Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda

This study has been completed.
Sponsor:
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: January 9, 2014
Last verified: January 2014
Results First Received: August 21, 2013  
Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: 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  
Number of Participants  
[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


No publications provided by University of Texas Southwestern Medical Center

Publications automatically indexed to this study:

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
Study First Received: January 10, 2008
Results First Received: August 21, 2013
Last Updated: January 9, 2014
Health Authority: United States: Food and Drug Administration