Efficacy Study of the Use of Sequential DFP-DFO Versus DFP (SEQDFPDFO)

Tracking Information
First Submitted Date ICMJE	August 11, 2008
First Posted Date ICMJE	August 13, 2008
Last Update Posted Date	August 13, 2008
Study Start Date ICMJE	September 2000
Actual Primary Completion Date	January 2008 (Final data collection date for primary outcome measure)
Current Primary Outcome Measures ICMJE; (submitted: August 12, 2008)	difference between multiple observations of the serum ferritin concentrations [ Time Frame: five-year treatment ]
Original Primary Outcome Measures ICMJE	Same as current
Change History	No Changes Posted
Current Secondary Outcome Measures ICMJE; (submitted: August 12, 2008)	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. [ Time Frame: five years ]
Original Secondary Outcome Measures ICMJE	Same as current
Current Other Outcome Measures ICMJE	Not Provided
Original Other Outcome Measures ICMJE	Not Provided
Descriptive Information
Brief Title ICMJE	Efficacy Study of the Use of Sequential DFP-DFO Versus DFP
Official Title ICMJE	Phase IV Study of the Use of Sequential DFP-DFO Versus DFP in Thalassemia Major Patients
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
Study Type ICMJE	Interventional
Study Phase	Phase 4
Study Design ICMJE	Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: None (Open Label); Primary Purpose: Treatment
Condition ICMJE	Beta-Thalassemia; Thalassemia Major
Intervention ICMJE	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 Other Names: DFP (Apotex, Canada) DFO (Biofutura Pharma S.p.A.,Italy); Drug: Deferiprone (DFP) DFP alone at 75 mg/kg divided into three oral daily doses Other Name: DFP (Apotex, Canada)
Study Arms	Experimental: 1 Sequential treatment including DFP at 75 mg/kg, divided into three oral daily doses, for four days per week and DFO by subcutaneous infusions (8-12h) at 50 mg/kg/day for the remaining three days per week Intervention: Drug: Deferiprone (DFP) and Deferoxamine (DFO); Active Comparator: 2 Deferiprone alone at 75 mg/kg divided into three oral daily doses Intervention: Drug: Deferiprone (DFP)
Publications *	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.
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status ICMJE	Completed
Actual Enrollment ICMJE; (submitted: August 12, 2008)	213
Original Actual Enrollment ICMJE	Same as current
Actual Study Completion Date	January 2008
Actual Primary Completion Date	January 2008 (Final data collection date for primary outcome measure)
Eligibility Criteria ICMJE	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
Sex/Gender	Sexes Eligible for Study: All
Ages	13 Years to 60 Years (Child, Adult)
Accepts Healthy Volunteers	No
Contacts ICMJE	Contact information is only displayed when the study is recruiting subjects
Listed Location Countries ICMJE	Italy
Removed Location Countries
Administrative Information
NCT Number ICMJE	NCT00733811
Other Study ID Numbers ICMJE	AOVCervello
Has Data Monitoring Committee	Yes
U.S. FDA-regulated Product	Not Provided
IPD Sharing Statement	Not Provided
Responsible Party	Aurelio Maggio, AO V Cervello
Study Sponsor ICMJE	Azienda Ospedaliera V. Cervello
Collaborators ICMJE	Not Provided
Investigators ICMJE	Study Chair: AURELIO MAGGIO, M.D. Azienda Ospedaliera V. Cervello
PRS Account	Azienda Ospedaliera V. Cervello
Verification Date	July 2008
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP