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

• Status: Completed
• Trial end date: 2020-09-29
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 4
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: ApoPharma; Chiesi Canada Corp
• Treatments: Deferiprone; Pharmaceutical Solutions

Criteria

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 level of serum ferritin greater than >200 μg/L but not more than 600 μg/L. Since SF level may be impacted by the presence of infection, it must additionally be verified that the child has had no signs of infection in the previous 7 days, including the day of screening, and that the level of C-reactive protein (CRP) is no greater than 20% higher than the normal range for the patient's age. If there are signs of infection and/or the CRP level is above this threshold, the SF level must be checked again a minimum of one week later.

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.