CUDC-907 Treatment in People With Metastatic and Locally Advanced Thyroid Cancer: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for CUDC-907 Treatment in People With Metastatic and Locally Advanced Thyroid Cancer (NCT03002623), a na phase 2 study. The primary outcome is Number of Participants With a Clinical Response (Complete Response (CR) + Partial Response (PR)) Assessed by the Response Evaluation Criteria in Solid Tumors (RECIST) v1.1.

2. Clinical Question

This question concerns tumor response during treatment, rather than a comparison of CUDC-907 against another treatment. The registry describes a population with advanced thyroid cancer and defines response using changes in target lesions and lymph nodes. Complete response requires disappearance of all target lesions, with pathological lymph nodes reduced to a short axis below 10 mm. Partial response requires at least a 30% decrease in the sum of target-lesion diameters from baseline. The endpoint combines these two response categories into a participant count.

The trial record lists enrollment of 7 and indicates that the study was terminated. Those details describe the recorded study, but they do not establish how many participants were evaluable for response or why the study ended. The reason for termination and the number assessed at the primary endpoint are not reported in the registry record. Without the observed response count and the population used for that assessment, the central clinical question cannot be answered from the posted results.

3. Trial Design

This was a phase II, single-group trial sponsored by the National Cancer Institute. The registry lists enrollment of 7. Allocation is recorded as not applicable, and masking as none, consistent with a design that does not describe a randomized, masked comparison. The listed study group received CUDC-907 for thyroid cancer.

The registry gives a start date of 2016-12-22 and a primary completion date of 2018-02-12. Its status is terminated. The reason for termination is not reported in the registry record. Nor does the record explain whether enrollment, follow-up, or other study activities differed from the intended plan.

The intervention description says that participants took CUDC-907 orally in tablet form for 5 days, then had 2 days off, repeating this schedule each week. Screening included medical history, physical examination, blood and urine tests, an electrocardiogram, symptom and activity review, imaging, and tumor tissue assessment. The description says a prior surgical sample could be used, with biopsy considered if such a sample was unavailable. Study visits during treatment repeated screening tests. The follow-up description in the registry summary is incomplete, so the planned follow-up procedures cannot be fully characterized.

The primary endpoint was a count of participants with complete or partial response by RECIST v1.1, assessed over approximately 6 months. Listed secondary outcomes include time to progression, survival after therapy, tumor-marker correlations with progression-free time, and serious and non-serious adverse events. These are planned or registered outcome topics; numerical results for them are not reported in the registry record. A statistical analysis plan, including the approach to missing observations and analysis populations, is not reported in the registry record.

Because the study has a single group, its response findings would describe outcomes among participants treated in the trial. They would not, by themselves, estimate a causal difference relative to an untreated or alternative-treatment group. No like-for-like control comparison is described.

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:

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 Number of Participants With a Clinical Response (Complete Response (CR) + Partial Response (PR)) Assessed by the Response Evaluation Criteria in Solid Tumors (RECIST) v1.1, measured over Approximately 6 months.

The primary endpoint counts participants whose best recorded tumor response met the registry definition of complete response or partial response under RECIST v1.1. A complete response requires disappearance of all target lesions. Any pathological lymph nodes, whether target or non-target, must have a short-axis measurement below 10 mm. A partial response requires at least a 30% decrease in the sum of target-lesion diameters, using the baseline sum as the reference.

This endpoint is used to describe measurable tumor change during treatment. Combining complete and partial response gives a count for participants meeting either response category, rather than separate counts for each category. It is a binary classification at the participant level: each participant either meets the response definition or does not, provided the necessary assessment is available. The registry gives the timeframe as approximately 6 months.

The endpoint definition is not itself a result. The registry record does not report the number who achieved complete response, the number who achieved partial response, or the combined response count. It also does not report the number assessed, how missing or unevaluable assessments were handled, or a confidence interval for the response count or rate. Consequently, the endpoint's clinical meaning can be described, but its observed outcome and precision cannot be evaluated from the posted results.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What does the primary count represent?

The primary endpoint is a participant count, not a measurement of the amount of tumor change averaged across participants. Each person's assessment is classified according to RECIST v1.1. The registered response definition groups complete response and partial response together. The result would ordinarily be reported as the count meeting that definition, with the number assessed made clear. The registry record does not report the observed count or the assessment denominator.

In general, a count endpoint can also be expressed as a proportion when the relevant denominator is known. A proportion makes the frequency easier to compare across groups of different sizes, but it does not remove uncertainty, and it depends on a clear definition of who was assessed. No response proportion is reported in the registry record, and none can be reconstructed from enrollment alone because the number assessed for the primary endpoint is not reported.

What would an uncertainty interval add?

In general, a confidence interval describes the range of effect values reasonably compatible with the observed data under the assumptions of the analysis. For a response proportion, an interval would help show how precisely the trial estimates response in its study population. A narrow interval would indicate greater precision than a wide interval, though neither would address all concerns about study design or generalizability. No confidence interval for the primary endpoint is reported in the registry record.

Uncertainty is especially important when a study has limited enrollment. A response count can change substantially in proportional terms when only a small number of assessments inform it. This is a general statistical consideration, not a claim about the unreported response result in this trial. The record does not state a sample-size justification or a target precision for the endpoint.

Why does the single-group design constrain inference?

In general, an observed outcome in a single treated group is descriptive. It does not directly show what would have happened to comparable participants under a different treatment or no study treatment. A randomized concurrent comparison can support a treatment contrast by balancing groups through allocation, subject to the conduct and analysis of the trial. This record describes no randomized control group: allocation is not applicable, masking is none, and the model is single group. Thus any reported response count would characterize the study group rather than establish a comparative effect.

Masking and allocation are separate design features. Allocation concerns how participants are assigned among groups; masking concerns whether treatment assignment is concealed from participants or study personnel. With no listed control group, allocation is not applicable in this record. The registry lists masking as none. The record does not detail whether response assessments were independently reviewed or otherwise protected from knowledge of treatment, so those procedures are not reported in the registry record.

What do the registered outcomes imply about the analysis?

The primary outcome uses RECIST v1.1 and a timeframe of approximately 6 months. The registry also lists outcomes for progression-free time and survival after therapy, as well as correlations between tumor features and progression-free time. Time-to-event outcomes generally account for both whether an event occurred and how long participants were observed before the event or censoring. In general, censoring occurs when an event time is not observed during available follow-up; interpretation depends on the assumptions and follow-up process. The registry record does not provide numerical analyses, censoring details, or methods for these outcomes.

The tumor-feature outcomes concern associations, not necessarily causal effects. In general, a correlation can describe whether two measured characteristics vary together, but it does not establish that one causes the other. The record does not report the measures, estimates, or analysis methods for the listed correlations.

The registry lists serious and non-serious adverse-event counts. Event counts can describe how many participants experienced events, but they do not by themselves indicate event rates over time, severity distributions beyond the registry's categories, or causal attribution. The record does not describe exposure-adjusted methods or adjudication procedures.

What analysis details are absent?

A statistical analysis plan is not reported in the registry record. The record does not describe the intended analysis population, handling of missing response assessments, rules for participants who stopped treatment, adjustment for multiple outcomes, or planned interim analyses. It also does not report estimates or tests for the primary endpoint. These omissions mean the posted information supports a description of the endpoint and design, but not a reconstruction of the trial's statistical analysis.

10. Multiplicity, Missing Data and Analysis Populations

The registry lists 7 outcome measures, including a primary response endpoint and secondary outcomes addressing progression-free time, survival after therapy, tumor-feature correlations, and adverse events. When a study evaluates multiple outcomes or multiple associations, the chance of obtaining an apparently notable result by chance can increase if each is tested separately. In general, a multiplicity strategy may prioritize an endpoint, adjust inference, or distinguish confirmatory from exploratory questions. A multiplicity plan is not reported in the registry record, so it is not possible to determine how these outcomes were intended to be interpreted together.

Missing data are also important for the response endpoint. A participant without an evaluable tumor assessment cannot automatically be classified as a responder or non-responder without an analysis rule. In general, different handling choices can lead to different estimates, particularly when missingness is related to disease status, tolerability, or follow-up. The registry record does not report the number of missing or unevaluable assessments, the reasons for missingness, or the rule used to handle them.

The analysis populations are not described. The record does not state which participants were included in the primary analysis or whether all enrolled participants were assessed according to a defined population. Enrollment is listed as 7, but it does not identify the endpoint denominator. Details about treatment exposure, withdrawals, and the disposition of participants are not reported in the registry record.

The record also does not report an interim-analysis plan, stopping rules, or a statistical analysis plan. The terminated status alone does not explain why the trial ended or establish how termination affected outcome availability. These gaps limit interpretation of both the primary endpoint and the secondary 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: death (50.0%).

The registry reports adverse-event counts by cancer group. For anaplastic thyroid cancer, 4 participants were listed as at risk for serious adverse events and 2 as affected. For poorly differentiated or variants of thyroid cancer, 3 were listed as at risk and 1 as affected. The corresponding non-serious-event figures are 4 at risk and 4 affected for anaplastic thyroid cancer, and 3 at risk and 3 affected for poorly differentiated or variants of thyroid cancer.

The registry lists deaths of 2 in the anaplastic thyroid cancer group and 1 in the poorly differentiated or variants group. It also lists Death as a top serious-event term at 50.0%. These are registry-recorded figures. The record does not explain whether the death counts overlap with the serious-event counts, provide a causal assessment, or clarify the denominator behind the top-term percentage. They should not be interpreted as evidence that CUDC-907 caused the deaths.

In general, an adverse-event count indicates that an event was recorded, not that the study treatment caused it. Counts also do not convey timing, duration, severity details, or comparative risk without additional information. The registry record does not report those details or a control-group safety comparison. The listed figures therefore describe the reported safety counts but do not establish a treatment-attributable safety effect.

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

The registry record establishes that a phase II, single-group study of CUDC-907 was conducted in people with advanced thyroid cancer and registered a RECIST-based clinical response endpoint. It also provides the endpoint definition, the approximate assessment timeframe, enrollment, study dates, termination status, and selected adverse-event counts. These registry entries describe the study's intended question and some recorded characteristics.

The record does not report the primary response count, the number assessed, or an uncertainty interval. It therefore cannot show how many participants met the response definition or how precisely that outcome was estimated. The absence of a reported result is not evidence that there were no responses; it means the registry record does not make the outcome available for interpretation.

Even if the response result were available, a single-group study would describe outcomes in its treated group rather than provide a direct randomized treatment comparison. Differences between observed outcomes and outcomes under another treatment cannot be estimated from a control group that is not part of the registered design. Generalizing beyond the participants studied would also require consideration of eligibility, enrollment, follow-up, and the completeness of outcome assessment.

The listed adverse-event counts likewise need cautious interpretation. They are counts by cancer group, with no control-group comparison or causal attribution reported. The registry's terminated status and enrollment figure do not resolve the missing outcome information, and the reason for termination is not reported. The record is thus informative about design and registered endpoints, but insufficient to establish the magnitude, precision, or comparative meaning of the primary outcome.

13. Why This Trial Matters Statistically

This trial illustrates why a registered endpoint and a reported endpoint result are different kinds of information. The RECIST v1.1 definition is specific: it identifies the tumor changes counted as complete or partial response. But without the observed count and the number assessed, the primary question remains unanswered by the registry record. Statistical interpretation depends on both a clear outcome definition and accessible results.

The single-group design is also important. A descriptive response outcome can characterize what was observed among trial participants, but it cannot by itself estimate how outcomes would differ under an alternative treatment. This distinction helps prevent a response count, if reported, from being mistaken for a comparative treatment effect.

The record also demonstrates why denominators and analysis populations matter. Enrollment is listed as 7, but the registry does not say how many participants had an evaluable primary assessment. Without that information, readers cannot tell how the response count relates to the enrolled group or how missing assessments were treated. Precision cannot be judged in the absence of a result and its uncertainty estimate.

Finally, the termination status and adverse-event figures require context. The record does not give a reason for termination or causal assessments for listed events. Statistical reasoning cannot fill those gaps by inference. Separating recorded details from unanswered questions is essential when interpreting a trial whose design is documented more fully than its outcomes.

14. Limitations

• The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
• Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.

The main limitation for interpretation is that the registry record does not report the primary clinical response result. It gives neither the count meeting the RECIST definition nor the number of participants assessed. No confidence interval or other measure of uncertainty is reported. As a result, the primary endpoint cannot be quantitatively evaluated from this record.

Enrollment is listed as 7, but the number enrolled is not necessarily the number treated or assessed for response. The participant disposition, analysis population, missing assessments, and reasons for missingness are not reported in the registry record. These omissions prevent evaluation of whether the primary result, if available elsewhere, would represent all enrolled participants or a selected subset.

The single-group design has no described randomized control group, so it cannot provide a direct comparative estimate. The study is marked terminated, but the reason and its implications for outcome availability are not reported. The start and primary completion dates are recorded, but they do not explain the conduct or completeness of follow-up.

The endpoint is described with a timeframe of approximately 6 months, while details of response assessment procedures and review are not reported. The registry summary's follow-up description is incomplete. A statistical analysis plan, multiplicity approach, missing-data method, interim-analysis plan, and sample-size justification are not reported in the registry record.

Safety reporting is limited to selected counts by cancer group and a top serious-event term. The record does not provide a control comparison, detailed event timing, or causal attribution. These limitations mean the posted information supports a careful description of the design and some event counts, but not a comprehensive efficacy or safety assessment.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: CUDC-907 Treatment in People With Metastatic and Locally Advanced Thyroid Cancer — NCT03002623.
• Registry record last updated 2018-12-21; status last verified 2018-12.
• Results first posted 2018-12-21.
• 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

NCT03002623 is a phase II, single-group trial of CUDC-907 in people with metastatic and locally advanced thyroid cancer, sponsored by the National Cancer Institute. The registry lists enrollment of 7, a start date of 2016-12-22, a primary completion date of 2018-02-12, and terminated status. The intervention was oral CUDC-907 on a weekly schedule of 5 treatment days followed by 2 days off.

The primary endpoint was the number of participants with complete or partial response by RECIST v1.1 over approximately 6 months. The registry record does not report the primary response result, the number assessed, or an uncertainty estimate. Secondary outcomes include progression-free time, survival after therapy, tumor-feature associations, and adverse events. Selected adverse-event counts are listed by cancer group, but causal attribution and a control comparison are not reported. The reason for termination, analysis populations, missing-data procedures, and statistical analysis plan are not reported in the registry record.