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Quercetin in Children With Fanconi Anemia; a Pilot Study

This study is currently recruiting participants.
See Contacts and Locations
Verified July 2017 by Children's Hospital Medical Center, Cincinnati
Sponsor:
Information provided by (Responsible Party):
Children's Hospital Medical Center, Cincinnati
ClinicalTrials.gov Identifier:
NCT01720147
First received: July 25, 2012
Last updated: July 5, 2017
Last verified: July 2017
  Purpose
Fanconi anemia (FA) is an autosomal recessive disease characterized by progressive bone marrow failure (BMF), congenital abnormalities and a predisposition to malignancy.

Condition Intervention Phase
Fanconi Anemia Drug: Quercetin (dietary supplement) Phase 1

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Prevention
Official Title: Quercetin in Children With Fanconi Anemia; a Pilot Study

Resource links provided by NLM:


Further study details as provided by Children's Hospital Medical Center, Cincinnati:

Primary Outcome Measures:
  • Measure the ability to administer twice daily oral quercetin therapy in patients with Fanconi Anemia (FA). [ Time Frame: 4 months (16 weeks) ]
  • Measure safety of oral quercetin therapy in patients with FA [ Time Frame: 4 months (16 weeks) ]
  • To measure pharmacokinetics (PK) of oral quercetin therapy in patients with FA [ Time Frame: 4 months (16 weeks) ]

Secondary Outcome Measures:
  • To measure the impact of quercetin therapy on reduction of Reactive Oxygen Species (ROS). [ Time Frame: 4 months (16 weeks) and 1 year ]
  • Number of participants with improved hematopoiesis. [ 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 ]

Estimated Enrollment: 32
Study Start Date: July 2012
Estimated Study Completion Date: December 2022
Estimated Primary Completion Date: December 2020 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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).

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).


Detailed Description:

Current therapies for children with Fanconi anemia (FA) and bone marrow failure, i.e. androgens or bone marrow transplantation, are associated with significant morbidity and mortality.

This is a pilot study aiming to assess feasibility, toxicity and pharmacokinetics of oral Quercetin therapy in patients with FA. This is a first step towards a clinical study of the efficacy of Quercetin therapy in delaying progression of BMF in FA.

Additional correlative studies will include assessment of impact of Quercetin on reduction of Reactive Oxygen Species (ROS), maintenance or improvement of hematopoietic stem cell (HSC) reserve, improvement of hematopoiesis (i.e. peripheral counts) and insulin sensitivity/glucose tolerance.

This study is an open-label single arm study.

Funding Source - FDA OOPD

Accrual closed for the main study. Expansion cohort added to observe additional patients at the desired dose.

Expansion Cohort:

The second and third cohort of participants completed the study treatment as expected, tolerating Quercetin well without any DLT. Based on the PK and ROS analyses, the dose was increased for the fourth cohort (subjects #10-12). To observe additional patients at the desired dose, an expansion cohort is being added. Up to 20 patients will be enrolled in the expansion cohort. The purpose of the expansion cohort is to assess the effects of quercetin supplement at the desired dose on clinical and biological endpoints.

Patients in the expansion cohort will receive quercetin treatment for the first 26 weeks. Patients who continue quercetin supplement clinically after 26 weeks, but discontinue quercetin before 1 year will be followed through the 1 year visit. Patients who discontinue quercetin supplement any time after the 1 year visit will be followed through the 2 year visit.

  Eligibility

Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Diagnosis of FA proven by DEB test
  • 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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01720147

Contacts
Contact: Stephanie Edwards, BSN 513-636-9292 stephanieL.edwards@cchmc.org
Contact: Sara Loveless 513-803-7656 Sara.Loveless@cchmc.org

Locations
United States, Ohio
Cincinnati Children's Hospital Medical Center Recruiting
Cincinnati, Ohio, United States, 45229
Principal Investigator: Parinda Mehta, MD         
Sponsors and Collaborators
Children's Hospital Medical Center, Cincinnati
Investigators
Principal Investigator: Parinda Mehta, MD Children's Hospital Medical Center, Cincinnati
  More Information

Responsible Party: Children's Hospital Medical Center, Cincinnati
ClinicalTrials.gov Identifier: NCT01720147     History of Changes
Other Study ID Numbers: 2011-2049
1R01FD004383-01A1 ( U.S. FDA Grant/Contract )
Study First Received: July 25, 2012
Last Updated: July 5, 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Keywords provided by Children's Hospital Medical Center, Cincinnati:
Fanconi Anemia
FA
Quercetin

Additional relevant MeSH terms:
Fanconi Anemia
Fanconi Syndrome
Anemia
Hematologic Diseases
Anemia, Hypoplastic, Congenital
Anemia, Aplastic
Bone Marrow Diseases
Genetic Diseases, Inborn
DNA Repair-Deficiency Disorders
Metabolic Diseases
Renal Tubular Transport, Inborn Errors
Kidney Diseases
Urologic Diseases
Metabolism, Inborn Errors
Quercetin
Antioxidants
Molecular Mechanisms of Pharmacological Action
Protective Agents
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on August 18, 2017