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 Repeat PET/CT Imaging of Patients With Amyloid 124I-AT-01 to Measure Changes in Organ-specific Amyloid Load (NCT05968846), a na phase 2 study. The primary outcome is To Measure Visual Changes and Quantitative Changes in Organ-specific Uptake of 124I-AT-01.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT05968846 |
| Phase | Phase 2 |
| Condition | Systemic Amyloidosis |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 19 |
| Arms in the primary outcome | n/a |
| Lead sponsor | University of Tennessee Graduate School of Medicine |
| Results posted | Yes |
2. Clinical Question
Population
Participants with systemic amyloidosis who had baseline PET/CT imaging in AMY1001 (NCT03678259) and repeat imaging in the current study. The registry reports enrollment of 19 participants.
Intervention
A single intravenous injection of 124I-AT-01, described as an iodine-124-labeled peptide, followed by PET/CT imaging. The study concerns whether uptake of the radiotracer can be assessed visually and quantitatively in specific organs.
Comparator
The registry describes a single-group study, not a randomized comparison. The imaging reference is each participant’s baseline PET/CT from AMY1001, with repeat PET/CT on Day 2 of the current study; this is not a separate control arm.
Primary question
Can visual and quantitative changes in organ-specific uptake of 124I-AT-01 be measured from baseline, and do those changes relate to disease-status changes in a way that could support monitoring organ-specific amyloid load over time?
The registry frames the work as an imaging-measurement question in systemic amyloidosis. Uptake is the imaging signal under study, while the proposed clinical relevance is whether its change corresponds to change in disease status. Those are related but distinct questions: an imaging measure may change, and a separate analysis is needed to establish whether that change tracks disease status. The registry record does not report results addressing either question.
3. Trial Design
The registry classifies this study as Phase 2 and single-group, with 19 participants enrolled. It lists no allocation method and no masking. These design features mean the registry does not describe randomized assignment or a masked comparison between treatment groups. The study is marked completed. Its recorded start date is 2022-12-07, and its primary completion date is 2023-08-01.
Participants received a single intravenous injection of 124I-AT-01, followed by PET/CT imaging. The primary outcome timeframe identifies baseline PET/CT imaging from AMY1001 (NCT03678259) and repeat PET/CT imaging on Day 2 of the current study. Thus, the stated reference for assessing change is an earlier scan, rather than a concurrent untreated or active-comparator group. The registry does not report further details about the interval between the baseline scan and repeat imaging.
The stated purpose was to measure visual and quantitative change in organ-specific uptake and to assess whether such changes correlate with disease-status changes. The primary outcome description focuses on visual change in radiotracer uptake from baseline, while its title also refers to quantitative change. Six outcome measures are listed in the record, but their results are not reported in the registry record. The registry lists no statistical analyses. Further details on the measurement procedures, scoring rules, analysis populations, or prespecified statistical plan are not reported in the registry record.
A within-participant comparison can focus on change over time, but it does not by itself separate imaging change from other changes that may occur over time. Without a randomized comparator, the study is not designed as a like-for-like comparison of assigned groups. This design is suited to the stated measurement question, but the registry record does not establish whether observed imaging changes correspond to disease-status changes.
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: Prior to assignment, potential participants were screened to verify their medical records, including confirming that participants must have been imaged with 124I-AT-01 as part of the AMY1001 study more than 12 months prior to the repeat imaging.
Overall Study
| Stage | All Participants |
|---|---|
| STARTED | 19 |
| COMPLETED | 19 |
| NOT COMPLETED | 0 |
Analysis population for the primary outcome, as defined in the registry: Summary of change in visual radiotracer uptake from baseline PET/CT imaging
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 | All Participants |
|---|---|---|
| Participants analysed | 19 | |
| Age, Continuous | Years | 72 (63 to 76) |
| Sex: Female, Male: Female | Participants | 8 |
| Sex: Female, Male: Male | Participants | 11 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 1 |
| Race (NIH/OMB): White | Participants | 18 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 19 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Time Since SA Diagnosis | Years | 7 (6 to 9) |
| Time Since baseline imaging in AMY1001 study (years) | Years | 2.9 (SD 0.9) |
| Amyloid Type: AL Kappa | Participants | 6 |
| Amyloid Type: AL Lambda | Participants | 3 |
| Amyloid Type: ATTR; Mutation type L58H | Participants | 3 |
| Amyloid Type: ATTR; Mutation type T60A | Participants | 5 |
| Amyloid Type: ATTR; Mutation type V30M and V122I | Participants | 1 |
| Amyloid Type: ATTR; Mutation type Wild type | Participants | 1 |
| Amyloid therapy at time of imaging of AL | Participants | 2 |
| Amyloid therapy at time of imaging of ATTR: Stabilizer only | Participants | 1 |
| Amyloid therapy at time of imaging of ATTR: Silencer only | Participants | 5 |
| Amyloid therapy at time of imaging of ATTR: Stabilizer and silencer | Participants | 4 |
6. Primary Endpoint
The registry lists the primary outcome as To Measure Visual Changes and Quantitative Changes in Organ-specific Uptake of 124I-AT-01, measured over Baseline PET/CT imaging from AMY1001 (NCT03678259) and repeat PET/CT imaging on Day 2 of the current study.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary outcome is described as measuring visual and quantitative changes in organ-specific uptake of 124I-AT-01 from baseline in participants with systemic amyloidosis. Its recorded unit is participants, and its timeframe compares baseline PET/CT imaging from AMY1001 (NCT03678259) with repeat imaging on Day 2 of the current study. The outcome’s population description refers specifically to change in visual radiotracer uptake from baseline.
The endpoint addresses whether PET/CT can detect change in the radiotracer signal in different organs. The registry’s stated rationale is that organ-specific uptake changes might correlate with changes in disease status and could therefore support monitoring organ-specific amyloid load over time. Measuring uptake and establishing a relationship with disease status are separate evidentiary steps; the registry’s stated purpose does not itself demonstrate that either relationship was observed.
General: a change-from-baseline endpoint uses a participant’s earlier measurement as the reference for a later measurement. This can make the question directly about within-person change, but interpretation depends on how the measurements are defined, collected, and compared. The registry record does not report the actual visual or quantitative change values, the scoring method, or an estimate of association with disease status. Consequently, the endpoint’s measurement goal can be described, but its observed magnitude and precision cannot be assessed from the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry identifies a focused imaging question: whether PET/CT can measure visual and quantitative changes in organ-specific uptake of 124I-AT-01. It describes baseline imaging from AMY1001 and repeat imaging on Day 2 of the current study. However, it does not report the primary outcome results. There is therefore no posted estimate of the number of participants with a change, the magnitude or direction of uptake change, or the precision of an estimate. The record also does not report an analysis relating uptake change to disease-status change.
The primary outcome is expressed in participants, and the registry’s population description refers to change in visual radiotracer uptake. The primary outcome title also refers to quantitative change. No numerical outcome result or analysis is listed. The record lists six outcome measures overall, but it does not report results for them. No analyses are listed in the registry record. These omissions mean the measurement objective and planned timeframe can be summarized, but the findings cannot be interpreted as evidence for or against the stated measurement aims.
General: a confidence interval describes the range of values reasonably compatible with an estimate under the statistical model used. Its width reflects precision, while the point estimate describes the estimated effect or change. Without an estimate and interval, precision cannot be evaluated. A p-value, if reported, would address compatibility with a specified null model; it would not by itself establish the size, importance, or reliability of an imaging change. Neither p-values nor confidence intervals for the primary outcome are reported in the registry record.
The single-group design also constrains interpretation. Comparing repeat imaging with baseline can describe change within participants, but without a separate randomized comparison it cannot establish what would have happened over the same period under another imaging or treatment condition. The record lists allocation as not applicable and describes no masking. These features do not invalidate the measurement question, but they limit what can be concluded about causes of change and comparative effects.
The registry states that the study aimed to determine whether uptake changes correlate with disease-status changes. A correlation analysis would address whether the two types of change vary together; it would not alone establish that one causes the other. The record does not report the disease-status measure, the association estimate, or its precision. No conclusion about tracking performance or monitoring utility can be drawn from the registry record.
Any sample-size illustrations associated with this study should be read as planning calculations, not as observed evidence. The stated assumptions are a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and an effect defined as a stated fraction of an observed effect. The trial itself is described as single-group, so the two-arm allocation is an illustration rather than its study design. Such calculations are not observed power and do not supply missing outcome findings. The registry record does not report a trial-specific sample-size justification.
9. Statistical Methods Explained
General: statistical methods translate observations into estimates and measures of uncertainty. For this study, the central measurement question concerns whether organ-specific uptake changes between baseline PET/CT and repeat imaging can be assessed. The registry identifies that objective and the comparison timeframe, but does not report the statistical analysis used to quantify change. Methodological interpretation should therefore distinguish the stated outcome from any particular analysis that might have been used.
What does change from baseline mean?
General: a change score represents a later measurement relative to an earlier reference measurement. It can be expressed as a difference, a category of change, or another prespecified summary. The choice affects interpretation. A participant count can summarize how many people meet a defined change criterion, while a quantitative summary can describe the scale and distribution of measured changes. The registry records the primary outcome unit as participants and describes visual change, while the outcome title also mentions quantitative change. It does not report the operational definitions or scoring rules.
With repeated measurements, the observations from one participant are related rather than independent. General: an analysis should account for this within-person relationship if it estimates an average change or compares repeated measurements. A simple comparison that treats the two scans as unrelated observations would not reflect that dependence. The registry record does not identify the analysis method, so it is not possible to determine how this issue was addressed.
What would an estimate and its uncertainty add?
General: an estimate describes the observed size or frequency of a change in the study sample. A confidence interval communicates uncertainty around that estimate under the chosen statistical model. A narrow interval indicates greater precision than a wide interval, all else equal, but precision does not by itself establish clinical relevance. The registry record does not report an estimate, confidence interval, or other uncertainty measure for the primary outcome.
General: a hypothesis test compares observed data with a null hypothesis under specified assumptions. A p-value describes how unusual the data would be if that null model and its assumptions were true. It is not the probability that the null hypothesis is true, and it does not measure the size of an effect. The registry record lists no analyses and reports no primary-outcome p-value. The stated significance level of 0.05 appears in the assumptions for planning illustrations; it is not a reported trial result or, by itself, evidence of the analysis plan.
Why does the single-group structure matter?
General: a comparison with baseline can characterize within-participant change, but time-related change may have explanations other than the imaging target of interest. A concurrent comparison group can help distinguish changes associated with a study condition from changes that would occur over time for other reasons, although the value of that comparison depends on its design. This registry describes a single-group study and allocation as not applicable, not a randomized comparison. The baseline scan from AMY1001 serves as the stated reference; it is not described as a concurrent control arm.
The record lists no masking. General: masking can reduce the influence of knowledge about study condition on measurement or interpretation, depending on who is masked and how outcomes are assessed. In a visual imaging outcome, the scoring process and reader procedures may be relevant. The registry does not report reader masking, the number of readers, or procedures for resolving differences, so no conclusion about these safeguards is possible.
How should sample-size illustrations be read?
General: sample-size calculations estimate how many observations a planned design would need to detect a specified effect with a chosen statistical threshold and power, under stated assumptions. They are planning tools, not evidence about what happened in the completed study. The stated illustration assumptions use a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and an effect defined as a stated fraction of the observed effect. The description specifies participants per arm for non-time-to-event calculations. These assumptions describe an illustration and should not be mistaken for the registry’s single-group design or for a trial-specific design justification.
The record does not report the statistical plan, a sample-size justification, the observed effect used for the illustrations, or analysis details. An illustration based on an observed effect is also not observed power: it does not tell whether the trial had a particular probability of detecting its actual result. Without the actual effect estimate and its uncertainty, the illustrations cannot establish how precisely the imaging question was answered.
What do counts and safety summaries convey?
General: a participant count describes how many people fall into a reported category, while the number at risk gives the group against which that count is understood. Counts alone do not describe event timing, severity beyond the category definition, or causal attribution. The registry reports safety counts, but does not provide a detailed event narrative or analysis. The outcome unit of participants likewise means that the primary endpoint is framed as a participant-level summary; it does not reveal the underlying measurements or their distribution.
Overall, the available record supports a description of the imaging objective, study structure, timeframe, and limited safety counts. It does not support reconstruction of an analysis, assessment of statistical precision, or evaluation of whether changes in uptake tracked disease status.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists six outcome measures, but does not report results for them or list any statistical analyses. It does not describe a multiplicity strategy, such as how interpretation would account for testing several outcomes or several organ-specific measures. Whether any adjustment or hierarchical testing plan was used is not reported in the registry record. Without those details, the risk of selectively emphasizing findings across multiple outcomes cannot be assessed.
Missing-data handling is also not reported in the registry record. The record does not state how unavailable scans, incomplete measurements, or participants without usable follow-up data were handled. It does not describe an analysis population or say whether every enrolled participant contributed to the primary outcome. Enrollment is reported as 19, but the number included in the primary outcome analysis is not reported in the registry record.
The registry does not report interim analyses, stopping rules, or a prespecified statistical plan. These details should not be inferred from the study’s completed status or from the presence of outcome descriptions. Without the analysis populations, missing-data procedures, and multiplicity plan, readers cannot determine how these issues affected the reported findings; no primary findings are reported in any case.
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 |
|---|---|---|---|
| All Participants | 0 | 19 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| All Participants | 0 of 19 |
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 |
|---|---|
| All Participants | 1 of 19 |
Most frequent non-serious adverse event terms
| Event term | Organ system | All Participants |
|---|---|---|
| Arthralgia | Musculoskeletal and connective tissue disorders | 1 of 19 |
The registry lists 19 participants at risk for serious adverse events and 0 participants affected. It lists 19 participants at risk for other adverse events and 1 participant affected. Deaths are reported as 0. No serious adverse event terms are listed.
These are counts in the registry safety summary. They do not provide event timing, individual clinical details, severity beyond the serious-versus-other grouping, or an assessment of whether an event was caused by the study intervention. The count of 1 refers to participants affected in the other-adverse-event category; the record does not provide a term for that event.
General: safety counts describe what was recorded within the stated group and observation period. They do not, by themselves, establish comparative risk or causality. Because the study is single-group and the registry does not report a comparator safety analysis, the counts should be read descriptively rather than as a comparison between groups.
12. What the Result Does, and Does Not, Mean
The record establishes that the study was designed to examine whether PET/CT could measure visual and quantitative changes in organ-specific uptake of 124I-AT-01. It identifies a baseline scan from AMY1001 and repeat imaging on Day 2 of the current study, and it describes the intended connection between uptake change and disease-status change. Those are the study’s objectives, not reported findings.
The registry does not report primary outcome results, quantitative changes, participant counts for the primary outcome, or analyses of associations with disease status. It therefore does not show how often uptake changed, how large any change was, how precise an estimate might be, or whether uptake change tracked disease-status change. The absence of these results in the registry record should not be interpreted as a finding of no change or no association.
General: evidence that a measurement can be repeated or can change is distinct from evidence that it reflects a clinical state or reliably tracks change in that state. Establishing a relationship requires defined measures and an analysis of their correspondence, with uncertainty and limitations made clear. The registry’s stated monitoring objective does not establish that the objective was achieved.
The study’s single-group structure also means it is not a randomized comparison of alternative conditions. Its baseline reference permits a change-over-time question, but does not establish what would have happened under a different condition. The safety counts are descriptive and do not establish causality or comparative safety. No treatment recommendation or conclusion about treatment effectiveness follows from the information reported in the registry record.
13. Why This Trial Matters Statistically
This study is statistically relevant because it focuses on measurement: whether an organ-specific imaging signal can be assessed over time and whether its change corresponds to disease-status change. In clinical research, a measurement tool’s usefulness depends not only on whether it produces a signal, but also on how consistently and precisely that signal is measured and how it relates to the question it is intended to answer.
The baseline-and-repeat structure makes the within-participant change question explicit. At the same time, the single-group design distinguishes the study from a randomized comparison. That distinction matters when readers interpret change: a before-and-after pattern can describe what was observed, while a comparator can help address alternative explanations. The registry record does not report the results needed to evaluate the imaging relationship.
The record also illustrates why endpoint definitions and analysis details matter. The primary outcome description refers to visual change and the title refers to visual and quantitative change, but the scoring rules, statistical methods, uncertainty estimates, and analysis populations are not reported. The registry lists six outcome measures and no analyses. Those gaps limit interpretation even though the study purpose and imaging timeframe are stated.
Finally, planning calculations and observed findings answer different questions. The stated sample-size assumptions are for illustrations, including equal allocation between two arms, whereas the registry describes a single-group study. Keeping those concepts separate helps prevent a planning scenario from being mistaken for a feature or result of the completed trial.
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 statistical interpretation is that the registry record does not report primary outcome results or any statistical analyses. The stated imaging objective can be described, but the size, direction, and precision of change cannot be evaluated. The record also does not report an analysis of whether uptake changes correlate with disease-status changes, despite identifying that relationship as part of the study’s purpose.
The study is described as single-group, with allocation listed as not applicable and masking as none. The baseline scan is from AMY1001, rather than a concurrent randomized comparator described in this record. This structure limits comparative interpretation and does not by itself establish the cause of any change over time. The registry does not report the interval between baseline imaging and repeat imaging.
Other relevant details are not reported in the registry record: the visual scoring and quantitative measurement procedures, analysis populations, missing-data handling, multiplicity strategy, statistical plan, and sample-size justification. Six outcome measures are listed, but their results are not reported. The number of participants included in the primary outcome analysis is not reported in the registry record.
The safety summary gives counts but no event terms for the one participant in the other-adverse-event affected count, and no detailed event information or causal assessment. These omissions limit interpretation of the safety record. Taken together, the registry supports a description of study intent and design, but not an assessment of the primary imaging findings or their precision.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Repeat PET/CT Imaging of Patients With Amyloid 124I-AT-01 to Measure Changes in Organ-specific Amyloid Load — NCT05968846.
- Registry record last updated 2026-09-02; status last verified 2024-12.
- Results first posted 2026-09-02.
- 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
This completed Phase 2 study enrolled 19 participants with systemic amyloidosis. The registry describes a single-group design with no masking and a single intravenous injection of 124I-AT-01 followed by PET/CT imaging. The primary outcome concerns visual and quantitative changes in organ-specific uptake, using baseline imaging from AMY1001 (NCT03678259) and repeat imaging on Day 2 of the current study.
The registry lists six outcome measures but reports no primary outcome results and no statistical analyses. It does not report whether uptake change correlated with disease-status change. Safety counts list 0 participants affected by serious adverse events, 1 affected by other adverse events, and 0 deaths. The record does not report a trial-specific statistical plan, missing-data procedures, or sample-size justification.