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

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 twice 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.

Inclusion Criteria:

1. Male or female aged ≥ 6 months to < 10 years

2. Confirmed diagnosis of beta-thalassemia, as determined by high performance liquid
chromatography (HPLC) or DNA testing

3. Started on a red blood cell (RBC) transfusion regimen

4. Screening levels of serum ferritin greater than >200 μg/L but not more than 600 μg/L
and of transferrin saturation greater than 30% but not more than 60%

Exclusion Criteria:

1. Prior use of iron chelation

2. Diagnosis of hepatitis B or C, or HIV infection

3. Evidence of abnormal liver or kidney function at screening: serum alanine transaminase
(ALT) level > 5 times upper limit of normal or creatinine levels >2 times upper limit
of normal

4. Disorders associated with neutropenia (absolute neutrophil count < 1.5 x 10^9/L) prior
to the initiation of study medication

5. A serious, unstable illness, as judged by the investigator, during the previous 3
months before screening/baseline visit including but not limited to hepatic, renal,
gastro-enterologic, respiratory, cardiovascular, endocrinologic, neurologic or
immunologic disease.

6. Presence of any medical condition which in the opinion of the investigator would cause
participation in the study to be unwise.