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 Brentuximab Vedotin (SGN-35) in Patients With Mycosis Fungoides With Variable CD30 Expression Level (NCT01396070), a na phase 2 study. The primary outcome is Overall Response Rate (ORR).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01396070 |
| Phase | Phase 2 |
| Condition | Non-Hodgkin Lymphoma (NHL), Cutaneous Lymphoma, Cutaneous T-cell Lymphoma (CTCL) |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 36 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Youn Kim |
| Results posted | Yes |
2. Clinical Question
Population
The study population was patients with mycosis fungoides or Sézary syndrome, conditions listed within the broader category of cutaneous T-cell lymphoma. The title specifies variable CD30 expression. The registry record gives an enrollment of 36, but does not provide further eligibility details or a breakdown by diagnosis or CD30 expression.
Intervention
Participants received brentuximab vedotin, also identified as SGN-35. The arm description specifies an intravenous dose of 1.8 mg/kg every 3 weeks. The record does not describe treatment duration or dose modifications.
Comparator
There was no concurrent control group. The study is recorded as single-group, with allocation listed as not applicable and masking listed as none. Its response findings therefore are not a randomized comparison against another treatment or a control condition.
Primary question
The primary endpoint was overall response rate, defined in the record by combining complete responses and partial responses. The timeframe is listed as 2 years. The registry record notes that 3 subjects were not evaluable, but does not report the number with complete or partial response or the resulting overall response rate.
In practical terms, the study asked whether responses meeting either of those categories were observed among the enrolled patients receiving the study drug. Combining complete and partial responses gives one summary of tumor response; it does not describe how long a response lasted or whether response differed by CD30 expression. Those questions require outcome details that are not reported in the registry record. Because the study had no comparator, it cannot by itself establish how response would compare with another treatment or with no study drug.
3. Trial Design
The registry record classifies this as a phase 2 exploratory pilot study with a single-group model. It lists an enrollment of 36 and a completed status. The recorded start is 2011-05 and primary completion is 2015-04. Those dates describe the registry record's study milestones; they do not provide the timing of individual participants' assessments or follow-up.
All participants were assigned to the brentuximab vedotin group described in the record. The treatment was specified as 1.8 mg/kg intravenously every 3 weeks. Allocation is listed as not applicable, and masking as none. Thus, the design does not include random allocation or a masked comparison. A single-group design can describe outcomes observed among people receiving the intervention, but cannot separate the intervention's contribution from other influences on those outcomes as a randomized comparison can.
The primary outcome was overall response rate, with a 2-year timeframe. The record describes the endpoint as the percentage calculated by combining complete response and partial response, and notes 3 subjects were not evaluable. It lists overall stable disease rate, overall partial response rate, and overall non-evaluable response among secondary outcomes. Detailed assessment criteria, assessment schedule, and definitions used to classify response are not reported in the registry record.
The record does not describe a control arm, a treatment allocation procedure, or an analysis plan. It also does not give detail on the number of participants in each diagnosis or CD30-expression category. The title indicates that CD30 expression varied, but the registry record does not report subgroup outcome results. These omissions limit how specifically the study's design and its response findings can be interpreted.
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 | Brentuximab Vedotin |
|---|---|
| STARTED | 36 |
| COMPLETED | 17 |
| NOT COMPLETED | 19 |
Reasons for not completing this period:
| Reason | Brentuximab Vedotin |
|---|---|
| Withdrawal by Subject | 2 |
| Death | 2 |
| Adverse Event | 8 |
| Physician Decision | 3 |
| Lack of Efficacy | 4 |
Analysis population for the primary outcome, as defined in the registry: The percentage was calculated by adding Complete response (CR) + Partial Response (PR) = Overall rate. 3 subjects were not evaluable.
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 | Brentuximab Vedotin |
|---|---|---|
| Participants analysed | 36 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 18 |
| Age, Categorical: >=65 years | Participants | 18 |
| Sex: Female, Male: Female | Participants | 11 |
| Sex: Female, Male: Male | Participants | 25 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 2 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 33 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 1 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 7 |
| Race (NIH/OMB): White | Participants | 25 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 4 |
| CD30 grouping at screening: < 10% CD30 positivity | participants | 17 |
| CD30 grouping at screening: 10% to 50% CD30 positivity | participants | 15 |
| CD30 grouping at screening: > 50% CD30 positivity | participants | 4 |
| Clinical Stage: Stage IB | Participants | 6 |
| Clinical Stage: Stage IIB | Participants | 16 |
| Clinical Stage: Stage IV/SS (includes IVA, IVA/SS and III) | Participants | 14 |
6. Primary Endpoint
The registry lists the primary outcome as Overall Response Rate (ORR), measured over 2 years.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | percentage |
| Endpoint classification | not reduced to a single comparison |
Overall response rate (ORR) is the primary endpoint. The registry record defines it as the percentage formed by combining complete response (CR) and partial response (PR). In general, a response endpoint is used to condense observed tumor response into a clinically interpretable summary. Combining complete and partial responses counts both categories as responses while retaining neither category's individual frequency in the combined measure.
The record lists the endpoint timeframe as 2 years and says that 3 subjects were not evaluable. It does not report the observed ORR, the separate counts of complete and partial responses, or the denominator used for the percentage. It also does not give the response criteria or the assessment schedule. Consequently, the precise operational meaning of the timeframe and the handling of non-evaluable participants cannot be reconstructed from the registry record.
ORR is a measure of response status, not response duration. It does not by itself tell readers how long a response lasted, how symptoms changed, or how outcomes compare with another treatment. In this single-group study, it is a descriptive endpoint for the study population rather than a randomized estimate of a treatment difference. The endpoint's name and definition are available, but the primary result needed to quantify response is not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies the outcome the study intended to summarize: overall response rate, combining complete and partial response. It also gives a timeframe of 2 years and states that 3 subjects were not evaluable. However, it does not report the observed response percentage, the underlying complete- and partial-response counts, or a confidence interval. The primary result therefore cannot be quantified from this record. No estimate of the precision of ORR can be made from the information reported.
This distinction matters: knowing the endpoint definition is not the same as knowing its result. The record establishes what response categories were to contribute to ORR, but it does not show how many participants met either category. The secondary outcome names include stable disease, partial response, and non-evaluable response, but numerical results for those outcomes are not reported in the registry record either. A reader should not infer an outcome from the study's title, status, or stated purpose.
The design also constrains interpretation. This was a single-group study, with no concurrent control and no masking. Its observed outcomes, if reported, would describe participants in the study; they would not constitute a randomized estimate comparing brentuximab vedotin with another treatment. General statistical principle: without a concurrent comparison, differences between observed outcomes and an external expectation can reflect factors other than the intervention. The registry record does not report an external benchmark or an analysis designed to address that issue.
The record's enrollment is 36, but enrollment alone does not show how many participants contributed to the primary endpoint. The note that 3 subjects were not evaluable signals that not every enrolled subject was classified for this endpoint, while the analysis population and missing-outcome rules are not reported in the registry record. Neither the percentage of evaluable subjects nor the response rate among them can be derived without the omitted outcome details.
The record contains serious and other adverse-event figures, but these are safety summaries, not evidence about the primary response endpoint. Their interpretation is also limited by missing details such as event counts by term and the observation period. Overall, the registry record supports a description of the study's question, design, and some safety reporting, but not a numerical conclusion about ORR or a comparative treatment effect.
9. Statistical Methods Explained
The methods information in the registry record is limited. It identifies an endpoint expressed as a percentage, reports the endpoint timeframe, and gives a note about non-evaluable subjects. It does not report an inferential analysis, confidence interval, statistical model, or detailed analysis plan. The explanations below distinguish general statistical ideas from methods specifically documented for this study.
What does the response percentage represent?
As a general concept, a response percentage expresses the share of a defined analysis population whose outcomes meet the response definition. Here the record defines the response category by combining complete response and partial response. To interpret such a percentage fully, readers need both the response count and the denominator, along with the rules used to classify outcomes. The record does not report the response counts or denominator for the primary endpoint, so the ORR cannot be checked or interpreted numerically from the registry record.
The record says that 3 subjects were not evaluable. General statistical principle: an outcome that cannot be classified is not automatically equivalent to no response. Its treatment in an analysis depends on prespecified rules, such as whether non-evaluable participants are excluded from a particular calculation or handled in another way. The registry record does not report the rule used. It would therefore be inappropriate to assume either a particular denominator or a particular result for those subjects.
What would a confidence interval add?
In general, a confidence interval describes the uncertainty around an estimated quantity under the assumptions of the statistical method used. For a response percentage, it would help readers assess how precisely the study estimates response in a population like the one enrolled. An interval does not remove design limitations, and it does not establish a causal comparison in a single-group study. No confidence interval for ORR is reported in the registry record, so its precision cannot be assessed from the posted information.
What does the single-group design allow?
General statistical principle: a single-group study can describe outcomes observed among participants who received the intervention. A concurrent control group helps distinguish outcomes associated with the intervention from outcomes that might also occur because of disease course, participant characteristics, other care, or measurement conditions. This record has no control arm and lists no allocation method. Accordingly, any response result from this design would be descriptive, not a randomized treatment contrast. The registry record does not report an adjusted comparison or another method for estimating such a contrast.
Masking is listed as none. In general, knowledge of treatment assignment can affect some aspects of care or assessment, depending on the outcome and procedures. The registry record does not describe safeguards used for response assessment, so their presence or absence cannot be determined. This is a design consideration, not a basis for assuming that response classifications were biased.
What do the analysis fields tell readers?
The record lists zero analyses. It does not provide a statistical plan, model specification, hypothesis-testing procedure, or interim-analysis information. The absence of reported analyses should not be filled in by assuming that a particular method was used. The record also does not state whether the primary outcome was analyzed as a proportion with a confidence interval, whether any subgroup analyses were planned, or how repeated assessments were handled.
The record does identify several secondary outcomes, but their names alone do not supply their definitions, estimates, or uncertainty. For example, the existence of a stable disease outcome does not establish how stable disease was defined or how many participants met that definition. Those details are not reported in the registry record.
How should planning illustrations be understood?
General statistical principle: a sample-size calculation is a planning exercise that uses assumptions about a future study, such as a target effect, significance level, allocation, and analysis method. It is not a measure of what the completed study achieved. The stated planning assumptions for the illustrations use a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; the effect to detect is described as a stated fraction of the observed effect. This hypothetical two-arm setup is distinct from the recorded single-group design.
Such planning illustrations are not observed power. Observed power is not a substitute for interpreting an estimate and its uncertainty; a planning calculation instead asks what design might be needed under specified assumptions. The record does not report the trial's own sample-size justification, and no planning values can be inferred from the assumptions alone. For time-to-event planning, the stated convention is a total number of events; for other planning illustrations, the convention is participants per arm. Those planning conventions do not change the actual design or supply the missing ORR result.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists one primary outcome and several secondary outcomes, but does not report how multiple outcomes were handled statistically. General statistical principle: testing or emphasizing several outcomes can increase the chance of a misleading positive finding if multiplicity is not considered. A prespecified hierarchy, adjustment, or other strategy can address that issue, but none is reported in the registry record. It is not possible to determine whether an approach was planned or applied.
The primary outcome description identifies 3 subjects as not evaluable. The record does not say why they were not evaluable, how their information was handled, or which participants were included in the primary analysis population. It also does not report whether other outcome data were missing. General statistical principle: missing outcomes can affect both the interpretation and precision of an estimate, particularly when the reasons for missingness relate to the outcome. No missing-data method or sensitivity analysis is reported in the registry record.
The record reports enrollment of 36, but does not state a separate analysis population for ORR. Enrollment should therefore not be treated as the endpoint denominator. Without the response counts and analysis rules, the primary percentage cannot be reproduced or evaluated for sensitivity to the non-evaluable cases.
No interim analyses, stopping rules, or statistical analysis plan are reported in the registry record. Their absence from the record does not establish that they were not considered; it means their status cannot be determined from the information reported. The record also does not provide subgroup analysis methods for variation in CD30 expression or for the listed diagnoses. Any interpretation of those potential subgroups would require details 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 |
|---|---|---|---|
| All Participants | 23 | 36 | 63.9% |
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 |
|---|---|
| All Participants | 32 of 36 |
Most frequent serious adverse event terms
| Event term | Organ system | All Participants |
|---|---|---|
| Diarrhea | Gastrointestinal disorders | 3 of 36 |
| Sepsis | Infections and infestations | 2 of 36 |
| Blood disorder | Blood and lymphatic system disorders | 2 of 36 |
| Skin Infection | Infections and infestations | 2 of 36 |
| Febrile Neutropenia | Blood and lymphatic system disorders | 2 of 36 |
| Confusion | Psychiatric disorders | 1 of 36 |
| Hypercalcemia | Blood and lymphatic system disorders | 1 of 36 |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | 1 of 36 |
| Thrombocytopenia | Blood and lymphatic system disorders | 1 of 36 |
| Pneumonia | Reproductive system and breast disorders | 1 of 36 |
Most frequent non-serious adverse event terms
| Event term | Organ system | All Participants |
|---|---|---|
| Skin eruption | Skin and subcutaneous tissue disorders | 4 of 36 |
| Dyspepsia | Gastrointestinal disorders | 3 of 36 |
| Weight Loss | Social circumstances | 3 of 36 |
| Peripheral Neuropathy | Nervous system disorders | 2 of 36 |
| Aberrant T-cell population (blood) | Blood and lymphatic system disorders | 2 of 36 |
| Dysphonia | Blood and lymphatic system disorders | 2 of 36 |
| Cough | Respiratory, thoracic and mediastinal disorders | 2 of 36 |
| Hyponatremia | Blood and lymphatic system disorders | 2 of 36 |
| Infusion Reaction | General disorders | 2 of 36 |
| Leukopenia | Investigations | 2 of 36 |
Serious events with the highest arm-level rate: diarrhea (8.3%), sepsis (5.6%), blood disorder (5.6%), skin infection (5.6%), febrile neutropenia (5.6%).
The registry record reports an all-participants safety group with 36 at risk for serious adverse events and 23 affected. For other adverse events, it reports 36 at risk and 32 affected. These are separate summaries; they should not be combined or interpreted as mutually exclusive groups. The record does not state the event definitions, exposure time, or whether the affected figures refer to participants with at least one event under specified collection rules.
The listed serious adverse-event terms and percentages are diarrhea, 8.3%; sepsis, 5.6%; blood disorder, 5.6%; skin infection, 5.6%; febrile neutropenia, 5.6%; and confusion, 2.8%. The registry record does not provide event counts for these terms, clarify whether participants could contribute to more than one term, or give further detail about severity or timing. Percentages should therefore be read as the reported values, not converted into counts or treated as a complete account of safety.
A death count is not reported in the registry record. These safety figures do not describe the primary response endpoint, and they do not by themselves support a comparison with another treatment because no control group is recorded.
12. What the Result Does, and Does Not, Mean
The study is recorded as a completed phase 2, single-group investigation of brentuximab vedotin in patients with mycosis fungoides or Sézary syndrome and variable CD30 expression. It identifies overall response rate as the primary outcome and defines that measure as combining complete and partial responses. These details explain what the study set out to summarize.
They do not establish the amount of response observed. The registry record does not report the primary ORR value, separate complete- and partial-response counts, or an interval describing uncertainty. Although it notes 3 subjects were not evaluable, it does not state how those cases were handled in the primary analysis. Without these details, readers cannot determine the response frequency or its precision.
Even if a response estimate were available, the single-group design would describe outcomes in the treated study population rather than provide a randomized comparison. There is no concurrent comparator in the registry record. General statistical principle: a one-group result cannot by itself determine how outcomes would have differed under another treatment or no study intervention. The record also does not report response results by diagnosis or CD30 expression, so it cannot support conclusions about those subgroups.
The safety figures provide a limited account of adverse-event reporting, including the number affected in the all-participants group and selected serious-event percentages. They do not give the full clinical context needed to compare risks, such as event counts by term or a comparator group. The registry record does not report a death count.
The most defensible reading is therefore narrow: the record documents a completed single-group study, its intervention, and its intended primary response measure, along with some safety summaries. It does not contain enough primary outcome information to quantify response, assess its precision, or support a comparative treatment claim. This is a limitation of what the registry record reports, not evidence that a particular outcome did or did not occur.
13. Why This Trial Matters Statistically
This trial is statistically informative as an example of the distinction between a clearly named endpoint and a fully reported result. The registry record specifies overall response rate and describes its component response categories, but does not report the numerical result. An endpoint definition is essential for interpretation, yet without the estimate and its uncertainty, readers cannot assess the magnitude or precision of the outcome.
The design also matters. A single-group phase 2 study can describe what was observed among participants receiving the intervention. It does not provide a randomized estimate of the difference between treatments. This distinction helps prevent descriptive outcomes from being read as comparative evidence. The record's lack of masking is another design feature relevant to how outcomes might have been assessed, although the record does not provide details of assessment procedures.
The note that 3 subjects were not evaluable draws attention to the analysis population. General statistical principle: a response percentage is interpretable only when its denominator and the handling of unclassifiable outcomes are clear. The record does not report those analysis details. This makes the missing response result especially consequential, because neither the estimate nor its construction can be checked.
Finally, planning assumptions should not be confused with a completed study's observed evidence. The stated planning illustration uses a hypothetical two-arm framework, while the recorded trial is single-group. Recognizing that distinction prevents planning calculations from being mistaken for trial design details or observed power. Together, these features show why endpoint definitions, analysis populations, uncertainty, and design must be read alongside any headline outcome.
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 trial-specific limitation for interpretation is that the registry record does not report the primary ORR result. It also omits the complete- and partial-response counts, the endpoint denominator, and a confidence interval. Although 3 subjects are noted as not evaluable, the record does not explain why or how they were treated in the analysis. This prevents reconstruction of the primary result.
The single-group design has no concurrent control, so it cannot supply a randomized comparison. Masking is listed as none. The registry record does not detail response-assessment procedures or report how potential assessment influences were addressed. It also does not give outcome estimates by mycosis fungoides versus Sézary syndrome or by CD30-expression level, despite those features in the study title.
Additional design and analysis details are not reported in the registry record, including response criteria, assessment schedule, duration of treatment, follow-up detail, statistical analysis plan, multiplicity strategy, and missing-data methods. The registry does not report an interim-analysis approach or the trial's own sample-size justification. These omissions do not establish that such plans or procedures were absent; they limit what can be verified from the record.
Safety interpretation is also constrained. The record supplies affected counts for serious and other adverse events and percentages for selected serious-event terms, but does not report event counts by term, exposure time, or a death count. With no control group, these summaries do not provide comparative safety estimates. The registry's completed status and study dates do not resolve these outcome-reporting gaps.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Brentuximab Vedotin (SGN-35) in Patients With Mycosis Fungoides With Variable CD30 Expression Level — NCT01396070.
- Registry record last updated 2017-04-05; status last verified 2017-02.
- Results first posted 2017-04-05.
- 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
NCT01396070 is recorded as a completed phase 2 exploratory pilot study of brentuximab vedotin in patients with mycosis fungoides and Sézary syndrome with variable CD30 expression. The design is single-group, allocation is listed as not applicable, and masking is listed as none. The registry records enrollment of 36 and study dates of 2011-05 to primary completion in 2015-04.
The intervention was brentuximab vedotin at 1.8 mg/kg intravenously every 3 weeks. Overall response rate was the primary outcome, defined by combining complete and partial responses, with a timeframe of 2 years. The record notes 3 subjects were not evaluable but does not report the primary result or its uncertainty.
Safety entries report 23 affected for serious adverse events and 32 for other adverse events, each among 36 at risk. Selected serious-event percentages are listed, while a death count is not reported. The absence of a comparator and the lack of numerical response results limit interpretation of both efficacy and comparative safety.