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 Efficacy and Safety Study of CC-4047 (Pomalidomide) to Treat Advanced Soft Tissue Sarcoma (NCT00717522), a na phase 2 study. The primary outcome is Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Deaths, or Discontinuations Due to AEs.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00717522 |
| Phase | Phase 2 |
| Condition | Soft Tissue Sarcoma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 7 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Celgene |
| Results posted | Yes |
2. Clinical Question
Population
Participants had advanced soft tissue sarcomas that had relapsed or were refractory to prior systemic anticancer therapy. The registry records an enrollment of 7 participants. The primary outcome population is described as all participants.
Intervention
The study evaluated pomalidomide, also called CC-4047 in the registry title. The recorded regimen was 7 mg taken orally once daily on days 1 through 21 of each 28-day cycle.
Comparator
There was no comparator group. The design is recorded as a single-group study, with no allocation and no masking. Consequently, the posted results do not compare pomalidomide with another treatment or a control condition.
Primary question
The primary outcome asks how many participants experienced adverse events, serious adverse events, deaths, or discontinuations due to adverse events. The registry also lists tumor response assessed using RECIST committee guidelines as a secondary outcome. The posted results summarized in the primary outcome are safety counts; a tumor-response result is not reported in the registry record.
The clinical question is therefore descriptive: what adverse-event experience was recorded among the participants who received the study regimen? Because the design has no comparison group, it cannot estimate how those outcomes would have differed under another treatment or without the study treatment. The record’s stated purpose includes evaluating safety and efficacy, but the available primary result addresses event counts rather than a comparative efficacy estimate.
3. Trial Design
This was a phase 2, multicenter, open-label, single-arm study of pomalidomide in people with advanced soft tissue sarcoma after relapse or refractory disease following systemic anticancer therapy. The official title describes a two-stage study, but details of the stages, their decision rules, or how the stages were implemented are not reported in the registry record.
The registry lists enrollment of 7 participants and records the study status as terminated. The recorded start date is 2008-08-01 and the primary completion date is 2009-01-01. The registry does not report why the study was terminated, so the status alone does not establish the reason or whether a planned enrollment target was reached.
Participants received pomalidomide 7 mg orally once daily on days 1 through 21 of each 28-day cycle. The study was open-label, meaning masking was not used. Allocation is recorded as not applicable, consistent with the single-group design. This setup can describe outcomes among enrolled participants, but it does not provide a concurrent control group for separating treatment-associated events from events that might occur from the disease, prior therapy, or other causes.
The primary outcome concerned participant counts for adverse events, serious adverse events, deaths, and discontinuations due to adverse events. The registry gives an adverse-event observation window from informed consent to 30 days after the treatment discontinuation visit. It reports median treatment duration as 49 days, with a range of 3 to 102 days. The secondary outcome was tumor response assessed by RECIST committee guidelines, but results for that outcome are not reported in the registry record.
4. Participant Flow and Accounting
The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.
Overall Study
| Stage | Pomalidomide |
|---|---|
| STARTED | 7 |
| COMPLETED | 0 |
| NOT COMPLETED | 7 |
Reasons for not completing this period:
| Reason | Pomalidomide |
|---|---|
| Disease Progression | 5 |
| Adverse Event | 1 |
| Death | 1 |
Analysis population for the primary outcome, as defined in the registry: All participants
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 | Pomalidomide |
|---|---|---|
| Participants analysed | 7 | |
| Age, Continuous | years | 58.9 (SD 13.52) |
| Sex: Female, Male: Female | Participants | 3 |
| Sex: Female, Male: Male | Participants | 4 |
| Race/Ethnicity, Customized: White | participants | 7 |
| Race/Ethnicity, Customized: Non-white | participants | 0 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Deaths, or Discontinuations Due to AEs, measured over AEs/SAEs were recorded from informed consent to 30 days post treatment discontinuation visit. Median treatment duration was 49 days (range: 3 to 102 days)..
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is a participant count covering adverse events, serious adverse events, deaths, or discontinuations due to adverse events. An adverse event is defined in the record as an untoward medical occurrence that may appear or worsen during the study, regardless of cause. A serious adverse event is defined by outcomes such as death, life-threatening illness, hospitalization, persistent or significant disability, congenital anomaly, or another important medical event.
This type of endpoint is used to describe the safety experience observed during treatment and the stated follow-up window. The registry specifies that events were recorded from informed consent through 30 days after the treatment discontinuation visit. It also describes toxicity grading using NCI Common Toxicity Criteria for Adverse Events, Version 3.0, with categories from mild through death. These definitions clarify what the record counts as an event and how severity may be classified.
The reported primary result is a count, not a rate adjusted for time at risk and not a comparison between treatment groups. The record lists 7 participants with other adverse events and 3 with serious adverse events. It does not provide separate posted counts for deaths or discontinuations due to adverse events. A combined endpoint title should not be read as evidence that each component was separately reported or occurred; the registry record does not report those component-specific results.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The posted safety counts describe a small single-arm study population: 7 participants were enrolled, and the registry lists 7 participants with other adverse events and 3 with serious adverse events. These are participant counts. They do not give the total number of individual event episodes, the duration of each event, or a comparison with an untreated or differently treated group. The count for deaths is not reported in the registry record, and a separate count for discontinuations due to adverse events is also not reported.
The record lists serious adverse-event terms including Cancer Pain at 28.6%, and Sarcoma Metastatic, Anaemia, Febrile Neutropenia, Leukopenia, and Neutropenia at 14.3% each. These are terms and percentages as recorded. They should not be treated as evidence that the terms were mutually exclusive, that each represents a different participant, or that the event was caused by pomalidomide. The registry’s definition of an adverse event explicitly does not require a particular etiology.
Precision is limited by the small enrollment and by the absence of reported confidence intervals or a comparative analysis. A count from a small group can change substantially with the experience of an individual participant, so the figures are best understood as a description of this enrolled group rather than a stable estimate for a broader population. The median treatment duration was 49 days, with a range of 3 to 102 days; participants therefore had different recorded treatment durations. The registry does not provide person-time analyses or information that would allow the counts to be adjusted for differing time at risk.
The study has no control arm, so the posted counts cannot identify what portion of observed events was attributable to the study treatment rather than disease or other factors. The registry reports 0 analyses. It does not report an inferential analysis, an adjusted estimate, or a result for the listed tumor-response outcome. The record’s terminated status is not accompanied by a reason, and does not by itself explain the size or pattern of the results.
Overall, the posted results document adverse-event experience among the enrolled participants, with serious events recorded for 3 participants. They do not establish a comparative safety profile or provide a precise estimate of event risk in a wider patient group. Death and adverse-event-related discontinuation counts are not reported in the registry record, despite appearing in the primary endpoint title.
9. Statistical Methods Explained
General statistical context: A single-arm study summarizes outcomes within the group that received the study intervention. Participant counts are direct descriptive statistics: they state how many people in the recorded population experienced an outcome. They do not, by themselves, show whether the outcome is more or less frequent than it would have been under another treatment or in the absence of treatment. That distinction matters particularly for adverse events, which may have multiple possible causes.
For this study, the registry identifies the primary outcome as a number of participants with adverse events, serious adverse events, deaths, or discontinuations due to adverse events. It specifies all participants as the outcome population. The posted safety group has 7 participants at risk for serious events and 7 at risk for other adverse events. The corresponding participant counts are 3 for serious events and 7 for other adverse events. The registry record does not report a separate result for deaths or discontinuations due to adverse events.
General statistical context: A confidence interval expresses uncertainty around an estimate under a statistical model and assumptions. It is not a guarantee that a particular range contains the true value, and its interpretation depends on the design and analysis. No confidence interval for the primary participant counts is reported in the registry record. Therefore, the precision of these counts cannot be quantified from the posted primary result using an interval estimate.
Counts also differ from event rates. A participant count records whether people experienced events, while an event rate may account for the amount of time participants were observed. This distinction can matter when follow-up or treatment duration varies. The registry reports a median treatment duration of 49 days and a range of 3 to 102 days, but it does not report an event-rate analysis or person-time denominators. The adverse-event recording window is stated as informed consent through 30 days after the treatment discontinuation visit.
General statistical context: In an open-label study, participants and study personnel are not masked to the assigned intervention. Knowledge of treatment can affect how subjective outcomes are reported, monitored, or classified. Adverse-event definitions and grading criteria provide structure for recording events, but they do not remove all potential sources of judgment or establish causation. The registry states that NCI Common Toxicity Criteria for Adverse Events, Version 3.0, were used for grades, and defines seriousness using clinical consequences such as hospitalization or life-threatening illness.
The record lists serious-event terms with percentages: Cancer Pain at 28.6%; Sarcoma Metastatic, Anaemia, Febrile Neutropenia, Leukopenia, and Neutropenia at 14.3% each. Such terms describe reported event categories. The record does not clarify in the posted summary whether a participant could contribute more than one term, nor should the percentages be interpreted as causal effects. The terms can overlap within a participant, and the count of participants with any serious event is a separate summary from the frequency of each term.
General statistical context: A comparative effect estimate requires a clear comparison, such as a difference between groups, and uncertainty around that estimate. Because this study is single-arm, there is no randomized or concurrent comparator in the record. A before-and-after comparison or comparison with external experience would require additional data and assumptions that are not reported. No comparative efficacy or safety effect can be recovered from the participant counts alone.
The study title describes a two-stage design, but the registry does not report the stage-specific sample sizes, stopping boundaries, decision criteria, or operational details. The sample-size justification and the statistical plan are not reported in the registry record. Interim analyses are also not reported. These omissions mean that the design label cannot be used to infer how decisions were made during the study.
The sample-size planning illustration uses a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the effect to detect expressed as a stated fraction of the observed effect. It is an illustration based on an observed effect, not the trial’s own design justification. Because this study itself has one group, those assumptions do not describe its allocation. Any such planning figures are hypothetical design illustrations, not observed power and not evidence that a treatment effect was established.
The registry lists 0 analyses. It does not report a model, adjusted result, hypothesis test, or interval estimate for the primary endpoint. The secondary outcome is tumor response assessed by RECIST committee guidelines, but a response result is not reported in the registry record. These gaps limit the statistical conclusions to the posted descriptive safety counts and event-term summaries.
10. Multiplicity, Missing Data and Analysis Populations
Multiplicity arises when a study evaluates several outcomes, time points, groups, or statistical tests. Without a prespecified strategy, repeated testing can increase the chance of a false-positive finding. The registry lists a primary safety outcome and a secondary tumor-response outcome. It does not report how multiplicity was handled, whether any outcomes had priority beyond their primary or secondary labels, or whether any adjustment to statistical testing was planned. The registry reports 0 analyses, so no multiplicity-adjusted or unadjusted hypothesis-test results are available in the posted record.
Missing-data methods are not reported in the registry record. The posted primary safety summary identifies 7 participants in the group and gives counts for serious and other adverse events, but it does not describe missing outcome data, incomplete follow-up, or how any such data would have been handled. The record reports a safety observation window and treatment-duration summary, but those details do not establish that every participant had complete observation through the same follow-up period.
Analysis populations are also not described beyond the primary outcome population being identified as all participants. The registry does not report whether analyses used an intention-to-treat, per-protocol, or safety population definition, nor does it describe exclusions from analysis. The enrollment of 7 should not be assumed to equal a separately defined analysis population unless the record states so.
Because the listed tumor-response outcome has no posted result, its missing result should not be interpreted as no response or as a negative finding. Likewise, missing component-specific counts for deaths and adverse-event-related discontinuations do not establish that no such outcomes occurred. Those results are not reported in the registry record.
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 |
|---|---|---|---|
| Pomalidomide | 3 | 7 | 42.9% |
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 |
|---|---|
| Pomalidomide | 7 of 7 |
Most frequent serious adverse event terms
| Event term | Organ system | Pomalidomide |
|---|---|---|
| Cancer Pain | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 2 of 7 |
| Sarcoma Metastatic | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 1 of 7 |
| Anaemia | Blood and lymphatic system disorders | 1 of 7 |
| Febrile Neutropenia | Blood and lymphatic system disorders | 1 of 7 |
| Leukopenia | Blood and lymphatic system disorders | 1 of 7 |
| Neutropenia | Blood and lymphatic system disorders | 1 of 7 |
| Tachycardia | Cardiac disorders | 1 of 7 |
| Dysphagia | Gastrointestinal disorders | 1 of 7 |
| Malnutrition | Metabolism and nutrition disorders | 1 of 7 |
| Muscle Spasms | Musculoskeletal and connective tissue disorders | 1 of 7 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Pomalidomide |
|---|---|---|
| Nausea | Gastrointestinal disorders | 3 of 7 |
| Anaemia | Blood and lymphatic system disorders | 3 of 7 |
| Neutropenia | Blood and lymphatic system disorders | 3 of 7 |
| Constipation | Gastrointestinal disorders | 2 of 7 |
| Dry mouth | Gastrointestinal disorders | 2 of 7 |
| Fatigue | General disorders | 2 of 7 |
| Thrombocytopenia | Blood and lymphatic system disorders | 2 of 7 |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | 2 of 7 |
| Hypoalbuminaemia | Metabolism and nutrition disorders | 2 of 7 |
| Hypocalcaemia | Metabolism and nutrition disorders | 2 of 7 |
Serious events with the highest arm-level rate: cancer pain (28.6%), sarcoma metastatic (14.3%), anaemia (14.3%), febrile neutropenia (14.3%), leukopenia (14.3%).
The serious-event summary lists 3 participants with serious adverse events among 7 participants at risk. The registry also lists event terms with percentages: Cancer Pain at 28.6%, and Sarcoma Metastatic, Anaemia, Febrile Neutropenia, Leukopenia, and Neutropenia at 14.3% each. These entries are descriptions of recorded serious-event categories, not estimates of causal risk attributable to pomalidomide.
Participant counts and event-term percentages answer different descriptive questions. The count of participants with a serious adverse event summarizes people with at least one such event; individual terms describe reported categories. The record does not establish that categories are mutually exclusive or that each category corresponds to a unique participant. Multiple terms may describe events in the same participant, so the percentages should not be added together.
The registry defines serious adverse events by clinical consequences, including death, life-threatening events, hospitalization, persistent or significant disability, congenital anomaly, or another important medical event. It records adverse events from informed consent through 30 days after the treatment discontinuation visit. A separate death count is not reported in the registry record. The listed serious-event terms should therefore be read as descriptive safety information, with the limitations of a small, uncontrolled study and without a causal comparison.
12. What the Result Does, and Does Not, Mean
The result establishes that adverse events were recorded in this small study group. The registry lists 7 participants with other adverse events and 3 participants with serious adverse events among the 7 enrolled participants. It also provides serious-event terms and percentages. These observations describe what was recorded during the study’s specified safety window; they are not estimates from a randomized comparison.
The counts do not show that pomalidomide caused each event. The registry’s adverse-event definition includes occurrences regardless of etiology, and the single-arm design offers no concurrent group to help distinguish treatment-associated events from disease-related events or other causes. Nor can the counts establish how the event experience would compare with another treatment. Such conclusions would require comparative data and an analysis not reported in the registry record.
The results also do not establish the absence of events that are not separately reported. Although deaths and discontinuations due to adverse events appear in the primary outcome title, component-specific counts are not reported. The secondary tumor-response outcome is listed, but its result is not reported in the registry record. Missing results should not be interpreted as zero events, no response, or evidence in either direction.
Precision is constrained by enrollment of 7 participants, the variation in treatment duration, and the absence of reported confidence intervals or event-rate analyses. The observed counts are informative as a record of experience among these participants, but they cannot by themselves establish how frequently similar events would occur in a larger or different population. The study’s terminated status does not supply an explanation for the results or prove that a particular enrollment or analysis plan was followed.
In short, the posted safety figures are descriptive evidence from a small, open-label, single-arm study. They should not be treated as proof of benefit, lack of benefit, or comparative safety. The registry does not report the inferential analyses or efficacy results needed to support those interpretations.
13. Why This Trial Matters Statistically
This study is statistically informative as an example of the limits of interpreting participant counts from a small, uncontrolled clinical study. A safety count can document events that occurred in an enrolled group, but a count alone does not quantify a comparative treatment effect. Without a control group, observed events cannot be separated statistically from events that might have occurred because of the underlying disease, prior treatment, or other circumstances.
The record also illustrates why outcome definitions and analysis details matter. The primary outcome title combines adverse events, serious adverse events, deaths, and discontinuations due to adverse events, while the posted results include participant counts for serious and other adverse events. A title that names several components does not substitute for component-specific results. Here, death and adverse-event-related discontinuation counts are not reported in the registry record.
Precision deserves attention as well. Enrollment was 7, and no confidence intervals or comparative estimates are reported. The counts therefore describe this group but do not quantify uncertainty in a way that supports broad generalization. Treatment duration ranged from 3 to 102 days, while the record does not provide a time-adjusted event analysis. That distinction is important when participants have differing exposure or observation time.
Finally, the official title calls the design two-stage, yet stage details and stopping rules are not reported. The registry records 0 analyses, and the statistical plan is not reported in the registry record. Those absences limit the ability to assess how the planned design relates to the posted outcomes. The study is a useful reminder to distinguish a design label from the documented analysis and to distinguish observed safety descriptions from comparative evidence.
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 important limitation is the single-arm design. With no comparator group, the study can describe events among participants who received pomalidomide but cannot estimate a comparative safety or efficacy effect. Open-label conduct also means masking was not used, which can influence reporting and classification of outcomes, particularly when assessment involves judgment.
Enrollment was 7, limiting the amount of observed information. The record reports counts but no confidence intervals, adjusted estimates, or event-rate analyses. Treatment duration varied, with a median of 49 days and a range of 3 to 102 days. Although the adverse-event observation window is specified, the registry does not provide time-adjusted comparisons or details sufficient to characterize each participant’s observation time.
The primary outcome title includes deaths and discontinuations due to adverse events, but separate results for those components are not reported in the registry record. The secondary tumor-response outcome is listed, but its result is not reported. The registry reports 0 analyses, and does not report missing-data handling, analysis-population definitions, multiplicity procedures, or the statistical plan. The two-stage design label is not accompanied by stage-specific details or decision rules.
The study is recorded as terminated, but the reason for termination is not reported in the registry record. The record also does not explain whether enrollment matched a planned target. These limitations mean that the safety counts should remain descriptive and should not be used to infer a treatment effect, a causal relationship, or results for outcomes without posted findings.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Efficacy and Safety Study of CC-4047 (Pomalidomide) to Treat Advanced Soft Tissue Sarcoma — NCT00717522.
- Registry record last updated 2019-11-19; status last verified 2019-11.
- Results first posted 2013-04-16.
- 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 describes a phase 2, multicenter, open-label, single-arm study of pomalidomide in advanced soft tissue sarcoma after relapse or refractory disease following systemic anticancer therapy. The recorded regimen was 7 mg orally once daily on days 1 through 21 of each 28-day cycle. Enrollment was 7, and the study status is terminated.
The primary outcome concerns participant counts for adverse events, serious adverse events, deaths, or discontinuations due to adverse events. The registry lists 7 participants with other adverse events and 3 with serious adverse events. Death and adverse-event-related discontinuation counts are not reported in the registry record. A secondary tumor-response outcome was listed, but its result is not reported.
The study has no comparator group, and the registry reports 0 analyses. The reason for termination, statistical plan, missing-data procedures, and details of the two-stage design are not reported in the registry record. The posted safety counts are descriptive observations from this small study and do not establish comparative or causal effects.