Irofulven in Treating Patients With Recurrent or Persistent Ovarian Epithelial or Primary Peritoneal Cancer: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Irofulven in Treating Patients With Recurrent or Persistent Ovarian Epithelial or Primary Peritoneal Cancer (NCT00053365), a na phase 2 study. The primary outcome is Frequency and Severity of Observed Adverse Events, Grade 3 or Higher According to the National Cancer Institute Common Toxicity Criteria (NCI CTC) v2.0.

2. Clinical Question

The central statistical question is therefore descriptive: what safety information is recorded for participants receiving irofulven, and how much can those counts establish about the primary endpoint? A single-group study can describe events observed during treatment, but it cannot provide a randomized comparison with another treatment or with no treatment. In this record, the adverse-event data distinguish participants affected by serious events from those affected by other events. Those categories should not be treated as interchangeable with the primary endpoint, which is specifically defined by event severity grade.

Progression-free survival is listed as a secondary outcome, but an analysis result is not reported in the registry record. Accordingly, the record does not answer a comparative question about disease progression, and the safety counts do not establish a treatment effect. The available information is best read as a limited description of study design, the stated outcome, and recorded safety summaries.

3. Trial Design

This was a phase II study sponsored by the National Cancer Institute. The registry lists a single-group model, no allocation, and no masking. It describes a study of irofulven in patients with recurrent or persistent ovarian epithelial or primary peritoneal cancer; the official title specifies platinum-sensitive disease. Enrollment was 61. The study began in 2003-06, reached primary completion in 2010-07, and is listed as completed.

Participants in the treatment group received irofulven intravenously over 30 minutes on days 1 and 8. Courses were scheduled to repeat every 21 days unless unacceptable toxicity or disease progression occurred. These details describe the planned administration in the registry; they do not establish how many participants received each course or how long individuals remained on treatment. Those details are not reported in the registry record.

The primary outcome concerns the frequency and severity of observed adverse events graded 3 or higher under National Cancer Institute Common Toxicity Criteria v2.0. The registry identifies the outcome unit as participants and gives a monitoring timeframe of every cycle while on treatment and 30 days after the last cycle. The stated outcome population is eligible and evaluable patients. Progression-free survival is listed among the secondary outcomes.

Because there is no comparator arm, the design supports a description of outcomes among treated participants, not a like-for-like estimate of differences between treatments. The registry does not report a protocol-level statistical analysis plan, details of eligibility criteria, the number of participants included in each outcome analysis, or the reasons that the safety denominator differs from total enrollment. These gaps limit how fully the study’s conduct and analysis can be reconstructed from the registry record alone.

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: This is the number of eligible and evaluable participants.

6. Primary Endpoint

The registry lists the primary outcome as Frequency and Severity of Observed Adverse Events, Grade 3 or Higher According to the National Cancer Institute Common Toxicity Criteria (NCI CTC) v2.0, measured over Assessed every cycle while on treatment, 30 days after the last cycle of treatment.

The primary endpoint is the frequency and severity of observed adverse events graded 3 or higher according to NCI CTC v2.0. It is recorded in participants, rather than as a continuous measurement. The stated assessment schedule is every treatment cycle while participants are on treatment and 30 days after the last cycle. The listed analysis population is eligible and evaluable patients.

In general, an adverse-event endpoint can describe the burden of events occurring during a study and can identify events that meet a defined severity threshold. A participant-count endpoint indicates how many people experienced events; it does not, by itself, describe how many events each person experienced, how long events lasted, or the timing of each event. Those distinctions matter when interpreting a safety summary.

The registry record does not report a numerical result for this primary endpoint. The separate adverse-event summary includes counts for participants affected by serious adverse events and other adverse events, but those categories cannot be substituted for a count of participants with events graded 3 or higher. Seriousness and severity grade are different classifications. Without the primary endpoint result and its detailed event-level or grade-level breakdown, the extent to which the stated endpoint was met cannot be assessed from the registry record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What a participant count means. The primary outcome is specified as a number of participants with observed adverse events graded 3 or higher. This is a binary-outcome structure at the participant level: a person either meets a defined event criterion or does not, according to the outcome rules. A count needs a clearly defined denominator to be interpretable. The registry names eligible and evaluable patients as the outcome population, but it does not give a numerical primary-endpoint count or the number included in that endpoint analysis.

In general, binary outcomes may be described using counts and proportions. A proportion conveys the share of an analysis population meeting the event definition, while a count alone conveys how many participants did so. Both depend on a consistent event definition and an appropriate denominator. No primary-endpoint proportion is reported in the registry record, and none can be inferred from the separate serious-event summary.

Why the study design matters

In general, random allocation helps create groups that can be compared while limiting systematic differences between them. This study is listed as single-group, with allocation not applicable and no masking. It therefore has no randomized control group. Observed events can be described among participants treated with irofulven, but the design does not isolate a difference attributable to treatment from other explanations, such as the underlying illness or other care. No adjustment method or comparative analysis is reported in the registry record.

Masking can reduce the influence of expectations on some aspects of assessment or reporting. The registry lists masking as none. That fact does not establish that the outcome assessment was biased; it means the record does not describe a masked study. The registry does not report further safeguards or procedures for event assessment.

What uncertainty measures add

In general, a confidence interval communicates statistical uncertainty around an estimate, conditional on the model and assumptions used. Wider intervals indicate less precision, while narrower intervals indicate greater precision under those assumptions. An interval does not correct for the lack of a comparator, incomplete outcome reporting, or differences between enrolled and analyzed participants. The registry record does not report confidence intervals for the primary safety endpoint.

For event counts, precision depends in part on the number of participants with relevant outcome information and on how common the outcome is. Sparse events can lead to imprecise estimates. No precision estimate for the stated primary endpoint is reported. The serious-event counts do not provide enough information to determine precision for the grade-based endpoint because the registry does not report that endpoint’s result or its denominator in numerical form.

Why event definitions and follow-up matter

Adverse-event classifications answer different questions. Severity grading describes the intensity or seriousness of an event according to a grading system; serious-event status is a separate classification. A grade 3-or-higher endpoint should therefore not be assumed to match a serious-event count. The registry names NCI CTC v2.0 for the primary endpoint but does not report the grade distribution or a cross-classification of grade and seriousness.

The stated monitoring timeframe extends through treatment and 30 days after the last cycle. In general, safety summaries depend on the period in which events are collected and on how follow-up is handled when participants stop treatment or leave observation. The registry does not report participant-level follow-up duration, completeness of event collection, or handling of events after the stated timeframe.

Planning illustrations are not trial results

The registry includes assumptions for a sample-size planning illustration: a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and an effect defined as a stated fraction of the observed effect. This is a planning framework, not a description of the study’s single-group design. It does not establish that the trial used those assumptions, and the registry record does not report the trial’s sample-size justification or corresponding planned sample size.

In general, a sample-size calculation estimates how many participants may be needed for a planned analysis under specified assumptions. Such a calculation is not observed power and does not show whether a result is statistically persuasive. The registry’s planning assumptions should not be interpreted as evidence that the actual study was designed or analyzed as a two-arm comparison. No planning output for this study is reported in the registry record.

What is absent from the reported analyses

The registry lists three outcome measures and zero analyses. It names progression-free survival as a secondary outcome but reports no analysis result for it. The statistical tests, model specifications, handling of multiple outcomes, missing-data methods, and any interim analyses are not reported in the registry record. These omissions prevent a detailed appraisal of model assumptions or of how uncertainty was quantified. They also mean that no unreported analysis should be inferred from the descriptive safety entries.

10. Multiplicity, Missing Data and Analysis Populations

The registry lists three outcome measures, including the primary adverse-event outcome and progression-free survival as a secondary outcome. It reports zero analyses. The other outcome measures and their results are not described in the available record, and no statistical analysis plan or multiplicity procedure is reported in the registry record.

In general, testing several outcomes can increase the chance of obtaining at least one apparently unusual result by chance. Multiplicity procedures are methods for managing that issue when multiple tests are planned. The record does not state whether outcomes were tested, whether any adjustment was planned, or whether a hierarchy of outcomes was specified. With no reported analysis results, there is no basis for judging how multiplicity was handled.

The primary outcome population is described as eligible and evaluable patients. The study enrolled 61 participants, while the safety summary uses 55 as its denominator. The record does not explain why these populations differ, identify exclusions, or state how missing outcome data were handled. In general, excluding participants with incomplete data can affect how representative an analysis is of everyone enrolled; the direction and extent of any effect depend on why data are missing and on the analysis method.

The registry also does not report whether outcome information was complete through the stated follow-up period, how participants who discontinued treatment were handled, or how missing progression-free survival information was treated. These are not details that can be inferred from the participant counts. The resulting uncertainty should be kept separate from the uncertainty in any statistical estimate, since no primary endpoint estimate is reported.

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: vomitting (5.5%), ataxia(incoordination) (5.5%), anorexia (3.6%), dehydration (3.6%), gi other (3.6%).

The adverse-event summary records 18 of 55 participants as affected by serious adverse events and 55 of 55 as affected by other adverse events. These are separate categories in the registry; they should not be added together, treated as mutually exclusive, or interpreted as the grade 3-or-higher primary endpoint. The record does not state whether participants could appear in both categories, and seriousness is not the same as severity grade.

The listed serious-event terms are Vomitting and Ataxia(Incoordination), each at 5.5%; and Anorexia, Dehydration, Gi Other, and Infection Without Neutropenia, each at 3.6%. These are the registry’s listed terms and percentages. The summary does not report the number of events per participant, timing, duration, grade, or the full set of safety outcomes. It therefore does not describe the entire safety profile.

The denominator of 55 differs from the enrollment of 61, and the reason is not reported in the registry record. These figures are descriptive of the recorded safety summary; they do not provide a comparison with another group or an estimate of how much risk is attributable to irofulven. The primary endpoint’s numerical result is not reported, so the serious-event summary should be read as separate safety information rather than as a substitute for that endpoint.

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

The record establishes that a completed phase II, single-group study evaluated irofulven in recurrent or persistent ovarian epithelial or primary peritoneal cancer, with the official title specifying platinum-sensitive disease. It describes the treatment schedule and identifies a primary safety outcome based on adverse events graded 3 or higher. The posted adverse-event summary includes counts for participants affected by serious and other adverse events, along with selected serious-event terms and percentages.

Those data do not establish whether irofulven is more or less effective than another treatment. There is no comparator arm, and progression-free survival is listed without a reported analysis result. The registry does not report a numerical result for the primary endpoint, so the record cannot show how many eligible and evaluable participants experienced grade 3-or-higher adverse events. The serious-event counts cannot fill that gap because serious status and severity grade are distinct classifications.

In general, a single-group study can contribute descriptive information about what was observed among treated participants. It cannot by itself distinguish treatment-related effects from other possible causes or estimate a comparative treatment effect. Even a complete outcome count would not replace a control group for that purpose. Likewise, a list of selected event terms is not equivalent to a complete event-level account or an estimate of comparative risk.

The registry record also does not report confidence intervals, statistical tests, missing-data procedures, or the detailed analysis population for the primary endpoint. As a result, neither the precision of the primary safety estimate nor the effect of missing outcome information can be evaluated from this record. The defensible interpretation is limited to the design and the descriptive safety entries that are explicitly recorded.

13. Why This Trial Matters Statistically

This study illustrates a basic distinction in clinical-trial interpretation: an observed event count and a comparative treatment effect are different quantities. A single-group design can record events among participants receiving a treatment, but without a concurrent control group it cannot directly estimate how those outcomes compare with an alternative. This distinction remains important even when a study is completed and has a stated primary endpoint.

The record also highlights the importance of matching the outcome definition to the available result. The primary endpoint is defined by adverse-event severity grade, while the posted safety summary separately reports serious adverse events and other adverse events. Treating those categories as equivalent would change the question being answered. A clear endpoint definition and an outcome result using that definition are necessary for interpretation.

Denominators are equally important. The record reports enrollment of 61 and uses 55 participants in the safety summary. Without an explanation of that difference, a reader cannot fully assess which participants contributed to the safety counts or how representative they are of those enrolled. The named eligible-and-evaluable population for the primary outcome does not resolve that gap because its numerical size is not reported.

Finally, the registry lists progression-free survival as a secondary outcome but reports no analysis. This underscores that naming an outcome is not the same as reporting its estimate, uncertainty, and analysis population. For biostatistical interpretation, the design, event definition, denominator, and completeness of results all matter. Here, the record supports a limited descriptive reading, while leaving the primary endpoint result and secondary outcome analysis unreported.

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 registry record gives limited information about eligibility criteria beyond the disease description, and it does not report the detailed characteristics of enrolled participants. It therefore does not allow assessment of how closely the study population represents other patients with recurrent or persistent ovarian epithelial or primary peritoneal cancer. The official title describes platinum-sensitive disease, but further detail on how that status was defined is not reported in the registry record.

The study has no control group and no masking. As a result, the record cannot support a randomized comparison, and outcome assessment may not have the protections that masking can offer. This does not establish that the recorded events were incorrectly assessed; it limits what can be ruled out from the design description alone.

The primary endpoint result is not reported, despite the endpoint being defined and its assessment timeframe being stated. The registry also does not report grade-level event counts, a complete event listing, confidence intervals, or the number of participants included in the primary endpoint analysis. Serious-event counts and selected terms do not resolve these omissions because they are not the same measure as the grade-based endpoint.

The safety denominator is 55, compared with enrollment of 61, and the reason for the difference is not reported in the registry record. There is no information on missing-data handling, participant-level follow-up, treatment exposure, or event ascertainment after discontinuation. Progression-free survival is listed as a secondary outcome, but no analysis result is reported. Together, these limitations restrict interpretation to the study description and partial safety summaries recorded in the registry.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Irofulven in Treating Patients With Recurrent or Persistent Ovarian Epithelial or Primary Peritoneal Cancer — NCT00053365.
• Registry record last updated 2019-07-24; status last verified 2019-07.
• Results first posted 2013-12-19.
• 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

NCT00053365 is listed as a completed phase II, single-group study sponsored by the National Cancer Institute. It enrolled 61 participants and studied irofulven for recurrent or persistent ovarian epithelial or primary peritoneal cancer; the official title specifies platinum-sensitive disease. The treatment schedule was intravenous administration over 30 minutes on days 1 and 8, with courses repeating every 21 days unless unacceptable toxicity or disease progression occurred.

The primary outcome was the frequency and severity of observed adverse events graded 3 or higher under NCI CTC v2.0, assessed each cycle and 30 days after the last cycle. Its numerical result is not reported in the registry record. The safety summary records 18 of 55 participants affected by serious adverse events and 55 of 55 affected by other adverse events, with selected serious-event terms and percentages. Progression-free survival is listed as a secondary outcome, but no analysis result is reported. The single-group design provides no randomized comparator.