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| Sponsor: | Novartis Pharmaceuticals |
|---|---|
| Information provided by (Responsible Party): | Novartis ( Novartis Pharmaceuticals ) |
| ClinicalTrials.gov Identifier: | NCT00171301 |
Purpose
To allow patients treated with deferasirox in the core study to continue iron chelation therapy for 2 years or until the drug became locally commercially available. To evaluate the long-term safety and efficacy of deferasirox by measuring treatment success, change in liver iron content (LIC) and change in serum ferritin levels. Safety was mainly assessed by incidence of adverse events (AEs)and clinically significant lab parameters.
| Condition | Intervention | Phase |
|---|---|---|
|
Beta-thalassemia Major Hemosiderosis Iron Overload Rare Anemia |
Drug: Deferasirox |
Phase IV |
| Study Type: | Interventional |
| Study Design: | Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | 1-year Extension to CICL670A2402 an Open-label, Multi-center Trial of the Efficacy and Safety of Long-term Treatment With Deferasirox (10 to 20 mg/kg/Day) in Beta-thalassemia Patients With Transfusional Hemosiderosis (Study Amended to 2- Year Duration) |
Liver MRI or Liver Biopsy was performed at the core study baseline (BL) and then 1 year and 2 years in the core study, baseline of the extension study and time of discontinuation from the extension visit (end of study). Liver iron content (LIC) is reported in milligram Iron per gram dry weight (mg Fe/g dw).
Absolute change in LIC from core study baseline to the end of the extension study is presented for participants with the following two core study baseline LIC levels: 1-<7 mg Fe/g dw and ≥7 mg Fe/g dw.
| Enrollment: | 233 |
| Study Start Date: | June 2005 |
| Study Completion Date: | May 2008 |
| Primary Completion Date: | May 2008 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Experimental: Deferasirox
Deferasirox was given orally once daily (10 to 20 mg/kg) to participants 2 years and older based on participant's body weight.
|
Drug: Deferasirox
Deferasirox was administered orally once daily. Deferasirox was available as 125 mg, 250 mg, and 500 mg tablets.
Other Name: Exjade
|
Iron accumulation is an inevitable consequence of chronic blood transfusions and results in serious complications in the absence of chelation treatment to remove excess iron. Deferasirox (Exjade, ICL670) is an oral chelator with high iron-binding potency and selectivity. This extension study aimed at collecting efficacy and safety data during 2 years of treatment with deferasirox in the extension study or until deferasirox became commercially available in the countries where the centers were located, whichever came first. The population comprised of β-thalassemia patients with transfusional hemosiderosis who could not be satisfactorily treated with deferoxamine or deferiprone.
Eligibility| Ages Eligible for Study: | 2 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Contacts and Locations| Egypt | |
| Novartis Investigative Site | |
| Cairo, Egypt | |
| Lebanon | |
| Novartis Investigative Site | |
| Beirut, Lebanon | |
| Oman | |
| Novartis Investigative Site | |
| Muscat, Oman | |
| Saudi Arabia | |
| Novartis Investigative Site | |
| Riyadh, Saudi Arabia | |
| Syrian Arab Republic | |
| Novartis Investigative Site | |
| Damascus, Syrian Arab Republic | |
| Study Director: | Novartis Pharmaceuticals | Novartis Pharmaceuticals |
More Information
| Responsible Party: | Novartis ( Novartis Pharmaceuticals ) |
| ClinicalTrials.gov Identifier: | NCT00171301 History of Changes |
| Other Study ID Numbers: | CICL670A2402E1 |
| Study First Received: | September 12, 2005 |
| Results First Received: | January 12, 2011 |
| Last Updated: | August 28, 2011 |
| Health Authority: | Lebanon: Institutional Review Board; Egypt: The Egyptian Society for Medical Ethics; Oman: The Research and Ethics Committee; Saudia Arabia: Clinical Research Committee; Syria: Institutional Review Board |
|
Transfusional hemosiderosis Beta-thalassemia major Deferasirox |
iron overload rare anemia iron overload due to transfusion |
|
Anemia Beta-Thalassemia Hemosiderosis Thalassemia Iron Overload Hematologic Diseases Anemia, Hemolytic, Congenital Anemia, Hemolytic Hemoglobinopathies |
Genetic Diseases, Inborn Iron Metabolism Disorders Metabolic Diseases Deferasirox Iron Chelating Agents Chelating Agents Molecular Mechanisms of Pharmacological Action Pharmacologic Actions |