Beta Thalassemia Transfusion Dependent Clinical Trial
Official title:
Multicenter, Open Label, Prospective Study to Evaluate the Efficacy and Safety of Deferasirox 30 mg/kg/Day for 52 Weeks, in Transfusion-dependent Beta-thalassemic Patients With Cardiac MRI T2* < 20 Msec
| Verified date | November 2011 |
| Source | Novartis |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | Italy: The Italian Medicines Agency |
| Study type | Interventional |
This study will assess the safety and efficacy of deferasirox in patients with cardiac MRI T2* < 20 msec.
| Status | Completed |
| Enrollment | 20 |
| Est. completion date | |
| Est. primary completion date | May 2011 |
| Accepts healthy volunteers | No |
| Gender | Both |
| Age group | 10 Years and older |
| Eligibility |
Inclusion Criteria: - ß-thalassemic patients receiving regular transfusions with an history of at least 20 transfusional events - Patients naïve for deferasirox - Serum ferritin levels =1000ng/ml (average of the last 6 months assays); - Cardiac MRI T2* >5 and <20 msec. - LVEF at MRI =56% Exclusion Criteria: - Patients that have already started deferasirox therapy - Existing contraindications to the performance of MRI procedure, including presence of metallic prostheses not compatible with the MRI procedure other than those approved as safe for use in MRI scanners (e.g., some types of aneurysm clips, shrapnel in proximity to vital organs such as the retina), pacemaker, intractable claustrophobia and incapability to follow the instruction for the correct performance of the MRI (e.g. to maintain a supine position, to hold breath, etc.) - Clinical conditions requiring intensive chelating therapy on the basis of Investigator's judgment - Stable average ALT levels >300 U/L in the preceding 12 months - Uncontrolled systemic hypertension - Estimated creatinine clearance <60 ml/min - History of nephrotic syndrome - History of clinically significant ocular toxicity related to the chelating therapy - Psychiatric disorders or drug dependence that might impair the capability of the patient to provide their Informed Consent or to be administered the study treatment - Known sensitivity to study drug(s) or class of study drug(s) - Patients with severe medical condition(s) that in the view of the investigator prohibits participation in the study - Use of any other investigational agent in the last 30 days. Other protocol-defined inclusion/exclusion criteria may apply. |
Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
| Country | Name | City | State |
|---|---|---|---|
| Italy | Novartis Investigative Site | Cagliari | |
| Italy | Novartis Investigative Site | Orbassano |
| Lead Sponsor | Collaborator |
|---|---|
| Novartis Pharmaceuticals |
Italy,
Piga A, Longo F, Origa R, Roggero S, Pinna F, Zappu A, Castiglioni C, Cappellini MD. Deferasirox for cardiac siderosis in ß-thalassaemia major: a multicentre, open label, prospective study. Br J Haematol. 2014 Nov;167(3):423-6. doi: 10.1111/bjh.12987. — View Citation
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Change from baseline in cardiac T2* value | Unsatisfactory response is a monthly MRI T2* improvement lower than 3% versus baseline. | 52 weeks | No |
| Secondary | Change from baseline in cardiac functions parameters (LVEF, LVESV and LVEDV) | 52 weeks | No | |
| Secondary | Change from baseline liver T2* value | 52 weeks | No |