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NCT01861106 · ClinicalTrials.gov registry record · Phase 2
Allogeneic Hematopoietic Stem Cell Transplant for GATA2 Mutations
A Phase 2 study of MDS and Immunodeficiency, sponsored by National Cancer Institute (NCI).
- Recruiting
- Registry status
- Phase 2
- Development phase
- 144
- Enrollment target
- 1
- Study location
NCT01861106: Recruiting Phase 2 study of MDS and Immunodeficiency, sponsored by National Cancer Institute (NCI).
NCT01861106 is a Phase 2 study of MDS and Immunodeficiency that is actively recruiting participants, run by National Cancer Institute (NCI). The registered enrollment target is 144 participants, above the 91-participant average among 22 other MDS trials with a reported enrollment target (58% higher). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01861106, a Phase 2 study of MDS and Immunodeficiency, is actively recruiting participants, sponsored by National Cancer Institute (NCI).
- RECRUITING
- Registry status
- Phase 2
- Development phase
- 144 participants
- Enrollment target
- 1
- Study location
Study 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 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.
Primary Outcome
Determination that engraftment has occurred, normal hematopoiesis has been restored and the clinical phenotype after allogeneic HSCT has been reversed
Conditions Studied
Interventions
- PROCEDURE Allogeneic HSCT
- DRUG Busulfan Test dose
- DRUG Fludarabine (Fludara, Berlex Laboratories)
- DRUG Busulfan (Busulfex)
- DRUG Cyclophosphamide (CTX, Cytoxan)
Study Locations (1)
Maryland
- National Institutes of Health Clinical Center - Bethesda
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 144 participants |
| Start Date | 2013-07-24 |
| Est. Completion | 2028-12-31 |
| Phase | Phase 2 |
What NCT01861106 shows while recruiting
NCT01861106 is an interventional study that assigns participants to a tested intervention. The registered 144 participants enrollment target is mid-sized for trials with a published cap, above the 91-participant average among 22 other MDS trials with a reported enrollment target (58% higher).
The record links to 3 conditions, with MDS appearing as the primary indexed condition, and to 5 interventions - of which Allogeneic HSCT is the first listed.
NCT01861106 reports a single indexed study location in Maryland.
Frequently Asked Questions
What is clinical trial NCT01861106 about?
NCT01861106 is a clinical study titled "Allogeneic Hematopoietic Stem Cell Transplant for GATA2 Mutations". 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 (...
What is the current status of trial NCT01861106?
This trial is currently recruiting. It is a Phase 2 study. The enrollment target is 144 participants. The study started on 2013-07-24. Estimated completion is 2028-12-31.
What conditions does trial NCT01861106 study?
This clinical trial studies the following conditions: MDS, Immunodeficiency, GATA2.
What interventions are being tested in trial NCT01861106?
The interventions under investigation include: Allogeneic HSCT (PROCEDURE), Busulfan Test dose (DRUG), Fludarabine (Fludara, Berlex Laboratories) (DRUG), Busulfan (Busulfex) (DRUG), Cyclophosphamide (CTX, Cytoxan) (DRUG).
Who is sponsoring clinical trial NCT01861106?
This trial is sponsored by National Cancer Institute (NCI), which has 3,257 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT01861106 being conducted?
This trial has 1 study location across Maryland. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Where NCT01861106's enrollment target sits among peer trials
144 3rd of 22 higher than 20 of 22 other MDS trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other MDS trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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