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Quercetin in Children With Fanconi Anemia; a Pilot Study

Primary Purpose

Fanconi Anemia

Status
Active
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Quercetin (dietary supplement)
Sponsored by
Children's Hospital Medical Center, Cincinnati
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional prevention trial for Fanconi Anemia focused on measuring Fanconi Anemia, FA, Quercetin

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Arm Type

Experimental

Arm Label

Quercetin - Dietary Supplement

Arm Description

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).

Outcomes

Primary Outcome Measures

Measure the ability to administer twice daily oral quercetin therapy in patients with Fanconi Anemia (FA).
Measure safety of oral quercetin therapy in patients with FA
To measure pharmacokinetics (PK) of oral quercetin therapy in patients with FA

Secondary Outcome Measures

To measure the impact of quercetin therapy on reduction of Reactive Oxygen Species (ROS).
Number of participants with improved hematopoiesis.
Measure the preservation of hematopoietic stem cell reserve in patients with FA
To measure the impact of quercetin therapy on changes in insulin sensitivity/glucose tolerance.

Full Information

First Posted
July 25, 2012
Last Updated
February 3, 2023
Sponsor
Children's Hospital Medical Center, Cincinnati
Collaborators
Food and Drug Administration (FDA)
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1. Study Identification

Unique Protocol Identification Number
NCT01720147
Brief Title
Quercetin in Children With Fanconi Anemia; a Pilot Study
Official Title
Quercetin in Children With Fanconi Anemia; a Pilot Study
Study Type
Interventional

2. Study Status

Record Verification Date
February 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
July 2012 (undefined)
Primary Completion Date
October 26, 2021 (Actual)
Study Completion Date
December 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Children's Hospital Medical Center, Cincinnati
Collaborators
Food and Drug Administration (FDA)

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief Summary
Fanconi anemia (FA) is an autosomal recessive disease characterized by progressive bone marrow failure (BMF), congenital abnormalities and a predisposition to malignancy.
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 will be able to continue quercetin supplement after the completion of the study period of 26 weeks if they wish. If patients decide to continue quercetin after the first 26 weeks, quercetin will be provided by the investigational pharmacy for up to 1 year. Patients who continue quercetin supplement 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. Patients previously enrolled in the initial phase of the study (cohorts 1-4) may be considered for enrollment on the expansion cohort of the study provided they meet all inclusion and exclusion criteria.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Fanconi Anemia
Keywords
Fanconi Anemia, FA, Quercetin

7. Study Design

Primary Purpose
Prevention
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
30 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Quercetin - Dietary Supplement
Arm Type
Experimental
Arm Description
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).
Intervention Type
Drug
Intervention Name(s)
Quercetin (dietary supplement)
Intervention Description
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).
Primary Outcome Measure Information:
Title
Measure the ability to administer twice daily oral quercetin therapy in patients with Fanconi Anemia (FA).
Time Frame
4 months (16 weeks)
Title
Measure safety of oral quercetin therapy in patients with FA
Time Frame
4 months (16 weeks)
Title
To measure pharmacokinetics (PK) of oral quercetin therapy in patients with FA
Time Frame
4 months (16 weeks)
Secondary Outcome Measure Information:
Title
To measure the impact of quercetin therapy on reduction of Reactive Oxygen Species (ROS).
Time Frame
4 months (16 weeks) and 1 year
Title
Number of participants with improved hematopoiesis.
Time Frame
4 months (16 weeks) and 1 year
Title
Measure the preservation of hematopoietic stem cell reserve in patients with FA
Time Frame
4 months (16 weeks) and 1 year
Title
To measure the impact of quercetin therapy on changes in insulin sensitivity/glucose tolerance.
Time Frame
4 month (16 weeks) and 1 year

10. Eligibility

Sex
All
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Parinda Mehta, MD
Organizational Affiliation
Children's Hospital Medical Center, Cincinnati
Official's Role
Principal Investigator
Facility Information:
Facility Name
Cincinnati Children's Hospital Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

Quercetin in Children With Fanconi Anemia; a Pilot Study

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