Phase 2
N=9
Combination Deferasirox and Deferiprone for Severe Iron Overload in Thalassemia
Thalassemia Major With Severe Transfusional Iron Overload
Bottom Line
View on ClinicalTrials.gov: NCT01709032 ↗Enrolled (actual)
9
Serious AEs
33.3%
Results posted
Mar 2019
Primary outcome: Primary: Number of Participants With Improvement in Liver Iron Concentration — 3 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Deferasirox and deferiprone (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Children's Hospital of Philadelphia
- Primary completion
- Aug 2015
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 | — |
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.
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
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.