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7206: Therapeutic Studies in Porphyria Cutanea Tarda


Porphyria Cutanea Tarda (PCT) is the most common porphyria that is generally diagnosed in adulthood. It is also the most responsive type of porphyria to treat. There are two current treatments for PCT. The first and more common treatment is repeated phlebotomies (removing blood, but that also includes the potential disadvantages of discomfort, inconvenience and expense. The second options is a low-dose treatment with medications that are normally used to treat malaria-- hydroxychloroquine or chloroquine. This treatment maybe more convenient and cost-effective but it is not option prescribed as primary treatment. This study compares these two treatments.

The research questions are:

  1. Is a low-dose regimen of hydroxychloroquine effective as phlebotomies for PCT treatment?
  2. Are people treated with low-dose hydroxychloroquine more likely to have relapses of their PCT?

About this Study

This study compares two treatments for PCT. The first phase of the study is when patients get the treatment, either phlebotomies or hydroxychloroquine. During this phase study visits are every 2-4 weeks. After the treatment phase when participants have reached remission participants will meet with their doctor every 3-6 months, for 3 years to see if any symptoms return. The procedures done at these visits include physical exams, standard lab work such as checking iron levels, and lab work to look for porphyrin levels in the blood and urine.

A total of 100 patients with well-documented PCT will be enrolled in this study.

For each visit, you will be asked to:

  • Keep a log of your study medication if you are treated with hydroxychloroquine
  • Fill out a questionnaire about your PCT symptoms
  • Give a blood sample
    • 1 to 2 teaspoons if you are treated by hydroxychloroquine
    • 1 unit (450 ml, which is approximately 1 pint) of blood at regular intervals if you are treated with phlebotomy.
  • Give a urine sample
  • Have your local doctor’s office send us medical records and lab results

Targeted Enrollment

To be eligible to participate, you must:

  • Be at least 18 years old
  • Have well-documented PCT
  • Be able to provide consent to participate after you are informed about what the study includes
  • Be willing to avoid becoming pregnant during treatment; if you are a woman capable of becoming pregnant, you must use an effective contraceptive method

You are not eligible to participate if you have:

  • Your skin problem is not due to PCT, or is due to another type of porphyria
  • You are pregnant or if you plan to become pregnant during treatment
  • You have had prior treatment by phlebotomy, hydroxychloroquine or chloroquine within one month of starting this study. The exception to this is if you have specific medical records and lab results from before and during the treatment
  • You are unwilling or unable to comply with study procedures and visit schedule
  • You have participated in this study before