Combined Chelation Treatment With Deferiprone and Deferoxamine in Thalassemia Major

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

UNKNOWN

Clinical Phase

PHASE4

Total Enrollment

65 participants

Study Classification

INTERVENTIONAL

Study Start Date

2004-05-31

Study Completion Date

2005-06-30

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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

See the medical conditions and disease areas that this research is targeting or investigating.

Beta-Thalassemia

Keywords

Explore important study keywords that can help with search, categorization, and topic discovery.

Randomized Controlled Trial Deferiprone Deferoxamine Iron chelation Beta Thalassemia Major

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

deferiprone

Intervention Type DRUG

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* 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)

Exclusion Criteria

* 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

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Ospedale Microcitemico, Via Jenner

Cagliari, Sardinia, Italy

Site Status

Countries

Review the countries where the study has at least one active or historical site.

Italy

References

Explore related publications, articles, or registry entries linked to this study.

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.

Reference Type DERIVED

PMID: 18298856 (View on PubMed)

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

02 065

Identifier Type: -

Identifier Source: org_study_id