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 Palatability and Tolerability of Deferasirox Taken With Meals, With Different Liquids or Crushed and Added to Food (NCT00845871), a na phase 4 study. The primary outcome is Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00845871 |
| Phase | Phase 4 |
| Condition | Transfusional Hemosiderosis |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 65 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Novartis Pharmaceuticals |
| Results posted | Yes |
2. Clinical Question
Population
Participants with transfusional hemosiderosis who met the study criteria and were taking, beginning, or resuming deferasirox. The study enrolled 65 participants across approximately 20 centers. For the primary analysis, the registry record defines the population as all enrolled participants, whether or not they received treatment.
Intervention
Deferasirox taken using different meal and administration methods. The title describes taking it with meals, with different liquids, or crushed and added to food. The regimen description gives a daily oral starting dose of 20 mg/kg, up to a maximum dose of 40 mg/kg/day.
Comparator
There was no separate control group. This was a single-group study with no random allocation and no masking. The administration conditions are not a randomized, like-for-like comparison, so differences between them cannot be interpreted as controlled treatment effects.
Primary question
What palatability ratings did participants report at Week 8 and Week 12 for deferasirox taken with food or by different administration methods? Ratings used a five-category Facial Hedonic scale, from “dislike extremely” to “like extremely.” For participants under 5 years of age, a parent or caregiver completed the scale.
3. Trial Design
This was a Phase 4, single-arm, open-label study of deferasirox. The registry record lists 65 enrolled participants and approximately 20 study centers. The study began in 2009-05, had a primary completion date of 2010-08, and is recorded as completed. Allocation was not applicable to the single-group design, and masking was none.
The brief summary describes a one-month run-in during which participants were instructed to take deferasirox according to their physician’s prescribing information. The treatment description gives a daily oral starting dose of 20 mg/kg, with a maximum dose of 40 mg/kg/day. The study title identifies the practical focus: palatability and tolerability when deferasirox is taken with meals, with different liquids, or crushed and added to food. The registry’s adverse-event group labels also identify breakfast, dinner, and no-meal conditions, each with liquid or soft food.
Because participants were not assigned to a separate control group, the administration conditions do not constitute a randomized comparison. The open-label design also means that participants and caregivers knew the administration method being assessed. These features matter especially for a subjective rating such as palatability. They do not make the recorded ratings unusable, but they limit what can be inferred from differences between conditions.
The registry record lists three secondary outcomes: adverse events, serious adverse events, discontinuations and interruptions; trough plasma concentration at Week 8, Week 12 and Week 16; and change from baseline in serum ferritin at Week 16. The specific eligibility criteria, treatment schedule beyond the stated regimen and run-in, and the reason for selecting the enrollment target 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: 82 participants were screened, out of which 65 participants enrolled into study. Study design comprised of a one-month run-in period where participants took deferasirox according to the physician, followed by a three-month assessment period. In assessment period, participants were instructed to document the selected method of administration and meal (breakfast, dinner, or no meal). Method of administration and meal type were kept same over week, but allowed to change once a new week began.
Run-in Period
| Stage | Deferasirox |
|---|---|
| STARTED | 65 |
| COMPLETED | 65 |
| NOT COMPLETED | 0 |
Assessment Period
| Stage | Deferasirox |
|---|---|
| STARTED | 65 |
| COMPLETED | 58 |
| NOT COMPLETED | 7 |
Reasons for not completing this period:
| Reason | Deferasirox |
|---|---|
| Adverse Event | 2 |
| Abnormal laboratory values | 1 |
| Abnormal test procedure results | 1 |
| Patient withdrew consent | 3 |
Analysis population for the primary outcome, as defined in the registry: The primary analysis was performed in the ITT population defined as all subjects enrolled regardless of any treatment received or not. As per the planned analysis, data were summarized by counts and percentages of each category of response when administered with food or with different methods of administration.
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: Analysis was performed in Intent-to-treat (ITT) population defined as all enrolled patients regardless of any treatment received or not. As per the planned analysis, data was summarized as overall by combining all Arms/Groups.
| Characteristic | Unit | Deferasirox |
|---|---|---|
| Participants analysed | 65 | |
| Age, Continuous | years | 27.0 (SD 22.88) |
| Sex: Female, Male: Female | Participants | 27 |
| Sex: Female, Male: Male | Participants | 38 |
| Race/Ethnicity, Customized: Caucasian | Participants | 26 |
| Race/Ethnicity, Customized: Black | Participants | 25 |
| Race/Ethnicity, Customized: Asian | Participants | 10 |
| Race/Ethnicity, Customized: Other | Participants | 4 |
| Taking Exjade prior to the study: Yes | Participants | 44 |
| Taking Exjade prior to the study: No | Participants | 21 |
6. Primary Endpoint
The registry lists the primary outcome as Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12, measured over Week 8 and Week 12.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | percentage of participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the percentage of participants in each category of palatability rating at Week 8 and Week 12. Participants rated the meal and method of administration using a five-point Facial Hedonic scale: “dislike extremely,” “somewhat dislike,” “neither like or dislike,” “somewhat like,” and “like extremely.” For participants under 5 years of age, a parent or caregiver completed the scale.
This endpoint is used to describe how acceptable the administration experience was to participants, rather than to measure a clinical change in transfusional hemosiderosis. Reporting the categories separately retains the distinction between strong dislike, mild dislike, a neutral response, mild liking, and strong liking. It does not assume that the distance between adjacent categories is equal. That is useful for an ordered response scale whose categories express direction and intensity but are not measurements on a continuous scale.
The planned summary was counts and percentages for each response category when deferasirox was administered with food or by different methods. The registry record identifies the primary analysis population as all enrolled participants, regardless of whether they received treatment. The endpoint’s category counts and percentages are not reported in the registry record. Consequently, the proportions of positive, neutral, or negative ratings cannot be described from the posted endpoint information.
Palatability ratings can help characterize the experience of taking a treatment in different ways. They are subjective reports, however, and are not by themselves evidence of a clinical treatment effect. Ratings can also be influenced by awareness of the administration method; the study was open-label.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central statistical feature is the absence of a randomized comparator. This study describes a group of participants’ experience with deferasirox under different meal and administration conditions; it does not estimate a controlled contrast between those conditions. The administration groups should not be read as independent treatment arms with comparable baseline characteristics, because the registry record does not describe random assignment.
The primary endpoint is categorical and is intended to be summarized as counts and percentages at Week 8 and Week 12. The registry record does not report those category-specific results. It therefore does not support a numerical account of how participants rated palatability, whether ratings differed by time point, or which administration condition received more favorable responses. A result cannot be reconstructed from the study title, the endpoint description, or the safety data.
The adverse-event results are descriptive. In the overall deferasirox group, 15 of 65 participants were reported with serious adverse events, and 52 of 65 with other adverse events; deaths were recorded as 0. The registry also gives counts by run-in and administration condition. Those counts describe the groups as labeled, but the groups are not randomized comparisons. Differences in their counts may reflect differences in the participants included, the size of the group at risk, or other features not detailed in the registry record. They do not establish that one administration method caused more or fewer events.
General statistical interpretation: percentages summarize the share of a defined group with an outcome, but their meaning depends on the denominator and on how participants were assigned to that group. Confidence intervals, when reported, describe uncertainty around an estimate under a statistical model; they do not correct for lack of randomization or remove possible bias from an open-label assessment. Confidence intervals for the primary palatability categories are not reported in the registry record.
The registry record states that the primary analysis used the intention-to-treat population, defined as all enrolled participants regardless of treatment received or not. This definition can preserve the originally defined population, but interpretation still depends on how missing ratings were handled. Missing-data procedures are not reported in the registry record. The posted information also does not report the category counts needed to evaluate the endpoint’s precision.
In short, the record establishes the study design, the rating instrument, the planned descriptive summary, and adverse-event counts. It does not provide the primary endpoint results or a controlled estimate comparing administration methods. The numerical safety summaries should be read as event descriptions within this study, not as evidence of comparative safety or causal effects.
9. Statistical Methods Explained
The primary outcome is a set of ordered response categories, not a single continuous measurement. Each category has a natural order from dislike to like, but the labels do not establish that the step from one category to the next represents an equal change in palatability. A general approach for this type of endpoint is to retain the category counts and percentages, as the registry record says was planned. Collapsing the categories into a single average would require additional assumptions and could hide differences in the response distribution.
What does a percentage describe?
General statistical explanation: a percentage is a proportion expressed on a percentage scale. It describes the number of participants with a response relative to the number included in the denominator. The denominator is essential: it identifies which participants contribute to the summary. The registry record says the primary analysis population included all enrolled participants, whether or not they received treatment, but the category-specific counts, percentages, and response denominators are not reported in the registry record. Without those details, the endpoint distribution cannot be assessed.
The planned summaries are descriptive. They would show how responses were distributed across the five categories at Week 8 and Week 12 and across the stated administration conditions. Descriptive summaries do not, by themselves, test whether any difference is statistically distinguishable from chance. Nor do they control for differences between participants in the conditions being described.
Why does the study design affect interpretation?
General statistical explanation: random allocation helps make groups comparable, on average, with respect to measured and unmeasured participant characteristics. In a single-group study without random allocation, that protection is absent. Here, the record identifies one study group and several meal or administration labels, but does not describe randomized assignment to those conditions. Thus, any observed difference in ratings or event counts across labels would be an association within the study, not an isolated effect of the administration method.
Open-label assessment is particularly relevant to a subjective endpoint. General statistical explanation: when participants know which method they are using, expectations or perceptions may influence how they report an experience. A caregiver’s report for a participant under 5 years of age is also a proxy assessment rather than a direct rating by that participant. These are interpretation considerations; the registry record does not quantify their influence.
What would uncertainty measures add?
General statistical explanation: a confidence interval gives a range of values compatible with the observed data under the assumptions of a statistical procedure. It can communicate precision more fully than a point estimate alone. For a categorical endpoint, uncertainty could be reported for category proportions or for a prespecified contrast, if an appropriate analysis were defined. The registry record does not report confidence intervals, a hypothesis test, or a prespecified contrast for the primary endpoint. No inferential conclusion about differences in palatability can therefore be drawn from the registry information.
For proportions, precision depends in part on how many participants contribute data to the relevant category summary. Small denominators generally make estimates less precise than large denominators. This is a general statistical principle, not a calculation of precision for this study: the primary category counts and denominators are not reported. The overall enrollment count alone does not reveal how many participants provided a rating at each time point or under each method.
How should repeated time points be understood?
The endpoint is assessed at Week 8 and Week 12. General statistical explanation: if the same participants contribute ratings at more than one time point, their responses may be related. Analyses of change or time trends should account for that dependence rather than treating repeated observations as unrelated. The registry record does not report participant-level response patterns, a model for repeated measurements, or a statistical comparison between the time points. The presence of two scheduled assessments should not be mistaken for evidence that a change occurred.
The registry states that the primary analysis was planned for the intention-to-treat population, defined as all enrolled participants regardless of treatment received or not, and that data were to be summarized by counts and percentages. It does not specify how missing ratings, partial participation, or participants without an assessment at a time point were handled. Such rules matter because the observed response distribution can depend on which participants contribute data.
What do the safety summaries represent?
The posted adverse-event data give, for each labeled group, the number at risk and the number affected by serious and other adverse events, as well as deaths. These are counts within the reported groups. General statistical explanation: a count is not a rate over time unless a time-at-risk denominator and exposure period are incorporated into the measure. The registry record does not describe a time-adjusted rate analysis. The safety figures should therefore be read as reported participant counts, not as person-time rates.
Over-dispersion is a general statistical concern when event counts vary more than a simple model expects; it can make standard errors too small if ignored. It is not possible to determine from the posted group counts whether over-dispersion is relevant, and the registry record does not report a count model or variance adjustment. In addition, the single-group design and nonrandomized condition labels preclude treating the group-level safety counts as a controlled comparison.
The record contains no listed analysis records for the outcomes. A detailed statistical analysis plan, model specifications, adjustment strategy, and diagnostic procedures are not reported in the registry record. The statistical methods that can be described with confidence are therefore limited to the planned descriptive summaries for the primary endpoint and the posted adverse-event counts.
10. Multiplicity, Missing Data and Analysis Populations
The registry record identifies one primary outcome and three secondary outcomes: adverse events, serious adverse events, discontinuation and interruption; trough plasma concentration at Week 8, Week 12 and Week 16; and change from baseline in serum ferritin at Week 16. Considering multiple outcomes or time points raises a general multiplicity issue: the more statistical comparisons made, the greater the opportunity for chance findings if each is interpreted as a separate confirmatory test. The registry record does not report a multiplicity adjustment, a hierarchy among secondary outcomes, or a detailed testing strategy. It also does not report inferential tests for the primary endpoint.
The primary analysis population is described as all enrolled participants, whether or not they received treatment. That is the registry’s stated intention-to-treat definition. The record says the planned primary summary was counts and percentages for each response category, but does not report how missing ratings were treated, whether participants could contribute at only one time point, or what denominators were used for the category summaries. These missing-data rules are important for interpreting percentages because the participants with recorded responses may differ from those without them.
The registry record does not report reasons for missing endpoint data, a missing-data model, or sensitivity analyses. It also does not report a sample-size justification for the trial’s own design. Any planning illustrations associated with the posted results are not the trial’s enrollment rationale or evidence of observed power. The record does not describe an interim analysis or a statistical analysis plan in sufficient detail to determine whether either was conducted.
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 (Run-in Phase) | 5 | 62 | 8.1% |
| Breakfast (Deferasirox With Soft Food) | 5 | 20 | 25.0% |
| Breakfast (Deferasirox With Liquid) | 0 | 18 | 0.0% |
| Dinner (Deferasirox With Soft Food) | 1 | 14 | 7.1% |
| Dinner (Deferasirox With Liquid) | 1 | 17 | 5.9% |
| No Meal (Deferasirox With Liquid) | 6 | 39 | 15.4% |
| No Meal (Deferasirox With Soft Food) | 0 | 30 | 0.0% |
| Deferasirox (Overall) | 15 | 65 | 23.1% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Deferasirox (Run-in Phase) | 0 of 62 |
| Breakfast (Deferasirox With Soft Food) | 0 of 20 |
| Breakfast (Deferasirox With Liquid) | 0 of 18 |
| Dinner (Deferasirox With Soft Food) | 0 of 14 |
| Dinner (Deferasirox With Liquid) | 0 of 17 |
| No Meal (Deferasirox With Liquid) | 0 of 39 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 4.90% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Deferasirox (Run-in Phase) | 30 of 62 |
| Breakfast (Deferasirox With Soft Food) | 10 of 20 |
| Breakfast (Deferasirox With Liquid) | 6 of 18 |
| Dinner (Deferasirox With Soft Food) | 6 of 14 |
| Dinner (Deferasirox With Liquid) | 6 of 17 |
| No Meal (Deferasirox With Liquid) | 18 of 39 |
Most frequent serious adverse event terms
| Event term | Organ system | Deferasirox (Run-in Phase) | Breakfast (Deferasirox With Soft Food) | Breakfast (Deferasirox With Liquid) | Dinner (Deferasirox With Soft Food) | Dinner (Deferasirox With Liquid) | No Meal (Deferasirox With Liquid) |
|---|---|---|---|---|---|---|---|
| Sickle cell anaemia with crisis | Congenital, familial and genetic disorders | 3 of 62 | 0 of 20 | 0 of 18 | 0 of 14 | 1 of 17 | 5 of 39 |
| Pyrexia | General disorders | 1 of 62 | 1 of 20 | 0 of 18 | 0 of 14 | 0 of 17 | 1 of 39 |
| Sepsis | Infections and infestations | 0 of 62 | 2 of 20 | 0 of 18 | 1 of 14 | 0 of 17 | 0 of 39 |
| Hypotension | Vascular disorders | 0 of 62 | 1 of 20 | 0 of 18 | 1 of 14 | 0 of 17 | 0 of 39 |
| Acute chest syndrome | Blood and lymphatic system disorders | 1 of 62 | 0 of 20 | 0 of 18 | 0 of 14 | 0 of 17 | 0 of 39 |
| Pancreatitis acute | Gastrointestinal disorders | 1 of 62 | 0 of 20 | 0 of 18 | 0 of 14 | 0 of 17 | 0 of 39 |
| Catheter related complication | General disorders | 0 of 62 | 1 of 20 | 0 of 18 | 0 of 14 | 0 of 17 | 0 of 39 |
| Cholelithiasis | Hepatobiliary disorders | 1 of 62 | 0 of 20 | 0 of 18 | 0 of 14 | 0 of 17 | 0 of 39 |
| Influenza | Infections and infestations | 0 of 62 | 0 of 20 | 0 of 18 | 0 of 14 | 0 of 17 | 1 of 39 |
| Perirectal abscess | Infections and infestations | 0 of 62 | 0 of 20 | 0 of 18 | 1 of 14 | 0 of 17 | 0 of 39 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Deferasirox (Run-in Phase) | Breakfast (Deferasirox With Soft Food) | Breakfast (Deferasirox With Liquid) | Dinner (Deferasirox With Soft Food) | Dinner (Deferasirox With Liquid) | No Meal (Deferasirox With Liquid) |
|---|---|---|---|---|---|---|---|
| Diarrhoea | Gastrointestinal disorders | 15 of 62 | 1 of 20 | 1 of 18 | 1 of 14 | 0 of 17 | 2 of 39 |
| Nausea | Gastrointestinal disorders | 9 of 62 | 1 of 20 | 0 of 18 | 2 of 14 | 1 of 17 | 3 of 39 |
| Pyrexia | General disorders | 7 of 62 | 2 of 20 | 0 of 18 | 3 of 14 | 0 of 17 | 4 of 39 |
| Abdominal pain | Gastrointestinal disorders | 6 of 62 | 1 of 20 | 2 of 18 | 2 of 14 | 0 of 17 | 1 of 39 |
| Headache | Nervous system disorders | 5 of 62 | 2 of 20 | 2 of 18 | 0 of 14 | 0 of 17 | 1 of 39 |
| Upper respiratory tract infection | Infections and infestations | 2 of 62 | 0 of 20 | 1 of 18 | 0 of 14 | 1 of 17 | 5 of 39 |
| Vomiting | Gastrointestinal disorders | 4 of 62 | 1 of 20 | 0 of 18 | 1 of 14 | 0 of 17 | 2 of 39 |
| Fatigue | General disorders | 2 of 62 | 1 of 20 | 2 of 18 | 2 of 14 | 0 of 17 | 1 of 39 |
| Abdominal pain upper | Gastrointestinal disorders | 2 of 62 | 0 of 20 | 0 of 18 | 1 of 14 | 1 of 17 | 2 of 39 |
| Pain in extremity | Musculoskeletal and connective tissue disorders | 1 of 62 | 2 of 20 | 1 of 18 | 0 of 14 | 0 of 17 | 2 of 39 |
Serious events with the highest arm-level rate: sickle cell anaemia with crisis (12.8%), sepsis (10.0%), perirectal abscess (7.1%), hypotension (7.1%), catheter related complication (5.0%).
The registry reports serious and other adverse-event counts for the run-in, meal and administration-condition groups, and an overall deferasirox group. Overall, 15 of 65 participants were reported with serious adverse events, and 52 of 65 with other adverse events; deaths were recorded as 0. In the run-in group, the corresponding counts were 5 of 62 and 30 of 62. The labeled administration groups report serious-event counts of 5 of 20 with breakfast and soft food, 0 of 18 with breakfast and liquid, 1 of 14 with dinner and soft food, 1 of 17 with dinner and liquid, 6 of 39 with no meal and liquid, and 0 of 30 with no meal and soft food.
These are descriptive counts, not randomized contrasts. The group labels and counts do not establish that an administration condition caused an event or changed its likelihood. The registry lists serious-event terms including sickle cell anaemia with crisis, sepsis, perirectal abscess, hypotension, catheter related complication, and pyrexia, with percentages of 12.8%, 10.0%, 7.1%, 7.1%, 5.0%, and 5.0%, respectively. The denominator for these term percentages is not reported in the registry record, so they should not be interpreted as percentages of all enrolled participants.
12. What the Result Does, and Does Not, Mean
The study addresses a practical question about the palatability and tolerability of deferasirox taken with meals, with different liquids, or crushed and added to food. Its primary endpoint is designed to describe participants’ ratings at Week 8 and Week 12, using an ordered five-category scale. The study also records adverse events and other secondary outcomes. These features make the trial a descriptive assessment of administration experience and reported safety events in the enrolled group.
The registry record does not report the primary endpoint’s category counts or percentages. As a result, the posted information does not establish what ratings participants gave, how responses were distributed, or whether ratings differed across time points or administration methods. It also does not report a controlled comparison. The record therefore cannot support a conclusion that one method was more palatable than another.
The safety counts document reported serious and other adverse events in the named groups, including the overall group. They should not be interpreted as proof that deferasirox or a particular administration method caused an event. A single-group, open-label study without random allocation cannot separate the effect of the administration method from other differences among participants or conditions. General statistical explanation: descriptive results characterize the observed study group; causal conclusions usually require a design and analysis that address alternative explanations.
The registry record reports trough plasma concentration and serum ferritin as secondary outcomes, but does not give their results in the information available for this analysis. No conclusion about those measures can be drawn from the outcome names alone. The same distinction applies to discontinuations and interruptions: they are listed as outcomes, but their results are not reported in the registry record.
Accordingly, the record is informative about the study’s question, design, rating scale, and reported adverse-event counts. It does not provide the primary palatability results or a randomized estimate of comparative effects. Those limits define what the posted data can and cannot establish; they are not a judgment about how any individual should take treatment.
13. Why This Trial Matters Statistically
This trial is statistically notable because its main outcome is a patient-reported, ordered rating rather than a laboratory measurement or a randomized treatment contrast. The five-category Facial Hedonic scale preserves distinctions from strong dislike to strong liking. Such categories are meaningful as ordered responses, but they do not automatically support treating the ratings as equally spaced numerical measurements. The registry’s planned use of counts and percentages is consistent with describing the distribution category by category.
The study also illustrates the difference between evaluating an administration experience and comparing treatment effects. Deferasirox was assessed under meal and administration conditions, but the design is single-group and open-label, with no random allocation. A difference between condition labels, if reported, would not by itself isolate the effect of the method. Participant characteristics, selection into a condition, and awareness of the method could all matter; the registry does not report analyses addressing these possibilities.
There is a second important statistical lesson in the gap between an endpoint’s planned analysis and its reported results. The record specifies the primary time points, response categories, and intended descriptive summaries, but does not report the category counts or percentages. Without those results, readers cannot evaluate the palatability distribution or its precision. A well-defined endpoint does not substitute for numerical outcome reporting.
Finally, the safety results show why group denominators and study design must be considered together. The registry reports numbers at risk and numbers affected, but the administration labels are not randomized comparison arms. The counts can describe events in the recorded groups while remaining insufficient for causal claims. Reading design, endpoint scale, denominators, and reporting completeness together is essential for a statistically careful interpretation of this study.
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 absence of random allocation and a separate control group is a central limitation for comparisons between administration methods. The condition labels do not guarantee that groups were comparable, and differences in their outcomes could have explanations other than the method itself. The study is also open-label. Because palatability is self-reported or, for participants under 5 years of age, reported by a parent or caregiver, awareness of the method could influence the assessment.
The registry record does not report primary category counts or percentages, so the principal outcome cannot be evaluated quantitatively from the posted endpoint information. It also does not report the response denominators at Week 8 and Week 12, confidence intervals, or a statistical comparison between time points or administration conditions. Missing-data handling and reasons for missing ratings are not reported in the registry record, limiting assessment of whether the recorded responses represent all enrolled participants.
The total enrollment was 65, and the adverse-event summaries show different numbers at risk across the labeled groups. These group counts should not be treated as balanced or directly comparable. The registry record does not provide a sample-size justification for the trial’s own design, so the intended precision or ability to detect a particular difference is not established by the enrollment number alone.
Although secondary outcomes include plasma concentration, serum ferritin, and discontinuation or interruption, their numerical results are not reported in the registry record. A detailed statistical analysis plan, multiplicity strategy, interim analysis information, and specific procedures for handling missing data are also not reported. These limitations restrict both endpoint interpretation and comparisons across the study conditions.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Palatability and Tolerability of Deferasirox Taken With Meals, With Different Liquids or Crushed and Added to Food — NCT00845871.
- Registry record last updated 2021-07-20; status last verified 2021-07.
- Results first posted 2021-07-20.
- 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
NCT00845871 is a completed Phase 4, single-arm, open-label study of deferasirox in participants with transfusional hemosiderosis. The registry lists 65 enrolled participants and approximately 20 centers. The study began in 2009-05 and had a primary completion date of 2010-08. The treatment description gives a daily oral starting dose of 20 mg/kg, up to a maximum of 40 mg/kg/day.
The primary outcome concerns percentages of participants in five palatability categories at Week 8 and Week 12. The intended summary was counts and percentages in the intention-to-treat population, defined as all enrolled participants regardless of treatment received. Primary category results are not reported in the registry record. Safety results include 15 of 65 participants with serious adverse events, 52 of 65 with other adverse events, and 0 deaths in the overall group. The nonrandomized, single-group design supports descriptive reporting, not causal comparisons of administration methods.