Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda

Porphyria Cutanea Tarda Clinical Trial

Official title:

Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00599326
• Other study ID #: CICL670A US17
• Secondary ID: IRB File Number
• Status: Completed
• Phase: Phase 3
• First received: January 10, 2008
• Last updated: January 9, 2014
• Start date: January 2008
• Est. completion date: April 2010

Study information

• Verified date: January 2014
• Source: University of Texas Southwestern Medical Center
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 10
• Est. completion date: April 2010
• Est. primary completion date: April 2010
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

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

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Porphyria Cutanea Tarda
• Porphyria, Erythropoietic
• Porphyrias

Intervention

Drug:
• Deferasirox
250 mg of deferasirox once daily for 6 months

Locations

Country	Name	City	State
United States	UT Southwestern Medical Center at Dallas - Dermatology Clinical Trials	Dallas	Texas

Sponsors (2)

Lead Sponsor	Collaborator
University of Texas Southwestern Medical Center	Novartis Pharmaceuticals

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	No
Secondary	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	No