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

• Sponsor: Novartis Pharmaceuticals (Industry)
• Overall Status: Completed
• CT.gov ID: NCT02993224
• Collaborator: (none)
• Enrollment: 148
• Locations: 17
• Arm: 1
• Actual Duration (Months): 43.5
• Patients Per Site: 8.7
• Patients Per Site Per Month: 0.2

Study Details

Study Description

Brief Summary

Study to evaluate patient preference of deferasirox film-coated tablet (FCT) or deferasirox dispersible tablet (DT) in patient with transfusion - dependent thalassemia or non-transfusion -dependent thalassemia as measured by preference questionnaire at Week 48

Condition or Disease	Intervention/Treatment	Phase
Transfusion-dependent Thalassemia; Non-transfusion-dependent Thalassemia	Drug: Deferasirox dispersable tablet (DT); Drug: Deferasirox film coated tablet (FCT)	Phase 2

Detailed Description

This was an open-label, multicenter, single arm, phase II study aimed at collecting data on preference for iron chelation therapy in patients with transfusion-dependent thalassemia (TDT) or non-transfusion-dependent thalassemia (NTDT) throughout a 48 week treatment period. Participants who were either chelator-naïve, or who were previously treated with iron chelators (excluding deferasirox) for at least 6 months continuously, were eligible to participate in this study.

The study was divided into 2 phases:

1. Core Phase:

• Screening phase which lasted for a maximum of 4 weeks to determine patient eligibility followed by

• Period 1: Participants were treated with deferasirox DT from Baseline visit Day 1 to Week 24

• Period 2: Participants were treated with deferasirox FCT from Week 25 to Week 48:

At the discretion of the investigator, patients could switch from deferasirox DT to deferasirox FCT at any time during Period 1 of the Core phase, and vice versa, from deferasirox FCT to deferasirox DT at any time during Period 2 of the Core phase. Re-switching treatments was not allowed within each period.

1. Extension Phase:

Participants could continue deferasirox FCT formulation as per the judgment of the investigator, through an extension phase for a maximum of 48 weeks months from the last dose of deferasirox FCT received at the end of period 2 in the Core Phase or until one of the end of study criteria defined is met, whichever came first. Participation in the extension phase was optional.

The end of study was defined as the earliest occurrence of one of the following:

• The patient reached Week 96 in the Extension phase.

• Deferasirox FCT was locally reimbursed for this indication (only applicable for the Extension phase)

• Another clinical study or post-trial access program became available that could continue to provide deferasirox FCT in this patient population and all patients ongoing were eligible to be transferred to that clinical study.

Study Design

• Study Type: Interventional
• Actual Enrollment: 148 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Other
• Official Title: Open-label, Multicenter, Single Arm, Phase II Study Assessing Treatment Patient Preference for New Deferasirox Formulation (Film-coated Tablet) Compared to the Reference Deferasirox Dispersible Tablet Formulation
• Actual Study Start Date: Jul 27, 2017
• Actual Primary Completion Date: Jan 29, 2020
• Actual Study Completion Date: Mar 11, 2021

Arms and Interventions

Arm

Intervention/Treatment

Experimental: 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

Drug: Deferasirox dispersable tablet (DT); Deferasirox DT was provided as 125 mg, 250 mg, 500 mg dispersible tablets for oral use. The strengths provided in an individual country could differ and reflected the strengths available commercially in each country. For iron chelation naive participants, the starting dose on Baseline Day 1 was 20 mg/kg/day in TDT and 10 mg/kg/day in NTDT. For iron chelation (deferoxamine and/or deferiprone) pre-treated participants, the starting dose was equivalent to the dose of deferoxamine received (for participants pre-treated with deferoxamine) and based on their serum ferritin levels (for participants pre-treated with deferiprone). Participants took deferasirox DT once daily for 24 weeks (core phase). The required number of deferasirox DT tablets were to be dispersed with gentle stirring in a glass of water.; Drug: Deferasirox film coated tablet (FCT); Deferasirox FCT was provided as 90 mg, 180 mg, 360 mg film coated tablets for oral use. The FCT starting dose on Week 25 was 14 mg/kg/day in TDT and 7 mg/kg/day in NTDT. Participants took deferasirox FCT once daily for 24 weeks during the core phase and up to 48 weeks during the extension phase. For patients with difficulties in swallowing deferasirox FCT, it was allowed to crush the film-coated tablets and administer the study drug by sprinkling the full dose on soft food (like yogurt or apple puree).

Outcome Measures

Primary Outcome Measures

1. Number of Participants Preferring Deferasirox FCT or DT at Week 48 Based on Preference Questionnaire (Item 2) [Week 48]

   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.

Secondary Outcome Measures

1. Number of Participants Preferring Deferasirox FCT, Deferasirox DT or Previous Iron Chelation Therapy at Week 28 Based on Preference Questionnaire (Item 2) [Week 28]

   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.

2. 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 and Week 24]

   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.

3. Number of Participants Selecting Each Reason for Treatment Preference as Assessed by the Preference Questionnaire at Week 28 and Week 48 [Week 28 and Week 48]

   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.

4. Percentage of Consumed Tablet Counts During Deferasirox DT and Deferasirox FCT Treatment Periods [Deferasirox DT: From Baseline up to Week 24. Deferasirox FCT: From Week 25 up to Week 48]

   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.

5. Change Over Time in Aftertaste Score of Palatability Questionnaire [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

6. Change Over Time in Palatability Score of Palatability Questionnaire [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

7. Change From Baseline in Adherence Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire [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.

8. Change From Baseline in Preference Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire [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.

9. Change From Baseline in Concerns Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire [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.

10. Change From Baseline in Gastrointestinal (GI) Symptom Score Based on GI Questionnaire [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.

11. Change From Baseline in Serum Ferritin Levels [From Baseline (Day 1) up to 96 weeks]

    Absolute change from baseline over time in serum ferritin levels

Eligibility Criteria

Criteria

• Ages Eligible for Study: 2 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

1. Prior to any screening procedures were performed, written informed consent/assent must be provided. For pediatric patients, consent was obtained from parent(s) or legal patient's representative. Investigators also obtained assent of patients according to local, regional or national guidelines.

2. Male and female patient aged ≥ 2 years

3. Deferasirox naïve patient or chelated naive patient or treated by other chelators for at least 6 months, such as:

4. Deferiprone/ DFP

5. Deferoxamine /DFO

6. Combination (DFO + DFP)

7. Subject was willing to discontinue current iron chelation therapy at least 5 days prior to study day 1 and for the duration of the study

8. Patients with transfusion-dependent thalassemia (independent of underlying condition) with transfusional iron overload as shown by a serum ferritin level of > 1000 ng/ml at screening and if available, LIC > 3 mg Fe/g dw within 6 months prior to screening

9. Patients with non-transfusion-dependent thalassemia with iron overload as shown by a serum ferritin level of ≥ 800 ng/ml at screening and if available, LIC ≥ 5 mg Fe/g dw within 6 months prior to screening

Exclusion Criteria:

1. Creatinine clearance below the contraindication limit in the locally approved prescribing information.

2. Serum creatinine level > 1.5 x ULN (upper limit of normal)

3. AST (SGOT) /ALT (SGPT) > 5 x ULN, unless LIC confirmed as >10 mg Fe/dw within 6 months prior to screening visit.

4. Significant proteinuria as indicated by a urinary protein/creatinine ratio > 0.5 mg/mg in a non-first void urine sample.

5. Patients with significant impaired gastrointestinal (GI) function or GI disease that might significantly alter the absorption of oral deferasirox (e.g. ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, or small bowel resection).

6. Clinical or laboratory evidence of active Hepatitis B or Hepatitis C (HBsAg in the absence of HBsAb OR HCV Ab positive with HCV RNA positive).

7. Patients with psychiatric or addictive disorders which prevent them from giving their informed consent or undergoing any of the treatment options or patients unwilling or unable to comply with the protocol

8. Patients with a known history of HIV seropositivity (Elisa or Western blot).

9. History of malignancy of any organ system, treated or untreated, within the past 5 years whether or not there is evidence of local recurrence or metastases, with the exception of localized basal cell carcinoma of the skin.

10. Patients participating in another clinical trial or receiving an investigational drug. Patients who have recently completed treatment with an investigational product must have ceased this treatment for at least five times the half-life of the investigational product.

11. History of hypersensitivity to any of the study drug or excipients.

12. Significant medical condition interfering with the ability to partake in this study (e.g. systemic uncontrolled hypertension, unstable cardiac disease not controlled by standard medical therapy, systemic disease (cardiovascular, renal, hepatic, etc.).

13. Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they were using effective methods of contraception during dosing of study treatment

14. Women were considered post-menopausal and not of child bearing potential if they had had 12 months of natural (spontaneous) amenorrhea with an appropriate clinical profile (e.g. age appropriate, history of vasomotor symptoms) or had had surgical bilateral oophorectomy (with or without hysterectomy) or tubal ligation at least six weeks ago. In the case of oophorectomy alone, only when the reproductive status of the woman had been confirmed by follow up hormone level assessment is she considered not of child bearing potential.

15. Sexually active males unless they used a condom during intercourse while taking drug and for 28 days after stopping study medication and should not father a child in this period. A condom was required to be used also by vasectomized men in order to prevent delivery of the drug via seminal fluid.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Novartis Investigative Site	Alexandria		Egypt	21131
2	Novartis Investigative Site	Cairo		Egypt	11562
3	Novartis Investigative Site	Zagazig		Egypt	44519
4	Novartis Investigative Site	Hazmiyeh	Beirut	Lebanon	PO BOX 213
5	Novartis Investigative Site	Rabat		Morocco	10102
6	Novartis Investigative Site	Al Ahsa	SAU	Saudi Arabia
7	Novartis Investigative Site	Jeddah		Saudi Arabia	21159
8	Novartis Investigative Site	Jeddah		Saudi Arabia	21589
9	Novartis Investigative Site	Riyadh		Saudi Arabia	11117
10	Novartis Investigative Site	Bangkok noi	Bangkok	Thailand	10700
11	Novartis Investigative Site	Bangkoknoi	Bangkok	Thailand	10700
12	Novartis Investigative Site	Bangkok		Thailand	10400
13	Novartis Investigative Site	Ankara		Turkey	06100
14	Novartis Investigative Site	Antalya		Turkey	07070
15	Novartis Investigative Site	Istanbul		Turkey	34093
16	Novartis Investigative Site	Hanoi		Vietnam
17	Novartis Investigative Site	Ho Chi Minh City		Vietnam	DISTRICT 1

Sponsors and Collaborators

• Novartis Pharmaceuticals

Investigators

None specified.

Study Documents (Full-Text)

• Study Protocol - Feb 12, 2019
• Statistical Analysis Plan - Apr 8, 2021

More Information

Publications

None provided.

• Responsible Party: Novartis Pharmaceuticals
• ClinicalTrials.gov Identifier: NCT02993224
• Other Study ID Numbers: CICL670FIC05; 2016-002282-61
• First Posted: Dec 15, 2016
• Last Update Posted: Oct 4, 2021
• Last Verified: Sep 1, 2021

• Individual Participant Data (IPD) Sharing Statement: Yes
• Plan to Share IPD: Yes
• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No
• Keywords provided by Novartis Pharmaceuticals: thalassemia; TDT; transfusion-dependent; non-transfusion-dependent; NTDT
• Additional relevant MeSH terms: Thalassemia; Deferasirox

Study Results

Participant Flow

Recruitment Details	Participants took part in 17 investigative sites in 7 countries

Arm/Group Title	Deferasirox DT Followed by Deferasirox FCT
Arm/Group Description	Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT
Period Title: Core Phase - Period 1
STARTED	148
COMPLETED	140
NOT COMPLETED	8
Period Title: Core Phase - Period 1
STARTED	140
COMPLETED	136
NOT COMPLETED	4
Period Title: Core Phase - Period 1
STARTED	116
COMPLETED	80
NOT COMPLETED	36

Baseline Characteristics

Arm/Group Title	Deferasirox DT Followed by Deferasirox FCT
Arm/Group Description	Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT
Overall Participants	148
Age (Years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [Years]	15.32 (13.824)
Sex: Female, Male (Count of Participants)
Female	82 55.4%
Male	66 44.6%
Race/Ethnicity, Customized (Count of Participants)
Indian (Indian subcontinent)	5 3.4%
Other	143 96.6%

Outcome Measures

1. Primary Outcome

Title	Number of Participants Preferring Deferasirox FCT or DT at Week 48 Based on Preference Questionnaire (Item 2)
Description	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.
Time Frame	Week 48

Outcome Measure Data

Analysis Population Description

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.

Arm/Group Title	Deferasirox DT Followed by Deferasirox FCT
Arm/Group Description	Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT
Measure Participants	134
Preference for deferasirox DT	10 6.8%
Preference for deferasirox FCT	121 81.8%
Preference for none of the above	3 2%

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Deferasirox DT Followed by Deferasirox FCT
	Comments	Preference for deferasirox DT vs deferasirox FCT
	Type of Statistical Test	Superiority
	Comments
Statistical Test of Hypothesis	p-Value	<.0001
	Comments
	Method	McNemar
	Comments
Method of Estimation	Estimation Parameter	Difference of proportion
	Estimated Value	0.83
	Confidence Interval	(2-Sided) 95%; 0.75 to 0.89
	Parameter Dispersion	Type: Value:
	Estimation Comments

2. Secondary Outcome

Title	Number of Participants Preferring Deferasirox FCT, Deferasirox DT or Previous Iron Chelation Therapy at Week 28 Based on Preference Questionnaire (Item 2)
Description	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.
Time Frame	Week 28

Outcome Measure Data

Analysis Population Description

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.

Arm/Group Title	Deferasirox DT Followed by Deferasirox FCT
Arm/Group Description	Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT
Measure Participants	69
Preference for deferasirox DT	6 4.1%
Preference for deferasirox FCT	60 40.5%
Preference for previous iron chelation therapy	3 2%
Preference for none of the above	0 0%

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Deferasirox DT Followed by Deferasirox FCT
	Comments	Preference of deferasirox FCT vs deferasirox DT
	Type of Statistical Test	Superiority
	Comments
Statistical Test of Hypothesis	p-Value	<0.0001
	Comments
	Method	McNemar
	Comments
Method of Estimation	Estimation Parameter	Difference of proportion
	Estimated Value	0.78
	Confidence Interval	(2-Sided) 95%; 0.65 to 0.88
	Parameter Dispersion	Type: Value:
	Estimation Comments

Statistical Analysis 2

Statistical Analysis Overview	Comparison Group Selection	Deferasirox DT Followed by Deferasirox FCT
	Comments	Preference for deferasirox FCT vs previous iron chelation therapy
	Type of Statistical Test	Superiority
	Comments
Statistical Test of Hypothesis	p-Value	<0.0001
	Comments
	Method	McNemar
	Comments
Method of Estimation	Estimation Parameter	Difference of proportion
	Estimated Value	0.83
	Confidence Interval	(2-Sided) 95%; 0.71 to 0.91
	Parameter Dispersion	Type: Value:
	Estimation Comments

3. Secondary Outcome

Title	Number of Participants Preferring Deferasirox DT or Previous Iron Chelation Therapy at Week 4 and Week 24 Based on Preference Questionnaire (Item 2)
Description	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.
Time Frame	Week 4 and Week 24

Outcome Measure Data

Analysis Population Description

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.

Arm/Group Title	Deferasirox DT Followed by Deferasirox FCT
Arm/Group Description	Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT
Measure Participants	70
Preference for deferasirox DT	57 38.5%
Preference for previous iron chelation therapy	11 7.4%
Preference for none of the above	2 1.4%
Preference for deferasirox DT	52 35.1%
Preference for previous iron chelation therapy	11 7.4%
Preference for none of the above	6 4.1%

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Deferasirox DT Followed by Deferasirox FCT
	Comments	Deferasirox DT vs previous iron chelation therapy at Week 4
	Type of Statistical Test	Superiority
	Comments
Statistical Test of Hypothesis	p-Value	< 0.0001
	Comments
	Method	McNemar
	Comments
Method of Estimation	Estimation Parameter	Difference of proportion
	Estimated Value	0.66
	Confidence Interval	(2-Sided) 95%; 0.51 to 0.77
	Parameter Dispersion	Type: Value:
	Estimation Comments

Statistical Analysis 2

Statistical Analysis Overview	Comparison Group Selection	Deferasirox DT Followed by Deferasirox FCT
	Comments	Deferasirox DT vs previous iron chelation therapy at Week 24
	Type of Statistical Test	Superiority
	Comments
Statistical Test of Hypothesis	p-Value	< 0.0001
	Comments
	Method	McNemar
	Comments
Method of Estimation	Estimation Parameter	Difference of proportion
	Estimated Value	0.59
	Confidence Interval	(2-Sided) 95%; 0.44 to 0.72
	Parameter Dispersion	Type: Value:
	Estimation Comments

4. Secondary Outcome

Title	Number of Participants Selecting Each Reason for Treatment Preference as Assessed by the Preference Questionnaire at Week 28 and Week 48
Description	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.
Time Frame	Week 28 and Week 48

Outcome Measure Data

Analysis Population Description

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

Arm/Group Title	Deferasirox DT Followed by Deferasirox FCT
Arm/Group Description	Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT
Measure Participants	139
Aftertaste (Week 28)	9 6.1%
Can correctly prepare the medicine (Week 28)	35 23.6%
Convenience (it's not a problem to take your medicine) (Week 28)	109 73.6%
Easier to remember to take the medicine (Week 28)	57 38.5%
Gain my personal time with family and friends (Week 28)	35 23.6%
No/ less pain on the injection site (Week 28)	31 20.9%
No/ less side effects (Week 28)	50 33.8%
Number of pills (Week 28)	46 31.1%
Number of times you have to take the medicine (Week 28)	42 28.4%
Other (Week 28)	5 3.4%
Taste (Week 28)	30 20.3%
Aftertaste (Week 48)	17 11.5%
Can correctly prepare the medicine (Week 48)	48 32.4%
Convenience (it's not a problem to take your medicine) (Week 48)	103 69.6%
Easier to remember to take the medicine (Week 48)	61 41.2%
Gain my personal time with family and friends (Week 48)	36 24.3%
No/ less pain on the injection site (Week 48)	37 25%
No/ less side effects (Week 48)	48 32.4%
Number of pills (Week 48)	41 27.7%
Number of times you have to take the medicine (Week 48)	47 31.8%
Other (Week 48)	0 0%
Taste (Week 48)	51 34.5%

5. Secondary Outcome

Title	Percentage of Consumed Tablet Counts During Deferasirox DT and Deferasirox FCT Treatment Periods
Description	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.
Time Frame	Deferasirox DT: From Baseline up to Week 24. Deferasirox FCT: From Week 25 up to Week 48

Outcome Measure Data

Analysis Population Description

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

Arm/Group Title	Deferasirox DT Followed by Deferasirox FCT
Arm/Group Description	Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT
Measure Participants	139
Deferasirox DT (Baseline up to Week 24)	98.68 (22.373)
Deferasirox FCT (From Week 25 up to Week 48)	95.07 (15.368)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Deferasirox DT Followed by Deferasirox FCT
	Comments	Compliance of deferasirox DT vs deferasirox FCT
	Type of Statistical Test	Superiority
	Comments
Statistical Test of Hypothesis	p-Value	0.1191
	Comments
	Method	ANCOVA
	Comments
Method of Estimation	Estimation Parameter	Least squares mean
	Estimated Value	-3.6
	Confidence Interval	(2-Sided) 95%; -8.1 to 0.9
	Parameter Dispersion	Type: Standard Error of the Mean; Value: 2.30
	Estimation Comments

6. Secondary Outcome

Title	Change Over Time in Aftertaste Score of Palatability Questionnaire
Description	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
Time Frame	Week 4, 24, 28 and 48

Outcome Measure Data

Analysis Population Description

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 at specified timepoints.

Arm/Group Title	Deferasirox DT Followed by Deferasirox FCT
Arm/Group Description	Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT
Measure Participants	139
From Week 4 to Week 24 in participants treated with deferasirox DT at Week 24	-0.1 (0.80)
From Week 4 to Week 24 in participants treated with deferasirox FCT at Week 24	-0.5 (1.22)
From Week 24 to Week 28 in participants treated with deferasirox FCT at Week 28	-0.5 (1.16)
From Week 24 to Week 48 in participants treated with deferasirox FCT at Week 48	-0.5 (1.08)

7. Secondary Outcome

Title	Change Over Time in Palatability Score of Palatability Questionnaire
Description	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
Time Frame	Week 4, 24, 28 and 48

Outcome Measure Data

Analysis Population Description

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 at specified timepoints.

Arm/Group Title	Deferasirox DT Followed by Deferasirox FCT
Arm/Group Description	Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT
Measure Participants	139
From Week 4 to Week 24- participants treated with deferasirox DT at Week 24	-0.1 (2.46)
From Week 4 to Week 24 in participants treated with deferasirox FCT at Week 24	0.0 (0.00)
From Week 24 to Week 28 in participants treated with deferasirox FCT at Week 28	1.1 (2.77)
From Week 24 to Week 48 in participants treated with deferasirox FCT at Week 48	1.3 (2.54)

8. Secondary Outcome

Title	Change From Baseline in Adherence Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire
Description	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.
Time Frame	Baseline (week 2 or, if missing, week 3), week 24, 28 and 48

Outcome Measure Data

Analysis Population Description

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 at specified timepoints

Arm/Group Title	Deferasirox DT Followed by Deferasirox FCT
Arm/Group Description	Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT
Measure Participants	139
Week 24- participants ≥ 10 years treated with deferasirox DT (PRO)	-0.3 (3.37)
Week 24 - participants ≥ 10 years treated with deferasirox FCT (PRO)	-1.0 (2.94)
Week 28 - participants ≥ 10 years treated with deferasirox FCT (PRO)	0.5 (3.59)
Week 48 - participants ≥ 10 years treated with deferasirox FCT (PRO)	0.5 (3.29)
Week 24 - participants < 10 years treated with deferasirox DT (ObsRO)	-0.2 (3.35)
Week 24 - participants < 10 years treated with deferasirox FCT (ObsRO)	-0.7 (4.16)
Week 28 - participants < 10 years treated with deferasirox FCT (ObsRO)	0.8 (3.68)
Week 48 - participants < 10 years treated with deferasirox FCT (ObsRO)	1.1 (3.79)

9. Secondary Outcome

Title	Change From Baseline in Preference Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire
Description	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.
Time Frame	Baseline (week 2 or, if missing, week 3), week 24, 28 and 48

Outcome Measure Data

Analysis Population Description

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 at specified timepoints

Arm/Group Title	Deferasirox DT Followed by Deferasirox FCT
Arm/Group Description	Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT
Measure Participants	139
Week 24 - participants ≥ 10 treated with deferasirox DT (PRO)	0.5 (1.21)
Week 24 - participants ≥ 10 treated with deferasirox FCT (PRO)	0.5 (3.70)
Week 28 - participants ≥ 10 treated with deferasirox FCT (PRO)	-1.1 (1.83)
Week 48 - participants ≥ 10 treated with deferasirox FCT (PRO)	-0.9 (2.02)
Week 24 - participants < 10 years treated with deferasirox DT (ObsRO)	0.0 (1.49)
Week 24 - participants < 10 years treated with deferasirox FCT (ObsRO)	0.0 (1.73)
Week 28 - participants < 10 years treated with deferasirox FCT (ObsRO)	-0.8 (1.74)
Week 48 - participants < 10 years treated with deferasirox FCT (ObsRO)	-0.9 (1.75)

10. Secondary Outcome

Title	Change From Baseline in Concerns Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire
Description	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.
Time Frame	Baseline (week 2 or, if missing, week 3), week 24, 28 and 48

Outcome Measure Data

Analysis Population Description

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 at specified timepoints

Arm/Group Title	Deferasirox DT Followed by Deferasirox FCT
Arm/Group Description	Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT
Measure Participants	139
Week 24 - participants ≥ 10 treated with deferasirox DT (PRO)	-0.1 (1.99)
Week 24 - participants ≥ 10 treated with deferasirox FCT (PRO)	-1.0 (1.15)
Week 28 - participants ≥ 10 treated with deferasirox FCT (PRO)	0.3 (1.84)
Week 48 - participants ≥ 10 treated with deferasirox FCT (PRO)	0.5 (1.80)
Week 24 - participants < 10 years treated with deferasirox DT (ObsRO)	-0.4 (1.33)
Week 24 - participants < 10 years treated with deferasirox FCT (ObsRO)	-0.7 (1.15)
Week 28 - participants < 10 years treated with deferasirox FCT (ObsRO)	0.1 (2.05)
Week 48 - participants < 10 years treated with deferasirox FCT (ObsRO)	0.1 (1.58)

11. Secondary Outcome

Title	Change From Baseline in Gastrointestinal (GI) Symptom Score Based on GI Questionnaire
Description	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.
Time Frame	Baseline (week -1 or, if missing, week -2), week 24, 28 and 48

Outcome Measure Data

Analysis Population Description

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 at specified timepoints

Arm/Group Title	Deferasirox DT Followed by Deferasirox FCT
Arm/Group Description	Participants were treated with deferasirox DT followed by deferasirox FCT (core phase). Those who entered the extension phase were treated with deferasirox FCT
Measure Participants	139
Week 24 - participants ≥ 10 years treated with deferasirox DT	1.4 (5.53)
Week 24 - participants ≥ 10 years treated with deferasirox FCT	5.8 (12.82)
Week 28 - participants ≥ 10 years treated with deferasirox FCT	-1.9 (4.33)
Week 48 -participants ≥ 10 years treated with deferasirox FCT	-2.2 (4.74)
Week 24 - participants < 10 years treated with deferasirox DT	0.2 (2.78)
Week 24 - participants < 10 years treated with deferasirox FCT	-2.0 (2.65)
Week 28 - participants < 10 years treated with deferasirox FCT	-0.5 (2.49)
Week 48 - participants < 10 years treated with deferasirox FCT	-0.6 (2.33)

12. Secondary Outcome

Title	Change From Baseline in Serum Ferritin Levels
Description	Absolute change from baseline over time in serum ferritin levels
Time Frame	From Baseline (Day 1) up to 96 weeks

Outcome Measure Data

Analysis Population Description

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 at specified timepoints

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

Adverse Events

Time Frame	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).
Adverse Event Reporting Description	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.
Arm/Group Title	Deferasirox DT (Core Phase)		Deferasirox FCT (Core Phase)		Deferasirox FCT (Extension Phase)
Arm/Group Description	Participants who were treated with deferasirox DT once daily in the core phase		Participants who were treated with deferasirox FCT once daily in the core phase		Participants who were treated with deferasirox FCT once daily in the extension phase
All Cause Mortality
	Deferasirox DT (Core Phase)		Deferasirox FCT (Core Phase)		Deferasirox FCT (Extension Phase)
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/148 (0%)		0/140 (0%)		0/116 (0%)
Serious Adverse Events
	Deferasirox DT (Core Phase)		Deferasirox FCT (Core Phase)		Deferasirox FCT (Extension Phase)
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	13/148 (8.8%)		6/140 (4.3%)		16/116 (13.8%)
Blood and lymphatic system disorders
Haemolysis	0/148 (0%)		1/140 (0.7%)		1/116 (0.9%)
Leukopenia	2/148 (1.4%)		0/140 (0%)		0/116 (0%)
Neutropenia	2/148 (1.4%)		0/140 (0%)		0/116 (0%)
Thrombocytopenia	0/148 (0%)		0/140 (0%)		1/116 (0.9%)
Cardiac disorders
Cardiac failure chronic	0/148 (0%)		1/140 (0.7%)		0/116 (0%)
Cardiac failure congestive	1/148 (0.7%)		0/140 (0%)		0/116 (0%)
Eye disorders
Optic nerve cupping	0/148 (0%)		0/140 (0%)		1/116 (0.9%)
Gastrointestinal disorders
Pancreatitis	0/148 (0%)		0/140 (0%)		1/116 (0.9%)
General disorders
Pyrexia	1/148 (0.7%)		1/140 (0.7%)		2/116 (1.7%)
Hepatobiliary disorders
Cholangitis acute	0/148 (0%)		0/140 (0%)		1/116 (0.9%)
Jaundice cholestatic	0/148 (0%)		0/140 (0%)		1/116 (0.9%)
Infections and infestations
Appendicitis	1/148 (0.7%)		0/140 (0%)		0/116 (0%)
Cellulitis	0/148 (0%)		1/140 (0.7%)		0/116 (0%)
Diarrhoea infectious	0/148 (0%)		0/140 (0%)		1/116 (0.9%)
Ecthyma	0/148 (0%)		0/140 (0%)		1/116 (0.9%)
Gastrointestinal infection	0/148 (0%)		0/140 (0%)		1/116 (0.9%)
Influenza	1/148 (0.7%)		0/140 (0%)		1/116 (0.9%)
Otitis media	0/148 (0%)		0/140 (0%)		1/116 (0.9%)
Respiratory tract infection	0/148 (0%)		0/140 (0%)		1/116 (0.9%)
Viral infection	0/148 (0%)		0/140 (0%)		1/116 (0.9%)
Injury, poisoning and procedural complications
Abdominal injury	0/148 (0%)		0/140 (0%)		1/116 (0.9%)
Femur fracture	0/148 (0%)		0/140 (0%)		1/116 (0.9%)
Gastrointestinal injury	0/148 (0%)		0/140 (0%)		1/116 (0.9%)
Road traffic accident	0/148 (0%)		0/140 (0%)		1/116 (0.9%)
Splenic rupture	0/148 (0%)		0/140 (0%)		1/116 (0.9%)
Ulna fracture	0/148 (0%)		0/140 (0%)		1/116 (0.9%)
Investigations
Alanine aminotransferase increased	2/148 (1.4%)		1/140 (0.7%)		1/116 (0.9%)
Aspartate aminotransferase increased	2/148 (1.4%)		1/140 (0.7%)		0/116 (0%)
Blood bilirubin increased	1/148 (0.7%)		0/140 (0%)		0/116 (0%)
Hepatic enzyme increased	1/148 (0.7%)		0/140 (0%)		0/116 (0%)
Metabolism and nutrition disorders
Hypokalaemia	0/148 (0%)		0/140 (0%)		1/116 (0.9%)
Metabolic acidosis	0/148 (0%)		0/140 (0%)		1/116 (0.9%)
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Cholangiocarcinoma	0/148 (0%)		0/140 (0%)		1/116 (0.9%)
Nervous system disorders
Polyneuropathy	1/148 (0.7%)		0/140 (0%)		0/116 (0%)
Renal and urinary disorders
Acute kidney injury	1/148 (0.7%)		0/140 (0%)		1/116 (0.9%)
Proteinuria	1/148 (0.7%)		1/140 (0.7%)		1/116 (0.9%)
Skin and subcutaneous tissue disorders
Angioedema	1/148 (0.7%)		0/140 (0%)		0/116 (0%)
Vascular disorders
Hypertension	1/148 (0.7%)		0/140 (0%)		0/116 (0%)
Other (Not Including Serious) Adverse Events
	Deferasirox DT (Core Phase)		Deferasirox FCT (Core Phase)		Deferasirox FCT (Extension Phase)
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	82/148 (55.4%)		59/140 (42.1%)		49/116 (42.2%)
Gastrointestinal disorders
Diarrhoea	16/148 (10.8%)		5/140 (3.6%)		2/116 (1.7%)
General disorders
Pyrexia	7/148 (4.7%)		8/140 (5.7%)		5/116 (4.3%)
Hepatobiliary disorders
Hepatomegaly	8/148 (5.4%)		0/140 (0%)		0/116 (0%)
Infections and infestations
Nasopharyngitis	6/148 (4.1%)		7/140 (5%)		3/116 (2.6%)
Pharyngitis	7/148 (4.7%)		6/140 (4.3%)		6/116 (5.2%)
Upper respiratory tract infection	8/148 (5.4%)		8/140 (5.7%)		4/116 (3.4%)
Urinary tract infection	9/148 (6.1%)		3/140 (2.1%)		3/116 (2.6%)
Investigations
Serum ferritin increased	8/148 (5.4%)		2/140 (1.4%)		1/116 (0.9%)
Urine protein/creatinine ratio increased	13/148 (8.8%)		12/140 (8.6%)		7/116 (6%)
Hepatic enzyme increased	5/148 (3.4%)		4/140 (2.9%)		6/116 (5.2%)
Renal and urinary disorders
Proteinuria	8/148 (5.4%)		9/140 (6.4%)		12/116 (10.3%)

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

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.