A Phase 1/2 interventional study of Deferasirox and deferiprone in Thalassemia Major With Severe Transfusional Iron Overload, sponsored by Children's Hospital of Philadelphia. Completed at 2 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2019-03-06.
Sponsored by Children's Hospital of Philadelphia · Phase 1/2, Interventional, and Treatment
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.
Death and disability from iron related damage to the heart remain the most serious issue facing transfusion-dependent patients with thalassemia. However, over the past decade there have been several reports of improved survival and fewer cardiac complications. This improvement may be related to the availability of three chelators and also the accurate measurement of iron stores in various organs (e.g. heart and liver) with magnetic resonance imaging, which allows for personalized, tailored medical care for patients. The chelator characteristics, side effect profiles, and ability to remove iron from specific organs differ among the chelators, suggesting that combination therapy may be beneficial. Using two drugs at lower doses may be more tolerable than escalating doses of a single drug and may improve iron removal. The combination of deferoxamine and deferiprone has been shown to be particularly beneficial for reducing cardiac iron, but it requires a painful injection/infusion, which hinders adherence. This pilot study aims to investigate the safety of an oral-only combination chelator regimen (deferasirox and deferiprone) in individuals with thalassemia major with poorly controlled iron overload and to assess how well this chelator combination lowers iron stores over one year.
416 studies on the registry are indexed under Thalassemia; 67 are open to participants now.
This study's enrollment of 9 is below the median of 37 across 277 interventional studies indexed under Thalassemia.
Browse Thalassemia studies →Children's Hospital of Philadelphia is the lead sponsor of 480 studies on the registry; 85 are open to participants now.
Of its 28 completed or terminated interventional studies of FDA-regulated products, 22 (79%) have results posted.
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Exclusion Criteria:
Drug: Deferasirox and deferiprone
Also known as: Exjade, Ferriprox
Number of Participants With Improvement in Liver Iron Concentration
Determine the safety of the combination of Deferasirox and Deferiprone for the treatment of subjects with Thalassemia Major and Severe Iron Overload by assessing change in liver iron concentration from baseline to follow-up
Time frame: 12 months
Number of Participants With Improvement in Cardiac T2* MRI
Improvement in Cardiac T2\* MRI from baseline to determine if there is a reduction of cardiac iron burden.
Time frame: 12 months
| Milestone | Deferasirox and Deferiprone |
|---|---|
| Started | 9 |
| Completed | 6 |
| Not completed | 3 |
Determine the safety of the combination of Deferasirox and Deferiprone for the treatment of subjects with Thalassemia Major and Severe Iron Overload by assessing change in liver iron concentration from baseline to follow-up
| Participants | Deferasirox and Deferiprone |
|---|---|
| Number of Participants With Improvement in Liver Iron Concentration | 3 |
Improvement in Cardiac T2\* MRI from baseline to determine if there is a reduction of cardiac iron burden.
| Participants | Deferasirox and Deferiprone Combination Chelation |
|---|---|
| Number of Participants With Improvement in Cardiac T2* MRI | 3 |
Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Deferasirox and Deferiprone | 0/9 (0%) | 3/9 (33.3%) | 8/9 (88.9%) |
| Event | Deferasirox and Deferiprone |
|---|---|
| viral syndromeGeneral disorders | 1/9 |
| perirectal abscessInfections and infestations | 1/9 |
| Rectal bleeding from hemorrhoidGastrointestinal disorders | 1/9 |
| Event | Deferasirox and Deferiprone |
|---|---|
| ArthralgiaMusculoskeletal and connective tissue disorders | 4/9 |
| DiarrheaGastrointestinal disorders | 4/9 |
| Nausea/vomitingGastrointestinal disorders | 3/9 |
| Abdominal painGastrointestinal disorders | 2/9 |
| TinnitusEar and labyrinth disorders | 1/9 |
| ALT elevatedHepatobiliary disorders | 1/9 |
| Age, Continuous(years) | Deferasirox and Deferiprone |
|---|---|
| Mean | 28.9 ± 5.6 |
| Sex: Female, Male(Participants) | Deferasirox and Deferiprone |
|---|---|
| Female | 5 |
| Male | 4 |
| Region of Enrollment(Participants) | Deferasirox and Deferiprone |
|---|---|
| United States | 9 |
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Children's Hospital of Philadelphia