- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01422915
Sorbent Therapy of the Cutaneous Porphyrias (EPP)
March 14, 2017 updated by: Peter Tishler, Brigham and Women's Hospital
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).
Study Overview
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 Type
Interventional
Enrollment (Actual)
4
Phase
- Phase 2
- Phase 3
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Massachusetts
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Boston, Massachusetts, United States, 02115
- Brigham & Women's Hospital
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
22 years to 60 years (Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
Inclusion Criteria:
- Adult over age 21
- healthy
Exclusion Criteria:
- Intercurrent illness
- pregnancy
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: colestipol treatment
2 grams morning and bedtime for 180 days
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2 grams morning and bedtime for 90 days.
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Photosensitivity, Assessed by Measuring the Number of Minutes of Sun Tolerance
Time Frame: At 60 days of treatment
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Minutes of sun tolerance
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At 60 days of treatment
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Protoporphyrin Concentration in Blood
Time Frame: Samples collected while on treatment (range 93-208 treatment days)
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Samples collected while on treatment (range 93-208 treatment days)
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
May 1, 2011
Primary Completion (Actual)
March 1, 2012
Study Completion (Actual)
March 1, 2012
Study Registration Dates
First Submitted
August 23, 2011
First Submitted That Met QC Criteria
August 24, 2011
First Posted (Estimate)
August 25, 2011
Study Record Updates
Last Update Posted (Actual)
April 25, 2017
Last Update Submitted That Met QC Criteria
March 14, 2017
Last Verified
March 1, 2017
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Digestive System Diseases
- Metabolic Diseases
- Skin Diseases
- Liver Diseases
- Genetic Diseases, Inborn
- Skin Diseases, Genetic
- Porphyrias, Hepatic
- Porphyria, Erythropoietic
- Porphyrias
- Protoporphyria, Erythropoietic
- Molecular Mechanisms of Pharmacological Action
- Antimetabolites
- Hypolipidemic Agents
- Lipid Regulating Agents
- Sequestering Agents
- Colestipol
Other Study ID Numbers
- 2010P002253
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Erythropoietic Protoporphyria
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University of UtahNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); University... and other collaboratorsTerminatedErythropoietic Protoporphyria (EPP) | X Linked Erythropoietic ProtoporphyriaUnited States
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Icahn School of Medicine at Mount SinaiNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); Rare... and other collaboratorsCompletedErythropoietic Protoporphyria | EPP | XLP | X-Linked Protoporphyria | XLPP | X-Linked Dominant Erythropoietic Protoporphyria | XLEPP | XLDPUnited States
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Amy K. Dickey, M.D.Wake Forest University Health Sciences; University of TexasRecruitingErythropoietic Protoporphyria | X-linked ProtoporphyriaUnited States
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