Clinical Trial: Study With Deferiprone and/or Desferrioxamine in Iron Overloaded Patients

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

Official Title: Randomized Open-label Phase III Study With Deferiprone and/or Desferrioxamine in Iron Overloaded Patients

Brief Summary: Comparison of efficacy and toxicity of the combination treatment of deferiprone and desferrioxamine with the single agent treatment of either drug

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. The human body has no active mechanism for the excretion of excess iron. Therefore multiply transfused patients will develop a secondary hemosiderosis, if no iron excretion is achieved 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 40-50mg/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-hydroxy-pyrid-4-one) is an orally active iron chelator investigated in various clinical trials since 1987. Dosages of 75 - 100mg/kg body weight/day of L1 have been found effective to maintain stable iron balance (urinary iron excretion of 0.5mg/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. However, only few controlled comparison studies with L1 and DFO have been performed so far in order to confirm the effectiveness of deferiprone.

The main side effects encountered during a deferiprone therapy are arthropathy, gastrointestinal symp
Sponsor: Lipomed

Current Primary Outcome: Clinical efficacy (Iron balance and liver iron concentration) [ Time Frame: At baseline and at 12 months ]

Original Primary Outcome: Clinical efficacy (Iron balance and liver iron concentration) at 12 months

Current Secondary Outcome:

  • Safety profile (general, hematologic, and organ toxicity) [ Time Frame: At 3-monthly intervals ]
  • Liver histology [ Time Frame: At baseline and at 12 months ]
  • Quality of life (patient's subjective of compliance and tolerance) [ Time Frame: At 3-monthly intervals ]
  • Actual treatment duration (ATD) [ Time Frame: At 12 months ]


Original Secondary Outcome:

  • Serum ferritin at 3-monthly intervals
  • Safety profile (general, hematologic, and organ toxicity) at 3-monthly intervals
  • Liver histology at 12 months
  • Quality of life (patient’s subjective of compliance and tolerance) at 3-monthly intervals
  • Actual treatment duration (ATD)


Information By: Lipomed

Dates:
Date Received: July 10, 2006
Date Started: January 2002
Date Completion:
Last Updated: November 16, 2012
Last Verified: November 2012