Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload
|ClinicalTrials.gov Identifier: NCT00235391|
Recruitment Status : Completed
First Posted : October 10, 2005
Results First Posted : May 2, 2011
Last Update Posted : June 7, 2011
|Condition or disease||Intervention/treatment||Phase|
|Thalassemia Sickle Cell Disease Diamond Blackfan Anemia Myelofibrosis||Drug: Deferasirox||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||1683 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Study to Provide Expanded Access of (Exjade®) Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload From Blood Transfusions Who Cannot Adequately be Treated With Other Locally Approved Iron Chelators|
|Study Start Date :||October 2005|
|Primary Completion Date :||October 2008|
|Study Completion Date :||October 2008|
Deferasirox was administered orally once a day, 30 minutes prior to breakfast. Dosage was based on participant's body weight. Starting dose was determined by the frequency of blood transfusions and recommended initial daily dose of deferasirox is 20 mg/kg body weight for patients receiving blood transfusion, 10 mg/kg for patients receiving less frequent transfusion/exchange transfusion and 30 mg/kg for patients receiving more frequent blood transfusions.
125 mg, 250 mg and 500 mg tablets. Dosage was calculated based on participant's body weight. Tablets were dispersed in water, orange or apple juice and taken orally once a day.
- Safety Profile of Deferasirox Based Upon Drug Administration and Reporting of Serious Adverse Events [ Time Frame: Baseline to end of study (Median exposure time to drug was approximately 30 weeks; Maximum exposure was 104 weeks) ]Safety as assessed by the number of participants with death, serious adverse events (SAE), and/or Adverse Events (AEs) leading to study drug interruption or discontinuation. Note: only treatment emergent AEs are summarized.
- The Change in Serum Ferritin Values From Baseline Through Completion of the Study [ Time Frame: Baseline to end of study (Median exposure time to drug was approximately 30 weeks; Maximum exposure was 104 weeks) ]The number of participants with Improvement, No Change or Worsening in Serum ferritin category levels at the end of the study compared to baseline. Serum ferritin levels in µg/L were divided into to 6 categories: (<1000), (1000-<2500), (2500-<4000), (4000-<5500), (5500-<7000) and (>=7000). Improvement was defined as a shift to a lower category at the end of study compared to the category at baseline. Worsening was defined as a shift to a higher category at the end of the study compared to the category at baseline. No change was no change in category at end of study from baseline.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00235391
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|Study Director:||Novartis Pharmaceuticals||Novartis Pharmaceuticals|