Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda
This study has been completed.
Sponsor:
University of Texas Southwestern Medical Center
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: September 18, 2012
Last verified: September 2012
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Purpose
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: | Endpoint Classification: Safety/Efficacy Study 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:
Genetics Home Reference related topics:
porphyria
Drug Information available for:
Deferasirox
U.S. FDA Resources
Further study details as provided by University of Texas Southwestern Medical Center:
Primary Outcome Measures:
- The elimination of all skin blistering. [ Time Frame: Within 6 months of treatment. ] [ Designated as safety issue: No ]
Secondary Outcome Measures:
- 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 ]
| Estimated 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.
Eligibility| Ages Eligible for Study: | 18 Years and older |
| Genders Eligible for Study: | Both |
| 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
Please refer to this study by its ClinicalTrials.gov identifier: NCT00599326
Locations
| 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
| Principal Investigator: | Amit Pandya, M.D. | UT Southwestern Medical Center at Dallas - Department of Dermatology |
More Information
No publications provided by University of Texas Southwestern Medical Center
Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
| Responsible Party: | Amit Pandya, Professor of Dermatology, University of Texas Southwestern Medical Center |
| ClinicalTrials.gov Identifier: | NCT00599326 History of Changes |
| Other Study ID Numbers: | CICL670A US17, IRB File Number 062007-047 |
| Study First Received: | January 10, 2008 |
| Last Updated: | September 18, 2012 |
| Health Authority: | United States: Food and Drug Administration |
Keywords provided by University of Texas Southwestern Medical Center:
|
Exjade PCT Study PCT Study Porphyria Cutanea Tarda |
Additional relevant MeSH terms:
|
Porphyrias Porphyria, Erythropoietic Porphyria Cutanea Tarda Porphyrias, Hepatic Metabolism, Inborn Errors Genetic Diseases, Inborn Skin Diseases, Metabolic Skin Diseases Metabolic Diseases |
Skin Diseases, Genetic Liver Diseases Digestive System Diseases Deferasirox Iron Chelating Agents Chelating Agents Molecular Mechanisms of Pharmacological Action Pharmacologic Actions |
ClinicalTrials.gov processed this record on May 22, 2013