Condition

Fanconi Anemia clinical trials and research

• 13Clinical trials
• 13NIH research projects
• 2New in 30 days
• 4Countries

Newest Fanconi Anemia clinical trials

All recruiting trials (13)

• Safe use of metformin and naproxen to help prevent cancer in people with Fanconi anemia

  This study tests whether metformin and naproxen can lower the chance of cancer developing in adults with Fanconi anemia.

  Recruiting at 2 sites Phase 1 Rockefeller University · Minneapolis, Minnesota and 1 other locations · NCT07901959

• Understanding congenital bone marrow failure and related disorders through a national network

  This project tests whether people with rare inherited bone marrow failure syndromes or related conditions really have the underlying genetic causes, by collecting data and samples across multiple centers.

  Recruiting at 4 sites Istituto Giannina Gaslini · Avellino and 3 other locations · NCT07877727

• MRI-based noninvasive detection and monitoring of oral lesions in people with Fanconi anemia

  This study tests whether MRI and dedicated dental MRI can help detect and track potential oral cancers or precancers in people with Fanconi anemia without surgery or biopsy.

  Recruiting University of Minnesota · Minneapolis, Minnesota · NCT07649031

• Using Afatinib to treat patients with Fanconi Anemia and advanced head and neck cancer

  This study is testing if Afatinib can help people with advanced head and neck cancer who also have Fanconi Anemia feel better and improve their condition.

  Recruiting at 2 sites Phase 1/2 Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau · Hanover and 1 other locations · NCT06648096

• Investigating how acetaldehyde contributes to oral cancer development

  This study is trying to see how drinking alcohol affects the development of oral cancer in healthy people and those with Fanconi anemia by looking at samples like saliva and urine.

  Recruiting Masonic Cancer Center, University of Minnesota · Minneapolis, Minnesota · NCT05973656

• Understanding Squamous Cell Carcinoma in People with Fanconi Anemia

  This study looks at how squamous cell carcinoma develops in people with Fanconi anemia to help catch it early and improve treatment options.

  Recruiting National Institutes of Health Clinical Center (CC) · Bethesda, Maryland · NCT05687149

• Transplanting depleted donor stem cells in patients with Fanconi Anemia using a new conditioning treatment

  This study is testing a new way to use donor stem cells for people with Fanconi Anemia by giving them a special treatment first to see if it helps their blood and immune system recover better and with fewer side effects.

  Recruiting Phase 1/2 Stanford University · Stanford, California · NCT04784052

• Reduced intensity bone marrow transplant for immune and bone marrow disorders

  This study is testing a gentler type of bone marrow transplant to see if it can help people with immune and bone marrow disorders recover better and live longer.

  Recruiting Phase 2 Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins · Baltimore, Maryland · NCT04232085

• Transplantation for inherited bone marrow failure disorders

  This study is testing a new way to do stem cell transplants for people with inherited bone marrow failure disorders to see if it can help them recover faster and reduce the risk of infections without needing extra medication.

  Recruiting Phase 2 Masonic Cancer Center, University of Minnesota · Minneapolis, Minnesota · NCT03579875

• Investigating genetic factors in childhood cancer

  This study is trying to find out if certain genes passed down in families can increase the risk of childhood cancer by looking at samples from kids who have had cancer and their relatives.

  Recruiting St. Jude Children's Research Hospital · Memphis, Tennessee · NCT03050268

• Investigation of genetics related to blood diseases

  This study is trying to understand the genetics behind blood diseases by collecting samples and health information from people with these conditions and their family members.

  Recruiting St. Jude Children's Research Hospital · Memphis, Tennessee · NCT02720679

• Transplant for Patients with Fanconi Anemia Using Adjusted Chemotherapy

  This study is testing a new way to use lower doses of chemotherapy before a stem cell transplant to see if it can safely help people with Fanconi anemia and serious blood disorders.

  Recruiting at 2 sites Phase 2 Children's Hospital Medical Center, Cincinnati · New York, New York and 2 other locations · NCT02143830

• Understanding cancer risks in inherited bone marrow failure syndromes

  This study is trying to understand the cancer risks in people with inherited bone marrow failure syndromes to help improve screening and prevention for families at risk.

  Recruiting at 2 sites National Institutes of Health Clinical Center (CC) · Bethesda, Maryland and 1 other locations · NCT00027274

NIH-funded research (13)

Early-stage science funded by the US National Institutes of Health. Most of these projects don't enroll patients, but they show what is being worked on.

• In vivo gene repair to restore healthy hematopoiesis in Fanconi anemia

  A study aims to repair faulty genes inside patients’ blood-forming cells to help people with Fanconi anemia regain healthy blood cell production.

  NIH research Children's Hosp of Philadelphia · Philadelphia, United States · NIH-11422867

• Understanding genetic dependencies when Fanconi anemia pathway is lost

  A study to find other genes or pathways that cancer cells rely on when the Fanconi anemia repair system is broken, with the aim of guiding future treatments.

  NIH research Columbia University Health Sciences · New York, United States · NIH-11421557

• Innovative cell and gene therapy approaches to treat Fanconi anemia

  A Stanford-led project aims to develop safer cell and gene therapies to help people with Fanconi anemia who have bone marrow failure.

  NIH research Stanford University · Stanford, United States · NIH-11416385

• How cells detect DNA damage and activate ATR, ATM, and Fanconi anemia repair systems

  Researchers are using high-resolution structural imaging to show how key repair proteins sense DNA damage, with relevance for people whose cancers come from DNA repair defects.

  NIH research Sloan-Kettering Inst Can Research · New York, United States · NIH-11313820

• In vivo gene therapy for Fanconi anemia

  A patient-focused overview of a potential in vivo gene therapy approach to correct Fanconi anemia, aiming to reduce the need for donor transplants.

  NIH research Fred Hutchinson Cancer Center · Seattle, United States · NIH-11297452

• Fanconi anemia: what happens inside cells

  Researchers want to learn how proteins called REV7 and CHAMP1 change DNA repair in people with Fanconi anemia and related genetic syndromes.

  NIH research Dana-Farber Cancer Inst · Boston, United States · NIH-11257267

• Understanding how cells detect and respond to DNA damage

  This study is looking at how our cells detect and fix damage to their DNA, which is really important for stopping cancer, and it aims to understand how certain proteins work together to repair serious DNA problems, with the hope that this knowledge can help develop new treatments to improve DNA repair in cancer cells.

  NIH research Sloan-Kettering Inst Can Research · New York, United States · NIH-11073113

• Overcoming drug resistance in ovarian cancer using the Fanconi Anemia pathway

  This study is looking at how to make chemotherapy work better for ovarian cancer patients by understanding a specific pathway in cancer cells that can make them resistant to treatment, with the hope of finding new ways to improve their care.

  NIH research George Washington University · Washington, United States · NIH-11059114

• New treatment approach for Fanconi anemia using enzymes

  This study is looking at a new treatment for kids with Fanconi anemia that uses special enzymes to help reduce harmful substances in the body, aiming to improve their blood health and overall well-being.

  NIH research Kinetiq Therapeutics LLC · Fort Worth, United States · NIH-11005464

• Understanding how chromothripsis affects cancer development

  This study is looking into how certain genetic changes happen in cancer, specifically focusing on a process called chromothripsis, and it aims to help patients understand more about how their cancer develops and find new ways to treat it.

  NIH research Ut Southwestern Medical Center · Dallas, United States · NIH-10996542

• Correcting FANCC mutations for treating Fanconi Anemia

  This study is testing a new gene therapy that uses cutting-edge technology to fix a problem in the FANCC gene for people with Fanconi Anemia, aiming to help their blood stem cells work better and provide a safer treatment option than traditional stem cell transplants.

  NIH research Indiana University Indianapolis · Indianapolis, United States · NIH-10886717

• New methods for gene therapy and bone marrow transplants in Fanconi anemia

  This study is looking for better ways to help people with Fanconi anemia by using safer treatments that avoid harsh chemotherapy and radiation before they get a bone marrow or stem cell transplant, making the process easier and reducing the chance of future cancers.

  NIH research Fred Hutchinson Cancer Center · Seattle, United States · NIH-10656502

• Using quercetin to prevent squamous cell carcinoma in children with Fanconi anemia

  This study is looking at whether quercetin, a natural substance, can help prevent squamous cell carcinoma in children with Fanconi anemia by reducing harmful effects in their bodies, offering a safer option than current treatments like radiation and chemotherapy.

  NIH research Cincinnati Childrens Hosp Med Ctr · Cincinnati, United States · NIH-10652481

Related topics

Quick answers

How many Fanconi Anemia clinical trials are recruiting right now?

13, as of November 12, 2026. 2 of them were first registered in the last 30 days. We update this list each time we refresh our data from ClinicalTrials.gov.

Where are these trials running?

In 4 countries. The most sites are in United States (11 trials), Germany (1 trial), Spain (1 trial), Italy (1 trial). Each study page lists its sites and which ones are recruiting.

What phases are they in?

Phase 2: 3, Phase 1/2: 2, Phase 1: 1. Trials with no phase listed are usually device, behavioural or observational studies.

How do I join one?

Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.

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