Busulfan (Busulfex)

DRUG4 trials

Sponsors

National Cancer Institute (NCI), He Huang

Conditions

DOCK8 DeficiencyGATA2Hematologic DiseaseHematologic DiseasesImmunodeficiencyMDS

Phase 1

Human Leukocyte Antigen (HLA) Mismatched Related Allogeneic Hematopoietic Stem Cell Transplantation

RecruitingNCT06809699

He HuangHematologic Diseases

Start: 2022-08-04End: 2027-10-31Target: 29Updated: 2025-02-05

Human Leukocyte Antigen (HLA) Mismatched Unrelated Allogeneic Hematopoietic Stem Cell Transplantation

RecruitingNCT06809712

He HuangHematologic Disease

Start: 2022-08-04End: 2027-10-31Target: 29Updated: 2025-02-05

Phase 2

Pilot Study of Reduced-Intensity Hematopoietic Stem Cell Transplant of DOCK8 Deficiency

Active, not recruitingNCT01176006

National Cancer Institute (NCI)DOCK8 Deficiency

Start: 2010-10-05End: 2028-10-31Updated: 2026-04-03

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

Related Papers

Hematopoietic cell transplantation for DOCK8 deficiency: Results from a prospective clinical trial.

2024-09-022 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