Beta-ThalassemiaPhase 4SEQDFPDFONCT00733811
Efficacy Study of the Use of Sequential DFP-DFO Versus DFP
A Phase 4 study testing Deferiprone (DFP) and Deferoxamine (DFO) and Deferiprone (DFP) in people with Beta-Thalassemia and Thalassemia Major, run by Azienda Ospedaliera V. Cervello at 1 site in Italy.
Trial 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… Read more about this study
- Who is it for?
- Ages 13 to 60 years
- Conditions the study is for
- Beta-Thalassemia, Thalassemia Major
- What is being tested?
- Deferiprone (DFP) and Deferoxamine (DFO) and Deferiprone (DFP)
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 213 participants
- Where
- 1 siteItaly
- Study dates
- Sep 2000to Jan 2008
- Phase
- Phase 4
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 13 to 60 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Thalassemia major patients with serum ferritin concentration between 800 to 3,000 ng/ml and were over 13 years of age
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Phase IV Study of the Use of Sequential DFP-DFO Versus DFP in Thalassemia Major Patients
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.
Read the 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
- Conditions
- Beta-Thalassemia, Thalassemia Major
- Being tested
- Deferiprone (DFP) and Deferoxamine (DFO), Deferiprone (DFP)
- Keywords
- thalassemia major, chelation treatment, secondary hemochromatosis
- Medical subject headings
- beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Deferiprone (DFP) and Deferoxamine (DFO)Drug
Also called DFP (Apotex, Canada), DFO (Biofutura Pharma S.p.A.,Italy)
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
Deferiprone (DFP)Drug
Also called DFP (Apotex, Canada)
DFP alone at 75 mg/kg divided into three oral daily doses
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: Drug: Deferiprone (DFP) and Deferoxamine (DFO) |
| 2 | Active comparator | Deferiprone alone at 75 mg/kg divided into three oral daily doses Receives: Drug: Deferiprone (DFP) |
What is being measured
The primary outcome is the main question the study is designed to answer.
difference between multiple observations of the serum ferritin concentrations
Measured: 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.
Measured: five years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Aug 2008 (estimated)
- Study startSep 2000
- Primary completionJan 2008
- Study completionJan 2008
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Beta-ThalassemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 213 participants (actual)
Sponsor and investigators
- Lead sponsor
- Azienda Ospedaliera V. Cervello
- Sponsor type
- University, hospital or other
- Investigators
- AURELIO MAGGIO, M.D., Study chair, Azienda Ospedaliera V. Cervello
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00733811
- Sponsor's protocol ID
- AOVCervello
- Acronym
- SEQDFPDFO
- Status last verified
- Jul 2008
- First submitted
- 11 Aug 2008
- Last updated
- 13 Aug 2008
- Results posted
- No
- Data monitoring committee
- Yes
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.
PubMed 19236376
Study at a glance
From the registry record, last updated by the sponsor on 13 Aug 2008. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase IV Study of the Use of Sequential DFP-DFO Versus DFP in Thalassemia Major Patients
- ClinicalTrials.gov ID
- NCT00733811
- Acronym / sponsor ID
- SEQDFPDFO / AOVCervello
- Conditions
- Beta-Thalassemia, Thalassemia Major
- Keywords
- thalassemia major, chelation treatment, secondary hemochromatosis
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 213 participants (actual)
- Lead sponsor
- Azienda Ospedaliera V. CervelloUniversity, hospital or other
- Study start
- Sep 2000
- Primary completion
- Jan 2008 (actual)
- Study completion
- Jan 2008 (actual)
- First posted
- 13 Aug 2008 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 1 | Experimental | Drug: Deferiprone (DFP) and Deferoxamine (DFO)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 |
| 2 | Active comparator | Drug: Deferiprone (DFP)Deferiprone alone at 75 mg/kg divided into three oral daily doses |
Who may qualify
- Ages
- 13 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (1 listed)
- Thalassemia major patients with serum ferritin concentration between 800 to 3,000 ng/ml and were over 13 years of age
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Deferiprone (DFP) and Deferoxamine (DFO)Also: DFP (Apotex, Canada), DFO (Biofutura Pharma S.p.A.,Italy)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 | Drug | 1 |
| Deferiprone (DFP)Also: DFP (Apotex, Canada)DFP alone at 75 mg/kg divided into three oral daily doses | Drug | 2 |
Primary outcome
difference between multiple observations of the serum ferritin concentrations
Time frame: five-year treatment
1 secondary outcome
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Aug 2008.
- Derived from this study: PubMed 19236376Maggio 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
AURELIO MAGGIO, M.D. study chair
Azienda Ospedaliera V. Cervello
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Deferiprone (DFP) and Deferoxamine (DFO) and Deferiprone (DFP) in people with Beta-Thalassemia and Thalassemia Major, run by Azienda Ospedaliera V. Cervello at 1 site in Italy.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 13 Aug 2008. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
difference between multiple observations of the serum ferritin concentrations (time frame: five-year treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00733811, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Aug 2008.