Efficacy and Safety of Deferasirox in Patients With Chronic Anemia and Transfusional Hemosiderosis
- Conditions
- Chronic AnemiaTransfusional Hemosiderosis
- Interventions
- Registration Number
- NCT00631163
- Lead Sponsor
- Novartis Pharmaceuticals
- Brief Summary
The overall purpose of this trial is to further evaluate the efficacy and safety of deferasirox, dosed initially according to the transfusional iron intake, in patients with transfusion dependant anemia related to disorders other than β-thalassemia and sickle cell disease.
During the study, the dose will be adjusted based on serum Ferritin.The overall purpose of the extension is to allow further treatment of patients who have already completed the core study, and to enable collection of long term efficacy and safety data. Patients will continue to receive Deferasirox at the dose they received at the end of the core study.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 102
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Deferasirox Deferasirox (ICL670) Participants received initial dose of 20 milligrams per kilogram (mg/kg) Deferasirox tablets was administered orally once daily (OD) based on the Participants body weight. The dose of Deferasirox was adjusted to either 10 mg/kg or 30 mg/kg based on the volumes of blood transfusions being administered.
- Primary Outcome Measures
Name Time Method Absolute Change From Baseline in Liver Iron Concentration (LIC) to Year 1 From the Baseline, Year 1 (End of core study) Liver iron concentration (LIC), a predictor of iron burden, was measured using relaxation rate magnetic resonance imaging (R2-MRI) technique. Relaxation rate was determined as R2 = 1/relaxation time (T2). The baseline value of LIC of participants was categorized as \< 7, ≥ 7 to \< 15, and ≥ 15 milligram of iron/tissue dry weight (mg Fe/g dw). A negative change from baseline favored study treatment in reducing LIC.
- Secondary Outcome Measures
Name Time Method Total Body Iron Elimination Rate (TBIE), Iron Intake, Iron Excretion/Iron Intake and Chelation Efficiency After 2 Years From the Baseline, Year 2 (End of extension study) Total body iron excretion (TBIE)was used to investigate the chelation efficacy of Deferasirox therapy. TBIE rate was estimated based on the iron influx as determined by the amount of red cells transfused and the change in total body iron (TBI) stores.
Correlation of LIC and Serum Ferritin at Core and Extension Study From the Baseline, Year 1 (End of core study), Year 2 (End of extension study) LIC, a predictor of iron burden, was measured using R2-MRI technique. Relaxation rate was determined as R2 = 1/T2. The baseline value of LIC was \< 7, ≥ 7 to \< 15, and ≥ 15 mg Fe/g dw. Serum ferritin was a marker for the monitoring of chelation therapy. Ferritin protein stores iron and provides overall iron levels, higher ferritin in blood showed more iron content. The correlation between absolute change in LIC and absolute change in serum ferritin was determined.
Absolute Change From Baseline in Liver Iron Concentration (LIC) in Japanese Subgroup From the Baseline, End of Year 1 (End of core study), End of Year 2 (End of extension study) Liver Iron Concentration (LIC), a predictor of iron burden, was measured using R2-MRI technique. Relaxation rate was determined as R2 = 1/T2. The baseline value of LIC was \< 7, ≥ 7 to \< 15, and ≥ 15 mg Fe/g dw. A negative change from baseline favoured study treatment in reducing LIC.
Absolute Serum Ferritin Levels Over 2 Years From the Baseline, Year 1 (End of core study), Year 2 (End of extension study) Serum ferritin was a marker for the monitoring of chelation therapy. Ferritin protein stores iron and provides overall iron levels, higher ferritin in blood showed more iron content.
Absolute Change From Baseline in Liver Iron Concentration (LIC) to End of Year 2 From the Baseline to End of Year 2 (End of extension study) Liver Iron Concentration (LIC), a predictor of iron burden, was measured using R2-MRI technique. Relaxation rate was determined as R2 = 1/T2. The baseline value of LIC of participants was categorized as \< 7, ≥ 7 to \< 15, and ≥ 15 mg Fe/g dw. A negative change from baseline favoured study treatment in reducing LIC.
Absolute Change From Baseline in Serum Ferritin Levels to Year 2 From the Baseline up to Year 2 (End of extension study) Serum ferritin was a marker for the monitoring of chelation therapy. Ferritin protein stores iron and provides overall iron levels, higher ferritin in blood showed more iron content.
Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Adverse Event of Special Interest (AESI), Discontinuation and Interruption From the Baseline up to Year 2 (End of extension study) Adverse events (AEs) were defined as any unfavorable and unintended diagnosis, symptom, sign (including an abnormal laboratory finding), syndrome or disease which either occurs during study, having been absent at baseline, or, if present at baseline, appears to worsen. Serious adverse events (SAEs) were defined as any untoward medical occurrences that result in death, are life threatening, require hospitalisation, cause persistent or significant disability/incapacity, result in congenital anomalies or birth defects, or are other conditions which in judgement of investigators represent significant hazards. Death was defined as a fatal event leading to permanent cessation of all vital functions of the body.
Number of Participants With Clinically Significant Ophthalmological Abnormalities At 2 years (End of extension study) Clinically significant changes in left eye and right eye were assessed by the investigator based on methods like visual acuity, slit lamp examination, tonometry and fundus oculi.
Trial Locations
- Locations (1)
Novartis Investigative Site
🇹🇷Izmir, Turkey