Efficacy Study of the Use of Sequential DFP-DFO Versus DFP

Beta-Thalassemia Clinical Trial

— SEQDFPDFO  

Official title:

Phase IV Study of the Use of Sequential DFP-DFO Versus DFP in Thalassemia Major Patients

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00733811
• Other study ID #: AOVCervello
• Status: Completed
• Phase: Phase 4
• First received: August 11, 2008
• Last updated: August 12, 2008
• Start date: September 2000
• Est. completion date: January 2008

Study information

• Verified date: July 2008
• Source: Azienda Ospedaliera V. Cervello
• Contact: n/a
• Is FDA regulated: No
• Health authority: Italy: Ministry of Health
• Study type: Interventional

Clinical Trial Summary

Changes in chelation treatment and transfusion practices, during the past two decades, have dramatically improved the prognosis of thalassemia major patients.Deferiprone (DFP) has been compared with deferoxamine (DFO), using different schedules of treatment, in the majority of the 13 clinical trials published between 1990 and 2008.No statistically significant difference was shown between these two interventions during, at most, 18 months of treatment.Three randomised trials that compared sequential DFP-DFO treatment versus DFO alone reported controversial results but this could be due to small sample sizes and short treatment duration. In fact, no trial with treatment duration longer than 18 months15, which reported on mortality, adverse events, serum ferritin concentrations, as well as costs has so far been published.

This long-term sequential DFP-DFO treatment versus DFP alone treatment trial was conducted to assess the impacts of these chelation treatments on serum ferritin concentrations, mortality, adverse events, and costs in thalassemia major patients.

Description:

The trial was designed as a multicentre randomised open-label trial with blinded data management and data analyses, to assess whether either treatment was superior to the other. The trial was performed on behalf of the Italian Society for the study of Thalassemia and Haemoglobinopathies (SoSTE). The investigators initiated, carried out, and controlled the trial, which was conducted without influence of the non-commercial sponsor.16

Recruitment information / eligibility

• Status: Completed
• Enrollment: 213
• Est. completion date: January 2008
• Est. primary completion date: January 2008
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 13 Years to 60 Years

Eligibility

Inclusion Criteria:

• Thalassemia major patients with serum ferritin concentration between 800 to 3,000 ng/ml and were over 13 years of age

Exclusion Criteria:

• Known intolerance to one of the trial treatments

• Platelet count < 100,000/mm3 or or leukocyte count < 3,000/mm3

• Severe liver damage indicated by ascites

• Heart failure

Study Design

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

Related Conditions & MeSH terms

• Beta-Thalassemia
• Thalassemia
• Thalassemia Major

Intervention

Drug:
• Deferiprone (DFP) and Deferoxamine (DFO)
Sequential treatment including DFP at 75 mg/kg for four days per week and DFO by subcutaneous infusions (8-12h) at 50mg/kg/day for the remaining three days per week
• Deferiprone (DFP)
DFP alone at 75 mg/kg divided into three oral daily doses

Locations

Country	Name	City	State
Italy	Ao V. Cervello	Palermo

Sponsors (1)

Lead Sponsor	Collaborator
Azienda Ospedaliera V. Cervello

Country where clinical trial is conducted

Italy, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	difference between multiple observations of the serum ferritin concentrations		five-year treatment	Yes
Secondary	the difference between the T2* signal at magnetic resonance imaging (MRI) of heart and liver at T0 and T1 time (see below); survival analysis; adverse events; treatment failures; and costs.		five years	Yes