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

• ClinicalTrials.gov Identifier: NCT00733811
• Recruitment Status: Completed
• First Posted: August 13, 2008
• Last Update Posted: August 13, 2008
• Sponsor: Azienda Ospedaliera V. Cervello
• Information provided by: Azienda Ospedaliera V. Cervello

Tracking Information

• First Submitted Date: August 11, 2008
• First Posted Date: August 13, 2008
• Last Update Posted Date: August 13, 2008
• Study Start Date: September 2000
• Actual Primary Completion Date: January 2008 (Final data collection date for primary outcome measure)
• Current Primary Outcome Measures (submitted: August 12, 2008): difference between multiple observations of the serum ferritin concentrations [ Time Frame: five-year treatment ]
• Original Primary Outcome Measures: Same as current
• Change History: No Changes Posted
• Current Secondary Outcome Measures (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: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: 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

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: Interventional
• Study Phase: Phase 4
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: None (Open Label); Primary Purpose: Treatment

Condition

• Beta-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
  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.

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: August 12, 2008): 213
• Original Actual Enrollment: 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

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: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Italy

Administrative Information

• NCT Number: NCT00733811
• Other Study ID Numbers: 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: Azienda Ospedaliera V. Cervello
• Collaborators: Not Provided

Investigators

• Study Chair: AURELIO MAGGIO, M.D.; Azienda Ospedaliera V. Cervello

• PRS Account: Azienda Ospedaliera V. Cervello
• Verification Date: July 2008