Doxorubicin-eluting LC Bead M1 for Patients With Hepatocellular Carcinoma: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Doxorubicin-eluting LC Bead M1 for Patients With Hepatocellular Carcinoma (NCT02007954), a na phase 2 study. The primary outcome is Success of DEBDOX-M1 Procedure as a Measure of Feasibility.

2. Clinical Question

The study asks whether small doxorubicin-eluting LC Bead M1 beads can be used feasibly and safely to deliver chemotherapy to hepatic lesions in patients with hepatocellular carcinoma. The registry describes the beads as 70-150 microns and the treatment as transarterial chemoembolization (TACE). Its stated rationale is that smaller beads may penetrate farther into the tumor bed, avoid premature proximal vessel occlusion, and support more consistent dosing. These are proposed advantages, not findings established by the study description.

This design can describe procedural experience and observed outcomes in the treated group. It cannot by itself establish how those outcomes compare with another treatment or with what would have happened without DEBDOX-M1. The registry also lists secondary outcomes concerning tumor response by EASL, qEASL, and mRECIST, downstaging or bridging to surgical interventions, and the AFP tumor marker before and after treatment. Results for these secondary outcomes are not reported in the registry record.

3. Trial Design

This is a Phase 2, single-group study sponsored by Yale University. The registry lists 24 enrolled patients and the status as completed. It records allocation as not applicable and masking as none. There is no randomized or masked comparison group, so the study is descriptive rather than a controlled estimate of treatment differences.

The intervention is DEBDOX, a device using LC-Bead M1 beads loaded with doxorubicin for TACE. The registry’s description identifies bead size as 70-150 microns and states that treatment delivers chemotherapy into liver tumor tissue through the hepatic artery. The study summary describes feasibility and safety as its purpose. The primary measure is technical success in using the beads to treat hepatic lesions, assessed over 6 months.

The registry says 24 patients received a total of 50 DEBDOX-M1 TACE procedures. Those are different units of experience: patients and procedures. Procedures performed in the same patient are not automatically independent observations, so an analysis based on procedure counts would need to account for that structure. The registry record does not report whether or how such dependence was addressed.

Follow-up described in the record includes monthly clinic visits and blood tests, including liver function and tumor markers, with imaging—usually MRI—every 1-2 months. The summary says patients were on study for 6 months and then exited from the study and were followed for survival. The record does not provide a detailed schedule of assessments for every outcome or a statistical analysis plan.

The registry lists a start date of 2014-02 and primary completion on 2015-08-11. It does not report a control arm, a treatment allocation procedure, masking, or a planned sample-size justification. The record describes continued eligibility for the smaller beads after study exit if recommended, but does not specify how that subsequent treatment was handled in outcome analyses.

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

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: The 24 patients received a total of 50 DEBDOX-M1 TACE procedures.

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.

6. Primary Endpoint

The registry lists the primary outcome as Success of DEBDOX-M1 Procedure as a Measure of Feasibility, measured over 6 months.

The primary endpoint is titled as success of the DEBDOX-M1 procedure as a measure of feasibility. Its description defines feasibility as achieving an acceptable level of technical success when using the beads to treat hepatic lesions in patients with hepatocellular carcinoma. The timeframe is 6 months, and the unit is described as a percentage of successful treatments.

A technical-success endpoint addresses whether the procedure can be carried out as intended. Such an endpoint is relevant to feasibility because procedural completion is a practical prerequisite for evaluating a treatment approach in a clinical study. It is not, on its own, a measure of tumor response, survival, or comparative benefit. Those questions require outcome definitions and evidence suited to them.

There is an ambiguity in the registry record: the primary measure is classified as a number, while its unit is described as a percentage. The record also says the 24 patients received a total of 50 DEBDOX-M1 TACE procedures, but it does not report the number of successful treatments or a percentage result. The definition of what counted as an acceptable level of technical success is not further specified in the record. As a result, the primary endpoint’s observed value and the precise operational threshold cannot be assessed from the registry record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry describes a single-group study, with no masking and allocation recorded as not applicable. The primary endpoint is a technical-success measure over 6 months. No detailed statistical analysis plan is reported in the registry record. The following explanations are general, and distinguish what the study design could describe from what the record actually reports.

What does a single-group design estimate?

In general, a single-group study describes outcomes among people who receive an intervention. It can document procedural experience and identify events observed during follow-up. Without a concurrent comparison group, it cannot isolate the effect of the intervention from other influences. A change over time, for example, cannot automatically be attributed to treatment because the study does not observe a randomized alternative group under the same conditions.

Here, the registry reports that 24 patients received a total of 50 procedures. These counts should not be treated as interchangeable. If outcomes are recorded for each procedure, multiple procedures in one patient may be related. General statistical methods for clustered observations account for the fact that outcomes within a person may be more alike than outcomes from different people. The registry record does not say whether the primary endpoint was analyzed per patient, per procedure, or with a method accounting for repeated procedures.

What does a feasibility endpoint measure?

In general, a technical-success endpoint asks whether an intervention could be delivered according to an operational definition. A percentage summarizes the proportion meeting that definition, but its interpretation depends on the denominator and the success criteria. A percentage based on patients answers a different question from one based on procedures. The registry describes the unit as a percentage of successful treatments and separately reports patient and procedure counts, but does not state the denominator for the endpoint or give its observed value. It also does not state the threshold that constitutes an acceptable level of success. Those omissions make the endpoint difficult to reproduce or interpret quantitatively.

The registry classifies the primary parameter as a number while describing the unit as a percentage. This may reflect a record-format distinction, but the registry record does not clarify it. The central information needed to interpret the endpoint is the count or percentage observed, the denominator, and the rule defining success; these are not reported for the primary endpoint.

How should uncertainty be understood?

In general, a confidence interval expresses the range of estimates reasonably compatible with the data under a specified statistical model. Its width conveys precision: a wider interval means greater uncertainty about the estimated quantity. A point estimate without an interval can conceal substantial uncertainty, particularly when the available experience is limited. Neither a confidence interval nor another measure of uncertainty for the primary endpoint is reported in the registry record. There is therefore no reported precision estimate to interpret.

A p-value, in general, evaluates how compatible data are with a specified null hypothesis under an analysis model. It does not give the probability that a treatment works, and it does not measure the size or clinical importance of an effect. No primary-endpoint p-value or hypothesis test is reported in the registry record. In a single-group feasibility study, estimation of the technical-success rate and its uncertainty may be more informative than a test against an unspecified benchmark, but the registry record does not report such an estimate.

What do the planning calculations mean?

General sample-size illustrations translate an assumed effect and statistical design into a planning quantity. They are not observed outcomes and are not evidence that a study had a particular probability of detecting an effect after it was conducted. The registry’s planning assumptions describe a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the effect to detect expressed as a stated fraction of an observed effect. They also distinguish total events for time-to-event calculations from participants per arm for other calculations. Those assumptions are planning illustrations, not the design of this single-group study; sample-size calculations should not be described as observed power. The registry does not report the study’s own sample-size justification.

General planning calculations depend on assumptions about the outcome, effect, allocation, and analysis. If a calculation uses an effect observed in a study, it is an illustration of what a future study might require under those assumptions, not an unbiased confirmation that the effect is real. The registry record does not report a randomized two-arm comparison for this trial, so the two-arm assumptions in such planning illustrations do not describe the treatment design used.

How do multiple outcomes affect interpretation?

The registry lists 10 outcome measures and 1 analysis. It names several secondary efficacy outcomes, but their results and any adjustment for examining multiple outcomes are not reported in the registry record. In general, testing many outcomes can increase the chance of at least one apparently unusual result if each is assessed separately. Multiplicity procedures can address that issue, but the choice depends on the analysis plan and the intended claims. No multiplicity strategy is reported in the registry record, so it cannot be determined whether or how multiple comparisons were handled.

In general, statistical methods should match the outcome type and structure. A count or proportion, a repeated measurement such as a tumor marker, and a time-to-event outcome do not necessarily call for the same method. The registry record does not report model specifications, covariate adjustment, handling of repeated measures, or a definition of the analysis population. The available information therefore supports a description of the registered questions and study structure, but not a reconstruction of the statistical analyses.

10. Multiplicity, Missing Data and Analysis Populations

The registry lists 10 outcome measures and 1 analysis. Named secondary outcomes include tumor response by EASL, qEASL, and mRECIST, the number of patients downstaged or bridged to surgical interventions, and AFP tumor marker values before and after treatment. Their numerical results are not reported in the registry record. The record also does not report whether outcome definitions or analysis priorities were changed during the study.

When a study assesses several outcomes, general statistical practice considers whether testing or interpreting them separately could increase the chance of emphasizing a result that arose by chance. A prespecified hierarchy or multiplicity adjustment can help align conclusions with the number of questions asked. No such approach is reported in the registry record. Because the secondary results are absent, the impact of multiplicity on interpretation cannot be evaluated.

The record does not report methods for missing outcome data, including how incomplete follow-up, unevaluable imaging, or missing laboratory measurements were handled. It does not report an analysis population definition, such as which participants or procedures were included in each outcome calculation. Nor does it describe whether all enrolled patients contributed to every outcome. These gaps matter because exclusions and missing observations can change which experience an estimate represents.

The study summary describes clinic visits and blood tests monthly, imaging usually every 1-2 months, and follow-up for survival after exit from the 6-month study period. It does not give completion rates for these assessments or explain the statistical treatment of missing visits. The registry record also does not report interim analyses or a statistical plan for handling repeated assessments. Consequently, it is not possible to assess how incomplete follow-up or multiple measurements affected the reported analyses.

11. Safety Results

The registry reports serious adverse events as affected participants divided by participants at risk.

Deaths

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 0% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: cardiac arrest (4.2%), hypoxia (4.2%), thromboembolic event (4.2%), renal failure (4.2%), respiratory failure (4.2%).

The registry reports safety counts for the DEBDOX group. It lists 24 at risk for serious adverse events and 4 affected. For other adverse events, it lists 24 at risk and 24 affected. It also records 1 death. These are descriptive counts from the registry record; they do not establish that DEBDOX-M1 or TACE caused the events.

The top serious terms listed are cardiac arrest, hypoxia, thromboembolic event, renal failure, respiratory failure, and upper gastrointestinal hemorrhage. Each term is shown with 4.2%. The registry record does not explain whether the terms represent separate patients, overlapping events, or event episodes, so they should not be added together or interpreted as a total. It also does not provide timing, duration, outcome, or a causal attribution for each term.

Serious adverse events and other adverse events are separate categories in the record. The figures alone do not convey clinical context, baseline risk, or whether an event was expected. With no comparator group, the counts cannot show whether event frequency differed from that under another treatment or without treatment. They describe what the registry reports for this study, not a comparative safety estimate.

12. What the Result Does, and Does Not, Mean

The registry record establishes that this Phase 2 study examined DEBDOX-M1 in patients with hepatocellular carcinoma using a single-group design. It describes a feasibility question centered on technical success and reports that 24 patients received a total of 50 DEBDOX-M1 TACE procedures. It also records serious and other adverse-event counts and lists several serious-event terms. Those details provide context about the treatment experience and the outcomes the study intended to assess.

The record does not report the primary endpoint’s observed result. It does not give the count of successful treatments, a percentage, an uncertainty interval, or a clearly stated denominator for that endpoint. The description says the endpoint concerns an acceptable level of technical success, but the record does not specify the operational success threshold. Thus, the registry information does not quantify feasibility.

The record also does not report numerical results for its listed tumor-response, downstaging or bridging, or AFP outcomes. It cannot establish from those outcome labels alone that tumor response occurred or that patients were downstaged or bridged to surgery. The safety counts describe events recorded among treated patients; they do not establish causation or comparative safety.

Because the study has no comparison group, it cannot estimate a treatment contrast against another approach. Even a complete set of single-group outcome results would describe outcomes in the treated group rather than demonstrate that the intervention caused them. General factors such as patient characteristics, disease course, and concurrent care can affect observed outcomes, and the design does not separate those influences from treatment effects.

The study’s proposed rationale for using smaller beads is a statement of motivation, not evidence that the proposed advantages were achieved. The registry describes the beads, treatment route, follow-up approach, and intended outcomes, but the missing primary and secondary results prevent a quantitative assessment of efficacy or feasibility. The appropriate conclusion is therefore limited to the study design and the safety counts recorded, without a claim that the treatment works or does not work.

13. Why This Trial Matters Statistically

This study is statistically notable because its primary question concerns feasibility, while its registry record does not report the primary endpoint result. For a technical-success question, the denominator and operational definition are central: a reader needs to know what counted as a successful treatment and whether the unit was a patient or a procedure. The record describes a percentage outcome but does not give its value or denominator, and it labels the parameter as a number. That combination leaves the main quantitative question unresolved.

The distinction between patients and procedures is also important. The registry reports 24 patients and a total of 50 procedures. In general, repeated procedures within a patient may have correlated outcomes. Treating each procedure as an independent observation can understate uncertainty if that correlation is ignored. The registry record does not specify the unit of analysis or whether any method accounted for repeated procedures.

The single-group structure defines the limits of inference. It can describe the experience with DEBDOX-M1 but cannot estimate a randomized comparison. This matters for both efficacy and safety: a count of events in a treated group is not a comparative risk estimate, and an observed tumor outcome would not by itself establish a treatment effect.

The registry lists several secondary outcomes, but their results and multiplicity approach are not reported. Multiple outcome definitions can offer different ways to characterize response, yet interpretation depends on prespecified methods and transparent results. Without those details, the reader cannot assess how outcomes were prioritized, how uncertainty was quantified, or whether selective emphasis influenced conclusions.

Finally, the record includes sample-size planning assumptions that describe an illustrative two-arm calculation, whereas the registered study is single-group. Keeping planning scenarios separate from the actual design prevents them from being mistaken for evidence about the study’s power or observed findings. The statistical value of the record lies in clarifying the research question and its design constraints, while also making clear how much outcome information is not reported.

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.

Several limitations specific to this study affect interpretation. It is a single-group study with no comparator and no masking. The registered design can describe the treated group but cannot provide a like-for-like estimate of how outcomes compare with another treatment. Differences from an external population would not resolve that limitation because the record does not report a controlled comparison.

The primary endpoint’s result is not reported in the registry record. The record does not give a successful-treatment count or percentage, specify the denominator, or operationalize the acceptable technical-success threshold. It describes the primary measure as a number but gives its unit as a percentage, adding ambiguity about how to read the outcome.

The registry reports both patients and procedures: 24 patients received a total of 50 procedures. It does not state whether the primary endpoint is calculated per patient or per procedure, or whether repeat procedures were handled as correlated observations. This limits the ability to understand the intended analysis unit and its uncertainty.

The registry names secondary efficacy outcomes but does not report their numerical results. It does not describe methods for missing data, analysis populations, multiplicity, interim analyses, or model selection. The absence of those details prevents an assessment of how incomplete follow-up or multiple endpoints may have affected the results.

Safety information is descriptive and lacks event-level timing and causal assessment. The listed serious-event terms and percentages do not establish attribution, and the registry does not explain whether terms overlap. The follow-up summary gives a general schedule but does not report assessment completion or the handling of observations after study exit. These limitations restrict interpretation to the registered design and reported counts.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Doxorubicin-eluting LC Bead M1 for Patients With Hepatocellular Carcinoma — NCT02007954.
• Registry record last updated 2017-08-11; status last verified 2014-07.
• Results first posted 2017-08-11.
• 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.

18. Record Summary

The registry record describes a completed Phase 2, single-group study of doxorubicin-eluting LC Bead M1 for hepatocellular carcinoma, sponsored by Yale University. It lists 24 enrolled patients and states that 24 patients received a total of 50 DEBDOX-M1 TACE procedures. Masking is recorded as none, and allocation as not applicable.

The primary question concerns technical success as a measure of feasibility over 6 months. The registry describes the unit as a percentage of successful treatments but does not report the endpoint result or clarify its denominator. Listed secondary outcomes include tumor response by EASL, qEASL, and mRECIST, downstaging or bridging to surgical interventions, and AFP before and after treatment; their results are not reported in the registry record.

Safety counts include 4 patients affected by serious adverse events among 24 at risk, 24 affected by other adverse events among 24 at risk, and 1 death. The record does not establish causality. It describes monthly clinic visits and blood tests, imaging usually every 1-2 months, and survival follow-up after study exit. The design supports descriptive reporting, not a controlled treatment comparison.