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 Romidepsin in Treating Patients With Recurrent and/or Metastatic Thyroid Cancer That Has Not Responded to Radioactive Iodine (NCT00098813), a na phase 2 study. The primary outcome is Tumor Major Response Rate (Including Stable Disease) as Measured by RECIST Criteria.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00098813 |
| Phase | Phase 2 |
| Condition | Recurrent Thyroid Cancer, Stage IV Follicular Thyroid Cancer, Stage IV Papillary Thyroid Cancer |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 20 |
| Arms in the primary outcome | n/a |
| Lead sponsor | National Cancer Institute (NCI) |
| Results posted | Yes |
2. Clinical Question
Population
Patients with recurrent and/or metastatic non-medullary thyroid carcinoma that had not responded to radioactive iodine. The registry describes papillary, follicular, and Hurthle cell variants; listed conditions include recurrent thyroid cancer and stage IV follicular and papillary thyroid cancer.
Intervention
Romidepsin as a single agent. The treatment description specifies intravenous administration over 4 hours on days 1, 8, and 15, repeating every 28 days for up to 12 courses if unacceptable toxicity or disease progression did not occur.
Comparator
There was no comparison group. The registry identifies a single-group study, with allocation listed as NA and masking as none. Outcomes therefore cannot be read as a randomized comparison with another treatment or with a control group.
Primary question
The primary outcome was the number of participants with a tumor major response, with stable disease included, according to RECIST criteria. The stated assessment period was from treatment start to 8 weeks. The actual number meeting this endpoint is not reported in the registry record.
In practical terms, the trial asked whether participants receiving romidepsin had a qualifying tumor status during the stated assessment period. Because stable disease was included in the endpoint wording, the measure was not limited to tumor shrinkage. It combined the response categories specified by the study title for this outcome, rather than reporting a separate count for each category.
The study enrolled 20 participants, but the registry record does not report how many met the primary endpoint. It also does not report an estimated response rate, confidence interval, or analysis comparing the result with a prespecified benchmark. The central question can therefore be described from the endpoint definition, but the posted record does not provide the outcome needed to quantify the result.
3. Trial Design
This was a phase II study of single-agent romidepsin in patients with radioiodine-refractory metastatic non-medullary thyroid carcinoma. The registry record gives an enrollment of 20 and lists the study as completed. The recorded start was 2004-10, and primary completion was 2009-08.
The design was single group, with no masking and allocation listed as NA. The treatment description states that participants received romidepsin intravenously over 4 hours on days 1, 8, and 15. Treatment repeated every 28 days for up to 12 courses, provided unacceptable toxicity or disease progression did not occur. Laboratory biomarker analysis and positron emission tomography are also listed as interventions; their role in the statistical analysis is not reported in the registry record.
With no concurrent comparator group, the design can describe outcomes among participants who received the study treatment, but it cannot by itself estimate a randomized treatment contrast. Differences from other patient groups or from an assumed course without treatment are not measured by this single-group structure. The registry record does not report a control condition or a comparative allocation procedure.
The record lists one primary outcome measure, a count of participants, with assessment from treatment start to 8 weeks. It does not report the observed primary outcome, the detailed eligibility criteria, the statistical analysis plan, or the rationale for the enrollment target. Those details should not be inferred from the phase designation or from the treatment schedule. The registry status is completed, but completion does not establish that a result or full analysis is posted.
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 | Treatment (Romidepsin) |
|---|---|
| STARTED | 20 |
| COMPLETED | 1 |
| NOT COMPLETED | 19 |
Reasons for not completing this period:
| Reason | Treatment (Romidepsin) |
|---|---|
| Death | 8 |
| Protocol Violation | 1 |
| Withdrawal by Subject | 7 |
| Progression of Disease | 1 |
| Other complicating disease | 1 |
| Reversal of RAI resistance | 1 |
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 | Treatment (Romidepsin) |
|---|---|---|
| Participants analysed | 20 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 12 |
| Age, Categorical: >=65 years | Participants | 8 |
| Age, Continuous | years | 53.5 (SD 34.64823228) |
| Sex: Female, Male: Female | Participants | 10 |
| Sex: Female, Male: Male | Participants | 10 |
| Region of Enrollment: United States | participants | 20 |
6. Primary Endpoint
The registry lists the primary outcome as Tumor Major Response Rate (Including Stable Disease) as Measured by RECIST Criteria, measured over From start of treatment to 8 weeks.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is titled “Tumor Major Response Rate (Including Stable Disease) as Measured by RECIST Criteria.” Its recorded data type is a number of participants, and its timeframe runs from treatment start to 8 weeks. Thus, the endpoint is intended to count participants who met the study’s qualifying tumor-status definition during that period. The inclusion of stable disease is important: the endpoint’s stated scope is broader than a measure restricted to tumor shrinkage.
As a general statistical concept, tumor-response endpoints provide a structured way to summarize disease status after treatment. A common advantage of a categorical endpoint is that it translates clinical assessments into countable outcomes. The use of RECIST indicates that the study named a standardized response framework, although the registry record does not provide the specific assessment procedures, response-category details, or rules for determining which assessment counted.
The registry identifies a count rather than reporting an actual count or rate. Although the title uses the word “rate,” the posted primary result is not reported in the registry record. The record also does not give an uncertainty interval or explain how missing or unevaluable assessments would be handled. Consequently, the endpoint’s intended meaning can be described, but its observed value and precision cannot be evaluated from the posted information.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record establishes the basic setting for interpreting the study: a phase II, single-group trial with 20 enrolled participants and a primary endpoint defined as the number with tumor major response, including stable disease, by RECIST criteria from treatment start to 8 weeks. It does not report the observed endpoint count. Without that count, there is no posted estimate of how many participants met the endpoint and no response proportion to interpret.
Precision is also not available. The record does not report a confidence interval for the endpoint, and there is no posted statistical analysis for it. A confidence interval is a general statistical way to convey the uncertainty around an estimate; without an estimate and its interval, readers cannot assess how narrowly or broadly the study’s result is determined. The enrollment of 20 alone does not reveal the outcome or its precision.
The design further limits what can be concluded. Since the trial had one treatment group and no comparator, its results would describe outcomes in that group. They would not constitute a randomized comparison against another treatment or a control condition. The registry record does not report an analysis that adjusts for differences between groups, and there is no second group in the recorded design from which to obtain such a contrast.
The term “major response” should be read according to the endpoint title, which explicitly includes stable disease. It should not be silently reinterpreted as tumor shrinkage alone. The registry record does not give separate counts for stable disease or other response categories, nor does it report the criteria or assessment details used to classify participants beyond naming RECIST.
Safety information is recorded separately from the primary endpoint. The registry lists 12 participants affected by at least one serious adverse event among 20 at risk, and 16 affected by other adverse events among 20 at risk. These are descriptive counts for the treatment group, not estimates of a difference from an untreated or comparator group. The posted record does not report a death count in the deaths field.
Overall, the posted information describes the trial’s question, design, treatment schedule, and some adverse-event counts, but it does not give the primary tumor outcome or its statistical uncertainty. No conclusion about the size, consistency, or comparative meaning of a tumor response can be drawn from an endpoint definition alone.
9. Statistical Methods Explained
The registry record supplies a limited statistical description. It identifies a single-group design, enrollment of 20, one primary outcome measure, and no posted analyses. It does not report the statistical methods used to analyze the primary endpoint, a confidence interval, a hypothesis test, an analysis population, or a prespecified statistical plan. The explanations below distinguish what is recorded from general concepts that help interpret this type of study.
What does a participant count represent?
The primary outcome is recorded as a number of participants meeting a tumor-status definition. In general, a binary participant-level outcome classifies each participant as meeting or not meeting the endpoint, after which the qualifying participants can be counted. If an estimate is expressed as a proportion, its interpretation depends on the defined analysis population and on how participants without an evaluable assessment are handled. Those details are not reported in the registry record, and the actual count is not posted.
The endpoint title calls the measure a response rate, while the recorded parameter is a number of participants. The record does not provide a calculated rate or specify the denominator used for one. Enrollment was 20, but it should not be assumed that every enrolled participant had an evaluable assessment or was included in a particular analysis. The registry record does not report those details.
What would uncertainty add?
As a general explanation, a confidence interval gives a range of values compatible with an estimate under a specified statistical method and its assumptions. For a participant-level binary outcome, the interval helps convey how much uncertainty remains around the estimated frequency. The interval’s width depends on the observed data and method; it cannot be recovered from the endpoint definition alone. No interval or method for calculating one is reported in the registry record.
A point estimate without its uncertainty can be difficult to interpret, especially in a small study. The enrollment of 20 describes the number recorded as enrolled, but it does not reveal how many met the primary outcome or how precisely that outcome was estimated. The absent result cannot be replaced by an estimate derived from enrollment alone.
What does the single-group structure allow?
As a general concept, a single-group study can describe outcomes observed among participants receiving an intervention. A comparative treatment effect requires a defined comparator and a method for contrasting outcomes between groups. This record identifies a single group and no masking; allocation is listed as NA. It therefore does not report a randomized treatment contrast. Any difference between the observed outcome and an external expectation would require a stated benchmark and assumptions, neither of which is reported as part of the primary analysis.
Randomization and masking are design features, not analysis results. Their absence does not determine what the observed endpoint was, and the endpoint definition does not establish that an outcome occurred. The registry record does not report a statistical adjustment intended to address the lack of a comparator.
What do the planning assumptions mean?
The record describes sample-size illustrations based on an assumed two-arm calculation, with equal allocation, a two-sided significance level of 0.05, a normal approximation, and an effect defined as a stated fraction of the observed effect. These are planning assumptions, not a description of the trial’s single-group design. A planning illustration asks what sample size would be needed under specified assumptions; it is not the number of participants observed in this trial and is not observed power.
The registry record does not report the trial’s own sample-size justification or establish that the illustrative two-arm assumptions were used to design this study. It also does not report an observed primary effect from which readers could verify a planning exercise. The illustrative assumptions should therefore be kept separate from the trial’s actual enrollment and from any interpretation of its outcome.
Can the statistical analysis be reproduced?
No. The record lists zero analyses and does not include the primary endpoint result, analysis method, confidence interval, hypothesis test, missing-data rules, or analysis population. There is no posted estimate to recalculate. General statistical methods can explain what an analysis might report, but they cannot supply the missing trial result or establish which methods the investigators used.
Over-dispersion is a general concern when count data vary more than a chosen model expects. It is not possible to assess whether that issue applies to this endpoint from the registry record: the primary outcome is a participant count, and no model or distributional analysis is reported. More broadly, statistical assumptions should not be attributed to this trial unless the record documents them.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists one primary outcome measure: the participant count for tumor major response, including stable disease, by RECIST criteria, assessed from treatment start to 8 weeks. It lists zero analyses. The presence of one listed primary outcome does not establish that the study had no other planned outcomes or analyses; the registry record does not give a complete statistical analysis plan.
Multiplicity arises, as a general statistical concept, when multiple hypotheses or outcomes are tested and the chance of a false-positive finding is managed through a planned approach. The record does not report whether multiplicity adjustments were planned, whether secondary outcomes were analyzed, or whether interim analyses were conducted. These matters are not reported in the registry record, so no particular approach should be assumed.
The primary endpoint’s observed value and its analysis are absent. The record does not specify how participants with missing, incomplete, or unevaluable tumor assessments were treated, whether the analysis was based on all enrolled participants, or whether another analysis population was used. It also does not describe procedures for withdrawals, protocol deviations, or missing data. The endpoint timeframe is stated, but the assessment and data-handling details are not.
These omissions affect interpretation rather than changing the endpoint definition. Without the result, analysis population, and handling rules, readers cannot determine the extent to which the analyzed participants corresponded to the enrolled group or how incomplete assessments may have influenced an estimate. The record also does not provide enough information to reconstruct the statistical analysis independently.
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 |
|---|---|---|---|
| Treatment (Romidepsin) | 12 | 20 | 60.0% |
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 |
|---|---|
| Treatment (Romidepsin) | 16 of 20 |
Most frequent serious adverse event terms
| Event term | Organ system | Treatment (Romidepsin) |
|---|---|---|
| Death-Disease Progression | General disorders | 2 of 20 |
| Dyspnea | Respiratory, thoracic and mediastinal disorders | 2 of 20 |
| AST, SGOT | Investigations | 1 of 20 |
| Arrhythmia | Cardiac disorders | 1 of 20 |
| Cardiac disorder | Cardiac disorders | 1 of 20 |
| Death-Sudden Death | General disorders | 1 of 20 |
| Dysphagia | Gastrointestinal disorders | 1 of 20 |
| Fever | General disorders | 1 of 20 |
| Fistula, GI-Small bowel NOS | Gastrointestinal disorders | 1 of 20 |
| Gastrointestinal disorder | Gastrointestinal disorders | 1 of 20 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Treatment (Romidepsin) |
|---|---|---|
| Fatigue | General disorders | 7 of 20 |
| Lymphocyte decrease | Investigations | 7 of 20 |
| Hypophosphatemia | Metabolism and nutrition disorders | 6 of 20 |
| Hyperglycemia | Metabolism and nutrition disorders | 5 of 20 |
| White blood cell decrease | Investigations | 3 of 20 |
| Neutrophil count decrease | Investigations | 3 of 20 |
| ALT, SGPT | Investigations | 2 of 20 |
| Hypocalcemia | Metabolism and nutrition disorders | 2 of 20 |
| Constipation | Gastrointestinal disorders | 2 of 20 |
| Hemoglobin decrease | Blood and lymphatic system disorders | 2 of 20 |
Serious events with the highest arm-level rate: abdominal pain (100.0%), death-disease progression (10.0%), dyspnea (10.0%), ast, sgot (5.0%), arrhythmia (5.0%).
For the treatment group, the registry record lists 20 participants at risk for serious adverse events and 12 affected. It also lists 20 at risk for other adverse events and 16 affected. These are group-level counts in the registry’s safety record. They do not indicate how many events each participant experienced, and the record does not provide a comparison with a control group.
The registry lists these serious adverse event terms and percentages: Abdominal pain, 100.0%; Death-Disease Progression, 10.0%; Dyspnea, 10.0%; AST, SGOT, 5.0%; Arrhythmia, 5.0%; and Cardiac disorder, 5.0%. The denominator or calculation basis for these term percentages is not reported in the registry record. They should therefore be read as the percentages recorded for the listed terms, not converted into participant counts or treated as percentages of all enrolled participants.
The deaths field is not populated with a count, so deaths are not reported in the registry record. The listed term “Death-Disease Progression” is an adverse-event term and does not, by itself, establish the total number of deaths. Serious and other adverse-event categories describe reported safety events; they do not establish that the study treatment caused them.
12. What the Result Does, and Does Not, Mean
The record documents a completed phase II study of romidepsin in a single group of 20 participants with radioiodine-refractory thyroid cancer. It specifies the treatment schedule and defines a primary tumor endpoint that includes stable disease. These registry entries describe what was studied and what the primary outcome was intended to count.
They do not establish the primary outcome. The actual number meeting the endpoint, a response estimate, and an uncertainty interval are not reported in the registry record. Consequently, the posted information does not quantify the tumor result or its precision. Neither the study’s phase nor its completed status can substitute for a reported endpoint result.
The single-group design also limits interpretation. Even if a group-level outcome were available, this design would describe observations among participants treated in the study; it would not provide a randomized comparison against another treatment. The record does not report a comparison group, a treatment-effect estimate, or a statistical analysis supporting a comparative claim.
The endpoint includes stable disease by its stated definition. It should not be described as a count of participants whose tumors shrank unless separate data support that description. The record does not report separate response categories, assessment details beyond the named RECIST criteria, or rules for missing assessments.
The safety record gives counts of participants affected by serious and other adverse events, plus a list of serious-event terms with percentages. These data are descriptive and do not by themselves establish cause. The deaths field has no reported count. In short, the registry record supports a description of study design, endpoint intent, and recorded safety summaries, but not a conclusion about the magnitude or comparative meaning of the primary tumor outcome.
13. Why This Trial Matters Statistically
This trial is statistically informative as an example of why a clearly named endpoint and a completed study are not enough to evaluate an outcome. The registry record identifies what was intended to be counted, the assessment period, the treatment group, and the enrollment. But it does not report the primary count or an analysis. Without those results, readers cannot assess the endpoint’s observed frequency or precision.
The single-group design is also central to interpretation. A description of outcomes among treated participants is different from a comparison of outcomes between randomized groups. Keeping that distinction clear prevents a within-group observation from being read as a controlled estimate of treatment effect. The record identifies no comparator and no masking.
The endpoint wording matters as well. Because stable disease is expressly included, the intended outcome is broader than tumor shrinkage alone. Statistical summaries are meaningful only when their definitions are retained; changing the endpoint’s meaning would misrepresent what the registry says was measured.
Finally, the safety counts illustrate the difference between reporting events and establishing their cause. Serious and other adverse events are listed for the treatment group, while the record does not provide a comparative safety analysis. The percentages attached to individual serious-event terms also lack a stated calculation basis in the record. Together, these features show why transparent reporting of results, denominators, analysis populations, and methods is necessary for readers to judge what the data can support.
14. Limitations
- The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
- Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.
The primary limitation for statistical interpretation is that the primary endpoint result is not reported in the registry record. The record provides no count of participants meeting the endpoint, no response estimate, and no confidence interval. It therefore cannot support an assessment of the observed tumor outcome or its precision.
The single-group design has no comparator. Outcomes in the treatment group cannot be interpreted as a randomized difference from another treatment or a control condition. The record does not report a benchmark for comparison or a comparative analysis.
Important analysis details are also absent. The registry record does not report the statistical analysis plan, the analysis population, the handling of missing or unevaluable tumor assessments, or the methods used to calculate uncertainty. It names RECIST but does not provide response-assessment details or separate counts for the categories included in the endpoint. Multiplicity procedures and interim analyses are not reported in the registry record.
The safety information has limits too. Counts are recorded for serious and other adverse events, but there is no comparator group or causal analysis. The calculation basis for the listed serious-event percentages is not specified, and the deaths field does not report a count. The listed Death-Disease Progression term should not be substituted for a total death count.
Finally, the planning assumptions describe a hypothetical two-arm sample-size calculation, while the trial itself is single group. The record does not report the trial’s own sample-size justification. Those illustrative assumptions cannot fill the gaps in the observed endpoint or analysis.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Romidepsin in Treating Patients With Recurrent and/or Metastatic Thyroid Cancer That Has Not Responded to Radioactive Iodine — NCT00098813.
- Registry record last updated 2014-05-28; status last verified 2013-10.
- Results first posted 2013-12-19.
- 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.
Continue through the Clinical Biostats statistical pathway
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18. Record Summary
The registry record describes a completed phase II, single-group study of romidepsin for recurrent and/or metastatic radioiodine-refractory non-medullary thyroid cancer. It records enrollment of 20, no masking, and treatment by intravenous infusion over 4 hours on days 1, 8, and 15, repeated every 28 days for up to 12 courses if unacceptable toxicity or disease progression did not occur.
The primary endpoint was the number of participants with tumor major response, including stable disease, by RECIST criteria, from treatment start to 8 weeks. Its observed result and statistical analysis are not reported in the registry record. The safety record lists 12 of 20 participants affected by serious adverse events and 16 of 20 affected by other adverse events; the deaths field has no reported count.