Quercetin Chemoprevention for Squamous Cell Carcinoma in Patients With Fanconi Anemia

Cincinnati Children's Hospital Medical Center

Status and phase

Active, not recruiting

Phase 2

Conditions

Fanconi Anemia

Squamous Cell Carcinoma

Treatments

Drug: Quercetin (dietary supplement)

Study type

Interventional

Funder types

Other

Identifiers

NCT03476330

6353

2018-0073

Details and patient eligibility

About

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)

Full description

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).

Enrollment

48 patients

Sex

All

Ages

2+ years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Diagnosis of FA
Able to take enteral medication
Patients ≥2 years

Exclusion criteria

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.