Porphyria Cutanea Tarda Clinical Trial
Official title:
Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda
To determine the efficacy and tolerability of deferasirox in the treatment of Porphyria
Cutanea Tarda.
Primary objective - the elimination of all blistering within 6 months of treatment.
Secondary objective - decrease in total body iron levels.
Phlebotomy is the standard therapy for Porphyria Cutanea Tarda (PCT), but it can be
inconvenient and cause anemia in some patients.
Deferasirox is a new class of tridentate iron chelators with high affinity and selectivity
for iron. The medication is administered orally, which if effective for PCT would make it a
more convenient and possibly more tolerable option for patients.
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Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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