Focus on Transfusion Dependent Thalassemia Patients Who Are Inadequate Responders to Deferasirox > 35mg/kgQD Clinical Trial
Official title:
A Prospective Single Arm Study to Assess the Efficacy and Safety of Deferasirox 20 mg/kg BID in Transfusion Dependent Thalassemia Patients Inadequately Responding to Current Treatment With Doses > 35mg/kg QD (Once a Day).
This is an open label, single arm, and multicenter study. The study will include the following phases. A screening phase which lasts for 4 weeks to determine patient eligibility. This phase will be followed by a 24 week Open label treatment phase. The study treatment is defined as deferasirox 20mg/kg BID 9Twice a day). Serum Ferritin Levels and MRI (Magmetic Resonance Imaging) LIC (Liver Iron Concentration) will be measured to evaluate the response to BID.
Study treatment is defined as deferasirox 20 mg/kg BID. The study treatment duration is 24
weeks. After the baseline visit, patient visits will occur weekly during the first month
because key safety parameters need to be performed weekly in the first month of treatment
and then every 4 weeks thereafter until week 24 End of Treatment (EOT).
Patients will have their first dose of study treatment at Visit 3. Safety assessments are
routinely performed including collection of AEs (Adverse Events), SAEs (Serious ADverse
Events<, vital signs, physical examination, ECG (Electrocardiograph), hematological and
biochemistry assessments.
Patients will continue therapy until intolerable toxicity, patient decision or after 24
weeks treatment duration at which point and End of Treatment (EOT) visit will be performed
and the End of Treatment CRF( Case Report Form) will be completed.
30 day Safety follow-up Patients who discontinue study drug before completing the study
should be scheduled for a visit as soon as possible, at which time all of the assessments
listed for the final visit will be performed. At a minimum, all patients who discontinue
study treatment, including those who refuse to return for a final visit, will be contacted
for safety evaluations during the 30 days following the last dose of study drug.
All patients must be followed for AEs and SAEs that may have occurred after discontinuation
from the study treatment. The safety follow-up visit will take place 30 days after the last
dose of study drug.
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