NCT00599326

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
10

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Jan 2008

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

January 1, 2008

Completed
9 days until next milestone

First Submitted

Initial submission to the registry

January 10, 2008

Completed
13 days until next milestone

First Posted

Study publicly available on registry

January 23, 2008

Completed
2.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 1, 2010

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

April 1, 2010

Completed
3.9 years until next milestone

Results Posted

Study results publicly available

February 10, 2014

Completed
Last Updated

February 10, 2014

Status Verified

January 1, 2014

Enrollment Period

2.2 years

First QC Date

January 10, 2008

Results QC Date

August 21, 2013

Last Update Submit

January 9, 2014

Conditions

Porphyria Cutanea Tarda

Keywords

Exjade PCT StudyPCT StudyPorphyria Cutanea Tarda

Outcome Measures

Primary Outcomes (1)

  • 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.

    Within 6 months of treatment.

Secondary Outcomes (1)

  • Number of Participants Showing Decrease in Ferritin and Urinary Porphyrin Level

    6 months

Study Arms (1)

A

EXPERIMENTAL
Drug: Deferasirox

Interventions

DeferasiroxDRUG

250 mg of deferasirox once daily for 6 months

Also known as: Exjade
A

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

UT Southwestern Medical Center at Dallas - Dermatology Clinical Trials

Dallas, Texas, 75390-8802, United States

Location

Related Publications (1)

  • 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.

    PMID: 22911183DERIVED

MeSH Terms

Conditions

Porphyria Cutanea Tarda

Interventions

Deferasirox

Condition Hierarchy (Ancestors)

Porphyrias, HepaticLiver DiseasesDigestive System DiseasesSkin Diseases, GeneticGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesSkin DiseasesSkin and Connective Tissue DiseasesPorphyriasMetabolic DiseasesNutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

BenzoatesAcids, CarbocyclicCarboxylic AcidsOrganic ChemicalsBenzene DerivativesHydrocarbons, AromaticHydrocarbons, CyclicHydrocarbonsTriazolesAzolesHeterocyclic Compounds, 1-RingHeterocyclic Compounds

Limitations and Caveats

10 patients were enrolled, of which 8 were compliant with therapy and had evaluable data. Of the 2 patients dropped from the study, one patient was lost to follow-up after 2 months and another was noncompliant with medication dosing.

Results Point of Contact

Title
Amit Pandya, M.D.
Organization
UT Southwestern Medical Center at Dallas
amit.pandya@utsouthwestern.edu
214-645-8300

Study Officials

  • Amit Pandya, M.D.

    UT Southwestern Medical Center at Dallas - Department of Dermatology

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
Yes

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Professor of Dermatology

Study Record Dates

First Submitted

January 10, 2008

First Posted

January 23, 2008

Study Start

January 1, 2008

Primary Completion

April 1, 2010

Study Completion

April 1, 2010

Last Updated

February 10, 2014

Results First Posted

February 10, 2014

Record last verified: 2014-01

Locations

US(1)