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 Human Immune Globulin in Treating Patients With Primary Amyloidosis That is Causing Heart Dysfunction (NCT00547365), a na phase 1, phase 2 study. The primary outcome is Tolerance for Human Immune Globulin Intravenous (IGIV), as Reflected by the Number and Severity of Toxicity Incidents Occurring in Ten Patients Receiving at Least One Infusion of IGIV..
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00547365 |
| Phase | Phase 1, Phase 2 |
| Condition | Multiple Myeloma, Plasma Cell Neoplasm |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 10 |
| Arms in the primary outcome | n/a |
| Lead sponsor | University of Tennessee |
| Results posted | Yes |
2. Clinical Question
Population
Patients with cardiac-associated light chain (AL) amyloidosis, described in the registry as primary amyloidosis causing heart dysfunction. The primary endpoint population is patients who had at least one infusion of human immune globulin intravenous (IGIV). The registry lists an enrollment of 10.
Intervention
Human immune globulin intravenous (IGIV), a biological intervention. The registry’s purpose describes studying side effects and the best dose, as well as therapeutic potential. The dose and dosing schedule are not reported in the registry record.
Comparator
There is no separate comparator arm: the design is listed as single group, with allocation not applicable and no masking. This is not a randomized or controlled comparison, so the record does not provide a like-for-like control group.
Primary question
How much toxicity, in number and severity of incidents, occurred among patients receiving at least one IGIV infusion during follow-up of up to 1 year? The endpoint is framed as tolerance. The registry record does not report the primary endpoint’s actual result or a statistical analysis of it.
The study record identifies a phase I/II investigation of IGIV in patients with cardiac-associated AL amyloidosis. Its stated rationale is that human immune globulin might affect abnormal cells, while its stated purpose is to study side effects and dose and to assess how well treatment works. These are the study’s aims, not findings.
The posted primary question is narrower than the broader therapeutic aim: it concerns tolerance, assessed through the number and severity of toxicity incidents. The record also lists two outcome measures, but details for a second outcome and results for either measure are not reported in the registry record. The available safety figures therefore should not be mistaken for a complete account of all trial outcomes.
Because the design has a single group, the study can describe what was recorded among participants who received IGIV, but it cannot make a controlled estimate of how those observations compare with what would have occurred without IGIV or under another treatment. The registry record does not report a comparator, treatment effect estimate, or analysis addressing that question.
3. Trial Design
This is a completed phase I/II study registered as NCT00547365. The sponsor is the University of Tennessee, and the registry lists enrollment of 10. The study dates recorded are a start in 2007-10 and primary completion in 2011-07. These dates and status describe the registry entry; they do not supply details about recruitment, follow-up completeness, or the timing of any individual participant’s treatment.
The design is listed as single group. Allocation is not applicable, and masking is listed as none. The intervention is human immune globulin intravenous (IGIV), classified as a biological intervention. There is no separate control arm in the registry record. Consequently, the study is not a randomized comparison, and its observations cannot isolate an intervention effect from changes over time, participant characteristics, or other influences.
The registry’s brief description says the phase I/II trial was intended to study side effects and the best dose, and to see how well IGIV works in patients with primary amyloidosis causing heart dysfunction. The intervention description says the aim was to analyze therapeutic potential in cardiac-associated AL amyloidosis. The specific dose levels, dose-escalation process, treatment schedule, eligibility criteria, and protocol procedures are not reported in the registry record.
The primary outcome is tolerance, expressed as the number and severity of toxicity incidents among patients who had at least one infusion. Its timeframe is up to 1 year. The registry lists two outcome measures and no analyses. It does not report a primary endpoint result, an analysis population count beyond the stated endpoint population definition, or a statistical analysis plan. These omissions limit what can be reconstructed about how the study was conducted and how its planned questions were evaluated.
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: Patients with primary light-chain (AL)-associated amyloidosis that caused heart dysfunction were on study.
Overall Study
| Stage | Human Immune Globulin Intravenous (IGIV) |
|---|---|
| STARTED | 10 |
| COMPLETED | 2 |
| NOT COMPLETED | 8 |
Reasons for not completing this period:
| Reason | Human Immune Globulin Intravenous (IGIV) |
|---|---|
| Physician Decision | 1 |
| Lost to Follow-up | 1 |
| Protocol Violation | 2 |
| death (not related to study) | 4 |
Analysis population for the primary outcome, as defined in the registry: All patients who had at least one infusion of human immune globulin intravenous.
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.
Population: Patients with cardiac-dominant AL amyloidosis, as determined from standard clinical tests (IVS, BNP),were entered on study.
| Characteristic | Unit | Human Immune Globulin Intravenous (IGIV) |
|---|---|---|
| Participants analysed | 10 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 4 |
| Age, Categorical: >=65 years | Participants | 6 |
| Sex: Female, Male: Female | Participants | 5 |
| Sex: Female, Male: Male | Participants | 5 |
| Region of Enrollment: United States | participants | 10 |
6. Primary Endpoint
The registry lists the primary outcome as Tolerance for Human Immune Globulin Intravenous (IGIV), as Reflected by the Number and Severity of Toxicity Incidents Occurring in Ten Patients Receiving at Least One Infusion of IGIV., measured over Up to 1 year.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | events |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is titled as tolerance for IGIV and is defined through the number and severity of toxicity incidents occurring among patients who received at least one infusion. Its unit is events, its parameter is a number, and its timeframe is up to 1 year. The endpoint population is described as all patients with at least one IGIV infusion. The registry does not provide an observed event count, a summary of severity, or a result for this endpoint.
A toxicity-event count is a way to describe the burden of recorded events during treatment and follow-up. Considering severity as well as number can distinguish a count of events from their clinical seriousness; an event count alone would not convey that distinction. In general, such an endpoint is relevant in an early-phase investigation because tolerability and safety are among the stated study aims. That is a general explanation of the endpoint’s role, not a claim about what happened in this study.
The endpoint title does not specify how toxicity was defined, graded, assessed, or attributed to IGIV. Those details are not reported in the registry record. Nor does the record state whether each event was counted separately, how recurrent events were handled, or how incomplete follow-up was addressed. Without the endpoint result and its operational details, the posted information does not support a quantitative account of tolerance over the stated follow-up timeframe.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record describes a completed, single-group study with enrollment of 10, but it does not post a result for the primary tolerance endpoint. In particular, it gives no total number of toxicity incidents, no severity breakdown, and no estimate with a confidence interval. It also lists no analyses. Thus, the central endpoint question cannot be answered from the posted outcome information.
The safety summary reports 0 serious adverse events among 10 participants at risk and 0 other adverse events among 10 participants at risk in the IGIV group. These are the posted figures and should be read as recorded counts for the categories shown. They do not substitute for the primary endpoint’s event count and severity description: the registry does not state that these adverse-event categories and the endpoint’s toxicity incidents are defined identically. The record lists no serious adverse-event terms. Deaths are not reported in the registry record.
These safety figures are descriptive. A zero count in a group of this size does not establish that an event is impossible, that no event would occur with longer observation, or that the intervention caused no harm. The record does not provide enough detail about event definitions, ascertainment, follow-up, or attribution to make broader safety claims. Likewise, the figures cannot establish a comparative safety advantage because there is no control group.
The broader stated aim included assessing how well IGIV works, but the posted information does not include an efficacy outcome result or an analysis. The registry lists two outcome measures, while only the primary measure’s description is available; details and results for the other measure are not reported in the registry record. No treatment effect, precision estimate, or inferential conclusion can therefore be drawn from the record.
In general, confidence intervals describe the range of estimates compatible with the data and a specified method, and help communicate precision. No confidence interval for the primary endpoint is reported. The lack of a posted endpoint estimate is not evidence of a null effect; it means the registry entry does not show an estimate to interpret. The appropriate reading is limited to the design and the recorded adverse-event counts, alongside the substantial uncertainty created by missing outcome results and the absence of a comparator.
9. Statistical Methods Explained
What does a single-group design allow?
The study is listed as single group, with no separate comparator. In general, a single-group study can describe outcomes observed among participants who receive an intervention. It does not, by itself, provide a concurrent estimate of what would have happened to comparable participants without that intervention. A change observed over time could reflect the intervention, the course of the underlying condition, other care, measurement variation, or several influences together. Without a control group, those explanations cannot be separated by a direct between-group comparison.
Allocation is listed as not applicable and masking as none. Those features are consistent with the registry’s single-group description, but they do not tell us which endpoint assessments were blinded or how outcome ascertainment was managed. Those details are not reported in the registry record. The absence of masking can matter in general when judgments about event presence or severity involve discretion, although the available record does not establish whether that issue affected this endpoint.
How should an event-count endpoint be understood?
The primary endpoint uses a number of toxicity incidents, with severity also named in the endpoint title. Event counts are different from the proportion of participants with at least one event: a participant may experience more than one incident, depending on the endpoint rules. The registry does not state whether repeated incidents were counted separately, how events were defined, or how severity was classified. It reports the unit as events, but gives no observed count or severity distribution.
In general, event counts can be unevenly distributed. Some participants may have no events while others have multiple events. When the variability in counts is greater than expected under a simple count model, that is called over-dispersion. Over-dispersion matters because a model that assumes too little variability can produce uncertainty estimates that are too narrow. This is a general statistical consideration, not a claim that the study’s data were over-dispersed. The record contains no event-count data or analysis with which to assess that issue.
Time at risk can also matter for event counts. Participants followed for different durations may have different opportunities for an event to be recorded. The registry gives a timeframe of up to 1 year, but does not report participant-level follow-up, exposure duration, or whether the endpoint was analyzed as a count over a fixed period or as a rate. It would therefore be inappropriate to infer an incidence rate or compare participants’ event experience using information absent from the record.
What can the posted safety summary tell us?
The adverse-event summary presents serious and other adverse events separately for the IGIV group, with participants at risk and participants affected. It reports 0 serious events among 10 at risk and 0 other events among 10 at risk. Such figures are descriptive counts. In general, a count of zero in a finite group does not show that the underlying risk is exactly zero. A reliable assessment also depends on how events were defined, how long participants were observed, and how completely events were collected. The registry does not supply those details.
Serious adverse events are a specific safety category and should not automatically be treated as interchangeable with all toxicities. The primary endpoint refers to toxicity incidents and severity; the adverse-event summary categorizes serious and other adverse events. Because the registry does not provide definitions or a mapping between these categories, the safety summary cannot fill in the missing primary endpoint result. The record also does not list serious-event terms, and deaths are not reported in the registry record.
What does an analysis require?
Statistical analysis starts with a defined outcome, a specified population, and rules for handling incomplete observations. The registry identifies the primary endpoint population as patients with at least one infusion, but does not report how many had an infusion in the final analysis, whether all were followed through the intended period, or how missing observations were handled. The study’s enrollment is 10, but enrollment alone does not establish how many contributed complete endpoint data.
No analyses are listed. The statistical methods, hypothesis, estimation approach, and precision measures are not reported in the registry record. A confidence interval, when available, communicates uncertainty around an estimate under a stated method; it would not repair the limitations of a single-group design or missing endpoint details. Here, no primary endpoint estimate or interval is posted.
The accompanying sample-size material is described as a planning illustration, not observed power. Its stated assumptions include a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. Those assumptions describe hypothetical planning calculations; they are not the design of this single-group trial. The record does not report the trial’s own sample-size justification, and the illustration should not be interpreted as evidence that the study had a particular probability of detecting an effect.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists two outcome measures, but only the primary tolerance endpoint is described in the available record. The other outcome’s definition, timing, analysis, and result are not reported in the registry record. No analyses are listed. The record does not report whether the investigators used a statistical analysis plan or how they handled multiple outcomes or repeated analyses.
In general, multiplicity arises when several outcomes, comparisons, time points, or analyses are considered. Examining multiple results can increase the chance that an apparently notable finding occurs by chance. Methods for addressing that issue depend on the planned analyses and the study’s goals. Here, the relevant analysis details are absent, so it is not possible to assess whether multiplicity was a concern or how it was handled. There is no basis for reconstructing an adjustment or selecting one after the fact.
The primary endpoint population is defined as all patients with at least one infusion, and the overall enrollment is 10. The registry does not state whether all enrolled participants received an infusion, how many were included in the endpoint assessment, or whether any data were missing. Missing-data methods, reasons for missingness, and sensitivity analyses are not reported in the registry record. In general, missing outcomes can affect both the estimate and its interpretation, particularly when missingness is related to health status or treatment experience; the record does not show whether that occurred.
The registry also does not describe an intention-to-treat population, a per-protocol population, or another analysis population beyond the endpoint’s wording. It does not report participant exclusions or deviations from treatment. These omissions prevent an assessment of whether the analysis population matched the enrolled group or the population implied by the stated endpoint.
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 |
|---|---|---|---|
| Human Immune Globulin Intravenous (IGIV) | 0 | 10 | 0.0% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 1% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Human Immune Globulin Intravenous (IGIV) | 0 of 10 |
The posted adverse-event figures show 0 serious adverse events among 10 participants at risk and 0 other adverse events among 10 participants at risk in the IGIV group. The registry lists no serious-event terms. These are descriptive safety counts for the categories displayed; they are not the missing result for the primary endpoint, which concerns the number and severity of toxicity incidents.
A zero recorded count does not establish that the risk is zero. Interpretation depends on event definitions, follow-up, exposure, and completeness of reporting. The record gives the primary endpoint timeframe as up to 1 year, but does not provide participant-level follow-up or details on adverse-event collection. Deaths are not reported in the registry record, which is different from a reported death count of zero.
Because the study has no comparator group, the figures cannot establish how the observed counts compare with another treatment or with no IGIV. They also do not establish that any event, or absence of an event, was caused by IGIV. The safest reading is limited to the counts posted and their reporting context.
12. What the Result Does, and Does Not, Mean
This record documents a completed phase I/II, single-group investigation of IGIV in cardiac-associated AL amyloidosis. Its stated aims included studying side effects and dose and assessing therapeutic potential. The posted primary endpoint focuses on tolerance, measured by the number and severity of toxicity incidents among patients who had at least one infusion, over a timeframe of up to 1 year.
What the posted results do show is limited: the adverse-event summary records 0 serious adverse events among 10 at risk and 0 other adverse events among 10 at risk. What it does not show is the primary endpoint result, a severity breakdown, an efficacy result, a confidence interval, or an analysis. The record lists two outcome measures but does not provide details and results for both. The absence of those results from the registry entry cannot be interpreted as evidence that the outcome was favorable, unfavorable, or unchanged.
In general, early-phase studies may provide descriptive information about treatment administration and observed events. This study’s single-group design cannot establish a causal treatment effect through comparison with a control group. Even a complete event description would need to be interpreted in light of the study population, follow-up, outcome definitions, and possible sources of bias. Those details are not all reported in the registry record.
The record therefore supports a careful description of the study’s design, aims, and posted safety counts, but not a conclusion about whether IGIV improved outcomes or how its risks compare with alternatives. It also does not report enough information to judge the precision of the primary endpoint estimate, because no estimate is posted. This distinction between a study’s intended question and the results actually available is essential when reading a registry entry.
13. Why This Trial Matters Statistically
This trial is statistically notable because its stated aims include both tolerance and therapeutic potential, while its registered design is single group. That design can describe observations among treated participants, but it does not provide a controlled comparison. The distinction matters: a treatment question about what would happen relative to another option cannot be answered by a within-group description alone.
The primary endpoint is an event count that also refers to severity. Such a measure can be informative when its definitions and results are fully reported. In this record, however, the endpoint’s observed count and severity breakdown are absent. The separate adverse-event summary gives zero recorded serious and other events in the group, but does not replace the endpoint result. Keeping these measures distinct avoids treating unlike summaries as equivalent.
The record also illustrates why reporting completeness affects statistical interpretation. Without endpoint results, analysis methods, missing-data information, or details for the second outcome measure, readers cannot evaluate an effect estimate, its precision, or the handling of multiple outcomes. The enrollment figure alone does not resolve those uncertainties.
Finally, planning calculations should be separated from observed evidence. The accompanying sample-size material is described as a planning illustration with two-arm assumptions, whereas this trial is single group. Such illustrations are hypothetical planning tools, not observed power and not a substitute for results. The statistical value of the record lies in showing the design and the limits of what is posted, rather than in supporting a treatment-effect conclusion.
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 central limitation for interpretation is that the registry record does not report the primary endpoint result. Although tolerance is defined through the number and severity of toxicity incidents, no observed count or severity summary is posted. The record also lists two outcome measures without supplying the other measure’s details and results. This prevents a complete account of the outcomes the study intended to assess.
The study is single group, with no comparator and no masking. This limits causal interpretation and prevents a like-for-like estimate against another treatment or control condition. The record does not report the dose, schedule, detailed eligibility criteria, endpoint definitions, or event-grading procedures. These omissions make it difficult to judge how treatment exposure and outcomes were operationalized.
Enrollment is listed as 10, but the registry does not report the number contributing complete primary endpoint data, participant-level follow-up, or missing-data handling. The primary endpoint timeframe is up to 1 year, but the record does not say how long each participant was observed. Deaths are not reported in the registry record. No analyses are listed, and the statistical plan, sample-size justification, and handling of multiple outcomes are not reported.
The safety summary records zero serious and zero other adverse events among 10 at risk, but the record does not explain event ascertainment or how these categories relate to the toxicity endpoint. These limitations are specific to the information available for this trial; they do not establish that procedures or analyses were absent, only that they are not reported in the registry record.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Human Immune Globulin in Treating Patients With Primary Amyloidosis That is Causing Heart Dysfunction — NCT00547365.
- Registry record last updated 2013-09-19; status last verified 2013-09.
- Results first posted 2013-09-19.
- 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
NCT00547365 is a completed phase I/II, single-group study sponsored by the University of Tennessee. The registry lists enrollment of 10 and an intervention of human immune globulin intravenous (IGIV) for patients with cardiac-associated AL amyloidosis. Allocation is not applicable and masking is none.
The primary endpoint concerns tolerance, measured by the number and severity of toxicity incidents among patients who received at least one infusion, over up to 1 year. The registry lists two outcome measures and no analyses. It does not report the primary endpoint result or the details and result of the other measure.
The posted safety summary records 0 serious adverse events among 10 at risk and 0 other adverse events among 10 at risk. No serious-event terms are listed; deaths are not reported in the registry record. The single-group design and missing outcome analyses do not support a controlled treatment-effect conclusion.