Imaging in MGUS, SMM and MM: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Imaging in MGUS, SMM and MM (NCT01237054), a na phase 2 study. The primary outcome is Count of Participants With Positive and Negative 18F-FDG PET CT and F18-NaF PET CT Imaging Results in Individuals With MGUS, SMM, and MM..

2. Clinical Question

The study’s broader stated objectives were to compare three imaging techniques and to relate imaging information to established clinical markers of progression from MGUS or SMM to MM. These objectives reflect an exploratory imaging question: whether different modalities identify findings that may be relevant across the diagnostic groups. They do not, by themselves, establish how well an imaging result predicts future progression. The record does not report whether the planned correlations or group comparisons were completed, nor does it provide estimates of diagnostic accuracy or prediction.

3. Trial Design

The registry describes a completed phase 2 pilot study with a single-group design and an enrollment of 31 participants. Eligible participants were adults diagnosed with MGUS, SMM, or MM. The record lists the National Cancer Institute as sponsor. It records a start date of 2010-10-17 and a primary completion date of 2011-08-09.

Participants were to undergo screening and provide baseline blood, urine, and bone marrow samples before imaging. The imaging protocol described three studies on separate days: standard 18F-FDG PET/CT, 18-NaF PET/CT using an experimental sodium fluoride-based drug, and DCE-MRI. The primary endpoint focuses on comparisons between the two PET/CT modalities, while the stated study objectives also cover comparison of all three modalities and correlations with clinical markers.

There was no random allocation, and masking is listed as none. This is therefore not a randomized trial of one imaging strategy against another, nor a blinded comparison against a separate control arm. Participants received the imaging procedures within one study group. Comparisons of modalities should be understood in that context: the design can describe observed imaging findings in the enrolled participants, but it does not isolate a treatment effect or establish that one modality is superior.

The primary outcome timeframe is 60 days. The registry description defines positive and negative imaging findings using modality-specific criteria: for NaF, a lesion was considered positive when focal uptake corresponded to a lesion identified on CT, and the FDG positivity criteria included focal abnormal uptake more intense than background, following parameters attributed in the registry to Zamagni et al. The registry record does not report the detailed analysis plan, participant flow, diagnosis-specific enrollment, or the number with evaluable scans. It also does not report a sample-size justification for the original study.

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 Count of Participants With Positive and Negative 18F-FDG PET CT and F18-NaF PET CT Imaging Results in Individuals With MGUS, SMM, and MM., measured over 60 days.

The primary endpoint counts participants with positive and negative 18F-FDG PET/CT and 18F-NaF PET/CT imaging results among people with MGUS, SMM, and MM. It is a count outcome: the intended summary is how many participants fall into each reported imaging-result category. The endpoint is not a time-to-progression measure, a treatment response, or a direct measure of clinical benefit.

The registry description gives operational criteria for classifying findings. For NaF, a lesion was considered positive when focal uptake corresponded to a lesion identified on CT; a result was negative when no uptake was seen in lesions. For FDG, positivity included focal abnormal uptake more intense than background, using criteria attributed in the record to Zamagni et al. These definitions matter because a count depends on how a positive finding is classified. The record does not provide additional detail on adjudication, reader agreement, or how multiple lesions within one participant were reduced to a participant-level result.

Comparing positive and negative counts across imaging modalities can describe whether the modalities produced different classifications in this pilot sample. It may help characterize their use in participants across the three diagnostic groups and motivate later research. Counts alone do not establish sensitivity, specificity, predictive value, or agreement; those interpretations require suitable reference definitions and analysis details. The registry record does not report the endpoint counts, estimates of these diagnostic properties, or a statistical comparison of the modalities. Accordingly, the endpoint’s intended measurement can be described, but its observed findings cannot be summarized from the registry record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

Statistical methods depend on the type of outcome and the way participants contribute observations. Here, the primary endpoint is described as counts of participants with positive and negative imaging results. General statistical explanation: a count reports how many observations fall into a category; it does not, by itself, indicate how uncertain the count is or whether two modalities differ beyond the observed sample. The registry record does not report the counts or an inferential analysis for the primary endpoint.

What does a count comparison describe?

A positive or negative count summarizes the classification assigned to participants under the stated imaging criteria. When the same participants undergo more than one imaging procedure, the results are potentially paired: each participant may contribute a result from each modality. General statistical explanation: paired data preserve the within-person relationship between results. A comparison that treats each modality’s observations as if they came from unrelated groups may not represent that relationship. The registry record does not state which statistical method was used for the modality comparison, whether the analysis used paired participant-level data, or how missing scans were handled.

Counts can also be presented separately for MGUS, SMM, and MM. Such stratification would make the diagnostic groups visible, but the registry does not report the number of participants in each group or the group-specific endpoint findings. Without those details, it is not possible to assess how stable a group-specific result might be. General statistical explanation: sparse counts can make estimates imprecise, particularly when outcomes are divided across several categories. That possibility is relevant to pilot studies, but the available record does not establish whether any particular category was sparse.

What does a confidence interval contribute?

General statistical explanation: a confidence interval expresses uncertainty around an estimate under the assumptions of the statistical method. It is different from a range of observed participant values and does not guarantee that the true value lies within a particular interval. For a count or proportion, an interval can help readers assess whether the data are compatible with a broad range of underlying values. The registry record does not report confidence intervals for the primary endpoint, so the precision of the intended comparison cannot be evaluated.

A p-value, when used, addresses compatibility between the observed data and a specified statistical null model. It is not the probability that the null hypothesis is true, and it does not describe the size or clinical importance of a difference. The registry record does not report p-values or identify a primary inferential test. No conclusion about statistical significance can therefore be drawn from the endpoint description alone.

Why do the analysis population and missing data matter?

General statistical explanation: an analysis population defines which participants contribute to a particular estimate. A study may enroll participants who do not complete every procedure or whose results cannot be evaluated. If the number analyzed differs across modalities, the comparison can reflect both the imaging classifications and differences in who contributed data. The registry reports enrollment of 31, but does not report endpoint-specific denominators, scan completion, exclusions, or a missing-data method. The analysis population for the primary endpoint is therefore not reported in the registry record.

Missingness can affect interpretation when the reasons for unavailable results are related to participant characteristics, disease status, or the test result itself. General statistical explanation: excluding incomplete observations may be reasonable in some circumstances, but it can introduce bias when the excluded observations differ systematically from those retained. The record does not describe whether results were missing, why any were missing, or whether any method was used to address them. It is not possible to determine the direction or scale of any resulting effect.

How should multiple analyses be understood?

The registry lists seven secondary outcomes and ten analyses. General statistical explanation: when many outcomes or comparisons are examined, the chance of at least one apparently notable result can increase if each test is interpreted separately. Statistical approaches to multiplicity can control or contextualize that risk, but the appropriate approach depends on the prespecified questions and analysis plan. The registry record does not report whether a multiplicity adjustment was planned or used, which analyses were confirmatory, or how the primary and secondary questions were prioritized.

What do planning illustrations mean?

General statistical explanation: a sample-size calculation describes how much information a future study might need under stated assumptions about the effect, variability, allocation, and statistical threshold. It is a planning tool, not a finding from participants. Any planning illustration based on an observed effect is not observed power and should not be mistaken for the trial’s original design justification. The registry record does not report the original sample-size justification. It does not report enough primary endpoint results to assess the precision of the modality comparison from the record alone.

The registry identifies this study as a pilot, and its design is single-group rather than randomized. General statistical explanation: pilot studies often help characterize procedures and generate questions for later research; they are not automatically designed to provide definitive estimates. The registry does not state a formal pilot objective, a target precision, or a decision rule for proceeding to another study. Those details are not reported in the registry record.

10. Multiplicity, Missing Data and Analysis Populations

The registry lists seven secondary outcomes and ten analyses in addition to the primary endpoint. Multiple outcomes can be useful when a study explores different aspects of imaging and clinical markers, but each additional comparison creates another opportunity for a result to appear noteworthy by chance. General statistical explanation: multiplicity procedures can address this issue by adjusting how evidence is evaluated across a family of tests, or by clearly separating confirmatory questions from exploratory ones. The registry record does not report whether such an adjustment was used or how the analyses were grouped or prioritized.

The statistical analysis plan is not reported in the registry record. In particular, the record does not state the inferential tests, the prespecified hypotheses, whether the primary comparison was paired, or how estimates and uncertainty were to be reported. It also does not report the endpoint results, confidence intervals, or p-values. Consequently, the strength and precision of evidence for any observed difference cannot be assessed.

Enrollment is reported as 31 participants, but the record does not report the number with evaluable imaging results, the number analyzed for each outcome, or the diagnostic-group breakdown. A participant may be enrolled without contributing complete data to every analysis. The handling and reasons for missing imaging, biomarker, or clinical-marker measurements are not reported in the registry record. Nor is it stated whether all enrolled participants were included in each analysis or whether any exclusions occurred.

These omissions limit assessment of both selection and uncertainty. General statistical explanation: if participants with unavailable results differ from those with complete results, the analyzed group may not represent the enrolled group. Without participant flow and missing-data methods, the extent of that concern cannot be determined. The registry record also does not report a sample-size justification for the original study, so the intended precision or power for the planned comparisons cannot be inferred.

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.

The registry reports 31 participants at risk in the serious-adverse-event and other-adverse-event groupings. It records zero participants affected in the serious category, zero affected in the other category, and zero deaths. These are the posted registry counts for the listed study group.

These figures should be read as registry-reported counts, not as proof that no adverse events occurred in any broader sense. The record does not report the event terms, a description of how events were collected, the observation period for safety reporting, or whether all participants contributed complete safety follow-up. No serious adverse-event terms are listed. The difference between no event recorded in these categories and certainty that no event occurred cannot be evaluated from the registry record.

The study involved imaging procedures, including an experimental sodium fluoride-based drug for 18-NaF PET/CT, but the adverse-event figures do not provide modality-specific counts. They cannot identify which procedure, if any, was associated with an event. Nor do these counts support comparisons of safety between imaging approaches, because the study was single-group and the record does not report separate modality-specific safety denominators or analyses. The counts are descriptive registry entries and should not be interpreted as a comparative safety estimate.

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

The record establishes that a completed phase 2 pilot study enrolled 31 participants with MGUS, SMM, or MM to undergo imaging procedures. Its primary endpoint was designed to count positive and negative results from 18F-FDG PET/CT and 18F-NaF PET/CT, with a stated timeframe of 60 days. The record also describes a broader interest in comparing imaging modalities and relating imaging information to clinical markers.

What the record does not establish is the observed imaging result. Positive and negative counts, diagnosis-specific results, and estimates comparing the modalities are not reported in the registry record. It therefore cannot show how often either modality classified a participant as positive, whether the modalities agreed, or whether one produced more positive findings in the enrolled sample. The absence of those results is not evidence that the modalities were equivalent or that no findings were observed.

The stated aims should also be distinguished from conclusions about disease progression. The study description refers to interest in markers associated with progression from MGUS or SMM to MM, but the primary endpoint is an imaging classification count. The registry record does not report a longitudinal progression outcome or an analysis demonstrating that imaging findings predict future progression. It does not establish clinical utility, diagnostic accuracy, or improved health outcomes.

The single-group design is suitable for describing procedures and observed findings in the enrolled participants, but it is not a randomized comparison of treatments. The imaging modalities were performed in the same study group, and the record does not report a blinded assessment or a separate control group. Any comparative interpretation would depend on participant-level results, evaluability, and analysis methods that are not reported.

The safety entries report zero affected participants in the listed serious and other adverse-event categories and zero deaths among 31 participants at risk. These are descriptive registry counts and do not answer every question about procedure-specific safety or completeness of follow-up. Overall, the registry supports a description of the study’s design and intended measurements, but not a conclusion about the comparative performance or clinical meaning of the imaging results.

13. Why This Trial Matters Statistically

This study raises a useful statistical question: how should imaging methods be compared when the same participants undergo more than one procedure and belong to clinically distinct groups? That question differs from comparing independent treatment arms. When observations come from the same person, the within-participant relationship can matter; when results are considered across MGUS, SMM, and MM, the group structure can also matter. The registry describes the intended comparisons, but does not report enough analysis detail or outcome results to determine how these features were handled.

The primary endpoint is a count of participant-level positive and negative classifications. This makes clear why outcome definitions matter: classification rules determine which findings enter each category. The registry provides criteria for positive imaging findings, but does not report reader agreement, adjudication, or diagnostic-accuracy analyses. Those details are important for interpreting whether a count reflects the performance of a modality, variation in reading, or the particular enrolled sample.

The pilot setting also highlights the difference between generating evidence and establishing a definitive result. General statistical explanation: exploratory studies can identify practical questions, characterize measurements, and inform future design, while precision and reproducibility require appropriate outcome reporting and analysis. The registry lists multiple secondary outcomes and analyses, yet does not report the analysis plan or multiplicity approach. Without those details, an isolated finding, if reported elsewhere, would need careful context.

Finally, the distinction between the stated aim and the available result is central. The study aimed to compare imaging techniques and relate imaging information to clinical markers, but the registry record does not report the corresponding primary outcome counts or correlation estimates. Recognizing this boundary helps avoid treating a research question as if it were an observed conclusion. The trial is statistically relevant for its measurement and design questions; the posted record alone does not resolve those questions.

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 interpreting this registry record is that the primary endpoint results are not reported. The positive and negative participant counts for 18F-FDG PET/CT and 18F-NaF PET/CT are unavailable, as are any estimates of difference, uncertainty, or statistical significance. The record therefore describes the intended outcome without showing what was observed.

The number enrolled is reported, but participant flow and analysis denominators are not. The record does not state how many participants had MGUS, SMM, or MM, how many completed each imaging procedure, or how many contributed evaluable results. It is also not reported whether results were excluded or missing, or how missing data were handled. These omissions prevent assessment of whether the analyzed participants corresponded to everyone enrolled.

The design is single-group, with no random allocation and no masking. The registry does not report a separate control group or a blinded imaging assessment. It also does not specify a statistical method for comparing the modalities or clarify whether paired results were analyzed as paired observations. These features limit the ability to assess comparative claims from the design description alone.

The record gives positivity criteria but does not report the reference standard, adjudication process, reader agreement, or measures of diagnostic accuracy. It lists seven secondary outcomes and ten analyses, while the multiplicity strategy and analysis plan are not reported. The original sample-size justification is also not reported in the registry record.

Finally, the primary endpoint concerns imaging classifications over a 60-day timeframe; the registry does not report a progression outcome or establish that imaging findings predict future disease course. These limitations are specific to what the record reports and do not establish that a procedure was ineffective, that findings were absent, or that the study had no value.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Imaging in MGUS, SMM and MM — NCT01237054.
• Registry record last updated 2026-02-06; status last verified 2026-01.
• Results first posted 2015-01-16.
• 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

This completed phase 2 pilot study enrolled 31 participants with MGUS, SMM, or MM. Participants were to undergo 18F-FDG PET/CT, 18-NaF PET/CT, and DCE-MRI on separate days. The design is single-group, with no random allocation and no masking; the primary endpoint timeframe is 60 days.

The primary endpoint counts positive and negative results for 18F-FDG PET/CT and 18F-NaF PET/CT. The registry lists seven secondary outcomes and ten analyses. It reports zero participants affected in the serious and other adverse-event categories and zero deaths among 31 at risk.

The primary imaging counts, analysis populations, missing-data handling, statistical analysis plan, multiplicity approach, and original sample-size justification are not reported in the registry record. The posted record therefore describes the study design and intended measurements but does not provide the results needed to assess the comparative imaging findings.