Quercetin in Children With Fanconi Anemia; a Pilot Study
Fanconi Anemia
About this trial
This is an interventional prevention trial for Fanconi Anemia focused on measuring Fanconi Anemia, FA, Quercetin
Eligibility Criteria
Inclusion Criteria:
- Diagnosis of FA proven by DEB test or molecular testing
- Able to take enteral medication
- All age groups, including adults
Exclusion Criteria:
- Patients with morphological evidence of myelodysplasia or leukemia
- Renal failure requiring dialysis
- Total bilirubin > 3 mg/dl and/or SGPT >200 at time of enrollment
- Patients who are pregnant or breastfeeding or are at risk of pregnancy and are unable to use acceptable methods of birth control during the length of the study
- Patients receiving cyclosporine or digoxin therapy or are unable to discontinue either treatment due to medical reasons
- Patients who have received quercetin supplementation or other antioxidants within the last 30 days
Sites / Locations
- Cincinnati Children's Hospital Medical Center
Arms of the Study
Arm 1
Experimental
Quercetin - Dietary Supplement
Quercetin will be given orally on a twice a day schedule starting with weight adjusted dose for a maximum total daily dose of 1500 mg/day, for 4 months (16 weeks). Pharmacokinetics (PK) data will be analyzed after each cohort of 3 patients and will be used to optimize the dosing schedule (if required) for subsequent patients. An expansion cohort has been added to the study protocol. Up to 20 patients may be enrolled. The dose utilized will be the same as the max weight adjusted dose that showed biological activity in our last cohort of patients (subjects #10-12 from above).