Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload

• Sponsor: Novartis Pharmaceuticals (Industry)
• Overall Status: Completed
• CT.gov ID: NCT00235391
• Collaborator: (none)
• Enrollment: 1,683
• Locations: 141
• Arm: 1
• Duration (Months): 36
• Patients Per Site: 11.9
• Patients Per Site Per Month: 0.3

Study Details

Study Description

Brief Summary

This is an open-label, non-randomized, multi-center trial designed to provide expanded access of deferasirox to patients with congenital disorders of red blood cells and chronic iron overload from blood transfusions who cannot adequately be treated with locally approved iron chelators.

Condition or Disease	Intervention/Treatment	Phase
Thalassemia; Sickle Cell Disease; Diamond Blackfan Anemia; Myelofibrosis	Drug: Deferasirox	Phase 3

Study Design

• Study Type: Interventional
• Actual Enrollment: 1683 participants
• Allocation: Non-Randomized
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: A Study to Provide Expanded Access of (Exjade®) Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload From Blood Transfusions Who Cannot Adequately be Treated With Other Locally Approved Iron Chelators
• Study Start Date: Oct 1, 2005
• Actual Primary Completion Date: Oct 1, 2008
• Actual Study Completion Date: Oct 1, 2008

Arms and Interventions

Arm

Intervention/Treatment

Experimental: Deferasirox Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Starting dose was determined by the frequency of blood transfusions and recommended initial daily dose of deferasirox is 20 mg/kg body weight for patients receiving blood transfusion, 10 mg/kg for patients receiving less frequent transfusion/exchange transfusion and 30 mg/kg for patients receiving more frequent blood transfusions.

Drug: Deferasirox; 125 mg, 250 mg and 500 mg tablets. Dosage was calculated based on participant's body weight. Tablets were dispersed in water, orange or apple juice and taken orally once a day.

Outcome Measures

Primary Outcome Measures

1. Safety Profile of Deferasirox Based Upon Drug Administration and Reporting of Serious Adverse Events [Baseline to end of study (Median exposure time to drug was approximately 30 weeks; Maximum exposure was 104 weeks)]

   Safety as assessed by the number of participants with death, serious adverse events (SAE), and/or Adverse Events (AEs) leading to study drug interruption or discontinuation. Note: only treatment emergent AEs are summarized.

Secondary Outcome Measures

1. The Change in Serum Ferritin Values From Baseline Through Completion of the Study [Baseline to end of study (Median exposure time to drug was approximately 30 weeks; Maximum exposure was 104 weeks)]

   The number of participants with Improvement, No Change or Worsening in Serum ferritin category levels at the end of the study compared to baseline. Serum ferritin levels in µg/L were divided into to 6 categories: (<1000), (1000-<2500), (2500-<4000), (4000-<5500), (5500-<7000) and (>=7000). Improvement was defined as a shift to a lower category at the end of study compared to the category at baseline. Worsening was defined as a shift to a higher category at the end of the study compared to the category at baseline. No change was no change in category at end of study from baseline.

Eligibility Criteria

Criteria

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

Inclusion Criteria:

• Male or female patients greater than or equal to 2 years of age

• Documented congenital disorder of red blood cells (e.g., β-thalassemia major, sickle cell anemia, diamond-blackfan anemia) requiring ongoing blood transfusions

• Cannot be adequately treated with a locally approved iron chelator due to one of the following reasons:

• Documented non-compliance, defined as having taken less than 50% of the prescribed chelation therapy doses in the 12 months prior to study entry

• Contraindications, unacceptable toxicities and/or documented poor response to locally approved iron chelators despite proper compliance

• History of at least 20 blood transfusions (equivalent to 100 mL/kg of packed red blood cells (PRBC])

• Serum ferritin value greater than or equal to 1000 µg/L

• Ability to comply with all study-related procedures, medications, and evaluations

Exclusion Criteria:

• Ongoing treatment with another iron chelator (Any other iron chelation therapy must be discontinued at least 24 hours prior to study entry.)

• Patients who meet the eligibility criteria for any other ongoing Novartis sponsored clinical study protocol with deferasirox and who have geographic access to these sites

• Patients unable to tolerate (or who have unacceptable toxicities to) prior treatment with deferasirox

• Serum creatinine above the upper limit of normal at screening.

• Patients with ALT ≥ 500 U/L at screening.

• Evidence of chelation-related cataracts or hearing loss within 4 weeks prior to baseline

• Pregnancy (as indicated by serum β-HCG pregnancy test at screening for all female patients with the potential to become pregnant) and patients who are breastfeeding

• Patients treated with systemic investigational drug within 4 weeks prior to or with topical investigational drug within 7 days prior to the baseline visit

Other protocol-defined inclusion/exclusion criteria may apply.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Arkansas Children's Hospital, UAMS College of Medicine	Little Rock	Arkansas	United States	72202
2	Alta Bates Comprehensive Cancer Center	Berkeley	California	United States	94704
3	Children's Hospital of Orange County	Orange	California	United States	92868
4	Children's Hospital and Health Center of San Diego	San Diego	California	United States	92123
5	Stanford University	Stanford	California	United States	94305
6	Alfred I. Dupong Hospital for Children	Wilmington	Delaware	United States	19803
7	Osler Medical, Inc.	Melbourne	Florida	United States	32901
8	Hematalogy Oncology Associates	Pensacola	Florida	United States	32501
9	Tampa Children's Hospital at St. Joseph's Hospital	Tampa	Florida	United States	33607
10	James A. Haley Veterans Hospital	Tampa	Florida	United States	33612
11	Backus Children's Hospital, Memorial Health University Medical Center	Savannah	Georgia	United States	31403
12	Hematalogy Oncology Clinic	Baton Rouge	Louisiana	United States	70808
13	Borgess Hospital	Kalamazoo	Michigan	United States	49048
14	Children's Hospitals and Clinics of Minnesota	Minneapolis	Minnesota	United States	55405
15	University of Mississippi Medical Center	Jackson	Mississippi	United States	39762
16	Children's Mercy Hospital	Kansas City	Missouri	United States	64108
17	The Cancer Institute of New Jersey	New Brunswick	New Jersey	United States	08903
18	Schneider Children's Hospital	New Hyde Park	New York	United States	11040
19	PCTI	Columbus	Ohio	United States	43205
20	The Children's Medical Center of Dayton	Dayton	Ohio	United States	45404
21	Oregon Health & Science University	Portland	Oregon	United States	97239
22	Hershey Medical Center	Hershey	Pennsylvania	United States	17033-0850
23	Children's Hospitals of Pittsburgh	Pittsburgh	Pennsylvania	United States	15213
24	Texas Children's Hospital	Houston	Texas	United States	77030
25	Children's Hospital of the Kings Daughters	Norfolk	Virginia	United States	23507
26	VCU Pediatric Hematology/Oncology	Richmond	Virginia	United States	23219
27	University of Washington Seattle Cancer Care Alliance	Seattle	Washington	United States	98195
28	Ziekenhuisnetwerk Antwerpen-AZ Middelheim	Antwerpen		Belgium
29	Centre Hospitalier Notre Dame et Reine	Charleroi		Belgium
30	CHR de la Citadelle	Liege		Belgium
31	Centre Hospitalier Chretien-Clinique Saint-Joseph	Montegnee		Belgium
32	The Ottawa Hospital-General Campus	Ottawa	Ontario	Canada
33	University of Alberta	Edmonton		Canada
34	CHUM-Hopital-Notre-Dame	Montreal		Canada
35	MUHC- Montreal Children's Hospital	Montreal		Canada
36	MUHC- Royal Victoria Hospital	Montreal		Canada
37	Hopital de l'Enfant-Jesus	Quebec		Canada
38	The Hospital for Sick Children,	Toronto		Canada
39	Toronto General Hospital-Hemoglobinopathy	Toronto		Canada
40	Burrard Medical Building	Vancouver		Canada
41	Charite-Universitatsmedizin Berlin	Berlin		Germany
42	Universitaetsklinik Dusseldorf	Duesseldorf		Germany
43	Johann Wolfgang von Goethe Universitat	Frankfurt am Main		Germany
44	Georg-August-Universitat Gottingen	Gottingen		Germany
45	Universitatskrankenhaus Hamburg-Eppendorf	Hamburg		Germany
46	Medizinische Hochschule Hannover	Hannover		Germany
47	Universitatsklinikum Koln	Köln		Germany
48	Klinikum Stuttgart Bismarckstrasse 8	Stuttgart		Germany
49	Universitatsklinikum Ulm	Ulm		Germany
50	Agia Sofia Hospital of Athens	Athens		Greece
51	General Hospital of Athens	Athens		Greece
52	Ippokration Hospital of Athens	Athens		Greece
53	General Hospital of Heraklion Benizeleio-Pananeio	Heraklion		Greece
54	University Hospital of Ioannina	Ioannina		Greece
55	General Hospital of Kalamata	Kalamata		Greece
56	Agia Sofia Hospital of Athens	Karditsa		Greece
57	General Hospital of Karditsa	Karditsa		Greece
58	General Hospital of Athens	Kerkyra		Greece
59	General Hospital of Korinthos	Korinthos		Greece
60	General Hospital of Larisa Tsakalof 1	Larisa		Greece
61	General Hospital of Mytilini Vostaneio	Mytilini		Greece
62	General State Hospital of Nikaia St. Panteleimon	Nikaia		Greece
63	University Hospital of Patras	Patra		Greece
64	General Hospital of Thessaloniki Agios Pavlos	Thessaloniki		Greece
65	General Hospital Thessalonikis Hippokratio	Thessaloniki		Greece
66	General Hospital of Volos	Volos		Greece
67	General Hospital of Xanthi	Xanthi		Greece
68	Presidio Ospedale Muscatello	Augusta		Italy
69	A.O. Ospedale Policlinico Consorziale di Bari	Bari		Italy
70	Presidio Ospedaliero Antonio Perrino	Brindisi		Italy
71	Ospedale Regionale Microcitemie	Cagliari		Italy
72	Ospedale Di Venere	Carbonara di Bari		Italy
73	Azienda Ospedali Vittorio Emanuele, Ferrarotto e San Bambino	Catania		Italy
74	Presidio Ospedaliero S. Bambino	Catania		Italy
75	PresidioOspedaliero S. Luigi Curro	Catania		Italy
76	Azienda Ospedaliera Pugliese Cicaccio	Catanzaro		Italy
77	Ospedale Civile dell'Annunziata	Cosenza		Italy
78	Ospedale San Giuseppe	Empoli		Italy
79	Azienda Ospedaliera Universitaria di Ferrara	Ferrara		Italy
80	Azienda Ospedaliera A. Meyer	Firenze		Italy
81	E.O. Ospedali Galliera	Genova		Italy
82	Ospedale Regionale Microcitemie	Itala		Italy
83	Ospedale Madonna delle Grazie	Matera		Italy
84	Az. Ospedaliera Universitaria Policlinico G. Martino	Messina		Italy
85	Fondazione Ospedale	Milano		Italy
86	Azienda Ospedaliero-Universitaria di Modena	Modena		Italy
87	AORN A. Cardarelli	Napoli		Italy
88	Azienda Ospedaliera Universitaria Federico II	Napoli		Italy
89	Azienda Ospedaliera V. Cervello	Palermo		Italy
90	Azienda Ospedaliera Villa Sofia-CTO	Palermo		Italy
91	Presidio Ospedaliero Giovanni di Cristina	Palermo		Italy
92	IRCCS Policlinico San Matteo	Pavia		Italy
93	Azienda Ospedaliera San Salvatore	Pesaro		Italy
94	Azienda Ospedaliero Universitaria Pisana	Pisa		Italy
95	Azienda Ospedaliera Civile- Maria Paterno	Ragusa		Italy
96	Azienda Ospedaliera Bianchi-Melacrino-Morelli	Reggio Calabria		Italy
97	Ospedale S. Eugenio	Roma		Italy
98	Ospedale nostra Signora di Bonaria	San Gavino Monreale- CA		Italy
99	Presidio Ospedaliero di Sassari-Ospedale SS	Sassari		Italy
100	Azienda Ospedaliera Ospedali Civili Riuniti di Sciacca	Sciacca		Italy
101	Ospedale Umberto I	Talassemie		Italy
102	Presidio Ospedaliero Centrale	Taranto		Italy
103	Ospedale Infantile Regina Margherita	Torino		Italy
104	AMC	Amsterdam		Netherlands
105	Haga Ziekenhuis	Den Haag		Netherlands
106	Catharina-ziekenhuis	Eindhoven		Netherlands
107	Erasmus MC	Rotterdam		Netherlands
108	Erasmus Medisch Centrum, locatie Sophia	Rotterdam		Netherlands
109	Hospital de Torrecardenas	Almeria		Spain
110	Hospital Infanta Cristina	Badajoz		Spain
111	Hospital Cruces	Baracaldo		Spain
112	Hospital Puerta del Mar	Cadiz		Spain
113	Hospital De Gran Canaria	Canara		Spain
114	Hospital Universitario La Paz	Madrid		Spain
115	Althaia : Xarxa Assistencial de Manresa	Manresa		Spain
116	Hospital Universitario Marques de Valdecilla	Santander		Spain
117	Hospital Virgen de la Salud	Toledo		Spain
118	Hospital Universitario La Fe	Valencia		Spain
119	Hospital Xeral de Vigo	Vigo		Spain
120	Kaohsiung Medical University Hospital	Kaohsiung		Taiwan
121	Mackay Memorial Hospital	Taipei		Taiwan
122	National Taiwan University Hospital	Taipei		Taiwan
123	Chang Gung Children's Hospital	Taoyuan		Taiwan
124	Tao-yuan General Hospital	Tau-Yuan County		Taiwan
125	Phramongkutklao Hospital	Bangkok		Thailand
126	Ramathibodi Hospital	Bangkok		Thailand
127	Songklanagarind Hospital	Bangkok		Thailand
128	Srinagarind Hospital	Khon Kaen		Thailand
129	Cukurova Universitesi	Adana		Turkey
130	Hacettepe Universitesi	Ankara		Turkey
131	Akdeniz Universitesi	Antalya		Turkey
132	Gaziantep Universitesi	Gaziantep		Turkey
133	Suleyman Demirel	Isparta		Turkey
134	Istanbul Universitesi	Istanbul		Turkey
135	Ege Universitesi Tip Fakultesi	Izmir		Turkey
136	Erciyes Universitesi	Kayseri		Turkey
137	Evelina Hospital St. Thomas' Hospital	London		United Kingdom
138	North Middlesex University Hospital	London		United Kingdom
139	The Royal Hospital London	London		United Kingdom
140	Whittington Hospital	London		United Kingdom
141	St. George's Hospital	Tooting		United Kingdom

Sponsors and Collaborators

• Novartis Pharmaceuticals

Investigators

• Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Study Documents (Full-Text)

None provided.

More Information

Additional Information:

• Visit NovartisClinicalTrials.com: Pre-qualify for a trial, and view a list of trials and participating study centers.

Publications

None provided.

• ClinicalTrials.gov Identifier: NCT00235391
• Other Study ID Numbers: CICL670A2203
• First Posted: Oct 10, 2005
• Last Update Posted: Jun 7, 2011
• Last Verified: Jun 1, 2011

• Keywords provided by , ,: Deferasirox; Congenital Anemias; Anemias; Red Blood Cell Disorders; Chronic Iron Overload; Transfusional Iron Overload; Iron Chelators; Oral Iron Chelators; Thalassemia; Sickle Cell Disease; Diamond Blackfan Anemia; Myelofibrosis; ICL670A
• Additional relevant MeSH terms: Anemia; Anemia, Sickle Cell; Primary Myelofibrosis; Thalassemia; Anemia, Diamond-Blackfan; Iron Overload; Deferasirox

Study Results

Participant Flow

Arm/Group Title	2 to < 6 Years	6 to < 12 Years	12 to < 16 Years	16 to < 50 Years	50 to < 65 Years	≥ 65 Years
Arm/Group Description	Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.	Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.	Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.	Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.	Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.	Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
Period Title: Overall Study
STARTED	97	200	172	1164	43	7
Deferasirox Treatment	97	200	172	1164	43	7
COMPLETED	85	182	148	944	31	3
NOT COMPLETED	12	18	24	220	12	4

Baseline Characteristics

Arm/Group Title	2 to < 6 Years	6 to < 12 Years	12 to < 16 Years	16 to < 50 Years	50 to < 65 Years	≥ 65 Years	Total
Arm/Group Description	Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.	Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.	Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.	Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.	Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.	Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.	Total of all reporting groups
Overall Participants	97	200	172	1164	43	7	1683
Age (years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [years]	3.48 (1.13)	8.52 (1.64)	13.48 (1.20)	28.91 (8.06)	54.58 (3.92)	70.14 (3.98)	24.27 (12.63)
Sex: Female, Male (Count of Participants)
Female	44 45.4%	101 50.5%	81 47.1%	633 54.4%	32 74.4%	5 71.4%	896 53.2%
Male	53 54.6%	99 49.5%	91 52.9%	531 45.6%	11 25.6%	2 28.6%	787 46.8%
Baseline Disease Characteristics (participants) [Number]
Beta-Thalassemia Major	74 76.3%	118 59%	114 66.3%	905 77.7%	10 23.3%	0 0%	1221 72.5%
Beta-Thalassemia Intermedia	1 1%	22 11%	16 9.3%	94 8.1%	20 46.5%	3 42.9%	156 9.3%
Sickle Cell Disease	8 8.2%	41 20.5%	31 18%	93 8%	2 4.7%	1 14.3%	176 10.5%
Diamond-Blackfan Anemia	4 4.1%	7 3.5%	8 4.7%	24 2.1%	0 0%	0 0%	43 2.6%
Other Diseases	10 10.3%	12 6%	3 1.7%	48 4.1%	11 25.6%	3 42.9%	87 5.2%
Prior Chelation Drug Therapy (participants) [Number]
Deferoxamine	80 82.5%	167 83.5%	137 79.7%	745 64%	31 72.1%	5 71.4%	1165 69.2%
Deferiprone	1 1%	8 4%	11 6.4%	146 12.5%	6 14%	1 14.3%	173 10.3%
Deferoxamine and Deferiprone	2 2.1%	16 8%	21 12.2%	269 23.1%	6 14%	0 0%	314 18.7%
Other Chelation Drug	6 6.2%	6 3%	3 1.7%	4 0.3%	0 0%	0 0%	19 1.1%
Prior Chelatation Drug Information Missing	8 8.2%	3 1.5%	0 0%	0 0%	0 0%	1 14.3%	12 0.7%
Reason for inadequate prior chelation therapy (participants) [Number]
Therapy non-compliance	57 58.8%	125 62.5%	106 61.6%	659 56.6%	25 58.1%	2 28.6%	974 57.9%
Therapy contraindication	1 1%	7 3.5%	6 3.5%	29 2.5%	1 2.3%	1 14.3%	45 2.7%
Therapy unacceptable toxicity	11 11.3%	29 14.5%	22 12.8%	131 11.3%	6 14%	1 14.3%	200 11.9%
Therapy poor response	20 20.6%	34 17%	38 22.1%	267 22.9%	8 18.6%	0 0%	367 21.8%
Therapy unacceptable discomfort	2 2.1%	2 1%	0 0%	78 6.7%	3 7%	2 28.6%	87 5.2%
Reason for inadequate therapy information missing	6 6.2%	3 1.5%	0 0%	0 0%	0 0%	1 14.3%	10 0.6%

Outcome Measures

1. Primary Outcome

Title	Safety Profile of Deferasirox Based Upon Drug Administration and Reporting of Serious Adverse Events
Description	Safety as assessed by the number of participants with death, serious adverse events (SAE), and/or Adverse Events (AEs) leading to study drug interruption or discontinuation. Note: only treatment emergent AEs are summarized.
Time Frame	Baseline to end of study (Median exposure time to drug was approximately 30 weeks; Maximum exposure was 104 weeks)

Outcome Measure Data

Analysis Population Description

The safety population, comprising all participants who received at least one dose of deferasirox during the study, was used in the analyses.

Arm/Group Title	2 to < 6 Years	6 to < 12 Years	12 to < 16 Years	16 to < 50 Years	50 to < 65 Years	≥ 65 Years
Arm/Group Description	Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.	Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.	Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.	Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.	Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.	Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
Measure Participants	97	200	172	1164	43	7
Number of deaths	0 0%	0 0%	1 0.6%	4 0.3%	0 0%	0 0%
Non-fatal SAEs	10 10.3%	22 11%	27 15.7%	129 11.1%	4 9.3%	2 28.6%
AEs leading to discontinuation	4 4.1%	5 2.5%	10 5.8%	75 6.4%	5 11.6%	2 28.6%
AEs leading dose adjustment/temporary interruption	15 15.5%	31 15.5%	28 16.3%	209 18%	11 25.6%	2 28.6%

2. Secondary Outcome

Title	The Change in Serum Ferritin Values From Baseline Through Completion of the Study
Description	The number of participants with Improvement, No Change or Worsening in Serum ferritin category levels at the end of the study compared to baseline. Serum ferritin levels in µg/L were divided into to 6 categories: (<1000), (1000-<2500), (2500-<4000), (4000-<5500), (5500-<7000) and (>=7000). Improvement was defined as a shift to a lower category at the end of study compared to the category at baseline. Worsening was defined as a shift to a higher category at the end of the study compared to the category at baseline. No change was no change in category at end of study from baseline.
Time Frame	Baseline to end of study (Median exposure time to drug was approximately 30 weeks; Maximum exposure was 104 weeks)

Outcome Measure Data

Analysis Population Description

Safety population defined as all participants who received at least one dose of study drug. This analysis did not include participants with unknown status at baseline and/or at the end of the study.

Arm/Group Title	2 to < 6 Years	6 to < 12 Years	12 to < 16 Years	16 to < 50 Years	50 to < 65 Years	≥ 65 Years
Arm/Group Description	Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.	Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.	Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.	Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.	Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.	Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
Measure Participants	97	200	172	1164	43	7
Improvement	17 17.5%	27 13.5%	26 15.1%	206 17.7%	4 9.3%	2 28.6%
No Change	63 64.9%	106 53%	87 50.6%	570 49%	31 72.1%	3 42.9%
Worsening	16 16.5%	63 31.5%	55 32%	341 29.3%	7 16.3%	1 14.3%

Adverse Events

Arm/Group Title	All Participants
Arm/Group Description	Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Tablets were dispersed in water, orange or apple juice.
All Cause Mortality
	All Participants
	Affected / at Risk (%)	# Events
Total	/ (NaN)
Serious Adverse Events
	All Participants
	Affected / at Risk (%)	# Events
Total	194/1683 (11.5%)
Blood and lymphatic system disorders
Acute chest syndrome	2/1683 (0.1%)
Anaemia	2/1683 (0.1%)
Anaemia haemolytic autoimmune	2/1683 (0.1%)
Coombs positive haemolytic anaemia	1/1683 (0.1%)
Haemolytic anaemia	1/1683 (0.1%)
Hypersplenism	3/1683 (0.2%)
Intravascular haemolysis	1/1683 (0.1%)
Leukopenia	1/1683 (0.1%)
Neutropenia	2/1683 (0.1%)
Pancytopenia	1/1683 (0.1%)
Reticulocytopenia	1/1683 (0.1%)
Splenomegaly	4/1683 (0.2%)
Cardiac disorders
Arrhythmia	1/1683 (0.1%)
Atrial fibrillation	2/1683 (0.1%)
Cardiac failure	6/1683 (0.4%)
Cardiac failure acute	1/1683 (0.1%)
Cardiac failure congestive	2/1683 (0.1%)
Congestive cardiomyopathy	1/1683 (0.1%)
Myopericarditis	1/1683 (0.1%)
Pericardial effusion	1/1683 (0.1%)
Tachycardia	1/1683 (0.1%)
Ventricular dysfunction	1/1683 (0.1%)
Congenital, familial and genetic disorders
Hip dysplasia	1/1683 (0.1%)
Sickle cell anaemia	2/1683 (0.1%)
Sickle cell anaemia with crisis	27/1683 (1.6%)
Thalassaemia	1/1683 (0.1%)
Thalassaemia beta	2/1683 (0.1%)
Ear and labyrinth disorders
Deafness unilateral	1/1683 (0.1%)
Middle ear effusion	1/1683 (0.1%)
Eye disorders
Retinopathy	1/1683 (0.1%)
Gastrointestinal disorders
Abdominal discomfort	1/1683 (0.1%)
Abdominal pain	3/1683 (0.2%)
Abdominal pain upper	2/1683 (0.1%)
Colitis	1/1683 (0.1%)
Constipation	1/1683 (0.1%)
Crohn's disease	1/1683 (0.1%)
Diarrhoea	1/1683 (0.1%)
Gastritis	4/1683 (0.2%)
Gastrointestinal haemorrhage	1/1683 (0.1%)
Haemorrhoids	1/1683 (0.1%)
Pancreatitis	1/1683 (0.1%)
Peritonitis	1/1683 (0.1%)
Proctitis	1/1683 (0.1%)
Vomiting	1/1683 (0.1%)
General disorders
Catheter thrombosis	1/1683 (0.1%)
Death	1/1683 (0.1%)
Local swelling	1/1683 (0.1%)
Malaise	3/1683 (0.2%)
Non-cardiac chest pain	5/1683 (0.3%)
Pain	3/1683 (0.2%)
Pyrexia	9/1683 (0.5%)
Hepatobiliary disorders
Biliary colic	1/1683 (0.1%)
Cholelithiasis	6/1683 (0.4%)
Hepatitis	2/1683 (0.1%)
Hyperbilirubinaemia	1/1683 (0.1%)
Liver disorder	1/1683 (0.1%)
Immune system disorders
Hypersensitivity	1/1683 (0.1%)
Infections and infestations
Abdominal infection	1/1683 (0.1%)
Acute tonsillitis	2/1683 (0.1%)
Appendicitis	1/1683 (0.1%)
Bacterial infection	1/1683 (0.1%)
Bronchitis	2/1683 (0.1%)
Bronchopneumonia	1/1683 (0.1%)
Catheter related infection	1/1683 (0.1%)
Catheter site infection	1/1683 (0.1%)
Cellulitis	2/1683 (0.1%)
Diarrhoea infectious	1/1683 (0.1%)
Ear infection	1/1683 (0.1%)
Epstein-Barr virus infection	3/1683 (0.2%)
Escherichia bacteraemia	1/1683 (0.1%)
Gastroenteritis	3/1683 (0.2%)
Gastrointestinal infection	1/1683 (0.1%)
Hepatitis C	1/1683 (0.1%)
Influenza	1/1683 (0.1%)
Liver abscess	1/1683 (0.1%)
Necrotising fasciitis	1/1683 (0.1%)
Osteomyelitis	2/1683 (0.1%)
Otitis media acute	1/1683 (0.1%)
Parvovirus infection	1/1683 (0.1%)
Perianal abscess	1/1683 (0.1%)
Pharyngitis	1/1683 (0.1%)
Pharyngitis streptococcal	1/1683 (0.1%)
Pneumonia	4/1683 (0.2%)
Pyelonephritis	1/1683 (0.1%)
Respiratory tract infection	2/1683 (0.1%)
Rhinitis	1/1683 (0.1%)
Sepsis	1/1683 (0.1%)
Tonsillitis	1/1683 (0.1%)
Upper respiratory tract infection	1/1683 (0.1%)
Viral infection	5/1683 (0.3%)
Injury, poisoning and procedural complications
Clavicle fracture	1/1683 (0.1%)
Comminuted fracture	1/1683 (0.1%)
Femur fracture	2/1683 (0.1%)
Forearm fracture	1/1683 (0.1%)
Haemolytic transfusion reaction	1/1683 (0.1%)
Post-traumatic pain	1/1683 (0.1%)
Road traffic accident	2/1683 (0.1%)
Spinal fracture	1/1683 (0.1%)
Suture rupture	1/1683 (0.1%)
Thermal burn	1/1683 (0.1%)
Tibia fracture	1/1683 (0.1%)
Upper limb fracture	1/1683 (0.1%)
Investigations
Alanine aminotransferase increased	2/1683 (0.1%)
Blood calcium increased	1/1683 (0.1%)
Blood creatinine increased	1/1683 (0.1%)
Cardiovascular evaluation	1/1683 (0.1%)
Protein urine present	1/1683 (0.1%)
Serum ferritin increased	1/1683 (0.1%)
Transaminases increased	1/1683 (0.1%)
Metabolism and nutrition disorders
Hyperglycaemia	1/1683 (0.1%)
Hypocalcaemia	1/1683 (0.1%)
Hypoglycaemic seizure	1/1683 (0.1%)
Musculoskeletal and connective tissue disorders
Arthralgia	1/1683 (0.1%)
Back pain	1/1683 (0.1%)
Fistula	1/1683 (0.1%)
Osteonecrosis	1/1683 (0.1%)
Pain in extremity	1/1683 (0.1%)
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Benign neoplasm of thymus	1/1683 (0.1%)
Hepatic neoplasm malignant recurrent	1/1683 (0.1%)
Non-small cell lung cancer	1/1683 (0.1%)
Nervous system disorders
Convulsion	1/1683 (0.1%)
Headache	2/1683 (0.1%)
Migraine	1/1683 (0.1%)
Paraesthesia	1/1683 (0.1%)
Spinal cord compression	1/1683 (0.1%)
Pregnancy, puerperium and perinatal conditions
Pregnancy	5/1683 (0.3%)
Psychiatric disorders
Anxiety	1/1683 (0.1%)
Depression	1/1683 (0.1%)
Suicide attempt	1/1683 (0.1%)
Renal and urinary disorders
Calculus ureteric	1/1683 (0.1%)
Nephrotic syndrome	1/1683 (0.1%)
Renal colic	2/1683 (0.1%)
Renal failure	2/1683 (0.1%)
Urethral stenosis	1/1683 (0.1%)
Urinary retention	1/1683 (0.1%)
Reproductive system and breast disorders
Haemorrhagic ovarian cyst	1/1683 (0.1%)
Uterine disorder	1/1683 (0.1%)
Respiratory, thoracic and mediastinal disorders
Adenoidal hypertrophy	1/1683 (0.1%)
Asthma	1/1683 (0.1%)
Dyspnoea	1/1683 (0.1%)
Epistaxis	1/1683 (0.1%)
Hypoxia	1/1683 (0.1%)
Pleural effusion	1/1683 (0.1%)
Pneumonitis	1/1683 (0.1%)
Pulmonary embolism	2/1683 (0.1%)
Skin and subcutaneous tissue disorders
Erythema multiforme	1/1683 (0.1%)
Pigmentation disorder	1/1683 (0.1%)
Rash	5/1683 (0.3%)
Rash maculo-papular	2/1683 (0.1%)
Swelling face	1/1683 (0.1%)
Urticaria	1/1683 (0.1%)
Surgical and medical procedures
Uterine dilation and curettage	1/1683 (0.1%)
Vascular disorders
Deep vein thrombosis	1/1683 (0.1%)
Hypotension	1/1683 (0.1%)
Other (Not Including Serious) Adverse Events
	All Participants
	Affected / at Risk (%)	# Events
Total	85/1683 (5.1%)
Skin and subcutaneous tissue disorders
Rash	85/1683 (5.1%)

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 (i.e., data from all sites) in the clinical trial.