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Trial record 1 of 1 for:    "Alpha-Thalassemia" | "Deferasirox"
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Efficacy and Safety of Deferasirox in Non-transfusion Dependent Thalassemia Patients With Iron Overload and a One Year Open-label Extension Study (THALASSA)

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ClinicalTrials.gov Identifier: NCT00873041
Recruitment Status : Completed
First Posted : April 1, 2009
Results First Posted : September 25, 2012
Last Update Posted : July 9, 2013
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Triple (Participant, Care Provider, Investigator);   Primary Purpose: Treatment
Condition Non-transfusion Dependent Thalassemia
Interventions Drug: deferasirox
Drug: placebo
Enrollment 166
Recruitment Details  
Pre-assignment Details There was a 4 week screening period to determine eligibility prior to randomization.
Arm/Group Title 5 mg/kg/Day Deferasirox 10 mg/kg/Day Deferasirox Placebo/Deferasirox
Hide Arm/Group Description Participants received a starting dose of 5 mg/kg/day deferasirox tablets orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation. In the Extension Study participants received deferasirox once daily (dose based on LIC) for 52 weeks. Participants received a starting dose of 10 mg/kg/day deferasirox tablets orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation. In the Extension Study participants received deferasirox once daily (dose based on LIC) for 52 weeks. Placebo tablet matching 5 mg/kg/day or 10 mg/kg/day orally in the morning each day for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation. In the Extension Study participants received deferasirox once daily (dose based on LIC) for 52 weeks.
Period Title: Core Study
Started 55 55 56
Completed 48 49 51
Not Completed 7 6 5
Reason Not Completed
Adverse Event             2             3             1
Withdrawal by Subject             1             2             2
Lost to Follow-up             3             1             0
Abnormal laboratory value             0             0             1
Protocol deviation             1             0             1
Period Title: Extension Study
Started 41 44 48
Completed 40 44 46
Not Completed 1 0 2
Reason Not Completed
Adverse Event             1             0             1
Administrative reasons             0             0             1
Arm/Group Title 5 mg/kg/Day Deferasirox 10 mg/kg/Day Deferasirox Placebo Total
Hide Arm/Group Description Participants received a starting dose of 5 mg/kg/day deferasirox tablets orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation. In the Extension Study participants received deferasirox once daily (dose based on LIC) for 52 weeks. Participants received a starting dose of 10 mg/kg/day deferasirox tablets orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation. In the Extension Study participants received deferasirox once daily (dose based on LIC) for 52 weeks. Placebo tablet matching 5 mg/kg/day or 10 mg/kg/day orally in the morning each day for 52 weeks. Participants received a starting dose of 5 or 10 mg/kg/day deferasirox tablets orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation. Total of all reporting groups
Overall Number of Baseline Participants 55 55 56 166
Hide Baseline Analysis Population Description
[Not Specified]
Age, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 55 participants 55 participants 56 participants 166 participants
<18 years 6 7 8 21
Between 18 and 65 years 49 47 48 144
>=65 years 0 1 0 1
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 55 participants 55 participants 56 participants 166 participants
Female
26
  47.3%
26
  47.3%
25
  44.6%
77
  46.4%
Male
29
  52.7%
29
  52.7%
31
  55.4%
89
  53.6%
1.Primary Outcome
Title Core Study: Change in Liver Iron Concentration (LIC) From Baseline to Week 52
Hide Description LIC was measured by magnetic resonance imaging technique at baseline and Week 52. Estimates were obtained from an Analysis of Covariance (ANCOVA) model for change in LIC between baseline and Week 52 with treatment as factor and baseline LIC as covariate.
Time Frame Baseline, Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set. The last available post-baseline LIC was carried forward if no LIC value was available at Week 52. Only patients with both baseline and at least one post-baseline value were included for this analysis.
Arm/Group Title 5 mg/kg/Day Deferasirox 10 mg/kg/Day Deferasirox Placebo
Hide Arm/Group Description:
Participants received a starting dose of 5 mg/kg/day deferasirox tablets orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation.
Participants received a starting dose of 10 mg/kg/day deferasirox tablets orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation.
Placebo tablet matching 5 mg/kg/day or 10 mg/kg/day orally in the morning each day for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation.
Overall Number of Participants Analyzed 51 54 54
Least Squares Mean (Standard Error)
Unit of Measure: mg iron (Fe)/g dry weight (dw)
-1.95  (0.500) -3.80  (0.484) 0.38  (0.486)
2.Primary Outcome
Title Extension Study: Percentage of Participants Reaching a Liver Iron Concentration (LIC) < 5 mg Fe/g dw From Core Baseline to End of Extension Study
Hide Description Liver iron concentration was measured at Core Baseline and at the end of the Extension Study. Magnetic Resonance Imaging (MRI) scans were analyzed at a central laboratory to determine the LIC value. The percentage of participants with LIC < 5 mgFe/g dw (milligram iron/gram dry weight) change from Baseline at the end of the Extension Study is reported.
Time Frame Core Baseline to End of Extension Study (up to 24 months)
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis consisted of all randomized participants. Patients with post-baseline LIC satisfying criterion at any time during the study are counted as responder. Patients with no baseline LIC or without any post-baseline LIC measurements will be assumed as non-responder.
Arm/Group Title Deferasirox Placebo/Deferasirox
Hide Arm/Group Description:
Participants received a starting dose of 5 mg/kg/day or 10 mg/kg/day deferasirox tablets orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation. In the Extension Study participants received deferasirox once daily (dose based on LIC) for 52 weeks.
Placebo tablet matching 5 mg/kg/day or 10 mg/kg/day orally in the morning each day for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation. In the Extension Study participants received deferasirox once daily (dose based on LIC) for 52 weeks.
Overall Number of Participants Analyzed 110 56
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: Percentage of participants
39.1
(30.5 to 48.4)
37.5
(26.0 to 56.0)
3.Secondary Outcome
Title Core Study: Change in Liver Iron Concentration (LIC) From Baseline to Week 24
Hide Description LIC was measured by magnetic resonance imaging technique at baseline and Week 24. Estimates were obtained from an Analysis of Covariance (ANCOVA) model for change in LIC between baseline and Week 24 with treatment as factor and baseline LIC as covariate.
Time Frame Baseline, Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set. The last available post-baseline LIC was carried forward if no LIC value was available at Week 24. Only patients with both baseline and at least one post-baseline value were included for this analysis.
Arm/Group Title 5 mg/kg/Day Deferasirox 10 mg/kg/Day Deferasirox Placebo
Hide Arm/Group Description:
Participants received a starting dose of 5 mg/kg/day deferasirox tablets orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation.
Participants received a starting dose of 10 mg/kg/day deferasirox tablets orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation.
Placebo tablet matching 5 mg/kg/day or 10 mg/kg/day orally in the morning each day for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation.
Overall Number of Participants Analyzed 49 48 51
Least Squares Mean (Standard Error)
Unit of Measure: mg iron (Fe)/g dry weight (dw)
-0.87  (0.448) -0.90  (0.450) -0.24  (0.439)
4.Secondary Outcome
Title Core Study: Change in Serum Ferritin Between Baseline and Fourth Quarter
Hide Description

Baseline serum ferritin average was the average of all available ferritin values from screening to last sample prior to the first intake of study drug.

Fourth quarter serum ferritin average was the average of all serum ferritin values obtained within days 286- End of Study.

Change from baseline: fourth quarter serum ferritin average - baseline serum ferritin average.

Time Frame Baseline, (Day 286 to End of Study [Day 365])
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis set (all randomized patients). Only participants with both baseline and post-baseline values are included in analyses. If serum ferritin was missing during the fourth quarter, the last available average of serum ferritin per quarter was used for the calculation of the change from baseline.
Arm/Group Title 5 mg/kg/Day Deferasirox 10 mg/kg/Day Deferasirox Placebo
Hide Arm/Group Description:
Participants received a starting dose of 5 mg/kg/day deferasirox tablets orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation.
Participants received a starting dose of 10 mg/kg/day deferasirox tablets orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation.
Placebo tablet matching 5 mg/kg/day or 10 mg/kg/day orally in the morning each day for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation.
Overall Number of Participants Analyzed 51 50 53
Mean (Standard Deviation)
Unit of Measure: μg/L
-130.47  (260.555) -249.16  (389.356) 128.63  (249.689)
5.Secondary Outcome
Title Core Study: Change in Serum Ferritin Between Baseline and Second Quarter
Hide Description

Baseline serum ferritin average was the average of all available ferritin values from screening to last sample prior to the first intake of study drug.

Second quarter serum ferritin average was the average of all serum ferritin values obtained within days 106-195.

Change from baseline: second quarter serum ferritin average - baseline serum ferritin average.

Time Frame Baseline, (Day 106 to Day 195)
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis set (all randomized patients). Only participants with both baseline and post-baseline values are included in analyses. If serum ferritin was missing during the second quarter, the last available average of serum ferritin per quarter was used for the calculation of the change from baseline.
Arm/Group Title 5 mg/kg/Day Deferasirox 10 mg/kg/Day Deferasirox Placebo
Hide Arm/Group Description:
Participants received a starting dose of 5 mg/kg/day deferasirox tablets orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation.
Participants received a starting dose of 10 mg/kg/day deferasirox tablets orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation.
Placebo tablet matching 5 mg/kg/day or 10 mg/kg/day orally in the morning each day for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation.
Overall Number of Participants Analyzed 52 54 56
Mean (Standard Deviation)
Unit of Measure: μg/L
8.20  (244.443) -17.75  (368.812) 106.45  (330.217)
6.Secondary Outcome
Title Core Study: Percentage of Participants With Adverse Events Graded Mild, Moderate and Severe
Hide Description Percentage of Participants with Mild, Moderate and Severe adverse events (AE) any primary system organ class regardless of study drug relationship. A patient with multiple occurrences of an AE is counted only once in the AE category for that treatment. A patient with multiple severity ratings for an AE while on a treatment is only counted once under the maximum rating.
Time Frame 52 Weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set included all randomized participants who received treatment.
Arm/Group Title 5 mg/kg/Day Deferasirox 10 mg/kg/Day Deferasirox Placebo
Hide Arm/Group Description:
Participants received a starting dose of 5 mg/kg/day deferasirox tablets orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation.
Participants received a starting dose of 10 mg/kg/day deferasirox tablets orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation.
Placebo tablet matching 5 mg/kg/day or 10 mg/kg/day orally in the morning each day for 52 weeks. Participants received a starting dose of 5 or 10 mg/kg/day deferasirox tablets orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation.
Overall Number of Participants Analyzed 55 55 56
Measure Type: Number
Unit of Measure: Percentage of participants
Mild 36.4 43.6 42.9
Moderate 27.3 16.4 21.4
Severe 12.7 18.2 16.1
7.Secondary Outcome
Title Core Study: Change in Liver Iron Concentration (LIC) From Baseline At Week 24 and Week 52 in Patients With Dose Increases After Week 24
Hide Description LIC was measured by magnetic resonance imaging technique at baseline, Week 24 and Week 52. Dose Doubling (Dose Increases) began at Week 24.
Time Frame Baseline, Week 24, Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set. The last available post-baseline LIC was carried forward if no LIC value was available at Week 24 and Week 52. Only patients with dose increases after week 24, with both baseline and at least one post-baseline value were included for this analysis.
Arm/Group Title 5 mg/kg/Day Deferasirox 10 mg/kg/Day Deferasirox Placebo
Hide Arm/Group Description:
Participants received a starting dose of 5 mg/kg/day deferasirox tablets orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation.
Participants received a starting dose of 10 mg/kg/day deferasirox tablets orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation.
Placebo tablet matching 5 mg/kg/day or 10 mg/kg/day orally in the morning each day for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation.
Overall Number of Participants Analyzed 26 25 30
Mean (Standard Deviation)
Unit of Measure: mg iron (Fe)/g dry weight (dw)
Change from baseline at Week 24 (n=26,24,30) 0.56  (2.992) 0.69  (3.131) 0.94  (2.693)
Change from baseline at Week 52 -1.82  (3.101) -4.02  (4.849) 0.62  (4.128)
8.Secondary Outcome
Title Core Study: Correlation Between Serum Ferritin and LIC (Liver Iron Concentration)
Hide Description

The correlation between serum ferritin and LIC was investigated using a scatter plot with a regression line for the following cases:

  • Baseline serum ferritin versus baseline LIC
  • Serum ferritin difference from baseline at fourth quarter versus difference from baseline in LIC at Week 52.

A value of 1.0 indicates a perfect correlation.

Time Frame Baseline, 52 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Participants from the Full Analysis Set (all randomized participants).
Arm/Group Title All Randomized Participants
Hide Arm/Group Description:
Participants received a starting dose of 5 mg/kg/day or 10 mg/kg/day deferasirox tablets or matching placebo orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation.
Overall Number of Participants Analyzed 165
Measure Type: Number
Unit of Measure: Correlation coefficient
Baseline 0.653
Week 52 (n=134) 0.609
9.Secondary Outcome
Title Core Study: Change From Baseline in Hemoglobin at Month 12
Hide Description Blood was collected for Hemoglobin at baseline and Month 12. Change from baseline= Month 12 hemoglobin - baseline hemoglobin.
Time Frame Baseline, Month 12
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (all randomized participants). Only patients with a value both at baseline and at considered timepoint are included in analyses.
Arm/Group Title 5 mg/kg/Day Deferasirox 10 mg/kg/Day Deferasirox Placebo
Hide Arm/Group Description:
Participants received a starting dose of 5 mg/kg/day deferasirox tablets orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation.
Participants received a starting dose of 10 mg/kg/day deferasirox tablets orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation.
Placebo tablet matching 5 mg/kg/day or 10 mg/kg/day orally in the morning each day for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation.
Overall Number of Participants Analyzed 47 48 49
Mean (Standard Deviation)
Unit of Measure: g/L
-1.8  (5.45) -0.7  (6.27) -2.8  (7.31)
10.Secondary Outcome
Title Core Study: Change From Baseline in Transferrin Saturation at Month 12
Hide Description Blood was collected for transferrin saturation at Baseline and Month 12. Change from baseline= Month 12 transferrin saturation - baseline transferrin saturation.
Time Frame Baseline, Month 12
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (all randomized patients). Only patients with a value both at baseline and at considered timepoint are included in the analyses.
Arm/Group Title 5 mg/kg/Day Deferasirox 10 mg/kg/Day Deferasirox Placebo
Hide Arm/Group Description:
Participants received a starting dose of 5 mg/kg/day deferasirox tablets orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation.
Participants received a starting dose of 10 mg/kg/day deferasirox tablets orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation.
Placebo tablet matching 5 mg/kg/day or 10 mg/kg/day orally in the morning each day for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation.
Overall Number of Participants Analyzed 47 47 47
Mean (Standard Deviation)
Unit of Measure: Percent saturation
-3.79  (14.234) -3.64  (22.443) 3.37  (10.083)
11.Secondary Outcome
Title Core Study: Change in Liver Iron Concentration (LIC) in Placebo Patients From Baseline to Week 52
Hide Description LIC was measured by magnetic resonance imaging technique at baseline and Week 52. The change in liver iron concentration for participants in the placebo arm was used to assess the iron accumulation rate.
Time Frame Baseline, Week 52
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set. The last available post-baseline LIC was carried forward if no LIC value was available at Week 52. Only patients with both baseline and at least one post-baseline value were included for this analysis.
Arm/Group Title Placebo
Hide Arm/Group Description:
Placebo tablet matching 5 mg/kg/day or 10 mg/kg/day orally in the morning each day for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation.
Overall Number of Participants Analyzed 54
Mean (Standard Deviation)
Unit of Measure: mg iron (Fe)/g dry weight (dw)
0.26  (3.501)
12.Secondary Outcome
Title Core Study: Percentage of Participants With Notable Abnormal Post-baseline Laboratory Results
Hide Description

The percentage of participants with notable laboratory results:

Platelet count: (<100 x 10^9/L)

Absolute neutrophils: (<1.5 x 10^9/L)

Alanine aminotransferase (ALT): (>5 x Upper limit normal (ULN) and >2 x baseline).

Aspartate aminotransferase (AST): (>5 x ULN and >2 x baseline)

Serum creatinine: (>33% increase from baseline and >ULN at ≥2 consecutive post-baseline values) Creatinine clearance: (<60 mL/min at ≥2 consecutive post-baseline values)

Urinary protein/creatinine ratio: (≥ 1.0 mg/mg at ≥2 consecutive post-baseline values)

Time Frame 52 Weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Set included all randomized participants who received treatment.
Arm/Group Title 5 mg/kg/Day Deferasirox 10 mg/kg/Day Deferasirox Placebo
Hide Arm/Group Description:
Participants received a starting dose of 5 mg/kg/day deferasirox tablets orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation.
Participants received a starting dose of 10 mg/kg/day deferasirox tablets orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation.
Placebo tablet matching 5 mg/kg/day or 10 mg/kg/day orally in the morning each day for 52 weeks. Participants received a starting dose of 5 mg/kg/day deferasirox tablets orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation.
Overall Number of Participants Analyzed 55 55 56
Measure Type: Number
Unit of Measure: Percentage of participants
Platelet count 5.5 5.5 10.7
Absolute neutrophils 5.5 3.6 5.4
ALT 0 0 1.8
AST 0 1.8 1.8
Serum creatinine 0 5.5 0
Creatinine clearance 1.8 1.8 0
Urinary protein/creatinine ratio 1.8 0 0
13.Secondary Outcome
Title Core Study: Percentage of Participants With Notably Abnormal Post-baseline Systolic Blood Pressure
Hide Description

Systolic blood pressure was measured at each visit after the patient rested in the sitting position for at least 3 minutes.

A Notably Abnormal Systolic Blood Pressure was defined as a measurement in one of the following two categories:

High: ≥180 with an increase from baseline ≥20 mmHg

Low: ≤90 with a decrease from baseline ≥20 mmHg

Time Frame Baseline, 52 Weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Set includes all randomized participants who received treatment.
Arm/Group Title 5 mg/kg/Day Deferasirox 10 mg/kg/Day Deferasirox Placebo
Hide Arm/Group Description:
Participants received a starting dose of 5 mg/kg/day deferasirox tablets orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation.
Participants received a starting dose of 10 mg/kg/day deferasirox tablets orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation.
Placebo tablet matching 5 mg/kg/day or 10 mg/kg/day orally in the morning each day for 52 weeks. Participants received a starting dose of 5 or 10 mg/kg/day deferasirox tablets orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation.
Overall Number of Participants Analyzed 55 55 56
Measure Type: Number
Unit of Measure: Percentage of participants
High 0.0 0.0 1.8
Low 10.9 5.5 16.1
14.Secondary Outcome
Title Core Study: Percentage of Participants With Notably Abnormal Post-baseline Diastolic Blood Pressure
Hide Description

Diastolic blood pressure was measured at each visit after the patient rested in the sitting position for at least 3 minutes.

A Notably Abnormal Diastolic Blood Pressure was defined as a measurement in one of the following two categories:

High: ≥105 with an increase from baseline ≥15 mmHg

Low: ≤50 with a decrease from baseline ≥15 mmHg

Time Frame Baseline, 52 Weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Set includes all randomized participants who received treatment.
Arm/Group Title 5 mg/kg/Day Deferasirox 10 mg/kg/Day Deferasirox Placebo
Hide Arm/Group Description:
Participants received a starting dose of 5 mg/kg/day deferasirox tablets orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation.
Participants received a starting dose of 10 mg/kg/day deferasirox tablets orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation.
Placebo tablet matching 5 mg/kg/day or 10 mg/kg/day orally in the morning each day for 52 weeks. Participants received a starting dose of 5 or 10 mg/kg/day deferasirox tablets orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation.
Overall Number of Participants Analyzed 55 55 56
Measure Type: Number
Unit of Measure: Percentage of participants
High 0.0 0.0 0.0
Low 14.5 10.9 14.3
15.Secondary Outcome
Title Core Study: Percentage of Participants With Notably Abnormal Post-baseline Pulse Rate
Hide Description

Pulse Rate was measured at each visit.

A Notably Abnormal Pulse Rate was defined as a measurement in one of the following two categories:

High: ≥120 with an increase from baseline ≥15 beats per minute (bpm)

Low: ≤50 with a decrease from baseline ≥15 bpm

Time Frame Baseline, 52 Weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Set includes all randomized participants who received treatment.
Arm/Group Title 5 mg/kg/Day Deferasirox 10 mg/kg/Day Deferasirox Placebo
Hide Arm/Group Description:
Participants received a starting dose of 5 mg/kg/day deferasirox tablets orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation.
Participants received a starting dose of 10 mg/kg/day deferasirox tablets orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation.
Placebo tablet matching 5 mg/kg/day or 10 mg/kg/day orally in the morning each day for 52 weeks. Participants received a starting dose of 5 or 10 mg/kg/day deferasirox tablets orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation.
Overall Number of Participants Analyzed 55 55 56
Measure Type: Number
Unit of Measure: Percentage of participants
High 1.8 0.0 3.6
Low 0.0 1.8 0.0
16.Secondary Outcome
Title Extension Study: Absolute Change in Serum Ferritin From Baseline to Eighth Quarter
Hide Description Blood was collected for serum ferritin at Core Baseline and monthly during the Eighth quarter of the Extension Study. Absolute change from Baseline: quarterly average – baseline average. A negative change from baseline indicated improvement.
Time Frame Core Baseline, Eighth Quarter (last 3 months of the study)
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set included all randomized participants. Only patients with a value both at baseline and at considered time point are included.
Arm/Group Title Deferasirox Placebo/Deferasirox
Hide Arm/Group Description:
Participants received a starting dose of 5 mg/kg/day or 10 mg/kg/day deferasirox tablets orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation. In the Extension Study participants received deferasirox once daily (dose based on LIC) for 52 weeks.
Placebo tablet matching 5 mg/kg/day or 10 mg/kg/day orally in the morning each day for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation. In the Extension Study participants received deferasirox once daily (dose based on LIC) for 52 weeks.
Overall Number of Participants Analyzed 84 46
Mean (Standard Deviation)
Unit of Measure: micrograms/liter
-565.9  (504.25) -504.3  (770.60)
17.Secondary Outcome
Title Extension Study: Change in Liver Iron Concentration (LIC) From Baseline at Month 24
Hide Description LIC was measured by magnetic resonance imaging technique at Baseline and Month 24. A negative change from baseline indicated improvement.
Time Frame Core Baseline, Month 24
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set. The last available post-baseline LIC was carried forward if no LIC value was available at Week 52. Only patients with both baseline and at least one post-baseline value were included for this analysis.
Arm/Group Title Deferasirox Placebo/Deferasirox
Hide Arm/Group Description:
Participants received a starting dose of 5 mg/kg/day or 10 mg/kg/day deferasirox tablets orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation. In the Extension Study participants received deferasirox once daily (dose based on LIC) for 52 weeks.
Placebo tablet matching 5 mg/kg/day or 10 mg/kg/day orally in the morning each day for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation. In the Extension Study participants received deferasirox once daily (dose based on LIC) for 52 weeks.
Overall Number of Participants Analyzed 84 46
Mean (Standard Deviation)
Unit of Measure: mg iron (Fe)/g dry weight (dw)
-7.1  (5.3) -6.7  (6.67)
18.Secondary Outcome
Title Extension Study: Correlation Between Serum Ferritin and LIC (Liver Iron Concentration)
Hide Description

The correlation between serum ferritin and LIC was investigated using a scatter plot with a regression line for serum ferritin difference from Baseline at Month 24 versus LIC difference from Baseline at Month 24.

A value of 1.0 indicates a perfect correlation.

Time Frame Core Baseline, Month 24
Hide Outcome Measure Data
Hide Analysis Population Description
Participants from the Extension Full Analysis Set (all randomized participants)in the Extension Study with data available for analysis.
Arm/Group Title All Randomized Participants
Hide Arm/Group Description:
Participants received a starting dose of 5 mg/kg/day or 10 mg/kg/day deferasirox tablets or matching placebo orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation.
Overall Number of Participants Analyzed 129
Measure Type: Number
Unit of Measure: Correlation coefficient
0.735
19.Secondary Outcome
Title Extension Study: Change From Baseline in Hemoglobin at Month 24
Hide Description Blood was collected for Hemoglobin at Baseline and Month 24. Change from Baseline= Month 24 hemoglobin - Baseline hemoglobin.
Time Frame Core Baseline, Month 24
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (all randomized participants). Only patients with a value both at baseline and at considered timepoint are included in analyses.
Arm/Group Title Deferasirox Placebo/Deferasirox
Hide Arm/Group Description:
Participants received a starting dose of 5 mg/kg/day deferasirox tablets orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation. In the Extension Study participants received deferasirox once daily (dose based on LIC) for 52 weeks.
Placebo tablet matching 5 mg/kg/day or 10 mg/kg/day orally in the morning each day for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation. In the Extension Study participants received deferasirox once daily (dose based on LIC) for 52 weeks.
Overall Number of Participants Analyzed 59 29
Mean (95% Confidence Interval)
Unit of Measure: g/L
-2.6
(-4.19 to -1.03)
-3.1
(-6.73 to 0.48)
20.Secondary Outcome
Title Extension Study: Change From Baseline in Transferrin Saturation at Month 24
Hide Description Blood was collected for transferrin saturation at Baseline and Month 24. Change from baseline= Month 24 transferrin saturation - baseline transferrin saturation.
Time Frame Core Baseline, Month 24
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (all randomized patients). Only patients with a value both at baseline and at considered timepoint are included in the analyses.
Arm/Group Title Deferasirox Placebo/Deferasirox
Hide Arm/Group Description:
Participants received a starting dose of 5 mg/kg/day deferasirox tablets orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation. In the Extension Study participants received deferasirox once daily (dose based on LIC) for 52 weeks.
Placebo tablet matching 5 mg/kg/day or 10 mg/kg/day orally in the morning each day for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation. In the Extension Study participants received deferasirox once daily (dose based on LIC) for 52 weeks.
Overall Number of Participants Analyzed 66 33
Mean (95% Confidence Interval)
Unit of Measure: Percent saturation
-5.01
(-11.00 to 0.98)
1.35
(-3.26 to 5.97)
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Deferasirox 5 mg/kg/Day Deferasirox 10 mg/kg/Day Placebo/Deferasirox Any Dose
Hide Arm/Group Description Participants received a starting dose of 5 mg/kg/day deferasirox tablets orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation. In the Extension Study participants received deferasirox once daily (dose based on LIC) for 52 weeks. Participants received a starting dose of 10 mg/kg/day deferasirox tablets orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation. In the Extension Study participants received deferasirox once daily (dose based on LIC) for 52 weeks. Placebo tablet matching 5 mg/kg/day or 10 mg/kg/day orally in the morning each day for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation. In the Extension Study participants received deferasirox once daily (dose based on LIC) for 52 weeks.
All-Cause Mortality
Deferasirox 5 mg/kg/Day Deferasirox 10 mg/kg/Day Placebo/Deferasirox Any Dose
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/--   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Deferasirox 5 mg/kg/Day Deferasirox 10 mg/kg/Day Placebo/Deferasirox Any Dose
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   11/55 (20.00%)   12/55 (21.82%)   16/56 (28.57%) 
Blood and lymphatic system disorders       
Anaemia  1  1/55 (1.82%)  2/55 (3.64%)  3/56 (5.36%) 
Haemolysis  1  0/55 (0.00%)  1/55 (1.82%)  0/56 (0.00%) 
Cardiac disorders       
Arrhythmia  1  0/55 (0.00%)  0/55 (0.00%)  1/56 (1.79%) 
Atrial fibrillation  1  0/55 (0.00%)  1/55 (1.82%)  0/56 (0.00%) 
Congenital, familial and genetic disorders       
Atrial septal defect  1  1/55 (1.82%)  0/55 (0.00%)  0/56 (0.00%) 
Eye disorders       
Cataract  1  1/55 (1.82%)  0/55 (0.00%)  0/56 (0.00%) 
Gastrointestinal disorders       
Abdominal pain  1  1/55 (1.82%)  1/55 (1.82%)  0/56 (0.00%) 
Abdominal tenderness  1  0/55 (0.00%)  1/55 (1.82%)  0/56 (0.00%) 
Duodenal ulcer  1  0/55 (0.00%)  1/55 (1.82%)  0/56 (0.00%) 
Food poisoning  1  0/55 (0.00%)  1/55 (1.82%)  0/56 (0.00%) 
Gastritis  1  0/55 (0.00%)  2/55 (3.64%)  0/56 (0.00%) 
Pancreatitis acute  1  0/55 (0.00%)  1/55 (1.82%)  0/56 (0.00%) 
General disorders       
Pyrexia  1  1/55 (1.82%)  3/55 (5.45%)  1/56 (1.79%) 
Hepatobiliary disorders       
Cholangitis  1  0/55 (0.00%)  1/55 (1.82%)  1/56 (1.79%) 
Cholecystitis  1  0/55 (0.00%)  0/55 (0.00%)  1/56 (1.79%) 
Cholecystitis acute  1  0/55 (0.00%)  1/55 (1.82%)  0/56 (0.00%) 
Cholecystitis chronic  1  0/55 (0.00%)  0/55 (0.00%)  1/56 (1.79%) 
Cholelithiasis  1  0/55 (0.00%)  1/55 (1.82%)  1/56 (1.79%) 
Hepatitis  1  0/55 (0.00%)  0/55 (0.00%)  1/56 (1.79%) 
Hepatotoxicity  1  1/55 (1.82%)  0/55 (0.00%)  0/56 (0.00%) 
Portal vein thrombosis  1  1/55 (1.82%)  0/55 (0.00%)  0/56 (0.00%) 
Infections and infestations       
Babesiosis  1  0/55 (0.00%)  0/55 (0.00%)  1/56 (1.79%) 
Cellulitis  1  1/55 (1.82%)  0/55 (0.00%)  0/56 (0.00%) 
Dengue fever  1  0/55 (0.00%)  0/55 (0.00%)  1/56 (1.79%) 
Gastroenteritis  1  3/55 (5.45%)  4/55 (7.27%)  2/56 (3.57%) 
Helicobacter gastritis  1  0/55 (0.00%)  1/55 (1.82%)  0/56 (0.00%) 
Influenza  1  0/55 (0.00%)  1/55 (1.82%)  0/56 (0.00%) 
Liver abscess  1  0/55 (0.00%)  0/55 (0.00%)  1/56 (1.79%) 
Osteomyelitis  1  0/55 (0.00%)  1/55 (1.82%)  0/56 (0.00%) 
Pneumonia  1  0/55 (0.00%)  0/55 (0.00%)  2/56 (3.57%) 
Respiratory tract infection  1  1/55 (1.82%)  0/55 (0.00%)  0/56 (0.00%) 
Viral upper respiratory tract infection  1  0/55 (0.00%)  0/55 (0.00%)  1/56 (1.79%) 
Injury, poisoning and procedural complications       
Ligament rupture  1  0/55 (0.00%)  0/55 (0.00%)  1/56 (1.79%) 
Lower limb fracture  1  0/55 (0.00%)  0/55 (0.00%)  1/56 (1.79%) 
Meniscus lesion  1  0/55 (0.00%)  0/55 (0.00%)  1/56 (1.79%) 
Pelvic fracture  1  1/55 (1.82%)  0/55 (0.00%)  0/56 (0.00%) 
Road traffic accident  1  0/55 (0.00%)  1/55 (1.82%)  0/56 (0.00%) 
Tibia fracture  1  0/55 (0.00%)  0/55 (0.00%)  1/56 (1.79%) 
Upper limb fracture  1  0/55 (0.00%)  1/55 (1.82%)  0/56 (0.00%) 
Investigations       
C-reactive protein  1  1/55 (1.82%)  0/55 (0.00%)  0/56 (0.00%) 
Weight decreased  1  1/55 (1.82%)  0/55 (0.00%)  0/56 (0.00%) 
Metabolism and nutrition disorders       
Dehydration  1  0/55 (0.00%)  0/55 (0.00%)  1/56 (1.79%) 
Nervous system disorders       
Optic neuritis  1  0/55 (0.00%)  0/55 (0.00%)  1/56 (1.79%) 
Syncope  1  1/55 (1.82%)  0/55 (0.00%)  0/56 (0.00%) 
Reproductive system and breast disorders       
Ovarian cyst ruptured  1  0/55 (0.00%)  0/55 (0.00%)  1/56 (1.79%) 
Skin and subcutaneous tissue disorders       
Pruritus  1  0/55 (0.00%)  1/55 (1.82%)  0/56 (0.00%) 
Rash  1  0/55 (0.00%)  1/55 (1.82%)  0/56 (0.00%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, 15.0
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Deferasirox 5 mg/kg/Day Deferasirox 10 mg/kg/Day Placebo/Deferasirox Any Dose
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   37/55 (67.27%)   45/55 (81.82%)   48/56 (85.71%) 
Blood and lymphatic system disorders       
Anaemia  1  6/55 (10.91%)  4/55 (7.27%)  3/56 (5.36%) 
Eye disorders       
Conjunctivitis  1  3/55 (5.45%)  1/55 (1.82%)  1/56 (1.79%) 
Gastrointestinal disorders       
Abdominal pain  1  4/55 (7.27%)  4/55 (7.27%)  9/56 (16.07%) 
Abdominal pain upper  1  4/55 (7.27%)  9/55 (16.36%)  7/56 (12.50%) 
Constipation  1  0/55 (0.00%)  0/55 (0.00%)  3/56 (5.36%) 
Diarrhoea  1  8/55 (14.55%)  7/55 (12.73%)  12/56 (21.43%) 
Dyspepsia  1  1/55 (1.82%)  3/55 (5.45%)  2/56 (3.57%) 
Food poisoning  1  0/55 (0.00%)  3/55 (5.45%)  4/56 (7.14%) 
Gastritis  1  3/55 (5.45%)  1/55 (1.82%)  2/56 (3.57%) 
Nausea  1  5/55 (9.09%)  9/55 (16.36%)  12/56 (21.43%) 
Tooth disorder  1  0/55 (0.00%)  0/55 (0.00%)  3/56 (5.36%) 
Vomiting  1  4/55 (7.27%)  3/55 (5.45%)  8/56 (14.29%) 
General disorders       
Asthenia  1  2/55 (3.64%)  1/55 (1.82%)  3/56 (5.36%) 
Fatigue  1  3/55 (5.45%)  7/55 (12.73%)  6/56 (10.71%) 
Oedema peripheral  1  1/55 (1.82%)  1/55 (1.82%)  4/56 (7.14%) 
Pain  1  3/55 (5.45%)  0/55 (0.00%)  1/56 (1.79%) 
Pyrexia  1  10/55 (18.18%)  5/55 (9.09%)  15/56 (26.79%) 
Infections and infestations       
Gastroenteritis  1  3/55 (5.45%)  5/55 (9.09%)  4/56 (7.14%) 
Gastroenteritis viral  1  0/55 (0.00%)  3/55 (5.45%)  2/56 (3.57%) 
Influenza  1  4/55 (7.27%)  6/55 (10.91%)  5/56 (8.93%) 
Nasopharyngitis  1  6/55 (10.91%)  5/55 (9.09%)  6/56 (10.71%) 
Pharyngitis  1  6/55 (10.91%)  2/55 (3.64%)  2/56 (3.57%) 
Rhinitis  1  2/55 (3.64%)  5/55 (9.09%)  3/56 (5.36%) 
Tonsillitis  1  5/55 (9.09%)  3/55 (5.45%)  5/56 (8.93%) 
Upper respiratory tract infection  1  9/55 (16.36%)  14/55 (25.45%)  14/56 (25.00%) 
Viral infection  1  2/55 (3.64%)  0/55 (0.00%)  4/56 (7.14%) 
Investigations       
Blood creatinine increased  1  1/55 (1.82%)  3/55 (5.45%)  2/56 (3.57%) 
Heart rate increased  1  0/55 (0.00%)  0/55 (0.00%)  3/56 (5.36%) 
Metabolism and nutrition disorders       
Decreased appetite  1  3/55 (5.45%)  1/55 (1.82%)  3/56 (5.36%) 
Musculoskeletal and connective tissue disorders       
Arthralgia  1  3/55 (5.45%)  2/55 (3.64%)  5/56 (8.93%) 
Back pain  1  2/55 (3.64%)  1/55 (1.82%)  6/56 (10.71%) 
Flank pain  1  1/55 (1.82%)  0/55 (0.00%)  3/56 (5.36%) 
Pain in extremity  1  2/55 (3.64%)  2/55 (3.64%)  3/56 (5.36%) 
Nervous system disorders       
Dizziness  1  4/55 (7.27%)  0/55 (0.00%)  1/56 (1.79%) 
Headache  1  3/55 (5.45%)  11/55 (20.00%)  12/56 (21.43%) 
Psychiatric disorders       
Insomnia  1  1/55 (1.82%)  3/55 (5.45%)  4/56 (7.14%) 
Respiratory, thoracic and mediastinal disorders       
Cough  1  3/55 (5.45%)  4/55 (7.27%)  8/56 (14.29%) 
Dyspnoea  1  1/55 (1.82%)  1/55 (1.82%)  3/56 (5.36%) 
Epistaxis  1  3/55 (5.45%)  1/55 (1.82%)  0/56 (0.00%) 
Oropharyngeal pain  1  4/55 (7.27%)  7/55 (12.73%)  3/56 (5.36%) 
Skin and subcutaneous tissue disorders       
Rash  1  3/55 (5.45%)  6/55 (10.91%)  5/56 (8.93%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, 15.0
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.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Study Director
Organization: Novartis Pharmaceuticals
Phone: 862-778-8300
Layout table for additonal information
Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT00873041     History of Changes
Obsolete Identifiers: NCT01185106
Other Study ID Numbers: CICL670A2209
EudraCT 2007-007000-15 ( Registry Identifier: EudraCT )
First Submitted: March 30, 2009
First Posted: April 1, 2009
Results First Submitted: June 20, 2012
Results First Posted: September 25, 2012
Last Update Posted: July 9, 2013