Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda

January 9, 2014 updated by: Amit Pandya, University of Texas Southwestern Medical Center

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Actual)

10

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Texas
      • Dallas, Texas, United States, 75390-8802
        • UT Southwestern Medical Center at Dallas - Dermatology Clinical Trials

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: A
Drug: Deferasirox
250 mg of deferasirox once daily for 6 months
Other Names:
  • Exjade

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
Within 6 months of treatment.

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
6 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

University of Texas Southwestern Medical Center

Collaborators

Novartis Pharmaceuticals

Investigators

  • Principal Investigator: Amit Pandya, M.D., UT Southwestern Medical Center at Dallas - Department of Dermatology

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

January 1, 2008

Primary Completion (Actual)

April 1, 2010

Study Completion (Actual)

April 1, 2010

Study Registration Dates

First Submitted

January 10, 2008

First Submitted That Met QC Criteria

January 10, 2008

First Posted (Estimate)

January 23, 2008

Study Record Updates

Last Update Posted (Estimate)

February 10, 2014

Last Update Submitted That Met QC Criteria

January 9, 2014

Last Verified

January 1, 2014

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CICL670A US17
  • IRB File Number 062007-047

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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