A Clinical Trial of Autologous Oxidized Tumor Cell Lysate Vaccine For Recurrent Ovarian, Fallopian Tube or Primary Peritoneal Cancer: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for A Clinical Trial of Autologous Oxidized Tumor Cell Lysate Vaccine For Recurrent Ovarian, Fallopian Tube or Primary Peritoneal Cancer (NCT01312389), a randomized phase 1, phase 2 study. The primary outcome is Number of Participants With Adverse Events.

2. Clinical Question

3. Trial Design

The registry describes a randomized, parallel-group Phase I/II clinical trial with no masking. The study concerned OC-L, an autologous vaccine based on oxidized tumor cell lysate, for recurrent ovarian, fallopian tube or primary peritoneal cancer. Its stated aims included feasibility, safety and immunogenicity, and the study summary described a duration of 24 months. The sponsor is Abramson Cancer Center at Penn Medicine. The registry lists a start date of 2011-04, primary completion of 2012-05, and a terminated status.

The design description separates Phase 1 from Phase 2. Phase 1 was planned to enroll 3 participants receiving tumor lysate/Montanide with Ampligen, using a 3+3 approach. The registry describes further enrollment depending on observed dose-limiting toxicities, but the posted description ends before the full stopping rule is stated. The record says the study was terminated after the first 3 Phase 1 participants were enrolled. It does not report why the study was terminated.

Phase 2 was planned as two arms. Arm A was to receive OC-L with Montanide by intradermal or subcutaneous injection in both groin regions. Arm B was to receive the same vaccine with intravenous Ampligen. The record lists 0 participants in each Phase 2 arm, so no Phase 2 outcomes are available for comparing the regimens. Although the overall design is described as randomized, the allocation sequence, its implementation and the specific randomization procedures are not reported in the registry record. No masking was planned.

The primary outcome was the number of participants with adverse events within 30 days of their last vaccination. The registry says that participants receiving at least one vaccination would be included in the toxicity analysis. Immune response and clinical response are listed as secondary outcomes, but corresponding analysis results are not posted. The record does not report further details of the 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.

Before assignment: Phase 1 patients were not to continue into Phase 2. If the study had continued, new patients would have been recruited into Phase 2.

Phase 1

Phase 2

Analysis population for the primary outcome, as defined in the registry: The study was terminated after the first 3 patients were enrolled in Phase 1

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: No subjects were enrolled in Phase 2 (Arm A or B)

6. Primary Endpoint

The registry lists the primary outcome as Number of Participants With Adverse Events, measured over within 30 days of last vaccination.

The primary endpoint is a count: the number of participants who experienced adverse events within 30 days of their last vaccination. Toxicities were to be graded using the NCI Common Toxicity Criteria, Version 4.0, and participants who received at least one vaccination were eligible for the toxicity analysis. The posted value for Phase 1 is 3 participants with adverse events. The result is a count of affected participants, not a measure of how many separate events occurred or how long they lasted.

Safety endpoints are used in early clinical research to describe observed harms and to support assessment of whether a regimen can be administered and studied further. Counting participants with events gives a direct summary of how many people in the observed group were affected. It does not, by itself, show the severity, timing, duration or causal relationship of every event. Those details require event-level reporting and grading information, which is not reported in the registry record beyond the stated grading method and the separate serious-event counts.

The count also needs to be read in light of the study’s small enrolled group and the fact that the Phase 2 arms had no participants. The endpoint does not estimate a difference between the planned Phase 2 regimens. Nor does it answer the listed secondary questions about immune or clinical response: those outcomes are named in the record, but their results are not reported.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The primary outcome is a count of participants with at least one adverse event during the specified period. This differs from a count of all event episodes: one participant may have more than one event, but the endpoint as named records whether participants were affected. The registry reports the Phase 1 participant count and the number affected. It does not provide event-level data or a more detailed breakdown of event frequency.

What does a count tell us?

A count is directly interpretable as the number of people meeting the endpoint definition in the observed group. It is useful for describing what happened in a small safety cohort. It does not, on its own, quantify uncertainty around the underlying rate or permit a reliable comparison with an unobserved group. In general, uncertainty estimates such as confidence intervals communicate the range of values compatible with data under stated assumptions. No confidence interval is reported for this primary outcome in the registry record, so the posted count should not be mistaken for a precise estimate of risk beyond the enrolled participants.

Counts also depend on the endpoint definition and the observation window. Here, the stated window is within 30 days of the last vaccination, and the toxicity description says all toxicities observed in that window would be included. The record says that participants receiving at least one vaccination would be included in the toxicity analysis. Details such as how the last vaccination date was established for participants who stopped early, or how events with incomplete timing information were treated, are not reported in the registry record.

Why does the planned comparison matter?

The trial is identified as randomized and parallel-group, and the planned Phase 2 arms differed in whether intravenous Ampligen accompanied the vaccine. Randomization is generally used to reduce systematic differences between groups at the point of assignment, making a between-group comparison more interpretable. But a randomized design can support such a comparison only when participants are assigned to the relevant groups and outcomes are available. The posted results show 0 participants in each Phase 2 arm. Thus, there is no observed Phase 2 contrast to estimate. The Phase 1 safety count does not substitute for that comparison.

The Phase 1 description refers to a 3+3 approach for dose-limiting toxicities. In general, such cohort-based escalation designs use observed toxicity information to guide whether additional participants are enrolled at a dose level. The registry’s Phase 1 description includes some conditional enrollment rules but is truncated before its final criterion. The exact full protocol, dose levels, decision rules and operational details are not reported in the registry record. The record reports enrollment of the first 3 participants and termination after those participants were enrolled, but does not explain why.

What can a planning calculation show?

Sample-size calculations are planning tools: they describe how many participants might be needed under specified assumptions to detect a chosen effect with a chosen statistical procedure. They are not evidence that a study had that number of participants, and they are not a measure of the probability that an observed result is true. The registry’s planning assumptions describe a two-sided significance level of 0.05, equal allocation between two arms and a normal approximation, with the target effect expressed as a fraction of the observed effect. The stated assumptions are for planning illustrations, not the trial’s own design.

For a time-to-event outcome, planning methods often relate the amount of information to the number of events rather than simply the number of participants. For a binary endpoint, planning typically depends on the event proportions being compared, the target difference, allocation and chosen statistical criteria. These are general methodological points. In this record, the binary endpoint does not have estimable values for a two-arm comparison because there are no Phase 2 participants in either arm. Consequently, no such comparison can be grounded in the posted Phase 2 outcomes. Planning illustrations cannot repair the absence of an observed comparison, and they should not be read as observed power.

Why does over-dispersion matter?

For some count outcomes, a statistical model may assume that variability follows a particular distribution. Over-dispersion means that the observed variability is greater than that model expects. If it is ignored, standard errors may be too small and uncertainty may be understated. This is a general consideration for count-data analysis. It does not alter the simple endpoint definition in this trial, which counts participants with adverse events, and the registry does not report an over-dispersion assessment or a fitted count model.

The statistical-analysis details necessary to evaluate model choice, covariate adjustment, handling of repeated events or confidence-interval construction are not reported in the registry record. No primary-outcome analyses are listed. The record identifies three outcome measures and two posted secondary outcomes, but the number of analyses is 0. The posted information therefore supports a descriptive reading of the observed counts, not a reconstruction of an inferential analysis plan.

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("Phase 1")
n      <- c(3)
events <- c(3)
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  Phase 1  3  3  100.0  29.2  100.0  43.9  100.0  39.8  99.4

11. Multiplicity, Missing Data and Analysis Populations

The registry lists immune response and clinical response as secondary outcomes in addition to the primary safety outcome. It does not report results or analyses for those secondary outcomes, and the number of analyses listed is 0. The multiplicity strategy—how the possibility of false-positive findings across several outcomes or comparisons would be controlled or interpreted—is not reported in the registry record. With no posted analyses for the secondary outcomes, there is no basis for assessing how multiple testing was handled.

The record identifies the toxicity population as participants who received at least one vaccination. It also says that the study was terminated after the first 3 Phase 1 participants were enrolled. However, it does not report participant-level follow-up completeness, whether all participants completed the 30-day adverse-event window, or how missing event information was handled. Missing-data rules, imputation methods and sensitivity analyses are not reported in the registry record. These absences do not establish that data were missing; they mean the handling of any missing information cannot be evaluated from the posted record.

The record does not describe an intention-to-treat analysis population, a per-protocol population or other analysis sets beyond the stated inclusion rule for toxicity. It also does not report whether participants were analyzed according to treatment received or assigned. Since the Phase 2 arms have 0 participants, those details would not create a Phase 2 comparison in the posted results. Randomization procedures, protocol deviations and the reason for termination are not reported in the registry record. As a result, the available information supports a descriptive summary of Phase 1 safety counts but not a full assessment of analysis populations, missing-data handling or multiplicity.

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.

Most frequent non-serious adverse event terms

The serious adverse-event summary lists 3 Phase 1 participants at risk and 0 affected by serious adverse events. It also lists 0 deaths. These are counts among the enrolled Phase 1 group, not comparative estimates. The Phase 2 arms each have 0 participants, so their zero counts do not indicate that participants in those arms had no events; no participants were enrolled in them according to the posted results.

The record separately lists other adverse events affecting 3 Phase 1 participants. Thus, the serious-event count of 0 should not be read as an absence of all adverse events. The registry does not list serious-event terms, and it does not provide a detailed event-by-event account or severity grades for the other events. The endpoint description states that toxicities were to be graded using NCI Common Toxicity Criteria, Version 4.0, within 30 days of the last vaccination. The grades themselves are not reported in the registry record.

These figures describe what was recorded in a group of 3 participants. A small descriptive safety summary cannot rule out events that were not observed, and it does not estimate a comparative difference between the planned Phase 2 regimens. The reason for study termination and the completeness of follow-up are not reported in the registry record, so neither should be inferred from the serious-event count.

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

The registry result establishes a limited descriptive fact: among the 3 Phase 1 participants, 3 were listed as having other adverse events, 0 were listed as having serious adverse events, and 0 deaths were recorded. The primary endpoint concerns participants with adverse events within 30 days of the last vaccination. It is therefore a short-window safety summary as defined in the record, not a comprehensive account of all possible events over longer follow-up.

The result does not establish that the regimen is safe or unsafe in a broader population. The small group gives little information about how often less common events might occur, and the record does not provide confidence intervals or detailed event descriptions. Absence of a recorded serious adverse event in this group is not proof that serious events cannot occur. Equally, the count of other adverse events does not by itself specify their severity, duration or clinical importance.

The result also does not establish whether Ampligen changes adverse-event frequency. Phase 2 Arm A and Phase 2 Arm B were planned to differ in the use of intravenous Ampligen, but both had 0 participants in the posted results. No randomized Phase 2 comparison is therefore available. The trial’s randomized designation describes the overall design, not an observed comparison among these empty Phase 2 arms.

The study summary names feasibility, safety and immunogenicity as aims, and the record lists immune response and clinical response as secondary outcomes. The posted data do not include analyses for those secondary questions. The safety count cannot be taken as evidence about immune response, clinical response, vaccine efficacy or disease control. No such conclusion follows from the adverse-event figures.

Finally, the record says that enrollment ended after the first 3 Phase 1 participants but does not report the reason for termination. That unexplained stopping context limits interpretation of the trial’s development and the extent to which the planned questions were pursued. The appropriate reading is therefore narrow: the registry documents safety counts for a small Phase 1 group, while comparative Phase 2 and secondary-outcome results remain unavailable in the registry record.

14. Why This Trial Matters Statistically

This trial is statistically instructive because it makes the distinction between a planned design and the data that were ultimately posted especially important. The registry identifies a randomized Phase I/II study, but the available participant counts show enrollment only in Phase 1. The planned Phase 2 arms have 0 participants. A randomized label cannot supply a treatment comparison when the groups needed for that comparison contain no participants.

The primary outcome is a participant count, which can make a small safety result easy to state but easy to overinterpret. Three participants had other adverse events, while 0 had serious adverse events. These counts are useful as a record of observed events in the enrolled group. They do not have the precision or scope needed to characterize event frequency across a wider population or assess differences between treatment strategies.

The record also illustrates why outcome definition and follow-up window matter. The adverse-event outcome is tied to the period within 30 days of the last vaccination, with toxicity grading specified in the description. That definition frames what the endpoint is intended to capture; it does not necessarily answer questions about events outside that period, event duration, or longer-term safety. Such details are not reported in the registry record.

Finally, the study’s stated secondary questions—immune response and clinical response—are distinct from its primary safety count. Their presence in a trial record does not mean that results are available or that a statistical comparison was completed. The record lists no analyses. This trial therefore offers a clear example of how registries can document a protocol’s aims and design while the posted results support only a narrower descriptive conclusion. Readers should keep planned comparisons, observed data and unanswered questions separate.

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 most direct limitation is the small enrolled Phase 1 group: the registry reports 3 participants and says the study was terminated after those participants were enrolled. This limits the range of conclusions that can be drawn from the adverse-event counts, particularly about uncommon events or the experience of a broader population. The Phase 2 arms each have 0 participants, so no outcomes are available to compare OC-L with Montanide alone against OC-L with Montanide plus intravenous Ampligen.

The primary outcome is reported as a count of affected participants, but the record does not provide a detailed list of the other adverse events, their grades, timing or duration. Although the stated method refers to NCI Common Toxicity Criteria, Version 4.0, individual toxicity grades are not reported. Serious-event terms are also not listed. Follow-up completeness for the 30-day window is not reported in the registry record.

The trial status is terminated, but the reason for termination is not reported. The Phase 1 description gives only a partial account of the 3+3 decision rules, so the complete protocol criteria are not available in the posted description. Randomization procedures, allocation concealment and any analysis procedures are not reported. No primary or secondary analyses are listed, and results for the named immune-response and clinical-response outcomes are not posted.

These limitations do not demonstrate that events or procedures were omitted; they define what cannot be assessed from the registry record. In particular, the posted counts should be treated as descriptive rather than as evidence of comparative safety or of clinical benefit.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: A Clinical Trial of Autologous Oxidized Tumor Cell Lysate Vaccine For Recurrent Ovarian, Fallopian Tube or Primary Peritoneal Cancer — NCT01312389.
• Registry record last updated 2021-11-15; status last verified 2021-10.
• Results first posted 2021-11-15.
• 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

This registry record describes a randomized, unmasked, parallel Phase I/II study of an autologous oxidized tumor cell lysate vaccine for recurrent ovarian, fallopian tube or primary peritoneal cancer. The sponsor is Abramson Cancer Center at Penn Medicine. The record lists a start date of 2011-04, primary completion of 2012-05 and a terminated status.

Enrollment stopped after the first 3 Phase 1 participants. The primary endpoint was the number of participants with adverse events within 30 days of their last vaccination. The posted Phase 1 results list 3 participants with other adverse events, 0 with serious adverse events and 0 deaths. Phase 2 Arm A and Phase 2 Arm B each have 0 participants, so no Phase 2 comparison is available. Immune response and clinical response are listed as secondary outcomes, but no analyses are reported. The reason for termination and further statistical-analysis details are not reported in the registry record.