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 Vorinostat in Treating Patients With Acute Myeloid Leukemia (NCT00305773), a randomized phase 2 study. The primary outcome is Confirmed Complete Response (CR) Rate.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00305773 |
| Phase | Phase 2 |
| Condition | Adult Acute Erythroid Leukemia (M6), Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0) |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 37 |
| Arms in the primary outcome | 2 |
| Lead sponsor | National Cancer Institute (NCI) |
| Results posted | Yes |
2. Clinical Question
Population
Adults with acute myeloid leukemia were eligible across a broad range of AML subtypes and disease settings listed in the registry, including recurrent, refractory, secondary, and untreated AML. The registry also lists several cytogenetic and morphologic AML categories. The enrolled population is described in the record through these eligibility conditions; a more detailed account of participant characteristics is not reported in the registry record.
Intervention
One study arm received oral vorinostat once daily on days 1-21 of each 21-day course. Treatment could continue for up to 17 courses, unless disease progression or unacceptable toxicity occurred.
Comparator
The other arm received oral vorinostat three times daily on days 1-14 of each 21-day course. It used the same stated course interval and maximum number of courses, with treatment subject to progression or unacceptable toxicity. Thus, the randomized comparison concerns two schedules of the same drug, rather than vorinostat versus a different drug or no treatment.
Primary question
The primary outcome was the confirmed complete response rate, measured as the number of participants with a complete response divided by the number evaluable. The central question was whether the observed response rates differed between the once-daily and thrice-daily schedules. The posted estimates are 0.0% for once-daily treatment and 4.5% for thrice-daily treatment. The registry reports a risk difference of 0.045, with a 95% confidence interval from -0.042 to 0.133 and an unadjusted p-value of 0.403. These estimates describe the trial results; they do not establish that one schedule is better.
3. Trial Design
This was a randomized, open-label, parallel-group phase II study sponsored by the National Cancer Institute. The registry records enrollment of 37 participants. Randomization assigned participants to one of two vorinostat schedules: once daily on days 1-21, or three times daily on days 1-14, with each course repeating every 21 days. Both schedules could be given for up to 17 courses in the absence of disease progression or unacceptable toxicity. The record does not report allocation concealment or other details of the randomization procedure.
The study was unmasked, so participants and study personnel were not blinded to the assigned schedule. The primary outcome was confirmed complete response rate, assessed up to 2 years. The registry describes confirmation using International Working Group criteria for AML response. It specifies that the response criteria must be met for at least 4 weeks and include blood-count and bone-marrow findings, as well as absence of circulating blasts, Auer rods, and extramedullary disease.
The registry lists time to progression, overall survival, and the number of participants with severe adverse events as secondary outcomes. Results for those secondary outcomes are not reported in the registry record described by the posted results. The record identifies the study as completed, with a start date of 2006-01 and primary completion in 2009-05. Additional details about the protocol, recruitment flow, participant characteristics, the randomization sequence, and follow-up completeness are not reported in the registry record.
Because both groups received vorinostat, the design addresses a schedule comparison within the study rather than a comparison with an untreated group or another therapy. The parallel structure means participants were assigned to a schedule rather than crossing between schedules. The study's open-label status is relevant when interpreting outcomes that could involve judgment or reporting, although the registry's response criteria include defined clinical and laboratory features.
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 | Arm A (Once Daily Vorinostat) | Arm B (Thrice Daily Vorinostat) |
|---|---|---|
| STARTED | 15 | 22 |
| COMPLETED | 1 | 4 |
| NOT COMPLETED | 14 | 18 |
Reasons for not completing this period:
| Reason | Arm A (Once Daily Vorinostat) | Arm B (Thrice Daily Vorinostat) |
|---|---|---|
| Death | 3 | 2 |
| Adverse Event | 1 | 2 |
| Alternative Treatment | 4 | 4 |
| Physician Decision | 1 | 1 |
| Withdrawal by Subject | 1 | 4 |
| Disease Progression | 1 | 3 |
| All other reasons | 3 | 2 |
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 | Arm A (Once Daily Vorinostat) | Arm B (Thrice Daily Vorinostat) | Total |
|---|---|---|---|---|
| Participants analysed | 15 | 22 | 37 | |
| Age, Continuous | years | 67 (41 to 79) | 67 (28 to 81) | 67 (28 to 81) |
| Sex: Female, Male: Female | Participants | 5 | 5 | 10 |
| Sex: Female, Male: Male | Participants | 10 | 17 | 27 |
| Region of Enrollment: United States | participants | 15 | 22 | 37 |
| Disease status: Relapsed | participants | 12 | 16 | 28 |
| Disease status: Untreated | participants | 3 | 6 | 9 |
| AML disease classification: Relapsed AML with good risk cytogenetics | participants | 0 | 0 | 0 |
| AML disease classification: All other relapsed AML | participants | 12 | 16 | 28 |
| AML disease classification: Untreated AML patients >= 65 years old | participants | 3 | 3 | 6 |
| AML disease classification: Untreated AML patients with MDS-AML | participants | 0 | 3 | 3 |
| AML disease classification: Untreated AML with >= 3 cytogenetic abnnormalities | participants | 0 | 0 | 0 |
6. Primary Endpoint
The registry lists the primary outcome as Confirmed Complete Response (CR) Rate, measured over Up to 2 years.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | 95% Confidence Interval |
| Unit | percentage of participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint was the confirmed complete response rate, expressed as the percentage of evaluable participants meeting the stated AML response criteria. A complete response required specified blood-count and bone-marrow findings to persist for at least 4 weeks, including no circulating blasts, less than 5% bone marrow blasts, and no extramedullary disease. The registry also lists requirements for neutrophil and platelet counts, marrow cellularity, trilineage maturation, and absence of Auer rods.
A response rate is a binary-outcome summary: each evaluable participant either meets the complete-response definition or does not. It can therefore be expressed as a count of responses relative to the evaluable group and as a percentage. The registry states that the rate was estimated by dividing the number of participants with a complete response by the total number evaluable. The reported arm sizes are 15 and 22; the posted counts indicate no responses in the once-daily arm and one in the thrice-daily arm.
In general, a response endpoint is used to describe whether a prespecified clinical state was reached during the stated assessment period. It can give a direct summary of response occurrence, while not by itself describing how long a response lasted, survival, or the balance of benefits and harms. The endpoint definition makes clear what qualified as a response, but the number evaluable and the handling of participants without an evaluable assessment are not reported in the registry record.
7. Results Posted in the Registry
- Arm B (Thrice Daily Vorinostat): 4.5% (1/22) versus 0.0% (0/15) on control; risk difference 4.5 percentage points (95% CI -4.2 to 13.2); unadjusted two-proportion z-test p = 0.403.
Risk difference: Arm B (Thrice Daily Vorinostat)
95% CI: -4.2 to 13.2 percentage points
Recalculated from the posted summary statistics
8. Statistical Interpretation
The posted complete response rate was 0.0% in the once-daily arm and 4.5% in the thrice-daily arm. The accompanying risk difference for thrice-daily compared with once-daily treatment was 0.045. Its 95% confidence interval ranged from -0.042 to 0.133, and the unadjusted p-value was 0.403. The point estimate is higher for the thrice-daily schedule, but the interval includes zero and also spans effects in either direction. The result is therefore imprecise and does not provide clear evidence of a difference between schedules.
The small number of observed responses is central to interpretation: the posted counts are 0 of 15 in the once-daily arm and 1 of 22 in the thrice-daily arm. With so few events, a single participant's outcome has a large influence on the estimated rate and on the between-arm contrast. The confidence intervals for the arm-specific rates are also broad: 0.0% to 23.0% for once-daily treatment and 0.4% to 24.0% for thrice-daily treatment. These intervals communicate that the observed rates do not pin down the underlying response probabilities precisely.
The unadjusted p-value is a measure of how compatible the observed data are with a specified null comparison under the analysis assumptions; it is not the probability that the null hypothesis is true, nor does it measure clinical importance. The reported value, 0.403, does not establish equivalence or prove that the schedules have the same response rate. A non-significant result can arise when the effect is small, when information is limited, or both. In this trial, the confidence interval and sparse response counts make limited precision especially relevant.
The registry labels the endpoint as a percentage of participants and describes it as a confirmed response rate. It also reports a risk-difference estimate and a confidence interval. No adjusted effect estimate or additional analysis entries are reported in the registry record. The posted comparison should therefore be read as the registry's unadjusted between-arm summary, not as a covariate-adjusted estimate. The record does not report details of the analysis population beyond the description that the denominator consists of evaluable participants.
The comparison is specific to the two schedules, population, follow-up, and endpoint definition recorded in this study. Both groups received vorinostat, so these results do not estimate an effect relative to no vorinostat or to another treatment. The registry lists other outcomes, including time to progression and overall survival, but their results are not reported in the registry record. The response result alone does not summarize those outcomes or the safety profile.
9. Statistical Methods Explained
The primary endpoint is binary at the participant level: an evaluable participant either met the complete-response definition or did not. The arm-specific response rate is the count meeting the definition divided by the number evaluable in that arm. The registry gives the rates as percentages and also posts counts and arm sizes. In general, a binary rate is a straightforward way to describe how often an event was observed in a group, but its precision depends strongly on the amount of information, particularly when the event is rare.
What does the risk difference represent?
The risk difference subtracts the event probability in the reference arm from the event probability in the other arm. It is an absolute contrast on the probability scale: a value of zero corresponds to equal estimated rates, a positive value means a higher estimated rate in the thrice-daily arm, and a negative value means a higher estimated rate in the once-daily arm. The posted estimate is 0.045 for thrice-daily relative to once-daily treatment. The registry's interval for this difference is -0.042 to 0.133. Because zero lies within that interval, the estimates are compatible with no difference as well as with differences in either direction.
How should the confidence intervals be read?
A confidence interval is a range of values produced by a statistical procedure that, over repeated applications under its assumptions, has the stated coverage. In practical interpretation, it shows which effect sizes are reasonably compatible with the data and method. It is not a guarantee that the true value lies in the particular interval, and it is not a range containing a stated percentage of individual participants. The arm-specific intervals are 0.0% to 23.0% and 0.4% to 24.0%; the risk-difference interval is -0.042 to 0.133. Their breadth reflects uncertainty in estimates based on few observed responses.
What does the p-value add?
The reported p-value is unadjusted and equals 0.403. In general, a p-value measures how unusual data at least as incompatible with a null model would be if that model and the analysis assumptions held. It does not give the probability that the null is true, the probability that chance alone caused the result, or the size of an effect. The p-value and confidence interval address related questions, but the interval is more informative about the range of effect sizes that remain compatible with the data. Neither establishes that the schedules are equivalent.
Why do sparse outcomes matter?
When outcomes are uncommon, a small number of events can dominate the estimated rate. Here, the posted response counts are 0 in the once-daily arm and 1 in the thrice-daily arm. That limited event information produces uncertainty even though the arm-specific percentages and the difference can be stated precisely to the displayed digits. Precision in the printed decimal places should not be confused with certainty about the underlying response rates.
In general, methods for binary outcomes can use different interval procedures, especially when event counts are small or an arm has no events. The registry record identifies the confidence interval but does not report the specific procedure used to calculate it. The statistical model, any continuity correction, and any other details of the primary analysis are not reported in the registry record. They should not be inferred from the displayed values alone.
What do the planning illustrations mean?
Sample-size illustrations based on an observed effect are planning exercises, not observed trial findings and not a measure of the probability that this study detected an effect. The stated planning approach uses a two-sided significance level of 0.05, equal allocation between arms, and a normal approximation; the effect to detect is expressed as a fraction of the observed effect. Such calculations are hypothetical and depend on their assumptions. They do not establish that the original trial was designed with those assumptions. The sample-size justification and the original design assumptions are not reported in the registry record.
For binary outcomes, planning estimates depend on the assumed event rates and the effect considered important to detect. If the observed effect is used as the planning basis, uncertainty in that observed effect carries into the illustration. A different target effect would yield a different planning requirement. These illustrations should therefore be interpreted as conditional planning scenarios, rather than evidence about the adequacy of the study or the observed response result.
The record reports no analysis entries. It does not report whether there were interim analyses, a prespecified statistical analysis plan, adjustment for baseline covariates, or a plan for handling missing outcome assessments. Those features cannot be reconstructed from the endpoint estimates. The term “unadjusted” applies to the posted p-value and should not be taken to imply any unreported details about the full analysis process.
10. Confidence Intervals and Precision
The response-rate estimates are based on few observed complete responses: no responses were posted for the once-daily arm and one for the thrice-daily arm. The registry reports 95% confidence intervals of 0.0% to 23.0% and 0.4% to 24.0%, respectively. These ranges are wide relative to the point estimates, indicating that the observed data do not locate either arm's underlying response rate with high precision.
For the between-arm comparison, the risk difference is 0.045, with a 95% confidence interval from -0.042 to 0.133. The interval crosses zero, leaving open the possibility of a lower response rate in the thrice-daily arm, no difference, or a higher rate. It also includes differences appreciably larger than the observed point estimate. The data therefore do not tightly distinguish among these possibilities.
In general, confidence intervals convey uncertainty about an estimated quantity under the chosen statistical method and its assumptions. Wider intervals indicate less precision; they do not mean the estimate is invalid. With sparse events, intervals can remain broad even when the reported estimates are given to several decimal places. The digits in the point estimate are a format, not a claim that the underlying effect is known to that degree.
The record does not report the interval-calculation method, and the evaluable denominator is described but not separately detailed. These gaps limit independent assessment of how the interval was constructed and how participants without an evaluable response were handled. The observed rates and their intervals should be interpreted as estimates for the registry's defined analysis, with those limitations in mind.
11. Multiplicity, Missing Data and Analysis Populations
The registry lists three secondary outcomes: time to progression, overall survival, and the number of participants with severe adverse events. Their results are not reported in the registry record. The primary response result is therefore the only efficacy estimate. The record does not report whether a formal multiplicity strategy was prespecified to account for testing multiple outcomes, comparisons, or analyses.
In general, when several outcomes or hypotheses are examined, the chance of obtaining at least one apparently unusual result can increase unless the analysis plan accounts for that multiplicity. That concern cannot be quantified for this study from the registry information. No analysis entries are reported, and the registry record does not describe interim analyses, outcome hierarchy, or any adjustment procedure.
The endpoint definition states that the response rate uses the number evaluable as its denominator. Arm sizes are reported as 15 and 22, but the record does not separately state whether these are the evaluable denominators or describe any exclusions from the response analysis. It also does not report the number of participants with missing response assessments, the reasons for missingness, or how missing outcomes were treated. Consequently, the effect of missing response data on the estimates cannot be assessed from the registry record.
The record does not provide enough information to characterize the primary analysis population as intention-to-treat, per-protocol, or another defined group. Nor does it report sensitivity analyses for missing outcomes. These omissions matter because the interpretation of a rate can depend on who was included in its denominator and how unavailable assessments were handled. They do not change the posted estimates, but they limit evaluation of their robustness.
12. 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 |
|---|---|---|---|
| Arm A (Once Daily Vorinostat) | 10 | 15 | 66.7% |
| Arm B (Thrice Daily Vorinostat) | 16 | 22 | 72.7% |
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 |
|---|---|
| Arm A (Once Daily Vorinostat) | 14 of 15 |
| Arm B (Thrice Daily Vorinostat) | 21 of 22 |
Most frequent serious adverse event terms
| Event term | Organ system | Arm A (Once Daily Vorinostat) | Arm B (Thrice Daily Vorinostat) |
|---|---|---|---|
| Fatigue | General disorders | 3 of 15 | 7 of 22 |
| Platelet count decreased | Investigations | 1 of 15 | 4 of 22 |
| Febrile neutropenia | Blood and lymphatic system disorders | 1 of 15 | 3 of 22 |
| Diarrhea | Gastrointestinal disorders | 0 of 15 | 4 of 22 |
| Nausea | Gastrointestinal disorders | 1 of 15 | 3 of 22 |
| Vomiting | Gastrointestinal disorders | 1 of 15 | 3 of 22 |
| Pneumonia | Infections and infestations | 1 of 15 | 2 of 22 |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | 1 of 15 | 2 of 22 |
| Blood disorder | Blood and lymphatic system disorders | 2 of 15 | 0 of 22 |
| Hemoglobin decreased | Blood and lymphatic system disorders | 0 of 15 | 2 of 22 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Arm A (Once Daily Vorinostat) | Arm B (Thrice Daily Vorinostat) |
|---|---|---|---|
| Platelet count decreased | Investigations | 14 of 15 | 19 of 22 |
| Neutrophil count decreased | Investigations | 13 of 15 | 19 of 22 |
| Fatigue | General disorders | 12 of 15 | 15 of 22 |
| Nausea | Gastrointestinal disorders | 6 of 15 | 13 of 22 |
| Diarrhea | Gastrointestinal disorders | 3 of 15 | 11 of 22 |
| Hemoglobin decreased | Blood and lymphatic system disorders | 7 of 15 | 6 of 22 |
| Anorexia | Metabolism and nutrition disorders | 4 of 15 | 9 of 22 |
| Dyspnea | Respiratory, thoracic and mediastinal disorders | 6 of 15 | 7 of 22 |
| Leukocyte count decreased | Investigations | 6 of 15 | 4 of 22 |
| Vomiting | Gastrointestinal disorders | 1 of 15 | 7 of 22 |
Serious events with the highest arm-level rate: fatigue (31.8%), diarrhea (18.2%), platelet count decreased (18.2%), febrile neutropenia (13.6%), nausea (13.6%).
The registry reports serious adverse events for 10 of 15 participants in the once-daily arm and 16 of 22 in the thrice-daily arm. It also reports other adverse events for 14 of 15 and 21 of 22 participants, respectively. These are participant counts, not counts of all individual episodes. A participant may experience more than one event, so a participant-level summary does not describe the total event burden or timing.
The listed serious adverse event terms include fatigue at 31.8%, diarrhea at 18.2%, and platelet count decreased at 18.2%, as well as febrile neutropenia, nausea, and vomiting at 13.6% each. The registry record does not provide further detail in the reported details about event duration, severity by term, attribution, or recurrence. Death counts are not reported in the registry record.
In general, adverse-event percentages describe the participants counted under the registry's reporting rules; they do not alone establish that an event was caused by a study drug or that the groups differ in safety. The arms also differ in size, so raw counts should be read alongside their denominators. No comparative safety estimate or statistical test is reported in the registry record. The serious-event summaries should be considered separately from the primary response endpoint.
13. What the Result Does, and Does Not, Mean
The registry results describe a randomized comparison of two vorinostat schedules in acute myeloid leukemia. The posted confirmed complete response rates are 0.0% for once-daily dosing and 4.5% for thrice-daily dosing. The estimated risk difference is 0.045, with a 95% confidence interval from -0.042 to 0.133 and an unadjusted p-value of 0.403. The observed response count was zero in the once-daily arm and one in the thrice-daily arm.
These data do not establish that the schedules have the same response rate, because the confidence interval remains compatible with differences in either direction. They also do not establish that the thrice-daily schedule has a higher response rate: the estimate is based on one observed response, the interval includes zero, and the p-value does not indicate a clear between-arm difference. The appropriate statistical reading is uncertainty, not proof of equivalence or proof of a schedule effect.
The result is limited to the primary endpoint and the two vorinostat schedules studied. It does not compare vorinostat with another drug or with no treatment. The registry lists time to progression and overall survival as secondary outcomes, but their results are not reported in the registry record. Likewise, the response endpoint does not describe response duration, long-term disease control, or overall survival.
The safety summaries report serious adverse events in both arms, but the posted counts do not by themselves establish a causal relationship or comparative safety. The registry does not report a comparative safety analysis in the registry record described. Treatment decisions require clinical context and evidence beyond this statistical summary; no treatment recommendation follows from these results alone.
More broadly, the estimates are shaped by the study's limited number of observed responses, its endpoint definition, and the information recorded in the registry. Details about the analysis population, missing assessments, and statistical procedures are not reported in the registry record. These considerations constrain how confidently the numerical findings can be generalized beyond the trial population and setting.
14. Why This Trial Matters Statistically
This trial illustrates a recurring issue in clinical research: a randomized design can yield a highly uncertain effect estimate when the primary outcome is rare and few events occur. Randomization structures the comparison between schedules, but it does not guarantee precision. The complete response result rests on no observed responses in one arm and one in the other, so a small change in event count would materially affect the estimated rates.
The contrast between the point estimate and its interval is instructive. The risk difference is positive, but its 95% confidence interval includes zero and extends in both directions. A reader should therefore avoid treating the point estimate alone as a settled description of the underlying difference. The interval communicates that the result is compatible with a range of effects, including no difference.
The registry also demonstrates why a p-value should not be used as a stand-alone verdict. The unadjusted p-value of 0.403 does not prove that schedules are equivalent, and it does not quantify the clinical value of either schedule. The confidence interval and event counts make the limits of the evidence more visible.
Finally, the record distinguishes an endpoint result from the broader clinical questions a trial may raise. Time to progression, overall survival, and severe adverse events are listed as secondary outcomes, but their results are not reported in the registry record. This makes clear that an isolated response-rate comparison cannot answer every question about outcomes or safety. Interpreting clinical trials requires attention to the endpoint, the precision of its estimate, the population analyzed, and the outcomes for which results are actually available.
15. 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.
- Participant counts were reconstructed from posted percentages and arm sizes, so small rounding differences are possible.
The primary estimate is based on very few complete responses, which limits precision and makes the observed arm rates sensitive to individual outcomes. The registry reports the arm sizes and response counts, but it does not separately describe the evaluable denominators or provide a participant flow showing exclusions and missing response assessments. The effect of missingness on the response estimate therefore cannot be assessed.
The record does not report the method used to calculate the confidence intervals, the full statistical analysis plan, or whether there were prespecified interim analyses. It reports no analysis entries. Details about the randomization procedure, allocation concealment, baseline balance, and analysis population are also not reported in the registry record. The open-label design is known, but the available record does not explain whether outcome assessment was blinded.
The study tested two schedules of the same drug. It does not provide a comparison with a different treatment or an untreated group. The listed secondary outcomes include time to progression and overall survival, but their results are not reported in the registry record. Thus, the response finding cannot be extended to those outcomes, and the available adverse-event summaries do not provide a full comparative safety analysis. Death counts are not reported in the registry record.
The registry lists a broad set of AML conditions, but detailed participant characteristics and subgroup results are not reported in the registry record. The extent to which the enrolled participants represent particular AML subtypes or disease settings cannot be assessed from the information available. In general, generalizability depends on the characteristics of those enrolled and the setting in which the study was conducted.
Finally, planning illustrations based on an observed effect are not evidence of the original design rationale or of observed power. The sample-size justification is not reported in the registry record. All these limitations affect how much can be concluded from the posted estimates, without changing the values themselves.
16. Related Tutorials
17. Related Statistical Calculators
18. Sources
- ClinicalTrials.gov: Vorinostat in Treating Patients With Acute Myeloid Leukemia — NCT00305773.
- Registry record last updated 2014-05-19; status last verified 2012-12.
- Results first posted 2013-05-16.
- 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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19. Record Summary
NCT00305773 is a completed, randomized, open-label, parallel-group phase II study sponsored by the National Cancer Institute. It enrolled 37 participants and compared once-daily with thrice-daily oral vorinostat schedules in acute myeloid leukemia. The primary outcome was confirmed complete response rate, assessed up to 2 years.
The posted rates were 0.0% for once-daily treatment and 4.5% for thrice-daily treatment. The risk difference was 0.045, with a 95% confidence interval from -0.042 to 0.133 and an unadjusted p-value of 0.403. The result is imprecise, with one response in the thrice-daily arm and none in the once-daily arm.
The registry lists time to progression, overall survival, and severe adverse events as secondary outcomes. Results for the efficacy secondary outcomes are not reported in the registry record. Serious adverse events were reported for 10 of 15 participants in the once-daily arm and 16 of 22 in the thrice-daily arm. Key analysis details, including the handling of missing response assessments, are not reported in the registry record.