Phase 2
N=48
Quercetin Chemoprevention for Squamous Cell Carcinoma in Patients With Fanconi Anemia
Fanconi Anemia · Squamous Cell Carcinoma
Bottom Line
View on ClinicalTrials.gov: NCT03476330 ↗Enrolled (actual)
48
Serious AEs
6.4%
Results posted
Apr 2026
Primary outcome: Primary: Number of Participants With at Least 20% Reduction of Buccal Micronuclei — 13 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Quercetin (dietary supplement) (Drug)
- Age
- Pediatric, Adult, Older Adult · 2+ yrs
- Sex
- All
- Sponsor
- Children's Hospital Medical Center, Cincinnati
- Primary completion
- Mar 2025
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With at Least 20% Reduction of Buccal Micronuclei |
13 | — |
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)
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.
Data sourced from ClinicalTrials.gov (NCT03476330). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.