Chronic Iron Overload Due to Transfusion-dependant Anemias
Conditions
Keywords
Iron overload, Chelation, Thalassemia, Myelodysplastic syndrome (MDS), Transfusional hemisiderosis, Deferasirox, ICL670, Dispersible tablet, Film-coated tablet
Brief summary
Assessed the new film-coated tablet formulation to the currently approved dispersible tablet formulation with regards to overall safety, Gastrointestinal (GI) tolerability, palatability, satisfaction and compliance
Interventions
DRUGDeferasirox dispersible tablet
Deferasirox DT was provided as 125 mg, 250 mg and 500 mg dispersible tablets for oral use.
DRUGDefearisox film-coated tablet
Deferasirox FCT was provided as 90 mg, 180 mg and 360 mg film-coated tablets for oral use.
Sponsors
Novartis Pharmaceuticals
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
10 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: * Male and female patients aged ≥ 10 years * Patients with transfusion-dependent thalassemia and iron overload, requiring deferasirox DT at doses of ≥ 30 mg/kg/day as per the investigator's decision OR Patients with very low, low or intermediate (int) risk myelodysplastic syndrome (MDS) and iron overload, requiring deferasirox DT at doses of ≥ 20 mg/kg/day as per the investigator's decision. * History of transfusion of at least 20 PRBC units and anticipated to be transfused with at least 8 units of PRBCs annually during the study * Serum ferritin \> 1000 ng/mL, measured at screening Visit 1 and screening Visit 2 (the mean value will be used for eligibility criteria). Key
Exclusion criteria
* Creatinine clearance below the contraindication limit in the locally approved prescribing information. Creatinine clearance will be estimated from serum creatinine at screening Visit 1 and screening Visit 2 and the mean value will be used for eligibility criteria. * Serum creatinine \> 1.5 xULN at screening measured at screening Visit 1 and screening Visit 2 (the mean value will be used for eligibility criteria). * ALT (SGPT) \> 5xULN, unless LIC confirmed as \>10 mg Fe/dw within 6 months prior to screening visit 1. * Significant proteinuria as indicated by a urinary protein/creatinine ratio \> 0.5 mg/mg in a non-first void urine sample at screening Visit 1 or screening Visit 2. * Patients with significant impaired gastrointestinal (GI) function or GI disease that may significantly alter the absorption of oral deferasirox (e.g. ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, or small bowel resection). * Liver disease with severity of Child-Pugh Class B or C
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Overall Safety as Measured by Frequency of Adverse Events | 28 weeks | The percentage of participants with adverse events, serious adverse events and deaths was assessed. |
| Overall Safety as Measured by Changes in Laboratory Values From Baseline | baseline (BL), 30 weeks | The percentage of participants with post-baseline laboratory values meeting specified criteria for notable/extended range was assessed. The following laboratory parameters were measured: platelet count, absolute neutrophils, serum creatinine , creatinine clearance, urinary protein/urinary creatinine ratio, alanine aminotransferase (ALT) and aspartate aminotransferase (AST). Note that within data categories, creat = creatinine, cons = consecutive, ULN = upper limit of normal and urin = urinary. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Palatability Questionnaire Score | weeks 2, 3, 13 and 24 (end of treatment or within 7 days of last dose) | The palatability questionnaire consisted of 4 items. The first item measured the taste and aftertaste of the medication and were scored a on a 5-point response scale. The second item offered an additional response option of no aftertaste. The last 2 items referred to whether the medication was taken, i.e. swallowed or vomited, and how the participant perceived the amount of medication to be taken. The palatability summary score was calculated using a scoring matrix from items 1, 3 and 4 scores and the score ranges from 0 - 11. Higher scores indicated the best palatability. A meaningful difference between two treatment arms was determined to be 1 point. |
| Weekly Average of Daily Scores of the Gastrointestinal (GI) Symptom Diary | weeks -1, 4, 8, 12, 16, 20, 24 | The GI symptom diary consisted of 6 items, five which were scored using a 0 - 10 rating scale with item appropriate anchors to rate the symptom, for example, Pain in your belly: 0 = no pain and 10 = worst pain. The GI diary summary score was created using the 10 point response scale for the 5 items. The GI symptom daily diary had a minimum score of 0 and a maximum score of 50. The weekly average score for the 7 days was calculated for each individual item and the GI summary score was created from these weekly averages. Higher scores indicated worse symptoms. A meaningful difference between two treatment arms was determined to be 0.3 point. |
| Number of Participants With Weekly Average Compliance of Medication Consumption | Weeks 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 23, 24 | A compliance questionnaire assessed whether the medication was taken. Weekly average compliance was calculated when there were at least four non-missing daily responses. |
| Weekly Dose Violation Rate | weeks 1, 4, 8, 12, 16, 20, 24 | The dose violation is defined as a dose either missed completely or not taken in accordance with the timing instruction (no later than 12:00 pm. The rate was calculated as \[number of dose violations/drug exposure (days)\] x 100. |
| Frequency of Selected Gastro-intestinal (GI) Adverse Events | 28 weeks | The percentage of participants with any GI adverse event, diarrhea, constipation, nausea, vomiting, abdominal pain was assessed. |
| Observed Maximum Plasma Concentration Following Drug Administration (Cmax) | week 1, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose; week 3, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose | Blood samples were collected to assess Cmax. |
| Time to Reach the Maximum Plasma Concentration After Drug Administration (Tmax) | week 1, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose; week 3, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose | Blood samples were collected to assess Tmax. |
| Dererasirox Plasma Concentration | Week 3, day 1, pre-dose (0 hour (h)) and 2 h post-dose; week 13, day 1, pre-dose (0 hour (h)) and 2 h post-dose; and week 21, day 1, pre-dose (0 hour (h)) and 2 h post-dose | Blood samples were collected to assess deferasirox concentration. Dose-adjusted calculations are presented: (concentration/actual dose)\*20 for participants on DFX-DT and (concentration/actual dose)\*14 for participants on DFX-FCT. |
| Area Under the Plasma Concentration-time Curve From Time Zero to the Last Quantifiable Concentration (AUClast) | week 1, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose; week 3, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose | Blood samples were collected to assess AUClast. |
| Mean Domain Scores of the Modified Satisfaction With Iron Chelation Therapy (Modified SICT) | weeks 2, 3, 13 and 24 (end of treatment or within 7 days of last dose) | The modified SICT consisted of 13 items that represent 3 domains: adherence, satisfaction and concerns. The adherence domain consisted of 7 items, 6 which were measured using a 5-point response scale and was calculated by summing the 6 items. The score range from 6 to 30 and higher scores indicated worse adherence. The satisfaction domain consisted of 3 items, 2 which were measured using a 5-point response scale and was calculated by summing the 2 items. The score range from 2 to 10 and higher scores indicated worse satisfaction. The concerns domain consisted of 3 items to address any concerns or worries with his/her medication. All 3 items were measured on a 5-point response scale and were calculated by summing the 3 items. The score range from 3 to 15 and higher scores indicated fewer concerns. For all three domains, the meaningful difference between two treatment arms was determined to be 1 point. |
Countries
Argentina, Austria, France, Germany, Greece, Italy, Lebanon, Malaysia, Mexico, Russia, Saudi Arabia, Spain, Thailand, United Arab Emirates, United Kingdom, United States
Participant flow
Pre-assignment details
Participants were randomized in a 1:1 ratio.
Participants by arm
| Arm | Count |
|---|---|
| Deferasirox Dispersible Tablet (DFX-DT) Iron chelation naïve participants received DFX-DT 20 mg/kg/day once daily orally from weeks 1 - 4. After week 4, the dose could be adjusted by +/- 5 to 10 mg/kg/day, with a maximum dose of 40 mg/kg/day. Iron chelation pre-treated participants were supposed to start on a dose that was equivalent to their pre-washout dose. | 86 |
| Deferasirox Film-coated Tablet (DFX-FCT) Participants received DFX-FCT 14 mg/kg/day once daily orally from weeks 1 - 4. After week 4, the dose could be adjusted by +/- 3.5 to 7 mg/kg/day, with a maximum dose of 28 mg/kg/day. Iron chelation pre-treated participants were supposed to start on a dose that was equivalent to their pre-washout dose | 87 |
| Total | 173 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Administrative problems | 0 | 1 |
| Overall Study | Adverse Event | 6 | 4 |
| Overall Study | Death | 0 | 1 |
| Overall Study | Participant/guardian decision | 0 | 2 |
| Overall Study | Physician Decision | 1 | 0 |
| Overall Study | Protocol deviation | 4 | 1 |
| Overall Study | Withdrawal by Subject | 2 | 1 |
Baseline characteristics
| Characteristic | Deferasirox Dispersible Tablet (DFX-DT) | Deferasirox Film-coated Tablet (DFX-FCT) | Total |
|---|---|---|---|
| Age, Continuous | 35.1 Years STANDARD_DEVIATION 18.6 | 34.6 Years STANDARD_DEVIATION 19.97 | 34.9 Years STANDARD_DEVIATION 19.25 |
| Sex: Female, Male Female | 47 Participants | 41 Participants | 88 Participants |
| Sex: Female, Male Male | 39 Participants | 46 Participants | 85 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 69 / 86 | 72 / 87 |
| serious Total, serious adverse events | 13 / 86 | 16 / 87 |
Outcome results
Primary
Overall Safety as Measured by Changes in Laboratory Values From Baseline
The percentage of participants with post-baseline laboratory values meeting specified criteria for notable/extended range was assessed. The following laboratory parameters were measured: platelet count, absolute neutrophils, serum creatinine , creatinine clearance, urinary protein/urinary creatinine ratio, alanine aminotransferase (ALT) and aspartate aminotransferase (AST). Note that within data categories, creat = creatinine, cons = consecutive, ULN = upper limit of normal and urin = urinary.
Time frame: baseline (BL), 30 weeks
Population: The safety set, which included all participants who received at least one dose of study drug, was analyzed.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Deferasirox Dispersible Tablet (DFX-DT) | Overall Safety as Measured by Changes in Laboratory Values From Baseline | platelet count, notable range: <100 x 10^9/L | 9.3 Percentage of participants |
| Deferasirox Dispersible Tablet (DFX-DT) | Overall Safety as Measured by Changes in Laboratory Values From Baseline | platelet count, extended range: <50 x 10^9/L | 3.5 Percentage of participants |
| Deferasirox Dispersible Tablet (DFX-DT) | Overall Safety as Measured by Changes in Laboratory Values From Baseline | absolute neutrophils, notable range: <1.5 x 10^9/L | 8.1 Percentage of participants |
| Deferasirox Dispersible Tablet (DFX-DT) | Overall Safety as Measured by Changes in Laboratory Values From Baseline | absolute neut., extended range: <0.5 x 10^9/L | 4.7 Percentage of participants |
| Deferasirox Dispersible Tablet (DFX-DT) | Overall Safety as Measured by Changes in Laboratory Values From Baseline | creat clearance, notable range: 2 cons <60mL/min | 7.0 Percentage of participants |
| Deferasirox Dispersible Tablet (DFX-DT) | Overall Safety as Measured by Changes in Laboratory Values From Baseline | creat clearance, extended range: 2 cons <40mL/min | 2.3 Percentage of participants |
| Deferasirox Dispersible Tablet (DFX-DT) | Overall Safety as Measured by Changes in Laboratory Values From Baseline | urin protein/urin creat ratio, 2 cons >1.0 mg/mg | 2.3 Percentage of participants |
| Deferasirox Dispersible Tablet (DFX-DT) | Overall Safety as Measured by Changes in Laboratory Values From Baseline | ALT, notable range: >5 x ULN and >2 x BL | 1.2 Percentage of participants |
| Deferasirox Dispersible Tablet (DFX-DT) | Overall Safety as Measured by Changes in Laboratory Values From Baseline | ALT, extended range: >10 x ULN and >2 x BL | 1.2 Percentage of participants |
| Deferasirox Dispersible Tablet (DFX-DT) | Overall Safety as Measured by Changes in Laboratory Values From Baseline | AST, notable range: >5 x ULN and >2 x BL | 0 Percentage of participants |
| Deferasirox Dispersible Tablet (DFX-DT) | Overall Safety as Measured by Changes in Laboratory Values From Baseline | AST, extended range: >10 x ULN and >2 x BL | 1.2 Percentage of participants |
| Deferasirox Dispersible Tablet (DFX-DT) | Overall Safety as Measured by Changes in Laboratory Values From Baseline | serum creat, 2 cons >33% incr. from BL and >ULN | 4.7 Percentage of participants |
| Deferasirox Film-coated Tablet (DFX-FCT) | Overall Safety as Measured by Changes in Laboratory Values From Baseline | creat clearance, extended range: 2 cons <40mL/min | 2.3 Percentage of participants |
| Deferasirox Film-coated Tablet (DFX-FCT) | Overall Safety as Measured by Changes in Laboratory Values From Baseline | platelet count, notable range: <100 x 10^9/L | 8.0 Percentage of participants |
| Deferasirox Film-coated Tablet (DFX-FCT) | Overall Safety as Measured by Changes in Laboratory Values From Baseline | ALT, extended range: >10 x ULN and >2 x BL | 0 Percentage of participants |
| Deferasirox Film-coated Tablet (DFX-FCT) | Overall Safety as Measured by Changes in Laboratory Values From Baseline | platelet count, extended range: <50 x 10^9/L | 5.7 Percentage of participants |
| Deferasirox Film-coated Tablet (DFX-FCT) | Overall Safety as Measured by Changes in Laboratory Values From Baseline | urin protein/urin creat ratio, 2 cons >1.0 mg/mg | 0 Percentage of participants |
| Deferasirox Film-coated Tablet (DFX-FCT) | Overall Safety as Measured by Changes in Laboratory Values From Baseline | absolute neutrophils, notable range: <1.5 x 10^9/L | 13.8 Percentage of participants |
| Deferasirox Film-coated Tablet (DFX-FCT) | Overall Safety as Measured by Changes in Laboratory Values From Baseline | AST, extended range: >10 x ULN and >2 x BL | 0 Percentage of participants |
| Deferasirox Film-coated Tablet (DFX-FCT) | Overall Safety as Measured by Changes in Laboratory Values From Baseline | absolute neut., extended range: <0.5 x 10^9/L | 0 Percentage of participants |
| Deferasirox Film-coated Tablet (DFX-FCT) | Overall Safety as Measured by Changes in Laboratory Values From Baseline | serum creat, 2 cons >33% incr. from BL and >ULN | 3.4 Percentage of participants |
| Deferasirox Film-coated Tablet (DFX-FCT) | Overall Safety as Measured by Changes in Laboratory Values From Baseline | ALT, notable range: >5 x ULN and >2 x BL | 1.1 Percentage of participants |
| Deferasirox Film-coated Tablet (DFX-FCT) | Overall Safety as Measured by Changes in Laboratory Values From Baseline | creat clearance, notable range: 2 cons <60mL/min | 2.3 Percentage of participants |
| Deferasirox Film-coated Tablet (DFX-FCT) | Overall Safety as Measured by Changes in Laboratory Values From Baseline | AST, notable range: >5 x ULN and >2 x BL | 1.1 Percentage of participants |
Primary
Overall Safety as Measured by Frequency of Adverse Events
The percentage of participants with adverse events, serious adverse events and deaths was assessed.
Time frame: 28 weeks
Population: The safety set, which included all participants who received at least one dose of study drug, was analyzed.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Deferasirox Dispersible Tablet (DFX-DT) | Overall Safety as Measured by Frequency of Adverse Events | SAEs | 15.1 Percentage of participants |
| Deferasirox Dispersible Tablet (DFX-DT) | Overall Safety as Measured by Frequency of Adverse Events | Deaths | 0 Percentage of participants |
| Deferasirox Dispersible Tablet (DFX-DT) | Overall Safety as Measured by Frequency of Adverse Events | Adverse events | 89.5 Percentage of participants |
| Deferasirox Film-coated Tablet (DFX-FCT) | Overall Safety as Measured by Frequency of Adverse Events | Adverse events | 89.7 Percentage of participants |
| Deferasirox Film-coated Tablet (DFX-FCT) | Overall Safety as Measured by Frequency of Adverse Events | SAEs | 18.4 Percentage of participants |
| Deferasirox Film-coated Tablet (DFX-FCT) | Overall Safety as Measured by Frequency of Adverse Events | Deaths | 1.1 Percentage of participants |
Secondary
Area Under the Plasma Concentration-time Curve From Time Zero to the Last Quantifiable Concentration (AUClast)
Blood samples were collected to assess AUClast.
Time frame: week 1, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose; week 3, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose
Population: The Pharmacokinetic subset A analysis set was considered for the analysis, but only participants with non-missing values were analyzed.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Deferasirox Dispersible Tablet (DFX-DT) | Area Under the Plasma Concentration-time Curve From Time Zero to the Last Quantifiable Concentration (AUClast) | week1 (n=14,15) | 1110 umol/L*h | Standard Deviation 495 |
| Deferasirox Dispersible Tablet (DFX-DT) | Area Under the Plasma Concentration-time Curve From Time Zero to the Last Quantifiable Concentration (AUClast) | week 3 (n=13,15) | 1590 umol/L*h | Standard Deviation 540 |
| Deferasirox Film-coated Tablet (DFX-FCT) | Area Under the Plasma Concentration-time Curve From Time Zero to the Last Quantifiable Concentration (AUClast) | week1 (n=14,15) | 1040 umol/L*h | Standard Deviation 405 |
| Deferasirox Film-coated Tablet (DFX-FCT) | Area Under the Plasma Concentration-time Curve From Time Zero to the Last Quantifiable Concentration (AUClast) | week 3 (n=13,15) | 2110 umol/L*h | Standard Deviation 987 |
Secondary
Dererasirox Plasma Concentration
Blood samples were collected to assess deferasirox concentration. Dose-adjusted calculations are presented: (concentration/actual dose)\*20 for participants on DFX-DT and (concentration/actual dose)\*14 for participants on DFX-FCT.
Time frame: Week 3, day 1, pre-dose (0 hour (h)) and 2 h post-dose; week 13, day 1, pre-dose (0 hour (h)) and 2 h post-dose; and week 21, day 1, pre-dose (0 hour (h)) and 2 h post-dose
Population: The Pharmacokinetic analysis set for all participants was considered for this analysis, but only participants with non-missing values were included in the analysis.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Deferasirox Dispersible Tablet (DFX-DT) | Dererasirox Plasma Concentration | week 3, pre-dose (n=63,70) | 39.6 umol/L | Standard Deviation 48.4 |
| Deferasirox Dispersible Tablet (DFX-DT) | Dererasirox Plasma Concentration | week 3, 2 hours post-dose (n=67,76) | 80.8 umol/L | Standard Deviation 52.2 |
| Deferasirox Dispersible Tablet (DFX-DT) | Dererasirox Plasma Concentration | week 13, pre-dose (n=69.56) | 37.1 umol/L | Standard Deviation 37.8 |
| Deferasirox Dispersible Tablet (DFX-DT) | Dererasirox Plasma Concentration | week 21, pre-dose (n=54,59) | 46.6 umol/L | Standard Deviation 46.4 |
| Deferasirox Dispersible Tablet (DFX-DT) | Dererasirox Plasma Concentration | week 21, 2 hours post-dose (n=59,64) | 89.8 umol/L | Standard Deviation 59.3 |
| Deferasirox Dispersible Tablet (DFX-DT) | Dererasirox Plasma Concentration | week 13, 2 hours post-dose (n=74,59) | 78.7 umol/L | Standard Deviation 39.5 |
| Deferasirox Film-coated Tablet (DFX-FCT) | Dererasirox Plasma Concentration | week 21, 2 hours post-dose (n=59,64) | 105 umol/L | Standard Deviation 51.2 |
| Deferasirox Film-coated Tablet (DFX-FCT) | Dererasirox Plasma Concentration | week 3, pre-dose (n=63,70) | 27.3 umol/L | Standard Deviation 20.4 |
| Deferasirox Film-coated Tablet (DFX-FCT) | Dererasirox Plasma Concentration | week 21, pre-dose (n=54,59) | 43.1 umol/L | Standard Deviation 36.8 |
| Deferasirox Film-coated Tablet (DFX-FCT) | Dererasirox Plasma Concentration | week 3, 2 hours post-dose (n=67,76) | 95.5 umol/L | Standard Deviation 53 |
| Deferasirox Film-coated Tablet (DFX-FCT) | Dererasirox Plasma Concentration | week 13, pre-dose (n=69.56) | 31.3 umol/L | Standard Deviation 22.9 |
| Deferasirox Film-coated Tablet (DFX-FCT) | Dererasirox Plasma Concentration | week 13, 2 hours post-dose (n=74,59) | 92.5 umol/L | Standard Deviation 39.1 |
Secondary
Frequency of Selected Gastro-intestinal (GI) Adverse Events
The percentage of participants with any GI adverse event, diarrhea, constipation, nausea, vomiting, abdominal pain was assessed.
Time frame: 28 weeks
Population: The safety set, which included all participants who received at least one dose of study drug, was analyzed.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Deferasirox Dispersible Tablet (DFX-DT) | Frequency of Selected Gastro-intestinal (GI) Adverse Events | Abdominal pain | 26.7 Percentage of participants |
| Deferasirox Dispersible Tablet (DFX-DT) | Frequency of Selected Gastro-intestinal (GI) Adverse Events | Vomiting | 22.1 Percentage of participants |
| Deferasirox Dispersible Tablet (DFX-DT) | Frequency of Selected Gastro-intestinal (GI) Adverse Events | Constipation | 15.1 Percentage of participants |
| Deferasirox Dispersible Tablet (DFX-DT) | Frequency of Selected Gastro-intestinal (GI) Adverse Events | Nausea | 26.7 Percentage of participants |
| Deferasirox Dispersible Tablet (DFX-DT) | Frequency of Selected Gastro-intestinal (GI) Adverse Events | Diarrhea | 34.9 Percentage of participants |
| Deferasirox Dispersible Tablet (DFX-DT) | Frequency of Selected Gastro-intestinal (GI) Adverse Events | Any GI adverse event | 61.6 Percentage of participants |
| Deferasirox Film-coated Tablet (DFX-FCT) | Frequency of Selected Gastro-intestinal (GI) Adverse Events | Diarrhea | 33.3 Percentage of participants |
| Deferasirox Film-coated Tablet (DFX-FCT) | Frequency of Selected Gastro-intestinal (GI) Adverse Events | Any GI adverse event | 58.6 Percentage of participants |
| Deferasirox Film-coated Tablet (DFX-FCT) | Frequency of Selected Gastro-intestinal (GI) Adverse Events | Abdominal pain | 26.4 Percentage of participants |
| Deferasirox Film-coated Tablet (DFX-FCT) | Frequency of Selected Gastro-intestinal (GI) Adverse Events | Nausea | 27.6 Percentage of participants |
| Deferasirox Film-coated Tablet (DFX-FCT) | Frequency of Selected Gastro-intestinal (GI) Adverse Events | Vomiting | 17.2 Percentage of participants |
| Deferasirox Film-coated Tablet (DFX-FCT) | Frequency of Selected Gastro-intestinal (GI) Adverse Events | Constipation | 8.0 Percentage of participants |
Secondary
Mean Domain Scores of the Modified Satisfaction With Iron Chelation Therapy (Modified SICT)
The modified SICT consisted of 13 items that represent 3 domains: adherence, satisfaction and concerns. The adherence domain consisted of 7 items, 6 which were measured using a 5-point response scale and was calculated by summing the 6 items. The score range from 6 to 30 and higher scores indicated worse adherence. The satisfaction domain consisted of 3 items, 2 which were measured using a 5-point response scale and was calculated by summing the 2 items. The score range from 2 to 10 and higher scores indicated worse satisfaction. The concerns domain consisted of 3 items to address any concerns or worries with his/her medication. All 3 items were measured on a 5-point response scale and were calculated by summing the 3 items. The score range from 3 to 15 and higher scores indicated fewer concerns. For all three domains, the meaningful difference between two treatment arms was determined to be 1 point.
Time frame: weeks 2, 3, 13 and 24 (end of treatment or within 7 days of last dose)
Population: The safety set, which included all participants who received at least one dose of study drug, was considered for the analysis. However, only participants with values at the given week were included in the analysis for that week.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Deferasirox Dispersible Tablet (DFX-DT) | Mean Domain Scores of the Modified Satisfaction With Iron Chelation Therapy (Modified SICT) | week 2, adherence (n=70,70) | 10.3 score on a scale | Standard Deviation 3.8 |
| Deferasirox Dispersible Tablet (DFX-DT) | Mean Domain Scores of the Modified Satisfaction With Iron Chelation Therapy (Modified SICT) | week 2, satisfaction/preference (n=70,70) | 5.2 score on a scale | Standard Deviation 2.24 |
| Deferasirox Dispersible Tablet (DFX-DT) | Mean Domain Scores of the Modified Satisfaction With Iron Chelation Therapy (Modified SICT) | week 2, concerns (n=70,70) | 12.9 score on a scale | Standard Deviation 2.94 |
| Deferasirox Dispersible Tablet (DFX-DT) | Mean Domain Scores of the Modified Satisfaction With Iron Chelation Therapy (Modified SICT) | week 3, adherence (n=58,51) | 10.9 score on a scale | Standard Deviation 4.09 |
| Deferasirox Dispersible Tablet (DFX-DT) | Mean Domain Scores of the Modified Satisfaction With Iron Chelation Therapy (Modified SICT) | week 3, satisfaction/preference (n=58,51) | 5.4 score on a scale | Standard Deviation 2.22 |
| Deferasirox Dispersible Tablet (DFX-DT) | Mean Domain Scores of the Modified Satisfaction With Iron Chelation Therapy (Modified SICT) | week 3, concerns (n=58,51) | 12.4 score on a scale | Standard Deviation 2.73 |
| Deferasirox Dispersible Tablet (DFX-DT) | Mean Domain Scores of the Modified Satisfaction With Iron Chelation Therapy (Modified SICT) | week 13, adherence (n=59,64) | 11.2 score on a scale | Standard Deviation 3.56 |
| Deferasirox Dispersible Tablet (DFX-DT) | Mean Domain Scores of the Modified Satisfaction With Iron Chelation Therapy (Modified SICT) | week 13, satisfaction/preference (n=59,64) | 5.4 score on a scale | Standard Deviation 2.14 |
| Deferasirox Dispersible Tablet (DFX-DT) | Mean Domain Scores of the Modified Satisfaction With Iron Chelation Therapy (Modified SICT) | week 13, concerns (n=59,64) | 12.7 score on a scale | Standard Deviation 2.5 |
| Deferasirox Dispersible Tablet (DFX-DT) | Mean Domain Scores of the Modified Satisfaction With Iron Chelation Therapy (Modified SICT) | week 24, adherence (n=63,60) | 12.5 score on a scale | Standard Deviation 5.32 |
| Deferasirox Dispersible Tablet (DFX-DT) | Mean Domain Scores of the Modified Satisfaction With Iron Chelation Therapy (Modified SICT) | week 24, satisfaction/preference (n=63,60) | 5.8 score on a scale | Standard Deviation 2.28 |
| Deferasirox Dispersible Tablet (DFX-DT) | Mean Domain Scores of the Modified Satisfaction With Iron Chelation Therapy (Modified SICT) | week 24, concerns (n=63,60) | 11.8 score on a scale | Standard Deviation 3.07 |
| Deferasirox Film-coated Tablet (DFX-FCT) | Mean Domain Scores of the Modified Satisfaction With Iron Chelation Therapy (Modified SICT) | week 24, satisfaction/preference (n=63,60) | 2.9 score on a scale | Standard Deviation 1.58 |
| Deferasirox Film-coated Tablet (DFX-FCT) | Mean Domain Scores of the Modified Satisfaction With Iron Chelation Therapy (Modified SICT) | week 2, adherence (n=70,70) | 7.6 score on a scale | Standard Deviation 2.14 |
| Deferasirox Film-coated Tablet (DFX-FCT) | Mean Domain Scores of the Modified Satisfaction With Iron Chelation Therapy (Modified SICT) | week 13, adherence (n=59,64) | 7.8 score on a scale | Standard Deviation 2.05 |
| Deferasirox Film-coated Tablet (DFX-FCT) | Mean Domain Scores of the Modified Satisfaction With Iron Chelation Therapy (Modified SICT) | week 2, satisfaction/preference (n=70,70) | 2.8 score on a scale | Standard Deviation 1.37 |
| Deferasirox Film-coated Tablet (DFX-FCT) | Mean Domain Scores of the Modified Satisfaction With Iron Chelation Therapy (Modified SICT) | week 24, adherence (n=63,60) | 7.5 score on a scale | Standard Deviation 2.41 |
| Deferasirox Film-coated Tablet (DFX-FCT) | Mean Domain Scores of the Modified Satisfaction With Iron Chelation Therapy (Modified SICT) | week 2, concerns (n=70,70) | 13.8 score on a scale | Standard Deviation 2.02 |
| Deferasirox Film-coated Tablet (DFX-FCT) | Mean Domain Scores of the Modified Satisfaction With Iron Chelation Therapy (Modified SICT) | week 13, satisfaction/preference (n=59,64) | 2.9 score on a scale | Standard Deviation 1.54 |
| Deferasirox Film-coated Tablet (DFX-FCT) | Mean Domain Scores of the Modified Satisfaction With Iron Chelation Therapy (Modified SICT) | week 3, adherence (n=58,51) | 7.7 score on a scale | Standard Deviation 2.06 |
| Deferasirox Film-coated Tablet (DFX-FCT) | Mean Domain Scores of the Modified Satisfaction With Iron Chelation Therapy (Modified SICT) | week 24, concerns (n=63,60) | 13.7 score on a scale | Standard Deviation 1.84 |
| Deferasirox Film-coated Tablet (DFX-FCT) | Mean Domain Scores of the Modified Satisfaction With Iron Chelation Therapy (Modified SICT) | week 3, satisfaction/preference (n=58,51) | 2.6 score on a scale | Standard Deviation 1.05 |
| Deferasirox Film-coated Tablet (DFX-FCT) | Mean Domain Scores of the Modified Satisfaction With Iron Chelation Therapy (Modified SICT) | week 13, concerns (n=59,64) | 13.6 score on a scale | Standard Deviation 1.87 |
| Deferasirox Film-coated Tablet (DFX-FCT) | Mean Domain Scores of the Modified Satisfaction With Iron Chelation Therapy (Modified SICT) | week 3, concerns (n=58,51) | 14.0 score on a scale | Standard Deviation 1.49 |
Secondary
Number of Participants With Weekly Average Compliance of Medication Consumption
A compliance questionnaire assessed whether the medication was taken. Weekly average compliance was calculated when there were at least four non-missing daily responses.
Time frame: Weeks 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 23, 24
Population: The safety set, which included all participants who received at least one dose of study drug, was analyzed.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Deferasirox Dispersible Tablet (DFX-DT) | Number of Participants With Weekly Average Compliance of Medication Consumption | week 7 | 55 Participants |
| Deferasirox Dispersible Tablet (DFX-DT) | Number of Participants With Weekly Average Compliance of Medication Consumption | week 2 | 64 Participants |
| Deferasirox Dispersible Tablet (DFX-DT) | Number of Participants With Weekly Average Compliance of Medication Consumption | week 3 | 62 Participants |
| Deferasirox Dispersible Tablet (DFX-DT) | Number of Participants With Weekly Average Compliance of Medication Consumption | week 4 | 58 Participants |
| Deferasirox Dispersible Tablet (DFX-DT) | Number of Participants With Weekly Average Compliance of Medication Consumption | week 5 | 56 Participants |
| Deferasirox Dispersible Tablet (DFX-DT) | Number of Participants With Weekly Average Compliance of Medication Consumption | week 6 | 62 Participants |
| Deferasirox Dispersible Tablet (DFX-DT) | Number of Participants With Weekly Average Compliance of Medication Consumption | week 1 | 56 Participants |
| Deferasirox Dispersible Tablet (DFX-DT) | Number of Participants With Weekly Average Compliance of Medication Consumption | week 8 | 56 Participants |
| Deferasirox Dispersible Tablet (DFX-DT) | Number of Participants With Weekly Average Compliance of Medication Consumption | week 9 | 53 Participants |
| Deferasirox Dispersible Tablet (DFX-DT) | Number of Participants With Weekly Average Compliance of Medication Consumption | week 10 | 52 Participants |
| Deferasirox Dispersible Tablet (DFX-DT) | Number of Participants With Weekly Average Compliance of Medication Consumption | week 11 | 50 Participants |
| Deferasirox Dispersible Tablet (DFX-DT) | Number of Participants With Weekly Average Compliance of Medication Consumption | week 12 | 50 Participants |
| Deferasirox Dispersible Tablet (DFX-DT) | Number of Participants With Weekly Average Compliance of Medication Consumption | week 13 | 49 Participants |
| Deferasirox Dispersible Tablet (DFX-DT) | Number of Participants With Weekly Average Compliance of Medication Consumption | week 14 | 51 Participants |
| Deferasirox Dispersible Tablet (DFX-DT) | Number of Participants With Weekly Average Compliance of Medication Consumption | week 15 | 48 Participants |
| Deferasirox Dispersible Tablet (DFX-DT) | Number of Participants With Weekly Average Compliance of Medication Consumption | week 16 | 48 Participants |
| Deferasirox Dispersible Tablet (DFX-DT) | Number of Participants With Weekly Average Compliance of Medication Consumption | week 17 | 43 Participants |
| Deferasirox Dispersible Tablet (DFX-DT) | Number of Participants With Weekly Average Compliance of Medication Consumption | week 18 | 43 Participants |
| Deferasirox Dispersible Tablet (DFX-DT) | Number of Participants With Weekly Average Compliance of Medication Consumption | week 19 | 40 Participants |
| Deferasirox Dispersible Tablet (DFX-DT) | Number of Participants With Weekly Average Compliance of Medication Consumption | week 20 | 40 Participants |
| Deferasirox Dispersible Tablet (DFX-DT) | Number of Participants With Weekly Average Compliance of Medication Consumption | week 21 | 39 Participants |
| Deferasirox Dispersible Tablet (DFX-DT) | Number of Participants With Weekly Average Compliance of Medication Consumption | week 22 | 38 Participants |
| Deferasirox Dispersible Tablet (DFX-DT) | Number of Participants With Weekly Average Compliance of Medication Consumption | week 23 | 36 Participants |
| Deferasirox Dispersible Tablet (DFX-DT) | Number of Participants With Weekly Average Compliance of Medication Consumption | week 24 | 30 Participants |
| Deferasirox Film-coated Tablet (DFX-FCT) | Number of Participants With Weekly Average Compliance of Medication Consumption | week 23 | 33 Participants |
| Deferasirox Film-coated Tablet (DFX-FCT) | Number of Participants With Weekly Average Compliance of Medication Consumption | week 1 | 53 Participants |
| Deferasirox Film-coated Tablet (DFX-FCT) | Number of Participants With Weekly Average Compliance of Medication Consumption | week 13 | 47 Participants |
| Deferasirox Film-coated Tablet (DFX-FCT) | Number of Participants With Weekly Average Compliance of Medication Consumption | week 2 | 64 Participants |
| Deferasirox Film-coated Tablet (DFX-FCT) | Number of Participants With Weekly Average Compliance of Medication Consumption | week 19 | 37 Participants |
| Deferasirox Film-coated Tablet (DFX-FCT) | Number of Participants With Weekly Average Compliance of Medication Consumption | week 3 | 56 Participants |
| Deferasirox Film-coated Tablet (DFX-FCT) | Number of Participants With Weekly Average Compliance of Medication Consumption | week 14 | 42 Participants |
| Deferasirox Film-coated Tablet (DFX-FCT) | Number of Participants With Weekly Average Compliance of Medication Consumption | week 4 | 58 Participants |
| Deferasirox Film-coated Tablet (DFX-FCT) | Number of Participants With Weekly Average Compliance of Medication Consumption | week 22 | 34 Participants |
| Deferasirox Film-coated Tablet (DFX-FCT) | Number of Participants With Weekly Average Compliance of Medication Consumption | week 5 | 58 Participants |
| Deferasirox Film-coated Tablet (DFX-FCT) | Number of Participants With Weekly Average Compliance of Medication Consumption | week 15 | 42 Participants |
| Deferasirox Film-coated Tablet (DFX-FCT) | Number of Participants With Weekly Average Compliance of Medication Consumption | week 6 | 51 Participants |
| Deferasirox Film-coated Tablet (DFX-FCT) | Number of Participants With Weekly Average Compliance of Medication Consumption | week 20 | 36 Participants |
| Deferasirox Film-coated Tablet (DFX-FCT) | Number of Participants With Weekly Average Compliance of Medication Consumption | week 7 | 48 Participants |
| Deferasirox Film-coated Tablet (DFX-FCT) | Number of Participants With Weekly Average Compliance of Medication Consumption | week 16 | 40 Participants |
| Deferasirox Film-coated Tablet (DFX-FCT) | Number of Participants With Weekly Average Compliance of Medication Consumption | week 8 | 46 Participants |
| Deferasirox Film-coated Tablet (DFX-FCT) | Number of Participants With Weekly Average Compliance of Medication Consumption | week 24 | 24 Participants |
| Deferasirox Film-coated Tablet (DFX-FCT) | Number of Participants With Weekly Average Compliance of Medication Consumption | week 9 | 45 Participants |
| Deferasirox Film-coated Tablet (DFX-FCT) | Number of Participants With Weekly Average Compliance of Medication Consumption | week 17 | 39 Participants |
| Deferasirox Film-coated Tablet (DFX-FCT) | Number of Participants With Weekly Average Compliance of Medication Consumption | week 10 | 46 Participants |
| Deferasirox Film-coated Tablet (DFX-FCT) | Number of Participants With Weekly Average Compliance of Medication Consumption | week 21 | 36 Participants |
| Deferasirox Film-coated Tablet (DFX-FCT) | Number of Participants With Weekly Average Compliance of Medication Consumption | week 11 | 42 Participants |
| Deferasirox Film-coated Tablet (DFX-FCT) | Number of Participants With Weekly Average Compliance of Medication Consumption | week 18 | 38 Participants |
| Deferasirox Film-coated Tablet (DFX-FCT) | Number of Participants With Weekly Average Compliance of Medication Consumption | week 12 | 41 Participants |
Secondary
Observed Maximum Plasma Concentration Following Drug Administration (Cmax)
Blood samples were collected to assess Cmax.
Time frame: week 1, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose; week 3, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose
Population: The Pharmacokinetic subset A analysis set was considered for the analysis, but only participants with non-missing values were analyzed
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Deferasirox Dispersible Tablet (DFX-DT) | Observed Maximum Plasma Concentration Following Drug Administration (Cmax) | week 1 (n=14,15) | 74.6 umol/L | Standard Deviation 30.7 |
| Deferasirox Dispersible Tablet (DFX-DT) | Observed Maximum Plasma Concentration Following Drug Administration (Cmax) | week 3 (n=14,15) | 118 umol/L | Standard Deviation 82.3 |
| Deferasirox Film-coated Tablet (DFX-FCT) | Observed Maximum Plasma Concentration Following Drug Administration (Cmax) | week 1 (n=14,15) | 79.3 umol/L | Standard Deviation 23.5 |
| Deferasirox Film-coated Tablet (DFX-FCT) | Observed Maximum Plasma Concentration Following Drug Administration (Cmax) | week 3 (n=14,15) | 139 umol/L | Standard Deviation 57.2 |
Secondary
Palatability Questionnaire Score
The palatability questionnaire consisted of 4 items. The first item measured the taste and aftertaste of the medication and were scored a on a 5-point response scale. The second item offered an additional response option of no aftertaste. The last 2 items referred to whether the medication was taken, i.e. swallowed or vomited, and how the participant perceived the amount of medication to be taken. The palatability summary score was calculated using a scoring matrix from items 1, 3 and 4 scores and the score ranges from 0 - 11. Higher scores indicated the best palatability. A meaningful difference between two treatment arms was determined to be 1 point.
Time frame: weeks 2, 3, 13 and 24 (end of treatment or within 7 days of last dose)
Population: The safety set, which included all participants who received at least one dose of study drug, was considered for the analysis. However, only participants with values at the given week were included in the analysis for that week.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Deferasirox Dispersible Tablet (DFX-DT) | Palatability Questionnaire Score | week 2 (n=69,70) | 9.0 score on a scale | Standard Deviation 3.01 |
| Deferasirox Dispersible Tablet (DFX-DT) | Palatability Questionnaire Score | week 3 (n=57,51) | 8.8 score on a scale | Standard Deviation 3.01 |
| Deferasirox Dispersible Tablet (DFX-DT) | Palatability Questionnaire Score | week 13 (n=59,62) | 9.3 score on a scale | Standard Deviation 2.84 |
| Deferasirox Dispersible Tablet (DFX-DT) | Palatability Questionnaire Score | week 24 (n=63,60) | 8.8 score on a scale | Standard Deviation 3.1 |
| Deferasirox Film-coated Tablet (DFX-FCT) | Palatability Questionnaire Score | week 24 (n=63,60) | 10.9 score on a scale | Standard Deviation 0.34 |
| Deferasirox Film-coated Tablet (DFX-FCT) | Palatability Questionnaire Score | week 2 (n=69,70) | 10.8 score on a scale | Standard Deviation 0.5 |
| Deferasirox Film-coated Tablet (DFX-FCT) | Palatability Questionnaire Score | week 13 (n=59,62) | 10.8 score on a scale | Standard Deviation 1.16 |
| Deferasirox Film-coated Tablet (DFX-FCT) | Palatability Questionnaire Score | week 3 (n=57,51) | 10.8 score on a scale | Standard Deviation 0.45 |
Secondary
Time to Reach the Maximum Plasma Concentration After Drug Administration (Tmax)
Blood samples were collected to assess Tmax.
Time frame: week 1, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose; week 3, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose
Population: The Pharmacokinetic subset A analysis set was considered for the analysis, but only participants with non-missing values were analyzed
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Deferasirox Dispersible Tablet (DFX-DT) | Time to Reach the Maximum Plasma Concentration After Drug Administration (Tmax) | week 1 (n=14,15) | 3.57 hour |
| Deferasirox Dispersible Tablet (DFX-DT) | Time to Reach the Maximum Plasma Concentration After Drug Administration (Tmax) | week 3 (n=14,15) | 2.85 hour |
| Deferasirox Film-coated Tablet (DFX-FCT) | Time to Reach the Maximum Plasma Concentration After Drug Administration (Tmax) | week 1 (n=14,15) | 2.00 hour |
| Deferasirox Film-coated Tablet (DFX-FCT) | Time to Reach the Maximum Plasma Concentration After Drug Administration (Tmax) | week 3 (n=14,15) | 2.02 hour |
Secondary
Weekly Average of Daily Scores of the Gastrointestinal (GI) Symptom Diary
The GI symptom diary consisted of 6 items, five which were scored using a 0 - 10 rating scale with item appropriate anchors to rate the symptom, for example, Pain in your belly: 0 = no pain and 10 = worst pain. The GI diary summary score was created using the 10 point response scale for the 5 items. The GI symptom daily diary had a minimum score of 0 and a maximum score of 50. The weekly average score for the 7 days was calculated for each individual item and the GI summary score was created from these weekly averages. Higher scores indicated worse symptoms. A meaningful difference between two treatment arms was determined to be 0.3 point.
Time frame: weeks -1, 4, 8, 12, 16, 20, 24
Population: The safety set, which included all participants who received at least one dose of study drug, was considered for the analysis. However, only participants with values at the given week were included in the analysis for that week.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Deferasirox Dispersible Tablet (DFX-DT) | Weekly Average of Daily Scores of the Gastrointestinal (GI) Symptom Diary | week 8 (n=59,51) | 1.4 score on a scale | Standard Deviation 2.45 |
| Deferasirox Dispersible Tablet (DFX-DT) | Weekly Average of Daily Scores of the Gastrointestinal (GI) Symptom Diary | week 16 (n=48,41) | 1.9 score on a scale | Standard Deviation 3.75 |
| Deferasirox Dispersible Tablet (DFX-DT) | Weekly Average of Daily Scores of the Gastrointestinal (GI) Symptom Diary | week 4 (n=60,64) | 1.8 score on a scale | Standard Deviation 3.49 |
| Deferasirox Dispersible Tablet (DFX-DT) | Weekly Average of Daily Scores of the Gastrointestinal (GI) Symptom Diary | week 20 (n40,39) | 1.5 score on a scale | Standard Deviation 3.27 |
| Deferasirox Dispersible Tablet (DFX-DT) | Weekly Average of Daily Scores of the Gastrointestinal (GI) Symptom Diary | week 12 (n=51,45) | 1.7 score on a scale | Standard Deviation 3.16 |
| Deferasirox Dispersible Tablet (DFX-DT) | Weekly Average of Daily Scores of the Gastrointestinal (GI) Symptom Diary | week 24 (n32,26) | 1.5 score on a scale | Standard Deviation 3.29 |
| Deferasirox Dispersible Tablet (DFX-DT) | Weekly Average of Daily Scores of the Gastrointestinal (GI) Symptom Diary | week -1 (n=69,65) | 1.4 score on a scale | Standard Deviation 2.1 |
| Deferasirox Film-coated Tablet (DFX-FCT) | Weekly Average of Daily Scores of the Gastrointestinal (GI) Symptom Diary | week 24 (n32,26) | 1.2 score on a scale | Standard Deviation 1.89 |
| Deferasirox Film-coated Tablet (DFX-FCT) | Weekly Average of Daily Scores of the Gastrointestinal (GI) Symptom Diary | week -1 (n=69,65) | 1.9 score on a scale | Standard Deviation 3.69 |
| Deferasirox Film-coated Tablet (DFX-FCT) | Weekly Average of Daily Scores of the Gastrointestinal (GI) Symptom Diary | week 4 (n=60,64) | 1.1 score on a scale | Standard Deviation 2.15 |
| Deferasirox Film-coated Tablet (DFX-FCT) | Weekly Average of Daily Scores of the Gastrointestinal (GI) Symptom Diary | week 8 (n=59,51) | 1.1 score on a scale | Standard Deviation 2.16 |
| Deferasirox Film-coated Tablet (DFX-FCT) | Weekly Average of Daily Scores of the Gastrointestinal (GI) Symptom Diary | week 12 (n=51,45) | 1.0 score on a scale | Standard Deviation 1.78 |
| Deferasirox Film-coated Tablet (DFX-FCT) | Weekly Average of Daily Scores of the Gastrointestinal (GI) Symptom Diary | week 16 (n=48,41) | 0.9 score on a scale | Standard Deviation 1.92 |
| Deferasirox Film-coated Tablet (DFX-FCT) | Weekly Average of Daily Scores of the Gastrointestinal (GI) Symptom Diary | week 20 (n40,39) | 0.9 score on a scale | Standard Deviation 1.44 |
Secondary
Weekly Dose Violation Rate
The dose violation is defined as a dose either missed completely or not taken in accordance with the timing instruction (no later than 12:00 pm. The rate was calculated as \[number of dose violations/drug exposure (days)\] x 100.
Time frame: weeks 1, 4, 8, 12, 16, 20, 24
Population: The safety set, which included all participants who received at least one dose of study drug, was considered for the analysis. However, only participants with values at the given week were included in the analysis for that week.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Deferasirox Dispersible Tablet (DFX-DT) | Weekly Dose Violation Rate | week 8 (n=56,46) | 18.0 percent dose violation | Standard Deviation 35.38 |
| Deferasirox Dispersible Tablet (DFX-DT) | Weekly Dose Violation Rate | week 16 (n=48,40) | 13.5 percent dose violation | Standard Deviation 31.08 |
| Deferasirox Dispersible Tablet (DFX-DT) | Weekly Dose Violation Rate | week 4 (n=58,58) | 15.8 percent dose violation | Standard Deviation 32.51 |
| Deferasirox Dispersible Tablet (DFX-DT) | Weekly Dose Violation Rate | week 20 (n=40,36) | 22.6 percent dose violation | Standard Deviation 38.38 |
| Deferasirox Dispersible Tablet (DFX-DT) | Weekly Dose Violation Rate | week 12 (n=50,41) | 15.7 percent dose violation | Standard Deviation 34.22 |
| Deferasirox Dispersible Tablet (DFX-DT) | Weekly Dose Violation Rate | week 24 (n=30,24) | 17.1 percent dose violation | Standard Deviation 34.26 |
| Deferasirox Dispersible Tablet (DFX-DT) | Weekly Dose Violation Rate | week 1 (n=56,53) | 17.7 percent dose violation | Standard Deviation 31.04 |
| Deferasirox Film-coated Tablet (DFX-FCT) | Weekly Dose Violation Rate | week 24 (n=30,24) | 10.1 percent dose violation | Standard Deviation 25.47 |
| Deferasirox Film-coated Tablet (DFX-FCT) | Weekly Dose Violation Rate | week 1 (n=56,53) | 15.8 percent dose violation | Standard Deviation 29.42 |
| Deferasirox Film-coated Tablet (DFX-FCT) | Weekly Dose Violation Rate | week 4 (n=58,58) | 6.7 percent dose violation | Standard Deviation 15.45 |
| Deferasirox Film-coated Tablet (DFX-FCT) | Weekly Dose Violation Rate | week 8 (n=56,46) | 8.4 percent dose violation | Standard Deviation 22.17 |
| Deferasirox Film-coated Tablet (DFX-FCT) | Weekly Dose Violation Rate | week 12 (n=50,41) | 10.7 percent dose violation | Standard Deviation 22.63 |
| Deferasirox Film-coated Tablet (DFX-FCT) | Weekly Dose Violation Rate | week 16 (n=48,40) | 10.0 percent dose violation | Standard Deviation 24.5 |
| Deferasirox Film-coated Tablet (DFX-FCT) | Weekly Dose Violation Rate | week 20 (n=40,36) | 11.3 percent dose violation | Standard Deviation 26.67 |