Phase 2 Completed N=9 Treatment

Combination Deferasirox and Deferiprone for Severe Iron Overload in Thalassemia

Thalassemia Major With Severe Transfusional Iron Overload

Source: ClinicalTrials.gov NCT01709032 ↗

Enrolled (actual)

Serious AEs

33.3%

Results posted

Mar 2019

Primary outcomePrimary: Number of Participants With Improvement in Liver Iron Concentration — 3 Participants

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.

Outcome Measures

Outcome	Result	p-value
PRIMARY Number of Participants With Improvement in Liver Iron Concentration	3	—
SECONDARY Number of Participants With Improvement in Cardiac T2* MRI	3	—

Eligibility 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 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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01709032). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.