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Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda

This study has been completed.
Novartis Pharmaceuticals
Information provided by (Responsible Party):
Amit Pandya, University of Texas Southwestern Medical Center Identifier:
First received: January 10, 2008
Last updated: January 9, 2014
Last verified: January 2014

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 3

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda

Resource links provided by NLM:

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

Primary Outcome Measures:
  • Number of Participants Showing Reduction or Elimination of Skin Blistering [ Time Frame: 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:
  • Number of Participants Showing Decrease in Ferritin and Urinary Porphyrin Level [ Time Frame: 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.

Enrollment: 10
Study Start Date: January 2008
Study Completion Date: April 2010
Primary Completion Date: April 2010 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: A Drug: Deferasirox
250 mg of deferasirox once daily for 6 months
Other Name: Exjade

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.


Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.
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Please refer to this study by its identifier: NCT00599326

United States, Texas
UT Southwestern Medical Center at Dallas - Dermatology Clinical Trials
Dallas, Texas, United States, 75390-8802
Sponsors and Collaborators
University of Texas Southwestern Medical Center
Novartis Pharmaceuticals
Principal Investigator: Amit Pandya, M.D. UT Southwestern Medical Center at Dallas - Department of Dermatology
  More Information

Publications automatically indexed to this study by Identifier (NCT Number):
Responsible Party: Amit Pandya, Professor of Dermatology, University of Texas Southwestern Medical Center Identifier: NCT00599326     History of Changes
Other Study ID Numbers: CICL670A US17
IRB File Number 062007-047
Study First Received: January 10, 2008
Results First Received: August 21, 2013
Last Updated: January 9, 2014

Keywords provided by Amit Pandya, University of Texas Southwestern Medical Center:
Exjade PCT Study
PCT Study
Porphyria Cutanea Tarda

Additional relevant MeSH terms:
Porphyria, Erythropoietic
Porphyria Cutanea Tarda
Porphyrias, Hepatic
Metabolic Diseases
Skin Diseases, Genetic
Genetic Diseases, Inborn
Skin Diseases
Liver Diseases
Digestive System Diseases
Iron Chelating Agents
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action processed this record on May 25, 2017