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

This study has been completed.
Sponsor:
Collaborator:
ASL Cagliari
Information provided by:
Ospedale Microcitemico
ClinicalTrials.gov Identifier:
NCT00800761
First received: December 1, 2008
Last updated: NA
Last verified: December 2001
History: No changes posted
  Purpose

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.


Condition Intervention Phase
Iron Overload
Cardiomyopathy
 Drug: Deferoxamine and Deferiprone
Drug: Deferoxamine
 Phase 4

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Increased Survival and Reversion of Iron-Induced Cardiac Disease in Patients With Thalassemia Major Receiving Intensive Combined Chelation Therapy

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Ospedale Microcitemico:

Primary Outcome Measures:
  • Our primary objective: to assess the prevalence of cardiovascular deaths and hospitalisations for cardiovascular disease in the 2 treatment groups [ Time Frame: 42 months ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • monitor the left ventricular ejection fraction (LVEF) and serum ferritin levels for evidence of improvement. [ Time Frame: 42 months ] [ Designated as safety issue: Yes ]

Study Start Date: December 2001
Study Completion Date: June 2006
Primary Completion Date: June 2006 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: Deferoxamine alone
comparison of deferoxamine subcutaneous 40mg/kg/die alone versus combined therapy deferoxamine-deferiprone
 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
Drug: Deferoxamine
deferoxamine vials,40 mg/kg,12 hours/die
Active Comparator: Deferoxamine plus 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
 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

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

Cardiomyopathy secondary to iron overload

Exclusion Criteria:

Heart failure

  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00800761

Locations
Italy
Adult Talassemic Center, Ospedale Microcitemico
Cagliari, Sardinia, Italy, 09121
Sponsors and Collaborators
Ospedale Microcitemico
ASL Cagliari
Investigators
Study Director: Maria E Lai, MD Department of Internal Medicine, University of Cagliari-Italy
  More Information

No publications provided

Responsible Party: Maria Eliana Lai, Prof, MD, Adult Thalassemic Center, Director, University
ClinicalTrials.gov Identifier: NCT00800761     History of Changes
Other Study ID Numbers: DFO-DFP in TM, DFOplusDFPLAI
Study First Received: December 1, 2008
Last Updated: December 1, 2008
Health Authority: Italy: Ethics Committee

Additional relevant MeSH terms:
Beta-Thalassemia
Heart Diseases
Thalassemia
Iron Overload
Cardiomyopathies
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
 Cardiovascular Diseases
Iron Metabolism Disorders
Metabolic Diseases
Deferoxamine
Deferiprone
Siderophores
Iron Chelating Agents
Chelating Agents
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions

ClinicalTrials.gov processed this record on May 16, 2013