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Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT00599326
First Posted: January 23, 2008
Last Update Posted: February 10, 2014
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Collaborator:
Novartis Pharmaceuticals
Information provided by (Responsible Party):
Amit Pandya, University of Texas Southwestern Medical Center
Results First Submitted: August 21, 2013  
Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition: Porphyria Cutanea Tarda
Intervention: Drug: Deferasirox

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Deferasirox Deferasirox : 250 mg of deferasirox once daily for 6 months with an increase to 500 mg/d after 2 months if new blisters continued to develop.

Participant Flow:   Overall Study
    Deferasirox
STARTED   10 
COMPLETED   8 
NOT COMPLETED   2 
Lost to Follow-up                1 
Noncompliant                1 



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Deferasirox Deferasirox : 250 mg of deferasirox once daily for 6 months with an increase to 500 mg/d after 2 months if new blisters continued to develop.

Baseline Measures
   Deferasirox 
Overall Participants Analyzed 
[Units: Participants]
 10 
Age 
[Units: Participants]
 
<=18 years   0 
Between 18 and 65 years   10 
>=65 years   0 
Age 
[Units: Years]
Mean (Standard Deviation)
 53.7  (5.907622195) 
Gender 
[Units: Participants]
 
Female   1 
Male   9 
Region of Enrollment 
[Units: Participants]
 
United States   10 


  Outcome Measures
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1.  Primary:   Number of Participants Showing Reduction or Elimination of Skin Blistering   [ Time Frame: Within 6 months of treatment. ]

2.  Secondary:   Number of Participants Showing Decrease in Ferritin and Urinary Porphyrin Level   [ Time Frame: 6 months ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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.


  More Information
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Certain Agreements:  
All Principal Investigators ARE employed by the organization sponsoring the study.


Results Point of Contact:  
Name/Title: Amit Pandya, M.D.
Organization: UT Southwestern Medical Center at Dallas
phone: 214-645-8300
e-mail: amit.pandya@utsouthwestern.edu


Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Responsible Party: Amit Pandya, University of Texas Southwestern Medical Center
ClinicalTrials.gov Identifier: NCT00599326     History of Changes
Other Study ID Numbers: CICL670A US17
IRB File Number 062007-047
First Submitted: January 10, 2008
First Posted: January 23, 2008
Results First Submitted: August 21, 2013
Results First Posted: February 10, 2014
Last Update Posted: February 10, 2014