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Trial record 13 of 22 for:    "Hemosiderosis" | "Chelating Agents"

Evaluating Use of Deferasirox as Compared to Deferoxamine in Treating Cardiac Iron Overload (CORDELIA)

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ClinicalTrials.gov Identifier: NCT00600938
Recruitment Status : Completed
First Posted : January 25, 2008
Results First Posted : August 27, 2014
Last Update Posted : August 27, 2014
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Transfusional Iron Overload
Transfusional Hemosiderosis
Interventions Drug: Core Study: Deferasirox
Drug: Core Study: Deferoxamine
Drug: Extension: deferoxamine to deferasirox
Drug: Extension: deferasirox to deferoxamine
Drug: Deferasirox
Drug: Deferoxamine
Enrollment 197
Recruitment Details  
Pre-assignment Details Patients randomized to 1 of 2 treatment grps ICL or DFO, 20 mg/kg/day once daily for 2 wks, followed by 30 mg/kg/day od for 1 wk and 40 mg/kg/day od (DFO) target dose of 50 to 60 mg/kg/day 8 to 12 hour for 5 to 7 days/wk. In ext. pts could either stay in the same grp from core or could switch grps: ICL to ICL, DFO to DFO, DFO to ICL and ICL to DFO
Arm/Group Title Deferasirox (ICL). For Extension Labeled as ICL to ICL Deferoxamine (DFO). For Extension Labeled as DFO to DFO DFO to ICL (Deferoxamine to Deferasirox) ICL to DFO (Deferasirox to Deferoxamine)
Hide Arm/Group Description 20 mg/kg/day once daily (od) for 2 weeks, followed by 30 mg/kg/day od for 1 week and a subsequent continuation of 40 mg/kg/day 50 mg/kg/day to 60 mg/kg/day infused subcutaneously in 8- to 12-hour intervals administered 5 to 7 days/week DFO to ICL” (patients who switched from DFO to deferasirox in extension) ICL to DFO” (patients who switched from deferasirox to DFO in extension)
Period Title: Core Study
Started 98 99 0 0
Core Safety Set 96 91 0 0
Per Protocol Set (PPS) 91 81 0 0
Completed 82 78 0 0
Not Completed 16 21 0 0
Reason Not Completed
Abnormal test procedure result(s)             3             2             0             0
Unsatisfactory therapeutic effect             1             2             0             0
Patient withdrew consent             7             12             0             0
Lost to Follow-up             3             2             0             0
Death             1             1             0             0
Protocol Violation             1             2             0             0
Period Title: Extension Study
Started 74 [1] 29 [2] 42 [2] 1 [1]
Extension Safety Set 73 [3] 29 42 1
Completed 65 24 33 0
Not Completed 9 5 9 1
Reason Not Completed
Adverse Event             1             0             2             0
Abnormal test procedure results             0             1             1             1
Unsatisfactory therapeutic effect             4             1             1             0
Withdrawal by Subject             2             2             3             0
Lost to Follow-up             1             1             2             0
Death             1             0             0             0
[1]
Of the 82 patients who completed the ICL core, 74 continued on the ICL ext. phase; 1 went to ICL-DFO
[2]
Of the 78 patients who completed the DFO core, 29 went on DFO ext. phase; 42 went to the DFO-ICL
[3]
1 patient did not receive drug in the ext. phase but received drug in the core phase
Arm/Group Title Core: Deferasirox (ICL) Core: Deferoxamine (DFO) Total
Hide Arm/Group Description 20 mg/kg/day once daily (od) for 2 weeks, followed by 30 mg/kg/day od for 1 week and a subsequent continuation of 40 mg/kg/day 50 mg/kg/day to 60 mg/kg/day infused subcutaneously in 8- to 12-hour intervals administered 5 to 7 days/week Total of all reporting groups
Overall Number of Baseline Participants 98 99 197
Hide Baseline Analysis Population Description
Per Protocol Set (PPS): randomized patients who received at least 6 months of study drug, had a T2* value assessed at least 150 days after randomization, and had no major protocol deviations. A total of 25 patients (7 in the deferasirox arm and 18 in the DFO arm) were excluded from PPS
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 98 participants 99 participants 197 participants
19.9  (6.53) 19.7  (6.32) 19.8  (6.41)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 98 participants 99 participants 197 participants
Female
40
  40.8%
42
  42.4%
82
  41.6%
Male
58
  59.2%
57
  57.6%
115
  58.4%
1.Primary Outcome
Title Core Study: Change From Baseline in Myocardial T2* (Magnetic Resonance T2-star (T2*) Technique for the Measurement of Tissue Iron) After 12 Months Treatment
Hide Description Non- inferiority in efficacy of deferasirox compared to deferoxamine (DFO) in treating cardiac iron overload as measured by T2*. A non-inferiority margin of 0.9 (90%) was applied. Due to limitations in performing heart biopsies, T2* (T2 star), a Magnetic Resonance (MR) relaxation parameter expressed in milliseconds, as is an important tool to noninvasively quantify cardiac iron concentration. Studies have shown that myocardial T2* evaluations may predict cardiac events, e.g., impaired (<56%) left ventricular ejection fraction (LVEF) is prevalent among patients with low T2*: found in 62% of patients with T2*<8 ms; 20% with T2* of 8-12 ms; and in 5% with T2* >12 ms (Tanner 2006)
Time Frame 12 Month
Hide Outcome Measure Data
Hide Analysis Population Description
Per Protocol Set (PPS) consisted of all randomized patients who received at least 6 months of randomized study drug, had a T2* value assessed at least 150 days after randomization, adhered to the major inclusion and none of the major exclusion criteria, and had no major protocol deviations.
Arm/Group Title Core: Deferasirox (ICL) Core: Deferoxamine (DFO)
Hide Arm/Group Description:
20 mg/kg/day once daily (od) for 2 weeks, followed by 30 mg/kg/day od for 1 week and a subsequent continuation of 40 mg/kg/day for 12 months.
50 mg/kg/day to 60 mg/kg/day infused subcutaneously in 8- to 12-hour intervals administered 5 to 7 days/week for 12 months.
Overall Number of Participants Analyzed 91 81
Geometric Mean (95% Confidence Interval)
Unit of Measure: Millisecond
1.12
(1.07 to 1.18)
1.07
(1.02 to 1.11)
2.Secondary Outcome
Title Core Study: Cardiac Function After 12 Months of Treatment With Deferasirox vs. Deferoxamine, by Change in Left Ventricular Ejection Fraction (LVEF)
Hide Description An absolute change from baseline in LVEF after 12 months treatment with deferasirox and compared to.DFO was tested using an analysis of covariance model including baseline left ventricular ejection fraction (LVEF) as a covariate.
Time Frame 12 Month
Hide Outcome Measure Data
Hide Analysis Population Description
Per Protocol Set (PPS) consisted of all randomized patients who received at least 6 months of randomized study drug, had a T2* value assessed at least 150 days after randomization, adhered to the major inclusion and none of the major exclusion criteria, and had no major protocol deviations.
Arm/Group Title Core: Deferasirox (ICL). For Extension Labeled as ICL to ICL Core: Deferoxamine (DFO). For Extension Labeled as DFO to DFO
Hide Arm/Group Description:
20 mg/kg/day once daily (od) for 2 weeks, followed by 30 mg/kg/day od for 1 week and a subsequent continuation of 40 mg/kg/day for 12 month
50 mg/kg/day to 60 mg/kg/day infused subcutaneously in 8- to 12-hour intervals administered 5 to 7 days/week for 12 month
Overall Number of Participants Analyzed 91 81
Least Squares Mean (Standard Error)
Unit of Measure: Percent
-0.5  (0.47) -0.0  (0.49)
3.Secondary Outcome
Title Core Study: Cardiac Function After 6 Months of Treatment With Deferasirox vs. Deferoxamine, by Change in Left Ventricular Ejection Fraction (LVEF)
Hide Description An absolute change from baseline in LVEF after 6 months treatment with deferasirox and DFO was summarized
Time Frame 6 Month
Hide Outcome Measure Data
Hide Analysis Population Description
Per Protocol Set (PPS) consisted of all randomized patients who received at least 6 months of randomized study drug, had a T2* value assessed at least 150 days after randomization, adhered to the major inclusion and none of the major exclusion criteria, and had no major protocol deviations
Arm/Group Title Core: Deferasirox (ICL). For Extension Labeled as ICL to ICL Deferoxamine (DFO). For Extension Labeled as DFO to DFO
Hide Arm/Group Description:
20 mg/kg/day once daily (od) for 2 weeks, followed by 30 mg/kg/day od for 1 week and a subsequent continuation of 40 mg/kg/day for 12 month
50 mg/kg/day to 60 mg/kg/day infused subcutaneously in 8- to 12-hour intervals administered 5 to 7 days/week
Overall Number of Participants Analyzed 85 73
Mean (Standard Deviation)
Unit of Measure: Percent
-0.95  (4.485) -0.37  (4.389)
4.Secondary Outcome
Title Core Study: Change From Baseline in Myocardial T2* After 6 Months Treatment
Hide Description Summary statistics of T2* ratio Month 6/baseline
Time Frame 6 Month
Hide Outcome Measure Data
Hide Analysis Population Description
Per Protocol Set (PPS) consisted of all randomized patients who received at least 6 months of randomized study drug, had a T2* value assessed at least 150 days after randomization, adhered to the major inclusion and none of the major exclusion criteria, and had no major protocol deviations
Arm/Group Title Core: Deferasirox (ICL). For Extension Labeled as ICL to ICL Core: Deferoxamine (DFO). For Extension Labeled as DFO to DFO
Hide Arm/Group Description:
20 mg/kg/day once daily (od) for 2 weeks, followed by 30 mg/kg/day od for 1 week and a subsequent continuation of 40 mg/kg/day for 12 month
50 mg/kg/day to 60 mg/kg/day infused subcutaneously in 8- to 12-hour intervals administered 5 to 7 days/week for 12 month
Overall Number of Participants Analyzed 85 73
Geometric Mean (95% Confidence Interval)
Unit of Measure: Ratio
1.04
(1.00 to 1.08)
1.04
(1.00 to 1.08)
5.Secondary Outcome
Title Core Study: Cardiac Function After 6 and 12 Months Treatment With Deferasirox vs. Deferoxamine, by Change in Left Ventricular End Systolic Volume Indices (LVESVI)
Hide Description An absolute change from baseline in LVESVI after 6 and 12 months treatment with deferasirox and DFO was summarized. Changes in cardiovascular magnetic resonance (CMR) measured left ventricular end systolic after 6 and 12 months treatment. Left ventricular (LV) end-systolic volume indexed to body surface area (ESVI) is a simple yet powerful echocardiographic marker of LV remodeling that can be measured easily. Left ventricular (LV) end-systolic volume (ESV) has been shown to be an important determinant of survival after myocardial infarction (MI)
Time Frame 6 Month, 12 Month
Hide Outcome Measure Data
Hide Analysis Population Description
Per Protocol Set (PPS) consisted of all randomized patients who received at least 6 months of randomized study drug, had a T2* value assessed at least 150 days after randomization, adhered to the major inclusion and none of the major exclusion criteria, and had no major protocol deviations
Arm/Group Title Core: Deferasirox (ICL). For Extension Labeled as ICL to ICL Core: Deferoxamine (DFO). For Extension Labeled as DFO to DFO
Hide Arm/Group Description:
20 mg/kg/day once daily (od) for 2 weeks, followed by 30 mg/kg/day od for 1 week and a subsequent continuation of 40 mg/kg/day for 12 month
50 mg/kg/day to 60 mg/kg/day infused subcutaneously in 8- to 12-hour intervals administered 5 to 7 days/week for 12 month
Overall Number of Participants Analyzed 91 81
Mean (Standard Deviation)
Unit of Measure: Milliliter
Change from baseline at 6 Month (n= 85, 73) 1.8  (8.021) 0.88  (8.919)
Change from Baseline at 12 Month/EOS (n= 91, 81) 1.57  (8.040) 0.10  (10.387)
6.Secondary Outcome
Title Core Study: Core Study: Cardiac Function After 6 and 12 Months of Treatment With Deferasirox vs. Deferoxamine, by Change in Left Ventricular End Diastolic Volume Indices (LVEDVI)
Hide Description An absolute change from baseline in LVEDVI after 6, and 12 months treatment with deferasirox and DFO was summarized
Time Frame 6 Month, 12 Month
Hide Outcome Measure Data
Hide Analysis Population Description
Per Protocol Set (PPS) consisted of all randomized patients who received at least 6 months of randomized study drug, had a T2* value assessed at least 150 days after randomization, adhered to the major inclusion and none of the major exclusion criteria, and had no major protocol deviations
Arm/Group Title Core; Deferasirox (ICL). For Extension Labeled as ICL to ICL Core: Deferoxamine (DFO). For Extension Labeled as DFO to DFO
Hide Arm/Group Description:
20 mg/kg/day once daily (od) for 2 weeks, followed by 30 mg/kg/day od for 1 week and a subsequent continuation of 40 mg/kg/day for 12 month
50 mg/kg/day to 60 mg/kg/day infused subcutaneously in 8- to 12-hour intervals administered 5 to 7 days/week
Overall Number of Participants Analyzed 91 81
Mean (Standard Deviation)
Unit of Measure: Percent
Change from Baseline at 6 Month (n= 85, 73) 1.81  (14.515) 1.48  (19.188)
Change from Baseline at 12 Month/EOS (n= 91, 81) 1.79  (13.122) 1.10  (20.485)
7.Secondary Outcome
Title Core Study: Cardiac Function After 6 and 12 Months of Treatment With Deferasirox vs. Deferoxamine, by Change in Left Ventricular Mass Indices (LVMI)
Hide Description An absolute change from baseline in LVMI after 6, and 12 months treatment with deferasirox and DFO was summarized
Time Frame 6 Month, 12 Month
Hide Outcome Measure Data
Hide Analysis Population Description
Per Protocol Set (PPS) consisted of all randomized patients who received at least 6 months of randomized study drug, had a T2* value assessed at least 150 days after randomization, adhered to the major inclusion and none of the major exclusion criteria, and had no major protocol deviations
Arm/Group Title Core: Deferasirox (ICL). For Extension Labeled as ICL to ICL Core: Deferoxamine (DFO). For Extension Labeled as DFO to DFO
Hide Arm/Group Description:
20 mg/kg/day once daily (od) for 2 weeks, followed by 30 mg/kg/day od for 1 week and a subsequent continuation of 40 mg/kg/day for 12 month
50 mg/kg/day to 60 mg/kg/day infused subcutaneously in 8- to 12-hour intervals administered 5 to 7 days/week for 12 month
Overall Number of Participants Analyzed 91 81
Mean (Standard Deviation)
Unit of Measure: gram/m^2
Change from Baseline at 6 Month (n= 85, 73) 1.01  (13.102) 3.32  (13.585)
Change from Baseline at 12 Month/EOS (n= 91, 81) 4.13  (15.055) 5.25  (14.973)
8.Secondary Outcome
Title Core Study: Cardiac Function and the Proportion of Patients Dropping Out Due to Cardiac Dysfunction After Treatment With Deferasirox vs. Deferoxamine
Hide Description The number of patients withdrawn from the study due to LVEF <50%, T2* <6 ms or significant decreases in T2* ≥ 33% from baseline was provided per treatment group.
Time Frame 12 Month
Hide Outcome Measure Data
Hide Analysis Population Description
Per Protocol Set (PPS) consisted of all randomized patients who received at least 6 months of randomized study drug, had a T2* value assessed at least 150 days after randomization, adhered to the major inclusion and none of the major exclusion criteria, and had no major protocol deviations
Arm/Group Title Core: Deferasirox (ICL). For Extension Labeled as ICL to ICL Core; Deferoxamine (DFO). For Extension Labeled as DFO to DFO
Hide Arm/Group Description:
20 mg/kg/day once daily (od) for 2 weeks, followed by 30 mg/kg/day od for 1 week and a subsequent continuation of 40 mg/kg/day for 12 month
50 mg/kg/day to 60 mg/kg/day infused subcutaneously in 8- to 12-hour intervals administered 5 to 7 days/week for 12 month
Overall Number of Participants Analyzed 98 99
Measure Type: Number
Unit of Measure: Participants
3 2
9.Secondary Outcome
Title Core Study: Safety and Tolerability of Deferasirox vs Deferoxamine Over the 12 Months Treatment Period.
Hide Description Number of patients with adverse events, serious adverse events and death
Time Frame 12 Month
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Set (SS) consisted of all randomized patients who received at least one dose of study drug and had at least one post-baseline safety assessment. Patients were analyzed according to treatment received. Treatment received is defined as first study drug administered
Arm/Group Title Core: Deferasirox (ICL). For Extension Labeled as ICL to ICL Core: Deferoxamine (DFO). For Extension Labeled as DFO to DFO
Hide Arm/Group Description:
20 mg/kg/day once daily (od) for 2 weeks, followed by 30 mg/kg/day od for 1 week and a subsequent continuation of 40 mg/kg/day
50 mg/kg/day to 60 mg/kg/day infused subcutaneously in 8- to 12-hour intervals administered 5 to 7 days/week for 12 month
Overall Number of Participants Analyzed 96 91
Measure Type: Number
Unit of Measure: Participants
At least one AE 65 69
Serious Adverse Events 10 10
Death. None were considered related to study drug. 1 1
10.Secondary Outcome
Title Core Study: Single and Repeated Dose Pharmacokinetics of Deferasirox, Area Under the Plasma Concentration-time Curve for a Dosing Interval (AUCtau)
Hide Description The plasma level of deferasirox (ICL670) obtained in this study was summarized descriptively. Plasma concentration was plotted by patient and by visit. Descriptive statistics included the mean, median, SD, and CV, min and max. deferasirox pharmacokinetics (PK) trough levels over the 12 months of treatment and obtained PK profiles for the 40 mg/kg/day deferasirox dose, area under the plasma concentration-time curve for a dosing interval (AUCtau)
Time Frame 12 Month
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic Analysis Set (PAS): PAS 2: All randomized patients who received the same dose of deferasirox for at least four consecutive days prior to PK sample collection and completed PK sample collection specified in the protocol at Visit 3 or Visit 4 (pre-dose, 1, 2, and 4 hours post-dose).
Arm/Group Title Deferasirox (ICL). For Extension Labeled as ICL to ICL
Hide Arm/Group Description:
20 mg/kg/day once daily (od) for 2 weeks, followed by 30 mg/kg/day od for 1 week and a subsequent continuation of 40 mg/kg/day
Overall Number of Participants Analyzed 13
Mean (Standard Deviation)
Unit of Measure: (h.ng/mL)
2129.70  (930.202)
11.Secondary Outcome
Title Core Study: Single and Repeated Dose Pharmacokinetics of Deferasirox, Maximum Plasma Concentration (Cmax)
Hide Description The plasma level of deferasirox (ICL670) obtained in this study was summarized descriptively. Plasma concentration was plotted by patient and by visit. Descriptive statistics included the mean, median, SD, and CV, min and max. deferasirox pharmacokinetics (PK) trough levels over the 12 months of treatment and obtained PK profiles for the 40 mg/kg/day deferasirox dose, maximum plasma concentration (Cmax)
Time Frame 12 Month
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic Analysis Set (PAS): PAS 2: All randomized patients who received the same dose of deferasirox for at least four consecutive days prior to PK sample collection and completed PK sample collection specified in the protocol at Visit 3 or Visit 4 (pre-dose, 1, 2, and 4 hours post-dose).
Arm/Group Title Deferasirox (ICL). For Extension Labeled as ICL to ICL
Hide Arm/Group Description:
20 mg/kg/day once daily (od) for 2 weeks, followed by 30 mg/kg/day od for 1 week and a subsequent continuation of 40 mg/kg/day
Overall Number of Participants Analyzed 13
Mean (Standard Deviation)
Unit of Measure: umol/L
150.09  (59.143)
12.Secondary Outcome
Title Core Study: Single and Repeated Dose Pharmacokinetics of Deferasirox, Time Points of Concentration Data
Hide Description The plasma level of deferasirox (ICL670) obtained in this study was summarized descriptively. Plasma concentration was plotted by patient and by visit. For trough concentration assessments, a 2-mL blood sample was to be taken on arrival at the study site, i.e. prior to the patient receiving the daily deferasirox dose (pre-dose blood sample). A second 2-mL blood sample was to be taken 2 hours later (post-dose sample). At all other visits (Visits 3 - 14), a pre-dose sample was to be taken. For PK profile assessments, 3 blood samples were taken after 1, 2, and 4 hours post-dose in addition to the 2-mL pre-dose
Time Frame Month 1 and month 2 (pre-dose, 1,2 and 4 hours post-dose)
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic Analysis Set (PAS): PAS 2: All randomized patients who received the same dose of deferasirox for at least four consecutive days prior to PK sample collection and completed PK sample collection specified in the protocol at Visit 3 or Visit 4 (pre-dose, 1, 2, and 4 hours post-dose).
Arm/Group Title Deferasirox (ICL). For Extension Labeled as ICL to ICL
Hide Arm/Group Description:
20 mg/kg/day once daily (od) for 2 weeks, followed by 30 mg/kg/day od for 1 week and a subsequent continuation of 40 mg/kg/day
Overall Number of Participants Analyzed 13
Mean (Standard Deviation)
Unit of Measure: (umol/L)
Month 1, 0 hour (predose) 32.25  (21.288)
Month 1, 1 hour (post dos) 96.32  (35.799)
Month 1, 2 hour (post dose) 136.47  (51.831)
Month 1, 4 hour (post dose) 133.33  (62.815)
Month 2, 0 hour (predose) 38.66  (29.887)
Month 2, 1 hour (post dose) 119.48  (38.709)
Month 2, 2 hour (post dose) 177.19  (55.343)
Month 2, 4 hour (post dose) 180.76  (57.877)
13.Secondary Outcome
Title Extension Study: Change From Baseline in Myocardial T2* After 24 Months Treatment
Hide Description The measured T2* values, the ratio (post-baseline / baseline T2*) at Month 6, 12, 18 and 24 was summarized for FAS population along with two-sided 95% CIs. The geometric means of the ratio was presented for all treatment groups
Time Frame Months 6, 12, 18 and 24
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): consisted of all patients enrolled in the extension. Patients were analyzed according to the treatment they were assigned to in the beginning of the extension phase.
Arm/Group Title Extension : ICL to ICL Extension: DFO to DFO Extension; DFO to ICL Extension: ICL to DFO
Hide Arm/Group Description:
Patients from core continued and received same dose 20 mg/kg/day once daily (od) for 2 weeks, followed by 30 mg/kg/day od for 1 week and a subsequent continuation of 40 mg/kg/day
Patients from core continued and received same 50 mg/kg/day to 60 mg/kg/day infused subcutaneously in 8- to 12-hour intervals administered 5 to 7 days/week
DFO to ICL” (patients who switched from DFO to deferasirox in extension)
ICL to DFO” (patients who switched from deferasirox to DFO in extension)
Overall Number of Participants Analyzed 74 29 42 1
Geometric Mean (95% Confidence Interval)
Unit of Measure: Ratio
Month 6 (n=71,26,40,1)
1.06
(1.01 to 1.11)
1.05
(0.98 to 1.12)
1.03
(0.98 to 1.09)
1.00 [1] 
(NA to NA)
Month 12(n=66, 29, 41, 1)
1.17
(1.11 to 1.24)
1.06
(0.97 to 1.15)
1.07
(1.01 to 1.14)
1.17 [1] 
(NA to NA)
Month 18 (n=68, 26, 39, 1)
1.24
(1.16 to 1.34)
1.18
(1.05 to 1.32)
1.13
(1.04 to 1.23)
1.05 [1] 
(NA to NA)
Month 24 (n=63, 25, 33, 1)
1.38
(1.28 to 1.49)
1.33
(1.13 to 1.55)
1.21
(1.09 to 1.34)
1.11 [1] 
(NA to NA)
[1]
95CI is not available for ICL to DFO group as there is only 1 patient.
14.Secondary Outcome
Title Extension Study: Cardiac Function From Baseline to Month 24 by Change in Left Ventricular Ejection Fraction (LVEF)
Hide Description Cardiac function endpoints (LVEF) obtained by CMR at baseline, Months 6, 12, 18 and 24 were summarized by means of descriptive statistics. These analyses were conducted for the measured values as well as for the absolute changes from baseline
Time Frame Months 6, 12, 18 and 24
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): consisted of all patients enrolled in the extension. Patients were analyzed according to the treatment they were assigned to in the beginning of the extension phase. Cardiac function parameters over time - excluding patients in Egypt sites.
Arm/Group Title Extension: ICL to ICL Extension: DFO to DFO Extension: DFO to ICL Extension: ICL to DFO
Hide Arm/Group Description:
Patients from core continued and received same dose 20 mg/kg/day once daily (od) for 2 weeks, followed by 30 mg/kg/day od for 1 week and a subsequent continuation of 40 mg/kg/day
Patients from core continued and received same 50 mg/kg/day to 60 mg/kg/day infused subcutaneously in 8- to 12-hour intervals administered 5 to 7 days/week
DFO to ICL” (patients who switched from DFO to deferasirox in extension)
ICL to DFO” (patients who switched from deferasirox to DFO in extension)
Overall Number of Participants Analyzed 74 29 42 1
Mean (Standard Deviation)
Unit of Measure: Percent
Month 6 (n=71,26,40,1) -1.1  (4.96) -1.8  (3.59) 0.1  (4.60) -1.0  (0)
Month 12 (n= 66,29,42,1) -0.5  (5.02) 0.3  (5.13) 0.0  (3.73) 0  (0)
Month 18 (n=68,26,39,1 ) -0.1  (4.84) -0.8  (5.14) -1.3  (3.74) -10.0  (0)
Month 24 (n= 63,25,33,1) 0.6  (4.72) -0.6  (5.02) 0.2  (4.82) -18.0  (0)
15.Secondary Outcome
Title Extension Study: Cardiac Function From Baseline to Month 24 by Change in Left Ventricular End Systolic Volume Indices (LVESVI)
Hide Description Cardiac function endpoints (LVESVI) obtained by CMR at baseline, Months 6, 12, 18 and 24 were summarized by means of descriptive statistics. These analyses were conducted for the measured values as well as for the absolute changes from baseline
Time Frame Months 6, 12, 18 and 24
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): consisted of all patients enrolled in the extension. Patients were analyzed according to the treatment they were assigned to in the beginning of the extension phase. Cardiac function parameters over time - excluding patients in Egypt sites.
Arm/Group Title Extension: ICL to ICL Extension: DFO to DFO Extension: DFO to ICL Extension: ICL to DFO
Hide Arm/Group Description:
Patients from core continued and received same 20 mg/kg/day once daily (od) for 2 weeks, followed by 30 mg/kg/day od for 1 week and a subsequent continuation of 40 mg/kg/day
Patients from core continued and received same 50 mg/kg/day to 60 mg/kg/day infused subcutaneously in 8- to 12-hour intervals administered 5 to 7 days/week
DFO to ICL” (patients who switched from DFO to deferasirox in extension)
ICL to DFO” (patients who switched from deferasirox to DFO in extension)
Overall Number of Participants Analyzed 74 29 42 1
Mean (Standard Deviation)
Unit of Measure: mL/m^2
Month 6 (n=69,26,40,1) 1.7  (7.98) 3.4  (10.40) 0  (8.22) 1  (0)
Month 12 (n=64,28,40,1 ) 1.5  (7.42) -0.8  (9.74) 0.6  (9.69) 2  (0)
Month 18 (n=67,24,35,1 ) 2.4  (10.58) 2.8  (11.54) 4.1  (6.88) 28.0  (0)
Month 24 (n=60,23,33,0 ) 1.6  (10.21) 4.3  (8.66) 1.7  (8.56) NA [1]   (NA)
[1]
no participant analyzed
16.Secondary Outcome
Title Extension Study: Cardiac Function From Baseline to Month 24 by Change in Left Ventricular End Diastolic Volume Indices (LVEDVI)
Hide Description Cardiac function endpoint (LVEDVI ) obtained by CMR at baseline, Months 6, 12, 18 and 24 were summarized by means of descriptive statistics. These analyses were conducted for the measured values as well as for the absolute changes from baseline
Time Frame Months 6, 12, 18 and 24
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): consisted of all patients enrolled in the extension. Patients were analyzed according to the treatment they were assigned to in the beginning of the extension phase. Cardiac function parameters over time - excluding patients in Egypt sites
Arm/Group Title Extension: ICL to ICL Extension: DFO to DFO Extension: DFO to ICL Extension: ICL to DFO
Hide Arm/Group Description:
Patients from core continued and received same 20 mg/kg/day once daily (od) for 2 weeks, followed by 30 mg/kg/day od for 1 week and a subsequent continuation of 40 mg/kg/day
Patients from core continued and received same 50 mg/kg/day to 60 mg/kg/day infused subcutaneously in 8- to 12-hour intervals administered 5 to 7 days/week
DFO to ICL” (patients who switched from DFO to deferasirox in extension)
ICL to DFO” (patients who switched from deferasirox to DFO in extension)
Overall Number of Participants Analyzed 74 29 42 1
Mean (Standard Deviation)
Unit of Measure: mL/m^2
Month 6 (n=69,26,40,1) 2.0  (14.19) 3.5  (21.95) 0.5  (18.77) 1.0  (0)
Month 12 (n=64,28,40,1) 2.0  (13.49) -0.6  (18.56) 3.0  (23.06) 4.0  (0)
Month 18 (n=67,24,35,1) 6.5  (17.80) 4.2  (19.75) 8.3  (16.08) 36.0  (0)
Month 24 (n=60,23,33,0) 3.4  (21.15) 9.5  (14.60) 5.4  (13.97) NA [1]   (NA)
[1]
No participant analyzed
17.Secondary Outcome
Title Extension Study: Cardiac Function From Baseline to Month 24 by Change in Left Ventricular Mass Indices (LVMI)
Hide Description Cardiac function endpoints (LVMI) obtained by CMR at baseline, Months 6, 12, 18 and 24 were summarized by means of descriptive statistics. These analyses were conducted for the measured values as well as for the absolute changes
Time Frame Months 6, 12, 18 and 24
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): consisted of all patients enrolled in the extension. Patients were analyzed according to the treatment they were assigned to in the beginning of the extension phase. Cardiac function parameters over time - excluding patients in Egypt sites
Arm/Group Title Extension: ICL to ICL Extension: DFO to DFO Extension: DFO to ICL Extension: ICL to DFO
Hide Arm/Group Description:
Patients from core continued and received same 20 mg/kg/day once daily (od) for 2 weeks, followed by 30 mg/kg/day od for 1 week and a subsequent continuation of 40 mg/kg/day
Patients from core continued and received same 50 mg/kg/day to 60 mg/kg/day infused subcutaneously in 8- to 12-hour intervals administered 5 to 7 days/week
DFO to ICL” (patients who switched from DFO to deferasirox in extension)
ICL to DFO” (patients who switched from deferasirox to DFO in extension)
Overall Number of Participants Analyzed 74 29 42 1
Mean (Standard Deviation)
Unit of Measure: gram/m^2
Month 6 (n=69,26,40,1) 1.4  (12.37) 1.8  (15.98) 3.2  (13.38) -6.0  (0)
Month 12 (n=64,28,40,1) 4.2  (13.90) 9.1  (14.19) 3.4  (16.54) 1  (0)
Month 18 (n=67,24,35,1) 4.8  (14.37) -0.1  (16.87) 4.0  (14.39) 37.0  (0)
Month 24 (n=60,23,33,0) 5.6  (13.00) 6.7  (14.96) 10.3  (13.32) NA [1]   (NA)
[1]
No participant analyzed
18.Secondary Outcome
Title Extension Study: The Cardiac Iron Concentration From T2* Values
Hide Description Cardiac iron concentration (derived from T2* values) at baseline, Months 6, 12, 18 and 24 were summarized by descriptive statistics. The absolute change from baseline at Months 6, 12, 18 and 24 were also summarized by treatment group. Lliver iron concentration is expressed in units (mg of iron / g of liver tissue dry weight (dw)
Time Frame Months 6, 12, 18 and 24
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): consisted of all patients enrolled in the extension. Patients were analyzed according to the treatment they were assigned to in the beginning of the extension phase
Arm/Group Title Extension: ICL to ICL Extension: DFO to DFO Extension: DFO to ICL Extension: ICL to DFO
Hide Arm/Group Description:
Patients from core continued and received same 20 mg/kg/day once daily (od) for 2 weeks, followed by 30 mg/kg/day od for 1 week and a subsequent continuation of 40 mg/kg/day
Patients from core continued and received same 50 mg/kg/day to 60 mg/kg/day infused subcutaneously in 8- to 12-hour intervals administered 5 to 7 days/week
DFO to ICL” (patients who switched from DFO to deferasirox in extension)
ICL to DFO” (patients who switched from deferasirox to DFO in extension)
Overall Number of Participants Analyzed 74 26 40 1
Mean (Standard Deviation)
Unit of Measure: mg Fe/g dw
Month 6 (n=71,26,40,1) -0.12  (0.587) -0.12  (0.539) -0.08  (0.465) 0  (0)
Month 12 (n=66,29,41,1) -0.38  (0.674) -0.12  (0.695) -0.14  (0.490) -0.77  (0)
Month 18 (n=68,26,39,1) -0.47  (0.789) -0.45  (0.797) -0.20  (0.607) -0.24  (0)
Month 24 (n=63,25,33,1) -0.70  (0.834) -0.69  (0.989) -0.34  (0.863) -0.52  (0)
19.Secondary Outcome
Title Extension Study: Change in Liver Iron Concentration (LIC) From Baseline at Month 24
Hide Description Results of liver iron content (LIC) measurements by MRI was summarized by descriptive statistics. The absolute value and the absolute change from baseline in LIC at Months 6, 12, 18 and 24 were provided by treatment group.
Time Frame Months 6, 12, 18 and 24
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): consisted of all patients enrolled in the extension. Patients were analyzed according to the treatment they were assigned to in the beginning of the extension phase
Arm/Group Title Extension: ICL to ICL Extension: DFO to DFO Extension: DFO to ICL Extension: ICL to DFO
Hide Arm/Group Description:
Patients from core continued and received same 20 mg/kg/day once daily (od) for 2 weeks, followed by 30 mg/kg/day od for 1 week and a subsequent continuation of 40 mg/kg/day
Patients from core continued and received same 50 mg/kg/day to 60 mg/kg/day infused subcutaneously in 8- to 12-hour intervals administered 5 to 7 days/week
DFO to ICL” (patients who switched from DFO to deferasirox in extension)
ICL to DFO” (patients who switched from deferasirox to DFO in extension)
Overall Number of Participants Analyzed 74 29 42 1
Mean (Standard Deviation)
Unit of Measure: mg Fe/g dw
Month 6 (n=71,26,38,1) -4.56  (7.467) -12.66  (7.821) -6.30  (7.375) -3.80  (0)
Month 12 (n=69,29,40,1) -10.22  (10.674) -19.44  (11.446) -7.98  (9.310) -3.90  (0)
Month 18 (n=70,23,38,1) -12.26  (13.938) -26.09  (13.443) -10.87  (11.116) -2.90  (0)
Month 24 (n=60,24,33,1) -15.74  (15.205) -26.02  (14.867) -10.96  (11.070) -3.20  (0)
20.Secondary Outcome
Title Extension Study: Change in Serum Ferritin From Baseline by Month
Hide Description Serum ferritin values was summarized by descriptive statistics. Absolute value and the absolute change from baseline in serum ferritin by month was provided by treatment group.
Time Frame Months 6, 12, 18 and 24
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS): consisted of all patients enrolled in the extension. Patients were analyzed according to the treatment they were assigned to in the beginning of the extension phase
Arm/Group Title Extension: ICL to ICL Extension: DFO to DFO Extension: DFO to ICL Extension: ICL to DFO
Hide Arm/Group Description:
Patients from core continued and received same 20 mg/kg/day once daily (od) for 2 weeks, followed by 30 mg/kg/day od for 1 week and a subsequent continuation of 40 mg/kg/day
Patients from core continued and received same 50 mg/kg/day to 60 mg/kg/day infused subcutaneously in 8- to 12-hour intervals administered 5 to 7 days/week
DFO to ICL” (patients who switched from DFO to deferasirox in extension)
ICL to DFO” (patients who switched from deferasirox to DFO in extension)
Overall Number of Participants Analyzed 74 29 42 1
Mean (Standard Deviation)
Unit of Measure: ug/L
Month 6 (n=72,28,38,0) -626.10  (1541.406) -1307.14  (1520.877) -1054.87  (1967.651) NA [1]   (NA)
Month 12 (n=70,29,40,1) -988.46  (2883.416) -1877.00  (1877.745) -1223.73  (1808.056) -498.00  (0)
Month 18 (n=66,24,39,1) -1962.14  (2225.438) -2426.92  (2685.817) -1494.82  (1511.257) -1067.00  (0)
Month 24 (n=59,24,30,0) -2239.03  (2178.564) -2724.00  (2693.583) -1513.23  (2278.144) NA [1]   (NA)
[1]
No participants analyzed
21.Secondary Outcome
Title Core Study: Single and Repeated Dose Pharmacokinetics of Deferasirox, Maximum Plasma Concentration (Tmax)
Hide Description The plasma level of deferasirox (ICL670) obtained in this study was summarized descriptively. Plasma concentration was plotted by patient and by visit. Descriptive statistics included the mean, median, SD, and CV, min and max. deferasirox pharmacokinetics (PK) trough levels over the 12 months of treatment and obtained PK profiles for the 40 mg/kg/day deferasirox dose, time to reach maximum plasma concentration (Tmax)
Time Frame 12 Month
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Hide Analysis Population Description
Pharmacokinetic Analysis Set (PAS): PAS 2: All randomized patients who received the same dose of deferasirox for at least four consecutive days prior to PK sample collection and completed PK sample collection specified in the protocol at Visit 3 or Visit 4 (pre-dose, 1, 2, and 4 hours post-dose).
Arm/Group Title Deferasirox (ICL). For Extension Labeled as ICL to ICL
Hide Arm/Group Description:
20 mg/kg/day once daily (od) for 2 weeks, followed by 30 mg/kg/day od for 1 week and a subsequent continuation of 40 mg/kg/day
Overall Number of Participants Analyzed 13
Median (Inter-Quartile Range)
Unit of Measure: (h)
4.00
(1.0 to 4.0)
Time Frame [Not Specified]
Adverse Event Reporting Description Core : The safety set consisted of all randomized patients who received at least one dose of study drug and had at least one post-baseline. safety assessment. Extension: one patient assigned to the deferasirox arm died before receiving treatment in the extension phase
 
Arm/Group Title Core Phase - ICL670 Core Phase - DFO Extension Phase - ICL to ICL Extension Phase - DFO to DFO Extension Phase - DFO to ICL Extension Phase - ICL to DFO
Hide Arm/Group Description Patients from core continued and received same 20 mg/kg/day once daily (od) for 2 weeks, followed by 30 mg/kg/day od for 1 week and a subsequent continuation of 40 mg/kg/day Patients from core continued and received same 50 mg/kg/day to 60 mg/kg/day infused subcutaneously in 8- to 12-hour intervals administered 5 to 7 days/week Patients from core continued and received same 20 mg/kg/day once daily (od) for 2 weeks, followed by 30 mg/kg/day od for 1 week and a subsequent continuation of 40 mg/kg/day Patients from core continued and received same 50 mg/kg/day to 60 mg/kg/day infused subcutaneously in 8- to 12-hour intervals administered 5 to 7 days/week DFO to ICL” (patients who switched from DFO to deferasirox in extension) ICL to DFO” (patients who switched from deferasirox to DFO in extension)
All-Cause Mortality
Core Phase - ICL670 Core Phase - DFO Extension Phase - ICL to ICL Extension Phase - DFO to DFO Extension Phase - DFO to ICL Extension Phase - ICL to DFO
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/--   --/--   --/--   --/--   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Core Phase - ICL670 Core Phase - DFO Extension Phase - ICL to ICL Extension Phase - DFO to DFO Extension Phase - DFO to ICL Extension Phase - ICL to DFO
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   10/96 (10.42%)   10/91 (10.99%)   14/73 (19.18%)   4/29 (13.79%)   9/42 (21.43%)   0/1 (0.00%) 
Blood and lymphatic system disorders             
Hypersplenism  1  0/96 (0.00%)  0/91 (0.00%)  1/73 (1.37%)  0/29 (0.00%)  0/42 (0.00%)  0/1 (0.00%) 
Neutropenia  1  0/96 (0.00%)  0/91 (0.00%)  0/73 (0.00%)  0/29 (0.00%)  1/42 (2.38%)  0/1 (0.00%) 
Cardiac disorders             
Arrhythmia  1  1/96 (1.04%)  0/91 (0.00%)  0/73 (0.00%)  0/29 (0.00%)  0/42 (0.00%)  0/1 (0.00%) 
Endocrine disorders             
Hypogonadism  1  0/96 (0.00%)  1/91 (1.10%)  0/73 (0.00%)  0/29 (0.00%)  1/42 (2.38%)  0/1 (0.00%) 
Gastrointestinal disorders             
Abdominal pain upper  1  1/96 (1.04%)  1/91 (1.10%)  1/73 (1.37%)  0/29 (0.00%)  1/42 (2.38%)  0/1 (0.00%) 
Colitis  1  1/96 (1.04%)  0/91 (0.00%)  1/73 (1.37%)  0/29 (0.00%)  0/42 (0.00%)  0/1 (0.00%) 
Diarrhoea  1  0/96 (0.00%)  1/91 (1.10%)  0/73 (0.00%)  0/29 (0.00%)  1/42 (2.38%)  0/1 (0.00%) 
Duodenal ulcer haemorrhage  1  0/96 (0.00%)  0/91 (0.00%)  1/73 (1.37%)  0/29 (0.00%)  0/42 (0.00%)  0/1 (0.00%) 
Dyspepsia  1  0/96 (0.00%)  0/91 (0.00%)  1/73 (1.37%)  0/29 (0.00%)  0/42 (0.00%)  0/1 (0.00%) 
Gastric haemorrhage  1  1/96 (1.04%)  0/91 (0.00%)  1/73 (1.37%)  0/29 (0.00%)  0/42 (0.00%)  0/1 (0.00%) 
Gastritis  1  0/96 (0.00%)  0/91 (0.00%)  1/73 (1.37%)  0/29 (0.00%)  0/42 (0.00%)  0/1 (0.00%) 
Ileus  1  0/96 (0.00%)  1/91 (1.10%)  0/73 (0.00%)  0/29 (0.00%)  1/42 (2.38%)  0/1 (0.00%) 
Oesophageal rupture  1  1/96 (1.04%)  0/91 (0.00%)  1/73 (1.37%)  0/29 (0.00%)  0/42 (0.00%)  0/1 (0.00%) 
Vomiting  1  1/96 (1.04%)  0/91 (0.00%)  1/73 (1.37%)  0/29 (0.00%)  0/42 (0.00%)  0/1 (0.00%) 
General disorders             
Face oedema  1  0/96 (0.00%)  1/91 (1.10%)  0/73 (0.00%)  0/29 (0.00%)  0/42 (0.00%)  0/1 (0.00%) 
Local swelling  1  0/96 (0.00%)  1/91 (1.10%)  0/73 (0.00%)  0/29 (0.00%)  0/42 (0.00%)  0/1 (0.00%) 
Pyrexia  1  1/96 (1.04%)  0/91 (0.00%)  1/73 (1.37%)  1/29 (3.45%)  0/42 (0.00%)  0/1 (0.00%) 
Hepatobiliary disorders             
Cholecystitis  1  0/96 (0.00%)  0/91 (0.00%)  1/73 (1.37%)  0/29 (0.00%)  0/42 (0.00%)  0/1 (0.00%) 
Cholelithiasis  1  1/96 (1.04%)  1/91 (1.10%)  1/73 (1.37%)  1/29 (3.45%)  0/42 (0.00%)  0/1 (0.00%) 
Infections and infestations             
Abdominal abscess  1  0/96 (0.00%)  1/91 (1.10%)  0/73 (0.00%)  0/29 (0.00%)  0/42 (0.00%)  0/1 (0.00%) 
Acute tonsillitis  1  1/96 (1.04%)  1/91 (1.10%)  0/73 (0.00%)  0/29 (0.00%)  1/42 (2.38%)  0/1 (0.00%) 
Amoebiasis  1  1/96 (1.04%)  0/91 (0.00%)  0/73 (0.00%)  0/29 (0.00%)  0/42 (0.00%)  0/1 (0.00%) 
Anal abscess  1  0/96 (0.00%)  0/91 (0.00%)  0/73 (0.00%)  1/29 (3.45%)  0/42 (0.00%)  0/1 (0.00%) 
Appendicitis  1  0/96 (0.00%)  1/91 (1.10%)  1/73 (1.37%)  0/29 (0.00%)  1/42 (2.38%)  0/1 (0.00%) 
Bronchitis  1  0/96 (0.00%)  0/91 (0.00%)  1/73 (1.37%)  0/29 (0.00%)  0/42 (0.00%)  0/1 (0.00%) 
Bronchopneumonia  1  0/96 (0.00%)  0/91 (0.00%)  0/73 (0.00%)  1/29 (3.45%)  0/42 (0.00%)  0/1 (0.00%) 
Gastroenteritis  1  1/96 (1.04%)  0/91 (0.00%)  1/73 (1.37%)  0/29 (0.00%)  1/42 (2.38%)  0/1 (0.00%) 
Gastrointestinal infection  1  0/96 (0.00%)  1/91 (1.10%)  0/73 (0.00%)  0/29 (0.00%)  1/42 (2.38%)  0/1 (0.00%) 
Herpes zoster  1  1/96 (1.04%)  0/91 (0.00%)  1/73 (1.37%)  0/29 (0.00%)  0/42 (0.00%)  0/1 (0.00%) 
Influenza  1  0/96 (0.00%)  0/91 (0.00%)  1/73 (1.37%)  0/29 (0.00%)  0/42 (0.00%)  0/1 (0.00%) 
Liver abscess  1  1/96 (1.04%)  0/91 (0.00%)  1/73 (1.37%)  0/29 (0.00%)  0/42 (0.00%)  0/1 (0.00%) 
Meningitis  1  0/96 (0.00%)  1/91 (1.10%)  0/73 (0.00%)  0/29 (0.00%)  0/42 (0.00%)  0/1 (0.00%) 
Pneumonia  1  0/96 (0.00%)  0/91 (0.00%)  0/73 (0.00%)  1/29 (3.45%)  0/42 (0.00%)  0/1 (0.00%) 
Tooth infection  1  0/96 (0.00%)  1/91 (1.10%)  0/73 (0.00%)  0/29 (0.00%)  0/42 (0.00%)  0/1 (0.00%) 
Urinary tract infection  1  1/96 (1.04%)  0/91 (0.00%)  1/73 (1.37%)  0/29 (0.00%)  0/42 (0.00%)  0/1 (0.00%) 
Wound infection  1  0/96 (0.00%)  0/91 (0.00%)  0/73 (0.00%)  0/29 (0.00%)  1/42 (2.38%)  0/1 (0.00%) 
Yersinia infection  1  0/96 (0.00%)  0/91 (0.00%)  1/73 (1.37%)  0/29 (0.00%)  0/42 (0.00%)  0/1 (0.00%) 
Injury, poisoning and procedural complications             
Radius fracture  1  0/96 (0.00%)  0/91 (0.00%)  1/73 (1.37%)  0/29 (0.00%)  0/42 (0.00%)  0/1 (0.00%) 
Metabolism and nutrition disorders             
Calcium deficiency  1  0/96 (0.00%)  0/91 (0.00%)  1/73 (1.37%)  0/29 (0.00%)  0/42 (0.00%)  0/1 (0.00%) 
Haemosiderosis  1  0/96 (0.00%)  1/91 (1.10%)  2/73 (2.74%)  0/29 (0.00%)  0/42 (0.00%)  0/1 (0.00%) 
Hyperglycaemia  1  1/96 (1.04%)  1/91 (1.10%)  0/73 (0.00%)  0/29 (0.00%)  1/42 (2.38%)  0/1 (0.00%) 
Hypocalcaemia  1  0/96 (0.00%)  0/91 (0.00%)  0/73 (0.00%)  0/29 (0.00%)  1/42 (2.38%)  0/1 (0.00%) 
Iron overload  1  0/96 (0.00%)  2/91 (2.20%)  2/73 (2.74%)  0/29 (0.00%)  1/42 (2.38%)  0/1 (0.00%) 
Musculoskeletal and connective tissue disorders             
Back pain  1  1/96 (1.04%)  0/91 (0.00%)  1/73 (1.37%)  0/29 (0.00%)  0/42 (0.00%)  0/1 (0.00%) 
Pain in jaw  1  0/96 (0.00%)  1/91 (1.10%)  0/73 (0.00%)  0/29 (0.00%)  0/42 (0.00%)  0/1 (0.00%) 
Nervous system disorders             
Grand mal convulsion  1  0/96 (0.00%)  1/91 (1.10%)  0/73 (0.00%)  0/29 (0.00%)  0/42 (0.00%)  0/1 (0.00%) 
Respiratory, thoracic and mediastinal disorders             
Cough  1  0/96 (0.00%)  0/91 (0.00%)  0/73 (0.00%)  1/29 (3.45%)  0/42 (0.00%)  0/1 (0.00%) 
Wheezing  1  0/96 (0.00%)  0/91 (0.00%)  0/73 (0.00%)  1/29 (3.45%)  0/42 (0.00%)  0/1 (0.00%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Core Phase - ICL670 Core Phase - DFO Extension Phase - ICL to ICL Extension Phase - DFO to DFO Extension Phase - DFO to ICL Extension Phase - ICL to DFO
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   60/96 (62.50%)   58/91 (63.74%)   56/73 (76.71%)   19/29 (65.52%)   36/42 (85.71%)   1/1 (100.00%) 
Blood and lymphatic system disorders             
Thrombocytosis  1  0/96 (0.00%)  4/91 (4.40%)  1/73 (1.37%)  2/29 (6.90%)  2/42 (4.76%)  0/1 (0.00%) 
Endocrine disorders             
Hypogonadism  1  2/96 (2.08%)  2/91 (2.20%)  0/73 (0.00%)  0/29 (0.00%)  2/42 (4.76%)  1/1 (100.00%) 
Eye disorders             
Conjunctivitis  1  1/96 (1.04%)  2/91 (2.20%)  4/73 (5.48%)  0/29 (0.00%)  3/42 (7.14%)  0/1 (0.00%) 
Gastrointestinal disorders             
Abdominal pain  1  7/96 (7.29%)  2/91 (2.20%)  7/73 (9.59%)  1/29 (3.45%)  3/42 (7.14%)  0/1 (0.00%) 
Abdominal pain upper  1  5/96 (5.21%)  5/91 (5.49%)  6/73 (8.22%)  3/29 (10.34%)  5/42 (11.90%)  0/1 (0.00%) 
Diarrhoea  1  12/96 (12.50%)  4/91 (4.40%)  10/73 (13.70%)  0/29 (0.00%)  9/42 (21.43%)  1/1 (100.00%) 
Nausea  1  6/96 (6.25%)  2/91 (2.20%)  4/73 (5.48%)  1/29 (3.45%)  4/42 (9.52%)  0/1 (0.00%) 
Vomiting  1  6/96 (6.25%)  1/91 (1.10%)  6/73 (8.22%)  0/29 (0.00%)  4/42 (9.52%)  0/1 (0.00%) 
General disorders             
Fatigue  1  1/96 (1.04%)  2/91 (2.20%)  0/73 (0.00%)  0/29 (0.00%)  3/42 (7.14%)  0/1 (0.00%) 
Injection site reaction  1  0/96 (0.00%)  3/91 (3.30%)  0/73 (0.00%)  0/29 (0.00%)  3/42 (7.14%)  0/1 (0.00%) 
Pyrexia  1  5/96 (5.21%)  5/91 (5.49%)  6/73 (8.22%)  3/29 (10.34%)  5/42 (11.90%)  1/1 (100.00%) 
Infections and infestations             
Acute tonsillitis  1  1/96 (1.04%)  3/91 (3.30%)  1/73 (1.37%)  1/29 (3.45%)  3/42 (7.14%)  0/1 (0.00%) 
Bronchitis  1  1/96 (1.04%)  3/91 (3.30%)  1/73 (1.37%)  0/29 (0.00%)  3/42 (7.14%)  0/1 (0.00%) 
Influenza  1  10/96 (10.42%)  6/91 (6.59%)  11/73 (15.07%)  1/29 (3.45%)  7/42 (16.67%)  0/1 (0.00%) 
Nasopharyngitis  1  8/96 (8.33%)  4/91 (4.40%)  12/73 (16.44%)  1/29 (3.45%)  6/42 (14.29%)  0/1 (0.00%) 
Otitis media  1  1/96 (1.04%)  0/91 (0.00%)  1/73 (1.37%)  0/29 (0.00%)  1/42 (2.38%)  1/1 (100.00%) 
Pharyngitis  1  3/96 (3.13%)  2/91 (2.20%)  4/73 (5.48%)  0/29 (0.00%)  3/42 (7.14%)  0/1 (0.00%) 
Tonsillitis  1  1/96 (1.04%)  2/91 (2.20%)  4/73 (5.48%)  1/29 (3.45%)  3/42 (7.14%)  0/1 (0.00%) 
Upper respiratory tract infection  1  8/96 (8.33%)  8/91 (8.79%)  13/73 (17.81%)  1/29 (3.45%)  9/42 (21.43%)  0/1 (0.00%) 
Urinary tract infection  1  0/96 (0.00%)  4/91 (4.40%)  1/73 (1.37%)  1/29 (3.45%)  4/42 (9.52%)  0/1 (0.00%) 
Injury, poisoning and procedural complications             
Ligament sprain  1  0/96 (0.00%)  3/91 (3.30%)  0/73 (0.00%)  0/29 (0.00%)  3/42 (7.14%)  0/1 (0.00%) 
Investigations             
Alanine aminotransferase increased  1  9/96 (9.38%)  5/91 (5.49%)  9/73 (12.33%)  2/29 (6.90%)  4/42 (9.52%)  1/1 (100.00%) 
Aspartate aminotransferase increased  1  7/96 (7.29%)  3/91 (3.30%)  8/73 (10.96%)  1/29 (3.45%)  2/42 (4.76%)  1/1 (100.00%) 
Blood creatinine increased  1  8/96 (8.33%)  2/91 (2.20%)  9/73 (12.33%)  1/29 (3.45%)  3/42 (7.14%)  0/1 (0.00%) 
Ejection fraction decreased  1  0/96 (0.00%)  0/91 (0.00%)  0/73 (0.00%)  0/29 (0.00%)  1/42 (2.38%)  1/1 (100.00%) 
Electrocardiogram QT prolonged  1  0/96 (0.00%)  1/91 (1.10%)  1/73 (1.37%)  1/29 (3.45%)  3/42 (7.14%)  0/1 (0.00%) 
Platelet count increased  1  2/96 (2.08%)  5/91 (5.49%)  3/73 (4.11%)  6/29 (20.69%)  0/42 (0.00%)  0/1 (0.00%) 
Protein urine present  1  11/96 (11.46%)  8/91 (8.79%)  6/73 (8.22%)  1/29 (3.45%)  7/42 (16.67%)  0/1 (0.00%) 
Metabolism and nutrition disorders             
Diabetes mellitus  1  3/96 (3.13%)  1/91 (1.10%)  5/73 (6.85%)  0/29 (0.00%)  1/42 (2.38%)  0/1 (0.00%) 
Musculoskeletal and connective tissue disorders             
Arthralgia  1  7/96 (7.29%)  4/91 (4.40%)  5/73 (6.85%)  2/29 (6.90%)  2/42 (4.76%)  1/1 (100.00%) 
Back pain  1  7/96 (7.29%)  4/91 (4.40%)  7/73 (9.59%)  1/29 (3.45%)  2/42 (4.76%)  0/1 (0.00%) 
Osteoporosis  1  5/96 (5.21%)  2/91 (2.20%)  3/73 (4.11%)  1/29 (3.45%)  2/42 (4.76%)  1/1 (100.00%) 
Nervous system disorders             
Headache  1  5/96 (5.21%)  5/91 (5.49%)  7/73 (9.59%)  2/29 (6.90%)  4/42 (9.52%)  0/1 (0.00%) 
Psychiatric disorders             
Depression  1  2/96 (2.08%)  1/91 (1.10%)  4/73 (5.48%)  0/29 (0.00%)  1/42 (2.38%)  0/1 (0.00%) 
Respiratory, thoracic and mediastinal disorders             
Cough  1  4/96 (4.17%)  2/91 (2.20%)  5/73 (6.85%)  0/29 (0.00%)  2/42 (4.76%)  0/1 (0.00%) 
Dyspnoea  1  4/96 (4.17%)  3/91 (3.30%)  1/73 (1.37%)  0/29 (0.00%)  3/42 (7.14%)  0/1 (0.00%) 
Nasal congestion  1  1/96 (1.04%)  1/91 (1.10%)  1/73 (1.37%)  0/29 (0.00%)  3/42 (7.14%)  0/1 (0.00%) 
Oropharyngeal pain  1  6/96 (6.25%)  2/91 (2.20%)  6/73 (8.22%)  0/29 (0.00%)  5/42 (11.90%)  1/1 (100.00%) 
Skin and subcutaneous tissue disorders             
Rash  1  4/96 (4.17%)  0/91 (0.00%)  4/73 (5.48%)  0/29 (0.00%)  1/42 (2.38%)  0/1 (0.00%) 
Urticaria  1  3/96 (3.13%)  3/91 (3.30%)  4/73 (5.48%)  3/29 (10.34%)  2/42 (4.76%)  0/1 (0.00%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
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 or disclosure of trial results in their entirety
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Study Director
Organization: Novartis Pharnaceuticals
Phone: 862-778-8300
EMail: trialandresults.registries@novartis.com
Layout table for additonal information
Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT00600938     History of Changes
Other Study ID Numbers: CICL670A2206
2007-000766-20 ( EudraCT Number )
First Submitted: January 14, 2008
First Posted: January 25, 2008
Results First Submitted: February 21, 2014
Results First Posted: August 27, 2014
Last Update Posted: August 27, 2014