Sorbent Therapy of the Cutaneous Porphyrias

Erythropoietic Protoporphyria Clinical Trial

Official title:

Sorbent Therapy of the Cutaneous Porphyrias

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01422915
• Other study ID #: 2010P002253
• Status: Completed
• Phase: Phase 2/Phase 3
• First received: August 23, 2011
• Last updated: April 29, 2013
• Start date: May 2011
• Est. completion date: March 2012

Study information

• Verified date: April 2013
• Source: Brigham and Women's Hospital
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Institutional Review Board
• Study type: Interventional

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).

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 4
• Est. completion date: March 2012
• Est. primary completion date: March 2012
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: Both
• Age group: 22 Years to 60 Years

Eligibility

Inclusion Criteria:

• Adult over age 21

• healthy

Exclusion Criteria:

• Intercurrent illness

• pregnancy

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

Intervention

Drug:
• Colestipol
1 gram tablets. Phase 1: 1 gram morning and bedtime for 90 days; Phase 2: 2 grams morning and bedtime for 90 days.

Locations

Country	Name	City	State
United States	Brigham & Women's Hospital	Boston	Massachusetts

Sponsors (1)

Lead Sponsor	Collaborator
Brigham and Women's Hospital

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Completion of Second Treatment Phase	Subjects receive 1 gm colestipol twice daily for 90 days, and then 2 gm colestipol twice daily for 90 days. Treatment is stopped thereafter.	At end of 180 days of treatment	Yes
Secondary	Completion of secondary treatment phase (Post Treatment)	Same as for the Primary Outcome Measure	360 days	Yes