We're building a better ClinicalTrials.gov. Check it out and tell us what you think!
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 1 of 49 for:    Porphyria Cutanea Tarda
Previous Study | Return to List | Next Study

Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00599326
Recruitment Status : Completed
First Posted : January 23, 2008
Results First Posted : February 10, 2014
Last Update Posted : February 10, 2014
Sponsor:
Collaborator:
Novartis Pharmaceuticals
Information provided by (Responsible Party):
Amit Pandya, University of Texas Southwestern Medical Center

Study Description
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information
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.


Condition or disease Intervention/treatment Phase
Porphyria Cutanea Tarda Drug: Deferasirox Phase 3

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 Design
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 10 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda
Study Start Date : January 2008
Actual Primary Completion Date : April 2010
Actual Study Completion Date : April 2010

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Porphyria
Drug Information available for: Deferasirox

Arms and Interventions
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Arm Intervention/treatment
Experimental: A Drug: Deferasirox
250 mg of deferasirox once daily for 6 months
Other Name: Exjade


Outcome Measures
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Primary Outcome Measures :
  1. 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 :
  1. 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.


Eligibility Criteria
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
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
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00599326


Locations
Layout table for location information
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
Investigators
Layout table for investigator information
Principal Investigator: Amit Pandya, M.D. UT Southwestern Medical Center at Dallas - Department of Dermatology
More Information
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Layout table for additonal information
Responsible Party: Amit Pandya, Professor of Dermatology, University of Texas Southwestern Medical Center
ClinicalTrials.gov Identifier: NCT00599326    
Other Study ID Numbers: CICL670A US17
IRB File Number 062007-047
First Posted: January 23, 2008    Key Record Dates
Results First Posted: February 10, 2014
Last Update Posted: February 10, 2014
Last Verified: January 2014
Keywords provided by Amit Pandya, University of Texas Southwestern Medical Center:
Exjade PCT Study
PCT Study
Porphyria Cutanea Tarda
Additional relevant MeSH terms:
Layout table for MeSH terms
Porphyria Cutanea Tarda
Porphyrias, Hepatic
Porphyria, Erythropoietic
Porphyrias
Metabolic Diseases
Skin Diseases, Genetic
Genetic Diseases, Inborn
Skin Diseases
 Liver Diseases
Digestive System Diseases
Deferasirox
Iron Chelating Agents
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action