OncoMatch/Clinical Trials/NCT01861106
Allogeneic Hematopoietic Stem Cell Transplant for GATA2 Mutations
Is NCT01861106 recruiting? Yes, currently enrolling (May 2026). This Phase 2 trial studies multiple treatments for gata2.
Treatment: Busulfan Test dose · Fludarabine (Fludara, Berlex Laboratories) · Busulfan (Busulfex) · Cyclophosphamide (CTX, Cytoxan) · Mycophenolate mofetil (MMF) · Tacrolimus · Equine Anti-Thymocyte Globulin — 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 6 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.
Check if I qualifyExtracted eligibility criteria
Cancer type
Myelodysplastic Syndrome
Biomarker criteria
Required: GATA2 mutation
Mutation in the GATA2 gene, or evidence of loss of expression of one allele of GATA2, by cDNA analysis performed by a CLIA certified laboratory, or the clinical syndrome of MonoMAC.
Required: GATA2 loss of expression of one allele
Mutation in the GATA2 gene, or evidence of loss of expression of one allele of GATA2, by cDNA analysis performed by a CLIA certified laboratory, or the clinical syndrome of MonoMAC.
Lab requirements
Kidney function
adult patients: <= 2.0 mg/dl and creatinine clearance >= 30 ml/min; pediatric patients (<18 years old): creatinine <1.5 mg/dl and a creatinine clearance, using the schwartz formula, > 30 ml/min/1.73m^2
Liver function
serum conjugated bilirubin < 2.5 mg/dl; serum alt and ast <= 5 times upper limit of normal
Cardiac function
left ventricular ejection fraction > 40%, preferably by 2-d echocardiogram obtained within 90 days prior to initiation of conditioning therapy
Structured fields extracted by AI. May contain errors — verify against the official protocol.
US trial sites
- National Institutes of Health Clinical Center · Bethesda, Maryland
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