A prospective single arm study to assess the efficacy and safety of deferasirox in transfusion dependent beta-thalassemia patients unresponsive to current treatment or with intolerance to single dose therapy
- Conditions
- Thalassemia.Beta thalassaemia
- Registration Number
- IRCT2014122920051N1
- Lead Sponsor
- ovartis
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Complete
- Sex
- All
- Target Recruitment
- 40
Inclusion: Aged 2 years; Serum ferritin >2000 ng/ml and unresponsive to current treatment with doses > 35 mg/kg QD (decrease in ferritin level less than 10%); patients who did not tolerate single dose therapy due to adverse effects
Exclusion: Patients will be excluded from this trial if they have one of the following criteria: positive serology for HBS Ag , HCV Ab, HIV Ab. Abnormal liver or kidney function tests;An alanine aminotranferase (ALT) level greater than 250 U/L;serum creatinine level above the upper limit of normal, and a urinary protein-creatinine ratio of greater than 0.5 mg/mg); a history of ocular toxicity related to iron chelation therapy;uncontrolled systemic hypertention, a prolonged corrected QT interval; systemic infection within 10days prior to enrollment; gastrointestinal absorption problem ;Patients who found complication of chelation therapy during trial as well as breast feeding and pregnant women.
Not provided
Study & Design
- Study Type
- interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Efficacy as reduction in serum ferritin levels as well as change in T2MRI. Timepoint: Base line,three month,six month,nine month,end of study. Method of measurement: For better evaluation of serum ferritin changes during one year, we define an index: Mean of Relative Change of Serum Ferritin (MRC-SF) from the baseline levels that will be calculated as the percentage of difference between mean serum ferritin levels of each occasion from mean baseline serum ferritin levels.
- Secondary Outcome Measures
Name Time Method Gastrointestinal symptoms and skin rash fatal, acute, irreversible renal failure. Timepoint: Baseline,three months,six months,nine months,End of study. Method of measurement: Exact follow up of the patients examination,laboratory data.;Safety. Timepoint: 12 years. Method of measurement: exact follow up of the patients examination and laboratory data.