Sorbent Therapy of the Cutaneous Porphyrias
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 4
- 试验地点
- 1
- 主要终点
- Photosensitivity, Assessed by Measuring the Number of Minutes of Sun Tolerance
研究概览
简要总结
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).
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 22 Years 至 60 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Adult over age 21
排除标准
- •Intercurrent illness
- •pregnancy
研究组 & 干预措施
colestipol treatment
2 grams morning and bedtime for 180 days
干预措施: Colestipol (Drug)
结局指标
主要结局
Photosensitivity, Assessed by Measuring the Number of Minutes of Sun Tolerance
时间窗: At 60 days of treatment
Minutes of sun tolerance
Protoporphyrin Concentration in Blood
时间窗: Samples collected while on treatment (range 93-208 treatment days)
1. erythrocyte protoporphyrin concentration, ug/dl 2. plasma protoporphyrin concentration, ug/dl
次要结局
未报告次要终点
研究者
Peter Tishler
physician
Brigham and Women's Hospital