Allo Stem Cell Transplant for GATA2 Mutations

Allogeneic Hematopoietic Stem Cell Transplant for Patients With Mutations in GATA2 or the MonoMAC Syndrome

What's the purpose of the trial?

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.
Trial status

Accepting patients

Phase
Phase 2
Enrollment
144
Last Updated
1 month ago
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Participating Centers

There is one center participating in this trial. Enter a location below to view the distance.

Experimental Treatments

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  • Allogeneic Stem Cell Transplant is a type of stem cell transplant that utilizes a donor's pre-collected stem cells to rescue the bone marrow from the toxic effects of very high dose chemotherapy.
  • Busulfan is a chemotherapy cancer medication that interferes with the growth and spread of cancer cells in the body, and may be used in the treatment of many different cancers.
  • Cyclophosphamide is a chemotherapy agent used in the treatment of cancer.
  • Fludarabine is a chemotherapy medication that may be used in the treatment of many different cancers.
  • Mycophenolate Mofetil is an immunosuppressive medication used to help prevent rejection in patients who have had a transplant.
  • Tacrolimus is an immunosuppressive medication used to help prevent rejection in patients who have had a transplant.

Arms / Cohorts

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Accepting patients

Arm A

Accepting patients

Arm B

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