About this trial
This trial looks at how to improve treatments for patients with Fanconi Anemia. The focus is on using lower doses of certain medicines before a bone marrow transplant. The goal is to reduce side effects while still effectively treating the condition. It involves patients with severe blood problems who need a transplant but do not have a perfect donor match.
The trial's ID number is NCT02143830. It is a Phase 2 trial, meaning it is looking at how well the treatment works and its safety. About 70 participants are expected to join this study.
Who can join
This study is open to children and adults aged 3 months and older. Anyone diagnosed with Fanconi Anemia or severe blood conditions may join. Both male and female participants are welcome. Some health conditions may prevent joining, but the trial team will confirm eligibility.
Conditions
Fanconi Anemia, Severe Marrow Failure, Myelodysplastic Syndrome (MDS), Acute Myelogenous Leukemia (AML)
The study team makes the final call on eligibility.
What happens in the study
Participants will receive one of three treatment plans based on their risk. All groups will get a combination of medicines, including busulfan, cyclophosphamide, and fludarabine, before a stem cell transplant. The treatment will vary depending on the patient's age and condition. Everyone will have a chance to receive supportive care after the transplant.
Where
This trial is running at 3 locations in the United States. 2 of them are currently recruiting.
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio
Fred Hutchinson Cancer Research Center
Seattle, Washington
Memorial Sloan Kettering Cancer Center
New York, New York