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 Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload (NCT00235391), a non-randomized phase 3 study. The primary outcome is Safety Profile of Deferasirox Based Upon Drug Administration and Reporting of Serious Adverse Events.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00235391 |
| Phase | Phase 3 |
| Condition | Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia |
| Allocation | Non-Randomized |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 1683 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Novartis Pharmaceuticals |
| Results posted | Yes |
2. Clinical Question
Population
People with congenital disorders of red blood cells and chronic iron overload from blood transfusions who could not be adequately treated with other locally approved iron chelators. The listed conditions are thalassemia, sickle cell disease, Diamond Blackfan anemia, and myelofibrosis. The safety population included participants who received at least one dose of deferasirox.
Intervention
Deferasirox, administered orally once a day. The recorded description says it was taken 30 minutes before breakfast, with dosage based on body weight and the starting dose determined by transfusion frequency.
Comparator
There was no concurrent comparator arm. The study was non-randomized and used a single-group model, so the registry record does not support a like-for-like treatment comparison.
Primary question
What safety events were recorded during treatment, including deaths, serious adverse events, and adverse events leading to study-drug interruption or discontinuation? The primary outcome is a count of participants, not a comparative estimate of treatment effect.
This is an expanded-access safety question rather than a randomized efficacy question. The registry record describes participants with chronic transfusion-related iron overload whose treatment options were limited by inadequate response to other locally approved iron chelators. It lists four underlying conditions, but does not report separate primary safety results for each condition. The record also does not provide a comparator group receiving another treatment or no deferasirox.
The main interpretation is therefore descriptive: the posted results count participants with specified safety events in the study population. Such counts can characterize what was reported during exposure, but without a comparable control group they cannot establish whether event frequencies were higher or lower because of treatment. The registry record also does not report an effectiveness result for the primary outcome. A change in serum ferritin is listed as a secondary outcome, but its results are not reported in the registry record.
3. Trial Design
The registry record identifies this as a phase 3, multi-center expanded-access study, with a single-group model, non-randomized allocation, and no masking. It was designed to provide access to deferasirox for people with congenital red blood cell disorders and chronic iron overload from transfusions who could not be adequately treated with other locally approved iron chelators. The record lists thalassemia, sickle cell disease, Diamond Blackfan anemia, and myelofibrosis as conditions. Enrollment was 1683, and the study status is completed.
Deferasirox was the sole intervention listed. The recorded administration was oral and once daily, 30 minutes before breakfast. Dose was based on body weight, and the starting dose was determined by the frequency of blood transfusions. The rest of the starting-dose description is not reported in the registry record. No other treatment arm or concurrent control is described.
The study began in 2005-10, and primary completion was recorded in 2008-10. The primary outcome concerned safety from baseline to the end of the study. The outcome description states that only treatment-emergent adverse events were summarized. Median exposure to the drug was approximately 30 weeks, and maximum exposure was 104 weeks. Those exposure durations matter when interpreting counts: participants did not necessarily contribute the same amount of time at risk.
The primary analysis population was the safety population, defined in the record as all participants who received at least one dose of deferasirox during the study. The registry record does not report a formal sample-size justification, a randomization procedure, or an inferential analysis plan. Because this was a single-group, open-label study, its design supports description of events during treatment, not a randomized estimate of how outcomes would differ under an alternative treatment.
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 | 2 to < 6 Years | 6 to < 12 Years | 12 to < 16 Years | 16 to < 50 Years | 50 to < 65 Years | ≥ 65 Years |
|---|---|---|---|---|---|---|
| STARTED | 97 | 200 | 172 | 1164 | 43 | 7 |
| Deferasirox Treatment | 97 | 200 | 172 | 1164 | 43 | 7 |
| COMPLETED | 85 | 182 | 148 | 944 | 31 | 3 |
| NOT COMPLETED | 12 | 18 | 24 | 220 | 12 | 4 |
Reasons for not completing this period:
| Reason | 2 to < 6 Years | 6 to < 12 Years | 12 to < 16 Years | 16 to < 50 Years | 50 to < 65 Years | ≥ 65 Years |
|---|---|---|---|---|---|---|
| Adverse Event | 4 | 5 | 10 | 72 | 5 | 2 |
| Abnormal laboratory value(s) | 3 | 5 | 4 | 27 | 1 | 2 |
| Unsatisfactory therapeutic effect | 0 | 2 | 2 | 17 | 0 | 0 |
| Condition no longer requires treatment | 3 | 0 | 0 | 11 | 1 | 0 |
| Protocol deviation | 0 | 0 | 1 | 11 | 0 | 0 |
| Withdrawal by Subject | 2 | 3 | 3 | 61 | 5 | 0 |
| Lost to Follow-up | 0 | 3 | 3 | 16 | 0 | 0 |
| Administrative problems | 0 | 0 | 0 | 1 | 0 | 0 |
| Death | 0 | 0 | 1 | 4 | 0 | 0 |
Analysis population for the primary outcome, as defined in the registry: The safety population, comprising all participants who received at least one dose of deferasirox during the study, was used in the analyses.
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.
Only the first 6 of 7 groups are shown.
| Characteristic | Unit | 2 to < 6 Years | 6 to < 12 Years | 12 to < 16 Years | 16 to < 50 Years | 50 to < 65 Years | ≥ 65 Years |
|---|---|---|---|---|---|---|---|
| Participants analysed | 97 | 200 | 172 | 1164 | 43 | 7 | |
| Age Continuous | years | 3.48 (SD 1.13) | 8.52 (SD 1.64) | 13.48 (SD 1.20) | 28.91 (SD 8.06) | 54.58 (SD 3.92) | 70.14 (SD 3.98) |
| Sex: Female, Male: Female | Participants | 44 | 101 | 81 | 633 | 32 | 5 |
| Sex: Female, Male: Male | Participants | 53 | 99 | 91 | 531 | 11 | 2 |
| Baseline Disease Characteristics: Beta-Thalassemia Major | participants | 74 | 118 | 114 | 905 | 10 | 0 |
| Baseline Disease Characteristics: Beta-Thalassemia Intermedia | participants | 1 | 22 | 16 | 94 | 20 | 3 |
| Baseline Disease Characteristics: Sickle Cell Disease | participants | 8 | 41 | 31 | 93 | 2 | 1 |
| Baseline Disease Characteristics: Diamond-Blackfan Anemia | participants | 4 | 7 | 8 | 24 | 0 | 0 |
| Baseline Disease Characteristics: Other Diseases | participants | 10 | 12 | 3 | 48 | 11 | 3 |
| Prior Chelation Drug Therapy: Deferoxamine | participants | 80 | 167 | 137 | 745 | 31 | 5 |
| Prior Chelation Drug Therapy: Deferiprone | participants | 1 | 8 | 11 | 146 | 6 | 1 |
| Prior Chelation Drug Therapy: Deferoxamine and Deferiprone | participants | 2 | 16 | 21 | 269 | 6 | 0 |
| Prior Chelation Drug Therapy: Other Chelation Drug | participants | 6 | 6 | 3 | 4 | 0 | 0 |
| Prior Chelation Drug Therapy: Prior Chelatation Drug Information Missing | participants | 8 | 3 | 0 | 0 | 0 | 1 |
| Reason for inadequate prior chelation therapy: Therapy non-compliance | participants | 57 | 125 | 106 | 659 | 25 | 2 |
| Reason for inadequate prior chelation therapy: Therapy contraindication | participants | 1 | 7 | 6 | 29 | 1 | 1 |
| Reason for inadequate prior chelation therapy: Therapy unacceptable toxicity | participants | 11 | 29 | 22 | 131 | 6 | 1 |
| Reason for inadequate prior chelation therapy: Therapy poor response | participants | 20 | 34 | 38 | 267 | 8 | 0 |
| Reason for inadequate prior chelation therapy: Therapy unacceptable discomfort | participants | 2 | 2 | 0 | 78 | 3 | 2 |
| Reason for inadequate prior chelation therapy: Reason for inadequate therapy information missing | participants | 6 | 3 | 0 | 0 | 0 | 1 |
6. Primary Endpoint
The registry lists the primary outcome as Safety Profile of Deferasirox Based Upon Drug Administration and Reporting of Serious Adverse Events, measured over Baseline to end of study (Median exposure time to drug was approximately 30 weeks; Maximum exposure was 104 weeks).
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary outcome is the safety profile of deferasirox, assessed through the number of participants with death, serious adverse events, and/or adverse events leading to study-drug interruption or discontinuation. The unit is participants, and the outcome parameter is a number of participants with an event. The record specifies that only treatment-emergent adverse events are summarized. The timeframe is baseline to the end of the study, with median drug exposure of approximately 30 weeks and maximum exposure of 104 weeks.
Counting participants is a direct way to describe how many people in the safety population had events that met the specified categories. The combined endpoint covers events of different kinds and severity: death, serious adverse events, and adverse events that led to treatment interruption or discontinuation. A participant might potentially meet more than one category, so category counts should not be added unless the record establishes that they are mutually exclusive. The registry record does not report a total count for the combined endpoint.
Safety monitoring is particularly relevant to an expanded-access study because the study's stated purpose was to provide treatment access in a population with chronic transfusion-related iron overload and limited suitability for other locally approved chelators. The endpoint does not, by itself, measure benefit or determine whether an event was caused by treatment. Nor does a participant count account for differences in exposure duration. The registry record gives exposure summaries, but does not report exposure-adjusted event rates or an analysis comparing events with a control group.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The posted primary outcome is descriptive. In the safety population of 1683 participants, 194 participants are recorded in the serious-adverse-event count, and 85 are recorded in the other adverse-event count. The latter category relates to adverse events leading to study-drug interruption or discontinuation, as described for the safety endpoint. These figures are counts of participants, not rates adjusted for time on treatment. The registry record does not report a death count, so the death component cannot be characterized from the posted results.
The serious-event terms listed most often are sickle cell anaemia with crisis (1.6%), pyrexia (0.5%), cardiac failure (0.4%), cholelithiasis (0.4%), non-cardiac chest pain (0.3%), and viral infection (0.3%). These entries describe terms and percentages in the registry record; they do not provide a treatment-versus-control comparison. The record does not state in the posted results how these terms were selected or provide a separate comparative analysis for them.
Several features limit what can be inferred. The study had no randomized comparator, no masking, and one treatment group. Participants could differ in underlying condition, transfusion history, health status, and time exposed, but condition-specific safety counts and exposure-adjusted comparisons are not reported in the registry record. As a result, the event counts cannot separate events associated with the underlying illnesses from events that might be associated with treatment, nor can they show what would have occurred under another treatment strategy.
The study lists a secondary outcome of change in serum ferritin from baseline through completion, but no result for that outcome is reported in the registry record. The record also lists zero analyses. Thus, the posted information does not provide inferential estimates, confidence intervals, or a formal statistical test for the primary safety outcome. Without such estimates, precision cannot be assessed beyond the raw participant counts and the exposure summaries.
These limitations do not make the counts meaningless: they document events reported in the study's defined safety population and give a descriptive account of treatment-emergent safety experience. But they must be read as observations within this particular single-group study, not as evidence that treatment caused, prevented, increased, or reduced events. A causal or comparative conclusion would require evidence the registry record does not report.
9. Statistical Methods Explained
What does a participant count represent?
For a binary safety outcome, each participant is classified according to whether the event occurred during the defined observation period. The reported count is the number of participants in the specified population who met that event definition. It is not necessarily the number of individual episodes: one person may experience more than one episode, while the participant count still records that person once for the relevant category. The registry record describes the primary outcome in terms of participants and reports 194 for serious adverse events and 85 for the other adverse-event category.
The denominator also matters. The safety population includes all participants who received at least one dose of deferasirox, and the adverse-event record gives 1683 participants at risk for both displayed categories. A count is easiest to interpret when its event definition, population, and observation period are clear. The record defines the safety population and states that only treatment-emergent events were summarized. It does not report a death count or a combined count for participants meeting any component of the primary endpoint.
Why is this not a treatment-effect estimate?
A treatment-effect estimate compares outcomes under different treatment conditions, or estimates a contrast supported by an appropriate study design. This study had one group, non-randomized allocation, and no masking. It therefore offers no concurrent randomized group against which to compare the observed safety counts. General statistical principle: a count observed during treatment does not, by itself, identify what would have happened to the same population under another treatment or without treatment. Differences in underlying illness, clinical circumstances, and exposure can affect observed event counts.
Because treatment assignment was not randomized, participants were not allocated to a like-for-like treatment comparison. Because the study was open-label, treatment was not masked. These features do not invalidate descriptive reporting, but they constrain causal interpretation. A comparison with external experience would require suitable data and methods; such a comparison is not reported in the registry record.
How should exposure time be considered?
Participants may contribute different amounts of time during which events can occur. The registry record reports median exposure of approximately 30 weeks and maximum exposure of 104 weeks, but does not provide exposure-adjusted event rates. General statistical principle: raw counts can be difficult to compare when time at risk differs, because people followed for longer have more opportunity to experience an event. Exposure-adjusted rates or time-to-event analyses can address some aspects of differing follow-up, subject to their assumptions and the completeness of time information. No such analysis is reported in the registry record.
The median and maximum exposure summaries describe the range of treatment exposure only in a limited way; they do not reveal each participant's follow-up duration or the timing of events. They also do not show whether people who stopped treatment continued to be followed for safety. Those details are not reported in the registry record.
What would uncertainty estimates add?
General statistical explanation: a confidence interval expresses the range of estimates that are reasonably compatible with observed data under a specified statistical model. It helps convey uncertainty around an estimate; it does not remove bias from a non-randomized design or establish causation. The registry record lists zero analyses and does not report confidence intervals, standard errors, or formal hypothesis tests for the primary safety outcome. Consequently, the precision of the reported counts is not quantified by an inferential interval in the posted results.
A p-value, when reported, addresses compatibility of data with a specified null model under stated assumptions. It is not a measure of clinical importance and does not establish a causal effect. No p-value or null hypothesis for the primary endpoint is reported in the registry record. There is therefore no basis to describe the primary outcome as statistically significant or not statistically significant.
What does over-dispersion mean in general?
General statistical explanation: count models often begin with assumptions about the expected relationship between the average count and its variability. Over-dispersion means observed variability is greater than the model allows under its initial assumptions. If ignored, uncertainty estimates can be too small and tests can appear more decisive than warranted. It is relevant mainly when modeling event counts or rates. The posted primary outcome is a participant count, and the registry record does not describe a count-model analysis or an assessment of over-dispersion. It would be inappropriate to infer that such a model was used.
How should the planning illustrations be read?
The registry record's sample-size assumptions describe a planning illustration based on an observed effect, using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. The illustration's effect is framed as a stated fraction of the observed effect. These assumptions are not a report of the trial's own design justification, and the record does not report that the study was planned as a randomized two-arm comparison. The actual study was single-group and non-randomized.
General statistical explanation: sample-size calculations describe how many participants a hypothetical design might require under its assumptions. They do not show the probability that the completed trial found a true effect, and they are not observed power. Planning for a fraction of an observed effect is a sensitivity exercise about a hypothetical target effect, not evidence that such an effect was detected. The registry record does not report a formal sample-size justification for the completed study. No numerical sample-size illustration values are reported in the registry record described for these outcomes.
The primary outcome is a participant count, while a planning calculation may use a modeled contrast and assumptions that are not the study's actual design. The planning framework should therefore not be mistaken for an analysis of the single-group safety data. The registry record's zero listed analyses further means there is no reported inferential calculation to interpret for the primary endpoint.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists a primary safety outcome and a secondary outcome measuring change in serum ferritin from baseline through completion. It reports two outcome measures overall, but no analyses are listed. The secondary outcome's results are not reported in the registry record. No family of statistical tests, multiplicity adjustment, hierarchy of hypotheses, or prespecified method for controlling false-positive findings is reported.
Multiplicity is a general concern when several outcomes or comparisons are tested: the more tests conducted, the greater the chance that at least one result appears unusual by chance under the relevant assumptions. Adjustment methods can address that issue, but the appropriate approach depends on the analysis plan and the purpose of the outcomes. Since the registry record reports no analyses or p-values, it is not possible to assess whether any adjustment was made. It would not be appropriate to infer an adjustment method or a confirmatory testing strategy.
Missing-data methods are also not reported in the registry record. The safety population is defined as all participants who received at least one dose, but the record does not describe missing outcome status, loss to follow-up, withdrawals, or how incomplete safety information was handled. For the serum ferritin outcome, the number with available measurements and the handling of missing measurements are not reported. These omissions limit assessment of whether the recorded participants and measurements represent the full enrolled group over the intended follow-up.
The primary safety definition includes death, serious adverse events, and adverse events leading to interruption or discontinuation, but no death count or combined endpoint total is reported. The registry record also does not state whether participants could be counted in multiple component categories. These reporting gaps prevent reconstruction of a unique composite outcome count from the posted category counts.
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 |
|---|---|---|---|
| All Participants | 194 | 1683 | 11.5% |
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 |
|---|---|
| All Participants | 85 of 1683 |
Most frequent serious adverse event terms
| Event term | Organ system | All Participants |
|---|---|---|
| Sickle cell anaemia with crisis | Congenital, familial and genetic disorders | 27 of 1683 |
| Pyrexia | General disorders | 9 of 1683 |
| Cardiac failure | Cardiac disorders | 6 of 1683 |
| Cholelithiasis | Hepatobiliary disorders | 6 of 1683 |
| Non-cardiac chest pain | General disorders | 5 of 1683 |
| Viral infection | Infections and infestations | 5 of 1683 |
| Pregnancy | Pregnancy, puerperium and perinatal conditions | 5 of 1683 |
| Rash | Skin and subcutaneous tissue disorders | 5 of 1683 |
| Splenomegaly | Blood and lymphatic system disorders | 4 of 1683 |
| Gastritis | Gastrointestinal disorders | 4 of 1683 |
Most frequent non-serious adverse event terms
| Event term | Organ system | All Participants |
|---|---|---|
| Rash | Skin and subcutaneous tissue disorders | 85 of 1683 |
Serious events with the highest arm-level rate: sickle cell anaemia with crisis (1.6%), pyrexia (0.5%), cardiac failure (0.4%), cholelithiasis (0.4%), non-cardiac chest pain (0.3%).
The safety record lists 1683 participants at risk for each of the two displayed event categories. It records 194 participants with serious adverse events and 85 in the other adverse-event category, which concerns events leading to study-drug interruption or discontinuation. These are participant counts over the study's reported observation period, not counts of event episodes and not exposure-adjusted rates.
The counts should not be added to create a total. The registry record does not say whether participants can appear in both categories, and the primary endpoint also includes death. A death count is not reported in the registry record. Nor is a separate total for participants with any component of the primary outcome reported.
The listed serious-event terms and percentages are sickle cell anaemia with crisis (1.6%), pyrexia (0.5%), cardiac failure (0.4%), cholelithiasis (0.4%), non-cardiac chest pain (0.3%), and viral infection (0.3%). These entries are descriptive, not comparative. The registry record does not report event rates adjusted for the differing exposure times; median exposure was approximately 30 weeks and maximum exposure was 104 weeks. The figures therefore describe what was recorded in this study, without establishing cause or comparison with an alternative.
12. What the Result Does, and Does Not, Mean
The result is a description of safety events recorded among participants who received at least one dose of deferasirox. The registry record reports participant counts for serious adverse events and for adverse events leading to study-drug interruption or discontinuation, alongside a list of serious-event terms and percentages. It also reports treatment exposure summaries. These data provide a record of observed events within the study population and its follow-up.
The result does not establish that deferasirox caused any particular event. Participants had underlying disorders associated with complex health circumstances, and the study did not include a randomized comparator. The record does not separate the contribution of treatment from the contribution of underlying illness, transfusion history, or other circumstances. Likewise, the event counts cannot show that treatment prevented events or reduced risk compared with another strategy.
The counts do not represent exposure-adjusted rates, and the record does not report confidence intervals or formal statistical tests. The primary endpoint includes death, but a death count is not reported. The listed categories may overlap, and no combined count is reported, so the category counts should not be summed. The registry record also does not report results for the secondary serum ferritin outcome.
General statistical interpretation: descriptive counts are useful for documenting observed experience, but they answer a narrower question than a comparative treatment-effect analysis. The strength and precision of any broader conclusion depend on a suitable comparison, clearly reported outcome definitions, complete follow-up, and analyses appropriate to the study design. Those elements are not reported for an inferential comparison in this registry record.
The appropriate reading is therefore bounded: the posted results describe safety events recorded in a completed, single-group expanded-access study. They do not provide a controlled estimate of relative risk, a causal conclusion, or a reported efficacy result. The absence of a reported death count or outcome analysis should be treated as missing information in the registry record, not as evidence that no such events or analyses occurred.
13. Why This Trial Matters Statistically
This trial is statistically informative because it illustrates the difference between a safety description and a comparative treatment evaluation. Its question concerned treatment-emergent events in an expanded-access population, and its primary endpoint was defined in participant counts. That design can document events observed during treatment, which is a distinct purpose from estimating how outcomes differ between treatment strategies.
The study's features make that distinction especially important. It was open-label, non-randomized, and single-group, with no concurrent control. Participants also had several listed underlying conditions, and the registry record does not report condition-specific safety analyses. A single overall count can summarize the study population but cannot show whether the event experience was similar across diagnoses or exposure histories.
The record also gives a reason to consider time at risk. Median exposure was approximately 30 weeks, while maximum exposure was 104 weeks. Because exposure duration can vary, raw participant counts do not fully describe event frequency over time. The registry record does not report exposure-adjusted rates or time-to-event analyses, so those differences cannot be evaluated statistically from the posted results.
Finally, the registry record lists no analyses and provides no confidence intervals or inferential tests for the primary outcome. This makes the record a useful example of how much descriptive safety reporting can convey—and what it cannot answer. Counts, event terms, and exposure summaries document observations, while uncertainty estimates and a suitable comparator would be needed for different questions about precision or relative effects. The posted data should be interpreted within that design and reporting scope.
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 is the absence of a concurrent comparator. Non-randomized allocation and a single-group model mean there is no like-for-like control comparison in the registry record. The open-label design also means treatment was not masked. These features limit causal interpretation of events observed during treatment, even though the study can describe those events.
The population covered several conditions: thalassemia, sickle cell disease, Diamond Blackfan anemia, and myelofibrosis. The registry record does not report condition-specific primary safety counts or analyses. It also does not provide subgroup results based on transfusion frequency, dose, or exposure duration. The recorded starting dose depended on transfusion frequency and dosage was based on body weight, but the full starting-dose details are not reported in the registry record.
Exposure varied: median exposure was approximately 30 weeks and maximum exposure was 104 weeks. No exposure-adjusted event rates or detailed follow-up times are reported. The primary outcome includes death, serious adverse events, and adverse events leading to interruption or discontinuation, but a death count and a combined count are not reported. The record does not clarify whether participants could contribute to more than one displayed category.
The registry record lists zero analyses. It does not report confidence intervals, hypothesis tests, missing-data handling, or results for the secondary serum ferritin outcome. It also does not report a formal sample-size justification. These omissions restrict evaluation of precision, completeness, and analysis choices. They should be understood as limitations of what is reported, not as proof that unreported events, procedures, or analyses did not occur.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload — NCT00235391.
- Registry record last updated 2011-06-07; status last verified 2011-06.
- Results first posted 2011-05-02.
- 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.
Continue through the Clinical Biostats statistical pathway
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18. Record Summary
This completed phase 3 study was a multi-center, open-label, non-randomized, single-group expanded-access study of deferasirox. Enrollment was 1683. The study began in 2005-10, and primary completion was recorded in 2008-10. It addressed people with congenital red blood cell disorders and chronic transfusion-related iron overload who could not be adequately treated with other locally approved iron chelators.
The primary outcome was a participant-count safety endpoint covering death, serious adverse events, and adverse events leading to study-drug interruption or discontinuation. The safety population consisted of participants who received at least one dose. The registry record reports serious adverse events for 194 participants and the other adverse-event category for 85; a death count and combined endpoint count are not reported. No analyses are listed, and the secondary serum ferritin result is not reported in the registry record.