Clinical Trial: Safety of ICL670 vs. Deferoxamine in Sickle Cell Disease Patients With Iron Overload Due to Blood Transfusions

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

Official Title: A Randomized, Open Label, Phase II Study on Safety and Efficacy of Long Term Treatment of ICL670 Relative to Deferoxamine in Sickle Cell Disease Patients With Transfusiona

Brief Summary: The purpose of this study is to determine if the new orally active iron chelator, ICL670, is as safe as deferoxamine in preventing accumulation of iron in the body while a patient is undergoing repeated blood transfusions.

Detailed Summary: Patients who require repeated blood transfusions accumulate iron in the body as blood cells contain iron and there is no natural body mechanism to eliminate it. After a while the iron levels get high enough to be toxic to the body. The current therapy of choice is deferoxamine which does a good job of removing excess iron, but is difficult to administer. Deferoxamine requires subcutaneous (under the skin) infusions over 4 to 8 hours nightly 3 to 7 nights per week. In addition to the need to wear an infusion pump nightly, adverse reactions around the site of the injection are frequent.
Sponsor: Novartis Pharmaceuticals

Current Primary Outcome: Evaluate the safety and tolerability of multiple doses of ICL670 [ Time Frame: 1 year ]

Original Primary Outcome:

Current Secondary Outcome:

  • Estimate the absolute and relative change of liver iron content (LIC) and total body iron excretion (TBIE) [ Time Frame: at baseline, after 24 weeks and at 1year (end of study) ]
  • Evaluate the pharmacokinetics [ Time Frame: 24 hours post-dose @ 4, 12, 24 and 52 weeks ]
  • Evaluate the relationship between pharmacokinetics, pharmacodynamics and safety variables [ Time Frame: at 24 and 52 weks pre-dose ]
  • Evaluate the relationship between hepatic iron and potential surrogate markers [ Time Frame: at screen, at washout, then every 2 weeks for the first 12 weeks followed by every 4 weeks ]


Original Secondary Outcome:

Information By: Novartis

Dates:
Date Received: August 11, 2003
Date Started: May 2003
Date Completion:
Last Updated: February 28, 2017
Last Verified: November 2016