Sorbent Therapy of the Cutaneous Porphyrias

Erythropoietic Protoporphyria Clinical Trial

Official title: Sorbent Therapy of the Cutaneous Porphyrias

Status Completed

Clinical Trial Summary

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).

Clinical Trial Description

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. ;

Study Design

Allocation: Non-Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Erythropoietic Protoporphyria
• Porphyrias

• NCT number: NCT01422915
• Study type: Interventional
• Source: Brigham and Women's Hospital
• Status: Completed
• Phase: Phase 2/Phase 3
• Start date: May 2011
• Completion date: March 2012