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Sorbent Therapy of the Cutaneous Porphyrias (EPP)

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ClinicalTrials.gov Identifier: NCT01422915
Recruitment Status : Completed
First Posted : August 25, 2011
Results First Posted : April 25, 2017
Last Update Posted : April 25, 2017
Sponsor:
Information provided by (Responsible Party):
Peter Tishler, Brigham and Women's Hospital

Study Description
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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).

Condition or disease Intervention/treatment Phase
Erythropoietic Protoporphyria Drug: Colestipol Phase 2 Phase 3

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 Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 4 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Sorbent Therapy of the Cutaneous Porphyrias
Study Start Date : May 2011
Actual Primary Completion Date : March 2012
Actual Study Completion Date : March 2012

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Porphyria

Arms and Interventions
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Arm Intervention/treatment
Experimental: colestipol treatment
2 grams morning and bedtime for 180 days
 Drug: Colestipol
2 grams morning and bedtime for 90 days.


Outcome Measures
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Primary Outcome Measures :
  1. Photosensitivity, Assessed by Measuring the Number of Minutes of Sun Tolerance [ Time Frame: At 60 days of treatment ]
    Minutes of sun tolerance

  2. 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


Eligibility Criteria
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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
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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
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United States, Massachusetts
Brigham & Women's Hospital
Boston, Massachusetts, United States, 02115
Sponsors and Collaborators
Brigham and Women's Hospital
More Information
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Responsible Party: Peter Tishler, physician, Brigham and Women's Hospital
ClinicalTrials.gov Identifier: NCT01422915    
Other Study ID Numbers: 2010P002253
First Posted: August 25, 2011    Key Record Dates
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:
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Protoporphyria, Erythropoietic
Porphyria, Erythropoietic
Porphyrias
Metabolic Diseases
Skin Diseases, Genetic
Genetic Diseases, Inborn
Skin Diseases
Porphyrias, Hepatic
 Liver Diseases
Digestive System Diseases
Colestipol
Hypolipidemic Agents
Antimetabolites
Molecular Mechanisms of Pharmacological Action
Lipid Regulating Agents
Sequestering Agents