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 Study to Assess Feasibility and Safety of Iron Repletion With Feraheme in Iron Deficient Patients With Durable Ventricular Assist Device Support (NCT04080908), a na phase 4 study. The primary outcome is Percentage of Patients Who Successfully Complete Ferumoxytol Injection Treatment.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT04080908 |
| Phase | Phase 4 |
| Condition | Anemia |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 1 |
| Arms in the primary outcome | n/a |
| Lead sponsor | NYU Langone Health |
| Results posted | Yes |
2. Clinical Question
Population
The intended population was people with laboratory evidence of iron deficiency after placement of a ventricular assist device (VAD), in the setting of durable VAD support. The registry lists anemia as a condition. The brief summary describes a pilot intended for 20 eligible subjects. The record does not provide additional eligibility criteria or describe the characteristics of the enrolled participant.
Intervention
The intervention was ferumoxytol injection, also called Feraheme in the study title and summary. The study was open label, so masking was listed as none. The record describes the study as testing the feasibility and safety of iron repletion with this treatment; it does not report a comparison with another treatment or a control group.
Comparator
There was no comparator arm. Allocation is listed as not applicable, and the study model is single group. Therefore, the trial cannot make a randomized, like-for-like comparison between ferumoxytol and another treatment or usual care. Any interpretation should remain within the limits of a single-arm pilot.
Primary question
The primary outcome was the percentage of participants who successfully completed ferumoxytol injection treatment by Month 6. The registry describes the measure as a mean with standard deviation. Although the outcome is defined, the registry record does not report a numerical result or analysis. As a result, it is not possible to estimate completion from the posted results or assess the precision of an observed completion estimate.
In practical statistical terms, the study was asking whether treatment completion could be assessed in the intended population and whether safety events were recorded during the study. The record does not explain the operational definition of successful completion, so the precise criteria for classifying a participant as completing treatment are not reported in the registry record. The record also does not report reasons for termination. Those omissions limit how specifically the study question can be interpreted.
3. Trial Design
The registry describes an open-label, single-arm pilot study of ferumoxytol injection in iron-deficient patients with durable VAD support. It is listed as Phase 4, with no allocation and no masking. Because there was no control group, the design supports descriptive reporting of treatment completion and adverse events, but not a randomized estimate of a difference between treatments. The open-label design also means that participants and study personnel were not masked to treatment, according to the registry entry.
The brief summary says the pilot was intended to test feasibility in 20 eligible subjects. The enrollment recorded in the registry is 1. The record lists a start date of 2020-01-15, a primary completion date of 2021-10-19, and a status of terminated. It does not report why the study was terminated or explain the difference between the intended enrollment described in the summary and the enrollment recorded in the registry.
NYU Langone Health is listed as the sponsor. The intervention is ferumoxytol injection, and the primary outcome concerns successful completion of that treatment by Month 6. The registry lists one outcome measure and no analyses. It does not report details of the statistical analysis plan, the planned analysis population, or whether any interim review was planned. The absence of those details should not be filled in by assuming customary procedures.
The study’s design and recorded enrollment constrain what can be learned from it. A single-arm pilot can describe what was recorded among participants in that group, but it cannot separate treatment effects from other influences using a concurrent control. With no reported primary-endpoint estimate, the record also does not allow a descriptive assessment of the observed completion outcome. The design may be useful for framing feasibility questions, but the posted record does not provide enough outcome information to answer them numerically.
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 | Ferumoxytol Injection Treatment |
|---|---|
| STARTED | 1 |
| COMPLETED | 1 |
| NOT COMPLETED | 0 |
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 | Ferumoxytol Injection Treatment |
|---|---|---|
| Participants analysed | 1 | |
| Age, Continuous | years | 41 (SD 0) |
| Sex: Female, Male: Female | Participants | 0 |
| Sex: Female, Male: Male | Participants | 1 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 1 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| 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 | 0 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Region of Enrollment: United States | participants | 1 |
6. Primary Endpoint
The registry lists the primary outcome as Percentage of Patients Who Successfully Complete Ferumoxytol Injection Treatment, measured over Month 6.
| Element | Registry entry |
|---|---|
| Result type | Mean |
| Measure of spread | Standard Deviation |
| Unit | percentage of participants |
| Endpoint classification | Continuous Endpoint |
The primary endpoint is the percentage of participants who successfully complete ferumoxytol injection treatment, assessed at Month 6. It targets a feasibility outcome: whether participants complete the treatment course. In a feasibility study, completion can describe whether the planned treatment process was carried through, rather than directly measuring a clinical benefit. The registry does not state the specific criteria used to define successful completion, so that operational definition is not reported in the registry record.
The registry characterizes the outcome as a mean with standard deviation. In general, when a percentage is formed from a participant-level indicator of completion, its mean can represent the average completion proportion in the analyzed group. The standard deviation describes variability in the measured values; it is not itself a measure of how precisely the mean is estimated. Precision is commonly conveyed with a confidence interval, which represents uncertainty around an estimate under the chosen statistical method.
No numerical primary-endpoint result, standard deviation, confidence interval, or analysis is reported in the registry record. Therefore, the endpoint definition alone does not reveal how many participants completed treatment or how uncertain an estimated completion percentage would be. The recorded enrollment of 1 does not substitute for a reported endpoint result: enrollment describes participation, not successful treatment completion. Nor should the safety counts be interpreted as completion data.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry identifies a clear primary question: the percentage of participants successfully completing ferumoxytol injection treatment by Month 6. However, it does not report a numerical outcome estimate or an analysis for that endpoint. The posted results therefore do not establish an observed completion percentage, its variability, or its precision. The endpoint’s title is a definition of what was intended to be measured, not a result.
Enrollment is recorded as 1, while the brief summary describes an intended pilot in 20 eligible subjects. These are different kinds of information: the summary describes the intended study size, whereas the enrollment field reports the enrollment recorded in the registry. The record does not explain the discrepancy. It also does not report the reason for the terminated status. No conclusion about why enrollment was limited or why the study ended can be drawn from these entries alone.
In general, a confidence interval helps readers judge the range of values compatible with an estimate under the analysis assumptions. It is not reported for the primary endpoint, and there is no reported estimate around which to assess uncertainty. A p-value is also not reported. Even if such a statistic were available, it would not by itself describe the size or practical importance of a result. Here, the absence of a reported endpoint estimate means those inferential questions cannot be evaluated from the registry record.
The design was single group, open label, and had no comparator. Descriptive counts in a single group can document what was recorded among participants, but cannot establish how outcomes would have differed under another treatment or under no treatment. This is especially important for a feasibility endpoint: without a comparator, the study is not a randomized comparison of treatment completion. The record does not provide enough information to assess potential influences on completion, such as the reasons for noncompletion or the criteria used to classify completion.
The safety entries report no serious adverse events among the participant listed at risk for serious events, and one other adverse event among the participant listed at risk for other events. These are descriptive registry counts. They do not establish the frequency of events beyond the recorded group, and the lack of a comparator prevents comparative safety interpretation. The record lists no deaths. These safety entries do not supply the missing primary-endpoint result.
Overall, the registry supports a limited description of the intended question, design, enrollment, and recorded safety counts. It does not support a numerical conclusion about treatment completion, a comparative treatment effect, or a precise estimate of safety. The terminated status and limited reported outcome information make careful separation of design intent from observed results essential.
9. Statistical Methods Explained
The analysis methods cannot be fully reconstructed from the registry record. It identifies a continuous outcome summarized as a mean with standard deviation, but lists no analyses and provides no numerical primary result. The statistical analysis plan, analysis population, handling of missing outcomes, and inferential procedures are not reported in the registry record. The following concepts explain how such methods are generally interpreted; they do not describe procedures confirmed to have been used in this study.
What does a mean describe?
In general, a mean is an average of measured values. For an outcome expressed as a percentage of participants completing treatment, the mean may summarize the proportion classified as completing in the analyzed group, depending on how the outcome was coded and analyzed. The registry lists the endpoint as a mean, but does not specify the participant-level coding or explain how the percentage was calculated. It also does not provide the observed mean. The endpoint label alone therefore cannot be used to infer the result.
A standard deviation describes how much individual measurements vary around their mean. It is not interchangeable with a confidence interval: the standard deviation characterizes variation in the observations, while a confidence interval expresses uncertainty in an estimated quantity under a statistical model. Neither a standard deviation nor a confidence interval is reported for this endpoint. In general, estimates based on limited observations tend to be less informative about variation and precision, but no numerical precision assessment can be made from this record.
What can a single-arm design estimate?
A single-arm design records outcomes in a group receiving the intervention, without a concurrent control group. It can be used to describe observed completion or event counts, if those results are reported. It does not provide a randomized comparison against another treatment. Differences from an external expectation or historical experience would require additional assumptions and information; the registry does not describe such a comparison. Accordingly, any interpretation should distinguish a descriptive observation from evidence of a treatment effect.
Open-label status means masking was listed as none. In general, lack of masking can matter when outcomes involve judgment, reporting, or decisions influenced by knowledge of treatment. The degree to which that concern applies depends on how outcomes are defined and collected. The record does not report details about outcome ascertainment, so the possibility and extent of such influence cannot be assessed for this study.
How should the planning illustration be read?
The assumptions described for the sample-size illustration are not the trial’s reported design. They specify a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with an effect defined as a stated fraction of the observed effect. The actual trial is single group, with allocation listed as not applicable. Thus, the illustrative assumptions do not describe a like-for-like analysis of this trial. Any sample-size outputs based on those assumptions are planning illustrations, not evidence of observed power and not a retrospective test of the study’s success.
In general, a sample-size calculation depends on the assumed effect, variability, allocation, significance level, and statistical method. If the observed effect is used as a starting point, changing the fraction of that effect changes the planning question. Such calculations do not establish that the assumed effect is true, nor do they repair missing outcome data. The registry does not report the study’s own sample-size justification or protocol-based power calculation. No conclusion about the original planning rationale should be inferred from the illustration’s assumptions.
What is not available for an inferential analysis?
The registry reports no analyses for the outcome and no numerical estimate from which to evaluate uncertainty. It also does not report a p-value, confidence interval, covariate adjustment, or a model specification. These items are not interchangeable: an estimate describes the observed effect or outcome, an interval conveys uncertainty under a method, and a test statistic addresses a particular null hypothesis. Without the estimate and analysis details, statistical significance and precision cannot be assessed.
General methods for percentages may account for the discrete nature of participant-level completion outcomes, and some methods may be preferable to a normal approximation in particular settings. The appropriate choice depends on the data and analysis plan. The registry does not report which method, if any, was used. There is also no reported variance estimate or outcome distribution to support assessment of model assumptions. It would therefore be inappropriate to assign a specific inferential method to the study or recalculate a result from the endpoint description alone.
The record reports an enrollment of 1, but does not state whether that participant belongs to the primary analysis population or whether the endpoint was observed. General statistical interpretation requires knowing who was included, what outcome data were available, and how missing values were handled. Those details are not reported in the registry record. Consequently, no numerical reanalysis or evaluation of the endpoint’s uncertainty is possible from the posted results.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists one primary outcome measure and no analyses. It does not report a statistical analysis plan or state whether any additional outcomes or hypotheses were planned outside the listed registry information. A multiplicity strategy, such as an adjustment for testing multiple outcomes, is not reported in the registry record. With no reported analyses or p-values, the effect of any multiplicity procedure cannot be assessed.
Missing-data handling is also not reported. The registry does not say whether the primary outcome was observed, how participants without an endpoint assessment would be treated, or whether any imputation or sensitivity analysis was planned. These details matter because analyses can answer different questions depending on how missing observations are handled. No missing-data method should be assumed.
The analysis population is not reported in the registry record. The enrollment field records 1, but that fact alone does not establish whether the participant was included in an endpoint analysis, completed the treatment, or had an assessment at Month 6. No participant flow or analysis denominator is reported for the primary endpoint. The outcome’s numerical result and analysis are absent, so the registry does not permit evaluation of exclusions or the relationship between enrollment and analysis.
In general, multiplicity concerns arise when several statistical questions are tested and the chance of a misleading result can increase if the testing strategy is not prespecified. Missingness concerns arise when outcome availability is incomplete or related to participant characteristics or outcomes. These are general considerations, not claims about what occurred in this study. The record does not provide enough detail to determine whether either issue affected an analysis.
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 |
|---|---|---|---|
| Ferumoxytol Injection Treatment | 0 | 1 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Ferumoxytol Injection Treatment | 0 of 1 |
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 |
|---|---|
| Ferumoxytol Injection Treatment | 1 of 1 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Ferumoxytol Injection Treatment |
|---|---|---|
| Hospitalization - planned heart transplant, ICD and VAD removal | Surgical and medical procedures | 1 of 1 |
| Pneumoperitoneum | Injury, poisoning and procedural complications | 1 of 1 |
| Steriod Induced Hyperglycemia | Endocrine disorders | 1 of 1 |
| left shoulder disorder | Musculoskeletal and connective tissue disorders | 1 of 1 |
The registry lists one participant at risk for serious adverse events, with none recorded as affected. It lists one participant at risk for other adverse events, with one recorded as affected, and lists no deaths. These are the safety counts recorded for the ferumoxytol injection treatment group. The registry does not list serious adverse-event terms among its top serious terms.
These counts are descriptive. They indicate what the record reports for the listed group, but do not estimate comparative risk because there was no control arm. The record does not provide further detail about the nature, timing, severity, or attribution of the other adverse event. It also does not report a broader safety analysis or explain how events were collected and classified. The serious-event count should not be taken as proof that serious events could not occur; it is a count in the registry record for a group with limited recorded enrollment.
Safety counts should also be kept separate from the primary endpoint. The primary outcome concerns successful treatment completion by Month 6, and no numerical result for that outcome is reported. An adverse-event count does not indicate whether treatment was completed, and the record does not describe any relationship between the event and completion.
12. What the Result Does, and Does Not, Mean
The record defines a feasibility and safety question for ferumoxytol injection in iron-deficient patients with durable VAD support. It reports an open-label, single-arm pilot design, an intended enrollment described as 20 eligible subjects in the brief summary, and registry enrollment of 1. It also reports a terminated status and limited safety counts. These registry entries describe the registered study and its recorded information; they do not supply a numerical answer to the primary question.
The primary endpoint is the percentage of participants who successfully complete treatment by Month 6. No numerical endpoint result or analysis is reported in the registry record. Therefore, the posted results do not show an observed completion percentage or its precision. The record also does not report the criteria for successful completion, the analysis population, or how missing outcome data were handled. It is not possible to determine from the endpoint title alone whether the intended feasibility outcome was achieved.
The safety entries describe counts within the single treatment group, not a comparison with another treatment. Without a control group, the record cannot estimate a difference in event rates between treatments. A recorded absence of serious adverse events in the listed group is not evidence that risk is absent, and the other adverse-event count does not establish an event rate beyond that group.
More broadly, the registry record does not establish whether ferumoxytol changes clinical outcomes, nor does it establish comparative effectiveness or safety. Those questions require appropriate outcome data and a design and analysis capable of addressing them. The available record is best read as documenting the study’s intended question, design, enrollment field, status, and limited adverse-event information, while leaving the central numerical endpoint unanswered.
13. Why This Trial Matters Statistically
This trial matters statistically because its stated purpose was feasibility, while the central registered endpoint is treatment completion. Feasibility outcomes can help describe whether a study procedure is carried through, but their interpretation depends on a clear operational definition and complete outcome reporting. The registry identifies the endpoint and its Month 6 timeframe, yet does not report the success criteria or a numerical result. This makes the distinction between defining an outcome and observing it particularly important.
The contrast between the intended enrollment described in the brief summary and the enrollment recorded in the registry also matters for interpretation. The record does not explain that difference or the terminated status. Without such context, readers cannot determine whether the discrepancy reflects a change in plans, recruitment experience, or another circumstance. No explanation should be inferred from the numbers alone.
The single-arm design illustrates a central limitation of descriptive evidence. Even when outcomes are reported, a single group cannot provide a randomized estimate of how outcomes compare with another treatment. In this record, the absence of a numerical primary result further limits descriptive interpretation. The safety counts are useful as recorded observations, but they cannot resolve comparative safety questions.
Finally, the planning assumptions described for sample-size illustrations do not match the single-group trial design: they posit equal allocation between two arms. This distinction helps prevent planning calculations from being mistaken for trial results or observed power. A sample-size illustration addresses a hypothetical design under stated assumptions; it does not fill gaps in outcome reporting or explain the trial’s conduct. The statistical importance of this record lies partly in recognizing those boundaries.
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 most direct limitation is that the numerical primary-endpoint result is not reported in the registry record, despite the endpoint being defined as successful treatment completion by Month 6. The record also lists no analyses. Without the estimate, the completion outcome cannot be described numerically, and its uncertainty cannot be assessed.
The recorded enrollment is 1, whereas the brief summary describes a pilot intended for 20 eligible subjects. The registry does not explain the discrepancy. The study is marked terminated, but the reason for termination is not reported in the registry record. These missing explanations prevent a clear account of how the study’s conduct related to its stated plan.
The record does not provide the operational definition of successful completion, additional eligibility criteria, participant characteristics, analysis population, or participant flow. Missing-data procedures and the statistical analysis plan are also not reported. Without those details, readers cannot determine how the outcome would have been classified or how the analysis would have treated incomplete information.
The single-arm, open-label design limits comparative interpretation. There is no randomized control group, and the record does not provide a comparative estimate. The safety entries are limited to the counts recorded for the treatment group and do not include detailed event descriptions or a comparative safety analysis. Finally, the assumptions described for planning illustrations involve two arms and equal allocation, unlike the registered single-group design. Those illustrations should not be treated as a description of the trial’s own design or analysis.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Study to Assess Feasibility and Safety of Iron Repletion With Feraheme in Iron Deficient Patients With Durable Ventricular Assist Device Support — NCT04080908.
- Registry record last updated 2023-07-18; status last verified 2023-06.
- Results first posted 2023-07-18.
- 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
The registry describes a Phase 4, open-label, single-arm pilot of ferumoxytol injection for iron repletion in iron-deficient patients with durable VAD support. NYU Langone Health is listed as the sponsor. The record lists anemia as a condition, no allocation, no masking, and a terminated status. It records a start date of 2020-01-15 and a primary completion date of 2021-10-19.
The brief summary describes an intended pilot in 20 eligible subjects, while registry enrollment is recorded as 1. The primary endpoint is the percentage of participants who successfully complete treatment by Month 6, summarized as a mean with standard deviation. No numerical endpoint result or analysis is reported. Safety entries list no serious adverse events, one other adverse event, and no deaths in the recorded group. The reason for termination and the enrollment discrepancy are not reported in the registry record.