Trial Outcomes & Findings for Open-label, Multicenter Study Assessing Preference for Deferasirox Film-coated Tablet Compared to Dispersible Tablet (NCT NCT02993224)

Last Updated: 2021-10-04

Results Overview

Number of participants preferring deferasirox FCT or DT as measured by preference questionnaire (item 2) at Week 48. The preference questionnaire was a 3-item questionnaire. At Week 48, the second item of the preference questionnaire asked the patients (or parents of young patients from 2 to 9 years old) which medicine did the patient "like best": "Tablet to dissolve in liquid" (=deferasirox DT), "Film coated tablet" (=deferasirox FCT), "Sprinkle powder on food" (=deferasirox FCT) and "I don't know" (=none of the above). The number of participants who selected each response option for item 2 was assessed. The analysis was performed for participants who answered the item 2 of the preference questionnaire.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

148 participants

Primary outcome timeframe

Week 48

Results posted on

2021-10-04

Participant Flow

Participants took part in 17 investigative sites in 7 countries

Participant milestones

Participant milestones
Measure	Deferasirox DT Followed by Deferasirox FCT Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT
Core Phase - Period 1 STARTED	148
Core Phase - Period 1 COMPLETED	140
Core Phase - Period 1 NOT COMPLETED	8
Core Phase- Period 2 STARTED	140
Core Phase- Period 2 COMPLETED	136
Core Phase- Period 2 NOT COMPLETED	4
Extension Phase STARTED	116
Extension Phase COMPLETED	80
Extension Phase NOT COMPLETED	36

Reasons for withdrawal

Reasons for withdrawal
Measure	Deferasirox DT Followed by Deferasirox FCT Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT
Core Phase - Period 1 Adverse Event	4
Core Phase - Period 1 Withdrawal by Subject	1
Core Phase - Period 1 Lost to Follow-up	3
Core Phase- Period 2 Adverse Event	1
Core Phase- Period 2 Unwillingness To Comply With Protocol Procedures And Prescribed Study Treatment	2
Core Phase- Period 2 Allogenic stem cell transplantation	1
Extension Phase Unwillingness To Comply With Protocol Procedures And Prescribed Study Treatment	1
Extension Phase Adverse Event	3
Extension Phase Withdrawal by Subject	3
Extension Phase Lost to Follow-up	2
Extension Phase Deferasirox FCT locally reimbursed	27

Baseline Characteristics

Open-label, Multicenter Study Assessing Preference for Deferasirox Film-coated Tablet Compared to Dispersible Tablet

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Deferasirox DT Followed by Deferasirox FCT n=148 Participants Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT
Age, Continuous	15.32 Years STANDARD_DEVIATION 13.824 • n=5 Participants
Sex: Female, Male Female	82 Participants n=5 Participants
Sex: Female, Male Male	66 Participants n=5 Participants
Race/Ethnicity, Customized Indian (Indian subcontinent)	5 Participants n=5 Participants
Race/Ethnicity, Customized Other	143 Participants n=5 Participants

PRIMARY outcome

Timeframe: Week 48

Population: Full Analysis Set (FAS): Participants to whom study treatment was assigned and who received at least one dose of each study treatment (DT and FCT). Only participants with available data for this outcome measure were included in this analysis.

Outcome measures

Outcome measures
Measure	Deferasirox DT Followed by Deferasirox FCT n=134 Participants Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT
Number of Participants Preferring Deferasirox FCT or DT at Week 48 Based on Preference Questionnaire (Item 2) Preference for deferasirox FCT	121 Participants
Number of Participants Preferring Deferasirox FCT or DT at Week 48 Based on Preference Questionnaire (Item 2) Preference for deferasirox DT	10 Participants
Number of Participants Preferring Deferasirox FCT or DT at Week 48 Based on Preference Questionnaire (Item 2) Preference for none of the above	3 Participants

SECONDARY outcome

Timeframe: Week 28

Population: Participants in the FAS \[to whom study treatment was assigned and who received at least one dose of each study treatment (DT and FCT)\] and who received iron chelation therapy prior to enrolling in the study. Only participants with available data for this outcome measure were included in this analysis.

Number of participants preferring deferasirox FCT, deferasirox DT or previous iron chelation therapy as measured by preference questionnaire (item 2) at Week 28. The preference questionnaire was a 3-item questionnaire. At Week 28, the second item of this questionnaire asked the patients (or parents of young patients from 2 to 9 years old) which medicine did the patient "like best": "Tablet to dissolve in liquid" (=deferasirox DT), "Film coated tablet (taken once a day)" (=deferasirox FCT), "Sprinkle powder on food" (=deferasirox FCT), "Tablet (taken 3 times a day)" (=previous iron chelation therapy), "Injection" (=previous iron chelation therapy) and "I don't know" (=none of the above). The number of participants who selected each response option for item 2 was assessed. This analysis was performed only for patients who had received iron chelation therapy prior to enrolling in the study and who answered the item 2 of the preference questionnaire.

Outcome measures

Outcome measures
Measure	Deferasirox DT Followed by Deferasirox FCT n=69 Participants Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT
Number of Participants Preferring Deferasirox FCT, Deferasirox DT or Previous Iron Chelation Therapy at Week 28 Based on Preference Questionnaire (Item 2) Preference for previous iron chelation therapy	3 Participants
Number of Participants Preferring Deferasirox FCT, Deferasirox DT or Previous Iron Chelation Therapy at Week 28 Based on Preference Questionnaire (Item 2) Preference for none of the above	0 Participants
Number of Participants Preferring Deferasirox FCT, Deferasirox DT or Previous Iron Chelation Therapy at Week 28 Based on Preference Questionnaire (Item 2) Preference for deferasirox DT	6 Participants
Number of Participants Preferring Deferasirox FCT, Deferasirox DT or Previous Iron Chelation Therapy at Week 28 Based on Preference Questionnaire (Item 2) Preference for deferasirox FCT	60 Participants

SECONDARY outcome

Timeframe: Week 4 and Week 24

Population: Participants in the FAS \[to whom study treatment was assigned and who received at least one dose of each study treatment (DT and FCT)\] and who received iron chelation therapy prior to enrolling in the study. Number analyzed signified number of participants with available data for this outcome measure at specified timepoints.

Number of participants preferring deferasirox DT or previous iron chelation therapy as measured by preference questionnaire (item 2) at Week 4 and 24. The preference questionnaire was a 3-item questionnaire. At Week 4 and 24, the second item of the preference questionnaire asked the patients (or parents of young patients from 2 to 9 years old) which medicine did the patient "like best": "Tablet to dissolve in liquid" (=deferasirox DT), "Tablet (taken 3 times a day)" (=previous iron chelation therapy), "Injection" (=previous iron chelation therapy) and "I don't know" (=none of the above). The number of participants who selected each response option for item 2 was assessed. This analysis was performed only for patients who had received iron chelation therapy prior to enrolling in the study and who answered item 2 of the preference questionnaire.

Outcome measures

Outcome measures
Measure	Deferasirox DT Followed by Deferasirox FCT n=70 Participants Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT
Number of Participants Preferring Deferasirox DT or Previous Iron Chelation Therapy at Week 4 and Week 24 Based on Preference Questionnaire (Item 2) Week 4 · Preference for previous iron chelation therapy	11 Participants
Number of Participants Preferring Deferasirox DT or Previous Iron Chelation Therapy at Week 4 and Week 24 Based on Preference Questionnaire (Item 2) Week 4 · Preference for deferasirox DT	57 Participants
Number of Participants Preferring Deferasirox DT or Previous Iron Chelation Therapy at Week 4 and Week 24 Based on Preference Questionnaire (Item 2) Week 4 · Preference for none of the above	2 Participants
Number of Participants Preferring Deferasirox DT or Previous Iron Chelation Therapy at Week 4 and Week 24 Based on Preference Questionnaire (Item 2) Week 24 · Preference for deferasirox DT	52 Participants
Number of Participants Preferring Deferasirox DT or Previous Iron Chelation Therapy at Week 4 and Week 24 Based on Preference Questionnaire (Item 2) Week 24 · Preference for previous iron chelation therapy	11 Participants
Number of Participants Preferring Deferasirox DT or Previous Iron Chelation Therapy at Week 4 and Week 24 Based on Preference Questionnaire (Item 2) Week 24 · Preference for none of the above	6 Participants

SECONDARY outcome

Timeframe: Week 28 and Week 48

Population: FAS: Participants to whom study treatment was assigned and who received at least one dose of each study treatment (DT and FCT).

The preference questionnaire was a 3 item questionnaire. The first item asked the patients (or parents of young patients from 2 to 9 years old) which medicine they were taking. The second item asked which of the medicines did the patient "Like best". Finally, the third item asked the patient why he/she preferred the medicine they chose in the second item. The number of participants who selected each response option for item 3 was assessed. Participants could select multiple reasons for treatment preference at each timepoint.

Outcome measures

Outcome measures
Measure	Deferasirox DT Followed by Deferasirox FCT n=139 Participants Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT
Number of Participants Selecting Each Reason for Treatment Preference as Assessed by the Preference Questionnaire at Week 28 and Week 48 Easier to remember to take the medicine (Week 28)	57 Participants
Number of Participants Selecting Each Reason for Treatment Preference as Assessed by the Preference Questionnaire at Week 28 and Week 48 No/ less pain on the injection site (Week 28)	31 Participants
Number of Participants Selecting Each Reason for Treatment Preference as Assessed by the Preference Questionnaire at Week 28 and Week 48 No/ less side effects (Week 28)	50 Participants
Number of Participants Selecting Each Reason for Treatment Preference as Assessed by the Preference Questionnaire at Week 28 and Week 48 Number of times you have to take the medicine (Week 28)	42 Participants
Number of Participants Selecting Each Reason for Treatment Preference as Assessed by the Preference Questionnaire at Week 28 and Week 48 Taste (Week 28)	30 Participants
Number of Participants Selecting Each Reason for Treatment Preference as Assessed by the Preference Questionnaire at Week 28 and Week 48 Aftertaste (Week 48)	17 Participants
Number of Participants Selecting Each Reason for Treatment Preference as Assessed by the Preference Questionnaire at Week 28 and Week 48 Can correctly prepare the medicine (Week 48)	48 Participants
Number of Participants Selecting Each Reason for Treatment Preference as Assessed by the Preference Questionnaire at Week 28 and Week 48 Convenience (it's not a problem to take your medicine) (Week 48)	103 Participants
Number of Participants Selecting Each Reason for Treatment Preference as Assessed by the Preference Questionnaire at Week 28 and Week 48 Easier to remember to take the medicine (Week 48)	61 Participants
Number of Participants Selecting Each Reason for Treatment Preference as Assessed by the Preference Questionnaire at Week 28 and Week 48 Gain my personal time with family and friends (Week 48)	36 Participants
Number of Participants Selecting Each Reason for Treatment Preference as Assessed by the Preference Questionnaire at Week 28 and Week 48 No/ less pain on the injection site (Week 48)	37 Participants
Number of Participants Selecting Each Reason for Treatment Preference as Assessed by the Preference Questionnaire at Week 28 and Week 48 No/ less side effects (Week 48)	48 Participants
Number of Participants Selecting Each Reason for Treatment Preference as Assessed by the Preference Questionnaire at Week 28 and Week 48 Number of pills (Week 48)	41 Participants
Number of Participants Selecting Each Reason for Treatment Preference as Assessed by the Preference Questionnaire at Week 28 and Week 48 Taste (Week 48)	51 Participants
Number of Participants Selecting Each Reason for Treatment Preference as Assessed by the Preference Questionnaire at Week 28 and Week 48 Aftertaste (Week 28)	9 Participants
Number of Participants Selecting Each Reason for Treatment Preference as Assessed by the Preference Questionnaire at Week 28 and Week 48 Can correctly prepare the medicine (Week 28)	35 Participants
Number of Participants Selecting Each Reason for Treatment Preference as Assessed by the Preference Questionnaire at Week 28 and Week 48 Convenience (it's not a problem to take your medicine) (Week 28)	109 Participants
Number of Participants Selecting Each Reason for Treatment Preference as Assessed by the Preference Questionnaire at Week 28 and Week 48 Gain my personal time with family and friends (Week 28)	35 Participants
Number of Participants Selecting Each Reason for Treatment Preference as Assessed by the Preference Questionnaire at Week 28 and Week 48 Number of pills (Week 28)	46 Participants
Number of Participants Selecting Each Reason for Treatment Preference as Assessed by the Preference Questionnaire at Week 28 and Week 48 Other (Week 28)	5 Participants
Number of Participants Selecting Each Reason for Treatment Preference as Assessed by the Preference Questionnaire at Week 28 and Week 48 Number of times you have to take the medicine (Week 48)	47 Participants
Number of Participants Selecting Each Reason for Treatment Preference as Assessed by the Preference Questionnaire at Week 28 and Week 48 Other (Week 48)	0 Participants

SECONDARY outcome

Timeframe: Deferasirox DT: From Baseline up to Week 24. Deferasirox FCT: From Week 25 up to Week 48

Population: FAS: Participants to whom study treatment was assigned and who received at least one dose of each study treatment (DT and FCT). Number analyzed signified number of participants with available data for this outcome measure for each treatment period

The percentage of consumed tablet counts (compliance) was calculated for each treatment period in the core phase: deferasirox DT (period 1) and deferasirox FCT (period 2). Compliance was defined as the total tablet count consumed divided by total tablet count prescribed and multiplied by 100. Total tablet count consumed was calculated as total number of tablets dispensed minus total number of tablets lost/wasted or returned. Total tablet count prescribed was calculated as the number of tablets that the patient should have taken during this period. If a patient did not return the study drug, the compliance was not calculated.

Outcome measures

Outcome measures
Measure	Deferasirox DT Followed by Deferasirox FCT n=139 Participants Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT
Percentage of Consumed Tablet Counts During Deferasirox DT and Deferasirox FCT Treatment Periods Deferasirox DT (Baseline up to Week 24)	98.68 Percentage of tablet counts Standard Deviation 22.373
Percentage of Consumed Tablet Counts During Deferasirox DT and Deferasirox FCT Treatment Periods Deferasirox FCT (From Week 25 up to Week 48)	95.07 Percentage of tablet counts Standard Deviation 15.368

SECONDARY outcome

Timeframe: Week 4, 24, 28 and 48

The palatability questionnaire consisted of 4 items, three items measuring taste and one item measuring aftertaste. The aftertaste item scored on a 5-point response scale with the response option: Very good = 1, Good = 2, Neither good nor bad = 3, Bad = 4, Very bad = 5. This item offered an additional response option of "no aftertaste". The aftertaste score was calculated among participants who had an aftertaste. Higher aftertaste scores indicated a worse aftertaste. For participants less than (\<) 10 years old, an observer palatability questionnaire was administered. Items and scoring algorithm remained the same as for participants greater than or equal to (≥) 10 years old. Change in aftertaste score over time was assessed

Outcome measures

Outcome measures
Measure	Deferasirox DT Followed by Deferasirox FCT n=139 Participants Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT
Change Over Time in Aftertaste Score of Palatability Questionnaire From Week 24 to Week 48 in participants treated with deferasirox FCT at Week 48	-0.5 Score on a Scale Standard Deviation 1.08
Change Over Time in Aftertaste Score of Palatability Questionnaire From Week 4 to Week 24 in participants treated with deferasirox DT at Week 24	-0.1 Score on a Scale Standard Deviation 0.80
Change Over Time in Aftertaste Score of Palatability Questionnaire From Week 4 to Week 24 in participants treated with deferasirox FCT at Week 24	-0.5 Score on a Scale Standard Deviation 1.22
Change Over Time in Aftertaste Score of Palatability Questionnaire From Week 24 to Week 28 in participants treated with deferasirox FCT at Week 28	-0.5 Score on a Scale Standard Deviation 1.16

SECONDARY outcome

Timeframe: Week 4, 24, 28 and 48

The palatability questionnaire consisted of 4 items, three items measuring taste and one item measuring aftertaste. Among the taste items, first one measured taste on a 5-point response scale. The other two items measured what happened after taking the medicine and how the perceived amount of liquid taken with the medicine was. Responses to these 3 items were combined and converted into a single palatability score using a scoring matrix: each combination of responses on each of 3 items corresponded to a predefined palatability score. E.g. if a participant responded "bad" to item 1, "vomited \<30min" to item 2 and "not enough" to item 3, then the palatability score assigned was 0. This score ranged from 0 to 11; higher scores indicated better palatability. For participants \<10 years old, an observer palatability questionnaire was administered. Items and scoring algorithm were the same as for participants ≥10 years old. Change in palatability score over time was assessed

Outcome measures

Outcome measures
Measure	Deferasirox DT Followed by Deferasirox FCT n=139 Participants Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT
Change Over Time in Palatability Score of Palatability Questionnaire From Week 4 to Week 24- participants treated with deferasirox DT at Week 24	-0.1 Score on a Scale Standard Deviation 2.46
Change Over Time in Palatability Score of Palatability Questionnaire From Week 4 to Week 24 in participants treated with deferasirox FCT at Week 24	0.0 Score on a Scale Standard Deviation 0.00
Change Over Time in Palatability Score of Palatability Questionnaire From Week 24 to Week 28 in participants treated with deferasirox FCT at Week 28	1.1 Score on a Scale Standard Deviation 2.77
Change Over Time in Palatability Score of Palatability Questionnaire From Week 24 to Week 48 in participants treated with deferasirox FCT at Week 48	1.3 Score on a Scale Standard Deviation 2.54

SECONDARY outcome

Timeframe: Baseline (week 2 or, if missing, week 3), week 24, 28 and 48

The mSICT patient reported outcome (PRO) consisted of 15 items that represented 3 domains: Adherence, Preference, and Concerns. The adherence domain score consisted of 6 adherence items, measured using a 5-point response scale. The adherence score was calculated by summing these 6 items, with scores ranging from 6 to 30. Higher scores indicated worse adherence. For participants \<10 years old, an observer version (ObsRO) was administered. The adherence score remained the same as for participants ≥10 years old.

Outcome measures

Outcome measures
Measure	Deferasirox DT Followed by Deferasirox FCT n=139 Participants Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT
Change From Baseline in Adherence Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire Week 24 - participants < 10 years treated with deferasirox FCT (ObsRO)	-0.7 Score on a scale Standard Deviation 4.16
Change From Baseline in Adherence Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire Week 48 - participants < 10 years treated with deferasirox FCT (ObsRO)	1.1 Score on a scale Standard Deviation 3.79
Change From Baseline in Adherence Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire Week 24- participants ≥ 10 years treated with deferasirox DT (PRO)	-0.3 Score on a scale Standard Deviation 3.37
Change From Baseline in Adherence Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire Week 24 - participants ≥ 10 years treated with deferasirox FCT (PRO)	-1.0 Score on a scale Standard Deviation 2.94
Change From Baseline in Adherence Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire Week 28 - participants ≥ 10 years treated with deferasirox FCT (PRO)	0.5 Score on a scale Standard Deviation 3.59
Change From Baseline in Adherence Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire Week 48 - participants ≥ 10 years treated with deferasirox FCT (PRO)	0.5 Score on a scale Standard Deviation 3.29
Change From Baseline in Adherence Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire Week 24 - participants < 10 years treated with deferasirox DT (ObsRO)	-0.2 Score on a scale Standard Deviation 3.35
Change From Baseline in Adherence Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire Week 28 - participants < 10 years treated with deferasirox FCT (ObsRO)	0.8 Score on a scale Standard Deviation 3.68

SECONDARY outcome

Timeframe: Baseline (week 2 or, if missing, week 3), week 24, 28 and 48

The mSICT patient reported outcome (PRO) consisted of 15 items that represented 3 domains: Adherence, Preference, and Concerns. The preference/satisfaction domain score consisted of 2 preference/satisfaction items, measured using a 5-point response scale. The preference score was calculated by summing these 2 items, with scores ranging from 2 to 10. Higher scores indicated worse satisfaction. For participants \< 10 years old, an observer version (ObsRO) was administered. Preference score remained the same as for participants ≥ 10 years old.

Outcome measures

Outcome measures
Measure	Deferasirox DT Followed by Deferasirox FCT n=139 Participants Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT
Change From Baseline in Preference Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire Week 24 - participants < 10 years treated with deferasirox FCT (ObsRO)	0.0 Score on a scale Standard Deviation 1.73
Change From Baseline in Preference Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire Week 24 - participants ≥ 10 treated with deferasirox DT (PRO)	0.5 Score on a scale Standard Deviation 1.21
Change From Baseline in Preference Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire Week 24 - participants ≥ 10 treated with deferasirox FCT (PRO)	0.5 Score on a scale Standard Deviation 3.70
Change From Baseline in Preference Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire Week 28 - participants ≥ 10 treated with deferasirox FCT (PRO)	-1.1 Score on a scale Standard Deviation 1.83
Change From Baseline in Preference Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire Week 48 - participants ≥ 10 treated with deferasirox FCT (PRO)	-0.9 Score on a scale Standard Deviation 2.02
Change From Baseline in Preference Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire Week 24 - participants < 10 years treated with deferasirox DT (ObsRO)	0.0 Score on a scale Standard Deviation 1.49
Change From Baseline in Preference Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire Week 28 - participants < 10 years treated with deferasirox FCT (ObsRO)	-0.8 Score on a scale Standard Deviation 1.74
Change From Baseline in Preference Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire Week 48 - participants < 10 years treated with deferasirox FCT (ObsRO)	-0.9 Score on a scale Standard Deviation 1.75

SECONDARY outcome

Timeframe: Baseline (week 2 or, if missing, week 3), week 24, 28 and 48

The mSICT patient reported outcome (PRO) consisted of 15 items that represented 3 domains: Adherence, Preference, and Concerns. The concerns domain score consisted of 3 items to address any concerns or worries with the medication. All 3 items were measured on a 5-point response scale. The concerns score was calculated by summing the 3 items, with scores ranging from 3 to 15. Higher scores indicated fewer concerns. For participants \< 10 years old, an observer version (ObsRO) was administered. Concerns score remained the same as for participants ≥ 10 years old.

Outcome measures

Outcome measures
Measure	Deferasirox DT Followed by Deferasirox FCT n=139 Participants Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT
Change From Baseline in Concerns Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire Week 24 - participants < 10 years treated with deferasirox DT (ObsRO)	-0.4 Score on a scale Standard Deviation 1.33
Change From Baseline in Concerns Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire Week 24 - participants ≥ 10 treated with deferasirox DT (PRO)	-0.1 Score on a scale Standard Deviation 1.99
Change From Baseline in Concerns Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire Week 24 - participants ≥ 10 treated with deferasirox FCT (PRO)	-1.0 Score on a scale Standard Deviation 1.15
Change From Baseline in Concerns Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire Week 28 - participants ≥ 10 treated with deferasirox FCT (PRO)	0.3 Score on a scale Standard Deviation 1.84
Change From Baseline in Concerns Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire Week 48 - participants ≥ 10 treated with deferasirox FCT (PRO)	0.5 Score on a scale Standard Deviation 1.80
Change From Baseline in Concerns Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire Week 24 - participants < 10 years treated with deferasirox FCT (ObsRO)	-0.7 Score on a scale Standard Deviation 1.15
Change From Baseline in Concerns Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire Week 28 - participants < 10 years treated with deferasirox FCT (ObsRO)	0.1 Score on a scale Standard Deviation 2.05
Change From Baseline in Concerns Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire Week 48 - participants < 10 years treated with deferasirox FCT (ObsRO)	0.1 Score on a scale Standard Deviation 1.58

SECONDARY outcome

Timeframe: Baseline (week -1 or, if missing, week -2), week 24, 28 and 48

The GI symptom score was calculated from responses to 5 questions of the GI questionnaire, each with a possible score of 1 to 5, for an overall possible score range of 5 to 25, where a lower score represents a less severe GI symptom and a higher score represents a more severe GI symptom. An observer GI symptom questionnaire was administered to those patients who were \< 10 years old. The questionnaire was completed by the parents of the participants. All items and the scoring algorithm remained the same as for participants ≥ 10 years old.

Outcome measures

Outcome measures
Measure	Deferasirox DT Followed by Deferasirox FCT n=139 Participants Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT
Change From Baseline in Gastrointestinal (GI) Symptom Score Based on GI Questionnaire Week 24 - participants ≥ 10 years treated with deferasirox DT	1.4 Score on a scale Standard Deviation 5.53
Change From Baseline in Gastrointestinal (GI) Symptom Score Based on GI Questionnaire Week 24 - participants ≥ 10 years treated with deferasirox FCT	5.8 Score on a scale Standard Deviation 12.82
Change From Baseline in Gastrointestinal (GI) Symptom Score Based on GI Questionnaire Week 28 - participants ≥ 10 years treated with deferasirox FCT	-1.9 Score on a scale Standard Deviation 4.33
Change From Baseline in Gastrointestinal (GI) Symptom Score Based on GI Questionnaire Week 48 -participants ≥ 10 years treated with deferasirox FCT	-2.2 Score on a scale Standard Deviation 4.74
Change From Baseline in Gastrointestinal (GI) Symptom Score Based on GI Questionnaire Week 24 - participants < 10 years treated with deferasirox DT	0.2 Score on a scale Standard Deviation 2.78
Change From Baseline in Gastrointestinal (GI) Symptom Score Based on GI Questionnaire Week 24 - participants < 10 years treated with deferasirox FCT	-2.0 Score on a scale Standard Deviation 2.65
Change From Baseline in Gastrointestinal (GI) Symptom Score Based on GI Questionnaire Week 28 - participants < 10 years treated with deferasirox FCT	-0.5 Score on a scale Standard Deviation 2.49
Change From Baseline in Gastrointestinal (GI) Symptom Score Based on GI Questionnaire Week 48 - participants < 10 years treated with deferasirox FCT	-0.6 Score on a scale Standard Deviation 2.33

SECONDARY outcome

Timeframe: From Baseline (Day 1) up to 96 weeks

Absolute change from baseline over time in serum ferritin levels

Outcome measures

Outcome measures
Measure	Deferasirox DT Followed by Deferasirox FCT n=139 Participants Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT
Change From Baseline in Serum Ferritin Levels Week 60 - participants treated with deferasirox FCT	980.644 microgram/liter (ug/L) Standard Deviation 994.4445
Change From Baseline in Serum Ferritin Levels Week 64 - participants treated with deferasirox FCT	863.579 microgram/liter (ug/L) Standard Deviation 854.8592
Change From Baseline in Serum Ferritin Levels Week 68 - participants treated with deferasirox FCT	921.725 microgram/liter (ug/L) Standard Deviation 860.8738
Change From Baseline in Serum Ferritin Levels Week 2 - participants treated with deferasirox DT	363.654 microgram/liter (ug/L) Standard Deviation 323.3828
Change From Baseline in Serum Ferritin Levels Week 4 - participants treated with deferasirox DT	393.126 microgram/liter (ug/L) Standard Deviation 457.8032
Change From Baseline in Serum Ferritin Levels Week 8 - participants treated with deferasirox DT	462.560 microgram/liter (ug/L) Standard Deviation 427.7138
Change From Baseline in Serum Ferritin Levels Week 12 - participants treated with deferasirox DT	510.455 microgram/liter (ug/L) Standard Deviation 548.6222
Change From Baseline in Serum Ferritin Levels Week 16 - participants treated with deferasirox DT	519.038 microgram/liter (ug/L) Standard Deviation 508.7432
Change From Baseline in Serum Ferritin Levels Week 20 - participants treated with deferasirox DT	540.618 microgram/liter (ug/L) Standard Deviation 548.2318
Change From Baseline in Serum Ferritin Levels Week 24 - participants treated with deferasirox DT	644.849 microgram/liter (ug/L) Standard Deviation 629.6687
Change From Baseline in Serum Ferritin Levels Week 28 - participants treated with deferasirox FCT	679.610 microgram/liter (ug/L) Standard Deviation 701.6219
Change From Baseline in Serum Ferritin Levels Week 32 - participants treated with deferasirox FCT	623.600 microgram/liter (ug/L) Standard Deviation 629.5417
Change From Baseline in Serum Ferritin Levels Week 36 - participants treated with deferasirox FCT	679.011 microgram/liter (ug/L) Standard Deviation 674.6716
Change From Baseline in Serum Ferritin Levels Week 40 - participants treated with deferasirox FCT	739.016 microgram/liter (ug/L) Standard Deviation 659.0840
Change From Baseline in Serum Ferritin Levels Week 44 - participants treated with deferasirox FCT	748.063 microgram/liter (ug/L) Standard Deviation 647.3000
Change From Baseline in Serum Ferritin Levels Week 48 - participants treated with deferasirox FCT	833.700 microgram/liter (ug/L) Standard Deviation 788.0650
Change From Baseline in Serum Ferritin Levels Week 52 - participants treated with deferasirox FCT	760.229 microgram/liter (ug/L) Standard Deviation 798.0836
Change From Baseline in Serum Ferritin Levels Week 56 - participants treated with deferasirox FCT	903.622 microgram/liter (ug/L) Standard Deviation 832.9872
Change From Baseline in Serum Ferritin Levels Week 72 - participants treated with deferasirox FCT	971.112 microgram/liter (ug/L) Standard Deviation 872.7306
Change From Baseline in Serum Ferritin Levels Week 76 - participants treated with deferasirox FCT	888.917 microgram/liter (ug/L) Standard Deviation 869.0805
Change From Baseline in Serum Ferritin Levels Week 80 - participants treated with deferasirox FCT	1005.758 microgram/liter (ug/L) Standard Deviation 868.4338
Change From Baseline in Serum Ferritin Levels Week 84 - participants treated with deferasirox FCT	1013.683 microgram/liter (ug/L) Standard Deviation 924.5828
Change From Baseline in Serum Ferritin Levels Week 88 - participants treated with deferasirox FCT	1117.849 microgram/liter (ug/L) Standard Deviation 985.0767
Change From Baseline in Serum Ferritin Levels Week 92 - participants treated with deferasirox FCT	1222.429 microgram/liter (ug/L) Standard Deviation 1027.2565
Change From Baseline in Serum Ferritin Levels Week 96 - participants treated with deferasirox FCT	1137.347 microgram/liter (ug/L) Standard Deviation 972.1286

Adverse Events

Deferasirox DT (Core Phase)

Serious events: 13 serious events

Other events: 82 other events

Deaths: 0 deaths

Deferasirox FCT (Core Phase)

Serious events: 6 serious events

Other events: 59 other events

Deaths: 0 deaths

Deferasirox FCT (Extension Phase)

Serious events: 16 serious events

Other events: 49 other events

Deaths: 0 deaths

Serious adverse events

Other adverse events

Other adverse events
Measure	Deferasirox DT (Core Phase) n=148 participants at risk Participants who were treated with deferasirox DT once daily in the core phase	Deferasirox FCT (Core Phase) n=140 participants at risk Participants who were treated with deferasirox FCT once daily in the core phase	Deferasirox FCT (Extension Phase) n=116 participants at risk Participants who were treated with deferasirox FCT once daily in the extension phase
Gastrointestinal disorders Diarrhoea	10.8% 16/148 • Adverse events (AEs) and serious AEs (including all-cause mortality data table) were collected from day of first dose to 30 days after last dose of study medication, assessed up to approximately 30 weeks for deferasirox DT treatment (core phase), 39 weeks for deferasirox FCT treatment (core phase) and 52 weeks for deferasirox FCT treatment (extension phase). Any sign or symptom that occurred during the study treatment plus the 30 days post treatment. Safety analyses were performed in all participants who received at least one dose of either study treatment (DT or FCT), and at least one safety assessment on or after Day 1. If, for a participant, AE start date was in deferasirox DT period and end date was in deferasirox FCT period, the participant was counted in both periods.	3.6% 5/140 • Adverse events (AEs) and serious AEs (including all-cause mortality data table) were collected from day of first dose to 30 days after last dose of study medication, assessed up to approximately 30 weeks for deferasirox DT treatment (core phase), 39 weeks for deferasirox FCT treatment (core phase) and 52 weeks for deferasirox FCT treatment (extension phase). Any sign or symptom that occurred during the study treatment plus the 30 days post treatment. Safety analyses were performed in all participants who received at least one dose of either study treatment (DT or FCT), and at least one safety assessment on or after Day 1. If, for a participant, AE start date was in deferasirox DT period and end date was in deferasirox FCT period, the participant was counted in both periods.	1.7% 2/116 • Adverse events (AEs) and serious AEs (including all-cause mortality data table) were collected from day of first dose to 30 days after last dose of study medication, assessed up to approximately 30 weeks for deferasirox DT treatment (core phase), 39 weeks for deferasirox FCT treatment (core phase) and 52 weeks for deferasirox FCT treatment (extension phase). Any sign or symptom that occurred during the study treatment plus the 30 days post treatment. Safety analyses were performed in all participants who received at least one dose of either study treatment (DT or FCT), and at least one safety assessment on or after Day 1. If, for a participant, AE start date was in deferasirox DT period and end date was in deferasirox FCT period, the participant was counted in both periods.
General disorders Pyrexia	4.7% 7/148 • Adverse events (AEs) and serious AEs (including all-cause mortality data table) were collected from day of first dose to 30 days after last dose of study medication, assessed up to approximately 30 weeks for deferasirox DT treatment (core phase), 39 weeks for deferasirox FCT treatment (core phase) and 52 weeks for deferasirox FCT treatment (extension phase). Any sign or symptom that occurred during the study treatment plus the 30 days post treatment. Safety analyses were performed in all participants who received at least one dose of either study treatment (DT or FCT), and at least one safety assessment on or after Day 1. If, for a participant, AE start date was in deferasirox DT period and end date was in deferasirox FCT period, the participant was counted in both periods.	5.7% 8/140 • Adverse events (AEs) and serious AEs (including all-cause mortality data table) were collected from day of first dose to 30 days after last dose of study medication, assessed up to approximately 30 weeks for deferasirox DT treatment (core phase), 39 weeks for deferasirox FCT treatment (core phase) and 52 weeks for deferasirox FCT treatment (extension phase). Any sign or symptom that occurred during the study treatment plus the 30 days post treatment. Safety analyses were performed in all participants who received at least one dose of either study treatment (DT or FCT), and at least one safety assessment on or after Day 1. If, for a participant, AE start date was in deferasirox DT period and end date was in deferasirox FCT period, the participant was counted in both periods.	4.3% 5/116 • Adverse events (AEs) and serious AEs (including all-cause mortality data table) were collected from day of first dose to 30 days after last dose of study medication, assessed up to approximately 30 weeks for deferasirox DT treatment (core phase), 39 weeks for deferasirox FCT treatment (core phase) and 52 weeks for deferasirox FCT treatment (extension phase). Any sign or symptom that occurred during the study treatment plus the 30 days post treatment. Safety analyses were performed in all participants who received at least one dose of either study treatment (DT or FCT), and at least one safety assessment on or after Day 1. If, for a participant, AE start date was in deferasirox DT period and end date was in deferasirox FCT period, the participant was counted in both periods.
Hepatobiliary disorders Hepatomegaly	5.4% 8/148 • Adverse events (AEs) and serious AEs (including all-cause mortality data table) were collected from day of first dose to 30 days after last dose of study medication, assessed up to approximately 30 weeks for deferasirox DT treatment (core phase), 39 weeks for deferasirox FCT treatment (core phase) and 52 weeks for deferasirox FCT treatment (extension phase). Any sign or symptom that occurred during the study treatment plus the 30 days post treatment. Safety analyses were performed in all participants who received at least one dose of either study treatment (DT or FCT), and at least one safety assessment on or after Day 1. If, for a participant, AE start date was in deferasirox DT period and end date was in deferasirox FCT period, the participant was counted in both periods.	0.00% 0/140 • Adverse events (AEs) and serious AEs (including all-cause mortality data table) were collected from day of first dose to 30 days after last dose of study medication, assessed up to approximately 30 weeks for deferasirox DT treatment (core phase), 39 weeks for deferasirox FCT treatment (core phase) and 52 weeks for deferasirox FCT treatment (extension phase). Any sign or symptom that occurred during the study treatment plus the 30 days post treatment. Safety analyses were performed in all participants who received at least one dose of either study treatment (DT or FCT), and at least one safety assessment on or after Day 1. If, for a participant, AE start date was in deferasirox DT period and end date was in deferasirox FCT period, the participant was counted in both periods.	0.00% 0/116 • Adverse events (AEs) and serious AEs (including all-cause mortality data table) were collected from day of first dose to 30 days after last dose of study medication, assessed up to approximately 30 weeks for deferasirox DT treatment (core phase), 39 weeks for deferasirox FCT treatment (core phase) and 52 weeks for deferasirox FCT treatment (extension phase). Any sign or symptom that occurred during the study treatment plus the 30 days post treatment. Safety analyses were performed in all participants who received at least one dose of either study treatment (DT or FCT), and at least one safety assessment on or after Day 1. If, for a participant, AE start date was in deferasirox DT period and end date was in deferasirox FCT period, the participant was counted in both periods.
Infections and infestations Nasopharyngitis	4.1% 6/148 • Adverse events (AEs) and serious AEs (including all-cause mortality data table) were collected from day of first dose to 30 days after last dose of study medication, assessed up to approximately 30 weeks for deferasirox DT treatment (core phase), 39 weeks for deferasirox FCT treatment (core phase) and 52 weeks for deferasirox FCT treatment (extension phase). Any sign or symptom that occurred during the study treatment plus the 30 days post treatment. Safety analyses were performed in all participants who received at least one dose of either study treatment (DT or FCT), and at least one safety assessment on or after Day 1. If, for a participant, AE start date was in deferasirox DT period and end date was in deferasirox FCT period, the participant was counted in both periods.	5.0% 7/140 • Adverse events (AEs) and serious AEs (including all-cause mortality data table) were collected from day of first dose to 30 days after last dose of study medication, assessed up to approximately 30 weeks for deferasirox DT treatment (core phase), 39 weeks for deferasirox FCT treatment (core phase) and 52 weeks for deferasirox FCT treatment (extension phase). Any sign or symptom that occurred during the study treatment plus the 30 days post treatment. Safety analyses were performed in all participants who received at least one dose of either study treatment (DT or FCT), and at least one safety assessment on or after Day 1. If, for a participant, AE start date was in deferasirox DT period and end date was in deferasirox FCT period, the participant was counted in both periods.	2.6% 3/116 • Adverse events (AEs) and serious AEs (including all-cause mortality data table) were collected from day of first dose to 30 days after last dose of study medication, assessed up to approximately 30 weeks for deferasirox DT treatment (core phase), 39 weeks for deferasirox FCT treatment (core phase) and 52 weeks for deferasirox FCT treatment (extension phase). Any sign or symptom that occurred during the study treatment plus the 30 days post treatment. Safety analyses were performed in all participants who received at least one dose of either study treatment (DT or FCT), and at least one safety assessment on or after Day 1. If, for a participant, AE start date was in deferasirox DT period and end date was in deferasirox FCT period, the participant was counted in both periods.
Infections and infestations Pharyngitis	4.7% 7/148 • Adverse events (AEs) and serious AEs (including all-cause mortality data table) were collected from day of first dose to 30 days after last dose of study medication, assessed up to approximately 30 weeks for deferasirox DT treatment (core phase), 39 weeks for deferasirox FCT treatment (core phase) and 52 weeks for deferasirox FCT treatment (extension phase). Any sign or symptom that occurred during the study treatment plus the 30 days post treatment. Safety analyses were performed in all participants who received at least one dose of either study treatment (DT or FCT), and at least one safety assessment on or after Day 1. If, for a participant, AE start date was in deferasirox DT period and end date was in deferasirox FCT period, the participant was counted in both periods.	4.3% 6/140 • Adverse events (AEs) and serious AEs (including all-cause mortality data table) were collected from day of first dose to 30 days after last dose of study medication, assessed up to approximately 30 weeks for deferasirox DT treatment (core phase), 39 weeks for deferasirox FCT treatment (core phase) and 52 weeks for deferasirox FCT treatment (extension phase). Any sign or symptom that occurred during the study treatment plus the 30 days post treatment. Safety analyses were performed in all participants who received at least one dose of either study treatment (DT or FCT), and at least one safety assessment on or after Day 1. If, for a participant, AE start date was in deferasirox DT period and end date was in deferasirox FCT period, the participant was counted in both periods.	5.2% 6/116 • Adverse events (AEs) and serious AEs (including all-cause mortality data table) were collected from day of first dose to 30 days after last dose of study medication, assessed up to approximately 30 weeks for deferasirox DT treatment (core phase), 39 weeks for deferasirox FCT treatment (core phase) and 52 weeks for deferasirox FCT treatment (extension phase). Any sign or symptom that occurred during the study treatment plus the 30 days post treatment. Safety analyses were performed in all participants who received at least one dose of either study treatment (DT or FCT), and at least one safety assessment on or after Day 1. If, for a participant, AE start date was in deferasirox DT period and end date was in deferasirox FCT period, the participant was counted in both periods.
Infections and infestations Upper respiratory tract infection	5.4% 8/148 • Adverse events (AEs) and serious AEs (including all-cause mortality data table) were collected from day of first dose to 30 days after last dose of study medication, assessed up to approximately 30 weeks for deferasirox DT treatment (core phase), 39 weeks for deferasirox FCT treatment (core phase) and 52 weeks for deferasirox FCT treatment (extension phase). Any sign or symptom that occurred during the study treatment plus the 30 days post treatment. Safety analyses were performed in all participants who received at least one dose of either study treatment (DT or FCT), and at least one safety assessment on or after Day 1. If, for a participant, AE start date was in deferasirox DT period and end date was in deferasirox FCT period, the participant was counted in both periods.	5.7% 8/140 • Adverse events (AEs) and serious AEs (including all-cause mortality data table) were collected from day of first dose to 30 days after last dose of study medication, assessed up to approximately 30 weeks for deferasirox DT treatment (core phase), 39 weeks for deferasirox FCT treatment (core phase) and 52 weeks for deferasirox FCT treatment (extension phase). Any sign or symptom that occurred during the study treatment plus the 30 days post treatment. Safety analyses were performed in all participants who received at least one dose of either study treatment (DT or FCT), and at least one safety assessment on or after Day 1. If, for a participant, AE start date was in deferasirox DT period and end date was in deferasirox FCT period, the participant was counted in both periods.	3.4% 4/116 • Adverse events (AEs) and serious AEs (including all-cause mortality data table) were collected from day of first dose to 30 days after last dose of study medication, assessed up to approximately 30 weeks for deferasirox DT treatment (core phase), 39 weeks for deferasirox FCT treatment (core phase) and 52 weeks for deferasirox FCT treatment (extension phase). Any sign or symptom that occurred during the study treatment plus the 30 days post treatment. Safety analyses were performed in all participants who received at least one dose of either study treatment (DT or FCT), and at least one safety assessment on or after Day 1. If, for a participant, AE start date was in deferasirox DT period and end date was in deferasirox FCT period, the participant was counted in both periods.
Infections and infestations Urinary tract infection	6.1% 9/148 • Adverse events (AEs) and serious AEs (including all-cause mortality data table) were collected from day of first dose to 30 days after last dose of study medication, assessed up to approximately 30 weeks for deferasirox DT treatment (core phase), 39 weeks for deferasirox FCT treatment (core phase) and 52 weeks for deferasirox FCT treatment (extension phase). Any sign or symptom that occurred during the study treatment plus the 30 days post treatment. Safety analyses were performed in all participants who received at least one dose of either study treatment (DT or FCT), and at least one safety assessment on or after Day 1. If, for a participant, AE start date was in deferasirox DT period and end date was in deferasirox FCT period, the participant was counted in both periods.	2.1% 3/140 • Adverse events (AEs) and serious AEs (including all-cause mortality data table) were collected from day of first dose to 30 days after last dose of study medication, assessed up to approximately 30 weeks for deferasirox DT treatment (core phase), 39 weeks for deferasirox FCT treatment (core phase) and 52 weeks for deferasirox FCT treatment (extension phase). Any sign or symptom that occurred during the study treatment plus the 30 days post treatment. Safety analyses were performed in all participants who received at least one dose of either study treatment (DT or FCT), and at least one safety assessment on or after Day 1. If, for a participant, AE start date was in deferasirox DT period and end date was in deferasirox FCT period, the participant was counted in both periods.	2.6% 3/116 • Adverse events (AEs) and serious AEs (including all-cause mortality data table) were collected from day of first dose to 30 days after last dose of study medication, assessed up to approximately 30 weeks for deferasirox DT treatment (core phase), 39 weeks for deferasirox FCT treatment (core phase) and 52 weeks for deferasirox FCT treatment (extension phase). Any sign or symptom that occurred during the study treatment plus the 30 days post treatment. Safety analyses were performed in all participants who received at least one dose of either study treatment (DT or FCT), and at least one safety assessment on or after Day 1. If, for a participant, AE start date was in deferasirox DT period and end date was in deferasirox FCT period, the participant was counted in both periods.
Investigations Serum ferritin increased	5.4% 8/148 • Adverse events (AEs) and serious AEs (including all-cause mortality data table) were collected from day of first dose to 30 days after last dose of study medication, assessed up to approximately 30 weeks for deferasirox DT treatment (core phase), 39 weeks for deferasirox FCT treatment (core phase) and 52 weeks for deferasirox FCT treatment (extension phase). Any sign or symptom that occurred during the study treatment plus the 30 days post treatment. Safety analyses were performed in all participants who received at least one dose of either study treatment (DT or FCT), and at least one safety assessment on or after Day 1. If, for a participant, AE start date was in deferasirox DT period and end date was in deferasirox FCT period, the participant was counted in both periods.	1.4% 2/140 • Adverse events (AEs) and serious AEs (including all-cause mortality data table) were collected from day of first dose to 30 days after last dose of study medication, assessed up to approximately 30 weeks for deferasirox DT treatment (core phase), 39 weeks for deferasirox FCT treatment (core phase) and 52 weeks for deferasirox FCT treatment (extension phase). Any sign or symptom that occurred during the study treatment plus the 30 days post treatment. Safety analyses were performed in all participants who received at least one dose of either study treatment (DT or FCT), and at least one safety assessment on or after Day 1. If, for a participant, AE start date was in deferasirox DT period and end date was in deferasirox FCT period, the participant was counted in both periods.	0.86% 1/116 • Adverse events (AEs) and serious AEs (including all-cause mortality data table) were collected from day of first dose to 30 days after last dose of study medication, assessed up to approximately 30 weeks for deferasirox DT treatment (core phase), 39 weeks for deferasirox FCT treatment (core phase) and 52 weeks for deferasirox FCT treatment (extension phase). Any sign or symptom that occurred during the study treatment plus the 30 days post treatment. Safety analyses were performed in all participants who received at least one dose of either study treatment (DT or FCT), and at least one safety assessment on or after Day 1. If, for a participant, AE start date was in deferasirox DT period and end date was in deferasirox FCT period, the participant was counted in both periods.
Investigations Urine protein/creatinine ratio increased	8.8% 13/148 • Adverse events (AEs) and serious AEs (including all-cause mortality data table) were collected from day of first dose to 30 days after last dose of study medication, assessed up to approximately 30 weeks for deferasirox DT treatment (core phase), 39 weeks for deferasirox FCT treatment (core phase) and 52 weeks for deferasirox FCT treatment (extension phase). Any sign or symptom that occurred during the study treatment plus the 30 days post treatment. Safety analyses were performed in all participants who received at least one dose of either study treatment (DT or FCT), and at least one safety assessment on or after Day 1. If, for a participant, AE start date was in deferasirox DT period and end date was in deferasirox FCT period, the participant was counted in both periods.	8.6% 12/140 • Adverse events (AEs) and serious AEs (including all-cause mortality data table) were collected from day of first dose to 30 days after last dose of study medication, assessed up to approximately 30 weeks for deferasirox DT treatment (core phase), 39 weeks for deferasirox FCT treatment (core phase) and 52 weeks for deferasirox FCT treatment (extension phase). Any sign or symptom that occurred during the study treatment plus the 30 days post treatment. Safety analyses were performed in all participants who received at least one dose of either study treatment (DT or FCT), and at least one safety assessment on or after Day 1. If, for a participant, AE start date was in deferasirox DT period and end date was in deferasirox FCT period, the participant was counted in both periods.	6.0% 7/116 • Adverse events (AEs) and serious AEs (including all-cause mortality data table) were collected from day of first dose to 30 days after last dose of study medication, assessed up to approximately 30 weeks for deferasirox DT treatment (core phase), 39 weeks for deferasirox FCT treatment (core phase) and 52 weeks for deferasirox FCT treatment (extension phase). Any sign or symptom that occurred during the study treatment plus the 30 days post treatment. Safety analyses were performed in all participants who received at least one dose of either study treatment (DT or FCT), and at least one safety assessment on or after Day 1. If, for a participant, AE start date was in deferasirox DT period and end date was in deferasirox FCT period, the participant was counted in both periods.
Renal and urinary disorders Proteinuria	5.4% 8/148 • Adverse events (AEs) and serious AEs (including all-cause mortality data table) were collected from day of first dose to 30 days after last dose of study medication, assessed up to approximately 30 weeks for deferasirox DT treatment (core phase), 39 weeks for deferasirox FCT treatment (core phase) and 52 weeks for deferasirox FCT treatment (extension phase). Any sign or symptom that occurred during the study treatment plus the 30 days post treatment. Safety analyses were performed in all participants who received at least one dose of either study treatment (DT or FCT), and at least one safety assessment on or after Day 1. If, for a participant, AE start date was in deferasirox DT period and end date was in deferasirox FCT period, the participant was counted in both periods.	6.4% 9/140 • Adverse events (AEs) and serious AEs (including all-cause mortality data table) were collected from day of first dose to 30 days after last dose of study medication, assessed up to approximately 30 weeks for deferasirox DT treatment (core phase), 39 weeks for deferasirox FCT treatment (core phase) and 52 weeks for deferasirox FCT treatment (extension phase). Any sign or symptom that occurred during the study treatment plus the 30 days post treatment. Safety analyses were performed in all participants who received at least one dose of either study treatment (DT or FCT), and at least one safety assessment on or after Day 1. If, for a participant, AE start date was in deferasirox DT period and end date was in deferasirox FCT period, the participant was counted in both periods.	10.3% 12/116 • Adverse events (AEs) and serious AEs (including all-cause mortality data table) were collected from day of first dose to 30 days after last dose of study medication, assessed up to approximately 30 weeks for deferasirox DT treatment (core phase), 39 weeks for deferasirox FCT treatment (core phase) and 52 weeks for deferasirox FCT treatment (extension phase). Any sign or symptom that occurred during the study treatment plus the 30 days post treatment. Safety analyses were performed in all participants who received at least one dose of either study treatment (DT or FCT), and at least one safety assessment on or after Day 1. If, for a participant, AE start date was in deferasirox DT period and end date was in deferasirox FCT period, the participant was counted in both periods.
Investigations Hepatic enzyme increased	3.4% 5/148 • Adverse events (AEs) and serious AEs (including all-cause mortality data table) were collected from day of first dose to 30 days after last dose of study medication, assessed up to approximately 30 weeks for deferasirox DT treatment (core phase), 39 weeks for deferasirox FCT treatment (core phase) and 52 weeks for deferasirox FCT treatment (extension phase). Any sign or symptom that occurred during the study treatment plus the 30 days post treatment. Safety analyses were performed in all participants who received at least one dose of either study treatment (DT or FCT), and at least one safety assessment on or after Day 1. If, for a participant, AE start date was in deferasirox DT period and end date was in deferasirox FCT period, the participant was counted in both periods.	2.9% 4/140 • Adverse events (AEs) and serious AEs (including all-cause mortality data table) were collected from day of first dose to 30 days after last dose of study medication, assessed up to approximately 30 weeks for deferasirox DT treatment (core phase), 39 weeks for deferasirox FCT treatment (core phase) and 52 weeks for deferasirox FCT treatment (extension phase). Any sign or symptom that occurred during the study treatment plus the 30 days post treatment. Safety analyses were performed in all participants who received at least one dose of either study treatment (DT or FCT), and at least one safety assessment on or after Day 1. If, for a participant, AE start date was in deferasirox DT period and end date was in deferasirox FCT period, the participant was counted in both periods.	5.2% 6/116 • Adverse events (AEs) and serious AEs (including all-cause mortality data table) were collected from day of first dose to 30 days after last dose of study medication, assessed up to approximately 30 weeks for deferasirox DT treatment (core phase), 39 weeks for deferasirox FCT treatment (core phase) and 52 weeks for deferasirox FCT treatment (extension phase). Any sign or symptom that occurred during the study treatment plus the 30 days post treatment. Safety analyses were performed in all participants who received at least one dose of either study treatment (DT or FCT), and at least one safety assessment on or after Day 1. If, for a participant, AE start date was in deferasirox DT period and end date was in deferasirox FCT period, the participant was counted in both periods.

Additional Information

Study director

Novartis Pharmaceuticals

Phone: 862-778-8300

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (ie, data from all sites) in the clinical trial.
Publication restrictions are in place

Restriction type: OTHER