Quercetin Chemoprevention for Squamous Cell Carcinoma in Patients With Fanconi Anemia
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| ClinicalTrials.gov Identifier: NCT03476330 |
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Recruitment Status :
Recruiting
First Posted : March 26, 2018
Last Update Posted : February 2, 2023
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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)
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Fanconi Anemia Squamous Cell Carcinoma | Drug: Quercetin (dietary supplement) | Phase 2 |
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 : | Interventional (Clinical Trial) |
| Estimated Enrollment : | 55 participants |
| Allocation: | N/A |
| Intervention Model: | Single Group Assignment |
| Masking: | None (Open Label) |
| Primary Purpose: | Prevention |
| Official Title: | Quercetin Chemoprevention for Squamous Cell Carcinoma in Patients With Fanconi Anemia |
| Actual Study Start Date : | May 8, 2018 |
| Estimated Primary Completion Date : | September 2024 |
| Estimated Study Completion Date : | September 2025 |
| Arm | Intervention/treatment |
|---|---|
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Experimental: Quercetin
All patients will be treated with oral quercetin.
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Drug: Quercetin (dietary supplement)
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. |
- Reduction of buccal micronuclei [ Time Frame: Up to 30 months ]Efficacy of Quercetin in reducing buccal micronuclei
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| Ages Eligible for Study: | 2 Years and older (Child, Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03476330
| Contact: Stephanie A Edwards, BSN, RN | 513-636-9292 | StephanieL.Edwards@cchmc.org |
| United States, Ohio | |
| Cincinnati Children's Hospital Medical Center | Recruiting |
| Cincinnati, Ohio, United States, 45229 | |
| Contact: Stephanie L Edwards, BSN, RN 513-636-9292 StephanieL.Edwards@cchmc.org | |
| Principal Investigator: Parinda A Mehta, MD | |
| Principal Investigator: | Parinda A Mehta, MD | Cincinnati Children's Hosptial Medical Center |
| Responsible Party: | Children's Hospital Medical Center, Cincinnati |
| ClinicalTrials.gov Identifier: | NCT03476330 |
| Other Study ID Numbers: |
2018-0073 6353 ( Other Grant/Funding Number: FDA Office of Orphan Products Development (OOPD) ) |
| First Posted: | March 26, 2018 Key Record Dates |
| Last Update Posted: | February 2, 2023 |
| Last Verified: | February 2023 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | No |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
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Hematopoietic cell transplantation |
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Neoplasms, Glandular and Epithelial Neoplasms by Histologic Type Neoplasms Neoplasms, Squamous Cell Quercetin Carcinoma Carcinoma, Squamous Cell Fanconi Syndrome Anemia Fanconi Anemia Hematologic Diseases Anemia, Hypoplastic, Congenital Anemia, Aplastic |
Congenital Bone Marrow Failure Syndromes Bone Marrow Failure Disorders Bone Marrow Diseases Genetic Diseases, Inborn DNA Repair-Deficiency Disorders Metabolic Diseases Renal Tubular Transport, Inborn Errors Kidney Diseases Urologic Diseases Antioxidants Molecular Mechanisms of Pharmacological Action Protective Agents Physiological Effects of Drugs |