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DRUG3 trials

Sponsors

National Cancer Institute (NCI)

Conditions

Autoimmune LymphoproliferativeCommon Variable ImmunodeficiencyGATA2Hematopoietic Stem Cell TransplantationImmune System DiseasesImmunodeficiencyLymphoproliferative DisordersMDS

Phase 2

Allogeneic Hematopoietic Stem Cell Transplant for GATA2 Mutations
RecruitingNCT01861106
National Cancer Institute (NCI)GATA2, Immunodeficiency, MDS
Start: 2013-07-24End: 2028-12-31Target: 144Updated: 2026-03-24
Allogeneic Hematopoietic Stem Cell Transplant for Patients With Inborn Errors of Immunity
RecruitingNCT04339777
National Cancer Institute (NCI)Autoimmune Lymphoproliferative, Common Variable Immunodeficiency, Immune System Diseases +2
Start: 2020-09-22End: 2027-11-30Target: 66Updated: 2025-09-16
A Phase II Study of Allogeneic Hematopoietic Stem Cell Transplant for Subjects With VEXAS (Vacuoles, E1 Enzyme, X-linked, Autoinflammatory, Somatic) Syndrome
RecruitingNCT05027945
National Cancer Institute (NCI)Hematopoietic Stem Cell Transplantation, Immunodeficiency
Start: 2023-02-23End: 2026-07-01Target: 54Updated: 2026-04-02

Related Papers

Clinical Characteristics, Management, and Hematopoietic Cell Transplantation of Patients with TLR8 Gain-of-Function.
2025-12-10
Sustained donor engraftment and clinical remission after hematopoietic cell transplantation for vexas syndrome, but with observed toxicities: Results from a prospective phase II clinical Trial
Blood2025-11-03
Clinical Manifestations Are Evolving and Progressive in Patients with Vexas Syndrome
Blood2023-11-024 citations
A novel <i>GATA2</i> distal enhancer mutation results in MonoMAC syndrome in 2 second cousins
Blood Advances2023-08-189 citations
Allogeneic Hematopoietic Stem-Cell Transplantation in Patients with GATA 2 Deficiency: Influence of Donor Stem Cell Source and Post-Transplantation Cyclophosphamide
Blood2020-11-041 citations
Sequencing of RNA in single cells reveals a distinct transcriptome signature of hematopoiesis in GATA2 deficiency
Blood Advances2020-06-1835 citations