Quercetin Chemoprevention for Squamous Cell Carcinoma in Patients With Fanconi Anemia

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)

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Key information

Conditions

Fanconi Anemia Anemia Anemia, Aplastic Anemia, Hypoplastic, Congenital Bone Marrow Diseases Bone Marrow Failure Disorders Carcinoma Carcinoma, Squamous Cell Congenital Bone Marrow Failure Syndromes Congenital, Hereditary, and Neonatal Diseases and Abnormalities DNA Repair-Deficiency Disorders Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Metabolic Diseases Neoplasms Neoplasms by Histologic Type Neoplasms, Glandular and Epithelial Neoplasms, Squamous Cell Nutritional and Metabolic Diseases Squamous Cell Carcinoma

Age range

2 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

About this study

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).

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Quercetin (dietary supplement)

Drug

Quercetin will be administered twice daily at an adjusted dose based on weight for a maximum total daily dose of 4000mg/day. If the patient is 70 kg or more, the dose will automatically be assigned at the maximum dose of 4000mg/day.

Primary outcomes

Number of Participants With at Least 20% Reduction of Buccal Micronuclei

Time frame: 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.

Sponsors and collaborators

Lead sponsor

Children's Hospital Medical Center, Cincinnati

Other

Registry information

Important dates

Study start: 2018
Primary completion: 2025
Study completion: 2026
First posted: Mar 26, 2018
Registry last updated: Apr 20, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed