Study of TBL 12 Sea Cucumber Extract for Patients With Untreated Asymptomatic Myeloma: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Study of TBL 12 Sea Cucumber Extract for Patients With Untreated Asymptomatic Myeloma (NCT01096810), a na phase 2 study. The primary outcome is Time to Progression.

2. Clinical Question

This is a question about the observed course of disease in participants treated within the study, not a direct comparison of TBL 12 with another treatment or with no treatment. Without a concurrent comparator, the study cannot establish how the observed course differs from what would have occurred under another care strategy. The registry lists antitumor effect as a secondary outcome, but does not report a result for it.

The endpoint uses progression criteria based on the International Uniform Response Criteria for Multiple Myeloma. The registry description lists laboratory changes, increases in bone marrow plasma cells, new bone lesions or soft tissue plasmacytomas, and hypercalcemia not attributable to another cause. The exact thresholds are part of the registry’s endpoint definition; the observed progression times and the number of participants who progressed are not reported in the registry record.

3. Trial Design

This is a Phase II, single-group study of TBL 12 for untreated asymptomatic multiple myeloma. The registry lists an enrollment of 20 and identifies the study status as terminated. It records no allocation to multiple groups and no masking. Consequently, the study is not a randomized comparison and has no concurrent control arm.

The intervention is described as an oral dose of 2 units (20 mL each) twice a day until disease progression. The study’s stated purpose includes assessing effects against asymptomatic multiple myeloma and observing side effects. Eligibility criteria include a diagnosis of asymptomatic multiple myeloma, adequate cardiac, liver and kidney function, and age 18 and older, among other criteria.

The recorded study start is 2008-09, and the primary completion date is 2014-03. These registry dates describe the study record; they do not by themselves establish how long any participant was followed. Participant-level follow-up duration is not reported in the registry record.

The primary endpoint is time to progression, with time measured in cycles. The registry defines a cycle as 28 days and specifies that time begins on the date of treatment and ends on the date of first documented progression. Although the record gives the endpoint’s summary type as a median with a full range, it does not report the median, range, or other participant-level endpoint results.

The record does not report a sample-size justification, an allocation plan beyond identifying a single group, or a detailed statistical analysis plan. The number of analyses recorded is zero. As a result, the design can be described from its registration fields, but the intended procedures for estimating the endpoint and handling the data cannot be reconstructed from the 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

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 Time to Progression, measured over From date of treatment until the date of first documented progression.

The primary endpoint is time to progression: the interval from the date of treatment to the date of first documented progression. It is measured in cycles, which the registry defines as 28-day cycles. The listed summary measure is the median, accompanied by the full range. A median describes the middle of the observed time values, while the range describes the lowest and highest observed values. Neither the median nor the range is reported in the registry record.

Progression is defined using the International Uniform Response Criteria for Multiple Myeloma. The listed criteria include an increase in serum or urine protein measures, an increase in bone marrow plasma cells, new lytic bone lesions or soft tissue plasmacytomas, or development of hypercalcemia not attributable to another cause. For several laboratory and marrow criteria, the definition also requires an absolute increase in addition to the stated relative increase. The registry’s exact thresholds are expressed as >25%, with additional absolute thresholds of 5 g/dL, 200 mg/24 hours, or 10%, depending on the measure.

Time to progression is used to describe how long participants remain without documented progression after starting the study intervention. It retains information about timing that a simple yes-or-no progression outcome would not capture. In general, time-to-event outcomes also require clear rules for defining the event and for handling participants whose event status is unknown when observation ends. The registry record does not report those handling rules, nor does it provide the number of observed progression events or a time-to-progression estimate. Therefore, the endpoint’s definition is available, but its study result and precision cannot be assessed from the registry record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

Statistical interpretation depends on separating the study’s design, its endpoint definition, its analysis procedures, and the results actually reported. In this record, the design and endpoint definition are available, but detailed analysis procedures and the primary outcome estimate are not. The distinction matters: a well-defined endpoint does not itself provide evidence about the endpoint’s observed value.

What does a single-group design allow?

A single-group study follows participants receiving the study intervention and describes what happens to them. It can be useful for recording outcomes and safety observations in the enrolled group. However, without a concurrent comparator, it cannot estimate a treatment contrast within the study. A contrast asks how outcomes differ between groups receiving different strategies under comparable conditions. This study has no such between-group contrast, and its record identifies no randomized allocation or masking.

Consequently, any observed course in the study group would need to be understood as an outcome in that group, rather than as a direct estimate of the difference attributable to the intervention. General factors such as the natural course of a condition, participant selection, and other care can affect outcomes in an uncontrolled study. These are general design considerations, not claims about what occurred in this particular trial.

How should time to progression be understood?

Time to progression is a time-to-event endpoint. The event is the first documented progression, defined according to the criteria in the registry. The measurement starts at treatment and is expressed in cycles, with a cycle defined as 28 days. A median time summarizes the point at which the ordered event-time distribution reaches its middle. A full range describes the span from the smallest to the largest observed times. The registry lists these as the summary measure and dispersion, but gives neither value.

Time-to-event analysis often needs to account for participants whose event has not been observed by the end of their available follow-up or whose follow-up ends for another reason. General methods can retain information from such participants up to the time they were last observed, under defined assumptions. The registry record does not state whether such methods were used, how follow-up ended for participants, or how censoring was defined. No method should be inferred from the endpoint label alone.

What does a confidence interval communicate?

In general, a confidence interval expresses uncertainty around an estimated quantity under the assumptions of the statistical method used. A narrower interval indicates greater precision under that method, while a wider interval indicates more uncertainty. Here, no time-to-progression estimate or confidence interval is reported in the registry record, so its precision cannot be evaluated. A full range is not a substitute for a confidence interval: the range describes the observed extremes, while an interval around an estimate characterizes uncertainty according to a statistical procedure.

What can the analysis fields tell us?

The registry records zero analyses. This means the record does not supply an analysis result for the primary endpoint. It also does not identify a statistical test, model, adjustment strategy, or method for handling incomplete observations. A result should not be reconstructed from the enrollment count or from the endpoint definition. The fact that a median and full range are specified as the intended summary does not establish that they were calculated or reported.

The record lists three outcome measures and names antitumor effect as a secondary outcome. It does not give secondary-outcome results or enough detail to identify the definition and analysis of every listed measure. In general, secondary outcomes can add context, but their interpretation depends on prespecified definitions, adequate data, and a clear account of how multiple outcomes were considered. Those details are not reported in the registry record.

What is the role of sample-size planning?

Sample-size planning asks how many participants or events may be needed to estimate or detect a specified effect with chosen statistical properties. Such planning depends on assumptions about the effect, variability or event rate, significance level, allocation, and analysis method. The registry record does not report the trial’s own sample-size justification or statistical design assumptions. Any planning illustration based on an observed effect is distinct from the trial’s original design and is not observed power. A planning calculation cannot replace an observed result, and it does not show that the study had a particular probability of detecting an effect.

More generally, power is a design concept tied to a hypothesized effect and specified assumptions. It is not a measure of whether a result is true, and it cannot be read off from a non-significant result. Since no primary result or analysis is recorded here, neither a test outcome nor a trial-specific power conclusion can be assessed.

Why does the progression definition matter?

An endpoint is interpretable only when the event is defined. The registry ties progression to named criteria involving protein measures, bone marrow plasma cells, new lesions or plasmacytomas, and hypercalcemia. This gives the reader a basis for understanding what was intended to count as progression. In general, consistent application of an endpoint definition is important because differing assessments can alter when an event is recorded. The record does not describe assessment schedules, adjudication procedures, or how ambiguous cases were resolved.

In summary, the statistical record identifies a single-group design and a specified time-to-progression endpoint, but it does not report the analysis or the endpoint result. General statistical principles can explain what the planned summaries would mean, but cannot supply the missing estimate, event count, uncertainty, or analysis details.

10. Multiplicity, Missing Data and Analysis Populations

Multiplicity concerns arise when a study evaluates more than one outcome, comparison, or analysis. In general, carrying out multiple tests can increase the chance of obtaining at least one apparently unusual result by chance, so an analysis plan may specify how outcomes are prioritized or how statistical error is handled. This registry lists three outcome measures and names antitumor effect as a secondary outcome, while identifying time to progression as primary. It does not report a multiplicity strategy, adjustment method, or detailed hierarchy for interpreting the outcomes. No multiple-testing procedure should be inferred.

Missing-data methods are also not reported in the registry record. The record does not describe whether any participants had incomplete outcome information, how missing measurements were handled, how participants without documented progression were treated, or whether sensitivity analyses were planned or conducted. These details are relevant in general because incomplete follow-up can affect time-to-event estimates and the population represented by an analysis.

The record does not define analysis populations, such as the set of participants included in an efficacy analysis, nor does it state whether all enrolled participants were included in safety summaries. Enrollment is listed as 20, and the adverse-event fields give 20 at risk in each category, but the record does not explain how those populations were defined or whether participants had different exposure or follow-up durations.

These omissions limit reproducibility and interpretation. They do not establish that the trial used a particular approach or that data were missing; they mean those analysis details are not reported in the registry record. The primary endpoint estimate is also not reported, preventing assessment of how any analysis decisions affected the result.

11. Safety Results

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

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 5% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: pneumococcal pneumonia (5.0%).

The registry’s adverse-event fields list 20 participants at risk and one affected in the serious-event category. In the other-event category, it also lists 20 at risk and one affected. These are counts recorded in the registry, not comparative estimates against an untreated or control group. The duration of exposure and the period over which participants were monitored for adverse events are not reported in the registry record.

Pneumococcal Pneumonia is listed among the serious-event terms, with a reported value of 5.0%. The record does not provide enough detail in these fields to establish the timing, duration, clinical course, or attribution of that event term. It should be read as a registry safety entry, not as evidence that TBL 12 caused the event or changed its risk.

Deaths are not reported in the registry record. This should not be interpreted as evidence that there were no deaths; the record contains no death count. Likewise, the small number of participants and absence of a comparator limit what these safety entries can establish about frequency or relative risk. The entries describe reported observations within this study, not a complete comparative safety assessment.

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

The record establishes that a Phase II, single-group study of TBL 12 was registered for untreated asymptomatic multiple myeloma, with time to progression specified as its primary endpoint. It also supplies the intervention description, progression criteria, and limited adverse-event entries. These details make it possible to understand the question the study set out to address and how progression was defined.

The principal result needed to answer that question is not reported in the registry record. There is no reported median time to progression, full range, event count, or uncertainty interval. The record also provides no result for the listed secondary outcome, antitumor effect. Without those findings, the observed disease course in the study group cannot be quantified from the record.

Even if a single-group endpoint estimate were available, it would describe outcomes in the participants enrolled and followed; it would not directly quantify a difference versus another treatment. Because there is no control group, the study cannot provide a randomized comparison or isolate an intervention effect from other influences. The registry itself identifies that the study is not a randomized comparison.

The listed enrollment of 20 does not specify the number contributing to each endpoint analysis or the completeness of follow-up. The record also does not report a statistical analysis plan, missing-data approach, or multiplicity procedure. These gaps make it impossible to evaluate analysis choices or the precision and robustness of an efficacy estimate.

The safety fields record one affected participant among 20 at risk in each of the serious and other-event categories and list Pneumococcal Pneumonia at 5.0% among serious-event terms. These entries should be interpreted descriptively and in the context of the study’s size and single-group design. They do not establish causation or comparative risk. The terminated status is recorded, but the reason for termination is not reported. Taken together, the record clarifies the planned question but does not supply the outcome evidence needed to answer it quantitatively.

13. Why This Trial Matters Statistically

This trial is statistically informative as an example of the difference between a defined research question and a reported result. The registry specifies a primary time-to-progression endpoint, its time origin, its event definition, and the summary form of median with full range. Those elements help readers understand what was to be measured. However, an endpoint definition is not an endpoint estimate, and the registry does not report the result.

The single-group design also makes the inferential boundary clear. Outcomes observed in a treated group can describe that group, but a comparator is needed for a direct estimate of how outcomes differ under another strategy. The record identifies no control group and no randomized allocation. This illustrates why design determines the questions a result can answer, independently of how carefully an outcome is measured.

The record also highlights why precision and analysis transparency matter. A median without an uncertainty measure, participant flow, event count, or follow-up description would be difficult to evaluate; in this case, even the median is not reported. The number enrolled alone cannot replace those details. Similarly, safety counts are most informative when the population, exposure period, ascertainment procedures, and attribution are clear. Those details are not reported in the registry record.

Finally, the record distinguishes registration information from analysis evidence. It gives a trial status and completion date, but not a reason for termination or an account of the analyses conducted. Recognizing these distinctions prevents readers from treating design intentions, registry status, or adverse-event entries as substitutes for a reported efficacy result. The statistical lesson is to keep the question, design, endpoint, analysis, and result separate when evaluating clinical evidence.

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 most direct limitation is that the primary endpoint result is not reported in the registry record. No median time to progression, full range, event count, or confidence interval is available. Without these, neither the observed timing of progression nor the precision of an estimate can be assessed. The secondary outcome named as antitumor effect also has no reported result.

The study is a single-group design with no concurrent comparator and no randomized allocation. It therefore cannot provide a like-for-like comparison of outcomes under different strategies. Any descriptive result from the group would not, on its own, establish what would have happened under another care strategy.

The registry does not report participant flow, the number included in efficacy analyses, follow-up duration, outcome assessment timing, or the handling of participants without documented progression. It also does not describe missing-data methods, analysis populations, or sensitivity analyses. These omissions prevent evaluation of whether the endpoint data represent all enrolled participants and how incomplete observations may have affected any estimate.

The statistical analysis plan and sample-size justification are not reported in the registry record. The record lists zero analyses, so no test, model, or uncertainty procedure can be examined. The reason for the terminated status is not reported, and the status alone does not explain why recruitment or follow-up ended.

Safety interpretation is limited by the registry entries’ lack of information about exposure and monitoring duration, event timing, attribution, and detailed outcomes. The counts are descriptive and have no control-group comparison. These limitations are specific to interpreting the available record; they do not establish that particular problems occurred during the trial.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Study of TBL 12 Sea Cucumber Extract for Patients With Untreated Asymptomatic Myeloma — NCT01096810.
• Registry record last updated 2016-07-22; status last verified 2016-06.
• Results first posted 2016-07-22.
• 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 describes NCT01096810 as a Phase II, single-group study of TBL 12 in people with untreated asymptomatic multiple myeloma. Enrollment is listed as 20, the status as terminated, and masking as none. The recorded study start is 2008-09, with primary completion in 2014-03.

TBL 12 is described as an oral dose of 2 units (20 mL each) twice a day until disease progression. The primary endpoint is time to progression, measured in cycles of 28 days and summarized as a median with full range. Progression criteria are specified, but the endpoint result is not reported in the registry record. Antitumor effect is listed as a secondary outcome, with no result reported.

The adverse-event fields list one affected participant among 20 at risk in each of the serious and other-event categories. Pneumococcal Pneumonia appears among serious-event terms at 5.0%. Deaths, the reason for termination, the analysis plan, and the trial’s sample-size justification are not reported in the registry record.