Quercetin Chemoprevention for Squamous Cell Carcinoma in Patients With Fanconi Anemia
Primary Purpose
Fanconi Anemia, Squamous Cell Carcinoma
Status
Recruiting
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Quercetin (dietary supplement)
Sponsored by
About this trial
This is an interventional prevention trial for Fanconi Anemia focused on measuring Hematopoietic cell transplantation
Eligibility Criteria
Inclusion Criteria:
- Diagnosis of FA
- Able to take enteral medication
- Patients ≥2 years
Exclusion Criteria:
- Renal failure requiring dialysis
- Total bilirubin >3 mg/dl and/or SGPT >200 at time of enrollment
- Patients receiving digoxin therapy, who are unable to discontinue either treatment due to medical reasons
- Patients who are pregnant or breastfeeding or are at risk of pregnancy or fathering a baby and are unable to use acceptable methods of birth control during the length of the study
- Patients who have received quercetin supplementation or other antioxidants within the last 30 days
- Patients receiving radiation therapy, chemotherapy or immunotherapy for treatment of SCC.
Sites / Locations
- Cincinnati Children's Hospital Medical CenterRecruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Quercetin
Arm Description
All patients will be treated with oral quercetin.
Outcomes
Primary Outcome Measures
Reduction of buccal micronuclei
Efficacy of Quercetin in reducing buccal micronuclei
Secondary Outcome Measures
Full Information
NCT ID
NCT03476330
First Posted
March 19, 2018
Last Updated
February 1, 2023
Sponsor
Children's Hospital Medical Center, Cincinnati
1. Study Identification
Unique Protocol Identification Number
NCT03476330
Brief Title
Quercetin Chemoprevention for Squamous Cell Carcinoma in Patients With Fanconi Anemia
Official Title
Quercetin Chemoprevention for Squamous Cell Carcinoma in Patients With Fanconi Anemia
Study Type
Interventional
2. Study Status
Record Verification Date
February 2023
Overall Recruitment Status
Recruiting
Study Start Date
May 8, 2018 (Actual)
Primary Completion Date
September 2024 (Anticipated)
Study Completion Date
September 2025 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Children's Hospital Medical Center, Cincinnati
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
Fanconi anemia (FA) is an autosomal recessive disease characterized by progressive bone marrow failure, variable congenital abnormalities and a predisposition to malignancy, particularly acute myeloid leukemia (AML) and squamous cell carcinoma (SCC). Improved transplant outcomes are modifying the natural history of Fanconi Anemia. Improved transplant survival, no radiation exposure, and almost no GVHD increases the importance of addressing later SCC even further. The investigators hypothesize that quercetin will prevent or delay the development of SCC and associated complications, there by ameliorating or delaying the need for potentially lethal treatment with chemotherapy and/or radiation therapy for the same.
Funding Source - FDA Office of Orphan Products Development (OOPD)
Detailed Description
Fanconi anemia (FA) is an autosomal recessive disease characterized by progressive bone marrow failure, variable congenital abnormalities and a predisposition to malignancy, particularly acute myeloid leukemia (AML) and squamous cell carcinoma (SCC). Currently, the only curative treatment option for the hematological complications of FA include hematopoietic cell transplantation (HCT). The investigators hypothesize that quercetin will prevent or delay the development of SCC and associated complications, there by ameliorating or delaying the need for potentially lethal treatment with chemotherapy and/or radiation therapy for the same.
This study is an open-label, single arm study. This study will enroll approximately 45 post-HCT patients with FA, and approximately 10 patients with FA without history of HCT. In both groups, patients with or without existing pre-malignant lesions or history of SCC will be allowed to participate, if they wish so and at the discretion of the PI. All patients will be treated with oral quercetin.
The investigators will determine the efficacy of Quercetin in reducing buccal micronuclei (a surrogate marker of DNA damage and susceptibility to squamous cell carcinoma due to genomic instability) in post-HCT patients with fanconi anemia (FA).
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Fanconi Anemia, Squamous Cell Carcinoma
Keywords
Hematopoietic cell transplantation
7. Study Design
Primary Purpose
Prevention
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
55 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Quercetin
Arm Type
Experimental
Arm Description
All patients will be treated with oral quercetin.
Intervention Type
Drug
Intervention Name(s)
Quercetin (dietary supplement)
Intervention Description
Quercetin will be administered twice daily at an adjusted dose based on weight for a maximum total daily dose of 4000mg/day. If the patient is 70 kg or more, the dose will automatically be assigned at the maximum dose of 4000mg/day.
Primary Outcome Measure Information:
Title
Reduction of buccal micronuclei
Description
Efficacy of Quercetin in reducing buccal micronuclei
Time Frame
Up to 30 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
2 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Diagnosis of FA
Able to take enteral medication
Patients ≥2 years
Exclusion Criteria:
Renal failure requiring dialysis
Total bilirubin >3 mg/dl and/or SGPT >200 at time of enrollment
Patients receiving digoxin therapy, who are unable to discontinue either treatment due to medical reasons
Patients who are pregnant or breastfeeding or are at risk of pregnancy or fathering a baby and are unable to use acceptable methods of birth control during the length of the study
Patients who have received quercetin supplementation or other antioxidants within the last 30 days
Patients receiving radiation therapy, chemotherapy or immunotherapy for treatment of SCC.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Stephanie A Edwards, BSN, RN
Phone
513-636-9292
Email
StephanieL.Edwards@cchmc.org
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Parinda A Mehta, MD
Organizational Affiliation
Cincinnati Children's Hosptial Medical Center
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
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Stephanie L Edwards, BSN, RN
Phone
513-636-9292
Email
StephanieL.Edwards@cchmc.org
First Name & Middle Initial & Last Name & Degree
Parinda A Mehta, MD
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
Quercetin Chemoprevention for Squamous Cell Carcinoma in Patients With Fanconi Anemia
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