Combination Deferasirox and Deferiprone for Severe Iron Overload in Thalassemia

• Status: Completed
• Trial end date: 2017-08-01
• Target enrollment: 0
• Participant gender: All

Summary

We hypothesize that the combination treatment with deferasirox and deferiprone will be well tolerated and will result in significant improvement in cardiac and liver iron levels.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Children's Hospital of Philadelphia
• Collaborators: Ann & Robert H Lurie Children's Hospital of Chicago; The Cooley's Anemia Foundation,; The Cooley’s Anemia Foundation,
• Treatments: Deferasirox; Deferiprone

Criteria

Inclusion Criteria:

• Alpha or beta thalassemia

• Receiving chronic transfusions (at least 20 transfusions in lifetime) with iron overload requiring treatment with chelation

• Serum ferritin >500 ng/ml

• Liver iron concentration equal to or greater than 10 mg/g dw (by R2 MRI) or 7 to 10 mg/g dw (by R2 MRI) and not improving OR cardiac T2* between 6 and <20 ms

• Women of childbearing age must have a negative pregnancy test

• Agree to use approved method of contraception for the duration of the study

• Subjects must have a good understanding of the study and be willing to comply with study procedures

Exclusion Criteria:

• Subjects with past history of unexplained neutropenia (ANC < 1500/mcL), clinically significant renal disease (creatinine above the upper limit of normal), proteinuria >300 mg/L, clinically significant liver disease (ALT > 5x upper limit of normal), pulmonary or cardiovascular disease

• History of other clinically relevant oral, endocrine, neurologic, psychiatric, immunologic, bone marrow or skin disorder that contraindicates dosing with deferasirox or deferiprone

• History of adverse reaction or known allergy to either deferasirox or deferiprone necessitating drug discontinuation

• Currently receiving treatment for active hepatitis

• Use of any investigational agent in the past 30 days

• Cardiac T2* <6 ms, left ventricular ejection fraction < 56%, and/or arrhythmia (certain subjects may be eligible if they have already had a trial of deferoxamine and deferiprone). Subjects who refuse to use deferoxamine after extensive consultation with at least 2 health care providers will also be allowed to participate.

• Pregnant or breastfeeding females

• Unwilling or unable to comply with study related procedures