Quercetin in Children With Fanconi Anemia; a Pilot Study

• Register: ClinicalTrials.gov
• Last refreshed on: 29 January 2024
• Main ID: NCT01720147
• Date of registration: 25/07/2012
• Prospective Registration: No
• Primary sponsor: Children's Hospital Medical Center, Cincinnati
• Public title: Quercetin in Children With Fanconi Anemia; a Pilot Study
• Scientific title: Quercetin in Children With Fanconi Anemia; a Pilot Study
• Date of first enrolment: July 2012
• Target sample size: 30
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT01720147
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Prevention. Masking: None (Open Label).
• Phase: Phase 1

Countries of recruitment

United States

Contacts

• Name: Parinda Mehta, MD
• Affiliation: Children's Hospital Medical Center, Cincinnati

Key inclusion & exclusion 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



Age minimum: N/A
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Fanconi Anemia

Intervention(s)

Drug: Quercetin (dietary supplement)

Primary Outcome(s)

To measure pharmacokinetics (PK) of oral quercetin therapy in patients with FA [Time Frame: 4 months (16 weeks)]

Measure safety of oral quercetin therapy in patients with FA [Time Frame: 4 months (16 weeks)]

Measure the ability to administer twice daily oral quercetin therapy in patients with Fanconi Anemia (FA). [Time Frame: 4 months (16 weeks)]

Secondary Outcome(s)

Number of participants with improved hematopoiesis. [Time Frame: 4 months (16 weeks) and 1 year]

To measure the impact of quercetin therapy on reduction of Reactive Oxygen Species (ROS). [Time Frame: 4 months (16 weeks) and 1 year]

Measure the preservation of hematopoietic stem cell reserve in patients with FA [Time Frame: 4 months (16 weeks) and 1 year]

To measure the impact of quercetin therapy on changes in insulin sensitivity/glucose tolerance. [Time Frame: 4 month (16 weeks) and 1 year]

Secondary ID(s)

1R01FD004383-01A1

2011-2049

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Food and Drug Administration (FDA)