Sorbent Therapy of the Cutaneous Porphyrias (EPP)

Mass General Brigham

Status and phase: Completed; Phase 3; Phase 2

Conditions: Erythropoietic Protoporphyria

Treatments: Drug: Colestipol

Study type: Interventional

Funder types: Other

Identifiers

NCT01422915

2010P002253

Details and patient eligibility

About

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 reduce sun sensitivity and lower erythrocyte porphyrin concentrations in subjects with erythropoietic protoporphyria (EPP).

Full description

Four adults with proven EPP volunteered as subjects for this study. First period: Subjects received 1 gm colestipol twice daily for ~45 days, then 2 gm twice daily for ~45 days. Labs included CBC; liver chemistries including cholesterol; serum iron, TIBC and ferritin; erythrocyte and plasma protoporphyrin concentrations; and completion of sun exposure questionnaire focused on cutaneous manifestations every 2-4 weeks. Second period: Subjects received colestipol tablets, 2 grams twice daily, completing the sun exposure questionnaire and protoporphyrin determinations ~monthly for 5-6 months.

Enrollment: 4 patients

Sex: All

Ages: 22 to 60 years old

Volunteers: Accepts Healthy Volunteers

Inclusion criteria

• Adult over age 21
• healthy

Exclusion criteria

• Intercurrent illness
• pregnancy

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

---

4 participants in 1 patient group

colestipol treatment

Experimental group

Description:

2 grams morning and bedtime for 180 days

Treatment:

Drug: Colestipol