Sorbent Therapy of the Cutaneous Porphyrias

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Peter Tishler, Brigham and Women's Hospital
ClinicalTrials.gov Identifier:
NCT01422915
First received: August 23, 2011
Last updated: April 29, 2013
Last verified: April 2013
  Purpose

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).


Condition Intervention Phase
Erythropoietic Protoporphyria
Drug: Colestipol
Phase 2
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Sorbent Therapy of the Cutaneous Porphyrias

Resource links provided by NLM:


Further study details as provided by Brigham and Women's Hospital:

Primary Outcome Measures:
  • Completion of Second Treatment Phase [ Time Frame: At end of 180 days of treatment ] [ Designated as safety issue: Yes ]
    Subjects receive 1 gm colestipol twice daily for 90 days, and then 2 gm colestipol twice daily for 90 days. Treatment is stopped thereafter.


Secondary Outcome Measures:
  • Completion of secondary treatment phase (Post Treatment) [ Time Frame: 360 days ] [ Designated as safety issue: Yes ]
    Same as for the Primary Outcome Measure


Enrollment: 4
Study Start Date: May 2011
Study Completion Date: March 2012
Primary Completion Date: March 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: colestipol treatment
  1. gram morning and bedtime for 90 days; then
  2. grams morning and bedtime for 90 days
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.

Detailed 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.

  Eligibility

Ages Eligible for Study:   22 Years to 60 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   Yes
Criteria

Inclusion Criteria:

  • Adult over age 21
  • healthy

Exclusion Criteria:

  • Intercurrent illness
  • pregnancy
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01422915

Locations
United States, Massachusetts
Brigham & Women's Hospital
Boston, Massachusetts, United States, 02115
Sponsors and Collaborators
Brigham and Women's Hospital
  More Information

No publications provided

Responsible Party: Peter Tishler, physician, Brigham and Women's Hospital
ClinicalTrials.gov Identifier: NCT01422915     History of Changes
Other Study ID Numbers: 2010P002253
Study First Received: August 23, 2011
Last Updated: April 29, 2013
Health Authority: United States: Institutional Review Board

Keywords provided by Brigham and Women's Hospital:
Sorbent
Resin
EPP
Cutaneous porphyria
Adult
over age 21
without intercurrent illness
not pregnant
willing to participate

Additional relevant MeSH terms:
Porphyrias
Porphyrias, Hepatic
Protoporphyria, Erythropoietic
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Skin Diseases, Metabolic
Skin Diseases
Metabolic Diseases
Liver Diseases
Digestive System Diseases
Skin Diseases, Genetic
Colestipol
Hypolipidemic Agents
Antimetabolites
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Lipid Regulating Agents
Therapeutic Uses
Sequestering Agents

ClinicalTrials.gov processed this record on September 16, 2014