Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda

This study has been completed.
Sponsor:
Collaborator:
Novartis Pharmaceuticals
Information provided by (Responsible Party):
Amit Pandya, University of Texas Southwestern Medical Center
ClinicalTrials.gov Identifier:
NCT00599326
First received: January 10, 2008
Last updated: January 9, 2014
Last verified: January 2014

January 10, 2008
January 9, 2014
January 2008
April 2010   (final data collection date for primary outcome measure)
Number of Participants Showing Reduction or Elimination of Skin Blistering [ Time Frame: Within 6 months of treatment. ] [ Designated as safety issue: No ]
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.
The elimination of all skin blistering. [ Time Frame: Within 6 months of treatment. ] [ Designated as safety issue: No ]
Complete list of historical versions of study NCT00599326 on ClinicalTrials.gov Archive Site
Number of Participants Showing Decrease in Ferritin and Urinary Porphyrin Level [ Time Frame: 6 months ] [ Designated as safety issue: No ]
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.
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 ]
Not Provided
Not Provided
 
Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda
Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda

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.

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.

Interventional
Phase 3
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Porphyria Cutanea Tarda
Drug: Deferasirox
250 mg of deferasirox once daily for 6 months
Other Name: Exjade
Experimental: A
Intervention: Drug: Deferasirox
Pandya AG, Nezafati KA, Ashe-Randolph M, Yalamanchili R. Deferasirox for porphyria cutanea tarda: a pilot study. Arch Dermatol. 2012 Aug;148(8):898-901. doi: 10.1001/archdermatol.2012.807.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
10
April 2010
April 2010   (final data collection date for primary outcome measure)

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.
Both
18 Years and older
No
Contact information is only displayed when the study is recruiting subjects
United States
 
NCT00599326
CICL670A US17, IRB File Number 062007-047
No
Amit Pandya, University of Texas Southwestern Medical Center
University of Texas Southwestern Medical Center
Novartis Pharmaceuticals
Principal Investigator: Amit Pandya, M.D. UT Southwestern Medical Center at Dallas - Department of Dermatology
University of Texas Southwestern Medical Center
January 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP