Iron OverloadPhase 4DFODFPTMNCT00800761
Intensive Combined Chelation Therapy for Iron-Induced Cardiac Disease in Patients With Thalassemia Major
A Phase 4 study testing Deferoxamine and Deferiprone and Deferoxamine in people with Iron Overload and Cardiomyopathy, run by Ospedale Microcitemico at 1 site in Italy.
Trial summary
Myocardial iron overload is the leading cause of death in patients with beta-thalassemia major (TM). Therapy with deferoxamine (DFO) combined with deferiprone (DFP) reduces myocardial iron and improves cardiac function. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Iron Overload, Cardiomyopathy
- What is being tested?
- Deferoxamine and Deferiprone and Deferoxamine
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- Not given
- Where
- 1 siteItaly
- Study dates
- Dec 2001to Jun 2006
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Cardiomyopathy secondary to iron overload
Who cannot join
- Heart failure
Trial locations and contacts
1 site in 1 country.
Italy1 site
Adult Talassemic Center, Ospedale Microcitemico
Cagliari, Sardinia, 09121
About this study
Official title: Increased Survival and Reversion of Iron-Induced Cardiac Disease in Patients With Thalassemia Major Receiving Intensive Combined Chelation Therapy
Myocardial iron overload is the leading cause of death in patients with beta-thalassemia major (TM). Therapy with deferoxamine (DFO) combined with deferiprone (DFP) reduces myocardial iron and improves cardiac function. However, the prognosis for TM patients with established cardiac disease switched from DFO monotherapy to combined DFP/DFO chelation is unknown. Twenty-eight TM patients with cardiac disease were enrolled in a prospective study lasting 42±6 months. Fifteen (9 high-ferritin and 6 low-ferritin) were placed on DFP/DFO (DFP, 75 mg/kg t.i.d.; DFO, 40-50 mg/kg over 8-12 h at night 5-7 d/wk), while 13 (5 high- and 8 low-ferritin) received DFO alone. No cardiac events were observed among high-ferritin patients on combination therapy, whereas 4 cardiac events (p=0.0049), including three deaths, occurred in high-ferritin patients on DFO monotherapy. These findings demonstrate that in TM patients with well-established cardiac disease combined iron-chelation therapy with DFP/DFO is superior to DFO monotherapy.
- Conditions
- Iron Overload, Cardiomyopathy
- Being tested
- Deferoxamine and Deferiprone, Deferoxamine
- Medical subject headings
- Iron Overload, Cardiomyopathies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Deferoxamine and DeferiproneDrug
comparison of two arms: the first one treated with deferoxamine subcutaneous vials,40 mg/kg,12 hours/die plus deferiprone tablets 75 mg/kg three times/die versus the second one treated with deferoxamine subcutaneous vials,40 mg/kg,12 hours/die
DeferoxamineDrug
deferoxamine vials,40 mg/kg,12 hours/die
| Study group | Role | What they receive |
|---|---|---|
| Deferoxamine alone | Active comparator | comparison of deferoxamine subcutaneous 40mg/kg/die alone versus combined therapy deferoxamine-deferiprone Receives: Drug: Deferoxamine and Deferiprone; Drug: Deferoxamine |
| Deferoxamine plus Deferiprone | Active comparator | comparison of two arms: the first one treated with deferoxamine subcutaneous vials,40 mg/kg,12 hours/die plus deferiprone tablets 75 mg/kg three times/die versus the second one treated with deferoxamine subcutaneous vials,40 mg/kg,12 hours/die Receives: Drug: Deferoxamine and Deferiprone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Our primary objective: to assess the prevalence of cardiovascular deaths and hospitalisations for cardiovascular disease in the 2 treatment groups
Measured: 42 months
Secondary outcomes (1)
monitor the left ventricular ejection fraction (LVEF) and serum ferritin levels for evidence of improvement.
Measured: 42 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Dec 2008 (estimated)
- Study startDec 2001
- Primary completionJun 2006
- Study completionJun 2006
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 Iron OverloadStudies 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
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
Sponsor and investigators
- Lead sponsor
- Ospedale Microcitemico
- Sponsor type
- University, hospital or other
- Collaborators
- Azienda Sanitaria Locale di Cagliari
- Investigators
- Maria E Lai, MD, Study director, Department of Internal Medicine, University of Cagliari-Italy
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00800761
- Sponsor's protocol ID
- DFO-DFP in TM
- Other IDs
- DFOplusDFPLAI
- Acronym
- DFODFPTM
- Status last verified
- Dec 2001
- First submitted
- 1 Dec 2008
- Last updated
- 2 Dec 2008
- Results posted
- No
- Data monitoring committee
- No
Publications
Padhani ZA, Gangwani MK, Sadaf A, Hasan B, Colan S, Alvi N, Das JK. Calcium channel blockers for preventing cardiomyopathy due to iron overload in people with transfusion-dependent beta thalassaemia. Cochrane Database Syst Rev. 2023 Nov 17;11(11):CD011626. doi: 10.1002/14651858.CD011626.pub3.
PubMed 37975597
Study at a glance
From the registry record, last updated by the sponsor on 2 Dec 2008. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Increased Survival and Reversion of Iron-Induced Cardiac Disease in Patients With Thalassemia Major Receiving Intensive Combined Chelation Therapy
- ClinicalTrials.gov ID
- NCT00800761
- Acronym / sponsor ID
- DFODFPTM / DFO-DFP in TM
- Conditions
- Iron Overload, Cardiomyopathy
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Lead sponsor
- Ospedale MicrocitemicoUniversity, hospital or other
- Collaborators
- Azienda Sanitaria Locale di Cagliari
- Study start
- Dec 2001
- Primary completion
- Jun 2006 (actual)
- Study completion
- Jun 2006 (actual)
- First posted
- 2 Dec 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.
Myocardial iron overload is the leading cause of death in patients with beta-thalassemia major (TM). Therapy with deferoxamine (DFO) combined with deferiprone (DFP) reduces myocardial iron and improves cardiac function. However, the prognosis for TM patients with established cardiac disease switched from DFO monotherapy to combined DFP/DFO chelation is unknown. Twenty-eight TM patients with cardiac disease were enrolled in a prospective study lasting 42±6 months. Fifteen (9 high-ferritin and 6 low-ferritin) were placed on DFP/DFO (DFP, 75 mg/kg t.i.d.; DFO, 40-50 mg/kg over 8-12 h at night 5-7 d/wk), while 13 (5 high- and 8 low-ferritin) received DFO alone. No cardiac events were observed among high-ferritin patients on combination therapy, whereas 4 cardiac events (p=0.0049), including three deaths, occurred in high-ferritin patients on DFO monotherapy. These findings demonstrate that in TM patients with well-established cardiac disease combined iron-chelation therapy with DFP/DFO is superior to DFO monotherapy.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Deferoxamine alone | Active comparator | Drug: Deferoxamine and Deferiprone, Drug: Deferoxaminecomparison of deferoxamine subcutaneous 40mg/kg/die alone versus combined therapy deferoxamine-deferiprone |
| Deferoxamine plus Deferiprone | Active comparator | Drug: Deferoxamine and Deferipronecomparison of two arms: the first one treated with deferoxamine subcutaneous vials,40 mg/kg,12 hours/die plus deferiprone tablets 75 mg/kg three times/die versus the second one treated with deferoxamine subcutaneous vials,40 mg/kg,12 hours/die |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (1 listed)
- Cardiomyopathy secondary to iron overload
Key exclusion criteria (1 listed)
- 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 |
|---|---|---|
| Deferoxamine and Deferipronecomparison of two arms: the first one treated with deferoxamine subcutaneous vials,40 mg/kg,12 hours/die plus deferiprone tablets 75 mg/kg three times/die versus the second one treated with deferoxamine subcutaneous vials,40 mg/kg,12 hours/die | Drug | Deferoxamine alone, Deferoxamine plus Deferiprone |
| Deferoxaminedeferoxamine vials,40 mg/kg,12 hours/die | Drug | Deferoxamine alone |
Primary outcome
Our primary objective: to assess the prevalence of cardiovascular deaths and hospitalisations for cardiovascular disease in the 2 treatment groups
Time frame: 42 months
1 secondary outcome
monitor the left ventricular ejection fraction (LVEF) and serum ferritin levels for evidence of improvement.
Time frame: 42 months
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 2 Dec 2008.
- Derived from this study: PubMed 37975597Padhani ZA, Gangwani MK, Sadaf A, Hasan B, Colan S, Alvi N, Das JK. Calcium channel blockers for preventing cardiomyopathy due to iron overload in people with transfusion-dependent beta thalassaemia. Cochrane Database Syst Rev. 2023 Nov 17;11(11):CD011626. doi: 10.1002/14651858.CD011626.pub3.
Related trials for Iron Overload
Active, not recruitingPhase 2
Treatment of Hemochromatosis
Hemochromatosis
Active, not recruitingObservational study
Ethnic Differences in Iron Absorption (FeGenes)
Anemia, Iron Deficiency, Iron Overload
Active, not recruitingObservational study
Heme and Non-heme Iron Intakes, Gut Microbiota, and Influence on Host Iron Absorption
Anemia, Iron Deficiency, Iron Overload
Active, not recruitingPhase 2
Efficacy and Safety of Vamifeport in Adult Participants With Homeostatic Iron Regulator Gene (HFE)-Related Hereditary Hemochromatosis
Homeostatic Iron Regulator Gene-related Hereditary Hemochromatosis
Not yet recruitingObservational study
Native T1 Mapping and ECV in Chronic Hemolytic Anemia
Hemolytic Anemia, Thalassemia
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.
Maria E Lai, MD study director
Department of Internal Medicine, University of Cagliari-Italy
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 Deferoxamine and Deferiprone and Deferoxamine in people with Iron Overload and Cardiomyopathy, run by Ospedale Microcitemico 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 1 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 2 Dec 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?
Our primary objective: to assess the prevalence of cardiovascular deaths and hospitalisations for cardiovascular disease in the 2 treatment groups (time frame: 42 months). 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 NCT00800761, 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 2 Dec 2008.