MIMAS: Safety Study of Crushed Deferasirox Film Coated Tablets in Pediatric Patients With Transfusional Hemosiderosis

• Sponsor: Novartis Pharmaceuticals (Industry)
• Overall Status: Completed
• CT.gov ID: NCT03372083
• Collaborator: (none)
• Enrollment: 44
• Locations: 10
• Arm: 1
• Actual Duration (Months): 22.6
• Patients Per Site: 4.4
• Patients Per Site Per Month: 0.2

Study Details

Study Description

Brief Summary

This study employed a prospective, single-arm, global multi-center interventional open-label, non-randomized design to identify and assess safety profile of the crushed deferasirox FCT when administered up to 24 weeks in pediatric patients aged ≥2 to <6 years with transfusional hemosiderosis. The study was designed to enroll a minimum of 40 patients. Forty-four patients were treated and analyzed.

Condition or Disease	Intervention/Treatment	Phase
Iron Overload	Drug: Deferasirox	Phase 4

Detailed Description

The study included a screening period (from Day 0-14) with two visits at least 7 days apart to assess eligibility of patients that were chelation naïve or on a prior iron chelator treatment other than DFX. For Patients on DFX treatment prior to study entry only one screening visit (screening visit 1) were to occur to determine eligibility. Any current chelation therapy except deferasirox were to be discontinued to undergo a 5-day washout period prior to commencing a 24 week treatment period with crushed deferasirox FCT.

All patients were to have weekly visits for the first month to monitor renal function. Hepatic function were to be assessed biweekly during the first month. Thereafter, monthly safety assessments were to be performed, including the monitoring of serum ferritin values and trends in order to adapt patient treatment.

Eligibility, application of dosing standards and adjustments, as well as safety and serum ferritin assessments as specified in the protocol.

The planned duration of treatment was 24 weeks followed by a 30-day safety follow up.

Study Design

• Study Type: Interventional
• Actual Enrollment: 44 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: A Single-arm Interventional Phase IV, Post-authorisation Study Evaluating the Safety of Pediatric Patients With Transfusional Hemosiderosis Treated With Deferasirox Crushed Film Coated Tablets
• Actual Study Start Date: Jan 16, 2018
• Actual Primary Completion Date: Dec 5, 2019
• Actual Study Completion Date: Dec 5, 2019

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Deferasirox Crushed deferasirox (ICL670) FCT for oral use daily. Deferasirox FCT dosing was based on subject's weight.	Drug: Deferasirox; Deferosirox was provided in tablet forms of 90, 180 and 360mg. Tablets were crushed in the home environment and administered by sprinkling the full dose on to soft food to be consumed immediately.; Other Names: ICL670

Outcome Measures

Primary Outcome Measures

1. Number of Participants With Selected Gastrointestinal Disorders up to 24 Weeks [Baseline (Week 1 Day 1) up to Week 24, plus 30 day safety follow-up.]

   To assess the safety of crushed deferasirox FCT with respect to selected gastrointestinal (GI) disorders (esophagitis, stomatitis, mouth ulceration, gastric ulcers, haemorrhage, abdominal pain, diarrhea, nausea, and vomiting). Only descriptive analysis performed.

Secondary Outcome Measures

1. Adverse Events Profile [Baseline (Week 1 Day 1) up to Week 24, plus 30 day safety follow-up.]

   Analysis of frequencies for treatment emergent Adverse Event (TEAEs), Serious Adverse Event TEAEs and Deaths due to AEs, through the monitoring of relevant clinical and laboratory safety parameters. Only descriptive analysis performed.

2. Number of Participants With Notable Changes in ECG Values From Baseline [Baseline (Week 1 Day 1) up to Week 24, plus 30 day safety follow-up.]

   Safety measured by the notable post-baseline changes in ECG values (PR, QRS, QT, QTcF and HR intervals) compared to baseline. Baseline was defined as the last non-missing value on or prior to the first dose. Only descriptive analysis performed.

3. Absolute Change From Baseline in Serum Ferritin (SF) [Baseline (BL), Week 4, Week 8, Week 12, Week 16, EOT (Week 24)]

   Absolute change from baseline over time in SF values up to 24 weeks of treatment were to be provided. Only descriptive analysis performed.

4. Number of Participants With Worst Post-baseline Values in Selected Chemistry Parameters [Baseline (Week 1 Day 1) up to Week 24, plus 30 day safety follow-up.]

   Safety measured by the worst post-baseline severity grade observed in a patient calculated using the normal/low/high classifications based on local laboratory normal ranges, regardless of the baseline status. Baseline was defined as the last non-missing value on or prior to the first dose. The selected chemistry parameters were: Alanine aminotransferase (ALT), Alkaline phosphatase (ALP), Aspartate aminotransferase (AST), total bilirubin, direct bilirubin, serum creatinine and Urine protein creatinine ratio (UPCR) (Protein/Creatinine represented UPCR). Only descriptive analysis performed.

5. Number of Participants With Clinically Significant Auditory Assessments Changes From Baseline [Baseline (Week 1 Day 1) up to Week 24, plus 30 day safety follow-up.]

   Safety measured by notable post-baseline changes in Auditory assessments (comprehensive audiometry threshold examination and speech recognition). Baseline was defined as the last non-missing value on or prior to the first dose. Only descriptive analysis performed.

6. Number of Participants With Clinically Significant Ocular Assessments Changes From Baseline [Baseline (Week 1 Day 1) up to Week 24, plus 30 day safety follow-up.]

   Safety measured by notable post-baseline changes in Ocular assessments (Distance visual acuity test, Applanation tonometry, lens photography, wide angle fundus photography of the retina and optic nerve). Ocular assessment were required at screening and end of Treatment; during treatment, they were to be performed at the discretion of the investigator based on patient reporting symptoms. Baseline was defined as the last non-missing value on or prior to the first dose. Only descriptive analysis performed.

7. Absolute Change From Baseline in Systolic and Diastolic Blood Pressures (mmHg) [Baseline (BL), Week 2, Week 3, Week 4, Week 8, Week 12, Week 16, Week 20, EOT (Week 24)]

   Absolute change from baseline over time in systolic and diastolic blood pressures measurements were to be provided. Only descriptive analysis performed.

8. Absolute Change From Baseline in Pulse Rate (Bpm) [Baseline (BL), Week 2, Week 3, Week 4, Week 8, Week 12, Week 16, Week 20, EOT (Week 24)]

   Absolute change from baseline over time in supine pulse rate was to be provided. Only descriptive analysis performed.

9. Absolute Change From Baseline in Body Temperature (°C) [Baseline (BL), Week 2, Week 3, Week 4, Week 8, Week 12, Week 16, Week 20, EOT (Week 24)]

   Absolute change from baseline over time in body temperature measurements was to be provided. Only descriptive analysis performed.

10. Absolute Change From Baseline in Body Weight (kg) [Baseline (BL), Week 4, Week 8, Week 12, Week 16, Week 20, EOT (Week 24)]

    Absolute change from baseline over time in body weight measurements was to be provided. Only descriptive analysis performed.

11. Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Participants Pre-treated With Deferasirox: Mean Change From Baseline in Adherence [Week 4, Week 12, EOT (Week 24)]

    The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT adherence domain consisted of 6 items from the child's perspective and 6 items from the caregiver's perspective, each with a possible score of 1 to 5, for an overall possible score range of 6 to 30. A higher score indicates poorer adherence. Only descriptive analysis performed.

12. Modified SICT in Participants Pre-treated With Deferasirox: Number of Participants With Type of Medicine Child Like Scoring [Baseline (BL), Week 4, Week 12, EOT (Week 24)]

    The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT preference domain consisted of 3 items including the type of medicine the child said he/she liked best (tablet to dissolve in liquid, tablet (taken once a day), tablet (taken 3 times a day), tablet crushed, sprinkle powder on food, injection and I don't know). These items were presented descriptively using frequency counts.

13. Modified SICT in Participants Pre-treated With Deferasirox: Number of Participants With Reasons Child Preferred Crushed Medicine Scoring [Baseline (BL), Week 4, Week 12, EOT (Week 24)]

    The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT preference domain consisted of 3 items including the reason child preferred crushed medicine (taste, aftertaste, convenience, number of pills, no/less side effects, can correctly prepare the medicine, easier to remember to take the medicine, number of times he/she has to take the medicine, no/less pain on the injection site, gain personal time with their family and friends, and other). These items were presented descriptively using frequency counts.

14. Modified SICT in Participants Pre-treated With Deferasirox: Number of Participants With Rank Based on Child's Preference Scoring [Baseline (BL), Week 4, Week 12, EOT (Week 24)]

    The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT preference domain consisted of 3 items including the rank of the medicine (tablet to dissolve in liquid, tablet taken once a day, tablet taken 3 times a day, tablet crushed, sprinkle powder on food and injection), with a range of 1 to 6 (1 being most preferred and 6 being least preferred), based on what the child prefers. These items were presented descriptively using frequency count.

15. Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Participants Pre-treated With Deferasirox: Mean Change From Baseline in Concerns [Week 4, Week 12, EOT (Week 24)]

    The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT concerns domain scale for child's response had a possible range from 2 to 10, based on two questions and the mSICT concerns domain scale for caregiver's responses had the possible range of 1 to 5 based on one question. A higher score indicated fewer concerns. Only descriptive analysis performed.

16. Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Chelation Naive Participants: Mean Change From Baseline in Adherence [Week 4, Week 12, EOT (Week 24)]

    The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT adherence domain consisted of 6 items from the child's perspective and 6 items from the caregiver's perspective, each with a possible score of 1 to 5, for an overall possible score range of 6 to 30. A higher score indicates poorer adherence. Only descriptive analysis performed.

17. Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Chelation Naive Participants: Number of Participants With Type of Medicine Child Like Scoring [Week 4, Week 12, EOT (Week 24)]

    The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT preference domain consisted of 3 items including the type of medicine the child said he/she liked best (tablet to dissolve in liquid, tablet (taken once a day), tablet (taken 3 times a day), tablet crushed, sprinkle powder on food, injection and I don't know). These items were presented descriptively using frequency counts.

18. Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Chelation Naive Participants: Number of Participants With Reasons Child Preferred Crushed Medicine Scoring [Week 4, Week 12, EOT (Week 24)]

    The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT preference domain consisted of 3 items including the reason child preferred crushed medicine (taste, aftertaste, convenience, number of pills, no/less side effects, can correctly prepare the medicine, easier to remember to take the medicine, number of times he/she has to take the medicine, no/less pain on the injection site, gain personal time with their family and friends, and other). These items were presented descriptively using frequency counts.

19. Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Chelation Naive Participants: Number of Participants With Rank Based on Child's Preference Scoring [Week 4, Week 12, EOT (Week 24)]

    The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT preference domain consisted of 3 items including the rank of the medicine (tablet to dissolve in liquid, tablet taken once a day, tablet taken 3 times a day, tablet crushed, sprinkle powder on food and injection), with a range of 1 to 6 (1 being most preferred and 6 being least preferred), based on what the child prefers. These items were presented descriptively using frequency counts.

20. Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Chelation Naive Participants: Mean Change From Baseline in Concerns [Week 4, Week 12, EOT (Week 24)]

    The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT concerns domain scale for child's response had a possible range from 2 to 10, based on two questions and the mSICT concerns domain scale for caregiver's responses had the possible range of 1 to 5 based on one question. A higher score indicated fewer concerns. Only descriptive analysis performed.

21. Palatability Score in Chelation Naive Participants [Week 4, Week 12, EOT (Week 24)]

    The palatability (taste and ability to consume medicine) questionnaire consisted of 4 items, three items measuring taste or ability to consume medicine and one item measuring aftertaste. The aftertaste item was treated separately. Among the taste items, first one measured taste on a five point response scale. The last two items measured what happened after taking the medicine, i.e., swallowed or vomited etc. and how the perceived amount of liquid taken with the medicine was, enough, not enough or too much. The palatability summary score was calculated from these three items using a scoring matrix and the score ranges from 0 to 11. A higher score indicates better palatability. Only descriptive analysis was performed.

22. Number of Chelation Naive Participants With Palatability After Taste Item Scoring [Week 4, Week 12, EOT (Week 24)]

    The palatability (taste and ability to consume medicine) questionnaire consisted of 4 items, three items measuring taste or ability to consume medicine and one item measuring aftertaste. The aftertaste item was treated as a separate item and scored on a 5-point response scale with the response format Very good = 1 (best), Good = 2, Neither good nor bad = 3, Bad = 4, Very bad = 5 (worst). Only descriptive analysis performed using frequency counts.

23. Palatability Score in Participants Pre-treated With Deferasirox [Week 4, Week 12, EOT (Week 24)]

    The palatability (taste and ability to consume medicine) questionnaire consisted of 4 items, three items measuring taste or ability to consume medicine and one item measuring aftertaste. The aftertaste item was treated separately. Among the taste items, first one measured taste on a five point response scale. The last two items measured what happened after taking the medicine, i.e., swallowed or vomited etc. and how the perceived amount of liquid taken with the medicine was, enough, not enough or too much. The palatability summary score was calculated from these three items using a scoring matrix and the score ranges from 0 to 11. A higher score indicates better palatability. Only descriptive analysis was performed.

24. Number of Participants Pre-treated With Deferasirox With Palatability After Taste Item Scoring [Baseline, Week 4, Week 12, EOT (Week 24)]

    The palatability (taste and ability to consume medicine) questionnaire consisted of 4 items, three items measuring taste or ability to consume medicine and one item measuring aftertaste. The aftertaste item was treated as a separate item and scored on a 5-point response scale with the response format Very good = 1 (best), Good = 2, Neither good nor bad = 3, Bad = 4, Very bad = 5 (worst). Only descriptive analysis performed using frequency counts.

25. GI Symptom Score in Chelation Naive Participants [Week 2, Week 3, Week 4, Week 8, Week 12, Week 16, Week 20, EOT (Week 24)]

    The GI symptom score was calculated from responses to 5 questions, 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. GI symptom scores were summarized using descriptive statistics at week 2, week 3, week 4, week 8, week 12, week 16, week 20 and EOT.

26. Number of Participants With GI Bowel Movements Item Scoring in Chelation Naive Participants [Week 2, Week 3, Week 4, Week 8, Week 12, Week 16, Week 20, EOT (Week 24)]

    The GI symptom questionnaire consisted of 6 items, 5 of which were scored using a 1-5 rating scale. The sixth item assessed bowel movement frequency during the past week, using 7 response options 0 = 0 ("None"), 1 = 1, 2 = 2, 3 = 3, 4 = 4, 5 = "5 - 10" and 6 = "11 or more". The GI bowel movements item score was presented descriptively using frequency counts.

27. GI Symptom Score in Participants Pre-treated With Deferasirox [Week 2, Week 3, Week 4, Week 8, Week 12, Week 16, Week 20, EOT (Week 24)]

    The GI symptom score was calculated from responses to 5 questions, 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. GI symptom scores were summarized using descriptive statistics at week 2, week 3, week 4, week 8, week 12, week 16, week 20 and EOT.

28. Number of Participants With GI Bowel Movements Item Scoring in Participants Pre-treated With Deferasirox [Baseline, Week 2, Week 3, Week 4, Week 8, Week 12, Week 16, Week 20, EOT (Week 24)]

    The GI symptom questionnaire consisted of 6 items, 5 of which were scored using a 1-5 rating scale. The sixth item assessed bowel movement frequency during the past week, using 7 response options 0 = 0 ("None"), 1 = 1, 2 = 2, 3 = 3, 4 = 4, 5 = "5 - 10" and 6 = "11 or more". The GI bowel movements item score was presented descriptively using frequency counts.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 2 Years to 6 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Key Inclusion Criteria:

1. Patients ≥2 to <6 years old diagnosed with transfusional hemosiderosis

2. Documented history of red blood cell transfusions

3. Written informed consent/assent before any study-specific procedures. The consent will be obtained from caregiver(s) or patient's legal representative. Investigators will also obtain assent of patients according to local, regional, or national regulations.

4. For patients on prior DFX: Serum ferritin (SF) >500 ng/mL, measured at screening visit 1 and requiring a DFX daily dose equivalent to FCT ≥ 7mg/kg/day.

5. For patients on a prior chelator other than DFX (e.g. deferiprone or deferoxamine) or chelation naive: Serum ferritin (SF) >1000 ng/mL measured at screening visits 1 and 2.

Key Exclusion Criteria:

1. Patients that receive more than one iron chelator at the same time as current iron chelation treatment. (Patients who have received combination therapy in their medical history but are currently being treated with a single ICT agent are eligible.)

2. Patients continuing on deferoxamine or deferiprone in addition to study treatment. (Patients switching to or continuing on deferasirox are eligible).

3. Unresolved adverse events if the patient was previously treated with deferiprone or deferoxamine or deferasirox.

4. Significant proteinuria as indicated by a urinary protein/creatinine ratio > 0.5 mg/mg in a non-first void sample urine measured at screening visit 1.

5. Serum creatinine > age adjusted ULN measured at any screening visit

6. Creatinine clearance below 90 mL/minute measured at any screening visit. Creatinine clearance using the Schwartz formula will be estimated from serum creatinine measured at each respective visit.

7. ALT and/or AST > 2.5 x ULN measured at screening visit 1.

8. Total bilirubin (TBIL) >1.5 x ULN measured at screening visit 1.

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

10. History of and/or laboratory evidence of active Hepatitis B or Hepatitis C (HBsAg in the absence of HBsAb OR HCV Ab positive with HCV RNA positive.

11. Liver disease with severity of Child-Pugh Class B or C.

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

13. Patients participating in another clinical trial or receiving an investigational drug.

14. Patients with a known history of HIV seropositivity.

15. Patients unwilling or unable to comply with the protocol.

16. 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.

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

18. Female patients who reach menarche and they or their caregivers refuse pregnancy testing and/or if there is a positive pregnancy test result.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Novartis Investigative Site	Zagazig		Egypt	44519
2	Novartis Investigative Site	Cona	FE	Italy	44100
3	Novartis Investigative Site	Cagliari	ITA	Italy	09121
4	Novartis Investigative Site	Napoli		Italy	80138
5	Novartis Investigative Site	Hazmiyeh	Beirut	Lebanon	PO BOX 213
6	Novartis Investigative Site	Muscat		Oman	123
7	Novartis Investigative Site	Bangkok noi	Bangkok	Thailand	10700
8	Novartis Investigative Site	Muang	Chiangmai	Thailand	50200
9	Novartis Investigative Site	Dubai		United Arab Emirates	9115
10	Novartis Investigative Site	London		United Kingdom	NW1 2BU

Sponsors and Collaborators

• Novartis Pharmaceuticals

Investigators

• Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Study Documents (Full-Text)

• Study Protocol - Apr 25, 2018
• Statistical Analysis Plan - Dec 16, 2019

More Information

Publications

None provided.

• Responsible Party: Novartis Pharmaceuticals
• ClinicalTrials.gov Identifier: NCT03372083
• Other Study ID Numbers: CICL670F2429; 2016-003482-25
• First Posted: Dec 13, 2017
• Last Update Posted: Aug 25, 2020
• Last Verified: Aug 1, 2020

• 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: Iron Chelation Therapy; Deferasirox; Asunra; Jadenu; Exjade; ICL 670; Pediatric
• Additional relevant MeSH terms: Hemochromatosis; Iron Overload; Hemosiderosis; Deferasirox

Study Results

Participant Flow

Recruitment Details	The study was conducted in 10 sites in 7 countries (one in Egypt, three in Italy, one in Lebanon, one in Oman, two in Thailand, one in the United Arab Emirates, and one in the United Kingdom).

Arm/Group Title	Deferasirox
Arm/Group Description	Crushed deferasirox (ICL670) FCT for oral use daily. Deferasirox FCT dosing was based on subject's weight.
Period Title: Overall Study
STARTED	44
COMPLETED	39
NOT COMPLETED	5

Baseline Characteristics

Arm/Group Title	Deferasirox
Arm/Group Description	Crushed deferasirox (ICL670) FCT for oral use daily. Deferasirox FCT dosing was based on subject's weight.
Overall Participants	44
Age (Years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [Years]	3.05 (1.056)
Sex: Female, Male (Count of Participants)
Female	17 38.6%
Male	27 61.4%
Race/Ethnicity, Customized (Number) [Number]
Caucasian	36 81.8%
Asian	8 18.2%
Baseline Body Mass Index (kilogram per square metre (kg/m^2)) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [kilogram per square metre (kg/m^2)]	16.40 (2.009)
Baseline iron chelation naive participants (Count of Participants)
Chelation naive = Yes	10 22.7%
Chelation naive = No	34 77.3%

Outcome Measures

1. Primary Outcome

Title	Number of Participants With Selected Gastrointestinal Disorders up to 24 Weeks
Description	To assess the safety of crushed deferasirox FCT with respect to selected gastrointestinal (GI) disorders (esophagitis, stomatitis, mouth ulceration, gastric ulcers, haemorrhage, abdominal pain, diarrhea, nausea, and vomiting). Only descriptive analysis performed.
Time Frame	Baseline (Week 1 Day 1) up to Week 24, plus 30 day safety follow-up.

Outcome Measure Data

Analysis Population Description

Safety set

Arm/Group Title	Deferasirox
Arm/Group Description	Crushed deferasirox (ICL670) FCT for oral use daily. Deferasirox FCT dosing was based on subject's weight.
Measure Participants	44
Oesophagitis	0 0%
Barrett's esophagitis	0 0%
Stomatitis	0 0%
Mouth ulceration	0 0%
Gastric ulcer	0 0%
Gastrointestinal haemorrhage	0 0%
Abdominal pain	2 4.5%
Diarrhoea	4 9.1%
Nausea	0 0%
Vomiting	2 4.5%

2. Secondary Outcome

Title	Adverse Events Profile
Description	Analysis of frequencies for treatment emergent Adverse Event (TEAEs), Serious Adverse Event TEAEs and Deaths due to AEs, through the monitoring of relevant clinical and laboratory safety parameters. Only descriptive analysis performed.
Time Frame	Baseline (Week 1 Day 1) up to Week 24, plus 30 day safety follow-up.

Outcome Measure Data

Analysis Population Description

Safety set

Arm/Group Title	Deferasirox
Arm/Group Description	Crushed deferasirox (ICL670) FCT for oral use daily. Deferasirox FCT dosing was based on subject's weight.
Measure Participants	44
On treatment-related AE	22 50%
On treatment related SAE	0 0%
On treatment Deaths	0 0%

3. Secondary Outcome

Title	Number of Participants With Notable Changes in ECG Values From Baseline
Description	Safety measured by the notable post-baseline changes in ECG values (PR, QRS, QT, QTcF and HR intervals) compared to baseline. Baseline was defined as the last non-missing value on or prior to the first dose. Only descriptive analysis performed.
Time Frame	Baseline (Week 1 Day 1) up to Week 24, plus 30 day safety follow-up.

Outcome Measure Data

Analysis Population Description

Safety set. For each parameter, only patients with a value at both baseline and post baseline were to be included.

Arm/Group Title	Deferasirox
Arm/Group Description	Crushed deferasirox (ICL670) FCT for oral use daily. Deferasirox FCT dosing was based on subject's weight.
Measure Participants	44
Number [Participants]	0 0%

4. Secondary Outcome

Title	Absolute Change From Baseline in Serum Ferritin (SF)
Description	Absolute change from baseline over time in SF values up to 24 weeks of treatment were to be provided. Only descriptive analysis performed.
Time Frame	Baseline (BL), Week 4, Week 8, Week 12, Week 16, EOT (Week 24)

Outcome Measure Data

Analysis Population Description

Full Analysis Set (FAS)

Arm/Group Title	Deferasirox
Arm/Group Description	Crushed deferasirox (ICL670) FCT for oral use daily. Deferasirox FCT dosing was based on subject's weight.
Measure Participants	44
Baseline (BL)	2152.7 (1039.06)
Change from BL @ Week 4	-118.4 (590.20)
Change from BL @ Week 8	-17.5 (665.68)
Change from BL @ Week 12	-52.8 (790.26)
Change from BL @ Week 16	71.9 (988.30)
Change from BL @ Week 20	-64.3 (791.92)
Change from BL @ EOT (Week 24)	-140.7 (824.01)

5. Secondary Outcome

Title	Number of Participants With Worst Post-baseline Values in Selected Chemistry Parameters
Description	Safety measured by the worst post-baseline severity grade observed in a patient calculated using the normal/low/high classifications based on local laboratory normal ranges, regardless of the baseline status. Baseline was defined as the last non-missing value on or prior to the first dose. The selected chemistry parameters were: Alanine aminotransferase (ALT), Alkaline phosphatase (ALP), Aspartate aminotransferase (AST), total bilirubin, direct bilirubin, serum creatinine and Urine protein creatinine ratio (UPCR) (Protein/Creatinine represented UPCR). Only descriptive analysis performed.
Time Frame	Baseline (Week 1 Day 1) up to Week 24, plus 30 day safety follow-up.

Outcome Measure Data

Analysis Population Description

Safety Set

Arm/Group Title	Deferasirox
Arm/Group Description	Crushed deferasirox (ICL670) FCT for oral use daily. Deferasirox FCT dosing was based on subject's weight.
Measure Participants	44
Normal	16 36.4%
Low	0 0%
High	28 63.6%
High & Low	0 0%
Missing	0 0%
Normal	25 56.8%
Low	2 4.5%
High	17 38.6%
High & Low	0 0%
Missing	0 0%
Normal	14 31.8%
Low	0 0%
High	30 68.2%
High & Low	0 0%
Missing	0 0%
Normal	16 36.4%
Low	0 0%
High	28 63.6%
High & Low	0 0%
Missing	0 0%
Normal	6 13.6%
Low	29 65.9%
High	6 13.6%
High & Low	3 6.8%
Missing	0 0%
Normal	16 36.4%
Low	0 0%
High	28 63.6%
High & Low	0 0%
Missing	0 0%
Normal	20 45.5%
Low	1 2.3%
High	17 38.6%
High & Low	6 13.6%
Missing	0 0%

6. Secondary Outcome

Title	Number of Participants With Clinically Significant Auditory Assessments Changes From Baseline
Description	Safety measured by notable post-baseline changes in Auditory assessments (comprehensive audiometry threshold examination and speech recognition). Baseline was defined as the last non-missing value on or prior to the first dose. Only descriptive analysis performed.
Time Frame	Baseline (Week 1 Day 1) up to Week 24, plus 30 day safety follow-up.

Outcome Measure Data

Analysis Population Description

Safety set. For each parameter, only patients with a value at both baseline and post baseline were to be included.

Arm/Group Title	Deferasirox
Arm/Group Description	Crushed deferasirox (ICL670) FCT for oral use daily. Deferasirox FCT dosing was based on subject's weight.
Measure Participants	44
Number [Participants]	0 0%

7. Secondary Outcome

Title	Number of Participants With Clinically Significant Ocular Assessments Changes From Baseline
Description	Safety measured by notable post-baseline changes in Ocular assessments (Distance visual acuity test, Applanation tonometry, lens photography, wide angle fundus photography of the retina and optic nerve). Ocular assessment were required at screening and end of Treatment; during treatment, they were to be performed at the discretion of the investigator based on patient reporting symptoms. Baseline was defined as the last non-missing value on or prior to the first dose. Only descriptive analysis performed.
Time Frame	Baseline (Week 1 Day 1) up to Week 24, plus 30 day safety follow-up.

Outcome Measure Data

Analysis Population Description

Safety set. For each parameter, only patients with a value at both baseline and post baseline were to be included.

Arm/Group Title	Deferasirox
Arm/Group Description	Crushed deferasirox (ICL670) FCT for oral use daily. Deferasirox FCT dosing was based on subject's weight.
Measure Participants	44
Number [Participants]	0 0%

8. Secondary Outcome

Title	Absolute Change From Baseline in Systolic and Diastolic Blood Pressures (mmHg)
Description	Absolute change from baseline over time in systolic and diastolic blood pressures measurements were to be provided. Only descriptive analysis performed.
Time Frame	Baseline (BL), Week 2, Week 3, Week 4, Week 8, Week 12, Week 16, Week 20, EOT (Week 24)

Outcome Measure Data

Analysis Population Description

Safety set. For each parameter, only patients with a value at both baseline and post baseline were to be included.

Arm/Group Title	Deferasirox
Arm/Group Description	Crushed deferasirox (ICL670) FCT for oral use daily. Deferasirox FCT dosing was based on subject's weight.
Measure Participants	44
Systolic BP-Baseline (BL)	100.0 (10.10)
Systolic BP-Change from BL @ Week 2	-0.1 (11.27)
Systolic BP-Change from BL @ Week 3	-2.3 (7.93)
Systolic BP-Change from BL @ Week 4	-0.3 (12.56)
Systolic BP-Change from BL @ Week 8	0.3 (8.53)
Systolic BP-Change from BL @ Week 12	-1.4 (12.16)
Systolic BP-Change from BL @ Week 16	-1.9 (11.76)
Systolic BP-Change from BL @ Week 20	-0.6 (11.67)
Systolic BP-Change from BL @ EOT (Week 24)	-2.6 (11.54)
Diastolic BP-Baseline (BL)	60.0 (7.85)
Diastolic BP-Change from BL @ Week 2	0.7 (6.13)
Diastolic BP-Change from BL @ Week 3	-2.1 (9.60)
Diastolic BP-Change from BL @ Week 4	1.0 (10.24)
Diastolic BP-Change from BL @ Week 8	-0.6 (13.21)
Diastolic BP-Change from BL @ Week 12	1.7 (11.42)
Diastolic BP-Change from BL @ Week 16	-0.5 (10.28)
Diastolic BP-Change from BL @ Week 20	-0.1 (11.70)
Diastolic BP-Change from BL @ EOT (Week 24)	-0.4 (7.89)

9. Secondary Outcome

Title	Absolute Change From Baseline in Pulse Rate (Bpm)
Description	Absolute change from baseline over time in supine pulse rate was to be provided. Only descriptive analysis performed.
Time Frame	Baseline (BL), Week 2, Week 3, Week 4, Week 8, Week 12, Week 16, Week 20, EOT (Week 24)

Outcome Measure Data

Analysis Population Description

Safety set. For each parameter, only patients with a value at both baseline and post baseline were to be included.

Arm/Group Title	Deferasirox
Arm/Group Description	Crushed deferasirox (ICL670) FCT for oral use daily. Deferasirox FCT dosing was based on subject's weight.
Measure Participants	44
Baseline (BL)	102.4 (10.73)
Change from BL @ Week 2	-1.1 (10.51)
Change from BL @ Week 3	-2.7 (16.18)
Change from BL @ Week 4	-3.1 (12.85)
Change from BL @ Week 8	-1.0 (15.47)
Change from BL @ Week 12	-0.3 (12.69)
Change from BL @ Week 16	-2.2 (8.99)
Change from BL @ Week 20	0.8 (12.55)
Change from BL @ EOT (Week 24)	1.1 (12.10)

10. Secondary Outcome

Title	Absolute Change From Baseline in Body Temperature (°C)
Description	Absolute change from baseline over time in body temperature measurements was to be provided. Only descriptive analysis performed.
Time Frame	Baseline (BL), Week 2, Week 3, Week 4, Week 8, Week 12, Week 16, Week 20, EOT (Week 24)

Outcome Measure Data

Analysis Population Description

Safety set. For each parameter, only patients with a value at both baseline and post baseline were to be included.

Arm/Group Title	Deferasirox
Arm/Group Description	Crushed deferasirox (ICL670) FCT for oral use daily. Deferasirox FCT dosing was based on subject's weight.
Measure Participants	44
Baseline (BL)	36.43 (0.416)
Change from BL @ Week 2	-0.03 (0.285)
Change from BL @ Week 3	-0.10 (0.353)
Change from BL @ Week 4	0.00 (0.360)
Change from BL @ Week 8	0.02 (0.336)
Change from BL @ Week 12	-0.03 (0.413)
Change from BL @ Week 16	0.10 (0.400)
Change from BL @ Week 20	0.03 (0.372)
Change from BL @ EOT (Week 24)	0.08 (0.453)

11. Secondary Outcome

Title	Absolute Change From Baseline in Body Weight (kg)
Description	Absolute change from baseline over time in body weight measurements was to be provided. Only descriptive analysis performed.
Time Frame	Baseline (BL), Week 4, Week 8, Week 12, Week 16, Week 20, EOT (Week 24)

Outcome Measure Data

Analysis Population Description

Safety set. For each parameter, only patients with a value at both baseline and post baseline were to be included.

Arm/Group Title	Deferasirox
Arm/Group Description	Crushed deferasirox (ICL670) FCT for oral use daily. Deferasirox FCT dosing was based on subject's weight.
Measure Participants	44
Baseline (BL)	15.26 (3.109)
Change from BL @ Week 4	0.08 (0.290)
Change from BL @ Week 8	0.19 (0.460)
Change from BL @ Week 12	0.31 (0.482)
Change from BL @ Week 16	0.25 (0.433)
Change from BL @ Week 20	0.43 (0.532)
Change from BL @ EOT (Week 24)	0.44 (0.600)

12. Secondary Outcome

Title	Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Participants Pre-treated With Deferasirox: Mean Change From Baseline in Adherence
Description	The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT adherence domain consisted of 6 items from the child's perspective and 6 items from the caregiver's perspective, each with a possible score of 1 to 5, for an overall possible score range of 6 to 30. A higher score indicates poorer adherence. Only descriptive analysis performed.
Time Frame	Week 4, Week 12, EOT (Week 24)

Outcome Measure Data

Analysis Population Description

Full Analysis Set (FAS). Only patients with a value at both baseline and post baseline were to be included.

Arm/Group Title	Deferasirox
Arm/Group Description	Crushed deferasirox (ICL670) FCT for oral use daily. Deferasirox FCT dosing was based on subject's weight.
Measure Participants	32
Child's perspective overall score-Week 4	-2.4
Child's perspective overall score-Week 12	-2.6
Child's perspective overall score-EOT	-1.9
Caregiver's perspective overall score-Week 4	-1.4
Caregiver's perspective overall score-Week 12	-1.4
Caregiver's perspective overall score-EOT	-1.0

13. Secondary Outcome

Title	Modified SICT in Participants Pre-treated With Deferasirox: Number of Participants With Type of Medicine Child Like Scoring
Description	The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT preference domain consisted of 3 items including the type of medicine the child said he/she liked best (tablet to dissolve in liquid, tablet (taken once a day), tablet (taken 3 times a day), tablet crushed, sprinkle powder on food, injection and I don't know). These items were presented descriptively using frequency counts.
Time Frame	Baseline (BL), Week 4, Week 12, EOT (Week 24)

Outcome Measure Data

Analysis Population Description

Full Analysis Set (FAS). Only patients with a value at both baseline and post baseline were to be included.

Arm/Group Title	Deferasirox
Arm/Group Description	Crushed deferasirox (ICL670) FCT for oral use daily. Deferasirox FCT dosing was based on subject's weight.
Measure Participants	32
Tablet to dissolve in liquid	0 0%
Tablet (taken once a day)	4 9.1%
Tablet (taken 3 times a day)	0 0%
Tablet crushed	11 25%
Sprinkle powder on food	14 31.8%
Injection	0 0%
I don't know	3 6.8%
Tablet to dissolve in liquid	0 0%
Tablet (taken once a day)	1 2.3%
Tablet (taken 3 times a day)	0 0%
Tablet crushed	14 31.8%
Sprinkle powder on food	14 31.8%
Injection	0 0%
I don't know	1 2.3%
Tablet to dissolve in liquid	1 2.3%
Tablet (taken once a day)	1 2.3%
Tablet (taken 3 times a day)	0 0%
Tablet crushed	13 29.5%
Sprinkle powder on food	12 27.3%
Injection	0 0%
I don't know	0 0%
Tablet to dissolve in liquid	0 0%
Tablet (taken once a day)	4 9.1%
Tablet (taken 3 times a day)	0 0%
Tablet crushed	9 20.5%
Sprinkle powder on food	15 34.1%
Injection	0 0%
I don't know	0 0%

14. Secondary Outcome

Title	Modified SICT in Participants Pre-treated With Deferasirox: Number of Participants With Reasons Child Preferred Crushed Medicine Scoring
Description	The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT preference domain consisted of 3 items including the reason child preferred crushed medicine (taste, aftertaste, convenience, number of pills, no/less side effects, can correctly prepare the medicine, easier to remember to take the medicine, number of times he/she has to take the medicine, no/less pain on the injection site, gain personal time with their family and friends, and other). These items were presented descriptively using frequency counts.
Time Frame	Baseline (BL), Week 4, Week 12, EOT (Week 24)

Outcome Measure Data

Analysis Population Description

Full Analysis Set (FAS). Only patients with a value at both baseline and post baseline were to be included.

Arm/Group Title	Deferasirox
Arm/Group Description	Crushed deferasirox (ICL670) FCT for oral use daily. Deferasirox FCT dosing was based on subject's weight.
Measure Participants	32
Taste	6 13.6%
Aftertaste	5 11.4%
Convenience	5 11.4%
Number of pills	0 0%
No/Less side effects	0 0%
Can correctly prepare the medicine	0 0%
Easier to remember to take the medicine	1 2.3%
Number of times he/she has to take the medicine	0 0%
No/Less pain on the injection site	0 0%
Gain personal time with their family and friends	2 4.5%
Other	1 2.3%
Taste	0 0%
Aftertaste	0 0%
Convenience	4 9.1%
Number of pills	0 0%
No/Less side effects	0 0%
Can correctly prepare the medicine	0 0%
Easier to remember to take the medicine	0 0%
Number of times he/she has to take the medicine	0 0%
No/Less pain on the injection site	0 0%
Gain personal time with their family and friends	2 4.5%
Other	8 18.2%
Taste	0 0%
Aftertaste	0 0%
Convenience	2 4.5%
Number of pills	0 0%
No/Less side effects	0 0%
Can correctly prepare the medicine	0 0%
Easier to remember to take the medicine	0 0%
Number of times he/she has to take the medicine	0 0%
No/Less pain on the injection site	0 0%
Gain personal time with their family and friends	3 6.8%
Other	8 18.2%
Taste	0 0%
Aftertaste	0 0%
Convenience	0 0%
Number of pills	0 0%
No/Less side effects	0 0%
Can correctly prepare the medicine	0 0%
Easier to remember to take the medicine	0 0%
Number of times he/she has to take the medicine	0 0%
No/Less pain on the injection site	0 0%
Gain personal time with their family and friends	7 15.9%
Other	3 6.8%

15. Secondary Outcome

Title	Modified SICT in Participants Pre-treated With Deferasirox: Number of Participants With Rank Based on Child's Preference Scoring
Description	The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT preference domain consisted of 3 items including the rank of the medicine (tablet to dissolve in liquid, tablet taken once a day, tablet taken 3 times a day, tablet crushed, sprinkle powder on food and injection), with a range of 1 to 6 (1 being most preferred and 6 being least preferred), based on what the child prefers. These items were presented descriptively using frequency count.
Time Frame	Baseline (BL), Week 4, Week 12, EOT (Week 24)

Outcome Measure Data

Analysis Population Description

Full Analysis Set (FAS). Only patients with a value at both baseline and post baseline were to be included.

Arm/Group Title	Deferasirox
Arm/Group Description	Crushed deferasirox (ICL670) FCT for oral use daily. Deferasirox FCT dosing was based on subject's weight.
Measure Participants	32
1	0 0%
2	1 2.3%
3	12 27.3%
4	7 15.9%
5	11 25%
6	1 2.3%
1	0 0%
2	11 25%
3	8 18.2%
4	3 6.8%
5	5 11.4%
6	3 6.8%
1	1 2.3%
2	7 15.9%
3	7 15.9%
4	4 9.1%
5	7 15.9%
6	0 0%
1	0 0%
2	8 18.2%
3	8 18.2%
4	7 15.9%
5	4 9.1%
6	1 2.3%
1	6 13.6%
2	3 6.8%
3	15 34.1%
4	6 13.6%
5	2 4.5%
6	0 0%
1	3 6.8%
2	0 0%
3	9 20.5%
4	18 40.9%
5	0 0%
6	0 0%
1	1 2.3%
2	0 0%
3	11 25%
4	13 29.5%
5	1 2.3%
6	0 0%
1	4 9.1%
2	0 0%
3	11 25%
4	13 29.5%
5	0 0%
6	0 0%
1	0 0%
2	0 0%
3	0 0%
4	14 31.8%
5	17 38.6%
6	1 2.3%
1	0 0%
2	1 2.3%
3	1 2.3%
4	6 13.6%
5	21 47.7%
6	1 2.3%
1	0 0%
2	2 4.5%
3	1 2.3%
4	6 13.6%
5	17 38.6%
6	0 0%
1	0 0%
2	1 2.3%
3	1 2.3%
4	3 6.8%
5	23 52.3%
6	0 0%
1	12 27.3%
2	14 31.8%
3	3 6.8%
4	3 6.8%
5	0 0%
6	0 0%
1	10 22.7%
2	9 20.5%
3	11 25%
4	0 0%
5	0 0%
6	0 0%
1	8 18.2%
2	9 20.5%
3	6 13.6%
4	2 4.5%
5	0 0%
6	1 2.3%
1	8 18.2%
2	9 20.5%
3	8 18.2%
4	2 4.5%
5	1 2.3%
6	0 0%
1	16 36.4%
2	13 29.5%
3	1 2.3%
4	1 2.3%
5	1 2.3%
6	0 0%
1	19 43.2%
2	8 18.2%
3	0 0%
4	1 2.3%
5	2 4.5%
6	0 0%
1	15 34.1%
2	8 18.2%
3	1 2.3%
4	1 2.3%
5	1 2.3%
6	0 0%
1	16 36.4%
2	10 22.7%
3	0 0%
4	2 4.5%
5	0 0%
6	0 0%
1	0 0%
2	0 0%
3	0 0%
4	0 0%
5	0 0%
6	32 72.7%
1	0 0%
2	0 0%
3	0 0%
4	1 2.3%
5	1 2.3%
6	28 63.6%
1	1 2.3%
2	0 0%
3	0 0%
4	0 0%
5	0 0%
6	25 56.8%
1	0 0%
2	0 0%
3	0 0%
4	1 2.3%
5	0 0%
6	27 61.4%

16. Secondary Outcome

Title	Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Participants Pre-treated With Deferasirox: Mean Change From Baseline in Concerns
Description	The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT concerns domain scale for child's response had a possible range from 2 to 10, based on two questions and the mSICT concerns domain scale for caregiver's responses had the possible range of 1 to 5 based on one question. A higher score indicated fewer concerns. Only descriptive analysis performed.
Time Frame	Week 4, Week 12, EOT (Week 24)

Outcome Measure Data

Analysis Population Description

Full Analysis Set (FAS). Only patients with a value at both baseline and post baseline were to be included.

Arm/Group Title	Deferasirox
Arm/Group Description	Crushed deferasirox (ICL670) FCT for oral use daily. Deferasirox FCT dosing was based on subject's weight.
Measure Participants	32
Child's perspective overall score-Week 4	1.1
Child's perspective overall score-Week 12	1.2
Child's perspective overall score-EOT	0.8
Caregiver's perspective overall score-Week 4	0.4
Caregiver's perspective overall score-Week 12	0.4
Caregiver's perspective overall score-EOT	0.2

17. Secondary Outcome

Title	Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Chelation Naive Participants: Mean Change From Baseline in Adherence
Description	The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT adherence domain consisted of 6 items from the child's perspective and 6 items from the caregiver's perspective, each with a possible score of 1 to 5, for an overall possible score range of 6 to 30. A higher score indicates poorer adherence. Only descriptive analysis performed.
Time Frame	Week 4, Week 12, EOT (Week 24)

Outcome Measure Data

Analysis Population Description

Full Analysis Set (FAS). Only patients with a value at both baseline and post baseline were to be included.

Arm/Group Title	Deferasirox
Arm/Group Description	Crushed deferasirox (ICL670) FCT for oral use daily. Deferasirox FCT dosing was based on subject's weight.
Measure Participants	12
Child's perspective overall score-Week 4	10.4 (2.98)
Child's perspective overall score-Week 12	9.8 (2.99)
Child's perspective overall score-EOT	10.3 (3.43)
Caregiver's perspective overall score-Week 4	9.0 (3.97)
Caregiver's perspective overall score-Week 12	9.9 (5.30)
Caregiver's perspective overall score-EOT	10.6 (5.03)

18. Secondary Outcome

Title	Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Chelation Naive Participants: Number of Participants With Type of Medicine Child Like Scoring
Description	The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT preference domain consisted of 3 items including the type of medicine the child said he/she liked best (tablet to dissolve in liquid, tablet (taken once a day), tablet (taken 3 times a day), tablet crushed, sprinkle powder on food, injection and I don't know). These items were presented descriptively using frequency counts.
Time Frame	Week 4, Week 12, EOT (Week 24)

Outcome Measure Data

Analysis Population Description

Full Analysis Set (FAS). Only patients with a value at both baseline and post baseline were to be included.

Arm/Group Title	Deferasirox
Arm/Group Description	Crushed deferasirox (ICL670) FCT for oral use daily. Deferasirox FCT dosing was based on subject's weight.
Measure Participants	12
Tablet to dissolve in liquid	0 0%
Tablet (taken once a day)	1 2.3%
Tablet (taken 3 times a day)	0 0%
Tablet crushed	5 11.4%
Sprinkle powder on food	3 6.8%
Injection	0 0%
I don't know	2 4.5%
Tablet to dissolve in liquid	1 2.3%
Tablet (taken once a day)	0 0%
Tablet (taken 3 times a day)	0 0%
Tablet crushed	5 11.4%
Sprinkle powder on food	3 6.8%
Injection	0 0%
I don't know	0 0%
Tablet to dissolve in liquid	4 9.1%
Tablet (taken once a day)	0 0%
Tablet (taken 3 times a day)	0 0%
Tablet crushed	3 6.8%
Sprinkle powder on food	2 4.5%
Injection	0 0%
I don't know	0 0%

19. Secondary Outcome

Title	Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Chelation Naive Participants: Number of Participants With Reasons Child Preferred Crushed Medicine Scoring
Description	The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT preference domain consisted of 3 items including the reason child preferred crushed medicine (taste, aftertaste, convenience, number of pills, no/less side effects, can correctly prepare the medicine, easier to remember to take the medicine, number of times he/she has to take the medicine, no/less pain on the injection site, gain personal time with their family and friends, and other). These items were presented descriptively using frequency counts.
Time Frame	Week 4, Week 12, EOT (Week 24)

Outcome Measure Data

Analysis Population Description

Full Analysis Set (FAS). Only patients with a value at both baseline and post baseline were to be included.

Arm/Group Title	Deferasirox
Arm/Group Description	Crushed deferasirox (ICL670) FCT for oral use daily. Deferasirox FCT dosing was based on subject's weight.
Measure Participants	12
Taste	0 0%
Aftertaste	1 2.3%
Convenience	2 4.5%
Number of pills	1 2.3%
No/Less side effects	0 0%
Can correctly prepare the medicine	1 2.3%
Easier to remember to take the medicine	1 2.3%
Number of times he/she has to take the medicine	1 2.3%
No/Less pain on the injection site	0 0%
Gain personal time with their family and friends	1 2.3%
Other	1 2.3%
Taste	0 0%
Aftertaste	0 0%
Convenience	2 4.5%
Number of pills	0 0%
No/Less side effects	0 0%
Can correctly prepare the medicine	0 0%
Easier to remember to take the medicine	0 0%
Number of times he/she has to take the medicine	0 0%
No/Less pain on the injection site	1 2.3%
Gain personal time with their family and friends	2 4.5%
Other	1 2.3%
Taste	0 0%
Aftertaste	0 0%
Convenience	1 2.3%
Number of pills	0 0%
No/Less side effects	1 2.3%
Can correctly prepare the medicine	1 2.3%
Easier to remember to take the medicine	2 4.5%
Number of times he/she has to take the medicine	0 0%
No/Less pain on the injection site	1 2.3%
Gain personal time with their family and friends	2 4.5%
Other	0 0%

20. Secondary Outcome

Title	Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Chelation Naive Participants: Number of Participants With Rank Based on Child's Preference Scoring
Description	The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT preference domain consisted of 3 items including the rank of the medicine (tablet to dissolve in liquid, tablet taken once a day, tablet taken 3 times a day, tablet crushed, sprinkle powder on food and injection), with a range of 1 to 6 (1 being most preferred and 6 being least preferred), based on what the child prefers. These items were presented descriptively using frequency counts.
Time Frame	Week 4, Week 12, EOT (Week 24)

Outcome Measure Data

Analysis Population Description

Full Analysis Set (FAS). Only patients with a value at both baseline and post baseline were to be included.

Arm/Group Title	Deferasirox
Arm/Group Description	Crushed deferasirox (ICL670) FCT for oral use daily. Deferasirox FCT dosing was based on subject's weight.
Measure Participants	12
1	2 4.5%
2	1 2.3%
3	2 4.5%
4	4 9.1%
5	1 2.3%
6	0 0%
1	2 4.5%
2	3 6.8%
3	2 4.5%
4	0 0%
5	2 4.5%
6	0 0%
1	4 9.1%
2	2 4.5%
3	1 2.3%
4	2 4.5%
5	0 0%
6	0 0%
1	1 2.3%
2	3 6.8%
3	5 11.4%
4	1 2.3%
5	0 0%
6	0 0%
1	0 0%
2	0 0%
3	2 4.5%
4	6 13.6%
5	1 2.3%
6	0 0%
1	0 0%
2	1 2.3%
3	3 6.8%
4	5 11.4%
5	0 0%
6	0 0%
1	1 2.3%
2	0 0%
3	1 2.3%
4	3 6.8%
5	4 9.1%
6	1 2.3%
1	0 0%
2	1 2.3%
3	2 4.5%
4	1 2.3%
5	5 11.4%
6	0 0%
1	0 0%
2	0 0%
3	0 0%
4	0 0%
5	9 20.5%
6	0 0%
1	1 2.3%
2	6 13.6%
3	1 2.3%
4	1 2.3%
5	1 2.3%
6	0 0%
1	1 2.3%
2	5 11.4%
3	2 4.5%
4	0 0%
5	0 0%
6	1 2.3%
1	1 2.3%
2	4 9.1%
3	4 9.1%
4	0 0%
5	0 0%
6	0 0%
1	5 11.4%
2	0 0%
3	0 0%
4	1 2.3%
5	3 6.8%
6	1 2.3%
1	5 11.4%
2	0 0%
3	1 2.3%
4	2 4.5%
5	1 2.3%
6	0 0%
1	4 9.1%
2	2 4.5%
3	1 2.3%
4	2 4.5%
5	0 0%
6	0 0%
1	0 0%
2	0 0%
3	1 2.3%
4	0 0%
5	1 2.3%
6	8 18.2%
1	1 2.3%
2	0 0%
3	0 0%
4	0 0%
5	0 0%
6	8 18.2%
1	0 0%
2	0 0%
3	0 0%
4	0 0%
5	0 0%
6	9 20.5%

21. Secondary Outcome

Title	Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Chelation Naive Participants: Mean Change From Baseline in Concerns
Description	The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT concerns domain scale for child's response had a possible range from 2 to 10, based on two questions and the mSICT concerns domain scale for caregiver's responses had the possible range of 1 to 5 based on one question. A higher score indicated fewer concerns. Only descriptive analysis performed.
Time Frame	Week 4, Week 12, EOT (Week 24)

Outcome Measure Data

Analysis Population Description

Full Analysis Set (FAS). Only patients with a value at both baseline and post baseline were to be included.

Arm/Group Title	Deferasirox
Arm/Group Description	Crushed deferasirox (ICL670) FCT for oral use daily. Deferasirox FCT dosing was based on subject's weight.
Measure Participants	12
Child's perspective overall score-Week 4	9.7 (0.65)
Child's perspective overall score-Week 12	9.4 (0.88)
Child's perspective overall score-EOT	8.9 (1.36)
Caregiver's perspective overall score-Week 4	3.7 (1.42)
Caregiver's perspective overall score-Week 12	3.9 (1.76)
Caregiver's perspective overall score-EOT	3.4 (1.59)

22. Secondary Outcome

Title	Palatability Score in Chelation Naive Participants
Description	The palatability (taste and ability to consume medicine) questionnaire consisted of 4 items, three items measuring taste or ability to consume medicine and one item measuring aftertaste. The aftertaste item was treated separately. Among the taste items, first one measured taste on a five point response scale. The last two items measured what happened after taking the medicine, i.e., swallowed or vomited etc. and how the perceived amount of liquid taken with the medicine was, enough, not enough or too much. The palatability summary score was calculated from these three items using a scoring matrix and the score ranges from 0 to 11. A higher score indicates better palatability. Only descriptive analysis was performed.
Time Frame	Week 4, Week 12, EOT (Week 24)

Outcome Measure Data

Analysis Population Description

Full Analysis Set (FAS). Only patients with a value at both baseline and post baseline were to be included.

Arm/Group Title	Deferasirox
Arm/Group Description	Crushed deferasirox (ICL670) FCT for oral use daily. Deferasirox FCT dosing was based on subject's weight.
Measure Participants	12
Week 4	10.4 (0.97)
Week 12	10.9 (0.33)
EOT (Week 24)	10.8 (0.67)

23. Secondary Outcome

Title	Number of Chelation Naive Participants With Palatability After Taste Item Scoring
Description	The palatability (taste and ability to consume medicine) questionnaire consisted of 4 items, three items measuring taste or ability to consume medicine and one item measuring aftertaste. The aftertaste item was treated as a separate item and scored on a 5-point response scale with the response format Very good = 1 (best), Good = 2, Neither good nor bad = 3, Bad = 4, Very bad = 5 (worst). Only descriptive analysis performed using frequency counts.
Time Frame	Week 4, Week 12, EOT (Week 24)

Outcome Measure Data

Analysis Population Description

Full Analysis Set (FAS). Only patients with a value at both baseline and post baseline were to be included.

Arm/Group Title	Deferasirox
Arm/Group Description	Crushed deferasirox (ICL670) FCT for oral use daily. Deferasirox FCT dosing was based on subject's weight.
Measure Participants	12
Very Good	3 6.8%
Good	4 9.1%
Neither good nor bad	3 6.8%
Bad	1 2.3%
Very bad	0 0%
Very Good	2 4.5%
Good	5 11.4%
Neither good nor bad	2 4.5%
Bad	0 0%
Very bad	0 0%
Very Good	1 2.3%
Good	4 9.1%
Neither good nor bad	3 6.8%
Bad	1 2.3%
Very bad	0 0%

24. Secondary Outcome

Title	Palatability Score in Participants Pre-treated With Deferasirox
Description	The palatability (taste and ability to consume medicine) questionnaire consisted of 4 items, three items measuring taste or ability to consume medicine and one item measuring aftertaste. The aftertaste item was treated separately. Among the taste items, first one measured taste on a five point response scale. The last two items measured what happened after taking the medicine, i.e., swallowed or vomited etc. and how the perceived amount of liquid taken with the medicine was, enough, not enough or too much. The palatability summary score was calculated from these three items using a scoring matrix and the score ranges from 0 to 11. A higher score indicates better palatability. Only descriptive analysis was performed.
Time Frame	Week 4, Week 12, EOT (Week 24)

Outcome Measure Data

Analysis Population Description

Full Analysis Set (FAS). Only patients with a value at both baseline and post baseline were to be included.

Arm/Group Title	Deferasirox
Arm/Group Description	Crushed deferasirox (ICL670) FCT for oral use daily. Deferasirox FCT dosing was based on subject's weight.
Measure Participants	32
Week 4	10.7 (1.29)
Week 12	10.3 (2.13)
EOT (Week 24)	10.6 (1.57)

25. Secondary Outcome

Title	Number of Participants Pre-treated With Deferasirox With Palatability After Taste Item Scoring
Description	The palatability (taste and ability to consume medicine) questionnaire consisted of 4 items, three items measuring taste or ability to consume medicine and one item measuring aftertaste. The aftertaste item was treated as a separate item and scored on a 5-point response scale with the response format Very good = 1 (best), Good = 2, Neither good nor bad = 3, Bad = 4, Very bad = 5 (worst). Only descriptive analysis performed using frequency counts.
Time Frame	Baseline, Week 4, Week 12, EOT (Week 24)

Outcome Measure Data

Analysis Population Description

Full Analysis Set (FAS). Only patients with a value at both baseline and post baseline were to be included.

Arm/Group Title	Deferasirox
Arm/Group Description	Crushed deferasirox (ICL670) FCT for oral use daily. Deferasirox FCT dosing was based on subject's weight.
Measure Participants	32
Very Good	5 11.4%
Good	5 11.4%
Neither good nor bad	15 34.1%
Bad	7 15.9%
Very bad	0 0%
Very Good	1 2.3%
Good	13 29.5%
Neither good nor bad	16 36.4%
Bad	0 0%
Very bad	0 0%
Very Good	4 9.1%
Good	9 20.5%
Neither good nor bad	12 27.3%
Bad	2 4.5%
Very bad	0 0%
Very Good	0 0%
Good	11 25%
Neither good nor bad	14 31.8%
Bad	3 6.8%
Very bad	0 0%

26. Secondary Outcome

Title	GI Symptom Score in Chelation Naive Participants
Description	The GI symptom score was calculated from responses to 5 questions, 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. GI symptom scores were summarized using descriptive statistics at week 2, week 3, week 4, week 8, week 12, week 16, week 20 and EOT.
Time Frame	Week 2, Week 3, Week 4, Week 8, Week 12, Week 16, Week 20, EOT (Week 24)

Outcome Measure Data

Analysis Population Description

Full Analysis Set (FAS). Only patients with a value at both baseline and post baseline were to be included.

Arm/Group Title	Deferasirox
Arm/Group Description	Crushed deferasirox (ICL670) FCT for oral use daily. Deferasirox FCT dosing was based on subject's weight.
Measure Participants	12
Week 2	6.8 (2.82)
Week 3	7.1 (2.85)
Week 4	7.0 (3.52)
Week 8	6.8 (2.64)
Week 12	6.2 (1.79)
Week 16	7.0 (2.18)
Week 20	5.9 (1.29)
EOT (Week 24)	6.8 (1.92)

27. Secondary Outcome

Title	Number of Participants With GI Bowel Movements Item Scoring in Chelation Naive Participants
Description	The GI symptom questionnaire consisted of 6 items, 5 of which were scored using a 1-5 rating scale. The sixth item assessed bowel movement frequency during the past week, using 7 response options 0 = 0 ("None"), 1 = 1, 2 = 2, 3 = 3, 4 = 4, 5 = "5 - 10" and 6 = "11 or more". The GI bowel movements item score was presented descriptively using frequency counts.
Time Frame	Week 2, Week 3, Week 4, Week 8, Week 12, Week 16, Week 20, EOT (Week 24)

Outcome Measure Data

Analysis Population Description

Full Analysis Set (FAS). Only patients with a value at both baseline and post baseline were to be included.

Arm/Group Title	Deferasirox
Arm/Group Description	Crushed deferasirox (ICL670) FCT for oral use daily. Deferasirox FCT dosing was based on subject's weight.
Measure Participants	12
3	1 2.3%
4	1 2.3%
5 to 10	8 18.2%
11 or more	2 4.5%
3	1 2.3%
4	0 0%
5 to 10	7 15.9%
11 or more	2 4.5%
3	0 0%
4	1 2.3%
5 to 10	8 18.2%
11 or more	2 4.5%
3	0 0%
4	3 6.8%
5 to 10	6 13.6%
11 or more	2 4.5%
3	0 0%
4	2 4.5%
5 to 10	6 13.6%
11 or more	1 2.3%
3	0 0%
4	2 4.5%
5 to 10	5 11.4%
11 or more	2 4.5%
3	0 0%
4	1 2.3%
5 to 10	7 15.9%
11 or more	2 4.5%
3	2 4.5%
4	0 0%
5 to 10	6 13.6%
11 or more	1 2.3%

28. Secondary Outcome

Title	GI Symptom Score in Participants Pre-treated With Deferasirox
Description	The GI symptom score was calculated from responses to 5 questions, 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. GI symptom scores were summarized using descriptive statistics at week 2, week 3, week 4, week 8, week 12, week 16, week 20 and EOT.
Time Frame	Week 2, Week 3, Week 4, Week 8, Week 12, Week 16, Week 20, EOT (Week 24)

Outcome Measure Data

Analysis Population Description

Full Analysis Set (FAS). Only patients with a value at both baseline and post baseline were to be included.

Arm/Group Title	Deferasirox
Arm/Group Description	Crushed deferasirox (ICL670) FCT for oral use daily. Deferasirox FCT dosing was based on subject's weight.
Measure Participants	32
Week 2	6.3 (1.91)
Week 3	6.0 (1.63)
Week 4	6.4 (1.81)
Week 8	6.4 (2.34)
Week 12	6.5 (1.83)
Week 16	6.7 (2.10)
Week 20	7.3 (2.37)
EOT (Week 24)	7.6 (2.95)

29. Secondary Outcome

Title	Number of Participants With GI Bowel Movements Item Scoring in Participants Pre-treated With Deferasirox
Description	The GI symptom questionnaire consisted of 6 items, 5 of which were scored using a 1-5 rating scale. The sixth item assessed bowel movement frequency during the past week, using 7 response options 0 = 0 ("None"), 1 = 1, 2 = 2, 3 = 3, 4 = 4, 5 = "5 - 10" and 6 = "11 or more". The GI bowel movements item score was presented descriptively using frequency counts.
Time Frame	Baseline, Week 2, Week 3, Week 4, Week 8, Week 12, Week 16, Week 20, EOT (Week 24)

Outcome Measure Data

Analysis Population Description

Full Analysis Set (FAS). Only patients with a value at both baseline and post baseline were to be included.

Arm/Group Title	Deferasirox
Arm/Group Description	Crushed deferasirox (ICL670) FCT for oral use daily. Deferasirox FCT dosing was based on subject's weight.
Measure Participants	32
3	0 0%
4	1 2.3%
5 to 10	30 68.2%
11 or more	1 2.3%
3	0 0%
4	1 2.3%
5 to 10	29 65.9%
11 or more	2 4.5%
3	0 0%
4	1 2.3%
5 to 10	26 59.1%
11 or more	1 2.3%
3	0 0%
4	1 2.3%
5 to 10	28 63.6%
11 or more	1 2.3%
3	1 2.3%
4	1 2.3%
5 to 10	29 65.9%
11 or more	1 2.3%
3	1 2.3%
4	0 0%
5 to 10	26 59.1%
11 or more	0 0%
3	0 0%
4	1 2.3%
5 to 10	29 65.9%
11 or more	1 2.3%
3	0 0%
4	1 2.3%
5 to 10	28 63.6%
11 or more	2 4.5%
3	0 0%
4	1 2.3%
5 to 10	25 56.8%
11 or more	2 4.5%

Adverse Events

Time Frame	Treatment-emergent adverse events (TEAEs) were collected from first dose of study treatment until end of study treatment plus 30 days post-treatment, assessed for approximately 28 weeks.
Adverse Event Reporting Description	Any sign or symptom that occurs during the study treatment and 30 days post treatment follow up. Maximum exposure to study treatment = 26 weeks
Arm/Group Title	Deferasirox
Arm/Group Description	Crushed deferasirox (ICL670) FCT for oral use daily. Deferasirox FCT dosing was based on subject's weight.
All Cause Mortality
	Deferasirox
	Affected / at Risk (%)	# Events
Total	0/44 (0%)
Serious Adverse Events
	Deferasirox
	Affected / at Risk (%)	# Events
Total	2/44 (4.5%)
General disorders
Pyrexia	1/44 (2.3%)
Infections and infestations
Bronchitis	1/44 (2.3%)
Other (Not Including Serious) Adverse Events
	Deferasirox
	Affected / at Risk (%)	# Events
Total	29/44 (65.9%)
Gastrointestinal disorders
Diarrhoea	4/44 (9.1%)
General disorders
Pyrexia	5/44 (11.4%)
Infections and infestations
Gastroenteritis	4/44 (9.1%)
Upper respiratory tract infection	10/44 (22.7%)
Investigations
Alanine aminotransferase increased	7/44 (15.9%)
Bilirubin conjugated increased	6/44 (13.6%)
Blood creatinine increased	9/44 (20.5%)
Urine protein/creatinine ratio increased	12/44 (27.3%)
Respiratory, thoracic and mediastinal disorders
Cough	6/44 (13.6%)
Rhinorrhoea	3/44 (6.8%)

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.