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

Status

Recruiting

Phases

Phase 3

Study type

Interventional

Source

TCTR

Registry ID

TCTR20240612005

Enrollment

Registered

2024-06-12

Start date

2023-04-05

Completion date

Unknown

Last updated

2026-08-03

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

None listed

Interventions

Enrolled patients will be prescribed deferasirox GPO (dispersible tablet) at the dose of 20-40 mg/kg/day to be taken once daily 30 minutes before meal.

Experimental Drug

Deferasirox

Eligibility

Sex/Gender

All

Age

18 Months to 20 Years

Inclusion criteria

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

Exclusion criteria: 1. Patients and/or guardians denied consent 2. Patients have been diagnosed with thalassemia 3. History of deferasirox allergy

Design outcomes

Primary

Measure	Time frame
Iron overload in IHA patients 12 months Serum ferritin levels	—

Secondary

Measure	Time frame
Safety of deferasirox in IHA 12 months Report of adverse events	—

Countries

Thailand

Contacts

Public ContactDuantida Songdej

Department of Pediatrics, Faculty of Medicine Ramathibodi Hospital, Mahidol University, Thailand

duantida.songdej@gmail.com022011749

Outcome results