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

NCT00733811 | Completed Phase 4 DMC

• 1 other identifier: AOVCervello
• Study Type: interventional
• Target: 213
• Locations: 1 country
• Sites: 1

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.

Conditions

Beta-Thalassemia; Thalassemia Major

Keywords

thalassemia major; chelation treatment; secondary hemochromatosis

Outcome Measures

Primary Outcomes (1)

• difference between multiple observations of the serum ferritin concentrations

  five-year treatment

Secondary Outcomes (1)

• 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

Study Arms (2)

1

EXPERIMENTAL

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

Drug: Deferiprone (DFP) and Deferoxamine (DFO)

2

ACTIVE COMPARATOR

Deferiprone alone at 75 mg/kg divided into three oral daily doses

Drug: Deferiprone (DFP)

Interventions

Deferiprone (DFP) and Deferoxamine (DFO) DRUG

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

Also known as: DFP (Apotex, Canada), DFO (Biofutura Pharma S.p.A.,Italy)

1

Deferiprone (DFP) DRUG

DFP alone at 75 mg/kg divided into three oral daily doses

Also known as: DFP (Apotex, Canada)

2

Eligibility Criteria

• Age: 13 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

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

You may not qualify if:

• 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

Sponsors & Collaborators

• Azienda Ospedaliera V. Cervello: lead

Study Sites (1)

Ao V. Cervello

Palermo, Italy

Location

Related Publications (1)

• 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.

  PMID: 19236376 DERIVED

MeSH Terms

Conditions

beta-Thalassemia

Interventions

Deferiprone; Isoflurophate; Deferoxamine

Condition Hierarchy (Ancestors)

Thalassemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Pyridones; Pyridines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Organofluorophosphonates; Organophosphonates; Organophosphorus Compounds; Organic Chemicals; Hydroxamic Acids; Hydroxylamines; Amines; Hydroxy Acids; Carboxylic Acids

Study Officials

• AURELIO MAGGIO, M.D.

  Azienda Ospedaliera V. Cervello

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: August 11, 2008
• First Posted: August 13, 2008
• Study Start: September 1, 2000
• Primary Completion: January 1, 2008
• Study Completion: January 1, 2008
• Last Updated: August 13, 2008

Record last verified: 2008-07

Locations

IT (1)