Sorbent Therapy of the Cutaneous Porphyrias

Phase 2

Completed

• Conditions: Erythropoietic Protoporphyria
• Interventions: Drug: Colestipol

• Registration Number: NCT01422915
• Lead Sponsor: Brigham and Women's Hospital

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

Detailed 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.

Study Timeline

• Study Start: 2011-05
• Study First Submitted: 2011-08-23
• Study First Submitted QC: 2011-08-24
• Study First Posted: 2011-08-25
• Primary Completion: 2012-03
• Study Completion: 2012-03
• Results First Submitted: 2013-08-20
• Status Verified: 2017-03
• Results First Submitted QC: 2017-03-14
• Last Update Submitted: 2017-03-14
• Results First Posted: 2017-04-25
• Last Update Posted: 2017-04-25

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 4

Inclusion Criteria

• Adult over age 21
• healthy

Exclusion Criteria

• Intercurrent illness
• pregnancy

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
colestipol treatment	Colestipol	2 grams morning and bedtime for 180 days

Primary Outcome Measures

Name	Time	Method
Photosensitivity, Assessed by Measuring the Number of Minutes of Sun Tolerance	At 60 days of treatment	Minutes of sun tolerance
Protoporphyrin Concentration in Blood	Samples collected while on treatment (range 93-208 treatment days)	1. erythrocyte protoporphyrin concentration, ug/dl; 2. plasma protoporphyrin concentration, ug/dl

Trial Locations

Locations (1)

Brigham & Women's Hospital; 🇺🇸 Boston, Massachusetts, United States