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Clinical Trial Details — Status: Active, not recruiting

Administrative data

NCT number NCT00103753
Other study ID # 02 065
Secondary ID
Status Active, not recruiting
Phase Phase 4
First received February 14, 2005
Last updated June 23, 2005
Start date May 2004
Est. completion date June 2005

Study information

Verified date February 2005
Source Royal Brompton & Harefield NHS Foundation Trust
Contact n/a
Is FDA regulated No
Health authority United Kingdom: National Health Service
Study type Interventional

Clinical 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. 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.


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.


Recruitment information / eligibility

Status Active, not recruiting
Enrollment 65
Est. completion date June 2005
Est. primary completion date
Accepts healthy volunteers No
Gender Both
Age group 18 Years and older
Eligibility 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

Study Design

Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Double-Blind, Primary Purpose: Treatment


Related Conditions & MeSH terms


Intervention

Drug:
deferiprone


Locations

Country Name City State
Italy Ospedale Microcitemico, Via Jenner Cagliari Sardinia

Sponsors (5)

Lead Sponsor Collaborator
Royal Brompton & Harefield NHS Foundation Trust Apotex Inc., CORDA, The Heart Charity, The Cooley’s Anemia Foundation,, The UK Thalassemia Society

Country where clinical trial is conducted

Italy, 

Outcome

Type Measure Description Time frame Safety issue
Primary Myocardial T2*
Secondary Liver T2*
Secondary LV and RV volumes and function in systole and diastole
Secondary Brachial artery reactivity
Secondary B-type natriuretic peptide
Secondary Patient compliance
Secondary Adverse events
Secondary Success of blinding
See also
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Withdrawn NCT04938635 - Efficacy and Safety Study of Multiple Doses of VIT-2763 in Adults With Transfusion-dependent Beta-thalassemia Phase 2
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Completed NCT06239389 - Comparison Of Efficacy And Safety Of Thalidomide Vs Hydroxyurea In Thalassemia Patients: A Single-Centre Pilot Study. Phase 2
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Enrolling by invitation NCT03655223 - Early Check: Expanded Screening in Newborns
Completed NCT03961828 - Hyalornic Acid Level in β-Thalassemic Children Treated for Hepatitis C Virus Phase 4
Recruiting NCT06065189 - Base-edited Autologous Hematopoietic Stem Cell Transplantation in Treating Patients With β-thalassemia Major Early Phase 1
Recruiting NCT04143724 - Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia Phase 2
Terminated NCT03381833 - A Study With LJPC-401 for the Treatment of Myocardial Iron Overload in Patients With Transfusion-Dependent Beta Thalassemia Phase 2
Not yet recruiting NCT01996683 - Efficacy and Safety of Efficacy and Safety of Continued Iron Chelation Therapy In Poly-transfused Thalassemia Patients With Low Serum Ferritin (< 500 ng/ml) N/A
Completed NCT02268409 - ACE-536 Extension Study - Beta Thalassemia Phase 2
Active, not recruiting NCT01016093 - Zoledronic Acid for the Prevention of Bone Loss Post-bone Marrow Transplantation for Thalassemia Major Patients Phase 2/Phase 3
Completed NCT01039636 - Safety and Pharmacokinetic Study of Escalating Multiple Doses of an Iron Chelator in Patients With Iron Overload Phase 1