Sorbent Therapy of the Cutaneous Porphyrias
The investigators demonstrated that cholestyramine is an effective binding agent in vitro for porphyrins. A few isolated case reports of treatment of individuals with a cutaneous porphyria suggest that cholestyramine and colestipol effectively remove porphyrins. Hypothesis: orally administered colestipol will effectively lower erythrocyte porphyrin concentrations in subjects with erythropoietic protoporphyria (EPP).
|Study Design:||Allocation: Non-Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Sorbent Therapy of the Cutaneous Porphyrias|
- Completion of Second Treatment Phase [ Time Frame: At end of 180 days of treatment ] [ Designated as safety issue: Yes ]Subjects receive 1 gm colestipol twice daily for 90 days, and then 2 gm colestipol twice daily for 90 days. Treatment is stopped thereafter.
- Completion of secondary treatment phase (Post Treatment) [ Time Frame: 360 days ] [ Designated as safety issue: Yes ]Same as for the Primary Outcome Measure
|Study Start Date:||May 2011|
|Study Completion Date:||March 2012|
|Primary Completion Date:||March 2012 (Final data collection date for primary outcome measure)|
Experimental: colestipol treatment
1 gram tablets. Phase 1: 1 gram morning and bedtime for 90 days; Phase 2: 2 grams morning and bedtime for 90 days.
Four adults with proven EPP volunteered as subjects for this study. Initial phase (pre-Rx): CBC; liver chemistries including cholesterol; serum iron, TIBC and ferritin; erythrocyte and plasma protoporphyrin concentrations; and completion of questionnaire focused on cutaneous manifestations are completed at weeks 2 and 4. Treatment Phase 1: colestipol tablets, 1 gram in morning and 1 gram at bedtime for 3 months, with repetition of same tests at end of months 2 and 3. Treatment Phase 2: colestipol tablets, 2 grams in morning and 2 grams at bedtime for 3 months, with repetition of same tests at end of months 2 and 3. Post-Treatment - repetition of same tests at end of months 1, 2 and 3.