Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda

• Status: Completed
• Trial end date: 2010-04-01
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: University of Texas Southwestern Medical Center
• Collaborator: Novartis Pharmaceuticals
• Treatments: Deferasirox

Criteria

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.