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 Vaccine Therapy Following Chemotherapy & Peripheral Stem Cell Transplantation in Treating Non-Hodgkin's Lymphoma (NCT00006478), a na phase 2 study. The primary outcome is Number of Participants With Humoral and Cellular Immune Response.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00006478 |
| Phase | Phase 2 |
| Condition | Lymphoma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 19 |
| Arms in the primary outcome | n/a |
| Lead sponsor | University of Nebraska |
| Results posted | Yes |
2. Clinical Question
The clinical question was whether patients with follicular lymphoma who received an idiotype vaccine after high-dose chemotherapy and autologous stem cell transplantation developed humoral and cellular immune responses. The registry describes the vaccine as being given with keyhole limpet hemocyanin (KLH) and a GM-CSF adjuvant; sargramostim is among the listed interventions.
This was a single-group study, not a comparison of vaccine therapy with a control treatment. The central question was therefore about immune responses observed in participants receiving the post-transplant vaccination approach, rather than a difference between treatment groups. The primary outcome was framed as the number of participants with humoral and cellular immune response.
Population
Patients with follicular lymphoma following high-dose chemotherapy and autologous stem cell transplantation. The registry reports 19 enrolled participants.
Intervention
Idiotype vaccine with KLH and GM-CSF adjuvant, following chemotherapy and transplantation. Listed interventions include autologous tumor cell vaccine, KLH, sargramostim, and adjuvant therapy.
Comparator
There was no separate comparator group. Allocation is listed as not applicable, and the study model is single group.
Primary question
How many participants had humoral and cellular immune responses to the idiotype vaccine?
The registry describes vaccinations as beginning at day 100 or up to 6 months after hematopoietic stem cell transplantation. It does not report a numerical primary outcome result, so the stated question cannot be answered from a reported response count in the registry record.
3. Trial Design
This was a Phase II, single-group trial sponsored by the University of Nebraska. The study title refers to vaccine therapy following chemotherapy and peripheral stem cell transplantation in non-Hodgkin's lymphoma; the official title and primary outcome description specify follicular lymphoma. The registry lists 19 participants as enrolled and gives the status as terminated.
Allocation is listed as not applicable, and masking is listed as none. There is no parallel control arm in the record, so the design does not provide a randomized, like-for-like comparison. The listed intervention components are autologous tumor cell vaccine, keyhole limpet hemocyanin, sargramostim, and adjuvant therapy. The primary outcome description refers to an idiotype vaccine with KLH and GM-CSF adjuvant after high-dose chemotherapy and autologous stem cell transplantation.
The registry gives a start date of 2000-10-11 and a primary completion date of 2008-04-03. The primary outcome timeframe includes measurement before the first immunization, before the 5th, 6th, and 7th immunization series, and 2 weeks after the 7th series, followed by annual measurement until disease progression. The arm description says vaccinations begin at day 100 or up to 6 months after transplantation.
The record states that 30 participants were to be enrolled so that 15 would be evaluable at the end of the immunization process. It also states that, of 19 enrolled, only 12 completed the vaccine series. The trial was halted prematurely, and no formal analysis was completed. The reasons for termination and the reasons participants did not complete the series are 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 | Vaccine Therapy |
|---|---|
| STARTED | 19 |
| COMPLETED | 12 |
| NOT COMPLETED | 7 |
Reasons for not completing this period:
| Reason | Vaccine Therapy |
|---|---|
| Vaccine could not be produced | 1 |
| Physician Decision | 6 |
Analysis population for the primary outcome, as defined in the registry: NO formal analysis was completed as this trial was halted prematurely. Thirty patients were to be enrolled in the protocol so that 15 patients would be evaluable at the end of the immunization process. Of the 19 patients enrolled on the trial, only 12 went on to complete the vaccine series.
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 | Vaccine Therapy |
|---|---|---|
| Participants analysed | 19 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 17 |
| Age, Categorical: >=65 years | Participants | 2 |
| Age, Continuous | years | 46.7 (SD 10.68) |
| Sex: Female, Male: Female | Participants | 10 |
| Sex: Female, Male: Male | Participants | 9 |
| Region of Enrollment: United States | participants | 19 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Participants With Humoral and Cellular Immune Response, measured over immune responses will be obtained prior to first immunization (baseline), prior to the 5th, 6th, 7th immunization series and 2 weeks following administration of the 7th immunization series. And then obtained annually until disease progression.
| Element | Registry entry |
|---|---|
| Result type | N/A |
| Measure of spread | not applicable |
| Unit | not stated |
| Endpoint classification | not reduced to a single comparison |
The primary outcome is the number of participants with humoral and cellular immune response to an idiotype vaccine given with KLH and a GM-CSF adjuvant after high-dose chemotherapy and autologous stem cell transplantation. Humoral and cellular responses represent distinct kinds of immune response, so the endpoint concerns whether participants showed the response types being assessed, not a clinical outcome such as disease progression or survival.
The registry specifies repeated assessment: before the first immunization, before the 5th, 6th, and 7th immunization series, 2 weeks after the 7th series, and annually until disease progression. Repeated measurements can describe the timing and persistence of a measured response, provided the response definitions and results are available. Those definitions, assay details, and time-specific numerical results are not reported in the registry record.
A participant count can be a straightforward way to describe how many people met an outcome definition in a single-group study. To interpret such a count, readers also need to know the number assessed at each time point and how missing assessments were handled. The registry record does not provide those details or a numerical primary outcome result. It states that no formal analysis was completed because the trial was halted prematurely. Thus, the endpoint is identified, but the record does not report an analyzed immune-response count or an estimate of its precision.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies an immune-response outcome but does not report its numerical result. It names the primary endpoint as the number of participants with humoral and cellular immune response, and describes planned assessment times, but it gives no response count, rate, or statistical estimate. Consequently, the posted record does not establish how many participants met either response definition or how responses changed across assessment times.
The record explicitly says that no formal analysis was completed because the trial was halted prematurely. That statement is important: the absence of a formal analysis is not evidence that the vaccine produced no response, nor is it evidence that a response occurred. It means the registered trial record does not supply an analyzed estimate to interpret. Any conclusion about the frequency of immune response would require outcome data and definitions not reported in the registry record.
The enrollment and completion figures describe different stages of participation. The record reports 19 enrolled and says 12 completed the vaccine series. It also states that 30 participants were to be enrolled so that 15 would be evaluable at the end of immunization. These figures do not provide an outcome denominator at each assessment, and they do not establish how many participants were eligible for each immune-response analysis. Reasons for non-completion and the handling of incomplete follow-up are not reported.
Because this was a single-group study with allocation listed as not applicable, it has no concurrent control group in the registry record. Even if an immune-response count were available, a single-group observation would describe participants in this study; it would not by itself estimate a contrast against an untreated or differently treated group. The record also gives no formal analysis, confidence interval, or other measure of uncertainty for the primary outcome.
The safety fields report 0 serious adverse events among 19 at risk and 0 other adverse events among 19 at risk. Those registry figures are descriptive and should not be confused with the missing immune-response analysis. The record does not provide event-specific serious adverse event terms, and the deaths field is not reported. Overall, the registry gives the trial framework and some participation and safety counts, but not the primary outcome results needed to quantify immune response.
9. Statistical Methods Explained
The registry record describes a single-group Phase II study and states that no formal analysis was completed because the trial was halted prematurely. It does not report a statistical analysis plan, a model for the primary outcome, a confidence interval, or a hypothesis test. The concepts below explain how such data are commonly approached in general; they do not describe analyses performed for this trial.
What does a single-group design estimate?
In general, a single-group study can describe outcomes among participants who received the study intervention. For a binary outcome such as whether a participant met a response definition, a basic summary might be the number meeting the definition and the number assessed. That description depends on clear criteria for response and a clearly stated denominator. It does not provide a randomized between-group treatment contrast. Without a concurrent control group, observed changes could not be separated from other influences using a treatment-versus-control comparison within the trial.
Here, the registry identifies the endpoint as a participant count for humoral and cellular immune response, but the numerical outcome and response criteria are not reported in the registry record. It also does not state whether the two response types were combined into one rule, reported separately, or assessed using another prespecified definition. Those details matter because different endpoint rules can yield different counts.
Why do repeated assessment times matter?
The listed timeframe includes baseline, assessments before later immunization series, an assessment 2 weeks after the 7th series, and annual assessments until disease progression. In general, repeated measurements can describe how a response develops or persists over time. They also create practical analysis questions: whether the same participants contribute at every time point, how to handle assessments that are missed, and whether the main comparison is with baseline or between later times. The registry record does not report the analysis approach for these repeated observations, or the number assessed at each time point.
How should uncertainty be understood?
In general, a confidence interval describes the range of values compatible with an estimate under a specified statistical method and its assumptions. It helps show the precision of an estimate; it is not a guarantee that the true value lies in the interval. No confidence interval or numerical primary outcome estimate is reported in the registry record. Therefore, the precision of any immune-response count cannot be evaluated from this record.
For a binary outcome, uncertainty depends in part on how many participants were assessed and how many met the endpoint definition. When only a small group is assessed, estimates can be imprecise. This is a general statistical consideration, not a calculated assessment of this trial: the registry does not give the response count or the analyzed denominator needed to quantify uncertainty.
What does premature termination change?
Early termination can leave fewer participants with complete outcome measurements than intended. The record reports 19 enrolled and 12 who completed the vaccine series, while the stated enrollment plan was 30 participants to obtain 15 evaluable participants at the end of immunization. These are participation figures, not a formal analysis population or a measure of statistical power. The number who completed the series does not reveal how many had usable data at each scheduled assessment.
The registry record does not report why the study was halted, whether any interim analyses were conducted, or whether the intended analysis changed. It says no formal analysis was completed; no additional explanation should be inferred. In general, an incomplete study can have less information than intended, and missing measurements can affect interpretation if participants with and without observed outcomes differ. The record does not describe methods for addressing missing data.
What do planning illustrations mean?
General sample-size planning calculations depend on assumptions such as the outcome being studied, the effect to detect, the significance level, allocation, and statistical method. The planning assumptions associated with this record describe illustrations using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. They are explicitly planning illustrations, not the trial's own design. Since this study is listed as single group and the registry does not report a numerical observed primary effect, such illustrations cannot be treated as evidence about the trial's power or as an analysis of its outcome.
Power is a property of a specified design and assumed effect under a statistical model; it is not a measure of whether the observed result is reliable after the fact. Sample-size illustrations are not observed power. In this record, the planned enrollment and the number who completed vaccination describe the study's progress, but they do not supply an estimate of the probability that a true effect would have been detected.
Finally, multiplicity can arise when multiple outcome types or repeated time points are examined. In general, multiple tests can increase the chance of at least one apparently notable result, so an analysis plan may specify a primary comparison or an adjustment strategy. The registry lists humoral and cellular immune response and multiple assessment times, but does not report a multiplicity strategy or formal testing. No conclusion about adjusted or unadjusted statistical significance can therefore be drawn.
10. Multiplicity, Missing Data and Analysis Populations
The registry describes two kinds of primary immune response—humoral and cellular—and a series of assessment times from baseline through later immunizations and annual follow-up until disease progression. It also lists secondary outcomes concerning vaccine safety, toxicity, and changes in quantitative Bcl-2. With multiple outcomes and repeated measurements, a statistical plan would ordinarily clarify which outcomes and time points are primary for inference and whether any adjustment for multiple testing is intended. Such a plan is not reported in the registry record.
The record states that no formal analysis was completed because the trial was halted prematurely. It reports no analysis population definitions, no test results, and no method for handling multiple outcomes or time points. Whether interim analyses were planned or conducted is not reported in the registry record. The record also does not describe missing-data procedures, such as how participants without a later measurement would contribute to an analysis.
For the primary outcome, 19 participants were enrolled and 12 completed the vaccine series. The protocol description says 30 participants were to be enrolled so that 15 would be evaluable at the end of the immunization process. These figures do not specify the number with outcome data at each time point, and completion of the vaccine series is not itself an analysis-population definition. The reasons for incomplete vaccination or follow-up, the response criteria, and the denominators used for any outcome summaries are not reported in the registry record.
These omissions limit interpretation of both the outcome and its uncertainty. They also prevent determining whether any observed findings were selected from multiple possible analyses. No adjustment, missing-data method, or analysis population should be assumed from the endpoint description alone.
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 |
|---|---|---|---|
| Vaccine Therapy | 0 | 19 | 0.0% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 0% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Vaccine Therapy | 0 of 19 |
The registry's safety figures list 19 participants at risk for serious adverse events and 0 affected. It also lists 19 at risk for other adverse events and 0 affected. These are the posted counts for the Vaccine Therapy group; the record does not provide event-specific terms among the top serious adverse events.
The figures should be read as registry-reported counts, not as a complete account of every aspect of safety monitoring. The record does not describe the collection rules, follow-up duration for these safety counts, or any analysis of risk. The deaths field is not reported in the registry record, so a death count cannot be inferred from the adverse-event figures. Likewise, zero reported events in these fields does not establish that an event was impossible or that the intervention is safe in other settings.
These safety counts are separate from the primary immune-response outcome, for which no numerical result is reported. The trial was terminated prematurely, and the registry says no formal analysis was completed. The displayed adverse-event counts therefore provide limited descriptive information and do not replace a detailed safety analysis.
12. What the Result Does, and Does Not, Mean
The registry record establishes that a Phase II, single-group trial of idiotype vaccination after high-dose chemotherapy and autologous stem cell transplantation was conducted, with 19 participants enrolled. It describes the primary question as whether participants had humoral and cellular immune responses and lists the intended assessment schedule. It also reports that 12 participants completed the vaccine series and that the trial was halted prematurely.
What the record does not establish is the number of participants who had an immune response, the size or persistence of any response, or the precision of an estimate. No numerical primary outcome result or formal analysis is reported. The absence of those results cannot be interpreted as either evidence for or evidence against an immune response.
The study also does not provide a randomized control comparison. As a general matter, a single-group outcome describes what was observed in the participants studied, but cannot alone quantify how outcomes would differ under another treatment or no treatment. The registry record does not support a comparative causal claim.
The safety fields report 0 serious adverse events and 0 other adverse events among 19 at risk, while the deaths field is not reported. These entries are descriptive registry counts, not proof of an absence of risk. The record does not supply enough detail to assess event timing, event types, or completeness of safety follow-up.
Overall, the available record is informative about the study's design, planned immune-response assessments, enrollment, vaccine-series completion, and reported adverse-event counts. It is not sufficient to establish the primary immune-response findings or to make a treatment-effect conclusion. The stated enrollment target and completion figures also do not substitute for outcome data or a formal statistical analysis.
13. Why This Trial Matters Statistically
This trial illustrates a common distinction between an endpoint being specified and an endpoint being reported with results. The registry identifies a concrete primary question: the number of participants with humoral and cellular immune response after vaccination following chemotherapy and autologous transplantation. It also gives a sequence of intended assessment times. Yet the record states that no formal analysis was completed and gives no numerical primary outcome result. Readers therefore have a clearly stated research question but no reported estimate answering it.
The record also makes the difference between enrollment and completion visible. It reports 19 enrolled, 12 completing the vaccine series, and an intended enrollment of 30 to yield 15 evaluable participants at the end of immunization. These are not interchangeable quantities. Enrollment describes entry into the trial, while completing the series describes receipt of the planned vaccination sequence; neither alone specifies how many participants had analyzable data at every outcome time.
Statistically, the single-group design defines what can be learned from the study. It can be used to describe outcomes within the treated group if outcome data are available, but it has no concurrent randomized comparator in the registry record. That limitation is separate from the missing numerical results: even a complete within-group response summary would not create a controlled treatment contrast.
The record is also a reminder that uncertainty and missingness cannot be resolved by the planned sample size alone. Without a reported response count, denominator, and analysis, precision cannot be assessed. The planned enrollment does not establish observed power, and incomplete participation does not reveal the direction or magnitude of any outcome.
These features make the study statistically instructive despite the absence of formal results: design, endpoint definition, follow-up structure, completion, and analysis reporting all shape what readers can conclude.
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.
Several trial-specific limitations follow directly from the registry record. The trial was terminated and no formal analysis was completed. The record reports no numerical result for the primary immune-response outcome, no response definitions, and no assay details. It is therefore not possible to assess the number of participants with humoral or cellular responses or the precision of any such estimate.
The study was single group, with allocation listed as not applicable and masking listed as none. There was no randomized comparator in the registry record. Any within-group response description, if available, would not itself provide a controlled comparison. The registry does not report a formal statistical analysis plan or explain whether interim analyses occurred.
Participation was incomplete relative to the stated plan: 19 participants enrolled, 12 completed the vaccine series, and the record says 30 were to be enrolled so that 15 would be evaluable at the end of immunization. Reasons for early termination and non-completion are not reported in the registry record. Nor does it provide the number assessed at each planned time point, the analysis population, or the handling of missing measurements. These gaps make it difficult to understand follow-up completeness or the denominators that would support an outcome summary.
The primary outcome timeframe extends through annual assessment until disease progression, but the record gives no time-specific results. It also does not explain how multiple response types or repeated assessments would be handled statistically. The safety fields report zero serious and zero other adverse events among 19 at risk, but detailed event terms and the deaths count are not reported. The reported entries therefore cannot substitute for a full safety analysis.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Vaccine Therapy Following Chemotherapy & Peripheral Stem Cell Transplantation in Treating Non-Hodgkin's Lymphoma — NCT00006478.
- Registry record last updated 2023-09-18; status last verified 2023-08.
- Results first posted 2010-11-17.
- 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
The registry record describes NCT00006478, a Phase II, single-group trial sponsored by the University of Nebraska. Its official title concerns idiotype vaccines following high-dose chemotherapy and hematopoietic stem cell transplantation for follicular lymphoma. The study is listed as terminated, with 19 participants enrolled; the record says 12 completed the vaccine series.
The primary outcome was the number of participants with humoral and cellular immune response, assessed at specified immunization time points and annually until disease progression. The registry states that no formal analysis was completed because the trial was halted prematurely, and it reports no numerical primary outcome result. The record lists safety, toxicity, and quantitative Bcl-2 changes as secondary outcomes.
The safety fields show 0 serious adverse events and 0 other adverse events among 19 at risk. Event-specific serious adverse event terms and the deaths count are not reported in the registry record.