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 Stem Cell Transplantation for Metastatic Solid Tumors (NCT00001880), a na phase 2 study. The primary outcome is Number of Participants Based on Tumor Response Criteria With Anti-tumor Effect Induce by Graft-versus-tumor Effect..
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00001880 |
| Phase | Phase 2 |
| Condition | Neoplasm Metastasis |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 42 |
| Arms in the primary outcome | n/a |
| Lead sponsor | National Heart, Lung, and Blood Institute (NHLBI) |
| Results posted | Yes |
2. Clinical Question
Population
The study concerned patients with progressive, incurable metastatic solid tumors whose disease was refractory to standard therapy. The registry summary describes a range of tumor types as potentially eligible, including cancers of the esophagus, stomach, colon, rectum, liver, biliary system, pancreas, lung, breast, prostate, bone, adrenal gland, bladder, and tumors of unknown primary origin. The exact eligibility criteria and the number enrolled for each tumor type are not reported in the registry record.
Intervention
The intervention was non-myeloablative allogeneic stem cell transplantation, described in the registry summary as a mini-transplant using stem cells collected from a sibling. The arm description specifies cyclosporin beginning on day -4, stem cells on Day 0, and intravenous methotrexate on days +1, +3, and +6. The study also included donor lymphocyte infusion as a possible subsequent intervention, with a secondary outcome tracking participants who received it to achieve tumor regression or prevent graft failure.
Comparator
There was no separate comparator group. The study was single-group and nonrandomized, so its registry record does not provide a concurrent control arm for estimating how outcomes would differ under another treatment or under no transplantation. Comparisons with outcomes from other studies would not be like-for-like randomized comparisons.
Primary question
The primary question was whether the transplantation could induce an anti-tumor effect through a graft-versus-tumor effect in patients with varied metastatic solid tumors refractory to standard therapy. Tumor response was assessed over one year using definitions for complete response, partial response, stable disease, and progressive disease. The registry record does not report how many participants met any of these response categories, so it does not establish the observed primary-endpoint result.
3. Trial Design
This was a phase 2, single-group study of non-myeloablative allogeneic stem cell transplantation in progressive and incurable metastatic solid tumors. The registry lists enrollment of 42 and identifies the study as terminated. It gives a start date of 1999-03-12 and a primary completion date of 2008-08-14. The reason for termination is not reported in the registry record.
The study was not randomized and had no masking. Its design therefore describes a treated group rather than a randomized comparison between the transplantation strategy and another intervention. The record lists no allocation method and identifies the model as single group. In this setting, the study can describe outcomes among participants who received the intervention, but the design alone cannot distinguish an intervention effect from other explanations for observed outcomes.
The intervention description combines cyclosporin, transplantation, and intravenous methotrexate on specified relative days. The registry summary describes the stem cells as collected from a sibling and the procedure as a mini-transplant. Donor lymphocyte infusion appears among the secondary outcomes as an intervention received by some participants for tumor regression or prevention of graft failure. The record does not report the criteria or decision process for administering it.
The primary outcome was the number of participants assessed by tumor-response criteria over one year. Secondary outcomes concerned engraftment, donor lymphocyte infusion, acute graft-versus-host disease grade 2 and higher, and chronic graft-versus-host disease. Although the record lists these outcomes, their participant-level results are not reported in the registry record. The protocol’s statistical plan, sample-size justification, and intended analysis populations are also 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 | Stem Cell Transplantation in Patients With Progressive and Incurable Metastatic Solid Tumors |
|---|---|
| STARTED | 42 |
| COMPLETED | 28 |
| NOT COMPLETED | 14 |
Reasons for not completing this period:
| Reason | Stem Cell Transplantation in Patients With Progressive and Incurable Metastatic Solid Tumors |
|---|---|
| Death | 14 |
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 | Stem Cell Transplantation in Patients With Progressive and Incurable Metastatic Solid Tumors |
|---|---|---|
| Participants analysed | 42 | |
| Age, Continuous: Male | years | 55 (24 to 67) |
| Age, Continuous: Female | years | 45 (28 to 65) |
| Sex: Female, Male: Female | Participants | 20 |
| Sex: Female, Male: Male | Participants | 22 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 1 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 41 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 3 |
| Race (NIH/OMB): White | Participants | 37 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 2 |
| Region of Enrollment: United States | participants | 42 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Participants Based on Tumor Response Criteria With Anti-tumor Effect Induce by Graft-versus-tumor Effect., measured over one year.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is a count of participants classified according to tumor-response criteria, with an assessment timeframe of one year. The stated objective was to identify an anti-tumor effect associated with a graft-versus-tumor effect after allogeneic peripheral blood stem cell transplantation in patients with varied metastatic solid tumors refractory to standard therapy.
The response definitions distinguish complete response, partial response, stable disease, and progressive disease. Complete response requires disappearance of all signs and symptoms of metastatic disease for at least one month. Partial response requires a 50% or greater decrease in the sum of the products of the longest perpendicular diameters of measured lesions lasting at least one month, with no new metastatic lesions. Stable disease is defined as measurements that do not meet the criteria for complete response, partial response, or progressive disease. Progressive disease is defined by a 25% or greater increase in the sum of the products of the longest perpendicular diameters compared with the smallest previous measurements, or by development of new metastatic disease.
These categories make the outcome clinically interpretable as a description of tumor status rather than a continuous measurement alone. However, the registry record does not report the number of participants in each category or a combined count representing anti-tumor effect. The endpoint definition is available; the observed primary-endpoint result is not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry describes an exploratory, single-group phase 2 study with a primary tumor-response question and a one-year timeframe. The primary outcome is framed as a participant count based on response criteria. However, the registry record does not report the count of participants with complete response, partial response, stable disease, or progressive disease. It also does not report a primary-endpoint estimate, uncertainty interval, or test result. Consequently, the posted record does not allow a reader to quantify the observed tumor-response pattern or its precision.
The study enrolled 42 according to the registry and is marked terminated. These design and status details do not substitute for outcome data. In particular, the record does not report why the study was terminated, whether the planned enrollment was reached, or whether any prespecified stopping rule was used. Those details should not be inferred from the status label.
The design also limits what can be concluded from any observed response counts. Because there was no randomized comparator group, response in the treated group would not by itself establish how outcomes would have differed under another strategy. The study’s aim was exploratory and concerned a range of metastatic solid tumors, but the registry does not report response results by tumor type. It therefore does not show whether response patterns differed across the included cancers.
Safety information is reported as counts for the study group: 21 participants affected by serious adverse events, 7 affected by other adverse events, and 14 deaths. The registry also lists serious adverse-event terms with percentages, including progressive disease at 28.6% and acute Graft VS Host Disease of GI Tract at 9.5%. These entries describe reported events; they do not, by themselves, establish that the transplantation caused each event. The record does not report event-level timing, severity grades beyond the named terms, attribution, or whether individuals could appear under more than one term.
Statistical analyses are not reported in the registry record. A count without a reported analysis cannot be treated as evidence of a statistically demonstrated effect, and absence of a posted result is not evidence that there was no response. The appropriate reading is limited: the registry records the study question, design, endpoint definitions, and some adverse-event counts, but does not provide the primary tumor-response findings needed to answer the central question.
Any planning illustrations based on the observed effect should be distinguished from the study’s own design and from observed power. The registry does not report a statistical plan or a sample-size justification. The displayed planning assumptions are not evidence that the study was designed around those assumptions, and the single-group trial design is not equivalent to the two-arm planning setup described for those illustrations.
9. Statistical Methods Explained
Statistical methods describe how an endpoint is defined, summarized, and compared, and how uncertainty or missing information is handled. The registry record identifies the primary outcome as a count of participants classified by tumor-response criteria. It does not report a statistical analysis plan, a model for the primary endpoint, an inferential test, or an uncertainty interval. The explanations below are general statistical context, not a description of analyses documented for this study.
What does a participant count describe?
For a binary outcome, each participant is classified as either having or not having the event being counted. A count can communicate how many participants had a particular outcome in the observed study group, but it is most informative when the event definition and the population included in the count are clear. This registry’s primary outcome uses multiple tumor-response categories rather than one clearly stated binary event. The registry describes the categories, but does not post the counts assigned to them. It is therefore not possible to recover a response count or a proportion from the record.
Counts and proportions answer different descriptive questions. A count reports the number of participants in a category; a proportion places that count in relation to a defined denominator. A denominator can depend on whether the analysis includes everyone enrolled, only those who received treatment, or only those with an assessable tumor evaluation. The intended analysis population and denominator for the primary endpoint are not reported in the registry record. No proportion should be inferred without that information and the corresponding outcome count.
Why do uncertainty intervals matter?
General statistical explanation: a confidence interval expresses the range of values compatible with the observed data under a specified method and assumptions. It helps readers distinguish a relatively precise estimate from one that is uncertain. The width of an interval depends in part on the amount of information and the variability in the outcome. A reported count without an interval can be a useful description, but it does not show the precision of an estimated treatment response. The registry record reports neither a primary response estimate nor an interval.
A confidence interval would not remove the limitations of a single-group design. It could describe uncertainty around an outcome in the treated group, but it could not create a concurrent counterfactual comparison. Estimating a treatment effect relative to another strategy generally requires a defensible comparator and an analysis suited to the design. This study has no randomized comparator group in the registry record.
What is the role of a statistical test?
General statistical explanation: a statistical test evaluates data against a specified null hypothesis using a prespecified method. Its interpretation depends on the hypothesis, analysis population, assumptions, and handling of multiple outcomes. A p-value alone does not measure the size or clinical importance of an effect, and a nonsignificant result does not prove that two strategies are equivalent. The registry record reports no primary-endpoint test or p-value and gives no statistical plan from which to identify a prespecified hypothesis.
The record lists five outcome measures and describes several response categories and secondary outcomes. That number of measures does not establish how many hypotheses were formally tested or whether any adjustment for multiplicity was planned. The record states zero analyses, and it does not report any analysis results. The presence of multiple outcomes is a reason to ask how findings were selected and interpreted, not a basis for assuming a particular correction method.
How should the planning illustrations be read?
General statistical explanation: sample-size planning asks how much information a future study would need to detect an effect under stated assumptions. Such calculations depend on the effect targeted, the significance level, power, allocation, outcome type, and analysis method. They are planning exercises, not evidence about an already completed study’s probability of finding a result. “Observed power” calculated after seeing an estimate is not a substitute for reporting the estimate and its uncertainty.
The planning description associated with the record specifies a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and an effect expressed as a stated fraction of the observed effect. It notes that time-to-event illustrations use a total event count under the Schoenfeld method, while other illustrations give participants per arm. These are planning assumptions, not a documented design justification for this single-group study. The sample-size justification for the trial is not reported in the registry record, and no planning illustration should be read as an observed trial result.
Why can over-dispersion matter?
General statistical explanation: over-dispersion occurs when observed variability is greater than a statistical model assumes. It can make standard errors too small and uncertainty intervals too narrow if the model does not accommodate the extra variability. It is relevant to some count-data models, especially where events may vary substantially among participants. The primary endpoint is described as a count of participants by response criteria, but the registry record does not report a count-data model or any over-dispersion assessment. There is no basis to claim that such a model was used or needed in the trial’s analysis.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists a primary outcome and several secondary outcomes, including engraftment, receipt of donor lymphocyte infusion, acute graft-versus-host disease grade 2 and higher, and chronic graft-versus-host disease. The primary response definition also contains multiple categories. Considering multiple outcomes or categories can raise multiplicity concerns when several statistical claims are tested: the chance of at least one apparently notable result may increase as the number of tests grows. General methods for addressing multiplicity include prespecifying a primary comparison, adjusting inference, or treating secondary findings as exploratory. The registry record does not report whether any such approach was planned or used.
Missing-data handling is also not reported in the registry record. The record does not say how participants without an assessable tumor evaluation were classified, whether any response assessments were missing, or whether any participants were excluded from the primary analysis. It likewise does not report the intended analysis population. These omissions matter because counts can change depending on who is included and how unavailable outcomes are treated.
The record reports zero analyses and does not post statistical analysis results. It is therefore not possible to determine whether analyses followed a prespecified plan, whether outcomes were selectively reported, or whether adjustment for multiple comparisons was applied. These are unknowns, not proof that a particular practice was or was not used. The registry record does not report an interim-analysis plan or stopping boundaries, so none should be assumed.
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 |
|---|---|---|---|
| Stem Cell Transplantation in Patients With Progressive and Incurable Metastatic Solid Tumors | 21 | 42 | 50.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Stem Cell Transplantation in Patients With Progressive and Incurable Metastatic Solid Tumors | 14 of 42 |
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 |
|---|---|
| Stem Cell Transplantation in Patients With Progressive and Incurable Metastatic Solid Tumors | 7 of 42 |
Most frequent serious adverse event terms
| Event term | Organ system | Stem Cell Transplantation in Patients With Progressive and Incurable Metastatic Solid Tumors |
|---|---|---|
| Progressive disease | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 12 of 42 |
| Acute Graft VS Host Disease of GI Tract | Gastrointestinal disorders | 4 of 42 |
| Mucositis with fever | Gastrointestinal disorders | 2 of 42 |
| Pseudomonas sepsis | Infections and infestations | 1 of 42 |
| Parainfluenza pneumonia and septic shock | Infections and infestations | 1 of 42 |
| Pulmonary aspergillus | Infections and infestations | 1 of 42 |
| Dyspnea with progressive mets to lungs | Respiratory, thoracic and mediastinal disorders | 1 of 42 |
| Fecal impaction | Gastrointestinal disorders | 1 of 42 |
| Community acquired pneumonia | Infections and infestations | 1 of 42 |
| Pneumonia | Infections and infestations | 1 of 42 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Stem Cell Transplantation in Patients With Progressive and Incurable Metastatic Solid Tumors |
|---|---|---|
| Hemorrhagic cystitis | Renal and urinary disorders | 4 of 42 |
| Cytomegalovirus disease | Infections and infestations | 3 of 42 |
Serious events with the highest arm-level rate: progressive disease (28.6%), acute graft vs host disease of gi tract (9.5%), mucositis with fever (4.8%), pseudomonas sepsis (2.4%), parainfluenza pneumonia and septic shock (2.4%).
The registry reports serious adverse events for the study group, with 21 participants affected and 42 at risk. It reports other adverse events affecting 7 participants, with 42 at risk, and records 14 deaths. These are registry counts; the record does not provide additional information about event timing, individual-level overlap, or attribution to the intervention.
The listed serious adverse-event terms include progressive disease at 28.6%, acute Graft VS Host Disease of GI Tract at 9.5%, mucositis with fever at 4.8%, pseudomonas sepsis at 2.4%, parainfluenza pneumonia and septic shock at 2.4%, and pulmonary aspergillus at 2.4%. These term-specific percentages should be read as registry-reported entries, not as estimates of comparative risk. The record does not report the underlying term counts or explain whether one participant could contribute to more than one term.
A serious adverse-event count is distinct from a causal assessment. The registry record does not report whether each event was judged related to transplantation, cyclosporin, methotrexate, or another factor. The event listing therefore describes safety outcomes recorded for the group but does not establish their cause or comparative frequency against an untreated or alternative-treatment group.
12. What the Result Does, and Does Not, Mean
The registry record establishes that this phase 2 study investigated non-myeloablative allogeneic stem cell transplantation in patients with progressive, incurable metastatic solid tumors refractory to standard therapy. It specifies a one-year tumor-response endpoint and gives operational definitions for complete response, partial response, stable disease, and progressive disease. It also records secondary safety and treatment-process outcomes, along with serious adverse-event counts and terms.
It does not report the primary tumor-response counts. As a result, the central endpoint cannot be summarized as a response rate or as a distribution across the response categories. No primary effect estimate or uncertainty interval is reported, and the registry gives no inferential analysis result. This absence does not mean that no participants responded; it means that the posted record does not provide the result needed to tell.
The single-group, nonrandomized design also means that any observed outcome within the treated group would not, on its own, establish what would have happened under another treatment. The study has no concurrent control group in the registry record. Thus, the design is suited to describing outcomes in the study group and exploring a treatment strategy, but it cannot support a randomized comparative-effect estimate.
The safety entries should likewise be interpreted as reported observations, not as proof of treatment causation. The registry does not provide event attribution or a control group for comparison. The listed adverse-event counts and terms are useful descriptive information, but they do not resolve how often comparable events would occur under another strategy.
Overall, the record documents the research question, intervention schedule, endpoint definitions, study status, and selected safety information. It does not provide the primary outcome results or analysis details needed to judge the magnitude, precision, or statistical evidence for a tumor response. No conclusion about effectiveness should be drawn from the endpoint description alone.
13. Why This Trial Matters Statistically
This study raises statistical issues that are central to interpreting exploratory clinical research. Its single-group design places the emphasis on describing outcomes in the treated group. Without randomization or a concurrent comparator, an observed tumor response could not by itself show how outcomes would differ under another treatment. That distinction between description and comparative inference is essential when reading results from an uncontrolled study.
The endpoint also illustrates the importance of clear outcome definitions. The registry distinguishes complete response, partial response, stable disease, and progressive disease using explicit criteria based on symptoms, lesion measurements, and new metastatic disease. Such definitions make the intended classifications more transparent. Yet a detailed endpoint definition is not a result: without the reported category counts and the population used in the analysis, readers cannot assess the observed response pattern.
The range of metastatic tumor types adds another interpretive challenge. The study’s objective was broad, but the registry does not report tumor-specific response results. A pooled result, if reported, would need careful interpretation because outcomes may vary by tumor type; the record does not provide information to assess that variation. This is a general concern for studies spanning heterogeneous patient groups, not evidence that any particular subgroup behaved differently.
Finally, the record separates what is known from what remains unavailable. It includes serious adverse-event information and lists secondary outcomes, but reports no primary-endpoint analysis and no statistical plan. This makes transparent reporting of denominators, analysis populations, missing outcomes, uncertainty, and multiplicity especially important. The study matters statistically as an example of why design, endpoint reporting, and analysis detail must be read together: none alone can answer the full clinical question.
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 for interpreting this record is that the tumor-response results are not reported in the registry record. Although the endpoint definitions and one-year timeframe are specified, there are no posted counts for complete response, partial response, stable disease, or progressive disease. The record also gives no primary effect estimate or uncertainty interval.
The study is single-group, nonrandomized, and has no comparator arm. Any outcomes observed among participants would therefore be descriptive and would not directly estimate a contrast with another treatment. The range of metastatic tumor types further limits interpretation of an overall finding without tumor-specific results; such results are not reported in the registry record.
The registry does not report the statistical plan, sample-size justification, analysis populations, missing-data methods, or whether interim analyses were conducted. It does not state how the primary outcome was summarized or whether any statistical testing was planned. The reason the study was terminated is not reported in the registry record. These gaps prevent assessment of whether the posted outcomes, if available elsewhere, followed a prespecified analysis strategy.
Safety reporting is also limited in the registry entry. Counts and term-specific percentages are listed, but the record does not report event attribution, timing, detailed participant-level overlap, or a comparator for contextualizing those events. The safety information can be described as recorded but cannot establish causation or comparative risk.
These limitations concern the information in the registry record and the study design; they do not establish that unreported analyses or assessments were never performed. They indicate that the record does not contain enough information to evaluate the primary response finding or its statistical precision.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Stem Cell Transplantation for Metastatic Solid Tumors — NCT00001880.
- Registry record last updated 2021-11-10; status last verified 2020-08.
- Results first posted 2021-11-10.
- 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 a phase 2, nonrandomized, single-group study of non-myeloablative allogeneic stem cell transplantation for progressive and incurable metastatic solid tumors refractory to standard therapy. It lists enrollment of 42, a terminated status, a start date of 1999-03-12, and a primary completion date of 2008-08-14.
The primary endpoint was the number of participants classified by tumor-response criteria over one year. The registry defines the response categories but does not report their participant counts or a primary statistical analysis. Secondary outcomes concern engraftment, donor lymphocyte infusion, and graft-versus-host disease.
Safety entries include 21 participants affected by serious adverse events, 7 affected by other adverse events, and 14 deaths. Serious adverse-event terms and percentages are also listed. The record does not report a comparator group, analysis plan, missing-data methods, or the reason for termination. The primary tumor-response result is not reported in the registry record.