Plain-English translation of NCT01825512 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
Read our Beta-thalassemia research guide →Phase 3 — Testing in thousands of people, comparing the treatment against what doctors currently use. This is the last big step before approval.
This trial tested , an oral medication that removes excess iron from the body, compared to another iron-removal medication called in children with inherited blood disorders. Children with conditions like thalassemia and sickle cell disease often need regular blood transfusions to survive, but this causes dangerous iron to build up in their bodies. The study examined whether this medication was as effective as the existing treatment at safely reducing iron levels.
While doctors have used this medication successfully in older children and adults, there was limited safety and effectiveness data for younger children. This trial was designed to fill that gap and provide doctors with better information about using the medication in very young patients.
Participants received either or as a daily oral medication, taken seven days a week. The dosage was adjusted based on the child's weight (75–100 mg/kg per day for , or 20–40 mg/kg per day for ). During the study, children had regular blood tests to measure iron levels, and children aged 10 and older underwent heart imaging scans (cardiac MRI) to assess how well the medication was protecting their heart from iron damage.
AI-generated summary from trial data · Aug 22, 2026 · Not medical advice
Albania
Cyprus
Egypt