Trial record 6 of 95 for:    "beta thalassemia"

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

This study has been completed.
Sponsor:
Information provided by:
Azienda Ospedaliera V. Cervello
ClinicalTrials.gov Identifier:
NCT00733811
First received: August 11, 2008
Last updated: August 12, 2008
Last verified: July 2008
  Purpose

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.


Condition Intervention Phase
Beta-Thalassemia
Thalassemia Major
Drug: Deferiprone (DFP) and Deferoxamine (DFO)
Drug: Deferiprone (DFP)
Phase 4

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Phase IV Study of the Use of Sequential DFP-DFO Versus DFP in Thalassemia Major Patients

Resource links provided by NLM:


Further study details as provided by Azienda Ospedaliera V. Cervello:

Primary Outcome Measures:
  • difference between multiple observations of the serum ferritin concentrations [ Time Frame: five-year treatment ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • 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 ] [ Designated as safety issue: Yes ]

Enrollment: 213
Study Start Date: September 2000
Study Completion Date: January 2008
Primary Completion Date: January 2008 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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
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)
Active Comparator: 2
Deferiprone alone at 75 mg/kg divided into three oral daily doses
Drug: Deferiprone (DFP)
DFP alone at 75 mg/kg divided into three oral daily doses
Other Name: DFP (Apotex, Canada)

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

Ages Eligible for Study:   13 Years to 60 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00733811

Locations
Italy
Ao V. Cervello
Palermo, Italy
Sponsors and Collaborators
Azienda Ospedaliera V. Cervello
Investigators
Study Chair: AURELIO MAGGIO, M.D. Azienda Ospedaliera V. Cervello
  More Information

No publications provided by Azienda Ospedaliera V. Cervello

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Aurelio Maggio, AO V Cervello
ClinicalTrials.gov Identifier: NCT00733811     History of Changes
Other Study ID Numbers: AOVCervello
Study First Received: August 11, 2008
Last Updated: August 12, 2008
Health Authority: Italy: Ministry of Health

Keywords provided by Azienda Ospedaliera V. Cervello:
thalassemia major
chelation treatment
secondary hemochromatosis

Additional relevant MeSH terms:
Beta-Thalassemia
Thalassemia
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
Deferoxamine
Deferiprone
Isoflurophate
Siderophores
Iron Chelating Agents
Chelating Agents
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Protease Inhibitors
Enzyme Inhibitors
Cholinesterase Inhibitors
Cholinergic Agents
Neurotransmitter Agents
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on April 15, 2014