Quercetin in Children With Fanconi Anemia; a Pilot Study

NCT01720147 | Completed Phase 1 DMC

• 2 other identifiers: 2011-20491R01FD004383-01A1
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Fanconi Anemia

Keywords

Fanconi Anemia; FA; Quercetin

Outcome Measures

Primary Outcomes (3)

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

  4 months (16 weeks)

• Measure safety of oral quercetin therapy in patients with FA

  4 months (16 weeks)

• To measure pharmacokinetics (PK) of oral quercetin therapy in patients with FA

  4 months (16 weeks)

Secondary Outcomes (4)

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

  4 months (16 weeks) and 1 year

• Number of participants with improved hematopoiesis.

  4 months (16 weeks) and 1 year

• Measure the preservation of hematopoietic stem cell reserve in patients with FA

  4 months (16 weeks) and 1 year

• To measure the impact of quercetin therapy on changes in insulin sensitivity/glucose tolerance.

  4 month (16 weeks) and 1 year

Study Arms (1)

Quercetin - Dietary Supplement

EXPERIMENTAL

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).

Drug: Quercetin (dietary supplement)

Interventions

Quercetin (dietary supplement) DRUG

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).

Quercetin - Dietary Supplement

Eligibility Criteria

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

You may qualify if:

• Diagnosis of FA proven by DEB test or molecular testing
• Able to take enteral medication
• All age groups, including adults

You may not qualify if:

• 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

Sponsors & Collaborators

• Children's Hospital Medical Center, Cincinnati: lead
• Food and Drug Administration (FDA): collaborator

Study Sites (1)

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Related Publications (1)

• Mehta PA, Nelson A, Loveless S, Lane A, Fukuda T, Teusink-Cross A, Elder D, Lagory D, Miller E, Cancelas JA, Howell J, Zhao J, Mizuno K, Myers KC, Lake K, McIntosh K, Setchell KDR, Luebbering N, Edwards S, Chihanga T, Wells SI, Davies SM. Phase 1 study of quercetin, a natural antioxidant for children and young adults with Fanconi anemia. Blood Adv. 2025 Apr 22;9(8):1927-1939. doi: 10.1182/bloodadvances.2024015053.

  PMID: 39820512 DERIVED

MeSH Terms

Conditions

Fanconi Anemia

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

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

Study Officials

• Parinda Mehta, MD

  Children's Hospital Medical Center, Cincinnati

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: PREVENTION
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 25, 2012
• First Posted: November 2, 2012
• Study Start: July 1, 2012
• Primary Completion: October 26, 2021
• Study Completion: October 26, 2021
• Last Updated: January 18, 2024

Record last verified: 2024-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)