Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda

Phase 3

Completed

• Conditions: Porphyria Cutanea Tarda
• Interventions: Drug: Deferasirox

• Registration Number: NCT00599326
• Lead Sponsor: University of Texas Southwestern Medical Center

Brief 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.

Detailed 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.

Study Timeline

• Study Start: 2008-01
• Study First Submitted: 2008-01-10
• Study First Submitted QC: 2008-01-10
• Study First Posted: 2008-01-23
• Primary Completion: 2010-04
• Study Completion: 2010-04
• Results First Submitted: 2013-08-21
• Status Verified: 2014-01
• Results First Submitted QC: 2014-01-09
• Last Update Submitted: 2014-01-09
• Results First Posted: 2014-02-10
• Last Update Posted: 2014-02-10

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 10

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
A	Deferasirox	-

Primary Outcome Measures

Name	Time	Method
Number of Participants Showing Reduction or Elimination of Skin Blistering	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.

Secondary Outcome Measures

Name	Time	Method
Number of Participants Showing Decrease in Ferritin and Urinary Porphyrin Level	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.

Trial Locations

Locations (1)

UT Southwestern Medical Center at Dallas - Dermatology Clinical Trials; 🇺🇸 Dallas, Texas, United States