Allogeneic Hematopoietic Stem Cell Transplant for GATA2 Mutations

Recruiting Phase 2 Interventional Study
GATA2 Immunodeficiency MDS
No Placebo Group Every participant receives an active treatment — no one gets a placebo. Prior Safety Data This treatment has already been tested in at least one earlier human trial.
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At a Glance
Age
6 – 70
Sex
Any
Trial phase
Phase 2
Study type
Interventional
Purpose
Treatment
Participants needed
144 (estimated)
Sponsor
National Cancer Institute (NCI) · NIH
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About This 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 mar…
Trial Locations
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Eligibility Criteria
* ELIGIBILITY CRITERIA: INCLUSION CRITERIA- Recipient 1. Patient age of 6-70 years. 2. 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. 3. Clinical history of at lea…
Contacts

Danielle E Pregent-Arnold, M.D.

(240) 281-3922

danielle.arnold@nih.gov

CONTACT