This study is testing if an allogeneic hematopoietic stem cell transplant (HSCT), also known as a stem cell transplant, can successfully treat GATA2 mutations. GATA2 mutations can lead to conditions like immunodeficiency (a weakened immune system) and myelodysplastic syndromes (MDS), which affect blood cell production. The transplant involves receiving healthy stem cells from a donor. Before the transplant, you would receive medications like Busulfan, Fludarabine, and Cyclophosphamide to prepare your body. Researchers will look to see if the transplant leads to successful engraftment (the new stem cells settling in) and restores normal blood cell production within one year. This study is open to individuals aged 6 to 70 years who have a GATA2 gene mutation or a related condition called MonoMAC. The current status of this study is unclear.

Study design

This is an interventional study, meaning participants will receive a specific treatment. It plans to enroll 144 participants.

What's involved

Participants will undergo a physical exam, medical history review, and blood sample collection for screening. Specific details about the number of visits or duration of participation are not specified in the trial record.

Compensation

Not stated in the trial record.

Follow-up

Researchers will assess the success of the transplant one year after it is completed.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.