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Trial record 4 of 28 for:    "porphyria" | ( Map: United States )

Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda

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.
ClinicalTrials.gov Identifier: NCT00599326
Recruitment Status : Completed
First Posted : January 23, 2008
Results First Posted : February 10, 2014
Last Update Posted : February 10, 2014
Sponsor:
Collaborator:
Novartis Pharmaceuticals
Information provided by (Responsible Party):
Amit Pandya, University of Texas Southwestern Medical Center

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Porphyria Cutanea Tarda
Intervention Drug: Deferasirox
Enrollment 10
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Deferasirox
Hide Arm/Group Description 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.
Period Title: Overall Study
Started 10
Completed 8
Not Completed 2
Reason Not Completed
Lost to Follow-up             1
Noncompliant             1
Arm/Group Title Deferasirox
Hide Arm/Group Description 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.
Overall Number of Baseline Participants 10
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 10 participants
<=18 years
0
   0.0%
Between 18 and 65 years
10
 100.0%
>=65 years
0
   0.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 10 participants
53.7  (5.907622195)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 10 participants
Female
1
  10.0%
Male
9
  90.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 10 participants
10
1.Primary Outcome
Title Number of Participants Showing Reduction or Elimination of Skin Blistering
Hide Description 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.
Time Frame Within 6 months of treatment.
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Deferasirox
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 8
Measure Type: Number
Unit of Measure: participants
Elimination 0
Reduction 8
2.Secondary Outcome
Title Number of Participants Showing Decrease in Ferritin and Urinary Porphyrin Level
Hide Description Patients with PCT usually have normal or elevated serum iron and ferritin levels as well as increased iron absorption. Phlebotomy is conducted to analyzes the ferritin levels. Urine collection is performed and samples of the urine are analyzed for porphyrin levels.
Time Frame 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Deferasirox
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 8
Measure Type: Number
Unit of Measure: participants
Decrease in ferritin level 8
Decrease in urinary porphyrin level 6
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Deferasirox
Hide Arm/Group Description 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.
All-Cause Mortality
Deferasirox
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Deferasirox
Affected / at Risk (%) # Events
Total   0/10 (0.00%)    
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Deferasirox
Affected / at Risk (%) # Events
Total   1/10 (10.00%)    
Gastrointestinal disorders   
Abdominal pain (Mild) * 1 [1]  1/10 (10.00%)  1
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, CTCAE 4.0
[1]
mild abdominal discomfort for 2 days in 1 patient, which resolved spontaneously.
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.
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
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