Combination Deferasirox and Deferiprone for Severe Iron Overload in Thalassemia

Completed · Phase 1/Phase 2

Conditions studied: Thalassemia Major With Severe Transfusional Iron Overload

In brief

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.

Key facts

Study ID

NCT01709032

Run by

Children's Hospital of Philadelphia

People needed

9

Starts

2012-09-01

Expected to finish

2017-08-01

Last updated by the study team

2019-03-06

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States

Full record on ClinicalTrials.gov

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