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 Use of Intravenous Ferric Carboxymaltose in Treatment of Anemia in Cancer Patients Undergoing Chemotherapy (NCT04246021), a na na study. The primary outcome is The Median Hb Change From Baseline to Week 12.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT04246021 |
| Phase | NA |
| Condition | Anemia, Cancer |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 84 |
| Arms in the primary outcome | n/a |
| Lead sponsor | King Hussein Cancer Center |
| Results posted | Yes |
2. Clinical Question
Population
The study concerns cancer patients with anemia undergoing chemotherapy, with or without radiotherapy. The registry record lists an enrollment of 84 and describes classification by baseline serum ferritin (sFr) and transferrin saturation (TSAT). It does not report the detailed eligibility criteria or participant characteristics beyond this description.
Intervention
The intervention was intravenous ferric carboxymaltose (FCM), also identified in the record by the names Ferrinject and ferrinject. The registered description says patients would receive one or two doses based on body weight and hemoglobin (Hb) level. The study was open-label.
Comparator
There was no separate control treatment group described. This was a nonrandomized, single-group study, with participants categorized by iron status rather than assigned to different treatments. Comparisons between those categories are not equivalent to randomized treatment comparisons.
Primary question
The primary endpoint asks for the median change in Hb from baseline to week 12, described for three iron-status groups. Those groups were absolute iron deficiency, functional iron deficiency, and others, based on sFr and TSAT criteria. The numerical median changes and their full ranges are not reported in the registry record.
The clinical question is therefore descriptive: how did Hb change over the stated follow-up period among patients treated with FCM, when results are classified by baseline iron status? The registry record also names secondary outcomes concerning response rate in relation to iron-deficiency status and the percentage of patients requiring blood transfusion or ESA treatment. It does not provide numerical findings for those outcomes in the information recorded. Because treatment was not randomized against a comparator, the study cannot by itself separate changes associated with FCM from changes related to cancer care, the passage of time, or other influences.
3. Trial Design
The registry describes a completed, nonrandomized, single-arm, open-label study. Its brief summary says it was stratified by iron-deficiency status. The registered intervention was ferric carboxymaltose, administered intravenously. The arm description states that patients would receive one or two doses based on body weight and Hb level; further dosing instructions in that description are incomplete in the registry record.
The study population was cancer patients with anemia undergoing chemotherapy, with or without radiotherapy. The registry lists an enrollment of 84. It does not provide detailed eligibility criteria, baseline characteristics, or information about how participants entered the study. Phase is recorded as NA, and allocation is also recorded as NA. Masking is recorded as none, consistent with the open-label description.
The primary outcome is the median Hb change from baseline to week 12, with the timeframe described as up to 12 weeks. The endpoint description divides participants into three groups using baseline sFr and TSAT: absolute iron deficiency (sFr < 30 ng/mL and TSAT < 20%), functional iron deficiency (sFr 30-800 ng/mL and TSAT < 20%), and others (TSAT >= 20%). The record gives group sizes of 26, 24, and 34, respectively. These are strata within the treated study, not randomized treatment arms.
The registered start date is 2016-06-15, the primary completion date is 2017-12-31, and the status is completed. The record lists five outcome measures and zero analyses. It does not report a statistical analysis plan, a sample-size justification, interim analyses, or the handling of deviations from the intended treatment. Those design and analysis details are not reported in the registry record.
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 | Ferric Carboxymaltose (Ferrinject) |
|---|---|
| STARTED | 84 |
| COMPLETED | 84 |
| NOT COMPLETED | 0 |
Analysis population for the primary outcome, as defined in the registry: median Hb change from baseline to week 12
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: A total of 84 adult cancer patients on active chemotherapy with hemoglobin (Hb) level ⩽11.0 g/dL were recruited. Based on serum ferritin (sFr) and transferrin saturation(TSAT), patients were divided into 3 groups: group I (absolute iron deficiency, n = 26) with sFr \< 30 ng/mL and TSAT \< 20%; group II (functional iron deficiency, n = 24) with sFr 30-800 ng/mL and TSAT \< 20%; and patients with TSAT ⩾ 20% were placed in group III as "others" (n = 34).
| Characteristic | Unit | Ferric Carboxymaltose (Ferrinject) |
|---|---|---|
| Participants analysed | 84 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 64 |
| Age, Categorical: >=65 years | Participants | 20 |
| Age, Continuous | years | 53.8 (SD 10.6) |
| Sex: Female, Male: Female | Participants | 70 |
| Sex: Female, Male: Male | Participants | 14 |
| Race and Ethnicity Not Collected | Participants | — |
| Region of Enrollment: Jordan | participants | 84 |
6. Primary Endpoint
The registry lists the primary outcome as The Median Hb Change From Baseline to Week 12, measured over up to 12 weeks.
| Element | Registry entry |
|---|---|
| Result type | Median |
| Measure of spread | Full Range |
| Unit | gm/dl |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is the median change in hemoglobin (Hb) from baseline to week 12, expressed in gm/dl. The registry describes the measure as a patient-level change summarized by its median and full range, with results separated by baseline iron-status group. The listed timeframe is up to 12 weeks.
Hb is a laboratory measure of the oxygen-carrying protein in red blood cells. In general, measuring change from baseline uses each participant’s starting value as a reference, so the summary concerns movement over follow-up rather than only the final Hb value. A median describes the middle of the observed changes when arranged in order; the full range describes the lowest and highest recorded changes. These summaries can help convey both a typical observed change and the spread of individual changes, although a range is sensitive to extreme observations.
The endpoint is stratified by sFr and TSAT categories, allowing the registry’s intended description to distinguish participants by baseline iron status. The categories are defined in the trial record, but a difference between their outcomes would not establish that iron status caused the difference. The study has no randomized comparator, and the record does not give the actual median changes, ranges, confidence intervals, or tests. Thus, the endpoint’s definition is available, but its numerical result and statistical precision are not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record defines a clinically interpretable outcome: change in Hb from baseline through week 12, summarized by median and full range within three baseline iron-status groups. It does not provide the numerical median change or range for any group. Consequently, the direction, size, and variability of the observed Hb changes cannot be described from the recorded results. No numerical estimate of the primary outcome or confidence interval is reported in the registry record.
The design also limits the comparisons that can be made. All participants received the same registered intervention, while the three groups reflect baseline iron-status classifications. The categories are not separate randomized treatment strategies, and there is no concurrent untreated or alternative-treatment group described. Any observed change over time could reflect several influences, including FCM, cancer treatment, changes in the underlying illness, or other care. Without a comparator, the study cannot identify the portion of change attributable to FCM alone.
Open-label conduct means masking was absent. This matters most for outcomes that can be influenced by expectations, decisions, or reporting; the extent of such influence depends on the outcome and its measurement process. Hb is a laboratory measure, but the record does not state laboratory procedures, assessor masking, or other safeguards. Those details are not reported in the registry record.
The record names secondary outcomes about response by iron-deficiency status and the need for blood transfusion or ESA treatment. Their definitions, numerical results, and uncertainty are not given in the record. It also lists five outcome measures but records zero analyses. This means the available registration information does not permit assessment of which analyses were performed or how estimates were calculated.
In general, a confidence interval communicates the range of effect estimates compatible with the data under the assumptions of an analysis. It helps distinguish a precisely estimated value from one surrounded by substantial uncertainty. No confidence interval for the primary endpoint is reported in the registry record, so the precision of any group-specific result cannot be assessed. The absence of a reported estimate is not evidence that the outcome did not change; it means the result is not available in this registry record.
The sample-size assumptions describe planning illustrations based on an observed effect, using a two-sided significance level of 0.05, equal allocation, and a normal approximation. Such illustrations concern hypothetical future planning, not the observed study’s power or the certainty of its findings. The actual illustration values are not reported in the registry record. Moreover, the registry does not report a study-specific sample-size justification, so these planning assumptions should not be treated as evidence about the original design.
9. Statistical Methods Explained
The statistical information that can be interpreted begins with the endpoint definition. The primary outcome is a change score: each participant’s Hb at baseline is compared with that participant’s Hb at follow-up, and the group summary is the median change with a full range. This is different from comparing only the group’s final Hb values. In general, change scores retain information about where participants began, but their interpretation depends on complete and comparable measurements at baseline and follow-up. The registry record does not describe the measurement schedule in detail or explain how missing Hb measurements were handled.
What does the median describe?
In general, the median is the central value after observations are ordered. It is less affected by extreme values than the arithmetic mean, which can be useful when individual changes are unevenly distributed. It does not show every participant’s experience, and it does not indicate how many participants had increases, decreases, or no change unless those details are separately reported. The full range gives the lowest and highest observed values, but can be strongly influenced by extreme observations. The record specifies median and full range as the summary approach, but does not report their numerical values for any iron-status group.
Why classify by baseline iron status?
The primary endpoint description uses sFr and TSAT to classify participants as having absolute iron deficiency, functional iron deficiency, or other status. The listed criteria are sFr < 30 ng/mL and TSAT < 20% for absolute iron deficiency; sFr 30-800 ng/mL and TSAT < 20% for functional iron deficiency; and TSAT >= 20% for the others group. Stratification can describe whether outcomes vary across clinically defined baseline categories. In general, it does not make those categories randomized. Differences between strata may reflect other participant characteristics or clinical circumstances as well as iron status.
What does the single-group design permit?
The record characterizes the study as nonrandomized and single-group, with no masking. The iron-status groups are subdivisions of that treated population, not a treatment group and a control group. In general, random assignment helps balance measured and unmeasured factors between treatment groups, while a concurrent comparator helps indicate what might have happened without the study treatment or with another treatment. Neither feature is described for this study. As a result, before-and-after changes can be described, but a causal treatment effect cannot be isolated from time-related change or other concurrent care using this design alone.
Open-label studies do not conceal treatment assignment from participants or study personnel. In general, awareness of treatment can influence subjective reports, care decisions, or additional interventions. The primary Hb measure is laboratory-based, but the registry record does not explain whether laboratory personnel were masked or provide details about measurement quality. The extent to which open-label conduct might affect a particular outcome therefore cannot be determined from the record.
What role does a confidence interval play?
In general, a confidence interval presents uncertainty around an estimated effect under specified statistical assumptions. A narrow interval indicates greater precision than a wide interval, though precision does not by itself establish that an effect is clinically important or causal. The registry record does not report confidence intervals for the primary endpoint, so readers cannot assess the statistical precision of the group summaries. A p-value, if reported, would address compatibility with a statistical null model rather than quantify the size or practical importance of an effect; no primary-outcome p-value is reported in the registry record.
How should planning illustrations be read?
The registry’s sample-size assumptions describe planning illustrations based on an observed effect, a two-sided significance level of 0.05, equal allocation, and a normal approximation. The assumed effect is described as a stated fraction of the observed effect. In general, a sample-size calculation estimates how many participants a hypothetical design would need under its assumptions; it is not an estimate of the probability that the completed study found a true effect. It is also not observed power. The numerical planning results and the effect used for those calculations are not reported in the registry record. These illustrations should not be mistaken for the study’s original design justification, which is not reported.
Over-dispersion is a general statistical concern when observed variability exceeds what a chosen model assumes. In general, a model that understates variability can give overly narrow uncertainty intervals or misleading tests. The registry record does not identify a model for the primary endpoint or report an assessment of distributional assumptions. It is therefore not possible to assess whether a model-based analysis was used or whether it accounted for unusual variability.
Finally, the record lists five outcome measures and zero analyses. This limits statistical interpretation beyond the endpoint definition: methods for estimating the summaries, handling missing observations, adjusting for covariates, or conducting subgroup comparisons are not reported in the registry record. No particular procedure should be inferred from the outcome description alone.
10. Multiplicity, Missing Data and Analysis Populations
The record lists five outcome measures, including the primary Hb-change endpoint and secondary outcomes concerning response in relation to baseline iron-deficiency status and the need for transfusion or ESA treatment. It records zero analyses. No multiplicity strategy is reported in the registry record. In general, when several outcomes or subgroup comparisons are examined, the chance of finding at least one apparently notable result can increase unless the analysis plan accounts for multiple testing. Without the analysis plan or reported results, it is not possible to determine which comparisons were conducted or how their interpretation addressed multiplicity.
Missing-data methods are not reported in the registry record. The primary endpoint requires baseline and follow-up Hb measurements, but the record does not state how many measurements were available, how missing follow-up values were treated, or whether the analysis included all enrolled participants. In general, complete-case summaries can differ from summaries that account for missing observations, especially if missingness is related to health status or outcomes. The direction and extent of any such effect cannot be inferred.
Analysis populations are also not described. The registry does not state whether the endpoint was analyzed among all enrolled participants, participants who received treatment, participants with a follow-up measurement, or another defined group. It does not report exclusions or protocol deviations. These omissions make it difficult to connect the listed enrollment with the population contributing to any endpoint result. The analysis population and missing-data handling are not reported in the registry record.
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 |
|---|---|---|---|
| Ferric Carboxymaltose (Ferinject) in Patients With Absolute Iron Deficiency Anemia | 0 | 26 | 0.0% |
| Ferric Carboxymaltose (Ferinject) in Patients With Functional Iron Deficiency | 0 | 24 | 0.0% |
| Others-Ferric Carboxymaltose(Ferinject) in Patients With Anemia | 0 | 34 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Ferric Carboxymaltose (Ferinject) in Patients With Absolute Iron Deficiency Anemia | 0 of 26 |
| Ferric Carboxymaltose (Ferinject) in Patients With Functional Iron Deficiency | 0 of 24 |
| Others-Ferric Carboxymaltose(Ferinject) in Patients With Anemia | 0 of 34 |
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 |
|---|---|
| Ferric Carboxymaltose (Ferinject) in Patients With Absolute Iron Deficiency Anemia | 17 of 26 |
| Ferric Carboxymaltose (Ferinject) in Patients With Functional Iron Deficiency | 6 of 24 |
| Others-Ferric Carboxymaltose(Ferinject) in Patients With Anemia | 16 of 34 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Ferric Carboxymaltose (Ferinject) in Patients With Absolute Iron Deficiency Anemia | Ferric Carboxymaltose (Ferinject) in Patients With Functional Iron Deficiency | Others-Ferric Carboxymaltose(Ferinject) in Patients With Anemia |
|---|---|---|---|---|
| hypophosphatemia | Investigations | 17 of 26 | 6 of 24 | 16 of 34 |
The serious adverse-event record lists three iron-status groups. Serious adverse events are recorded for 0 participants in each group, and deaths are recorded as 0 in each group. The other adverse-event counts are 17 in the absolute iron-deficiency group, 6 in the functional iron-deficiency group, and 16 in the others group. The record lists 26, 24, and 34 participants at risk in those respective groups. No serious adverse-event terms are listed.
These figures are counts, not estimates of comparative safety. In general, the absence of a recorded serious adverse event in a group does not establish that a treatment carries no serious risk, particularly when the record does not describe event definitions, ascertainment procedures, follow-up completeness, or the analysis population. The counts of other adverse events do not by themselves indicate severity, cause, duration, or whether events were judged related to treatment. The registry record does not provide that detail.
The groups are baseline iron-status strata within a single-group study, not randomized treatment comparisons. Differences in their event counts should therefore not be interpreted as evidence that risk differs because of iron status or treatment. The record does not report an adjusted safety analysis or a concurrent control group.
12. What the Result Does, and Does Not, Mean
The registry record identifies a completed, open-label study of intravenous FCM in cancer patients with anemia undergoing chemotherapy, with or without radiotherapy. It defines a primary outcome: median Hb change from baseline to week 12, summarized by baseline iron-status group. It also records serious and other adverse-event counts for those groups. These details describe what the study set out to assess and what safety counts were entered in the record.
The record does not report numerical primary endpoint results, including median changes or full ranges. It therefore does not establish the magnitude or direction of Hb change in any group, or how precisely any change was estimated. Numerical results for the named secondary outcomes are also not reported in the registry record. The absence of those findings from the record should not be interpreted as evidence of no change or no effect.
The design does not include a randomized comparator. The iron-status categories are not treatment arms, and the open-label, single-group structure cannot isolate the effect of FCM from other factors that may influence Hb over time. The study can be understood as a description of outcomes in a treated population, but the registry information does not support a controlled estimate of what would have happened under another treatment or without FCM.
The listed adverse-event figures report zero serious adverse events and zero deaths in each group, alongside counts of other adverse events. They do not establish that no safety concerns occurred: the record does not provide the detailed event terms, definitions, attribution, or completeness needed for that conclusion. Nor do the counts support a comparative safety claim, because no control group is described.
In general, statistical results should be considered alongside their precision, design, outcome definitions, and completeness. Here, the endpoint definition and study structure are available, but numerical efficacy results and important analysis details are not reported in the registry record. Conclusions should remain limited to those documented features.
13. Why This Trial Matters Statistically
This study is statistically informative as an example of the distinction between stratification and randomization. The registry describes three groups formed from baseline iron-status criteria, but all are within a nonrandomized, single-group study of FCM. Group-specific summaries can describe outcomes in those categories; they do not create a treatment-control contrast. This distinction matters whenever subgroup labels might otherwise be mistaken for separate treatment assignments.
The endpoint also illustrates the difference between defining a statistical measure and reporting its result. The record specifies a median change in Hb with a full range and a follow-up timeframe. Yet it does not report the numerical summaries. Without those estimates, readers cannot assess the observed change, its variability, or its precision. An endpoint description alone is not a finding.
For clinical researchers, the record highlights why complete reporting includes more than an outcome name. Analysis populations, missing-data handling, model assumptions, and multiplicity procedures affect how results can be interpreted. Those details are not reported in the registry record. The record’s sample-size assumptions are planning illustrations, not a substitute for a study-specific design justification or an observed result.
The safety counts provide another useful distinction: counts of recorded events are not comparative risk estimates. In a single-group study, they do not show how event frequency compares with what would have occurred under another treatment. The registry supports a clear description of design and registered outcomes, but the missing numerical primary results and analysis details restrict statistical conclusions.
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 important trial-specific limitation for interpreting the primary endpoint is that its numerical results are not reported in the registry record. Although the endpoint is defined as median Hb change with a full range through week 12, the record does not provide the group-specific summaries or uncertainty estimates. This prevents assessment of the size, direction, variability, and precision of the primary outcome.
The study is described as nonrandomized, single-group, and open-label. There is no separate comparator treatment group. The iron-status categories are based on baseline measurements, so differences between categories may reflect factors beyond iron status. The design cannot isolate treatment effects from concurrent cancer care, changes over time, or other influences on Hb.
The participant information is limited. The registry lists an enrollment of 84 and describes the three iron-status groups, but detailed eligibility criteria and participant characteristics are not reported in the registry record. The record does not specify how many participants contributed primary endpoint data, how missing measurements were handled, or which analysis population was used.
Analysis methods are also incompletely documented. The record lists five outcome measures and zero analyses, but does not report a statistical analysis plan, multiplicity handling, model assumptions, or a study-specific sample-size justification. The dosing description says one or two doses were based on body weight and Hb level, but the registered description is incomplete. These limitations constrain interpretation of both the intended assessment and the recorded safety counts.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Use of Intravenous Ferric Carboxymaltose in Treatment of Anemia in Cancer Patients Undergoing Chemotherapy — NCT04246021.
- Registry record last updated 2022-01-04; status last verified 2021-12.
- Results first posted 2021-12-08.
- 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 record describes a completed, nonrandomized, open-label, single-group study of intravenous ferric carboxymaltose in cancer patients with anemia undergoing chemotherapy, with or without radiotherapy. Enrollment is listed as 84. Participants were classified by baseline iron status, and the primary endpoint was median Hb change from baseline to week 12, with a full-range summary.
The record names three iron-status groups and lists secondary outcomes relating to response by iron-deficiency status and need for transfusion or ESA treatment. It records five outcome measures and zero analyses. Numerical primary and secondary outcome findings are not reported in the registry record. The listed serious adverse-event and death counts are zero in each group; other adverse-event counts are also recorded.
Because there is no randomized comparator, the study describes outcomes within a treated population rather than providing a controlled treatment-effect estimate. Missing-data handling, analysis populations, and the statistical analysis plan are not reported in the registry record.