A Prospective, Open-label Study of [68Ga]Ga-DOTA-TATE in Patients With Neuroendocrine Neoplasms (NENs) and Healthy Volunteers in Japan: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for A Prospective, Open-label Study of [68Ga]Ga-DOTA-TATE in Patients With Neuroendocrine Neoplasms (NENs) and Healthy Volunteers in Japan (NCT06240741), a na phase 3 study. The primary outcome is Number of [68Ga]Ga-DOTA-TATE Positive Participants (TP Participants) Among CIM Positive Participants (TP or FN Participants).

2. Clinical Question

3. Trial Design

The registry describes a prospective, open-label, multicenter, single-arm, Phase III study of [68Ga]Ga-DOTA-TATE for diagnosis in patients with NENs and healthy volunteers in Japan. Allocation is listed as not applicable, masking as none, and the model as single group. These design features matter because the central question is diagnostic classification against CIM, rather than a comparison of assigned treatments. CIM is described as the standard of truth for the diagnostic assessment.

The study record lists an enrollment of 71 and a status of completed. The recorded start date is 2024-03-21, and the primary completion date is 2024-12-27. The intervention description specifies intravenous [68Ga]Ga-DOTA-TATE, with a weight-based dose and a maximum total dose. A 68Ge/68Ga Generator is also listed as an intervention; the registry record does not give further detail about its role in the study.

For patients with confirmed or suspected NENs, the efficacy population description requires results from both PET/CT and CIM. This pairing allows each participant's PET/CT classification to be compared with the reference classification. The description also includes healthy volunteers, for whom PET/CT results are required, but the rest of the eligibility wording is incomplete in the registry record. The record does not report how participants were recruited, the number enrolled in each group, or how many met the efficacy criteria.

Open-label status means masking is listed as absent; however, the record does describe central reads for imaging classifications. It does not report whether readers were masked to other information or to one another's interpretations. The record also does not report a randomized comparison, so the study should not be interpreted as estimating a comparative treatment effect between independent arms.

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.

Before assignment: Therefore, efficacy results related to the diagnostic performance are presented as a single combined population of NEN patients and HVs. Baseline characteristics, other efficacy (e.g. number of lesions), safety, and PK results are presented separately due to the differences between the NEN patient and HV populations. These approaches are as per the protocol and the statistical analysis plan.

Overall Study

Analysis population for the primary outcome, as defined in the registry: The Efficacy Analysis Set (EFF) comprised all enrolled participants who were administered with \[68Ga\]Ga-DOTA-TATE. Patients with confirmed/suspected NENs had to have both results of \[68Ga\]Ga-DOTA-TATE PET/CT imaging and CIM. HVs had to have results of \[68Ga\]Ga-DOTA-TATE PET/CT imaging, with a valid measurement without a protocol deviation with impact, and who are CIM positive participants (TP or FN participants).

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 [68Ga]Ga-DOTA-TATE Positive Participants (TP Participants) Among CIM Positive Participants (TP or FN Participants), measured over Day 1.

The primary endpoint is the number of [68Ga]Ga-DOTA-TATE-positive participants among participants classified as CIM-positive. The registry defines subject-level sensitivity as the proportion of PET/CT-positive participants, termed true positive (TP), among CIM-positive participants, termed TP or false negative (FN). A TP participant had at least one lesion identified on both PET/CT and CIM by central read. An FN participant had no lesions identified on PET/CT but at least one lesion identified on CIM by central read.

Sensitivity is used to describe how often the test under evaluation identifies participants who are positive according to the reference method. It is a measure of diagnostic agreement for the reference-positive group; it is not, by itself, a measure of the probability that a positive test corresponds to disease in an individual. The registry also lists related outcomes, including subject-level positive predictive value, negative predictive value, accuracy, and region-level measures. These address different aspects or units of diagnostic performance and should not be treated as interchangeable with the primary endpoint.

The primary timeframe is Day 1. The registry record does not report the observed TP count, the number of CIM-positive participants, or the resulting sensitivity estimate. It also does not report an uncertainty interval or a statistical test for this endpoint.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What does sensitivity mean in this study?

In general, sensitivity describes the share of reference-positive cases that a test also classifies as positive. For this study, the reference classification is CIM, and the test under evaluation is [68Ga]Ga-DOTA-TATE PET/CT. The registry calls participants positive on both methods true positive (TP), and participants negative on PET/CT but positive on CIM false negative (FN). The primary endpoint counts TP participants among those classified as TP or FN by CIM. This definition makes the denominator dependent on the number of participants with positive CIM results and the numerator dependent on their PET/CT classifications.

The endpoint is at the participant level. The registry separately lists region-level outcomes, which concern a different unit of analysis. Participant-level sensitivity and region-level sensitivity answer related but distinct questions. A region-based analysis may account for multiple anatomical regions within a participant, whereas a participant-based analysis gives each participant a classification. The record does not provide the detailed region definitions or the statistical handling of multiple regions.

Why is a reference method important?

In general, diagnostic accuracy measures need a rule for deciding which cases count as positive or negative. Here, CIM is stated to be the standard of truth. PET/CT results are compared with CIM classifications, and that comparison defines the primary endpoint. The interpretation is therefore conditional on the reference method and its application. If the reference classification is uncertain or differs from the underlying disease state, a measure calculated against it describes agreement with that reference rather than an error-free account of disease.

The registry specifies that TP and FN classifications are based on whether lesions were seen by central read. It does not report the detailed reading procedures, reader masking, adjudication of disagreements, or criteria for determining lesion status beyond those definitions. Those details would help readers assess how reproducible the classifications might be and whether knowledge of one imaging result could influence another reading.

What can counts tell the reader?

A count is the number of participants assigned to a defined classification. The primary outcome is explicitly a count, while sensitivity is its proportion-based interpretation using the CIM-positive group as the denominator. Counts are useful because they show the underlying number of classified participants, but a count alone does not describe performance without its denominator. Conversely, a proportion without its underlying counts may conceal how much information supports the estimate.

General statistical interpretation also considers uncertainty around an estimate. A confidence interval, when reported, describes a range of values compatible with the data under the method's assumptions; it is not a guarantee that the true value lies within that particular interval. No confidence interval or other uncertainty measure for the primary endpoint is reported in the registry record. Without the event count, denominator, and uncertainty measure, the precision of the primary finding cannot be evaluated.

How do the secondary diagnostic measures differ?

In general, positive predictive value describes the share of test-positive classifications that are also positive by the reference method, while negative predictive value describes the share of test-negative classifications that are also negative by the reference method. Accuracy describes the share of classifications that agree with the reference across positive and negative cases. Specificity concerns the reference-negative group and the frequency with which the test also classifies that group as negative. These measures use different subsets or combinations of classifications, so none is a substitute for sensitivity.

The registry lists subject-level PPV, NPV, and accuracy, as well as region-level sensitivity, specificity, and PPV. It also lists counts of participants with concordant positive or negative results. These measures can provide complementary descriptions, but their interpretation depends on the eligible analysis population and the number of reference-positive and reference-negative cases. The registry record does not report the results, denominators, or uncertainty for these secondary measures.

What does the record say about the analysis plan?

The registry record lists 31 outcome measures and 0 analyses. It does not report the statistical analysis methods for the primary or secondary outcomes, nor does it state whether estimates were adjusted for any participant characteristics. The record does not report a hypothesis-testing procedure, confidence-interval method, or prespecified handling of multiple endpoints. It also does not report an interim analysis plan. These omissions mean that readers cannot compare a stated analysis plan with the posted results or evaluate whether particular analytic choices were prespecified.

In general, analysis methods should match the outcome's structure and the sampling unit. For a diagnostic count and proportion, it is important to identify the analysis population, the numerator and denominator, and the method used to quantify uncertainty. If observations are grouped within participants, as may be relevant to region-level outcomes, statistical dependence can affect uncertainty estimates. The registry record does not describe whether or how such dependence was addressed, so no specific method should be assumed.

General explanations of over-dispersion or other model assumptions are not evidence that such issues arose in this study. No model details are reported, and the endpoint is described as a participant count and proportion rather than as a reported model estimate. The absence of analysis results should not be filled in by applying an assumed method to the enrollment total or to the safety counts.

10. Multiplicity, Missing Data and Analysis Populations

The registry lists 31 outcome measures, including a primary diagnostic endpoint and a range of secondary participant-level and region-level measures. When a study assesses multiple outcomes, general statistical practice distinguishes the primary question from additional or exploratory questions and considers how repeated testing may affect interpretation. The registry record does not report a multiplicity strategy, such as a prespecified adjustment or a hierarchy for interpreting secondary outcomes. It is therefore not possible to determine from the record how the family of outcomes was intended to be handled inferentially.

The record lists 0 analyses and does not post results for the primary or secondary diagnostic outcomes. It does not report a missing-data method, rules for excluding participants with incomplete imaging, or the number of participants with missing CIM or PET/CT classifications. The efficacy population description requires paired imaging results for patients with confirmed or suspected NENs, but eligibility rules alone do not show how many participants fulfilled those requirements or what happened to incomplete records.

The record states that the Efficacy Analysis Set comprised enrolled participants who received [68Ga]Ga-DOTA-TATE, with additional imaging requirements for patients and healthy volunteers. The healthy-volunteer description is incomplete in the registry record. The number analyzed in each group and any deviations from the stated population are not reported. Without those details, the relationship between enrollment and the analysis denominator cannot be assessed.

In general, missing data can affect diagnostic estimates if participants with incomplete or unevaluable results differ from those with complete results. That is a general methodological concern, not a finding about this study. The registry record does not report the extent, reasons, or handling of missing observations, so the direction or size of any potential effect cannot be judged.

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: intra-abdominal haemorrhage (2.1%), vomiting (2.1%).

The safety record gives separate counts for participants with serious adverse events and other adverse events in the confirmed or suspected NEN group and the healthy-volunteer group. In the NEN group, 2 of 48 participants are recorded with serious adverse events and 8 of 48 with other adverse events. In the healthy-volunteer group, 0 of 23 are recorded with serious adverse events and 2 of 23 with other adverse events. The record reports 0 deaths in each group.

The listed top serious adverse event terms are Intra-abdominal haemorrhage and Vomiting, each reported as 2.1%. These terms and percentages are registry safety entries; the record does not provide enough context to infer causality or to determine whether a term occurred in isolation or alongside other events. Serious adverse-event counts should also be kept distinct from the broader other-adverse-event counts.

These figures are descriptive and should not be read as a randomized comparison. The study is single-arm, and the patient and healthy-volunteer groups are not like-for-like controls. The registry record does not report detailed event narratives, timing, severity gradations, exposure-adjusted rates, or an attribution assessment for these entries. The counts therefore describe what is recorded for the study groups, without establishing a comparative safety effect or a causal relationship to the imaging agent.

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

The study's primary question is whether [68Ga]Ga-DOTA-TATE PET/CT classifies as positive those participants whom CIM classifies as positive. That is a diagnostic sensitivity question, not a question about whether a treatment improves a health outcome. The registry record defines the endpoint and the reference classification but does not report the result. As a result, the posted information does not establish the observed sensitivity, the number of missed CIM-positive participants, or how precisely the endpoint was estimated.

The record also does not report numerical results for the listed secondary diagnostic measures. Their presence in the outcome list does not demonstrate that PET/CT results agreed with CIM at a particular level, nor does it show performance across regions. Nor can a diagnostic comparison with CIM by itself establish that every classification corresponds to the underlying disease state. CIM is the study's stated standard of truth, and interpretation is tied to that choice of reference.

The enrollment of 71 and the completion status describe the study record, but enrollment does not reveal the number of participants included in each endpoint analysis. The efficacy criteria require imaging results, and the record does not report the final analysis denominators or missing-data handling. Safety counts are available, but they do not supply the absent diagnostic results or establish a causal safety relationship.

In general, diagnostic estimates are most informative when reported with the relevant counts, denominators, and uncertainty measures, along with enough methods detail to understand how classifications were made. Those results and details are not reported in the registry record. The appropriate interpretation is therefore limited: the study was designed to evaluate diagnostic performance against CIM, while the posted record does not allow a conclusion about the observed performance or its precision.

13. Why This Trial Matters Statistically

This study is statistically relevant because it frames the evaluation of an imaging test as a diagnostic classification problem. Rather than comparing assigned treatments, it compares PET/CT findings with CIM, the stated standard of truth. The distinction determines what the primary measure means: subject-level sensitivity describes classification among CIM-positive participants, rather than a treatment effect or a general measure of agreement across all participants.

The study also lists outcomes at more than one level. Participant-level outcomes summarize classifications for individuals, while region-level outcomes address diagnostic performance by region. In general, those units can carry different statistical dependencies and answer different questions. Clear definitions of the unit of analysis, reader procedures, and handling of repeated regions are important for interpreting estimates. The registry record lists these outcome types but does not provide their results or detailed analysis methods.

The inclusion of healthy volunteers alongside patients adds another population to the study description, but does not make the design a randomized controlled comparison. The groups have distinct roles in the stated diagnostic study, and the record does not report group-specific efficacy results or fully specify the healthy-volunteer analysis criteria. Treating the groups as equivalent comparison arms would therefore misrepresent the design.

Finally, the gap between the detailed endpoint definitions and the absence of posted analysis results is important for evidence interpretation. A study can describe what it intends to measure without publishing the estimate needed to assess the magnitude or precision of that measure. Here, the registry record identifies the primary diagnostic question and lists multiple related outcomes, but the outcome findings and analysis methods needed to evaluate them are 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.

The principal limitation for interpretation is that the registry record does not report the primary endpoint result. It gives no TP count, CIM-positive denominator, sensitivity estimate, or uncertainty measure. Although multiple secondary diagnostic outcomes are listed, their results are also not reported. The diagnostic performance of [68Ga]Ga-DOTA-TATE PET/CT therefore cannot be assessed from the posted record.

The record is open-label and lists masking as none. It notes central reads for the lesion classifications used to define TP and FN, but does not report reader masking, how disagreement was handled, or the detailed reading procedures. The extent to which these processes could influence classification cannot be assessed from the available description.

The efficacy population description is incomplete for healthy volunteers, and the record does not give the number of participants analyzed in each group. It also does not report the number of missing or unevaluable imaging results, reasons for missingness, exclusions, or the method for handling incomplete data. These omissions limit understanding of how the enrolled group relates to the endpoint analysis population.

The registry record does not report statistical methods for the diagnostic endpoints, the approach to uncertainty estimation, adjustment for multiple outcomes, or an interim-analysis plan. It lists 31 outcome measures but 0 analyses. Region-level outcomes also appear among the listed measures, while the handling of potentially related observations is not described. The general statistical consequences of these omissions cannot be quantified for this study.

The single-arm design is appropriate to distinguish from a randomized comparison, but it does not provide a randomized treatment contrast. CIM is the stated reference, so diagnostic estimates would reflect agreement with that method rather than validation against an independently described error-free standard. Finally, the safety entries are limited descriptive counts without detailed event context or causal attribution.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: A Prospective, Open-label Study of [68Ga]Ga-DOTA-TATE in Patients With Neuroendocrine Neoplasms (NENs) and Healthy Volunteers in Japan — NCT06240741.
• Registry record last updated 2026-02-03; status last verified 2026-01.
• Results first posted 2026-02-03.
• 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

NCT06240741 is a completed, prospective, open-label, multicenter, single-arm, Phase III study of [68Ga]Ga-DOTA-TATE PET/CT in patients with NENs and healthy volunteers in Japan. The record lists an enrollment of 71, a start date of 2024-03-21, and a primary completion date of 2024-12-27. CIM is identified as the standard of truth for the primary diagnostic assessment.

The primary endpoint is the count of PET/CT-positive participants among CIM-positive participants, corresponding to subject-level sensitivity. The registry record defines the classification but does not report the endpoint result or results for the listed secondary diagnostic outcomes. It lists 31 outcome measures and 0 analyses. Safety counts are reported by study group, including serious adverse events, other adverse events, and deaths. The absence of posted diagnostic estimates and statistical methods limits interpretation of the study's diagnostic findings.