Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda
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|ClinicalTrials.gov Identifier: NCT00599326|
Recruitment Status : Completed
First Posted : January 23, 2008
Results First Posted : February 10, 2014
Last Update Posted : February 10, 2014
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.
|Condition or disease||Intervention/treatment||Phase|
|Porphyria Cutanea Tarda||Drug: Deferasirox||Phase 3|
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.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||10 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda|
|Study Start Date :||January 2008|
|Actual Primary Completion Date :||April 2010|
|Actual Study Completion Date :||April 2010|
250 mg of deferasirox once daily for 6 months
Other Name: Exjade
- 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.
- 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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00599326
|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|