Quercetin in Children With Fanconi Anemia; a Pilot Study
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- Measure the ability to administer twice daily oral quercetin therapy in patients with Fanconi Anemia (FA).
研究概览
简要总结
Fanconi anemia (FA) is an autosomal recessive disease characterized by progressive bone marrow failure (BMF), congenital abnormalities and a predisposition to malignancy.
详细描述
Current therapies for children with Fanconi anemia (FA) and bone marrow failure, i.e. androgens or bone marrow transplantation, are associated with significant morbidity and mortality.
This is a pilot study aiming to assess feasibility, toxicity and pharmacokinetics of oral Quercetin therapy in patients with FA. This is a first step towards a clinical study of the efficacy of Quercetin therapy in delaying progression of BMF in FA.
Additional correlative studies will include assessment of impact of Quercetin on reduction of Reactive Oxygen Species (ROS), maintenance or improvement of hematopoietic stem cell (HSC) reserve, improvement of hematopoiesis (i.e. peripheral counts) and insulin sensitivity/glucose tolerance.
This study is an open-label single arm study.
Funding Source - FDA OOPD
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
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of FA proven by DEB test or molecular testing
- •Able to take enteral medication
- •All age groups, including adults
排除标准
- •Patients with morphological evidence of myelodysplasia or leukemia
- •Renal failure requiring dialysis
- •Total bilirubin > 3 mg/dl and/or SGPT >200 at time of enrollment
- •Patients who are pregnant or breastfeeding or are at risk of pregnancy and are unable to use acceptable methods of birth control during the length of the study
- •Patients receiving cyclosporine or digoxin therapy or are unable to discontinue either treatment due to medical reasons
- •Patients who have received quercetin supplementation or other antioxidants within the last 30 days
研究组 & 干预措施
Quercetin - Dietary Supplement
Quercetin will be given orally on a twice a day schedule starting with weight adjusted dose for a maximum total daily dose of 1500 mg/day, for 4 months (16 weeks). Pharmacokinetics (PK) data will be analyzed after each cohort of 3 patients and will be used to optimize the dosing schedule (if required) for subsequent patients.
An expansion cohort has been added to the study protocol. Up to 20 patients may be enrolled. The dose utilized will be the same as the max weight adjusted dose that showed biological activity in our last cohort of patients (subjects #10-12 from above).
干预措施: Quercetin (dietary supplement) (Drug)
结局指标
主要结局
Measure the ability to administer twice daily oral quercetin therapy in patients with Fanconi Anemia (FA).
时间窗: 4 months (16 weeks)
To measure pharmacokinetics (PK) of oral quercetin therapy in patients with FA
时间窗: 4 months (16 weeks)
Measure safety of oral quercetin therapy in patients with FA
时间窗: 4 months (16 weeks)
次要结局
- To measure the impact of quercetin therapy on reduction of Reactive Oxygen Species (ROS).(4 months (16 weeks) and 1 year)
- To measure the impact of quercetin therapy on changes in insulin sensitivity/glucose tolerance.(4 month (16 weeks) and 1 year)
- Number of participants with improved hematopoiesis.(4 months (16 weeks) and 1 year)
- Measure the preservation of hematopoietic stem cell reserve in patients with FA(4 months (16 weeks) and 1 year)