Porphyria Cutanea Tarda Clinical Trial
Official title:
A Phase II, Open Label Clinical Trial Exploring the Safety and the Efficacy of Oral Deferasirox in Patients Newly Diagnosed With Porphyria Cutanea Tarda (PCT) and Non-transfusion Iron Overload
While clinical phlebotomy is current standard practice for alleviating non-transfusion iron overload in patients with PCT, it may not be suitable for all patients. For example, some patients are unwilling to be adequately phlebotomized because of inconvenience, as phlebotomy can be cumbersome, especially during the induction treatment phase requiring frequent clinic visits (twice a month, for at least 6 months) or because of venous access difficulties. Other patients are unable to undergo phlebotomy due to medical reasons such as anemia or cardiopulmonary disorders. It is postulated such patients with PCT who have non-transfusion iron overload could benefit from treatment with deferasirox (Exjade®), a once daily oral iron chelator licensed in several countries, including the EU, for treating transfusion iron overload in adult and pediatric patients. Although there is some data on the efficacy and safety of deferasirox in patients with HH, who, like those with PCT, have non-transfusional iron overload, there is a need to evaluate the safety and efficacy of deferasirox treatment of non-transfusion iron overload in patients with PCT.
The primary objective is to assess the safety of deferasirox in treating non-transfusion
iron overload in patients with PCT.
The secondary objective is to assess the effectiveness of deferasirox treatment :
After 3 and 6 months to:
•Lower serum ferritin from abnormal to normal standard ranges specified for males and
females in this patient population.
After 6 months to :
•Lower liver iron content after 24 weeks of treatment measured by liver MRI T2
After 3 and 6 months to :
- Improve clinical symptoms, i.e. improvement in skin lesions (reduction or no new bullae
formation), and skin fragility (photographs will be used).
- Reduce porphyrin levels.
;
Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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