Quercetin Chemoprevention for Squamous Cell Carcinoma in Patients With Fanconi Anemia

NCT ID: NCT03476330

Last Updated: 2025-04-27

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 48 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2018-05-08
• Study Completion Date: 2026-03-31

Brief Summary

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)

Detailed 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).

Conditions

Fanconi Anemia; Squamous Cell Carcinoma

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: PREVENTION
• Blinding Strategy: NONE

Study Groups

Quercetin

All patients will be treated with oral quercetin.

Group Type: EXPERIMENTAL

Quercetin (dietary supplement)

Intervention Type: 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.

Interventions

Quercetin (dietary supplement)

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.

Intervention Type: DRUG

Eligibility Criteria

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.

• Minimum Eligible Age: 2 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Children's Hospital Medical Center, Cincinnati

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Parinda A Mehta, MD

Role: PRINCIPAL_INVESTIGATOR

Cincinnati Children's Hosptial Medical Center

Locations

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Countries

United States

Other Identifiers

6353

Identifier Type: OTHER_GRANT

Identifier Source: secondary_id

2018-0073

Identifier Type: -

Identifier Source: org_study_id