A Phase 2 interventional study of Combo Chelation with Deferasirox (Exjade) and Deferoxamine (DFO) in Thalassemia and Iron Overload, sponsored by Elliott Vichinsky. Completed at 1 site in United States. Open to participants aged 8 Years and older. Per ClinicalTrials.gov, last updated 2021-08-12.
Sponsored by Elliott Vichinsky · Phase 2, Interventional, and Treatment
This is a pilot study looking at the safety and efficacy of giving combination chelation with deferasirox and deferoxamine. The hypothesis is that combination chelation is safe in decreasing overall iron in patients with thalassemia.
This was a phase two pilot clinical trial designed to evaluate the safety and efficacy of the combination of deferasirox and deferoxamine in transfusion dependent thalassemia with a range of systemic iron burden. The duration of combined therapy was 12 months. Changes in liver iron concentration, ferritin, myocardial iron, and serum creatinine was monitored.
250 studies on the registry are indexed under Thalassemia; 43 are open to participants now.
This study's enrollment of 22 is below the median of 33 across 170 interventional studies indexed under Thalassemia.
Browse Thalassemia studies →This is the only study on the registry with Elliott Vichinsky as lead sponsor.
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Exclusion Criteria:
All subjects received Deferasirox (Exjade) and Deferoxamine (DFO) dosing based on the iron overload at baseline.
Drug: Combo Chelation with Deferasirox (Exjade) and Deferoxamine (DFO)
Drug page: Deferasirox
All subjects will be given Deferasirox 20-30 mg/kg for 7 days per week. All subject will be given Deferoxamine 50 mg/kg for 3-7 days per week. The number of days for Deferoxamine will be determined by liver iron concentration at baseline.
Also known as: Deferasirox, (Exjade), Deferoxamine (DFO)
Efficacy of Combined Treatment With Deferasirox and Deferoxamine Over 12 Months
Change in liver iron concentration from baseline to 12 months with the use of combined chelation therapy. The change was calculated as the liver iron concentration at 12 months minus the value at baseline.
Time frame: 12 months
Change in Serum Creatinine During 12 Months Combined Chelation Therapy
Comparison of average serum creatinine over 12 months of combined chelation therapy compared with baseline serum creatinine.
Time frame: 12 months
| Milestone | Deferasirox (Exjade) and Deferoxamine (DFO) |
|---|---|
| Started | 22 |
| Completed | 18 |
| Not completed | 4 |
Change in liver iron concentration from baseline to 12 months with the use of combined chelation therapy. The change was calculated as the liver iron concentration at 12 months minus the value at baseline.
| mg/g | Deferasirox (Exjade) and Desferal (DFO) |
|---|---|
| Efficacy of Combined Treatment With Deferasirox and Deferoxamine Over 12 Months | -6.2 (-23.1 to 2.7) |
Comparison of average serum creatinine over 12 months of combined chelation therapy compared with baseline serum creatinine.
| mg/dl | Deferasirox (Exjade) and Desferal (DFO) |
|---|---|
| Change in Serum Creatinine During 12 Months Combined Chelation Therapy | 0.09 (0.05 to 0.12) |
Collected over 1 year. Non-serious events are listed at a 2.5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Deferasirox (Exjade) and Deferoxamine (DFO) | 1/22 (4.5%) | 2/22 (9.1%) | 1/22 (4.5%) |
| Event | Deferasirox (Exjade) and Deferoxamine (DFO) |
|---|---|
| CholelithiasisHepatobiliary disorders | 1/22 |
| Central venous line infectionInfections and infestations | 1/22 |
| DeathCardiac disorders | 1/22 |
| Event | Deferasirox (Exjade) and Deferoxamine (DFO) |
|---|---|
| Transfusion reactionBlood and lymphatic system disorders | 1/22 |
| Age, Categorical(Participants) | Deferasirox (Exjade) and Deferoxamine (DFO) |
|---|---|
| Age, Categorical — <=18 years | 6 |
| Age, Categorical — Between 18 and 65 years | 16 |
| Age, Categorical — >=65 years | 0 |
| Sex/Gender, Customized(Participants) | Deferasirox (Exjade) and Deferoxamine (DFO) |
|---|---|
| Sex/Gender Not Collected | 22 |
This study is completed, as verified in Jul 2021. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Deferasirox