Transfusional Hemosiderosis
Conditions
Keywords
Exjade, deferasirox, palatability, tolerability, food, iron overload, Serum Iron overload due to transfusions
Brief summary
This single-arm, open-label, multi-center study enrolled 65 patients from approximately 20 centers. All patients who met the study criteria and were taking, beginning or resuming treatment with Deferasirox were allowed. The study will began with a one month run-in phase, where all patients were instructed to take Deferasirox according to their physician's prescribing information.
Detailed description
Following the run-in phase, patients entered a three month, assessment phase. During the assessment phase, patients were given five general options for taking Deferasirox including with or without meals, crushed and added to a soft food or mixed in a liquid of choice.
Interventions
DRUGdeferasirox:
Participants were administered daily with deferasirox starting dose of 20 mg/kg orally to a maximum dose of 40 mg/kg/day.
Sponsors
Novartis Pharmaceuticals
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
2 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Male or female patients with thalassemia major, sickle cell disease (SCD), low or intermediate 1 (INT 1) risk myelodysplastic syndrome (MDS) or other anemias and transfusional hemosiderosis. * Patients who were on, starting, or resuming treatment with Exjade. * Patients who were \>2 years (i.e., 2 years of age or older).
Exclusion criteria
* Serum creatinine above the upper limit of normal (ULN) for age. * Alanine aminotransferase (ALT) \>2.5 times the ULN.-High risk intermediate-2 or high risk MDS or acute leukemia.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 8 and Week 12 | Palatability was assessed by participants based on a five-point Facial Hedonic scale defined as: dislike extremely; somewhat dislike; neither like or dislike; somewhat like; like extremely for the meal and method of administration. For participants under 5 years of age, the scale was completed by parent or caregiver. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption | Day 1 up to Week 16 | Adverse events (AEs) were defined as any unfavorable and unintended diagnosis, symptom, sign (including an abnormal laboratory finding), syndrome or disease which either occurs during study, having been absent at baseline, or, if present at baseline, appears to worsen. Serious adverse events (SAEs) were defined as any untoward medical occurrences that result in death, are life threatening, require (or prolong) hospitalization, cause persistent or significant disability/incapacity, result in congenital anomalies or birth defects, or are other conditions which in judgement of investigators represent significant hazards. Subjects who had permanently terminated from the treatment or kept the treatment on hold/deviated from protocol due to adverse event were defined as subjects with permanent discontinuation and temporary interruption, respectively. |
| Trough Plasma Concentration of Deferasirox at Week 8, Week 12 and Week 16 | Pre-dose (0), 1, 2, 4 and 6 hour (post-dose) at Week 8, 12 and 16 | Blood samples were drawn at every visit as close as possible to 24 hours post dose from each subject participating in the study and trough plasma concentrations were estimated. |
| Change From Baseline in Serum Ferritin at Week 16 | Baseline, Week 16 (End of study) | Ferritin protein stores iron and provides overall iron levels. Higher ferritin in blood showed higher iron content. Fluctuations from normal serum ferritin levels (500 ng/mL) observed at two consecutive visits led to dose adjustment of deferasirox. |
Countries
United States
Participant flow
Recruitment details
The study was conducted at 17 centres in United States.
Pre-assignment details
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.
Participants by arm
| Arm | Count |
|---|---|
| Deferasirox Participants were administered daily with deferasirox at a minimum starting dose of 20 mg/kg/day along with different food and liquids consistently for 12 weeks of assessment period. For participants receiving \> 30 mg/kg/day, a maximum of 40 mg/kg/day was allowed. | 65 |
| Total | 65 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Assessment Period | Abnormal laboratory values | 1 |
| Assessment Period | Abnormal test procedure results | 1 |
| Assessment Period | Adverse Event | 2 |
| Assessment Period | Patient withdrew consent | 3 |
Baseline characteristics
| Characteristic | Deferasirox |
|---|---|
| Age, Continuous | 27.0 years STANDARD_DEVIATION 22.88 |
| Race/Ethnicity, Customized Asian | 10 Participants |
| Race/Ethnicity, Customized Black | 25 Participants |
| Race/Ethnicity, Customized Caucasian | 26 Participants |
| Race/Ethnicity, Customized Other | 4 Participants |
| Sex: Female, Male Female | 27 Participants |
| Sex: Female, Male Male | 38 Participants |
| Taking Exjade prior to the study No | 21 Participants |
| Taking Exjade prior to the study Yes | 44 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk | EG004 affected / at risk | EG005 affected / at risk | EG006 affected / at risk | EG007 affected / at risk |
|---|---|---|---|---|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 62 | 0 / 20 | 0 / 18 | 0 / 14 | 0 / 17 | 0 / 39 | 0 / 30 | 0 / 65 |
| other Total, other adverse events | 30 / 62 | 10 / 20 | 6 / 18 | 6 / 14 | 6 / 17 | 18 / 39 | 9 / 30 | 52 / 65 |
| serious Total, serious adverse events | 5 / 62 | 5 / 20 | 0 / 18 | 1 / 14 | 1 / 17 | 6 / 39 | 0 / 30 | 15 / 65 |
Outcome results
Primary
Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12
Palatability was assessed by participants based on a five-point Facial Hedonic scale defined as: dislike extremely; somewhat dislike; neither like or dislike; somewhat like; like extremely for the meal and method of administration. For participants under 5 years of age, the scale was completed by parent or caregiver.
Time frame: Week 8 and Week 12
Population: 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.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Breakfast (Deferasirox With Soft Food) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 12, Neither like or dislike | 16.7 percentage of participants |
| Breakfast (Deferasirox With Soft Food) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 12, Dislike extremely | 0 percentage of participants |
| Breakfast (Deferasirox With Soft Food) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 12, Like extremely | 50 percentage of participants |
| Breakfast (Deferasirox With Soft Food) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 8, Like extremely | 40 percentage of participants |
| Breakfast (Deferasirox With Soft Food) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 12, Somewhat like | 33.3 percentage of participants |
| Breakfast (Deferasirox With Soft Food) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 8, Neither like or dislike | 20 percentage of participants |
| Breakfast (Deferasirox With Soft Food) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 8, Somewhat like | 30 percentage of participants |
| Breakfast (Deferasirox With Soft Food) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 8, Somewhat dislike | 0 percentage of participants |
| Breakfast (Deferasirox With Soft Food) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 8, Dislike extremely | 10 percentage of participants |
| Breakfast (Deferasirox With Soft Food) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 12, Somewhat dislike | 0 percentage of participants |
| Breakfast (Deferasirox With Liquid) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 8, Somewhat dislike | 28.6 percentage of participants |
| Breakfast (Deferasirox With Liquid) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 8, Somewhat like | 0 percentage of participants |
| Breakfast (Deferasirox With Liquid) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 12, Like extremely | 50 percentage of participants |
| Breakfast (Deferasirox With Liquid) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 8, Dislike extremely | 0 percentage of participants |
| Breakfast (Deferasirox With Liquid) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 8, Like extremely | 42.9 percentage of participants |
| Breakfast (Deferasirox With Liquid) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 12, Neither like or dislike | 25 percentage of participants |
| Breakfast (Deferasirox With Liquid) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 12, Dislike extremely | 0 percentage of participants |
| Breakfast (Deferasirox With Liquid) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 12, Somewhat like | 0 percentage of participants |
| Breakfast (Deferasirox With Liquid) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 8, Neither like or dislike | 28.6 percentage of participants |
| Breakfast (Deferasirox With Liquid) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 12, Somewhat dislike | 25 percentage of participants |
| Dinner (Deferasirox With Soft Food) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 8, Like extremely | 66.7 percentage of participants |
| Dinner (Deferasirox With Soft Food) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 8, Dislike extremely | 0 percentage of participants |
| Dinner (Deferasirox With Soft Food) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 12, Like extremely | 33.3 percentage of participants |
| Dinner (Deferasirox With Soft Food) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 12, Somewhat like | 66.7 percentage of participants |
| Dinner (Deferasirox With Soft Food) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 8, Somewhat dislike | 0 percentage of participants |
| Dinner (Deferasirox With Soft Food) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 8, Neither like or dislike | 33.3 percentage of participants |
| Dinner (Deferasirox With Soft Food) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 12, Neither like or dislike | 0 percentage of participants |
| Dinner (Deferasirox With Soft Food) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 8, Somewhat like | 0 percentage of participants |
| Dinner (Deferasirox With Soft Food) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 12, Somewhat dislike | 0 percentage of participants |
| Dinner (Deferasirox With Soft Food) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 12, Dislike extremely | 0 percentage of participants |
| Dinner (Deferasirox With Liquid) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 8, Like extremely | 28.6 percentage of participants |
| Dinner (Deferasirox With Liquid) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 8, Dislike extremely | 0 percentage of participants |
| Dinner (Deferasirox With Liquid) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 8, Somewhat dislike | 14.3 percentage of participants |
| Dinner (Deferasirox With Liquid) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 8, Neither like or dislike | 28.6 percentage of participants |
| Dinner (Deferasirox With Liquid) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 8, Somewhat like | 28.6 percentage of participants |
| Dinner (Deferasirox With Liquid) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 12, Dislike extremely | 0 percentage of participants |
| Dinner (Deferasirox With Liquid) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 12, Somewhat dislike | 0 percentage of participants |
| Dinner (Deferasirox With Liquid) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 12, Neither like or dislike | 33.3 percentage of participants |
| Dinner (Deferasirox With Liquid) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 12, Somewhat like | 33.3 percentage of participants |
| Dinner (Deferasirox With Liquid) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 12, Like extremely | 33.3 percentage of participants |
| No Meal (Deferasirox With Liquid) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 8, Neither like or dislike | 29.4 percentage of participants |
| No Meal (Deferasirox With Liquid) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 8, Somewhat dislike | 5.9 percentage of participants |
| No Meal (Deferasirox With Liquid) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 8, Somewhat like | 17.6 percentage of participants |
| No Meal (Deferasirox With Liquid) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 12, Like extremely | 26.1 percentage of participants |
| No Meal (Deferasirox With Liquid) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 12, Somewhat like | 8.7 percentage of participants |
| No Meal (Deferasirox With Liquid) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 8, Dislike extremely | 0 percentage of participants |
| No Meal (Deferasirox With Liquid) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 12, Dislike extremely | 4.3 percentage of participants |
| No Meal (Deferasirox With Liquid) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 12, Neither like or dislike | 56.5 percentage of participants |
| No Meal (Deferasirox With Liquid) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 12, Somewhat dislike | 4.3 percentage of participants |
| No Meal (Deferasirox With Liquid) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 8, Like extremely | 47.1 percentage of participants |
| No Meal Deferasirox With Soft Food) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 8, Somewhat dislike | 14.3 percentage of participants |
| No Meal Deferasirox With Soft Food) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 12, Somewhat like | 10 percentage of participants |
| No Meal Deferasirox With Soft Food) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 12, Somewhat dislike | 10 percentage of participants |
| No Meal Deferasirox With Soft Food) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 8, Neither like or dislike | 28.6 percentage of participants |
| No Meal Deferasirox With Soft Food) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 12, Like extremely | 30 percentage of participants |
| No Meal Deferasirox With Soft Food) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 12, Neither like or dislike | 50 percentage of participants |
| No Meal Deferasirox With Soft Food) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 8, Somewhat like | 42.9 percentage of participants |
| No Meal Deferasirox With Soft Food) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 8, Dislike extremely | 0 percentage of participants |
| No Meal Deferasirox With Soft Food) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 12, Dislike extremely | 0 percentage of participants |
| No Meal Deferasirox With Soft Food) | Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12 | Week 8, Like extremely | 14.3 percentage of participants |
Secondary
Change From Baseline in Serum Ferritin at Week 16
Ferritin protein stores iron and provides overall iron levels. Higher ferritin in blood showed higher iron content. Fluctuations from normal serum ferritin levels (500 ng/mL) observed at two consecutive visits led to dose adjustment of deferasirox.
Time frame: Baseline, Week 16 (End of study)
Population: The analysis was performed in the ITT population. The 'n' signifies those subjects evaluable for this measure at specified time points for each group respectively. As per the planned analysis, data was summarized as overall by combining all Arms/Groups.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Breakfast (Deferasirox With Soft Food) | Change From Baseline in Serum Ferritin at Week 16 | Age 2 to < 10 years | -198.1 Nanogram/mlillilitre | Standard Deviation 653.11 |
| Breakfast (Deferasirox With Soft Food) | Change From Baseline in Serum Ferritin at Week 16 | Age 10 to < 60 years | 38.3 Nanogram/mlillilitre | Standard Deviation 859.45 |
| Breakfast (Deferasirox With Soft Food) | Change From Baseline in Serum Ferritin at Week 16 | Age ≥ 60 years | -593 Nanogram/mlillilitre | Standard Deviation 1683.39 |
Secondary
Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption
Adverse events (AEs) were defined as any unfavorable and unintended diagnosis, symptom, sign (including an abnormal laboratory finding), syndrome or disease which either occurs during study, having been absent at baseline, or, if present at baseline, appears to worsen. Serious adverse events (SAEs) were defined as any untoward medical occurrences that result in death, are life threatening, require (or prolong) hospitalization, cause persistent or significant disability/incapacity, result in congenital anomalies or birth defects, or are other conditions which in judgement of investigators represent significant hazards. Subjects who had permanently terminated from the treatment or kept the treatment on hold/deviated from protocol due to adverse event were defined as subjects with permanent discontinuation and temporary interruption, respectively.
Time frame: Day 1 up to Week 16
Population: The analysis was performed in the Safety Set (SAF) population, defined as all participants who received at least one dose of study medication. 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.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Breakfast (Deferasirox With Soft Food) | Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption | AEs leading to permanent discontinuation | 0 Participants |
| Breakfast (Deferasirox With Soft Food) | Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption | AEs | 39 Participants |
| Breakfast (Deferasirox With Soft Food) | Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption | Death | 0 Participants |
| Breakfast (Deferasirox With Soft Food) | Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption | SAEs | 5 Participants |
| Breakfast (Deferasirox With Soft Food) | Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption | AEs leading to temporary interruption | 4 Participants |
| Breakfast (Deferasirox With Liquid) | Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption | AEs leading to permanent discontinuation | 2 Participants |
| Breakfast (Deferasirox With Liquid) | Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption | SAEs | 5 Participants |
| Breakfast (Deferasirox With Liquid) | Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption | AEs leading to temporary interruption | 2 Participants |
| Breakfast (Deferasirox With Liquid) | Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption | AEs | 10 Participants |
| Breakfast (Deferasirox With Liquid) | Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption | Death | 0 Participants |
| Dinner (Deferasirox With Soft Food) | Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption | Death | 0 Participants |
| Dinner (Deferasirox With Soft Food) | Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption | SAEs | 0 Participants |
| Dinner (Deferasirox With Soft Food) | Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption | AEs | 6 Participants |
| Dinner (Deferasirox With Soft Food) | Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption | AEs leading to permanent discontinuation | 0 Participants |
| Dinner (Deferasirox With Soft Food) | Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption | AEs leading to temporary interruption | 1 Participants |
| Dinner (Deferasirox With Liquid) | Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption | AEs leading to permanent discontinuation | 0 Participants |
| Dinner (Deferasirox With Liquid) | Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption | AEs | 6 Participants |
| Dinner (Deferasirox With Liquid) | Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption | SAEs | 1 Participants |
| Dinner (Deferasirox With Liquid) | Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption | AEs leading to temporary interruption | 0 Participants |
| Dinner (Deferasirox With Liquid) | Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption | Death | 0 Participants |
| No Meal (Deferasirox With Liquid) | Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption | AEs | 6 Participants |
| No Meal (Deferasirox With Liquid) | Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption | Death | 0 Participants |
| No Meal (Deferasirox With Liquid) | Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption | AEs leading to permanent discontinuation | 0 Participants |
| No Meal (Deferasirox With Liquid) | Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption | AEs leading to temporary interruption | 0 Participants |
| No Meal (Deferasirox With Liquid) | Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption | SAEs | 1 Participants |
| No Meal Deferasirox With Soft Food) | Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption | AEs | 24 Participants |
| No Meal Deferasirox With Soft Food) | Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption | Death | 0 Participants |
| No Meal Deferasirox With Soft Food) | Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption | AEs leading to temporary interruption | 2 Participants |
| No Meal Deferasirox With Soft Food) | Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption | SAEs | 6 Participants |
| No Meal Deferasirox With Soft Food) | Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption | AEs leading to permanent discontinuation | 0 Participants |
| No Meal Deferasirox With Soft Food) | Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption | AEs leading to temporary interruption | 0 Participants |
| No Meal Deferasirox With Soft Food) | Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption | SAEs | 0 Participants |
| No Meal Deferasirox With Soft Food) | Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption | AEs leading to permanent discontinuation | 1 Participants |
| No Meal Deferasirox With Soft Food) | Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption | AEs | 11 Participants |
| No Meal Deferasirox With Soft Food) | Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption | Death | 0 Participants |
Secondary
Trough Plasma Concentration of Deferasirox at Week 8, Week 12 and Week 16
Blood samples were drawn at every visit as close as possible to 24 hours post dose from each subject participating in the study and trough plasma concentrations were estimated.
Time frame: Pre-dose (0), 1, 2, 4 and 6 hour (post-dose) at Week 8, 12 and 16
Population: The 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 was summarized as overall by combining all Arms/Groups.
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Breakfast (Deferasirox With Soft Food) | Trough Plasma Concentration of Deferasirox at Week 8, Week 12 and Week 16 | Week 8, Deferasirox 20 mg/kg | 36.30 micromoles per litre (μmol/L) |
| Breakfast (Deferasirox With Soft Food) | Trough Plasma Concentration of Deferasirox at Week 8, Week 12 and Week 16 | Week 12, Deferasirox 20 mg/kg | 66.95 micromoles per litre (μmol/L) |
| Breakfast (Deferasirox With Soft Food) | Trough Plasma Concentration of Deferasirox at Week 8, Week 12 and Week 16 | Week 16, Deferasirox 20 mg/kg | 27.75 micromoles per litre (μmol/L) |
| Breakfast (Deferasirox With Soft Food) | Trough Plasma Concentration of Deferasirox at Week 8, Week 12 and Week 16 | Week 8, Deferasirox 30 mg/kg | 20.15 micromoles per litre (μmol/L) |
| Breakfast (Deferasirox With Soft Food) | Trough Plasma Concentration of Deferasirox at Week 8, Week 12 and Week 16 | Week 12, Deferasirox 30 mg/kg | 34.30 micromoles per litre (μmol/L) |
| Breakfast (Deferasirox With Soft Food) | Trough Plasma Concentration of Deferasirox at Week 8, Week 12 and Week 16 | Week 16, Deferasirox 30 mg/kg | 54.50 micromoles per litre (μmol/L) |