Phase 3 N=10 Treatment

Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda

Porphyria Cutanea Tarda

Bottom Line

View on ClinicalTrials.gov: NCT00599326 ↗

Enrolled (actual)

Serious AEs

0.0%

Results posted

Feb 2014

Primary outcome: Primary: Number of Participants Showing Reduction or Elimination of Skin Blistering — 0; 8 participants

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: Deferasirox (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: University of Texas Southwestern Medical Center
Primary completion: Apr 2010

Outcome Measures

Outcome	Result	p-value
PRIMARY Number of Participants Showing Reduction or Elimination of Skin Blistering	0; 8	—
SECONDARY Number of Participants Showing Decrease in Ferritin and Urinary Porphyrin Level	8; 6	—

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.

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.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00599326). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.