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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)

Primary Purpose

Non-transfusion Dependent Thalassemia

Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
deferasirox
placebo
Sponsored by
Novartis Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Non-transfusion Dependent Thalassemia focused on measuring Thalassemia, thalassemia intermedia, alpha-thalassemia, beta-thalassemia, deferasirox, iron overload, non-transfusion dependent

Eligibility Criteria

10 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Core Inclusion Criteria:

  • Male or female aged ≥ 10 years with non-transfusion dependent syndromes, not requiring transfusion within 6 months prior to study start. Note: there was a local country amendment for Greece only to change the age specific inclusion criteria to ≥ 18 years old
  • Liver iron concentration ≥ 5 mg/g dry weight measured by Magnetic resonance imaging (MRI) before study start
  • Serum ferritin >300 ng/mL at screening

Core Exclusion Criteria:

  • Hemoglobin S (HbS)-variants of thalassemia syndromes
  • Anticipated regular transfusion program during the study. Patients having a sporadic transfusion (e.g. in case of infection) throughout the study course will not be excluded
  • Any blood transfusion 6 months prior to study start
  • Creatinine clearance ≤ 60 mL/min at screening
  • Serum creatinine above the upper limit of normal at both screening visits
  • Significant proteinuria as indicated by a urine protein/urine creatinine ratio > 1.0 mg/mg
  • Alanine aminotransferase (ALT) of > 5 x the upper limit of normal at both screening visits
  • Concomitant therapy with hydroxyurea, erythropoietin, butyrate
  • History of deferasirox treatment
  • Pediatric patients: a patient's weight of below 20 kg

Extension Inclusion Criteria:

  • Patients who completed the core CICL670A2209 clinical trial
  • Written informed consent obtained prior entry to one year extension study CICL670A2209

Extension Exclusion Criteria:

  • Patients with a continuous increase in serum creatinine ≥ 33% above the baseline value and > ULN who did not improve after drug interruption or dose reduction in the core study
  • Patients with a continuous increase in ALT greater than 2 times the baseline value and > 5 times ULN who did not improve after drug interruption or dose reduction in the core study
  • Patients with progressive proteinuria, as assessed by the investigator, who did not improve after drug interruption or dose reduction in the core study
  • Significant medical condition interfering with the ability to partake in this study (e.g.systemic uncontrolled hypertension, unstable cardiac disease not controlled by standard medical therapy, systemic disease (cardiovascular, renal, hepatic, etc.)

Other protocol-defined inclusion/exclusion criteria may apply

Sites / Locations

  • Children's Hospital & Research Center Oakland
  • Children's Memorial Hospital/Division of Hematology/Oncology
  • New York Presbyterian Hospital/Weill Medical College of Cornell University
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Experimental

Experimental

Placebo Comparator

Placebo Comparator

Arm Label

5 mg/kg/day deferasirox

10 mg/kg/day deferasirox

5 mg/kg/day placebo

10 mg/kg/day placebo

Arm 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 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.

Placebo tablet matching 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.

Outcomes

Primary Outcome Measures

Core Study: Change in Liver Iron Concentration (LIC) From Baseline to Week 52
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.
Extension Study: Percentage of Participants Reaching a Liver Iron Concentration (LIC) < 5 mg Fe/g dw From Core Baseline to End of Extension Study
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.

Secondary Outcome Measures

Core Study: Change in Liver Iron Concentration (LIC) From Baseline to Week 24
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.
Core Study: Change in Serum Ferritin Between Baseline and Fourth Quarter
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.
Core Study: Change in Serum Ferritin Between Baseline and Second Quarter
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.
Core Study: Percentage of Participants With Adverse Events Graded Mild, Moderate and Severe
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.
Core Study: Change in Liver Iron Concentration (LIC) From Baseline At Week 24 and Week 52 in Patients With Dose Increases After Week 24
LIC was measured by magnetic resonance imaging technique at baseline, Week 24 and Week 52. Dose Doubling (Dose Increases) began at Week 24.
Core Study: Correlation Between Serum Ferritin and LIC (Liver Iron Concentration)
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.
Core Study: Change From Baseline in Hemoglobin at Month 12
Blood was collected for Hemoglobin at baseline and Month 12. Change from baseline= Month 12 hemoglobin - baseline hemoglobin.
Core Study: Change From Baseline in Transferrin Saturation at Month 12
Blood was collected for transferrin saturation at Baseline and Month 12. Change from baseline= Month 12 transferrin saturation - baseline transferrin saturation.
Core Study: Change in Liver Iron Concentration (LIC) in Placebo Patients From Baseline to Week 52
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.
Core Study: Percentage of Participants With Notable Abnormal Post-baseline Laboratory Results
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)
Core Study: Percentage of Participants With Notably Abnormal Post-baseline Systolic Blood Pressure
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
Core Study: Percentage of Participants With Notably Abnormal Post-baseline Diastolic Blood Pressure
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
Core Study: Percentage of Participants With Notably Abnormal Post-baseline Pulse Rate
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
Extension Study: Absolute Change in Serum Ferritin From Baseline to Eighth Quarter
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.
Extension Study: Change in Liver Iron Concentration (LIC) From Baseline at Month 24
LIC was measured by magnetic resonance imaging technique at Baseline and Month 24. A negative change from baseline indicated improvement.
Extension Study: Correlation Between Serum Ferritin and LIC (Liver Iron Concentration)
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.
Extension Study: Change From Baseline in Hemoglobin at Month 24
Blood was collected for Hemoglobin at Baseline and Month 24. Change from Baseline= Month 24 hemoglobin - Baseline hemoglobin.
Extension Study: Change From Baseline in Transferrin Saturation at Month 24
Blood was collected for transferrin saturation at Baseline and Month 24. Change from baseline= Month 24 transferrin saturation - baseline transferrin saturation.

Full Information

First Posted
March 30, 2009
Last Updated
May 20, 2013
Sponsor
Novartis Pharmaceuticals
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1. Study Identification

Unique Protocol Identification Number
NCT00873041
Brief Title
Efficacy and Safety of Deferasirox in Non-transfusion Dependent Thalassemia Patients With Iron Overload and a One Year Open-label Extension Study
Acronym
THALASSA
Official Title
A Randomized, Double-blind, Placebo-controlled, Phase II Study to Evaluate Efficacy and Safety of Deferasirox in Non-transfusion-dependent Thalassemia Patients With Iron Overload
Study Type
Interventional

2. Study Status

Record Verification Date
May 2013
Overall Recruitment Status
Completed
Study Start Date
November 2008 (undefined)
Primary Completion Date
June 2011 (Actual)
Study Completion Date
June 2012 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Novartis Pharmaceuticals

4. Oversight

5. Study Description

Brief Summary
CICL670A2209: This study will evaluate the safety and efficacy of deferasirox in non-transfusion dependent thalassemia patients with iron overload. Patients will be treated either with active treatment (deferasirox) or placebo for 12 months (core study phase). Patients who complete the core study phase will be offered to continue their study with the active treatment (deferasirox) in a 12 months extension phase. During the core and extension, the effects of treatment on iron overload in the liver will be evaluated using magnetic resonance imaging (MRI) assessments. CICL670A2209E1: A one-year open-label extension to a randomized, double-blind, placebo-controlled, phase II study to evaluate efficacy and safety of deferasirox in non-transfusion dependent thalassemia patients with iron overload (Thalassa).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Non-transfusion Dependent Thalassemia
Keywords
Thalassemia, thalassemia intermedia, alpha-thalassemia, beta-thalassemia, deferasirox, iron overload, non-transfusion dependent

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigator
Allocation
Randomized
Enrollment
166 (Actual)

8. Arms, Groups, and Interventions

Arm Title
5 mg/kg/day deferasirox
Arm Type
Experimental
Arm 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.
Arm Title
10 mg/kg/day deferasirox
Arm Type
Experimental
Arm Description
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.
Arm Title
5 mg/kg/day placebo
Arm Type
Placebo Comparator
Arm Description
Placebo tablet matching 5 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.
Arm Title
10 mg/kg/day placebo
Arm Type
Placebo Comparator
Arm Description
Placebo tablet matching 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.
Intervention Type
Drug
Intervention Name(s)
deferasirox
Intervention Description
Supplied as 125 mg, 250 mg and 500 mg tablets.
Intervention Type
Drug
Intervention Name(s)
placebo
Intervention Description
Supplied as matching 125 mg, 250 mg and 500 mg tablets.
Primary Outcome Measure Information:
Title
Core Study: Change in Liver Iron Concentration (LIC) From Baseline to Week 52
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
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
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)
Secondary Outcome Measure Information:
Title
Core Study: Change in Liver Iron Concentration (LIC) From Baseline to Week 24
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
Title
Core Study: Change in Serum Ferritin Between Baseline and Fourth Quarter
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])
Title
Core Study: Change in Serum Ferritin Between Baseline and Second Quarter
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)
Title
Core Study: Percentage of Participants With Adverse Events Graded Mild, Moderate and Severe
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
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
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
Title
Core Study: Correlation Between Serum Ferritin and LIC (Liver Iron Concentration)
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
Title
Core Study: Change From Baseline in Hemoglobin at Month 12
Description
Blood was collected for Hemoglobin at baseline and Month 12. Change from baseline= Month 12 hemoglobin - baseline hemoglobin.
Time Frame
Baseline, Month 12
Title
Core Study: Change From Baseline in Transferrin Saturation at Month 12
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
Title
Core Study: Change in Liver Iron Concentration (LIC) in Placebo Patients From Baseline to Week 52
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
Title
Core Study: Percentage of Participants With Notable Abnormal Post-baseline Laboratory Results
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
Title
Core Study: Percentage of Participants With Notably Abnormal Post-baseline Systolic Blood Pressure
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
Title
Core Study: Percentage of Participants With Notably Abnormal Post-baseline Diastolic Blood Pressure
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
Title
Core Study: Percentage of Participants With Notably Abnormal Post-baseline Pulse Rate
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
Title
Extension Study: Absolute Change in Serum Ferritin From Baseline to Eighth Quarter
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)
Title
Extension Study: Change in Liver Iron Concentration (LIC) From Baseline at Month 24
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
Title
Extension Study: Correlation Between Serum Ferritin and LIC (Liver Iron Concentration)
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
Title
Extension Study: Change From Baseline in Hemoglobin at Month 24
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
Title
Extension Study: Change From Baseline in Transferrin Saturation at Month 24
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

10. Eligibility

Sex
All
Minimum Age & Unit of Time
10 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Core Inclusion Criteria: Male or female aged ≥ 10 years with non-transfusion dependent syndromes, not requiring transfusion within 6 months prior to study start. Note: there was a local country amendment for Greece only to change the age specific inclusion criteria to ≥ 18 years old Liver iron concentration ≥ 5 mg/g dry weight measured by Magnetic resonance imaging (MRI) before study start Serum ferritin >300 ng/mL at screening Core Exclusion Criteria: Hemoglobin S (HbS)-variants of thalassemia syndromes Anticipated regular transfusion program during the study. Patients having a sporadic transfusion (e.g. in case of infection) throughout the study course will not be excluded Any blood transfusion 6 months prior to study start Creatinine clearance ≤ 60 mL/min at screening Serum creatinine above the upper limit of normal at both screening visits Significant proteinuria as indicated by a urine protein/urine creatinine ratio > 1.0 mg/mg Alanine aminotransferase (ALT) of > 5 x the upper limit of normal at both screening visits Concomitant therapy with hydroxyurea, erythropoietin, butyrate History of deferasirox treatment Pediatric patients: a patient's weight of below 20 kg Extension Inclusion Criteria: Patients who completed the core CICL670A2209 clinical trial Written informed consent obtained prior entry to one year extension study CICL670A2209 Extension Exclusion Criteria: Patients with a continuous increase in serum creatinine ≥ 33% above the baseline value and > ULN who did not improve after drug interruption or dose reduction in the core study Patients with a continuous increase in ALT greater than 2 times the baseline value and > 5 times ULN who did not improve after drug interruption or dose reduction in the core study Patients with progressive proteinuria, as assessed by the investigator, who did not improve after drug interruption or dose reduction in the core study Significant medical condition interfering with the ability to partake in this study (e.g.systemic uncontrolled hypertension, unstable cardiac disease not controlled by standard medical therapy, systemic disease (cardiovascular, renal, hepatic, etc.) Other protocol-defined inclusion/exclusion criteria may apply
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Novartis Pharmaceuticals
Organizational Affiliation
Novartis Pharmaceuticals
Official's Role
Study Director
Facility Information:
Facility Name
Children's Hospital & Research Center Oakland
City
Oakland
State/Province
California
ZIP/Postal Code
94609-1809
Country
United States
Facility Name
Children's Memorial Hospital/Division of Hematology/Oncology
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60614-3394
Country
United States
Facility Name
New York Presbyterian Hospital/Weill Medical College of Cornell University
City
New York
State/Province
New York
ZIP/Postal Code
10021
Country
United States
Facility Name
Novartis Investigative Site
City
Athens
Country
Greece
Facility Name
Novartis Investigative Site
City
Patras
Country
Greece
Facility Name
Novartis Investigative Site
City
Thessaloniki
Country
Greece
Facility Name
Novartis Investigative Site
City
Cagliari
Country
Italy
Facility Name
Novartis Investigative Site
City
Genova
Country
Italy
Facility Name
Novartis Investigative Site
City
Milano
Country
Italy
Facility Name
Novartis Investigative Site
City
Napoli
Country
Italy
Facility Name
Novartis Investigative Site
City
Rome
Country
Italy
Facility Name
Novartis Investigative Site
City
Beirut
Country
Lebanon
Facility Name
Novartis Investigative Site
City
Ampang Selangor
Country
Malaysia
Facility Name
Novartis Investigative Site
City
Kuala Lumpur
Country
Malaysia
Facility Name
Novartis Investigative Site
City
Taipei
Country
Taiwan
Facility Name
Novartis Investigative Site
City
Bangkok
Country
Thailand
Facility Name
Novartis Investigative Site
City
Adana
Country
Turkey
Facility Name
Novartis Investigative Site
City
Ankara
Country
Turkey
Facility Name
Novartis Investigative Site
City
Istanbul
Country
Turkey
Facility Name
Novartis Investigative Site
City
Izmir
Country
Turkey
Facility Name
Novartis Investigative Site
City
London
Country
United Kingdom

12. IPD Sharing Statement

Citations:
PubMed Identifier
25212456
Citation
Taher AT, Porter JB, Viprakasit V, Kattamis A, Chuncharunee S, Sutcharitchan P, Siritanaratkul N, Origa R, Karakas Z, Habr D, Zhu Z, Cappellini MD. Defining serum ferritin thresholds to predict clinically relevant liver iron concentrations for guiding deferasirox therapy when MRI is unavailable in patients with non-transfusion-dependent thalassaemia. Br J Haematol. 2015 Jan;168(2):284-90. doi: 10.1111/bjh.13119. Epub 2014 Sep 12.
Results Reference
derived
PubMed Identifier
23553596
Citation
Taher AT, Porter JB, Viprakasit V, Kattamis A, Chuncharunee S, Sutcharitchan P, Siritanaratkul N, Galanello R, Karakas Z, Lawniczek T, Habr D, Ros J, Zhang Y, Cappellini MD. Deferasirox demonstrates a dose-dependent reduction in liver iron concentration and consistent efficacy across subgroups of non-transfusion-dependent thalassemia patients. Am J Hematol. 2013 Jun;88(6):503-6. doi: 10.1002/ajh.23445. Epub 2013 May 13.
Results Reference
derived
Links:
URL
http://www.novartisclinicaltrials.com/etrials/searchTrial.do?trialID=717
Description
Visit NovartisClinicalTrials.com: Pre-qualify for a trial, and view a list of trials and participating study centers.

Learn more about this trial

Efficacy and Safety of Deferasirox in Non-transfusion Dependent Thalassemia Patients With Iron Overload and a One Year Open-label Extension Study

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