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
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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.
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|Ages Eligible for Study:||18 Years and older (Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- diagnosis of Porphyria Cutanea Tarda based on clinical exam and 24-hour urine porphyrin levels
- have Porphyria Cutanea Tarda for at least 3 months prior to enrollment with active blistering (3 blisters or erosions per month)
- women of childbearing potential must use an effective method of contraception during the study, however this cannot include hormonal contraception (oral contraceptives, hormone patches, Depo-Provera injections, NUVA Ring, etc.)
- treatment naive patients or patients unresponsive or intolerant of phlebotomy
- Ferritin level is greater than or equal to 25ng/mL
- patients with serum creatinine above the upper limit of normal
- patients receiving phlebotomy who are controlled on this therapy
- pregnant or breast feeding females
- patients with liver transaminases more than 5 times the upper limit of normal
- patients with a history of hypersensitivity to deferasirox
- patients with a history of pre-existing renal condition, or receiving medication that depresses renal function
- patients on other chelators
- history of non-compliance to medical regimens.
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|
|Responsible Party:||Amit Pandya, Professor of Dermatology, University of Texas Southwestern Medical Center|
|Other Study ID Numbers:||
IRB File Number 062007-047
|First Posted:||January 23, 2008 Key Record Dates|
|Results First Posted:||February 10, 2014|
|Last Update Posted:||February 10, 2014|
|Last Verified:||January 2014|
Exjade PCT Study
Porphyria Cutanea Tarda
Porphyria Cutanea Tarda
Skin Diseases, Genetic
Genetic Diseases, Inborn
Digestive System Diseases
Iron Chelating Agents
Molecular Mechanisms of Pharmacological Action