Effect of Oral Iron Therapy on Erythrocyte Protoporphyrin Levels in the Erythropoietic Protoporphyrias
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 16
- 试验地点
- 6
- 主要终点
- Change in Erythrocyte Protoporphyrin Levels
研究概览
简要总结
In the medical literature there are conflicting reports on whether iron improves symptoms in patients with EPP and XLP. Giving iron to people who are iron deficient is thought to improve EPP symptoms. However, this has never been systematically tested. Therefore, the purpose of this study is to determine the effect of oral iron for EPP and XLP patients.
详细描述
This is a clinical trial, which means its purpose is to study an intervention or treatment. In this study all patients with EPP or XLP will be given a standard dose of iron pills and monitored for one year. There is currently no effective Food and Drug Administration (FDA) approved treatment for EPP or XLP in the US. Giving iron to patients with low ferritin (a measure of body iron stores) levels may help improve their EPP symptoms by decreasing erythrocyte protoporphyrin levels.
Patients will be asked come to the study site once every three months over the course of a year for a total of five visits. At these visits the study doctors will check in with participants and some blood and urine samples will be taken. Participants will not be charged for any of the lab tests that are being done for part of this study alone. In between these study visits there will be phone call to check in and see how participants are doing. All patients in this study will receive iron pills at no cost to them.
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
- •Enrollment in the Longitudinal Study of the Porphyrias
- •Male or female age ≥18 years
- •History of nonblistering cutaneous photosensitivity
- •Biochemical findings - A marked increase in erythrocyte protoporphyrin (total erythrocyte protoporphyrin >400 ug/dL) with a predominance of metal-free protoporphyrin
- •Serum ferritin ≤30 ng/mL at baseline
- •Able to tolerate oral iron
排除标准
- •History of liver or bone marrow transplant or clinically significant liver dysfunction as determined by the investigator
- •Known or suspected allergy to oral iron based on patient report
- •Clinical evidence of active and ongoing GI bleeding
- •Use of any other clinical or experimental therapy in the past 3 months
- •Individuals with elevations of porphyrins in plasma or erythrocytes due to other diseases (i.e. secondary porphyrinemia) such as liver and bone marrow diseases
- •Patients with any clinically significant comorbid conditions, which in the opinion of the investigator, precludes participation
研究组 & 干预措施
Oral Iron
standard dose of iron pills
干预措施: Oral Iron (Drug)
结局指标
主要结局
Change in Erythrocyte Protoporphyrin Levels
时间窗: Baseline and at 12 months
Relative difference in erythrocyte protoporphyrin levels between baseline and 12 months after treatment
次要结局
- Mean Change in EPP-specific Quality of Life Questionnaire(Baseline and 12 months)
研究者
Manisha C Balwani
Associate Professor
Icahn School of Medicine at Mount Sinai