This page provides an independent statistical analysis and educational interpretation of publicly reported results. ClinicalTrials.gov provides the official trial registry record. Numerical trial results on this page are restricted to the results posted in the ClinicalTrials.gov record; calculations added by Clinical Biostats are labelled as such.
1. Trial at a Glance
This page covers the registry results for Study for the Treatment of Transfusional Iron Overload in Myelodysplastic Patients (NCT00117507), a na phase 4 study. The primary outcome is Number of Participants With Adverse Events and Serious Adverse Events.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00117507 |
| Phase | Phase 4 |
| Condition | Myelodysplastic Syndromes, Iron Overload |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 24 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Novartis Pharmaceuticals |
| Results posted | Yes |
2. Clinical Question
Population
The study concerned adult, transfusion-dependent patients with myelodysplastic syndrome (MDS) and iron overload. The brief summary specifies that enrolled patients had low-risk or intermediate (INT-1) MDS according to International Prognostic Scoring System criteria. The primary safety population is defined as participants who received at least 1 dose of study drug and had at least 1 post-baseline safety assessment. The registry record does not provide further eligibility details or a separate description of the characteristics of the participants included in the posted safety counts.
Intervention
Participants received oral deferasirox. The summary states that treatment began at 20mg/kg/day and continued for 12 months; the arm description also gives this dose and duration. The trial was open-label, so treatment assignment was not masked.
Comparator
There was no comparator group: the study is recorded as a single-group trial, and allocation is listed as not applicable. The observed outcomes therefore describe the participants treated in this study, rather than a contrast between deferasirox and a control treatment or placebo.
Primary question
The primary outcome was the number of participants with adverse events and serious adverse events up to Week 52. The record describes adverse events as untoward medical occurrences after written informed consent, including unfavorable or unintended signs, abnormal laboratory findings, symptoms, or diseases from the first dose through the end of study treatment. The central question addressed by the posted primary outcome is how many participants in the defined safety population had these events during the stated observation period. It is a safety and tolerability question, not a randomized comparison of treatment effects.
3. Trial Design
This was an open-label, single-group Phase 4 study of deferasirox for transfusional iron overload in patients with low-risk or INT-1 MDS. The registry lists Novartis Pharmaceuticals as the sponsor and gives the study status as completed. The start is recorded as 2005-09 and primary completion as 2008-01.
The brief summary says that 30 patients were to be enrolled and that 24 patients were actually enrolled. Participants received oral deferasirox at 20mg/kg/day, with treatment described as lasting 12 months. The primary outcome’s timeframe is stated as up to Week 52. These descriptions identify the intended treatment period and outcome window; they do not establish that every participant completed treatment or had outcome information throughout that period.
Because there was no control group and no random allocation, the study cannot provide a like-for-like comparison with an untreated group or another treatment group. Its open-label design also means participants and study personnel were not masked to treatment. These features are relevant when interpreting event reporting and the scope of conclusions: the posted primary data are counts observed in the treated group, not estimates of a randomized between-group effect.
The record lists secondary outcomes concerning serum ferritin, liver iron concentration, transfusion requirements, serum erythropoietin, urinary hepcidin, transferrin saturation, and labile plasma iron. Numerical results for those outcomes are not reported in the registry record described for this analysis. The registry record does not report a detailed statistical analysis plan, a sample-size justification, interim analyses, or further information about treatment adherence and follow-up.
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: A total 24 participants were enrolled in the study of which 9 participants completed the study and 15 participants discontinued from the study.
Overall Study
| Stage | Deferasirox |
|---|---|
| STARTED | 24 |
| COMPLETED | 9 |
| NOT COMPLETED | 15 |
Reasons for not completing this period:
| Reason | Deferasirox |
|---|---|
| Adverse Event | 4 |
| Abnormal laboratory values | 3 |
| Unsatisfactory therapeutic effect | 1 |
| Patient withdrew consent | 4 |
| Death | 3 |
Analysis population for the primary outcome, as defined in the registry: The safety set consisted of all participants who received at least 1 dose of study drug and had at least 1 post-baseline safety assessment.
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: Intent-to-treat (ITT) population consisted of all patients who were registered in the study, whether or not they received treatment.
| Characteristic | Unit | Deferasirox |
|---|---|---|
| Participants analysed | 24 | |
| Age, Continuous | years | 68.6 (SD 8.58) |
| Sex: Female, Male: Female | Participants | 8 |
| Sex: Female, Male: Male | Participants | 16 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Participants With Adverse Events and Serious Adverse Events, measured over Up To Week 52.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint counts participants with adverse events and serious adverse events up to Week 52. An adverse event is defined broadly in the record: it may be an unfavorable or unintended sign, abnormal laboratory finding, symptom, or disease occurring after written informed consent. The described event period runs from the first dose of study treatment until the end of study treatment.
Counting participants with events is a direct way to describe how many people in the safety population experienced an event, rather than how many separate event episodes occurred. The record’s unit is participants. That distinction matters because one participant may have more than one event, while a participant count records that person once for the relevant category. The posted primary outcome is therefore a participant-level safety summary, not a total event count, an event rate over person-time, or a measure of treatment benefit.
Safety and tolerability were the stated focus of this open-label study. In general, participant counts can help describe the observed burden of reported adverse events during a defined period. They do not by themselves show whether events were caused by the study drug, how long they lasted, their clinical consequences, or how the observed experience compares with what would have occurred without treatment. Those questions require additional information or a suitable comparison that is not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The posted primary results report 23 participants with adverse events and 11 participants with serious adverse events in the deferasirox group. The group’s listed at-risk count is 24 for each of these categories. The record also lists 3 deaths. These are descriptive counts for the study group; they are not a comparison with a control group, and the registry record does not report an adjusted or causal analysis of these events.
The record lists the following serious adverse event terms and percentages: Pyrexia, 16.7%; Cardiac failure congestive, 8.3%; Chest pain, 8.3%; Urinary tract infection, 8.3%; Dyspnoea, 8.3%; and Splenic infarction, 4.2%. These entries identify reported terms and their posted percentages. The record does not explain whether participants could contribute to more than one term, give event-level timing or duration, or state how the listed terms relate to the total serious adverse event participant count. The percentages should therefore be read as the registry’s reported values, not recomputed or interpreted as mutually exclusive categories.
In general, a participant count is useful for showing how many people had at least one event in a category, but it gives limited information about event frequency and severity. Counts also do not show the distribution of follow-up time. The stated observation window is up to Week 52, but the registry record does not report individual follow-up duration or missing safety assessments. It is consequently not possible to determine from these counts alone whether participants had comparable observation time.
The study had no control arm. Without a like-for-like comparator, the observed counts cannot estimate how many events would have occurred under another treatment or without study treatment. The open-label design may also affect how symptoms are noticed, reported, assessed, or classified. These considerations do not invalidate the descriptive counts, but they constrain what those counts can establish.
The primary outcome concerns safety. Although the registry lists several secondary outcomes related to iron measures and transfusion requirements, their numerical results are not reported in the registry record. No conclusion about changes in those measures follows from the primary safety counts. The record also does not give confidence intervals or other uncertainty estimates for the primary counts. The results should therefore be read as the posted observations in this study, with their precision and causal interpretation limited by the information recorded.
9. Statistical Methods Explained
The primary outcome is a count of participants with adverse events and serious adverse events. For a binary participant-level outcome, each participant is classified according to whether the event category applies during the specified period. The reported count indicates how many participants were classified as having that outcome. It is different from counting every event episode: several events in one participant can still contribute a single participant to a participant-level count.
The registry identifies the unit as participants and defines the safety set as participants who received at least 1 dose of study drug and had at least 1 post-baseline safety assessment. This definition specifies who is eligible for the safety summary. The group’s at-risk counts are 24 for adverse events and 24 for serious adverse events. The registry record does not report participant-level data, event dates, or additional details needed to reconstruct the classifications independently.
In general, a proportion can be used to express a participant count relative to its denominator, but no new proportion is calculated. A proportion still gives only a summary of the observed participants and period. It does not account for differences in follow-up duration, and it does not convey how many event episodes occurred. If participants have different amounts of observation time, an event rate based on person-time may answer a different question; person-time results are not reported in the registry record.
In general, confidence intervals describe the uncertainty around an estimated quantity under a statistical model. For participant-level binary outcomes, an interval can communicate the range of values reasonably compatible with the observed data and the method used. The registry record does not report confidence intervals for these primary counts. Without them, the posted counts do not show the statistical precision of an underlying event proportion. An interval would also not resolve limitations from the single-group design, incomplete follow-up information, or possible differences in event assessment.
The single-group design shapes the analysis. There is no randomized contrast, so an analysis of the study group alone can describe its observed event experience but cannot isolate the effect of deferasirox from other causes. For example, the record describes participants as having MDS and transfusional iron overload, but does not provide an untreated or alternative-treatment group against which background event occurrence can be assessed. An observed event during treatment is not, on its own, evidence that treatment caused it.
Open-label conduct is another design feature relevant to measurement. In general, masking can reduce the influence of knowledge of treatment on event reporting or assessment. Since masking is listed as none, the study was not masked. The registry record does not report whether independent reviewers assessed events, how event severity was graded, or how investigators determined seriousness or relatedness to treatment. Those details should not be assumed.
The record gives a planning statement for sample-size illustrations: two-sided significance level 0.05, equal allocation between two arms, normal approximation, and an effect to detect defined as a stated fraction of the observed effect. It also states that time-to-event planning uses a total number of events, while other planning results use participants per arm. These are assumptions for planning illustrations, not evidence that the trial itself used a two-arm design. The actual study is recorded as single-group, and no numerical sample-size illustration results are reported in the registry record. Such planning illustrations are not observed power and do not change the interpretation of the trial’s observed counts.
The registry record lists no analyses for the outcome set described in the registry record, and it does not report a statistical model or inferential test for the primary endpoint. No adjusted estimates, hypothesis-test results, or multiplicity strategy for the primary and secondary outcomes are reported in the registry record. It is not possible to infer from the endpoint counts whether any formal testing was planned or performed outside the posted record.
In general, over-dispersion is a concern when outcome variability exceeds what a chosen statistical model expects. It can make standard errors too small and uncertainty appear narrower than warranted if the model is not adapted. The posted primary outcome is a participant-level binary count, and no model-based variance estimate is reported. The registry record provides no basis for assessing over-dispersion or a model adjustment for it. For these data, the more immediate interpretive issue is that only descriptive counts are posted and there is no comparator group.
The time definition also matters. The primary outcome is specified as up to Week 52, while the treatment description states 12 months. The record does not report whether every participant remained under safety observation for the full period, how discontinuation was handled, or how events after treatment cessation were treated. These details affect how a reader might understand the risk period and are not recoverable from the reported counts alone.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists several secondary outcomes in addition to the primary safety outcome, including changes in serum ferritin and liver iron concentration, transfusion requirements, serum erythropoietin, urinary hepcidin, transferrin saturation, and labile plasma iron. Numerical results for those outcomes are not reported in the registry record. The record does not report how testing across the primary and secondary outcomes was handled, whether a multiplicity adjustment was planned, or which outcomes were intended to support confirmatory claims. No multiplicity strategy should be inferred from the outcome list alone.
Missing-data handling is not reported in the registry record. In particular, the record does not state how participants without a post-baseline safety assessment were treated, how missing event information was handled, or whether any imputation was used. The safety-set definition requires at least 1 post-baseline safety assessment, but the record does not give the number of enrolled participants excluded from that set or explain any exclusions. The listed at-risk count for each primary category is 24, while the reported enrollment is also 24; this does not establish complete follow-up through Week 52.
The record does not report a detailed analysis population for the secondary outcomes, a missing-data sensitivity analysis, or a statistical analysis plan. These omissions limit assessment of how incomplete follow-up may have affected the posted results. They also mean the secondary outcomes cannot be interpreted as a complete set of trial findings from the 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 |
|---|---|---|---|
| Deferasirox | 11 | 24 | 45.8% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Deferasirox | 3 of 24 |
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 |
|---|---|
| Deferasirox | 23 of 24 |
Most frequent serious adverse event terms
| Event term | Organ system | Deferasirox |
|---|---|---|
| Pyrexia | General disorders | 4 of 24 |
| Cardiac failure congestive | Cardiac disorders | 2 of 24 |
| Chest pain | General disorders | 2 of 24 |
| Urinary tract infection | Infections and infestations | 2 of 24 |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | 2 of 24 |
| Splenic infarction | Blood and lymphatic system disorders | 1 of 24 |
| Arrhythmia | Cardiac disorders | 1 of 24 |
| Left ventricular hypertrophy | Cardiac disorders | 1 of 24 |
| Ascites | Gastrointestinal disorders | 1 of 24 |
| Diarrhoea | Gastrointestinal disorders | 1 of 24 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Deferasirox |
|---|---|---|
| Diarrhoea | Gastrointestinal disorders | 15 of 24 |
| Nausea | Gastrointestinal disorders | 11 of 24 |
| Abdominal pain | Gastrointestinal disorders | 9 of 24 |
| Oedema peripheral | General disorders | 6 of 24 |
| Weight decreased | Investigations | 6 of 24 |
| Cough | Respiratory, thoracic and mediastinal disorders | 5 of 24 |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | 5 of 24 |
| Constipation | Gastrointestinal disorders | 4 of 24 |
| Vomiting | Gastrointestinal disorders | 4 of 24 |
| Upper respiratory tract infection | Infections and infestations | 4 of 24 |
Serious events with the highest arm-level rate: pyrexia (16.7%), cardiac failure congestive (8.3%), chest pain (8.3%), urinary tract infection (8.3%), dyspnoea (8.3%).
The deferasirox group has 11 participants listed with serious adverse events and 23 listed with adverse events; the at-risk count is 24 for each category. The record separately lists 3 deaths. These are counts of participants or deaths as labelled in the registry, not a causal assessment. The record does not state that the deaths were caused by deferasirox, nor does it provide a comparison group that could establish whether the observed counts differ from another treatment experience.
The serious adverse event terms are reported with these percentages: Pyrexia, 16.7%; Cardiac failure congestive, 8.3%; Chest pain, 8.3%; Urinary tract infection, 8.3%; Dyspnoea, 8.3%; and Splenic infarction, 4.2%. In general, a listed term and percentage describe the registry’s reporting for that term; they do not convey timing, duration, recurrence, or treatment relatedness. The record does not clarify overlap between terms or explain how these term-level percentages were derived. They should not be added together or treated as mutually exclusive.
Seriousness and severity are not interchangeable concepts. The registry record does not provide event severity grades or a detailed seriousness definition in the information. These figures are best read as descriptive safety reporting during the stated period, not as proof about cause or comparative risk.
12. What the Result Does, and Does Not, Mean
The results establish that the registry reports adverse events and serious adverse events among participants in this single-group study. The posted primary counts are 23 participants with adverse events and 11 with serious adverse events, with 24 listed at risk in each category. The record also lists 3 deaths and several serious adverse event terms with percentages. These observations describe what was recorded for this study’s safety population and specified observation period.
They do not establish that deferasirox caused any particular event. Events occurring after treatment begins may have multiple possible causes, and this record has no randomized comparison group to distinguish treatment-associated effects from background illness, other factors, or chance. The study’s open-label design and the absence of reported detail on event assessment further limit causal interpretation.
The counts do not show how often each participant experienced events, how long events lasted, how severe they were, or how event occurrence varied over time. The registry record does not report individual follow-up, confidence intervals, or a comparative analysis. Therefore, the posted values do not provide a measure of precision for an underlying risk estimate or a basis for comparing treatment strategies.
The study’s stated focus is safety and tolerability, but the primary counts alone do not summarize all aspects of tolerability. The registry lists secondary outcomes related to iron status and transfusion requirements, but numerical results for them are not reported in the registry record. No conclusion about those outcomes should be drawn from the safety counts.
In general, a completed single-group study can document observed experience and help describe the kinds of events recorded in its participants. Its findings are most informative when interpreted within the population, treatment schedule, and observation period described, and alongside the design limitations. They do not establish how the same participants would have fared under a different treatment or without treatment.
13. Why This Trial Matters Statistically
This trial illustrates why the design and the endpoint must be read together. The primary outcome is a participant count for adverse events and serious adverse events, which is suited to a descriptive safety summary. But the single-group design offers no randomized comparator. As a result, even clearly reported event counts cannot by themselves separate treatment-associated events from events that may occur in the setting of MDS, iron overload, or other circumstances.
The registry also makes the distinction between enrollment and the safety population important. It reports 24 enrolled participants, defines the safety set by treatment exposure and a post-baseline assessment, and lists 24 at risk for each primary event category. A reader should not assume from these values that follow-up was complete for every participant through Week 52; completeness of follow-up is not reported in the registry record.
The study is also a reminder that a list of registered secondary outcomes is not equivalent to a set of reported findings. Outcomes relating to ferritin, liver iron concentration, transfusion requirements, and other measures are named, but their numerical results are not reported in the registry record. Statistical interpretation should stay anchored to the results that are posted rather than treating planned or listed outcomes as observed evidence.
Finally, planning assumptions for sample-size illustrations are distinct from the design and analysis of the completed trial. The record’s planning statement describes assumptions for illustrations, while the actual study is recorded as single-group. Keeping these concepts separate prevents planning calculations from being mistaken for observed power or for a randomized comparison. Overall, this study is useful as a case in descriptive safety reporting and in recognizing the limits imposed by design, reporting, and missing uncertainty estimates.
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 main limitation for interpretation is the absence of a comparator group. The trial is recorded as single-group, with allocation not applicable, so the reported event counts cannot be used to estimate a comparative effect. The open-label design may influence event reporting or assessment, and the registry record does not describe independent adjudication or how investigators assessed treatment relatedness.
The record does not report confidence intervals, a statistical analysis plan, a sample-size justification for the trial, or a formal inferential analysis for the primary endpoint. Although planning assumptions for illustrations are listed, numerical illustration results are not reported in the registry record. Those assumptions should not be mistaken for the trial’s own statistical design or for evidence of observed power.
Details needed to evaluate completeness are also absent. The record does not report participant-level follow-up, withdrawals, missing safety assessments, treatment discontinuation, or how incomplete event information was handled. The primary window is up to Week 52, and the treatment description states 12 months, but the available counts do not show the duration of observation for each participant.
The serious adverse event terms are reported with percentages, but the record does not explain overlap among terms or give their timing, duration, severity, or relationship to study treatment. The separately listed deaths are not linked to a cause in the reported information. Numerical results for the registered secondary outcomes are not reported in the registry record. These limitations constrain both causal interpretation and assessment of outcomes beyond the primary safety counts.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Study for the Treatment of Transfusional Iron Overload in Myelodysplastic Patients — NCT00117507.
- Registry record last updated 2021-06-24; status last verified 2021-06.
- Results first posted 2021-06-24.
- Data retrieved from ClinicalTrials.gov on 2026-11-09.
- Group sizes, means, event counts and sponsor-reported p-values are reproduced as posted. Effect estimates, confidence intervals and p-values not attributed to the sponsor are independently calculated from the posted summary statistics.
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18. Record Summary
The registry describes a completed Phase 4, open-label, single-group study of oral deferasirox for transfusional iron overload in adults with low-risk or INT-1 MDS. Novartis Pharmaceuticals is listed as sponsor. The study started in 2005-09 and reached primary completion in 2008-01.
The summary states that 30 patients were planned and 24 were enrolled. Deferasirox was given at 20mg/kg/day for 12 months. The primary outcome counted participants with adverse events and serious adverse events up to Week 52. The posted group counts are 23 with adverse events, 11 with serious adverse events, and 3 deaths; the at-risk count is 24 for each primary category.
The record lists secondary outcomes concerning iron measures, transfusion requirements, and related biomarkers, but their numerical results are not reported in the registry record. There is no control group, and the reported safety counts are descriptive rather than comparative.