Deferasirox BID (Twice a Day) in Transfusion Dependent Thalassemia Patients With Inadequate Response to High Doses
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|ClinicalTrials.gov Identifier: NCT01948817|
Recruitment Status : Withdrawn
First Posted : September 24, 2013
Last Update Posted : April 20, 2017
|Condition or disease||Intervention/treatment||Phase|
|Focus on Transfusion Dependent Thalassemia Patients Who Are Inadequate Responders to Deferasirox > 35mg/kgQD||Drug: Deferasirox||Phase 2|
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.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||0 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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).|
|Study Start Date :||February 2014|
|Estimated Primary Completion Date :||February 2016|
|Estimated Study Completion Date :||February 2016|
Deferasirox 20mg/kg taken BID
Deferasirox 20mg/kg taken BID
- Mean relative change in SF (Serum Ferritin) [ Time Frame: Baseline, 24 Weeks ]To evaluate the efficacy of a twice-daily dose regimen of deferasirox in inadequate-responders to a once daily dose regimen as measured by relative change in serum ferritin from baseline to 24 weeks of treatment
- Mean Absolute/relative change in LIC [ Time Frame: Baseline, 24 Weeks ]To evaluate the efficacy of BID deferasirox in liver iron removal as measured by MRI by absolute/relative change from baseline to 24 weeks of treatment
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01948817
|Study Director:||Novartis Pharmaceuticals||Novartis Pharmaceuticals|