Efficacy and Safety of Deferasirox in Non-transfusion Dependent Thalassemia Patients With Iron Overload and a One Year Open-label Extension Study
NCT ID: NCT00873041
Last Updated: 2013-07-09
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE2
166 participants
INTERVENTIONAL
2008-11-30
2012-06-30
Brief Summary
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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).
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
TRIPLE
Study Groups
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5 mg/kg/day deferasirox
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.
deferasirox
Supplied as 125 mg, 250 mg and 500 mg tablets.
placebo
Supplied as matching 125 mg, 250 mg and 500 mg tablets.
10 mg/kg/day deferasirox
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.
deferasirox
Supplied as 125 mg, 250 mg and 500 mg tablets.
placebo
Supplied as matching 125 mg, 250 mg and 500 mg tablets.
5 mg/kg/day placebo
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
Supplied as matching 125 mg, 250 mg and 500 mg tablets.
10 mg/kg/day placebo
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.
placebo
Supplied as matching 125 mg, 250 mg and 500 mg tablets.
Interventions
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deferasirox
Supplied as 125 mg, 250 mg and 500 mg tablets.
placebo
Supplied as matching 125 mg, 250 mg and 500 mg tablets.
Eligibility Criteria
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Inclusion Criteria
* Serum ferritin \>300 ng/mL at screening
* Patients who completed the core CICL670A2209 clinical trial
* Written informed consent obtained prior entry to one year extension study CICL670A2209
Exclusion Criteria
* 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
* 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.)
10 Years
ALL
No
Sponsors
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Novartis Pharmaceuticals
INDUSTRY
Responsible Party
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Principal Investigators
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Novartis Pharmaceuticals
Role: STUDY_DIRECTOR
Novartis Pharmaceuticals
Locations
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Children's Hospital & Research Center Oakland
Oakland, California, United States
Children's Memorial Hospital/Division of Hematology/Oncology
Chicago, Illinois, United States
New York Presbyterian Hospital/Weill Medical College of Cornell University
New York, New York, United States
Novartis Investigative Site
Athens, , Greece
Novartis Investigative Site
Pátrai, , Greece
Novartis Investigative Site
Thessaloniki, , Greece
Novartis Investigative Site
Cagliari, , Italy
Novartis Investigative Site
Genova, , Italy
Novartis Investigative Site
Milan, , Italy
Novartis Investigative Site
Napoli, , Italy
Novartis Investigative Site
Rome, , Italy
Novartis Investigative Site
Beirut, , Lebanon
Novartis Investigative Site
Ampang Selangor, , Malaysia
Novartis Investigative Site
Kuala Lumpur, , Malaysia
Novartis Investigative Site
Taipei, , Taiwan
Novartis Investigative Site
Bangkok, , Thailand
Novartis Investigative Site
Adana, , Turkey (Türkiye)
Novartis Investigative Site
Ankara, , Turkey (Türkiye)
Novartis Investigative Site
Istanbul, , Turkey (Türkiye)
Novartis Investigative Site
Izmir, , Turkey (Türkiye)
Novartis Investigative Site
London, , United Kingdom
Countries
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References
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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.
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.
Related Links
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Visit NovartisClinicalTrials.com: Pre-qualify for a trial, and view a list of trials and participating study centers.
Other Identifiers
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EudraCT 2007-007000-15
Identifier Type: REGISTRY
Identifier Source: secondary_id
CICL670A2209
Identifier Type: -
Identifier Source: org_study_id
NCT01185106
Identifier Type: -
Identifier Source: nct_alias
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