Sorbent Therapy of the Cutaneous Porphyrias (EPP)
Primary Purpose
Erythropoietic Protoporphyria
Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Colestipol
Sponsored by
About this trial
This is an interventional treatment trial for Erythropoietic Protoporphyria focused on measuring Sorbent, Resin, EPP, Cutaneous porphyria, Adult, over age 21, without intercurrent illness, not pregnant, willing to participate
Eligibility Criteria
Inclusion Criteria:
- Adult over age 21
- healthy
Exclusion Criteria:
- Intercurrent illness
- pregnancy
Sites / Locations
- Brigham & Women's Hospital
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
colestipol treatment
Arm Description
2 grams morning and bedtime for 180 days
Outcomes
Primary Outcome Measures
Photosensitivity, Assessed by Measuring the Number of Minutes of Sun Tolerance
Minutes of sun tolerance
Protoporphyrin Concentration in Blood
erythrocyte protoporphyrin concentration, ug/dl
plasma protoporphyrin concentration, ug/dl
Secondary Outcome Measures
Full Information
NCT ID
NCT01422915
First Posted
August 23, 2011
Last Updated
March 14, 2017
Sponsor
Brigham and Women's Hospital
1. Study Identification
Unique Protocol Identification Number
NCT01422915
Brief Title
Sorbent Therapy of the Cutaneous Porphyrias
Acronym
EPP
Official Title
Sorbent Therapy of the Cutaneous Porphyrias
Study Type
Interventional
2. Study Status
Record Verification Date
March 2017
Overall Recruitment Status
Completed
Study Start Date
May 2011 (undefined)
Primary Completion Date
March 2012 (Actual)
Study Completion Date
March 2012 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Brigham and Women's Hospital
4. Oversight
Data Monitoring Committee
No
5. Study Description
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).
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Erythropoietic Protoporphyria
Keywords
Sorbent, Resin, EPP, Cutaneous porphyria, Adult, over age 21, without intercurrent illness, not pregnant, willing to participate
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
4 (Actual)
8. Arms, Groups, and Interventions
Arm Title
colestipol treatment
Arm Type
Experimental
Arm Description
2 grams morning and bedtime for 180 days
Intervention Type
Drug
Intervention Name(s)
Colestipol
Intervention Description
2 grams morning and bedtime for 90 days.
Primary Outcome Measure Information:
Title
Photosensitivity, Assessed by Measuring the Number of Minutes of Sun Tolerance
Description
Minutes of sun tolerance
Time Frame
At 60 days of treatment
Title
Protoporphyrin Concentration in Blood
Description
erythrocyte protoporphyrin concentration, ug/dl
plasma protoporphyrin concentration, ug/dl
Time Frame
Samples collected while on treatment (range 93-208 treatment days)
10. Eligibility
Sex
All
Minimum Age & Unit of Time
22 Years
Maximum Age & Unit of Time
60 Years
Accepts Healthy Volunteers
Accepts Healthy Volunteers
Eligibility Criteria
Inclusion Criteria:
Adult over age 21
healthy
Exclusion Criteria:
Intercurrent illness
pregnancy
Facility Information:
Facility Name
Brigham & Women's Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Sorbent Therapy of the Cutaneous Porphyrias
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