Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda

The University of Texas System (UT)

Status and phase: Completed; Phase 3

Conditions: Porphyria Cutanea Tarda

Treatments: Drug: Deferasirox

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT00599326

CICL670A US17

IRB File Number 062007-047

Details and patient eligibility

About

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.

Full description

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.

Enrollment: 10 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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10 participants in 1 patient group

A

Experimental group

Treatment:

Drug: Deferasirox