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 Rituximab Plus Lenalidomide in Patients With Mucosa Associated Lymphoid Tissue (NCT01611259), a na phase 2 study. The primary outcome is Objective Responses in Patients With MALT Lymphoma Presenting With Measureable Disease.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01611259 |
| Phase | Phase 2 |
| Condition | Lymphoma of the Mucosa Associated Lymphoid Tissue (MALT) |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 50 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Arbeitsgemeinschaft medikamentoese Tumortherapie |
| Results posted | Yes |
2. Clinical Question
Population
Patients with lymphoma of the mucosa-associated lymphoid tissue (MALT) type and measurable disease. The registry describes 50 participants enrolled; 48 were included in the safety assessment. Two participants received treatment but had no efficacy assessment.
Intervention
Rituximab plus lenalidomide in an open-label, single-group phase II study. The regimen describes 28-day cycles, with rituximab on day 1 and lenalidomide taken daily for 21 days. The number of cycles depended on response or disease status.
Comparator
There was no concurrent comparator group. The registry describes allocation as not applicable and masking as none. The response-rate thresholds in the study description are benchmarks for interpreting the combination, not results from a control arm.
Primary question
Among patients with measurable MALT lymphoma, what proportion had an objective response by the 40-week assessment? The registry describes a response rate below 40% as grounds for rejecting the combination as ineffective and a minimum response rate of 60% as the definition of an active combination. The observed response count and response proportion are not reported in the registry record, so the study’s result against those benchmarks cannot be determined from the posted results.
3. Trial Design
This was an open-label, phase II, single-group study of rituximab plus lenalidomide in patients with MALT lymphoma and measurable disease. The registry lists enrollment of 50 and describes no random allocation or masking. With no concurrent control group, the study was designed to assess responses in participants receiving the combination, not to estimate a randomized treatment contrast.
The regimen description specifies 28-day cycles. Rituximab was given intravenously at 375 mg/m² on day 1, and lenalidomide was taken orally at 20 mg daily for 21 days. The description assigns 6 cycles to patients with complete response and 8 cycles to participants with stable disease or partial remission. The registry does not report further treatment-delivery details.
The study start is listed as 2012-05, primary completion as 2014-05, and status as completed. These dates describe the registry record; they do not establish when individual participants were assessed or how long each participant was followed.
Safety assessment included 48 participants. The registry says two participants received treatment but had no efficacy assessment and were excluded from both intention-to-treat and per-protocol efficacy analyses. The reason those assessments were unavailable, and the criteria used to define those analysis populations, are not reported in the registry record. No detailed statistical analysis plan, sample-size justification, or interim-analysis plan is reported there.
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: 2 patients were withdrawn before first response evaluation and were replaced. 2 patients withdrew their consents prior to study treatment and did not experience AEs to other study procedures. These 2 patients were therefore excluded from intent-to-treat (ITT) efficacy assessments, safety assessments and listing of baseline characteristics.
Overall Study
| Stage | Rituximab and Lenalidomide |
|---|---|
| STARTED | 48 |
| COMPLETED | 34 |
| NOT COMPLETED | 14 |
Reasons for not completing this period:
| Reason | Rituximab and Lenalidomide |
|---|---|
| Physician Decision | 1 |
| Lack of Efficacy | 2 |
| Adverse Event | 4 |
| Withdrawal by Subject | 7 |
Analysis population for the primary outcome, as defined in the registry: 48 patients were included into safety assessment, two of them received treatment but no efficacy assessment was available. Therefore, these two patients were neither included into Intention-to-treat nor Per-protocol efficacy analysis.
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: Two of the enrolled patients withdrew their consent prior to study treatment and did not experience adverse events due to other study procedures. These 2 patients were therefore excluded from intent-to-treat (ITT) and per-protocol (PP) efficacy assessments, safety assessments and listing of baseline characteristics.
| Characteristic | Unit | Rituximab and Lenalidomide |
|---|---|---|
| Participants analysed | 48 | |
| Age, Continuous | years | 65 (33 to 85) |
| Sex: Female, Male: Female | Participants | 29 |
| Sex: Female, Male: Male | Participants | 19 |
6. Primary Endpoint
The registry lists the primary outcome as Objective Responses in Patients With MALT Lymphoma Presenting With Measureable Disease, measured over 40 weeks.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the number of participants with objective responses among patients with measurable MALT lymphoma. The study description frames the main question as the proportion of patients responding to rituximab plus lenalidomide, with assessment at 40 weeks. An objective response endpoint classifies participants according to whether their disease meets prespecified response criteria; the particular criteria and assessment procedures are not reported in the registry record.
A response proportion is used in a single-group phase II study to describe how often the defined outcome occurred among the assessed participants. It can be compared with a threshold specified in the study description, but it does not by itself show how outcomes would have differed under another treatment or no treatment. The registry’s stated benchmarks are a response rate below 40% for rejecting the combination as ineffective and a minimum response rate of 60% for defining an active combination.
The response count, response proportion, and uncertainty around the estimate are not reported in the registry record. Therefore, the posted results do not establish whether the observed response rate met either benchmark. The primary endpoint’s title refers to participants with measurable disease, while the registry also notes that two treated participants had no efficacy assessment and were excluded from both efficacy analysis populations.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry describes a phase II study intended to evaluate objective responses to rituximab plus lenalidomide in patients with measurable MALT lymphoma. Its primary question is a single-group response-rate question, with the study description identifying below 40% and a minimum of 60% as interpretive thresholds. These thresholds are not observed findings. The response count and response proportion are not reported in the registry record, so it is not possible to determine whether the study crossed either threshold.
The design has no concurrent comparison group. Any response proportion, if reported, would describe outcomes among participants assessed while receiving this regimen. It would not isolate the effect of the combination from other influences or establish what would have happened to comparable participants under a different treatment. The registry lists no random allocation and no masking. This should be kept in mind when interpreting any efficacy result that might be reported elsewhere; the posted results do not supply such a result.
The enrollment figure is 50, while the safety assessment included 48 participants. The registry states that two participants received treatment but had no efficacy assessment and were excluded from both intention-to-treat and per-protocol efficacy analyses. This distinction matters because safety and efficacy summaries do not necessarily use the same analysis population. The record does not explain why the efficacy assessments were unavailable, whether the missing assessments were related to participants’ outcomes, or how missing endpoint information was handled beyond the exclusions stated.
Safety findings are reported for the 48 participants in the safety assessment: 18 were affected by serious adverse events, and 47 were affected by other adverse events. Those counts describe the posted safety data, not treatment effects compared with another group. The listed serious-event terms have reported percentages, but the registry does not report a comparative safety analysis. A death count is not reported in the registry record.
The registry indicates 3 outcome measures and 0 analyses. It does not report a response estimate, confidence interval, hypothesis-test result, or multiplicity procedure. Consequently, statistical precision and the strength of evidence relative to the study’s response-rate thresholds cannot be evaluated from the posted results. The absence of these results is not evidence that the response rate was high or low; it means the efficacy question cannot be answered from this record.
9. Statistical Methods Explained
The statistical interpretation begins with the study design. This is a single-group phase II study, rather than a randomized comparison. In a single-group design, the principal efficacy summary is usually the observed proportion of assessed participants who meet the outcome definition. That proportion describes the study participants and endpoint as analyzed. It does not estimate a treatment difference against a concurrent control group. No response proportion or response count is reported in the registry record.
What does a response proportion represent?
For a binary endpoint, each participant is classified as having or not having the defined outcome. The response proportion is the number meeting the response definition divided by the analysis population for that endpoint. The registry identifies the endpoint as objective responses in participants with measurable disease and gives a 40-week timeframe, but it does not report the numerator, the denominator used for the efficacy estimate, or the response criteria. As a result, no response proportion can be interpreted from the posted results.
In general, a confidence interval describes the uncertainty around an estimated quantity under the statistical model used. For a response proportion, it would indicate a range of values compatible with the observed data and method, rather than guarantee that the true value lies in that range. No confidence interval or method for calculating one is reported in the registry record. Without an estimate and its uncertainty, it is not possible to assess how precisely the response rate was measured or how clearly it compares with the stated thresholds.
How should the response-rate benchmarks be read?
The primary-outcome description states that a response rate below 40% would lead to rejecting the combination as ineffective, and defines an active combination at a minimum response rate of 60%. These are design-context benchmarks, not reported estimates or statistical test results. The record does not provide a formal hypothesis-testing procedure, an acceptance rule for rates between the stated benchmarks, or a reported conclusion against either threshold. The benchmarks therefore do not substitute for the missing observed response result.
In general, a hypothesis test assesses how compatible observed data are with a specified statistical model or null hypothesis. A p-value, when reported, is not the probability that a treatment works or the probability that a finding is due to chance. No p-value or test statistic for the primary endpoint is reported in the registry record. The specific inferential method and its assumptions are also not reported.
What does the planning illustration mean?
The described sample-size calculations are planning illustrations based on a fraction of an observed effect, not the trial’s own design justification. The stated illustration assumptions include a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. Those assumptions describe a hypothetical two-arm planning exercise; they do not match this study’s single-group design. The observed effect and the resulting participant counts for the illustration are not reported in the registry record. Such planning counts are not observed power and do not show the probability that this completed study detected an effect.
In general, planning calculations depend on the assumed effect, variability, analysis method, and design. Changing those assumptions can change the number of participants indicated by a calculation. A planning illustration based on an observed effect is not a substitute for a prespecified sample-size justification, and no such justification is reported in the registry record. No sample-size target rationale or adjustment for participants without efficacy assessments is reported.
Why do the analysis populations matter?
The registry says 48 participants were included in safety assessment and that two treated participants lacked efficacy assessment and were excluded from both intention-to-treat and per-protocol efficacy analysis. In general, an intention-to-treat analysis retains participants in the group to which they were assigned, while a per-protocol analysis applies eligibility or adherence rules specified by the study. Because this study has no randomized groups, the exact operational definitions of these populations are important; those definitions are not reported. The record also does not state how the missing efficacy assessments were handled in any other way.
The study lists adverse events and influence on T-cell subsets as secondary outcomes, in addition to the primary response outcome. The registry reports 3 outcome measures and 0 analyses. It does not describe a multiplicity adjustment or a hierarchy for testing several outcomes. In general, when multiple hypotheses are tested, the chance of at least one false-positive result can increase unless an appropriate strategy is used. The record does not allow assessment of whether or how that issue was addressed. These missing analysis details limit interpretation, but they do not imply a particular direction or size of treatment effect.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists a primary response outcome and secondary outcomes concerning the number and severity of adverse events and the influence of rituximab plus lenalidomide on T-cell subsets. It reports 3 outcome measures and 0 analyses. No multiplicity strategy, testing hierarchy, or adjustment for examining multiple outcomes is reported in the registry record. In general, multiplicity matters when several outcomes or hypotheses are examined because interpreting each test in isolation can overstate the evidence from a set of tests. The record does not show whether formal tests were planned or performed.
For efficacy, the registry states that 48 participants were included in safety assessment, while two participants who received treatment had no efficacy assessment. Those two participants were excluded from both intention-to-treat and per-protocol efficacy analyses. The record does not report the reasons for missing assessments, the number included in each efficacy analysis population, or any imputation or sensitivity analysis. It also does not define the analysis populations in detail.
In general, missing outcome data can affect an estimate if participants without assessments differ in relevant ways from those assessed. The direction and size of any such effect cannot be inferred from the fact of missingness alone. Because the efficacy result itself is not reported, the impact of these exclusions on the response estimate cannot be evaluated. The registry’s population description establishes that safety and efficacy assessment populations differed, but it does not provide enough detail to reconstruct or compare efficacy analyses.
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 |
|---|---|---|---|
| Rituximab and Lenalidomide | 18 | 48 | 37.5% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 4.17% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Rituximab and Lenalidomide | 47 of 48 |
Most frequent serious adverse event terms
| Event term | Organ system | Rituximab and Lenalidomide |
|---|---|---|
| Pyrexia | General disorders | 2 of 48 |
| Rash | Skin and subcutaneous tissue disorders | 2 of 48 |
| Infusion related reaction | Injury, poisoning and procedural complications | 1 of 48 |
| Basal cell carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 1 of 48 |
| Anemia | Blood and lymphatic system disorders | 1 of 48 |
| Pain | General disorders | 1 of 48 |
| Diarrhea | Gastrointestinal disorders | 1 of 48 |
| Gastric haemorrhage | Gastrointestinal disorders | 1 of 48 |
| Vomiting | Gastrointestinal disorders | 1 of 48 |
| Renal failure | Renal and urinary disorders | 1 of 48 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Rituximab and Lenalidomide |
|---|---|---|
| Pruritus | Skin and subcutaneous tissue disorders | 23 of 48 |
| Rash | Skin and subcutaneous tissue disorders | 23 of 48 |
| Fatigue | General disorders | 17 of 48 |
| Neutrophil count | Investigations | 12 of 48 |
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | 11 of 48 |
| Vertigo | Nervous system disorders | 11 of 48 |
| Cough | Respiratory, thoracic and mediastinal disorders | 11 of 48 |
| Constipation | Gastrointestinal disorders | 10 of 48 |
| Diarrhoea | Gastrointestinal disorders | 9 of 48 |
| Chills | General disorders | 9 of 48 |
Serious events with the highest arm-level rate: pyrexia (4.2%), rash (4.2%), infusion related reaction (2.1%), basal cell carcinoma (2.1%), anemia (2.1%).
The safety assessment included 48 participants. The registry reports that 18 were affected by serious adverse events and 47 by other adverse events. These are participant counts in the treated group; they do not compare the combination with a control group, because the study had no concurrent comparator. The registry does not report a death count.
The listed serious adverse-event terms and percentages are pyrexia, 4.2%; rash, 4.2%; infusion related reaction, 2.1%; basal cell carcinoma, 2.1%; anemia, 2.1%; and pain, 2.1%. These percentages should be read as the registry’s reported figures, not converted into counts or treated as comparative risks. The record does not provide further detail about event timing, recurrence, severity grading beyond the serious-event designation, or participant-level overlap among event categories.
In general, adverse-event summaries describe events observed during a study and do not alone establish that the treatment caused them. Without a comparison group and fuller event-level information, these figures cannot quantify excess risk relative to another treatment or the background rate.
12. What the Result Does, and Does Not, Mean
The registry record establishes that an open-label, single-group phase II study evaluated rituximab plus lenalidomide in patients with measurable MALT lymphoma. It identifies objective response by 40 weeks as the primary endpoint and states response-rate benchmarks for interpreting the combination. It also reports safety findings for 48 participants and notes that two treated participants had no efficacy assessment.
The record does not report the primary efficacy result. There is no response count or proportion, and no confidence interval or hypothesis-test result. Therefore, the posted results do not show whether the response rate was below 40%, reached the minimum of 60%, or fell between those benchmarks. This absence does not indicate that no responses occurred; it means the efficacy outcome cannot be determined from the registry record.
The single-group design also limits what a response result could establish. A response proportion would describe participants in this study under its assessment procedures. Without a concurrent comparator, it would not estimate a comparative treatment effect. The registry does not report the response criteria, detailed assessment methods, or statistical analysis plan needed to evaluate how the endpoint was defined and analyzed.
The safety record reports 18 participants affected by serious adverse events and 47 affected by other adverse events among the 48 participants assessed for safety. These are descriptive counts. They do not show how event rates compare with those under another treatment, and the registry does not report a death count. The listed serious-event percentages provide additional descriptive information but not a causal or comparative estimate.
Accordingly, the posted record supports a description of the design, treatment schedule, analysis-population distinction, and reported safety counts. It does not support a conclusion about the study’s observed response rate, statistical precision, or comparative benefit and risk. Those questions require results and methods not reported in the registry record.
13. Why This Trial Matters Statistically
This trial illustrates a central distinction in phase II research: a study can specify an important efficacy question and decision benchmarks without the registry record reporting the result needed to answer that question. The primary endpoint is a response proportion, and the description identifies below 40% and a minimum of 60% as benchmarks. Without the observed response count, estimate, and uncertainty, neither benchmark can be assessed against the study’s findings.
The single-group design also clarifies the scope of inference. A response result would describe outcomes in the participants studied; it would not provide a randomized comparison with another treatment. Recognizing that distinction helps prevent a descriptive response rate from being interpreted as a controlled treatment effect.
The difference between the safety and efficacy populations is another statistically relevant feature. Safety assessment included 48 participants, while two treated participants had no efficacy assessment and were excluded from both efficacy analysis populations. This makes transparent reporting of denominators, missing outcomes, and population definitions important for interpreting an efficacy estimate. The registry records the exclusions but not the reasons for missingness or the detailed analysis rules.
Finally, the record demonstrates why statistical conclusions depend on more than a study’s phase and enrollment. The response criteria, estimate, uncertainty, analytic method, and handling of missing assessments all contribute to interpretation. These details are not reported in the registry record. The available safety counts remain descriptive and cannot serve as a substitute for the missing primary efficacy result or a comparative safety analysis.
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 most consequential limitation of the posted record is that it does not report the primary efficacy result. The response count and response proportion are absent, as are uncertainty estimates and inferential results. This prevents evaluation of the study’s stated response-rate benchmarks. The registry also does not report the criteria used to define objective response or the procedures used to assess it.
The study is open-label and single-group, with no concurrent comparator. Consequently, the record does not provide a randomized estimate of a treatment contrast. The registry lists no allocation and no masking, and does not report an analysis plan that would clarify how potential assessment or selection issues were addressed.
The safety population included 48 participants, while two treated participants lacked efficacy assessment and were excluded from both intention-to-treat and per-protocol efficacy analyses. The reason for missing assessments, the detailed definitions of analysis populations, and any sensitivity analysis are not reported in the registry record. These omissions limit assessment of how the exclusions might relate to the endpoint, although the direction or size of any effect cannot be inferred.
Safety findings are descriptive. The record does not report a death count, comparative event rates, or detailed event-level information such as timing and participant-level overlap among terms. The statistical analysis plan, sample-size justification, and interim-analysis plan are also not reported in the registry record. The described planning illustration uses two-arm assumptions despite the single-group study design, and its observed effect and resulting participant counts are not reported.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Rituximab Plus Lenalidomide in Patients With Mucosa Associated Lymphoid Tissue — NCT01611259.
- Registry record last updated 2017-11-06; status last verified 2017-06.
- Results first posted 2017-11-06.
- 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 an open-label, single-group phase II study of rituximab plus lenalidomide for patients with measurable MALT lymphoma. Enrollment was 50; the study is listed as completed, with a start of 2012-05 and primary completion of 2014-05. The regimen used 28-day cycles, with cycle number dependent on response or disease status.
The primary endpoint was objective response at 40 weeks. The record states response-rate benchmarks but does not report the observed response count or proportion, confidence interval, or test result. It reports 48 participants in the safety assessment, including 18 affected by serious adverse events and 47 by other adverse events. Two treated participants had no efficacy assessment and were excluded from both efficacy analysis populations.
The registry lists 3 outcome measures and 0 analyses. Detailed response criteria, missing-data methods, multiplicity handling, and a statistical analysis plan are not reported in the registry record.