OncoMatch/Clinical Trials/NCT01861106
Allogeneic Hematopoietic Stem Cell Transplant for GATA2 Mutations
Official title: Allogeneic Hematopoietic Stem Cell Transplant for Patients With Mutations in GATA2 or the MonoMAC Syndrome
Is NCT01861106 recruiting? Yes, currently enrolling (Oct 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
Treatments studied
Chemotherapy
Other
Cancer type
Myelodysplastic Syndrome
Biomarker criteria
Required: GATA2 mutation
Mutation in the GATA2 gene
Required: GATA2 loss of expression of one allele
evidence of loss of expression of one allele of GATA2, by cDNA analysis performed by a CLIA certified laboratory
Demographics
Prior therapy
Cannot have received: investigational agent
Exception: virus-specific cytotoxic T-cells for the treatment of viral infection/reactivation prior to allo HSCT
Patients who are receiving any other investigational agents with the exception of virus-specific cytotoxic T-cells for the treatment of viral infection/reactivation prior to allo HSCT
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
Showing up to 5 US sites.
See all sites on ClinicalTrials.gov →Frequently asked questions
Is NCT01861106 currently recruiting?
Yes, this trial is currently recruiting patients.
Are there prior therapy exclusions?
Yes. Prior investigational agent disqualifies patients from enrollment.
Does this trial require GATA2?
Yes, GATA2 mutation is a required biomarker for enrollment.
Does this trial require GATA2?
Yes, GATA2 loss of expression of one allele is a required biomarker for enrollment.
Is there an age limit?
Yes. Patients must be 70 years or younger and at least 6 years old.
Could you qualify for this trial?
Enter your biomarker results to see how this trial's eligibility criteria match your specific cancer profile.
Check if I qualifyRelated pages