Clinical Trial: A Protocol to Allow Treatment With ICL670 for Patients With or at Risk of Life-threatening Complications of Transfusional Iron Overload Who Are Unable to Tolerate Other Iron Chelators Because of Documented Severe Toxicity

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

Official Title: A Protocol to Allow Treatment With ICL670 for Patients With or at Risk of Life-threatening Complications of Transfusional Iron Overload Who Are Unable to Tolerate Other Iron Chelators Because of Docum

Brief Summary: The purpose of this open-label, non-comparative, multi-center protocol was to further evaluate safety and to provide treatment with ICL670 to patients who had or were at risk of life threatening complications due to transfusional iron overload with a documented inability to tolerate any of the commercially available iron chelators due to severe toxicity rendering continued therapy either impossible or hazardous. Patients who were also ineligible for all on-going registration trials with ICL670 were included in the study. In exceptional cases, patients with a degree of iron overload which was not immediately life-threatening and who were ineligible for the registration trials were also enrolled provided they had a well-documented, sound justification for alternative chelation therapy.

Detailed Summary:
Sponsor: Novartis Pharmaceuticals

Current Primary Outcome: To evaluate the safety profile and to provide treatment with ICL670 for patients with or at risk of life-threatening complications due to transfusional iron overload who are unable to tolerate other iron chelators because of documented severe toxicity. [ Time Frame: 0 - 163 weeks ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • To estimate the absolute and relative change of liver iron concentration (LIC), to be measured using appropriate methodology available at individual centers. [ Time Frame: Yearly ]
  • To evaluate the role of serum ferritin, serum iron, transferrin and transferrin saturation in monitoring iron burden in these patients. [ Time Frame: Quarterly ]
  • To evaluate the relationship between changes in LIC and serum ferritin, transferring saturation and serum iron. [ Time Frame: Yearly ]


Original Secondary Outcome: Same as current

Information By: Novartis

Dates:
Date Received: January 5, 2010
Date Started: June 2003
Date Completion:
Last Updated: February 21, 2017
Last Verified: February 2017