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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
Brigham and Women's Hospital
About
Eligibility
Locations
Arms
Outcomes
Full info

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

22 Years - 60 Years (Adult)All SexesAccepts Healthy Volunteers

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

First Posted
August 23, 2011
Last Updated
March 14, 2017
Sponsor
Brigham and Women's Hospital
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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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