Clinical Trial: Allogeneic Hematopoietic Stem Cell Transplantation With JSP191-Based Conditioning in Participants With GATA2 Deficiency
Study Status: NOT_YET_RECRUITING
Recruit Status: NOT_YET_RECRUITING
Condition: GATA2
Study Type: INTERVENTIONAL
Official Title: A Phase II Study of Allogeneic Hematopoietic Stem Cell Transplantation With JSP191-Based Conditioning in Participants With GATA2 Deficiency
Brief Summary:
Background:People with GATA2 deficiency have a mutation on the GATA2 gene.This gene affects immune function.People with this disease are prone to serious infections; in time, they may develop blood cancers.A hematopoietic stem cell (HSC) transplant can cure GATA2 deficiency, but using stem cells donated by other people can cause serious side effects.Objective:To test a new drug (JSP191) to see if it can make HSC transplants safer.Eligibility:People aged 6 to 70 years who have GATA2 deficiency.Design:Participants…
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