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 Adjuvant Chemotherapy for High Risk Uterine Leiomyosarcoma (NCT00282087), a na phase 2 study. The primary outcome is Two-year Progression-free Survival Among Women Treated With This Adjuvant Regimen for High Risk Uterine LMS.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00282087 |
| Phase | Phase 2 |
| Condition | Leiomyosarcoma, Uterine Neoplasm |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 47 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Sarcoma Alliance for Research through Collaboration |
| Results posted | Yes |
2. Clinical Question
The trial asks whether progression-free survival can be described after adjuvant chemotherapy for women with high-risk uterine leiomyosarcoma who have undergone surgical resection and have no evidence of disease remaining. The registry record identifies two-year progression-free survival as the primary outcome. It also lists tolerability and toxicity, along with associations between progression-free survival and several clinical or tumor characteristics, as secondary outcomes.
Population
Women treated after surgical resection of high-risk uterine leiomyosarcoma, with no evidence of disease remaining. The registry record lists leiomyosarcoma and uterine neoplasm as conditions.
Intervention
Adjuvant chemotherapy with gemcitabine and docetaxel followed by doxorubicin. The regimen description specifies drug doses and administration schedules.
Comparator
There is no separate comparison group in this single-group trial. Allocation is listed as NA, so the design does not provide a like-for-like control group.
Primary question
What percentage of participants has two-year progression-free survival after treatment with this adjuvant regimen? The registry record lists a 95% Confidence Interval but does not report the outcome estimate.
This question is descriptive for the treated group. Without a concurrent control group, the trial cannot directly estimate how outcomes would have differed under another treatment or no adjuvant treatment. The registry record does not report a numerical primary outcome result, so it does not establish the observed two-year progression-free survival in this trial.
3. Trial Design
This is a completed phase II trial with a single-group model and no masking. The registry lists enrollment of 47. The study start is recorded as 2006-01, and primary completion as 2012-01. The sponsor is Sarcoma Alliance for Research through Collaboration.
The treatment is described as gemcitabine/docetaxel followed by doxorubicin. The registry gives gemcitabine at 900 mg/m2 IV over 90 minutes on days 1 and 8, docetaxel at 75 mg/m2 IV on day 8, and doxorubicin at 60 mg/m2 IVP every 21 days for 4 cycles. It also describes dexamethasone pre-medication before docetaxel and recommends central venous catheter access for doxorubicin.
The brief summary describes adjuvant treatment after surgical resection when no evidence of disease remains. The principal objective is progression-free survival. The primary outcome title specifies two-year progression-free survival, with assessment every 3 months up to two years. The outcome is expressed as a percentage of participants, and a 95% Confidence Interval is listed as the dispersion measure.
Because this is a single-group study, its design does not include a randomized comparator. Allocation is listed as NA, and the registry identifies no masking. These features mean that observed outcomes in the treated group cannot, by themselves, distinguish treatment effects from other explanations for outcomes over time. The registry record does not report a detailed statistical analysis plan, eligibility criteria, or a sample-size justification. The trial's completed status indicates its registry status, not that results for every outcome are reported.
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 | Women Treated With Adjuvant Regimen for High Risk Uterine LMS |
|---|---|
| STARTED | 47 |
| COMPLETED | 46 |
| NOT COMPLETED | 1 |
Reasons for not completing this period:
| Reason | Women Treated With Adjuvant Regimen for High Risk Uterine LMS |
|---|---|
| Death | 1 |
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 | Women Treated With Adjuvant Regimen for High Risk Uterine LMS |
|---|---|---|
| Participants analysed | 47 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 44 |
| Age, Categorical: >=65 years | Participants | 3 |
| Age, Continuous | years | 53 (SD 16) |
| Sex: Female, Male: Female | Participants | 47 |
| Sex: Female, Male: Male | Participants | 0 |
| Region of Enrollment: United States | participants | 47 |
6. Primary Endpoint
The registry lists the primary outcome as Two-year Progression-free Survival Among Women Treated With This Adjuvant Regimen for High Risk Uterine LMS, measured over Every 3 months up to two years.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | 95% Confidence Interval |
| Unit | percentage of participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is titled two-year progression-free survival among women treated with the adjuvant regimen. It is expressed as a percentage of participants, and the registry specifies a 95% Confidence Interval. The timeframe is every 3 months up to two years.
Progression-free survival is used in clinical research to describe how long participants remain alive without disease progression, according to the study's definitions and assessment procedures. In general, a time-to-event endpoint can capture both whether an event occurs and when it occurs. That can be useful when outcomes are followed over time rather than recorded only at a single visit. This general explanation does not establish which event definitions, assessment rules, or censoring procedures this trial used.
The registry record does not report a numerical estimate for the primary endpoint, its confidence interval limits, the number of progression or death events, or the analysis population. It also does not give the operational definition of progression-free survival. Consequently, the endpoint's intended subject is identifiable, but its observed value and the precision of that value cannot be assessed from the registry record.
A confidence interval is a statistical expression of uncertainty around an estimate. In general, it describes a range of values compatible with the data under the method used; it is not a guarantee that the true value lies within that range. Without the reported estimate, interval limits, and analysis method, the listed 95% Confidence Interval cannot be interpreted numerically.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record describes a treatment group and identifies two-year progression-free survival as the primary outcome. It does not report the percentage observed, the interval limits, or the number of events contributing to that outcome. Therefore, the posted registry information does not establish the trial's observed progression-free survival or how precisely it was estimated.
The absence of a reported estimate is not evidence that the outcome was favorable or unfavorable. It also does not show that the treatment did or did not affect progression-free survival. The record supplies the endpoint title and assessment timeframe, but not the result needed to describe the outcome in this trial.
Interpretation is also limited by the single-group design. In general, outcomes in an untreated or differently treated control group help distinguish what occurred under the study regimen from what might have occurred otherwise. This trial has no such concurrent comparator, and its allocation is listed as NA. As a result, even a reported outcome percentage would describe participants in the trial rather than provide a direct estimate of a treatment contrast.
The record lists a 95% Confidence Interval as the measure of dispersion. In general, interval width conveys precision: a wider interval indicates greater uncertainty about the estimate than a narrower interval, under the method's assumptions. The limits are not reported, so precision cannot be judged. The record also does not state the method used to estimate the interval.
Secondary outcomes include tolerability and toxicity and associations between progression-free survival and age, menopausal status at diagnosis, uterine serosal involvement, mitotic rate, estrogen receptor status, progesterone receptor status, and 1988 FIGO stage. The registry record names these outcomes but does not report their results. The listed outcomes should not be read as evidence that any association was found.
Overall, the registry record establishes the trial's setting, intervention, design, and intended primary outcome. It does not supply numerical efficacy results or enough analysis detail to assess the primary endpoint. Any interpretation of treatment benefit, comparative effectiveness, or subgroup patterns would go beyond the reported information.
9. Statistical Methods Explained
The registry identifies the primary outcome as a percentage of participants and lists a 95% Confidence Interval. It does not report the primary estimate, the confidence limits, the calculation method, or the analysis population. The explanations below distinguish general statistical concepts from methods documented for this trial.
What does a percentage outcome represent?
General explanation: A percentage outcome expresses the share of an analysis group meeting a specified condition. For progression-free survival, interpretation depends on how the condition is defined, how participants are followed, and how events and incomplete follow-up are handled. The registry record identifies two-year progression-free survival as the endpoint, but does not provide a numerical percentage or the detailed rules used to derive it.
A percentage calculated from participants observed at a particular time and a time-to-event estimate that accounts for different follow-up durations are not necessarily the same quantity. The registry's outcome description does not state the estimator or how follow-up ending before the endpoint assessment was handled. It is therefore not possible to infer the calculation from the outcome title alone.
How should the confidence interval be understood?
General explanation: A confidence interval communicates sampling uncertainty around an estimate under a statistical method and its assumptions. The estimate is the central result; the interval indicates the range of values that remain compatible with the observed data under that method. Greater precision generally corresponds to a narrower interval. An interval does not describe all sources of uncertainty, such as bias from study design or incomplete outcome reporting.
The registry lists a 95% Confidence Interval, but does not report its limits or method. Without those details, readers cannot evaluate the interval's width, check its relation to the estimate, or determine whether it was calculated using a time-to-event method or another approach. The confidence level alone is not enough to reconstruct the result.
What can a single-group design establish?
General explanation: A single-group study can describe outcomes among participants who received a regimen. A concurrent comparison group is needed for a direct estimate of how outcomes differ between treatment strategies. When there is no control group, differences between the observed outcome and an external expectation can be affected by patient selection, clinical setting, follow-up, and other factors.
Here, the registry lists a single-group model, allocation as NA, and masking as none. The design therefore does not support a randomized treatment comparison. The record does not report a statistical adjustment that would create a comparator, nor should an adjustment be assumed.
What do the planning assumptions mean?
The stated sample-size convention describes a planning illustration based on an observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. This is a planning framework, not a description of the trial's single-group design. The registry record does not report that these assumptions were used to design the trial. Sample-size illustrations are hypothetical planning quantities, not observed power and not evidence that an effect was detected. No numerical planning result is stated in the registry record.
General explanation: Planning calculations ask what sample size might be needed under specified assumptions about an effect, variability, significance level, and desired power. Their relevance depends on whether those assumptions are suitable for the proposed future design. A hypothetical two-arm calculation should not be mistaken for a comparison performed in a single-group trial.
What cannot be assessed from the registry record?
The registry does not report the primary endpoint estimate, confidence limits, event count, analysis population, or detailed statistical methods. The progression-free survival definition, censoring rules, handling of missing assessments, and any sensitivity analyses are not reported in the registry record. These omissions prevent independent assessment of how the endpoint was constructed and whether the reported precision reflects the follow-up actually available.
General explanation: In time-to-event analyses, censoring can occur when a participant's event status is not observed through the full follow-up period. Valid interpretation depends on how censoring is defined and on assumptions about its relationship to the event. Missing outcome assessments can also affect an estimate if missingness is related to participants' health or prognosis. These concepts explain why analysis details matter; they do not imply that any particular censoring or missing-data method was used in this trial.
10. Exact Estimates for Each Arm
No two-arm comparison is shown for this trial, but each arm's result can still be summarized with exact interval estimates computed from the posted counts. The Clopper-Pearson interval is exact and conservative; the Wilson interval is a common closer-to-nominal alternative; the Bayesian credible interval uses a uniform Beta(1, 1) prior. For small groups the three can differ noticeably.
| Arm | Participants | Events | Observed % | Clopper-Pearson 95% CI | Wilson 95% CI | Bayesian 95% credible interval |
|---|---|---|---|---|---|---|
| Women Treated With Adjuvant Regimen for High Risk | 46 | 36 | 78.3% | 63.6% to 89.1% | 64.4% to 87.7% | 64.3% to 87.7% |
R code and output
arm <- c("Women Treated With Adjuvant Regimen for High Risk ")
n <- c(46)
events <- c(36)
for (i in seq_along(n)) {
ci <- binom.test(events[i], n[i])$conf.int # Clopper-Pearson exact interval
cr <- qbeta(c(0.025, 0.975), 1 + events[i], 1 + n[i] - events[i]) # Bayesian, uniform Beta(1,1) prior
wi <- (function(x, n) { p <- x / n; z <- qnorm(0.975); d <- 1 + z^2 / n
c((p + z^2 / (2 * n) - z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d, (p + z^2 / (2 * n) + z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d) })(events[i], n[i])
cat(sprintf("ARM|%s|%d|%d|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f\n", arm[i], n[i], events[i], 100 * events[i] / n[i],
100 * ci[1], 100 * ci[2], 100 * wi[1], 100 * wi[2], 100 * cr[1], 100 * cr[2]))
}
ARM Women Treated With Adjuvant Regimen for High Risk 46 36 78.3 63.6 89.1 64.4 87.7 64.3 87.711. Multiplicity, Missing Data and Analysis Populations
The registry lists 10 outcome measures and 9 analyses. The primary outcome is two-year progression-free survival. Secondary outcomes include tolerability and toxicity, as well as correlations between progression-free survival and age, menopausal status at diagnosis, uterine serosal involvement, mitotic rate, estrogen receptor status, progesterone receptor status, and 1988 FIGO stage.
The registry record does not report a multiplicity adjustment, a prespecified hierarchy among secondary outcomes, or a strategy for controlling the chance of false-positive findings across multiple analyses. General explanation: When several outcomes or subgroup associations are tested, the chance of at least one apparently notable result can increase. A stated adjustment or analysis hierarchy can help readers understand how that issue was addressed. No such approach should be inferred when it is not described.
Results for the listed secondary outcomes are not reported in the registry record. It is therefore not possible to determine which analyses were completed, how many participants contributed to each, or whether any reported associations were exploratory or confirmatory.
The registry record also does not describe missing-data handling, the analysis population for the primary endpoint, exclusions after enrollment, or sensitivity analyses. These details matter because incomplete follow-up or unavailable assessments can affect the population contributing to an estimate. Their absence from the registry record does not establish that data were missing or that a particular handling method was used; it means the approach is not reported.
12. 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 |
|---|---|---|---|
| Women Treated With Adjuvant Regimen for High Risk Uterine LMS | 9 | 47 | 19.1% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 4% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Women Treated With Adjuvant Regimen for High Risk Uterine LMS | 14 of 47 |
Most frequent serious adverse event terms
| Event term | Organ system | Women Treated With Adjuvant Regimen for High Risk Uterine LMS |
|---|---|---|
| Chest Pain | Cardiac disorders | 2 of 47 |
| Neutropenia | Blood and lymphatic system disorders | 2 of 47 |
| Neutrophils | Investigations | 2 of 47 |
| Perforation of bowel | Gastrointestinal disorders | 1 of 47 |
| Hernia | Injury, poisoning and procedural complications | 1 of 47 |
| Colostomy Reversal | Gastrointestinal disorders | 1 of 47 |
| Diarrhea | Gastrointestinal disorders | 1 of 47 |
| Throat Pain | Respiratory, thoracic and mediastinal disorders | 1 of 47 |
| Bilirubin | Investigations | 1 of 47 |
| Platelets | Investigations | 1 of 47 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Women Treated With Adjuvant Regimen for High Risk Uterine LMS |
|---|---|---|
| myelosuppression | Endocrine disorders | 14 of 47 |
| Hypersensitivity | Immune system disorders | 10 of 47 |
| neuropathy | Nervous system disorders | 3 of 47 |
| Edema | Skin and subcutaneous tissue disorders | 2 of 47 |
Serious events with the highest arm-level rate: chest pain (4.3%), neutropenia (4.3%), neutrophils (4.3%), perforation of bowel (2.1%), hernia (2.1%).
The registry lists 47 participants at risk for serious adverse events and reports 9 affected. It also lists 47 at risk for other adverse events and reports 14 affected. These are separate registry categories; the record does not state whether participants in the categories overlap. Deaths are not reported in the registry record.
The registry's top serious adverse event terms include Chest Pain and Neutropenia at 4.3% each; Neutrophils at 4.3%; and Perforation of bowel, Hernia, and Colostomy Reversal at 2.1% each. These percentages are reported as terms, not as a complete account of every event or a comparison with another treatment group. The registry does not report event-specific severity, timing, duration, attribution, or the number of affected participants for each term.
General explanation: Adverse-event terms can describe events rather than unique people, and a person may have more than one event. A list of common terms is not a substitute for the full safety dataset. Without a control group, the reported counts do not establish whether events occurred more or less often than they would under another treatment strategy.
13. What the Result Does, and Does Not, Mean
The registry record documents a completed phase II, single-group study of adjuvant gemcitabine/docetaxel followed by doxorubicin for high-risk uterine leiomyosarcoma. It identifies two-year progression-free survival as the primary outcome and lists several safety and clinical or tumor-characteristic outcomes. It does not report numerical results for the primary or secondary outcomes.
This means the record can be used to understand what the study intended to evaluate and how the treatment regimen was described, but not to state the observed progression-free survival. The listed 95% Confidence Interval indicates that uncertainty was intended to accompany the primary outcome measure; its limits and the estimate are not reported. No conclusion about the size or precision of the primary result can be drawn.
The single-group design also limits what any observed outcome could establish. General explanation: A treated group's outcome is descriptive. Without a concurrent control group, it is not a direct estimate of the difference between treatment options. Differences from outcomes in another population or study can reflect differences in eligibility, care, follow-up, or patient characteristics, in addition to treatment.
The safety record reports affected counts and selected serious adverse event terms. Those entries describe what is recorded in the registry; they do not establish comparative safety or attribute an event to the regimen. The record does not report deaths.
Accordingly, the evidence available in the registry record supports a description of the study design, regimen, intended outcomes, and selected adverse-event entries. It does not support a claim that the treatment improved or failed to improve progression-free survival, nor does it establish how outcomes compare with another approach. Such conclusions would require reported outcome estimates and, for comparative claims, an appropriate comparison.
14. Why This Trial Matters Statistically
This trial is statistically informative as an example of the distinction between an outcome question and a reported outcome result. The registry specifies a two-year progression-free survival endpoint, its percentage scale, a 95% Confidence Interval, and a follow-up timeframe. Yet it does not report the estimate or interval limits. Naming an endpoint is not equivalent to reporting its findings.
The design also matters for interpretation. A single-group phase II study can describe outcomes among treated participants, but it does not provide a randomized control comparison. This distinction should remain clear even if a numerical estimate is available: precision around a treated-group outcome does not remove the limitations of a non-comparative design.
The record lists several secondary outcomes, including toxicity and associations with clinical or tumor characteristics. Such outcomes can be valuable for describing a study's scope, but interpretation requires results and information about analysis choices. The registry does not report a multiplicity strategy or results for these outcomes.
Finally, the planning assumptions are explicitly an illustration rather than a report of the trial's design. Keeping planning calculations separate from observed evidence prevents a hypothetical sample-size exercise from being mistaken for trial power or a treatment comparison. The statistical lesson is to distinguish design, outcome definition, estimate, uncertainty, and causal interpretation; each requires different information.
15. 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 principal limitation for interpreting outcomes is that the registry record does not report the primary progression-free survival estimate or its confidence limits. It also does not report results for the listed secondary outcomes. The record therefore does not permit a numerical account of efficacy, subgroup associations, or the precision of the primary result.
The single-group design has no concurrent control, and allocation is listed as NA. This prevents a direct like-for-like treatment comparison. Masking is listed as none. The registry record does not describe outcome-assessment procedures or whether assessors were independent.
The record does not report detailed eligibility criteria, the operational definition of progression-free survival, censoring rules, analysis populations, missing-data methods, or sensitivity analyses. It also does not report a sample-size justification or a detailed statistical plan. These are limitations of the information reported in the registry record; they do not show what may have been documented elsewhere.
The listed safety terms and affected counts do not provide full event-level detail or a comparator. The record does not report deaths. It is also not possible to assess the results of the planned correlations with age, menopausal status, uterine serosal involvement, mitotic rate, estrogen receptor status, progesterone receptor status, or 1988 FIGO stage because those results are not reported.
16. Related Tutorials
17. Related Statistical Calculators
18. Sources
- ClinicalTrials.gov: Adjuvant Chemotherapy for High Risk Uterine Leiomyosarcoma — NCT00282087.
- Registry record last updated 2014-12-01; status last verified 2014-11.
- Results first posted 2014-12-01.
- 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.
Continue through the Clinical Biostats statistical pathway
Use this trial's endpoint as a starting point for deeper study of effect measures, confidence intervals, and sample-size planning.
19. Record Summary
NCT00282087 is a completed phase II, single-group trial titled Adjuvant Chemotherapy for High Risk Uterine Leiomyosarcoma. The registry lists enrollment of 47 and identifies Sarcoma Alliance for Research through Collaboration as sponsor. The treatment is gemcitabine/docetaxel followed by doxorubicin, given as adjuvant therapy after surgical resection.
The primary outcome is two-year progression-free survival, expressed as a percentage of participants, with a 95% Confidence Interval and assessments every 3 months up to two years. The registry record does not report the primary estimate or confidence limits. It lists tolerability and toxicity and several clinical or tumor-characteristic associations as secondary outcomes, but their results are not reported.
The safety entries list 9 affected among 47 at risk for serious adverse events and 14 affected among 47 at risk for other adverse events. The record reports selected serious adverse event terms and does not report deaths.