Monoclonal Antibody Therapy in Treating Patients Scheduled for Surgery to Remove Ovarian Cancer: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Monoclonal Antibody Therapy in Treating Patients Scheduled for Surgery to Remove Ovarian Cancer (NCT00006099), a non-randomized phase 1 study. The primary outcome is Number of Subjects With Grade 3 Adverse Events.

2. Clinical Question

The registry’s stated purpose was to study monoclonal antibody therapy in patients with ovarian cancer. Its rationale describes antibodies as a way to locate tumor cells and potentially deliver tumor-killing substances. That is the stated rationale, not evidence that the antibody achieved those aims in this trial. The record concerns a presurgical pilot, and its central measurable question was framed around toxicity rather than a comparative measure of tumor response or survival.

With 1 patient entering the study and no randomized comparison, the record cannot establish a treatment effect relative to another therapy or no therapy. The two infusion routes are listed as arms, but the record does not support interpreting them as balanced groups. It also does not provide a reported primary-endpoint result that would allow the reader to quantify the Grade 3 adverse-event outcome.

3. Trial Design

This was a Phase 1, non-randomized study with no masking and a sequential model. Its official title identifies it as a pilot trial of humanized 3S193 monoclonal antibody in presurgical patients with ovarian cancer. Ludwig Institute for Cancer Research is listed as the sponsor. The registry record reports an enrollment of 1 and a status of terminated.

The study began on 2000-08-02, and its primary completion date is listed as 2002-02-26. These registry dates do not explain when the participant received treatment, how long follow-up was completed, or why the study was terminated. The reason for termination is not reported in the registry record.

The intervention was hu3S193 administered through routes described in separate arm entries. For intraperitoneal infusion, the record specifies a 5 mg dose radiolabeled with 5 mCi of 111In. It also describes intraperitoneal administration of 10 mCi 99mTc-sulphur colloid in 500 ml of normal saline to assess distribution. The intravenous arm description specifies a 5 mg dose radiolabeled with 5 mCi of 111In, diluted in 100 mL of 5% human serum albumin and administered over a 30 minute period.

Although both routes appear in the arm descriptions, the safety record lists 1 at risk in the intraperitoneal arm and 0 at risk in the intravenous arm. Therefore, the record does not document participants at risk in both arms. Non-random allocation and the absence of a like-for-like control comparison make a route-to-route efficacy interpretation inappropriate. The registry record does not describe a planned sample-size justification, an allocation procedure beyond non-randomization, or a statistical analysis plan.

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

Analysis population for the primary outcome, as defined in the registry: The 1 patient who entered the study.

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: The 1 patient who entered the study.

6. Primary Endpoint

The registry lists the primary outcome as Number of Subjects With Grade 3 Adverse Events, measured over up to 30 days.

The primary endpoint is titled “Number of Subjects With Grade 3 Adverse Events.” It is a count of participants meeting an adverse-event severity criterion, rather than a measure of tumor response, survival, or change in a continuous clinical measure. The specified timeframe is up to 30 days. The endpoint description says toxicities were to be graded using the Common Toxicity Criteria (CTC) Scale, version 2.0, March 1998.

In general, a toxicity endpoint can describe the occurrence of harms during a defined observation period. A severity threshold focuses attention on events considered more serious under the chosen grading system. The record also states that the study was to be terminated upon an adverse event of Grade 3 or greater severity considered definitely related to hu3S193. This stopping condition gives the endpoint particular relevance to the stated safety focus; it does not establish that such an event occurred.

The listed endpoint population is the 1 patient who entered the study. However, the registry record has no analysis entries and does not report an endpoint-specific count, an estimate, or an uncertainty interval. The separate adverse-event figures list affected counts for serious and other adverse events, but those figures do not substitute for a reported primary-endpoint analysis. In particular, the registry record does not state how its adverse-event categories map to the Grade 3 endpoint or report a Grade 3 count. The endpoint should therefore be read as the prespecified or registered measurement, not as a result that can be recovered from the available record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What does a count endpoint represent?

A count of participants with an event records how many people in a defined population experienced the outcome. It differs from a continuous endpoint, which records a measurement on a scale, and from a time-to-event endpoint, which incorporates when an event occurred. Here, the registered primary outcome is a participant count for Grade 3 adverse events, with a timeframe of up to 30 days. The registry record identifies the population as the 1 patient who entered the study.

For a count to be interpreted, the event definition, observation period, and population need to be clear. The record supplies a severity framework through the CTC Scale, version 2.0, March 1998, and describes a stopping condition based on severity and attribution to hu3S193. It does not supply the primary endpoint’s outcome count or an analysis entry. The general definition of the endpoint is therefore available, while the trial-specific estimate is not.

Why does the denominator matter?

In general, an event count is interpreted alongside the number of participants observed for that event. The denominator indicates how much information the count represents. With very few observations, each participant’s outcome has a large influence on the apparent pattern. Even if no events are observed, the true event probability remains uncertain; absence of observed events is not proof that the event cannot occur.

The posted safety entries make this denominator issue especially important. The intraperitoneal arm has 1 participant at risk in each of the serious and other adverse-event categories, while the intravenous arm has 0 at risk in both. A zero affected count paired with no participants at risk is not an estimate of safety among exposed participants. The record also gives no arm-by-arm primary endpoint result. These safety counts should be read as descriptive registry entries, not as a comparative statistical analysis.

What can a comparison establish?

In general, randomized allocation helps make groups comparable at the start of a study, supporting a causal interpretation of outcome differences when the trial is otherwise appropriately conducted. This trial was non-randomized, and the record states that the arms are not a like-for-like control comparison. Thus, even if numerical outcomes were available for both routes, a difference could not automatically be attributed to route of administration. The posted safety entries do not show participants at risk in both arms in any case.

The registry does not report a randomized control group, masking, or a comparative estimate. Masking is listed as none. These are descriptions of design, not a statistical adjustment that can correct for group differences. The record does not describe covariate adjustment or another method for addressing confounding.

How should uncertainty be understood?

In general, a confidence interval gives a range of values compatible with the data under a specified statistical method. Its width reflects, among other things, the amount of information and the variability in the observations. Sparse binary outcomes can yield imprecise estimates, especially when event counts are very low. A confidence interval does not establish causation and should be interpreted with the design and endpoint definition.

No confidence interval, standard error, p-value, or effect estimate for the primary endpoint is reported in the registry record. The record lists 0 analyses. It is therefore not possible to assess statistical precision or distinguish a possible underlying event rate from the observed count using a reported inferential analysis. General statements about uncertainty do not replace an analysis of the trial’s own outcomes.

What analysis plan is documented?

The registry record does not report a statistical analysis plan, an interim analysis plan, a sample-size justification, or a method for handling missing data. It records a sequential model, but that label alone does not describe the statistical rules used to enroll, stop, or analyze participants. The stopping condition described for a Grade 3 or more severe adverse event considered definitely related to hu3S193 is a clinical safety rule in the endpoint description; the record does not specify a broader monitoring or analysis procedure.

In general, a prespecified analysis plan helps clarify the outcome definitions, populations, comparisons, and methods before results are evaluated. Without a reported plan, readers cannot determine which analyses were intended beyond the registered endpoint description. The absence of analyses in the registry record also means there is no posted basis for evaluating any inferential claims.

Are planning calculations trial results?

In general, sample-size calculations are planning illustrations that depend on assumptions about the target effect, variability, allocation, and statistical method. They are not observed results and do not show the probability that a completed trial has demonstrated an effect. The registry record’s available trial details do not report an observed primary-endpoint effect or a numerical primary analysis. Any planning illustration should therefore remain distinct from evidence observed in this study, and should not be described as observed power.

10. Exact Estimates for Each Arm

No two-arm comparison is shown for this trial, but each arm's result can still be summarized with exact interval estimates computed from the posted counts. The Clopper-Pearson interval is exact and conservative; the Wilson interval is a common closer-to-nominal alternative; the Bayesian credible interval uses a uniform Beta(1, 1) prior. For small groups the three can differ noticeably.

R code and output

arm    <- c("Intraperitoneal (IP) Infusion of 111In-hu3S193")
n      <- c(1)
events <- c(0)
for (i in seq_along(n)) {
  ci <- binom.test(events[i], n[i])$conf.int          # Clopper-Pearson exact interval
  cr <- qbeta(c(0.025, 0.975), 1 + events[i], 1 + n[i] - events[i])   # Bayesian, uniform Beta(1,1) prior
  wi <- (function(x, n) { p <- x / n; z <- qnorm(0.975); d <- 1 + z^2 / n
    c((p + z^2 / (2 * n) - z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d, (p + z^2 / (2 * n) + z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d) })(events[i], n[i])
  cat(sprintf("ARM|%s|%d|%d|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f\n", arm[i], n[i], events[i], 100 * events[i] / n[i],
              100 * ci[1], 100 * ci[2], 100 * wi[1], 100 * wi[2], 100 * cr[1], 100 * cr[2]))
}

ARM  Intraperitoneal (IP) Infusion of 111In-hu3S193  1  0  0.0  0.0  97.5  0.0  79.3  1.3  84.2

11. Multiplicity, Missing Data and Analysis Populations

The registry record lists 1 outcome measure and 0 analyses. It does not report a multiplicity adjustment, a hierarchy of outcomes, or a plan for controlling error across multiple statistical tests. Because no analyses are listed, there are no reported test results to assess for multiplicity. The primary endpoint is identified, but the record does not provide an inferential analysis of it.

Missing-data methods are not reported in the registry record. It does not state how an incomplete observation period, a missed assessment, or an unavailable toxicity grade would be handled. The primary endpoint population is described as the 1 patient who entered the study, but that description does not explain whether the patient completed follow-up through the stated timeframe or how any missing assessments were treated.

The analysis populations are also not described beyond the primary endpoint’s stated population. The record does not specify an intention-to-treat population, a safety population definition, or a per-protocol population. The safety entries show at-risk counts by arm, but they do not define the population used for analysis or explain inclusion rules. In general, conclusions can depend on which participants are included and how missing outcomes are handled. Here, those methods are not reported, and there are no analysis entries to clarify them.

12. Safety Results

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

Deaths

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 0% reporting threshold.

The safety entries are counts, not comparative effect estimates. For intraperitoneal infusion, the registry records 1 at risk and 0 affected for serious adverse events, and 1 at risk and 0 affected for other adverse events. Deaths are recorded as 0. For intravenous infusion, the record lists 0 at risk and 0 affected in the serious and other adverse-event categories, with 0 deaths.

The zero affected values in the intravenous arm must be read alongside its zero at-risk values: they do not describe observed outcomes among participants at risk in that arm. The registry does not report a like-for-like comparison between routes, and its safety figures do not provide a comparative risk estimate. No serious adverse-event terms are listed.

These entries should not be substituted for the primary endpoint result. The endpoint specifically counts subjects with Grade 3 adverse events up to 30 days, while the safety entries are organized as serious and other adverse-event counts. The registry record does not state that these categories are equivalent or provide a Grade 3 endpoint count. The reason for termination is not reported in the registry record.

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

The record documents a Phase 1 pilot of hu3S193 in presurgical patients with ovarian cancer, with 1 patient enrolled and non-randomized allocation. Its primary endpoint was a count of subjects with Grade 3 adverse events over a timeframe of up to 30 days. The endpoint description also states a stopping condition for a Grade 3 or more severe adverse event considered definitely related to hu3S193.

What the record does not establish is equally important. It does not report a numerical result or analysis for the primary endpoint, and it lists 0 analyses. The adverse-event entries report counts by arm, but they do not provide an endpoint-specific Grade 3 result or a comparison of routes. The intravenous arm has 0 at risk, so its recorded zeros cannot be interpreted as evidence about event frequency among treated participants by that route.

This trial does not provide a randomized comparison, and the registry explicitly cautions that the arms are not a like-for-like control comparison. No efficacy outcome or treatment-effect estimate is reported. Consequently, the record cannot establish whether hu3S193 affected tumor outcomes, whether either route performed better, or how outcomes compare with an alternative treatment.

In general, a small pilot may describe study procedures and collect initial observations, but its findings must be interpreted in light of the amount of information and the design. Here, the enrollment and reported analysis information are too limited to support broad conclusions about efficacy or safety. The record’s terminated status is known; the reason for termination is not reported in the registry record. The absence of a reported result should not be mistaken for a favorable or unfavorable endpoint finding.

14. Why This Trial Matters Statistically

This trial is statistically notable because the endpoint and the available result information are not the same thing. The registry clearly identifies a Grade 3 adverse-event count as the primary endpoint and specifies a timeframe and grading framework. Yet it lists 0 analyses and does not report a numerical result for that endpoint. Distinguishing a registered question from an analyzed result is essential when interpreting clinical-trial records.

The design also illustrates why denominators and comparators matter. Enrollment was 1, allocation was non-randomized, and the registry cautions that the arms are not a like-for-like control comparison. The safety entries list a participant at risk in the intraperitoneal arm but none in the intravenous arm. Descriptive zeros without a denominator cannot be taken as evidence of no risk, and unequal or absent exposure across groups cannot support a fair route comparison.

More generally, a count observed in a very small study conveys limited information about the frequency of an uncommon or serious event. Precision depends on the amount of observed information, and the registry does not provide an uncertainty interval or inferential estimate for the primary endpoint. A stopping condition may be relevant to safety oversight, but the record does not describe the broader monitoring or analysis methods and does not report why the study was terminated.

The record therefore matters as an example of how careful statistical reading separates protocol-level intent, arm descriptions, safety counts, and analyzed endpoint results. Those components answer different questions. Keeping them distinct prevents route descriptions or zeros in safety fields from being overread as evidence of comparative benefit or definitive safety.

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.

The main trial-specific limitation is the limited amount of outcome information: enrollment was 1, and the registry lists 0 analyses. Although the primary endpoint is defined, its numerical result is not reported. The record therefore does not permit an estimate of the observed Grade 3 adverse-event count from an endpoint analysis or an assessment of its precision.

The design does not provide a randomized comparison. Allocation was non-randomized, masking was none, and the registry cautions that the arms are not a like-for-like control comparison. The safety entries show 0 at risk in the intravenous arm, so the posted data do not support a route comparison. The record does not report patient characteristics, detailed eligibility criteria, treatment received by the participant, or completion of follow-up.

The safety categories and the primary endpoint are not explicitly reconciled. The registry reports serious and other adverse-event counts, but it does not state how these categories correspond to Grade 3 events or provide a primary endpoint count. No serious adverse-event terms are listed. The reason for terminated status is not reported in the registry record.

Statistical limitations include the absence of a reported analysis plan, inferential methods, uncertainty intervals, missing-data methods, analysis populations beyond the endpoint description, and sample-size justification. The registry does not report whether any assessments were missing or how they would have been handled. These gaps restrict interpretation of both the endpoint and the safety entries; they should not be filled by assumptions about the protocol or conduct.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Monoclonal Antibody Therapy in Treating Patients Scheduled for Surgery to Remove Ovarian Cancer — NCT00006099.
• Registry record last updated 2023-10-04; status last verified 2023-10.
• Results first posted 2021-08-12.
• 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.

19. Record Summary

NCT00006099 is a Phase 1 pilot of hu3S193 in presurgical patients with ovarian cancer, sponsored by Ludwig Institute for Cancer Research. It was non-randomized, unmasked, and sequential, with enrollment of 1. The registry lists the study as terminated; the reason is not reported.

The primary endpoint was the number of subjects with Grade 3 adverse events up to 30 days, graded according to the CTC Scale, version 2.0, March 1998. The registry record lists 0 analyses and does not report a numerical primary-endpoint result. Safety entries show 1 at risk and 0 affected in the intraperitoneal arm, no participants at risk in the intravenous arm, and 0 deaths in both arms. These counts do not establish a comparative safety result or an efficacy effect.