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Sorbent Therapy of the Cutaneous Porphyrias (EPP)

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01422915
First Posted: August 25, 2011
Last Update Posted: April 25, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Peter Tishler, Brigham and Women's Hospital
  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 reduce sun sensitivity and 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: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Sorbent Therapy of the Cutaneous Porphyrias

Resource links provided by NLM:


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

Primary Outcome Measures:
  • 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) ]
    1. erythrocyte protoporphyrin concentration, ug/dl
    2. plasma protoporphyrin concentration, ug/dl


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
2 grams morning and bedtime for 180 days
Drug: Colestipol
2 grams morning and bedtime for 90 days.

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.
  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   22 Years to 60 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Criteria

Inclusion Criteria:

  • Adult over age 21
  • healthy

Exclusion Criteria:

  • Intercurrent illness
  • pregnancy
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01422915


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

Responsible Party: Peter Tishler, physician, Brigham and Women's Hospital
ClinicalTrials.gov Identifier: NCT01422915     History of Changes
Other Study ID Numbers: 2010P002253
First Submitted: August 23, 2011
First Posted: August 25, 2011
Results First Submitted: August 20, 2013
Results First Posted: April 25, 2017
Last Update Posted: April 25, 2017
Last Verified: March 2017

Keywords provided by Peter Tishler, 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
Porphyria, Erythropoietic
Porphyrias, Hepatic
Protoporphyria, Erythropoietic
Metabolic Diseases
Skin Diseases, Genetic
Genetic Diseases, Inborn
Skin Diseases
Liver Diseases
Digestive System Diseases
Colestipol
Hypolipidemic Agents
Antimetabolites
Molecular Mechanisms of Pharmacological Action
Lipid Regulating Agents
Sequestering Agents