Clinical Trial: Study Using Deferiprone Alone or in Combination With Desferrioxamine in Iron Overloaded Transfusion-dependent Patients

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

Official Title: Retrospective and Prospective Multicenter Study Using Deferiprone (L1) Alone or in Combination With Desferrioxamine for the Treatment of Iron Overload in Transfusion-dependent Patients

Brief Summary: Systematical (retro- and prospective) investigation of the long-term safety (toxicity assessment according to CTCAE v3.0) and efficacy of deferiprone either given alone or in combination with desferrioxamine

Detailed Summary:

Patients with refractory anemias requiring regular blood transfusions accumulate iron at the rate of approximately 0.5 mg/kg/day, which may lead to serious organ toxicity, e.g. to the heart, liver and endocrine organs. The human body has no active mechanism for the excretion of excess iron. Therefore multiply transfused patients will develop a secondary hemosiderosis, if excess iron is not excreted by a chelating agent. Symptoms of iron-overload occur when body iron stores reach 10-20 g. At higher levels severe, even fatal complications, particularly cardiac failure, may develop.

Desferrioxamine (DFO, Desferal) is the established and commonly used iron-chelating drug, but is expensive and must be given by slow subcutaneous or intravenous infusion for 8-12 hours a day during 5-7 days weekly at a dosage of 40-50 mg/kg body weight/day. This often leads to failure of compliance of the patient and therefore to inefficient iron chelation. Further, some patients are hypersensitive to desferrioxamine and others suffer from toxicity, e.g. to the ears or eyes.

Deferiprone (L1; CP20; 1,2 dimethyl-3-hydroxypyrid-4-one) is an orally active iron chelator investigated in various clinical trials since 1987. Dosages of 75 - 100 mg/kg body weight/day of L1 have been considered effective to maintain stable iron balance (urinary iron excretion of 0.5 mg/kg/day) and to reduce serum ferritin levels between 6% and 25% within one year of treatment in iron-overloaded thalassemic patients. There exists long-term experience with patients who have received deferiprone continuously for more than 10 years so far. The main side effects encountered during a deferiprone therapy are arthropathy, gastrointestinal symptoms, headache, and mild zinc deficiency. These adverse reactions are usually reversed on reducing the dose or discontinuing the drug. Except
Sponsor: Lipomed

Current Primary Outcome:

  • Liver Iron Concentration (LIC) by SQUID [ Time Frame: Yearly ]
  • Long-term safety profile [ Time Frame: Long-term ]


Original Primary Outcome:

  • Liver Iron Concentration (LIC) by SQUID at yearly control visits
  • Long-term safety profile


Current Secondary Outcome:

  • Serum ferritin [ Time Frame: At quarterly control visits ]
  • Urinary Iron Excretion (UIE) [ Time Frame: At six-monthly control visits ]
  • Heart iron content (optional) by MRI T2* and MRI SIR [ Time Frame: Yearly ]


Original Secondary Outcome:

  • Serum ferritin at quarterly control visits
  • Urinary Iron Excretion (UIE) at six-monthly control visits
  • Heart iron content (optional) by MRI T2* and MRI SIR at yearly control visits


Information By: Lipomed

Dates:
Date Received: July 6, 2006
Date Started: March 2005
Date Completion:
Last Updated: December 9, 2011
Last Verified: December 2011