Clinical Trial: Evaluation of the Safety, Tolerability, Pharmacokinetics (PK) and Effects on Liver Iron Concentration of ICL670 Relative to Deferoxamine(DFO).

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

Official Title: A One Year Open Label, Non-comparative Extension to a Randomized, Multicenter, Phase II Study to Evaluate the Safety, Tolerability, Pharmacokinetics (PK) and Effects on Liver Iron Concentration (LIC)

Brief Summary: The safety, tolerability, effects on liver iron concentration and pharmacokinetics of ICL670 is studied in sickle cell disease patients with transfusional hemosiderosis.

Detailed Summary: The treatment period started once the patient completed the core study and signed informed consent. It is continued for up to 4 years. Safety parameters were assessed every 4 weeks. Eye and Ear examinations were performed on a yearly basis. To further investigate the extent of iron overload, serum ferritin, iron, and transferrin were monitored every four weeks. The Program Safety Board monitored the safety of ICL670 during the study to evaluate and categorize any serious case reported in association with ICL670.
Sponsor: Novartis Pharmaceuticals

Current Primary Outcome: Number of Participants With Adverse Events After Start of ICL670 [ Time Frame: 0 - 60 months ]

Safety as assessed by the number of participants with adverse event or death after the start of ICL670.


Original Primary Outcome: To evaluate the long-term safety and tolerability profile of ICL670 in sickle cell disease patients with transfusional iron overload requiring chelation therapy [ Time Frame: 0 - 60 months ]

Current Secondary Outcome: Change in Serum Ferritin From Start of ICL670 to End of Study [ Time Frame: 0 - 60 months ]

The main efficacy variable was change in serum ferritin in response to therapy with ICL670. Due to variability of serum ferritin, end of study was considered as the mean of at most the last 3 available observations after the start of ICL670.


Original Secondary Outcome:

  • To allow patients treated with either ICL670 or DFO satisfactorily completing the week 52 assessment of the core protocol ICL670A0109 to continue iron chelation therapy with ICL670 [ Time Frame: 0 - 60 months ]
  • Change in serum ferritin over time in response to therapy with ICL670 [ Time Frame: 0 60 months ]
  • To determine LIC by MRI in a subset of patients (Although it was specified as a secondary objective in the protocol, MRI data were not collected in the extension phase of the trial). [ Time Frame: 0 - 60 months ]
  • Determinations relative to iron metabolism (serum ferritin, total iron, transferrin and transferrin saturation) [ Time Frame: 0 - 60 months ]


Information By: Novartis

Dates:
Date Received: March 15, 2010
Date Started: July 2004
Date Completion:
Last Updated: May 3, 2011
Last Verified: May 2011