A Phase II, Multi-center, Single-arm, Prospective Study to Evaluate the Safety and Efficacy of Deferasirox in Beta-thalassemia Major Patients After Hematopoietic Stem Cell Transplantation.
Overview
- Phase
- Phase 4
- Intervention
- ICL670
- Conditions
- Iron Overload After Hematopoietic Stem Cell Transplantation (HSCT) in Patients With Beta-thalassemia Major
- Sponsor
- Novartis Pharmaceuticals
- Enrollment
- 27
- Locations
- 1
- Primary Endpoint
- Number of Participants With Adverse Events, Serious Adverse Events and Deaths as a Measure of Safety and Tolerability
- Status
- Completed
- Last Updated
- 9 years ago
Overview
Brief Summary
This was a prospective, single-arm, multicenter, national, phase II clinical study. The purpose of this Phase II study was to examine the safety and efficacy of deferasirox to decrease iron overload (IOL) in the posttransplant period in patients with beta-thalassemia major.
Investigators
Eligibility Criteria
Inclusion Criteria
- Not provided
Exclusion Criteria
- Not provided
Arms & Interventions
ICL670
Oral dose of ICL670 at 10 mg/kg daily
Intervention: ICL670
Outcomes
Primary Outcomes
Number of Participants With Adverse Events, Serious Adverse Events and Deaths as a Measure of Safety and Tolerability
Time Frame: 12 months
To determine the safety; incidence, type and severity of adverse events including renal, hepatic, biochemistry and hematologic parameters of deferasirox in the treatment of iron overload after hematopoietic stem cell transplantation (HSCT) in patients with beta-thalassemia major in 12 months period
Secondary Outcomes
- Change in Serum Ferritin Level.(Baseline, 12 Months)
- The Percentage of Patients Reaching Serum Ferritin Levels Lower Than 500 μg/L(Week 28 and Week 52)
- Change in the Further Parameters of Iron Overload (Cardiac Iron Concentration by Magnetic Resonance Imaging (MRI Examination)(Baseline, 12 month)
- Change in the Further Parameters of Iron Overload (Liver Iron Concentration by Magnetic Resonance Imaging (MRI Examination)(Baseline, 12 month)