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 Bendamustine and Bortezomib Combination Therapy in Indolent Non-Hodgkin's Lymphoma(NHL) (NCT00426855), a na phase 1, phase 2 study. The primary outcome is Optimal Bendamustine Dosage for Further Studies.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00426855 |
| Phase | Phase 1, Phase 2 |
| Condition | Lymphoma, Non-Hodgkin |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 12 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Peter Moosmann |
| Results posted | Yes |
2. Clinical Question
Population
People with relapsed or refractory indolent non-Hodgkin's lymphoma or B-CLL, as described in the official title. The registry record lists non-Hodgkin's lymphoma as the condition and records enrollment of 12 participants.
Intervention
Combination treatment with bendamustine and bortezomib. The registry lists both drugs and describes a combination chemotherapy group.
Comparator
There is no separate comparator group. The study is recorded as single-group, with allocation marked NA and masking recorded as none.
Primary question
The stated primary outcome concerns the optimal bendamustine dosage for further studies, measured in mg/m^2. The registry record does not state a selected dosage or report an outcome value.
The study's stated purpose was to determine whether the bendamustine and bortezomib combination was safe and tolerable in people with indolent non-Hodgkin's lymphoma or chronic lymphocytic leukemia. Its official title narrows the intended population to relapsed or refractory disease and identifies the work as a single-center phase 2 study; the registry phase field lists phase 1 and phase 2.
These details frame a dose-oriented, early-phase question rather than a comparative treatment-effect question. With no comparator group, the record cannot support a direct randomized contrast between the combination and another treatment or control. The primary outcome title suggests that dose selection for later study was the focus, but the registry does not describe the dose-selection procedure or give its result. Accordingly, the central question that can be answered from this record is what was planned and recorded, not whether the regimen achieved a particular clinical benefit.
3. Trial Design
The registry describes a completed, single-group study with no masking. It lists phase 1 and phase 2 and identifies the work in the official title as a single-center study. Enrollment was 12. The listed intervention group received combination chemotherapy with bendamustine and bortezomib; the record does not give a separate control arm. Allocation is marked NA, consistent with the record's single-group design rather than a randomized comparison.
The study's brief summary states that its purpose was to assess whether the combination was safe and tolerable in patients with indolent non-Hodgkin's lymphoma or chronic lymphocytic leukemia. The official title specifies relapsed or refractory indolent non-Hodgkin's lymphoma or B-CLL. The registry condition list names non-Hodgkin's lymphoma. These descriptions are not a substitute for detailed eligibility criteria, which are not reported in the registry record.
The primary outcome is titled “Optimal Bendamustine Dosage for Further Studies.” Its unit is mg/m^2, and the stated timeframe is three weeks after treatment termination. No primary outcome result is listed, and the record contains no analysis records. The registry gives a start of 2007-01 and primary completion of 2009-02, with overall status marked completed. The treatment schedule, dose-escalation or dose-selection rules, and details of any phase transition are not reported in the registry record. The design should therefore be read as a small, noncomparative early-phase investigation, without attributing unrecorded protocol details to it.
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 | Group 1 |
|---|---|
| STARTED | 12 |
| COMPLETED | 12 |
| NOT COMPLETED | 0 |
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 | Group 1 |
|---|---|---|
| Participants analysed | 12 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 4 |
| Age, Categorical: >=65 years | Participants | 8 |
| Sex: Female, Male: Female | Participants | 10 |
| Sex: Female, Male: Male | Participants | 2 |
6. Primary Endpoint
The registry lists the primary outcome as Optimal Bendamustine Dosage for Further Studies, measured over Three weeks after treatment termination.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | mg/m^2 |
| Endpoint classification | not reduced to a single comparison |
The primary outcome is titled “Optimal Bendamustine Dosage for Further Studies,” is expressed in mg/m^2, and has a timeframe of three weeks after treatment termination. Its stated purpose is dose selection for further studies. In early-phase research, dose-focused outcomes can help characterize a regimen for subsequent evaluation; that is a general explanation of the role such an outcome may serve, not a claim about what this study established.
The registry classifies the measure as a number and gives no outcome description or population detail. It does not define how “optimal” dosage was determined, identify a dose-selection criterion, or state a selected dose. No numerical primary outcome result is reported in the registry record. Consequently, the endpoint label communicates the intended focus, but it does not allow readers to determine the result, its uncertainty, or the rule used to reach a dosage decision.
The timeframe places the assessment three weeks after treatment termination. It does not, by itself, establish when the dosage decision was made or how safety and tolerability informed it. Those operational details are not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record supports a limited descriptive reading. This was a completed, single-group study of bendamustine and bortezomib, with enrollment of 12 and a primary outcome focused on optimal bendamustine dosage for further studies. The record does not provide a numerical result for that outcome. It also contains no analysis records, so there is no posted inferential estimate, confidence interval, or hypothesis-test result to interpret.
The absence of a comparator is important. A single-group study can describe what was observed among its participants, but this record does not set up a like-for-like contrast against another treatment or control. It therefore cannot estimate a comparative effect from a randomized comparison. Nor should the dose-oriented endpoint be treated as a measure of clinical benefit: the outcome is framed as dosage selection, and no efficacy outcome result is included in the record.
The safety information records 1 participant with a serious adverse event among 12 at risk. It also records 12 participants with other adverse events among 12 at risk. Thrombocytopenia is listed among the leading serious adverse-event terms at 8.3%. These registry counts describe the recorded categories; they do not provide event timing, duration, severity grading beyond the serious designation, attribution to treatment, or participant-level detail. A count of participants affected is not the same as a count of all events, and the record does not explain the rules used to classify events.
Precision cannot be assessed for the primary outcome because no estimate or interval is posted. The small enrolled cohort and lack of a comparison group also limit the scope of any descriptive interpretation. These are design and reporting considerations, not evidence for or against a treatment effect. The record gives no basis for claiming that the combination works or does not work, or for extending these observations to a broader patient population.
When reading any planning illustrations associated with the analysis, they should be understood as hypothetical calculations under stated assumptions, not as observed power or as a reconstruction of this study's design. The registry does not report its sample-size justification or a statistical analysis plan. The sponsor's own analysis would take precedence if any recalculated result conflicted with it, but no sponsor analysis result is recorded.
9. Statistical Methods Explained
Statistical methods need to be interpreted in light of what the registry actually records. This is a single-group study with allocation marked NA and masking marked none. Enrollment was 12, and the primary measure is a numerical dosage outcome. The record lists no analyses. Thus, the available information describes the design and intended outcome, but not a statistical result for the primary endpoint.
What does a single-group design allow?
In general, a single-group study can summarize observations within the enrolled participants. Without a separate comparator, it does not provide a concurrent treatment contrast. An observed outcome in one group cannot by itself distinguish the effect of the intervention from other explanations, such as the course of illness or features of who entered the study. This general limitation is especially relevant when readers are looking for a comparative treatment effect. The registry's allocation and model entries identify no randomized control comparison, so such a contrast should not be inferred.
The study's stated primary focus is optimal bendamustine dosage for further studies. A dose-selection outcome may be approached through different rules and statistical methods. The record does not state which rule was used, how dose levels were evaluated, or how observations were combined into a selected dosage. It would be inappropriate to reconstruct those methods from the endpoint title alone.
What would an estimate and confidence interval mean?
In general, an estimate is a numerical summary of the data, while a confidence interval describes the statistical uncertainty around an estimate under a specified method and its assumptions. A narrow interval typically indicates greater precision than a wide one, but precision is not the same as clinical importance. The registry record gives neither an estimate nor an interval for the primary outcome. No confidence level or interval method is reported, so neither the magnitude nor the precision of the primary result can be evaluated.
Similarly, a p-value would relate data to a stated statistical hypothesis under a specified analysis. It does not measure the probability that a treatment works, and it does not communicate the size or clinical relevance of an effect. No p-value or hypothesis test is recorded for the primary outcome. The absence of an analysis record should not be interpreted as a nonsignificant result; it means that no inferential result is reported in the registry record.
How should planning calculations be read?
General planning illustrations may show how a sample size would vary under chosen assumptions. The stated assumptions for those illustrations are a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; the target effect is described as a stated fraction of the observed effect. These are planning assumptions, not the documented design of this single-group study. In particular, an equal-allocation, two-arm calculation does not turn this study into a two-arm trial. It is not a report of the study's own sample-size justification.
The stated planning approach distinguishes time-to-event outcomes, for which the illustration gives total events using the Schoenfeld method, from other outcomes, for which it gives participants per arm. These conventions describe how planning quantities are expressed; they do not supply a result for this trial. No planning sample-size values or observed-effect estimate are reported in the registry record, and no sample-size justification from the investigators is reported. Such illustrations are hypothetical planning exercises, never observed power.
In general, over-dispersion means that observed variability exceeds what a particular simple model assumes. If present, it can make uncertainty estimates from that model too small unless the analysis accounts for the extra variation. The registry record does not report a dispersion estimate, a fitted model, or a method for addressing over-dispersion. The primary outcome's measure is listed as a number, but that alone does not establish which probability model or variance assumptions would be suitable.
What can the adverse-event counts establish?
Safety counts are descriptive summaries of participants recorded as affected, not a comparative risk estimate. General statistical comparisons require clearly defined groups and consistent outcome ascertainment. This record has one group, so it offers no concurrent comparator for the adverse-event figures. It also does not report an analysis plan, uncertainty intervals, or adjusted safety comparisons. The figures should be read as counts and a listed term, without adding an inferential interpretation.
The distinction between participants and events matters generally: a participant may experience more than one event, while a registry count of affected participants does not state the total number of events. The record's adverse-event summaries do not describe repeated events or provide detail sufficient to reconstruct event rates over person-time. Therefore, calculations beyond the recorded counts and percentage would require unsupported assumptions.
Overall, the statistical methods used to select a dosage, summarize the primary outcome, handle missing values, and conduct any inferential analyses are not reported in the registry record. General concepts can explain what such methods would mean, but they cannot fill those reporting gaps or recover an unposted result.
10. Multiplicity, Missing Data and Analysis Populations
The registry record contains no analysis records and does not report a statistical analysis plan. It therefore does not describe whether multiple outcomes, dose levels, subgroups, or repeated assessments were considered, or whether any multiplicity adjustment was planned or used. No adjustment method or family of hypotheses is reported. The absence of this information is not evidence that multiplicity was addressed, nor does it establish that multiple testing occurred.
Missing-data handling is also not reported in the registry record. There is no description of how participants without an endpoint measurement would be treated, whether any imputation was planned, or how withdrawals and incomplete follow-up were handled. Enrollment of 12 is stated, but the record does not provide a primary-endpoint analysis population or the number contributing data to the dosage outcome. It would be inappropriate to assume that every enrolled participant was analyzed for the primary measure.
The record does not define an intention-to-treat population, a per-protocol population, or a safety analysis population. Nor does it report rules for excluding participants or observations. These omissions matter because analysis populations and missing-data decisions can affect a study's reported estimate and its interpretation. Without those details, the primary endpoint cannot be independently reconstructed from the registry record.
The single-group design also limits the relevance of multiplicity concepts tied to comparisons between arms; it does not eliminate the possibility of multiple dose decisions or outcomes. Since those details are unavailable, readers should not infer a prespecified hierarchy, adjustment strategy, or complete analysis framework.
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 |
|---|---|---|---|
| Group 1 | 1 | 12 | 8.3% |
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 |
|---|---|
| Group 1 | 12 of 12 |
Most frequent serious adverse event terms
| Event term | Organ system | Group 1 |
|---|---|---|
| Thrombocytopenia | Blood and lymphatic system disorders | 1 of 12 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Group 1 |
|---|---|---|
| Diarrhea | Gastrointestinal disorders | 10 of 12 |
| Nausea | Gastrointestinal disorders | 8 of 12 |
| Vomiting | Gastrointestinal disorders | 6 of 12 |
| Fatigue | Psychiatric disorders | 6 of 12 |
| Leukopenia | Blood and lymphatic system disorders | 5 of 12 |
| Dehydration | Cardiac disorders | 2 of 12 |
| Neuropathy, sensory | Nervous system disorders | 2 of 12 |
| Neutropenia | Blood and lymphatic system disorders | 2 of 12 |
| Stomatitis | Gastrointestinal disorders | 1 of 12 |
| Neuropathy, motor | Nervous system disorders | 1 of 12 |
Serious events with the highest arm-level rate: thrombocytopenia (8.3%).
The registry records 12 participants at risk for serious adverse events and 1 participant affected. For other adverse events, it records 12 at risk and 12 affected. Thrombocytopenia is listed among the leading serious adverse-event terms at 8.3%. These are descriptive registry entries for the single group, not comparisons between treatment arms.
Counts of affected participants do not necessarily equal counts of events: a person may have more than one event, and the record does not state the total event count. It also does not give event-level timing, duration, severity grades other than the serious classification, or whether events were judged related to treatment. The meaning and scope of the “other” adverse-event category are not explained in the registry record. The percentage for thrombocytopenia should be read as the listed registry figure, not as a comparative estimate.
These entries provide a brief account of recorded safety categories, but no confidence intervals or formal safety analysis are reported. With no comparator group, they cannot show how these counts compare with another treatment or control. They also do not establish whether the combination is safe or unsafe; they describe only the safety information recorded for this study.
12. What the Result Does, and Does Not, Mean
The record establishes that a completed study evaluated bendamustine and bortezomib as a combination in a single group, with an enrollment of 12. Its stated purpose concerned safety and tolerability, and its primary outcome was framed as optimal bendamustine dosage for further studies. The registry reports selected adverse-event counts, including 1 participant affected by a serious adverse event among 12 at risk, and 12 affected by other adverse events among 12 at risk.
It does not report the selected dosage, a primary outcome value, an estimate of uncertainty, or an inferential analysis. Therefore, the principal dose-focused question cannot be answered from the recorded results. The adverse-event summaries also lack enough detail for an assessment of event timing, attribution, recurrence, or comparative safety. Their presence in the registry should not be mistaken for a complete clinical results report.
The design does not include a separate control group. Consequently, the record does not provide a randomized comparison or an estimate of how outcomes differ from another treatment. It cannot establish a treatment effect, whether favorable or unfavorable. The endpoint's focus on dosage for later studies is distinct from an endpoint demonstrating clinical benefit.
These boundaries do not erase the study's descriptive value: the record documents the study's stated purpose, design features, enrollment, endpoint framing, and reported safety counts. But unanswered questions remain, including how dosage was selected, which participants contributed primary-outcome data, how missing data were handled, and what the numerical primary result was. Those details are not reported in the registry record. A cautious reading separates what is recorded from what would require a fuller results report.
13. Why This Trial Matters Statistically
This study is statistically notable for the contrast between a dose-oriented early-phase question and the limited results recorded in the registry. The primary outcome is explicitly framed around identifying an optimal bendamustine dosage for further studies. For that question to be interpretable, readers would need the selected dosage, the decision rule, and information about the observations used. The registry record does not report those details or a numerical endpoint result.
The single-group design makes the scope of inference clear. It can provide descriptive observations in the enrolled cohort, but it is not a randomized, like-for-like comparison. This distinction helps prevent a dose-selection study from being read as evidence of comparative treatment benefit. The lack of a control group is a design feature, not a statistical flaw by itself; whether the design addresses its intended question depends on methods and results that are not reported in the registry record.
The small enrollment of 12 also puts emphasis on transparent reporting of uncertainty and participant flow. The record does not provide a primary-outcome estimate, confidence interval, analysis population, or missing-data approach. Those omissions leave readers unable to judge precision or reproduce the primary analysis. The safety counts offer a limited descriptive view, but they lack contextual and event-level detail.
Finally, planning calculations must remain distinct from study results. Hypothetical assumptions about two-arm allocation and detection of fractions of an observed effect are not the design or sample-size justification for this single-group trial. Separating planning illustrations from observed evidence is essential to avoid treating a modeled scenario as a result from the enrolled participants.
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 limitations arise specifically from the registry information for this study. The enrollment was 12, and the design is recorded as single-group, with no masking and allocation marked NA. There is no separate comparator group. This precludes a direct randomized comparison within the study and restricts interpretation to the reported group-level observations.
The main limitation for evaluating the primary question is that the record names an optimal dosage outcome but supplies no selected dose or numerical result. It does not explain the dose-selection rule, provide the primary outcome description, or identify the population contributing to the endpoint. The timeframe is stated as three weeks after treatment termination, but the record does not explain the assessment procedures or the relationship between that timing and the selection decision.
The record contains no analysis entries and does not report a statistical analysis plan, confidence interval, hypothesis test, or sample-size justification. Missing-data methods, analysis populations, multiplicity procedures, and details of any dose-level comparisons are likewise not reported in the registry record. These gaps prevent assessment of the methods behind the intended outcome and preclude independent reconstruction of the analysis.
Safety reporting is also limited to summarized counts and a listed serious adverse-event term. The record does not provide event-level detail, attribution, timing, duration, or a comparison group. The study's official title names relapsed or refractory indolent non-Hodgkin's lymphoma or B-CLL, while the condition list names non-Hodgkin's lymphoma; detailed eligibility criteria are not reported. Generalizability should therefore not be assumed beyond the population described in the record. These limitations identify what cannot be established from this registry entry; they do not imply a particular unreported result.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Bendamustine and Bortezomib Combination Therapy in Indolent Non-Hodgkin's Lymphoma(NHL) — NCT00426855.
- Registry record last updated 2015-04-03; status last verified 2015-04.
- Results first posted 2015-04-03.
- 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 identifies NCT00426855 as a completed phase 1/2, single-center study of bendamustine and bortezomib in relapsed or refractory indolent non-Hodgkin's lymphoma or B-CLL. It records a single group, no masking, enrollment of 12, a start of 2007-01, and primary completion of 2009-02.
The stated study purpose was to assess safety and tolerability. The primary outcome concerns optimal bendamustine dosage for further studies, in mg/m^2, at three weeks after treatment termination. No primary outcome result or analysis record is reported. The registry lists 1 participant with a serious adverse event among 12 at risk, and 12 participants with other adverse events among 12 at risk; thrombocytopenia is listed at 8.3% among the leading serious adverse-event terms.
The record does not report a comparator, a selected dosage, a statistical analysis plan, sample-size justification, missing-data methods, or a primary endpoint estimate. Its findings therefore cannot be interpreted as a comparative treatment effect.