Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda

• Sponsor: University of Texas Southwestern Medical Center (Other)
• Overall Status: Completed
• CT.gov ID: NCT00599326
• Collaborator: Novartis Pharmaceuticals (Industry)
• Enrollment: 10
• Location: 1
• Arm: 1
• Duration (Months): 27
• Patients Per Site Per Month: 0.4

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Porphyria Cutanea Tarda	Drug: Deferasirox	Phase 3

Detailed Description

Phlebotomy is the standard therapy for Porphyria Cutanea Tarda (PCT), but it can be inconvenient and cause anemia in some patients.

Deferasirox is a new class of tridentate iron chelators with high affinity and selectivity for iron. The medication is administered orally, which if effective for PCT would make it a more convenient and possibly more tolerable option for patients.

Study Design

• Study Type: Interventional
• Actual Enrollment: 10 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda
• Study Start Date: Jan 1, 2008
• Actual Primary Completion Date: Apr 1, 2010
• Actual Study Completion Date: Apr 1, 2010

Arms and Interventions

Arm	Intervention/Treatment
Experimental: A	Drug: Deferasirox; 250 mg of deferasirox once daily for 6 months; Other Names: Exjade

Outcome Measures

Primary Outcome Measures

1. Number of Participants Showing Reduction or Elimination of Skin Blistering [Within 6 months of treatment.]

   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.

Secondary Outcome Measures

1. Number of Participants Showing Decrease in Ferritin and Urinary Porphyrin Level [6 months]

   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.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	UT Southwestern Medical Center at Dallas - Dermatology Clinical Trials	Dallas	Texas	United States	75390-8802

Sponsors and Collaborators

• University of Texas Southwestern Medical Center
• Novartis Pharmaceuticals

Investigators

• Principal Investigator: Amit Pandya, M.D., UT Southwestern Medical Center at Dallas - Department of Dermatology

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Amit Pandya, Professor of Dermatology, University of Texas Southwestern Medical Center
• ClinicalTrials.gov Identifier: NCT00599326
• Other Study ID Numbers: CICL670A US17; IRB File Number 062007-047
• First Posted: Jan 23, 2008
• Last Update Posted: Feb 10, 2014
• Last Verified: Jan 1, 2014

• Keywords provided by Amit Pandya, Professor of Dermatology, University of Texas Southwestern Medical Center: Exjade PCT Study; PCT Study; Porphyria Cutanea Tarda
• Additional relevant MeSH terms: Porphyria Cutanea Tarda; Porphyrias, Hepatic; Porphyria, Erythropoietic; Porphyrias; Deferasirox

Study Results

Participant Flow

Arm/Group Title	Deferasirox
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

Baseline Characteristics

Arm/Group Title	Deferasirox
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 Participants	10
Age (Count of Participants)
<=18 years	0 0%
Between 18 and 65 years	10 100%
>=65 years	0 0%
Age (years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [years]	53.7 (5.907622195)
Sex: Female, Male (Count of Participants)
Female	1 10%
Male	9 90%
Region of Enrollment (participants) [Number]
United States	10 100%

Outcome Measures

1. Primary Outcome

Title	Number of Participants Showing Reduction or Elimination of Skin Blistering
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.

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Deferasirox
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.
Measure Participants	8
Elimination	0 0%
Reduction	8 80%

2. Secondary Outcome

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

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Deferasirox
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.
Measure Participants	8
Decrease in ferritin level	8 80%
Decrease in urinary porphyrin level	6 60%

Adverse Events

Arm/Group Title	Deferasirox
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 (%)	# Events
Total	/ (NaN)
Serious Adverse Events
	Deferasirox
	Affected / at Risk (%)	# Events
Total	0/10 (0%)
Other (Not Including Serious) Adverse Events
	Deferasirox
	Affected / at Risk (%)	# Events
Total	1/10 (10%)
Gastrointestinal disorders
Abdominal pain (Mild)	1/10 (10%)	1

Limitations/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.

More Information

Certain Agreements

All Principal Investigators ARE employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.