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 Iron Isomaltoside/Ferric Derisomaltose vs Iron Sucrose for Treatment of Iron Deficiency Anemia in Non-Dialysis-Dependent Chronic Kidney Disease (NCT02940860), a randomized phase 3 study. The primary outcome is Change in Hemoglobin (Hb) From Baseline to Week 8.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02940860 |
| Phase | Phase 3 |
| Condition | Iron Deficiency Anaemia, Iron Deficiency Anemia, Chronic Kidney Disease |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 1538 |
| Arms in the primary outcome | 2 |
| Lead sponsor | Pharmacosmos A/S |
| Results posted | Yes |
2. Clinical Question
Population
People with iron deficiency anaemia and non-dialysis-dependent chronic kidney disease. The primary analysis population is described as the intention-to-treat population, including all randomized subjects. The registry description also says the trial concerned subjects for whom oral iron preparations were ineffective or could not be used, or whose screening hemoglobin, in the investigators’ opinion, was sufficiently low to require rapid repletion of iron stores.
Intervention
Intravenous iron isomaltoside/ferric derisomaltose, listed in the registry under both names and also identified by the brand names Monofer®/Monoferric® in the official title.
Comparator
Intravenous iron sucrose. The registry describes both treatments as administered intravenously. Treatment schedules, doses, and any differences in administration beyond route are not reported in the registry record.
Primary question
What was the change in hemoglobin from baseline to week 8 in each randomized group, and what does the reported between-group estimate indicate about the comparison? The primary outcome is a continuous measure, reported as least-squares means with confidence intervals. The registry labels the between-group analysis as non-inferiority, but the non-inferiority margin and the decision criterion are not reported in the registry record. The results can therefore be described, but the formal non-inferiority decision rule cannot be independently assessed from the registry information.
3. Trial Design
This was a phase III, randomized, open-label, parallel-group trial comparing intravenous iron isomaltoside/ferric derisomaltose with intravenous iron sucrose. The registry records enrollment of 1538 participants and a completed status. The listed start date is 2016-11-29, and the primary completion date is 2018-05-29. The sponsor was Pharmacosmos A/S.
The trial concerned iron deficiency anaemia in people with non-dialysis-dependent chronic kidney disease. The primary outcome population is the intention-to-treat population, defined in the registry as all randomized subjects. The group counts for the primary outcome are 1027 for iron isomaltoside/ferric derisomaltose and 511 for iron sucrose. Those counts describe the posted primary outcome results; the planned allocation ratio is not reported in the registry record.
Open-label means the registry records no masking. This matters because participants and investigators knew the assigned treatment, although the primary outcome itself is a measured hemoglobin concentration. The record does not specify whether laboratory assessors or analysts were masked. Randomization and a parallel-group design support a between-group comparison, but details of sequence generation, allocation concealment, stratification, and site procedures are not reported in the registry record.
The official title calls the study a comparative safety and efficacy trial. The registry lists 20 outcome measures and 32 analyses. It identifies the primary measure as change in hemoglobin from baseline to week 8 and lists multiple secondary safety and efficacy outcomes. Further information about treatment duration, dose schedules, eligibility criteria beyond the stated clinical population, or the protocol’s planned statistical procedures is 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 | Iron Isomaltoside/Ferric Derisomaltose | Iron Sucrose |
|---|---|---|
| STARTED | 1027 | 511 |
| COMPLETED | 971 | 476 |
| NOT COMPLETED | 56 | 35 |
Reasons for not completing this period:
| Reason | Iron Isomaltoside/Ferric Derisomaltose | Iron Sucrose |
|---|---|---|
| Non-serious AE | 1 | 2 |
| Serious AE | 7 | 3 |
| Withdrawal by Subject | 19 | 9 |
| Lost to Follow-up | 13 | 7 |
| Physician Decision | 5 | 3 |
| Sponsor Decision | 0 | 1 |
| Protocol Violation | 0 | 2 |
| Death | 3 | 2 |
| Moved, poor venous access, transport | 6 | 4 |
| Not treated | 2 | 2 |
Analysis population for the primary outcome, as defined in the registry: Intention to treat (ITT). All randomised subjects.
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 | Iron Isomaltoside/Ferric Derisomaltose | Iron Sucrose | Total |
|---|---|---|---|---|
| Participants analysed | 1027 | 511 | 1538 | |
| Age, Continuous | years | 68.3 (SD 12.3) | 69.3 (SD 12.3) | 68.6 (SD 12.3) |
| Age, Customized: Between 18 and 65 years | participants | 337 | 151 | 488 |
| Age, Customized: 65-84 years | participants | 608 | 316 | 924 |
| Age, Customized: > 84 years | participants | 82 | 44 | 126 |
| Sex: Female, Male: Female | Participants | 633 | 329 | 962 |
| Sex: Female, Male: Male | Participants | 394 | 182 | 576 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 476 | 248 | 724 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 551 | 263 | 814 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 2 | 2 | 4 |
| Race (NIH/OMB): Asian | Participants | 16 | 11 | 27 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 1 | 0 | 1 |
| Race (NIH/OMB): Black or African American | Participants | 264 | 117 | 381 |
| Race (NIH/OMB): White | Participants | 731 | 375 | 1106 |
| Race (NIH/OMB): More than one race | Participants | 2 | 0 | 2 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 11 | 6 | 17 |
| Race/Ethnicity, Customized: Mexican | participants | 4 | 3 | 7 |
| Race/Ethnicity, Customized: Indigenous Mexican | participants | 1 | 0 | 1 |
| Race/Ethnicity, Customized: Mixed (Caucasian and Native american) | participants | 1 | 0 | 1 |
| Race/Ethnicity, Customized: Hispanic | participants | 4 | 3 | 7 |
| Race/Ethnicity, Customized: Central Indian | participants | 1 | 0 | 1 |
| Race/Ethnicity, Customized: Guyanese | participants | 1 | 0 | 1 |
| Race/Ethnicity, Customized: Other (Mixed: not declared) | participants | 1 | 0 | 1 |
| Region of Enrollment: United States | participants | 1027 | 511 | 1538 |
| Current smoker: YES | participants | 118 | 53 | 171 |
| Current smoker: NO | participants | 909 | 458 | 1367 |
6. Primary Endpoint
The registry lists the primary outcome as Change in Hemoglobin (Hb) From Baseline to Week 8, measured over Baseline to week 8.
| Element | Registry entry |
|---|---|
| Result type | Least Squares Mean |
| Measure of spread | 95% Confidence Interval |
| Unit | g/dL |
| Endpoint classification | Continuous Endpoint |
The primary endpoint is change in hemoglobin concentration from baseline to week 8, measured in g/dL. Hemoglobin is the blood measure used to represent the response of interest in the registry description. The stated aim was to evaluate the effect of iron isomaltoside/ferric derisomaltose compared with iron sucrose in subjects with non-dialysis-dependent chronic kidney disease and iron deficiency anaemia. The registry defines response in terms of the ability to increase hemoglobin from baseline to week 8.
The result is reported as a least-squares mean for each treatment group, with a 95% confidence interval. The posted means are 1.22 for iron isomaltoside/ferric derisomaltose and 1.14 for iron sucrose. The registry also reports a between-group mean difference of 0.08, with a 95% confidence interval from -0.06 to 0.23, under an analysis labeled non-inferiority.
In general, a continuous outcome retains information about the size of change, rather than reducing each participant’s result to whether a threshold was or was not reached. A week-8 endpoint also gives the comparison a defined assessment time. It does not, by itself, describe the time course of change at other visits or the durability of any response after week 8. Those issues are not answered by this endpoint.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The posted primary results show increases in hemoglobin in both groups on the reported least-squares mean scale: 1.22 g/dL for iron isomaltoside/ferric derisomaltose and 1.14 g/dL for iron sucrose. The reported between-group mean difference is 0.08 g/dL, with a 95% confidence interval from -0.06 to 0.23. The interval includes zero, so the registry estimate is compatible with a small difference in either direction as well as with no between-group difference. The interval also shows that the estimate is not exact: the plausible values represented by this interval extend from a modest negative difference to a positive difference.
The registry labels this analysis non-inferiority. A non-inferiority conclusion depends on a prespecified margin and a decision rule: the confidence interval must be assessed against that margin, not merely against zero. The margin and decision rule are not reported in the registry record. Consequently, the label and estimate can be reported, but the formal basis for concluding non-inferiority cannot be independently checked from this record. The registry’s own analysis should be distinguished from a simple test of whether the difference is zero.
The arm-specific confidence intervals are 1.14 to 1.31 for iron isomaltoside/ferric derisomaltose and 1.03 to 1.26 for iron sucrose. These intervals describe uncertainty around each reported group mean; they are not substitutes for the confidence interval around the between-group estimate. For comparing treatments, the reported between-group estimate and its own interval are the directly relevant quantities.
The primary analysis is specified for all randomized subjects, which the registry calls the intention-to-treat population. The record does not report how missing week-8 measurements were handled, whether every randomized participant contributed to the endpoint, or whether additional analysis populations were used. Those details limit assessment of how closely the posted estimate follows the stated population definition.
The endpoint is a measure of hemoglobin change, not a direct measure of every aspect of clinical benefit, safety, treatment burden, or longer-term outcomes. Nor does a confidence interval establish that the treatments are identical. It expresses the precision of the estimated contrast under the analysis used. These results should therefore be read as an estimate of the week-8 hemoglobin comparison in the recorded population, with uncertainty, rather than as a complete account of all possible treatment effects.
9. Statistical Methods Explained
The primary outcome is continuous: each participant’s hemoglobin change is represented on a numerical scale in g/dL. The registry reports least-squares means for the two groups and a mean difference for their comparison. In general, a least-squares mean is a model-based group estimate; the registry does not identify the model or list covariates for this analysis. It is therefore not possible to describe which adjustments, if any, produced these estimates.
A confidence interval gives a range of values compatible with the estimate under the statistical method and its assumptions. The 95% confidence intervals around the group means describe uncertainty about those means. The separate 95% interval around the difference is the key interval for interpreting the treatment contrast. Its inclusion of zero means the reported interval does not exclude a zero difference at that confidence level. It does not prove that the groups are equivalent, and it does not by itself resolve the non-inferiority question.
For non-inferiority, the scientific question is whether the result rules out a loss larger than a margin chosen in advance. The margin, its rationale, and the exact decision criterion are not reported in the registry record. General statistical principle: a confidence interval can be compared with a non-inferiority margin only when the margin and the direction of the comparison are specified. A result that is not statistically distinguishable from zero is not automatically a demonstration of non-inferiority.
The record does not state the model, standard error method, handling of protocol deviations, or missing-data approach for the primary outcome. It also does not state whether the analysis was adjusted for baseline hemoglobin or other variables. These omissions mean that the posted estimates can be interpreted as reported, but their model details cannot be reconstructed from the registry record.
How to read binary outcomes
Several secondary outcomes are binary: participants either met a stated threshold or did not during the specified period. The recalculated comparisons use risk differences, which express the difference between the proportions in the two groups. A risk difference of zero is the null value for this measure. The recalculated estimates are unadjusted, based on the posted event counts and denominators; they are not the same as a regression-adjusted analysis.
In general, the confidence interval for a risk difference conveys the range of contrasts consistent with the data and calculation method. If it includes zero, the data do not exclude no difference at the corresponding confidence level. If it does not include zero, the result is a nominal signal under that calculation. Neither case addresses the full set of trial outcomes or the consequences of making many comparisons. The registry also reports logistic-regression odds ratios for two secondary outcomes. An odds ratio and a risk difference use different scales and should not be treated as interchangeable, especially when event frequencies are not rare.
The posted secondary comparisons include unadjusted p-values, while the registry record does not report a multiplicity adjustment for the set of secondary outcomes. In general, unadjusted p-values do not account for the increased chance of chance findings when many outcomes are examined. The binary endpoint calculations should therefore be read as exploratory descriptions of the posted data, not as a complete confirmatory testing strategy.
What planning illustrations mean
Planning estimates based on observed secondary effects are illustrations, not the trial’s actual sample-size justification and not observed power. The stated assumptions for those illustrations are a two-sided significance level of 0.05, equal allocation between the two arms, and a normal approximation; the targeted effect is a stated fraction of the observed effect. The record does not report whether these assumptions were used to plan the trial or whether they reflect its design. The original sample-size calculation and its assumptions are not reported in the registry record.
General statistical principle: planning estimates depend on the effect size chosen, the event frequencies assumed, the significance level, the desired power, and the allocation plan. Using an effect observed after a trial to illustrate future sample sizes can be informative for planning scenarios, but it does not show the probability that the completed trial detected an effect. Smaller effects generally require larger studies to detect under otherwise comparable assumptions. These calculations also do not correct for multiplicity unless an adjustment is explicitly built into the assumptions; no such adjustment is stated for the illustrations.
Finally, safety counts and efficacy outcomes answer different questions. Adverse-event counts describe events recorded among participants at risk in each group. They do not establish that an event was caused by treatment. General statistical principle: interpretation of safety comparisons depends on event definitions, observation time, exposure, ascertainment, and the analysis plan. Those details are not all reported in the registry record, so counts should be treated as descriptive.
10. Analyses Reported by the Sponsor
| Method | Estimate | 95% CI | P-value |
|---|---|---|---|
| not stated | Mean Difference (Final Values) 0.08 | -0.06 to 0.23 | not reported |
The registry reports a between-group mean difference of 0.08 g/dL for the primary outcome, with a 95% confidence interval from -0.06 to 0.23, and classifies the analysis as non-inferiority. A method and p-value are not listed for this analysis. The arm-level least-squares means are also posted with their confidence intervals.
No recalculated primary comparison is available in the posted results, so there is no independent unadjusted recalculation to set against the registry-reported analysis. The registry record does not indicate a discrepancy for this result. The registry-reported analysis therefore takes precedence for describing the primary comparison.
The estimate alone does not reveal whether the prespecified non-inferiority criterion was met. The non-inferiority margin, its justification, and the decision rule are not reported in the registry record. Without them, it is not possible to verify the formal non-inferiority interpretation or to determine how the confidence interval was judged against the planned criterion. This limitation is separate from the numerical estimate itself: the reported difference and interval remain interpretable as the registry’s stated result, while the design rule required to evaluate the non-inferiority claim is unavailable.
For two secondary outcomes, the registry reports logistic-regression odds ratios and associated confidence intervals and p-values. These adjusted analyses are distinct from simple unadjusted calculations based on counts. Where the registry reports its own analysis, the two approaches should be identified separately rather than combined as if they were the same estimate.
11. Multiplicity, Missing Data and Analysis Populations
The registry lists 20 outcome measures and 32 analyses, with 18 secondary outcomes posted. A number of secondary outcomes cover related clinical or laboratory measures, and some are assessed across several visits or time windows. The record does not report a multiplicity plan, such as a prespecified hierarchy, sequential testing procedure, or adjustment to control the overall error rate across these analyses.
General statistical principle: interpreting each p-value as if it were the only test can overstate the strength of evidence when many hypotheses are examined. A nominally small unadjusted p-value may not remain persuasive after accounting for the full set of tests. Because the registry does not describe a correction strategy, secondary p-values should be treated as unadjusted and interpreted cautiously. The absence of a reported strategy does not establish that none was used in the full protocol; it means the strategy is not reported in the registry record.
The primary population is identified as intention-to-treat, all randomized subjects. The registry does not report the number with observed week-8 measurements, how missing hemoglobin values were handled, or whether imputation or another missing-data method was used. It also does not describe sensitivity analyses for missing outcomes. These details matter because an intention-to-treat label does not, on its own, specify how unavailable measurements enter a model or what assumptions underlie the analysis.
The record does not report whether per-protocol or safety populations were used for additional efficacy analyses, nor does it provide population-specific results for the primary outcome. The outcome counts for the posted comparisons can differ from the total enrollment, but the reasons for those denominators are not detailed in the registry record. No conclusion should be drawn about the amount or pattern of missing data from the counts alone.
For the primary non-inferiority analysis, the margin and the analysis population required by the protocol are not described beyond the stated intention-to-treat population. The record also does not explain how protocol deviations or treatment discontinuations were handled. These gaps prevent a full assessment of whether the analysis followed the planned non-inferiority framework. They do not change the reported estimates, but they limit the certainty with which those estimates can be linked to the intended decision procedure.
12. 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 |
|---|---|---|---|
| Iron Isomaltoside/Ferric Derisomaltose | 83 | 1019 | 8.1% |
| Iron Sucrose | 50 | 506 | 9.9% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Iron Isomaltoside/Ferric Derisomaltose | 3 of 1019 |
| Iron Sucrose | 3 of 506 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 2% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Iron Isomaltoside/Ferric Derisomaltose | 56 of 1019 |
| Iron Sucrose | 34 of 506 |
Most frequent serious adverse event terms
| Event term | Organ system | Iron Isomaltoside/Ferric Derisomaltose | Iron Sucrose |
|---|---|---|---|
| Cardiac failure congestive | Cardiac disorders | 7 of 1019 | 11 of 506 |
| Acute kidney injury | Renal and urinary disorders | 7 of 1019 | 5 of 506 |
| Chronic kidney disease | Renal and urinary disorders | 5 of 1019 | 3 of 506 |
| End stage renal disease | Renal and urinary disorders | 5 of 1019 | 2 of 506 |
| Urinary tract infection | Infections and infestations | 3 of 1019 | 2 of 506 |
| Hyperkalaemia | Metabolism and nutrition disorders | 4 of 1019 | 1 of 506 |
| Chest pain | General disorders | 2 of 1019 | 3 of 506 |
| Cellulitis | Infections and infestations | 1 of 1019 | 3 of 506 |
| Chronic obstructive pulmonary disease | Respiratory, thoracic and mediastinal disorders | 2 of 1019 | 2 of 506 |
| Fall | Injury, poisoning and procedural complications | 2 of 1019 | 2 of 506 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Iron Isomaltoside/Ferric Derisomaltose | Iron Sucrose |
|---|---|---|---|
| Urinary tract infection | Infections and infestations | 22 of 1019 | 14 of 506 |
| Hyperkalaemia | Metabolism and nutrition disorders | 22 of 1019 | 9 of 506 |
| Hypertension | Vascular disorders | 14 of 1019 | 13 of 506 |
Serious events with the highest arm-level rate: cardiac failure congestive (2.2%), acute kidney injury (1.0%), chronic kidney disease (0.6%), cellulitis (0.6%), chest pain (0.6%).
The registry reports serious adverse events and other adverse events separately for the two treatment groups, with denominators of 1019 for iron isomaltoside/ferric derisomaltose and 506 for iron sucrose. Serious adverse events affected 83 and 50 participants, respectively; other adverse events affected 56 and 34 participants, respectively. The registry records 3 deaths in each group. These are descriptive counts, not evidence that treatment caused or prevented an event.
The listed serious adverse-event terms include cardiac failure congestive at 2.2%, acute kidney injury at 1.0%, chronic kidney disease at 0.6%, cellulitis at 0.6%, chest pain at 0.6%, and end stage renal disease at 0.5%. The registry record does not provide the event-level details, timing, exposure-adjusted rates, or attribution needed to interpret these terms as treatment-related effects. The denominator or method underlying each listed percentage is not specified in the record.
General statistical principle: safety counts are affected by how events are defined, sought, coded, and observed, as well as by the time participants were followed. The registry identifies serious and other adverse-event counts, but does not report all those details. Comparisons should therefore remain descriptive. Equal death counts do not establish equivalent safety, and differences in event counts do not by themselves establish a causal treatment effect.
13. What the Result Does, and Does Not, Mean
The primary result describes the estimated change in hemoglobin from baseline to week 8 in the randomized groups. The registry reports a mean difference of 0.08 g/dL and a 95% confidence interval from -0.06 to 0.23. This is an estimate with uncertainty, not a precise measurement of a fixed treatment effect. The interval includes zero and spans values on either side of it, so it does not rule out every modest difference in either direction.
The result does not establish that the treatments are identical. A failure to distinguish an estimate from zero is not proof of equivalence. It also does not, by itself, establish non-inferiority: that conclusion requires comparison with a prespecified margin, which is not reported in the registry record. The registry’s non-inferiority label should be read alongside this missing information, rather than as a substitute for the margin and planned decision rule.
The result is specific to the recorded primary endpoint and population: hemoglobin change through week 8 among the stated intention-to-treat population. It does not answer whether the groups differ on longer-term outcomes, every secondary measure, treatment burden, or every aspect of safety. Nor can these results be generalized beyond the recorded clinical population without additional evidence.
The secondary results add outcome-specific information but are not a single unified test of efficacy or safety. Some unadjusted findings may appear more distinct than others, yet the registry does not report a multiplicity plan. The adverse-event counts likewise describe recorded events and do not establish causation. Missing details about data handling, analysis models, and event ascertainment further limit conclusions.
In general, statistical interpretation separates the estimated effect, its precision, the hypothesis-testing framework, and the clinical importance of the outcome. The posted confidence interval addresses precision under the analysis used. The registry does not report a threshold for a clinically important hemoglobin difference, nor the formal non-inferiority margin. The result can therefore be stated numerically and interpreted cautiously, but the record does not support a broader claim about clinical importance or a fully verified non-inferiority conclusion.
14. Why This Trial Matters Statistically
This trial illustrates why the label attached to an analysis is not enough to interpret its statistical conclusion. The primary comparison is designated as non-inferiority, but the registry’s mean difference and confidence interval cannot be judged against the intended non-inferiority boundary because that margin is not reported. Readers need both the estimate and the rule used to make the decision.
It also demonstrates the difference between a group estimate and a contrast. Each treatment group has its own least-squares mean and confidence interval, while the mean difference has a separate interval. Comparing the arm-level intervals by eye is not a replacement for the reported interval on the contrast. For treatment comparisons, the direct between-group estimate is the relevant summary.
The record further highlights the distinction between an unadjusted re-calculation from event counts and a registry-reported regression analysis. These methods can target related questions while using different statistical scales or adjustment. Keeping them separate helps prevent an unadjusted calculation from being mistaken for the trial’s own analysis.
Finally, a trial can report many outcomes and analyses, but a large list of results does not automatically provide a set of equally strong confirmations. Without a reported multiplicity plan, secondary findings require cautious interpretation. Planning illustrations based on observed effects are also not evidence of the trial’s original design or its achieved power. These distinctions are useful when reading registry results across both efficacy and safety outcomes.
15. 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 registry record gives limited detail about the primary statistical model. It reports least-squares means and a mean difference but does not identify the model specification, covariates, standard error method, or handling of baseline values. The non-inferiority margin and decision rule are also not reported, which prevents independent evaluation of the stated non-inferiority analysis.
The trial is recorded as open-label, with no masking. The record does not explain whether outcome assessors or analysts were masked, or how the lack of masking was considered in outcome assessment. Randomization is recorded, but sequence generation, allocation concealment, stratification, and site-level procedures are not reported in the registry record.
Missing-data handling is not described. The record does not state how many randomized participants had a week-8 hemoglobin result, how unavailable values were treated, or whether sensitivity analyses were conducted. The stated intention-to-treat population therefore does not provide enough information to assess the practical implementation of that analysis population. The reasons for the differing denominators across posted outcomes are also not reported.
The registry lists numerous outcomes and analyses but does not describe how multiplicity was handled or identify a hierarchy for secondary outcomes. This limits the confirmatory interpretation of nominal secondary findings. For the safety data, definitions, observation time, event attribution, and exposure-adjusted analyses are not reported in the registry record, so the counts cannot establish causal differences.
Finally, the record does not include the original sample-size justification or explain whether the planning illustrations based on observed secondary effects resemble the actual design assumptions. The posted data are therefore informative about the reported estimates, but do not provide all protocol and analysis details needed for a complete statistical audit.
16. Related Tutorials
17. Related Statistical Calculators
18. Sources
- ClinicalTrials.gov: Iron Isomaltoside/Ferric Derisomaltose vs Iron Sucrose for Treatment of Iron Deficiency Anemia in Non-Dialysis-Dependent Chronic Kidney Disease — NCT02940860.
- Registry record last updated 2020-10-06; status last verified 2020-02.
- Results first posted 2020-03-06.
- 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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19. Record Summary
NCT02940860 is a completed phase III, randomized, open-label, parallel-group comparison of intravenous iron isomaltoside/ferric derisomaltose and intravenous iron sucrose in people with iron deficiency anaemia and non-dialysis-dependent chronic kidney disease. The registry records enrollment of 1538 participants and identifies Pharmacosmos A/S as sponsor.
The primary endpoint is change in hemoglobin from baseline to week 8 in the intention-to-treat population. The registry reports least-squares means of 1.22 g/dL and 1.14 g/dL, and a mean difference of 0.08 g/dL with a 95% confidence interval from -0.06 to 0.23. The analysis is labeled non-inferiority, but the margin and decision rule are not reported in the registry record.
Secondary efficacy and safety outcomes provide additional descriptive information. Their multiplicity strategy, missing-data methods, and several analysis details are not reported in the registry record, so secondary findings and adverse-event counts require cautious interpretation.