Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

10 participants

Study Classification

INTERVENTIONAL

Study Start Date

2008-01-31

Study Completion Date

2010-04-30

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.

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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.

Conditions

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Porphyria Cutanea Tarda

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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A

Group Type EXPERIMENTAL

Deferasirox

Intervention Type DRUG

250 mg of deferasirox once daily for 6 months

Interventions

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Deferasirox

250 mg of deferasirox once daily for 6 months

Intervention Type DRUG

Other Intervention Names

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Exjade

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.

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No