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 Liposomal Doxorubicin Followed By Bexarotene in Treating Patients With Cutaneous T-Cell Lymphoma (NCT00255801), a na phase 2 study. The primary outcome is Median Progression-free Survival.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00255801 |
| Phase | Phase 2 |
| Condition | Lymphoma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 37 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Memorial Sloan Kettering Cancer Center |
| Results posted | Yes |
2. Clinical Question
Population
The study concerns patients with cutaneous T-cell lymphoma, described in the official title as advanced stage disease. The registry lists lymphoma as the condition and an enrollment of 37. Further eligibility details and a fuller description of the analyzed population are not reported in the registry record.
Intervention
The planned sequence begins with intravenous pegylated liposomal doxorubicin hydrochloride, identified as Doxil, every two weeks for 8 doses over 16 weeks. Responses are assessed after that treatment. Patients then receive oral bexarotene, identified as Targretin, for at least 16 weeks; those achieving a complete or partial response may continue bexarotene until relapse.
Comparator
There is no separate comparator group. The registry identifies a single-group study and no allocation to competing arms. Consequently, the record does not provide a randomized, concurrent estimate of how outcomes would have differed under another treatment or under no study treatment.
Primary question
The primary endpoint is median progression-free survival, with a timeframe of 3 years and units of months. The clinical question is how long patients in this treatment sequence remain free of progression, as defined by the study’s assessment criteria. The numerical median, its uncertainty, and an analysis result are not reported in the registry record, so the endpoint’s observed value cannot be described from this record.
3. Trial Design
This is a phase II, single-group study sponsored by Memorial Sloan Kettering Cancer Center. The registry lists allocation as not applicable and masking as none. Enrollment is recorded as 37. The record marks the study completed, with a start in 2005-11 and primary completion in 2017-10.
The planned regimen is sequential rather than a comparison between parallel treatment arms. Patients receive intravenous Doxil every two weeks for 8 doses, a course described as 16 weeks. Responses are then assessed, followed by oral Targretin for at least 16 weeks. Patients who achieve a complete response or partial response may continue Targretin until relapse. These descriptions state the planned treatment sequence; the registry record does not report how many patients completed each stage or their treatment exposure.
A single-group phase II design can describe outcomes after a specified regimen, but without a concurrent control group it cannot isolate the regimen’s effect from other explanations for observed outcomes. The registry record does not report a statistical analysis plan, sample-size justification, interim analyses, or an account of protocol changes. It also does not report numerical primary endpoint results or analysis records. The study’s completion status therefore should not be mistaken for evidence that a result is available in this 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 | Doxil and Targretin® (Bexarotene) |
|---|---|
| STARTED | 37 |
| COMPLETED | 37 |
| NOT COMPLETED | 0 |
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 | Doxil and Targretin® (Bexarotene) |
|---|---|---|
| Participants analysed | 37 | |
| Age, Continuous | years | 56 (27 to 81) |
| Sex: Female, Male: Female | Participants | 17 |
| Sex: Female, Male: Male | Participants | 20 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 7 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 30 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 1 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 13 |
| Race (NIH/OMB): White | Participants | 22 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 1 |
| Region of Enrollment: United States | Participants | 37 |
6. Primary Endpoint
The registry lists the primary outcome as Median Progression-free Survival, measured over 3 years.
| Element | Registry entry |
|---|---|
| Result type | Median |
| Measure of spread | Full Range |
| Unit | months |
| Endpoint classification | Time-to-Event Analysis |
The primary endpoint is median progression-free survival, measured in months, with a stated timeframe of 3 years. Progression-free survival is a time-to-event measure: it describes the time from a defined starting point until a qualifying event. The median is the point in the estimated event-time distribution by which half of the relevant population has had an event, subject to the endpoint definition and analysis methods.
The registry describes assessments for response and progression. These include imaging for specified stage IV patients with positive findings before treatment, blood and flow-cytometry assessments for patients with Sézary syndrome, and skin assessments using the modified Severity-Weighted Assessment Tool. Progression for the described skin and lymph-node assessments includes an increase of at least 25% in the mSWAT skin score and an increase of at least 50% in the summed products of the greatest diameters of involved lymph nodes for the specified patients.
Time-to-event endpoints can account for differing observation times, including participants whose event has not been observed by the end of follow-up. The registry record does not report the numerical median, its confidence interval, the analysis population, or sufficient details to establish how censoring and the endpoint time origin were handled.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record names median progression-free survival as the primary endpoint but does not report a numerical estimate or an analysis result. It therefore does not establish what median progression-free survival was in this study or how precisely it was estimated. A timeframe of 3 years identifies the stated endpoint timeframe; it is not itself an outcome value or a statement that every participant was followed for that duration.
The study is single-group, with no randomized or concurrent comparator. Any outcome observed among participants receiving the planned sequence would describe that group under the study’s conditions. It would not, by itself, show how outcomes would compare with a different treatment or with no treatment. Differences in patient characteristics, disease course, other care, and follow-up can affect outcomes in a study without a control group. The registry record does not report an adjusted comparison that addresses those issues.
The regimen is sequential: Doxil is followed by Targretin after response assessment. The record describes the intended schedule, but does not report the number who began or completed each stage, the distribution of treatment duration, or whether all participants received the sequence as planned. These omissions limit interpretation of outcomes in relation to actual exposure.
The registry includes response assessment criteria and safety counts, but these do not substitute for the missing primary endpoint estimate. Nor do they establish a causal treatment effect. The record lists 37 participants at risk for serious adverse events, 15 affected, and 12 deaths in the treatment group. Those figures describe registry safety reporting; they do not identify the causes of deaths or establish that the treatment caused an event.
Overall, the registry record communicates the study’s question, planned regimen, endpoint, and some safety reporting. It does not supply the primary outcome estimate, uncertainty around that estimate, or detailed analysis methods. Conclusions about the magnitude or precision of the progression-free survival result cannot be drawn from the information recorded.
9. Statistical Methods Explained
What does a median time-to-event endpoint represent?
General statistical explanation: A time-to-event endpoint records the time until a defined event occurs. The median is a location measure of the event-time distribution, rather than an arithmetic average. It is often useful when follow-up duration differs or when event times are not distributed symmetrically. For progression-free survival, the event definition and time origin are essential: they determine which outcomes count as events and when measurement begins.
In this trial, the named primary endpoint is median progression-free survival in months, with a timeframe of 3 years. The registry provides progression assessment criteria, including skin and lymph-node criteria, but does not report the numerical median or all analysis details. In general, time-to-event analyses also need rules for observations without a recorded event by the end of follow-up. Such observations are commonly treated as censored at an appropriate time, but the registry record does not report the censoring rules used for this study.
Why does uncertainty matter?
General statistical explanation: A confidence interval communicates the range of estimates compatible with the data under the analysis method and its assumptions. A narrow interval indicates greater precision than a wide interval, but neither interval alone establishes that a treatment caused an outcome. Interpretation depends on the study design, endpoint definition, follow-up, and analysis population as well as the estimate.
No confidence interval for the primary endpoint is reported in the registry record. Without an estimate and its uncertainty, readers cannot assess the precision of the study’s primary outcome from this record. A completed status does not fill that gap.
What does a single-group design allow?
General statistical explanation: A single-group study can describe observed outcomes and adverse events among participants enrolled under a particular study regimen. It lacks a concurrent treatment comparison, so it cannot directly estimate a between-group treatment contrast. Historical comparisons may be affected by differences in eligibility, disease severity, assessment practices, and follow-up. No historical comparator or comparative analysis is reported in the registry record.
The study lists allocation as not applicable and masking as none. These features mean the record does not describe random assignment or masking. They do not, on their own, establish that assessment was biased; rather, they identify design features relevant when considering how outcomes were measured and interpreted.
How should the sequential regimen be understood?
General statistical explanation: In a sequential regimen, later treatment exposure depends on reaching the later stage of the protocol. If analyses include only people who reached that stage, their outcomes may not represent everyone who entered the study. A clear report would describe how many participants entered each treatment stage, how response groups were defined, and which population was used for each analysis. Those details are not reported in the registry record. The treatment description states that response is assessed after Doxil and that some responders may continue Targretin until relapse; it does not provide observed stage-by-stage participation or outcomes.
What role do assessment criteria play?
General statistical explanation: Prespecified, reproducible criteria help make event classification more consistent. This record describes several forms of assessment: imaging for specified stage IV patients with positive pretreatment findings, blood or flow-cytometry measures for patients with Sézary syndrome, and skin assessment using mSWAT. It also gives progression thresholds for the specified skin and lymph-node measures. Using different assessments for relevant clinical presentations can make endpoint classification more tailored, but a complete interpretation also requires knowing how assessments were timed, reconciled, and applied when information was unavailable. Those operational details are not reported in the registry record.
What can a planning illustration say?
General statistical explanation: Sample-size illustrations based on an observed effect are hypothetical planning exercises. They show how a future study’s size might relate to a chosen effect assumption and statistical design. They are not observed power, evidence that the original study was adequately powered, or a replacement for its analysis results. The registry record does not report its sample-size justification or a statistical design rationale. It also does not report primary endpoint analyses from which to assess the precision of an observed time-to-event estimate.
10. Multiplicity, Missing Data and Analysis Populations
The registry record identifies median progression-free survival as the primary endpoint and Maximum Therapeutic Response as a secondary outcome. It lists no analysis records. It does not report a statistical analysis plan explaining how outcomes were tested, whether any interim analyses were conducted, or whether multiplicity was considered. Therefore, the record does not establish whether or how the chance of false-positive findings across multiple outcomes or analyses was addressed.
Details of missing-data handling are also not reported in the registry record. For a time-to-event endpoint, interpretation depends on how follow-up ends for participants without a recorded event, how censoring times are assigned, and whether loss to follow-up may be related to prognosis. The registry gives a 3-year timeframe but does not state how incomplete follow-up was handled or provide a follow-up summary.
The registry does not report the analysis population for the primary endpoint, such as whether it included all enrolled participants or a subset defined by treatment exposure or evaluability. It also does not report the numbers assessed at each treatment stage or the handling of participants who did not complete the planned sequence. Those details matter because the regimen includes response assessment before bexarotene and allows some responders to continue treatment until relapse.
The enrollment entry is 37, but the record does not provide a primary endpoint result or analysis population with which to interpret that enrollment in relation to the outcome. A sample-size justification and the basis for choosing enrollment are not reported in the registry record. These omissions leave uncertainty about outcome reporting, analysis decisions, missing observations, and the scope of any conclusions.
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 |
|---|---|---|---|
| Doxil and Targretin® (Bexarotene) | 15 | 37 | 40.5% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Doxil and Targretin® (Bexarotene) | 12 of 37 |
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 |
|---|---|
| Doxil and Targretin® (Bexarotene) | 37 of 37 |
Most frequent serious adverse event terms
| Event term | Organ system | Doxil and Targretin® (Bexarotene) |
|---|---|---|
| Infection, other | Infections and infestations | 5 of 37 |
| Rigors/chills | General disorders | 2 of 37 |
| Cardiac Arrhythmia, other | Cardiac disorders | 1 of 37 |
| Cough | Respiratory, thoracic and mediastinal disorders | 1 of 37 |
| Death not assoc w CTCAE term-Disease prog NOS | General disorders | 1 of 37 |
| Fever (in the absence of neutropenia) | General disorders | 1 of 37 |
| Fracture | Injury, poisoning and procedural complications | 1 of 37 |
| Gastritis (incl bile reflux gastritis) | Gastrointestinal disorders | 1 of 37 |
| Inf norm ANC/gr1/2 neut-Cellulitis(skin) | Infections and infestations | 1 of 37 |
| Inf unknown ANC-Blood | Infections and infestations | 1 of 37 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Doxil and Targretin® (Bexarotene) |
|---|---|---|
| Hyperglycemia | Metabolism and nutrition disorders | 11 of 37 |
| White blood cells | Investigations | 9 of 37 |
| Cholesterol, high | Investigations | 7 of 37 |
| Triglyceride, high | Metabolism and nutrition disorders | 7 of 37 |
| Fatigue | General disorders | 4 of 37 |
| Neutrophils | Investigations | 4 of 37 |
| Lymphopenia | Investigations | 3 of 37 |
| Pruritus/itching | Skin and subcutaneous tissue disorders | 3 of 37 |
| Pain - Back | Musculoskeletal and connective tissue disorders | 2 of 37 |
| Rash: hand-foot skin reaction | Skin and subcutaneous tissue disorders | 2 of 37 |
Serious events with the highest arm-level rate: infection, other (13.5%), rigors/chills (5.4%), cardiac arrhythmia, other (2.7%), cough (2.7%), death not assoc w ctcae term-disease prog nos (2.7%).
The registry reports one treatment group, with 37 participants at risk for serious adverse events and 15 affected. For other adverse events, it reports 37 at risk and 37 affected. It also lists 12 deaths. These are descriptive registry counts; the record does not establish that the treatment caused the events or deaths, and it does not report causes for the deaths in this safety summary.
The most frequently listed serious terms are Infection, other (13.5%) and Rigors/chills (5.4%). Cardiac Arrhythmia, other, Cough, Death not assoc w CTCAE term-Disease prog NOS, and Fever (in the absence of neutropenia) are each listed at 2.7%. These percentages are reported as recorded and should not be treated as comparative risks against another treatment, because the study has no comparator group.
Serious adverse event reporting is distinct from a complete account of every symptom, its severity, duration, or relationship to treatment. The registry record does not provide individual event narratives or a causal assessment for the listed terms. The safety figures should therefore be read as a summary of reported events in the study group, not as proof of attribution or as a comparison of safety between regimens.
12. What the Result Does, and Does Not, Mean
This record establishes that a phase II single-group study evaluated a planned sequence of liposomal doxorubicin followed by bexarotene in cutaneous T-cell lymphoma. It identifies median progression-free survival as the primary endpoint, describes the planned treatment schedule, and records selected safety counts and serious adverse event terms. Those details clarify what the study was designed to examine.
The record does not report the numerical primary endpoint result, its uncertainty, or a comparison with another treatment. It therefore does not permit a conclusion about the observed median progression-free survival or its precision. The absence of a concurrent control also means that any outcomes observed within the group would not alone show what would have happened under a different treatment strategy.
The presence of response criteria does not indicate how many participants responded, and the safety counts do not show whether events were caused by treatment. The record does not provide enough detail to assess treatment completion, follow-up completeness, the primary analysis population, missing-data handling, or the statistical methods used.
These limitations are about what the registry record reports, not proof that analyses or additional information do not exist elsewhere. Within this record, however, the study’s design and intended endpoint are clearer than its outcome findings. No clinical recommendation follows from these registry entries alone.
13. Why This Trial Matters Statistically
This study is statistically notable for the distinction between a treatment sequence and a comparative treatment trial. Its single-group design can describe outcomes among participants receiving the planned sequence, but cannot provide the direct randomized contrast that helps separate treatment effects from differences in patient characteristics, disease course, or other care. That distinction should shape interpretation before considering any endpoint estimate.
The primary endpoint is time-to-event, which requires attention to the event definition, time origin, follow-up, and censoring. The registry gives progression criteria for relevant assessments, including skin and lymph-node measures, but does not report a numerical median or detailed analysis rules. This illustrates why endpoint descriptions and endpoint results are separate parts of trial interpretation.
The sequential treatment plan also raises an analysis-population question: patients may reach the bexarotene stage only after the preceding treatment and response assessment. Without stage-specific participation and analysis details, it is not possible to tell how exposure and eligibility for later treatment relate to reported outcomes.
Finally, a completed study record does not necessarily contain a complete outcome analysis. Here, the record includes design and safety information but lists no analysis records and gives no primary endpoint estimate. Statistical interpretation must therefore distinguish what the protocol description says the study intended to measure from what the registry actually reports as a result.
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 is that the registry record does not report a numerical result for median progression-free survival, despite naming it as the primary endpoint and stating a timeframe of 3 years. No confidence interval or other measure of uncertainty is reported. This prevents assessment of the endpoint’s magnitude and precision.
The study is single-group, with allocation not applicable and masking none. Without a concurrent comparator, outcomes cannot directly establish a comparative treatment effect. The registry also does not report details of the primary analysis population, follow-up completeness, censoring, missing-data handling, or the statistical analysis plan.
The record describes a sequential regimen but does not report how many participants received each stage, completed treatment, or were assessed for response. It does not report response results or the numerical primary endpoint analysis. The listed progression criteria cover specified clinical assessments, while additional details about assessment timing and adjudication are not reported in the registry record.
Safety reporting includes counts and selected serious adverse event terms, but the record does not provide causal attribution for the listed events or causes for the deaths in the safety summary. These limits mean the safety information is descriptive and cannot support a controlled comparison. Sample-size justification, interim analyses, and any protocol changes are not reported in the registry record.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Liposomal Doxorubicin Followed By Bexarotene in Treating Patients With Cutaneous T-Cell Lymphoma — NCT00255801.
- Registry record last updated 2018-08-16; status last verified 2018-07.
- Results first posted 2018-08-16.
- 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
NCT00255801 is a completed phase II, single-group study sponsored by Memorial Sloan Kettering Cancer Center. It concerns cutaneous T-cell lymphoma and records an enrollment of 37. The planned sequence is intravenous Doxil every two weeks for 8 doses over 16 weeks, followed by oral Targretin for at least 16 weeks, with continued treatment until relapse permitted for patients achieving a complete or partial response.
The primary endpoint is median progression-free survival in months, with a stated timeframe of 3 years. The registry describes response and progression assessments but does not report a numerical primary endpoint result or analysis records. Safety reporting lists 15 participants affected by serious adverse events among 37 at risk, 37 affected by other adverse events among 37 at risk, and 12 deaths. The single-group design and missing endpoint analysis limit comparative and outcome interpretation.