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 SS1P and Pentostatin Plus Cyclophosphamide for Mesothelioma (NCT01362790), a non-randomized phase 1, phase 2 study. The primary outcome is Response Assessment.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01362790 |
| Phase | Phase 1, Phase 2 |
| Condition | Mesothelioma, Adenocarcinoma of Lung, Pancreatic Neoplasms |
| Allocation | Non-Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 55 |
| Arms in the primary outcome | n/a |
| Lead sponsor | National Cancer Institute (NCI) |
| Results posted | Yes |
2. Clinical Question
The study examined a treatment regimen combining SS1P with pentostatin and cyclophosphamide in participants with mesothelioma or selected other cancers. Its stated rationale was that SS1P is designed to target cells expressing mesothelin, while pentostatin and cyclophosphamide suppress immune activity and might reduce immune responses against SS1P. The registry record describes a pilot and Phase 2 study, rather than a randomized comparison of the regimen with a control.
Population
Participants had mesothelioma, lung adenocarcinoma or pancreatic cancer. The registry record reports overall enrollment of 55. It also states that no participants were enrolled in the Phase 2 Lung Adenocarcinoma Pilot group.
Intervention
The treatment approach combined SS1P with pentostatin and cyclophosphamide. The registry lists several pilot and expansion groups using different regimens, including groups for pleural and peritoneal mesothelioma, pancreatic adenocarcinoma and lung adenocarcinoma.
Comparator
There was no randomized control group. Allocation was non-randomized, and the registry record describes parallel groups. Differences between groups therefore should not be treated as like-for-like randomized treatment comparisons.
Primary question
The primary endpoint was response assessment using EORTC modified RECIST criteria. The registry record defines complete response, partial response, progressive disease and stable disease, but does not report the response results. The primary endpoint timeframe is listed as 52 months and 4 days.
Statistically, the key question is what the posted outcomes can establish about response and safety in the enrolled groups. The answer is constrained by the absence of posted response counts and analyses, the non-randomized design, and the fact that the groups represent different pilot or expansion regimens and cancer populations. Those features limit interpretation of between-group differences and do not permit a controlled estimate of treatment effect.
3. Trial Design
The registry describes a completed Phase 1/Phase 2 study with non-randomized allocation, no masking and a parallel-group model. Overall enrollment was 55. The sponsor was the National Cancer Institute. The study start date is listed as 2011-05-11, and primary completion is listed as 2016-08-03.
The listed groups include mesothelioma pilot regimens, peritoneal and pleural mesothelioma expansion groups, a mesothelioma dose-de-escalation group, a pancreatic adenocarcinoma pilot group and a lung adenocarcinoma expansion group. These are not randomized arms intended to serve as interchangeable controls. In addition, the registry states that no participants were enrolled in the Phase 2 Lung Adenocarcinoma Pilot group. Comparisons across the remaining groups may reflect differences in cancer type, treatment regimen or study phase, as well as other factors; the registry record does not provide an adjustment plan that would resolve such differences.
The interventions listed are pentostatin, cyclophosphamide and SS1P biological product lots. The arm descriptions show that schedules differed across regimens. For example, Regimen A descriptions include pentostatin on days 1, 5 and 9 during a 30 day first cycle, followed by dosing on day 1 of subsequent 21-day cycles. Regimen B descriptions use different cycle lengths and dosing schedules. These details indicate that the study record encompasses more than one treatment schedule; they do not establish that the schedules were compared under randomized conditions.
The registry record reports secondary outcomes of overall survival, progression-free survival and duration of response. It lists 8 outcome measures and 0 analyses. The primary endpoint timeframe is 52 months and 4 days. A rationale for the total enrollment target, a formal sample-size justification, details of interim analyses and the statistical plan are not reported in the registry record. Consequently, the enrollment total alone does not establish the precision the investigators intended or the statistical power of any analysis.
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: 55 participants were enrolled. No data was collected for 10/55 participants who were screened for neutralizing antibodies and were positive, thus not enrolled into a Group /Arm, nor treated. 45/55 participants were treated on this study.
Only the first 6 of 7 groups are shown.
Pilot Phase & Phase 2
| Stage | Mesothelioma Pilot Phase Regimen A | Mesothelioma Pilot Phase Regimen B | Phase 2 Peritoneal Mesothelioma Pilot Expansion Phase | Phase 2 Pleural Mesothelioma Pilot Expansion Phase | Mesothelioma Positive Ca Dose De-escalation Pilot Regimen A | Phase 2 Pancreatic Adenocarcinoma Pilot Phase Regimen A |
|---|---|---|---|---|---|---|
| STARTED | 11 | 8 | 7 | 15 | 0 | 4 |
| COMPLETED | 1 | 0 | 2 | 1 | 0 | 0 |
| NOT COMPLETED | 10 | 8 | 5 | 14 | 0 | 4 |
Reasons for not completing this period:
| Reason | Mesothelioma Pilot Phase Regimen A | Mesothelioma Pilot Phase Regimen B | Phase 2 Peritoneal Mesothelioma Pilot Expansion Phase | Phase 2 Pleural Mesothelioma Pilot Expansion Phase | Mesothelioma Positive Ca Dose De-escalation Pilot Regimen A | Phase 2 Pancreatic Adenocarcinoma Pilot Phase Regimen A |
|---|---|---|---|---|---|---|
| Changed treatment, too early to assess | 1 | 0 | 0 | 0 | 0 | 0 |
| Adverse Event | 0 | 1 | 0 | 1 | 0 | 2 |
| Physician Decision | 0 | 0 | 0 | 1 | 0 | 0 |
| Developed antibodies | 7 | 2 | 3 | 8 | 0 | 0 |
| Refused further treatment | 0 | 1 | 0 | 0 | 0 | 0 |
| Progressive disease | 1 | 2 | 2 | 3 | 0 | 2 |
| Treatment delay >3 weeks | 1 | 0 | 0 | 0 | 0 | 0 |
| Complicating disease/intercurr. illness | 0 | 2 | 0 | 0 | 0 | 0 |
| Death on study | 0 | 0 | 0 | 1 | 0 | 0 |
Dose De-escalation of Regimen A
| Stage | Mesothelioma Pilot Phase Regimen A | Mesothelioma Pilot Phase Regimen B | Phase 2 Peritoneal Mesothelioma Pilot Expansion Phase | Phase 2 Pleural Mesothelioma Pilot Expansion Phase | Mesothelioma Positive Ca Dose De-escalation Pilot Regimen A | Phase 2 Pancreatic Adenocarcinoma Pilot Phase Regimen A |
|---|---|---|---|---|---|---|
| STARTED | 0 | 0 | 0 | 0 | 8 | 0 |
| COMPLETED | 0 | 0 | 0 | 0 | 2 | 0 |
| NOT COMPLETED | 0 | 0 | 0 | 0 | 6 | 0 |
Reasons for not completing this period:
| Reason | Mesothelioma Pilot Phase Regimen A | Mesothelioma Pilot Phase Regimen B | Phase 2 Peritoneal Mesothelioma Pilot Expansion Phase | Phase 2 Pleural Mesothelioma Pilot Expansion Phase | Mesothelioma Positive Ca Dose De-escalation Pilot Regimen A | Phase 2 Pancreatic Adenocarcinoma Pilot Phase Regimen A |
|---|---|---|---|---|---|---|
| Adverse Event | 0 | 0 | 0 | 0 | 1 | 0 |
| Deveoped antibodies | 0 | 0 | 0 | 0 | 3 | 0 |
| Progressive disease | 0 | 0 | 0 | 0 | 2 | 0 |
Analysis population for the primary outcome, as defined in the registry: No pts. were enrolled in the Phase 2 Lung Adenocarcinoma Pilot group.
5. Baseline Characteristics
The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.
Population: No pts were enrolled in Grp7.
Only the first 6 of 7 groups are shown.
| Characteristic | Unit | Mesothelioma Pilot Phase Regimen A | Mesothelioma Pilot Phase Regimen B | Phase 2 Peritoneal Mesothelioma Pilot Expansion Phase | Phase 2 Pleural Mesothelioma Pilot Expansion Phase | Phase 2 Pancreatic Adenocarcinoma Pilot Phase Regimen A | Phase 2 Lung Adenocarcinoma Pilot Expansion Phase Regimen A |
|---|---|---|---|---|---|---|---|
| Participants analysed | 11 | 8 | 7 | 15 | 4 | 0 | |
| Age, Categorical: <=18 years | Participants | 0 | 0 | 0 | 0 | 0 | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 9 | 7 | 6 | 7 | 4 | 0 |
| Age, Categorical: >=65 years | Participants | 2 | 1 | 1 | 8 | 0 | 0 |
| Age, Continuous | years | 55.27 (SD 7.68) | 60.55 (SD 16.41) | 49.91 (SD 16.32) | 64.21 (SD 7.65) | 52.53 (SD 6.77) | — |
| Sex: Female, Male: Female | Participants | 4 | 2 | 2 | 5 | 1 | — |
| Sex: Female, Male: Male | Participants | 7 | 6 | 5 | 10 | 3 | — |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 | 0 | 0 | 1 | 0 | — |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 11 | 8 | 7 | 14 | 4 | — |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 | 0 | 0 | — |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 | 0 | 0 | — |
| Race (NIH/OMB): Asian | Participants | 1 | 0 | 0 | 1 | 0 | — |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 | 0 | 0 | — |
| Race (NIH/OMB): Black or African American | Participants | 0 | 0 | 1 | 0 | 0 | — |
| Race (NIH/OMB): White | Participants | 10 | 8 | 6 | 14 | 4 | — |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 0 | 0 | 0 | — |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 | 0 | 0 | — |
| Region of Enrollment: United States | Participants | 11 | 8 | 7 | 15 | 4 | — |
| Count of Participants with a Peritoneum and Pleural Tumor Site: Peritoneum | Participants | 2 | 1 | 7 | 0 | 0 | — |
| Count of Participants with a Peritoneum and Pleural Tumor Site: Pleural | Participants | 9 | 7 | 0 | 15 | 0 | — |
| Count of Participants with a Peritoneum and Pleural Tumor Site: Pancreatic | Participants | 0 | 0 | 0 | 0 | 4 | — |
6. Primary Endpoint
The registry lists the primary outcome as Response Assessment, measured over 52 months and 4 days.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is titled Response Assessment and is measured as a count of participants. The registry says response was assessed using the European Organization for Research and Treatment of Cancer modified Response Evaluation Criteria in Solid Tumors. Complete response means complete disappearance of all target lesions. Partial response requires at least a 30% decrease in the sum of the longest diameter of target lesions. Progressive disease requires at least a 20% increase in that sum. Stable disease means neither enough shrinkage for partial response nor enough increase for progressive disease, using the smallest sum of longest diameters since treatment started as the reference.
General statistical context: an endpoint based on response categories turns observations of tumor measurements into clinically interpretable classifications. Counts in each category can describe how many participants met each definition, while the criteria make the classification rule explicit. These counts do not by themselves establish that treatment caused a response, particularly in a non-randomized study without a control group.
The registry record gives a primary endpoint timeframe of 52 months and 4 days, but the response counts and results are not reported in the registry record. The primary endpoint population description also states that no participants were enrolled in the Phase 2 Lung Adenocarcinoma Pilot group. The extent to which other groups contributed response observations is not detailed in the available record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record describes the endpoint and reports overall enrollment, but it does not report the primary response results. It is therefore not possible to describe the number of participants with complete response, partial response, progressive disease or stable disease from this record. Nor are an effect estimate, confidence interval or inferential test for response reported. The endpoint definition tells readers how response was classified; it does not tell readers what response distribution was observed.
The overall enrollment was 55, across several non-randomized groups and cancer populations. That total should not be read as the number contributing to every outcome or regimen. In particular, the record states that no participants were enrolled in the Phase 2 Lung Adenocarcinoma Pilot group. Group-specific endpoint denominators and response results are not reported in the registry record, so precision and completeness of response estimates cannot be assessed.
The study's non-randomized allocation and absence of masking matter for interpretation. Participants were not assigned randomly to parallel groups, and the groups included different diseases and regimens. As a result, any contrast among those groups would not automatically represent a causal treatment comparison. Differences could be associated with group composition or treatment schedules, and no adjusted analysis is reported in the registry record. No randomized control comparison is available to separate treatment effects from other group differences.
The registry lists overall survival, progression-free survival and duration of response as secondary outcomes, but does not report their numerical results in the registry record by the record. It lists 0 analyses. The presence of an outcome name is not equivalent to a posted analysis, and no conclusion about the magnitude or precision of any secondary endpoint can be drawn from its listing alone.
Safety information is reported as counts of participants affected by serious and other adverse events in the groups. Those counts describe observed events within the recorded groups, not an estimate of comparative risk from a randomized trial. The registry also lists deaths, but does not state in these figures whether deaths were caused by treatment. Serious adverse-event terms and their percentages are listed, yet the percentage denominator is not reported in the registry record.
Overall, the posted information supports a description of the study structure, response criteria and recorded safety counts. It does not support a quantitative estimate of response benefit, a comparison with an untreated or alternative-treatment group, or a conclusion about the effectiveness of the regimen. Such interpretations require outcome results and analysis details that are not reported in the registry record.
9. Statistical Methods Explained
The registry describes a response endpoint measured as a count of participants classified using EORTC modified RECIST criteria. It lists 8 outcome measures and 0 analyses. The record therefore supplies a definition for the primary endpoint, but not the response counts or statistical analyses needed to estimate response frequency or uncertainty. The statistical concepts below are general explanations of how such information is usually interpreted; they are not claims about analyses conducted in this study.
What does a response count describe?
General: a count gives the number of participants meeting a specified outcome definition. For a categorical response endpoint, each participant is assigned a category according to the stated criteria. Interpreting a count requires knowing the number of participants assessed and the number included in the analysis. A count alone does not give a response rate unless its denominator is also known. Neither response counts nor group-specific response denominators are reported in the registry record.
The endpoint definitions are based on changes in the sum of the longest diameter of target lesions. Complete response is defined as disappearance of all target lesions; partial response uses at least a 30% decrease; progressive disease uses at least a 20% increase; stable disease covers neither sufficient decrease nor sufficient increase, relative to the smallest sum since treatment started. These rules clarify how the endpoint is classified, but they do not supply the observed classifications.
What would an estimate and confidence interval mean?
General: an estimate summarizes an observed outcome, while a confidence interval describes the uncertainty around that estimate under the statistical model used. A narrower interval indicates greater precision under that model; a wider interval indicates less precision. Confidence intervals do not repair weaknesses in design, such as lack of randomization, and they do not make non-comparable groups equivalent. No response estimate or confidence interval is reported in the registry record, so response precision cannot be evaluated.
How should a hypothesis test be read?
General: a p-value is a measure of how compatible the observed data are with a specified null model, under the assumptions of the analysis. It is not the probability that a treatment works, and it does not measure the size or clinical importance of an effect. A p-value is most informative when considered with the estimate, its uncertainty, the analysis plan and the study design. The registry lists 0 analyses and reports no p-value for the primary endpoint.
What does the non-randomized structure imply?
General: random allocation can help balance known and unknown participant characteristics across comparison groups. Without randomization, group differences may be related to participant selection, disease type, regimen or other factors. The study record says allocation was non-randomized and masking was absent. It also describes multiple groups covering different cancers and schedules. This means a comparison among groups would need careful interpretation; the registry record does not report a statistical adjustment plan or a randomized control comparison.
The parallel-group label means groups were followed within the same study structure; it does not mean the groups were randomized or directly comparable. Differences between the pleural mesothelioma, peritoneal mesothelioma, pancreatic adenocarcinoma and other listed groups cannot be attributed to regimen alone from the information reported. The absence of enrollment in the Phase 2 Lung Adenocarcinoma Pilot group also means that group cannot contribute participant-level endpoint observations.
What do the planning assumptions mean?
General: sample-size planning asks how much information a future or planned study may need to detect a chosen effect under specified assumptions. The registry-related planning description states a two-sided significance level of 0.05, equal allocation between two arms and a normal approximation. It describes planning for fractions of an observed effect. These are planning assumptions, not a description of the trial's observed results or proof that the study had a particular probability of detecting an effect.
For time-to-event planning, the description refers to a total number of events using the Schoenfeld approach; for other outcomes it refers to participants per arm. Such calculations depend on the assumed effect and other inputs. The registry record does not report the trial's own sample-size justification. Any sample-size values displayed as planning illustrations should not be interpreted as observed power, as the number enrolled in this study, or as evidence that the study was adequately powered.
Why can over-dispersion matter?
General: in count outcomes, a simple statistical model may assume that variability follows a particular relationship with the mean. Over-dispersion occurs when the observed variability is greater than that assumption allows. If ignored, standard errors may be too small and uncertainty may be understated. This concept is relevant to some count-data analyses, but the registry record does not report a count model, an over-dispersion assessment or any model-based results for this study.
More broadly, model choice cannot substitute for missing outcome data or resolve design limitations on its own. Because the response outcomes and analyses are not reported, there is no basis in the registry record for evaluating model fit, assumptions, uncertainty or robustness of the primary response analysis.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists a primary response endpoint and secondary outcomes of overall survival, progression-free survival and duration of response. It also reports 8 outcome measures and 0 analyses. The registry record does not describe a multiplicity strategy, such as a prespecified hierarchy among endpoints or a method for controlling the chance of false-positive findings when multiple outcomes are tested. This does not establish that no such plan existed; its details are not reported in the registry record.
General statistical context: testing multiple outcomes or making repeated comparisons can increase the chance of at least one apparently unusual result under a set of null hypotheses. Prespecified ordering or adjustment methods can address that issue, but their use and consequences must be documented to interpret inferential results. No endpoint-specific tests or p-values are reported, so the effect of multiplicity cannot be assessed.
Missing-data handling is also not reported in the registry record. The record does not describe how participants without an evaluable response, with incomplete follow-up, or with missing survival information were handled. It does not report imputation, censoring rules, sensitivity analyses or reasons for missing observations. Those details matter because analyses may differ depending on whether missing outcomes are excluded, treated as events, censored or handled through another method.
The record does not define analysis populations, such as which participants were included in each endpoint analysis. Overall enrollment of 55 does not establish the denominator for each outcome or group. The primary endpoint population description notes that no participants were enrolled in the Phase 2 Lung Adenocarcinoma Pilot group, but the analysis populations for the other groups are not described. These reporting gaps limit assessment of completeness and reproducibility.
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 |
|---|---|---|---|
| Mesothelioma Pilot Phase Regimen A | 6 | 11 | 54.5% |
| Mesothelioma Pilot Phase Regimen B | 8 | 8 | 100.0% |
| Phase 2 Peritoneal Mesothelioma Pilot Expansion Phase | 3 | 7 | 42.9% |
| Phase 2 Pleural Mesothelioma Pilot Expansion Phase | 9 | 15 | 60.0% |
| Mesothelioma Positive Ca Dose De-escalation Pilot Regimen A | 1 | 8 | 12.5% |
| Phase 2 Pancreatic Adenocarcinoma Pilot Phase Regimen A | 1 | 4 | 25.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Mesothelioma Pilot Phase Regimen A | 5 of 11 |
| Mesothelioma Pilot Phase Regimen B | 8 of 8 |
| Phase 2 Peritoneal Mesothelioma Pilot Expansion Phase | 3 of 7 |
| Phase 2 Pleural Mesothelioma Pilot Expansion Phase | 9 of 15 |
| Mesothelioma Positive Ca Dose De-escalation Pilot Regimen A | 1 of 8 |
| Phase 2 Pancreatic Adenocarcinoma Pilot Phase Regimen A | 1 of 4 |
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 |
|---|---|
| Mesothelioma Pilot Phase Regimen A | 11 of 11 |
| Mesothelioma Pilot Phase Regimen B | 8 of 8 |
| Phase 2 Peritoneal Mesothelioma Pilot Expansion Phase | 7 of 7 |
| Phase 2 Pleural Mesothelioma Pilot Expansion Phase | 15 of 15 |
| Mesothelioma Positive Ca Dose De-escalation Pilot Regimen A | 8 of 8 |
| Phase 2 Pancreatic Adenocarcinoma Pilot Phase Regimen A | 4 of 4 |
Most frequent serious adverse event terms
| Event term | Organ system | Mesothelioma Pilot Phase Regimen A | Mesothelioma Pilot Phase Regimen B | Phase 2 Peritoneal Mesothelioma Pilot Expansion Phase | Phase 2 Pleural Mesothelioma Pilot Expansion Phase | Mesothelioma Positive Ca Dose De-escalation Pilot Regimen A | Phase 2 Pancreatic Adenocarcinoma Pilot Phase Regimen A |
|---|---|---|---|---|---|---|---|
| Death NOS | General disorders | 5 of 11 | 5 of 8 | 2 of 7 | 4 of 15 | 0 of 8 | 1 of 4 |
| Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, specify | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 0 of 11 | 3 of 8 | 1 of 7 | 4 of 15 | 1 of 8 | 0 of 4 |
| Fever | General disorders | 1 of 11 | 1 of 8 | 0 of 7 | 0 of 15 | 0 of 8 | 0 of 4 |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | 1 of 11 | 1 of 8 | 0 of 7 | 0 of 15 | 0 of 8 | 0 of 4 |
| Atrial fibrillation | Cardiac disorders | 1 of 11 | 0 of 8 | 1 of 7 | 0 of 15 | 0 of 8 | 0 of 4 |
| Acute kidney injury | Renal and urinary disorders | 0 of 11 | 0 of 8 | 0 of 7 | 1 of 15 | 0 of 8 | 0 of 4 |
| Lung infection | Infections and infestations | 0 of 11 | 0 of 8 | 0 of 7 | 1 of 15 | 0 of 8 | 0 of 4 |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | 0 of 11 | 1 of 8 | 0 of 7 | 0 of 15 | 0 of 8 | 0 of 4 |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | 0 of 11 | 0 of 8 | 0 of 7 | 1 of 15 | 0 of 8 | 0 of 4 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Mesothelioma Pilot Phase Regimen A | Mesothelioma Pilot Phase Regimen B | Phase 2 Peritoneal Mesothelioma Pilot Expansion Phase | Phase 2 Pleural Mesothelioma Pilot Expansion Phase | Mesothelioma Positive Ca Dose De-escalation Pilot Regimen A | Phase 2 Pancreatic Adenocarcinoma Pilot Phase Regimen A |
|---|---|---|---|---|---|---|---|
| Lymphocyte count decreased | Investigations | 11 of 11 | 8 of 8 | 7 of 7 | 15 of 15 | 8 of 8 | 4 of 4 |
| Hypoalbuminemia | Metabolism and nutrition disorders | 11 of 11 | 7 of 8 | 6 of 7 | 12 of 15 | 8 of 8 | 4 of 4 |
| Hyponatremia | Metabolism and nutrition disorders | 8 of 11 | 5 of 8 | 7 of 7 | 13 of 15 | 8 of 8 | 4 of 4 |
| Hyperglycemia | Metabolism and nutrition disorders | 10 of 11 | 6 of 8 | 5 of 7 | 15 of 15 | 6 of 8 | 2 of 4 |
| Anemia | Blood and lymphatic system disorders | 8 of 11 | 7 of 8 | 4 of 7 | 11 of 15 | 8 of 8 | 4 of 4 |
| White blood cell decreased | Investigations | 8 of 11 | 7 of 8 | 5 of 7 | 11 of 15 | 7 of 8 | 3 of 4 |
| Nausea | Gastrointestinal disorders | 8 of 11 | 5 of 8 | 5 of 7 | 13 of 15 | 7 of 8 | 2 of 4 |
| Fatigue | General disorders | 7 of 11 | 4 of 8 | 4 of 7 | 9 of 15 | 7 of 8 | 4 of 4 |
| Edema limbs | General disorders | 6 of 11 | 5 of 8 | 3 of 7 | 11 of 15 | 5 of 8 | 2 of 4 |
| Aspartate aminotransferase increased | Injury, poisoning and procedural complications | 7 of 11 | 4 of 8 | 3 of 7 | 7 of 15 | 6 of 8 | 4 of 4 |
Serious events with the highest arm-level rate: death nos (62.5%), neoplasms benign, malignant and unspecified (incl cysts and polyps) - other, specify (37.5%), atrial fibrillation (14.3%), fever (12.5%), respiratory failure (12.5%).
The registry reports serious adverse-event counts by study group, including the number at risk and the number affected, alongside counts for other adverse events and deaths. In Mesothelioma Pilot Phase Regimen A, 11 participants were at risk for serious events and 6 were affected; the record lists 11 at risk and 11 affected for other events, with 5 deaths. The corresponding serious-event counts are 8 and 8 for Mesothelioma Pilot Phase Regimen B, 7 and 3 for the peritoneal mesothelioma expansion group, 15 and 9 for the pleural mesothelioma expansion group, 8 and 1 for the dose-de-escalation group, and 4 and 1 for the pancreatic adenocarcinoma pilot group. The lung adenocarcinoma expansion group has 0 participants at risk and 0 affected.
The registry also lists serious-event terms and percentages: Death NOS, 62.5%; neoplasms benign, malignant and unspecified (including cysts and polyps) - Other, specify, 37.5%; atrial fibrillation, 14.3%; fever, respiratory failure and hypoxia, each 12.5%. The denominator used for these percentages is not reported in the registry record.
These figures describe recorded events, not causal attribution. The death counts do not establish that deaths were caused by study treatment. Because allocation was non-randomized and groups differed, counts across groups are not randomized safety comparisons. They should be read with their group denominators and reporting context, not as direct evidence of comparative risk.
12. What the Result Does, and Does Not, Mean
The registry record establishes that a completed, non-randomized Phase 1/Phase 2 study evaluated SS1P with pentostatin and cyclophosphamide across several pilot and expansion groups. It identifies the primary response criteria, names secondary outcomes and reports group-level safety counts. It also documents overall enrollment of 55 and notes that no participants entered the Phase 2 Lung Adenocarcinoma Pilot group.
Those details do not establish the observed response distribution. The registry record does not report the primary response counts, an effect estimate, a confidence interval or a statistical test. It also does not report numerical results for the listed secondary outcomes in the information summarized. Therefore, the degree of response, the precision of any response estimate and the pattern of survival outcomes cannot be determined from this record.
The study's structure also limits what can be inferred from comparisons. Allocation was non-randomized, there was no masking, and the listed groups include different cancers and treatment schedules. A difference between groups would not, by itself, show that one regimen caused a better or worse outcome. The registry record does not identify a randomized control group or provide an adjustment strategy for group differences.
The adverse-event counts provide descriptive safety information for participants recorded in each group. They do not establish that an event was caused by treatment or permit a fair randomized comparison of safety. The listed deaths should not be interpreted as treatment-related deaths on the basis of these counts alone.
General statistical context: statistical conclusions depend on both the numerical results and the study design. Even a precise estimate cannot eliminate bias from non-randomized allocation, while a well-defined endpoint cannot substitute for the outcome data needed to estimate it. Here, the record supports a description of endpoint definitions, design and reported safety counts, but not a quantitative claim about treatment effect or comparative effectiveness.
13. Why This Trial Matters Statistically
This study illustrates why an endpoint definition and an endpoint result are different kinds of information. The registry provides detailed response categories based on EORTC modified RECIST criteria, but does not provide the response counts or analyses. A reader can understand the intended measurement rule without being able to determine the observed response frequency, its precision or how it varied across the enrolled groups.
It also illustrates the statistical implications of a non-randomized, multi-group design. The record includes pilot and expansion groups across mesothelioma, pancreatic adenocarcinoma and lung adenocarcinoma, with different treatment schedules. Such group structure can be useful for describing study experience, but it does not create a randomized control comparison. An apparent difference among groups could reflect differences in participants, cancer type or regimen rather than a treatment effect.
The absence of participants in the Phase 2 Lung Adenocarcinoma Pilot group is another important reporting detail: a named group does not necessarily contribute outcome data. Similarly, overall enrollment cannot be assumed to be the denominator for every endpoint. Clear group-specific denominators and analysis populations are necessary to understand how much information supports each result.
Finally, the record reports safety counts but does not establish causal attribution or provide the denominator basis for the listed serious-event percentages. Taken together, these features show why statistical interpretation depends not only on an outcome label or a count, but also on design, denominators, analysis methods and uncertainty. The missing response results and analysis details sharply constrain conclusions that can be drawn from the registry record.
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 specific to this study are apparent from the registry record. Allocation was non-randomized and masking was absent. The listed groups were not a randomized comparison, and they encompass different cancer populations and treatment regimens. Consequently, cross-group outcome contrasts would not isolate the effect of treatment without additional design or analysis information, which is not reported in the registry record.
The response endpoint is defined, but response counts and results are not reported. The record also lists 0 analyses, so there is no reported inferential analysis to evaluate. While overall enrollment was 55, group-specific denominators for response and secondary outcomes are not posted by the record. The no-enrollment statement for the Phase 2 Lung Adenocarcinoma Pilot group further means the group cannot provide participant outcome data.
The registry lists overall survival, progression-free survival and duration of response as secondary outcomes, but numerical results, follow-up summaries and analysis methods for those outcomes are not reported in the registry record. Missing-data rules, censoring details, analysis populations, multiplicity handling and statistical model assumptions are also not reported. The trial's own sample-size justification and interim-analysis plans are not reported in the registry record.
Safety counts are descriptive and do not establish treatment causality. The percentage denominators for the listed serious-event terms are not reported. The record's death counts should not be interpreted as deaths caused by treatment. These limitations mean that both efficacy and comparative safety conclusions remain constrained by the available reporting.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: SS1P and Pentostatin Plus Cyclophosphamide for Mesothelioma — NCT01362790.
- Registry record last updated 2019-06-06; status last verified 2019-06.
- Results first posted 2019-06-06.
- 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 NCT01362790, a completed Phase 1/Phase 2 study sponsored by the National Cancer Institute. Overall enrollment was 55. The study used non-randomized allocation, no masking and a parallel-group model. Its groups covered mesothelioma, pancreatic adenocarcinoma and lung adenocarcinoma, with different pilot or expansion regimens combining SS1P, pentostatin and cyclophosphamide. No participants were enrolled in the Phase 2 Lung Adenocarcinoma Pilot group.
The primary endpoint was participant response assessed using EORTC modified RECIST criteria, with a listed timeframe of 52 months and 4 days. Secondary outcomes included overall survival, progression-free survival and duration of response. The record lists 8 outcome measures and 0 analyses. Response results and inferential analyses are not reported in the registry record. Group-level serious and other adverse-event counts and death counts are reported, but they do not establish causality or randomized comparisons. Multiplicity procedures, missing-data handling, analysis populations and the trial's own sample-size justification are not reported in the registry record.