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

History of Changes

Tracking Information

First Received Date ^ICMJE

August 11, 2008

Last Updated Date

August 12, 2008

Start Date ^ICMJE

September 2000

Primary Completion Date

January 2008 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures ^ICMJE

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

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

Enrollment ^ICMJE

213

Completion Date

January 2008

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

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

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