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

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Erythropoietic Protoporphyria Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Genetic Diseases, Inborn Liver Diseases Metabolic Diseases Nutritional and Metabolic Diseases Porphyrias Porphyrias, Hepatic Protoporphyria, Erythropoietic Skin Diseases Skin Diseases, Genetic Skin and Connective Tissue Diseases

Age range

22 year–60 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2 / Phase 3

Primary location

Brigham & Women's Hospital

Boston, Massachusetts, 02115, United States

About this study

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.

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

Adult over age 21
healthy

Exclusion criteria

Intercurrent illness
pregnancy

Treatment and study plan

Colestipol

Drug

2 grams morning and bedtime for 90 days.

Primary outcomes

Photosensitivity, Assessed by Measuring the Number of Minutes of Sun Tolerance

Time frame: At 60 days of treatment

Minutes of sun tolerance
Protoporphyrin Concentration in Blood

Time frame: Samples collected while on treatment (range 93-208 treatment days)
- erythrocyte protoporphyrin concentration, ug/dl
- plasma protoporphyrin concentration, ug/dl

Sponsors and collaborators

Lead sponsor

Brigham and Women's Hospital

Other

Registry information

Acronym: EPP

Important dates

Study start: 2011
Primary completion: 2012
Study completion: 2012
First posted: Aug 25, 2011
Registry last updated: Apr 25, 2017

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed