Quercetin Chemoprevention for Squamous Cell Carcinoma in Patients With Fanconi Anemia

NCT03476330 | Completed Phase 2 Results DMC FDA Drug

• 2 other identifiers: 2018-00736353
• Study Type: interventional
• Target: 48
• Locations: 1 country
• Sites: 1

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)

Conditions

Fanconi Anemia; Squamous Cell Carcinoma

Keywords

Hematopoietic cell transplantation

Outcome Measures

Primary Outcomes (1)

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

  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.

  1 year

Study Arms (1)

Quercetin

EXPERIMENTAL

All patients will be treated with oral quercetin.

Drug: Quercetin (dietary supplement)

Interventions

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.

Quercetin

Eligibility Criteria

• Age: 2 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Children's Hospital Medical Center, Cincinnati: lead

Study Sites (1)

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

MeSH Terms

Conditions

Fanconi Anemia; Carcinoma, Squamous Cell

Interventions

Quercetin; Dietary Supplements

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Flavonols; Flavonoids; Chromones; Benzopyrans; Pyrans; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Food; Diet, Food, and Nutrition; Physiological Phenomena; Food and Beverages

Results Point of Contact

• Title: Parinda Mehta, MD
• Organization: Cincinnati Children's Hospital Medical Center

parinda.mehta@cchmc.org

513-636-5917

Study Officials

• Parinda A Mehta, MD

  Cincinnati Children's Hosptial Medical Center

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: PREVENTION
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 19, 2018
• First Posted: March 26, 2018
• Study Start: May 8, 2018
• Primary Completion: March 6, 2025
• Study Completion: January 9, 2026
• Last Updated: April 20, 2026
• Results First Posted: April 20, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)