| Primary | Overall Safety as Measured by Frequency of Adverse Events | The percentage of participants with adverse events, serious adverse events and deaths was assessed. | The safety set, which included all participants who received at least one dose of study drug, was analyzed. | Posted | | Number | | Percentage of participants | | 28 weeks | | | | ID | Title | Description |
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| OG000 | 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. | | OG001 | 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 |
| | | Title | Denominators | Categories |
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| Adverse events | | | | SAEs | | |
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| 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. | The safety set, which included all participants who received at least one dose of study drug, was analyzed. | Posted | | Number | | Percentage of participants | | baseline (BL), 30 weeks | | | | ID | Title | Description |
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| OG000 | 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. | | OG001 | 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 |
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| 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. | The safety set, which included all participants who received at least one dose of study drug, was analyzed. | Posted | | Number | | Percentage of participants | | 28 weeks | | | | ID | Title | Description |
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| OG000 | 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. | | OG001 | 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 |
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| 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. | 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. | Posted | | Mean | Standard Deviation | score on a scale | | weeks 2, 3, 13 and 24 (end of treatment or within 7 days of last dose) | | | | ID | Title | Description |
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| OG000 | 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. |
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| 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. | 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. | Posted | | Mean | Standard Deviation | score on a scale | | weeks 2, 3, 13 and 24 (end of treatment or within 7 days of last dose) | | | | ID | Title | Description |
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| OG000 | 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. | | OG001 | Deferasirox Film-coated Tablet (DFX-FCT) | |
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| 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. | 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. | Posted | | Mean | Standard Deviation | score on a scale | | weeks -1, 4, 8, 12, 16, 20, 24 | | | | ID | Title | Description |
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| OG000 | 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. | | OG001 | Deferasirox Film-coated Tablet (DFX-FCT) | |
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| 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. | The safety set, which included all participants who received at least one dose of study drug, was analyzed. | Posted | | Number | | Participants | | 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 | | | | ID | Title | Description |
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| OG000 | 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. | | OG001 | 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 |
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| 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. | 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. | Posted | | Mean | Standard Deviation | percent dose violation | | weeks 1, 4, 8, 12, 16, 20, 24 | | | | ID | Title | Description |
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| OG000 | 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. | | OG001 | 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 |
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| Secondary | Area Under the Plasma Concentration-time Curve From Time Zero to the Last Quantifiable Concentration (AUClast) | Blood samples were collected to assess AUClast. | The Pharmacokinetic subset A analysis set was considered for the analysis, but only participants with non-missing values were analyzed. | Posted | | Mean | Standard Deviation | umol/L*h | | 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 | | | | ID | Title | Description |
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| OG000 | 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. | | OG001 | 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 |
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| Secondary | Observed Maximum Plasma Concentration Following Drug Administration (Cmax) | Blood samples were collected to assess Cmax. | The Pharmacokinetic subset A analysis set was considered for the analysis, but only participants with non-missing values were analyzed | Posted | | Mean | Standard Deviation | umol/L | | 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 | | | | ID | Title | Description |
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| OG000 | 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. | | OG001 | 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 |
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| Secondary | Time to Reach the Maximum Plasma Concentration After Drug Administration (Tmax) | Blood samples were collected to assess Tmax. | The Pharmacokinetic subset A analysis set was considered for the analysis, but only participants with non-missing values were analyzed | Posted | | Median | Full Range | hour | | 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 | | | | ID | Title | Description |
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| OG000 | 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. | | OG001 | 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 |
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| 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. | The Pharmacokinetic analysis set for all participants was considered for this analysis, but only participants with non-missing values were included in the analysis. | Posted | | Mean | Standard Deviation | umol/L | | 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 | | | | ID | Title | Description |
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| OG000 | 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. | | OG001 | 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 |
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