Clinical Trial: Quercetin in Children With Fanconi Anemia; a Pilot Study

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Quercetin in Children With Fanconi Anemia; a Pilot Study

Brief Summary: Fanconi anemia (FA) is an autosomal recessive disease characterized by progressive bone marrow failure (BMF), congenital abnormalities and a predisposition to malignancy.

Detailed Summary:

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


Sponsor: Children's Hospital Medical Center, Cincinnati

Current Primary Outcome:

  • Measure the ability to administer twice daily oral quercetin therapy in patients with Fanconi Anemia (FA). [ Time Frame: 4 months (16 weeks) ]
  • Measure safety of oral quercetin therapy in patients with FA [ Time Frame: 4 months (16 weeks) ]
  • To measure pharmacokinetics (PK) of oral quercetin therapy in patients with FA [ Time Frame: 4 months (16 weeks) ]


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • To measure the impact of quercetin therapy on reduction of Reactive Oxygen Species (ROS). [ Time Frame: 4 months (16 weeks) and 1 year ]
  • Number of participants with improved hematopoiesis. [ Time Frame: 4 months (16 weeks) and 1 year ]
  • Measure the preservation of hematopoietic stem cell reserve in patients with FA [ Time Frame: 4 months (16 weeks) and 1 year ]
  • To measure the impact of quercetin therapy on changes in insulin sensitivity/glucose tolerance. [ Time Frame: 4 month (16 weeks) and 1 year ]


Original Secondary Outcome:

  • To measure the impact of quercetin therapy on reduction of Reactive Oxygen Species (ROS). [ Time Frame: 4 months (16 weeks) and 1 year ]
  • Number of participants with improved hematopoiesis. [ Time Frame: 4 months (16 weeks) and 1 year ]
  • Measure the preservation of hematopoietic stem cell reserve in patients with FA [ Time Frame: 4 months (16 weeks) and 1 year ]
  • Number of participants with changes in insulin sensitivity/glucose tolerance. [ Time Frame: 4 month (16 weeks) and 1 year ]


Information By: Children's Hospital Medical Center, Cincinnati

Dates:
Date Received: July 25, 2012
Date Started: July 2012
Date Completion: October 2017
Last Updated: August 19, 2016
Last Verified: August 2016