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 After Stem Cell Transplant in Treating Patients With High-Risk Lymphoma (NCT00561418), a na phase 1 study. The primary outcome is Safety and Tolerability of Vorinostat (SAHA) After Autologous Stem Cell Transplantation.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00561418 |
| Phase | Phase 1 |
| Condition | Lymphoma, Small Intestine Cancer |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 23 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Ohio State University Comprehensive Cancer Center |
| Results posted | Yes |
2. Clinical Question
Population
Patients with high-risk lymphoma after autologous hematopoietic stem cell transplantation were the intended population. The registry lists lymphoma and small intestine cancer as conditions, but does not report further eligibility details or describe how many participants had each condition.
Intervention
Vorinostat, also called SAHA, was administered orally. The regimen was to begin approximately day +60 after transplantation, with 21 consecutive treatment days in each 28-day cycle, for up to 11 cycles. The description also refers to dose escalations.
Comparator
There was no separate comparator group. The study is recorded as a single-group trial with allocation listed as not applicable. Its results therefore do not provide a randomized, like-for-like comparison with another treatment or with no treatment.
Primary question
The primary outcome concerns the safety and tolerability of vorinostat after autologous transplantation, assessed through adverse events. The registry describes a phase I study of side effects and dose, with assessment over up to 3 years. A numerical dose-selection rule is not reported.
The study was sponsored by Ohio State University Comprehensive Cancer Center and enrolled 23 participants. It is marked completed. The registry lists clinical benefit, duration of response, and time to progression as secondary outcomes, but numerical results for these outcomes are not reported in the registry record. The central statistical question is therefore descriptive: what adverse-event counts were recorded among participants in the vorinostat group? The record does not support estimating a treatment difference against a control group, nor does it provide enough outcome information to characterize benefit or time-to-event patterns.
3. Trial Design
This was a phase I, single-group study of vorinostat after autologous hematopoietic stem cell transplantation for high-risk lymphoma. The registry lists 23 participants as enrolled and marks the study completed. The sponsor was Ohio State University Comprehensive Cancer Center. The recorded study start is 2007-11, and primary completion is recorded as 2012-11.
Participants received oral vorinostat according to a regimen beginning approximately day +60 after transplantation. Treatment was scheduled for 21 consecutive days of a 28-day cycle, for up to 11 cycles. The arm description mentions dose escalations, but the escalation sequence, dose levels, and rules for moving between levels are not reported in the registry record. The intervention list also includes correlative studies; their procedures and analysis are not described in detail.
The design is recorded as single group, with allocation not applicable and masking none. There is no separate control arm. Consequently, the study can describe events observed in participants receiving the regimen, but it cannot make a randomized comparison with another group. Differences from an external population would not be controlled by this design.
The stated primary outcome is safety and tolerability, expressed as a number of patients, with assessment over up to 3 years. The registry identifies adverse-event grading using NCI CTCAE version 3.0. Clinical benefit, duration of response, and time to progression are listed as secondary outcomes. Numerical results for those outcomes are not reported in the registry record. A formal statistical analysis plan, detailed eligibility criteria, analysis populations, and sample-size justification are also 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 | Vorinostat (SAHA) |
|---|---|
| STARTED | 23 |
| COMPLETED | 23 |
| NOT COMPLETED | 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 | Vorinostat (SAHA) |
|---|---|---|
| Participants analysed | 23 | |
| Age, Continuous | years | 55 (25 to 75) |
| Sex: Female, Male: Female | Participants | 4 |
| Sex: Female, Male: Male | Participants | 19 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 23 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 1 |
| Race (NIH/OMB): White | Participants | 22 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Region of Enrollment: United States | patients | 23 |
6. Primary Endpoint
The registry lists the primary outcome as Safety and Tolerability of Vorinostat (SAHA) After Autologous Stem Cell Transplantation, measured over Up to 3 years.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | patients |
| Endpoint classification | Binary Endpoint |
The primary outcome is safety and tolerability of vorinostat after autologous stem cell transplantation. It is expressed as a number of patients and concerns adverse events assessed over up to 3 years. The registry identifies NCI CTCAE version 3.0 as the grading system: Grade 1 is mild, Grade 2 moderate, Grade 3 severe, and Grade 4 life-threatening or disabling.
In a phase I study, systematic adverse-event assessment is used to describe tolerability and inform evaluation of dose. The registry’s stated purpose includes studying side effects and the best dose. However, it does not report a specific rule for deciding which dose to select, such as a defined event threshold, nor does it provide dose-level results in the record. The safety counts should therefore be read as descriptive group-level observations rather than as a complete dose-selection analysis.
The record does not define the exact adverse-event set included in the primary outcome, explain whether counts refer to participants with at least one event or to a narrower category, or give a detailed timing rule beyond the stated timeframe. The reported serious and other adverse-event counts provide some safety information, but do not describe severity grades, event types, recurrence, or exposure at each dose. Those details are not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry reports 23 participants at risk for serious adverse events in the vorinostat group, with 0 affected. For other adverse events, 23 were at risk and 12 were affected. These are counts within a single treatment group. They describe what was recorded among the enrolled group; they do not estimate a difference between vorinostat and a comparator, because no comparator group was included.
The serious-event count is a point-in-time summary over the reported study outcome, not proof that serious events are impossible or that the true event risk is zero. With a group of this size, an observed count can be informative about what occurred in the trial while remaining imprecise about the range of event rates that might occur in a broader population. The registry does not report confidence intervals for these counts. It also does not report event-specific serious adverse-event terms, and no top serious terms are listed.
The count of 12 affected by other adverse events indicates that these events were recorded in the group. It does not, by itself, show the number, duration, grade, or clinical importance of individual events. Nor does it establish that the events were caused by vorinostat. The record does not provide event-level detail, dose-specific safety summaries, or a comparison that could help distinguish treatment-associated events from other causes.
The study’s primary focus is safety and tolerability, not a randomized estimate of efficacy. Although clinical benefit, duration of response, and time to progression are named as secondary outcomes, numerical results for them are not reported in the registry record. No inferential analyses are listed. As a result, the posted record does not support a quantitative conclusion about those outcomes.
Planning illustrations based on an observed effect should not be interpreted as observed power or as the trial’s original design justification. The stated illustration assumptions include a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. Those assumptions describe a hypothetical comparison and do not match the recorded single-group design. The registry does not report the trial’s own sample-size justification. Any interpretation should keep the distinction clear: the observed safety counts are descriptive, while planning calculations are hypothetical and are not additional clinical findings.
9. Statistical Methods Explained
The study is described as phase I and single group. This design determines what statistical conclusions can be drawn. A single-group study can count participants with recorded events and describe treatment exposure as documented. Without a concurrent comparator, it cannot isolate a treatment effect from differences in patient characteristics, disease course, supportive care, or other influences. General statistical interpretation therefore emphasizes counts and their context rather than comparative estimates.
What do the adverse-event counts represent?
The registry gives the number at risk and the number affected for serious adverse events and other adverse events. These are participant counts, not event totals: a participant may have more than one event, but the record does not give the number of events per person. It also does not state whether the categories are mutually exclusive, describe repeat events, or provide event-level listings. The reported values are best read as the number of participants recorded as affected in each category.
The primary outcome is expressed as a number of patients. This is a descriptive measure. A count can be useful for showing how many participants experienced a recorded outcome, but its interpretation depends on the group size, length of follow-up, outcome definition, and completeness of observation. The registry states a timeframe of up to 3 years, but does not give person-time, individual follow-up duration, or the handling of participants who stopped treatment or left follow-up early.
Why is uncertainty relevant?
A confidence interval is a general statistical tool for expressing uncertainty around an estimate. For a participant count or event proportion, an interval can indicate how much the estimate might vary because the trial includes a limited number of participants. A count of zero does not imply that the underlying chance of an event is exactly zero. In this record, confidence intervals are not reported, so readers cannot use posted interval estimates to judge the precision of the safety counts. General uncertainty principles remain relevant even when only counts are available.
In a comparative trial, an effect estimate might contrast event risks in two arms. Here, there is only one arm, so such a contrast cannot be obtained from the study design. General principles for comparative inference, including adjustment for baseline imbalances, do not create a missing control group. Nor can statistical adjustment substitute for a randomized comparison when the necessary participant-level information and a comparison group are absent.
What does the grading system add?
The registry identifies NCI CTCAE version 3.0 and defines Grades 1 through 4, ranging from mild to life-threatening or disabling. Grading provides a framework for classifying event severity. The recorded safety summary, however, does not show how many participants had events at each grade or identify the particular event types. Therefore, the grading framework is known, but grade-specific results are not reported in the registry record. A general severity scale cannot be used to infer the distribution of observed grades when those counts are absent.
How should the planning assumptions be read?
The sample-size illustrations are described as planning calculations based on an observed effect, using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. For time-to-event outcomes, the stated convention is a total number of events; for other outcomes, it is participants per arm. These are general planning conventions, not results from the treatment group. The illustrations are not observed power and are not evidence that the trial had a particular probability of detecting an effect.
The assumed equal allocation between two arms does not describe this study, which is recorded as single group. Thus, the illustrations should not be mistaken for a reconstruction of the actual enrollment plan or a claim about how this phase I study was designed. The registry does not report its own sample-size justification, the effect size targeted in its original planning, or a formal statistical plan. Without those details, the planning assumptions cannot establish whether enrollment was adequate for a particular safety or efficacy question.
What is not available for interpretation?
The record lists no analyses. It does not report inferential estimates, confidence intervals, p-values, or numerical results for the secondary outcomes. This absence limits statistical assessment: it is not possible to evaluate the precision of secondary outcome estimates, the handling of time-to-event censoring, or any adjusted analysis from the registry information. General statistical concepts can clarify what such methods would mean, but they cannot fill in unreported analyses or results.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists a primary safety and tolerability outcome and three secondary outcomes: clinical benefit, duration of response, and time to progression. Considering several outcomes can raise a multiplicity issue because, in general, the chance of at least one apparently unusual result can increase when many statistical tests are performed. That concern is usually addressed through a prespecified hierarchy, adjustment procedure, or a clear distinction between confirmatory and exploratory analyses. No multiplicity procedure or outcome-testing hierarchy is reported in the registry record. Numerical results for the secondary outcomes are also not reported, so their statistical evidence cannot be assessed.
The registry lists no analyses. It does not report an analysis population, such as whether all enrolled participants or only participants who began treatment were included in each outcome summary. The number enrolled is 23, but the record does not provide a participant flow, treatment exposure by person, or outcome-specific denominators beyond the safety counts. The handling of withdrawals, missed assessments, and incomplete follow-up is not reported in the registry record.
Missing-data methods are also not reported. In general, incomplete outcome information can affect descriptive counts and time-to-event estimates, and methods depend on why observations are missing and what information remains available. No assumptions or sensitivity analyses for missing data are reported. The stated primary timeframe is up to 3 years, but the record does not explain how participants with shorter observation were handled.
The record does not report interim analyses, stopping rules, a formal statistical analysis plan, or a sample-size justification. These omissions do not establish that no such procedures existed; they mean that they cannot be evaluated from the registry record. Given the single-group design and absence of posted analyses, the most direct statistical information available is the reported adverse-event counts.
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 |
|---|---|---|---|
| Vorinostat (SAHA) | 0 | 23 | 0.0% |
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 |
|---|---|
| Vorinostat (SAHA) | 12 of 23 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Vorinostat (SAHA) |
|---|---|---|
| fatigue | Investigations | 12 of 23 |
| Lymphopenia | Investigations | 11 of 23 |
| Thrombocytopenia | Injury, poisoning and procedural complications | 11 of 23 |
| Leukopenia | Investigations | 10 of 23 |
| anemia | Blood and lymphatic system disorders | 10 of 23 |
For serious adverse events, the registry reports 23 participants at risk and 0 affected in the vorinostat group. For other adverse events, it reports 23 at risk and 12 affected. These are participant counts. They should not be read as numbers of individual event episodes, because a participant may have experienced more than one event and event totals are not reported.
The record provides no top serious-event terms, and it does not give event names, grade-specific counts, timing, duration, or dose-level breakdowns. Although the primary outcome description identifies NCI CTCAE version 3.0, the safety summary does not show the distribution across its severity grades. The registry’s stated assessment timeframe is up to 3 years; individual follow-up durations and exposure time are not reported.
The zero serious-event count means that no affected participants are recorded in that category for this group. It is not proof that the underlying risk is zero. The other-event count likewise does not by itself establish causation, severity, or clinical importance. Without a comparator group, these counts cannot show whether event frequency differed from what might occur without vorinostat or under another regimen. Confidence intervals and event-level analyses are not reported in the registry record.
12. What the Result Does, and Does Not, Mean
The record describes a phase I study in which 23 participants were enrolled into a single vorinostat group after autologous stem cell transplantation. Its primary focus was safety and tolerability, assessed through adverse events over up to 3 years. The posted safety counts are 0 participants affected by serious adverse events among 23 at risk and 12 affected by other adverse events among 23 at risk. Those are the available numerical safety findings in the registry record.
These observations do not establish that vorinostat caused or prevented an adverse event. The study has no separate control group, and the record does not provide event-level detail, dose-level counts, or uncertainty intervals. The absence of a recorded serious adverse event in this group should not be interpreted as proof that serious events cannot occur. The counts also cannot characterize the frequency of all possible events outside the observed group or follow-up.
The registry identifies clinical benefit, duration of response, and time to progression as secondary outcomes, but numerical results for them are not reported. Consequently, the posted information does not quantify those outcomes or show how they compare with another treatment. A safety-focused phase I record should not be treated as a comparative efficacy study, and no conclusion about clinical benefit follows from the event counts alone.
The record also does not establish the best dose. Although the brief purpose refers to studying dose and the regimen description mentions dose escalations, dose-specific findings and a selection rule are not reported. Nor are the statistical plan, analysis populations, missing-data methods, or original sample-size justification reported. These gaps constrain assessment of both safety precision and the intended role of any secondary analyses.
In short, the posted results describe recorded adverse-event counts in one group. They are useful as a limited account of what was observed in this study, but they do not provide a randomized treatment comparison, a full safety profile, or numerical evidence on the listed secondary outcomes.
13. Why This Trial Matters Statistically
This study illustrates why design and outcome type matter when reading clinical trial results. A phase I, single-group study can provide descriptive safety information during treatment and follow-up. It cannot, by design, provide a randomized contrast with a control group. The recorded safety counts therefore answer a narrower question than a comparative trial would: how many participants in this group were recorded as affected, rather than how the treatment changed event risk relative to another option.
The primary outcome is expressed as a number of patients, while the registry also names response and time-to-progression outcomes. Those outcome types require different summaries and, for time-to-event outcomes, attention to follow-up and censoring. The record does not report numerical analyses for the secondary outcomes, so their presence in the outcome list should not be mistaken for posted evidence about them. The distinction between an outcome being named and an outcome being analyzed is important for transparent interpretation.
The safety information also shows why counts need context. A zero count in a group is an observation, not a guarantee about future risk; an affected-participant count does not communicate event severity or recurrence without further detail. Grading criteria are identified, but grade-specific results are absent. That difference between a defined measurement framework and a complete report of its results is central to evaluating what the registry can support.
Finally, planning illustrations are not equivalent to the actual design rationale. The stated illustration assumptions include two-arm equal allocation even though the study is recorded as single group. Keeping planning calculations separate from observed outcomes avoids implying that the trial had a comparator or a particular level of power. The registry does not report its original sample-size justification. This is a useful reminder that statistical interpretation depends on both the available numbers and the design that generated them.
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 main design limitation is the absence of a control group. As a single-group study, it can report events in the vorinostat group but cannot estimate a randomized difference against another treatment or against no treatment. Masking is listed as none, and allocation is not applicable. These design features limit comparative interpretation, especially for outcomes that may be influenced by disease characteristics, transplant recovery, concomitant care, or follow-up practices.
The enrollment was 23, and the record does not report confidence intervals for the adverse-event counts. The precision of the safety findings therefore cannot be evaluated through posted interval estimates. The primary outcome is described as a patient count over up to 3 years, but person-time and individual follow-up durations are not given. It is not possible to assess how observation time varied across participants.
Safety reporting is limited to counts of participants affected by serious and other adverse events. No serious-event terms are listed, and event types, grades, timing, recurrence, and dose-level results are not reported. Although the grading system is specified, grade-specific findings are absent. This limits assessment of the nature and severity of the events and the relationship, if any, to dose or treatment exposure.
The record lists secondary outcomes but reports no numerical results for them, and it lists no analyses. Statistical methods, analysis populations, missing-data handling, interim procedures, stopping rules, and a sample-size justification are not reported in the registry record. The dose-escalation description lacks dose levels and decision rules. These omissions prevent a fuller evaluation of outcome completeness, analysis choices, and dose selection.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Vorinostat After Stem Cell Transplant in Treating Patients With High-Risk Lymphoma — NCT00561418.
- Registry record last updated 2015-07-16; status last verified 2015-07.
- Results first posted 2015-07-16.
- 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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18. Record Summary
NCT00561418 is a completed phase I, single-group study sponsored by Ohio State University Comprehensive Cancer Center. It enrolled 23 participants and evaluated oral vorinostat after autologous hematopoietic stem cell transplantation for high-risk lymphoma. The regimen was scheduled to begin approximately day +60 after transplantation, with 21 treatment days per 28-day cycle for up to 11 cycles, with dose escalations.
The primary outcome was safety and tolerability over up to 3 years, assessed using NCI CTCAE version 3.0. The registry reports 0 participants affected by serious adverse events among 23 at risk and 12 affected by other adverse events among 23 at risk. Clinical benefit, duration of response, and time to progression are listed as secondary outcomes, but numerical results are not reported in the registry record.
The study had no comparator group. The registry does not report analyses, a formal statistical plan, missing-data methods, analysis populations, dose-selection rules, or the original sample-size justification.