An Extension Study of Iron Chelation Therapy With Deferas... | NCT00171210

Label	Type	Description	Intervention Names
Deferasirox	Experimental	All participants received Deferasirox (ICL670) orally once a day. Dosage based on body weight.	Drug: Deferasirox

Name	Type	Description	Arm Group Labels	Other Names
Deferasirox	Drug	Tablets taken orally once a day.	Deferasirox

Measure	Description	Time Frame
Long Term Safety and Tolerability Profile of ICL670 Based on the Number of Participants Who Experienced Any Adverse Event	Adverse events results are based on preferred terms with at least 7% of participants in any group.	up to 5 years

Measure	Description	Time Frame
Long-term Effect of ICL670 on Hepatic Iron Stores Measured by Means of Liver Iron Content (LIC) as Assessed by Liver Biopsy	Mean absolute change of LIC from start of Deferasirox (ICL670) treatment to the end of study assessed by liver biopsy. Reported in milligrams of Iron per gram dry weight (mg Fe/g dw).	Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)

Name	Affiliation	Role
Novartis Pharmaceuticals	Novartis Pharmaceuticals	Study Director

Facility	Status	City	State	ZIP	Country	Contacts
Children's Hospital Los Angeles		Los Angeles	California	90027	United States
Children's Hospital and Research Center at Oakland

PubMed Identifier	Type	Citation	Retractions
21628399	Derived	Cappellini MD, Bejaoui M, Agaoglu L, Canatan D, Capra M, Cohen A, Drelichman G, Economou M, Fattoum S, Kattamis A, Kilinc Y, Perrotta S, Piga A, Porter JB, Griffel L, Dong V, Clark J, Aydinok Y. Iron chelation with deferasirox in adult and pediatric patients with thalassemia major: efficacy and safety during 5 years' follow-up. Blood. 2011 Jul 28;118(4):884-93. doi: 10.1182/blood-2010-11-316646. Epub 2011 May 31.

ID	Title	Description
FG000	Crossover	Participants treated with Deferoxamine (DFO) during the core study and crossed over to receive Deferasirox (ICL670) orally once a day during the extension study. Dosage based on body weight.
FG001	ICL670

Type

Title

Description

Population Description

Reporting Status

Anticipated Posting Date

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Time Frame

Units Analyzed

Denominator Units Selected

Arm/Group Information

Denominators

Classes

Analyses

Primary

Long Term Safety and Tolerability Profile of ICL670 Based on the Number of Participants Who Experienced Any Adverse Event

Adverse events results are based on preferred terms with at least 7% of participants in any group.

All patients enrolled into core study and who consented to participate in extension contributed to the pool of data on long term safety follow-up. Analyses included all patients who received at least 1 dose of ICL670 in the core/extension study, that is, analyses also included ICL670 patients from the core group who did not continue into extension.

Posted

Number

Participants

up to 5 years

ID	Title	Description
OG000	Crossover	Participants treated with Deferoxamine (DFO) during the core study and crossed over to receive Deferasirox (ICL670) orally once a day during the extension study. Dosage based on body weight.
OG001	ICL670	Participants treated with Deferasirox (ICL670) orally once a day during the core study and continued this treatment in the extension study. Dosage based on body weight.

Units	Counts
Participants	OG000259 OG001296

Title

Denominators

Categories

Pyrexia

Title	Measurements
	OG000109 OG001113

Cough

Title	Measurements
	OG000

Secondary

Long-term Effect of ICL670 on Hepatic Iron Stores Measured by Means of Liver Iron Content (LIC) as Assessed by Liver Biopsy

Mean absolute change of LIC from start of Deferasirox (ICL670) treatment to the end of study assessed by liver biopsy. Reported in milligrams of Iron per gram dry weight (mg Fe/g dw).

Population includes only those participants from the full analysis set (defined as all participants who received at least 1 dose of ICL670 in the core/extension study, that is, analyses also included ICL670 participants from the core group who did not continue into extension) and had LIC Biopsy data at Start of ICL670 treatment and End of Study.

Posted

Mean

Standard Deviation

mg Fe/g dw

Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)

ID	Title	Description
OG000	Crossover	Participants treated with Deferoxamine (DFO) during the core study and crossed over to receive Deferasirox (ICL670) orally once a day during the extension study. Dosage based on body weight.
OG001	ICL670	Participants treated with Deferasirox (ICL670) orally once a day during the core study and continued this treatment in the extension study. Dosage based on body weight.

Units

Secondary

Long-term Effect of ICL670 on Hepatic Iron Stores Measured by Means of Liver Iron Content (LIC) as Assessed by SQUID

Mean absolute change in LIC from start of Deferasirox (ICL670) treatment to the end of the study assessed by Superconducting Quantum Interfering Device (SQUID) measurement used as a non-invasive alternative to Biopsy for pediatric participants. Reported in milligrams of Iron per gram dry weight (mg Fe/g dw).

Population includes only those participants from the full analysis set (defined as all participants who received at least 1 dose of ICL670 in the core/extension study, that is, analyses also included ICL670 participants from the core group who did not continue into extension) and had LIC SQUID data at Start of ICL670 treatment and End of Study.

Posted

Mean

Standard Deviation

mg Fe/g dw

Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)

ID	Title	Description
OG000	Crossover	Participants treated with Deferoxamine (DFO) during the core study and crossed over to receive Deferasirox (ICL670) orally once a day during the extension study. Dosage based on body weight.
OG001	ICL670	Participants treated with Deferasirox (ICL670) orally once a day during the core study and continued this treatment in the extension study. Dosage based on body weight.

Secondary

Long-term Effect of Treatment With ICL670 on the Changes in Serum Ferritin Levels From Start of ICL670 Treatment to End of Study

Mean Absolute Change in serum ferritin (ug/L) from start of treatment with Deferasirox (ICL670) to end of study taking into account the therapeutic goal which will either be to maintain iron balance or to induce negative iron balance. End of study taken as the mean of, at most, the last three available results after start of treatment with ICL670.

Posted

Mean

Standard Deviation

μg/L

Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)

ID	Title	Description
OG000	Crossover	Participants treated with Deferoxamine (DFO) during the core study and crossed over to receive Deferasirox (ICL670) orally once a day during the extension study. Dosage based on body weight.
OG001	ICL670	Participants treated with Deferasirox (ICL670) orally once a day during the core study and continued this treatment in the extension study. Dosage based on body weight.

Secondary

Change in Surrogate Marker: Serum Transferrin From Start of Treatment With ICL670 to End of Study

Measurement of the relative change in percent of potential surrogate marker: Serum Transferrin (g/L) from start of treatment with Deferasirox (ICL670) to end of study.

(Serum Transferrin at the End of Study-Serum Transferrin at Start of ICL670)/Serum Transferrin at Start of ICL670*100.

Posted

Mean

Standard Deviation

Percent change

Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)

ID	Title	Description
OG000	Crossover	Participants treated with Deferoxamine (DFO) during the core study and crossed over to receive Deferasirox (ICL670) orally once a day during the extension study. Dosage based on body weight.
OG001	ICL670	Participants treated with Deferasirox (ICL670) orally once a day during the core study and continued this treatment in the extension study. Dosage based on body weight.

Secondary

Change in Surrogate Marker: Serum Iron From Start of Treatment With ICL670 to End of Study

Measurement of the relative change of potential surrogate markers: Serum Iron (µmol/L) from start of treatment with Deferasirox (ICL670) to end of study.

(Serum Iron at the End of Study-Serum Iron at Start of ICL670)/Serum Iron at Start of ICL670*100.

Posted

Mean

Standard Deviation

Percent change

Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)

ID	Title	Description
OG000	Crossover	Participants treated with Deferoxamine (DFO) during the core study and crossed over to receive Deferasirox (ICL670) orally once a day during the extension study. Dosage based on body weight.
OG001	ICL670	Participants treated with Deferasirox (ICL670) orally once a day during the core study and continued this treatment in the extension study. Dosage based on body weight.

Secondary

Change in Surrogate Marker: Transferrin Saturation From Start of Treatment With ICL670 to End of Study

Measurement of the relative change of potential surrogate marker: Transferrin Saturation (Percent) from start of treatment with Deferasirox (ICL670) to end of study.

(Transferrin Saturation at the End of Study-Tranferrin Saturation at Start of ICL670)/Transferrin Saturation at Start of ICL670*100.

Posted

Mean

Standard Deviation

Percent change

Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)

ID	Title	Description
OG000	Crossover	Participants treated with Deferoxamine (DFO) during the core study and crossed over to receive Deferasirox (ICL670) orally once a day during the extension study. Dosage based on body weight.
OG001	ICL670	Participants treated with Deferasirox (ICL670) orally once a day during the core study and continued this treatment in the extension study. Dosage based on body weight.

Secondary

Absolute Change in Liver Iron Content From Start of ICL670 Treatment to End of Study Measured by Biopsy

Measurement of median absolute change in liver iron content (LIC) from start of treatment with Deferasirox (ICL670) to end of study obtained through biopsy. Absolute change = End of study value - start of treatment value. LIC is expressed in mg of iron per gram of liver dry weight (mg Fe/g dw).

Posted

Median

Full Range

mg Fe/g dw

Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)

ID	Title	Description
OG000	Crossover	Participants treated with Deferoxamine (DFO) during the core study and crossed over to receive Deferasirox (ICL670) orally once a day during the extension study. Dosage based on body weight.
OG001	ICL670	Participants treated with Deferasirox (ICL670) orally once a day during the core study and continued this treatment in the extension study. Dosage based on body weight.

Secondary

Relative Change in Liver Iron Content From Start of ICL670 Treatment to End of Study Measured by Biopsy

Relative change in liver iron content (LIC) as measured by biopsy and calculated by: End of study value - Start of ICL670 treatment value (absolute change) / Start of ICL670 treatment value.

Posted

Median

Full Range

percent of start value

Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)

ID	Title	Description
OG000	Crossover	Participants treated with Deferoxamine (DFO) during the core study and crossed over to receive Deferasirox (ICL670) orally once a day during the extension study. Dosage based on body weight.
OG001	ICL670	Participants treated with Deferasirox (ICL670) orally once a day during the core study and continued this treatment in the extension study. Dosage based on body weight.

Units

Secondary

Absolute Change in Liver Iron Content From Start of ICL670 Treatment to End of Study Measured by SQUID

Measurement of the median absolute change in liver iron content (LIC) from start of treatment with Deferasirox (ICL670) to end of study obtained through Superconducting Quantum Interfering Device (SQUID). Absolute change = End of study value - start of treatment value. LIC is expressed in mg of iron per gram of liver dry weight (mg Fe/g dw).

Population includes only those participants from the full analysis set (defined as all participants who received at least 1 dose of ICL670 in the core/extension study, that is, analyses also included ICL670 participants from the core group who did not continue into extension) and had LIC SQUID data at Start of ICL670 treatment and End of Study.

Posted

Median

Full Range

mg Fe/g dw

Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)

ID	Title	Description
OG000	Crossover	Participants treated with Deferoxamine (DFO) during the core study and crossed over to receive Deferasirox (ICL670) orally once a day during the extension study. Dosage based on body weight.
OG001	ICL670	Participants treated with Deferasirox (ICL670) orally once a day during the core study and continued this treatment in the extension study. Dosage based on body weight.

Secondary

Relative Change in Liver Iron Content From Start of ICL670 Treatment to End of Study as Measured by SQUID

Relative change in liver iron content (LIC) measured by Superconducting Quantum Interfering Device (SQUID), calculated by: End of study value - Start of ICL670 treatment value (absolute change) / Start of ICL670 treatment value.

Population includes only those participants from the full analysis set (defined as all participants who received at least 1 dose of ICL670 in the core/extension study, that is, analyses also included ICL670 participants from the core group who did not continue into extension) and had LIC SQUID data at Start of ICL670 treatment and End of Study.

Posted

Median

Full Range

percent of start value

Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)

ID	Title	Description
OG000	Crossover	Participants treated with Deferoxamine (DFO) during the core study and crossed over to receive Deferasirox (ICL670) orally once a day during the extension study. Dosage based on body weight.
OG001	ICL670	Participants treated with Deferasirox (ICL670) orally once a day during the core study and continued this treatment in the extension study. Dosage based on body weight.

Secondary

Change of Total Body Iron Excretion Rate (TBIE) From Start of ICL670 Treatment to the End of Study

Median change in TBIE (mg/kg/day) from start of treatment with Deferasirox (ICL670) to end of study.

Posted

Median

Full Range

mg/kg/day

Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)

ID	Title	Description
OG000	Crossover	Participants treated with Deferoxamine (DFO) during the core study and crossed over to receive Deferasirox (ICL670) orally once a day during the extension study. Dosage based on body weight.
OG001	ICL670	Participants treated with Deferasirox (ICL670) orally once a day during the core study and continued this treatment in the extension study. Dosage based on body weight.

Units	Counts
Participants

Serious Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Anaemia	Blood and lymphatic system disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0012 affected259 at risk
Anaemia haemolytic autoimmune	Blood and lymphatic system disorders	MedDRA	Systematic Assessment	EG0002 affected296 at risk EG0010 affected259 at risk
Haemolysis	Blood and lymphatic system disorders	MedDRA	Systematic Assessment	EG0000 affected296 at risk EG0011 affected259 at risk
Hypersplenism	Blood and lymphatic system disorders	MedDRA	Systematic Assessment	EG0008 affected296 at risk EG0016 affected259 at risk
Leukopenia	Blood and lymphatic system disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Lymphadenopathy	Blood and lymphatic system disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Neutropenia	Blood and lymphatic system disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0011 affected259 at risk
Neutrophilia	Blood and lymphatic system disorders	MedDRA	Systematic Assessment	EG0000 affected296 at risk EG0011 affected259 at risk
Splenomegaly	Blood and lymphatic system disorders	MedDRA	Systematic Assessment	EG0005 affected296 at risk EG0011 affected259 at risk
Arrhythmia	Cardiac disorders	MedDRA	Systematic Assessment	EG0002 affected296 at risk EG0010 affected259 at risk
Atrial flutter	Cardiac disorders	MedDRA	Systematic Assessment	EG0002 affected296 at risk EG0010 affected259 at risk
Cardiac failure	Cardiac disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Cardiac failure congestive	Cardiac disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Cardio-respiratory arrest	Cardiac disorders	MedDRA	Systematic Assessment	EG0000 affected296 at risk EG0011 affected259 at risk
Cardiogenic shock	Cardiac disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Myocardial infarction	Cardiac disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Tachycardia	Cardiac disorders	MedDRA	Systematic Assessment	EG0000 affected296 at risk EG0011 affected259 at risk
Ventricular tachycardia	Cardiac disorders	MedDRA	Systematic Assessment	EG0002 affected296 at risk EG0010 affected259 at risk
Accessory spleen	Congenital, familial and genetic disorders	MedDRA	Systematic Assessment	EG0000 affected296 at risk EG0011 affected259 at risk
Thalassaemia beta	Congenital, familial and genetic disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0011 affected259 at risk
Vertigo	Ear and labyrinth disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Cataract	Eye disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Lenticular opacities	Eye disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0011 affected259 at risk
Papilloedema	Eye disorders	MedDRA	Systematic Assessment	EG0000 affected296 at risk EG0011 affected259 at risk
Vision blurred	Eye disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Abdominal pain	Gastrointestinal disorders	MedDRA	Systematic Assessment	EG0008 affected296 at risk EG0014 affected259 at risk
Abdominal pain lower	Gastrointestinal disorders	MedDRA	Systematic Assessment	EG0000 affected296 at risk EG0011 affected259 at risk
Abdominal pain upper	Gastrointestinal disorders	MedDRA	Systematic Assessment	EG0002 affected296 at risk EG0010 affected259 at risk
Acute abdomen	Gastrointestinal disorders	MedDRA	Systematic Assessment	EG0000 affected296 at risk EG0012 affected259 at risk
Appendicitis perforated	Gastrointestinal disorders	MedDRA	Systematic Assessment	EG0000 affected296 at risk EG0011 affected259 at risk
Colitis	Gastrointestinal disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Diarrhoea	Gastrointestinal disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0011 affected259 at risk
Duodenal ulcer	Gastrointestinal disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Dyspepsia	Gastrointestinal disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Gastric ulcer	Gastrointestinal disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Gastrointestinal haemorrhage	Gastrointestinal disorders	MedDRA	Systematic Assessment	EG0000 affected296 at risk EG0011 affected259 at risk
Ileus	Gastrointestinal disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0011 affected259 at risk
Ileus paralytic	Gastrointestinal disorders	MedDRA	Systematic Assessment	EG0000 affected296 at risk EG0011 affected259 at risk
Inguinal hernia	Gastrointestinal disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Nausea	Gastrointestinal disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Oesophagitis	Gastrointestinal disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Pancreatitis	Gastrointestinal disorders	MedDRA	Systematic Assessment	EG0002 affected296 at risk EG0010 affected259 at risk
Pancreatitis acute	Gastrointestinal disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Peptic ulcer	Gastrointestinal disorders	MedDRA	Systematic Assessment	EG0002 affected296 at risk EG0010 affected259 at risk
Peritoneal haemorrhage	Gastrointestinal disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Peritonitis	Gastrointestinal disorders	MedDRA	Systematic Assessment	EG0000 affected296 at risk EG0011 affected259 at risk
Varices oesophageal	Gastrointestinal disorders	MedDRA	Systematic Assessment	EG0000 affected296 at risk EG0011 affected259 at risk
Vomiting	Gastrointestinal disorders	MedDRA	Systematic Assessment	EG0004 affected296 at risk EG0010 affected259 at risk
Asthenia	General disorders	MedDRA	Systematic Assessment	EG0003 affected296 at risk EG0010 affected259 at risk
Chest pain	General disorders	MedDRA	Systematic Assessment	EG0002 affected296 at risk EG0010 affected259 at risk
Death	General disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Malaise	General disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Oedema peripheral	General disorders	MedDRA	Systematic Assessment	EG0000 affected296 at risk EG0011 affected259 at risk
Pain	General disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Pyrexia	General disorders	MedDRA	Systematic Assessment	EG00010 affected296 at risk EG0016 affected259 at risk
Bile duct stone	Hepatobiliary disorders	MedDRA	Systematic Assessment	EG0000 affected296 at risk EG0011 affected259 at risk
Biliary colic	Hepatobiliary disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Biliary tract disorder	Hepatobiliary disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Cholangitis	Hepatobiliary disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Cholecystitis	Hepatobiliary disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0011 affected259 at risk
Cholecystitis acute	Hepatobiliary disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Cholecystitis chronic	Hepatobiliary disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0011 affected259 at risk
Cholelithiasis	Hepatobiliary disorders	MedDRA	Systematic Assessment	EG0007 affected296 at risk EG0016 affected259 at risk
Cholestasis	Hepatobiliary disorders	MedDRA	Systematic Assessment	EG0000 affected296 at risk EG0011 affected259 at risk
Hepatitis	Hepatobiliary disorders	MedDRA	Systematic Assessment	EG0002 affected296 at risk EG0010 affected259 at risk
Hepatitis acute	Hepatobiliary disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0011 affected259 at risk
Jaundice	Hepatobiliary disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Jaundice cholestatic	Hepatobiliary disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0011 affected259 at risk
Liver injury	Hepatobiliary disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Acute tonsillitis	Infections and infestations	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Appendicitis	Infections and infestations	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0012 affected259 at risk
Bronchitis	Infections and infestations	MedDRA	Systematic Assessment	EG0003 affected296 at risk EG0010 affected259 at risk
Bronchopneumonia	Infections and infestations	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Escherichia sepsis	Infections and infestations	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Gastroenteritis	Infections and infestations	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0013 affected259 at risk
Gastroenteritis viral	Infections and infestations	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Gastrointestinal infection	Infections and infestations	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
HIV infection	Infections and infestations	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Hepatitis C	Infections and infestations	MedDRA	Systematic Assessment	EG0000 affected296 at risk EG0011 affected259 at risk
Infection	Infections and infestations	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Infectious mononucleosis	Infections and infestations	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Lower respiratory tract infection	Infections and infestations	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Lung abscess	Infections and infestations	MedDRA	Systematic Assessment	EG0000 affected296 at risk EG0011 affected259 at risk
Meningitis	Infections and infestations	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Meningitis bacterial	Infections and infestations	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Orchitis	Infections and infestations	MedDRA	Systematic Assessment	EG0000 affected296 at risk EG0011 affected259 at risk
Pharyngitis	Infections and infestations	MedDRA	Systematic Assessment	EG0002 affected296 at risk EG0011 affected259 at risk
Pharyngitis streptococcal	Infections and infestations	MedDRA	Systematic Assessment	EG0000 affected296 at risk EG0011 affected259 at risk
Pilonidal cyst	Infections and infestations	MedDRA	Systematic Assessment	EG0000 affected296 at risk EG0011 affected259 at risk
Pneumonia	Infections and infestations	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0011 affected259 at risk
Pneumonia viral	Infections and infestations	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Pyelocystitis	Infections and infestations	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Pyelonephritis acute	Infections and infestations	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Septic shock	Infections and infestations	MedDRA	Systematic Assessment	EG0002 affected296 at risk EG0010 affected259 at risk
Sinusitis	Infections and infestations	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Streptococcal bacteraemia	Infections and infestations	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Tonsillitis	Infections and infestations	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Tooth abscess	Infections and infestations	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Upper respiratory tract infection	Infections and infestations	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Urinary tract infection	Infections and infestations	MedDRA	Systematic Assessment	EG0003 affected296 at risk EG0010 affected259 at risk
Viral infection	Infections and infestations	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Wound infection	Infections and infestations	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Yersinia infection	Infections and infestations	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Ankle fracture	Injury, poisoning and procedural complications	MedDRA	Systematic Assessment	EG0000 affected296 at risk EG0011 affected259 at risk
Cervical vertebral fracture	Injury, poisoning and procedural complications	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Femoral neck fracture	Injury, poisoning and procedural complications	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Femur fracture	Injury, poisoning and procedural complications	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0012 affected259 at risk
Foot fracture	Injury, poisoning and procedural complications	MedDRA	Systematic Assessment	EG0000 affected296 at risk EG0011 affected259 at risk
Head injury	Injury, poisoning and procedural complications	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Heat stroke	Injury, poisoning and procedural complications	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Jaw fracture	Injury, poisoning and procedural complications	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Ligament rupture	Injury, poisoning and procedural complications	MedDRA	Systematic Assessment	EG0000 affected296 at risk EG0011 affected259 at risk
Limb injury	Injury, poisoning and procedural complications	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Lower limb fracture	Injury, poisoning and procedural complications	MedDRA	Systematic Assessment	EG0000 affected296 at risk EG0011 affected259 at risk
Post procedural haemorrhage	Injury, poisoning and procedural complications	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Procedural pain	Injury, poisoning and procedural complications	MedDRA	Systematic Assessment	EG0002 affected296 at risk EG0010 affected259 at risk
Road traffic accident	Injury, poisoning and procedural complications	MedDRA	Systematic Assessment	EG0002 affected296 at risk EG0011 affected259 at risk
Tendon rupture	Injury, poisoning and procedural complications	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Transfusion reaction	Injury, poisoning and procedural complications	MedDRA	Systematic Assessment	EG0002 affected296 at risk EG0010 affected259 at risk
Alanine aminotransferase increased	Investigations	MedDRA	Systematic Assessment	EG0000 affected296 at risk EG0011 affected259 at risk
Blood creatinine increased	Investigations	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Blood potassium decreased	Investigations	MedDRA	Systematic Assessment	EG0000 affected296 at risk EG0011 affected259 at risk
Blood sodium decreased	Investigations	MedDRA	Systematic Assessment	EG0000 affected296 at risk EG0011 affected259 at risk
Cardiovascular function test abnormal	Investigations	MedDRA	Systematic Assessment	EG0000 affected296 at risk EG0011 affected259 at risk
Cells in urine	Investigations	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Haemoglobin decreased	Investigations	MedDRA	Systematic Assessment	EG0000 affected296 at risk EG0011 affected259 at risk
Hepatic enzyme increased	Investigations	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Liver function test abnormal	Investigations	MedDRA	Systematic Assessment	EG0000 affected296 at risk EG0011 affected259 at risk
Serum ferritin increased	Investigations	MedDRA	Systematic Assessment	EG0000 affected296 at risk EG0011 affected259 at risk
Streptococcal identification test positive	Investigations	MedDRA	Systematic Assessment	EG0000 affected296 at risk EG0011 affected259 at risk
Transaminases increased	Investigations	MedDRA	Systematic Assessment	EG0003 affected296 at risk EG0012 affected259 at risk
Dehydration	Metabolism and nutrition disorders	MedDRA	Systematic Assessment	EG0000 affected296 at risk EG0011 affected259 at risk
Diabetes mellitus	Metabolism and nutrition disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Arthralgia	Musculoskeletal and connective tissue disorders	MedDRA	Systematic Assessment	EG0002 affected296 at risk EG0010 affected259 at risk
Back pain	Musculoskeletal and connective tissue disorders	MedDRA	Systematic Assessment	EG0002 affected296 at risk EG0011 affected259 at risk
Bone cyst	Musculoskeletal and connective tissue disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Bone lesion	Musculoskeletal and connective tissue disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Bone pain	Musculoskeletal and connective tissue disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Osteonecrosis	Musculoskeletal and connective tissue disorders	MedDRA	Systematic Assessment	EG0000 affected296 at risk EG0011 affected259 at risk
Osteoporotic fracture	Musculoskeletal and connective tissue disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Pain in extremity	Musculoskeletal and connective tissue disorders	MedDRA	Systematic Assessment	EG0000 affected296 at risk EG0011 affected259 at risk
Pathological fracture	Musculoskeletal and connective tissue disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Rheumatoid arthritis	Musculoskeletal and connective tissue disorders	MedDRA	Systematic Assessment	EG0000 affected296 at risk EG0011 affected259 at risk
Spinal osteoarthritis	Musculoskeletal and connective tissue disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Myolipoma	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	MedDRA	Systematic Assessment	EG0000 affected296 at risk EG0011 affected259 at risk
Pituitary tumour benign	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Cerebral haemorrhage	Nervous system disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Cerebral ischaemia	Nervous system disorders	MedDRA	Systematic Assessment	EG0000 affected296 at risk EG0011 affected259 at risk
Cerebrovascular disorder	Nervous system disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Clumsiness	Nervous system disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Depressed level of consciousness	Nervous system disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Dizziness	Nervous system disorders	MedDRA	Systematic Assessment	EG0002 affected296 at risk EG0010 affected259 at risk
Epilepsy	Nervous system disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Headache	Nervous system disorders	MedDRA	Systematic Assessment	EG0002 affected296 at risk EG0010 affected259 at risk
Loss of consciousness	Nervous system disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Psychomotor hyperactivity	Nervous system disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Syncope	Nervous system disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Abortion spontaneous	Pregnancy, puerperium and perinatal conditions	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Pregnancy	Pregnancy, puerperium and perinatal conditions	MedDRA	Systematic Assessment	EG0000 affected296 at risk EG0012 affected259 at risk
Depression	Psychiatric disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Dysthymic disorder	Psychiatric disorders	MedDRA	Systematic Assessment	EG0000 affected296 at risk EG0011 affected259 at risk
Insomnia	Psychiatric disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Suicide attempt	Psychiatric disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Calculus ureteric	Renal and urinary disorders	MedDRA	Systematic Assessment	EG0000 affected296 at risk EG0011 affected259 at risk
Calculus urinary	Renal and urinary disorders	MedDRA	Systematic Assessment	EG0002 affected296 at risk EG0010 affected259 at risk
Glomerulonephropathy	Renal and urinary disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Hydronephrosis	Renal and urinary disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Ketonuria	Renal and urinary disorders	MedDRA	Systematic Assessment	EG0000 affected296 at risk EG0011 affected259 at risk
Nephrolithiasis	Renal and urinary disorders	MedDRA	Systematic Assessment	EG0002 affected296 at risk EG0010 affected259 at risk
Nephropathy	Renal and urinary disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Proteinuria	Renal and urinary disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0011 affected259 at risk
Renal colic	Renal and urinary disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Renal failure	Renal and urinary disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Renal tubular disorder	Renal and urinary disorders	MedDRA	Systematic Assessment	EG0000 affected296 at risk EG0011 affected259 at risk
Acute pulmonary oedema	Respiratory, thoracic and mediastinal disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Cough	Respiratory, thoracic and mediastinal disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Dyspnoea	Respiratory, thoracic and mediastinal disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0011 affected259 at risk
Lung disorder	Respiratory, thoracic and mediastinal disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Wheezing	Respiratory, thoracic and mediastinal disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Dermal cyst	Skin and subcutaneous tissue disorders	MedDRA	Systematic Assessment	EG0000 affected296 at risk EG0011 affected259 at risk
Dermatitis allergic	Skin and subcutaneous tissue disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Dyshidrosis	Skin and subcutaneous tissue disorders	MedDRA	Systematic Assessment	EG0000 affected296 at risk EG0011 affected259 at risk
Henoch-Schonlein purpura	Skin and subcutaneous tissue disorders	MedDRA	Systematic Assessment	EG0000 affected296 at risk EG0011 affected259 at risk
Hyperhidrosis	Skin and subcutaneous tissue disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Rash	Skin and subcutaneous tissue disorders	MedDRA	Systematic Assessment	EG0002 affected296 at risk EG0010 affected259 at risk
Cholecystectomy	Surgical and medical procedures	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Splenectomy	Surgical and medical procedures	MedDRA	Systematic Assessment	EG0002 affected296 at risk EG0011 affected259 at risk
Deep vein thrombosis	Vascular disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk
Hypotension	Vascular disorders	MedDRA	Systematic Assessment	EG0001 affected296 at risk EG0010 affected259 at risk

Other Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Palpitations	Cardiac disorders	MedDRA	Systematic Assessment	EG00017 affected296 at risk EG00111 affected259 at risk
Ear pain	Ear and labyrinth disorders	MedDRA	Systematic Assessment	EG00016 affected296 at risk EG00114 affected259 at risk
Abdominal pain	Gastrointestinal disorders	MedDRA	Systematic Assessment	EG00074 affected296 at risk EG00157 affected259 at risk
Abdominal pain upper	Gastrointestinal disorders	MedDRA	Systematic Assessment	EG00051 affected296 at risk EG00152 affected259 at risk
Constipation	Gastrointestinal disorders	MedDRA	Systematic Assessment	EG00017 affected296 at risk EG00113 affected259 at risk
Diarrhoea	Gastrointestinal disorders	MedDRA	Systematic Assessment	EG00068 affected296 at risk EG00173 affected259 at risk
Dyspepsia	Gastrointestinal disorders	MedDRA	Systematic Assessment	EG00021 affected296 at risk EG00117 affected259 at risk
Nausea	Gastrointestinal disorders	MedDRA	Systematic Assessment	EG00062 affected296 at risk EG00152 affected259 at risk
Toothache	Gastrointestinal disorders	MedDRA	Systematic Assessment	EG00022 affected296 at risk EG00114 affected259 at risk
Vomiting	Gastrointestinal disorders	MedDRA	Systematic Assessment	EG00072 affected296 at risk EG00161 affected259 at risk
Asthenia	General disorders	MedDRA	Systematic Assessment	EG00029 affected296 at risk EG00131 affected259 at risk
Fatigue	General disorders	MedDRA	Systematic Assessment	EG00029 affected296 at risk EG00122 affected259 at risk
Influenza like illness	General disorders	MedDRA	Systematic Assessment	EG00012 affected296 at risk EG00117 affected259 at risk
Oedema peripheral	General disorders	MedDRA	Systematic Assessment	EG00017 affected296 at risk EG00115 affected259 at risk
Pyrexia	General disorders	MedDRA	Systematic Assessment	EG000110 affected296 at risk EG001108 affected259 at risk
Cholelithiasis	Hepatobiliary disorders	MedDRA	Systematic Assessment	EG00018 affected296 at risk EG0015 affected259 at risk
Acute tonsillitis	Infections and infestations	MedDRA	Systematic Assessment	EG00028 affected296 at risk EG00121 affected259 at risk
Bronchitis	Infections and infestations	MedDRA	Systematic Assessment	EG00042 affected296 at risk EG00141 affected259 at risk
Ear infection	Infections and infestations	MedDRA	Systematic Assessment	EG00022 affected296 at risk EG0017 affected259 at risk
Gastroenteritis	Infections and infestations	MedDRA	Systematic Assessment	EG00037 affected296 at risk EG00129 affected259 at risk
Influenza	Infections and infestations	MedDRA	Systematic Assessment	EG00070 affected296 at risk EG00165 affected259 at risk
Nasopharyngitis	Infections and infestations	MedDRA	Systematic Assessment	EG00073 affected296 at risk EG00156 affected259 at risk
Pharyngitis	Infections and infestations	MedDRA	Systematic Assessment	EG00054 affected296 at risk EG00157 affected259 at risk
Rhinitis	Infections and infestations	MedDRA	Systematic Assessment	EG00052 affected296 at risk EG00145 affected259 at risk
Sinusitis	Infections and infestations	MedDRA	Systematic Assessment	EG00021 affected296 at risk EG00113 affected259 at risk
Tonsillitis	Infections and infestations	MedDRA	Systematic Assessment	EG00029 affected296 at risk EG00122 affected259 at risk
Tooth abscess	Infections and infestations	MedDRA	Systematic Assessment	EG00015 affected296 at risk EG0018 affected259 at risk
Upper respiratory tract infection	Infections and infestations	MedDRA	Systematic Assessment	EG00052 affected296 at risk EG00132 affected259 at risk
Urinary tract infection	Infections and infestations	MedDRA	Systematic Assessment	EG00023 affected296 at risk EG00114 affected259 at risk
Transfusion reaction	Injury, poisoning and procedural complications	MedDRA	Systematic Assessment	EG00032 affected296 at risk EG00136 affected259 at risk
Blood creatinine increased	Investigations	MedDRA	Systematic Assessment	EG00046 affected296 at risk EG00122 affected259 at risk
Arthralgia	Musculoskeletal and connective tissue disorders	MedDRA	Systematic Assessment	EG00044 affected296 at risk EG00139 affected259 at risk
Back pain	Musculoskeletal and connective tissue disorders	MedDRA	Systematic Assessment	EG00059 affected296 at risk EG00149 affected259 at risk
Bone pain	Musculoskeletal and connective tissue disorders	MedDRA	Systematic Assessment	EG00015 affected296 at risk EG00114 affected259 at risk
Musculoskeletal pain	Musculoskeletal and connective tissue disorders	MedDRA	Systematic Assessment	EG00015 affected296 at risk EG0014 affected259 at risk
Osteoporosis	Musculoskeletal and connective tissue disorders	MedDRA	Systematic Assessment	EG00017 affected296 at risk EG00115 affected259 at risk
Pain in extremity	Musculoskeletal and connective tissue disorders	MedDRA	Systematic Assessment	EG00030 affected296 at risk EG00120 affected259 at risk
Headache	Nervous system disorders	MedDRA	Systematic Assessment	EG00084 affected296 at risk EG00181 affected259 at risk
Cough	Respiratory, thoracic and mediastinal disorders	MedDRA	Systematic Assessment	EG000110 affected296 at risk EG00185 affected259 at risk
Epistaxis	Respiratory, thoracic and mediastinal disorders	MedDRA	Systematic Assessment	EG0008 affected296 at risk EG00114 affected259 at risk
Nasal congestion	Respiratory, thoracic and mediastinal disorders	MedDRA	Systematic Assessment	EG00010 affected296 at risk EG00114 affected259 at risk
Oropharyngeal pain	Respiratory, thoracic and mediastinal disorders	MedDRA	Systematic Assessment	EG00062 affected296 at risk EG00150 affected259 at risk
Productive cough	Respiratory, thoracic and mediastinal disorders	MedDRA	Systematic Assessment	EG00015 affected296 at risk EG00112 affected259 at risk
Rash	Skin and subcutaneous tissue disorders	MedDRA	Systematic Assessment	EG00033 affected296 at risk EG00124 affected259 at risk
Urticaria	Skin and subcutaneous tissue disorders	MedDRA	Systematic Assessment	EG00023 affected296 at risk EG00114 affected259 at risk

ID	Term
D019189	Iron Metabolism Disorders
D008659	Metabolic Diseases
D009750	Nutritional and Metabolic Diseases

ID	Term
D001565	Benzoates
D000146	Acids, Carbocyclic
D002264	Carboxylic Acids
D009930	Organic Chemicals
D001555	Benzene Derivatives
D006841	Hydrocarbons, Aromatic
D006844	Hydrocarbons, Cyclic
D006838	Hydrocarbons
D014230	Triazoles
D001393	Azoles
D006573	Heterocyclic Compounds, 1-Ring
D006571	Heterocyclic Compounds

ID	Title	Description
BG000	Crossover	Participants treated with Deferoxamine (DFO) during the core study and crossed over to receive Deferasirox (ICL670) orally once a day during the extension study. Dosage based on body weight.
BG001	ICL670	Participants treated with Deferasirox (ICL670) orally once a day during the core study and continued this treatment in the extension study. Dosage based on body weight.
BG002	Total	Total of all reporting groups

ID	Title	Description	Deaths (Affected)	Deaths (At Risk)	Serious Events (Affected)	Serious Events (At Risk)	Other Events (Affected)	Other Events (At Risk)
EG000	ICL670	Participants treated with Deferasirox (ICL670) orally once a day during the core study continued this treatment in the extension study. Dosage based on body weight.			102	296	266	296
EG001	Crossover	Participants treated with Deferoxamine (DFO) during the core study and crossed over to receive Deferasirox (ICL670) orally once a day during the extension study. Dosage based on body weight.			61	259	236	259