Clinical trial NCT00733811: Efficacy Study of the Use of Sequential DFP-DFO Versus DFP
Official title: Phase IV Study of the Use of Sequential DFP-DFO Versus DFP in Thalassemia Major Patients
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Beta-Thalassemia
Thalassemia Major
Keywords: thalassemia major
chelation treatment
secondary hemochromatosis
Lead sponsor: Azienda Ospedaliera V. Cervello
Sponsor class: OTHER
First posted: 2008-08-13
Enrollment: 213
Sex: ALL
Minimum age (years): 13
Maximum age (years): 60
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief 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.

Detailed 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

Eligibility criteria: 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

Primary outcomes:
measure: difference between multiple observations of the serum ferritin concentrations
timeFrame: five-year treatment
Secondary outcomes:
measure: 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.
timeFrame: five years
Other outcomes:
Locations:
city: Palermo
country: Italy
facility: Ao V. Cervello
Study officials:
affiliation: Azienda Ospedaliera V. Cervello
name: AURELIO MAGGIO, M.D.
role: STUDY_CHAIR
References:
citation: Maggio A, Vitrano A, Capra M, Cuccia L, Gagliardotto F, Filosa A, Romeo MA, Magnano C, Caruso V, Argento C, Gerardi C, Campisi S, Violi P, Malizia R, Cianciulli P, Rizzo M, D'Ascola DG, Quota A, Prossomariti L, Fidone C, Rigano P, Pepe A, D'Amico G, Morabito A, Gluud C. Long-term sequential deferiprone-deferoxamine versus deferiprone alone for thalassaemia major patients: a randomized clinical trial. Br J Haematol. 2009 Apr;145(2):245-54. doi: 10.1111/j.1365-2141.2009.07609.x. Epub 2009 Feb 19.