An Open-Label Single-Arm Clinical Trial to Evaluate the Safety and Efficacy of Deferasirox in Pediatric Patients with Transfusion-Dependent Rare Inherited Hemolytic Anemia
Phase 3
Recruiting
- Conditions
- - Transfusion-dependent RBC membranopathy- Transfusion-dependent RBC enzymopathy- Transfusion-dependent KLF1 insufficiencyDeferasirox in non-thalassemic inherited hemolytic anemia (IHA)
- Registration Number
- TCTR20240612005
- Lead Sponsor
- Faculty of Medicine Ramathibodi Hospital, Mahidol University, Thailand
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 20
Inclusion Criteria
1. Patients diagnosed with IHA (RBC membranopathy, enzymopathy, or KLF1 insufficiency) aged 18 months to 20 years who have been followed up at Hematology Clinic, Department of Pediatrics, Faculty of Medicine Ramathibodi Hospital, Mahidol University, Thailand
2. Transfusion-dependent patients (have been receiving RBC transfusion at the interval of 8 weeks or shorter for at least 6 months)
3. Serum ferritin level 1,000 ng/mL or more measured for 2 consecutive times (at least 1 month apart)
Exclusion Criteria
1. Patients and/or guardians denied consent
2. Patients have been diagnosed with thalassemia
3. History of deferasirox allergy
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Iron overload in IHA patients 12 months Serum ferritin levels
- Secondary Outcome Measures
Name Time Method Safety of deferasirox in IHA 12 months Report of adverse events