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|
- Number of Participants Showing Reduction or Elimination of Skin Blistering [ Time Frame: Within 6 months of treatment. ] [ Designated as safety issue: No ]The present trial was undertaken to determine if oral deferasirox could be useful in the treatment of PCT. Monthly clinic visits with a physical examination was conducted to assess the skin for blisters.
- Number of Participants Showing Decrease in Ferritin and Urinary Porphyrin Level [ Time Frame: 6 months ] [ Designated as safety issue: No ]Patients with PCT usually have normal or elevated serum iron and ferritin levels as well as increased iron absorption. Phlebotomy is conducted to analyzes the ferritin levels. Urine collection is performed and samples of the urine are analyzed for porphyrin levels.
|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|