- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03476330
Quercetin Chemoprevention for Squamous Cell Carcinoma in Patients With Fanconi Anemia
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)
Study Overview
Status
Conditions
Intervention / Treatment
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).
Study Type
Enrollment (Actual)
Phase
- Phase 2
Contacts and Locations
Study Contact
- Name: Stephanie A Edwards, BSN, RN
- Phone Number: 513-636-9292
- Email: StephanieL.Edwards@cchmc.org
Study Locations
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Ohio
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Cincinnati, Ohio, United States, 45229
- Cincinnati Children's Hospital Medical Center
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Description
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.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Prevention
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Quercetin
All patients will be treated with oral quercetin.
|
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.
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Reduction of buccal micronuclei
Time Frame: Up to 30 months
|
Efficacy of Quercetin in reducing buccal micronuclei
|
Up to 30 months
|
Collaborators and Investigators
Investigators
- Principal Investigator: Parinda A Mehta, MD, Cincinnati Children's Hosptial Medical Center
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Estimated)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Metabolic Diseases
- Neoplasms by Histologic Type
- Neoplasms
- Kidney Diseases
- Urologic Diseases
- Neoplasms, Glandular and Epithelial
- Bone Marrow Diseases
- Hematologic Diseases
- Genetic Diseases, Inborn
- DNA Repair-Deficiency Disorders
- Anemia, Hypoplastic, Congenital
- Anemia, Aplastic
- Congenital Bone Marrow Failure Syndromes
- Bone Marrow Failure Disorders
- Renal Tubular Transport, Inborn Errors
- Neoplasms, Squamous Cell
- Female Urogenital Diseases
- Female Urogenital Diseases and Pregnancy Complications
- Urogenital Diseases
- Male Urogenital Diseases
- Carcinoma
- Anemia
- Fanconi Syndrome
- Fanconi Anemia
- Carcinoma, Squamous Cell
- Physiological Effects of Drugs
- Molecular Mechanisms of Pharmacological Action
- Protective Agents
- Antioxidants
- Quercetin
Other Study ID Numbers
- 2018-0073
- 6353 (FDA Office of Orphan Products Development (OOPD))
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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