Allogeneic Hematopoietic Stem Cell Transplant for GATA2 Mutations
Status:
Recruiting
Trial end date:
2023-12-31
Target enrollment:
Participant gender:
Summary
Background:
- GATA2 deficiency is a disease caused by mutations in the GATA2 gene. It can cause different
types of leukemia and other diseases. Researchers want to see if a stem cell transplant can
be used to treat this condition. A stem cell transplant will give stem cells from a matching
donor (related or unrelated) to a recipient. It will allow the donor stem cells to produce
healthy bone marrow and blood cells that will attack the recipient s cancer cells.
Objectives:
- To see if stem cell transplants are successful at treating GATA2 mutations and related
conditions.
Eligibility:
- Recipients who are between 8 and 70 years of age and have GATA2 deficiency.
Design:
- All participants will be screened with a physical exam and medical history. Blood
samples will be collected. Recipients will have imaging studies and other tests.
- Recipients will have chemotherapy or radiation to prepare for the transplant. On the day
of the transplant, they will receive the donated stem cells.
- Recipients will stay in the hospital until their condition is stable after transplant.
- Frequent blood tests and scans will be required for the first 6 months after the
transplant, followed by less frequent visits over time.