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 Universal Granulocyte Macrophage-colony Stimulating Factor (GM-CSF)-Producing and GM.CD40L for Autologous Tumor Vaccine in Mantle Cell Lymphoma (NCT00101101), a na phase 2 study. The primary outcome is Rate of Immunological Response to Vaccination.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00101101 |
| Phase | Phase 2 |
| Condition | Lymphoma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 43 |
| Arms in the primary outcome | n/a |
| Lead sponsor | H. Lee Moffitt Cancer Center and Research Institute |
| Results posted | Yes |
2. Clinical Question
Population
The trial concerned patients with relapsed or de novo stage II, stage III, or stage IV mantle cell lymphoma. The primary endpoint population was participants who received at least one vaccine injection. The registry record does not report how many participants met that endpoint-specific definition.
Intervention
The study evaluated an autologous tumor cell-based vaccine formulated with a universal GM-CSF-producing and CD40L-expressing bystander cell line, together with IL-2, after combination chemotherapy. The registry describes conventional therapy that could include chemotherapy with or without rituximab, with type and duration at the individual clinician’s discretion.
Comparator
This was a single-group study, not a randomized comparison. The allocation field is recorded as NA, and there is no like-for-like control group in the registry record. Consequently, the record does not support a direct comparison of vaccine treatment with an alternative treatment or with no vaccine.
Primary question
The primary question was whether participants showed an immunological response to vaccination, assessed using interferon gamma secretion by peripheral blood mononuclear cells, delayed type hypersensitivity (DTH) reactions to irradiated autologous tumor cells, and lymphocyte accumulation at DTH and vaccine injection sites. The endpoint timeframe was 4 months per participant. The registry record describes these assessments but does not report the response count or rate.
3. Trial Design
The registry identifies this as a phase II, single-group trial of an autologous tumor cell-based vaccine for patients with mantle cell lymphoma. Enrollment is recorded as 43, and the status is completed. The recorded start was 2004-07 and primary completion was 2012-10. These dates describe the trial record; they do not establish how long any individual participant was followed.
The stated study population included patients with relapsed or de novo stage II, stage III, or stage IV mantle cell lymphoma. The primary endpoint population was narrower in one specific respect: participants had to receive at least one vaccine injection. The number meeting that definition is not reported in the registry record.
The treatment description places vaccine therapy and IL-2 after combination chemotherapy. The record describes 3-6 cycles of chemotherapy with or without rituximab, at the discretion of the individual clinician. It lists cyclophosphamide, doxorubicin, vincristine, prednisone, and dexamethasone among the drug interventions. The arm description includes 6 courses of CHOP or 3 courses of hyperfractionated chemotherapy, but the latter description is incomplete in the registry record. The full regimen received by each participant is not reported.
Masking is recorded as none. Because this is a single-group study and not a randomized comparison, the results cannot provide a randomized estimate of how outcomes differ from a control treatment. The registry lists secondary outcomes of related serious adverse events and median event-free survival, in addition to the primary immunological-response outcome. Outcome results beyond the reported adverse-event counts 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 and Conventional Therapy |
|---|---|
| STARTED | 43 |
| COMPLETED | 23 |
| NOT COMPLETED | 20 |
Reasons for not completing this period:
| Reason | Vaccine and Conventional Therapy |
|---|---|
| never received vaccine | 20 |
Analysis population for the primary outcome, as defined in the registry: Participants who received at least one vaccine injection.
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: All participants who started the study.
| Characteristic | Unit | Vaccine and Conventional Therapy |
|---|---|---|
| Participants analysed | 43 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 18 |
| Age, Categorical: >=65 years | Participants | 25 |
| Age, Continuous | participants | 65 (47 to 81) |
| Sex: Female, Male: Female | Participants | 9 |
| Sex: Female, Male: Male | Participants | 34 |
| Region of Enrollment: United States | participants | 43 |
6. Primary Endpoint
The registry lists the primary outcome as Rate of Immunological Response to Vaccination, measured over 4 months per participant.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint was the rate of immunological response to vaccination, with the measure recorded as participants. The registry defines the endpoint using several immune assessments: in vitro interferon gamma secretion by peripheral blood mononuclear cells, delayed type hypersensitivity (DTH) reaction to irradiated autologous tumor cells, and lymphocyte accumulation at DTH and vaccine injection sites. The recorded timeframe was 4 months per participant.
The assessment description specifies DTH skin testing within 2 weeks before the first vaccine and again after the fourth vaccine. Irradiated autologous tumor cells were injected intradermally, and the injection site was inspected 48 hours later for induration and erythema. Biopsies were used to assess granulocytic and lymphocytic accumulation. The description records a 3mm punch biopsy, with the vaccine-site biopsy obtained 2-5 days after the second vaccine. A pathologist graded cell accumulation at the sites.
As a general statistical concept, an immunological endpoint can indicate whether a measurable immune response was observed under the endpoint’s specified criteria. It is not automatically equivalent to a clinical outcome such as event-free survival. The registry record does not report the response count, rate, or criteria used to combine the listed assessments into an overall response classification. The endpoint’s observed result and its precision therefore cannot be evaluated from the reported record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central interpretive limitation is that the registry record describes the primary endpoint but does not report its result. It gives no number of participants classified as having an immunological response, no response rate, and no uncertainty estimate for that endpoint. The outcome’s assessment procedures are detailed, but procedural detail cannot substitute for the result itself.
The record reports enrollment of 43. That enrollment figure should not be treated as the primary endpoint denominator: the endpoint population is defined as participants who received at least one vaccine injection, and the number in that population is not reported. It is therefore not possible to determine the endpoint denominator from the record, or to assess whether all enrolled participants contributed endpoint data.
The trial was single group, with allocation recorded as NA. It has no randomized control group for a direct comparison. As a general statistical principle, observed outcomes in an uncontrolled study may reflect the studied treatment and other influences, including the course of the underlying disease, other treatment, participant selection, and measurement. The registry record does not provide a controlled estimate that separates these influences.
The record does give adverse-event counts for the Vaccine and Conventional Therapy group: 2 participants with serious adverse events among 43 at risk, and 11 participants with other adverse events among 43 at risk. These counts describe the recorded group; they do not establish that events were caused by the vaccine, chemotherapy, or IL-2. The secondary outcome is named as occurrence of related serious adverse events, but the group counts alone do not provide a detailed attribution analysis. The registry also lists Leukocyte, Granulocyte, and Cough among the top serious-event terms, each with a reported 2.3%.
Median event-free survival is listed as a secondary outcome, but its result, follow-up detail, and uncertainty are not reported in the registry record. The status marked completed does not mean that every outcome result is available in the record. Overall, the posted information supports a description of the study design, endpoint procedures, and certain event counts, but not an estimate of immunological response or a comparative treatment effect.
9. Statistical Methods Explained
Statistical methods determine how outcomes are summarized, how comparisons are made, and how uncertainty is represented. The registry record identifies a phase II, single-group study and lists outcome measures, but it does not report the statistical analysis methods used for the primary outcome. The intended response definition, treatment of incomplete assessments, and method for estimating uncertainty are not reported in the registry record.
What does a single-group design allow?
This study has one registered group, titled Vaccine and Conventional Therapy, and is not a randomized comparison. In general, a single-group design can describe outcomes among participants who received the study regimen. It cannot by itself estimate what those same participants’ outcomes would have been under a different regimen. A control group helps distinguish changes associated with an intervention from changes that might occur for other reasons; random assignment is one method of making groups comparable at baseline. Neither a randomized allocation nor a control group is described for this trial.
The treatment description also allows variation in conventional therapy: chemotherapy with or without rituximab, with type and duration at the individual clinician’s discretion. In general, variation in concurrent care can complicate interpretation of outcomes attributed to one component of a treatment strategy. The record does not describe an analysis that accounts for differences in chemotherapy or other treatment received.
How should a response rate be understood?
In general, a response rate is a proportion: the number meeting a response definition relative to the number assessed in the relevant population. The registry defines the primary population as participants who received at least one vaccine injection, but does not report the size of that population or the number classified as responders. Thus, a primary response rate cannot be read from the registry record. The definition also combines several kinds of immune assessment in its description. How those measures were used to determine an overall response is not reported in the registry record.
A confidence interval is a statistical range expressing uncertainty around an estimate under a specified method. For a response rate, it can help show how much precision the observed count and denominator support. No confidence interval for the primary endpoint is reported in the registry record. Without the response count, denominator, and interval, the magnitude and precision of an estimated response cannot be assessed.
What do the planning assumptions mean?
The registry’s planning illustration uses a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It describes detectable effects as a stated fraction of an observed effect. These are planning assumptions, not observed trial outcomes. The illustration’s two-arm allocation assumption does not describe this trial’s single-group design, and the registry does not report the trial’s own sample-size justification. Any sample-size illustration should be read as a hypothetical planning calculation, not as evidence that the trial had a particular probability of detecting an effect. It is not observed power.
In general, a significance level is a threshold used in a specified testing procedure; it does not measure the probability that a treatment works. A two-sided procedure considers departures in either direction. The registry record does not report a primary-endpoint hypothesis test, p-value, or confidence interval, so no inferential result for immunological response can be interpreted from the stated planning assumptions.
Why can event counts differ from event totals?
For binary outcomes, counts describe participants with an event, rather than a continuous measurement such as a mean. A participant can potentially experience more than one event type, so counts for categories should not automatically be added to infer a total number of affected participants. The serious and other adverse-event figures are presented as participant counts in the registered group. The registry does not report enough detail to establish how event categories overlap or how each event was attributed.
Over-dispersion is a general issue for count data when variability exceeds what a chosen probability model expects. It can make standard errors and confidence intervals too narrow if the model does not allow for that extra variability. That concept does not establish that over-dispersion occurred in this trial: the registry record does not report a count model or a statistical analysis of event counts. The listed adverse-event counts should therefore be read descriptively.
Likewise, time-to-event analyses generally use event times and account for censoring, when follow-up ends before the event is observed. Event-free survival is listed as a secondary outcome, but the registry record does not report the analysis method, number of events, follow-up distribution, or median result. No time-to-event estimate can be reconstructed from the information recorded.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists three outcome measures, including the primary immunological-response endpoint and secondary outcomes of related serious adverse events and median event-free survival. It records no analyses. The statistical plan for choosing among outcomes, handling multiple tests, or defining any hierarchy is not reported in the registry record.
Multiplicity is a general concern when several outcomes or several statistical tests are examined. Repeated testing can increase the chance of a seemingly notable result arising by chance unless the analysis plan accounts for the testing strategy. This general concern does not show that such testing occurred in this trial. The record does not provide results or a multiplicity procedure from which to assess that issue.
The primary endpoint population is participants who received at least one vaccine injection. The number meeting that definition, the number with complete immune assessments, and the number included in any analysis are not reported in the registry record. It is not reported how missing assessments were handled, whether participants were excluded from endpoint summaries, or whether any missing values were imputed.
In general, missing-data methods matter because results can depend on whether participants with incomplete measurements differ from those with complete measurements. The registry record does not report reasons for missing endpoint data or an analysis of their possible effect. The record also does not describe an intention-to-treat population or another analysis population for the primary endpoint. These omissions limit assessment of who contributed to the outcome and how robust any estimated response might be.
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 and Conventional Therapy | 2 | 43 | 4.7% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 5% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Vaccine and Conventional Therapy | 11 of 43 |
Most frequent serious adverse event terms
| Event term | Organ system | Vaccine and Conventional Therapy |
|---|---|---|
| Leukocyte | Blood and lymphatic system disorders | 1 of 43 |
| Granulocyte | Blood and lymphatic system disorders | 1 of 43 |
| Cough | Respiratory, thoracic and mediastinal disorders | 1 of 43 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Vaccine and Conventional Therapy |
|---|---|---|
| Injection site reaction/extravasation changes | Skin and subcutaneous tissue disorders | 11 of 43 |
| Fatigue (asthenia, lethargy, malaise) | General disorders | 8 of 43 |
| Hemoglobin | Blood and lymphatic system disorders | 6 of 43 |
| Pain - Joint | General disorders | 4 of 43 |
| Platelets | Blood and lymphatic system disorders | 3 of 43 |
| Cough | Respiratory, thoracic and mediastinal disorders | 3 of 43 |
| Fever (in the absence of neutropenia) | General disorders | 2 of 43 |
| Neutrophils/granulocytes | Blood and lymphatic system disorders | 2 of 43 |
| Leukocytes (total WBC) | Blood and lymphatic system disorders | 2 of 43 |
| Pain - Head/headache | General disorders | 2 of 43 |
Serious events with the highest arm-level rate: leukocyte (2.3%), granulocyte (2.3%), cough (2.3%).
The registry reports 2 participants with serious adverse events among 43 at risk in the Vaccine and Conventional Therapy group. It also reports 11 participants with other adverse events among 43 at risk. These are participant counts, not independently calculated rates. The registry’s listed top serious-event terms are Leukocyte, Granulocyte, and Cough, each reported as 2.3%.
The secondary outcome is described as the occurrence of related serious adverse events. However, the figures do not provide event-level detail or show how investigators judged each event’s relationship to treatment. They should not be interpreted as proof that a particular intervention caused an event. The record does not report deaths for the group, so death information is not reported in the registry record.
As a general statistical caution, adverse-event term counts may not represent mutually exclusive groups: one participant may have more than one type of event. The registry record does not describe overlap among terms or provide a distinct-participant total by event category. The counts are therefore best read as recorded safety summaries, not added together or treated as a causal comparison. Because the study has no control group, these figures do not quantify how event frequency differs from another treatment or background risk.
12. What the Result Does, and Does Not, Mean
The registry record establishes that a completed phase II, single-group trial evaluated an autologous tumor cell-based vaccine with GM.CD40L and IL-2 after conventional therapy for mantle cell lymphoma. It describes an immunological primary endpoint and the assessments used to evaluate it. It also records adverse-event counts for the Vaccine and Conventional Therapy group.
The primary result needed to answer whether participants met the immunological-response endpoint is not reported in the registry record. Neither the response count nor the response rate is available, and the endpoint-specific denominator is not reported. Consequently, the record does not establish the observed magnitude or precision of an immunological response. The listed procedures explain what investigators assessed, not what the assessments found.
The adverse-event counts document events in the recorded group, but do not establish cause. The trial’s single-group design also means that the record cannot provide a randomized treatment comparison. Descriptive outcomes from a group exposed to a treatment regimen do not isolate the treatment’s effect from other influences. The registry record does not report event-free survival results, so that secondary outcome cannot be interpreted from the available information either.
As a general distinction, an immune measurement is not interchangeable with a clinical endpoint. An immune response can be a biological observation, while an outcome such as event-free survival concerns a participant’s clinical course over time. The registry lists both kinds of outcome but provides no reported result for either in the record.
Accordingly, the posted information supports a cautious account of the trial’s design, endpoint procedures, and safety counts. It does not establish that treatment improves clinical outcomes, nor does it establish the absence of an effect. Those conclusions would require outcome results and, for comparative claims, an appropriate comparison. The registry record does not report those results.
13. Why This Trial Matters Statistically
This trial is statistically notable for the distinction between a detailed endpoint description and an endpoint result. The record specifies how several immune features were assessed, including interferon gamma secretion, DTH testing, and tissue-cell accumulation. Yet it does not report the number meeting the primary response definition or the associated rate. This illustrates why measurement procedures and observed estimates answer different questions.
It also illustrates how the analysis population affects interpretation. Although enrollment is recorded as 43, the primary outcome applies to participants who received at least one vaccine injection. The record does not state how many participants met that definition. Enrollment alone cannot therefore serve as a confirmed denominator for the primary endpoint.
The single-group design matters for causal interpretation. Without a randomized control group, observed immune and safety outcomes are descriptive of the study group rather than estimates of a difference caused by the vaccine strategy. The treatment description also includes clinician discretion in chemotherapy type and duration, adding variation to the treatment context described in the record.
Finally, the record distinguishes a planning illustration from a trial’s observed statistical evidence. Planning assumptions can help frame hypothetical study design questions, but they are not trial results and do not substitute for an estimate with uncertainty. In this case, the listed planning assumptions include a two-arm framework even though the registered trial is single group. Recognizing that difference prevents a planning calculation from being mistaken for a result from this trial.
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 primary limitation is the absence of reported primary endpoint results. The registry describes the immunological assessments but does not report the response count, response rate, or uncertainty around an estimate. The number of participants who received at least one vaccine injection is also not reported, preventing confirmation of the endpoint denominator.
The registered design is single group, with allocation recorded as NA. There is no like-for-like control comparison, so the record cannot estimate a treatment difference against a comparator. The arm description indicates that chemotherapy type and duration could be at the individual clinician’s discretion and that chemotherapy could be given with or without rituximab. The complete treatment received by each participant is not reported; the description of one chemotherapy option is incomplete in the registry record.
The registry does not report how the listed immune assessments were combined into an overall response classification, how missing measurements were handled, or which participants were included in an analysis. It also does not report a statistical analysis plan, uncertainty estimates, or analysis methods for the primary endpoint. These gaps prevent evaluation of the endpoint definition’s operational details and the precision of any result.
For safety, the record provides serious and other adverse-event participant counts and several serious-event terms, but not detailed event-level attribution or category overlap. Death information is not reported in the registry record. Event-free survival is listed as a secondary outcome, but its result and analysis details are not reported. The registry’s completed status does not resolve these reporting gaps.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Universal Granulocyte Macrophage-colony Stimulating Factor (GM-CSF)-Producing and GM.CD40L for Autologous Tumor Vaccine in Mantle Cell Lymphoma — NCT00101101.
- Registry record last updated 2023-08-22; status last verified 2023-08.
- Results first posted 2013-09-04.
- 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
NCT00101101 is a completed phase II, single-group trial of an autologous tumor cell-based vaccine using a GM-CSF-producing and CD40L-expressing bystander cell line, with IL-2 after conventional therapy, in mantle cell lymphoma. Enrollment is recorded as 43. The primary endpoint was immunological response to vaccination, assessed over 4 months per participant among those who received at least one vaccine injection.
The registry describes immune-assessment procedures but does not report the primary response result or endpoint denominator. It lists secondary outcomes of related serious adverse events and median event-free survival; the latter result is not reported in the registry record. The Vaccine and Conventional Therapy group has 2 participants with serious adverse events and 11 with other adverse events, among 43 at risk. The single-group design does not provide a randomized comparison.