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 Vosaroxin and Decitabine in Older Patients With Acute Myeloid Leukemia and High-risk Myelodysplastic Syndrome (NCT01893320), a na phase 1, phase 2 study. The primary outcome is Maximum Tolerated Dose (MTD) of Vosaroxin in Combination With Decitabine.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01893320 |
| Phase | Phase 1, Phase 2 |
| Condition | Leukemia |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 66 |
| Arms in the primary outcome | n/a |
| Lead sponsor | M.D. Anderson Cancer Center |
| Results posted | Yes |
2. Clinical Question
Population
The study concerns older patients with acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS). The registry lists leukemia as the condition and gives no narrower description of the eligible population in the primary-outcome record.
Intervention
The treatment under study is vosaroxin in combination with decitabine. The registry lists both as drugs and describes a single treatment group. Its arm description begins with a schedule for the combination but is truncated, so it does not provide a complete account of the treatment regimen.
Comparator
There is no separate control group or randomized comparator. The registry identifies a single-group model and no allocation. Consequently, the study does not provide a like-for-like comparison with another treatment or with a control group.
Primary question
The primary question is what maximum tolerated dose of vosaroxin, in combination with decitabine, is identified under the registry’s dose-limiting-toxicity definition. The record also describes a broader research aim of studying whether the combination can help control AML or MDS and assessing safety, but it does not report a numerical primary-outcome result.
3. Trial Design
This is a phase I/II, single-group study sponsored by M.D. Anderson Cancer Center. The registry lists an enrollment of 66 and a completed status. The recorded study start is 2013-07-18, and the primary completion date is 2021-01-11. Masking is listed as none, and allocation is listed as not applicable. There is no randomized control group in the registered design.
The intervention is the combination of vosaroxin and decitabine. The arm descriptions distinguish a phase I maximum-tolerated-dose group from a combination-treatment group, but the registry excerpt does not give a complete treatment schedule. The primary outcome is the maximum tolerated dose (MTD) of vosaroxin in combination with decitabine. A secondary outcome is participants with a response.
The adverse-event record lists 7 participants at risk in the phase I MTD group and 59 in the combination group. Those counts are consistent with the registered enrollment of 66, but the record does not explain the relationship between the groups in enough detail to infer individual treatment histories or analysis populations. Nor does the single-group design support a direct treatment-versus-control comparison. Results from the listed groups should therefore be read as descriptive safety information, not as evidence from randomized arms that are comparable by design.
The registry record does not report a sample-size justification, detailed statistical analysis plan, or numerical primary-outcome result. The presence of phase I and phase II labels does not by itself establish what formal procedures were used for dose escalation, response evaluation, or decision-making.
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 | Phase I MTD Vosaroxin | Vosaroxin + Decitabine |
|---|---|---|
| STARTED | 7 | 59 |
| COMPLETED | 7 | 59 |
| NOT COMPLETED | 0 | 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 | Phase I MTD Vosaroxin | Vosaroxin + Decitabine | Total |
|---|---|---|---|---|
| Participants analysed | 7 | 59 | 66 | |
| Age, Categorical: <=18 years | Participants | 0 | 0 | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 2 | 9 | 11 |
| Age, Categorical: >=65 years | Participants | 5 | 50 | 55 |
| Age, Continuous | years | 69 (41 to 75) | 69 (60 to 78) | 69 (41 to 78) |
| Sex: Female, Male: Female | Participants | 0 | 24 | 24 |
| Sex: Female, Male: Male | Participants | 7 | 35 | 42 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 0 | 3 | 3 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 | 3 | 3 |
| Race (NIH/OMB): White | Participants | 7 | 49 | 56 |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 | 4 | 4 |
| Region of Enrollment: United States | participants | 7 | 59 | 66 |
6. Primary Endpoint
The registry lists the primary outcome as Maximum Tolerated Dose (MTD) of Vosaroxin in Combination With Decitabine, measured over 21 days.
| 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 is the maximum tolerated dose of vosaroxin in combination with decitabine, expressed in mg/m^2. The registry defines MTD as the highest daily oral dose evaluated at which fewer than 33% of patients experience a dose-limiting toxicity (DLT). It describes a non-hematologic DLT as a clinically significant grade 3 or 4 adverse event or abnormal laboratory value, assessed by the treating physician as related to study drug and unrelated to disease progression, intercurrent illness, or concomitant medications, during the first 28 days on study. A hematologic DLT is defined as severe myelosuppression with a hypoplastic marrow with less than 5% cellularity and no evidence of leukemia 42 days from the start of therapy.
In general, an MTD endpoint is used in dose-finding research to identify a dose in relation to a prespecified toxicity rule. It is not itself a measure of comparative effectiveness. The registry lists a timeframe of 21 days, while the endpoint definition refers to a 28-day period for non-hematologic DLTs and a 42-day assessment for the hematologic definition. That difference in wording makes the intended timing harder to interpret. The endpoint description refers to a daily oral dose, while the arm description begins by describing intravenous vosaroxin; the registry record does not resolve this difference. A numerical MTD result is not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registered study asks a dose-finding and safety question about vosaroxin combined with decitabine in older patients with AML or high-risk MDS. Its primary outcome is defined through dose-limiting toxicity, rather than a comparison of response or survival between treatment groups. This distinction matters: an MTD result, if reported, would describe a dose selected under the registry’s toxicity rule. It would not by itself establish how the combination compares with another treatment or how much clinical benefit it produces.
The registry record does not report a numerical MTD, a dose-level result, or a statistical analysis for the primary endpoint. It lists participants with a response as a secondary outcome, but provides no response result in the registry entries. The study’s brief summary states that its goals are to learn whether the combination can help control AML or MDS and to study safety; those goals should not be mistaken for reported findings. Without outcome estimates, their uncertainty, and the corresponding analysis details, the effect or precision of the treatment cannot be assessed from this record.
The adverse-event data provide descriptive counts for two groups. The phase I MTD group lists 6 of 7 participants with a serious adverse event and 2 of 7 with another adverse event; the combination group lists 48 of 59 with a serious adverse event and 19 of 59 with another adverse event. Deaths are listed as 0 and 7, respectively. These are group-specific reported counts, not a randomized comparison. The group sizes differ, and the registry does not report an adjusted comparison or establish that the groups are interchangeable. The counts should not be interpreted as causal contrasts between treatment strategies.
The record lists serious adverse-event terms with percentages, including febrile neutropenia at 42.9%, lung infection and oral mucositis at 28.6% each, sepsis at 18.6%, and bone pain and dehydration at 14.3% each. The registry entries do not specify the denominator or group assignment for each of these percentages. They are therefore best read as terms and percentages reported in the safety record, not as evidence that any one group experienced those rates.
In general, a confidence interval communicates the range of estimates compatible with the data and the analysis assumptions; it is not reported for the primary endpoint in the registry record. The absence of a reported estimate and interval means the primary result’s precision cannot be assessed. The design also lacks a randomized comparator, so it cannot isolate the combination’s effect from other explanations for observed outcomes. These limits concern what the registry record can establish, not a conclusion that the treatment did or did not work.
9. Statistical Methods Explained
The registry describes a phase I/II, single-group study with no masking and no allocation. The primary outcome is an MTD defined by dose-limiting toxicity, and the safety record presents counts for a phase I MTD group and a combination group. The record does not list statistical analyses for the outcomes. The following concepts explain how such information is generally interpreted; they do not establish which methods investigators used.
What does an MTD rule mean?
In general, dose-finding studies use observed toxicities at evaluated doses to inform a dose-selection decision. An MTD definition sets a rule linking the dose under consideration to the frequency or proportion of participants experiencing DLTs. The registry’s definition identifies a highest daily oral dose under a threshold of fewer than 33% experiencing DLTs. To interpret an actual MTD finding, readers would need the doses evaluated, the numbers treated at each dose, the DLT counts and classifications, and the decision procedure used to select the dose. Those details and a numerical MTD result are not reported in the registry record.
The registry defines two kinds of DLT. Non-hematologic toxicities use a clinical and attribution-based definition, including a grade 3 or 4 event or abnormal laboratory value judged related to study drug. The hematologic definition instead specifies severe myelosuppression, a marrow cellularity criterion, and no evidence of leukemia at a later assessment. In general, explicit criteria help make endpoint classification more consistent, but clinician attribution and evaluation timing can still affect which events count. The registry does not report how disagreements, incomplete assessments, or other ambiguous cases were handled.
Why do timing definitions matter?
The listed endpoint timeframe is 21 days. The description separately refers to non-hematologic DLTs during the first 28 days and a hematologic assessment 42 days from treatment start. In general, follow-up windows define which events can contribute to an endpoint. Different windows can change which toxicities are captured and when a dose decision can be made. The registry record does not clarify how the listed timeframe relates to the two windows in the definition. Its endpoint definition also refers to a daily oral dose, while the arm description begins with intravenous vosaroxin. The record does not explain that difference, so the dose metric and schedule cannot be reconciled from the registry description alone.
What can descriptive safety counts show?
Counts of participants with serious or other adverse events describe how many participants in each listed group were recorded as affected. In general, these counts do not show the timing, duration, severity distribution, recurrence, or relationship of every event to treatment. A participant count is also different from an event count when one participant can experience more than one event. The registry’s safety summary does not provide the full detail needed to reconstruct event histories. The named adverse-event terms and their percentages are reported separately from group-specific counts, without a denominator or group assignment in the registry record for interpretation.
For a randomized comparison, an analysis might estimate a between-group difference and quantify its uncertainty. That framework does not apply directly to the two groups: the registered design is single-group, and the record says the arms are not a like-for-like control comparison. In general, descriptive comparisons across groups can be affected by differences in who entered each group, when they were treated, and what treatment they received. The registry does not report adjusted comparisons that address such issues.
How should planning illustrations be read?
In general, a sample-size calculation describes how many participants or events a hypothetical design might require under stated assumptions. The registry-related planning assumptions describe a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and an effect expressed as a fraction of the observed effect. They are explicitly planning illustrations based on the observed effect, not the trial’s own design. They should not be read as observed power or as evidence that the study was planned with those assumptions. The record does not include the numerical effect estimates or calculated sample sizes needed to interpret particular planning scenarios.
In general, over-dispersion means that variability in count data is greater than a simple statistical model expects. If a count outcome has over-dispersion, a model that assumes less variability can produce uncertainty estimates that are too narrow. That concept is not a reported analysis for this trial: the registry record lists no statistical analyses, and the primary outcome is an MTD rather than a reported count-model result.
More broadly, no confidence interval, p-value, adjusted estimate, or model specification for the primary outcome is reported in the registry record. Without such information, readers cannot assess the precision of an estimated MTD or determine how any formal inference was conducted. General statistical concepts can clarify what those quantities would mean, but they cannot fill in results or methods that are absent from the record.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists a primary outcome for MTD and a secondary outcome titled participants with a response. It also lists two outcome measures, but no analyses are reported. The record does not describe a multiplicity strategy, such as a prespecified hierarchy or adjustment for testing more than one outcome. Without that information, it is not possible to tell whether or how the chance of false-positive findings across multiple analyses was addressed. This is especially relevant when interpreting several outcomes or subgroup findings, although no such analysis results are reported in the registry record.
Missing-data handling is not reported in the registry record. It does not specify how participants without a completed toxicity assessment, response evaluation, or other outcome measurement were handled, nor whether any imputation or sensitivity analysis was used. For an MTD endpoint, incomplete follow-up can matter because the endpoint definition depends on observing toxicities within specified windows. The available record does not establish whether all participants had complete assessments under those definitions.
The analysis populations are also not reported in the registry record. The safety information lists 7 participants at risk in the phase I MTD group and 59 in the combination group, but it does not explain which participants were included in each analysis, how group membership was defined, or whether the same participants contributed to every outcome. The registered enrollment is 66; that total alone does not identify the analysis population for each endpoint.
These omissions limit reproducibility and interpretation. They do not establish that analyses were not performed outside the registry record; they mean the record does not report them. The registry also does not provide a detailed statistical analysis plan, interim-analysis procedures, or a sample-size justification.
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 |
|---|---|---|---|
| Phase I MTD Vosaroxin | 6 | 7 | 85.7% |
| Vasoroxin + Decitabine | 48 | 59 | 81.4% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Phase I MTD Vosaroxin | 0 of 7 |
| Vasoroxin + Decitabine | 7 of 59 |
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 |
|---|---|
| Phase I MTD Vosaroxin | 2 of 7 |
| Vasoroxin + Decitabine | 19 of 59 |
Most frequent serious adverse event terms
| Event term | Organ system | Phase I MTD Vosaroxin | Vasoroxin + Decitabine |
|---|---|---|---|
| Febrile Neutropenia | Blood and lymphatic system disorders | 3 of 7 | 18 of 59 |
| Lung Infection | Infections and infestations | 2 of 7 | 13 of 59 |
| Sepsis | Infections and infestations | 1 of 7 | 11 of 59 |
| Infection | Infections and infestations | 0 of 7 | 8 of 59 |
| Mucositis Oral | Gastrointestinal disorders | 2 of 7 | 4 of 59 |
| Catheter Related Infection | Infections and infestations | 0 of 7 | 3 of 59 |
| Dehydration | Metabolism and nutrition disorders | 1 of 7 | 2 of 59 |
| Respiratory Failure | Respiratory, thoracic and mediastinal disorders | 0 of 7 | 3 of 59 |
| Urinary Tract Infection | Infections and infestations | 1 of 7 | 2 of 59 |
| Blood Bilirubin Increased | Investigations | 0 of 7 | 2 of 59 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Phase I MTD Vosaroxin | Vasoroxin + Decitabine |
|---|---|---|---|
| Hypokalemia | Metabolism and nutrition disorders | 0 of 7 | 4 of 59 |
| Blood Bilirubin Increase | Investigations | 1 of 7 | 2 of 59 |
| Fall | Injury, poisoning and procedural complications | 0 of 7 | 3 of 59 |
| Acute Kidney Injury | Renal and urinary disorders | 1 of 7 | 1 of 59 |
| Gait disturbance | General disorders | 0 of 7 | 2 of 59 |
| Alanine Aminotransferase Increased | Investigations | 0 of 7 | 1 of 59 |
| Arthritis | Musculoskeletal and connective tissue disorders | 0 of 7 | 1 of 59 |
| Aspartate Aminotransferase Increase | Investigations | 0 of 7 | 1 of 59 |
| Burn | Skin and subcutaneous tissue disorders | 0 of 7 | 1 of 59 |
| Conjunctivitis | Eye disorders | 0 of 7 | 1 of 59 |
Serious events with the highest arm-level rate: febrile neutropenia (42.9%), lung infection (28.6%), mucositis oral (28.6%), sepsis (18.6%), bone pain (14.3%).
The safety record lists two groups. In the phase I MTD group, 6 of 7 participants are listed with a serious adverse event and 2 of 7 with another adverse event; deaths are listed as 0. In the combination group, 48 of 59 participants are listed with a serious adverse event and 19 of 59 with another adverse event; deaths are listed as 7. These counts describe what the registry reports for each group. They do not establish that events were caused by treatment, and they are not a randomized comparison.
The registry also lists serious adverse-event terms and percentages: febrile neutropenia, 42.9%; lung infection, 28.6%; oral mucositis, 28.6%; sepsis, 18.6%; bone pain, 14.3%; and dehydration, 14.3%. The registry record does not state the denominator or group assignment for these percentages in the registry record. The terms should therefore not be attributed to a particular group or treated as directly comparable rates. In general, an adverse-event summary does not by itself show event timing, recurrence, severity details, or treatment attribution. Those details are not reported in the registry record.
12. What the Result Does, and Does Not, Mean
The record establishes that a phase I/II study of vosaroxin with decitabine was registered for older patients with AML or high-risk MDS, with an MTD outcome and safety assessment. It reports an enrollment of 66, a completed status, and group-specific adverse-event counts. It also provides definitions for the primary toxicity endpoint and lists response as a secondary outcome. These details describe the registered study and the data reported in its safety record.
However, the registry record does not report a numerical MTD result or a response result. It therefore does not permit a reader to determine what dose was selected under the MTD rule, how many participants responded, or how precisely either result was estimated. The stated research aim of learning whether the combination can help control AML or MDS is not itself a finding. The registry entries do not justify a conclusion that the treatment does or does not work.
The safety counts show that participants in both listed groups were recorded as having serious and other adverse events, and that deaths were recorded in the combination group. These observations are descriptive. Because the study is single-group and the two groups are not a like-for-like control comparison, the counts cannot identify what would have occurred under another treatment or without the combination. They also do not, on their own, establish that a particular event or death was caused by study treatment.
In general, a confidence interval would help describe the uncertainty around an estimated outcome, while a comparison group could help estimate a treatment contrast under appropriate design and analysis assumptions. Neither a primary-outcome estimate with uncertainty nor a randomized comparator is reported. The interpretation is consequently limited to the registered design, endpoint definitions, and descriptive safety figures, rather than a claim about comparative benefit or harm.
There are also unresolved details in the endpoint wording: the listed timeframe differs from the assessment windows in the definition, and the MTD description refers to an oral dose while the arm description begins with intravenous vosaroxin. The registry record does not clarify these points. They limit how confidently the operational endpoint and dose schedule can be reconstructed.
13. Why This Trial Matters Statistically
This trial is statistically notable because its primary outcome is a dose-selection endpoint, not a conventional comparison of clinical outcomes between randomized groups. The MTD rule connects a candidate dose to dose-limiting toxicity, so interpreting the endpoint requires details about doses, participant exposure, toxicity classification, assessment windows, and the selection procedure. Although the registry provides a toxicity definition, it does not report the dose-level results or the final MTD.
The study is registered as phase I/II and single-group, with no masking and no allocation. That design can provide descriptive observations about treatment exposure and safety, but it does not create a randomized control contrast. The two groups in the safety record should not be treated as equivalent trial arms: the registry explicitly says this is not a randomized comparison, and it does not report a like-for-like control comparison. This distinction prevents descriptive differences from being interpreted as causal treatment effects.
The record also illustrates why endpoint specifications need to be internally interpretable. The stated timeframe is 21 days, while the toxicity definitions refer to 28-day and 42-day assessments. The MTD definition refers to a daily oral dose, whereas the arm description begins with intravenous administration. Without clarification, readers cannot fully reconstruct the assessment schedule and dose metric from the registry record.
Finally, the record distinguishes between study aims and reported results. It describes goals of studying control of AML or MDS and safety, but does not report a numerical primary outcome or response result. The safety counts and listed adverse-event terms remain useful as descriptive record entries, while their interpretation is constrained by the absence of analysis details, population definitions, and a comparative design.
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 primary outcome’s numerical result is not reported in the registry record. That omission prevents interpretation of the selected MTD and its precision. The record also does not provide dose-level participant counts, dose-limiting-toxicity counts by dose, or the procedure used to make a dose-selection decision. Although the endpoint definition describes non-hematologic and hematologic DLTs, it does not report how cases were adjudicated or how incomplete assessments were handled.
The timing and dose wording leave additional ambiguity. The outcome timeframe is listed as 21 days, but the definition refers to non-hematologic DLTs during the first 28 days and a hematologic assessment 42 days from treatment start. The MTD definition refers to a highest daily oral dose, while the arm description begins with intravenous vosaroxin. The registry record does not reconcile these statements.
The study has a single-group model and no randomized comparator. The phase I MTD and combination groups in the safety record are not a like-for-like control comparison. The record does not explain group membership or provide a detailed account of analysis populations, and it does not report adjusted estimates. The safety-term percentages are not accompanied by denominators or group assignments. As a result, the safety information is descriptive and cannot establish comparative risks or treatment causation.
The registry lists participants with a response as a secondary outcome but does not report a result. Statistical analyses, missing-data procedures, multiplicity handling, interim analyses, and sample-size justification are not reported in the registry record. The planning assumptions refer to illustrations based on an observed effect, but the numerical estimates needed to interpret any planning scenario are not reported in the registry record. These gaps limit both reproducibility and the conclusions that can be drawn from the registry record.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Study of Vosaroxin and Decitabine in Older Patients With Acute Myeloid Leukemia and High-risk Myelodysplastic Syndrome — NCT01893320.
- Registry record last updated 2022-02-09; status last verified 2022-01.
- Results first posted 2022-02-09.
- 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
NCT01893320 is a completed phase I/II, single-group study sponsored by M.D. Anderson Cancer Center. It studies vosaroxin with decitabine in older patients with AML or high-risk MDS. The registry records enrollment of 66, no masking, and no allocation. The primary outcome is the MTD of vosaroxin in combination with decitabine; participants with a response is listed as a secondary outcome.
The primary endpoint is defined using dose-limiting toxicities, but its numerical result is not reported in the registry record. The record lists safety counts for a phase I MTD group and a combination group, including serious and other adverse events and deaths. Because the design is not randomized and the groups are not a like-for-like control comparison, these counts are descriptive rather than comparative treatment-effect estimates. Analysis methods, missing-data handling, analysis populations, and a sample-size justification are not reported in the registry record.