Quercetin Chemoprevention for Squamous Cell Carcinoma in Patients With Fanconi Anemia

Sponsor

Children's Hospital Medical Center, Cincinnati (Other)

Overall Status

Recruiting

CT.gov ID

NCT03476330

Collaborator

(none)

Enrollment

Location

Arm

87.8

Anticipated Duration (Months)

0.6

Patients Per Site Per Month

Study Details

Study Description

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)

Condition or Disease	Intervention/Treatment	Phase
Fanconi Anemia Squamous Cell Carcinoma	Drug: Quercetin (dietary supplement)	Phase 2

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).

Study Design

Study Type:

Interventional

Anticipated Enrollment :

55 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Prevention

Official Title:

Quercetin Chemoprevention for Squamous Cell Carcinoma in Patients With Fanconi Anemia

Actual Study Start Date :

May 8, 2018

Anticipated Primary Completion Date :

Sep 1, 2024

Anticipated Study Completion Date :

Sep 1, 2025

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Quercetin All patients will be treated with oral quercetin.	Drug: 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.

Arm

Intervention/Treatment

Experimental: Quercetin

All patients will be treated with oral quercetin.

Drug: 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.

Outcome Measures

Primary Outcome Measures

Reduction of buccal micronuclei [Up to 30 months]
Efficacy of Quercetin in reducing buccal micronuclei

Eligibility Criteria

Criteria

Ages Eligible for Study:

2 Years and Older

Sexes Eligible for Study:

All

Accepts 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.