NCT00115349

Combination Therapy Compared With Single-Drug Therapy in Patients With Cardiac Diseases

Thalassemia Clinical Research Network - Cardiac L1/DFO Trial

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 20 (actual)
• Sponsor: Carelon Research · Academic / Other
• Sex: All
• Age: 18 Years – 100 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to determine whether left ventricular function improves more rapidly with deferoxamine (DFO) and deferiprone (L1) combination therapy than with DFO monotherapy in patients with thalassemia and decreased ejection fractions. Secondary aims include evaluating changes in myocardial iron burden using T2\* and estimating the relative incidence and severity of chelator-induced toxicity.

Detailed description

DESIGN NARRATIVE: Participants will be randomized to 1 year of treatment with L1/DFO combination therapy or DFO monotherapy. At baseline, 6 months, and 1 year on therapy, cardiac function will be assessed by MRI measurement of left ventricular ejection fraction (LVEF), T2\*, Holter monitoring, and electrocardiography. Additional monitoring for safety includes weekly blood testing, monthly visits, and periodic eye and ear exams.

Conditions

• Cardiovascular Diseases
• Heart Diseases
• Beta-Thalassemia

Interventions

Type	Name	Description
DRUG	Deferoxamine	Deferoxamine will be given daily for 12-24h/day 7 days a week either subcutaneous or intravenous at up to 50-60 mg/kg/day.
DRUG	Deferiprone (L1)	The dose of L1, 75mg/kg in three divided oral doses, is the maximum dose at which toxicity has been tested in prospective trials

Timeline

• Start date: 2005-06-01
• Primary completion: 2008-07-01
• Completion: 2009-04-01
• First posted: 2005-06-22
• Last updated: 2018-03-01
• Results posted: 2014-01-14

Locations

6 sites across 1 country: United States