A Study of 123I-CMICE-013 Radiopharmaceutical in Healthy Volunteers: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for A Study of 123I-CMICE-013 Radiopharmaceutical in Healthy Volunteers (NCT01558362), a na phase 1 study. The primary outcome is Biodistribution of the 123I-CMICE-013 Within the Lungs.

2. Clinical Question

In statistical terms, the central question is descriptive: what lung uptake was recorded over the imaging period after administration? Without a control group or a reported endpoint estimate, the record cannot support a comparative estimate of uptake or a claim about differences between treatments. The study’s healthy-volunteer population also defines the scope of the question; its findings would not, on their own, establish what the imaging measure would be in people with coronary artery disease.

3. Trial Design

This was a completed Phase 1 study sponsored by Ottawa Heart Institute Research Corporation. The registry lists enrollment of 12 and study dates from 2012-04 to primary completion in 2012-09. The official title describes intravenous administration to healthy adult volunteers and identifies safety, tolerance, pharmacokinetics and nuclear medicine imaging as study aims.

The design is listed as single-group, with allocation recorded as not applicable and masking as none. The intervention was 123I-CMICE-013. Rest and stress doses are listed separately in the adverse-event record, and the primary endpoint description says the rest and stress scans were performed one week apart. These dose labels do not create a randomized comparison: the record does not identify an untreated or active-control arm.

For each scan, the stated interval from enrollment to completion of imaging was 24 hours. SPECT imaging was performed immediately after injection, then after 90 minutes, 4 hours, 6 hours and 24 hours. The primary endpoint was a continuous measure expressed as a percentage of the total injected dose, with the mean and standard deviation specified as the summary statistics.

The registry record does not report a statistical analysis plan, a sample-size justification, or details of any interim analyses. Although it lists 7 outcome measures, the record does not provide numerical results for the primary endpoint. It lists two secondary outcomes concerning total effective dose, separately for men and women. The study’s stated scope is safety, biodistribution, pharmacokinetics, radiation dosimetry and imaging in healthy volunteers; no treatment-effect comparison is defined.

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

Analysis population for the primary outcome, as defined in the registry: Normal, healthy volunteers, with no pre-existing medical conditions were recruited. The volunteers were six (6) males and six (6) females and ranged from 18 to 42 years of age.

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 Biodistribution of the 123I-CMICE-013 Within the Lungs, measured over From enrollment to completion of imaging was 24 hours for each scan. Rest and stress scans were performed one week apart..

The primary endpoint is the biodistribution of 123I-CMICE-013 within the lungs, measured as a percentage of the total injected dose. In the endpoint description, SPECT imaging was carried out immediately after injection and at 90 minutes, 4 hours, 6 hours and 24 hours. A region of interest was manually drawn on planar images. Counts in the full-body region of interest on the initial images were treated as corresponding to 100% of the injected dose, and organ uptake was calculated relative to that reference.

The stated summary measure is the mean, with standard deviation as the measure of dispersion. The mean describes the average lung uptake among the observations included in an analysis; the standard deviation describes how spread out those observations are around that average. General statistical context: biodistribution measurements can help characterize where a radiopharmaceutical is detected over time. The registry’s endpoint description therefore links the imaging measurements to the study’s stated interest in distribution and localization.

The record does not report the mean, standard deviation, number of observations contributing at each imaging time, or a numerical result by scan condition. It also does not describe how missing or unreadable images were handled. Consequently, the endpoint definition is available, but the magnitude and precision of the observed lung uptake are not reported in the registry record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry defines the primary measure as a continuous outcome: lung biodistribution expressed as a percentage of total injected dose. It specifies the mean as the summary parameter and standard deviation as the measure of dispersion. These labels describe how a result would be summarized, but they do not supply the result itself. No mean or standard deviation for the primary endpoint is reported in the registry record.

What does the mean describe?

General statistical context: the mean is the arithmetic average of the observations included in an analysis. For a continuous imaging measure, it gives a central summary of the recorded uptake. It does not show whether all observations were close to that value or whether the average was influenced by values farther from the center. The registry does not state how many observations contributed to each time-specific estimate, or whether results were combined across the imaging times, rest and stress scans, or participants.

What does the standard deviation add?

General statistical context: the standard deviation describes spread around the mean in the units of the measurement. A larger standard deviation indicates more variability in the measurements; a smaller one indicates less. It is not a confidence interval and does not, by itself, quantify the precision of the mean. The record specifies standard deviation but does not report its value for the primary endpoint.

How was uptake defined?

The endpoint description says that a region of interest was manually drawn on planar images. Counts in the full-body region of interest on the initial images were taken to correspond to 100% of the injected dose. Uptake in an organ was then calculated as a percentage of that reference. Imaging was performed immediately after injection and at 90 minutes, 4 hours, 6 hours and 24 hours. This defines the measurement process in the record; details such as reader training, repeat readings, or assessment of measurement reliability are not reported.

What can repeated imaging contribute?

General statistical context: measurements at multiple times can describe how an imaging signal is distributed over the observation period. Measurements taken from the same participant can be related, so an analysis that compares time points may need to account for within-participant correlation. The registry record does not report whether time points were analyzed separately or jointly, how repeated measurements were modeled, or how the rest and stress scan data were handled statistically. The stated scan interval was 24 hours for each scan, with rest and stress scans one week apart.

What does the design allow?

The study is listed as single-group, with allocation not applicable and no masking. General statistical context: a single-group descriptive study can characterize measurements in the participants observed, but it does not by itself isolate a treatment effect from other explanations. The registry does not identify a control group for the lung endpoint. The rest-dose and stress-dose labels in the safety record should not be treated as randomized comparison arms; the record describes rest and stress scans one week apart.

What uncertainty and analysis details are missing?

A confidence interval is a general statistical way to express uncertainty around an estimate, given an analysis and its assumptions. The record does not report a confidence interval, a p-value, or another uncertainty measure for the primary endpoint. It also does not report a statistical analysis plan, handling of missing measurements, analysis populations, or interim analyses. Those details should not be inferred from the stated use of a mean and standard deviation.

The record lists 7 outcome measures and no analysis records. The stated sample-size illustration assumptions describe two-sided significance level 0.05, equal allocation between two arms, a normal approximation, and an effect defined as a stated fraction of the observed effect. Such an illustration is not the trial’s design, particularly for this single-group study, and any sample-size illustration is not observed power. Numerical sample-size estimates are not reported in the registry record. No recalculated comparison is presented for the primary endpoint, because the necessary outcome summary values are not reported.

10. Multiplicity, Missing Data and Analysis Populations

The registry lists 7 outcome measures. The primary outcome identified in the record is lung biodistribution, summarized by a mean and standard deviation. Two secondary outcomes are named: total effective dose of 123I-CMICE-013 for men and total effective dose of 123I-CMICE-013 for women. Numerical results for these outcomes are not reported in the registry record.

Multiplicity refers, in general, to the increased chance of a misleading result when many outcomes or comparisons are tested without accounting for the number of tests. The registry record does not report a multiplicity adjustment or a prespecified hierarchy among outcomes. It also does not report an analysis plan describing which outcomes would be treated as confirmatory or exploratory. Therefore, it is not possible to determine how multiple outcomes were handled analytically.

The record lists no analysis records. That does not establish that no analyses were conducted; it means analysis details and results are not available in the registry record. The registry does not report the analysis populations, whether all enrolled volunteers contributed to each endpoint, or how missing observations, withdrawals, or unusable images were handled. It also does not report whether observations were excluded or imputed.

These omissions matter for interpretation because the measurement schedule includes several imaging times and rest and stress scans. General statistical context: missing measurements and choices about which observations enter an analysis can affect both the estimated mean and its uncertainty. Without a reported analysis population and missing-data approach, the completeness and basis of any endpoint estimate cannot be assessed. In this record, the endpoint estimate itself is also not reported.

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 non-serious adverse event terms

The adverse-event record lists separate rest-dose and stress-dose groups. For each group, the record lists 12 participants at risk for serious adverse events and 0 with a serious adverse event. It also lists 12 at risk for other adverse events and 2 affected. Deaths are listed as 0 in each group. The record does not provide the terms or descriptions of the other adverse events in the information available.

These are descriptive counts, not evidence of a comparative safety effect. The study is single-group, with no randomized control group, and the rest-dose and stress-dose labels do not represent a like-for-like randomized comparison. The record does not explain whether the same volunteers contributed to both dose-group records, so the figures should be read within each listed group rather than combined.

General statistical context: a count of zero observed serious events does not establish that the underlying risk is zero. The precision of a safety estimate depends on the amount and structure of the observed data, and event counts alone do not describe all possible harms. The registry record does not report confidence intervals or further detail about event ascertainment. No treatment recommendation follows from these counts.

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

This record describes a Phase 1 imaging study of intravenous 123I-CMICE-013 in healthy adult volunteers. Its primary endpoint was lung biodistribution as a percentage of total injected dose, using imaging immediately after injection and at 90 minutes, 4 hours, 6 hours and 24 hours. The endpoint definition and measurement approach are reported, but the mean and standard deviation are not. As a result, the record does not show the observed magnitude or variability of lung uptake.

The study’s stated aims also include safety and tolerability, pharmacokinetics, radiation dosimetry, and distribution and localization. The record lists total effective dose outcomes for men and women, but numerical results for those outcomes are not reported. The safety record lists no serious adverse events or deaths in either dose group and lists other adverse events in each. These registry counts describe what was recorded; they do not establish a comparison with another treatment or with no treatment.

The design is single-group, with no allocation and no masking. Rest and stress scans are described as occurring one week apart, but they are not a randomized control comparison. General statistical context: without a suitable comparator, observed measurements cannot isolate what would have happened under an alternative intervention. The record also does not report an analysis plan, uncertainty estimates, missing-data handling, or analysis populations.

The recruited volunteers were described as healthy and without pre-existing medical conditions. Although coronary artery disease appears in the registry’s condition listing, the record does not describe a patient population with that disease. Accordingly, the registry record does not establish how the imaging findings apply to patients with coronary artery disease, whether the measure predicts clinical outcomes, or whether the radiopharmaceutical is superior to another tracer. Those questions are outside what can be concluded from the reported information.

13. Why This Trial Matters Statistically

This trial is statistically notable for the distinction between a clearly specified measurement and a reported result. The registry describes the lung endpoint, its units, the imaging schedule and the summary statistics intended for it. Yet it does not report the endpoint’s mean or standard deviation. A precise measurement definition helps readers understand what was intended to be measured, but it cannot substitute for the numerical estimate and its uncertainty.

The design also matters. A single-group study can provide descriptive observations in the volunteers enrolled, but it does not provide a randomized control comparison. The registry’s rest-dose and stress-dose labels relate to scans performed one week apart; they should not be mistaken for independent treatment arms with like-for-like control conditions. This limits the kinds of comparative conclusions the record can support.

General statistical context: imaging protocols that collect measurements over time can produce correlated observations within a participant. Transparent reporting of the analysis method, time-point handling, and missing-data approach helps readers assess whether the summary reflects the intended measurements. Those details are not reported in the registry record. The safety counts are likewise descriptive and are not accompanied by uncertainty estimates or event descriptions.

The record lists safety, pharmacokinetics, dosimetry and imaging among its aims, alongside a primary biodistribution outcome and two named dose outcomes. It does not report numerical results for those outcomes or a statistical analysis plan. Thus, the record is useful for identifying the intended scope and measurement framework, while leaving the observed endpoint magnitudes and their precision unresolved. That distinction is important when evaluating what can—and cannot—be inferred statistically from a Phase 1 registry record.

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 interpreting the posted results is that the registry record does not report the numerical result for the primary lung biodistribution endpoint. The mean and standard deviation are specified as summary measures, but their values are not reported. No confidence interval or other uncertainty estimate is reported either. This prevents assessment of the measured uptake’s magnitude, variability, and precision.

The study is listed as single-group, with no allocation and no masking, and no randomized comparator is described. The rest-dose and stress-dose labels do not establish a control comparison. Although scans are described as occurring one week apart, the record does not report how rest and stress endpoint data were analyzed or whether a direct contrast was estimated.

The population was described as healthy volunteers without pre-existing medical conditions, including six males and six females ranging from 18 to 42 years. The registry lists coronary artery disease as a condition, but the recruited population description does not indicate that participants had that condition. The record therefore does not establish applicability to patients with coronary artery disease.

Other limitations in the registry record include the absence of reported analysis populations, missing-data handling, an analysis plan, and details of interim analyses. The record does not state the number of observations contributing at each imaging time or describe image-reading reliability. It lists two secondary total effective dose outcomes but does not report their numerical results. The adverse-event listing gives counts but no event terms or descriptions for the other adverse events. These gaps limit assessment of both the endpoint analyses and the completeness of safety reporting.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: A Study of 123I-CMICE-013 Radiopharmaceutical in Healthy Volunteers — NCT01558362.
• Registry record last updated 2025-03-26; status last verified 2025-01.
• Results first posted 2025-03-26.
• Data retrieved from ClinicalTrials.gov on 2026-11-09.
• Group sizes, means, event counts and sponsor-reported p-values are reproduced as posted. Effect estimates, confidence intervals and p-values not attributed to the sponsor are independently calculated from the posted summary statistics.

18. Record Summary

The registry identifies a completed Phase 1, single-group study of intravenous 123I-CMICE-013 in healthy adult volunteers, sponsored by Ottawa Heart Institute Research Corporation. Enrollment is listed as 12, with study dates from 2012-04 to primary completion in 2012-09. The primary endpoint is lung biodistribution as a percentage of total injected dose, summarized by the mean and standard deviation.

Imaging was described immediately after injection and at 90 minutes, 4 hours, 6 hours and 24 hours. Rest and stress scans were performed one week apart. The record lists two secondary outcomes for total effective dose, separately for men and women, and lists no analysis records.

The adverse-event record lists 0 serious adverse events and 0 deaths in each dose group, with 2 affected by other adverse events in each. Numerical primary and secondary outcome results, an analysis plan, analysis populations and missing-data methods are not reported in the registry record.