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 Extended Evaluation of Deferasirox Film-coated Tablet (FCT) Formulation (NCT02720536), a na phase 3 study. The primary outcome is Change From Baseline Red Blood Cells (RBC) (10^12 Cells/L) at Month 6 and Month 12.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02720536 |
| Phase | Phase 3 |
| Condition | Chronic Iron Overload |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 53 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Novartis Pharmaceuticals |
| Results posted | Yes |
2. Clinical Question
Population
Patients with chronic iron overload who completed study CICL670F2201. The registry record lists an enrollment of 53 and describes the primary endpoint population as members of the safety analysis set with a measurement at baseline and the relevant follow-up time point. Eligibility details beyond the stated study context are not reported in the registry record.
Intervention
Deferasirox film-coated tablets. Treatment was to be administered daily for up to 24 months, with each patient’s daily dose calculated from actual body weight. The registry record identifies deferasirox as the drug intervention.
Comparator
There was no concurrent comparator group: the study is described as open-label and single-arm. Allocation is listed as NA, and masking as none. The registry record therefore does not describe a randomized comparison with another treatment or with a control group.
Primary question
The primary endpoint concerns change from baseline in red blood cell (RBC) count, measured as 10^12 cells/L, at Month 6 and Month 12. The study was intended to collect additional safety and efficacy data among patients completing the earlier study. The registry record does not report the numerical RBC changes, their variability, or an analysis comparing follow-up measurements with baseline.
In plain terms, the study asks what safety and laboratory outcome information is observed during continued deferasirox treatment in this group. Its single-arm design can describe measurements and adverse events among treated participants, but it cannot isolate treatment effects from changes that might have occurred over time for other reasons. The registry lists serum ferritin changes as secondary outcomes, including absolute change and percentage relative change at Month 6 and Month 12. Numerical results for those outcomes are not reported in the registry record.
3. Trial Design
This was an open-label, multicenter, single-arm Phase III study of deferasirox film-coated tablets. It was designed to collect additional safety and efficacy data from patients completing study CICL670F2201. The listed condition is chronic iron overload, and the registry record lists 53 participants as the enrollment. The study status is completed.
The recorded start date is 2016-08-16, and the primary completion date is 2019-07-23. Treatment was described as daily for up to 24 months. Dose calculation was based on each patient’s actual body weight. The record does not give a more detailed dosing schedule, dose range, duration actually received by participants, or adherence information.
Open-label means that masking was not used, consistent with the registry entry of none. The study model is single group, with allocation listed as NA. Consequently, the design does not include a concurrent randomized control arm. The absence of a comparator is central to interpretation: changes measured during follow-up are descriptive observations in treated participants, not a controlled estimate of the difference attributable to treatment.
The primary endpoint is mean change from baseline in RBC count at Month 6 and Month 12, with standard deviation specified as the measure of dispersion. The endpoint description says that a change at each time point is calculated only for subjects with a value at baseline and at that particular time point. The population is described as the safety analysis set, restricted separately at each time point to participants with both measurements. The number included at each assessment is not reported in the registry record.
The record lists serum ferritin change and percentage relative change at Month 6 and Month 12 among the secondary outcomes. It also lists 10 outcome measures overall. Although the study is marked completed, numerical results for the primary endpoint and these secondary outcomes are not reported in the registry record. The record lists no analyses. The protocol’s rationale for enrollment, sample-size justification, and any planned interim analyses 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.
Before assignment: All patients completed study CICL670F2201 (NCT02125877) prior to entry into this study
Overall Study
| Stage | Deferasirox |
|---|---|
| STARTED | 53 |
| COMPLETED | 34 |
| NOT COMPLETED | 19 |
Reasons for not completing this period:
| Reason | Deferasirox |
|---|---|
| Primary reason (PR) - subject withdrawal | 11 |
| PR - pregnancy | 2 |
| PR - unsatisfactory therapeutic effect | 2 |
| PR - death | 1 |
| PR - adverse event | 1 |
| PR - abnormal lab value(s) | 1 |
| PR- unwilling to comply with procedures | 1 |
Analysis population for the primary outcome, as defined in the registry: Safety analysis set
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 | Deferasirox |
|---|---|---|
| Participants analysed | 53 | |
| Age, Customized: <18 | Participants | 3 |
| Age, Customized: ≥18 - <50 | Participants | 46 |
| Age, Customized: ≥ 50 - <65 | Participants | 1 |
| Age, Customized: ≥ 65 | Participants | 3 |
| Sex: Female, Male: Female | Participants | 35 |
| Sex: Female, Male: Male | Participants | 18 |
| Race/Ethnicity, Customized: Caucasian | Participants | 50 |
| Race/Ethnicity, Customized: Asian | Participants | 1 |
| Race/Ethnicity, Customized: Other | Participants | 2 |
| Main underlying disease: MDS with very low risk as per the IPSS - R | participants | 2 |
| Main underlying disease: MDS with low risk as per the IPSS - R | participants | 1 |
| Main underlying disease: MDS with INT risk as per the IPSS - R | participants | 1 |
| Main underlying disease: Transfusion-dependent thalassemia | participants | 49 |
6. Primary Endpoint
The registry lists the primary outcome as Change From Baseline Red Blood Cells (RBC) (10^12 Cells/L) at Month 6 and Month 12, measured over Baseline, 6 and 12 months.
| Element | Registry entry |
|---|---|
| Result type | Mean |
| Measure of spread | Standard Deviation |
| Unit | 10^12 cells/L |
| Endpoint classification | Continuous Endpoint |
The primary endpoint is mean change from baseline in red blood cell count, reported in 10^12 cells/L, at Month 6 and Month 12. The registry describes measurements at baseline and at those follow-up times. For each follow-up, the change is calculated as the post-baseline value minus the baseline value. The endpoint is restricted to participants in the safety analysis set who have measurements at both baseline and that follow-up time.
RBC count is a continuous laboratory measure. A mean change gives the average direction and size of the observed change in the analyzed participants; the standard deviation describes how dispersed individual changes are around that average. Reporting both can help distinguish an average pattern from the degree of variation among participants. These are general statistical interpretations, not a claim about the results of this study.
Repeated measurement at follow-up can describe how the laboratory measure changes over the observed period. However, because this is a single-arm study, a change from baseline does not by itself establish why the change occurred. Without a concurrent comparator, natural variation, disease course, other care, and measurement variability cannot be separated from an effect of treatment using this design alone.
The registry record does not report the mean changes, standard deviations, confidence intervals, or tests for the primary endpoint. It therefore does not provide a numerical estimate of the observed RBC change or its precision. The endpoint definition is available, but the results needed to quantify the change are not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record establishes the study’s design and intended outcome measurements, but it does not give numerical primary endpoint results. In particular, it does not report the mean RBC change from baseline at Month 6 or Month 12, the corresponding standard deviations, or the numbers of participants contributing measurements at those time points. Without those results, the magnitude and precision of the observed change cannot be assessed from the registry record.
The endpoint is defined as a within-participant change, calculated only when both baseline and the relevant follow-up value are available. That definition means the analyzed group can differ between follow-up time points if measurement availability differs. The record does not report the number of participants with paired measurements at each time point, so the degree to which the analyzed sets differ is not known. Nor does the record describe how missing laboratory measurements were handled beyond the stated requirement for values at both times.
The study is single-arm and open-label, with no comparator. Its outcome descriptions can support a descriptive account of measurements and safety observations among participants treated with deferasirox. They do not provide a controlled contrast that separates treatment-associated change from other influences. This is a limitation of what the design can establish, not a finding that any observed change is or is not due to treatment.
The registry lists serum ferritin change and percentage relative change at Month 6 and Month 12 as secondary outcomes. It lists 10 outcome measures overall, but no numerical results for these outcomes are reported in the registry record. No analyses are listed. As a result, there are no posted estimates or inferential results available in this record to evaluate alongside the primary endpoint.
The safety summary lists 53 participants at risk for serious adverse events and 13 affected, and 53 at risk for other adverse events and 48 affected. It also lists 1 death. These are counts in the registry summary; the record does not establish from these figures whether an event was caused by the intervention. The listed serious-event terms and their percentages describe reported events, but their interpretation depends on the registry’s event definitions and ascertainment, details that are not fully described in the available record.
Overall, the posted information is most informative about the study’s setting, intended measurements, and summarized safety counts. It is not enough to estimate the size or precision of laboratory changes, assess statistical significance, or make a controlled comparison. Those conclusions would require outcome results and analysis details that are not reported in the registry record.
9. Statistical Methods Explained
What does a mean change describe?
The primary outcome is continuous: RBC count is measured on a numerical scale, and the analysis is described in terms of a mean. In general, a mean change is the average of individual differences between a follow-up measurement and baseline. The registry defines change as post-baseline minus baseline. A positive or negative mean would indicate the direction of the average change under that convention, but the actual mean values are not reported in the registry record.
The endpoint also specifies standard deviation. In general, standard deviation describes how spread out individual values are around the mean. It is a description of variation among observations, not a measure of how precisely the mean has been estimated. Precision is commonly conveyed with a confidence interval, which gives a range of values compatible with the data under the method’s assumptions. Confidence intervals for the primary outcome are not reported in the registry record.
Why use paired measurements?
The endpoint description requires a baseline value and a value at the particular follow-up time point for each included participant. This is a paired approach: each participant’s follow-up measurement is compared with that participant’s own baseline. In general, pairing accounts for the fact that measurements from the same person are related. It can make the change more informative than comparing unrelated groups, but the resulting mean still describes the participants with available paired observations. The registry does not report how many paired observations contributed at either follow-up.
The record describes the population as the safety analysis set, narrowed at each time point to those with both measurements. Therefore the analyzed set is defined separately for each assessment. If participants lack one or both measurements, they do not contribute to that time point’s defined change calculation. The record does not state whether missingness was evaluated, whether any imputation was used, or whether additional sensitivity analyses were conducted.
What can a single-group design estimate?
In general, a single-group study can describe the measurements observed over time among participants who receive the intervention. With no concurrent control group, however, it cannot directly estimate the difference between outcomes under treatment and outcomes under an alternative condition. Changes can coincide with treatment without being attributable to treatment alone. The study is also open-label, and masking is listed as none. The record does not describe a blinded outcome assessment.
This design context matters even if numerical mean changes were available. A confidence interval around a within-group mean change would quantify statistical uncertainty for that estimate under its analysis assumptions; it would not remove the design’s lack of a comparator. No confidence intervals or tests for the primary endpoint are reported in the registry record.
How should multiple outcomes be considered?
The registry lists 10 outcome measures. The primary outcome includes assessments at Month 6 and Month 12, while the secondary outcomes include serum ferritin change and percentage relative change at those follow-up times. In general, examining multiple outcomes or time points creates multiple opportunities to observe apparently notable results by chance. Statistical plans sometimes address this through prespecified prioritization or multiplicity adjustments. The registry record does not report whether such procedures were planned or used, and no analyses are listed.
Without numerical estimates and analysis details, it is not possible to determine how the outcomes were analyzed, whether any comparisons were prespecified, or how uncertainty across outcomes was handled. The endpoint descriptions identify what was intended to be measured, but do not substitute for a statistical analysis plan or posted results.
What does the sample-size illustration mean?
The registry’s sample-size assumptions describe a planning illustration based on the observed effect, not the trial’s own design. The illustration uses a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; its target effect is a stated fraction of the observed effect. For time-to-event outcomes, the illustration concerns total events using the Schoenfeld approach; other outcomes are expressed as participants per arm.
Those assumptions describe hypothetical planning calculations. They do not change this study’s single-group design, establish that participants were assigned equally between arms, or indicate that the trial had a particular probability of detecting an effect. Any sample-size values associated with that illustration are planning quantities, not observed participant counts or observed power. The record does not report a trial-specific sample-size justification or the rationale for the enrollment of 53.
More broadly, statistical significance and practical interpretation are distinct. A p-value, if reported, would summarize how compatible data are with a specified null model and analysis assumptions; it would not measure the probability that a treatment works. No p-values or other analyses are reported in the registry record. The available information therefore supports explanation of the intended endpoint and design, but not a re-analysis of treatment effects.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 10 outcome measures, including the primary RBC change at Month 6 and Month 12 and secondary serum ferritin changes at Month 6 and Month 12. Assessing multiple outcomes and time points raises a general multiplicity issue: the more results considered, the more care is needed in distinguishing planned primary questions from exploratory findings. A protocol may specify a hierarchy of outcomes or a method to adjust for multiple testing. Such a hierarchy, adjustment, or other multiplicity strategy is not reported in the registry record.
The registry record lists no analyses, and numerical outcome results are not reported. It is therefore not possible to determine whether the planned outcomes were analyzed as stated, whether additional outcomes were examined, or whether multiplicity was addressed in an analysis.
For the primary endpoint, the record specifies that each time-point change uses participants with both a baseline and the relevant follow-up measurement. This is a complete-pair definition for that outcome and time point. In general, restricting an analysis to participants with observed paired values can affect how representative the analyzed group is if measurement availability is related to participant characteristics or outcomes. The registry does not report missing-measurement counts, reasons for missingness, or an assessment of whether missingness could influence interpretation.
The record does not report imputation, a missing-data model, sensitivity analyses, or the use of an intention-to-treat population. It describes the primary population as the safety analysis set, with the paired-measurement restriction applied at each time point. Other analysis populations and their definitions are not reported in the registry record. These omissions limit evaluation of whether missing data or analysis-population choices could affect the results.
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 |
|---|---|---|---|
| Deferasirox | 13 | 53 | 24.5% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Deferasirox | 1 of 53 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 5% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Deferasirox | 48 of 53 |
Most frequent serious adverse event terms
| Event term | Organ system | Deferasirox |
|---|---|---|
| Atrial fibrillation | Cardiac disorders | 1 of 53 |
| Cardiac failure | Cardiac disorders | 1 of 53 |
| Goitre | Endocrine disorders | 1 of 53 |
| Biliary colic | Hepatobiliary disorders | 1 of 53 |
| Cholecystitis | Hepatobiliary disorders | 1 of 53 |
| Cholestasis | Hepatobiliary disorders | 1 of 53 |
| Hepatic failure | Hepatobiliary disorders | 1 of 53 |
| Diverticulitis | Infections and infestations | 1 of 53 |
| Lower respiratory tract infection | Infections and infestations | 1 of 53 |
| Lymph gland infection | Infections and infestations | 1 of 53 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Deferasirox |
|---|---|---|
| Diarrhoea | Gastrointestinal disorders | 14 of 53 |
| Headache | Nervous system disorders | 14 of 53 |
| Pyrexia | General disorders | 13 of 53 |
| Nausea | Gastrointestinal disorders | 12 of 53 |
| Vomiting | Gastrointestinal disorders | 12 of 53 |
| Cough | Respiratory, thoracic and mediastinal disorders | 12 of 53 |
| Abdominal pain upper | Gastrointestinal disorders | 11 of 53 |
| Abdominal pain | Gastrointestinal disorders | 10 of 53 |
| Asthenia | General disorders | 10 of 53 |
| Influenza | Infections and infestations | 9 of 53 |
Serious events with the highest arm-level rate: atrial fibrillation (1.9%), cardiac failure (1.9%), goitre (1.9%), biliary colic (1.9%), cholecystitis (1.9%).
The safety summary lists 53 participants at risk for serious adverse events, with 13 affected. For other adverse events, it lists 53 at risk and 48 affected. The record also lists 1 death. These are the posted counts; the registry does not provide enough detail in this summary to determine the timing, duration, severity distribution, or relationship of each event to treatment.
The listed serious adverse event terms include atrial fibrillation, cardiac failure, goitre, biliary colic, cholecystitis, and cholestasis, each shown with 1.9%. Those entries should be read as reported terms and percentages, not as a combined category or as evidence of causation. The registry record does not state in this summary whether the death was related to treatment.
Adverse-event counts describe what was recorded among participants in the safety summary. They do not, by themselves, establish that deferasirox caused an event, nor do they provide a comparison with an untreated or alternative-treatment group. Because the study is single-arm, these figures are descriptive and should be interpreted in that design context.
12. What the Result Does, and Does Not, Mean
The registry record describes a completed, open-label, single-arm study in which patients with chronic iron overload who completed study CICL670F2201 received daily deferasirox film-coated tablets for up to 24 months. It identifies RBC change from baseline at Month 6 and Month 12 as the primary outcome and serum ferritin measures as secondary outcomes. It also provides summarized safety counts.
Those design and outcome details establish what the study was intended to observe. They do not reveal the size or direction of the RBC or serum ferritin changes, because numerical outcome results are not reported in the registry record. The record also does not report estimates of uncertainty or inferential analyses. Consequently, the registry information does not support a numerical assessment of the primary endpoint’s magnitude or precision.
Even if within-group changes were reported, the single-arm design would not provide a controlled estimate of how outcomes would differ under another treatment or no intervention. A change over time may reflect several influences, and the design has no concurrent comparison group to separate them. This does not invalidate the descriptive purpose of the study, but it limits causal interpretation.
The adverse-event counts offer a description of events recorded in the study group. They do not establish event attribution, and there is no control group for comparative safety interpretation. The single death is listed in the registry summary, but its relationship to treatment is not reported in the registry record.
Accordingly, the posted record supports a description of the study’s design, intended endpoints, and summarized safety observations. It does not show whether the primary endpoint changed by a particular amount, whether a statistical test was significant, or whether outcomes differed from a comparator. Those conclusions require outcome estimates and analysis information not reported in the registry record.
13. Why This Trial Matters Statistically
This study illustrates an important distinction between collecting follow-up data and estimating a comparative treatment effect. Its single-group structure can describe laboratory measurements and adverse events among participants treated with deferasirox, while leaving the counterfactual question—what would have happened to comparable participants under another condition—unanswered. That distinction is essential when interpreting within-group changes.
The outcome definition also highlights the role of analysis populations. The primary change is calculated only for participants with measurements at baseline and the relevant follow-up. This is a clear operational definition, but evaluation of the resulting estimate depends on knowing how many observations contributed and whether missingness might have influenced who remained in each analysis. Those details are not reported in the registry record.
Multiplicity is another statistical consideration because the record lists 10 outcome measures and identifies more than one follow-up time for the primary and secondary outcomes. Prespecified prioritization and transparent analysis methods help readers interpret a collection of results. The registry record does not report the statistical plan or analyses, so the handling of multiple outcomes cannot be assessed.
Finally, the record separates the description of an endpoint from its results. It states what was measured, how change was defined, and which dispersion measure was intended, but provides no numerical primary outcome results. This makes it possible to understand the intended analysis while also recognizing that the posted record does not permit evaluation of the magnitude or precision of the laboratory findings. The safety counts add descriptive context, not a controlled comparison or proof of event causation.
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 principal limitation for interpreting the posted results is that numerical findings for the primary and secondary outcomes are not reported in the registry record. The primary RBC mean changes and standard deviations are absent, as are participant counts contributing at each time point. The record lists 10 outcome measures but provides no analyses. Therefore, neither effect magnitude nor statistical precision can be evaluated from the registry information.
The single-arm, open-label design has no concurrent comparator. It can describe outcomes during treatment but cannot distinguish treatment-associated changes from other explanations using a controlled contrast. Masking is listed as none, and the record does not describe blinded outcome assessment.
The primary endpoint’s paired-measurement rule means participants need both baseline and follow-up values for inclusion at a time point. The number and characteristics of participants excluded because of missing measurements, and reasons for missingness, are not reported in the registry record. The record also does not report imputation or sensitivity analyses.
The statistical plan, multiplicity approach, sample-size justification, and interim analyses are not reported in the registry record. While the record includes hypothetical sample-size assumptions, those describe a planning illustration rather than the design of this single-arm study. The safety summary provides counts and listed terms, but does not report event attribution or enough detail to interpret timing and exposure. These limitations constrain interpretation of both the efficacy-related endpoints and the safety observations.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Extended Evaluation of Deferasirox Film-coated Tablet (FCT) Formulation — NCT02720536.
- Registry record last updated 2020-03-03; status last verified 2020-02.
- Results first posted 2020-03-03.
- 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
NCT02720536 is a completed, open-label, multicenter, single-arm Phase III study titled “Extended Evaluation of Deferasirox Film-coated Tablet (FCT) Formulation.” It enrolled 53 patients with chronic iron overload who completed study CICL670F2201. The recorded start date is 2016-08-16, and the primary completion date is 2019-07-23.
Participants were to receive daily deferasirox film-coated tablets for up to 24 months, with daily dose calculated from actual body weight. The primary outcome was mean change from baseline in RBC count at Month 6 and Month 12, with standard deviation specified. Secondary outcomes included serum ferritin change and percentage relative change at those time points.
The record lists 10 outcome measures and no analyses. Numerical efficacy results are not reported in the registry record. The safety summary lists 13 participants affected by serious adverse events, 48 affected by other adverse events, and 1 death among 53 at risk in each category. The single-group design supports descriptive interpretation, not a controlled comparison.