Intensive Combined Chelation Therapy for Iron-Induced Cardiac Disease in Patients With Thalassemia Major

Cardiomyopathy Clinical Trial

— DFODFPTM  

Official title:

Increased Survival and Reversion of Iron-Induced Cardiac Disease in Patients With Thalassemia Major Receiving Intensive Combined Chelation Therapy

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00800761
• Other study ID #: DFO-DFP in TM
• Secondary ID: DFOplusDFPLAI
• Status: Completed
• Phase: Phase 4
• First received: December 1, 2008
• Last updated: December 1, 2008
• Start date: December 2001
• Est. completion date: June 2006

Study information

• Verified date: December 2001
• Source: Ospedale Microcitemico
• Contact: n/a
• Is FDA regulated: No
• Health authority: Italy: Ethics Committee
• Study type: Interventional

Clinical Trial Summary

Myocardial iron overload is the leading cause of death in patients with beta-thalassemia major (TM). Therapy with deferoxamine (DFO) combined with deferiprone (DFP) reduces myocardial iron and improves cardiac function. However, the prognosis for TM patients with established cardiac disease switched from DFO monotherapy to combined DFP/DFO chelation is unknown. Twenty-eight TM patients with cardiac disease were enrolled in a prospective study lasting 42±6 months. Fifteen (9 high-ferritin and 6 low-ferritin) were placed on DFP/DFO (DFP, 75 mg/kg t.i.d.; DFO, 40-50 mg/kg over 8-12 h at night 5-7 d/wk), while 13 (5 high- and 8 low-ferritin) received DFO alone. No cardiac events were observed among high-ferritin patients on combination therapy, whereas 4 cardiac events (p=0.0049), including three deaths, occurred in high-ferritin patients on DFO monotherapy. These findings demonstrate that in TM patients with well-established cardiac disease combined iron-chelation therapy with DFP/DFO is superior to DFO monotherapy.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 0
• Est. completion date: June 2006
• Est. primary completion date: June 2006
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

Cardiomyopathy secondary to iron overload

Exclusion Criteria:

Heart failure

Study Design

Allocation: Non-Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Beta-Thalassemia
• Cardiomyopathies
• Cardiomyopathy
• Heart Diseases
• Iron Overload
• Thalassemia

Intervention

Drug:
• Deferoxamine and Deferiprone
comparison of two arms: the first one treated with deferoxamine subcutaneous vials,40 mg/kg,12 hours/die plus deferiprone tablets 75 mg/kg three times/die versus the second one treated with deferoxamine subcutaneous vials,40 mg/kg,12 hours/die
• Deferoxamine
deferoxamine vials,40 mg/kg,12 hours/die

Locations

Country	Name	City	State
Italy	Adult Talassemic Center, Ospedale Microcitemico	Cagliari	Sardinia

Sponsors (2)

Lead Sponsor	Collaborator
Ospedale Microcitemico	Azienda Sanitaria Locale di Cagliari

Country where clinical trial is conducted

Italy, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Our primary objective: to assess the prevalence of cardiovascular deaths and hospitalisations for cardiovascular disease in the 2 treatment groups		42 months	Yes
Secondary	monitor the left ventricular ejection fraction (LVEF) and serum ferritin levels for evidence of improvement.		42 months	Yes