Clinical trial NCT00599326: 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
Overall status: COMPLETED
Phase: PHASE3
Conditions: Porphyria Cutanea Tarda
Keywords: Exjade PCT Study
PCT Study
Porphyria Cutanea Tarda
Lead sponsor: University of Texas Southwestern Medical Center
Sponsor class: OTHER
Collaborators: name: Novartis Pharmaceuticals
First posted: 2008-01-23
Enrollment: 10
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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.

Primary outcomes:
measure: Number of Participants Showing Reduction or Elimination of Skin Blistering
timeFrame: Within 6 months of treatment.
Secondary outcomes:
measure: Number of Participants Showing Decrease in Ferritin and Urinary Porphyrin Level
timeFrame: 6 months
Other outcomes:
Locations:
city: Dallas
country: United States
facility: UT Southwestern Medical Center at Dallas - Dermatology Clinical Trials
state: Texas
Study officials:
affiliation: UT Southwestern Medical Center at Dallas - Department of Dermatology
name: Amit Pandya, M.D.
role: PRINCIPAL_INVESTIGATOR
References:
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.