Trial Outcomes & Findings for Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda (NCT NCT00599326)

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

10 participants

Primary outcome timeframe

Within 6 months of treatment.

Results posted on

2014-02-10

Participant Flow

Participant milestones

Participant milestones
Measure	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.
Overall Study STARTED	10
Overall Study COMPLETED	8
Overall Study NOT COMPLETED	2

Reasons for withdrawal

Reasons for withdrawal
Measure	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.
Overall Study Lost to Follow-up	1
Overall Study Noncompliant	1

Baseline Characteristics

Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Deferasirox n=10 Participants 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.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	10 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	53.7 years STANDARD_DEVIATION 5.907622195 • n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants
Sex: Female, Male Male	9 Participants n=5 Participants
Region of Enrollment United States	10 participants n=5 Participants

PRIMARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Deferasirox n=8 Participants 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.
Number of Participants Showing Reduction or Elimination of Skin Blistering Elimination	0 participants
Number of Participants Showing Reduction or Elimination of Skin Blistering Reduction	8 participants

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Deferasirox n=8 Participants 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.
Number of Participants Showing Decrease in Ferritin and Urinary Porphyrin Level Decrease in ferritin level	8 participants
Number of Participants Showing Decrease in Ferritin and Urinary Porphyrin Level Decrease in urinary porphyrin level	6 participants

Adverse Events

Deferasirox

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Deferasirox n=10 participants at risk 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.
Gastrointestinal disorders Abdominal pain (Mild)	10.0% 1/10 • Number of events 1

Additional Information

Amit Pandya, M.D.

UT Southwestern Medical Center at Dallas

Phone: 214-645-8300

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place