Clinical Trial Archives - Fanconi Cancer Foundation https://fanconi.org/researches-category/clinical-trial/ Pioneering Research for a Brighter Tomorrow. Tue, 09 Apr 2024 09:03:47 +0000 en-US hourly 1 https://wordpress.org/?v=7.0 https://fanconi.org/wp-content/uploads/2024/04/cropped-fanconi-favicon-32x32.png Clinical Trial Archives - Fanconi Cancer Foundation https://fanconi.org/researches-category/clinical-trial/ 32 32 Clinical trial to investigate the safety and efficacy of Afatinib when administered as therapy in FA patients https://fanconi.org/supported-researches/clinical-trial-to-investigate-the-safety-and-efficacy-of-afatinib-when-administered-as-therapy-in-fa-patients/ Wed, 20 Mar 2024 12:37:08 +0000 https://wpbox7.net/ce/dc7ad561/?post_type=app_research&p=2464 Individuals with FA have a higher risk of developing head and neck squamous cell carcinoma (HSNCC) at young ages. Long-term survival rates remain low because often these patients cannot tolerate conventional chemotherapy and radiation treatments due to issues related to toxicity. This study is the first clinical trial focused on a therapy for FA-related HNSCC. […]

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]]> Individuals with FA have a higher risk of developing head and neck squamous cell carcinoma (HSNCC) at young ages. Long-term survival rates remain low because often these patients cannot tolerate conventional chemotherapy and radiation treatments due to issues related to toxicity. This study is the first clinical trial focused on a therapy for FA-related HNSCC. It will investigate the efficacy and safety of the drug, Afatinib, when administered to people with FA who are diagnosed with unresectable and/or advanced squamous cell carcinoma of the oral cavity, oropharynx, hypopharynx, or larynx.

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]]> Phase 1: Pilot Study of Metformin for Patients with Fanconi Anemia https://fanconi.org/supported-researches/phase-1-pilot-study-of-metformin-for-patients-with-fanconi-anemia/ Wed, 20 Mar 2024 08:55:38 +0000 https://wpbox7.net/ce/dc7ad561/?post_type=app_research&p=2800 Grant description: Low blood counts due to bone marrow failure are a common complication of Fanconi anemia (FA) which affect health and quality of life. Metformin improves blood counts in mice with FA and may protect against DNA damage. Laboratory studies suggest that Metformin may be protective against aldehydes, which are toxic to FA mice […]

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]]> Grant description:

Low blood counts due to bone marrow failure are a common complication of Fanconi anemia (FA) which affect health and quality of life. Metformin improves blood counts in mice with FA and may protect against DNA damage. Laboratory studies suggest that Metformin may be protective against aldehydes, which are toxic to FA mice and to FA patients. Metformin is an FDA-approved oral medication used for many decades to treat diabetes and insulin resistance. Metformin is currently being studied in other clinical trials as an anti-aging and anti-cancer drug, but it is not known how Metformin will affect people with FA. In this study which is opening at Boston Children’s Hospital, we want to learn whether Metformin improves blood counts in people with FA. You may be eligible for this study if you have FA and low blood counts, are between the ages of 6-35 years and have not had a hematopoietic stem cell transplant (bone marrow transplant). As a participant in this study, you will be provided with Metformin for 6 months. It is not known whether Metformin improves blood counts in patients with Fanconi anemia.

Grant follow-up:

The goal of this clinical trial was to determine whether the drug metformin was efficacious at improving blood counts and safe for non-diabetic patients with FA. The results from the study confirm that metformin is safe and tolerable for non-diabetic patients with FA and showed that 4 of the 13 patients who received metformin for at least one month had a favorable hematologic response, suggesting the drug may improve blood counts for a subset of patients with FA.

The research team recently published results from this clinical trial in the journal, Blood, and are working to develop a Phase II multi-institutional clinical trial.

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]]> Quercetin chemoprevention for Squamous cell carcinoma in patients with Fanconi anemia https://fanconi.org/supported-researches/quercetin-chemoprevention-for-squamous-cell-carcinoma-in-patients-with-fanconi-anemia/ Wed, 20 Mar 2024 08:54:42 +0000 https://wpbox7.net/ce/dc7ad561/?post_type=app_research&p=2799 Excessive toxicity from chemotherapy and radiation makes treatment for SCC in FA quite challenging and leads to dismal outcomes, in fact fatal outcomes in most patients. Thus, there is clearly a need for a new approach both for prevention and/or treatment that has fewer and less severe side effects. Our previous study showed that a […]

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]]> Excessive toxicity from chemotherapy and radiation makes treatment for SCC in FA quite challenging and leads to dismal outcomes, in fact fatal outcomes in most patients. Thus, there is clearly a need for a new approach both for prevention and/or treatment that has fewer and less severe side effects. Our previous study showed that a naturally occurring antioxidant Quercetin, is safe and well tolerated in pre-HCT patients with FA. In this study we propose to utilize Quercetin to prevent development of SCC in post-transplant FA patients. We expect this approach to work, as Quercetin has been reported to lead to prevention of SCC in mice (non-FA). In our hands, Quercetin decreases tumor growth in FA head and neck cancer cell lines, by killing the tumor cells. In our previous study, we see evidence of decreased DNA damage in oral mucosa brushings from patients with FA (pre-HCT) after treatment with quercetin for one month. Based on these strong and promising data we propose a pilot study of Quercetin treatment for 6 months at least (with an option of up to total of 2 years) in 20 post-transplant patients with FA. Our clinical and laboratory tests will confirm the beneficial effect of quercetin (e.g. decreased oxidative stress-in blood and saliva, along with decrease in ongoing DNA damage in buccal mucosa compared to baseline). Expected positive impact is that success will lead to a new prevention strategy for SCC in post-HCT patients with FA that will eliminate or at least delay the development of SCC. This is an innovative approach in our opinion, as Quercetin acts on all of the pathways implicated in SCC development in FA, i.e. oxidative stress, NF kappa B, and aldehyde mediated tumor growth. This study will confirm a simple and feasible approach for the prevention of SCC with use of oral quercetin.

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]]> Pilot Study of Metformin for Patients with Fanconi Anemia https://fanconi.org/supported-researches/pilot-study-of-metformin-for-patients-with-fanconi-anemia/ Wed, 20 Mar 2024 08:37:19 +0000 https://wpbox7.net/ce/dc7ad561/?post_type=app_research&p=2791 The objective of this proposal is to conduct a pilot study of metformin to treat FA. This pilot study will explore whether Metformin is safe and efficacious in improving hematopoiesis in patients with Fanconi Anemia. We will focus on pediatric and young adult patients with FA to determine safety, tolerability, and preliminary efficacy of Metformin […]

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]]> The objective of this proposal is to conduct a pilot study of metformin to treat FA.
This pilot study will explore whether Metformin is safe and efficacious in improving hematopoiesis in patients with Fanconi Anemia. We will focus on pediatric and young adult patients with FA to determine safety, tolerability, and preliminary efficacy of Metformin in Fanconi Anemia patients. This study will also integrate biological correlative studies to glean mechanistic insights which may inform future studies. The anticipate enrolling 20 patients, with a study duration of 2 years.

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