NCT00879242

Brief Summary

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

Trial Health

80
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
20

participants targeted

Target at below P25 for phase_2

Geographic Reach
1 country

2 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

February 1, 2009

Completed
2 months until next milestone

First Submitted

Initial submission to the registry

April 8, 2009

Completed
1 day until next milestone

First Posted

Study publicly available on registry

April 9, 2009

Completed
2.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 1, 2011

Completed
Last Updated

December 12, 2016

Status Verified

November 1, 2011

Enrollment Period

2.2 years

First QC Date

April 8, 2009

Last Update Submit

December 8, 2016

Conditions

Beta Thalassemia Transfusion Dependent

Keywords

Thalassemiacardiac MRI T2*

Outcome Measures

Primary Outcomes (1)

  • Change from baseline in cardiac T2* value

    Unsatisfactory response is a monthly MRI T2\* improvement lower than 3% versus baseline.

    52 weeks

Secondary Outcomes (2)

  • Change from baseline in cardiac functions parameters (LVEF, LVESV and LVEDV)

    52 weeks

  • Change from baseline liver T2* value

    52 weeks

Study Arms (1)

Deferasirox

EXPERIMENTAL

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

Interventions

DeferasiroxDRUG

Dispersible tables of 125, 250 and 500 mg at the dose of 30 mg/kg/day, oral administration.

Deferasirox

Eligibility Criteria

Age10 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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%

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Novartis Investigative Site

Cagliari, Italy

Location

Novartis Investigative Site

Orbassano, Italy

Location

Related Publications (1)

  • Piga A, Longo F, Origa R, Roggero S, Pinna F, Zappu A, Castiglioni C, Cappellini MD. Deferasirox for cardiac siderosis in beta-thalassaemia major: a multicentre, open label, prospective study. Br J Haematol. 2014 Nov;167(3):423-6. doi: 10.1111/bjh.12987. Epub 2014 Jun 30. No abstract available.

    PMID: 24976388RESULT

MeSH Terms

Conditions

Thalassemia

Interventions

Deferasirox

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, CongenitalAnemia, HemolyticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesHemoglobinopathiesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

BenzoatesAcids, CarbocyclicCarboxylic AcidsOrganic ChemicalsBenzene DerivativesHydrocarbons, AromaticHydrocarbons, CyclicHydrocarbonsTriazolesAzolesHeterocyclic Compounds, 1-RingHeterocyclic Compounds

Study Officials

  • Novartis Pharmaceuticals

    Novartis Pharmaceuticals

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

April 8, 2009

First Posted

April 9, 2009

Study Start

February 1, 2009

Primary Completion

May 1, 2011

Last Updated

December 12, 2016

Record last verified: 2011-11

Locations

IT(2)