Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda

Study Overview
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Brief Summary
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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.

Detailed Description
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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.

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

Conditions
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Porphyria Cutanea Tarda

Intervention / Treatment
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* Drug: Deferasirox


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 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 Exclusion Criteria: 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.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: A<br> | Drug: Deferasirox<br>* 250 mg of deferasirox once daily for 6 months<br>* Other names: Exjade;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants Showing Reduction or Elimination of Skin Blistering | 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. | Within 6 months of treatment. | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants Showing Decrease in Ferritin and Urinary Porphyrin Level | 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. | 6 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Exjade PCT Study, PCT Study, Porphyria Cutanea Tarda