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Fanconi Anemia clinical trials

View clinical trials related to Fanconi Anemia.

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NCT ID: NCT04248439 Active, not recruiting - Clinical trials for Fanconi Anemia Complementation Group A

Gene Therapy for Fanconi Anemia, Complementation Group A

Start date: July 15, 2020
Phase: Phase 2
Study type: Interventional

The objective of this study is to assess the therapeutic efficacy of a hematopoietic cell-based gene therapy for patients with Fanconi anemia, subtype A (FA-A). Hematopoietic stem cells from mobilized peripheral blood of patients with FA-A will be transduced ex vivo (outside the body) with a lentiviral vector carrying the FANCA gene. After transduction, the corrected stem cells will be infused intravenously back to the patient with the goal of preventing bone marrow failure.

NCT ID: NCT04069533 Active, not recruiting - Clinical trials for Fanconi Anemia Complementation Group A

Lentiviral-mediated Gene Therapy for Pediatric Patients With Fanconi Anemia Subtype A

Start date: November 28, 2019
Phase: Phase 2
Study type: Interventional

This is an open-label Phase II clinical trial to evaluate the efficacy of a hematopoietic cell-based gene therapy for pediatric patients with Fanconi Anemia, subtype A (FA-A). Hematopoietic stem cells from mobilized peripheral blood of patients with FA-A will be transduced ex vivo (outside the body) with a lentiviral vector carrying the FANCA gene. After transduction, the corrected stem cells will be infused intravenously back to the patient with the goal of preventing bone marrow failure.

NCT ID: NCT03814408 Active, not recruiting - Clinical trials for Fanconi Anemia Complementation Group A

A Clinical Trial to Evaluate the Safety of RP-L102 in Pediatric Subjects With Fanconi Anemia Subtype A

Start date: January 11, 2019
Phase: Phase 1
Study type: Interventional

The objective of this study is to assess the therapeutic safety and preliminary efficacy of a hematopoietic cell-based gene therapy consisting of autologous CD34+ enriched cells transduced with a lentiviral vector carrying the FANCA gene in subjects with Fanconi anemia subtype A (FA-A).

NCT ID: NCT03476330 Active, not recruiting - Clinical trials for Squamous Cell Carcinoma

Quercetin Chemoprevention for Squamous Cell Carcinoma in Patients With Fanconi Anemia

Start date: May 8, 2018
Phase: Phase 2
Study type: Interventional

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)

NCT ID: NCT00542373 Active, not recruiting - Clinical trials for Tobacco Use Disorder

Widefield Fluorescence and Reflectance Imaging Systems and Oral Tissue Samples in Monitoring Participants at Risk for Developing Oral Cancer

Start date: August 27, 2007
Phase: N/A
Study type: Interventional

This clinical trial studies widefield fluorescence and reflectance imaging, fluorescence spectroscopy, and tissue samples in regularly examining (monitoring) participants at risk for developing oral cancer. All tissue and cells are made of tiny particles. Some of these particles give off small amounts of light. This light is called fluorescence. Fluorescent imaging use instruments that shine different wavelengths (colors) of light in the mouth taking fluorescence pictures through a portable head light or by taking fluorescent and reflectance pictures through a dental microscope using a digital camera. Fluorescent spectroscopy uses a small probe placed gently against the lining of the mouth and the tissue is exposed to small amounts of fluorescent light that is then collected with a special camera and a computer to be analyzed. Checking mouth tissue samples under a microscope may also help detect abnormal cells. Diagnostic procedures, such as fluorescence and reflectance imaging, fluorescence spectroscopy imaging, and tissue samples, may help doctors detect pre-cancer or early cancer when it may be easier to treat.

NCT ID: NCT00006127 Active, not recruiting - Fanconi's Anemia Clinical Trials

Phase I Study of Amifostine in Patients With Bone Marrow Failure Related to Fanconi's Anemia

Start date: April 2000
Phase: Phase 1
Study type: Interventional

OBJECTIVES: I. Evaluate the toxicity of amifostine in patients with bone marrow failure related to Fanconi's anemia. II. Determine the efficacy of this treatment regimen in this patient population. III. Evaluate the effect of this treatment regimen on bone marrow progenitor cell proliferation and peripheral blood mononuclear cell apoptosis in these patients.

NCT ID: NCT00005896 Active, not recruiting - Fanconi's Anemia Clinical Trials

Phase I Pilot Study of CD34 Enriched, Fanconi's Anemia Complementation Group C Gene Transduced Autologous Peripheral Blood Stem Cell Transplantation in Patients With Fanconi's Anemia

Start date: March 2000
Phase: Phase 1
Study type: Interventional

OBJECTIVES: I. Determine the safety of transferring the Fanconi anemia complementation group C (FACC) gene to hematopoietic progenitors by retroviral mediated gene transfer in patients with Fanconi's anemia, complementation group C. II. Determine the extent of engraftment following this treatment regimen without prior ablation of recipient marrow in these patients. III. Determine the ability of this treatment regimen to correct the cell phenotype and improve hematopoietic function in these patients.