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Fanconi Anemia | Quercetin in Children With Fanconi Anemia; a Pilot Study

Fanconi Anemia research study

What is the primary objective of this study?

Fanconi anemia (FA) is an autosomal recessive disease characterized by progressive bone marrow failure (BMF), congenital abnormalities and a predisposition to malignancy.

Who is eligible to participate?

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

Which medical condition, disease, disorder, syndrome, illness, or injury is researched?

Fanconi Anemia

Study Interventions

Interventions can include giving participants drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available or noninvasive approaches such as surveys, education, and interviews.

Drug: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).

Study Arms

Research studies and clinical trials typically have two or more research arms. An arm is a group of people who receive the same treatment in the study.

Quercetin - Dietary SupplementQuercetin 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).

Study Status

Recruiting

Start Date: July 2012

Completed Date: December 2022

Phase: Phase 1

Type: Interventional

Design:

Primary Outcome: Measure the ability to administer twice daily oral quercetin therapy in patients with Fanconi Anemia (FA).

Secondary Outcome: To measure the impact of quercetin therapy on reduction of Reactive Oxygen Species (ROS).

Study sponsors, principal investigator, and references

Principal Investigator: Parinda Mehta, MD

Lead Sponsor: Children's Hospital Medical Center, Cincinnati

Collaborator: Food and Drug Administration (FDA)

More information:https://clinicaltrials.gov/show/NCT01720147

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