Allogeneic Hematopoietic Stem Cell Transplant for GATA2 Mutations
Public ClinicalTrials.gov record NCT01861106. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
Allogeneic Hematopoietic Stem Cell Transplant for Patients With Mutations in GATA2 or the MonoMAC Syndrome
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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....
Study identification
- NCT ID
- NCT01861106
- Recruitment status
- Recruiting
- Study type
- Interventional
- Phase
- Phase 2
- Enrollment
- 144 participants
Conditions and interventions
Conditions
Interventions
- Allogeneic HSCT Procedure
- Busulfan Test dose Drug
- Fludarabine (Fludara, Berlex Laboratories) Drug
- Busulfan (Busulfex) Drug
- Cyclophosphamide (CTX, Cytoxan) Drug
- Total Body Irradiation (TBI) Procedure
- Mycophenolate mofetil (MMF) Drug
- Tacrolimus Drug
- Equine Anti-Thymocyte Globulin Biological
Procedure · Drug · Biological
Eligibility (public fields only)
- Age range
- 6 Years to 70 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Jul 23, 2013
- Primary completion
- Dec 30, 2027
- Completion
- Dec 30, 2028
- Last update posted
- Sep 9, 2026
2013 – 2028
United States locations
- U.S. sites
- 1
- U.S. states
- 1
- U.S. cities
- 1
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| National Institutes of Health Clinical Center | Bethesda | Maryland | 20892 | Recruiting |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT01861106, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Sep 9, 2026 · Synced Sep 13, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT01861106 live on ClinicalTrials.gov.