A Study of Long-term Treatment With Deferasirox in Patients With Beta-thalassemia and Transfusional Hemosiderosis

February 28, 2017 updated by: Novartis

A Study of Efficacy and Safety of Long-term Treatment With Deferasirox in Patients With Beta-thalassemia and Transfusional Hemosiderosis

Because patients with beta-thalassemia are unable to actively eliminate iron from the body, toxic and eventually lethal levels of iron can accumulate as a result of repeated blood transfusions. This study will evaluate the efficacy, safety and tolerability of deferasirox.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

252

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Egypt
      • Cairo, Egypt
        • Novartis Investigative Site
  • Lebanon
      • Beirut, Lebanon, Riad El Solh 1107 2020
        • Ali Taher
  • Oman
      • Muscat, Oman
        • Novartis Investigative Site
  • Saudi Arabia
      • Riyadh, Saudi Arabia
        • Novartis Investigative Site
  • Syrian Arab Republic
      • Damascus, Syrian Arab Republic
        • Novartis Investigative Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

2 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Beta-thalassemia outpatients with transfusional hemosiderosis and unable to be chelated with deferoxamine (DFO) due to DFO being contra-indicated and/or due to documented unacceptable toxicity of DFO or documented poor response to DFO despite proper compliance, or documented non-compliance to DFO, with serum ferritin ≥ 500 ng/ml and LIC ≥ 2 mg/Fe/g dw liver
  • Beta-thalassemia outpatients with transfusional hemosiderosis treated with deferiprone

Exclusion Criteria:

  • Means levels of ALT > 300 U/L
  • Serum creatinine above upper limit of normal
  • Active hepatitis C or chronic hepatitis B receiving specific treatment

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Deferasirox
Drug: deferasirox

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Liver Iron Concentration (as measured by biopsy)
Time Frame: at baseline and after 1 yeor of ICL670 treatment
at baseline and after 1 yeor of ICL670 treatment

Secondary Outcome Measures

Outcome Measure
Time Frame
Iron balance, i.e. Total Body Iron Excretion (based on the iron influx as determined by the amount of red cells transfused and the change in total body iron (TBI) stores)
Time Frame: after 1 year of ICL670 treatment
after 1 year of ICL670 treatment
Putative surrogate markers such as serum ferritin serum iron, serum transferrin and transferrin saturation
Time Frame: monthly assessments for 1 year
monthly assessments for 1 year
Adverse events
Time Frame: during 1 year ICL670 treatment
during 1 year ICL670 treatment
hematology and biochemistry, urinalysis
Time Frame: monthly assessments for 1 year ICL670 treatment
monthly assessments for 1 year ICL670 treatment
ECG and Echocardiography
Time Frame: 6-monthly for 1 year ICL670 treatment
6-monthly for 1 year ICL670 treatment

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Novartis

Investigators

  • Principal Investigator: Ali Taher, Ass.Prof., American University of Beirut Medical Center

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

May 1, 2004

Primary Completion (Actual)

November 1, 2006

Study Completion

December 7, 2022

Study Registration Dates

First Submitted

September 12, 2005

First Submitted That Met QC Criteria

September 14, 2005

First Posted (Estimate)

September 15, 2005

Study Record Updates

Last Update Posted (Actual)

March 1, 2017

Last Update Submitted That Met QC Criteria

February 28, 2017

Last Verified

November 1, 2016

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CICL670A2402

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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