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 Pilot Study of Multi-Modality Imaging in Patients With Hematologic Malignancies (NCT02042924), a na na study. The primary outcome is Difference in Percentage of Proliferating Bone Marrow Between Baseline and 100 Days.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02042924 |
| Phase | NA |
| Condition | Hematologic Malignancy |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 2 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Masonic Cancer Center, University of Minnesota |
| Results posted | Yes |
2. Clinical Question
The study asked whether imaging could characterize variation in bone-marrow proliferation and marrow composition in patients receiving a myeloablative transplant regimen involving total body irradiation (TBI) or total marrow irradiation (TMI). The registry describes FLT PET/CT as the measure of cellular proliferation and whole-body water-fat MRI as the measure of marrow composition. It also identifies feasibility of PET and MRI imaging as an objective for future trial planning.
The primary question concerned the difference in the percentage of proliferating bone marrow between baseline and 100 days after transplant. The registry specifies assessment across the skull, proximal humeri, ribs, clavicles, cervical spine, thoracic spine, lumbar spine, sacrum, pelvis, and proximal femur. This is a within-person imaging question: the listed schedule calls for scans before the preparative regimen and again 100 days after transplant. It is not a randomized comparison between imaging strategies or a test of one transplant regimen against another.
The intended interpretation is therefore descriptive and exploratory. The study sought information about the distribution and variability of imaging measures, as well as whether the imaging schedule was feasible. The record does not report numerical endpoint results, a quantified assessment of heterogeneity, or a feasibility result. It states that only one patient was enrolled in each cohort and that an analysis would not be meaningful. The registry record does not report what those cohorts were, nor does it give a comparison of TBI with TMI.
3. Trial Design
This was a single-group pilot study, with enrollment of 2 participants. The registry lists allocation as NA and masking as none. It gives the phase as NA and the study type as “other.” These entries do not describe a randomized or masked treatment comparison. The record is marked terminated; it does not report a reason for termination.
Participants were described as receiving a myeloablative transplant using TBI or TMI in the preparative regimen. The imaging plan was one FLT PET/CT and one MRI before the preparative regimen, followed by another of each scan 100 days after transplant. The registry lists FLT PET/CT as a radiation intervention and MRI as a device intervention. These are imaging procedures within the study description, not treatment arms being compared for clinical effectiveness.
The enrollment was too small for the registry to consider an analysis meaningful: its primary-outcome population note says that only one patient was enrolled in each cohort. The cohort definitions and any further details about allocation to cohorts are not reported in the registry record. The record does not provide a statistical analysis plan, sample-size justification, or explanation of how the pilot’s observations were intended to inform future trial design.
The registry lists 3 outcome measures and 0 analyses. The primary outcome is identified as the difference in percentage of proliferating bone marrow between baseline and 100 days. No numerical results for that outcome are reported. Consequently, the design and intended measurement schedule can be described, but the record does not support an estimate of the imaging changes or a comparison between patient groups.
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 | Imaging Studies |
|---|---|
| STARTED | 2 |
| COMPLETED | 1 |
| NOT COMPLETED | 1 |
Reasons for not completing this period:
| Reason | Imaging Studies |
|---|---|
| Early Death | 1 |
Analysis population for the primary outcome, as defined in the registry: Only one patient was enrolled in each cohort and therefore any analysis would not be meaningful.
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 | Imaging Studies |
|---|---|---|
| Participants analysed | 1 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 1 |
| Age, Categorical: >=65 years | Participants | 0 |
| Sex: Female, Male: Female | Participants | 0 |
| Sex: Female, Male: Male | Participants | 1 |
| Region of Enrollment: United States | participants | 1 |
6. Primary Endpoint
The registry lists the primary outcome as Difference in Percentage of Proliferating Bone Marrow Between Baseline and 100 Days, measured over 100 days.
| Element | Registry entry |
|---|---|
| Result type | N/A |
| Measure of spread | not applicable |
| Unit | not stated |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is the difference in the percentage of proliferating bone marrow between baseline and 100 days. The registry specifies that this measure is to be calculated at the skull, proximal humeri, ribs, clavicles, cervical spine, thoracic spine, lumbar spine, sacrum, pelvis, and proximal femur. FLT PET/CT is the named imaging modality associated with cellular proliferation in the study summary.
As a general statistical concept, measuring the same person at two time points can describe within-person change while accounting for the fact that measurements from one person are related. Examining multiple anatomical sites can also describe how the imaging measure varies across the skeleton. Such measurements are relevant to the study’s stated objective of estimating heterogeneity in cellular proliferation.
The registry record does not report the observed endpoint values, a summary of the baseline-to-follow-up differences, or uncertainty around any estimate. It also does not report a detailed endpoint calculation method, including how percentage proliferation was defined or summarized across sites. The stated purpose is exploratory measurement and future study design, not a comparison of clinical outcomes. With only one patient in each cohort, the record explicitly says that an analysis would not be meaningful.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry describes an imaging pilot with enrollment of 2 participants and a planned baseline scan and repeat scan 100 days after transplant. It identifies a primary endpoint based on the difference in percentage of proliferating bone marrow. However, it does not post numerical results for that endpoint. There is therefore no reported estimate of change to interpret, and no reported measure of precision such as a confidence interval.
The record’s statement that only one patient was enrolled in each cohort is central to interpretation. A very small number of observations cannot characterize between-person variability reliably. It also leaves little basis for distinguishing a pattern that might reflect a broader population from a pattern specific to an individual. The registry itself notes that an analysis would not be meaningful; that caution should take precedence over any attempt to infer a group effect from the study description.
The study summary frames the work as an effort to estimate heterogeneity in FLT-PET uptake and marrow composition and to assess imaging feasibility. The record does not provide results for either objective. It does not state whether both time points were completed for each participant, whether the images were evaluable, or whether the planned imaging procedures were feasible. Those findings are not reported in the registry record.
No statistical analysis is listed, and the registry reports 0 analyses. No p-value, effect estimate, confidence interval, or model result is posted. It would therefore be inappropriate to characterize the primary endpoint as showing an increase, decrease, or absence of change. Likewise, the record does not permit a comparison of TBI and TMI recipients or an assessment of how imaging findings relate to clinical outcomes.
General interpretation principle: an estimate and its uncertainty are both needed to judge how precisely a study describes a quantity. With no posted endpoint estimate and a sample of 2, neither the direction nor precision of any imaging change can be established from this record. The information supports describing the intended pilot design and its limitations, not drawing an efficacy conclusion about transplant preparation or imaging.
9. Statistical Methods Explained
The registry record identifies a single-group imaging study, not a randomized comparison. Allocation is listed as NA, masking as none, and the study phase as NA. It lists 2 enrolled participants and 0 analyses. The primary outcome is a baseline-to-100-days difference in percentage of proliferating bone marrow. No analysis method, statistical model, or numerical result is reported in the registry record.
What a within-person change measures
General explanation: when a measurement is taken from the same participant at two time points, a change score describes the difference between those measurements for that participant. This differs from comparing unrelated groups, because the two observations from one person may be more alike than observations from different people. A paired analysis, when suitable and prespecified, uses that relationship. The registry gives a paired imaging schedule, but does not report whether a paired statistical analysis was planned or performed.
A change score is interpretable only with a clear definition of the measurement at each time point and of how the difference is calculated. Here, the outcome title identifies percentage of proliferating bone marrow and the two time points, while the outcome description names anatomical sites. The record does not report additional operational details, such as image-processing rules, how site-level measurements would be combined, or how missing site measurements would be handled.
Variation and heterogeneity
General explanation: heterogeneity means that a quantity varies across people, locations, or other relevant units. A study may describe this variation using individual measurements, summaries of spread, or a model that separates different sources of variation. The choice depends on the study question and the amount and structure of the data. With only one patient in each cohort, the registry says an analysis would not be meaningful; the posted record does not provide a quantified estimate of heterogeneity.
The outcome covers multiple skeletal sites. Measurements at different sites within one person are not necessarily independent. General statistical methods for clustered or repeated measurements can account for such dependence, but the record does not name a method or model for doing so. It also does not report whether the primary endpoint would be analyzed site by site or as a single overall summary.
Precision and uncertainty
General explanation: a confidence interval gives a range of values compatible with an estimate under a specified statistical procedure. Its width reflects uncertainty and depends on factors including sample size and variability. A point estimate without an uncertainty measure can give an incomplete picture, while a very small study may yield substantial uncertainty even when a numerical estimate can be calculated. No confidence interval or endpoint estimate is reported for this study.
A p-value, when calculated under a defined model, describes how compatible the observed data are with a specified null hypothesis. It does not measure the size or clinical importance of an effect. No p-value or hypothesis test is reported. The record also does not specify a null hypothesis, significance level for an outcome analysis, or adjustment for multiple comparisons.
Why distributional assumptions matter
General explanation: conventional methods often rely on assumptions about the distribution of measurements or model errors. When data are sparse, those assumptions are difficult to assess from the data. Over-dispersion means that observed variability exceeds what a chosen model expects; ignoring it can make uncertainty appear smaller than it is. This is a general consideration, not a finding from this study. The registry provides no outcome data with which to evaluate distributional assumptions or over-dispersion.
The study summary also mentions whole-body water-fat MRI to assess marrow composition. That objective is distinct from the named primary endpoint of proliferating bone marrow. The registry does not report a statistical method or result for the MRI composition measure, nor does it specify how that measure would be related to FLT PET/CT findings.
Planning illustrations, if considered alongside the posted results, are not observed power. The record gives generic assumptions for such illustrations, but posts no observed endpoint effect from which to interpret an effect-based planning calculation. Any such illustration is a planning exercise, not evidence that the study had a particular probability of detecting a real effect.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 3 outcome measures, but only the primary endpoint is described in detail in the available record. No analysis is listed. The record does not report how the other outcome measures were defined, whether they were prespecified as primary or secondary, or how any statistical testing across outcomes or anatomical sites would be handled.
Multiplicity arises when several outcomes, sites, time points, or hypotheses are examined. General statistical explanation: the more tests conducted, the greater the chance that at least one result may appear notable by chance if no adjustment or interpretive strategy is used. Approaches can include prespecifying a single primary test, controlling an error rate, or treating analyses as exploratory. The registry record does not report a multiplicity strategy. The named endpoint itself spans multiple skeletal sites, but the record does not state whether these sites would be tested separately or combined.
Missing-data handling is also not reported in the registry record. The planned schedule includes baseline and 100-day imaging, but the record does not state whether both scans were completed for each participant, how incomplete scans would be handled, or whether any imputation method was planned. The registry does not describe analysis populations beyond the primary-outcome note that only one patient was enrolled in each cohort and that an analysis would not be meaningful.
With 2 participants and no posted analysis, there is no basis to assess how missing observations or multiplicity affected results. The registry record does not report interim analyses, a statistical analysis plan, or a method for defining evaluable imaging data.
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 |
|---|---|---|---|
| Imaging Studies | 0 | 2 | 0.0% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 0% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Imaging Studies | 1 of 2 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Imaging Studies |
|---|---|---|
| Sweating | General disorders | 1 of 2 |
The registry lists the Imaging Studies group with 2 participants at risk for serious adverse events and 0 participants affected by serious adverse events. For other adverse events, it lists 2 participants at risk and 1 affected. These counts describe the safety entries in the record; they do not provide event-level clinical details.
The registry does not list any serious adverse-event terms. The number of deaths is null, so a death count is not reported in the registry record; null should not be read as a reported count of zero. The record also does not identify the type, timing, or severity of the other adverse event, or whether it was considered related to imaging.
General statistical explanation: adverse-event counts in a very small group are descriptive and provide limited information about how often an event might occur in a broader population. The affected-participant count is not a comparative safety estimate because the study has no control group. The safety entries also do not establish causation. They should be read as the registry’s recorded counts, not as a comprehensive assessment of risk.
12. What the Result Does, and Does Not, Mean
This record documents a small pilot intended to examine multi-modality imaging around transplant in patients with hematologic malignancies. It specifies FLT PET/CT and MRI before the preparative regimen and again 100 days after transplant, and it identifies a primary endpoint based on the difference in percentage of proliferating bone marrow. It also states that the study sought to inform future trials and assess imaging feasibility.
The record does not report numerical imaging results, a completed analysis, or a feasibility finding. It therefore does not establish the direction or size of any imaging change, the precision of a change estimate, or whether the imaging measures differed by transplant preparation. It also does not report a clinical outcome comparison or establish that any imaging pattern predicts a clinical outcome.
The enrollment of 2 participants and the registry note that only one patient was enrolled in each cohort sharply limit what can be inferred about variation between people. The registry explicitly says analysis would not be meaningful. Even a descriptive observation from so few participants could not, by itself, characterize the range of imaging patterns in a wider patient population.
General statistical interpretation: a pilot can help identify practical questions and methods for later research, but its value depends on what data are collected and reported. In this case, the registry record describes the intended measurements but does not post the findings needed to judge feasibility or quantify heterogeneity. The record supports an account of study intent and design, not a conclusion about clinical benefit, harm, or the effectiveness of imaging or transplant approaches.
13. Why This Trial Matters Statistically
This study is statistically notable for the gap between a broad measurement objective and a very small enrolled sample. The stated aims include estimating heterogeneity in cellular proliferation and marrow composition, which concern variation across measurements or participants. The record reports enrollment of 2 and notes one patient in each cohort, while also stating that an analysis would not be meaningful. Those details make clear why a pilot of this size cannot provide a stable population-level description of heterogeneity.
The scheduled imaging also illustrates an important design distinction. Repeated measurements in the same participant can be useful for describing within-person change, while multiple anatomical sites can contribute information about spatial variation. At the same time, repeated and site-specific observations are related; they do not automatically function as independent participants. The registry does not report an analysis plan addressing this structure.
The record’s stated intention to inform future trials makes reporting of operational detail especially relevant. Information about image completion, evaluability, measurement definitions, and variability could help guide later work, but those findings are not reported in the registry record. Without them, the pilot’s contribution to planning cannot be assessed from the posted outcomes.
This is also a useful example of why study purpose and reported evidence should be separated. The record describes what investigators intended to measure, but contains no analysis result for the primary endpoint. Statistical interpretation must therefore remain focused on design, data availability, and limitations rather than inferred effects.
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 largest limitation is enrollment of 2 participants. The primary-outcome note says one patient was enrolled in each cohort and that analysis would not be meaningful. The record does not describe the cohorts, so their composition or any between-cohort contrast cannot be assessed.
The study is marked terminated, but a reason for termination is not reported in the registry record. The record does not state whether all planned baseline and 100-day scans were completed, whether the images were evaluable, or whether participants withdrew. Consequently, the available information cannot establish completeness of the planned measurements.
No primary endpoint result, other outcome result, or analysis is posted. The record does not report an analysis plan, endpoint calculation details beyond the stated measure and sites, handling of multiple sites, missing-data methods, or a sample-size justification. It also does not report feasibility findings, despite feasibility being an objective in the study summary.
The record describes imaging in patients undergoing transplant preparation involving TBI or TMI, but does not provide a like-for-like control comparison. It cannot support a comparative conclusion about those regimens or about clinical outcomes. The listed adverse-event counts are also very limited and lack event descriptions; the death count is not reported in the registry record.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Pilot Study of Multi-Modality Imaging in Patients With Hematologic Malignancies — NCT02042924.
- Registry record last updated 2017-12-05; status last verified 2017-12.
- Results first posted 2017-06-09.
- 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.
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18. Record Summary
NCT02042924 is a terminated, single-group pilot study of multi-modality imaging in patients with hematologic malignancies. It enrolled 2 participants and lists FLT PET/CT and MRI before the transplant preparative regimen and again 100 days after transplant. The summary describes an objective of estimating heterogeneity in cellular proliferation and marrow composition and assessing imaging feasibility.
The primary outcome is the difference in percentage of proliferating bone marrow between baseline and 100 days, across named skeletal sites. The registry states that only one patient was enrolled in each cohort and that an analysis would not be meaningful. It lists 3 outcome measures and 0 analyses, and does not report numerical primary results or a reason for termination.
The safety record lists 0 participants with serious adverse events and 1 with other adverse events among 2 at risk. A death count is not reported in the registry record.