Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 10
- 试验地点
- 1
- 主要终点
- Number of Participants Showing Reduction or Elimination of Skin Blistering
研究概览
简要总结
To determine the efficacy and tolerability of deferasirox in the treatment of Porphyria Cutanea Tarda.
Primary objective - the elimination of all blistering within 6 months of treatment.
Secondary objective - decrease in total body iron levels.
详细描述
Phlebotomy is the standard therapy for Porphyria Cutanea Tarda (PCT), but it can be inconvenient and cause anemia in some patients.
Deferasirox is a new class of tridentate iron chelators with high affinity and selectivity for iron. The medication is administered orally, which if effective for PCT would make it a more convenient and possibly more tolerable option for patients.
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •diagnosis of Porphyria Cutanea Tarda based on clinical exam and 24-hour urine porphyrin levels
- •have Porphyria Cutanea Tarda for at least 3 months prior to enrollment with active blistering (3 blisters or erosions per month)
- •women of childbearing potential must use an effective method of contraception during the study, however this cannot include hormonal contraception (oral contraceptives, hormone patches, Depo-Provera injections, NUVA Ring, etc.)
- •treatment naive patients or patients unresponsive or intolerant of phlebotomy
- •Ferritin level is greater than or equal to 25ng/mL
排除标准
- •patients with serum creatinine above the upper limit of normal
- •patients receiving phlebotomy who are controlled on this therapy
- •pregnant or breast feeding females
- •patients with liver transaminases more than 5 times the upper limit of normal
- •patients with a history of hypersensitivity to deferasirox
- •patients with a history of pre-existing renal condition, or receiving medication that depresses renal function
- •patients on other chelators
- •history of non-compliance to medical regimens.
研究组 & 干预措施
A
干预措施: Deferasirox (Drug)
结局指标
主要结局
Number of Participants Showing Reduction or Elimination of Skin Blistering
时间窗: Within 6 months of treatment.
The present trial was undertaken to determine if oral deferasirox could be useful in the treatment of PCT. Monthly clinic visits with a physical examination was conducted to assess the skin for blisters.
次要结局
- Number of Participants Showing Decrease in Ferritin and Urinary Porphyrin Level(6 months)
研究者
Amit Pandya
Professor of Dermatology
University of Texas Southwestern Medical Center