Effect of Deferasirox on Patients With Cardiac MRI T2* < 20 Msec

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT00879242
First received: April 8, 2009
Last updated: November 14, 2011
Last verified: November 2011
  Purpose

This study will assess the safety and efficacy of deferasirox in patients with cardiac MRI T2* < 20 msec.


Condition Intervention Phase
Beta Thalassemia Transfusion Dependent
Drug: Deferasirox
Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
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

Resource links provided by NLM:


Further study details as provided by Novartis:

Primary Outcome Measures:
  • Change from baseline in cardiac T2* value [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]
    Unsatisfactory response is a monthly MRI T2* improvement lower than 3% versus baseline.


Secondary Outcome Measures:
  • Change from baseline in cardiac functions parameters (LVEF, LVESV and LVEDV) [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]
  • Change from baseline liver T2* value [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]

Enrollment: 20
Study Start Date: February 2009
Primary Completion Date: May 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Deferasirox
30 mg/kg/day. The daily dose can be increased to 40 mg/kg in case of unsatisfactory response after 12 weeks of treatment.
Drug: Deferasirox
Dispersible tables of 125, 250 and 500 mg at the dose of 30 mg/kg/day, oral administration.

  Eligibility

Ages Eligible for Study:   10 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • β-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.

  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00879242

Locations
Italy
Novartis Investigative Site
Cagliari, Italy
Novartis Investigative Site
Orbassano, Italy
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  More Information

No publications provided

Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT00879242     History of Changes
Other Study ID Numbers: CICL670AIT07, EUDRACT Code :2008-003230-22
Study First Received: April 8, 2009
Last Updated: November 14, 2011
Health Authority: Italy: The Italian Medicines Agency

Keywords provided by Novartis:
Thalassemia
cardiac MRI T2*

Additional relevant MeSH terms:
Beta-Thalassemia
Thalassemia
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
Deferasirox
Iron Chelating Agents
Chelating Agents
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions

ClinicalTrials.gov processed this record on April 17, 2014