Quercetin Chemoprevention for Squamous Cell Carcinoma in Patients With Fanconi Anemia
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 48
- 试验地点
- 1
- 主要终点
- Reduction of buccal micronuclei
研究概览
简要总结
Fanconi anemia (FA) is an autosomal recessive disease characterized by progressive bone marrow failure, variable congenital abnormalities and a predisposition to malignancy, particularly acute myeloid leukemia (AML) and squamous cell carcinoma (SCC). Improved transplant outcomes are modifying the natural history of Fanconi Anemia. Improved transplant survival, no radiation exposure, and almost no GVHD increases the importance of addressing later SCC even further. The investigators hypothesize that quercetin will prevent or delay the development of SCC and associated complications, there by ameliorating or delaying the need for potentially lethal treatment with chemotherapy and/or radiation therapy for the same.
Funding Source - FDA Office of Orphan Products Development (OOPD)
详细描述
Fanconi anemia (FA) is an autosomal recessive disease characterized by progressive bone marrow failure, variable congenital abnormalities and a predisposition to malignancy, particularly acute myeloid leukemia (AML) and squamous cell carcinoma (SCC). Currently, the only curative treatment option for the hematological complications of FA include hematopoietic cell transplantation (HCT). The investigators hypothesize that quercetin will prevent or delay the development of SCC and associated complications, there by ameliorating or delaying the need for potentially lethal treatment with chemotherapy and/or radiation therapy for the same.
This study is an open-label, single arm study. This study will enroll approximately 45 post-HCT patients with FA, and approximately 10 patients with FA without history of HCT. In both groups, patients with or without existing pre-malignant lesions or history of SCC will be allowed to participate, if they wish so and at the discretion of the PI. All patients will be treated with oral quercetin.
The investigators will determine the efficacy of Quercetin in reducing buccal micronuclei (a surrogate marker of DNA damage and susceptibility to squamous cell carcinoma due to genomic instability) in post-HCT patients with fanconi anemia (FA).
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Prevention
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of FA
- •Able to take enteral medication
- •Patients ≥2 years
排除标准
- •Renal failure requiring dialysis
- •Total bilirubin >3 mg/dl and/or SGPT >200 at time of enrollment
- •Patients receiving digoxin therapy, who are unable to discontinue either treatment due to medical reasons
- •Patients who are pregnant or breastfeeding or are at risk of pregnancy or fathering a baby and are unable to use acceptable methods of birth control during the length of the study
- •Patients who have received quercetin supplementation or other antioxidants within the last 30 days
- •Patients receiving radiation therapy, chemotherapy or immunotherapy for treatment of SCC.
研究组 & 干预措施
Quercetin
All patients will be treated with oral quercetin.
干预措施: Quercetin (dietary supplement) (Drug)
结局指标
主要结局
Reduction of buccal micronuclei
时间窗: Up to 30 months
Efficacy of Quercetin in reducing buccal micronuclei
Number of Participants With at Least 20% Reduction of Buccal Micronuclei
时间窗: 1 year
Efficacy of Quercetin in reducing buccal micronuclei. A 20% reduction in the average total number of micronuclei will be considered a success of the intervention.
次要结局
未报告次要终点