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 A Study for Participants With Relapsed Cutaneous T-Cell Lymphoma (NCT00744991), a na phase 2 study. The primary outcome is Percentage of Participants With Confirmed Complete Response (CR) or Partial Response (PR) Among Mycosis Fungoides (MF) and Sezary Syndrome (SS) Participants (Response Rate).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00744991 |
| Phase | Phase 2 |
| Condition | Cutaneous T-Cell Lymphoma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 25 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Eli Lilly and Company |
| Results posted | Yes |
2. Clinical Question
Population
The study concerned participants with cutaneous T-cell lymphoma (CTCL) who had failed prior therapies. The primary endpoint population is described as all participants who received at least 1 dose of study drug. The endpoint description distinguishes mycosis fungoides (MF) from Sezary syndrome (SS), using different disease measures to classify response. The number of treated participants included in the endpoint analysis is not reported in the registry record.
Intervention
The intervention was single-agent enzastaurin. The study was open label, so treatment masking was not used. The registry record does not report a dose or administration schedule. It describes a phase 2 study and gives the purpose as assessing efficacy and safety in participants with CTCL who had failed prior therapies.
Comparator
This was a single-group study, with no comparator group identified. Allocation is recorded as not applicable. The study therefore does not provide a randomized, concurrent control comparison of enzastaurin with another treatment or with no study treatment. Comparisons with other studies or with outcomes expected under different care are not reported in the registry record.
Primary question
The primary endpoint was the percentage of participants with confirmed complete response (CR) or partial response (PR), assessed using modified Severity-Weighted Assessment Tool (mSWAT) criteria for MF and mSWAT plus Sezary cell count for SS. The response assessment period ran from baseline until measured progressive disease, up to 9 cycles of 28 days each. The observed response percentage and its 95% confidence interval are not reported in the registry record.
3. Trial Design
This phase 2 study evaluated single-agent enzastaurin in participants with relapsed cutaneous T-cell lymphoma (CTCL). The registry record describes the study as open label and multicenter. It identifies a single-group model, with allocation recorded as not applicable and masking recorded as none. There was no concurrent control arm, so the design does not support a randomized comparison between treatment groups.
The study's stated purpose was to determine the efficacy and safety of enzastaurin in participants with CTCL who had failed prior therapies. The condition listed is CTCL. Enrollment is recorded as 25. That enrollment figure should not be assumed to be the number included in the primary endpoint analysis: the endpoint population is defined as participants who received at least 1 dose, and the number who met that definition is not reported in the registry record.
The primary response assessment was planned from baseline until measured progressive disease, with a limit of up to 9 cycles, each lasting 28 days. The registry record gives a start date of 2008-09, a primary completion date of 2010-01, and a completed status. It does not report the treatment dose, treatment duration for individual participants, or the timing of any specific assessments beyond the primary endpoint timeframe.
Secondary outcomes listed include duration of response, time to progression, time to objective response, health-state utility, pruritus measures, change in itch-related quality-of-life scores, and adverse events or deaths. Results for these secondary outcomes are not reported in the registry record. The record also does not describe a comparator, treatment allocation process, or masking procedure beyond identifying the study as single group, allocation not applicable, and unmasked.
4. Participant Flow and Accounting
The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.
Before assignment: This study had 2 stages. Stage 2 would occur if the minimum level of efficacy (confirmed complete or partial anti-tumor response) was observed in Stage 1. The study was considered complete at the end of Stage 1.
Overall Study
| Stage | Enzastaurin |
|---|---|
| STARTED | 25 |
| Received at Least 1 Dose | 25 |
| COMPLETED | 0 |
| NOT COMPLETED | 25 |
Reasons for not completing this period:
| Reason | Enzastaurin |
|---|---|
| Progressive Disease | 11 |
| Withdrawal by Subject | 8 |
| Physician Decision | 3 |
| Adverse Event | 2 |
| Protocol Entry Criteria Unmet | 1 |
Analysis population for the primary outcome, as defined in the registry: All participants who received at least 1 dose of study drug.
5. Baseline Characteristics
The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.
Population: Participants who received at least 1 dose of study drug.
| Characteristic | Unit | Enzastaurin |
|---|---|---|
| Participants analysed | 25 | |
| Age, Continuous | years | 64 (26 to 83) |
| Sex: Female, Male: Female | Participants | 13 |
| Sex: Female, Male: Male | Participants | 12 |
| Race/Ethnicity, Customized: Caucasian | Participants | 18 |
| Race/Ethnicity, Customized: African | Participants | 4 |
| Race/Ethnicity, Customized: Hispanic | Participants | 3 |
| Region of Enrollment: United States | Participants | 25 |
| Number of Participants with Cutaneous T-Cell Lymphoma (CTCL) Variant: Mycosis Fungoides (MF) | Participants | 19 |
| Number of Participants with Cutaneous T-Cell Lymphoma (CTCL) Variant: Sezary Syndrome (SS) | Participants | 6 |
6. Primary Endpoint
The registry lists the primary outcome as Percentage of Participants With Confirmed Complete Response (CR) or Partial Response (PR) Among Mycosis Fungoides (MF) and Sezary Syndrome (SS) Participants (Response Rate), measured over Baseline to measured Progressive Disease (PD) [up to 9 cycles (28-day cycles)].
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | 95% Confidence Interval |
| Unit | percentage of participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the percentage of participants with a confirmed complete response (CR) or partial response (PR). It is a binary response outcome at the participant level: each participant is classified according to whether a confirmed CR or PR occurred during the stated assessment period, and the percentage is calculated among participants who received at least 1 dose of study drug. The registry record identifies a 95% confidence interval as the measure of uncertainty, but does not report the observed percentage or interval bounds.
Response was assessed differently according to disease subtype. For mycosis fungoides (MF), the modified Severity-Weighted Assessment Tool (mSWAT) uses a weighted score of skin involvement, ranging from 0 for unaffected to 400 for severely affected. A CR requires no detectable malignant disease for at least 4 weeks; a PR requires an mSWAT score reduction of at least 50% from baseline. For Sezary syndrome (SS), mSWAT and Sezary cell count are used. A CR requires Sezary cells below 5%; a PR requires a reduction in Sezary cells of at least 50% from baseline.
Response rate is used to describe how often participants meet a prespecified response definition during the assessment period. The confirmed-response requirement and explicit criteria make the endpoint more specific than an unconfirmed change in disease measures. However, a response percentage alone would not describe how long a response lasts, how symptoms change, or what adverse events occur. Those topics appear among the secondary outcomes, but their results are not reported in the registry record. The endpoint's observed value and its precision are also not reported, so the magnitude of response cannot be interpreted from this record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies a response-rate endpoint but does not report the observed response rate, the response count, or the bounds of the 95% confidence interval. As a result, the primary efficacy result cannot be described numerically from the registry record. Enrollment is recorded as 25, but the primary analysis population is participants who received at least 1 dose; the number treated and the number with an evaluable response are not reported. Enrollment alone does not establish the denominator used for the endpoint.
In general, a confidence interval expresses uncertainty around an estimated quantity. For a response percentage, a 95% confidence interval would describe the range of values compatible with the data under the method used to calculate it. It does not mean that a particular participant has a 95% chance of responding, and it does not resolve design limitations such as the absence of a control group. Here, the endpoint's confidence-interval method and numerical bounds are not reported in the registry record, so neither the estimate nor its precision can be assessed.
The single-group design is important to interpretation. Any observed responses would describe outcomes among participants treated in this study, without a randomized comparison that could separate treatment-associated outcomes from other influences. The registry record does not provide a control group, and it does not report analyses comparing enzastaurin with an alternative. Therefore, a response rate, if available elsewhere, would be a within-study description rather than a controlled estimate of a difference between treatments.
The endpoint combines response criteria tailored to MF and SS. This allows the assessment to reflect disease-specific measures, but the record does not report how many participants had each subtype or provide subtype-specific response results. It also does not report the response numerator, the treated denominator, the number with missing or unevaluable assessments, or the handling of those participants. These omissions prevent assessment of whether the reported endpoint population matches enrollment and whether missing assessments could affect the estimate.
The safety results that are reported are counts for participants with serious and other adverse events, along with selected serious-event terms and percentages. These are descriptive safety data. They do not establish a comparison with background event rates or another treatment, and the record does not provide exposure-adjusted rates or a participant-level account of overlap between event categories. Death counts are not reported in the registry record.
Overall, the available record describes the study design, response definition, and selected safety findings, but it does not contain the numerical primary response result or results for the listed secondary outcomes. Statistical interpretation should therefore remain limited to what is explicitly reported: the endpoint's definition and assessment period, the single-group design, the stated enrollment, and the available safety counts. The record does not support a numerical conclusion about response frequency or comparative efficacy.
9. Statistical Methods Explained
The central statistical outcome is a binary response measure: whether a participant had a confirmed complete response (CR) or partial response (PR) during the endpoint assessment period. The registry record defines the response rate as the number of participants with CR or PR from the start of study treatment until progressive disease or recurrence, divided by the number of participants treated, then multiplied by 100. The primary endpoint population is described as all participants who received at least 1 dose of study drug. The actual response count, treated denominator, and calculated percentage are not reported in the registry record.
What does a confidence interval add?
A percentage is an estimate based on the participants observed in a study. A confidence interval communicates uncertainty around that estimate. In general, a narrower interval indicates greater precision, while a wider interval indicates less precision. The width depends in part on the amount of information in the data and on the method used to calculate the interval. The registry record specifies a 95% confidence interval for the primary endpoint, but its numerical limits and calculation method are not reported. Without the estimate and interval, readers cannot judge either the response frequency or its precision.
A confidence interval should not be read as a guarantee about an individual participant's outcome. Nor does it address whether an observed response was caused by the treatment. The interval pertains to uncertainty in an estimated response rate under statistical assumptions; the single-group design remains a separate limitation for causal interpretation. No numerical interval or inferential comparison is reported in the registry record.
How is response defined?
The endpoint uses disease-specific response assessments. For MF, the mSWAT is a weighted measure of the percentage of total body surface area attributed to skin lesions, transformed into a score from 0, unaffected, to 400, severely affected. A CR is defined as no detectable malignant disease for at least 4 weeks, and a PR as an mSWAT reduction of at least 50% from baseline. For SS, assessment uses mSWAT and Sezary cell count measured by flow cytometry. A CR requires Sezary cells below 5%, and a PR requires a Sezary cell reduction of at least 50% from baseline.
These definitions make the endpoint depend on both the participant's disease subtype and the specified measurement criteria. Confirmation is part of the endpoint, so an isolated qualifying measurement is not necessarily sufficient. The registry record does not report the number of participants in each subtype, separate MF and SS results, or details about the timing and confirmation of individual assessments.
What does the single-group model imply?
In a single-group study, the primary response percentage describes outcomes among the treated participants who meet the analysis-population definition. There is no randomized comparator in this study. Consequently, there is no between-group treatment contrast, and the result cannot by itself show how outcomes would have differed under another treatment or no study treatment. General statistical interpretation distinguishes estimation of an outcome within a group from estimation of a comparative effect between groups.
The allocation field is recorded as not applicable, and masking is recorded as none. The study is described as open label. These design features should be considered when interpreting measured outcomes, especially outcomes involving clinical assessment or participant reporting. The registry record does not describe procedures for blinded endpoint review or other steps that might address assessment concerns.
What can be inferred from the safety figures?
The safety summary reports participants affected by serious adverse events and other adverse events, alongside selected serious-event terms. These are descriptive counts and percentages, not a randomized comparison. In general, adverse-event categories may overlap: one participant can experience more than one event, and participants with serious events may also be represented in other-event reporting. Therefore, event-category counts should not be added to infer a unique participant total. The registry record does not give exposure-adjusted rates, event onset times, or a detailed account of how events were collected.
The record gives serious-event term percentages for dehydration, pain, bacteraemia, cellulitis, genital herpes, and pneumonia. These listed values describe the reported terms; they do not establish that the terms are exhaustive or mutually exclusive. Death counts are not reported in the registry record, even though deaths are named among the secondary outcomes.
What planning illustrations can and cannot say
Planning illustrations use the stated assumptions of a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. The effect to detect is defined as a stated fraction of the observed effect. For time-to-event outcomes, the illustration concerns the total number of events using the Schoenfeld method; for other outcomes, it concerns participants per arm. These are planning quantities, not observed trial results or observed power.
The study itself is single group, so a two-arm planning setup is not a description of its treatment allocation. Planning illustrations based on an observed effect also depend on that effect being available and meaningful for the hypothetical comparison. The registry record does not report the primary observed response effect, and it does not state that these planning assumptions were used to design the trial. The sample-size justification and the trial's own statistical plan are not reported in the registry record.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists multiple secondary outcomes alongside the primary response endpoint. These include duration of response, time to progression, time to objective response, health-state utility, pruritus experience, itch-related quality-of-life scores, and adverse events or deaths. When several outcomes are examined, the chance of at least one apparently notable result can increase if each is tested separately. Statistical approaches can address multiplicity, for example by prespecifying a hierarchy or adjusting inference, but the registry record does not report a multiplicity strategy or a detailed statistical analysis plan.
The record indicates 8 outcome measures and 0 analyses. This does not establish what analyses were performed outside the registry entry; it means analyses are not reported in the record. Numerical results for the primary endpoint and the listed secondary outcomes are not reported. No p-values or test results are available for interpretation.
The primary population is defined as participants who received at least 1 dose of study drug. The number meeting that definition, the endpoint denominator, the response count, and the treatment of unevaluable participants are not reported in the registry record. The record also does not describe how missing response assessments were handled, whether imputation was used, or whether sensitivity analyses were conducted. These details matter because the response rate depends on who is included in its denominator and how participants without a classifiable response are treated.
Duration of response and other time-related outcomes can require rules for censoring participants whose event is not observed during follow-up. The registry record does not report censoring rules, follow-up completeness, or analysis methods for these outcomes. It also does not report interim analyses, subgroup-analysis methods, or whether outcome definitions changed. The absence of these details in the registry record should not be read as evidence that a particular method was or was not used.
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 |
|---|---|---|---|
| Enzastaurin | 5 | 25 | 20.0% |
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 |
|---|---|
| Enzastaurin | 19 of 25 |
Most frequent serious adverse event terms
| Event term | Organ system | Enzastaurin |
|---|---|---|
| Dehydration | Metabolism and nutrition disorders | 2 of 25 |
| Pain | General disorders | 1 of 25 |
| Bacteraemia | Infections and infestations | 1 of 25 |
| Cellulitis | Infections and infestations | 1 of 25 |
| Genital herpes | Infections and infestations | 1 of 25 |
| Pneumonia | Infections and infestations | 1 of 25 |
| Sepsis | Infections and infestations | 1 of 25 |
| International normalised ratio increased | Investigations | 1 of 25 |
| Hyperkalaemia | Metabolism and nutrition disorders | 1 of 25 |
| Hypoglycaemia | Metabolism and nutrition disorders | 1 of 25 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Enzastaurin |
|---|---|---|
| Diarrhoea | Gastrointestinal disorders | 6 of 25 |
| Fatigue | General disorders | 6 of 25 |
| Oedema peripheral | General disorders | 5 of 25 |
| Nausea | Gastrointestinal disorders | 3 of 25 |
| Constipation | Gastrointestinal disorders | 2 of 25 |
| Chills | General disorders | 2 of 25 |
| Staphylococcal infection | Infections and infestations | 2 of 25 |
| Urinary tract infection | Infections and infestations | 2 of 25 |
| Decreased appetite | Metabolism and nutrition disorders | 2 of 25 |
| Hyperkalaemia | Metabolism and nutrition disorders | 2 of 25 |
Serious events with the highest arm-level rate: dehydration (8.0%), pain (4.0%), bacteraemia (4.0%), cellulitis (4.0%), genital herpes (4.0%).
The safety summary reports 5 participants with serious adverse events among 25 at risk in the enzastaurin group. It also reports 19 participants with other adverse events among 25 at risk. These are separate reported categories; the counts should not be added to infer a total number of affected participants, because the record does not state whether participants overlap across categories.
The selected serious adverse event terms and their reported percentages are dehydration, 8.0%; pain, 4.0%; bacteraemia, 4.0%; cellulitis, 4.0%; genital herpes, 4.0%; and pneumonia, 4.0%. These are term-level figures, not a complete description of all safety events. The registry record does not report the total number of events for each term, event timing, duration, severity beyond the serious-event classification, or treatment relatedness. It also does not report deaths in the safety summary.
These figures describe events recorded in a single-group study. They do not provide a concurrent control comparison, and they cannot establish how event frequency compares with another treatment. The registry record does not report exposure-adjusted rates or the duration of individual participants' treatment and observation. For these reasons, the counts are best read as descriptive safety information, not as comparative estimates.
12. What the Result Does, and Does Not, Mean
The study's primary question concerned the frequency of confirmed complete or partial response among participants with relapsed cutaneous T-cell lymphoma who received enzastaurin. The registry record sets out the response criteria and identifies a 95% confidence interval, but it does not report the response percentage, its numerator or denominator, or the interval bounds. The primary result therefore cannot be quantified from the registry record.
Even if a response estimate were available, this design would describe outcomes in a single treated group. Because there was no randomized comparator, the study cannot provide a controlled estimate of how outcomes differ from those under another treatment. A within-group response percentage is not, by itself, a measure of the treatment's comparative effect. The record does not report an analysis that addresses that comparison.
The response endpoint is also distinct from other clinically relevant outcomes. It does not by itself measure duration of response, time to progression, symptom experience, quality of life, or safety. The registry record lists these as secondary outcomes but does not report their results. Consequently, the available information does not permit a complete description of participants' disease course, symptom changes, or experience of adverse events over time.
The safety figures provide counts of participants with serious and other adverse events and selected serious-event term percentages. They are useful as descriptive observations from the study group, but they do not establish relative safety versus a control group. Death counts are not reported in the registry record. The reported safety information should not be treated as a complete accounting of event burden, since event overlap, exposure duration, and detailed event-level information are not specified.
The registry record supports a clear description of the study's design and endpoint definitions, along with selected safety counts. It does not support a numerical conclusion about how often response occurred, how precise that estimate was, or how outcomes compare with another treatment. Those distinctions keep the interpretation within the limits of the available record.
13. Why This Trial Matters Statistically
This trial is statistically informative as an example of the difference between defining an outcome and reporting its result. The registry record gives a detailed primary response definition, identifies the analysis population as participants who received at least 1 dose, and specifies a 95% confidence interval. Yet the observed response percentage and interval are not reported. A complete statistical interpretation needs both the outcome definition and the numerical estimate with its uncertainty.
The study also illustrates how a single-group design shapes the question that can be answered. A response rate in treated participants is an estimate of outcome frequency in that group. Without a randomized comparator, it is not a treatment contrast. This distinction matters even when the endpoint is clinically recognizable and its measurement criteria are clearly described.
Another statistical feature is the use of separate response criteria for MF and SS. Disease-specific assessment can make an endpoint more appropriate to different clinical presentations, while also making it important to report the composition of the study population and results by subtype. The registry record does not report subtype counts or separate subtype response estimates, so readers cannot examine whether the combined endpoint conceals different patterns.
The safety summary provides another teaching point: participant counts across adverse-event categories should not be added unless overlap is known. Serious events and other events may involve some of the same participants. Term-specific percentages likewise describe listed safety events but do not replace a full account of event timing, exposure, and follow-up.
Finally, planning illustrations and observed trial results answer different questions. The stated planning assumptions describe hypothetical two-arm calculations, whereas the actual study is single group. Planning quantities are not evidence of achieved precision or observed power. Keeping design, endpoint estimation, safety description, and hypothetical planning separate helps prevent overinterpretation.
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 main limitation for interpreting efficacy is that the registry record does not report the primary response rate, response count, treated denominator, or 95% confidence interval. Although enrollment is recorded as 25, the endpoint population is defined by having received at least 1 dose, and the number in that population is not reported. The record therefore does not show how many participants contributed to the primary result.
The single-group, open-label design has no randomized comparator. It cannot provide a direct estimate of the difference between enzastaurin and another treatment. The record does not describe a blinded response review or report comparative analyses. These design features limit what can be concluded about comparative efficacy, independently of the missing primary numerical result.
The endpoint combines disease-specific criteria for MF and SS, but the record does not report the number of participants with each subtype or separate response findings. It also does not report how missing or unevaluable assessments were handled, the endpoint calculation method beyond its general definition, or sensitivity analyses. The confidence-interval method is not reported.
Results for the listed secondary outcomes are not reported in the registry record. Specific limitations include the absence of results for response duration, progression, time to response, participant-reported health and itch measures, and adverse events or deaths beyond the selected safety figures. Follow-up completeness, event exposure time, and detailed safety collection methods are not reported. The record also does not report the statistical analysis plan, sample-size justification, multiplicity strategy, interim analyses, or subgroup-analysis methods.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: A Study for Participants With Relapsed Cutaneous T-Cell Lymphoma — NCT00744991.
- Registry record last updated 2020-10-19; status last verified 2020-09.
- Results first posted 2020-10-19.
- 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
NCT00744991 is a completed phase 2, open-label, multicenter, single-group study of single-agent enzastaurin in participants with relapsed cutaneous T-cell lymphoma who had failed prior therapies. Enrollment is recorded as 25. The study start is listed as 2008-09 and primary completion as 2010-01. The sponsor is Eli Lilly and Company.
The primary endpoint was the percentage of participants with confirmed CR or PR, using mSWAT criteria for MF and mSWAT plus Sezary cell count for SS. Its stated assessment period was baseline until measured progressive disease, up to 9 cycles of 28 days each. The observed response result and 95% confidence interval are not reported in the registry record. Selected safety counts and serious-event term percentages are reported, but death counts and results for the listed secondary outcomes are not reported.