Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda

This study is currently recruiting participants.

Verified by University of Texas Southwestern Medical Center, February 2009

First Received: January 10, 2008 Last Updated: February 27, 2009 History of Changes

Sponsors and Collaborators:	University of Texas Southwestern Medical Center Novartis Pharmaceuticals
Information provided by:	University of Texas Southwestern Medical Center
ClinicalTrials.gov Identifier:	NCT00599326

Purpose

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.

Condition	Intervention	Phase
Porphyria Cutanea Tarda	Drug: Deferasirox	Phase III

Genetics Home Reference related topics: porphyria

MedlinePlus related topics: Porphyria

Drug Information available for: Deferasirox

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Open Label, Active Control, Single Group Assignment, Safety/Efficacy Study
Official Title:	Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda

Further study details as provided by University of Texas Southwestern Medical Center:

Primary Outcome Measures:

The elimination of all skin blistering. [ Time Frame: Within 6 months of treatment. ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

Assess for a decrease in total body iron levels along with efficacy, safety and tolerability of deferasirox. [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]

Estimated Enrollment:	10
Study Start Date:	January 2008
Estimated Study Completion Date:	June 2010
Estimated Primary Completion Date:	June 2009 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
A: Experimental	Drug: Deferasirox 250 mg of deferasirox once daily for 6 months

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.

Eligibility

Ages Eligible for Study:	18 Years and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

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.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00599326

Contacts

Contact: Christina Carrigan, R.N.	214-645-8968	christina.carrigan@utsouthwestern.edu
Contact: Memoree Lee	214-645-8971	memoree.lee@utsouthwestern.edu

Locations

United States, Texas
UT Southwestern Medical Center at Dallas - Dermatology Clinical Trials	Recruiting
Dallas, Texas, United States, 75390-8802
Principal Investigator: Amit Pandya, M.D.
Sub-Investigator: Radha Yalamanchili, M.D.

Sponsors and Collaborators

University of Texas Southwestern Medical Center

Novartis Pharmaceuticals

Investigators

Principal Investigator:

Amit Pandya, M.D.

UT Southwestern Medical Center at Dallas - Department of Dermatology

More Information

No publications provided

Responsible Party:	Department of Dermatology - UT Southwestern Medical Center at Dallas ( Amit Pandya, M.D. - Professor of Dermatology )
Study ID Numbers:	CICL670A US17, IRB File Number 062007-047
Study First Received:	January 10, 2008
Last Updated:	February 27, 2009
ClinicalTrials.gov Identifier:	NCT00599326 History of Changes
Health Authority:	United States: Food and Drug Administration

Keywords provided by University of Texas Southwestern Medical Center:

Exjade PCT Study
PCT Study
Porphyria Cutanea Tarda

Study placed in the following topic categories:

Liver Diseases
Metabolic Diseases
Porphyrias
Skin Diseases
Deferasirox
Porphyria, Congenital Erythropoietic
Metabolism, Inborn Errors
Digestive System Diseases
Genetic Diseases, Inborn

Porphyrias, Hepatic
Chelating Agents
Porphyria, Erythropoietic
Porphyria
Iron
Metabolic Disorder
Porphyria Cutanea Tarda
Skin Diseases, Genetic

Additional relevant MeSH terms:

Liver Diseases
Metabolic Diseases
Molecular Mechanisms of Pharmacological Action
Porphyrias
Skin Diseases
Deferasirox
Iron Chelating Agents
Pharmacologic Actions
Metabolism, Inborn Errors

Digestive System Diseases
Genetic Diseases, Inborn
Skin Diseases, Metabolic
Porphyrias, Hepatic
Chelating Agents
Porphyria, Erythropoietic
Porphyria Cutanea Tarda
Skin Diseases, Genetic

ClinicalTrials.gov processed this record on May 07, 2009