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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).
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| colestipol treatment | Experimental | 2 grams morning and bedtime for 180 days |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Colestipol | Drug | 2 grams morning and bedtime for 90 days. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Photosensitivity, Assessed by Measuring the Number of Minutes of Sun Tolerance | Minutes of sun tolerance | At 60 days of treatment |
| Protoporphyrin Concentration in Blood |
| Samples collected while on treatment (range 93-208 treatment days) |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Brigham & Women's Hospital | Boston | Massachusetts | 02115 | United States |
All 4 subjects were enrolled in the study. Initially, baseline blood protoporphyrin concentrations and answers to sun sensitivity questionnaire were obtained.
Dates: February and March 2011.Location types: suburbs of Massachusetts (3 subjects) and in New Jersey (1).
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| ID | Title | Description |
|---|---|---|
| FG000 | Colestipol Treatment | Colestipol 2 grams twice daily for 5-6 months. Completion of sun sensitivity questionnaire and blood protoporphyrin concentrations. were obtained monthly. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Colestipol Therapy of Protoporphyria | 4 subjects were assigned, all between 18-65 years of age. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Photosensitivity, Assessed by Measuring the Number of Minutes of Sun Tolerance | Minutes of sun tolerance | All of the original 4 subjects had bona fide erythropoietic protoporphyria (EPP). One subject was removed during Perdiod 1. The data from the same 3 subjects who completed periods 1 and 2 were analyzed for the study results. Data collected was not tractable for statistical analysis given the range of results | Posted | Mean | Standard Deviation | minutes | At 60 days of treatment |
|
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | 1st Period - Colestipol Optimal Dosage |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abnormal liver enzymes | Hepatobiliary disorders | Systematic Assessment | Abnormal liver enzymes on one occasion. |
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1 subject was removed during Period 1 because of abnormal liver chemistries.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Peter V. Tishler | BrighamHospital | 617-525-2123 | repvt@channing.harvard.edu |
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| ID | Term |
|---|---|
| D046351 | Protoporphyria, Erythropoietic |
| ID | Term |
|---|---|
| D017094 | Porphyrias, Hepatic |
| D008107 | Liver Diseases |
| D004066 | Digestive System Diseases |
| D012873 | Skin Diseases, Genetic |
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| ID | Term |
|---|---|
| D003084 | Colestipol |
| ID | Term |
|---|---|
| D011073 | Polyamines |
| D000588 | Amines |
| D009930 | Organic Chemicals |
| D011108 | Polymers |
| D046911 |
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| Participants |
|
| Age, Continuous | Mean | Standard Deviation | years |
|
| Age, Customized | For baseline in Period 1 and Period 2: ~monthly completion of sun sensitivity questionnaire; donate whole blood for RBC and plasma protoporphyrin | Number | participants |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| Units | Counts |
|---|
| Participants |
|
|
| Primary | Protoporphyrin Concentration in Blood |
| Posted | Mean | Standard Deviation | ug/dl | Samples collected while on treatment (range 93-208 treatment days) |
|
|
|
| 1 |
| 4 |
| 0 |
| 4 |
|
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| D030342 |
| Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D012871 | Skin Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D011164 | Porphyrias |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| Macromolecular Substances |
| D001697 | Biomedical and Dental Materials |
| D008420 | Manufactured Materials |
| D013676 | Technology, Industry, and Agriculture |