Sorbent Therapy of the Cutaneous Porphyrias
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Purpose
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 lower erythrocyte porphyrin concentrations in subjects with erythropoietic protoporphyria (EPP).
| Condition | Intervention | Phase |
|---|---|---|
|
Erythropoietic Protoporphyria |
Drug: Colestipol |
Phase 2 Phase 3 |
| Study Type: | Interventional |
| Study Design: | Allocation: Non-Randomized Endpoint Classification: Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | Sorbent Therapy of the Cutaneous Porphyrias |
- Completion of Second Treatment Phase [ Time Frame: At end of 180 days of treatment ] [ Designated as safety issue: Yes ]Subjects receive 1 gm colestipol twice daily for 90 days, and then 2 gm colestipol twice daily for 90 days. Treatment is stopped thereafter.
- Completion of secondary treatment phase (Post Treatment) [ Time Frame: 360 days ] [ Designated as safety issue: Yes ]Same as for the Primary Outcome Measure
| Enrollment: | 4 |
| Study Start Date: | May 2011 |
| Study Completion Date: | March 2012 |
| Primary Completion Date: | March 2012 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
Experimental: colestipol treatment
|
Drug: Colestipol
1 gram tablets. Phase 1: 1 gram morning and bedtime for 90 days; Phase 2: 2 grams morning and bedtime for 90 days.
|
Detailed Description:
Four adults with proven EPP volunteered as subjects for this study. Initial phase (pre-Rx): CBC; liver chemistries including cholesterol; serum iron, TIBC and ferritin; erythrocyte and plasma protoporphyrin concentrations; and completion of questionnaire focused on cutaneous manifestations are completed at weeks 2 and 4. Treatment Phase 1: colestipol tablets, 1 gram in morning and 1 gram at bedtime for 3 months, with repetition of same tests at end of months 2 and 3. Treatment Phase 2: colestipol tablets, 2 grams in morning and 2 grams at bedtime for 3 months, with repetition of same tests at end of months 2 and 3. Post-Treatment - repetition of same tests at end of months 1, 2 and 3.
Eligibility| Ages Eligible for Study: | 22 Years to 60 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | Yes |
Inclusion Criteria:
- Adult over age 21
- healthy
Exclusion Criteria:
- Intercurrent illness
- pregnancy
Contacts and Locations More Information
No publications provided
| Responsible Party: | Peter Tishler, physician, Brigham and Women's Hospital |
| ClinicalTrials.gov Identifier: | NCT01422915 History of Changes |
| Other Study ID Numbers: | 2010P002253 |
| Study First Received: | August 23, 2011 |
| Last Updated: | April 29, 2013 |
| Health Authority: | United States: Institutional Review Board |
Keywords provided by 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:
|
Porphyrias Porphyria, Erythropoietic Protoporphyria, Erythropoietic Metabolism, Inborn Errors Genetic Diseases, Inborn Skin Diseases, Metabolic Skin Diseases Metabolic Diseases Skin Diseases, Genetic Porphyrias, Hepatic |
Liver Diseases Digestive System Diseases Colestipol Hypolipidemic Agents Antimetabolites Molecular Mechanisms of Pharmacological Action Pharmacologic Actions Lipid Regulating Agents Therapeutic Uses |
ClinicalTrials.gov processed this record on May 16, 2013