Belinostat and Carboplatin in Treating Patients With Recurrent or Persistent Ovarian Epithelial Cancer, Fallopian Tube Cancer, or Primary Peritoneal Cancer That Did Not Respond to Carboplatin or Cisplatin: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Belinostat and Carboplatin in Treating Patients With Recurrent or Persistent Ovarian Epithelial Cancer, Fallopian Tube Cancer, or Primary Peritoneal Cancer That Did Not Respond to Carboplatin or Cisplatin (NCT00993616), a na phase 2 study. The primary outcome is Frequency and Severity of Observed Adverse Effects Graded According to NCI CTCAE Version 3.0.

2. Clinical Question

3. Trial Design

This was a phase II, single-group trial sponsored by the National Cancer Institute. The registry describes 29 participants enrolled and identifies one treatment arm receiving belinostat and carboplatin. Allocation is listed as not applicable, and masking as none. There is no concurrent control arm in the record, so the design is suited to describing outcomes among treated participants rather than making a randomized comparison between groups.

The treatment schedule specified intravenous belinostat over 30 minutes on days 1-5 and intravenous carboplatin over 30-60 minutes on day 3. Courses were scheduled every 21 days, for up to 6 courses, unless disease progression or unacceptable toxicity occurred. The arm description also mentions continued treatment for patients who were clinically responding or who, in the opinion of their physic… The remainder of that statement is not reported in the registry record, so its complete criteria cannot be described.

The trial began in 2009-12 and reached primary completion in 2012-07. Its status is completed. The registry record does not report the rationale for the enrollment target or a sample-size justification. It also does not report an interim-analysis plan or a detailed statistical analysis plan. These omissions matter when interpreting a phase II record: the design label and enrollment alone do not reveal what decision thresholds or precision targets investigators intended to use.

The reported adverse-event population is eligible and treated patients, with 27 listed at risk for serious adverse events and 27 listed at risk for other adverse events. This is distinct from the total enrollment of 29. The registry record does not explain the difference between those counts, so no explanation for participant inclusion or exclusion from the safety summaries can be inferred.

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

Reasons for not completing this period:

5. Baseline Characteristics

The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.

Population: Number of eligible and evaluable participants

6. Primary Endpoint

The registry lists the primary outcome as Frequency and Severity of Observed Adverse Effects Graded According to NCI CTCAE Version 3.0, measured over Every cycle during treatment and 30 days after the end of treatment.

The primary endpoint is the frequency and severity of observed adverse effects, graded according to NCI CTCAE Version 3.0. The unit is participants, and the stated timeframe is every cycle during treatment and 30 days after the end of treatment. In other words, the outcome concerns how many participants experienced adverse effects and how severe those effects were under the registry’s grading system. It is not a time-to-event outcome or a reported measure of tumor response.

Safety outcomes are used in clinical research to describe the types and severity of unwanted events observed during treatment and a stated follow-up period. A grading framework helps organize severity consistently, while the participant unit communicates how many people were affected rather than how many event episodes occurred. In general, such summaries help readers understand what was observed in the treated group; by themselves, they do not establish that treatment caused an event.

The registry record reports adverse-event summaries, including serious adverse events and other adverse events. It does not report a detailed breakdown of all grades, the event-level data, or the full endpoint description beyond the stated title and timeframe. It also does not report an efficacy endpoint result. The safety endpoint therefore supports a descriptive reading of the posted adverse-event counts, not a conclusion about comparative safety or cancer outcomes.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry identifies the study as phase II and single group, with allocation listed as not applicable and masking as none. These design features determine what kinds of statistical conclusions are available. Since there is no concurrent control group, the record can describe the experience of participants who received the regimen but cannot estimate a randomized between-group treatment contrast.

What does a single-group design show?

In general, a single-group study records outcomes among participants receiving a treatment regimen. It can describe observed event counts and, when reported, response or follow-up patterns. Without a comparison group, however, changes or events cannot be separated statistically from other influences such as disease course, participant characteristics, or background event rates. This is why descriptive counts should not be expressed as evidence that the regimen caused or prevented a particular outcome.

The record reports 29 enrolled participants, while the safety summaries list 27 at risk for serious adverse events and 27 at risk for other adverse events. Those denominators belong to the posted safety summaries. The reason for the difference between enrollment and the at-risk groups is not reported in the registry record. A statistical analysis population should be defined by its inclusion rules, and those rules are not described in enough detail to reconstruct them.

How should participant counts be read?

The outcome unit is participants. A participant count tells how many people were recorded as affected, not how many separate episodes occurred. A person can experience more than one type of event, and the registry record does not provide event-level details that would show overlap among terms. For that reason, the serious-event counts and term percentages should not be combined or treated as mutually exclusive categories.

In general, a denominator gives the number of participants considered at risk for the summary, while the affected count gives the number with the event category recorded. Here, the registry lists 27 at risk for each of the serious and other adverse-event summaries. It does not provide enough detail to infer how the categories were operationalized, whether participants could appear in both, or how the specific term percentages were derived. The stated values should therefore be read as registry summaries rather than recalculated from assumptions about the underlying data.

What does adverse-event grading contribute?

The primary outcome says adverse effects were graded according to NCI CTCAE Version 3.0. In general, a common grading system provides standardized language for describing severity across events and participants. It helps distinguish severity categories rather than treating every recorded event as equivalent. The registry entries in the registry record do not include a complete severity distribution, so the grading reference alone does not show how many events fell into each grade. The record also does not report the individual records needed to examine event onset, duration, recurrence, or overlap.

The timeframe spans every cycle during treatment and 30 days after treatment ended. In general, repeated safety monitoring can capture events at different points during exposure and shortly after treatment. The registry record gives the observation schedule in broad terms but does not report cycle-specific counts or a more detailed follow-up schedule. It is therefore not possible to assess how event occurrence varied over time from the listed summaries.

Are there inferential results?

No analyses are listed in the registry record. There are no reported p-values, confidence intervals, adjusted estimates, or model results in the registry record describing this trial. In general, a p-value measures how compatible observed data are with a specified null hypothesis under a statistical model; it does not measure the size or clinical importance of an effect. A confidence interval communicates uncertainty around an estimate under the chosen model. Neither can be interpreted when no corresponding analysis is reported.

The record includes a description of planning illustrations based on an observed effect, using a two-sided significance level of 0.05, equal allocation between two hypothetical arms, and a normal approximation. Such illustrations are not the trial’s own design justification and are not observed power. They do not turn this single-group study into a two-arm comparison. The record does not report the trial’s sample-size justification or a statistical plan that would clarify its planned inferential approach.

In general, over-dispersion occurs when variation in outcome data exceeds what a statistical model assumes. It can make model-based uncertainty estimates unreliable if not accounted for. No model or outcome distribution is reported for this trial, so there is no basis to state whether over-dispersion was assessed or relevant to an analysis. More broadly, the missing analytic detail prevents assessment of model assumptions, adjustment choices, or robustness of statistical results.

10. Multiplicity, Missing Data and Analysis Populations

The registry record does not report a statistical analysis plan, an interim-analysis plan, or a prespecified approach to multiplicity. It lists no analyses. It is therefore not possible to determine whether investigators planned several statistical tests, how they would control the chance of false-positive findings across tests, or whether any interim review could affect interpretation. These methods are not reported in the registry record.

The primary outcome describes adverse effects graded according to NCI CTCAE Version 3.0, but the record does not report a complete grade distribution, participant-level data, or cycle-specific summaries. It also does not describe the handling of missing outcome observations, withdrawals, or participants with incomplete follow-up. The reason that 29 participants were enrolled while 27 are listed at risk in the safety summaries is not reported in the registry record. Without those details, the completeness and analysis-population rules cannot be assessed.

In general, missing-data methods matter because the people with observed outcomes may differ from those without observed outcomes. A complete-case summary, for example, describes participants with recorded data and may not represent everyone enrolled if missingness is related to outcomes. This is a general statistical concern, not a claim about what happened in this trial. The registry does not state how missing safety information was handled.

Likewise, the record does not specify whether the primary outcome was assessed using a formal statistical comparison or only descriptive summaries. No efficacy results are reported in the registry record. These gaps limit assessment of prespecification, multiplicity, missingness, and the relationship between planned and reported analyses.

11. Safety Results

The registry reports serious adverse events as affected participants divided by participants at risk.

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 5% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: obstruction, gastrointestinal - small bowel not otherwise specified (7.4%), allergic reaction/hypersensitivity (3.7%), platelets (3.7%), dehydration (3.7%), hemorrhage, gastrointestinal - upper gastrointestinal not otherwise specified (3.7%).

The serious adverse-event summary lists 9 affected participants among 27 at risk. The other adverse-event summary lists 27 affected participants among 27 at risk. These are participant counts, not a count of all individual event episodes. The record does not state whether a participant could be represented in more than one event category, so the term counts should not be added together.

The most frequently listed serious-event term is Obstruction, Gastrointestinal - Small Bowel Not otherwise specified at 7.4%. Allergic Reaction/Hypersensitivity, Platelets, Dehydration, Hemorrhage, Gastrointestinal - Upper Gastrointestinal Not Otherwise Specified, and Infection with normal or Grade 1 Or 2 Absolute neutrophil count: Blood are each listed at 3.7%. The registry does not provide enough detail to determine the number of episodes, timing, duration, or severity grade for each term.

These figures describe events recorded in the treated group; they do not establish that the regimen caused them. Because the study has no concurrent control group, the posted values cannot be directly compared with outcomes under another treatment. The deaths field is not populated, so deaths are not reported in the registry record; that field should not be read as confirmation that there were none.

12. What the Result Does, and Does Not, Mean

The record describes a phase II study of belinostat and carboplatin in a single group of patients with recurrent or persistent platinum-resistant ovarian, fallopian tube, or primary peritoneal cancer. Its primary outcome concerns the frequency and severity of adverse effects during treatment and for 30 days after treatment ended. The posted safety summaries report 9 of 27 participants affected by serious adverse events and 27 of 27 affected by other adverse events.

Those counts are descriptive. They do not by themselves establish that treatment caused the recorded events, and there is no concurrent control group against which to compare them. The record does not provide a full grade breakdown, detailed event histories, or an explanation of the difference between 29 enrolled participants and the 27 participants listed at risk in each safety summary.

The results do not establish whether the combination improves cancer outcomes. Efficacy results are not reported in the registry record, and no analyses are listed. No p-values, confidence intervals, or treatment-effect estimates are reported. Consequently, neither the size nor the precision of a comparative efficacy effect can be assessed from the posted record.

More generally, statistical evidence is strongest when the design, outcome definitions, analysis populations, and uncertainty estimates are sufficiently described to support the intended comparison. Here, the design is single group and the analysis details are limited. The evidence available supports describing the reported safety counts, not drawing conclusions about comparative effectiveness, the cause of individual events, or outcomes outside the population and observation period recorded.

These limits are about what the registry record reports, not a judgment about the value of the research question. A phase II study can provide descriptive information relevant to later research, but its results need to be interpreted according to its design and reporting detail. No treatment recommendation follows from these registry summaries.

13. Why This Trial Matters Statistically

This trial is statistically informative as an example of how design constrains interpretation. The study is phase II but single group, with no masking and no allocation to a control arm. Its safety counts can describe events among treated participants, while the design does not support a randomized estimate of how outcomes compare with another treatment. Distinguishing those goals is essential: a descriptive safety summary and a comparative treatment effect answer different questions.

The difference between total enrollment and the at-risk safety counts also illustrates why analysis populations need clear definitions. The record lists 29 enrolled participants and 27 at risk in each safety summary, but does not explain the population rules. Without those rules, readers cannot evaluate how participants entered the summaries or whether incomplete observations affected them.

The outcome is defined as frequency and severity, and it names a grading framework and observation period. Yet the available summary does not report full grade distributions or detailed timing. This shows how an endpoint can be recognizable while key information needed to assess severity patterns and follow-up completeness remains unavailable.

Finally, the record lists no analyses and does not report efficacy results or a statistical plan. The planning illustration description is not a substitute for a trial-specific sample-size justification and does not create a control comparison for a single-group study. The statistical lesson is that design, endpoint definitions, denominators, and analysis reporting must be considered together. Counts can be useful, but their meaning depends on the population and comparison the study was designed to support.

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 principal trial-specific limitation is its single-group design. With allocation listed as not applicable and no masking, there is no concurrent randomized group to support a treatment contrast. The observed adverse-event summaries therefore cannot be interpreted as comparative estimates or as evidence that the regimen caused any listed event.

The registry reports 29 participants enrolled, but 27 are listed at risk in the serious and other adverse-event summaries. The explanation and analysis-population rules are not reported in the registry record. This limits assessment of how representative the safety summaries are of everyone enrolled and how incomplete observations were handled.

Although the primary endpoint refers to frequency and severity under NCI CTCAE Version 3.0, a full severity distribution and cycle-specific results are not reported. Individual event details, timing, duration, and overlap are also not reported in the registry record. The deaths field is unpopulated, so death outcomes cannot be characterized from that field.

Efficacy results, statistical analyses, a statistical analysis plan, and the trial’s sample-size justification are not reported in the registry record. The schedule and treatment details are stated, but the arm description’s final continuation criterion is incomplete in the record. These reporting limits prevent assessment of planned versus completed treatment, inferential methods, and the precision of any efficacy estimate.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Belinostat and Carboplatin in Treating Patients With Recurrent or Persistent Ovarian Epithelial Cancer, Fallopian Tube Cancer, or Primary Peritoneal Cancer That Did Not Respond to Carboplatin or Cisplatin — NCT00993616.
• Registry record last updated 2019-07-23; status last verified 2019-07.
• Results first posted 2014-01-21.
• 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.

18. Record Summary

NCT00993616 is a completed phase II, single-group trial sponsored by the National Cancer Institute. It studied belinostat with carboplatin in recurrent or persistent platinum-resistant ovarian, fallopian tube, or primary peritoneal cancer. Enrollment was 29; the trial began in 2009-12 and reached primary completion in 2012-07.

The primary outcome was the frequency and severity of observed adverse effects, graded according to NCI CTCAE Version 3.0, during each treatment cycle and 30 days after treatment ended. The registry lists 27 participants at risk in each safety summary, with 9 affected by serious adverse events and 27 affected by other adverse events. It does not report efficacy results, statistical analyses, the trial’s sample-size justification, or a detailed statistical plan.