Clinical Trial: Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda

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

Official Title: Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda

Brief Summary:

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.


Detailed Summary:

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.


Sponsor: University of Texas Southwestern Medical Center

Current Primary Outcome: Number of Participants Showing Reduction or Elimination of Skin Blistering [ Time Frame: Within 6 months of treatment. ]

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.


Original Primary Outcome: The elimination of all skin blistering. [ Time Frame: Within 6 months of treatment. ]

Current Secondary Outcome: Number of Participants Showing Decrease in Ferritin and Urinary Porphyrin Level [ Time Frame: 6 months ]

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.


Original Secondary Outcome: Assess for a decrease in total body iron levels along with efficacy, safety and tolerability of deferasirox. [ Time Frame: 6 months ]

Information By: University of Texas Southwestern Medical Center

Dates:
Date Received: January 10, 2008
Date Started: January 2008
Date Completion:
Last Updated: January 9, 2014
Last Verified: January 2014