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.
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda|
- The elimination of all skin blistering. [ Time Frame: Within 6 months of treatment. ] [ Designated as safety issue: No ]
- Assess for a decrease in total body iron levels along with efficacy, safety and tolerability of deferasirox. [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]
|Study Start Date:||January 2008|
|Study Completion Date:||April 2010|
|Primary Completion Date:||April 2010 (Final data collection date for primary outcome measure)|
250 mg of deferasirox once daily for 6 months
Other Name: Exjade
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.
|United States, Texas|
|UT Southwestern Medical Center at Dallas - Dermatology Clinical Trials|
|Dallas, Texas, United States, 75390-8802|
|Principal Investigator:||Amit Pandya, M.D.||UT Southwestern Medical Center at Dallas - Department of Dermatology|