Clinical Trial: Safety and Efficacy of Deferasirox in Patients With Transfusion Dependent Iron Overload - a Non-comparative Extension Study

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: A 5-year Open Label, Non-comparative Extension to a Randomized, Open-label, Phase IIa Study to Evaluate Safety, Tolerability and the Effects on Liver Iron Concentration of Repeated Doses of 10 and 20

Brief Summary: The purpose of this study is to assess the safety and the effects on liver iron of Deferasirox when given for a long treatment period in patients with transfusion dependent iron overload.

Detailed Summary:
Sponsor: Novartis Pharmaceuticals

Current Primary Outcome: The Relative Change From Baseline in Liver Iron Content (LIC) After Prolonged Use of Deferasirox [ Time Frame: Baseline to 7 Years ]

The mean percentage change in liver iron content (LIC) as assessed by superconducting quantum interference device (SQUID) was evaluated by comparing the LIC at the start of Deferasirox treatment to the LIC at the end of the 5 year extension study for participants who were treated with Deferasirox for more than 3.5 years. LIC is expressed in milligrams of iron per gram of liver dry weight (mgFe/g dw). Relative change = 1- (Change in LIC from Baseline/Baseline level) x 100.


Original Primary Outcome: To evaluate the long term safety and tolerability profile of ICL670A after administration of multiple doses. [ Time Frame: 5 years ]

Current Secondary Outcome: Relative Change in Serum Ferritin From Baseline to 3.5 Years [ Time Frame: Baseline to 3.5 years ]

The mean percentage change in serum ferritin was evaluated by comparing the serum ferritin level at the start of Deferasirox treatment to the serum ferritin level collected 18 months following the start of the extension study. Serum ferritin is measured in micrograms per Liter. Relative Change = 1- (Change in ferritin level from Baseline/Baseline level) x 100.


Original Secondary Outcome:

  • To evaluate the long term effect of ICL670A on hepatic iron stores measured by means of SQID and the total body iron stores. [ Time Frame: 5 years ]
  • To evaluate long-term serum ferritin and other surrogate markers as potential measures for the safe dose titration for ICL670A. [ Time Frame: 5 years ]


Information By: Novartis

Dates:
Date Received: October 16, 2009
Date Started: February 2003
Date Completion:
Last Updated: July 18, 2011
Last Verified: July 2011