 50 mg/m2 IV on Days 1, 4, 8 and 11 of | 0 of 2 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 5% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Dose Level 1: Vosaroxin 50 mg/m2 IV on Days 1 and 4 of 28 Day | 3 of 4 |
| Dose Level 2: Vosaroxin 72 mg/m2 IV on Days 1 and 4 of 28 Day | 3 of 4 |
| Dose Level 3: Vosaroxin 50 mg/m2 IV on Days 1, 4, 8 and 11 of | 2 of 2 |
Most frequent serious adverse event terms
| Event term | Organ system | Dose Level 1: Vosaroxin 50 mg/m2 IV on Days 1 and 4 of 28 Day | Dose Level 2: Vosaroxin 72 mg/m2 IV on Days 1 and 4 of 28 Day | Dose Level 3: Vosaroxin 50 mg/m2 IV on Days 1, 4, 8 and 11 of |
|---|---|---|---|---|
| Febrile Neutropenia | Blood and lymphatic system disorders | 3 of 4 | 0 of 4 | 1 of 2 |
| Non- Cardiogenic Shock | General disorders | 0 of 4 | 1 of 4 | 0 of 2 |
| Sudden Death | General disorders | 1 of 4 | 0 of 4 | 0 of 2 |
| Respiratory Distress | Respiratory, thoracic and mediastinal disorders | 1 of 4 | 0 of 4 | 0 of 2 |
| Disease Progression | Blood and lymphatic system disorders | 0 of 4 | 0 of 4 | 1 of 2 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Dose Level 1: Vosaroxin 50 mg/m2 IV on Days 1 and 4 of 28 Day | Dose Level 2: Vosaroxin 72 mg/m2 IV on Days 1 and 4 of 28 Day | Dose Level 3: Vosaroxin 50 mg/m2 IV on Days 1, 4, 8 and 11 of |
|---|---|---|---|---|
| Diarrhea | Gastrointestinal disorders | 2 of 4 | 2 of 4 | 2 of 2 |
| Nausea | Gastrointestinal disorders | 2 of 4 | 3 of 4 | 1 of 2 |
| Fatigue | General disorders | 3 of 4 | 1 of 4 | 1 of 2 |
| Febrile Neutropenia | Blood and lymphatic system disorders | 3 of 4 | 1 of 4 | 1 of 2 |
| Constipation | Gastrointestinal disorders | 3 of 4 | 1 of 4 | 0 of 2 |
| Epistaxis | General disorders | 2 of 4 | 1 of 4 | 1 of 2 |
| Mucositis | Gastrointestinal disorders | 1 of 4 | 1 of 4 | 2 of 2 |
| Vomiting | Gastrointestinal disorders | 1 of 4 | 2 of 4 | 0 of 2 |
| Bacterimia | Infections and infestations | 0 of 4 | 1 of 4 | 2 of 2 |
| Oral Thrush | Infections and infestations | 1 of 4 | 1 of 4 | 0 of 2 |
Serious events with the highest arm-level rate: febrile neutropenia (75.0%), disease progression (50.0%), non- cardiogenic shock (25.0%), sudden death (25.0%), respiratory distress (25.0%).
The serious adverse-event record presents counts by dose level. It lists participants at risk and participants affected, plus deaths. For Dose Level 1, the counts are 4 at risk, 3 affected, and 1 death. For Dose Level 2, they are 4 at risk, 1 affected, and 1 death. For Dose Level 3, they are 2 at risk, 2 affected, and 0 deaths. These are descriptive registry figures; the record does not provide confidence intervals or explain the observation time used for the counts.
The registry also lists serious-event terms and percentages: Febrile Neutropenia, 75.0%; Disease Progression, 50.0%; Non- Cardiogenic Shock, 25.0%; Sudden Death, 25.0%; and Respiratory Distress, 25.0%. The denominator and calculation details for these term percentages are not reported in the registry record. They should not be read as dose-specific percentages or as event rates per unit of follow-up. The record also does not establish whether listed events were considered related to vosaroxin.
These safety summaries describe what was recorded, not what caused an event. Cohort counts are small and the study was sequential rather than a randomized comparison. Differences between dose levels therefore do not establish that a dose caused more or fewer events. The available safety information should be interpreted alongside the limits of the registry definitions and missing analysis details.
12. What the Result Does, and Does Not, Mean
The study record documents an investigation of escalating vosaroxin doses in adults with intermediate 2 or high-risk MDS after failure of hypomethylating agent-based therapy. Its primary statistical purpose was dose determination using dose-limiting toxicity in the first cycle and a stated maximum-tolerated-dose definition. The record also lists response and transfusion outcomes, and it contains descriptive serious adverse-event counts by dose level.
What the registry record does not establish is equally important. It does not report the observed dose-limiting toxicity findings, an identified maximum tolerated dose, numerical response results, or transfusion results. It lists 0 analyses. Completion status and the presence of outcome definitions do not demonstrate that an endpoint was met or not met. The absence of a reported estimate is not evidence that there was no effect; it leaves the effect and its precision unknown from this record.
The serious adverse-event figures show recorded counts for the listed dose levels, but they do not support causal conclusions. The study had no randomized comparator described, dose cohorts had different dose or schedule descriptions, and the allocation method is not stated. The listed serious-event term percentages also lack a reported denominator and calculation details. These limitations preclude interpreting the percentages as directly comparable risks or attributing events to the intervention.
In general, a dose-finding result and an efficacy result answer different questions. Identifying a tolerability boundary would characterize a dose-selection decision under the study’s definitions; it would not by itself establish improvement in response or other clinical outcomes. Here, even that dose-selection result is not reported. The most defensible reading is therefore descriptive: the registry identifies the study’s question, eligibility, design, outcome definitions, and safety counts, while leaving the analyzed primary and secondary outcomes unavailable.
13. Why This Trial Matters Statistically
This study illustrates a statistical distinction between dose finding and comparative evaluation. Its primary outcome was defined around dose-limiting toxicity and maximum tolerated dose, not a randomized treatment-effect contrast. That distinction matters because a dose-escalation study can inform how tolerability was assessed across tested cohorts, while it cannot automatically provide the same kind of evidence as a randomized comparison.
The sequential, open-label design also makes clear why cohort descriptions require context. The registry lists different doses and schedules, and it does not state how participants were allocated to cohorts. Thus, any differences in recorded counts across cohorts could reflect more than the dose itself. The enrollment of 10 and the listed cohort sizes indicate that the safety summaries are based on small groups; in general, small counts carry substantial uncertainty, even when the counts themselves are reported exactly.
The record also demonstrates why an outcome definition is not the same as an outcome result. The maximum-tolerated-dose rule is stated, but no dose-limiting toxicity findings or selected dose are reported. Response and transfusion outcomes are named, but numerical findings are not reported. With 0 analyses recorded, statistical uncertainty and the effect estimates cannot be assessed.
Finally, the registry’s safety terms and counts require careful distinction between description and causal interpretation. Without a randomized comparator and with unspecified allocation, the reported events can be described but not attributed to treatment or compared as if cohorts were exchangeable. This is a useful statistical lesson for interpreting early-phase records: design, definitions, denominators, and analysis results all matter, and missing results cannot be reconstructed from the study’s stated aims.
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.
Several limitations specific to this trial record constrain interpretation. The registry does not report the primary endpoint result: there are no numerical dose-limiting toxicity findings and no identified maximum tolerated dose. It also does not report numerical response or transfusion results, despite listing both as secondary outcomes. There are 0 analyses recorded, so no estimates of uncertainty or inferential results are available.
The study is described as sequential and open-label, with participants receiving vosaroxin according to dose cohort. No randomized comparator is described, and the allocation method is not stated. Dose levels also differ in dose and schedule descriptions, limiting direct interpretation of cohort differences. The Dose Level 3 schedule text is incomplete in the registry record, and the full schedule is not reported in the registry record.
The registry does not give the detailed dose-escalation algorithm, the operational criteria for dose-limiting toxicity, or how incomplete assessments were handled. It does not report the statistical analysis plan, interim analyses, sample-size justification, or methods for missing data and multiplicity. It is also not reported how the adverse-event term percentages were calculated or what denominators they use. These gaps prevent a complete assessment of the planned and executed statistical methods.
Finally, the adverse-event counts are descriptive and come from small dose cohorts. The record does not provide uncertainty estimates, exposure-adjusted rates, or attribution details for the listed events. These limitations restrict both comparisons across cohorts and conclusions about the cause of safety events.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Vosaroxin for Intermediate 2 or High-risk MDS After Failure With Hypomethylating Agent-based Therapy — NCT01980056.
- Registry record last updated 2019-02-19; status last verified 2019-01.
- Results first posted 2018-12-28.
- 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
NCT01980056 is a completed, open-label Phase 1/2 sequential study sponsored by Weill Medical College of Cornell University. It enrolled 10 participants and evaluated vosaroxin in intermediate 2 or high-risk MDS after failure of hypomethylating agent-based therapy. The study began on 2013-10-25, with primary completion on 2015-01-19.
The primary outcome concerned dose determination using dose-limiting toxicity in the first cycle and a stated maximum-tolerated-dose definition. Secondary outcomes concerned response and transfusions. The registry lists 0 analyses and does not report numerical results for the primary or secondary outcomes. Descriptive serious adverse-event counts and listed event terms are available, but their interpretation is limited by small cohorts, the nonrandomized design, and unreported denominator and analysis details.