NCT03591575

Safety and Efficacy of Early Treatment With Deferiprone in Infants and Young Children

Safety and Efficacy of Early-start Deferiprone Treatment in Infants and Young Children Newly Diagnosed With Transfusion-dependent Beta Thalassemia

• Status: Completed
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 64 (actual)
• Sponsor: Chiesi Canada Corp · Industry
• Sex: All
• Age: 6 Months – 9 Years
• Healthy volunteers: Not accepted

Summary

This study is looking at the effects of giving early treatment of deferiprone to young children with beta thalassemia who have started receiving regular blood transfusions but have not yet reached the criteria for starting on iron chelation therapy. Half the patients in the study will receive deferiprone, and the other half will receive placebo, for up to 12 months.

Detailed description

This study will give deferiprone to infants and young children with thalassemia who have started receiving regular blood transfusions but whose iron load is not yet at the level where chelation treatment would normally begin. The purpose is to see if doing this will postpone the build-up of iron without causing serious side effects. Half the children in the study will be given deferiprone at a dose that is lower than what is normally prescribed, and the other half will be given placebo. All patients will receive the assigned product three times a day for up to 12 months. Tests for signs of iron overload will be done monthly, and a patient whose iron load reaches the level where chelation therapy would normally begin will be immediately taken out of the study and started on standard chelation therapy.

Conditions

• Beta Thalassemia Major Anemia
• Iron Overload

Interventions

Type	Name	Description
DRUG	Deferiprone oral solution	Liquid formulation of deferiprone, with a concentration of 80 mg/mL
DRUG	Placebo	Liquid solution that matches deferiprone oral solution in appearance and taste

Timeline

• Start date: 2018-11-09
• Primary completion: 2020-09-29
• Completion: 2020-09-29
• First posted: 2018-07-19
• Last updated: 2024-03-15
• Results posted: 2024-03-15

Locations

4 sites across 2 countries: Egypt, Indonesia

Regulatory

• FDA-regulated drug study