FANCONI ANEMIA

This trial is testing a modified bone marrow transplant procedure designed to cure patients with rare, inherited disorders where the immune system or bone marrow doesn't work properly. The approach uses a less intense pre-transplant treatment and a specific drug after the transpl…

This program provides expanded access to a special machine that filters stem cells before a transplant. The goal is to reduce a dangerous side effect called graft-versus-host disease (GVHD) by removing certain immune cells. It is for patients, including children over 1 month old,…

This study is testing a modified stem cell transplant procedure for patients with Fanconi anemia who have developed severe blood problems like bone marrow failure or leukemia. The goal is to see if using lower doses of certain chemotherapy drugs and removing another medication ca…

This study is testing a modified stem cell transplant procedure for people with inherited bone marrow failure disorders like Fanconi anemia. The goal is to see if removing specific immune cells from the donor cells before transplant can reduce the risk of a serious complication c…

This trial is testing a new way to perform stem cell transplants for people with Fanconi anemia, a rare genetic disorder that causes bone marrow failure. The goal is to use a special, processed donor graft and an experimental antibody called JSP-191 during the pre-transplant cond…

This study aims to understand why people with inherited bone marrow failure syndromes have a higher risk of developing cancer. Researchers will follow 4,000 participants, including patients with these rare genetic conditions and their family members, to track cancer rates and ide…

This study aims to understand how and why people with Fanconi anemia develop certain cancers, particularly in the mouth, throat, and genital areas. Researchers will regularly screen 200 participants aged 8 and older over 10 years to track early warning signs and disease progressi…

This study aims to discover new genes that cause cancer to run in families, especially those affecting children. Researchers will collect health information, blood, and sometimes leftover tumor samples from families where multiple members have had cancer. The goal is to build a l…

This study aims to collect blood, saliva, and health information from people with blood diseases and their family members to build a research library. The goal is to discover unknown genetic causes and understand why disease severity varies between people. This is a research stud…

This study aims to learn about the anxiety people with Fanconi anemia feel during their required cancer screenings. Researchers will observe 20 adults during their regular screening visits, taking notes on their body language, conversations, and environment. The goal is to better…