Back to resultsPilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda
Primary Purpose
Porphyria Cutanea Tarda
Status
Completed
Phase
Phase 3
Locations
United States
Study Type
Interventional
Intervention
Deferasirox
Sponsored by
About this trial
This is an interventional treatment trial for Porphyria Cutanea Tarda focused on measuring Exjade PCT Study, PCT Study, Porphyria Cutanea Tarda
Eligibility 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.
Sites / Locations
- UT Southwestern Medical Center at Dallas - Dermatology Clinical Trials
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
A
Arm Description
Outcomes
Primary Outcome Measures
Number of Participants Showing Reduction or Elimination of Skin Blistering
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
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.
Full Information
NCT ID
NCT00599326
First Posted
January 10, 2008
Last Updated
January 9, 2014
Sponsor
University of Texas Southwestern Medical Center
Collaborators
Novartis Pharmaceuticals
1. Study Identification
Unique Protocol Identification Number
NCT00599326
Brief Title
Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda
Official Title
Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda
Study Type
Interventional
2. Study Status
Record Verification Date
January 2014
Overall Recruitment Status
Completed
Study Start Date
January 2008 (undefined)
Primary Completion Date
April 2010 (Actual)
Study Completion Date
April 2010 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
University of Texas Southwestern Medical Center
Collaborators
Novartis Pharmaceuticals
4. Oversight
Data Monitoring Committee
No
5. Study Description
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.
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Porphyria Cutanea Tarda
Keywords
Exjade PCT Study, PCT Study, Porphyria Cutanea Tarda
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
10 (Actual)
8. Arms, Groups, and Interventions
Arm Title
A
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Deferasirox
Other Intervention Name(s)
Exjade
Intervention Description
250 mg of deferasirox once daily for 6 months
Primary Outcome Measure Information:
Title
Number of Participants Showing Reduction or Elimination of Skin Blistering
Description
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.
Time Frame
Within 6 months of treatment.
Secondary Outcome Measure Information:
Title
Number of Participants Showing Decrease in Ferritin and Urinary Porphyrin Level
Description
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.
Time Frame
6 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Amit Pandya, M.D.
Organizational Affiliation
UT Southwestern Medical Center at Dallas - Department of Dermatology
Official's Role
Principal Investigator
Facility Information:
Facility Name
UT Southwestern Medical Center at Dallas - Dermatology Clinical Trials
City
Dallas
State/Province
Texas
ZIP/Postal Code
75390-8802
Country
United States
12. IPD Sharing Statement
Citations:
PubMed Identifier
22911183
Citation
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.
Results Reference
derived
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Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda
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