Beta-ThalassemiaPhase 4NCT00103753
Combined Chelation Treatment With Deferiprone and Deferoxamine in Thalassemia Major
A Phase 4 study testing deferiprone in people with Beta-Thalassemia, run by Royal Brompton & Harefield NHS Foundation Trust at 1 site in Italy.
Trial summary
Thalassemia major is a genetic disorder affecting hemoglobin synthesis, rendering individuals dependent upon lifelong blood transfusions. Consequently, iron overload occurs and patients have shortened life expectancy with the most common cause of death being heart failure. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Beta-Thalassemia
- What is being tested?
- deferiprone
- How many people
- 65 participants
- Where
- 1 siteItaly
- Study dates
- May 2004to Jun 2005
- 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
- Beta thalassemia major
- Maintaining pre-transfusion hemoglobin of 9 g/dL
- Myocardial T2* between 8 and 20 ms
- Ability to give informed consent
- Male or female
- Age >18 years
- Any ejection fraction
- Confirmation of effective contraception throughout the trial (both men and women)
Who cannot join
- Implant incompatible with MR (magnetic resonance), such as pacemaker, claustrophobia, or other condition making CMR impossible or inadvisable
- Neutropenia within 12 months (ANC <1.5 x10^9/L), unless normal at screening
- Thrombocytopenia within 12 months (<50 x10^9/L), unless normal at screening
- Liver enzymes > 3 times upper limit of normal
- Patients who have previously received deferiprone for a total of more than 6 months over the last 5 years.
- Patients with a previous reaction to deferiprone
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Randomized, Placebo Controlled, Double Blind Trial of the Effect of Combined Therapy With Deferoxamine and Deferiprone on Myocardial Iron in Thalassemia Major Using Cardiovascular Magnetic Resonance
Thalassemia major is a genetic disorder affecting hemoglobin synthesis, rendering individuals dependent upon lifelong blood transfusions. Consequently, iron overload occurs and patients have shortened life expectancy with the most common cause of death being heart failure. This trial tests whether the combination of traditional therapy (deferoxamine) with a newer drug (deferiprone) will prove more effective in removing cardiac iron than deferoxamine alone.
Read the detailed description
Thalassemia Major (TM) is a hereditary anemia resulting from a single gene defect that results in abnormal red cell production. The survival of affected individuals is dependent upon lifelong blood transfusions. Unfortunately, this causes total body iron overload, and 50% of the patients in the UK are dead by the age of 35. Approximately 70% of these deaths result from heart failure which results as a consequence of cardiac iron toxicity.
A Cardiovascular Magnetic Resonance (CMR) technique (which exploits the fact that T2* signal decay relates to tissue iron) developed at the Royal Brompton Hospital provides a non-invasive and reproducible assessment of cardiac iron. CMR therefore provides a very useful method to assess response to new treatments in this condition. Using cardiac T2* as a primary endpoint, we will investigate whether the oral chelator, deferiprone in combination with traditional treatment (deferoxamine), is superior in removing cardiac iron as compared to deferoxamine alone. This trial will provide the first randomized controlled, double-blinded, evidence for the efficacy of combination treatment in TM.
- Conditions
- Beta-Thalassemia
- Being tested
- deferiprone
- Keywords
- Randomized Controlled Trial, Deferiprone, Deferoxamine, Iron chelation, Beta Thalassemia Major
- Medical subject headings
- beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
deferiproneDrug
What is being measured
The primary outcome is the main question the study is designed to answer.
Myocardial T2*
Secondary outcomes (7)
Liver T2*
LV and RV volumes and function in systole and diastole
Brachial artery reactivity
B-type natriuretic peptide
Patient compliance
Adverse events
Success of blinding
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Feb 2005 (estimated)
- Study startMay 2004
- Study completionJun 2005
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
- Single group
- Masking
- Double blind
- Enrolment
- 65 participants
Sponsor and investigators
- Lead sponsor
- Royal Brompton & Harefield NHS Foundation Trust
- Sponsor type
- University, hospital or other
- Collaborators
- CORDA, The Heart Charity, The Cooley's Anemia Foundation,, Apotex Inc., The UK Thalassemia Society
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00103753
- Sponsor's protocol ID
- 02 065
- Status last verified
- Feb 2005
- First submitted
- 14 Feb 2005
- Last updated
- 24 Jun 2005
- Results posted
- No
Publications
Tanner MA, Galanello R, Dessi C, Smith GC, Westwood MA, Agus A, Pibiri M, Nair SV, Walker JM, Pennell DJ. Combined chelation therapy in thalassemia major for the treatment of severe myocardial siderosis with left ventricular dysfunction. J Cardiovasc Magn Reson. 2008 Feb 25;10(1):12. doi: 10.1186/1532-429X-10-12.
PubMed 18298856
Study at a glance
From the registry record, last updated by the sponsor on 24 Jun 2005. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Placebo Controlled, Double Blind Trial of the Effect of Combined Therapy With Deferoxamine and Deferiprone on Myocardial Iron in Thalassemia Major Using Cardiovascular Magnetic Resonance
- ClinicalTrials.gov ID
- NCT00103753
- Acronym / sponsor ID
- 02 065
- Conditions
- Beta-Thalassemia
- Keywords
- Randomized Controlled Trial, Deferiprone, Deferoxamine, Iron chelation, Beta Thalassemia Major
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 65 participants
- Lead sponsor
- Royal Brompton & Harefield NHS Foundation TrustUniversity, hospital or other
- Collaborators
- CORDA, The Heart Charity, The Cooley's Anemia Foundation,, Apotex Inc., The UK Thalassemia Society
- Study start
- May 2004
- Study completion
- Jun 2005
- First posted
- 15 Feb 2005 (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.
Thalassemia major is a genetic disorder affecting hemoglobin synthesis, rendering individuals dependent upon lifelong blood transfusions. Consequently, iron overload occurs and patients have shortened life expectancy with the most common cause of death being heart failure. This trial tests whether the combination of traditional therapy (deferoxamine) with a newer drug (deferiprone) will prove more effective in removing cardiac iron than deferoxamine alone.
Detailed description
Thalassemia Major (TM) is a hereditary anemia resulting from a single gene defect that results in abnormal red cell production. The survival of affected individuals is dependent upon lifelong blood transfusions. Unfortunately, this causes total body iron overload, and 50% of the patients in the UK are dead by the age of 35. Approximately 70% of these deaths result from heart failure which results as a consequence of cardiac iron toxicity.
A Cardiovascular Magnetic Resonance (CMR) technique (which exploits the fact that T2* signal decay relates to tissue iron) developed at the Royal Brompton Hospital provides a non-invasive and reproducible assessment of cardiac iron. CMR therefore provides a very useful method to assess response to new treatments in this condition. Using cardiac T2* as a primary endpoint, we will investigate whether the oral chelator, deferiprone in combination with traditional treatment (deferoxamine), is superior in removing cardiac iron as compared to deferoxamine alone. This trial will provide the first randomized controlled, double-blinded, evidence for the efficacy of combination treatment in TM.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- Double blind
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Beta thalassemia major
- Maintaining pre-transfusion hemoglobin of 9 g/dL
- Myocardial T2* between 8 and 20 ms
- Ability to give informed consent
- Male or female
Key exclusion criteria (6 listed)
- Implant incompatible with MR (magnetic resonance), such as pacemaker, claustrophobia, or other condition making CMR impossible or inadvisable
- Neutropenia within 12 months (ANC <1.5 x10^9/L), unless normal at screening
- Thrombocytopenia within 12 months (<50 x10^9/L), unless normal at screening
- Liver enzymes > 3 times upper limit of normal
- Patients who have previously received deferiprone for a total of more than 6 months over the last 5 years.
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 | Drug | – |
Primary outcome
Myocardial T2*
7 secondary outcomes
Liver T2*
LV and RV volumes and function in systole and diastole
Brachial artery reactivity
B-type natriuretic peptide
Patient compliance
Adverse events
Success of blinding
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Jun 2005.
- Derived from this study: PubMed 18298856Tanner MA, Galanello R, Dessi C, Smith GC, Westwood MA, Agus A, Pibiri M, Nair SV, Walker JM, Pennell DJ. Combined chelation therapy in thalassemia major for the treatment of severe myocardial siderosis with left ventricular dysfunction. J Cardiovasc Magn Reson. 2008 Feb 25;10(1):12. doi: 10.1186/1532-429X-10-12.
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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.
The record lists no contact. Use the official record to reach the sponsor.
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 in people with Beta-Thalassemia, run by Royal Brompton & Harefield NHS Foundation Trust 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 8 inclusion and 6 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: status not confirmed, as of the sponsor's last update on 24 Jun 2005. 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?
Results are not available in the source record.
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?
Myocardial T2*. 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 NCT00103753, 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 24 Jun 2005.